UNITED STATES SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 10-K

☒ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the Fiscal Year Ended December 31, ~~2021~~2022

☐TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

Commission File Number: 001-31918

~~IDERA PHARMACEUTICALS~~Aceragen, ~~INC~~Inc.

(Exact name of Registrant as specified in its charter)


Delaware	04-3072298
(State or other jurisdiction	(I.R.S. Employer
of incorporation or organization)	Identification No.)

505 Eagleview Blvd., Suite 212	19341
Exton, Pennsylvania	(Zip Code)
(Address of principal executive offices)

(484) 348-1600

(Registrant’s telephone number, including area code)

Securities registered pursuant to Section 12(b) of the Act


Title of Each Class:	Trading Symbol	Name of Each Exchange on Which Registered
Common Stock, $.001 par value	IDRA	Nasdaq Capital Market
Common Stock, $0.001 par value	ACGN	Nasdaq Capital Market

Securities registered pursuant to Section 12(g) of the Act: None

Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. Yes ☐ No ☒

Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or 15(d) of the Securities Act. Yes ☐ No ☒

Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒ No ☐

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒ No ☐

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.


Large accelerated filer	☐	Accelerated filer	☐

Non-accelerated filer	☒	Smaller reporting company	☒

		Emerging growth company	☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.☐

Indicate by check mark whether the registrant has filed a report on and attestation to its management’s assessment of the effectiveness of its internal control over financial reporting under Section 404(b) of the Sarbanes-Oxley Act (15 U.S.C. 7262(b)) by the registered public accounting firm that prepared or issued its audit report.☐

If securities are registered pursuant to Section 12(b) of the Act, indicate by check mark whether the financial statements of the registrant included in the filing reflect the correction of an error to previously issued financial statements.☐

Indicate by check mark whether any of those error corrections are restatements that required a recovery analysis of incentive-based compensation received by any of the registrant’s executive officers during the relevant recovery period pursuant to § 240.10D-1(b).☐

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act.) Yes ☐ No ☒

The aggregate market value of the voting and non-voting common equity held by non-affiliates of the registrant was $6324.80 million based on the last sale price of the registrant’s common stock as reported on the Nasdaq Capital Market on June 30, ~~2021~~2022 (the last business day of the registrant’s most recently completed second fiscal quarter).

As of ~~March~~April 3113, ~~2022~~2023, the registrant had 528,~~924~~423,~~870~~504 shares of common stock outstanding.

DOCUMENTS INCORPORATED BY REFERENCE

~~Portions of the registrant’s definitive proxy statement for its 2022 annual meeting of stockholders are incorporated by reference into Part III of this Form 10-K where indicated~~None. ~~Such definitive proxy statement will be filed with the U.S. Securities and Exchange Commission within 120 days after the year ended December 31, 2021.~~

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~~IDERA PHARMACEUTICALS~~ACERAGEN, INC.

FORM 10-K

TABLE OF CONTENTS



		Page

PART I.

Item 1.	Business	1
Item 1A.	Risk Factors	2429
Item 1B.	Unresolved Staff Comments	3857
Item 2.	Properties	3857
Item 3.	Legal Proceedings	3857
Item 4.	Mine Safety Disclosures	3857

PART II.

Item 5.	Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities	3958
Item 6.	Reserved	3958
Item 7.	Management’s Discussion and Analysis of Financial Condition and Results of Operations	4059
Item 7A.	Quantitative and Qualitative Disclosures about Market Risk	5071
Item 8.	Financial Statements and Supplementary Data	5071
Item 9.	Changes in and Disagreements with Accountants on Accounting and Financial Disclosure	5072
Item 9A.	Controls and Procedures	5072
Item 9B.	Other Information	5173
Item 9C	Disclosure Regarding Foreign Jurisdictions that Prevent Inspections	5173

PART III.

Item 10.	Directors, Executive Officers and Corporate Governance	5274
Item 11.	Executive Compensation	5278
Item 12.	Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters	5292
Item 13.	Certain Relationships and Related Transactions, and Director Independence	5395
Item 14.	Principal Accountant Fees and Services	5397

PART IV.

Item 15.	Exhibits and Financial Statement Schedules	5498
Item 16.	Form 10-K Summary	60106

__________________________________

Unless otherwise stated or the context ~~otherwise~~requires ~~indicates~~otherwise, references in this Annual Report on Form 10-K to “~~Idera~~Aceragen,” the “company,” the “Company”,” “we,” “us,” ~~and~~or “our” refer to Aceragen, Inc. (formerly known as Idera Pharmaceuticals, Inc.) and its subsidiaries, taken together. The Aceragen logo and other trademarks or service marks of the Company appearing in this Annual Report on Form 10-K are the property of Aceragen, Inc. All other brand names or trademarks are the property of their respective owners.

~~IMO®~~Website ~~and~~addresses ~~Idera®~~referenced in this Annual Report on Form 10-K are ~~our~~provided ~~trademarks.~~for ~~All~~convenience ~~other~~only, ~~trademarks~~and the content on the referenced websites does not constitute a part of, and ~~service~~are ~~marks~~specifically ~~appearing~~not inincorporated by reference into, this Annual Report on Form 10-K ~~are the property of their respective owners~~.

~~Website~~All ~~addresses~~share ~~referenced~~and inper ~~this~~share ~~Annual~~amounts, ~~Report~~including onthe ~~Form~~exercise ~~10-K~~or ~~are~~conversion ~~provided~~price ~~for~~of ~~convenience~~any ~~only,~~of ~~and~~our ~~the~~securities, ~~content~~reflect, onas applicable, the ~~referenced~~occurrence ~~websites~~of ~~does~~a ~~not~~1-for-17 ~~constitute~~reverse asplit ~~part~~ of ~~this~~our ~~Annual~~common ~~Report~~stock that occurred on ~~Form~~January ~~10-K~~17, 2023.

All share and per share amounts, including the exercise or conversion price of any of our securities, reflect, as applicable, the occurrence of a 1-for-8 reverse split of our common stock that occurred on July 27, 2018.

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NOTE REGARDING FORWARD-LOOKING STATEMENTS

This Annual Report on Form 10-K (“Form 10-K”) and the documents we incorporate by reference contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”). All statements, other than statements of historical fact, included or incorporated in this report regarding, among other things, our cash resources and projected cash runway, financial position, our strategy, strategic alternatives, future operations, clinical trials (including, ~~collaborations~~without limitation, ~~intellectual~~the ~~property~~anticipated timing enrollment, ~~cash~~and ~~resources~~results thereof), ~~financial~~collaborations, ~~position~~intellectual property, future revenues, projected costs, fundraising and/or financing plans, prospects, the ongoing impacts of the coronavirus (“COVID-19”) pandemic, the benefits related to the Aceragen Acquisition (as defined below), the benefits related to the Reverse Stock Split (as defined below) and the plans and objectives of management are forward-looking statements. The words “believes,” “anticipates,” “estimates,” “plans,” “expects,” “intends,” “may,” “could,” “should,” “potential,” “likely,” “projects,” “continue,” “will,” “schedule,” “would,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. We cannot guarantee that we will actually achieve the plans, intentions, or expectations disclosed in our forward-looking statements and you should not place undue reliance on our forward-looking statements. These forward-looking statements involve known and unknown risks, uncertainties, and other factors, which may be beyond our control, and which may cause the actual results, performance, or achievements of the Company to be materially different from future results, performance, or achievements expressed or implied by such forward-looking statements.

There are ~~several~~a number of important factors that could cause our actual results to differ materially from those indicated or implied by forward-looking statements. These important factors include those set forth below under Part I, Item 1A “Risk Factors” and in our other disclosures and filings with the Securities and Exchange Commission (“SEC”). These factors and the other cautionary statements made in this ~~Annual Report on~~ Form 10-K and the documents we incorporate by reference should be read as being applicable to all related forward-looking statements whenever they appear in this Form 10-K and the documents we incorporate by reference.

In addition, any forward-looking statements represent our estimates only as of the date that this ~~Annual Report on~~ Form 10-K is filed with the SEC and should not be relied upon as representing our estimates as of any subsequent date. All forward-looking statements included in this Form 10-K are made as of the date hereof and are expressly qualified in their entirety by this cautionary notice. We disclaim any intention or obligation to update or revise any forward-looking statement, whether as a result of new information, future events, or otherwise, except as may be required by law.

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PART I.

Item 1.

Business.

~~Overview~~Prior to January 17, 2023, we were known as Idera Pharmaceuticals, Inc. On September 28, 2022 we completed the Aceragen Acquisition, whereby we acquired all of the outstanding equity interests in Aceragen, Inc., a private company, as further described below. In connection with the Aceragen Acquisition and related transactions, we changed our name to Aceragen, Inc. Unless the context indicates otherwise, references in this section to the “Company,” “Aceragen,” “Idera,” “we,” “us,” “our” and similar terms refer to Aceragen, Inc. (f/k/a Idera Pharmaceuticals, Inc.) and our consolidated subsidiaries. References to “Legacy Aceragen” refer to Aceragen, Inc. and its subsidiaries prior to the consummation of the Aceragen Acquisition.

WeAll ~~are~~share aand ~~biopharmaceutical~~per ~~company~~share ~~with~~amounts, aincluding ~~business strategy focused on~~ the ~~clinical~~exercise ~~development,~~or ~~and~~conversion ~~ultimately~~price ~~the~~of ~~commercialization,~~any of ~~drug~~our ~~candidates~~securities, ~~for rare disease indications characterized by small~~reflect, ~~well-defined~~as patient populations with serious unmet medical needs. Our current focus is to identify and acquire rights to novel development and commercial stage rare disease programs through new business development opportunitiesapplicable, ~~including additional strategic alternatives. We have in~~ the ~~past~~occurrence ~~and~~of ~~may~~a in1-for-17 ~~the~~reverse ~~future~~split ~~explore~~of ~~collaborative~~our ~~alliances~~common tostock ~~support~~that ~~development~~occurred ~~and~~on ~~commercialization~~January of17, ~~any of our drug candidates~~2023.

Until May 2021, we were developing intratumoral tilsotolimod for the treatment of anti-PD1 refractory metastatic melanoma in combination with ipilimumab, an anti-CTLA4 antibody marketed as Yervoy® by Bristol Myers Squibb Company (“BMS”), in a Phase 3 registration trial (ILLUMINATE-301). During the first quarter of 2021, we announced that ILLUMINATE-301 did not meet its primary endpoint of Objective Response Rate (“ORR”). Based on subsequent evaluation of the full data set, in May 2021 we announced that we would not continue the trial to its Overall Survival (“OS”) primary endpoint.Overview

We are a clinical-stage biopharmaceutical company with a business strategy focused on the clinical development, and ultimately the commercialization, of drug candidates for rare disease indications characterized by small, well-defined patient populations with serious unmet medical needs. Our current focus is to develop and optimize commercial value of ACG-701 (patented formulation of sodium fusidate) and ACG-801 (recombinant human acid ceramidase (rhAC)) for appropriate patients. We have in the past and may in the future explore collaborative alliances to support development and commercialization of any of our drug candidates. We may also seek to identify and potentially acquire rights to novel development and commercial-stage rare disease programs through new business development opportunities, including additional strategic alternatives.

As discussed in more detail below, in September 2022, we acquired Legacy Aceragen, a privately-held biotechnology company focused on addressing rare, orphan pulmonary, and rheumatic diseases for which there are limited or no available treatments. Legacy Aceragen owned or controlled the intellectual property related to ACG-701 (patented formulation of sodium fusidate) and ACG-801 (recombinant human acid ceramidase (rhAC)). Following the Aceragen Acquisition, our business strategy is to develop and optimize commercial value of ACG-701 and ACG-801 for appropriate patients. Accordingly, we are developing ACG-701 to treat cystic fibrosis (“CF”) pulmonary exacerbations (“PEx”) and melioidosis, a severe, life-threatening infection, and ACG-801 to treat patients suffering from a genetic mutation in the ASAH 1 gene, also known as Farber disease.

Until December 2021, we were developing tilsotolimod, via intratumoral injection, for the treatment of solid tumors in combination with nivolumab, an anti-PD1 antibody marketed as Opdivo® by Bristol Myers Squibb Company (“BMS”), and/or ipilimumab, an anti-CTLA4 antibody marketed as Yervoy® by BMS. Due to Phase 3 results in anti-PD-1 refractory advanced melanoma (ILLUMINATE-301), reported in March 2021, which showed the study failed to meet its primary endpoint, as well as a decision in December 2021 to discontinue enrollment in ILLUMINATE-206, our Phase 2 study in solid tumors, Company-sponsored development of tilsotolimod in oncology was discontinued. Although clinical trials with tilsotolimod have not yet translated into a new treatment alternative for patients, we believe that data supporting tilsotolimod’s mechanism of action and encouraging safety profile from across the array of pre-clinical and clinical work to date, together with its intellectual property protection, are noteworthy.

~~Through~~Business ~~December~~Acquisition 2021, we were also evaluating intratumoral tilsotolimod in combination with nivolumab, an anti-PD1 antibody marketed as Opdivo® by BMS, and ipilimumab for the treatment of multiple solid tumors in a multicohort Phase 2 trial. In December 2021, we announced that preliminary data from the second 10 patients dosed in the safety cohort of ILLUMINATE-206 showed a safety profile consistent with the first 10 patients in ILLUMINATE-206 and with prior studies. No further enrollment in ILLUMINATE-206 is planned at this time.

~~While~~On ~~our~~September ~~clinical~~28, ~~trials~~2022 (“Acquisition Date”), in accordance with ~~tilsotolimod~~the ~~have~~terms ~~not~~of ~~yet~~an ~~translated~~Agreement ~~into~~and aPlan ~~new~~of ~~treatment~~Merger ~~alternative~~(the “Merger Agreement”), we acquired 100% of the outstanding security interests of Legacy Aceragen in a “stock-for-stock” ~~patients~~transaction, ~~data~~whereby ~~supporting~~all ~~tilsotolimod’s~~Legacy ~~mechanism~~Aceragen outstanding equity interests were exchanged for a combination of ~~action~~shares ~~and~~of ~~encouraging~~our ~~safety~~common ~~profile~~stock, ~~from~~shares ~~across~~of ~~the~~Series ~~array~~Z preferred stock, par value $0.01 per share (“Series Z Preferred Stock”), and shares of ~~pre~~the newly-~~clinical~~designated ~~and~~Series ~~clinical~~X ~~work~~non-voting topreferred ~~date~~stock, ~~together~~par ~~with~~value ~~its~~$0.01 ~~intellectual~~per ~~property~~share ~~protection~~(“Series X Preferred Stock”) (such acquisition, ~~are~~the ~~noteworthy~~“Aceragen Acquisition”). As aUnder ~~result,~~the interms ~~December~~of ~~2021,~~the weMerger ~~announced~~Agreement, ~~that~~Legacy weAceragen ~~will~~stockholders ~~consider~~received an(i) ~~out-licensing~~451,608 ~~arrangement~~shares soof ~~that~~our ~~tilsotolimod’s~~common ~~full~~stock ~~potential~~(inclusive ~~may~~of ~~continue~~shares subject to berepurchase), ~~explored~~(ii) on80,656 ~~behalf~~shares of ~~patients~~Series ~~who~~Z doPreferred ~~not~~Stock ~~respond~~(inclusive of shares subject to ~~traditional~~repurchase), ~~immunotherapy~~and (iii) five shares of Series X Preferred Stock. In addition, all outstanding options and warrants to purchase

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Legacy Aceragen common stock were converted into stock options and warrants to purchase shares of our common stock and Series Z Preferred Stock on terms substantially identical to those in effect prior to the Aceragen Acquisition, except for adjustments to the underlying number of shares and the exercise price based on the Merger Agreement exchange ratio.

Pursuant to the Merger Agreement, we held a Special Meeting of Stockholders’ on January 12, 2023 (the “Special Meeting”) at which our stockholders approved, among other matters: (i) the conversion of Series Z Preferred Stock into shares of common stock in accordance with Nasdaq Listing Rule 5635(a) (the “Conversion Proposal”) and (ii) a proposal to amend our Charter to effect a reverse stock split of all of the Company’s issued and outstanding shares of common stock (the “Reverse Stock Split Proposal” and, together with the Conversion Proposal, the “Merger Agreement Meeting Proposals”).

See “Item 7—Management’s Discussion and Analysis of Financial Condition and Results of Operations” included elsewhere in this Form 10-K for further discussion of the Aceragen Acquisition and related transactions.

Recent ~~Events and Updates~~Developments

~~Reduction~~On April 13, 2023, the Board approved certain cost-cutting measures with a view to preserving capital to support our continuing operations. As part of this plan, we have commenced the furlough of 12 employees, representing approximately 46% of our workforce. Additionally, certain of our employees and executive officers will defer portions of their respective base salaries in amounts that exceed $200,000, with such deferrals having a retroactive effective date of April 5, 2023. We will continue to review operations for other opportunities to reduce costs and pursue financing opportunities. For more information, please see Item 9B of this Form 10-~~Force~~K.

In April 2021, we began to implement a reduction-in-force, which affected approximately 50% of our workforce. The decision was made to align our workforce to our needs considering the topline data results from ILLUMINATE-301’s ORR endpoint and the subsequent decision not to continue to the study’s OS endpoint. In connection with these actions, we incurred termination costs during the second and third quarters of 2021, which included severance, benefits, and related costs, totaling approximately $1.3 million. No further reduction in force was taken related to the decision to stop enrollment in ILLUMINATE-206.

We are actively evaluating other novel therapeutic assets, including developmental and potentially commercial-stage assets, which may represent an opportunity to expand our pipeline.

Nasdaq Compliance

As previously disclosed, on November 26, 2021, we received a deficiency letter (the “Nasdaq Letter”) from the Nasdaq Listing Qualifications Department, notifying us that we are not in compliance with Nasdaq Listing Rule 5550(a)(2), which requires us to maintain a minimum bid price of at least $1 per share for continued listing on The Nasdaq Capital Market (the “Minimum Bid Requirement”). The Company’s failure to comply with the Minimum Bid Requirement was based on the Company’s common stock per share price being below the $1 threshold for a period of 30 consecutive business days. In accordance with Nasdaq Listing Rule 5810(c)(3)(A) (the “Compliance Period Rule”), the Company has been provided an initial period of 180 calendar days (the “Compliance Date”), to regain compliance with the Minimum Bid Requirement. If, at any time before the Compliance Date, the bid price for the Company’s common stock closes at $1.00 or more per share for a minimum of 10 consecutive business

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days, as required under Nasdaq requirements, the Staff will provide written notification to the Company that it complies with the Minimum Bid Requirement, unless the Staff exercises its discretion to extend this 10-day period pursuant to Nasdaq Listing Rule 5810(c)(3)(H).

If the Company does not regain compliance with the Minimum Bid Requirement by the Compliance Date, the Company may be eligible for an additional 180 calendar day compliance period (the “Second Compliance Period”). To qualify, the Company would need to meet the continued listing requirement for the market value of publicly held shares and all other initial listing standards of the Nasdaq Capital Market, with the exception of the Minimum Bid Requirement, and provide written notice to the Staff of its intention to cure the deficiency during the Second Compliance Period.

Neither the Nasdaq Letter nor the Company’s noncompliance with the Minimum Bid Requirement have an immediate effect on the listing or trading of the Company’s common stock, which continues to trade on The Nasdaq Capital Market under the symbol “IDRA.”

Clinical Development

~~Tilsotolimod~~Our ~~(IMO-2125)~~ Pipeline

~~Tilsotolimod~~We isare adeveloping ~~synthetic~~ACG-701 ~~phosphorothioate oligonucleotide that acts as a direct agonist of TLR9~~ to ~~stimulate~~treat ~~the~~CF ~~innate~~PEx and ~~adaptive~~melioidosis, ~~immune systems. It was developed for administration via intratumoral injection in combination with systemically administered checkpoint inhibitors~~ and ~~costimulation~~ACG-801 ~~therapies for the treatment of various solid tumors. We referred~~ to ~~our~~treat ~~tilsotolimod~~Farber ~~development~~disease, ~~program~~each asof ~~the~~which ~~ILLUMINATE~~is ~~development~~discussed ~~program. See additional information under the heading “Collaborative Alliances” for information on the development of tilsotolimod~~ in ~~collaboration~~greater ~~with~~detail ~~AbbVie Inc~~below. ~~(“AbbVie”) for patients with head and neck squamous cell carcinoma (“HNSCC”).~~

ACG-701 (sodium fusidate)

~~ILLUMINATE~~ACG-~~206~~701 ~~- Phase 2 Trial of Tilsotolimod (IMO-2125) in Combination with Nivolumab and Ipilimumab~~ for ~~the~~Cystic ~~treatment~~Fibrosis ofPulmonary ~~Solid Tumors~~Exacerbations

ACG-701 is a proprietary oral, loading dose formulation of sodium fusidate being developed as a potential treatment for acute PEx associated with CF, a major factor driving lung function decline in people living with CF.

CF is a progressive, genetic disease hallmarked by inflammatory and infectious pulmonary exacerbations that are the primary cause of morbidity and mortality for CF patients. PEx are a sudden worsening of respiratory symptoms caused by lung infections that often require intravenous (“IV”) antibiotic treatment. Current standard of care for acute PEx in CF is multifaceted, involving antibiotics, chest physiotherapy, inhaled medications to promote secretion clearance, and anti-inflammatory agents.

Sodium fusidate has an established clinical efficacy and safety profile from more than 50 years of use in other countries, including as part of CF PEx treatment guidelines in the United Kingdom and Australia. Despite this, the compound has never been approved by the U.S. Food and Drug Administration (“FDA”) and represents a new and potentially powerful approach in the United States to address the infection, inflammation, and enhanced mucin expression that are hallmark features of CF PEx. According to the Cystic Fibrosis Foundation, in 2022, it was estimated that 105,000 patients have been diagnosed and were living with CF globally, with approximately 40,000 patients in the United States. If approved, ACG-701 would represent the first oral product in the United States indicated for the treatment of CF PEx.

In ~~September~~December ~~2019~~2022, we initiated the REPRIEVE study (NCT05641298), a Phase 2 randomized, ~~open~~double-~~label~~blinded, ~~global,~~placebo-controlled ~~multicohort~~ study toevaluating ~~evaluate~~ACG-701 for the ~~safety~~treatment ~~and~~of ~~effectiveness~~CF ofPEx ~~tilsotolimod~~(the ~~administered~~“REPRIEVE ~~intratumorally~~Study”), inwhich ~~combination~~is ~~with~~being ~~nivolumab~~funded ~~and~~in ~~ipilimumab~~part ~~for~~by ~~the~~$3.5 ~~treatment~~million ofin ~~solid~~support ~~tumors.~~from Wethe ~~refer~~Cystic toFibrosis ~~this~~Foundation. ~~study~~The asREPRIEVE ~~ILLUMINATE-206.~~Study, which is

The first solid tumor investigated under ILLUMINATE-206 was relapsed/refractory Microsatellite-Stable Colorectal Cancer (“MSS-CRC”) in immunotherapy-naïve patients (the “MSS-CRC Study”). To investigate the safety profile of the combination of tilsotolimod, nivolumab and ipilimumab, ILLUMINATE-206 was designed with a stepwise approach to ipilimumab dosage. An initial group of ten patients in the safety cohort of the MSS-CRC Study, many of whom were heavily pre-treated and rapidly progressing, received 8 mg of intratumoral tilsotolimod and 3 mg/kg of intravenous (IV) nivolumab every two weeks, along with 1 mg/kg of IV ipilumab every eight weeks (the “Low-Dose, Low-Frequency Cohort”). This regimen was generally well tolerated; no patients discontinued treatment due to adverse events (“AEs”) and no patients experienced Grade 4 or 5 AEs. As of the response data cutoff date, per Response Evaluation Criteria in Solid Tumors V1.1 (“RECIST V1.1”) criteria, one patient experienced stable disease (“SD”) and nine patients progressed. Investigators reported that six of the progressing patients had stability or reduction in size of injected lesions and six had stability or reduction in overall size of uninjected lesions.

Based on these results, we enrolled an additional ten patients in the MSS-CRC Study. Changes in the study design intended to improve potential outcomes in the targeted patient population included increasing the frequency of ipilimumab dosing to every three weeks and limiting the number of allowed prior lines of treatment to two. Accordingly, the second group of ten patients enrolled in the MSS-CRC Study received 8 mg of intratumoral tilsotolimod (total of 9 doses over approximately 28 weeks) and 3 mg/kg of intravenous (IV) nivolumab every three

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~~weeks~~expected ~~followed~~to byenroll ~~480~~80 mgpatients, will evaluate the efficacy and safety of IVACG-701 ~~nivolumab~~plus ~~every~~optimized ~~four~~background ~~weeks,~~therapy ~~along~~(OBT) ~~with~~compared 1to ~~mg/kg~~oral placebo plus OBT and capture multiple clinical events inclusive of IVpatient-reported ~~ipilimumab~~outcomes, ~~every~~FEV1, ~~three~~and ~~weeks~~antimicrobial regimen changes through day 14. The study opened for ~~four~~patient ~~doses~~enrollment (in March 2023 at clinical sites in the ~~“Low-Dose,~~United ~~High~~States. Patients enrolled in the REPRIEVE Study will either be administered oral ACG-~~Frequency~~701 ~~Cohort”~~tablets twice a day for 14 days plus OBT or placebo tablets twice a day for 14 days plus OBT. The REPRIEVE Study will apply a desirability of outcome ranking (DOOR) analysis to the results. Data from the REPRIEVE Study is expected in the first half of 2024.

ACG-701 has received Orphan Drug designations for the treatment of CF patients from the FDA. In addition, we have also received Fast Track and Qualified Infectious Disease Product (“QIDP”) designation for ACG-701 for the treatment of CF PEx. If approved, QIDP will provide an additional five-year extension of regulatory exclusivity to any exclusivities that the product may otherwise qualify.

ACG-701 for Melioidosis

ACG-701 is also being developed for the treatment of melioidosis, a life-threatening infection that can affect numerous organ systems, including the lungs. The pathogen that causes melioidosis, B. pseudomallei, is endemic in Southeast Asia and is classified as a Tier 1 biothreat agent by the U.S. government. The U.S. Department of Defense’s Defense Threat Reduction Agency (“DTRA”) is supporting the development of ACG-701 as a potential medical countermeasure against this pathogen and has awarded us funding of up to $49.7 million, of which $30.0 million remains available as of December 31, 2022.

In May 2022, we initiated the TERRA study (NCT05105035), a Phase 2 randomized, double-blind, placebo-controlled study evaluating ACG-701 for the treatment of melioidosis in hospitalized patients (the “TERRA Study”). The TERRA Study, which is actively recruiting and expected to enroll 125 patients, will evaluate the efficacy, safety and tolerability of ACG-701 given in combination with IV ceftazidime or IV meropenem and capture multiple clinical events inclusive of mortality, organ failure, sepsis, and treatment modifications through day 14. Patients enrolled in the study will be administered either IV ceftazidime or IV meropenem per standard of care and either placebo or ACG-701 every 12 hours for days 1-14. Day 1 dosing consists of two doses of 1500mg ACG-701 administered 12 hours apart, and day 2-14 dosing consists of 600mg of ACG-701 administered every 12 hours.

An independent data monitoring committee (“iDMC”) is responsible for overseeing the safety and efficacy data. In February 2023, we announced that the iDMC for the TERRA Study met and recommended that the TERRA Study continue without modification. A data read-out from the TERRA Study is expected in the fourth quarter of 2023.

~~In December 2021, we announced that preliminary data from the second ten patients dosed in the safety cohort of ILLUMINATE~~ACG-~~206~~801 ~~showed~~(acid ~~a safety profile consistent with the first ten patients in ILLUMINATE-206 and with prior studies.~~ ceramidase)

ACG-801 for Farber Disease

ACG-801, recombinant human acid ceramidase, is an investigational biological enzyme replacement therapy being developed for the treatment of Farber disease. Farber disease is a severe, progressive monogenic lysosomal storage disorder, involving mutations in the acid ceramidase gene that lead to toxic levels of ceramide accumulation. Acid ceramidase acts in the lysosome to metabolize ceramide, a pro-inflammatory lipid. Loss of acid ceramidase function leads to abnormal accumulation of ceramide, causing macrophage-driven inflammation and multi-organ disease affecting bone, cartilage, the immune system, central nervous system, and the lungs. Patients with the most severe phenotype of Farber disease die early in life, most commonly from respiratory failure. As of December 31, 2022, we estimated that worldwide prevalence of Farber disease exceeded 1,000 patients. We are not aware of any competitive development programs seeking to treat Farber disease and there are no Farber disease-specific treatments currently approved by the FDA.

We are planning a single, harmonized trial for regulatory submission for both FDA and European Medicines Agency (“EMA”) approval, known as the ADVANCE Study, which, as discussed in greater detail below, has been partially funded by NovaQuest (as defined below). A randomized, double-blind, placebo-controlled study of Farber disease patients, the ADVANCE study will measure nodule changes and capture patient-specific disease burden improvement through week 28. We have had regular interactions with the FDA, including a clinical-

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focused Type C meeting in January 2023 to discuss trial design. Following resolution of the clinical hold placed on ACG-801 pertaining to manufacturing and quality issues, which we filed a response to in March 2023, we expect to initiate ADVANCE Study clinical activities in the first quarter of 2024.

The FDA has granted Orphan and Fast Track designations for ACG-801. The FDA has also designated ACG-801 as a product for rare pediatric diseases. If the ACG-801 marketing application is ultimately determined to meet the FDA’s criteria to be a rare pediatric disease application, ACG-801 may be eligible for a Rare Pediatric Disease priority review voucher provided that the marketing application is approved before the program sunsets. Additionally, ACG-801 was granted Orphan Drug Designation by the EMA for Farber disease.

Discontinued Programs

Tilsotolimod (IMO-2125)

OfTilsotolimod ~~the~~is ~~eight patients who had~~ a ~~post-baseline~~synthetic ~~disease~~phosphorothioate ~~assessment~~oligonucleotide ~~evaluated~~that ~~per~~acts ~~RECIST~~as ~~v1.1,~~a ~~one~~direct ~~experienced~~agonist SDof ~~with~~TLR9 ~~disease~~to ~~control~~stimulate ~~for more than six months;~~ the ~~remaining~~innate ~~patients~~and ~~experienced~~adaptive ~~Progressive~~immune ~~Disease (“PD”)~~systems. ~~However,~~It ~~one of the RECIST v1.1 PD patients~~ was ~~determined~~developed tofor ~~have~~administration ~~experienced~~via ~~pseudo-progression,~~intratumoral ~~meaning~~injection ~~that the initial increase from baseline~~ in ~~overall~~combination ~~tumor~~with ~~burden~~systemically ~~was~~administered ~~followed~~checkpoint byinhibitors ~~a decrease from baseline in overall tumor burden~~ and, ~~based~~costimulation ontherapies for the ~~total~~treatment ~~decrease~~of ~~from~~various ~~baseline,~~solid ~~was considered an Immune-Related Partial Response (“irPR”) by Immune-Related RECIST (“irRECIST”)~~tumors. ~~The~~We ~~patient~~referred ~~continued in active treatment, as allowed per protocol, through February 2022, when they withdrew due~~ to ~~travel~~our ~~constraints.~~tilsotolimod Asdevelopment aprogram ~~result,~~as wethe ~~are~~ILLUMINATE indevelopment ~~process of concluding all study-related activities for ILLUMINATE-206~~program.

AsIn ~~further~~the ~~discussed~~first inquarter ~~this~~of ~~annual~~2018, ~~report~~we ~~under~~initiated ~~the~~a ~~caption~~Phase ~~“Item~~3 1.trial ~~Business~~of -intratumoral ~~Collaborative~~tilsotolimod ~~Alliances”,~~in combination with ipilimumab in ~~March~~patients ~~2019~~with anti-PD-1 refractory melanoma, which we ~~entered~~referred ~~into~~to as ILLUMINATE-301. This trial compared the results of the tilsotolimod–ipilimumab combination to those of ipilimumab alone in a ~~clinical~~1:1 randomization. The family of primary endpoints of the trial ~~collaboration~~consisted ~~and~~of ~~supply~~Objective ~~agreement~~Response ~~with~~Rate ~~BMS,~~(“ORR”) ~~under~~by ~~which~~blinded ~~BMS~~independent ~~agreed~~central toreview ~~manufacture~~RECIST v1.1 and ~~supply~~median ~~ipilimumab~~Overall ~~and~~Survival ~~nivolumab~~(“OS”). In March 2021, atwe reported that ILLUMINATE-301 did not meet its ~~cost~~primary ~~and~~endpoint ~~for~~of noORR. ~~charge~~In toMay us2021, ~~for~~following ~~use~~evaluation inof the full data set, we announced we would not continue ILLUMINATE-~~206~~301 to its OS primary endpoint.

In September 2019, we initiated a Phase 2, open-label, global, multicohort study to evaluate the safety and effectiveness of tilsotolimod administered intratumorally in combination with nivolumab and ipilimumab for the treatment of solid tumors, which we referred to as ILLUMINATE-206. The first solid tumor investigated under ILLUMINATE-206 was relapsed/refractory Microsatellite-Stable Colorectal Cancer (“MSS-CRC”) in immunotherapy-naïve patients (the “MSS-CRC Study”). In December 2021, based on an analysis of preliminary data from the safety cohort of ILLUMINATE-206, we announced that further enrollment in the trial had been discontinued. The decision to discontinue ILLUMINATE-206 was not related to safety concerns.

~~ILLUMINATE-301~~Following -the ~~Phase~~announcement 3that ~~Trial~~all ~~of Tilsotolimod (IMO~~Company-~~2125)~~sponsored indevelopment ~~Combination~~of ~~with~~tilsotolimod ~~Ipilimumab~~was discontinued in ~~Patients~~December ~~with~~2021, ~~Anti~~all study-~~PD1~~related ~~Refractory~~activities ~~Melanoma~~concluded.

InCollaborative ~~the~~Alliances first quarter of 2018, we initiated a Phase 3 trial of intratumoral tilsotolimod in combination with ipilimumab in patients with anti-PD-1 refractory melanoma, which we referred to as ILLUMINATE-301. This trial compared the results of the tilsotolimod–ipilimumab combination to those of ipilimumab alone in a 1:1 randomization. The family of primary endpoints of the trial consisted of ORR by blinded independent central review RECIST v1.1 and ~~median~~Clinical ~~OS.~~Funding Arrangements

AsOur ~~discussed~~current ~~above,~~collaborative inalliances ~~March~~and ~~2021,~~clinical wefunding ~~reported~~arrangements ~~that~~include ~~ILLUMINATE-301~~agreements ~~did~~with ~~not~~the ~~meet~~Cystic ~~its~~Fibrosis ~~primary~~Foundation, ~~endpoint~~DTRA of(as ~~ORR.~~defined Inbelow), ~~May~~and ~~2021~~Scriptr Global, ~~following~~Inc. ~~evaluation~~(“Scriptr”), ofeach ~~the~~discussed ~~full~~further ~~data~~below. ~~set,~~We wemay ~~announced~~seek weto ~~would~~enter ~~not~~into ~~continue~~new ~~ILLUMINATE-301~~funding arrangements or collaborative alliances to ~~its~~support OSdevelopment ~~primary~~and ~~endpoint~~commercialization of additional drug candidates.

~~Collaborative~~Cystic ~~Alliances~~Fibrosis Foundation

~~Our~~On ~~current~~December ~~alliances~~13, ~~include~~2021, ~~collaborations~~Legacy Aceragen entered into a therapeutic development and award agreement (the “CFF Agreement”) with ~~Scriptr~~The ~~Global~~Cystic Fibrosis Foundation (“CFF”), ~~Inc~~pursuant to which CFF agreed to fund the REPRIEVE Study, as described above under the heading “Clinical Development,” in an amount up to $3.5 million (the “~~Scriptr~~CFF Award”), ~~AbbVie~~of which $1.0 million had been received as of December 31, 2022. In consideration for the CFF Award and ~~BMS~~CFF’s license of CFF Know-How, Legacy Aceragen agreed to pay CFF royalties of up to $21.0 Wemillion ~~may~~as ~~seek~~follows: (a) one-time royalty equal to ~~enter~~3x ~~into~~the ~~new~~CFF ~~collaborative~~Award ~~alliances~~if a product resulting from the REPRIEVE Study is approved for commercial sale; (b) an amount equal to ~~support~~1x ~~development~~the ~~and~~CFF ~~commercialization~~Award in the event of ~~additional~~a ~~drug~~license ~~candidates.~~transaction or change in control, as defined in the CFF Agreement; and (c) a one-time royalty equal to 1x the CFF

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Award upon (i) attainment of reaching net sales of $250 million and (ii) upon attainment of reaching net sales of $500 million.

Department of Defense’s Defense Threat Reduction Agency (“DTRA”)

In connection with the Aceragen Acquisition, we assumed development contracts awarded to Legacy Aceragen in June 2020 and August 2021 which provide for funding of up to $49.7 million in the aggregate by the Department of Defense’s DTRA for the clinical and regulatory development of ACG-701 as a potential medical countermeasure against the pathogen that causes melioidosis, B. Pseudomallei, as further discussed above. As of December 31, 2022, $19.7 million had been used and $30.0 million remains available for use through December 2026.

Collaboration with Scriptr

In February 2021, we entered into a collaboration and option agreement with Scriptr, pursuant to which (i) Scriptr and Idera will conduct a research collaboration utilizing Scriptr Platform Technology (“SPT”) to identify, research, and develop gene therapy candidates (each, a “Collaboration Candidate”) for the treatment, palliation, diagnosis, or prevention of (a) myotonic dystrophy type 1 (“DM1 Field”) and (b) Friedreich’s Ataxia (“FA Field”) on a Research Program-by-Research Program basis, as applicable, and (ii) we were granted an exclusive option, in our sole discretion, to make effective the Scriptr License Agreement, (as defined below,) for a given Research Program, (as defined below,) to make use of Collaboration Candidates and related intellectual property (collectively, the “Scriptr Agreement”).

Pursuant to the Scriptr Agreement, Scriptr will use commercially reasonable efforts to carry out research activities set forth in accordance with the applicable DM1 Field and FA Field research plans, including certain pre-clinical proof of concept studies, to identify research Collaboration Candidates utilizing SPT (each, a “Research Program”). Following the completion of activities under a given Research Program, Scriptr will prepare and submit to us a comprehensive data package (each, a “Data Package”) that summarizes, on a Research Program-by-Research Program basis, any Collaboration Candidates researched under the Research Program, including any data and results. Upon receipt of a Data Package, we have, in our sole discretion, up to two-hundred seventy (270) calendar days to make effective the exclusive license agreement entered into by and between Scriptr and us, pursuant to which, among other things, Scriptr grants us exclusive rights and licenses with respect to the development, manufacture and commercialization of licensed candidates and products, subject to certain conditions and limitations (the “Scriptr License Agreement”), for a given Research Program (each licensed Research Program, a “Licensed Program”). The Scriptr License Agreement provides for customary development milestones on candidates developed under a Licensed Program and royalties on licensed products, if any.

In partial consideration of the rights granted by Scriptr to us under the Scriptr Agreement, we made a one-time, non-creditable, and non-refundable payment to Scriptr during the first quarter of 2021. In order to fund the Research Programs, we reimburse Scriptr for costs incurred by or on behalf of Scriptr in connection with the conduct of each Research Program during the research term in accordance with the applicable Research Program budget and payment schedule. We incurred $0.5 million and $2.1 million in research and development expenses under the Scriptr Agreement during the years ended December 31, 2022 and 2021, respectively.

Academic and Research Collaborations

We have entered into research collaborations with scientists at leading academic research institutions. These research collaborations allow us to augment our internal research capabilities and obtain access to specialized knowledge and expertise. In general, our research collaborations may require us to supply compounds and pay various amounts to support the research. Under these research agreements, if a collaborator, solely or jointly with us, creates any invention, we may own exclusively such invention, have an automatic paid-up, royalty-free, non-exclusive license, or have an option to negotiate an exclusive, worldwide, royalty-bearing license to such invention. Inventions developed solely by our scientists in connection with research collaborations are owned exclusively by us. These collaborative agreements are non-exclusive and may be terminated with limited notice.

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toOther usMaterial a comprehensive data package (each, a “Data Package”) that summarizes, on a Research Program-by-Research Program basis, any Collaboration Candidates researched under the Research Program, including any data and results. Upon receipt of a Data Package, we have, in our sole discretion, up to two-hundred seventy (270) calendar days to make effective the exclusive license agreement entered into by and between Scriptr and us, pursuant to which, among other things, Scriptr grants us exclusive rights and licenses with respect to the development, manufacture and commercialization of licensed candidates and products, subject to certain conditions and limitations (the “Scriptr License Agreement”), for a given Research Program (each licensed Research Program, a “Licensed Program”). The Scriptr License Agreement provides for customary development milestones on candidates developed under a Licensed Program and royalties on licensed products, if any.Contracts

InNovaQuest partial consideration of the rights granted by Scriptr to us under the Scriptr Agreement, we made a one-time, non-creditable and non-refundable payment to Scriptr during the first quarter of 2021. In order to fund the Research Programs, we will reimburse Scriptr for costs incurred by or on behalf of Scriptr in connection with the conduct of each Research Program during the research term in accordance with the applicable Research Program budget and payment schedule. We incurred approximately $2.1 million in research and development expenses under the Scriptr Agreement during the year ended December 31, 2021. Agreements

~~Collaboration~~NovaQuest ~~with~~Side ~~AbbVie~~Letter

~~Effective~~At ~~August~~closing ~~27,~~of ~~2019~~the Aceragen Acquisition, we entered into a ~~clinical~~side ~~trial~~letter ~~collaboration and supply~~ agreement (the “~~AbbVie~~NovaQuest ~~Agreement~~Side Letter”) with ~~AbbVie~~NovaQuest Co-Investment Fund XV, aL.P. ~~global~~(“NovaQuest”), ~~research-based~~pursuant ~~biopharmaceutical~~to ~~company,~~which: (i) NovaQuest was granted customary observer rights to ~~conduct~~have a ~~clinical~~managing ~~study~~director toor ~~evaluate~~investment ~~the~~professional ~~efficacy~~of ~~and~~NovaQuest ~~safety~~Capital Management, L.L.C. attend all meetings of ~~combinations~~our board of andirectors ~~OX40~~and ~~agonist~~all ~~(ABBV-368)~~meetings of the Audit Committee, ~~tilsotolimod~~Compensation Committee, ~~nab-paclitaxel~~and Nominating and~~/or~~ anCorporate ~~anti~~Governance Committee thereof in a non-~~programmed~~voting ~~cell~~capacity ~~death~~until 1such time as NovaQuest no longer beneficially owns shares of Series X Preferred Stock (~~PD-1)~~as ~~antagonist~~defined below); (~~ABBV-181~~ii). we ~~Under~~agreed ~~the~~to ~~AbbVie~~grant ~~Agreement,~~NovaQuest customary registration rights in the event we ~~agreed~~in the future grant registration rights to ~~provide~~any ainvestor ~~clinical~~or ~~trial~~equity ~~supply~~holder ofwith ~~tilsotolimod~~respect to ~~AbbVie~~shares ~~and~~of ~~AbbVie~~our ~~will~~capital ~~sponsor,~~stock; ~~fund~~and (iii) we acknowledged and ~~conduct~~agreed that pursuant to the ~~study~~terms ~~entitled~~of “Athe ~~Phase~~Series X ~~1b,~~Preferred ~~Multicenter~~Stock Certificate of Designation, ~~Open-Label~~we ~~Study~~shall tonot ~~Determine~~authorize or issue shares of its capital stock unless the ~~Safety,~~same ~~Tolerability,~~ranks ~~Pharmacokinetics~~junior to the Series X Preferred Stock, ~~and~~or ~~Preliminary~~increase ~~Efficacy~~the authorized number of ~~ABBV-368~~shares ~~plus~~of ~~Tilsotolimod~~Series X ~~and~~Preferred ~~Other~~Stock ~~Therapy~~or ~~Combinations~~any inadditional ~~Subjects~~class ~~with~~of ~~Recurrent/Metastatic~~capital ~~Head~~stock ~~and~~unless ~~Neck~~the ~~Squamous~~same ~~Cell~~ranks ~~Carcinoma”~~junior (to the ~~“AbbVie~~Series X ~~Study”).~~Preferred Stock, in each case until such time as NovaQuest no longer beneficially owns shares of Series X Preferred Stock.

InNovaQuest ~~December~~Purchase ~~2021,~~Agreement ~~AbbVie~~(Related ~~announced the discontinuation of further patient enrollment in the AbbVie Study. The decision~~ to ~~discontinue the AbbVie Study was not related to safety concerns. Current patient treatment and follow~~ACG-~~up is ongoing.~~ 801)

~~Collaboration~~Pursuant to the Stock and Warrant Purchase Agreement, dated as of March 24, 2021, by and between Legacy Aceragen and NovaQuest, as amended by that Amendment, dated October 25, 2021, and as such agreement may be amended from time to time (the “Purchase Agreement”), NovaQuest agreed to provide up to $35.0 million in product-based financing to support the development of ACG-801 for Farber disease. The financing was to be provided through (i) $15.0 million in proceeds from the sale of Legacy Aceragen capital stock and warrants to purchase shares of Legacy Aceragen capital stock, and (ii) up to $20.0 million in capital contributions for development funding relating to the treatment of Farber disease (“Capital Contributions”). The Capital Contributions were to be paid by NovaQuest in quarterly installments for Legacy Aceragen’s eligible expenses associated with ~~Bristol~~the development of ACG-~~Meyers~~801 ~~Squibb~~for Farber disease (“ACG-801 Product”). Prior to the Aceragen Acquisition, Legacy Aceragen received $20.0 million in Capital Contributions, representing the total eligible Capital Contributions provided for under the Purchase Agreement.

WeNovaQuest, ~~entered~~holders ~~into~~of ~~two~~Series X ~~clinical~~Preferred ~~collaboration~~Stock ~~agreements~~(as ~~with~~defined ~~BMS~~below), ~~the~~are ~~first~~entitled to receive distributions on shares of Series X Preferred Stock as set forth in ~~May~~(a) the ~~2018~~Purchase Agreement, and ~~the~~(b) that ~~second~~certain inSales ~~March~~Distribution ~~2019~~and PRV Agreement, todated ~~support~~as ~~ILLUMINATE-301~~of October 25, 2021, by and ~~ILLUMINATE-206~~between Legacy Aceragen and NovaQuest, ~~respectively.~~as such agreement may be amended from time to time (the “PRV Agreement”), prior and in preference to any declaration or payment of any other distribution or dividend (other than dividends on shares of Common Stock payable in shares of Common Stock).

~~Under~~Pursuant to the ~~May~~terms ~~2018~~and ~~BMS~~conditions of the Purchase Agreement, ~~BMS~~we ~~granted~~must usmake a ~~non-exclusive,~~distribution ~~non-transferrable,~~to ~~royalty-free~~NovaQuest ~~license~~of (35% of the net proceeds we receive in connection with a transaction (a “PRV Sale Transaction”), pursuant to which we sell any of our right, title, and interest in and to ~~sublicense~~a priority review voucher (“PRV”) ~~under~~granted ~~its~~by ~~intellectual~~the ~~property~~FDA toin ~~use~~connection ~~ipilimumab~~with regulatory approval of ACG-801 Product. Further, in ~~ILLUMINATE~~the event that we do not receive a PRV in connection with regulatory approval of ACG-~~301~~801 ~~and~~Product ~~agreed~~by tothe ~~manufacture~~FDA, ~~and~~or ~~supply~~do ~~ipilimumab~~not complete a PRV Sale Transaction within 12 months after our receipt of a PRV, atthen ~~its~~we ~~cost~~shall ~~and~~make ~~for~~a nodistribution ~~charge~~to NovaQuest equal to us$35 million in two equal installments, ~~for~~the ~~use~~first inof ~~ILLUMINATE~~which shall be effected within 45 days after the date we receive regulatory approval of ACG-~~301~~801 Product from the FDA (the “U.S. Approval Date”) and the second of which shall be effected within one year after the U.S. Approval Date.

~~Under~~In addition, pursuant to the ~~March~~terms ~~2019~~and ~~BMS~~conditions of the Purchase Agreement, ~~BMS~~after ~~granted~~first uscommercial asale ~~non-exclusive,~~of ~~non~~ACG-~~transferrable,~~801 ~~royalty-free~~Product ~~license~~and continuing for each fiscal quarter until the Distribution End Date (~~with~~as defined therein), we shall make a ~~right~~distribution to ~~sublicense)~~NovaQuest ~~under~~(the ~~its~~“Required ~~intellectual~~Net ~~property~~Sales Distribution”) equal to ~~use~~the ~~ipilimumab~~product ~~and~~of ~~nivolumab~~the inRequired ~~ILLUMINATE-206~~Net ~~and~~Sales ~~agreed~~Distribution toRate ~~manufacture~~(as ~~and~~defined ~~supply~~below) ~~both~~multiplied ~~ipilimumab~~by ~~and~~the ~~nivolumab,~~aggregate attotal ~~its~~of ~~cost~~the ~~and~~net sales of ACG-801 Product for nosuch ~~charge~~fiscal quarter. The Required Net Sales Distribution Rate shall initially be 15%; provided that once the aggregate of all distributions paid to usNovaQuest equals $140 million, ~~for~~the ~~use~~Required inNet ~~ILLUMINATE-206.~~Sales

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Distribution Rate shall decrease to 5%. The Required Net Sales Distribution shall end after the date on which the last of the following has occurred: (i) we pay $140 million to NovaQuest; (ii) the last valid patent covering ACG-801 Product in the United States and European Union has expired; and (iii) regulatory exclusivity for ACG-801 Product in both the United States and the European Union has expired.

~~Academic~~Pursuant to the terms and ~~Research~~conditions ~~Collaborations~~of the Purchase Agreement, we must use commercially reasonable efforts to (i) develop ACG-801 in a manner that ensures that Aceragen is reasonably likely to obtain regulatory approval from the FDA for Farber disease no later than September 1, 2024 and to obtain regulatory approval from the European Medicines Agency (EMA) no later than October 1, 2024; (ii) identify a third-party purchaser of a PRV, if we receive a PRV, and consummate a PRV Sales Transaction (as defined therein) within 12 months of receipt of the PRV; and (iii) commercialize ACG-801 Product in each jurisdiction in which regulatory approval is received.

WeNovaQuest ~~have~~PRV ~~entered~~Agreement ~~into~~(Related ~~research collaborations with scientists at leading academic research institutions. These research collaborations allow us~~ to augment our internal research capabilities and obtain access to specialized knowledge and expertise. In general, our research collaborations may require us to supply compounds and pay various amounts to support the ~~research.~~Arrevus Under these research agreements, if a collaborator, solely or jointly with us, creates any invention, we may own exclusively such invention, have an automatic paid-up, royalty-free non-exclusive license or have an option to negotiate an exclusive, worldwide, royalty-bearing license to such invention. Inventions developed solely by our scientists in connection with research collaborations are owned exclusively by us. These collaborative agreements are non-exclusive and may be terminated with limited notice. Acquisition)

4Pursuant to the terms and conditions of the PRV Agreement related to Legacy Aceragen’s acquisition of Arrevus, Inc., we must make a distribution to NovaQuest of 10% of the net proceeds we receive in connection with a PRV Sales Transaction pursuant to which we sell any of our right, title, and interest in and to a PRV that we receive in connection with regulatory approval of ACG-701. Further, in the event that we do not receive a PRV in connection with regulatory approval of ACG-701 by the FDA or do not complete a PRV Sales Transaction within 12 months after our receipt of such PRV, then we must make certain satisfaction milestone payments in favor of NovaQuest.

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Research and Development Expenses

WePursuant ~~are~~to ~~committed~~the toterms ~~redefining~~and conditions of the ~~treatment~~PRV ofAgreement, ~~certain~~we ~~cancers~~must ~~and~~use ~~rare~~commercially ~~diseases~~reasonable ~~and~~efforts ~~have~~to ~~historically~~(i) develop ~~dedicated~~ACG-701 in a ~~significant~~manner ~~portion~~that ofensures ~~our~~that ~~resources~~we toare ~~our~~reasonably ~~efforts~~likely onto ~~the~~obtain ~~discovery~~regulatory ~~and~~approval ~~development~~from ofthe ~~our~~FDA ~~drug~~for ~~candidates. For~~ACG-701 ~~the~~by ~~years~~no ~~ended~~later than December 31, ~~2021~~2024, ~~2020~~(ii) if we receive a PRV in connection with regulatory approval of ACG-701, identify a third-party purchaser of such PRV and ~~2019,~~consummate wea ~~spent~~PRV ~~approximately~~Sales ~~$16.4~~Transaction ~~million,~~within ~~$24.8~~12 months ~~million~~of receipt of such PRV, and ~~$34~~(iii) commercialize ACG-701 in each jurisdiction for which regulatory approval is received.9 ~~million,~~After ~~respectively,~~first oncommercial ~~research~~sale of ACG-701 and ~~development~~continuing ~~activities.~~for Weeach ~~plan~~fiscal toquarter ~~continue~~until tothe ~~invest~~Distribution inEnd ~~research~~Date ~~and~~(as ~~development.~~defined ~~Accordingly~~therein), we ~~anticipate~~shall amake ~~significant~~the ~~portion~~Required Net Sales Distribution equal to product of ~~our~~5% ~~operating~~multiplied ~~expenses~~by ~~will~~the ~~continue~~aggregate tototal beof ~~related~~net tosales ~~research~~of ~~and~~ACG-701 ~~development~~for insuch ~~2022~~fiscal ~~and~~quarter. ~~beyond~~The Required Net Sales Distribution shall end after we pay $50 million to NovaQuest.

5Customers

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Patents, Proprietary Rights and Trade Secrets

~~Our~~The success depends in part on our ability to obtain and maintain proprietary protection for our drug candidates, technology and know-how, to operate without infringing the proprietary rights of others and to prevent others from infringing our proprietary rights. We use a variety of methods to seek to protect our proprietary position, including filing U.S. ~~and~~ ~~foreign~~Government ~~patent~~accounted ~~applications~~for ~~related~~all toof our ~~proprietary~~revenues ~~technology,~~for ~~inventions and improvements that are important to~~ the ~~development~~year ofended ~~our business. We also rely on trade secrets~~December 31, ~~know-how, continuing technological innovation, and in-licensing opportunities to develop and maintain our proprietary position~~2022.

WeIntellectual ~~have devoted and continue to devote a substantial amount of our resources into establishing intellectual property protection for:~~ Property

● Novel chemical entities that function as agonists of TLR3, TLR7, TLR8 or TLR9;

● Novel chemical entities that function as antagonists of TLR7, TLR8 or TLR9; and

● Composition and use of our nucleic acid chemistry compounds to treat and prevent a variety of diseases.

OnOur ~~November~~success 5depends in part on our ability to obtain and maintain proprietary protection for our product candidates, ~~2019~~technology and know-how, to operate without infringing the ~~U.S.~~proprietary ~~Patent~~rights of others and ~~Trademark~~to ~~Office~~prevent ~~(“USPTO”)~~others ~~issued~~from toinfringing usour Uproprietary rights.S. ~~Patent~~We ~~No.~~use ~~10,463,686~~a ~~entitled~~variety ~~“Immune~~of ~~Modulation~~methods ~~With~~to ~~TLR9~~seek ~~Agonists~~to ~~For~~protect ~~Cancer~~our ~~Treatment~~proprietary position,” ~~which~~including ~~includes~~filing ~~tilsotolimod~~U.S. and ~~The~~foreign patent ~~includes~~applications 24related ~~claims~~to ~~directed~~our toproprietary ~~methods~~technology, ofinventions ~~treating~~and ~~melanoma~~improvements ~~with~~that ~~intratumoral~~are ~~administration~~important to the development of ~~tilsotolimod~~our inbusiness. ~~combination~~We ~~with~~also ~~certain~~rely ~~immune~~on ~~checkpoint~~trade ~~inhibitor~~secrets, ~~therapies~~know-how, ~~including~~continuing ~~inhibitors~~technological ofinnovation, ~~the~~and ~~CTLA~~in-4licensing opportunities to develop and ~~PD-1/PD-L1~~maintain ~~pathways~~our proprietary position. ~~The~~We ~~patent~~also isintend ~~expected~~to rely on regulatory exclusivities, when available, to ~~expire~~protect inthe ~~September~~market ~~2037~~for any of our product candidates that receive regulatory approval.

OnOur ~~September~~intellectual ~~15,~~property ~~2020,~~is ~~the~~critical ~~USPTO~~to ~~issued~~our ~~U.S.~~business ~~Patent~~and ~~No.~~we 10strive to protect it,~~772,907~~ (including by seeking to obtain and maintain patent protection in the ~~“‘907~~United ~~Patent”)~~States and internationally to uscover our product candidates, ~~entitled~~their ~~“Immune~~methods ~~Modulation~~of ~~with~~use, ~~TLR9~~and ~~Agonists~~processes for ~~Cancer~~their ~~Treatment,”~~manufacture ~~which~~and ~~includes~~any ~~our~~other ~~investigational~~inventions ~~therapy~~that ~~tilsotolimod~~are commercially important to the development of our business. ~~The~~Our ~~‘907~~patent ~~Patent~~portfolio includes 26patent ~~claims~~and ~~directed~~patent toapplications ~~methods~~that ofwe ~~treating~~own ~~colorectal~~or ~~cancer~~exclusively ~~(“CRC”)~~license with ~~intratumoral~~composition ~~administration~~ of ~~tilsotolimod~~matter inand ~~combination~~method of use claims with ~~certain~~respect ~~immune~~to ~~checkpoint~~our ~~inhibitor~~product ~~therapies~~candidates, ~~including~~ACG-701 ~~CTLA~~and ACG-4801. Our policy is to pursue, ~~PD-1~~maintain and defend patent rights whether developed internally or ~~PD-L1~~licensed ~~inhibitors.~~from third parties and to protect the technology, inventions and improvements that are commercially important to the development of our business.

On November 17, 2020, the USPTO issued to us U.S. Patent No. 10,835,550 (the “‘550 Patent”) entitled “Immune Modulation with TLR9 Agonists for Cancer Treatment,” which includes our investigational therapy tilsotolimod. The ‘550 Patent includes 29 claims directed to methods of treating HNSCC with intratumoral administration of tilsotolimod in combination with certain immune checkpoint inhibitor therapies, including CTLA-4, PD-1 or PD-L1 inhibitors. 7

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Our patent portfolios for our ACG-701 ad ACG-801 candidates are summarized below.

ACG-801

OnWe ~~January~~exclusively ~~18,~~license ~~2022~~from Mt. Sinai 4 patent families that claim certain compositions, ~~the~~method ~~USPTO~~of ~~issued~~making and methods of use with respect to usacid ~~U.S.~~ceramidase ~~Patent~~and NoACG-801. 11One of the patent families includes granted patents in the United States,~~224~~ Europe,~~611~~ ~~(the~~Japan, ~~“‘611~~Europe, ~~Patent”~~Israel, Mexico, and Hong-Kong, ~~together~~and ~~with~~pending ~~‘907~~applications ~~Patent~~in Brazil, Canada, Europe, Hong-Kong, Japan, Israel, Mexico, Saudi Arabia, and United States. The granted and pending claims in this patent family are directed to certain compositions of acid ceramidase and methods of producing the ~~‘550~~compositions. ~~Patent~~The 20-year term for patents in this family runs through 2034, absent any available patent term adjustments or extension. A second patent family currently includes pending patent applications in the “United States, Australia, Canada, Europe, Hong-Kong, Japan, Mexico, Jordan, and New ~~Patents”)~~Zealand ~~entitled~~that ~~“immune~~claim ~~Modulation~~certain ~~with~~methods ~~TLR9~~of ~~Agonists~~treating ~~for~~Farber ~~Cancer~~Disease ~~Treatment,”~~using ~~which~~acid ceramidase and ACG-801. A third patent family currently includes ~~our~~granted ~~investigational~~patents ~~therapy~~in ~~tilsotolimod~~the United States, Europe, Hong-Kong, and Japan, and pending applications in the United States, Canada, China, Europe, Hong-Kong, and Japan. The ~~‘611~~granted ~~Patent~~and ~~includes~~pending 30 claims in this family are directed to certain methods of treating ~~kidney~~infections ~~cancer~~in patients with ~~intratumoral~~cystic ~~administration~~fibrosis, ofchronic ~~tilsotolimod~~obstructive pulmonary disease, or an open wound. The 20-year term for patents in ~~combination~~this family runs through 2033, absent any available patent term adjustments or extension. A fourth patent family currently includes granted patents in the United States and Europe, and pending applications in each of these jurisdictions with claims directed to certain ~~immune~~methods ~~checkpoint~~of ~~inhibitor~~using ~~therapies,~~acid ~~including~~ceramidase ~~CTLA~~to produce chondrocytes. The 20-4year term for patents in this family runs through 2032, ~~PD-1~~absent orany ~~PD-L1~~available ~~inhibitors~~patent term adjustments or extensions.

We own 3 patent families directed to certain therapeutic uses of acid ceramidase and ACG-801, including for treating Farber Disease. One of the patent families includes pending applications in United States, Australia, Brazil, Canada, Columbia, China, Europe, Hong Kong, Israel, Japan, South Korea, Mexico, Russia, New Zealand, Philippines, and Thailand that claim certain methods of treating Farber Disease using acid ceramidase and ACG-801. The 20-year term for patents in this family runs through 2039, absent any available patent term adjustments or extensions. A second patent family currently includes pending patent applications in United States, Australia, Brazil, Canada, China, Columbia, Europe, Hong Kong, Israel, Japan, South Korea, Mexico, Russia, South Africa, New Zealand, and Philippines that claim certain biomarkers of and methods of assessing Farber Disease. The 20-year term for patents in this family runs through 2039, absent any available patent term adjustments or extensions. A third patent family includes pending patent applications in United States and Argentina with claims directed to treating certain inflammatory disorders associated with Farber Disease. The 20-year term for patents in this family runs through 2041.

ACG-701

~~The~~We ~~New~~own ~~Patents~~2 ~~expand~~patent ~~protection~~families ofdirected to ACG-701 and certain therapeutic uses thereof. One patent family includes patents issued in the ~~first~~U.S. ~~tilsotolimod~~and ~~method-~~Canada that claim certain therapeutic uses of fusidic acid for treating bacterial infections. The 20-~~use~~year ~~patent~~term for patents in this family runs through 2030, ~~which~~absent ~~was~~any ~~directed~~availably topatent ~~methods~~term ofadjustments ~~treating~~or ~~metastatic~~extensions. ~~melanoma~~A ~~and~~second ~~was~~patent family includes a patent issued in ~~November~~Japan ~~2019~~and a pending application in the U.S. ~~The~~that ~~New~~claims ~~Patents~~compositions ~~provide~~of ~~exclusivity~~fusidic acid. The 20-year term for ~~certain~~patents ~~uses~~in ofthis ~~tilsotolimod~~family runs through ~~September~~2032, ~~2037.~~absent any available patent term adjustments.

The status of our patent portfolios changes frequently in the ordinary course of our business. As of March 13, 2023, we owned or exclusively licensed approximately 10 U.S. patents and patent applications and about 111 patents and patent applications throughout the rest of the world that relate to our ACG-701 ad ACG-801 candidates.

As of March 15, 2022, we owned approximately 56 U.S. patents and patent applications and about 192 patents and patent applications throughout the rest of the world for our TLR-targeted immune modulation technologies. These patents and patent applications include claims covering the chemical compositions of matter and methods of use of our IMO compounds, such as IMO-8400, IMO-9200 and tilsotolimod (IMO-2125), as well as other compounds. These patents and patent applications (if granted) expire at various dates ranging from 2020 to 2042. With respect to IMO-8400, we have ten issued U.S. patents that cover the chemical composition of matter of IMO-8400 and certain methods of its use, the latest of which expires in 2031. With respect to IMO-9200, we have nine issued U.S. patents that cover the chemical composition for IMO-9200 and methods of its use, the latest of which expires in 2034. With respect to tilsotolimod, we have issued U.S. patents that cover the chemical composition of matter of tilsotolimod that will expire between 2023 and 2026, and we have additional U.S. patents 8

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~~that~~TLR-targeted ~~cover~~immune ~~methods~~modulation of its use, the latest of which will expire in 2037. We have pending applications in the United States and outside of the United States that cover methods of treatment or use of tilsotolimod, which, if granted, will expire between 2035 and 2042.technologies

Prior to the Aceragen Acquisition, we devoted a substantial amount of our resources into establishing intellectual property protection for:

●

novel chemical entities that function as agonists of TLR3, TLR7, TLR8 or TLR9;

●

novel chemical entities that function as antagonists of TLR7, TLR8 or TLR9; and

●

composition and use of our nucleic acid chemistry compounds to treat and prevent a variety of diseases.

As of March 15, 2023, we owned approximately 44 U.S. patents and patent applications and about 177 patents and patent applications throughout the rest of the world for our TLR-targeted immune modulation technologies. These patents and patent applications include claims covering the chemical compositions of matter and methods of use of our IMO compounds, such as IMO-8400, IMO-9200 and tilsotolimod (IMO-2125), as well as other compounds. These patents and patent applications (if granted) expire at various dates ranging from 2023 to 2041. With respect to IMO-8400, we have ten issued U.S. patents that cover the chemical composition of matter of IMO-8400 and certain methods of its use, the latest of which expires in 2031. With respect to IMO-9200, we also have ten issued U.S. patents that cover the chemical composition for IMO-9200 and methods of its use, the latest of which expires in 2034. With respect to tilsotolimod, we have issued U.S. patents that cover the chemical composition of matter of tilsotolimod that will expire between 2023 and 2026, and we have additional U.S. patents that cover methods of its use, the latest of which will expire in 2037 assuming payment of all maintenance fees.

Because patent applications in the United States and many foreign jurisdictions are typically not published until 18 months after filing, or in some cases not at all, and because publications of discoveries in the scientific literature often lag behind actual discoveries, neither we nor our licensors can be certain that we or they were the first to make the inventions claimed in issued patents or pending patent applications, or that we or they were the first to file for protection of the inventions set forth in these patent applications.

Litigation may be necessary to defend against or assert claims of infringement, to enforce patents issued to us, to protect trade secrets or know-how owned by us, or to determine the scope and validity of the proprietary rights of others or to determine the appropriate term for an issued patent. In addition, USPTO may declare interference proceedings to determine the priority of inventions with respect to our patent applications or reexamination or reissue proceedings to determine if the scope of a patent should be narrowed. Litigation or any of these other proceedings could result in substantial costs to and diversion of effort by us, even if the eventual outcome is favorable to us, and could have a material adverse effect on our business, financial condition and results of operations. These efforts by us may not be successful.

The term of individual patents depends upon the legal term for patents in the countries in which they are obtained. In most countries, including the United States, the patent term is 20 years from the earliest filing date of a non-provisional patent application. In the United States, a patent’s term may be lengthened by patent term adjustment, which compensates a patentee for administrative delays by the USPTO in examining and granting a patent. A patent’s term may also be shortened if a patent is terminally disclaimed over an earlier filed patent. The term of a patent that covers a drug, biological product, or medical device approved pursuant to a pre-market approval may also be eligible for patent term extension when U.S. Food and Drug Administration (“FDA”) approval is granted, provided statutory and regulatory requirements are met. The length of the patent term extension is related to the length of time the drug is under regulatory review and development. The Drug Price Competition and Patent Term Restoration Act of 1984 (the “Hatch-Waxman Act”) permits a patent term extension of up to five years beyond the expiration date set for the patent. Patent extension cannot extend the remaining term of a patent beyond a total of 14 years from the date of product approval, only one patent applicable to each regulatory review period may be granted an extension and only those claims reading on the approved drug are extended. Similar provisions are available in Europe and other foreign jurisdictions to extend the term of a patent that covers an approved drug.

We may rely, in some circumstances, on trade secrets and confidentiality agreements to protect our technology. Although trade secrets are difficult to protect, wherever possible, we use confidentiality agreements to protect the proprietary nature of our technology. We regularly implement confidentiality agreements with our employees, consultants, scientific advisors, and other contractors and collaborators. However, there can be no assurance that these agreements will not be breached, that we will have adequate remedies for any breach, or that our trade secrets and/or proprietary information will not otherwise become known or be independently discovered by competitors. To the extent that our employees, consultants or contractors use intellectual property owned by others in their work for us, disputes may also arise as to the rights in related or resulting know-how and inventions.

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competitors. To the extent that our employees, consultants or contractors use intellectual property owned by others in their work for us, disputes may also arise as to the rights in related or resulting know-how and inventions.

~~Manufacturing~~In addition to patents, we rely upon unpatented trade secrets, know-how and continuing technological innovation to develop and maintain a competitive position. We seek to protect our proprietary information, in part, through confidentiality agreements with our employees, collaborators, contractors and consultants, and invention assignment agreements with our employees and some of our collaborators. The confidentiality agreements are designed to protect our proprietary information and, in the case of agreements or clauses requiring invention assignment, to grant us ownership of technologies that are developed through a relationship with a third party.

We do not currently own or operate manufacturing facilities for the production of clinical or commercial quantities of any of our drug candidates. We currently rely and expect to continue to rely on other companies to manufacture our drug candidates for preclinical and clinical development. We source our bulk drug manufacturing requirements from a limited number of contract manufacturers through the issuance of work orders on an as-needed basis. We currently do not have any long-term supply contracts. We depend and will continue to depend on our contract manufacturers to manufacture our drug candidates in accordance with current Good Manufacturing Practices (“cGMP”) regulations for use in clinical trials. We will ultimately depend on contract manufacturers for the manufacture of our products for commercial sale, if and when our drug candidates are approved. Contract manufacturers are subject to extensive governmental regulation.

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Competition

The biotechnology and pharmaceutical industries are characterized by rapidly advancing technologies, intense competition and a strong emphasis on proprietary products. We are currently developing ACG-701, a patented formulation of sodium fusidate, to treat CF PEx and melioidosis, and ACG-801, recombinant human acid ceramidase (rhAC), to treat patients suffering from Farber disease. We recognize that other companies, including large pharmaceutical companies, may be developing or have plans to develop products and technologies that may compete with ~~those~~our drug candidates, ACG-701 and ACG-801, or any other drug candidates we may ~~acquire.~~develop ~~Many~~or ofacquire ~~our~~in ~~competitors~~the ~~have~~future. ~~substantially~~Our ~~greater financial~~products, ~~technical~~if approved, ~~and~~will ~~human~~compete ~~resources~~with ~~than~~novel wetherapies ~~have.~~developed Inby ~~addition~~biopharmaceutical companies, ~~many~~academic ofresearch ~~our~~institutions, ~~competitors~~governmental ~~have~~agencies, ~~significantly~~and ~~greater~~public ~~experience~~and ~~than~~private weresearch ~~have~~institutions, in ~~undertaking~~addition ~~preclinical~~to ~~studies~~standard ~~and~~of ~~human~~care ~~clinical~~treatments. ~~trials~~Our competitors compete with us on the level of ~~new~~the ~~pharmaceutical~~technologies ~~products~~employed, ~~obtaining~~or ~~FDA~~on ~~and~~the ~~other~~level ~~regulatory~~of ~~approvals~~development of ~~products~~product ~~for~~candidates. ~~use~~In inaddition, ~~health~~many ~~care~~small ~~and~~biotechnology ~~manufacturing,~~companies ~~and~~have ~~marketing~~formed ~~and~~collaborations ~~selling~~with ~~approved~~large, ~~products.~~established ~~Our~~companies ~~competitors~~to ~~may~~obtain ~~discover,~~support ~~develop~~for ortheir ~~commercialize~~research, ~~products~~development, orand ~~other~~commercialization ~~novel~~of ~~technologies~~products that ~~are~~may ~~more~~potentially ~~effective,~~directly ~~safer,~~compete with our current or ~~less~~future ~~costly~~product ~~than~~candidates. ~~any~~We anticipate that we ~~may~~will ~~develop.~~continue ~~Our~~to ~~competitors~~face ~~may~~increasing ~~also~~competition ~~obtain~~as ~~FDA~~new ortherapies ~~other~~and ~~regulatory~~combinations ~~approval~~thereof, ~~for~~technologies, ~~their~~and ~~products~~data ~~more~~emerge ~~rapidly~~for ~~than~~rare wediseases ~~may~~which ~~obtain~~we ~~approval~~are ~~for ours~~pursuing.

Many of our competitors, either alone or with their strategic partners, have substantially greater financial, technical, and human resources than we do and significantly greater experience in the discovery and development of product candidates, obtaining FDA and other regulatory approvals of treatments, and the commercialization of those treatments. Accordingly, our competitors may be more successful than us in obtaining approval for treatments and achieving widespread market acceptance.

WeACG-701 ~~anticipate~~is ~~that~~currently ~~the~~being ~~competition~~developed ~~with~~to ~~our~~treat ~~drug~~patient ~~candidates~~suffering orfrom ~~technologies~~CF ~~that~~PEx weand ~~may~~melioidosis. ~~acquire~~We ~~will~~are beaware ~~based~~of onseveral acompanies ~~number~~that may compete with ACG-701 for the treatment of ~~factors~~melioidosis, including, ~~product~~AN2 ~~efficacy~~Therapeutics, ~~safety~~Inc., ~~availability~~Bugworks Research, Inc., Entasis Therapeutics, Inc., Soligenix, Inc., and ~~price~~Venatorx Pharmaceuticals, Inc. ~~The~~While ~~timing~~there ofare ~~market~~currently ~~introduction~~no ofFDA-approved ~~products~~drugs ~~and~~specifically ~~competitive~~for ~~products~~the ~~will~~treatment ~~also~~of ~~affect~~melioidosis, ~~competition~~there ~~among~~are ~~products.~~several Weantimicrobial ~~expect~~drugs used off-label for the ~~relative~~treatment ~~speed~~of ~~with~~melioidosis, ~~which~~such weas ~~can~~beta-lactams ~~develop~~(e.g., ~~products~~ceftazidime, ~~complete~~certain ~~the~~beta-lactam-beta-lactamase ~~clinical~~inhibitor ~~trials~~combinations), ~~and~~carbapenems, ~~approval~~trimethoprim-sulfamethoxazole ~~processes~~(TMP-SMX), and ~~supply~~doxycycline, ~~commercial~~depending ~~quantities~~on the phase of ~~the~~treatment, with which our drug candidate may compete. While there are FDA-approved products tofor the ~~market~~treatment of CF and several companies developing therapies to beaddress ~~important~~the ~~competitive~~underlying ~~factors.~~cause ~~Our~~of ~~competitive~~CF, ~~position~~there ~~will~~are ~~also~~currently ~~depend~~no ~~upon~~FDA-approved ~~our~~drugs ~~ability~~specifically tofor ~~attract~~the ~~and~~treatment ~~retain~~of ~~qualified~~CF ~~personnel~~PEx and, to ~~obtain~~the ~~patent~~best ~~protection~~of orour ~~otherwise~~knowledge, ~~develop~~no ~~proprietary~~company’s ~~products~~programs orare ~~processes,~~currently ~~protect~~aimed ~~our~~directly ~~intellectual~~at ~~property~~addressing CF PEx. However, ~~and~~there tomay ~~secure~~also ~~sufficient~~be ~~capital~~unexpected ~~resources~~or ~~for~~unknown ~~the~~competitors ~~period~~that ~~between~~we ~~technological~~are ~~conception~~not ~~and~~presently ~~commercial~~aware ~~sales~~of.

~~Risks~~ACG-801 ~~related~~is being initially developed for the treatment of patients suffering from Farber disease, for which there is currently no approved therapies. While we are aware of several companies developing enzyme replacement therapies for other lysosomal storage diseases, to ~~our~~the ~~competitors~~best ~~and~~of our ~~competitive~~knowledge, ~~position~~no company’s programs are ~~discussed~~currently inaimed ~~further~~directly ~~detail~~at inaddressing ~~the~~Farber ~~section~~disease. ~~entitled~~However, ~~“Risk~~there ~~Factors”~~may be unexpected or unknown competitors of ~~this~~which ~~Annual~~we ~~Report~~are onnot ~~Form~~presently ~~10-K~~aware.

Our commercial potential for our current drug candidates, ACG-701 and ACG-801, or any other drug candidates or technologies we may acquire, could be reduced or eliminated if our competitors develop and commercialize products that are safer, more effective, have fewer or less severe side effects, are more convenient or are less expensive than products that we may develop. Our competitors also may obtain FDA, EMA, or other regulatory approval for their products more rapidly than we may obtain approval for our products, which could result in our competitors establishing a strong market position before we are able to enter the market or make our development

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~~Government~~more ~~Regulation~~complicated. The key competitive factors affecting the success of all of our programs are likely to be efficacy, safety, and convenience.

These competitors may also vie for a similar pool of qualified scientific and management talent, sites, and patient populations for clinical trials, as well as for technologies complementary to, or necessary for, our products. Further, our competitive position will also depend upon our ability to obtain patent protection or otherwise develop proprietary products or processes, protect our intellectual property, and to secure sufficient capital resources for the period between technological conception and commercial sales.

Government authorities in the United States, at the federal, state and local level, and in other countries and jurisdictions, including the European Union, extensively regulate, among other things, the research, development, testing, manufacture, quality control, approval, packaging, storage, recordkeeping, labeling, advertising, promotion, distribution, marketing and sales, pricing, post-approval monitoring and reporting, and import and export of pharmaceutical products. The processes for obtaining regulatory approvals in the United States and in foreign countries and jurisdictions, along with subsequent compliance with applicable statutes and regulations and other regulatory authorities, require the expenditure of substantial time and financial resources. Regulatory requirements are also continually evolving. By example, in light of the COVID-19 pandemic, the FDA has issued a number of guidance documents to assist companies navigating product development, and manufacturing concerns raised by COVID-19.Manufacturing

~~Review~~We do not currently own or operate, and ~~Approval~~currently have no plans to establish, any manufacturing facilities for the production of ~~Drugs~~preclinical, clinical, or commercial quantities of ACG-701 or ACG-801, or any other product candidate. We currently rely and expect to continue to rely on third-party contract development and manufacturing organizations (“CDMOs”) for all of our requirements of raw materials, drug substance, and drug product. We are not currently party to any long-term agreements with our current CDMOs. We intend to rely on CDMOs for clinical and commercial manufacturing of ACG-701 and ACG-801, as well as the clinical and commercial manufacturing of our other product candidates and any other product candidates that we may identify in the ~~United~~future. ~~States~~Although we rely on CDMOs, we have employees and third-party consultants with extensive manufacturing experience to oversee the relationships with our contract manufacturers.

InGovernment the United States, the FDA regulates drugs under the Federal Food, Drug, and Cosmetic Act (“FDCA”) and associated implementing regulations and guidance. The failure to comply with the FDCA and other applicable U.S. requirements at any time during the product development process, approval process or after approval may subject an applicant and/or sponsor to a variety of administrative or judicial sanctions, including refusal by the FDA to approve pending applications, withdrawal of an approval, imposition of a clinical hold, issuance of warning letters and other types of enforcement letters, product recalls, product seizures, operating restrictions such as the total or partial suspension of production or distribution, import bans, injunctions, fines, refusals of government contracts, restitution, disgorgement of profits, or civil or criminal investigations and penalties brought by the FDA and the Department of Justice (“DOJ”) or other governmental entities, including state agencies. Regulation

AnGovernment ~~applicant~~authorities ~~seeking~~in the United States, at the federal, state and local level, and in other countries and jurisdictions, including the European Union, extensively regulate, among other things, the research, development, testing, manufacture, quality control, approval, topackaging, ~~market~~storage, recordkeeping, labeling, advertising, promotion, distribution, marketing and ~~distribute~~sales, apricing, ~~new~~post-approval ~~drug~~monitoring ~~product~~and reporting, and import and export of pharmaceutical products. The processes for obtaining regulatory approvals in the United States ~~must~~and ~~typically~~in ~~undertake~~foreign countries and jurisdictions, along with subsequent compliance with applicable statutes and regulations and other regulatory authorities, require the ~~following:~~expenditure of substantial time and financial resources. Regulatory requirements are also continually evolving. By example, the FDA and the EU regulatory authorities have also issued a growing body of guidance documents that provide the agency’s interpretation of regulatory requirements.

●Review ~~completion~~and Approval of ~~preclinical~~Drugs ~~laboratory tests, animal studies~~ and ~~formulation~~Biologics ~~studies~~ in ~~compliance with~~ the ~~FDA’s~~United ~~good~~States ~~laboratory practice (“GLP”) regulations;~~

●In ~~submission~~the toUnited States, the FDA ofregulates andrugs ~~investigational~~under ~~new~~the ~~drug~~Federal Food, Drug, and Cosmetic Act (“~~IND~~FDCA”) ~~application~~and associated implementing regulations and guidance. In the United States, ~~which~~biologics ~~must~~are ~~take~~regulated ~~effect~~under ~~before~~both ~~human~~the ~~clinical~~FDCA ~~trials~~and the Public Health Services Act (“PHSA”). The failure to comply with the FDCA and other applicable U.S. requirements at any time during the product development process, approval process or after approval may ~~begin~~subject inan applicant and/or sponsor to a variety of administrative or judicial sanctions, including refusal by the ~~United~~FDA ~~States;~~to approve pending applications, withdrawal of an approval, imposition of a clinical hold, issuance of warning letters and other types of enforcement letters, product recalls, product seizures, operating restrictions such as the total or partial suspension of production or distribution, import bans, injunctions, fines, refusals of government contracts, restitution, disgorgement of profits, or civil or criminal investigations and penalties brought by the FDA and the Department of Justice (“DOJ”) or other governmental entities, including state agencies.

An applicant seeking approval to market and distribute a new drug or biologic product in the United States must typically undertake the following:

●

completion of preclinical laboratory tests, animal studies and formulation studies in compliance with the FDA’s good laboratory practice (“GLP”) regulations and other applicable laws or regulations;

●

submission to the FDA of an investigational new drug (“IND”) application, which must take effect before human clinical trials may begin in the United States;

●

initial and continuing approval by an independent institutional review board (“IRB”), representing each clinical site before each clinical trial may be initiated;

●Table ~~performance~~ of ~~adequate and well-controlled human clinical trials in accordance with good clinical practices (“GCP”) to establish the safety and efficacy of the proposed drug product for each indication;~~Contents

● performance of adequate and well-controlled human clinical trials in accordance with good clinical practices (“GCP”) to establish the safety and efficacy of the proposed product for each indication;

● preparation and submission to the FDA of a new drug application (“NDA”) for drug products or biologic license application (“BLA”) for biologic products;

●

review of the product candidate by an FDA advisory committee, where appropriate or if applicable;

● satisfactory completion of one or more FDA inspections or remote regulatory assessments of the manufacturing facility or facilities at which the product, or components thereof, are produced to assess compliance with cGMP requirements and to assure that the facilities, methods and controls are adequate to preserve the product’s identity, strength, quality and purity;

● satisfactory completion of one or more FDA inspections of the manufacturing facility or facilities at which the product, or components thereof, are produced to assess compliance with cGMP requirements and to assure that the facilities, methods and controls are adequate to preserve the product’s identity, strength, quality and purity;

●

satisfactory completion of FDA audits of clinical trial sites to assure compliance with GCPs and the integrity of the clinical data;

● payment of user fees and securing FDA approval of the marketing application; and

● payment of user fees and securing FDA approval of the NDA; and

●

compliance with any post-approval requirements, including Risk Evaluation and Mitigation Strategies (“REMS”) where applicable, and post-approval studies required by the FDA.

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Preclinical Studies and Submission of an IND

Before an applicant begins testing a product candidate ~~with potential therapeutic value~~ in humans, the product candidate enters the preclinical testing stage. Preclinical studies include laboratory evaluation of the chemistry, pharmacology, toxicity, purity, and stability, among other attributes, of the manufactured drug or biologic substance or active pharmaceutical ingredient and the formulated ~~drug or drug~~ product, as well as in vitro and animal studies to assess the safety and activity of the drug or biologic for initial testing in humans and to establish a rationale for therapeutic use. The conduct of preclinical studies is subject to federal regulations and requirements, including GLP regulations. The results of the preclinical tests, together with manufacturing information, analytical data, any available clinical data or literature and plans for clinical studies, among other things, are submitted to the FDA as part of an IND. Additional preclinical testing, such as animal tests of reproductive adverse events and carcinogenicity, may continue after the IND is submitted.

Human Clinical Studies in Support of ana ~~NDA~~Marketing Application

Clinical trials involve the administration of the investigational product to human subjects under the supervision of qualified investigators in accordance with GCP requirements, which include, among other things, the requirement that all research subjects provide their informed consent in writing before their participation in any clinical trial. Special clinical trial ethical considerations also must be taken into account if a study involves children. Clinical trials are conducted under written study protocols detailing, among other things, the objectives of the study, inclusion and exclusion criteria, the parameters to be used in monitoring safety and the effectiveness criteria to be evaluated, and a statistical analysis plan. A protocol for each clinical trial and any subsequent protocol amendments must be submitted to the FDA as part of the IND. Once an IND is in effect, unapproved product candidates may be shipped in interstate commerce for use in an investigational clinical trial and the investigational product may be administered to humans as part of a clinical trial. An IND automatically becomes effective 30 days after receipt by the FDA, unless before that time the FDA raises concerns or questions related to a proposed clinical trial and places the trial on clinical hold or partial clinical hold. In such a case, the IND sponsor and the FDA must resolve any outstanding concerns before the clinical trial can begin. Depending on the conditions under development, multiple INDs may be required for the same drug product.

InA ~~addition~~protocol tofor ~~the~~each ~~foregoing~~clinical ~~IND~~trial ~~requirements,~~and anany ~~IRB~~subsequent ~~representing~~protocol ~~each~~amendments ~~institution~~must ~~participating~~be insubmitted to the ~~clinical~~FDA ~~trial~~as ~~must~~part ~~review~~of ~~and approve~~ the ~~plan~~IND. ~~for~~Under ~~any~~newly ~~clinical~~enacted ~~trial~~legislation, ~~before~~diversity itplans ~~commences~~will atalso ~~that~~be ~~institution,~~required. ~~and~~Once ~~the~~an ~~IRB~~IND ~~must~~is ~~conduct~~in ~~continuing~~effect, ~~review~~unapproved ~~and~~product ~~reapprove~~candidates ~~the~~may ~~study~~be atshipped ~~least~~in ~~annually.~~interstate ~~The~~commerce ~~IRB~~for ~~must~~use ~~review~~in ~~and~~an ~~approve,~~investigational ~~among~~clinical ~~other~~trial ~~things,~~and the ~~study~~investigational ~~protocol,~~product ~~protocol~~may ~~amendments,~~be ~~communications~~administered to ~~study~~humans ~~subjects~~as ~~and~~part ~~informed~~of ~~consent~~a ~~information~~clinical totrial. beAn ~~provided~~IND toautomatically ~~study~~becomes ~~subjects.~~effective An30 ~~IRB~~days ~~must~~after ~~operate~~receipt inby ~~compliance~~the ~~with~~ FDA, ~~regulations.~~unless ~~Information~~before ~~about~~that ~~certain~~time ~~clinical~~the ~~trials~~FDA ~~must~~raises beconcerns ~~submitted~~or ~~within~~questions ~~specific~~related ~~timeframes~~ to ~~the~~a ~~National~~proposed ~~Institutes~~clinical oftrial ~~Health~~and ~~(“NIH”)~~places ~~for~~the ~~public~~trial ~~dissemination~~ on ~~their~~clinical ~~ClinicalTrials.gov~~hold ~~website. Sponsors~~ or ~~distributors~~partial ofclinical ~~investigational~~hold. ~~products~~In ~~for~~such ~~the~~a ~~diagnosis~~case, ~~monitoring,~~the orIND ~~treatment~~sponsor ofand ~~one~~the orFDA ~~more~~must ~~serious~~resolve ~~disease~~any oroutstanding ~~conditions~~concerns ~~must~~before ~~also~~the ~~have~~clinical atrial ~~publicly~~can ~~available~~begin. ~~policy~~Clinical onholds ~~evaluating~~also ~~and~~may ~~responding~~be toimposed ~~requests~~by ~~for~~the ~~expanded~~FDA ~~access.~~at ~~Investigators~~any ~~must~~time ~~also~~before ~~provide~~or ~~certain~~during ~~information~~trials due to ~~clinical~~safety ~~trial~~concerns ~~sponsors~~or tonon-compliance. ~~allow~~Depending on the ~~sponsors~~conditions tounder ~~make~~development, ~~certain~~multiple ~~financial~~INDs ~~disclosures~~may tobe required for the ~~FDA~~same product.

In addition to the foregoing IND requirements, an IRB representing each institution participating in the clinical trial must review and approve the plan for any clinical trial before it commences at that institution, and the IRB must conduct continuing review and reapprove the study at least annually. The IRB must review and approve, among other things, the study protocol, protocol amendments, communications to study subjects and informed consent information to be provided to study subjects. An IRB must operate in compliance with FDA regulations. Information

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about certain clinical trials must be submitted within specific timeframes to the National Institutes of Health (“NIH”) for public dissemination on their ClinicalTrials.gov website. Sponsors or distributors of investigational products for the diagnosis, monitoring, or treatment of one or more serious disease or conditions must also have a publicly available policy on evaluating and responding to requests for expanded access. Investigators must also provide certain information to clinical trial sponsors to allow the sponsors to make certain financial disclosures to the FDA.

Additionally, some trials are overseen by an independent group of qualified experts organized by the trial sponsor, known as a data monitoring committee (“DMC”). This group provides recommendations as to whether a trial should move forward at designated check points based on access that only the group maintains to available data from the study. Suspension or termination of development during any phase of clinical trials can occur if it is determined that the participants or patients are being exposed to an unacceptable health risk. Other reasons for suspension or termination may be made by us based on evolving business objectives and/or competitive climate.

Human clinical trials are typically conducted in three sequential phases, which may overlap or be combined:

Phase 1: The ~~drug~~product is initially introduced into healthy human subjects or patients with the target disease (e.g. cancer) or condition and tested for safety, dosage tolerance, structure-activity relationships, mechanism of action, absorption, metabolism, distribution, excretion, and pharmacokinetics and, if possible, to gain an early indication of its effectiveness and to determine optimal dosage.

Phase 2: The ~~drug~~product is administered to a limited patient population to identify possible adverse effects and safety risks, to preliminarily evaluate the efficacy of the product for specific targeted diseases and to determine dosage tolerance and optimal dosage.

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Phase 3: The ~~drug~~product is administered to an expanded patient population, generally at geographically dispersed clinical trial sites, in well-controlled clinical trials to generate enough data to statistically evaluate the efficacy and safety of the product for approval, to establish the overall risk-benefit profile of the product, and to provide adequate information for the labeling of the product.

~~The~~In ~~manufacture~~some ofcases, ~~investigational~~the ~~drugs~~FDA ~~for~~may ~~the~~condition ~~conduct~~approval of ~~human~~an ~~clinical~~NDA ~~trials~~or isBLA ~~subject~~on tothe ~~cGMP~~applicant’s ~~requirements.~~agreement ~~Investigational~~to ~~drugs~~conduct ~~and~~additional ~~active~~clinical ~~ingredients~~trials ~~imported~~to ~~into~~further assess the ~~United~~product’s ~~States~~safety ~~are~~and ~~also~~effectiveness ~~subject~~after toNDA ~~regulation~~or byBLA ~~the FDA~~approval. ~~Further,~~Such ~~the~~post-approval ~~export~~trials ofare ~~investigational~~typically ~~products~~referred ~~outside~~to ~~the~~as ~~United~~Phase 4 ~~States~~studies. isThe ~~subject~~results toof ~~regulatory~~Phase ~~requirements~~4 ofstudies can confirm or refute the ~~receiving~~effectiveness ~~country~~of asa ~~well~~product ascandidate, ~~U.S.~~and ~~export~~can ~~requirements~~provide ~~under~~important ~~the~~safety ~~FDCA~~information.

~~Progress~~The ~~reports~~manufacture ~~detailing the results~~ of ~~the~~investigational ~~clinical~~products ~~trials must be submitted at least annually to the FDA and to the IRB and more frequently if serious adverse events occur. In addition, IND safety reports must be submitted to the FDA~~ for ~~any of~~ the ~~following:~~conduct serious and unexpected suspected adverse reactions; findings from other studies or animal or in vitro testing that suggest a significant risk in humans exposed to the drug; and any clinically important increase in the case of ahuman ~~serious suspected adverse reaction over that listed in the protocol or investigator brochure. Phase 1, Phase 2 and Phase 3~~ clinical trials ~~may~~is ~~not~~subject beto ~~completed~~cGMP ~~successfully within any specified period, or at all~~requirements. ~~Furthermore,~~Investigational ~~the~~products ~~FDA,~~and ~~the~~active ~~sponsor~~ingredients orimported ~~the~~into ~~data monitoring committee for a clinical trial may suspend or terminate~~ the ~~clinical~~United ~~trial~~States ~~at any time on various grounds, including a finding that the research subjects~~ are ~~being~~also ~~exposed~~subject to anregulation ~~unacceptable~~by ~~health~~the ~~risk~~FDA. ~~Similarly~~Further, anthe ~~IRB~~export ~~can suspend or terminate approval~~ of ainvestigational ~~clinical~~products ~~trial~~outside ~~at its institution, or an institution it represents, if~~ the ~~clinical~~United ~~trial~~States is ~~not~~subject ~~being~~to ~~conducted~~regulatory ~~in accordance with the IRB’s~~ requirements orof if the ~~drug~~receiving ~~has~~country ~~been~~as ~~associated~~well ~~with~~as ~~unexpected serious harm to patients~~U.S. ~~The~~export ~~FDA~~requirements ~~will~~under ~~typically inspect one or more clinical sites to assure compliance with GCP and~~ the ~~integrity of the clinical data submitted~~FDCA.

Progress reports detailing the results of the clinical trials must be submitted at least annually to the FDA and to the IRB and more frequently if serious adverse events occur. In addition, IND safety reports must be submitted to the FDA for any of the following: serious and unexpected suspected adverse reactions; findings from other studies or animal or in vitro testing that suggest a significant risk in humans exposed to the product; and any clinically important increase in the case of a serious suspected adverse reaction over that listed in the protocol or investigator brochure. Phase 1, Phase 2 and Phase 3 clinical trials may not be completed successfully within any specified period, or at all. Furthermore, the FDA, the sponsor or the data monitoring committee for a clinical trial may suspend or terminate the clinical trial at any time on various grounds, including a finding that the research subjects are being exposed to an unacceptable health risk. Similarly, an IRB can suspend or terminate approval of a clinical trial at its institution, or an institution it represents, if the clinical trial is not being conducted in accordance with the IRB’s requirements or if the product has been associated with unexpected serious harm to patients. The FDA will typically inspect or conduct a remote regulatory assessment of one or more clinical sites to assure compliance with GCP and the integrity of the clinical data submitted.

Section 505(b)(2) NDAs

NDAs for most new drug products are based on clinical studies conducted by or for the product sponsor. These applications are submitted under Section 505(b)(1) of the FDCA. The FDA is, however, authorized to approve an alternative type of NDA under Section 505(b)(2) of the FDCA. This type of application allows the applicant to rely, in part, on the FDA’s previous findings of safety and efficacy for a similar product, or published literature. Specifically, Section 505(b)(2) applies to NDAs for a drug for which the investigations made to show whether or not the drug is safe for use and effective in use and relied upon by the applicant for approval of the application “were not conducted by or for the applicant and for which the applicant has not obtained a right of reference or use from the person by or for whom the investigations were conducted.”

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alternative type of NDA under Section 505(b)(2) of the FDCA. This type of application allows the applicant to rely, in part, on the FDA’s previous findings of safety and efficacy for a similar product or published literature. Specifically, Section 505(b)(2) applies to NDAs for a drug for which the investigations made to show whether or not the drug is safe for use and effective in use and relied upon by the applicant for approval of the application “were not conducted by or for the applicant and for which the applicant has not obtained a right of reference or use from the person by or for whom the investigations were conducted.”

Thus, Section 505(b)(2) authorizes the FDA to approve an NDA based on safety and effectiveness data that was not developed by the applicant. NDAs filed under Section 505(b)(2) may provide an alternate and potentially more expeditious pathway to FDA approval for new or improved formulations or new uses of previously approved products. If the Section 505(b)(2) applicant can establish that reliance on the FDA’s previous approval or a similar product or that the applicant’s reliance on published data is scientifically appropriate, such as through bridging studies, the applicant may eliminate the need to conduct certain preclinical or clinical studies of the new product. Companies using this pathway, however, must conduct studies to support any differences from an approved product. The FDA may then approve the new drug candidate for all or some of the label indications for which the referenced product has been approved, as well as for any new indication sought by the Section 505(b)(2) applicant.

Submission of ana ~~NDA~~Marketing Application to the FDA

Assuming successful completion of required clinical testing and other requirements, the results of the preclinical and clinical studies, together with detailed information relating to the product’s chemistry, manufacture, controls and proposed labeling, among other things, are submitted to the FDA as part of an NDA, for drug products, or BLA for biologic products, requesting approval to market the ~~drug~~ product for one or more indications. Under federal law, the submission of most NDAs and BLAs is subject to a substantial application user fee. The sponsor of an approved NDA or BLA is also subject to an annual program fee. ~~Exceptions~~Depending oron ~~waivers~~the ~~for~~product ~~these~~and ~~fees~~fee, ~~exist for~~ a ~~small~~ company ~~(fewer~~may ~~than~~qualify ~~500 employees, including employees and affiliates) satisfying certain requirements and products with orphan drug designation~~ for ~~a particular indication are not subject to a~~ fee ~~provided~~exceptions ~~there~~or ~~are no other intended uses in the NDA and provided other exemption requirements are met~~waivers.

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The FDA conducts a preliminary review of an NDA or BLA within 60 calendar days of its receipt and strives to inform the sponsor by the 74th day after the FDA’s receipt of the submission of any filing review issues. The FDA may request additional information rather than accept an ~~NDA~~application for filing. In this event, the application must be resubmitted with the additional information. The resubmitted application is also subject to review before the FDA accepts it for filing. Once the submission is accepted for filing, the FDA begins an in-depth substantive review.

The FDA has agreed to specified performance goals under the Prescription Drug User Fee Act (“PDUFA”) guidelines in the review process of NDAs and BLAs. Under PDUFA, 90% of applications seeking approval of New Molecular Entities (“NMEs”), are meant to be reviewed within ten months from the date on which the FDA accepts the NDA or BLA for filing, and 90% of applications for NMEs that have been designated for “priority review” are meant to be reviewed within six months of the filing date. For applications seeking approval of ~~drugs~~products that are not NMEs, the ten-month and six-month review periods run from the date that the FDA receives the application. The review process may be extended by the FDA for three additional months to consider new information or clarification provided by the applicant to address an outstanding deficiency identified by the FDA following the original submission. ~~This review period may change as the PDUFA statute must be reauthorized by the U.S. Congress by September 2022.~~

Before approving ana ~~NDA~~marketing application, the FDA typically will inspect or conduct a remote regulatory assessment of the facility or facilities where the product is or will be manufactured. These pre-approval inspections cover all facilities associated with ana ~~NDA~~marketing application submission, including ~~drug~~ component manufacturing (such as active pharmaceutical ingredients), finished drug product manufacturing, and control testing laboratories. The FDA will not approve an application unless it determines that the manufacturing processes and facilities are in compliance with cGMP requirements and adequate to assure consistent production of the product within required specifications. Additionally, before approving an NDA or BLA,, the FDA will typically inspect or conduct a remote regulatory assessment of one or more clinical sites to assure compliance with GCP.

In addition, as a condition of approval, the FDA may require an applicant to develop a REMS. REMS use risk minimization strategies beyond the professional labeling to ensure that the benefits of the product outweigh the potential risks. To determine whether a REMS is needed, the FDA will consider the size of the population likely to use the product, seriousness of the disease, expected benefit of the product, expected duration of treatment, seriousness of known or potential adverse events, and whether the product is a new molecular entity. REMS can include medication guides, physician communication plans for healthcare professionals, and elements to assure safe use (“ETASU”). ETASU may include, but are not limited to, special training or certification for prescribing or dispensing, dispensing only under certain circumstances, special monitoring, and the use of patient registries. The FDA may require a REMS before approval or post-approval if it becomes aware of a serious risk associated with use of the product. The requirement for a REMS can materially affect the potential market and profitability of a product. The REMS strategy must be approved by the FDA. In addition, the REMS must include a timetable to assess the strategy at 18 months, three years, and seven years after the strategy’s approval.

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include medication guides, physician communication plans for healthcare professionals, and elements to assure safe use (“ETASU”). ETASU may include, but are not limited to, special training or certification for prescribing or dispensing, dispensing only under certain circumstances, special monitoring, and the use of patient registries. The FDA may require a REMS before approval or post-approval if it becomes aware of a serious risk associated with use of the product. The requirement for a REMS can materially affect the potential market and profitability of a product. The REMS strategy must be approved by the FDA. In addition, the REMS must include a timetable to assess the strategy at 18 months, three years, and seven years after the strategy’s approval.

The FDA is required to refer an application for a novel drug to an advisory committee or explain why such referral was not made. Typically, an advisory committee is a panel of independent experts, including clinicians and other scientific experts, that reviews, evaluates and provides a recommendation as to whether the application should be approved and under what conditions. The FDA is not bound by the recommendations of an advisory committee, but it considers such recommendations carefully when making decisions.

Fast Track, Breakthrough Therapy and Priority Review Designations

The FDA is authorized to designate certain products for expedited review or increased regulatory guidance if they are intended to address an unmet medical need in the treatment of a serious or life-threatening disease or condition. These programs are fast track designation, breakthrough therapy designation and priority review designation.

Specifically, the FDA may designate a product for fast track review if it is intended, whether alone or in combination with one or more other ~~drugs~~products, for the treatment of a serious or life-threatening disease or condition, and it demonstrates the potential to address unmet medical needs for such a disease or condition. For fast track products, sponsors may have greater interactions with the FDA and the FDA may initiate review of sections of a fast track product’s ~~NDA~~marketing application before the application is complete. This rolling review may be available if the FDA determines, after preliminary evaluation of clinical data submitted by the sponsor, that a fast track product may be effective. The sponsor must also provide, and the FDA must approve, a schedule for the submission of the remaining information and the sponsor must pay applicable user fees. However, the FDA’s time period goal for reviewing a fast track application does not begin until the last section of the marketing application is submitted. In addition, the fast track designation may be withdrawn by the FDA if the FDA believes that the designation is no longer supported by data emerging in the clinical trial process.

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fast track application does not begin until the last section of the NDA is submitted. In addition, the fast track designation may be withdrawn by the FDA if the FDA believes that the designation is no longer supported by data emerging in the clinical trial process.

In 2012, Congress enacted the Food and Drug Administration Safety and Innovation Act (“FDASIA”). This law established a new regulatory scheme allowing for expedited review of products designated as “breakthrough therapies.” A product may be designated as a breakthrough therapy if it is intended, either alone or in combination with one or more other drugs or biologics, to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the product may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. The FDA may take certain actions with respect to breakthrough therapies, including holding meetings with the sponsor throughout the development process; providing timely advice to the product sponsor regarding development and approval; involving more senior staff in the review process; assigning a cross-disciplinary project lead for the review team; and taking other steps to design the clinical trials in an efficient manner. Like fast track designation, breakthrough designation may be rescinded if the product no longer meets the qualifying criteria.

Third, the FDA may designate a product for priority review if it is a ~~drug~~product that treats a serious condition and, if approved, would provide a significant improvement in safety or effectiveness. The FDA determines, on a case-by-case basis, whether the proposed ~~drug~~product represents a significant improvement when compared with other available therapies. Significant improvement may be illustrated by evidence of increased effectiveness in the treatment of a condition, elimination or substantial reduction of a treatment-limiting drug reaction, documented enhancement of patient compliance that may lead to improvement in serious outcomes, and evidence of safety and effectiveness in a new subpopulation. A priority designation is intended to direct overall attention and resources to the evaluation of such applications, and to shorten the FDA’s goal for taking action on a marketing application from ten months to six months.

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Accelerated Approval Pathway

The FDA may grant accelerated approval to a drug or biologic for a serious or life-threatening condition that provides meaningful therapeutic advantage to patients over existing treatments based upon a determination that the ~~drug~~product has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit. The FDA may also grant accelerated approval for such a condition when the product has an effect on an intermediate clinical endpoint that can be measured earlier than an effect on irreversible morbidity or mortality (“IMM”), and that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit, taking into account the severity, rarity, or prevalence of the condition and the availability or lack of alternative treatments. ~~Drugs~~Products granted accelerated approval must meet the same statutory standards for safety and effectiveness as those granted traditional approval.

For the purposes of accelerated approval, a surrogate endpoint is a marker, such as a laboratory measurement, radiographic image, physical sign, or other measure that is thought to predict clinical benefit, but is not itself a measure of clinical benefit. Surrogate endpoints can often be measured more easily or more rapidly than clinical endpoints. An intermediate clinical endpoint is a measurement of a therapeutic effect that is considered reasonably likely to predict the clinical benefit of a ~~drug~~product, such as an effect on IMM. The FDA has limited experience with accelerated approvals based on intermediate clinical endpoints, but has indicated that such endpoints generally may support accelerated approval where the therapeutic effect measured by the endpoint is not itself a clinical benefit and basis for traditional approval, if there is a basis for concluding that the therapeutic effect is reasonably likely to predict the ultimate clinical benefit of a ~~drug~~product.

The accelerated approval pathway is most often used in settings in which the course of a disease is long, and an extended period of time is required to measure the intended clinical benefit of a ~~drug~~product, even if the effect on the surrogate or intermediate clinical endpoint occurs rapidly. Thus, accelerated approval has been used extensively in the development and approval of ~~drugs~~products for treatment of a variety of cancers in which the goal of therapy is generally to improve survival or decrease morbidity and the duration of the typical disease course requires lengthy and sometimes large trials to demonstrate a clinical or survival benefit.

The accelerated approval pathway is ~~usually~~ contingent on a sponsor’s agreement to conduct, in a diligent manner, additional post-approval confirmatory studies to verify and describe the ~~drug~~product’s clinical benefit. As a result, a ~~drug~~product candidate approved on this basis is subject to rigorous post-marketing compliance requirements, including the completion of Phase 4 or post-approval clinical trials to confirm the effect on the clinical endpoint. By the date of approval of an accelerated approval product, FDA must specify the conditions for the required post approval studies, including enrollment targets, the study protocol, milestones, and target completion dates. The FDA may also require that the confirmatory Phase 4 studies be commenced prior to the FDA granting a product accelerate approval. Reports on the progress of the required Phase 4 confirmatory studies must be submitted to the FDA every 180 days after approval. Failure to conduct required post-approval studies, or confirm a clinical benefit during post-marketing studies, would allow the FDA to withdraw the product from the market on an expedited basis through a statutorily defined streamlined process. All promotional materials for product candidates approved under accelerated regulations are subject to prior review by the FDA.

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the completion of Phase 4 or post-approval clinical trials to confirm the effect on the clinical endpoint. Failure to conduct required post-approval studies, or confirm a clinical benefit during post-marketing studies, would allow the FDA to withdraw the drug from the market on an expedited basis. All promotional materials for drug candidates approved under accelerated regulations are subject to prior review by the FDA.

The FDA’s Decision on anMarketing ~~NDA~~Application

On the basis of the FDA’s evaluation of the NDA or BLA and accompanying information, including the results of the inspection of the manufacturing facilities, the FDA may issue an approval letter or a complete response letter. An approval letter authorizes commercial marketing of the product with specific prescribing information for specific indications. A complete response letter generally outlines the deficiencies in the submission and may require substantial additional testing or information in order for the FDA to reconsider the application. If and when those deficiencies have been addressed to the FDA’s satisfaction in a resubmission of the ~~NDA~~marketing application, the FDA will issue an approval letter. The FDA has committed to reviewing such resubmissions in two or six months depending on the type of information included. Even with submission of this additional information, the FDA ultimately may decide that the application does not satisfy the regulatory criteria for approval.

If the FDA approves a product, it may limit the approved indications for use for the product, require that contraindications, warnings or precautions be included in the product labeling, require that post-approval studies, including Phase 4 clinical trials, be conducted to further assess the ~~drug~~product’s safety after approval, require testing and surveillance programs to monitor the product after commercialization, or impose other conditions, including distribution restrictions or other risk management mechanisms, including REMS, which can materially affect the potential market and profitability of the product. The FDA may prevent or limit further marketing of a product based on the results of post-market studies or surveillance programs. After approval, many types of changes to the approved product, such as adding new indications, manufacturing changes and additional labeling claims, are subject to further testing requirements and FDA review and approval.

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distribution restrictions or other risk management mechanisms, including REMS, which can materially affect the potential market and profitability of the product. The FDA may prevent or limit further marketing of a product based on the results of post-market studies or surveillance programs. After approval, many types of changes to the approved product, such as adding new indications, manufacturing changes and additional labeling claims, are subject to further testing requirements and FDA review and approval.

Post-Approval Requirements

~~Drugs~~Products manufactured or distributed pursuant to FDA approvals are subject to pervasive and continuing regulation by the FDA, including, among other things, requirements relating to recordkeeping, periodic reporting, product sampling and distribution, advertising and promotion and reporting of adverse experiences and production issues with the product. After approval, most changes to the approved product, such as adding new indications or other labeling claims, are subject to prior FDA review and approval. There also are continuing, annual user fee requirements for any marketed products and the establishments at which such products are manufactured, as well as new application fees for supplemental applications with clinical data. Many states also regulate the distribution of drug product samples and commercial product.

In addition, ~~drug~~product manufacturers and other entities involved in the manufacture and distribution of approved drugs and biologics are required to register their establishments with the FDA and state agencies, are required to list their distributed products, and are subject to periodic unannounced inspections by the FDA and these state agencies for compliance with cGMP requirements. The information that must be submitted to FDA regarding manufactured products was expanded through the Coronavirus Aid, Relief, and Economic Security Act (“CARES Act”) to include the volume of ~~drugs~~products produced during the prior year. Changes to the manufacturing process are strictly regulated and often require prior FDA approval before being implemented. FDA regulations also require investigation and correction of any deviations from cGMP and impose reporting and documentation requirements upon the sponsor and any third-party manufacturers that the sponsor may decide to use. Accordingly, manufacturers must continue to expend time, money, and effort in the area of production and quality control to maintain cGMP compliance.

Once an approval is granted, the FDA may withdraw the approval if compliance with regulatory requirements and standards is not maintained or if problems occur after the product reaches the market. Later discovery of previously unknown problems with a product, including adverse events of unanticipated severity or frequency, or with manufacturing processes, or failure to comply with regulatory requirements, may result in revisions to the approved labeling to add new safety information; imposition of post-market studies or clinical trials to assess new safety risks; or imposition of distribution or other restrictions under a REMS program. Other potential consequences include, among other things:

●

restrictions on the marketing or manufacturing of the product, suspension of the approval, complete withdrawal of the product from the market or product recalls;

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●

warning letters, untitled letters, cyber letters, or holds or termination of post-approval clinical trials;

●

refusal of the FDA to approve pending marketing applications or supplements to approved marketing applications, or suspension or revocation of product license approvals;

●

product seizure or detention, product recalls, or refusal to permit the import or export of products; or

●

FDA debarment, injunctions, fines, consent decrees, corporate integrity agreements, suspension and debarment from government contracts, refusal of orders under existing government contracts, exclusion from participation in federal and state healthcare programs, restitution, disgorgement, or the imposition of civil or criminal penalties, including fines and imprisonment, and adverse publicity.

●Sponsors ~~refusal~~are further subject to various requirements related to FDA product shortage and manufacturing volume reporting, supply chain security, such as risk management plan requirements, and the promotion of supply chain redundancy. FDA post-approval requirements are further continually evolving. For example, in March 2020, the U.S. Congress passed the CARES Act, which includes various provisions regarding FDA toproduct ~~approve~~shortage ~~pending~~and ~~NDAs~~manufacturing orvolume ~~supplements~~reporting torequirements, ~~approved~~as ~~NDAs~~well as provisions regarding supply chain security, orsuch ~~suspension~~as orrisk ~~revocation~~management plan requirements, and the promotion of ~~product~~supply ~~license~~chain ~~approvals;~~redundancy and domestic manufacturing. The executive branch has also taken steps to promote domestic manufacturing, and the Consolidated Appropriations

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Act of 2023, and the reauthorization of the Prescription Drug User Fee Act, which were passed in 2022, included several changes to the FDC Act.

●The ~~product~~FDA ~~seizure~~strictly orregulates ~~detention~~the marketing, orlabeling, ~~refusal~~advertising and promotion of prescription products placed on the market. This regulation includes, among other things, standards and regulations for direct-to-consumer ~~permit~~advertising, communications regarding unapproved uses, industry-sponsored scientific and educational activities, and promotional activities involving the ~~import~~Internet and social media. Promotional claims about a drug’s safety or ~~export~~effectiveness are prohibited before the product is approved. After approval, a product may not be promoted for uses that are not approved by the FDA, as reflected in the product’s prescribing information. All statements must be consistent with the FDA-approved label, truthful, and non-misleading. Additionally, claims about product benefits and risks must be substantiated adequately while maintaining a fair balance between the two. For instance, it is impermissible to make claims about the superiority of our products; over other treatments without sufficient evidence, which often requires head-to-head clinical studies for substantiation. In the United States, health care professionals are generally permitted to prescribe products for such uses not described in the product’s labeling, known as off-label uses, because the FDA does not regulate the practice of medicine. However, FDA regulations impose rigorous restrictions on manufacturers’ communications, prohibiting the promotion of off-label uses. It may be permissible, under very specific, narrow conditions, for a manufacturer to engage in nonpromotional, non-misleading communication regarding off-label information, such as distributing scientific or medical journal information.

~~FDA~~If ~~debarment,~~a ~~injunctions,~~company ~~fines,~~is ~~consent~~found ~~decrees,~~to ~~corporate~~have ~~integrity~~promoted ~~agreements~~off-label uses, ~~suspension~~it may become subject to adverse public relations and ~~debarment~~administrative ~~from~~and ~~government~~judicial ~~contracts~~enforcement by the FDA, ~~refusal~~the ofDOJ, ~~orders~~or ~~under~~the ~~existing~~Office ~~government~~of ~~contracts,~~the ~~exclusion~~Inspector ~~from~~General ~~participation~~of inthe ~~federal~~Department of Health and Human Services, as well as state ~~healthcare~~authorities. ~~programs,~~This ~~restitution,~~could ~~disgorgement,~~subject ora ~~the~~company ~~imposition~~to a range of ~~civil~~penalties orthat ~~criminal~~could ~~penalties~~have a significant commercial impact, including civil and criminal fines and ~~imprisonment,~~agreements that materially restrict the manner in which a company promotes or distributes products. The federal government has levied large civil penalties and ~~adverse~~criminal ~~publicity~~fines against companies for alleged improper promotion and has also requested that companies enter into consent decrees or permanent injunctions under which specified promotional conduct is changed or curtailed.

~~FDA~~In ~~post-approval~~addition, ~~requirements~~the ~~are~~distribution ~~continually~~of ~~evolving.~~prescription ~~For~~pharmaceutical ~~example,~~products inis ~~March~~subject ~~2020,~~to the ~~U.S.~~Prescription ~~Congress~~Drug ~~passed~~Marketing Act (“PDMA”), and its implementing regulations, as well as the ~~CARES~~Drug Supply Chain Security Act, ~~which~~(“DSCSA”). The ~~includes~~PDMA ~~various~~regulate ~~provisions~~and ~~regarding~~limit ~~FDA~~the distribution and tracing of prescription drug ~~shortage~~samples ~~and~~at ~~manufacturing~~the ~~volume~~federal ~~reporting~~level. ~~requirements,~~The asDSCSA ~~well~~imposes asrequirements ~~provisions~~to ~~regarding~~ensure ~~supply~~accountability ~~chain~~in ~~security~~distribution, ~~such~~which asrequires ~~risk~~certain ~~management~~licensing ~~plan~~and ~~requirements~~licensing standards, and ~~the~~to ~~promotion~~identify ofand ~~supply~~remove ~~chain~~counterfeit ~~redundancy~~ and ~~domestic~~other ~~manufacturing.~~illegitimate products from the market.

~~The~~Additional ~~FDA~~controls ~~strictly regulates the marketing, labeling, advertising and promotion of prescription drug products placed on the market. This regulation includes, among other things, standards and regulations~~ for direct-to-consumer advertising, communications regarding unapproved uses, industry-sponsored scientific and educational activities, and promotional activities involving the Internet and social media. Promotional claims about a drug’s safety or effectiveness are prohibited before the drug is approved. After approval, a drug product may not be promoted for uses that are not approved by the FDA, as reflected in the product’s prescribing information. In the United States, health care professionals are generally permitted to prescribe drugs for such uses not described in the drug’s labeling, known as off-label uses, because the FDA does not regulate the practice of medicine. However, FDA regulations impose rigorous restrictions on manufacturers’ communications, prohibiting the promotion of off-label uses. It may be permissible, under very specific, narrow conditions, for a manufacturer to engage in nonpromotional, non-misleading communication regarding off-label information, such as distributing scientific or medical journal information.biologics

IfIn aaddition ~~company~~to isthe ~~found~~above requirements, biologic products are subject to ~~have~~additional ~~promoted~~controls ~~off-label~~and ~~uses~~requirements. Biologic products are subject, itnot ~~may~~only ~~become~~to ~~subject~~drug cGMPs, but also to ~~adverse~~a ~~public~~special ~~relations~~set ~~and~~of ~~administrative~~manufacturing ~~and~~controls ~~judicial~~that ~~enforcement~~are byintended ~~the~~to ~~FDA,~~ensure ~~the~~product ~~DOJ~~quality. Due to risks involved with biologic products, orFDA ~~the~~can ~~Office~~immediately ofsuspend ~~the~~licenses ~~Inspector~~in ~~General~~situations ofwhere there is a danger to public health. FDA can also prepare or procure biologic products in the ~~Department~~event of ~~Health~~shortages and ~~Human~~critical ~~Services,~~public ashealth ~~well~~needs. After asa ~~state~~BLA ~~authorities.~~is ~~This~~approved, ~~could~~the ~~subject~~product amay ~~company~~also be subject to official lot release as a ~~range~~condition of ~~penalties~~approval, meaning that ~~could~~the ~~have~~manufacturer amust ~~significant~~submit ~~commercial~~samples ~~impact,~~of ~~including~~each ~~civil~~lot ~~and~~of ~~criminal~~product ~~fines~~to ~~and~~the ~~agreements~~FDA ~~that~~together ~~materially~~with ~~restrict~~a release protocol showing the ~~manner~~results inof ~~which~~all of the manufacturer’s tests. Manufacturers subject to lot release may not release a ~~company~~biologic ~~promotes~~product orfor ~~distributes~~distribution ~~drug~~until ~~products~~authorized by FDA. The ~~federal~~FDA ~~government~~may ~~has~~also ~~levied~~perform ~~large~~certain ~~civil~~confirmatory ~~penalties~~tests ~~and~~on ~~criminal~~lots ~~fines~~of ~~against~~some ~~companies~~products before releasing the lots for ~~alleged~~distribution ~~improper~~by ~~promotion~~the ~~and~~manufacturer. In ~~has~~addition, ~~also~~the ~~requested~~FDA ~~that~~conducts ~~companies~~laboratory ~~enter~~research ~~into~~related ~~consent~~to ~~decrees~~the orregulatory ~~permanent~~standards ~~injunctions~~on ~~under~~the ~~which~~safety, ~~specified~~purity, ~~promotional~~potency, ~~conduct~~and iseffectiveness ~~changed~~of orbiological ~~curtailed~~products.

In addition, the distribution of prescription pharmaceutical products is subject to the Prescription Drug Marketing Act (“PDMA”), and its implementing regulations, as well as the Drug Supply Chain Security Act (“DSCSA”). The PDMA regulate and limit the distribution and tracing of prescription drug samples at the federal level. The DSCSA imposes requirements to ensure accountability in distribution, that requires certain licensing and licensing standards, and to identify and remove counterfeit and other illegitimate products from the market.

Abbreviated New Drug Applications for Generic Drugs

In 1984, with passage of the Hatch-Waxman Amendments to the FDCA, Congress authorized the FDA to approve generic drugs under abbreviated approval requirements. To obtain approval of a generic drug, an applicant must submit an Abbreviated New Drug Application (“ANDA”) to the agency. In support of such applications, a generic manufacturer may rely on the preclinical and clinical testing previously conducted for a drug product previously approved under an NDA, known as the Reference Listed Drug (“RLD”). ~~Many states also regulate the distribution of drug product samples and commercial product.~~

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Specifically, in order for an ANDA to be approved, the FDA must find that the generic version is identical to the RLD with respect to the active ingredients, the route of administration, the dosage form, conditions of use, and the strength of the drug, among other requirements. Certain differences, however, between the reference listed drug and ANDA product may be permitted pursuant to a suitability petition. Certain labeling differences may also be permitted if information in the reference listed drug’s label is protected by patent or exclusivities. At the same time, the FDA must also determine that the generic drug is “bioequivalent” to the innovator drug. Under the statute, a generic drug is bioequivalent to a RLD if “the rate and extent of absorption of the drug do not show a significant difference from the rate and extent of absorption of the listed drug.”

Upon approval of an ANDA, the FDA indicates whether the generic product is “therapeutically equivalent” to the RLD in its publication “Approved Drug Products with Therapeutic Equivalence Evaluations,” also referred to as the “Orange Book.” Physicians and pharmacists consider a therapeutic equivalent generic drug to be fully substitutable for the RLD, subject to state law requirements. In addition, by operation of certain state laws and numerous health insurance programs, the FDA’s designation of therapeutic equivalence often results in automatic substitution of the generic drug at the pharmacy.

In an effort to increase competition in the drug and biologic product marketplace, Congress, the executive branch, and FDA have taken certain legislative and regulatory steps. For example, measures have been proposed and implemented to facilitate product importation. Moreover, the 2020 Further Consolidated Appropriations Act included provisions requiring that sponsors of approved drug products, including those subject to REMS, provide samples of the approved products to persons developing 505(b)(2) NDA or ANDA drug products within specified timeframes, in sufficient quantities, and on commercially reasonable market-based terms. Failure to do so can subject the approved product sponsor to civil actions, penalties, and responsibility for attorney’s fees and costs of the civil action. This same bill also includes provisions with respect to shared and separate REMS programs for reference and generic drug products.

Hatch-Waxman Exclusivities

Under the Hatch-Waxman Amendments, the FDA may not approve an ANDA or 505(b)(2) application until any applicable period of non-patent exclusivity for the RLD has expired. The FDCA provides a period of five years of non-patent exclusivity for a new drug containing a new chemical entity. For the purposes of this provision, a new chemical entity (“NCE”), is a drug that contains no active moiety that has previously been approved by the FDA in any other NDA. An active moiety is the molecule or ion, excluding those appended portions of the molecule that cause the drug to be an ester, salt, or other noncovalent derivative, responsible for the physiological or pharmacological action of the drug substance. In cases where such exclusivity has been granted, an ANDA or 505(b)(2) application may not be filed with the FDA until the expiration of five years unless the submission is accompanied by a Paragraph IV certification, in which case the applicant may submit its application four years following the original product approval. The FDCA also provides for a period of three years of exclusivity if the NDA includes reports of one or more new clinical investigations, other than bioavailability or bioequivalence studies, that were conducted by or for the applicant and are essential to the approval of the application. This three-year exclusivity period prevents FDA from making a drug approval effective for the same changes to a previously approved drug product, such as a new dosage form, route of administration, combination or indication, as the product that holds the exclusivity. The three-year and five-year exclusivities, however, do not prevent the filing or approval of full NDAs.

Hatch-Waxman Patent Certification and the 30-Month Stay

Upon approval of an NDA or a supplement thereto, NDA sponsors are required to list with the FDA each patent with claims that cover the applicant’s product or an approved method of using the product. Each of the patents listed by the NDA sponsor is published in the Orange Book. When an ANDA or 505(b)(2) applicant files its application with the FDA, the applicant is required to provide a certification to the FDA concerning any patents listed for the reference product in the Orange Book, except for patents covering methods of use for which the ANDA applicant is not seeking approval.

Specifically, the applicant must certify with respect to each patent that:

●

the required patent information has not been filed;

●

the listed patent has expired;

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●

the listed patent has not expired, but will expire on a particular date and approval is sought after patent expiration; or

●

the listed patent is invalid, unenforceable or will not be infringed by the new product.

A certification that the new product will not infringe the already approved product’s listed patents or that such patents are invalid or unenforceable is called a Paragraph IV certification. If the applicant does not challenge the listed patents or indicates that it is not seeking approval of a patented method of use, the application will not be approved until all the listed patents claiming the referenced product have expired (other than method of use patents involving indications for which the ANDA applicant is not seeking approval). The applicant may also elect to submit a “Section VIII” statement certifying that its proposed label does not contain (or carves out) any language regarding the patented method-of-use rather than certifying to a listed method-of-use patent.

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patents involving indications for which the ANDA applicant is not seeking approval). The applicant may also elect to submit a “Section VIII” statement certifying that its proposed label does not contain (or carves out) any language regarding the patented method-of-use rather than certifying to a listed method-of-use patent.

If the ANDA or 505(b)(2) applicant has provided a Paragraph IV certification to the FDA, the applicant must also send notice of the Paragraph IV certification to the NDA and patent holders within certain specified timeframes. The NDA and patent holders may then initiate a patent infringement lawsuit in response to the notice of the Paragraph IV certification. The filing of a patent infringement lawsuit within 45 days after the receipt of a Paragraph IV certification automatically prevents the FDA from approving the ANDA or 505(b)(2) application until the earlier of 30 months after the receipt of the Paragraph IV notice, expiration of the patent, or a decision in the infringement case that is favorable to the ANDA applicant.

~~Pediatric~~Biologic ~~Studies~~Exclusivity and ~~Exclusivity~~ Biosimilars

Under the ~~Pediatric~~Biologic ~~Research~~Product ~~Equity~~Competition and Innovation Act (~~“PREA”~~BPCIA), ofa ~~2003,~~product ansponsor ~~NDA~~may orapply ~~supplement~~to ~~thereto~~FDA ~~must~~for ~~contain~~approval ~~data~~of a biologic product that ~~are~~is ~~adequate~~“biosimilar to” ~~assess~~or ~~the~~“interchangeable ~~safety~~with” ~~and~~a ~~effectiveness~~previously ofapproved biological product. For the ~~drug~~FDA to approve a biosimilar product, ~~for~~it ~~the~~must ~~claimed~~find ~~indications~~that inthere is ~~all~~a ~~relevant~~high ~~pediatric~~degree ~~subpopulations,~~of ~~and~~similarity to ~~support~~the ~~dosing~~reference product, notwithstanding minor differences in clinically inactive components, and ~~administration~~that ~~for~~there ~~each~~are ~~pediatric~~no ~~subpopulation~~clinically ~~for~~meaningful ~~which~~differences between the reference product isand proposed ~~safe~~biosimilar product in terms of safety, purity and ~~effective~~potency. ~~With~~Biosimilarity ~~enactment~~must ofbe ~~the~~shown ~~FDASIA~~through inanalytical ~~2012~~studies, ~~sponsors~~animal ~~must~~studies, ~~also~~and ~~submit~~at ~~pediatric~~least ~~study~~one ~~plans~~clinical ~~prior~~trial, toabsent ~~the~~a ~~assessment~~waiver ~~data.~~by ~~Those~~the ~~plans~~FDA. There must ~~contain~~be anno ~~outline~~difference ofbetween the ~~proposed~~reference ~~pediatric~~product ~~study~~and ora ~~studies~~biosimilar ~~the~~in ~~applicant~~mechanism ~~plans~~of toaction, ~~conduct~~conditions of use, ~~including~~route ~~study~~of ~~objectives~~administration, ~~and~~dosage ~~design~~form, ~~any~~and ~~deferral~~strength. orFor ~~waiver~~the ~~requests,~~FDA ~~and~~to ~~other~~approve ~~information~~a ~~required~~biosimilar byproduct ~~regulation.~~as ~~The~~interchangeable ~~applicant,~~with ~~the~~a ~~FDA~~reference product, the FDA ~~and~~must find that the ~~FDA’s~~biosimilar ~~internal~~product ~~review~~can ~~committee~~be ~~must~~expected ~~then~~to ~~review~~produce the ~~information~~same ~~submitted,~~clinical ~~consult~~results ~~with~~as ~~each~~the ~~other~~reference product, and (for ~~agree~~products ~~upon~~administered amultiple ~~final~~times) ~~plan.~~that ~~The~~the ~~FDA~~biologic orand the ~~applicant~~reference biologic may ~~request~~be answitched ~~amendment~~after one has been previously administered without increasing safety risks or risks of diminished efficacy relative to ~~the~~exclusive ~~plan~~use atof ~~any~~the ~~time.~~reference biologic.

If a biosimilar application is submitted to FDA, the biosimilar product sponsor and reference product sponsor may exchange certain patent and product information for the purpose of determining whether there should be a legal patent challenge. Based on the outcome of negotiations surrounding the exchanged information, the reference product sponsor may bring a patent infringement suit and injunction proceedings against the biosimilar product sponsor. The biosimilar applicant may also be able to bring an action for declaratory judgment concerning the patent.

Like the Hatch Waxman Act for small molecule drugs, the BPCIA provides for certain periods of regulatory exclusivity for biologic products. Specifically, a biosimilar product application may not be submitted to the FDA until four years following the date of approval of the reference product and the FDA may not approve a biosimilar product until 12 years from the date of approval of the reference product. Not all biologic products, however, will qualify for exclusivity. For instance, certain changes and supplements to an approved BLA, and subsequent applications filed by the same sponsor, manufacturer, licensor, predecessor in interest, or other related entity do not qualify for the 12 year exclusivity period. Biologic product exclusivity also does not block FDA approval of a full BLA for a competing product.

The FDA maintains a publicly-available online database of licensed biological products, which is commonly referred to as the “Purple Book.” The Purple Book lists product names, dates of licensure, and applicable periods of exclusivity. Further, the reference product sponsor must provide patent information and patent expiry dates to FDA following the exchange of patent information between biosimilar and reference product sponsors. This information is then published in the Purple Book.

FDA Efforts to Increase Competition

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subject the approved product sponsor to civil actions, penalties, and responsibility for attorney’s fees and costs of the civil action. This same bill also includes provisions with respect to shared and separate REMS programs for reference and generic drug products.

Pediatric Studies, Exclusivity, and Vouchers

Under the Pediatric Research Equity Act (“PREA”) of 2003, an NDA, BLA, or supplement thereto must contain data that are adequate to assess the safety and effectiveness of the product for the claimed indications in all relevant pediatric subpopulations, and to support dosing and administration for each pediatric subpopulation for which the product is safe and effective. With enactment of the FDASIA in 2012, sponsors must also submit pediatric study plans prior to the assessment data. Those plans must contain an outline of the proposed pediatric study or studies the applicant plans to conduct, including study objectives and design, any deferral or waiver requests, and other information required by regulation. The applicant, the FDA, and the FDA’s internal review committee must then review the information submitted, consult with each other, and agree upon a final plan. The FDA or the applicant may request an amendment to the plan at any time.

The FDA may, on its own initiative or at the request of the applicant, grant deferrals for submission of some or all pediatric data until after approval of the product for use in adults, or full or partial waivers from the pediatric data requirements. Additional requirements and procedures relating to deferral requests and requests for extension of deferrals are contained in the FFDCA. Unless otherwise required by regulation, the pediatric data requirements do not apply to products with orphan designation.

InPediatric ~~addition,~~exclusivity ~~the~~is ~~FDA~~another ~~Reauthorization~~type ~~Act~~ of ~~2017~~non-patent ~~introduced~~marketing aexclusivity ~~new~~in ~~provision~~the ~~regarding~~United ~~required~~States ~~pediatric~~and, ~~studies.~~if ~~Under this statute~~granted, provides for ~~product~~the ~~candidates~~attachment ~~intended~~of ~~for~~an ~~the~~additional ~~treatment~~six months of ~~adult~~marketing ~~cancer~~protection ~~which~~to ~~are~~the ~~directed~~term atof ~~molecular~~any ~~targets~~existing ~~that~~regulatory exclusivity, including the ~~FDA~~non-patent ~~determines~~and toorphan beexclusivity ~~substantially~~and ~~relevant~~on tolisted ~~the~~patents. ~~growth~~This orsix-month ~~progression~~exclusivity ofmay be granted if a sponsor submits pediatric ~~cancer,~~data ~~original~~that ~~application~~fairly ~~sponsors~~respond ~~must~~to ~~submit,~~a ~~with~~written request from the ~~marketing~~FDA ~~application,~~for ~~reports~~such ~~from~~data. ~~molecularly~~The ~~targeted~~data ~~pediatric~~do ~~cancer~~not ~~investigations~~need ~~designed~~to show the product to ~~yield~~be ~~clinically~~effective ~~meaningful~~in the pediatric ~~study~~population ~~data~~studied; rather, ~~gathered~~if ~~using~~the ~~appropriate~~clinical ~~formulations~~trial ~~for~~is ~~each~~deemed ~~applicable~~to ~~age~~fairly ~~group,~~respond to ~~inform~~the ~~potential~~FDA’s ~~pediatric~~request, ~~labeling~~the additional protection is granted. ~~The~~If ~~FDA~~reports ~~may,~~of onrequested ~~its~~pediatric ~~own~~studies ~~initiative~~are orsubmitted atto and accepted by the ~~request~~FDA ofwithin the ~~applicant~~applicable time limits, ~~grant~~whatever ~~deferrals~~statutory or ~~waivers~~regulatory periods of ~~some~~exclusivity or ~~all~~orange ofbook ~~this~~listed ~~data,~~patents asthat ~~above~~cover the product are extended by six months. ~~Unlike~~This ~~PREA~~is not a patent term extension, ~~orphan~~but ~~products~~it ~~are~~effectively ~~not~~extends ~~exempt~~the ~~from~~regulatory ~~this~~period ~~requirement~~during which the FDA cannot approve another application.

~~Pediatric~~As ~~exclusivity~~a isfurther ~~another~~incentive ~~type~~to ofdevelop ~~non-patent~~products ~~marketing~~for ~~exclusivity~~pediatric indiseases, the ~~United~~FDCA ~~States~~established ~~and,~~a ifprogram ~~granted,~~under ~~provides~~which ~~for~~ the ~~attachment~~FDA ofawards anpriority ~~additional~~review ~~six~~vouchers ~~months~~to sponsors of ~~marketing~~rare ~~protection~~pediatric todisease ~~the~~products ~~term~~that ofmeet ~~any~~certain ~~existing~~criteria. ~~regulatory~~The ~~exclusivity,~~rare ~~including~~disease must be serious or life-threatening in which the ~~non~~serious or life-~~patent~~threatening ~~and~~manifestations ~~orphan~~primarily ~~exclusivity~~affect ~~and~~individuals onaged ~~listed~~from ~~patents~~birth to 18 years. ~~This~~The ~~six-month~~product ~~exclusivity~~must ~~may~~contain beno ~~granted~~active ifingredient an(including ~~NDA~~any ~~sponsor~~ester ~~submits~~or ~~pediatric~~salt ~~data~~of the active ingredient) that ~~fairly~~has ~~respond~~been topreviously aapproved ~~written~~in ~~request~~any ~~from~~other application and the ~~FDA~~application must meet certain additional qualifying criteria, including eligibility for ~~such~~FDA ~~data~~priority review. ~~The~~If ~~data~~FDA dodetermines ~~not~~that ~~need~~a toproduct ~~show~~is ~~the~~for ~~product~~a torare bepediatric ~~effective~~disease inand the ~~pediatric~~qualifying ~~population~~application ~~studied;~~criteria ~~rather~~are met, ifFDA ~~the~~may ~~clinical~~award ~~trial~~a ispriority ~~deemed~~review tovoucher. ~~fairly~~This ~~respond~~voucher may be redeemed to ~~the~~receive ~~FDA’s~~priority ~~request,~~review ~~the~~of ~~additional~~a ~~protection~~subsequent ismarketing ~~granted.~~application Iffor ~~reports~~a different product. Use of ~~requested~~a ~~pediatric~~priority ~~studies~~review ~~are~~voucher ~~submitted~~is subject to an FDA user fee. These vouchers are also transferable and ~~accepted~~may be bought and sold. Vouchers may also be revoked by ~~the~~FDA under certain circumstances and sponsors of approved rare pediatric disease products must submit certain reports to FDA. Changes ~~within~~to the ~~statutory~~FDCA, ~~time limits~~however, ~~whatever~~have ~~statutory~~limited orfuture ~~regulatory~~pediatric ~~periods~~priority ofreview ~~exclusivity~~vouchers. orUnder ~~patent~~the ~~protection~~law’s ~~cover~~sunset provision, the ~~product~~drug ~~are~~or ~~extended~~biologic bymust ~~six~~be ~~months.~~designated ~~This~~by isFDA ~~not~~for a ~~patent~~rare ~~term~~pediatric ~~extension~~disease no later than September 30, ~~but~~2024, itand ~~effectively~~approved ~~extends~~no ~~the~~later ~~regulatory~~than ~~period~~September ~~during~~30, ~~which~~2026, unless the ~~FDA~~law ~~cannot~~is ~~approve~~reauthorized ~~another~~by ~~application~~Congress.

17Orphan Drug Designation and Exclusivity

Under the Orphan Drug Act, the FDA may designate a drug or biologic product as an “orphan drug” if it is intended to treat a rare disease or condition (generally meaning that it affects fewer than 200,000 individuals in the United States, or more in cases in which there is no reasonable expectation that the cost of developing and making a product available in the United States for treatment of the disease or condition will be recovered from sales of the product in the United States). Additionally, sponsors must present a plausible hypothesis for clinical superiority to obtain orphan designation if there is a product already approved by the FDA that that is considered by the FDA to be the same as the already approved product and is intended for the same indication. This hypothesis must be demonstrated to obtain orphan exclusivity. A company must request orphan product designation before submitting

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a marketing application. If the request is granted, the FDA will disclose the identity of the therapeutic agent and its potential use. Orphan product designation does not convey any advantage in or shorten the duration of the regulatory review and approval process, although it does convey certain advantages such as tax benefits and exemption from Prescription Drug User Fee Act fees.

If a product with orphan status receives the first FDA approval for the disease or condition for which it has such designation or for a select indication or use within the rare disease or condition for which it was designated, the product generally will receive orphan product exclusivity. Orphan ~~Drug~~product ~~Designation~~exclusivity means that the FDA may not approve any other applications for the same product for the same indication for seven years, except in certain limited circumstances. Competitors may receive approval of different products for the indication for which the orphan product has exclusivity and ~~Exclusivity~~may obtain approval for the same product but for a different indication. If a product designated as an orphan product ultimately receives marketing approval for an indication broader than what was designated in its orphan product application, it may not be entitled to exclusivity.

~~Under the~~ Orphan ~~Drug~~drug ~~Act,~~exclusivity ~~the~~will ~~FDA~~not ~~may~~bar ~~designate~~approval aof ~~drug~~another product asunder ancertain ~~“orphan~~circumstances, ~~drug”~~including if ~~it is intended to treat~~ a ~~rare~~subsequent ~~disease~~product oris ~~condition~~deemed ~~(generally~~to ~~meaning~~be ~~that it affects fewer than 200,000 individuals in~~ the ~~United~~same ~~States,~~as ~~or more in cases in which there is no reasonable expectation that~~ the ~~cost~~product ofwith ~~developing~~exclusivity and ~~making a drug product available in the United States~~ for ~~treatment of~~ the ~~disease~~same or condition ~~will~~is beshown ~~recovered~~to ~~from~~be ~~sales~~clinically ofsuperior to the ~~product).~~approved ~~A company must request orphan~~ product ~~designation~~on ~~before submitting an NDA. If~~ the ~~request~~basis ~~is granted, the FDA will disclose the identity~~ of ~~the~~greater ~~therapeutic~~efficacy ~~agent~~or ~~and~~safety, ~~its~~or ~~potential~~providing ~~use.~~a ~~Orphan~~major ~~product~~contribution ~~designation~~to ~~does~~patient ~~not~~care, ~~convey any advantage in~~ or ~~shorten~~if the ~~duration~~company ofwith ~~the~~orphan ~~regulatory~~drug ~~review~~exclusivity ~~and~~is ~~approval~~not ~~process,~~able ~~although~~to itmeet ~~does~~market ~~convey certain advantages such as tax benefits and exemption from Prescription Drug User Fee Act fees~~demand.

IfPresently, athe ~~product~~exact ~~with~~scope of orphan ~~status~~drug ~~receives~~exclusivity ~~the~~is ~~first~~currently ~~FDA~~in ~~approval~~flux. ~~for~~In 2021, the ~~disease~~scope orof ~~condition~~orphan product exclusivity for ~~which~~the itdrug ~~has~~Firdapse ~~such~~was ~~designation~~the orsubject ~~for~~of a ~~select~~legal ~~indication~~challenge orand ~~use~~reversal ~~within~~in the ~~rare~~Catalyst ~~disease~~Pharms., orInc. ~~condition~~v. ~~for~~Becerra ~~which~~judicial itdecision. ~~was~~The ~~designated,~~court ~~the~~ruled ~~product~~that ~~generally~~the ~~will receive~~ orphan ~~product~~drug exclusivity. ~~Orphan~~for ~~product~~Firdapse ~~exclusivity~~prevented ~~means~~another ~~that~~company’s ~~the~~application ~~FDA~~for ~~may~~the ~~not~~same ~~approve~~drug ~~any~~from ~~other~~being ~~applications~~approved for the ~~same~~entire ~~product~~disease or condition for ~~the~~which ~~same~~orphan ~~indication~~drug ~~for~~designation ~~seven~~was ~~years~~granted, ~~except~~despite inthe ~~certain~~fact ~~limited~~that ~~circumstances.~~the ~~Competitors~~approved ~~may~~product ~~receive~~indication ~~approval~~was ofnarrower. ~~different~~This ~~products~~decision ~~for~~differed from the ~~indication~~FDA’s ~~for~~interpretation ~~which~~of the FDCA, which held that orphan drug exclusivity only protected a product’s ~~has~~approved ~~exclusivity~~indication. ~~and~~In ~~may~~January ~~obtain~~2023, ~~approval~~the ~~for~~FDA published a notice in the ~~same~~Federal ~~product~~Register ~~but~~indicating ~~for~~that ait ~~different~~interprets ~~indication~~the Catalyst Pharms. Ifdecision anarrowly ~~drug~~and orplans ~~drug~~to ~~product~~continue ~~designated~~limiting asthe anscope of orphan ~~product~~drug ~~ultimately~~exclusivity ~~receives~~to ~~marketing~~the ~~approval~~uses ~~for~~or anindications ~~indication~~for ~~broader~~which ~~than~~a ~~what~~drug ~~was~~is ~~designated~~approved. inThe ~~its~~scope of orphan ~~product~~drug ~~application,~~exclusivity itwill ~~may~~likely ~~not~~ be ~~entitled~~an toevolving ~~exclusivity~~area.

~~Orphan~~Qualified ~~drug~~Infectious ~~exclusivity~~Disease will not bar approval of another product under certain circumstances, including if a subsequent product with the same drug for the same condition is shown to be clinically superior to the approved product on the basis of greater efficacy or safety, or providing a major contribution to patient care, or if the company with orphan drug exclusivity is not able to meet market demand. Products

For certain infectious disease products, the FDCA provides further development incentives through additional periods of regulatory exclusivity. Specifically, qualified infectious disease products (QIDPs) are eligible for the extension of existing periods of regulatory exclusivities by an additional five years. This exclusivity extension is subject to certain statutory limits. A QIDP is an antibacterial or antifungal drug for human use intended to treat serious or life-threatening infections, including those caused by an antibacterial or antifungal resistant pathogen, including novel or emerging infectious pathogens; or qualifying pathogens designated by the FDA. Sponsors must request, any time before submitting an NDA, that the FDA designate the product as a QIDP. QIDP designation may be withdrawn if FDA finds that the request for designation contained an untrue statement of material fact.

Patent Term Restoration and Extension

A patent claiming a new drug or biologic product may be eligible for a limited patent term extension under the Drug Price Competition and Patent Term Restoration Act of 1984, which permits a patent restoration of up to five years for patent term lost during product development and the FDA regulatory review. The restoration period granted is typically one-half the time between the effective date of an IND and the submission date of ana ~~NDA~~marketing application, plus the time between the submission date of ana ~~NDA~~marketing application and the ultimate approval date. Patent term restoration cannot be used to extend the remaining term of a patent past a total of 14 years from the product’s approval date. Only one patent applicable to an approved ~~drug~~ product is eligible for the extension, and the application for the extension must be submitted prior to the expiration of the patent in question. A patent that covers multiple drugs or biologics for which approval is sought can only be extended in connection with one of the approvals. The USPTO reviews and approves the application for any patent term extension or restoration in consultation with the FDA.

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Review and Approval of Drug Products in the European Union

In order to market any product outside of the United States, a company must also comply with numerous and varying regulatory requirements of other countries and jurisdictions regarding quality, safety and efficacy and governing, among other things, clinical trials, marketing authorization, commercial sales and distribution of drug products. Whether or not it obtains FDA approval for a product, the company would need to obtain the necessary approvals by the comparable foreign regulatory authorities before it can commence clinical trials or marketing of the product in those countries or jurisdictions. The approval process ultimately varies between countries and jurisdictions and can involve additional product testing and additional administrative review periods. The time required to obtain approval in other countries and jurisdictions might differ from and be longer than that required to obtain FDA approval. Regulatory approval in one country or jurisdiction does not ensure regulatory approval in another, but a failure or delay in obtaining regulatory approval in one country or jurisdiction may negatively impact the regulatory process in others.

Clinical Trial Approval in the EU

Pursuant to the previous European Clinical Trials Directive, a system for the approval of clinical trials in the European Union (“EU”) has been implemented through national legislation of the member states. Under this system, an applicant ~~must~~obtained ~~obtain~~ approval from the competent national authority of a EU member state in which the clinical trial was conducted. In April 2014, the EU adopted a new Clinical Trials Regulation (which came fully into force from January 2022), which will be directly applicable to and binding without the need for any national implementing legislation. Under the new coordinated procedure for the approval of clinical trials, the sponsor of a multi-state clinical trial will be required to submit a single application for approval of a clinical trial to a reporting EU Member State (RMS) through an EU Portal. The submission procedure will be the same irrespective of whether the clinical trial is to be conducted in a single EU member state or in more than one EU member state. The Clinical Trials Regulation also aims to streamline and simplify the rules on safety reporting for clinical trials. The applicant may only start a clinical trial after a competent ethics committee has issued a favorable opinion. Clinical trial applications must be accompanied by an investigational medicinal product dossier with supporting information prescribed by the applicable EU legislation and corresponding national laws of the member states and further detailed in applicable guidance documents.

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trial is to be conducted. Furthermore, the applicant may only start a clinical trial after a competent ethics committee has issued a favorable opinion. Clinical trial application must be accompanied by an investigational medicinal product dossier with supporting information prescribed by the European Clinical Trials Directive and corresponding national laws of the member states and further detailed in applicable guidance documents. In April 2014, the EU adopted a new Clinical Trials Regulation, which will be directly applicable to and binding without the need for any national implementing legislation. Under the new coordinated procedure for the approval of clinical trials, the sponsor of a clinical trial will be required to submit a single application for approval of a clinical trial to a reporting EU Member State (RMS) through an EU Portal. The submission procedure will be the same irrespective of whether the clinical trial is to be conducted in a single EU member state or in more than one EU member state. The Clinical Trials Regulation also aims to streamline and simplify the rules on safety reporting for clinical trials.

Orphan Drug Designation and Exclusivity in the European Union

Regulation 141/2000 provides that a drug shall be designated as an orphan drug if its sponsor can establish: that the product is intended for the diagnosis, prevention, or treatment of a life-threatening or chronically debilitating condition affecting not more than five in ten thousand persons in the [EU] when the application is made, or that the product is intended for the diagnosis, prevention, or treatment of a life-threatening, seriously debilitating, or serious and chronic condition in the [EU] and that without incentives it is unlikely that the marketing of the drug in the [EU] would generate sufficient return to justify the necessary investment. For either of these conditions, the applicant must demonstrate that there exists no satisfactory method of diagnosis, prevention, or treatment of the condition in question that has been authorized in the [EU] or, if such method exists, the drug will be of significant benefit to those affected by that condition.

Regulation 847/2000 sets out criteria and procedures governing designation of orphan drugs in the EU. Specifically, an application for designation as an orphan product can be made any time prior to the filing of an application for approval to market the product. Marketing authorization for an orphan drug leads to a ten-year period of market exclusivity. This period may, however, be reduced to six years if, at the end of the fifth year, it is established that the product no longer meets the criteria for orphan drug designation, for example because the product is sufficiently profitable not to justify market exclusivity. Market exclusivity can be revoked only in very selected cases, such as consent from the marketing authorization holder, inability to supply sufficient quantities of the product, demonstration of “clinically relevant superiority” by a similar medicinal product, or, after a review by the Committee for Orphan Medicinal Products, requested by a member state in the fifth year of the marketing exclusivity period (if the designation criteria are believed to no longer apply). Medicinal products designated as orphan drugs pursuant to Regulation 141/2000 shall be eligible for other incentives made available by the [EU] and by the EU member states to support research into, and the development and availability of, orphan drugs.

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Pharmaceutical Coverage, Pricing and Reimbursement

Significant uncertainty exists as to the coverage and reimbursement status of products approved by the FDA and other government authorities. Sales of products will depend, in part, on the extent to which the costs of the products will be covered by third-party payors, including government healthcare programs in the United States such as Medicare and Medicaid, commercial health insurers and managed care organizations. The process for determining whether a payor will provide coverage for a product may be separate from the process for setting the price or reimbursement rate that the payor will pay for the product once coverage is approved. Third-party payors are increasingly challenging the prices charged, examining the medical necessity, ~~and~~ reviewing the cost-effectiveness of medical products and services, and imposing controls to manage costs. Our rebate payments may increase, or our prices be adjusted under value-based purchasing arrangements based on evidence-based measures or outcomes-based measures for a patient or beneficiary based on use of our drug. Third-party payors may also limit coverage to specific products on an approved list, or formulary, which might not include all of the approved products for a particular indication, or may impose other market access or utilization management controls.

In order to secure coverage and reimbursement for any product that might be approved for sale, a company may need to conduct expensive pharmacoeconomic studies in order to demonstrate the medical necessity and cost-effectiveness of the product, in addition to the costs required to obtain FDA or other comparable regulatory approvals. A payor’s decision to provide coverage for a drug product does not imply that an adequate reimbursement rate will be approved. Third-party reimbursement may not be sufficient to maintain price levels high enough to realize an appropriate return on investment in product development.

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The containment of healthcare costs also has become a priority of federal, state and foreign governments and the prices of drugs have been a focus in this effort. Governments have shown significant interest in implementing cost-containment programs, including price controls, increases in rebates paid, restrictions on reimbursement, and requirements for substitution of generic products. Adoption of price controls and cost-containment measures, and adoption of more restrictive policies in jurisdictions with existing controls and measures, could further limit a company’s revenue generated from the sale of any approved products. Coverage policies and third-party reimbursement rates may change at any time. Even if favorable coverage and reimbursement status is attained for one or more products for which a company or its collaborators receive marketing approval, less favorable coverage policies and reimbursement rates may be implemented in the future. In the EU, pricing and reimbursement schemes vary widely from country to country. Some countries provide that drug products may be marketed only after a reimbursement price has been agreed. Some countries may require the completion of additional studies that compare the cost-effectiveness of a particular drug candidate to currently available therapies, or so-called health technology assessments, in order to obtain reimbursement or pricing approval. For example, the EU provides options for its member states to restrict the range of drug products for which their national health insurance systems provide reimbursement and to control the prices of medicinal products for human use. EU member states may approve a specific price for a drug product or may instead adopt a system of direct or indirect controls on the profitability of the company placing the drug product on the market. Other EU member states allow companies to fix their own prices for drug products, but monitor and control company profits. The downward pressure on health care costs in general, particularly prescription drugs, has become intense. As a result, increasingly high barriers are being erected to the entry of new products. In addition, in some countries, cross-border imports from low-priced markets exert competitive pressure that may reduce pricing within a country. Any country that has price controls or reimbursement limitations for drug products may not allow favorable reimbursement and pricing arrangements.

Healthcare Law and Regulation

Healthcare providers, physicians and third-party payors play a primary role in the recommendation and prescription of drug products that are granted marketing approval. Arrangements with providers, consultants, third-party payors and customers are subject to broadly applicable fraud and abuse and other healthcare laws and regulations. Such restrictions under applicable federal and state healthcare laws and regulations, include the following:

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the federal Anti-Kickback Statute prohibits, among other things, persons from knowingly and willfully soliciting, offering, receiving or providing remuneration, directly or indirectly, in cash or in kind, to induce or reward either the referral of an individual for, or the purchase, order or recommendation of, any good or service, for which payment may be made, in whole or in part, under a federal healthcare program such as Medicare and Medicaid;

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the federal False Claims Act imposes civil penalties, and provides for civil whistleblower or qui tam actions, against individuals or entities for knowingly presenting, or causing to be presented, to the federal government, claims for payment that are false or fraudulent or knowingly making, using, or causing to be made or used a false record or statement material to a false or fraudulent claim, or making a false statement to avoid, decrease or conceal an obligation to pay money to the federal government, with potential substantial liability including mandatory treble damages at trial and significant per-claim penalties;

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the Affordable Care Act included a provision requiring certain providers and suppliers of items and services to federal healthcare programs to report and return overpayments within sixty days after they are “identified” (the “Overpayment Statute”), after which the recipient of the overpayment incurs federal civil False Claims Act liability;

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the Affordable Care Act authorized the imposition of civil monetary penalties on manufactures participating in the 340B program for failure to charge the statutory ceiling price, and required HHS to promulgate regulations establishing the standards for implementing this Civil Monetary Penalty, or CMP, authority. CMS’ final CMP rule went into effect January 1, 2019;

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the federal Health Insurance Portability and Accountability Act of 1996 (“HIPAA”), imposes criminal and civil liability for executing a scheme to defraud any healthcare benefit program or making false statements relating to healthcare matters;

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HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act and their respective implementing regulations, including the Final Omnibus Rule published in January 2013, also imposes obligations, including mandatory contractual terms, with respect to safeguarding the privacy, security and transmission of individually identifiable health information and sanctions for failing to meet those obligations;

● HIPAA imposes specific privacy rules with respect to disclosure of protected health information from covered entities for research purposes, such as clinical trials;

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the federal false statements statute prohibits knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false statement in connection with the delivery of or payment for healthcare benefits, items or services;

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the federal transparency requirements under the Health Care Reform Law, known as the federal Physician Payments Sunshine Act, require manufacturers of covered drugs, devices, biologics and medical supplies to report to the Centers for Medicare & Medicaid Services (“CMS”) within the Department of Health and Human Services information related to certain payments and other transfers of value to US-licensed physicians, physician assistants, nurse practitioners, certified registered nurse anesthetists and certified nurse midwifes and US teaching hospitals and to physician ownership and investment interests held by physicians and their immediate family members; and

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analogous state and foreign laws and regulations, such as state anti-kickback and false claims laws, may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third-party payors, including private insurers.

Some state laws require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government in addition to requiring drug manufacturers to report information related to payments to physicians and other health care providers or marketing expenditures. State and foreign laws also govern the privacy and security of health information in some circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts.

Healthcare Reform in the United States

A primary trend in the United States healthcare industry and elsewhere is cost containment. There have been a number of federal and state proposals during the last several years regarding the pricing of pharmaceutical and ~~biopharmaceutical products, limiting coverage and reimbursement for drugs and other medical products, government control and other changes to the healthcare system in the United States.~~

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biopharmaceutical products, limiting coverage and reimbursement for drugs and other medical products, government control and other changes to the healthcare system in the United States.

By way of example, the United States and state governments continue to propose and pass legislation designed to reduce the cost of healthcare. In March 2010, the United States Congress enacted the Patient Protection and Affordable Care Act (the “PPACA”) which, among other things, includes changes to the coverage and payment for products under government health care programs. Moreover, in 2017, the U.S. Congress modified and amended certain provisions of the PPACA, which could have an impact on coverage and reimbursement for healthcare items and services covered by the federal and state healthcare programs as well as plans in the private health insurance market. The so-called “individual mandate” was repealed as part of tax reform legislation adopted in December 2017. Legal challenges to the PPACA may continue to arise and there may continue to be future efforts to modify, repeal, or otherwise invalidate all, or certain provisions of the Affordable Care Act. The Biden administration is expected to continue to take measures to further facilitate the implementation of the PPACA. Among the provisions of the PPACA of importance to potential drug candidates are:

● an annual, nondeductible fee on any entity that manufactures, or imports specified branded prescription drugs and biologic agents, apportioned among these entities according to their market share in certain government healthcare programs, although this fee would not apply to sales of certain products approved exclusively for orphan indications;

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expansion of eligibility criteria for Medicaid programs by, among other things, allowing states to offer Medicaid coverage to certain individuals with income at or below 133% of the federal poverty level, thereby potentially increasing a manufacturer’s Medicaid rebate liability;

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expanded manufacturers’ rebate liability under the Medicaid Drug Rebate Program by increasing the minimum rebate for both branded and generic drugs and revising the definition of “average manufacturer price” (“AMP”) for calculating and reporting Medicaid drug rebates on outpatient prescription drug prices and extending rebate liability to prescriptions for individuals enrolled in Medicare Advantage plans;

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addressed a new methodology by which rebates owed by manufacturers under the Medicaid Drug Rebate Program are calculated for drugs that are inhaled, infused, instilled, implanted or injected;

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expanded the types of entities eligible for the 340B drug discount program;

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established the Medicare Part D coverage gap discount program by requiring manufacturers to provide a point-of-sale-discount off the negotiated price of applicable brand drugs to eligible beneficiaries during their coverage gap period as a condition for the manufacturers’ outpatient drugs to be covered under Medicare Part D;

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established a new Patient-Centered Outcomes Research Institute to oversee, identify priorities in, and conduct comparative clinical effectiveness research, along with funding for such research;

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established the Independent Payment Advisory Board (“IPAB”) which has authority to recommend certain changes to the Medicare program to reduce expenditures by the program that could result in reduced payments for prescription drugs, subsequently repealed through the Bipartisan Budget Act of 2018; and

● established the Center for Medicare and Medicaid Innovation within CMS to test innovative payment and service delivery models to lower Medicare and Medicaid spending, potentially including prescription drug spending. Other legislative changes have been proposed and adopted since the PPACA was enacted. These changes include the Budget Control Act of 2011, which, among other things, led to aggregate reductions to Medicare payments to providers of up to 2% per fiscal year that started in 2013 , were paused from May 1, 2020 through December 31, 2021, and will continue through 2031 unless additional Congressional action is taken, and the American Taxpayer Relief Act of 2012, which, among other things, reduced Medicare payments to several types of providers and increased the statute of limitations period for the government to recover overpayments to providers from three to five years. These new laws may result in additional reductions in Medicare and other healthcare funding and otherwise affect the prices we may obtain for any of our product candidates for which we may obtain regulatory approval or the frequency with which any such product candidate is prescribed or used.

Other legislative changes have been proposed and adopted since the PPACA was enacted. These changes include the Budget Control Act of 2011, which, among other things, led to aggregate reductions to Medicare payments to providers of up to 2% per fiscal year that started in 2013 , were paused from May 1, 2020 through December 31, 2021, and will continue through 2031 unless additional Congressional action is taken, and the American Taxpayer Relief Act of 2012, which, among other things, reduced Medicare payments to several types of providers and increased the statute of limitations period for the government to recover overpayments to providers from three to five years. These new laws may result in additional reductions in Medicare and other healthcare funding and

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otherwise affect the prices we may obtain for any of our product candidates for which we may obtain regulatory approval or the frequency with which any such product candidate is prescribed or used.

More recently, Congress amended the Medicaid statute, effective October 1, 2019, to exclude prices paid by secondary manufacturers for an authorized generic drug (but not a product approved under the BLA process) from the NDA holder’s AMP for the brand, thereby increasing the rebate amount and the 340B price for the brand. This was implemented by CMS in a final rule issued December 31, 2020. The rule also expanded the definition of products identified as “line extensions” and, in certain circumstances, required inclusion of patient copay assistance in Medicaid best price (effective January 1, 2023), thereby potentially increasing Medicaid rebates paid by manufacturers for such drugs. 340B program guidance regulations on civil monetary penalties for statutory violations, which had been finalized in early 2017 but deferred, also recently went into effect. On November 27, 2020, CMS issued an interim final rule implementing a Most Favored Nation payment model under which reimbursement for certain Medicare Part B drugs and biologicals will be based on a price that reflects the lowest per capita Gross Domestic Product-adjusted (GDP-adjusted) price of any non-U.S. member country of the Organization for Economic Co-operation and Development (OECD) with a GDP per capita that is at least sixty percent of the U.S. GDP per capita. This rule now has been rescinded, but similar programs have been described in recent legislative proposals.

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These healthcare reforms, as well as other healthcare reform measures that may be adopted in the future, may result in additional reductions in Medicare and other healthcare funding, payment of increased rebates, more rigorous coverage criteria, new payment methodologies and additional downward pressure on the price for any approved product and/or the level of reimbursement physicians receive for administering any approved product. Reductions in reimbursement levels may negatively impact the prices or the frequency with which products are prescribed or administered. Any reduction in reimbursement from Medicare or other government programs may result in a similar reduction in payments from private payors. Since enactment of the PPACA, there have been numerous legal challenges and congressional actions to repeal and replace provisions of the law.

Further, there have been several recent U.S. congressional inquiries and proposed federal and proposed and enacted state legislation designed to, among other things, bring more transparency to drug pricing, review the relationship between pricing and manufacturer patient programs, reduce the costs of drugs under Medicare and reform government program reimbursement methodologies for drug products. At the federal level, Congress and the recent administrations have indicated that they will continue to seek new legislative and/or administrative measures to control drug costs. At the state level, individual states are increasingly aggressive in passing legislation and implementing regulations designed to control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing. In addition, regional health care authorities and individual hospitals are increasingly using bidding procedures to determine what pharmaceutical products and which suppliers will be included in their prescription drug and other health care programs. These measures could reduce the ultimate demand for our products, once approved, or put pressure on our product pricing.

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Human Capital Resources

Our vision is to translate scientific breakthroughs into important new medicine. We have a culture where patients are at the center of all we do, with core values that connect us to each other and our stakeholders and define who we are, what we stand for, and how we work.

WeAt ~~are~~March ~~focused on effective attraction~~31, ~~development~~2023, ~~and~~our ~~retention~~total ~~of,~~workforce ~~and~~consisted ~~compensation~~of ~~and~~26 ~~benefits~~full-time toemployees, ~~human~~of ~~resource~~whom ~~talent,~~17 ~~including~~are ~~workforce~~engaged ~~and~~in ~~management~~research and development. However, ~~diversity~~on ~~and~~April ~~inclusion~~13, ~~initiatives~~2023, ~~succession~~the ~~planning,~~Board ~~and~~approved ~~corporate~~certain ~~culture~~cost-cutting ~~and~~measures ~~leadership~~with ~~quality,~~a ~~which~~view ~~are~~to ~~vital~~preserving capital to support our ~~success~~continuing operations. AtAs ~~December~~part 31of this plan, ~~2021~~we have commenced the furlough of 12 employees, ~~our~~representing ~~total~~approximately 46% of our workforce. ~~consisted~~For more information, see Item 9B of 13this ~~full~~Form 10-~~time~~K. None of our employees~~. We~~ are represented by labor unions or covered by collective bargaining agreements, and we consider our ~~relations~~relationship with ~~our~~ employees to be good. We also utilize the services of several independent consultants to support our research and development and general and administrative operations.

~~During~~We ~~2021~~are focused on effective identification, asrecruitment, wedevelopment, ~~worked~~and retention of, and compensation and benefits to, ~~manage~~human ~~through~~resource talent, including workforce and management development, diversity, equity, and inclusion initiatives, succession planning, and corporate culture and leadership quality, which are vital to our success. Attracting, developing and retaining talented employees to support the ~~effects~~growth of ~~the~~our business is an integral part of our human capital ~~aspects~~strategy ofand ~~the~~critical ~~ongoing~~to ~~COVID~~our long-19term ~~pandemic~~success. The principal purposes of our incentive plans are to attract, ~~all~~retain and motivate selected employees, ~~were~~consultants ~~provided~~and ~~the~~directors. ~~option~~Accordingly, ofthese ~~working~~individuals ~~remotely~~are oreligible atto ~~our~~receive ~~Exton~~stock-based compensation awards and cash-based performance bonus awards. Additionally, ~~Pennsylvania~~we ~~office~~offer ~~with~~a ~~appropriate~~package ~~safeguards~~of competitive employee-specific benefits, including 401(k) plan matching contributions.

Corporate Information

We were incorporated in Delaware in 1989 and our office headquarters is located at 505 Eagleview Boulevard, Suite 212, Exton, Pennsylvania 19341. Our internet address is www.aceragen.com. The information contained on, or that can be accessed through, our website is not part of this Form 10-K. We have included our website in this Form 10-K solely as an inactive textual reference.

This Form 10-K contains references to our trademarks and trademarks belonging to other entities. Solely for convenience, trademarks and trade names referred to in this Form 10-K, including logos, artwork, and other visual displays, may appear without the ® or TM symbols, but such references are not intended to indicate, in any way, that we will not assert, to the fullest extent under applicable law, our rights or the rights of the applicable licensor to these trademarks and trade names. We do not intend our use or display of other companies’ trade names or trademarks to imply a relationship with, or endorsement or sponsorship of us by, any other company.

~~Information~~ Available ~~on the Internet~~Information

~~Our internet address is www.iderapharma.com.~~ We make available free of charge through our ~~web~~website ~~site~~ our Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and amendments to these reports filed or furnished pursuant to Section 13(a) or 15(d) of the Exchange Act as soon as reasonably practicable after we electronically file or furnish such materials to the SEC. The SEC maintains an internet site at www.sec.gov containing reports, proxies and information statements and other information regarding issuers that file electronically with the SEC.

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Item 1A.

Risk Factors.

RISK FACTORS

Investing in our securities involves a high degree of risk. You should carefully consider the risks and uncertainties described below in addition to the other information included or incorporated by reference in this Form 10-K before purchasing our common stock. Our business, financial condition and results of operations could be materially and adversely affected by any of these and currently unknown risks or uncertainties. In that case, the market price of our common stock could decline, and you may lose all or part of your investment in our securities.

~~Risks~~Summary ~~Relating~~of toPrincipal ~~Our~~Risk ~~Financial Position and Need for Additional Capital~~Factors

Our stock price has been and may continue to be volatile, and the value of an investment in our common stock may decline.

● We ~~historically~~will ~~have~~need ~~experienced~~to ~~significant~~raise ~~volatility~~additional ~~in our stock price. Since December 31~~capital, ~~2021,~~and ~~our~~if ~~common~~we ~~stock~~are ~~has~~unable traded as low as $0.41 per share. The realization of any of the risks described in these risk factors or other unforeseen risks could have an adverse effect on the market price of our common stock. The trading price of our common stock is likely to ~~continue~~do toso, bewe ~~highly~~will ~~volatile~~not ~~and could~~ be ~~subject~~able to ~~declines~~continue inas ~~response to numerous factors, including disappointing results in~~ a ~~clinical~~going ~~program, as was the case following the announcement of topline results for ILLUMINATE-301~~concern. Other risk factors include results from clinical trials; FDA regulatory actions; announcements by us or our competitors of acquisitions, regulatory approvals, clinical milestones, new products, significant contracts, commercial relationships or capital commitments; additions or departures of key personnel; commencement of, or our involvement in, litigation; and any major change in our Board of Directors or management.

~~From~~● ~~time~~Adverse todevelopments ~~time,~~affecting wefinancial ~~estimate~~institutions, ~~the~~companies ~~timing~~in of the ~~potential~~financial ~~accomplishment~~services ofindustry ~~clinical and other development goals~~ or ~~milestones. These estimated milestones may include~~ the ~~commencement~~financial orservices ~~completion~~industry ~~of clinical trials. Also~~generally, ~~from~~such ~~time~~as to time, we expect that we will publicly announce the anticipated timing of some of these milestones. All these estimated milestones are based on numerous assumptions. These milestones may change and the actual ~~timing~~events ofor ~~meeting~~concerns ~~these~~involving ~~milestones may vary dramatically from our estimates~~liquidity, indefaults ~~some cases for reasons beyond our control. If we do not meet these estimated milestones,~~ or ~~the anticipated timing thereof~~non-performance, ascould ~~publicly~~adversely ~~announced,~~affect our ~~stock~~operations ~~price~~and ~~may decline~~liquidity.

● We ~~may~~expect ~~not~~that bewe ~~able~~will continue to ~~comply~~incur ~~with~~net ~~Nasdaq’s~~losses ~~continued~~in ~~listing~~the ~~standards~~foreseeable future.

● Our ~~common~~ stock ~~trades~~price onhas ~~The~~been ~~Nasdaq~~and ~~Capital~~may ~~Market~~continue ~~(“Nasdaq”)~~to ~~under the symbol “IDRA.” There is also no guarantee that we will~~ be ~~able~~volatile, toand ~~perpetually~~the ~~satisfy~~value ~~Nasdaq’s continued listing requirements to maintain our listing on Nasdaq for any periods~~ of ~~time.~~an ~~Our~~investment ~~failure to continue to meet these requirements may result~~ in our ~~securities~~common ~~being~~stock ~~delisted~~may ~~from Nasdaq~~decline.

On● ~~November 26,~~We ~~2021, we~~may ~~received a deficiency letter (the “Nasdaq Letter”) from the Nasdaq Listing Qualifications Department, notifying us that we are~~ not inbe ~~compliance~~able ~~with Nasdaq Listing Rule 5550(a)(2), which requires the Company~~ to ~~maintain a minimum bid price of at least $1 per share for continued listing on Nasdaq (the “Minimum Bid Requirement”). The Company’s failure to~~ comply with ~~the Minimum Bid Requirement was based on the Company’s common stock per share price being below the $1 threshold for a period of 30 consecutive business days. In accordance with~~ Nasdaq Listing Rule 5810(c)(3)(A) (the “Compliance Period Rule”), the Company has been provided an initial period of 180 calendar days (the “Compliance Date”), to regain compliance with the Minimum Bid Requirement. If, at any time before the Compliance Date, the bid price for the Company’s ~~common~~continued ~~stock~~listing ~~closes at $1~~standards.00 or more per share for a minimum of 10 consecutive business days, as required under Nasdaq requirements, the Staff will provide written notification to the Company that it complies with the Minimum Bid Requirement, unless the Staff exercises its discretion to extend this 10-day period pursuant to Nasdaq Listing Rule 5810(c)(3)(H).

If● ~~the~~Our ~~Company~~recent ~~does~~organizational ~~not regain compliance with the Minimum Bid Requirement by the Compliance Date~~changes, ~~the~~including ~~Company may be eligible for an additional 180 calendar day compliance period (~~the ~~“Second~~Aceragen ~~Compliance Period”). To qualify~~Acquisition, ~~the~~and ~~Company~~any ~~would~~acquisitions ~~need~~or tobusiness ~~meet~~development ~~the~~opportunities ~~continued~~we ~~listing~~pursue ~~requirement~~may ~~for~~not ~~the~~be ~~market value of publicly held~~ successful.

24● As a small biopharmaceutical-focused company with limited resources, we may be unable to attract and retain qualified personnel.

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We depend on our senior management team, and the loss of one or more of our executive officers or key employees or an inability to attract and retain highly skilled employees could compromise our ability to pursue our growth strategy and grow our business.

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We are depending heavily on the development, regulatory approval, and commercialization of product candidates. If we are unable to successfully develop and commercialize product candidates, or experience significant delays in doing so, our business may be materially harmed.

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If our clinical trials are unsuccessful, delayed or terminated, we may not be able to develop and commercialize our product candidates.

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The technologies on which we rely are unproven and may not result in any approved and marketable products.

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We face substantial competition, which may result in others discovering, developing, or commercializing products before or more successfully than us.

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Even if we complete the necessary preclinical studies and clinical trials, the marketing approval process is expensive, time-consuming, and uncertain and may prevent us from obtaining approvals for the commercialization of some or all of our product candidates.

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We may not be able to obtain or maintain anticipated periods of regulatory exclusivity for our product candidates.

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A breakthrough therapy, fast track, or other expedited designation for our product candidates may not lead to a faster development or regulatory review or approval process, and it does not increase the likelihood that those product candidates will receive marketing approval.

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We have only limited experience in regulatory affairs and our product candidates are based on new technologies; these factors may affect our ability or the time we require to obtain necessary regulatory approvals.

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We are subject to extensive and costly governmental regulation, the violation of which could expose us to criminal sanctions, civil penalties, contractual damages, reputational harm, and diminished profits and future earnings.

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We depend on information technology, infrastructure, and data to conduct our business. Any significant disruption, or cyber-attacks, could have a material adverse effect on our business.

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Climate change, environmental, social and governance and sustainability initiatives may result in regulatory or structural industry changes that could require significant operational changes and expenditures, reduce demand for the Company’s products and adversely affect our business.

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~~shares~~● ~~and~~Our ~~all~~existing ~~other initial listing standards of the Nasdaq Capital Market~~collaborations, ~~with~~clinical ~~the~~funding ~~exception of the Minimum Bid Requirement~~arrangements, and ~~provide~~any ~~written~~collaborations ~~notice~~or toarrangements ~~the~~we ~~Staff~~enter ofinto ~~its~~in ~~intention to cure~~ the ~~deficiency~~future ~~during~~may ~~the~~not ~~Second~~be ~~Compliance Period~~successful.

~~Neither~~● ~~the~~If ~~Nasdaq~~we ~~Letter~~are ~~nor~~unable ~~the~~to ~~Company’s~~establish ~~noncompliance~~additional ~~with~~collaborative ~~the Minimum Bid Requirement have an immediate effect on the listing or trading of the Company’s common stock~~alliances, ~~which~~our ~~will~~business ~~continue~~may tobe ~~trade~~materially ~~on The Nasdaq Capital Market under the symbol “IDRA~~harmed.”

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We are subject to substantial customer concentration. If we fail to retain our revenues from the U.S. Government consistent with historical performance or acquire new customers cost-effectively, our business could be adversely affected.

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If we are unable to obtain and maintain patent protection for our discoveries, the value of our technology and any product candidates could be adversely affected.

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We do not own or license patents or patent applications that claim the active pharmaceutical ingredient in our ACG-701 and ACG-801 product candidates.

●

Licensing of intellectual property is of critical importance to our business and involves complex legal, business and scientific issues. Our rights to develop and commercialize ACG-801 and future product candidates may be subject to terms and conditions of licenses granted to us by third parties. If we fail to comply with our obligations under our current or future license agreements, we could lose important intellectual property rights and the ability to continue to develop and commercialize products.

●

We may not be successful in obtaining all rights necessary to develop and commercialize our current or future product candidates.

●

We may become involved in expensive patent litigation or other proceedings, which could result in our incurring substantial costs and expenses or substantial liability for damages, require us to stop our development and commercialization efforts or result in our patents being invalidated, interpreted narrowly or limited.

●

Our intellectual property may be infringed by a third party.

●

If we are unable to protect the confidentiality of our trade secrets, our business and competitive position would be harmed.

●

We may be subject to claims by third parties asserting that we have misappropriated their intellectual property, or claiming ownership of what we regard as our own intellectual property.

●

We rely on government funding for certain aspects of our research and development activities and we may develop intellectual property through such activities and therefore may be subject to federal regulations. Compliance with such regulations may limit our exclusive rights, and limit our ability to contract with non-U.S. manufacturers.

●

Even if the compounds we may develop are successful in clinical trials and receive regulatory approvals, we or our collaboration partners may not be able to successfully commercialize them.

●

Because we have limited manufacturing experience, and no manufacturing facilities or infrastructure, we are dependent on third-party manufacturers to manufacture product candidates for us.

●

We have no experience selling, marketing or distributing potential products and no internal capability to do so.

●

If third parties on whom we rely for clinical and preclinical trials do not perform as contractually required or as we expect, we may not be able to obtain regulatory approval for or commercialize our drug candidates and our business may suffer.

●

We face a risk of product liability claims and may not be able to obtain insurance.

●

Provisions in our restated certificate of incorporation (“Charter”) and second amended and restated bylaws (“Bylaws”) may prevent a change in control that stockholders may consider desirable.

●

Six stockholders beneficially own approximately 69% of our outstanding common stock. If these significant stockholders choose to act together, they could exert substantial influence over our business, and the interests of these stockholders may conflict with those of other stockholders.

●

The issuance or sale of shares of our common stock could depress the trading price of our common stock.

●

Because we do not intend to pay dividends on our common stock, investor returns will be limited to any increase in the value of our stock.

●

Our Series X Preferred Stock have rights, preferences and privileges that are not held by, and are preferential to, the rights of our common stock, which could adversely affect our liquidity and financial condition, and may result in the interests of Series X Preferred Stock holders differing from those of the holders of common stock.

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Risks Relating to Our Financial Position and Need for Additional Capital

Despite recent cost-saving measures, we will need to raise additional capital, and if we are unable to do so, we will not be able to continue as a going concern.

As of December 31, 2022, we had $12.0 million in cash and cash equivalents. We believe based on our current operating plan, our existing cash and cash equivalents will enable us to fund our operations into May 2023. Accordingly, we need to raise additional capital to continue to fund our operations and service our obligations in the future. If we are unable to raise additional capital when needed, we will not be able to continue as a going concern. We do not currently have any products approved for sale and do not generate any revenue from product sales. Accordingly, we expect to rely primarily on equity and/or debt financings and our clinical funding arrangements (e.g., contract with Department of Defense’s DTRA) to fund our continued operations.

We are seeking and expect to continue to seek additional funding through financings of equity and/or debt securities, strategic research and development collaborations, clinical funding arrangements, or the sale or license of technology assets. We may also engage in strategic alternative conversations of mergers, acquisition, or sale of the company or assets. We believe the key factors that will affect our ability to obtain funding are: (i) the results of our clinical development activities in our product candidates we develop on the timelines anticipated; (ii) the time and expense required to submit a marketing application for our product candidates; (iii) the cost, timing, and outcome of regulatory reviews; (iv) the receptivity of the capital markets to financings by biotechnology companies generally and companies with product candidates and technologies similar to ours specifically; (v) receptivity of the capital markets to any in-licensing, product acquisition or other transaction we may enter into; and (vi) ability to enter into additional collaborations and the success of such collaborations.

Financing may not be available to us when we need it, or on favorable or acceptable terms, or at all. We could be required to seek funds through collaborative alliances or through other means that may require us to relinquish rights to some of our technologies, product candidates or products that we would otherwise pursue on our own. In addition, if we raise additional funds by issuing equity securities, our existing stockholders may experience dilution, or an equity financing that involves existing stockholders may cause a concentration of ownership. Debt financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends, and are likely to include rights that are senior to the holders of our common stock. Any additional debt or equity financing may contain terms which are not favorable to us or to our stockholders, such as liquidation and other preferences, or liens or other restrictions on our assets. As discussed in Note 16 to the consolidated financial statements appearing elsewhere in this Form 10-K, additional equity financings may also result in cumulative changes in ownership over a three-year period in excess of 50% which would limit the amount of net operating loss and tax credit carryforwards that we may utilize in any one year. If we are unable to obtain adequate funding on a timely basis or at all, we will be required to terminate, modify or delay clinical trials of our product candidates, or relinquish rights to portions of our technology, product candidates and/or products.

In addition, we recently undertook staff furloughs and salary reductions for certain of our employees, and we may undertake other cost-saving initiatives in the future. The actions we announced on April 13, 2023, as well as future restructuring or cost-saving initiatives, may not achieve our goal of preserving capital or raising additional capital.

If we are unable to raise additional capital when required or on acceptable terms, or we are unsuccessful in our cost-saving measures, we may be required to:

●

significantly delay, scale back, or discontinue the development or commercialization of our product candidates;

●

seek strategic alliances, or amend existing alliances, for research and development programs at an earlier stage than otherwise would be desirable or that we otherwise would have sought to develop independently, or on terms that are less favorable than might otherwise be available in the future;

●

dispose of technology assets, or relinquish or license on unfavorable terms, our rights to technologies or any of our product candidates that we otherwise would seek to develop or commercialize ourselves;

●

pursue the sale of our company to a third party at a price that may result in a loss on investment for our stockholders; or

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●

file for bankruptcy or cease operations altogether.

IfAny weof ~~fail~~these toevents comply with Nasdaq rules and requirements, including, without limitation, the Minimum Bid Requirement, our stock may be delisted. In addition, even if we demonstrate compliance with the Minimum Bid Requirement, we will have to continue to meet other objective and subjective listing requirements to continue to be listed on Nasdaq. Delisting from Nasdaq could ~~make trading our common stock more difficult for investors, potentially leading to declines in our share price and liquidity. Without a Nasdaq listing, stockholders may~~ have a ~~difficult~~material ~~time~~adverse ~~getting~~effect aon ~~quote for the sale or purchase of~~ our ~~common stock~~business, ~~the~~operating ~~sale or purchase of our common stock would likely be made more difficult~~results, and ~~the trading volume and liquidity of our common stock could decline~~prospects. Delisting from Nasdaq could also result in negative publicity and could also make it more difficult for us to raise additional capital. The absence of such a listing may adversely affect the acceptance of our common stock as currency or the value accorded by other parties. Further, if we are delisted, we would also incur additional costs under state blue sky laws in connection with any sales of our securities. These requirements could severely limit the market liquidity of our common stock and the ability of our stockholders to sell our common stock in the secondary market. If our common stock is delisted by Nasdaq, our common stock may be eligible to trade on an over-the-counter quotation system, such as the OTCQB Market, where an investor may find it more difficult to sell our stock or obtain accurate quotations as to the market value of our common stock. In the event our common stock is delisted from Nasdaq, we may not be able to list our common stock on another national securities exchange or obtain quotation on an over-the counter quotation system.

WeAdverse ~~will~~developments ~~need~~affecting ~~additional~~financial ~~financing~~institutions, ~~which~~companies ~~may~~in bethe ~~difficult~~financial toservices ~~obtain~~industry onor ~~terms~~the ~~attractive~~financial toservices usindustry orgenerally, atsuch ~~all.~~as Ifactual weevents ~~are~~or ~~unable~~concerns toinvolving ~~raise capital when needed~~liquidity, wedefaults ~~could~~or benon-performance ~~forced~~by tofinancial ~~delay~~institutions, ~~reduce~~could oradversely ~~eliminate~~affect our ~~product~~current ~~development~~financial ~~programs~~condition, oroperations ~~commercialization~~and ~~efforts~~liquidity.

WeActual ~~expect~~events ~~that~~involving welimited ~~will~~liquidity, ~~need~~defaults, tonon-performance ~~raise~~or ~~additional~~other ~~funds~~adverse indevelopments ~~order~~that toaffect ~~complete~~financial ~~the~~institutions ~~development~~or ~~of,~~other ~~seek~~companies ~~regulatory~~in ~~approvals~~the ~~for,~~financial ~~and~~services ~~commercialization~~industry ofor ~~our~~the ~~drug~~financial ~~candidates~~services ~~for~~industry ~~rare~~generally, ~~disease~~or ~~and~~concerns toor ~~continue~~rumors toabout ~~fund~~any ~~our~~events ~~operations.~~of Wethese ~~are~~kinds, ~~seeking~~have ~~and~~in ~~expect~~the topast ~~continue~~and tomay ~~seek~~in ~~additional~~the ~~funding~~future ~~through~~lead ~~financings~~to ofmarket-wide ~~equity~~liquidity orproblems. ~~debt~~For ~~securities~~example, ~~collaborations,~~on orMarch ~~the~~10, ~~sale~~2023, orone ~~license~~ of ~~assets. We believe the key factors that will affect~~ our ~~ability~~banking topartners, ~~obtain~~Silicon ~~funding~~Valley ~~are:~~Bank, (i“SVB”), ~~the~~was ~~results~~closed ofby ~~our~~the ~~clinical~~California ~~development~~Department ~~activities~~of inFinancial ~~our~~Protection ~~drug~~and ~~candidates~~Innovation, wewhich ~~develop~~appointed on the ~~timelines~~Federal ~~anticipated;~~Deposit ~~(ii)~~Insurance ~~the~~Corporation, ~~time~~or ~~and~~the ~~expense~~Federal ~~required~~Deposit toInsurance ~~submit~~Corporation ~~an NDA for our drug candidates;~~ (~~iii~~“FDIC”) ~~the cost~~, ~~timing,~~as ~~and~~receiver. ~~outcome~~As ofmore ~~regulatory~~fully ~~reviews;~~described ~~(iv)~~in ~~the~~Note ~~receptivity~~19, of“Subsequent ~~the~~Events,” ~~capital~~as ~~markets~~of toMarch ~~financings~~10, by2023, ~~biotechnology~~we ~~companies~~had ~~generally~~approximately ~~and~~56% ~~companies~~of ~~with~~our ~~drug~~cash ~~candidates~~ and ~~technologies~~cash ~~similar~~equivalent tobalances ~~ours~~in ~~specifically;~~segregated ~~(v)~~custodial ~~receptivity~~accounts ofheld ~~the~~by ~~capital~~a ~~markets to any in~~third-~~licensing,~~party ~~product~~custodian ~~acquisition~~for orwhich ~~other~~SVB ~~transaction~~was weour ~~may~~agent ~~enter~~and/or ~~into;~~SVB ~~and~~Asset ~~(vi)~~Management, ~~ability~~an toaffiliate ~~enter~~of ~~into~~SVB, ~~additional~~was ~~collaborations~~the ~~and~~advisor at the ~~success~~time ofSVB ~~such~~was ~~collaborations~~closed.

~~Financing~~On ~~may~~March ~~not~~12, be2023, ~~available~~the toDepartment usof ~~when~~the ~~we need it~~Treasury, orthe onFederal ~~favorable or acceptable terms~~Reserve, orand atthe ~~all.~~FDIC Wejointly ~~could~~released bea ~~required~~statement ~~to seek funds through collaborative alliances or through other means~~ that ~~may~~depositors ~~require~~at usSVB towould ~~relinquish~~have ~~rights~~access to ~~some~~their ~~of our technologies~~funds, ~~drug~~even ~~candidates~~those ~~or drugs that we would otherwise pursue on our own. In addition, if we raise additional~~ funds byin ~~issuing~~excess ~~equity~~of ~~securities,~~the ~~our~~standard ~~existing~~FDIC ~~stockholders~~insurance ~~may experience dilution~~limits, orunder ana ~~equity~~systemic ~~financing~~risk ~~that~~exception. ~~involves~~As ~~existing stockholders may cause a concentration~~ of ~~ownership.~~March ~~Debt financing~~13, ~~if available~~2023, ~~may~~we ~~involve~~had ~~agreements~~access ~~that~~to ~~include covenants limiting or restricting~~ our ~~ability~~cash toand ~~take~~cash ~~specific~~equivalents ~~actions,~~at ~~such~~SVB; ~~as incurring additional debt~~however, ~~making~~there ~~capital~~is ~~expenditures~~uncertainty orin ~~declaring~~the ~~dividends,~~markets ~~and~~regarding ~~are~~the ~~likely~~stability toof ~~include~~regional ~~rights~~banks ~~that~~and ~~are senior to~~ the ~~holders~~safety of ~~our~~deposits ~~common~~in ~~stock.~~excess ~~Any~~of ~~additional~~the ~~debt~~FDIC orinsured ~~equity~~deposit ~~financing~~limits. ~~may~~There ~~contain~~is ~~terms~~no ~~which~~guarantee ~~are~~that ~~not~~the ~~favorable~~U.S. toDepartment usof ~~or to our stockholders~~Treasury, ~~such~~FDIC ~~as liquidation~~ and ~~other~~Federal ~~preferences,~~Reserve orBoard ~~liens~~will orprovide ~~other~~access ~~restrictions~~to onuninsured ~~our~~funds ~~assets. As discussed~~ in ~~Note 13 to~~ the ~~financial~~future ~~statements appearing elsewhere~~ in ~~this~~the ~~Form~~event ~~10-K,~~of ~~additional~~the ~~equity~~closure ~~financings~~of ~~may~~other ~~also~~banks ~~result~~or infinancial ~~cumulative~~institutions ~~changes~~ in ~~ownership over~~ a ~~three-year~~timely ~~period~~fashion inor ~~excess~~at ofall. ~~50%~~Furthermore, ~~which would limit the amount of net operating loss and tax credit carryforwards that~~ we may ~~utilize~~be inimpacted ~~any~~by ~~one~~other ~~year.~~disruptions Ifto wethe ~~are~~U.S. ~~unable~~banking tosystem ~~obtain~~caused ~~adequate~~by ~~funding~~the onrecent adevelopments ~~timely~~involving ~~basis~~SVB, orincluding atpotential ~~all,~~delays wein ~~will~~our beability ~~required~~ to ~~terminate~~transfer funds, ~~modify~~make ~~or delay clinical trials of our drug candidates~~payments, or ~~relinquish~~receive ~~rights~~funds towhether ~~portions~~held ofwith ~~our~~SVB ~~technology, drug candidates and/~~or ~~products.~~other banks.

We expect that we will continue to incur net losses in the foreseeable future.

25During the year ended December 31, 2022, we incurred a net loss of $23.4 million, as compared to net income of $98.1 million for the year ended December 31, 2021. As of December 31, 2022, we had an accumulated deficit of $758.8 million and a cash and cash equivalents balance of $12.0 million. We expect to incur substantial operating losses in future periods and will require additional capital as we seek to advance any future product candidates through development to commercialization. We do not expect to generate product revenue, sales-based milestones or royalties until we successfully complete development of and obtain marketing approval for our product candidates, either alone or in collaboration with third parties, which may not occur or may take a number of years. To commercialize any future product candidates, we need to complete clinical development and comply with comprehensive regulatory requirements. We are subject to numerous risks and uncertainties similar to those of other companies of the same size within the biotechnology industry, such as uncertainty of clinical trial outcomes, uncertainty of additional funding and history of operating losses.

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As of December 31, 2021, we had an accumulated deficit of $735.5 million and a cash and cash equivalents balance of $32.5 million. We expect to incur substantial operating losses in future periods and will require additional capital as we seek to advance any future drug candidates through development to commercialization. We do not expect to generate product revenue, sales-based milestones or royalties until we successfully complete development of and obtain marketing approval for any future drug candidates, either alone or in collaboration with third parties, which may not occur or may take a number of years. To commercialize any future drug candidates, we need to complete clinical development and comply with comprehensive regulatory requirements. We are subject to numerous risks and uncertainties similar to those of other companies of the same size within the biotechnology industry, such as uncertainty of clinical trial outcomes, uncertainty of additional funding and history of operating losses.

Even if we succeed in receiving marketing approval for and commercializing any product candidate, we will continue to incur substantial research and development and other expenditures to develop and market additional potential indications or products. The size of our future net losses will depend, in part, on the rate of future growth of our expenses and our ability to generate revenue. Our prior losses and expected future losses have had and will continue to have an adverse effect on our stockholders’ equity and working capital.

Our stock price has been and may continue to be volatile, and the value of an investment in our common stock may decline.

We historically have experienced significant volatility in our stock price. Since December 31, 2022, our common stock has traded as high as 12.07 and as low as 2.14 per share. The realization of any of the risks described in these risk factors or other unforeseen risks could have an adverse effect on the market price of our common stock. The trading price of our common stock is likely to continue to be highly volatile and could be subject to declines in response to numerous factors, including disappointing results in a clinical program, as was the case following the announcement of topline results for ILLUMINATE-301. Other risk factors include results from clinical trials; FDA

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regulatory actions; announcements by us or our competitors of acquisitions, regulatory approvals, clinical milestones, new products, significant contracts, commercial relationships, or capital commitments; additions or departures of key personnel; commencement of, or our involvement in, litigation; and any major change in our Board of Directors or management.

From time to time, we estimate the timing of the potential accomplishment of clinical and other development goals or milestones. These estimated milestones may include the commencement or completion of clinical trials. Also, from time to time, we expect that we will publicly announce the anticipated timing of some of these milestones. All estimated milestones are based on numerous assumptions. These milestones may change and the actual timing of meeting these milestones may vary dramatically from our estimates, in some cases for reasons beyond our control. If we do not meet these estimated milestones, or the anticipated timing thereof, as publicly announced, our stock price may decline.

We may not be able to comply with Nasdaq’s continued listing standards.

Our Common Stock trades on The Nasdaq Capital Market (“Nasdaq”) under the symbol “ACGN.” We cannot assure you that our securities will continue to be listed on Nasdaq.

As previously reported, on November 26, 2021, we received a deficiency letter (the “First Nasdaq Letter”) from the Nasdaq Listing Qualifications Department (the “Staff”), notifying us that we were not in compliance with Nasdaq Listing Rule 5550(a)(2), which requires us to maintain a minimum bid price of at least $1 per share for continued listing (the “Minimum Bid Requirement”). Our failure to comply with the Minimum Bid Requirement was based on the Common Stock per share price being below the $1.00 threshold for a period of 30 consecutive business days. Pursuant to the First Nasdaq Letter, we had 180 calendar days from November 26, 2021 to regain compliance with the Minimum Bid Requirement.

Also as previously reported, on May 26, 2022, we received a second notice (the “Second Nasdaq Letter”) from the Staff indicating that, while we had not regained compliance with the Minimum Bid Requirement, the Staff had determined that we were eligible for an additional 180-day period, or until November 21, 2022, to regain compliance with the Minimum Bid Requirement.

On November 22, 2022, the Company was notified by the Staff that, based upon the Company’s continued non-compliance with the Minimum Bid Requirement, the Company’s securities were subject to delisting unless the Company timely requested a hearing before the Nasdaq Hearings Panel (the “Panel”).

The Company timely requested a hearing before the Panel, which request stayed any further delisting action by the Staff at least pending the conclusion of the Company’s hearing before the Panel and the expiration of any extension period that may be granted by the Panel to the Company following the hearing. The Company had its Panel hearing on January 5, 2023. On January 10, 2023, the Company received a letter on behalf of the Panel granting an extension until January 20, 2023, for the Company to regain compliance with the continued listing standards.

Following the successful completion of the Company’s 1-to-17 reverse stock split, on January 25, 2023, the Company received a written notice from the Panel that the Company satisfied all initial listing requirements of Nasdaq, as required by the Panel.

While we regained compliance and are currently in compliance with Nasdaq continued listing requirements, there is no guarantee that we will be able to perpetually satisfy Nasdaq’s continued listing requirements to maintain our listing on Nasdaq for any periods of time. Our failure to continue to meet these requirements may result in our securities being delisted from Nasdaq.

As of December 31, 2022, we had an accumulated deficit of $758.8 million and total stockholders’ equity of $11.8 million. We expect to incur substantial operating losses in future periods and will require additional capital as we seek to advance any future product candidates through development to commercialization. Additionally, since December 31, 2022, our common stock has traded as low as 2.14 per share. If we fail to comply with Nasdaq rules and requirements, including, without limitation, with (i) the Minimum Bid Requirement, or (ii) Nasdaq Listing Rule 5550(b)(1), which requires the Company to maintain a minimum of $2.5 million in stockholders’ equity (the “Minimum Equity Requirement”), our stock may be delisted. In addition, even if we demonstrate compliance

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with the Minimum Bid Requirement and Minimum Equity Requirement, we will have to continue to meet other objective and subjective listing requirements to continue to be listed on Nasdaq. Delisting from Nasdaq could make trading our common stock more difficult for investors, potentially leading to declines in our share price and liquidity. Without a Nasdaq listing, stockholders may have a difficult time getting a quote for the sale or purchase of our common stock, the sale or purchase of our common stock would likely be made more difficult, and the trading volume and liquidity of our common stock could decline. Delisting from Nasdaq could also result in negative publicity and could also make it more difficult for us to raise additional capital. The absence of such a listing may adversely affect the acceptance of our common stock as currency or the value accorded by other parties. Further, if we are delisted, we would also incur additional costs under state blue sky laws in connection with any sales of our securities. These requirements could severely limit the market liquidity of our common stock and the ability of our stockholders to sell our common stock in the secondary market. If our common stock is delisted by Nasdaq, our common stock may be eligible to trade on an over-the-counter quotation system, such as the OTCQB Market, where an investor may find it more difficult to sell our stock or obtain accurate quotations as to the market value of our common stock. In the event our common stock is delisted from Nasdaq, we may not be able to list our common stock on another national securities exchange or obtain quotation on an over-the counter quotation system.

Risks Relating to Our Business, Strategy, and Industry

Our recent organizational changes, ~~undertaken~~including tothe ~~align~~Aceragen toAcquisition, ~~our~~and ~~focus~~any onacquisitions ~~new~~or business development opportunities we pursue may not be successful.

InAs ~~April~~part ~~2021,~~of ~~following~~our ~~the~~business ~~announcement~~strategy, ~~that~~we ~~ILLUMINATE-301~~review ~~did~~and ~~not~~intend ~~meet~~to ~~its~~continue ~~primary~~to ~~endpoint~~review ofacquisition ~~ORR,~~opportunities that we ~~decided~~believe towould ~~implement~~be aadvantageous ~~reduction-in-force~~or ~~affecting~~complementary ~~approximately~~to ~~50%~~the development of our ~~workforce~~business. ~~starting~~During at the ~~second~~third quarter of ~~2021.~~2022, ~~The~~we ~~objective~~acquired ofLegacy ~~this~~Aceragen, ~~workforce~~and ~~reduction~~we ~~was~~may toacquire ~~realign~~additional ~~our~~businesses, ~~workforce~~assets, toor ~~meet~~technologies ~~our needs~~ in ~~light of~~ the ~~outcome~~future. ofIf ~~ILLUMINATE-301’s~~we ~~ORR~~make ~~endpoint.~~any ~~In May 2021~~acquisitions, we ~~announced~~could ~~that~~take weany ~~would~~or ~~not~~all ~~continue~~of ~~ILLUMINATE-301~~the ~~toward~~following ~~its OS endpoint. In connection with these~~ actions, weany ~~have~~one ~~incurred~~of ~~termination~~which ~~costs,~~could ~~which~~adversely ~~include~~affect ~~severance,~~our ~~benefits~~business, ~~and~~financial ~~related costs~~condition, ~~totaling~~results ~~$1.3~~of ~~million~~operations inor ~~2021.~~share price:

We● ~~believe~~use these changes were needed to streamline our organization and reallocate our resources to better align with our current strategic goals, including our current focus on new portfolio opportunities. However, these restructuring activities may yield unintended consequences and costs, such as the loss of institutional knowledge and expertise, attrition beyond our intended reduction-in-force, a ~~reduction~~significant inportion ~~morale among our remaining employees, and the risk that we may not achieve the anticipated benefits, all~~ of ~~which may have an adverse effect on~~ our ~~results~~available ~~of operations or financial condition. In addition~~cash, ~~while~~if positions have been eliminated certain functions necessary to our reduced operations remain, and we may be unsuccessful in distributing the duties and obligations of departed employees among our remaining employees. We may also discover that the reductions in workforce and cost cutting measures will make it difficult for us to pursue new opportunities and initiatives and require us to hire qualified replacement personnel, which may require us to incur additional and unanticipated costs and expenses. Moreover, there is no assurance we will be successful in our pursuit of any new business development opportunities, including additional strategic alternatives. Our failure to successfully accomplish any of the above activities and goals may have a material adverse impact on our business, financial condition, and results of operations. ;

As● require a ~~small~~significant ~~biopharmaceutical-focused~~devotion ~~company~~of ~~with~~management’s ~~limited~~time and resources, wein ~~may~~the bepursuit ~~unable~~or toconsummation ~~attract~~of ~~and~~any ~~retain qualified personnel.~~ acquisition;

We● ~~are~~incur ~~a small company with 13 full-time employees as of December 31~~debt, ~~2021.~~which ~~Any~~may ~~future~~not ~~growth~~be ~~will~~available ~~require~~to ~~hiring~~us ~~additional~~on ~~qualified~~favorable ~~personnel.~~terms ~~Also, because of the specialized scientific nature of our business, we face intense competition for qualified employees~~ and ~~consultants~~may ~~from~~adversely ~~biopharmaceutical~~affect ~~companies, research organizations and academic institutions. Failure to attract and retain qualified personnel would materially harm~~ our ~~ability to compete effectively and grow our business.~~liquidity;

If● weissue ~~lose~~equity ~~any of our officers~~ or ~~key~~equity-based ~~employees,~~securities ~~our~~that ~~management~~would ~~and~~dilute ~~technical~~existing ~~expertise~~stockholders’ ~~could~~ownership ~~be weakened significantly.~~percentage;

~~Our~~● ~~success~~assume ~~largely~~contingent ~~depends on the skills, experience,~~ and ~~efforts~~other ofliabilities; ~~our executive officers, especially our President~~ and Chief Executive Officer, Mr. Vincent Milano. We do not maintain key person life insurance policies covering any of our employees. The loss of any of our executive officers could weaken our management and technical expertise significantly and harm our business.

26● take charges in connection with such acquisitions.

For example, prior to the Aceragen Acquisition, Legacy Aceragen was obligated to pay an aggregate amount of $8.0 million to certain former stockholders of Arrevus, Inc. (the “Former Stockholders”). As a result of this obligation and subsequent to the Aceragen Acquisition, on January 31, 2023 the Company issued 12% convertible unsecured promissory notes to certain of the Former Stockholders in an aggregate amount of approximately $5.9 million, which convertible notes bear annual interest at 12%. Under the terms of the convertible notes, at the holder’s election, any or all of the then outstanding principal and accrued interest may be converted into shares of Company’s common stock. The terms of the convertible notes provide the Former Stockholders with customary registration rights covering the common stock issued following any conversion of the convertible notes.

Acquisitions also entail numerous other risks, including, without limitation: difficulties in assimilating acquired operations, products, technologies and personnel; unanticipated costs; diversion of management’s attention from existing operations; risks of entering markets in which we have limited or no prior experience; risk relating to obtaining regulatory approvals; unanticipated costs or liabilities; and potential loss of key employees from either our existing business or the acquired organization. Acquisitions may result in accounting charges for restructuring and other expenses, amortization of purchased technology and intangible assets and stock-based compensation expense, any of which could materially and adversely affect our operating results. We may not be able to realize the anticipated synergies, innovation, operational efficiencies, benefits of or successfully integrate with our existing business the businesses, products, technologies or personnel that we acquire, and our failure to do so could harm our business, operating results, and potentially our share price.

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WeAs ~~are~~a ~~depending~~small ~~heavily~~biopharmaceutical-focused oncompany ~~the~~with ~~development,~~limited ~~regulatory approval~~resources, ~~and commercialization of drug candidates. If~~ we ~~are~~may be unable to ~~successfully~~attract ~~develop~~ and ~~commercialize~~retain ~~drug~~qualified ~~candidates, or experience significant delays in doing so, our business may be materially harmed~~personnel.

WeAs ~~have~~of ~~made~~March ~~and~~31, ~~intend~~2023, towe ~~continue~~had to26 ~~make a significant investment of our~~ full-time ~~and~~employees. ~~financial~~We ~~resources~~are ina ~~the~~small ~~development~~company and ~~commercialization~~any offuture ~~our~~growth ~~drug candidates. Our ability to generate product revenues~~ will ~~depend~~require ~~heavily~~hiring onadditional ~~our~~qualified ~~ability~~personnel. ~~to successfully develop~~Also, ~~obtain~~because ~~regulatory~~of ~~approval~~the ~~for,~~specialized ~~and~~scientific ~~commercialize~~nature of our ~~drug~~business, ~~candidates. If~~ we ~~fail~~face tointense ~~obtain~~competition ~~regulatory~~for ~~approval~~qualified employees and ~~successfully~~consultants ~~commercialize~~from ~~our~~biopharmaceutical ~~drug candidates~~companies, ~~our~~research ~~business~~organizations ~~would be materially~~ and ~~adversely~~academic ~~impacted~~institutions. ~~Even~~ ifFailure ~~our~~to ~~drug~~attract ~~candidates~~and ~~receive~~retain ~~regulatory~~qualified ~~approval,~~personnel wewould ~~will~~materially ~~incur~~harm ~~significant~~our ~~expenses~~ability to ~~support~~compete ~~its~~effectively ~~commercialization~~ and ~~launch,~~grow ~~which~~our ~~investment may never be realized if sales are insufficient~~business.

Our success depends largely upon the continued services of our executive officers and other key employees. We do not maintain “key person” insurance for our executive officers. From time to time there may be changes in our senior management team resulting from the hiring or departure of executives, which could disrupt our business. For example, in 2022, in connection with the Aceragen Acquisition, Vincent Milano resigned as Chief Executive Officer of the Company. Mr. Milano was replaced by John Taylor, the Chief Executive Officer of Legacy Aceragen. All of our employees’ employment is at-will, including the employment of our Chief Executive Officer and our Chief Financial Officer, which means that any of these employees could leave our employment at any time. The replacement of one or more of our executive officers or other key employees would likely involve significant time and costs and may significantly delay or prevent the achievement of our business objectives.

We have made and intend to continue to make a significant investment of our time and financial resources in the development and commercialization of our product candidates. Our ability to generate product revenues will depend heavily on our ability to successfully develop, obtain regulatory approval for, and commercialize our product candidates. If we fail to obtain regulatory approval and successfully commercialize our product candidates, our business would be materially and adversely impacted. Even if our product candidates receive regulatory approval, we will incur significant expenses to support its commercialization and launch, which investment may never be realized if sales are insufficient.

If we experience delays or difficulties in the enrollment of patients in clinical trials, our receipt of necessary regulatory approvals could be delayed or prevented.

We may not be able to initiate or continue clinical trials for our ~~drug~~product candidates if we are unable to locate and enroll a sufficient number of eligible patients to participate in these trials as required by the FDA or similar regulatory authorities outside the U.S. Enrollment may be particularly difficult for our product candidates that are intended for rare and orphan diseases.

In addition, some of our competitors have ongoing clinical trials for ~~drug~~product candidates that treat the same indications as our ~~drug~~product candidates, and patients who would otherwise be eligible for our clinical trials may instead enroll in clinical trials of our competitors'’ ~~drug~~product candidates. Our inability to enroll a sufficient number of patients for our clinical trials could also require us to abandon one or more clinical trials altogether. Enrollment delays may result in increased development costs for our ~~drug~~product candidates, which would cause the value of our company to decline and limit our ability to obtain additional financing.

If our clinical trials are unsuccessful, delayed or terminated, we may not be able to develop and commercialize our ~~drug~~product candidates.

~~Clinical~~We ~~trials~~have ~~are~~not ~~lengthy,~~yet ~~complex,~~been ~~and~~able ~~expensive~~to ~~processes~~commence ~~with~~our ~~uncertain~~ADVANCE ~~results~~study for ACG-801 due to an FDA clinical hold. WeWhile ~~may~~we ~~not~~expect to be able to ~~complete~~resolve ~~any~~the clinical ~~trial~~hold ofand ancommence ~~investigational~~the ~~product~~ADVANCE ~~within~~study ~~any~~in ~~specified~~the ~~time~~second ~~period.~~half ~~Moreover~~of 2023, ~~clinical~~there ~~trials~~is ~~may~~no ~~not~~guarantee ~~show~~that ~~our~~FDA ~~investigational~~will ~~products~~agree and will permit us to ~~have~~begin anthe ~~acceptable~~study. ~~safety~~Additionally, there is a risk that we may experience additional clinical holds and ~~efficacy~~will ~~profile.~~not ~~The~~be ~~FDA,~~able ~~IRBs,~~to obtain timely or ~~other~~any ~~equivalent~~approval ~~foreign~~for ~~regulatory~~our ~~agencies~~product ~~may~~candidates, ~~not~~and ~~allow~~we usmay need to ~~complete~~carry ~~these~~out additional clinical trials, non-clinical studies, CMC assessments, and /or ~~commence~~remediation. Clinical trials are lengthy, complex, and ~~complete~~expensive ~~any~~processes ~~other~~with ~~clinical~~uncertain ~~trials~~results. We may not be able to

Numerous unforeseen events may occur during, or as a result of, preclinical testing, nonclinical testing or the clinical trial process that could delay or inhibit the ability to receive regulatory approval or to commercialize drug products. For example, setbacks in clinical trials may result in enhanced scrutiny by regulators or IRBs of clinical trials of our drug candidates, which could result in regulators or IRBs prohibiting the commencement of clinical trials, requiring additional nonclinical studies as a precondition to commencing clinical trials or imposing restrictions on the design or scope of clinical trials that could slow enrollment of trials, increase the costs of trials or limit the significance of the results of trials. Such setbacks could also adversely impact the desire of investigators to enroll patients in, and the desire of patients to enroll in, clinical trials of our drug candidates. 35

Other events that could delay or inhibit conduct of our clinical trials include: (i) nonclinical or clinical data may not be readily interpreted, which may lead to delays and/or misinterpretation; (ii) our nonclinical tests, including toxicology studies, or clinical trials may produce negative or inconclusive results; (iii) we might have to suspend or terminate our clinical trials if the participating subjects experience serious adverse events or undesirable side effects or are exposed to unacceptable health risks; (iv) regulators or IRBs may hold, suspend or terminate clinical research for various reasons, including noncompliance with regulatory requirements, issues identified through inspections of manufacturing or clinical trial operations or clinical trial sites; (v) we, along with our collaborators and subcontractors, may not employ, in any capacity, persons who have been debarred under by FDA or similar foreign regulatory authorities; (vi) we or our contract manufacturers may be unable to manufacture sufficient quantities of our drug candidates for use in clinical trials; (vii) the cost of our clinical trials may be greater than we currently anticipate making continuation and/or completion improbable; (viii) our investigators and contract research organizations may not follow the applicable regulatory requirements; and (ix) our drug candidates may not cause the desired effects or may cause undesirable side effects or our drug candidates may have other unexpected characteristics.

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Incomplete ~~conducting~~any clinical ~~trials,~~trial weof ~~cannot~~an beinvestigational ~~certain~~product ~~that~~within any ~~planned~~specified ~~clinical trial will begin on~~ time, ifperiod or at all. ~~Delays~~Moreover, ~~in commencing~~ clinical trials ofmay ~~potential~~not ~~products~~show ~~could increase~~ our ~~drug~~investigational ~~candidate~~products ~~development~~to ~~costs,~~have ~~delay~~an ~~any~~acceptable ~~potential~~safety ~~revenues,~~and ~~reduce~~efficacy ~~the~~profile. ~~potential~~The ~~length~~FDA, ofIRBs, ~~patent~~Drug ~~exclusivity~~Safety ~~and~~Monitoring ~~reduce~~Boards, ~~the~~or ~~probability~~other ~~that~~equivalent aforeign ~~potential product will receive~~ regulatory ~~approval. Significant~~agencies ~~clinical~~may ~~trial~~not ~~delays also could~~ allow ~~our~~us ~~competitors~~ to ~~bring~~complete ~~products~~these totrials ~~market~~or ~~before~~commence ~~we do~~ and ~~impair~~complete ~~our~~any ~~ability~~other toclinical ~~commercialize our drug candidates~~trials.

Numerous unforeseen events may occur during, or as a result of, preclinical testing, nonclinical testing or the clinical trial process that could delay or inhibit the ability to receive regulatory approval or to commercialize products. For example, setbacks in clinical trials may result in enhanced scrutiny by regulators or IRBs of clinical trials of our product candidates. Regulators or IRBs may also prohibit the commencement or continuation of clinical trials, data monitoring committees may recommend that studies be modified or discontinued, regulatory authorities may require additional nonclinical studies as a precondition to commencing clinical trials or imposing restrictions on the design or scope of clinical trials that could slow enrollment of trials, increase the costs of trials or limit the significance of the results of trials. Such setbacks could also adversely impact the desire of investigators to enroll patients in, and the desire of patients to enroll in, clinical trials of our product candidates.

Other events that could delay or inhibit conduct of our clinical trials, or prevent receipt of product approval, include: (i) nonclinical or clinical data may not be readily interpreted, which may lead to delays and/or misinterpretation; (ii) our nonclinical tests, including toxicology studies, or clinical trials may produce negative or inconclusive results; (iii) we might have to suspend or terminate our clinical trials if the participating subjects experience serious adverse events or undesirable side effects or are exposed to unacceptable health risks; (iv) regulators or IRBs may hold, suspend or terminate clinical research for various reasons, including noncompliance with regulatory requirements, issues identified through inspections of manufacturing or clinical trial operations or clinical trial sites; (v) we, along with our collaborators and subcontractors, may not employ, in any capacity, persons who have been debarred under by FDA or similar foreign regulatory authorities; (vi) we or our contract manufacturers may be unable to manufacture sufficient quantities of our product candidates for use in clinical trials meeting the applicable quality standards or regulatory authorities may disagree with our manufacturing and control methods; (vii) the cost of our clinical trials may be greater than we currently anticipate making continuation and/or completion improbable; (viii) our investigators and contract research organizations may not follow the applicable regulatory requirements requiring that studies be terminated or repeated; (ix) FDA and regulatory authorities may not agree with the design or analysis of the results from our clinical studies; (x) there may be changes in approval requirements that may necessitate that we conduct additional development work; and (ix) our product candidates may not cause the desired effects or may cause undesirable side effects or our product candidates may have other unexpected characteristics. These risks may be increased for product candidates intended for the treatment of diseases or conditions where there may be limited clinical experience; where there may not be validated or established measures to show improvement of a subject’s condition; where we are using new, unvalidated, or unestablished, endpoints, tests or methodologies; or where the product candidates are new or novel.

In conducting clinical trials, we cannot be certain that any planned clinical trial will begin on time, if at all. Delays in commencing clinical trials of potential products could increase our product candidate development costs, delay any potential revenues, reduce the potential length of patent exclusivity and reduce the probability that a potential product will receive regulatory approval. Significant clinical trial delays also could allow our competitors to bring products to market before we do and impair our ability to commercialize our product candidates.

The technologies on which we rely are unproven and may not result in any approved and marketable products.

Our technologies or therapeutic approaches are relatively new and unproven. Further, the chemical and pharmacological properties of our ~~drug~~product candidates may not be fully recognized in preclinical studies and small-scale clinical trials, and such compounds may interact with human biological systems in unforeseen, ineffective or harmful ways that we have not yet identified. Pre-clinical trials and early -stage clinical trials may not be indicative of results that may be obtained in later stage trials. As a result of these factors, we may never succeed in obtaining regulatory approval to market any product.

We face substantial competition, which may result in others discovering, developing, or commercializing ~~drugs~~products before or more successfully than us.

The development and commercialization of new products is highly competitive. There are many other companies, public and private, actively engaged in discovery, development, and commercializing products and technologies that may compete with our ~~drug~~product ~~candidate~~candidates and rare disease program. Some potentially competitive products have been in development or commercialized for years. Many of the marketed products have been accepted by the ~~medical community, patients, and third-party payors. Our ability to compete may be affected by the previous adoption of such products by the medical community, patients, and third-party payors.~~

We recognize that other companies, including large pharmaceutical companies, may be developing or have plans to develop products and technologies that may compete with ours. Many of our competitors have substantially greater financial, technical, and human resources than we have and/or may have significantly greater experience than we have in undertaking preclinical studies and human clinical trials of new pharmaceutical products, obtaining FDA and other regulatory approvals of products for use in health care and manufacturing, and marketing and selling approved products. We anticipate that the competition with our drug candidates and technologies will be based on a number of factors including product efficacy, safety, availability, and price. The timing of market introduction of our drug candidates and competitive products will also affect competition among products. We expect the relative speed with which we can develop products, complete the clinical trials and approval processes, and supply commercial quantities of the products to the market to be important competitive factors. 36

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medical community, patients, and third-party payors. Our ability to compete may be affected by the previous adoption of such products by the medical community, patients, and third-party payors.

We recognize that other companies, including large pharmaceutical companies, may be developing or have plans to develop products and technologies that may compete with ours. Many of our competitors have substantially greater financial, technical, and human resources than we have and/or may have significantly greater experience than we have in undertaking preclinical studies and human clinical trials of new pharmaceutical products, obtaining FDA and other regulatory approvals of products for use in health care and manufacturing, and marketing and selling approved products. We anticipate that the competition with our product candidates and technologies will be based on a number of factors including product efficacy, safety, availability, reimbursement, insurance coverage, and price. The timing of market introduction of our product candidates and competitive products will also affect competition among products. We expect the relative speed with which we can develop products, complete the clinical trials and approval processes, and supply commercial quantities of the products to the market to be important competitive factors.

Information regarding our competitors is discussed in further detail in the section entitled “Business” of this Form 10-K.

Our business could be adversely affected by the effects of health epidemics, such as the ongoing COVID-19 global pandemic, including disruptions to our clinical trials or the delay of regulatory approvals.

Our business may be adversely affected by the effects of health epidemics, including the ongoing worldwide COVID-19 pandemic. The COVID-19 pandemic has caused significant volatility and uncertainty globally. This has resulted in an economic downturn and may disrupt our business and delay our clinical trials and regulatory approvals. This may also result in an interruption or issues with respect to the manufacture and supply of our product candidates. Quarantines and similar government orders have been enacted in each of the geographies in which we are conducting our clinical trials and may impact the ability of patients to participate in our trials. The ~~patient populations that are eligible for our clinical trials may be immune-compromised and at higher risk for becoming infected with~~ COVID-19. As COVID-19 affects the parts of the world where we are conducting our clinical trials, and the patients involved with these clinical trials become infected with COVID-19, we may have more adverse events and deaths in our clinical trials. The COVID-19 pandemic may also require that changes be made to any clinical trials or product manufacturing that may ultimately have an adverse impact. Additionally, if global health concerns continue to prevent the FDA from conducting its regular inspections, reviews, or other regulatory activities, it could significantly impact the ability of the FDA to timely review and process our regulatory submissions, which could have a material adverse effect on our business. Such concerns could also affect the ability of our personnel to perform their normal responsibilities and could result in temporary closures of our facilities.

The COVID-19 pandemic continues to evolve. The ultimate impact of the COVID-19 pandemic is highly uncertain and subject to change. We do not yet know the full extent of potential delays or impacts on our business, our clinical trials, healthcare systems or the global economy. However, any one or a combination of these events could have an adverse effect on the operation of and results from our clinical trials, which could prevent or delay us from obtaining approval for our ~~drug~~product candidates, or on our employee resources.

28Risks Relating to Regulatory Approval and Marketing and Other Compliance Matters

We are not permitted to market our product candidates in the U.S. or in other countries until we, or any future collaborators, receive marketing approval from the FDA or marketing approval from applicable regulatory authorities outside of the U.S. The approval process is lengthy, often taking a number of years, is uncertain, and is expensive. Securing marketing approval requires the submission of extensive preclinical and clinical data and supporting information to regulatory authorities for each therapeutic indication to establish the product candidate’s safety and efficacy. Information about the product manufacturing process to, and inspection of manufacturing facilities by, the regulatory authorities is also required. Any delay in obtaining or failure to obtain required approvals could materially adversely affect our ability or that of any collaborators we may have to generate revenue from the particular product candidate, which likely would result in significant harm to our financial position and adversely impact our stock price.

Our failure to obtain marketing approval in foreign jurisdictions would prevent our product candidates from being marketed abroad which subjects us to additional business risks that could adversely affect our operations.

We, and any future collaborators, must obtain separate marketing approvals and comply with numerous and varying regulatory requirements in foreign jurisdictions. The approval procedure varies among countries and can involve additional studies. The time required to obtain approval may differ substantially from that required to obtain FDA approval. In addition, in many countries outside of the U.S., it is required that the product be approved for reimbursement before the product can be approved for sale in that country. We, and any future collaborators,

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~~Risks~~may ~~Related~~not toobtain ~~Regulatory~~approvals from regulatory authorities outside of the U.S. on a timely basis, if at all. Approval ~~and~~by ~~Marketing~~the FDA does not ensure approval by regulatory authorities in foreign jurisdictions, and ~~Other~~approval ~~Compliance~~by ~~Matters~~one regulatory authority outside of the U.S. does not ensure approval by regulatory authorities in other jurisdictions or by the FDA.

Even if we, ~~complete~~or ~~the~~any ~~necessary~~future ~~preclinical~~collaborators, ~~studies~~obtain ~~and~~marketing ~~clinical~~approvals ~~trials~~for our product candidates, the ~~marketing~~terms ~~approval~~of ~~process~~approvals isand ~~expensive,~~ongoing ~~time-consuming,~~regulation ~~and~~of ~~uncertain~~our ~~and~~products may ~~prevent~~limit ushow ~~from~~we, ~~obtaining~~or ~~approvals~~they, ~~for~~manufacture ~~the~~and ~~commercialization~~market ofour ~~some~~products, orwhich ~~all~~could ofmaterially impair our ~~drug~~ability ~~candidates~~to generate revenue.

We ~~are not permitted to market our drug candidates in the U.S. or in other countries until we~~, orand any future collaborators, ~~receive~~must ~~approval~~comply ofwith anrequirements ~~NDA~~concerning ~~from~~advertising ~~the~~and ~~FDA~~promotion orfor ~~marketing~~any ~~approval from applicable regulatory authorities outside~~ of ~~the~~our ~~U.S.~~product ~~The~~candidates ~~approval~~for ~~process~~which iswe ~~lengthy,~~or ~~often~~they ~~taking~~obtain amarketing ~~number~~approval. ofSuch ~~years,~~promotional iscommunications ~~uncertain,~~are ~~and~~subject isto ~~expensive.~~a ~~Securing~~variety ~~marketing approval requires the submission~~ of ~~extensive~~legal ~~preclinical~~ and ~~clinical~~regulatory ~~data~~restrictions and ~~supporting~~must ~~information~~be toconsistent ~~regulatory~~with ~~authorities~~the ~~for~~information ~~each~~in ~~therapeutic indication to establish~~ the ~~drug candidate'~~product’s ~~safety~~approved ~~and efficacy~~labeling. ~~Information~~Thus, ~~about the product manufacturing process to~~we, and ~~inspection~~any offuture ~~manufacturing facilities by~~collaborators, ~~the~~will ~~regulatory~~not ~~authorities~~be isable ~~also required. Any delay in obtaining or failure~~ to ~~obtain~~promote ~~required approvals could materially adversely affect our ability or that of~~ any ~~collaborators~~products we ~~may~~develop ~~have~~for toindications ~~generate~~or ~~revenue~~uses ~~from~~for ~~the particular drug candidate,~~ which ~~likely~~they ~~would~~are ~~result~~not ~~in significant harm to our financial position and adversely impact our stock price~~approved.

~~Our~~In ~~failure~~addition, manufacturers of approved products and those manufacturers’ facilities are required to ~~obtain~~comply ~~marketing~~with ~~approval~~extensive inFDA ~~foreign~~requirements, ~~jurisdictions~~including ~~would~~ensuring ~~prevent~~that ~~our~~quality ~~drug~~control ~~candidates~~and ~~from~~manufacturing ~~being~~procedures ~~marketed~~conform ~~abroad~~to Good Manufacturing Practices (“cGMPs”), which ~~subjects~~include usrequirements relating to ~~additional~~quality ~~business~~control ~~risks~~and ~~that~~quality ~~could~~assurance ~~adversely~~as ~~affect~~well as the corresponding maintenance of records and documentation and reporting requirements. We, our ~~operations~~contract manufacturers, our future collaborators and their contract manufacturers could be subject to periodic unannounced inspections by the FDA, and other regulatory authorities to monitor and ensure compliance with cGMPs.

Accordingly, Weassuming we, ~~and~~or ~~any~~our future collaborators, ~~must~~receive ~~obtain separate~~ marketing ~~approvals and comply with numerous and varying regulatory requirements in foreign jurisdictions. The~~ approval ~~procedure~~for ~~varies~~one ~~among~~or ~~countries~~more ~~and~~of ~~can~~our ~~involve~~product ~~additional studies. The time required to obtain approval may differ substantially from that required to obtain FDA approval. In addition~~candidates, ~~in many countries outside of the U.S.~~we, ~~it is required that the drug be approved for reimbursement before the drug can be approved for sale in that country. We,~~ and ~~any~~our future collaborators, ~~may~~and ~~not~~our ~~obtain~~and ~~approvals~~their ~~from~~contract ~~regulatory~~manufacturers ~~authorities~~will ~~outside~~continue ofto ~~the~~expend ~~U.S.~~time, onmoney aand ~~timely~~effort ~~basis,~~in ~~if at~~ all. ~~Approval~~areas byof ~~the FDA does not ensure approval by~~ regulatory ~~authorities~~compliance, inincluding ~~foreign~~manufacturing, ~~jurisdictions~~production, ~~and~~product ~~approval~~surveillance byand ~~one~~quality ~~regulatory authority outside of the U~~control.S. ~~does not ensure approval by regulatory authorities in other jurisdictions or by the FDA.~~

~~Even~~If if we, orand ~~any~~our future collaborators, ~~obtain~~are ~~marketing~~not ~~approvals~~able ~~for~~to ~~our~~comply ~~drug~~with ~~candidates~~post-approval regulatory requirements, ~~the~~we, ~~terms~~and ofour ~~approvals~~future ~~and~~collaborators, ~~ongoing~~could ~~regulation~~have ofthe marketing approvals for our ~~drugs~~products ~~may~~withdrawn ~~limit~~by ~~how~~regulatory weauthorities and our, or ~~they~~our future collaborators’, ~~manufacture~~ability ~~and~~to market ~~our~~any ~~drugs~~future products could be limited, which could ~~materially~~adversely ~~impair~~affect our ability to ~~generate~~achieve ~~revenue~~or sustain profitability. Further, the cost of compliance with post-approval regulations may have a negative effect on our operating results and financial condition.

WeMoreover, legislative and ~~any~~regulatory ~~future~~proposals ~~collaborators,~~have ~~must~~been ~~comply~~made ~~with~~to ~~requirements~~expand ~~concerning~~post-approval ~~advertising~~requirements and ~~promotion~~restrict ~~for~~promotional ~~any~~activities ofrelating to our drug ~~candidates~~and ~~for~~biologic ~~which~~products. weWe cannot be sure whether additional legislative changes will be enacted, or ~~they~~whether ~~obtain~~FDA ~~marketing~~regulations, ~~approval.~~guidance ~~Such~~or ~~promotional~~interpretations ~~communications~~will ~~are~~be ~~subject~~changed, toor awhat ~~variety~~the impact of ~~legal~~such ~~and~~changes ~~regulatory~~on ~~restrictions~~the ~~and~~marketing ~~must~~approvals of our product candidates, if any, may be. ~~consistent~~In ~~with~~addition, ~~the~~increased ~~information~~scrutiny inby Congress of the ~~drug~~FDA’s ~~approved~~approval ~~labeling.~~process ~~Thus,~~may ~~we,~~significantly ~~and~~delay ~~any~~or ~~future~~prevent ~~collaborators~~marketing approval, ~~will~~as ~~not~~well beas ~~able~~subject tous ~~promote~~and any ~~drugs~~collaborators weto ~~develop~~more ~~for~~stringent ~~indications~~product orlabeling ~~uses~~and ~~for~~post-marketing ~~which~~testing ~~they~~and ~~are~~other ~~not approved~~requirements.

InAny ~~addition, manufacturers~~ of ~~approved~~our ~~drugs~~product ~~and~~candidates ~~those~~for ~~manufacturers’~~which ~~facilities~~we, ~~are~~or ~~required~~our tofuture ~~comply with extensive FDA requirements~~collaborators, ~~including~~obtain ~~ensuring~~marketing ~~that~~approval ~~quality~~in ~~control~~the ~~and~~future ~~manufacturing~~could ~~procedures~~be ~~conform~~subject to ~~cGMPs,~~post-approval ~~which~~restrictions ~~include~~or ~~requirements~~withdrawal ~~relating~~from ~~to quality control and quality assurance as well as~~ the ~~corresponding~~market ~~maintenance of records~~ and ~~documentation~~we, and ~~reporting requirements. We,~~ our ~~contract~~future ~~manufacturers~~collaborators, ~~our~~may ~~future~~be ~~collaborators~~subject ~~and~~to ~~their~~substantial ~~contract~~penalties ~~manufacturers~~if ~~could~~we, beor ~~subject~~they, fail to ~~periodic~~comply ~~unannounced~~with ~~inspections~~regulatory byrequirements ~~the~~or ~~FDA~~if we, ~~and~~or ~~other~~they, ~~regulatory~~experience ~~authorities~~unanticipated toproblems ~~monitor~~with ~~and~~our ~~ensure~~products ~~compliance~~following ~~with cGMPs~~approval.

~~Accordingly,~~Any ~~assuming~~of our product candidates for which we, or our future collaborators, ~~receive~~obtain marketing approval ~~for~~in ~~one~~the orfuture, ~~more~~as ofwell ~~our~~as ~~drug~~the ~~candidates,~~manufacturing weprocesses, ~~and~~post-approval ~~our~~studies ~~future~~and ~~collaborators~~measures, ~~and~~labeling, ~~our~~advertising and ~~their~~promotional ~~contract~~activities ~~manufacturers~~for such product, among other things, will ~~continue~~be subject to ~~expend~~continual ~~time,~~requirements ~~money~~of and ~~effort~~review inby ~~all~~the ~~areas~~FDA ofand other regulatory ~~compliance~~authorities. These requirements include submissions of safety and other post-marketing information and reports, ~~including~~registration and listing requirements, requirements relating to manufacturing, ~~production~~quality control, quality assurance and corresponding maintenance of records and documents, requirements regarding the distribution of samples to physicians and recordkeeping. Even if marketing approval of a product ~~surveillance~~candidate is granted, the approval may be subject to limitations on the indicated uses for which the product may be marketed or to the conditions of approval, including the requirement to implement a Risk Evaluation and ~~quality~~Mitigation ~~control~~Strategy, which could include requirements for a restricted distribution system.

IfThe ~~we,~~FDA ~~and~~may ~~our~~also ~~future~~impose ~~collaborators,~~requirements ~~are~~for ~~not~~costly ~~able to comply with~~ post-~~approval regulatory requirements, we, and our future collaborators, could have the~~ marketing ~~approvals~~studies ~~for~~or ~~our~~clinical ~~drugs~~trials ~~withdrawn by regulatory authorities~~ and ~~our,~~surveillance ~~or our future collaborators’, ability~~ to ~~market~~monitor ~~any~~the ~~future~~safety ~~drugs could be limited, which could adversely affect our ability to achieve~~ or ~~sustain~~efficacy ~~profitability. Further, the cost~~ of ~~compliance~~a ~~with~~product. ~~post-approval~~The ~~regulations~~FDA ~~may~~and ~~have~~other aagencies, ~~negative~~including ~~effect~~the onDOJ, ~~our~~closely ~~operating~~regulate ~~results~~ and ~~financial condition.~~

Moreover, legislative and regulatory proposals have been made to expand post-approval requirements and restrict promotional activities relating to our drugs. We cannot be sure whether additional legislative changes will be enacted, or whether FDA regulations, guidance or interpretations will be changed, or what the impact of such changes on the 38

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monitor the post-approval marketing ~~approvals~~and promotion of ~~our~~products ~~drug~~to ~~candidates,~~ensure ifthat ~~any~~they are manufactured, ~~may~~marketed, ~~be.~~and Indistributed ~~addition,~~only ~~increased~~for ~~scrutiny~~the byapproved ~~Congress~~indications and in accordance with the provisions of the approved labeling. The FDA's ~~approval~~imposes ~~process~~stringent ~~may~~restrictions ~~significantly~~on ~~delay~~manufacturers’ orcommunications ~~prevent~~regarding ~~marketing~~off-label ~~approval~~use and if we, asor ~~well~~our asfuture ~~subject~~collaborators, usdo ~~and~~not market any ~~collaborators~~of toour ~~more~~product ~~stringent~~candidates ~~product~~for ~~labeling~~which ~~and~~we, ~~post-~~or they, receive marketing ~~testing~~approval ~~and~~for ~~other~~only ~~requirements~~their approved indications, we, or they, may be subject to warnings or enforcement action for off-label promotion.

~~Any~~In addition, later discovery of previously unknown adverse events or other problems with our ~~drug~~products ~~candidates~~or ~~for~~their ~~which~~manufacturers weor manufacturing processes, or failure to comply with regulatory requirements both before and after product approval, may yield various results, including: (i) litigation involving patients taking our ~~future~~product; ~~collaborators~~(ii) restrictions on such products, ~~obtain~~manufacturers ~~marketing~~or ~~approval~~manufacturing inprocesses; (iii) restrictions on the ~~future~~labeling ~~could~~or bemarketing ~~subject~~of a product; (iv) restrictions on product distribution or use; (v) requirements to conduct post-~~approval~~marketing ~~restrictions~~studies or clinical trials; (vi) warning letters or untitled letters, as well as other enforcement and adverse actions; (vii) withdrawal of the products from the market; ~~and~~(viii) ~~we,~~refusal ~~and~~to ~~our~~approve ~~future~~pending ~~collaborators,~~applications ~~may~~or besupplements ~~subject~~ to ~~substantial~~approved ~~penalties~~applications ifthat we submit; (ix) recall of products; (x) fines, restitution or ~~they,~~disgorgement ~~fail~~of profits or revenues; (xi) suspension or withdrawal of marketing approvals; (xii) damage to ~~comply~~relationships with ~~regulatory~~any ~~requirements~~potential collaborators; (xiii) unfavorable press coverage and damage to our reputation; (xiv) refusal to permit the import or ifexport ~~we,~~of products; (xv) product seizure; or ~~they,~~(xvi) ~~experience~~injunctions ~~unanticipated~~or ~~problems~~the ~~with~~imposition ~~our~~of ~~drugs~~civil ~~following~~or ~~approval.~~criminal penalties.

~~Any~~We ofmay ~~our~~not drug candidates for which we, or our future collaborators, obtain marketing approval in the future, as well as the manufacturing processes, post-approval studies and measures, labeling, advertising and promotional activities for such drug, among other things, will be ~~subject~~able to ~~continual~~obtain requirements of and review by the FDA and other regulatory authorities. These requirements include submissions of safety and other post-marketing information and reports, registration and listing requirements, requirements relating to manufacturing, quality control, quality assurance and corresponding maintenance of records and documents, requirements regarding the distribution of samples to physicians and recordkeeping. Even if marketing approval of a drug candidate is granted, the approval may be subject to limitations on the indicated uses for which the drug may be marketed or tomaintain ~~the~~anticipated ~~conditions~~periods of ~~approval,~~regulatory ~~including~~exclusivity ~~the requirement to implement a Risk Evaluation and Mitigation Strategy, which could include requirements~~ for aour ~~restricted~~product ~~distribution system~~candidates.

~~The~~We ~~FDA~~currently ~~may~~anticipate ~~also~~that ~~impose~~our ~~requirements~~product ~~for~~candidates ~~costly~~may ~~post-marketing~~be ~~studies~~eligible orfor ~~clinical~~certain ~~trials~~periods ~~and~~of ~~surveillance~~regulatory toexclusivity. By ~~monitor~~example, the ~~safety~~FDA orhas ~~efficacy~~granted ofus aorphan drug. ~~The~~designation ~~FDA~~for ~~and~~ACG-701 ~~other~~(sodium ~~agencies,~~fusidate) ~~including~~for the treatment ~~DOJ,~~of ~~closely~~CF ~~regulate~~PEx patients and ~~monitor~~for ACG-801 (recombinant human acid ceramidase) for the ~~post~~treatment of Farber disease. Additionally, ACG-~~approval~~801 ~~marketing~~was ~~and~~granted ~~promotion~~orphan ofdrug ~~drugs~~designation toby ~~ensure~~the ~~that~~EMA ~~they~~for ~~are~~Farber ~~manufactured,~~disease. ~~marketed~~We ~~and~~also ~~distributed~~have ~~only~~received QIDP designation for ACG-701 for the ~~approved~~treatment ~~indications~~of CF PEx and inwe ~~accordance~~anticipate ~~with~~that, depending on the ~~provisions~~product candidate, we may be eligible for certain periods of exclusivity under the ~~approved~~Hatch ~~labeling.~~Waxman ~~The~~Act ~~FDA~~and ~~imposes~~the ~~stringent~~Biologic ~~restrictions~~Price onCompetition ~~manufacturers’~~and ~~communications~~Innovation ~~regarding~~Act. ~~off-label~~There ~~use~~can ~~and~~be ifno assurance that we, will obtain any anticipated periods of regulatory exclusivity or ~~our~~that ~~future~~we ~~collaborators,~~will dobe ~~not~~able ~~market~~to ~~any~~maintain ofthe ~~our~~designations ~~drug~~that ~~candidates~~are necessary for ~~which~~such ~~we,~~exclusivities. We oralso ~~they,~~may ~~receive~~not ~~marketing~~be ~~approval~~able ~~for~~to ~~only~~obtain ~~their~~additional ~~approved~~necessary ~~indications~~designations in the future. Moreover, even if we, orinitially ~~they~~receive periods of regulatory exclusivity following product approval, such exclusivities may be ~~subject~~lost toor ~~warnings~~overcome orunder ~~enforcement~~certain ~~action~~circumstances, ~~for~~and ~~off-label~~the ~~promotion.~~scope of such exclusivities may not provide sufficient protection from competition.

InA ~~addition~~breakthrough therapy, ~~later~~fast ~~discovery~~track, ~~of previously unknown adverse events~~ or other ~~problems~~expedited ~~with~~designation for our ~~drugs or their manufacturers or manufacturing processes, or failure to comply with regulatory requirements both before and after~~ product ~~approval,~~candidates may ~~yield~~not ~~various~~lead ~~results,~~to ~~including: (i) litigation involving patients taking our drug; (ii) restrictions on such drugs, manufacturers or manufacturing processes; (iii) restrictions on the labeling or marketing of~~ a ~~drug;~~faster ~~(iv)~~development ~~restrictions on drug distribution~~ or ~~use;~~regulatory ~~(v)~~review ~~requirements to conduct post-marketing studies~~ or ~~clinical~~approval ~~trials; (vi) warning letters or untitled letters~~process, ~~as well as other enforcement~~ and ~~adverse~~it ~~actions;~~does ~~(vii)~~not ~~withdrawal~~increase of the ~~drugs~~likelihood ~~from the market; (viii) refusal to approve pending applications or supplements to approved applications~~ that wethose ~~submit;~~product ~~(ix)~~candidates ~~recall~~will ofreceive ~~drugs; (x) fines, restitution or disgorgement of profits or revenues; (xi) suspension or withdrawal of~~ marketing approvals; (xii) damage to relationships with any potential collaborators; (xiii) unfavorable press coverage and damage to our reputation; (xiv) refusal to permit the import or export of drugs; (xv) drug seizure; or (xvi) injunctions or the imposition of civil or criminal penaltiesapproval.

WeThe ~~may~~FDA ~~not~~has begranted ~~able~~us toFast ~~obtain~~Track ordesignation ~~maintain~~for ~~orphan~~ACG-701 ~~drug~~(sodium ~~exclusivity~~fusidate) for ~~applications~~the treatment of ~~our~~CF ~~drug~~PEx ~~candidates.~~patients and for ACG-801 (recombinant human acid ceramidase) for the treatment of Farber disease.

~~The~~We ~~FDA~~ may ~~designate~~also seek a ~~product~~breakthrough astherapy, anfast ~~orphan~~track, ~~drug~~or ifother itdesignation isfor aappropriate ~~drug~~product ~~intended~~candidates. toDesignations ~~treat~~such aas ~~rare~~these ~~disease~~are orwithin ~~condition,~~the ~~which~~discretion isof ~~generally~~the ~~defined~~FDA, asor aother ~~patient~~comparable ~~population~~regulatory ofauthorities. ~~fewer~~The ~~than~~receipt ~~200,000~~of ~~individuals~~a indesignation ~~the~~for ~~U.S. Generally, if~~ a product ~~with~~candidate anmay ~~orphan~~not ~~drug~~result ~~designation~~in ~~subsequently~~a ~~receives~~faster ~~the~~development ~~first~~process, ~~marketing~~review or approval ~~for~~compared ~~the~~to ~~indication~~products considered for ~~which~~approval itunder ~~has~~conventional ~~such~~FDA ~~designation,~~procedures ~~the~~and ~~product~~does isnot ~~entitled~~assure toultimate aapproval ~~period~~by ofthe ~~seven~~FDA. ~~years~~In ofaddition, ~~marketing~~even ~~exclusivity.~~if ~~Orphan~~one ~~drug~~or ~~exclusivity~~more ~~may~~of beour ~~lost~~product ifcandidates ~~the~~qualify ~~FDA~~under ~~determines~~one ~~that~~of ~~the~~FDA’s ~~request~~designation ~~for~~programs, ~~designation~~the ~~was~~FDA ~~materially~~may ~~defective~~later ordecide ifthat the ~~manufacturer~~products isno ~~unable~~longer tomeet ~~assure~~the ~~sufficient~~conditions ~~quantity~~for ofqualification ~~the~~or ~~drug~~decide tothat ~~meet~~ the ~~needs~~time ofperiod ~~patients~~for ~~with~~FDA ~~the~~review ~~rare~~or ~~disease~~approval orwill ~~condition~~not be shortened.

InWe ~~June~~have ~~2017,~~only ~~the~~limited ~~FDA~~experience ~~granted~~in usregulatory ~~orphan~~affairs ~~drug~~and ~~designation~~our for tilsotolimod for the treatment of melanoma Stages IIb to IV. However, there can be no assurance that we will obtain orphan drug designation or exclusivity for any other disease indications for which we develop tilsotolimod, or for any other drug candidates. There is also no guarantee that we will be able to obtain orphan drug exclusivity if any product candidates ~~with orphan designation~~ are ~~approved. Even~~based ifon wenew ~~obtain~~technologies; ~~orphan~~these ~~drug~~factors ~~exclusivity for a product, that exclusivity~~ may ~~not~~affect ~~effectively~~our ~~protect~~ability ~~the product from competition because different drugs can be approved for the same condition~~ or the ~~same~~time ~~drug~~we ~~can~~require be approved for different conditions. Even after an orphan drug is approved, the FDA can subsequently approve the same drug for the same condition if the FDA concludes that the later drug is clinically superior in that it is shown to beobtain ~~safer,~~necessary ~~more~~regulatory ~~effective or makes a major contribution to patient care~~approvals.

30We have never obtained regulatory approval for, or commercialized, a product. It is possible that the FDA may refuse to accept any or all of our planned marketing applications for substantive review or may conclude, after review of our data, that our applications are insufficient to obtain regulatory approval of any of our product candidates. The FDA may also require that we conduct additional clinical or manufacturing validation studies, which may be costly and time-consuming, and submit that data before it will reconsider our applications. Depending on the extent of these or any other FDA required studies, approval of any marketing application that we

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Asubmit ~~breakthrough~~may ~~therapy,~~be ~~fast~~significantly ~~track~~delayed, orpossibly ~~other expedited designation~~ for ~~our~~years, ~~drug~~or ~~candidates~~ may ~~not~~require ~~lead~~us to aexpend ~~faster~~more ~~development~~resources orthan ~~regulatory~~we ~~review~~have available or ~~approval~~can ~~process, and it does not increase the likelihood that those drug candidates will receive marketing approval~~secure.

WeA ~~may~~variety ~~seek~~of ~~a breakthrough therapy~~factors, ~~fast~~including ~~track,~~inadequate orfunding ~~other designation~~ for ~~appropriate drug candidates. Designations such as these are within~~ the ~~discretion~~FDA, of the ~~FDA.~~SEC ~~The~~and ~~receipt~~other ofgovernment aagencies ~~designation~~could ~~for~~hinder atheir ~~drug~~ability ~~candidate~~to ~~may~~hire ~~not~~and ~~result~~retain inkey aleadership ~~faster~~and ~~development~~other ~~process~~personnel, ~~review~~prevent ornew ~~approval~~products ~~compared~~and toservices ~~drugs~~from ~~considered~~being ~~for~~developed ~~approval~~or ~~under~~commercialized ~~conventional~~in ~~FDA~~a ~~procedures~~timely ~~and~~manner ~~does~~or ~~not~~otherwise ~~assure~~prevent ~~ultimate~~those ~~approval~~agencies byfrom ~~the~~performing ~~FDA.~~normal Inbusiness ~~addition,~~functions ~~even~~on ifwhich ~~one~~the oroperation ~~more~~ of our ~~drug~~business ~~candidates~~may ~~qualify under one of FDA’s designation programs~~rely, ~~the~~which ~~FDA~~could ~~may~~negatively ~~later~~impact ~~decide~~our ~~that the products no longer meet the conditions for qualification or decide that the time period for FDA review or approval will not be shortened~~business.

WeThe ~~have~~ability ~~only~~of ~~limited~~the ~~experience~~FDA into ~~regulatory~~review ~~affairs~~and approve new products can be affected by a variety of factors, including government budget and ~~our~~funding ~~drug~~levels, ~~candidates~~ability ~~are~~to ~~based~~hire onand ~~new~~retain ~~technologies;~~key personnel, accept payments of user fees, and statutory, regulatory, and policy changes. Average review times at the agency have fluctuated in recent years as a result of these and other factors. ~~may~~In ~~affect~~addition, government funding of government agencies on which our ~~ability~~operations ormay ~~the~~rely, ~~time~~including wethose ~~require~~that fund research and development activities, is subject to ~~obtain~~the ~~necessary~~political ~~regulatory~~process, ~~approvals.~~which is inherently fluid and unpredictable.

WeDisruptions ~~have~~at ~~never~~the ~~obtained~~FDA ~~regulatory~~and ~~approval~~other ~~for,~~agencies ormay ~~commercialized,~~also aslow ~~drug.~~the Ittime isnecessary ~~possible~~for ~~that~~new ~~the~~therapeutic ~~FDA~~candidates ~~may refuse~~ to ~~accept~~be ~~any~~reviewed and/or ~~all~~approved ofby ~~our~~necessary ~~planned~~government ~~NDAs~~agencies, ~~for~~which ~~substantive~~could ~~review~~adversely oraffect ~~may~~our ~~conclude~~business. For example, ~~after~~over ~~review~~the oflast ~~our~~several ~~data~~years, ~~that~~the ~~our~~U.S. ~~applications~~government ~~are~~has ~~insufficient~~shut todown ~~obtain~~several ~~regulatory~~times. ~~approval~~If ofa ~~any~~prolonged ofgovernment ~~our~~shutdown ~~drug~~occurs, ~~candidates.~~or ~~The~~if ~~FDA~~global ~~may~~health ~~also~~concerns ~~require~~(such ~~that~~as wethose ~~conduct~~relating ~~additional~~to ~~clinical~~the COVID-19 pandemic) continue to prevent the FDA or ~~manufacturing~~other ~~validation~~regulatory ~~studies,~~authorities ~~which~~from ~~may~~conducting betheir ~~costly~~regular ~~and~~inspections, ~~time-consuming~~reviews, ~~and~~or ~~submit~~other ~~that~~regulatory ~~data~~activities, ~~before~~ it ~~will~~could ~~reconsider~~significantly ~~our~~impact ~~applications.~~the ~~Depending~~ability onof the ~~extent~~FDA ofto ~~these~~timely orreview ~~any~~and ~~other~~process ~~FDA~~our ~~required~~regulatory ~~studies~~submissions, ~~approval~~which ofcould ~~any~~have ~~NDA~~a ~~that~~material weadverse ~~submit~~effect ~~may~~on beour ~~significantly~~business. ~~delayed~~Further, ~~possibly~~future ~~for~~government ~~years,~~shutdowns orcould ~~may~~impact ~~require~~our usability to ~~expend~~access ~~more~~the ~~resources~~public ~~than~~markets weand ~~have~~obtain ~~available~~necessary orcapital ~~can~~in ~~secure.~~order to properly capitalize and continue our operations.

Our product candidates are subject, and any future commercial products will be subject to costly, extensive and rigorous domestic and foreign government regulation, as discussed under the caption “Government Regulation” within Item 1 of this Form 10-K. These requirements are continually evolving, which will require us to adapt our practices and processes, which we may not be able to do.

In addition, our future arrangements with third party payors, healthcare providers and physicians may expose us to broadly applicable fraud and abuse and other healthcare laws and regulations that may constrain the business or financial arrangements and relationships through which we market, sell and distribute any ~~drugs~~products for which we obtain marketing approval. These include, but are not limited to, the following: the Anti-Kickback Statute; the Foreign Corrupt Practices Act; the False Claims Act; privacy laws such as HIPAA; transparency requirements; and analogous state and foreign laws. Additionally, some state laws require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government and require ~~drug~~ manufacturers to report information related to drug pricing and to certain payments and other transfers of value to physicians, other healthcare providers, and healthcare entities, or marketing expenditures.

Efforts to ensure that our business arrangements with third parties will comply with applicable healthcare laws and regulations will involve substantial costs. It is possible that governmental authorities will conclude that our business practices may not comply with current or future statutes, regulations or case law involving applicable fraud and abuse or other healthcare laws and regulations. If our operations are found to be in violation of any of these laws or any other governmental regulations that may apply to us, we may be subject to significant civil, criminal and administrative penalties, damages, fines, imprisonment, exclusion from participation in government funded healthcare programs, such as Medicare and Medicaid, suspension and debarment from procurement and non-procurement transactions, and the curtailment or restructuring of our operations. If any of the physicians or other healthcare providers or entities with whom we expect to do business is found to be not in compliance with applicable laws, they may be subject to criminal, civil, or administrative sanctions, including exclusions from government funded healthcare programs.

~~We depend on information technology, infrastructure, and data to conduct our business. Any significant disruption, or cyber-attacks, could have a material adverse effect on our business.~~ 40

We are dependent upon information technology, infrastructure and data. Computer systems, including ours and those of our suppliers, partners and service providers, contain sensitive confidential information or intellectual property, and are vulnerable to service interruption or destruction, cyber-attacks (both malicious and random) and

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~~other~~We ~~natural~~depend oron ~~man-made~~information ~~incidents or disasters~~technology, ~~which may be prolonged or go undetected. Such events are increasing in their frequency~~infrastructure, ~~sophistication~~ and ~~intensity,~~data ~~and have become increasingly difficult~~ to ~~detect.~~conduct ~~A significant interruption of~~ our ~~information technology could adversely affect our ability to manage and keep our operations running efficiently and effectively~~business. AnAny ~~incident~~significant ~~that results in a wider or sustained~~ disruption, ~~to our business~~ or ~~products~~cyber-attacks, could have a material adverse effect on our business~~, financial condition and results of operations~~.

~~Likewise~~We are dependent upon information technology, infrastructure, and data. ~~privacy~~Computer orsystems, ~~security~~including ~~breaches~~ours byand ~~employees~~those orof ~~others~~our ~~may~~suppliers, ~~pose~~partners, aand ~~risk~~service ~~that~~providers, contain sensitive ~~data,~~confidential ~~including~~information ~~our~~or intellectual property, ~~trade~~and ~~secrets~~are orvulnerable ~~personal~~to ~~information~~service ofinterruption ~~our~~or ~~employees~~destruction, ~~patients~~cyber-attacks or(both ~~other~~malicious ~~business~~and ~~partners~~random) ~~may~~and beother ~~exposed~~natural toor ~~unauthorized~~man-made ~~persons~~incidents or todisasters, ~~the~~which ~~public.~~may ~~There can~~ be noprolonged ~~assurance~~or ~~that~~go ~~our~~undetected. ~~efforts,~~Such orevents ~~the~~are ~~efforts~~increasing ofin ~~our~~their ~~partners~~frequency, sophistication, and ~~vendors~~intensity, ~~will~~and ~~prevent~~have ~~service~~become ~~interruptions,~~increasingly ordifficult ~~identify~~to ~~breaches~~detect. inA significant interruption of our ~~systems,~~information ~~that~~technology could adversely affect our ~~business~~ability ~~and~~to ~~operations~~manage and~~/or~~ ~~result~~keep inour ~~the~~operations ~~loss~~running ofefficiently ~~critical~~and oreffectively. ~~sensitive~~An ~~information,~~incident ~~which~~that ~~could~~results ~~result~~ in ~~financial,~~a ~~legal,~~wider ~~business~~ or ~~reputational~~sustained ~~harm~~disruption to ~~us. In addition,~~ our ~~liability~~business ~~insurance may not be sufficient in type~~ or ~~amount~~products tocould ~~cover~~have usa ~~against~~material ~~claims~~adverse ~~related~~effect toon ~~security~~our ~~breaches~~business, ~~cyberattacks~~financial condition and ~~other~~results ~~related~~of ~~breaches~~operations.

Likewise, data privacy or security breaches by employees or others may pose a risk that sensitive data, including our intellectual property, trade secrets or personal information of our employees, patients, or other business partners may be exposed to unauthorized persons or to the public. There can be no assurance that our efforts, or the efforts of our partners and vendors, will prevent service interruptions, or identify breaches in our systems, that could adversely affect our business and operations and/or result in the loss of critical or sensitive information, which could result in financial, legal, business, or reputational harm to us. In addition, our liability insurance may not be sufficient in type or amount to cover us against claims related to security breaches, cyberattacks, and other related breaches.

Climate change, environmental, social and governance (“ESG”) and sustainability are a growing global movement. These matters have garnered continuous political and social attention leading to the introduction of national, regional, and local legislation, regulatory requirements, reporting obligations, and policy changes. Further, there is a growing societal demand to limit greenhouse gas emissions and support global initiatives. Compliance with these international agreements and measures may necessitate operational changes, impose taxes or require purchases of emission credits, leading to significant capital expenditures. Furthermore, future legislative, regulatory, or policy changes may emerge that require additional modifications to reduce greenhouse gas emissions from our operations, which may result in substantial capital expenditures.

The growing focus on climate change, ESG, and sustainability has led to government investigations and litigation, both public and private, which may result in increased costs or adverse effects on our business or financial results. Furthermore, entities that offer investors information regarding corporate governance and similar issues have developed rating systems for assessing companies on their ESG approach. Some investors use these ratings to inform their investment and voting decisions. If we receive unfavorable ESG ratings, it may lead to a rise in negative investor sentiment towards us, which could harm our securities’ price and our ability to access capital at reasonable costs.

Any or all of these ESG and sustainability initiatives may result in significant operational changes and expenditures, cause us reputational harm, and could materially adversely affect our business, financial condition, and results of operations.

Risks Relating to Collaborators

Our existing collaborations, clinical funding arrangements, and any collaborations or arrangements we enter into in the future may not be successful.

Our current collaboration and clinical funding agreements, as more fully described within Item 1 of this Form 10-K, or any collaborations we may enter into in the future, may not be successful. The success of our collaborative alliances, if any, will depend heavily on the efforts and activities of our collaborators. Our existing collaborations and any potential future collaborations have risks, including the following: (i) our collaborators may control the development (and timing thereof) of the ~~drug~~product candidates being developed with our technologies and compounds ~~and/or the companion diagnostic to be developed for use in conjunction with our drug candidates~~; (ii) our collaborators may control the public release of information regarding the developments; (iii) disputes may arise in the future with respect to the ownership of or right to use technology and intellectual property developed with our collaborators; (iv) disagreements with our collaborators could delay or terminate the development of our products, or result in litigation or arbitration; (v) we may have difficulty enforcing the contracts if any of our collaborators fail to perform; (vi) our collaborators may terminate their collaborations with us, which could make it difficult for us to attract new collaborators or adversely affect the perception of us in the business or financial communities; (vii) our collaboration agreements are likely to be for fixed terms and subject to termination by our collaborators in the event of a material breach or lack of scientific progress by us; (viii) our collaborators may challenge our intellectual property rights or utilize our intellectual property rights in such a way as to invite litigation that could jeopardize or invalidate our intellectual property rights or expose us to potential liability; (ix) our collaborators may not comply with all applicable regulatory requirements; (x) our collaborators may under fund or not commit sufficient resources to the testing or development of our drug candidates; and (xi) our collaborators may develop alternative products either on their own or in collaboration with others, or encounter conflicts of interest or changes in business strategy or other business issues. Additionally, our collaborators will face the same development risks that we do and may not be successful in their efforts. Given these risks, it is possible that any collaborative alliance into which we enter may not be successful.

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developed with our collaborators; (iv) disagreements with our collaborators could delay or terminate the development of our products, or result in litigation or arbitration; (v) we may have difficulty enforcing the contracts if any of our collaborators fail to perform; (vi) our collaborators may terminate their collaborations with us, which could make it difficult for us to attract new collaborators or adversely affect the perception of us in the business or financial communities; (vii) our collaboration agreements are likely to be for fixed terms and subject to termination by our collaborators in the event of a material breach or lack of scientific progress by us; (viii) our collaborators may challenge our intellectual property rights or utilize our intellectual property rights in such a way as to invite litigation that could jeopardize or invalidate our intellectual property rights or expose us to potential liability; (ix) our collaborators may not comply with all applicable regulatory requirements; (x) our collaborators may under fund or not commit sufficient resources to the testing or development of our product candidates; and (xi) our collaborators may develop alternative products either on their own or in collaboration with others, or encounter conflicts of interest or changes in business strategy or other business issues. Additionally, our collaborators will face the same development risks that we do and may not be successful in their efforts. Given these risks, it is possible that any collaborative alliance into which we enter may not be successful.

If we are unable to establish additional collaborative alliances, our business may be materially harmed.

Collaborators provide the necessary resources and ~~drug~~product development experience to advance our compounds in their programs. We have entered into and expect to continue to seek to enter into collaborative alliances with pharmaceutical companies. Upfront payments and milestone payments received from collaborations help to provide us with the financial resources for our internal research and development programs. We believe additional resources will be required to advance compounds. If we do not reach agreements with additional collaborators in the future or if the terms of such a collaborative alliance on are not favorable to us, we may not be able to obtain the expertise and resources necessary to achieve our business objectives, our ability to advance our compounds will be jeopardized and we may fail to meet our business objectives. Moreover, collaborations are complex and time consuming to negotiate, document, and implement. We may not be successful in our efforts to establish and implement collaborations on a timely basis.

We are subject to substantial customer concentration risk. The U.S. Government accounted for all of the Company’s revenues for the year ended December 31, 2022. These revenues are from collaboration and license agreements and a contract with the U.S. Government. If these agreements are terminated or fees and funding are reduced under these agreements, our business could be adversely affected.

Risks Relating to Intellectual Property

If we are unable to obtain and maintain patent protection for our discoveries, the value of our technology and ~~products~~any ~~will~~product candidates could be adversely affected.

32Our commercial success and ability to develop and commercialize products depends in significant part on our ability to: (i) obtain and maintain valid and enforceable patents and other intellectual property rights in the U.S. and other countries with respect to our technology and product candidates; (ii) obtain licenses to the proprietary rights of others on commercially reasonable terms; (iii) operate without infringing upon the proprietary rights of others; (iv) prevent others from infringing on our proprietary rights; and (v) protect our trade secrets. We rely on trade secret, patent, copyright and trademark laws, and confidentiality, licensing and other agreements with employees and third parties, all of which offer only limited protection. We seek to protect our proprietary position by filing and prosecuting patent applications in the U.S. and abroad related to our novel technologies and product candidates that are important to our business.

If the scope of the patent protection we or our licensors obtain is not sufficiently broad, we may not be able to prevent others from developing and commercializing technology and products similar or identical to ours. The degree of patent protection we require to successfully compete in the marketplace may be unavailable or severely limited and may not adequately protect our rights or permit us to gain or keep any competitive advantage. We cannot provide any assurances that any of our patents have, or that any of our pending patent applications that

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~~Our~~mature ~~ability~~into issued patents will include, claims with a scope sufficient to ~~develop~~protect our current and ~~commercialize~~future ~~drugs~~product ~~depends~~candidates inor ~~significant~~otherwise ~~part~~provide onany ~~our~~competitive ~~ability~~advantage. In addition, to: ~~(i)~~the ~~obtain~~extent ~~and~~that ~~maintain~~we ~~valid~~license ~~and~~intellectual ~~enforceable~~property ~~patents;~~in ~~(ii)~~the ~~obtain~~future, we cannot provide assurances that those licenses towill remain in force. In addition, the ~~proprietary~~laws of foreign countries may not protect our rights to the same extent as the laws of ~~others~~the onUnited ~~commercially~~States. ~~reasonably~~Furthermore, ~~terms;~~patents ~~(iii)~~have ~~operate~~a ~~without~~limited ~~infringing~~lifespan. ~~upon~~In the ~~proprietary~~United ~~rights~~States, the natural expiration of ~~others;~~a ~~(iv)~~patent ~~prevent~~is ~~others~~generally ~~from~~20 ~~infringing~~years onafter ~~our~~it ~~proprietary~~is ~~rights~~filed. Various extensions may be available; however, the life of a patent, and ~~(v)~~the ~~protect~~protection ~~our~~it ~~trade~~affords, ~~secrets~~is limited. Given the amount of time required for the development, testing and regulatory review of new product candidates, patents protecting such candidates might expire before or shortly after such candidates are commercialized.

WeOur dopatents ~~not~~and ~~know whether any of our currently~~ pending patent applications, orif ~~those~~issued, ~~patent~~may ~~applications~~not ~~that~~provide weus ~~license~~with ~~will~~any ~~result~~meaningful inprotection ~~the~~or ~~issuance~~prevent ofcompetitors ~~any~~from ~~patents.~~designing ~~Our~~around ~~issued~~our ~~patents~~patent ~~and~~claims ~~those~~to ~~that~~circumvent ~~may~~our bepatents ~~issued~~by indeveloping ~~the~~similar ~~future,~~ or ~~those~~alternative ~~licensed~~technologies toor ~~us,~~therapeutics ~~may be challenged, invalidated, held unenforceable, narrowed~~ in ~~the course of~~ a ~~post~~non-~~issuance~~infringing ~~proceeding~~manner. orFor ~~circumvented~~example, ~~and~~a ~~the~~third ~~rights~~party ~~granted thereunder~~ may ~~not~~develop ~~provide~~a ~~us proprietary protection or~~ competitive ~~advantages~~therapy ~~against~~that ~~competitors~~provides ~~with~~benefits similar ~~technology.~~to ~~Moreover,~~one ~~intellectual~~or ~~property~~more ~~laws~~of ~~may~~our ~~change~~product ~~and~~candidates ~~negatively~~but ~~impact~~that ~~our~~falls ~~ability~~outside tothe ~~obtain~~scope ~~issued~~of ~~patents covering~~ our ~~technologies~~patent orprotection. toIf ~~enforce~~the ~~any~~patent ~~patents~~protection ~~that~~provided ~~issue.~~by ~~Because of~~ the ~~extensive~~patents ~~time~~and ~~required~~patent ~~for~~applications ~~development,~~we ~~testing,~~hold ~~and~~or ~~regulatory~~pursue ~~review~~with ofrespect ato ~~potential~~our product, itcandidates is ~~possible~~not ~~that,~~sufficiently ~~before~~broad ~~any~~to ofimpede ~~our~~such ~~products can be commercialized~~competition, ~~any~~our ~~related~~ability ~~patent~~to ~~may~~successfully ~~expire~~commercialize orour ~~remain~~product incandidates ~~force~~could ~~for~~be ~~only~~negatively ~~a short period following commercialization~~affected, ~~thus~~which ~~reducing~~would ~~any~~harm ~~advantage~~our ~~provided by the patent~~business.

~~Because~~Patent ~~patent~~positions ~~applications~~of inlife ~~the~~sciences ~~U.S.~~companies can be uncertain and ~~many~~involve ~~foreign~~complex ~~jurisdictions~~factual ~~are~~and ~~typically~~legal ~~not~~questions. ~~published~~No ~~until~~consistent 18policy ~~months~~governing ~~after~~the ~~filing,~~scope orof claims allowable in ~~some~~the ~~cases~~pharmaceutical ~~not~~and atbiotechnology ~~all,~~fields ~~and~~have ~~because~~emerged ~~publications~~in the United States. The scope of ~~discoveries~~patent protection in jurisdictions outside of the ~~scientific~~United ~~literature~~States ~~often~~is ~~lag~~also ~~behind~~uncertain. ~~actual~~Changes ~~discoveries,~~in ~~neither~~either wethe ~~nor~~patent ~~our~~laws ~~licensors~~or ~~can~~their beinterpretation ~~certain~~in ~~that~~any jurisdictions in which we orseek ~~they~~patent ~~were~~protection ~~the~~may ~~first~~diminish our ability to ~~make~~protect ~~the~~our inventions ~~claimed~~and inmaintain ~~issued~~and ~~patents~~enforce orour ~~pending~~intellectual ~~patent~~property ~~applications~~rights; and, ormore ~~that~~generally, wemay oraffect ~~they~~the ~~were~~value ~~the~~of ~~first~~our tointellectual ~~file~~property, ~~for~~including ~~protection~~the narrowing of the ~~inventions~~scope ~~set~~of ~~forth~~our inpatents ~~these~~and ~~patent~~any ~~applications~~that we may license.

The patent prosecution process is complex, expensive, time-consuming and inconsistent across jurisdictions. We may not be able to file, prosecute, maintain, enforce, or license all necessary or desirable patent rights at a commercially reasonable cost or in a timely manner or at all. In addition, we may not pursue or obtain patent protection in all relevant markets. It is possible that we will fail to identify important patentable aspects of our research and development efforts in time to obtain appropriate, or any, patent protection. While we enter into non-disclosure and confidentiality agreements with parties who have access to confidential or patentable aspects of our research and development efforts, including for example, our employees, external academic scientific collaborators, CROs, contract manufacturers, consultants, advisors and other third parties, any of these parties may breach the agreements and disclose our confidential or proprietary information before a patent application is filed, thereby endangering our ability to seek patent protection. In addition, publications of discoveries in the scientific and scholarly literature often lag behind the actual discoveries, and patent applications in the United States and other jurisdictions are typically not published until 18 months after filing, or in some cases not at all. Consequently, we cannot be certain that we were the first to file for patent protection on the inventions claimed in our patents or pending patent applications.

The issuance, scope, validity, enforceability and commercial value of our patent rights are highly uncertain. Pending patent applications cannot be enforced against third parties unless, and until, patents issue from such applications, and then only to the extent the issued claims cover the technology. There can be no assurance that our patent applications or any patent applications that we may license in the future will result in patents being issued. Further, the scope of the invention claimed in a patent application can be significantly reduced before the patent is issued, and this scope can be reinterpreted after issuance. Even if patent applications we currently own or that we may license in the future issue as patents, they may not issue in a form that will provide us with adequate protection to prevent competitors or other third parties from competing with us, or otherwise provide us with a competitive advantage. Any patents that eventually issue may be challenged, narrowed or invalidated by third parties. Consequently, we do not know whether our technology or any of our product candidates will be protectable or remain protected by valid and enforceable patent rights. Our competitors or other third parties may be able to evade our patent rights by developing new products that are similar to our product candidates, biosimilars of our product candidates, or alternative technologies or products in a non-infringing manner.

The issuance or grant of a patent is not irrefutable as to its inventorship, scope, validity or enforceability, and our patents may be challenged in the courts or patent offices in the United States and abroad. There may be prior art of which we are not aware that may affect the validity or enforceability of a patent claim. There also may be prior art of which we are aware, but which we do not believe affects the validity or enforceability of a claim, which may, nonetheless, ultimately be found to affect the validity or enforceability of a claim. We may in the future, become

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subject to a third-party pre-issuance submission of prior art or opposition, derivation, revocation, re-examination, post-grant and inter partes review, or interference proceeding and other similar proceedings challenging our patent rights or the patent rights of others in the U.S. Patent and Trademark Office, or USPTO, or other foreign patent office. An unfavorable determination in any such submission, proceeding or litigation could reduce the scope of, or invalidate, our patent rights, allow third parties to commercialize our technology or products and compete directly with us, without payment to us, or limit or extinguish our ability to manufacture or commercialize products without infringing third-party patent rights.

In addition, given the amount of time required for the development, testing and regulatory review of new product candidates, patents protecting such candidates might expire before or shortly after such candidates are commercialized. As a result, our intellectual property may not provide us with sufficient rights to exclude others from commercializing products similar or identical to ours. Moreover, some of our owned and in-licensed patents and patent applications may in the future be co-owned with third parties. If we are unable to obtain an exclusive license to any such third-party co-owners’ interest in such patents or patent applications, such co-owners may be able to license their rights to other third parties, including our competitors, and our competitors could market competing products and technology. In addition, we or our licensors may need the cooperation of any such co-owners of our owned and in-licensed patents in order to enforce such patents against third parties, and such cooperation may not be provided to us or our licensors. Any of the foregoing could have a material adverse effect on our competitive position, business, financial conditions, results of operations and prospects.

We do not own or license patents or patent applications that claim the active pharmaceutical ingredient in our ACG-701 and ACG-801 product candidates.

The active pharmaceutical ingredient in ACG-701, sodium fusidate, is an old compound that was published in the 1960s, has been approved for therapeutic use in jurisdictions outside the United States and is included in ex-US cystic fibrosis treatment guidelines. We do not own or license patents that claim sodium fusidate or its acid form, fusidic acid, as a composition of matter. We do own patents in the United States and in other jurisdictions that claim certain methods of treatment using ACG-701, and we seek additional patent claims on certain compositions. There can be no assurances that our granted patents will, or that any of our pending patent applications that grant as patents will, effectively prevent others from commercializing competitive technologies and products. There is a risk that others, including companies that make generic pharmaceuticals, may develop products that are the same as or similar to ACG-701 for the same or similar uses as us, and that our patents will not effectively prevent them from commercializing their products.

The active pharmaceutical ingredient in ACG-801 is a recombinant form of acid ceramidase. Human acid ceramidase is a naturally occurring protein and patentability of such proteins is limited under current patent law. We do not own or license patents that claim recombinant human acid ceramidase as a composition of matter. We own patents in the United States and in other jurisdictions that claim certain compositions of and methods of treatment using acid ceramidase, and we seek additional patent claims on certain compositions. There can be no assurances that our granted patents will, or that any of our pending patent applications that grant as patents will, effectively prevent others from commercializing competitive technologies and products. There is a risk that others, including companies that make biosimilar pharmaceuticals, may develop products that are similar to ACG-801 for the same or similar uses as us, and that our patents will not effectively prevent them from commercializing their products.

Licensing of intellectual property is of critical importance to our business and involves complex legal, business and scientific issues. Our rights to develop and commercialize ACG-801 are subject to the terms and conditions of our license agreement with Icahn School of Medicine at Mount Sinai, and our rights to develop and commercialize future product candidates may be subject to terms and conditions of licenses granted to us by third parties. If we fail to comply with our obligations under our current or future intellectual property license agreements or otherwise experience disruptions to our business relationships with our current or any future licensors, we could lose intellectual property rights that are important to our business and lose the ability to continue to develop and commercialize the related product.

We are and expect to continue to be reliant upon third-party licensors for certain patent and other intellectual property rights that are important or necessary to the development of our technology and product candidates. In particular, ACG-801 is dependent on our Amended and Restated License Agreement with Icahn School of Medicine at Mount Sinai (“Mt. Sinai License”). Pursuant to the Mt. Sinai License we are the exclusive licensee of patent

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rights and know-how of Mt. Sinai. The Mt. Sinai License imposes, and we expect that any future license agreement will impose, specified diligence, milestone payment, royalty, commercialization, development and other obligations on us and require us to meet development timelines, or to exercise diligent or commercially reasonable efforts to develop and commercialize licensed products, in order to maintain the licenses.

Furthermore, Mt. Sinai has, and future licensors may have, the right to terminate the license if we materially breach the agreement and fail to cure such breach within a specified period or in the event we undergo certain bankruptcy events. In spite of our best efforts, our current or any future licensors might conclude that we have materially breached our license agreements and might therefore terminate the license agreements. If the Mt. Sinai License is terminated, and we lose our intellectual property rights under the Mt. Sinai License, this may result in complete termination of our development and commercialization activities with respect to ACG-801. If the Mt. Sinai License is terminated, or if the underlying intellectual property fails to provide the intended exclusivity, our competitors or other third parties could have the freedom to seek regulatory approval of, and to market, products and technologies identical or competitive to ours and we may be required to cease our development and commercialization of certain of our product candidates and technology. In addition, we may seek to obtain additional licenses from Mr. Sinai or a future licensor and, in connection with obtaining such licenses, we may agree to amend our existing licenses in a manner that may be more favorable to the licensors, including by agreeing to terms that could enable third parties, including our competitors, to receive licenses to a portion of the intellectual property that is subject to our existing licenses and to compete with any product candidates we may develop and our technology. Any of the foregoing could have a material adverse effect on our competitive position, business, financial condition, results of operations and prospects. Disputes may arise regarding intellectual property subject to a licensing agreement, including:

●

the scope of rights granted under the license agreement and other interpretation-related issues;

●

our or our licensors’ ability to obtain, maintain and defend intellectual property and to enforce intellectual property rights against third parties;

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the extent to which our technology, product candidates and processes infringe, misappropriate or otherwise violate the intellectual property of the licensor that is not subject to the license agreement;

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the sublicensing of patent and other intellectual property rights under our license agreements;

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our diligence, development, regulatory, commercialization, financial or other obligations under the license agreement and what activities satisfy those diligence obligations;

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the inventorship and ownership of inventions and know-how resulting from the joint creation or use of intellectual property by our current or future licensors and us and our partners; and

●

the priority of invention of patented technology.

In addition, our license agreement with Mt. Sinai is, and future license agreements are likely to be, complex, and certain provisions in such agreements may be susceptible to multiple interpretations. The resolution of any contract interpretation disagreement that may arise could narrow what we believe to be the scope of our rights to the relevant intellectual property or technology, or increase what we believe to be our diligence, development, regulatory, commercialization, financial or other obligations under the relevant agreement. In addition, if disputes over intellectual property that we have licensed or any other dispute related to our license agreements prevent or impair our ability to maintain our current license agreements on commercially acceptable terms, we may be unable to successfully develop and commercialize the affected product candidates and technology. Any of the foregoing could have a material adverse effect on our business, financial condition, results of operations and prospects.

License agreements we may enter into in the future may be non-exclusive. Accordingly, third parties may also obtain non-exclusive licenses from such licensors with respect to the intellectual property licensed to us under such license agreements. Accordingly, these license agreements may not provide us with exclusive rights to use such licensed patent and other intellectual property rights, or may not provide us with exclusive rights to use such patent and other intellectual property rights in all relevant fields of use and in all territories in which we may wish to develop or commercialize our technology and any product candidates we may develop in the future.

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Moreover, some of our in-licensed patent and other intellectual property rights may in the future be subject to third party interests such as co-ownership. If we are unable to obtain an exclusive license to such third-party co-owners’ interest, in such patent and other intellectual property rights, such third-party co-owners may be able to license their rights to other third parties, including our competitors, and our competitors could market competing products and technology. We or our licensors may need the cooperation of any such co-owners of our licensed patent and other intellectual property rights in order to enforce them against third parties, and such cooperation may not be provided to us or our licensors.

Additionally, under the terms of the Mt. Sinai License we do not control the preparation, filing, prosecution, maintenance, enforcement and defense of patents and patent applications that we exclusively license. Although we have a right to have our comments considered in connection with patent prosecution and maintenance, we cannot be certain that Mt. Sinai will prepare, file, prosecute and maintain the licensed patents and patent applications in a manner that is consistent with the best interests of with our business. If Mt. Sinai fails to prosecute or maintain or loses rights to any licensed patents or applications, the rights we have licensed may be reduced or eliminated, which could adversely affect our ability to develop and commercialize ACG-801. If we enter into additional licenses in the future with Mt. Sinai or other licensors we may not control the preparation, filing, prosecution, maintenance, enforcement and defense of patents and patent applications that we license from such licensors. It is possible that our licensors’ filing, prosecution and maintenance of the licensed patents and patent applications, enforcement of patents against infringers or defense of such patents against challenges of validity or claims of enforceability may be less vigorous than if we had conducted them ourselves, and accordingly, we cannot be certain that these patents and patent applications will be prepared, filed, prosecuted, maintained, enforced and defended in a manner consistent with the best interests of our business. If our licensors fail to file, prosecute, maintain, enforce and defend such patents and patent applications, or lose rights to those patents or patent applications, the rights we have licensed may be reduced or eliminated, our right to develop and commercialize any of our technology and any product candidates we may develop that are the subject of such licensed rights could be adversely affected and we may not be able to prevent competitors or other third parties from making, using and selling competing products.

Furthermore, our owned and in-licensed patent rights may be subject to a reservation of rights by the licensor and or one or more third parties. For example, the research resulting in certain of our in-licensed patent rights and technology was funded in part by the U.S. government. Consequently, the U.S. government may have certain rights in to such patents. Certain patents that we exclusively license from Mt. Sinai are the results of research that was funded in part by the U.S. government and certain of our development activities for ACG-701 are funded in part by the U.S. government. When new technologies are developed with government funding, in order to secure ownership of patent rights related to the technologies, the recipient of such funding is required to comply with certain government regulations, including timely disclosing the inventions claimed in such patent rights to the U.S. government and timely electing title to such inventions. A failure to meet these obligations may lead to a loss of rights or the unenforceability of relevant patents or patent applications. In addition, the U.S. government may have certain rights in such patent rights, including a non-exclusive license authorizing the U.S. government to use the invention or to have others use the invention on its behalf. If the U.S. government decides to exercise these rights, it is not required to engage us as its contractor in connection with doing so. The U.S. government’s rights may also permit it to disclose the funded inventions and technology, which may include our confidential information, to third parties and to exercise march-in rights to use or allow third parties to use the technology that was developed using U.S. government funding. The U.S. government may exercise its march-in rights if it determines that action is necessary because we or our licensors failed to achieve practical application of the U.S. government-funded technology, because action is necessary to alleviate health or safety needs, to meet requirements of federal regulations, or to give preference to U.S. industry. In addition, our rights in such U.S. government-funded inventions may be subject to certain requirements to manufacture any product candidates we may develop embodying such inventions in the United States. Any of the foregoing could harm our business, financial condition, results of operations and prospects significantly.

We may not be successful in obtaining all rights necessary to develop and commercialize our current or future product candidates

We currently have exclusively licensed certain intellectual property from Mt. Sinai that is relevant for ACG-801. Our current and future product candidates may require the use of additional intellectual property rights held by third parties, and our ability to develop and commercialize such product candidates will depend, in part, on our ability to acquire, in-license or use any relevant intellectual property rights. We may be unable to secure such licenses or

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otherwise acquire or in-license intellectual property rights from third parties that we identify as necessary for any product candidates we may develop and our technology on commercially reasonable terms, or at all. Even if we are able to in-license any such necessary intellectual property, it could be on non-exclusive terms, thereby giving our competitors and other third parties access to the same intellectual property licensed to us, and the applicable licensors could require us to make substantial licensing and royalty payments. The licensing or acquisition of third-party intellectual property rights is a competitive area, and several more established companies may pursue strategies to license or acquire third-party intellectual property rights that we may consider attractive or necessary. These established companies may have a competitive advantage over us due to their size, capital resources and clinical development and commercialization capabilities. In addition, companies that perceive us to be a competitor may be unwilling to assign or license rights to us.

We sometimes collaborate with non-profit and academic institutions to accelerate our preclinical research or development under written agreements with these institutions. Typically, these institutions provide us with an option to negotiate a license to any of the institution’s rights in technology resulting from the collaboration. Regardless of such option, we may be unable to negotiate a license within the specified timeframe or under terms that are acceptable to us. If we are unable to do so, the institution may offer the intellectual property rights to third parties, potentially blocking our ability to pursue our research program and develop and commercialize our product candidates.

Third parties may own or control patents or patent applications and require us to seek licenses, which could increase our development and commercialization costs, or prevent us from developing or marketing products.

Although we have ~~many~~ issued patents and pending patent applications in the U.S. and other countries, we may not have rights under certain third-party patents or patent applications related to our compounds under development and product candidates. Third parties may own or control these patents and patent applications in the U.S. and abroad. In particular, we are aware of certain third-party U.S. patents that contain claims related to immunostimulatory polynucleotides and their use to stimulate an immune response, as well as to antisense technology. Although we do not believe any of our TLR or antisense compounds under development infringe any valid claim of these patents, we cannot be assured that the holder of such patents would not seek to assert such patents against us or, if the holder did, that the courts would not interpret the claims of such patents more broadly than we believe appropriate and determine that we are in infringement of such patents. In addition, there may be other patents and patent applications related to our current or future ~~drug~~product candidates of which we are not aware. Therefore, in some cases, in order to develop, manufacture, sell or import some of our ~~drug~~product candidates, we or our collaborators may choose to seek, or be required to seek, licenses under third-party patents issued in the U.S. and abroad or under third-party patents that might issue from U.S. and foreign patent applications. In such an event, we would be required to pay license fees or royalties or both to the licensor. If licenses are not available to us on acceptable terms, we or our collaborators may not be able to develop, manufacture, sell, or import these products, or may be delayed in doing so. Either of these results could have a material adverse effect on our business.

Competitors may infringe our patents or the patents of our licensors. There has been substantial litigation and other proceedings regarding patent and other intellectual property rights in the biotechnology industry. We may become a party to various types of patent litigation or other proceedings regarding intellectual property rights from time to time even under circumstances where we are not practicing and do not intend to practice any of the intellectual property involved in the proceedings. In addition to litigation, we may become involved in patent office proceedings, including oppositions, reexaminations, supplemental examinations and inter partes reviews involving our patents or the patents of third parties. We may initiate such proceedings or have such proceedings brought against us. An adverse determination in any such proceeding, or in litigation, could reduce the scope of, or invalidate, our patent rights, allow third parties to commercialize our technology or products and compete directly with us, without payment to us, or result in our inability to manufacture or commercialize products without infringing third-party patent rights. In addition, if the breadth or strength of protection provided by our patents and patent applications is threatened, it could dissuade companies from collaborating with us to license, develop or commercialize current or future ~~drug~~product candidates. An adverse determination in a proceeding involving a patent in our portfolio could result in the loss of protection or a narrowing in the scope of protection provided by that patent.

33Even if resolved in our favor, litigation or other proceedings relating to intellectual property claims may cause us to incur significant expenses and could distract our scientific, technical and management personnel from their normal responsibilities. In addition, there could be public announcements of the results of hearings, motions or other interim proceedings or developments and if securities analysts or investors perceive these results to be negative, it could have a substantial adverse effect on the price of our common stock. Such litigation or proceedings could

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~~patent~~substantially inincrease our ~~portfolio~~operating ~~could~~losses ~~result~~and inreduce the ~~loss~~resources ofavailable ~~protection~~for ordevelopment aactivities ~~narrowing~~or inany ~~the~~future ~~scope~~sales, ofmarketing ~~protection~~or ~~provided~~distribution ~~by that patent~~activities.

We may not have sufficient financial or other resources to adequately conduct such litigation or proceedings. The cost to us of any patent litigation or other proceeding, even if resolved in our favor, could be substantial. Some of our competitors may be able to sustain the cost of such litigation or proceedings more effectively than we can because of their substantially greater financial resources. If any patent litigation or other proceeding is resolved against us, we or our collaborators may be enjoined from developing, manufacturing, selling, or importing our ~~drugs~~products without a license from the other party and we may be held liable for significant damages. We may not be able to obtain any required license on commercially acceptable terms or at all. In a patent office proceeding, such as an opposition, reexamination, or inter partes review, our patents may be narrowed or invalidated. Uncertainties resulting from the initiation and continuation of patent litigation or other proceedings could have a material adverse effect on our ability to compete in the marketplace. Patent litigation and other proceedings may also absorb significant management time.

Our intellectual property may be infringed by a third party.

Third parties may infringe one or more of our issued patents or trademarks. We cannot predict if, when or where a third party may infringe one or more of our issued patents or trademarks. To counter infringement, we may be required to file infringement claims, which can be expensive and time-consuming. Moreover, there is no assurance that we would be successful in proving that a third party is infringing one or more of our issued patents or trademarks. Any claims we assert against perceived infringers could also provoke these parties to assert counterclaims against us, alleging that we infringe their intellectual property. In addition, in a patent infringement proceeding, a court may decide that a patent of ours is invalid or unenforceable, in whole or in part, construe the patent’s claims narrowly and/or refuse to stop the other party from using the technology at issue on the grounds that our patents do not cover the technology in question, any of which may adversely affect our business. Even if we are successful in proving in a court of law that a third party is infringing one or more of our issued patents or trademarks there can be no assurance that we would be successful in halting their infringing activities.

If we are unable to protect the confidentiality of our trade secrets, our business and competitive position would be harmed.

In addition to the protection afforded by patents, we rely on trade secret protection and confidentiality agreements to protect proprietary know-how that is not patentable or that we elect not to patent, processes for which patents are difficult to enforce and any other elements of our product candidate discovery and development processes that involve proprietary know-how, information or technology that is not covered by patents. However, trade secrets can be difficult to protect and some courts inside and outside the United States are less willing or unwilling to protect trade secrets. We seek to protect our proprietary technology and processes, in part, by entering into confidentiality agreements with our employees, consultants, scientific advisors, contractors and other parties who have access to such technology and processes. However, we may not be able to prevent the unauthorized disclosure or use of our technical know-how or other trade secrets by the parties to these agreements. Monitoring unauthorized uses and disclosures is difficult, and we do not know whether the steps we have taken to protect our proprietary technologies will be effective. If any of the collaborators, scientific advisors, employees and consultants who are parties to these agreements breach or violate the terms of any of these agreements, we may not have adequate remedies for any such breach or violation. As a result, we could lose our trade secrets and third parties could use our trade secrets to compete with any product candidates we may develop and our technology. Additionally, we cannot guarantee that we have entered into such agreements with each party that may have or has had access to our trade secrets or proprietary technology and processes.

We also seek to preserve the integrity and confidentiality of our data and trade secrets by maintaining physical security of our premises and physical and electronic security of our information technology systems; however, such systems and security measures may be breached, and we may not have adequate remedies for any breach.

In addition, our trade secrets may otherwise become known or be independently discovered by competitors or other third parties. Competitors or third parties could purchase any product candidates we may develop or our technology and attempt to replicate some or all of the competitive advantages we derive from our development efforts, willfully infringe our intellectual property rights, design around our intellectual property rights or develop their own competitive technologies that fall outside the scope of our intellectual property rights. If any of our trade

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secrets were to be lawfully obtained or independently developed by a competitor or other third party, we would have no right to prevent them, or those to whom they communicate such trade secrets, from using that technology or information to compete with us. If our trade secrets are not adequately protected so as to protect our market against competitors’ products, our business, financial condition, results of operations and prospects could be materially and adversely affected.

Changes in patent laws could increase the uncertainties and costs surrounding the prosecution of our patent applications and the enforcement or defense of our issued patents.

As is the case with other pharmaceutical companies, our success is heavily dependent on intellectual property, particularly patents. Obtaining and enforcing patents in the pharmaceutical industry involve technological and legal complexity, and obtaining and enforcing pharmaceutical patents is costly, time-consuming, and inherently uncertain. Changes in either the patent laws or interpretation of the patent laws in the U.S. and other countries could increase uncertainties and costs of prosecuting patent applications and the enforcement or defense of patents, and may diminish the value of our patents or narrow the scope of our patent protection. For example, prior to March 2013, in the United States, the first to invent the claimed invention was entitled to the patent, while outside the United States, the first to file a patent application was entitled to the patent. After March 2013, under the Leahy-Smith America Invents Act, or the America Invents Act, the United States transitioned to a first inventor to file system in which, assuming that other requirements for patentability are met, the first inventor to file a patent application will be entitled to the patent on an invention regardless of whether a third-party was the first to invent the claimed invention. The America Invents Act also includes a number of significant changes that affect the way patent applications are prosecuted and also may affect patent litigation. These include allowing third-party submission of prior art to the USPTO during patent prosecution and additional procedures to attack the validity of a patent by USPTO-administered post-grant proceedings, including post-grant review, inter partes review, and derivation proceedings. However, the America Invents Act and its implementation and any future changes to the patent law could increase the uncertainties and costs surrounding the prosecution of our patent applications and the enforcement or defense of our issued patents, all of which could have an adverse effect on our business, financial condition, results of operations, and prospects. In addition, the U.S. Supreme Court has ruled on several patent cases in recent years, either narrowing the scope of patent protection available in certain circumstances or weakening the rights of patent owners in certain situations. In addition to increasing uncertainty with regard to our ability to obtain patents in the future, this combination of events has created uncertainty with respect to the value of patents, once obtained. Depending on decisions by Congress, the federal courts, and the USPTO, the laws and regulations governing patents could change in unpredictable ways that would weaken our ability to obtain new patents or to enforce existing patents and patents we may obtain in the future. Recent patent reform legislation could increase the uncertainties and costs surrounding the prosecution of our patent applications and the enforcement or defense of our issued patents.

We may not be able to protect our intellectual property rights throughout the world.

Filing, prosecuting and defending patents on our product candidates and any future product candidates throughout the world would be prohibitively expensive, and our or our licensors’ intellectual property rights in some countries outside the U.S. can be less extensive than those in the U.S. In addition, the laws and practices of some foreign countries, particularly those relating to pharmaceuticals, do not protect intellectual property rights to the same extent as federal and state laws in the U.S. For example, novel formulations and methods of medical treatment and manufacturing processes may not be patentable in certain jurisdictions, and the requirements for patentability may differ in certain countries, particularly developing countries. Furthermore, generic drug manufacturers or other competitors may challenge the scope, validity or enforceability of our patents, requiring us to engage in complex, lengthy and costly litigation or other proceedings. Generic drug manufacturers may develop, seek approval for, and launch generic versions of our products. Many countries, including EU countries, India, Japan and China, have compulsory licensing laws under which a patent owner may be compelled under certain circumstances to grant licenses to third parties. In those countries, we may have limited remedies if patents are infringed or if we are compelled to grant a license to a third party, which could materially diminish the value of our patents. This could limit our potential revenue opportunities. Accordingly, our efforts to enforce intellectual property rights around the world may be inadequate to obtain a significant commercial advantage from our intellectual property.

We may not be able to prevent third parties from practicing our inventions in all countries outside the U.S., or from selling or importing products made using our inventions into or within the U.S. or other jurisdictions. Competitors may use our technologies in jurisdictions where we have not obtained patent protection to develop their own

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products, and may export otherwise infringing products to territories where we have patent protection, but where enforcement is not as strong as that in the U.S. These products may compete with our products in jurisdictions where we do not have any issued patents and our patent claims or other intellectual property rights may not be effective or sufficient to prevent them from competing with us in these jurisdictions.

Many companies have encountered significant problems in protecting and defending intellectual property rights in certain foreign jurisdictions. Proceedings to enforce our patent rights in foreign jurisdictions could result in substantial cost and divert our efforts and attention from other aspects of our business, could put our patents at risk of being invalidated or interpreted narrowly and our patent applications at risk of not issuing and could provoke third parties to assert claims against us. We may not prevail in any lawsuits that we initiate and the damages or other remedies awarded, if any, may not be commercially meaningful.

Patent terms may not adequately protect our competitive position on our product candidates.

Depending upon the timing, duration and specifics of development, testing and regulatory approval of our product candidates, such as ACG-701 and ACG-801, patents protecting such candidates might expire before or shortly after such candidate is commercialized. We plan to seek extension of patent terms in the United States and in other countries, if available, if we have patents that are eligible for term extension when a product candidate is approved. In the United States the Drug Price Competition and Patent Term Restoration Act of 1984, or the Hatch-Waxman Amendments, permit the term of a patent to be extended up to five years as compensation for patent term lost during the FDA regulatory review process. A patent term extension cannot extend the remaining term of a patent beyond a total of 14 years from the date of product approval, only one patent may be extended and only those claims covering the approved drug, a method for using it, which is limited to the approved indication, or a method for manufacturing it may be extended. However, we may not be granted an extension because of, for example, failing to exercise due diligence during the testing phase or regulatory review phase, failing to apply within applicable deadlines, failing to apply prior to expiration of relevant patents, or otherwise failing to satisfy applicable requirements. Moreover, the applicable time period or the scope of patent protection afforded could be less than we request. If we are unable to obtain patent term extension or term of any such extension is less than we request, our competitors may obtain approval of competing products and commercialize those products following our patent expiration, and our business, financial condition, results of operations, and prospects could be materially harmed.

Obtaining and maintaining our patent protection depends on compliance with various procedural, document submissions, fee payment and other requirements imposed by governmental patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements.

Periodic maintenance fees on any issued patent are due to be paid to the USPTO and foreign patent agencies in several stages over the lifetime of the patent. We rely on our service providers or our licensors to pay these fees. The USPTO and various foreign governmental patent agencies require compliance with a number of procedural, documentary, fee payment and other similar provisions during the patent application process. We rely on reputable law firms or our licensors for compliance with patent agency requirements. While an inadvertent lapse can in many cases be cured by payment of a late fee or by other means in accordance with the applicable rules, there are situations in which noncompliance can result in abandonment or lapse of the patent or patent application, resulting in partial or complete loss of patent rights in the relevant jurisdiction. Non-compliance events that could result in abandonment or lapse of a patent or patent application include, but are not limited to, failure to respond to official actions within prescribed time limits, non-payment of fees and failure to properly legalize and submit formal documents. If we or our licensors fail to maintain the patents and patent applications covering our product candidates, our competitive position would be adversely affected.

We may be subject to claims by third parties asserting that we have misappropriated their intellectual property, or claiming ownership of what we regard as our own intellectual property.

Some of our employees were previously employed at universities or at other biotechnology or pharmaceutical companies, including our competitors or potential competitors. Some of these employees, including each member of our senior management, executed proprietary rights, non-disclosure and non-competition agreements, or similar agreements, in connection with such previous employment. Although we try to ensure that our employees do not use the proprietary information or know-how of others in their work for us, we may be subject to claims that we or these employees have used or disclosed intellectual property, including trade secrets or other proprietary information, of any such third party. Litigation may be necessary to defend against such claims. If we fail in

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defending any such claims, in addition to paying monetary damages, we may lose valuable intellectual property rights or personnel or sustain damages. Such intellectual property rights could be awarded to a third party, and we could be required to obtain a license from such third party to commercialize our technology or products. Such a license may not be available on commercially reasonable terms or at all. Even if we are successful in defending against such claims, litigation could result in substantial costs and be a distraction to management.

We rely on government funding for certain aspects of our research and development activities and we may develop intellectual property through such activities and therefore may be subject to federal regulations such as “march-in” rights, certain reporting requirements and a preference for U.S. based companies. Compliance with such regulations may limit our exclusive rights, and limit our ability to contract with non-U.S. manufacturers.

In August 2021, we entered into a contract with Department of Defense’s Defense Threat Reduction Agency (DTRA contract) for the completion of pre-clinical and clinical development activities for ACG-701 for the treatment of melioidosis. We may generate intellectual property rights through the use of this or other U.S. government funding and are therefore subject to certain federal regulations. Also, the research that lead to certain patents and technology that we exclusively license from Mt. Sinai used U.S. government funding. As a result, the U.S. government may have certain rights to intellectual property embodied in our current or future product candidates pursuant to the Bayh-Dole Act of 1980 (Bayh-Dole Act), and implementing regulations. These U.S. government rights in certain inventions developed under a government-funded program include a non-exclusive, non-transferable, irrevocable worldwide license to use inventions for any governmental purpose. In addition, the U.S. government has the right to require us to grant exclusive, partially exclusive, or non-exclusive licenses to any of these inventions to a third party if it determines that: (i) adequate steps have not been taken to commercialize the invention; (ii) government action is necessary to meet public health or safety needs; or (iii) government action is necessary to meet requirements for public use under federal regulations (also referred to as “march-in rights”). The U.S. government also has the right to take title to these inventions if we to disclose the invention to the government and fail to file an application to register the intellectual property in the specified manner and within specified time limits. These time limits have recently been changed by regulation, and may change in the future. Intellectual property generated under a government funded program is also subject to certain reporting requirements, compliance with which may require us to expend substantial resources. In addition, the U.S. government requires that any products embodying the subject invention or produced through the use of the subject invention be manufactured substantially in the U.S. The manufacturing preference requirement can be waived if the owner of the intellectual property can show that reasonable but unsuccessful efforts have been made to grant licenses on similar terms to potential licensees that would be likely to manufacture substantially in the U.S. or that under the circumstances domestic manufacture is not commercially feasible. This preference for U.S. manufacturers may limit our ability to contract with non-U.S. product manufacturers for products covered by such intellectual property. To the extent any of our current or future intellectual property is generated through the use of U.S. government funding, the provisions of the Bayh-Dole Act may similarly apply.

Numerous factors may limit any potential competitive advantage provided by our intellectual property rights.

The degree of future protection afforded by our intellectual property rights, whether owned or in-licensed, is uncertain because intellectual property rights have limitations, and may not adequately protect our business, provide a barrier to entry against our competitors or potential competitors, or permit us to maintain our competitive advantage. Moreover, if a third party has intellectual property rights that cover the practice of our technology, we may not be able to fully exercise or extract value from our intellectual property rights. The factors that may limit any potential competitive advantage provided by our intellectual property rights include:

●

others may be able to make compounds or formulations that are similar to our product candidates but that are not covered by the claims of the patents that we own or control;

●

we may not be able to obtain and/or maintain necessary licenses on reasonable terms or at all;

●

we or any strategic partners might not have been the first to make the inventions covered by the issued patents or pending patent applications that we own or control;

●

we might not have been the first to file patent applications covering certain of our inventions;

●

others may independently develop similar or alternative technologies or duplicate any of our technologies without infringing our intellectual property rights;

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●

it is possible that our pending patent applications will not lead to issued patents;

●

issued patents that we own or control may not provide us with any competitive advantages, or may be held invalid or unenforceable as a result of legal challenges;

●

our competitors might conduct research and development activities in the U.S. and other countries that provide a safe harbor from patent infringement claims for certain research and development activities, as well as in countries where we do not have patent rights and then use the information learned from such activities to develop competitive products for sale in our major commercial markets;

●

we may not be able to maintain the confidentiality of our trade secrets or other proprietary information;

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others may independently develop similar or alternative technologies without infringing our intellectual property rights;

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we may not develop additional proprietary technologies that are patentable; and

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the patents of others may have an adverse effect on our business.

Risks Relating to Product Manufacturing, Marketing and Sales, and Reliance on Third Parties

Even if the compounds we may develop are successful in clinical trials and receive regulatory approvals, we or our collaboration partners may not be able to successfully commercialize them.

Even if the compounds were successful in clinical development and receive regulatory approvals, it may never reach or remain on the market, be successfully developed into commercial products or gain market acceptance among physicians, patients, healthcare payors or the medical community for a number of reasons including: (i) it may be found ineffective or cause harmful side effects; (ii) it may be difficult to manufacture on a scale necessary for commercialization; (iii) it may experience excessive product loss due to contamination, equipment failure, inadequate transportation or storage, improper installation or operation of equipment, vendor or operator error, natural disasters or other catastrophic events, inconsistency in yields or variability in product characteristics; (iv) it may be uneconomical to produce; (v) the timing of market introduction of the compounds we may develop and competitive products may be inopportune; (vi) political and legislative changes may make the commercialization of any product candidates we may develop in the future, more difficult; (vii) we may fail to obtain reimbursement approvals or pricing that is cost effective for patients as compared to other available forms of treatment or that covers the cost of production and other expenses; (viii) they may not compete effectively with existing or future alternatives; (ix) we may be unable to develop commercial operations and to sell marketing rights; (x) it may fail to achieve market acceptance; or (xi) we may be precluded from commercialization of a product due to proprietary rights of third parties.

Because we have limited manufacturing experience, and no manufacturing facilities or infrastructure, we are dependent on third-party manufacturers to manufacture ~~drug~~product candidates for us.

We have limited manufacturing experience and no manufacturing facilities, infrastructure or clinical or commercial scale manufacturing capabilities. In order to continue to develop our ~~drug~~product candidates, apply for regulatory approvals, and ultimately commercialize products, we need to develop, contract for or otherwise arrange for the necessary manufacturing capabilities. We currently rely upon third parties to produce material for nonclinical and clinical testing purposes and expect to continue to do so in the future. We also expect to rely upon third parties to produce materials that may be required for the commercial production of our ~~drug~~product candidates, if approved. Our current and anticipated future dependence upon others for the manufacture of our ~~drug~~product candidates may adversely affect our future profit margins and our ability to develop product candidates and commercialize any product candidates on a timely and competitive basis. We currently do not have any long-term supply contracts.

34There are a limited number of manufacturers who operate under the FDA’s cGMP regulations capable of manufacturing our product candidates. As a result, we may have difficulty finding manufacturers for our product candidates suitable for our needs. If we are unable to arrange for third-party manufacturing of our product

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~~margins~~candidates ~~and~~on ~~our~~a ~~ability~~timely tobasis, ~~develop~~or ~~drug~~on ~~candidates~~acceptable ~~and~~terms, ~~commercialize~~we ~~any~~may ~~drug~~not ~~candidates~~be onable ato ~~timely~~complete ~~and~~development ~~competitive~~of ~~basis.~~our Weproduct ~~currently~~candidates door ~~not~~market ~~have any long-term supply contracts~~them.

~~There~~Any ~~are~~contract amanufacturers ~~limited~~with ~~number~~which ofwe ~~manufacturers~~enter ~~who~~into ~~operate~~manufacturing ~~under~~arrangements will be subject to extensive regulatory requirements and ongoing periodic, unannounced inspections or remote regulatory assessments by the FDA's, or foreign equivalent, and corresponding state and foreign agencies or their designees to ensure compliance with cGMP requirements and other governmental regulations ~~capable~~and ofcorresponding ~~manufacturing~~foreign standards. Any failure by our ~~drug~~third-party ~~candidates.~~manufacturers Asto acomply ~~result~~with such requirements, weregulations ~~may~~or ~~have~~standards ~~difficulty~~could ~~finding~~lead ~~manufacturers~~to ~~for~~a ~~our~~delay ~~drug~~in ~~candidates~~the ~~suitable~~conduct ~~for~~of our ~~needs.~~clinical Iftrials, weor ~~are~~a ~~unable~~delay in, or failure to ~~arrange~~obtain, ~~for~~regulatory ~~third-party~~approval ~~manufacturing~~of any of our ~~drug~~product candidates. onSuch afailure ~~timely~~could ~~basis~~also result in sanctions being imposed, including fines, injunctions, civil penalties, delays, suspension or onwithdrawal ~~acceptable~~of ~~terms~~approvals, weproduct ~~may~~seizures ~~not~~or berecalls, ~~able~~imposition toof ~~complete~~operating ~~development~~restrictions, total or partial suspension of ~~our~~production ~~drug~~or ~~candidates~~distribution, or ~~market~~criminal ~~them~~prosecution.

~~Any~~Additionally, contract manufacturers ~~with~~may ~~which~~not webe ~~enter~~able ~~into~~to ~~manufacturing~~manufacture ~~arrangements~~our ~~will~~product becandidates ~~subject~~at a cost or in quantities necessary to ~~extensive~~make ~~regulatory~~them ~~requirements~~commercially ~~and~~viable. ~~ongoing~~Furthermore, ~~periodic~~changes in the manufacturing process or procedure, ~~unannounced~~including ~~inspections~~a bychange in the ~~FDA,~~location where the drug substance or ~~foreign~~product ~~equivalent,~~is ~~and~~manufactured ~~corresponding~~or ~~state~~a ~~and~~change ~~foreign~~of ~~agencies~~a orthird-party ~~their~~manufacturer, ~~designees~~may torequire ~~ensure~~prior ~~compliance~~FDA review and approval in accordance with ~~cGMP~~the ~~requirements~~FDA’s cGMP and ~~other~~marketing ~~governmental~~application regulations and ~~corresponding~~may ~~foreign~~require ~~standards.~~that ~~Any~~we ~~failure~~conduct bybridging ~~our~~studies ~~third-party~~to ~~manufacturers~~demonstrate tothat ~~comply~~the ~~with~~product ~~such~~produced ~~requirements,~~under ~~regulations~~prior processes and procedures or ~~standards~~by ~~could~~prior ~~lead~~manufacturers is comparable to afuture ~~delay~~product. inContract ~~the~~manufacturers ~~conduct~~may ofalso ~~our~~be ~~clinical~~subject ~~trials,~~to orcomparable aforeign ~~delay~~requirements. ~~in,~~This orreview ~~failure~~may tobe ~~obtain,~~costly ~~regulatory~~and ~~approval~~time-consuming ofand ~~any~~could delay or prevent the launch of ~~our~~a ~~drug~~product ~~candidates~~candidate. ~~Such~~The ~~failure~~FDA ~~could~~or similar foreign regulatory agencies at any time may also ~~result~~implement innew ~~sanctions~~standards ~~being~~or ~~imposed,~~change ~~including~~their ~~fines,~~interpretation ~~injunctions,~~and ~~civil~~enforcement ~~penalties,~~of ~~delays~~existing standards for manufacture, ~~suspension~~packaging or ~~withdrawal~~testing of ~~approvals,~~products. ~~product~~If ~~seizures~~we or ~~recalls,~~our ~~imposition~~contract ofmanufacturers ~~operating~~are ~~restrictions~~unable to comply, ~~total~~we or ~~partial~~they ~~suspension~~may ofbe ~~production~~subject orto ~~distribution~~regulatory action, orcivil ~~criminal~~actions ~~prosecution~~or penalties.

Additionally, contract manufacturers may not be able to manufacture our drug candidates at a cost or in quantities necessary to make them commercially viable. Furthermore, changes in the manufacturing process or procedure, including a change in the location where the drug substance or drug product is manufactured or a change of a third-party manufacturer, may require prior FDA review and approval in accordance with the FDA's cGMP and NDA regulations. Contract manufacturers may also be subject to comparable foreign requirements. This review may be costly and time-consuming and could delay or prevent the launch of a drug candidate. The FDA or similar foreign regulatory agencies at any time may also implement new standards or change their interpretation and enforcement of existing standards for manufacture, packaging or testing of products. If we or our contract manufacturers are unable to comply, we or they may be subject to regulatory action, civil actions or penalties.

We have no experience selling, marketing or distributing potential products and no internal capability to do so.

Advancing compounds through Phase 3 development and regulatory approval will require us to begin commercialization preparation activities and incur related expenses. These activities will include, among other things, the development of an in-house marketing organization and sales force, a market access and payor reimbursement strategy and a distribution function, which will require significant capital expenditures, management resources and time. If we are unable to adequately prepare the market for the potential future commercialization of compounds, we may not be able to generate product revenue once marketing authorization is obtained.

If we are unable or decide not to establish internal sales, marketing and distribution capabilities, we will pursue collaborative arrangements regarding the sales and marketing of our products; however, there can be no assurance that we will be able to establish or maintain such collaborative arrangements on commercially reasonable terms, or if we are able to do so, that they will have effective sales forces. Any revenue we receive will depend upon the efforts of such third parties, which may not be successful. We may have little or no control over the marketing and sales efforts of such third parties and our revenue from product sales may be lower than if we had commercialized our product candidates ourselves. We also face competition in our search for third parties to assist us with the sales and marketing efforts of our product candidates. Finally, regardless of whether we contract out our sales and marketing functions, we will be responsible for the marketing and promotion of our products and may be held responsible should any products be improperly marketed or promoted.

We do not have the ability to independently conduct the clinical or preclinical trials required to obtain regulatory approval for our ~~drug~~product candidates. We depend on independent investigators, contract research organizations (“CROs”), and other third-party service providers in the conduct of the trials of our ~~drug~~product candidates and expect to continue to do so. We expect to contract with CROs, investigators, and other third parties for future clinical and preclinical trials but there is no guarantee that we will be able to at all or on favorable terms. We rely heavily on these parties for successful execution of our trials, but do not control many aspects of their activities. We are responsible for ensuring that each of our trials is conducted in accordance with the applicable regulations and protocols for the trial. Third parties may not complete activities on schedule, or at all, or may not conduct our trials in accordance with regulatory requirements or our protocols. If these third parties fail to carry out their obligations,

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~~requirements or our protocols. If these third parties fail to carry out their obligations,~~ we may need to enter into new arrangements with alternative third parties. This could be difficult, costly or impossible. There can be no assurance that we will be able to sustain our relationships with these third-party entities, secure additional partnerships, or identify alternative sites or CROs in the event of any termination of our existing agreements or should we need to establish alternative arrangements. Moreover, if third parties fail to carry out their obligations and comply with the applicable regulatory requirements, our preclinical or clinical trials may need to be extended, delayed, terminated or repeated, and we may not be able to obtain regulatory approval in a timely fashion, or at all, for the applicable ~~drug~~product candidate, or to commercialize such ~~drug~~product candidate being tested in such trials. If we seek to conduct any of these activities ourselves in the future, we will need to recruit appropriately trained personnel and add to our research, clinical, quality and corporate infrastructure. Moreover, if we need to replace any third parties, we may not be able to do so in a timely fashion or on commercially reasonable terms.

The commercial success of any product candidates that we may develop will depend upon the degree of market acceptance by physicians, patients, third-party payors, and others in the medical community.

~~The~~Any ~~commercial~~products ~~success~~that ofwe ~~any~~ultimately ~~drug~~bring ~~candidates~~to ~~that~~the wemarket, ~~may~~if ~~develop~~they ~~will~~receive ~~depend~~marketing ~~upon~~approval, ~~the~~may ~~degree~~not ofgain market acceptance by physicians, patients, third-party payors, ~~and~~or others in the medical community. If our products do not achieve an adequate level of acceptance, we may not generate product revenue and we may not become profitable. The degree of market acceptance of our products, if approved for commercial sale, will depend on a number of factors, including: (i) the prevalence and severity of any side effects; (ii) the efficacy and potential advantages over alternative treatments; (iii) the ability to offer our product candidates for sale at competitive prices; (iv) relative convenience and ease of administration; (v) the willingness of the target patient population to try new therapies and of physicians to prescribe these therapies; (vi) the strength of marketing and distribution support and the timing of market introduction of competitive products; and (vii) publicity concerning our products or competing products and treatments.

~~Any~~Even ~~products~~if ~~that~~a wepotential ~~ultimately~~product ~~bring~~displays toa ~~the~~favorable ~~market,~~efficacy ifand ~~they~~safety ~~receive marketing approval~~profile, ~~may not gain~~ market acceptance byof ~~physicians,~~the ~~patients,~~product ~~third-party~~will ~~payors~~not orbe ~~others~~known inuntil ~~the~~after ~~medical~~it ~~community~~is launched. ~~For~~Our ~~example,~~efforts ~~current~~to ~~cancer~~educate ~~treatments~~patients, ~~including chemotherapy and radiation therapy are well-established in~~ the medical community, and ~~doctors~~third-party ~~may~~payors ~~continue~~about ~~to rely on these treatments. If~~ our ~~products~~product docandidates ~~not achieve an adequate level of acceptance, we~~ may ~~not~~require ~~generate~~significant ~~product~~resources ~~revenue~~ and we may ~~not~~never ~~become~~be ~~profitable~~successful. ~~The~~Such ~~degree~~efforts of market acceptance of our products, if approved for commercial sale, will depend on a number of factors, including: (i) the prevalence and severity of any side effects; (ii) the efficacy and potential advantages over alternative treatments; (iii) the ability to ~~offer~~educate ~~our drug candidates for sale at competitive prices; (iv) relative convenience and ease of administration; (v)~~ the ~~willingness~~marketplace ofmay ~~the~~require ~~target~~more ~~patient~~resources ~~population~~than toare ~~try~~required ~~new~~by ~~therapies~~conventional ~~and~~methods ofused ~~physicians~~by ~~to prescribe these therapies; (vi) the strength of marketing and distribution support and the timing of market introduction of competitive products; and (vii) publicity concerning~~ our ~~products or competing products and treatments~~competitors.

Even if a potential product displays a favorable efficacy and safety profile, market acceptance of the product will not be known until after it is launched. Our efforts to educate patients, the medical community, and third-party payors about our drug candidates may require significant resources and may never be successful. Such efforts to educate the marketplace may require more resources than are required by conventional methods used by our competitors.

If we are unable to obtain adequate reimbursement from third-party payors for any products that we may develop or acceptable prices for those products, our revenues and prospects for profitability will suffer.

Most patients rely on Medicare, Medicaid, private health insurers, and other third-party payors to pay for their medical needs, including any drugs we may market. If third-party payors do not provide adequate coverage or reimbursement for any products that we may develop, our revenues and prospects for profitability will suffer.

Third-party payors are challenging the prices charged for medical products and services, and many third-party payors limit reimbursement for newly-approved products. These third-party payors may base their coverage and reimbursement on the coverage and reimbursement rate paid by carriers for Medicare beneficiaries. Furthermore, many such payors are investigating or implementing methods for reducing health care costs, such as the establishment of capitated or prospective payment systems. Cost containment pressures have led to an increased emphasis on the use of cost-effective products by health care providers, which could limit the price we might establish for products that we or our current or future collaborators may develop or sell, which would result in lower product revenues or royalties payable to us. In particular, third-party payors may limit the indications for which they will reimburse patients who use any products that we may develop or impose other patient access or utilization controls or limitations.

We face a risk of product liability claims and may not be able to obtain insurance.

Our business exposes us to the risk of product liability claims that is inherent in the manufacturing, testing, and marketing of prescription ~~drugs~~products. We face a risk of product liability exposure related to the testing of our ~~drug~~product candidates in clinical trials and will face an even greater risk if we commercially sell any products. Regardless of merit or eventual outcome, liability claims and product recalls may result in: (i) decreased demand for our ~~drug~~product candidates and products; (ii) damage to our reputation; (iii) regulatory investigations that could require costly recalls or product modifications; (iv) withdrawal of clinical trial participants; (v) costs to defend related ligation; (vi) substantial monetary awards to clinical trial participants or patients; (vii) loss of revenue; (viii) the diversion of

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management'’s attention away from managing our business; and (ix) the inability to commercialize any products that we may develop.

Although we have product liability and clinical trial liability insurance that we believe is adequate, this insurance is subject to deductibles and coverage limitations. We may not be able to obtain or maintain adequate protection against potential liabilities. If we are unable to obtain insurance at acceptable cost or otherwise protect against potential product liability claims, we will be exposed to significant liabilities, which may materially and adversely affect our business and financial position. These liabilities could also prevent or interfere with our commercialization efforts.

Risks Relating to Ownership of Our Preferred and Common Stock

Provisions in our restated certificate of incorporation (“Charter”) and second amended and restated bylaws (“Bylaws”) and Delaware law, may prevent a change in control that stockholders may consider desirable.

Section 203 of the General Corporation Law of the State of Delaware (the “DGCL”) and our Charter and Bylaws contain provisions that might enable our management to resist a takeover of our company or discourage a third party from attempting to take over our company. These provisions include: (i) a classified board of directors; (ii) limitations on the removal of directors; (iii) limitations on stockholder proposals at meetings of stockholders; (iv) the inability of stockholders to act by written consent or to call special meetings; and (v) the ability of our board of directors to designate the terms of and issue new series of preferred stock without stockholder approval. These provisions could: (i) have the effect of delay, defer, or prevent a change in control of us or a change in our management that stockholders may consider favorable or beneficial or (ii) discourage proxy contests and make it more difficult for stockholders to elect directors and take other corporate actions.

The Company’s Bylaws provide, to the fullest extent permitted by law, that the Court of Chancery of the State of Delaware will be the exclusive forum for certain legal actions between the Company and its stockholders, which could increase costs to bring a claim, discourage claims or limit the ability of the Company’s stockholders to bring a claim in a judicial forum viewed by the stockholders as more favorable for disputes with the Company or the Company’s directors, officers or other employees.

Our Bylaws provide to the fullest extent permitted by law that unless the Company consents in writing to the selection of an alternative forum, the Court of Chancery of the State of Delaware will be the sole and exclusive forum for any (i) derivative action or proceeding brought on behalf of the Company, (ii) any action asserting a claim of breach of a fiduciary duty owed by any director, officer, other employee or stockholder of the Company to the Company or its stockholders, (iii) any action arising pursuant to any provision of the DGCL, the Company’s Charter or the Bylaws, (iv) any action to interpret, apply, enforce or determine the validity of the Charter or the Bylaws, or (v) any action asserting a claim governed by the internal affairs doctrine. The choice of forum provision may increase costs to bring a claim, discourage claims or limit a stockholder’s ability to bring a claim in a judicial forum that it finds favorable for disputes with the Company or its directors, officers or other employees, which may discourage such lawsuits against the Company or its directors, officers and other employees. Alternatively, if a court were to find the choice of forum provision contained in the Company’s Bylaws to be inapplicable or unenforceable in an action, the Company may incur additional costs associated with resolving such action in other jurisdictions. The exclusive forum provision in our Bylaws would not apply to claims brought under the Exchange Act or the Securities Act, or any other claim for which the federal courts have exclusive jurisdiction. Additionally, such provision will not relieve us of our duty to comply with the federal securities laws and the rules and regulations thereunder, and stockholders will not be deemed to have waived our compliance with these laws, rules and regulations.

~~Risks~~Six ~~Relating~~stockholders beneficially own approximately 69% of our outstanding common stock in the aggregate. If these significant stockholders choose to ~~Ownership~~act together, they could exert substantial influence over our business, and the interests of ~~Our~~these ~~Common~~stockholders ~~Stock~~may conflict with those of other stockholders.

~~Provisions~~Following inthe Series Z Preferred Stock Conversion (as defined below) there is a concentration of ownership of our ~~certificate~~outstanding common stock because approximately 69% of ~~incorporation~~our ~~and~~outstanding common stock is beneficially owned by~~-laws~~ ~~and~~six ~~Delaware~~stockholders. ~~law~~As of March 1, ~~may~~2023: ~~prevent~~(i) entities affiliated with Pillar Invest Corporation (the “Pillar Investment Entities”) beneficially owned approximately 11.2% of our outstanding common stock; (ii) NovaQuest beneficially owned approximately 9.8% of our outstanding common stock; (iii) Dr. Atul Chopra, a ~~change~~founder inand ~~control~~a ~~that~~member ~~stockholders~~of ~~may~~Legacy ~~consider~~Aceragen’s ~~desirable~~board of directors, beneficially owned approximately 17.5% of our outstanding common stock; and (iv) Mr. John Taylor, Mr. Daniel Salain, and Mr. Andrew Jordan (“Executives”) individually beneficially owned

Section 203 of the General Corporation Law of the State of Delaware (the “DGCL”) and our certificate of incorporation and by-laws contain provisions that might enable our management to resist a takeover of our company or discourage a third party from attempting to take over our company. These provisions include: (i) a classified board of directors; (ii) limitations on the removal of directors; (iii) limitations on stockholder proposals at meetings of stockholders; (iv) the inability of stockholders to act by written consent or to call special meetings; and (v) the ability of our board of directors to designate the terms of and issue new series of preferred stock without stockholder approval. These provisions could: (i) have the effect of delay, defer or prevent a change in control of us or a change in our management that stockholders may consider favorable or beneficial or (ii) discourage proxy contests and make it more difficult for stockholders to elect directors and take other corporate actions. 55

The Company’s amended and restated bylaws (“Bylaws”) provide, to the fullest extent permitted by law, that the Court of Chancery of the State of Delaware will be the exclusive forum for certain legal actions between the Company and its stockholders, which could increase costs to bring a claim, discourage claims or limit the ability of the Company’s stockholders to bring a claim in a judicial forum viewed by the stockholders as more favorable for disputes with the Company or the Company’s directors, officers or other employees.

Our Bylaws provide to the fullest extent permitted by law that unless the Company consents in writing to the selection of an alternative forum, the Court of Chancery of the State of Delaware will be the sole and exclusive forum for any (i) derivative action or proceeding brought on behalf of the Company, (ii) any action asserting a claim of breach of a fiduciary duty owed by any director, officer, other employee or stockholder of the Company to the Company or its stockholders, (iii) any action arising pursuant to any provision of the DGCL, the Company’s Certificate of Incorporation or the Bylaws, (iv) any action to interpret, apply, enforce or determine the validity of the Certificate of Incorporation or the Bylaws, or (v) any action asserting a claim governed by the internal affairs doctrine. The choice of forum provision may increase costs to bring a claim, discourage claims or limit a stockholder’s ability to bring a claim in a judicial forum that it finds favorable for disputes with the Company or its directors, officers or other employees, which may discourage such lawsuits against the Company or its directors, officers and other employees. Alternatively, if a court were to find the choice of forum provision contained in the Company’s Bylaws to be inapplicable or unenforceable in an action, the Company may incur additional costs associated with resolving such action in other jurisdictions. The exclusive forum provision in our Bylaws would not apply to claims brought under the Exchange Act or the Securities Act, or any other claim for which the federal courts have exclusive jurisdiction. Additionally, such provision will not relieve us of our duty to comply with the federal securities laws and the rules and regulations thereunder, and stockholders will not be deemed to have waived our compliance with these laws, rules and regulations.

Approximately 24% of our outstanding common stock is held (28% beneficially owned) by three stockholders. If these significant stockholders choose to act together, they could exert substantial influence over our business, and the interests of these stockholders may conflict with those of other stockholders.

There is a concentration of ownership of our outstanding common stock because approximately 24% of our outstanding common stock is owned by three stockholders. As of December 31, 2021: (i) Baker Bros. Advisors LP, and certain of its affiliated funds (collectively, “Baker Brothers”) beneficially owned 3.9% of our outstanding common stock, which excludes all convertible securities as a result of certain beneficial ownership limitations as discussed in

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~~Note~~17.5%, 1517.5% ofand ~~this Form 10-K; (ii) entities affiliated with Pillar Invest Corporation (the “Pillar Investment Entities”) beneficially owned 19~~5.94% of our ~~outstanding~~ common stock;, ~~and~~respectively (~~iii)~~“Pillar ~~Castellina~~Investment ~~Ventures Ltd. (“Castellina~~Entities” and, together with ~~Baker~~NovaQuest, ~~Brothers~~Dr. Chopra, and ~~Pillar Investment Entities~~Executives, the “Significant Securityholders”) ~~beneficially owned 4~~.2% ~~of our outstanding common stock.~~ If any of our Significant Securityholders acted together, they could be able to exert substantial influence over our business. Additionally, the interests of the Significant Securityholders may be different from or conflict with the interests of our other stockholders. This concentration of voting power with the Significant Securityholders could delay, defer or prevent a change of control, entrench our management and the board of directors or delay or prevent a merger, consolidation, takeover or other business combination involving us on terms that other stockholders may desire. In addition, conflicts of interest could arise in the future between us, on the one hand, and either of our Significant Securityholders on the other hand, concerning potential competitive business activities, business opportunities, the issuance of additional securities and other matters.

The issuance or sale of shares of our common stock could depress the trading price of our common stock.

If (i) we issue additional shares of our common stock or rights to acquire shares of our common stock in other future transactions, (ii) any of our existing stockholders sells a substantial amount of our common stock, or (iii) the market perceives that such issuances or sales may occur, then the trading price of our common stock may significantly decrease. In addition, our issuance of additional shares of common stock will dilute the ownership interests of our existing common stockholders.

Because we do not intend to pay dividends on our common stock, investor returns will be limited to any increase in the value of our stock.

We have never declared or paid any cash dividends on our common stock. We currently intend to retain all available funds and any future earnings to support our operations and finance the growth and development of our business and do not anticipate declaring or paying any cash dividends on our common stock for the foreseeable future.

Our Series X Preferred Stock have rights, preferences and privileges that are not held by, and are preferential to, the rights of our common stock, which could adversely affect our liquidity and financial condition, and may result in the interests of the holders of our Series X Preferred Stock differing from those of the holders of common stock.

The Series X Preferred Stock ranks senior to our common stock with respect to dividend rights on the distribution of assets on any voluntary or involuntary liquidation, dissolution, or winding up of our affairs. The holders of our Series X Preferred Stock are entitled to receive distributions on shares of Series X Preferred Stock as set forth in (a) the Purchase Agreement, and (b) the PRV Agreement (any such distributions under the Purchase Agreement and the PRV Agreement, the “Preferred Distributions”), prior and in preference to any declaration or payment of any other distribution or dividend (other than dividends on shares of Common Stock payable in shares of Common Stock).

In addition, holders of Series X Preferred Stock are entitled to receive a distribution in the event either (i) Aceragen receives any proceeds from the sale of a PRV granted by the FDA in connection with regulatory approval of a ACG-801 (recombinant human acid ceramidase) or ACG-701 (sodium fusidate) product or (ii) Aceragen does not receive such a PRV or does not complete a PRV sale within a certain period after receipt. The holders of Series X Preferred Stock are also entitled to net sales distributions based upon future net sales of the ACG-801 and ACG-701.

The holders of our Series X Preferred Stock also have the right, subject to certain exceptions, to require us to repurchase all or any portion of the Series X Preferred Stock upon certain change of control events or Product Divestiture (as defined in the PRV Agreement) of a ACG-801 product without NovaQuests’ consent with respect to certain change of control events or Product Divestiture (as defined in the PRV Agreement), and Aceragen may, and NovaQuest may require us to, redeem the Series X Preferred Stock at a price equal to the fair market value thereof or make certain distributions to the holders of Series X Preferred Stock.

These dividend, distribution, and share repurchase obligations could impact our liquidity and reduce the amount of cash flows available for general corporate purposes. Our obligations to the holders of the Series X Preferred Stock could also limit our ability to obtain additional financing or increase our borrowing costs, which could have an adverse effect on our financial condition. These preferential rights could also result in divergent interests between the holders of shares of Series X Preferred Stock and holders of our common stock.

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Item 1B. Unresolved Staff Comments.

None.

Item 2. Properties.

We lease approximately 11,000 square feet of office space located in Exton, Pennsylvania. The lease expires on May 31, 2025. We may terminate the lease at any point as long as we remain a member of the landlord’s group and require a space with more square footage. We have specified rights to sublease this facility.

In connection with the Aceragen Acquisition, we acquired an operating lease for an office in Basel, Switzerland which expired on March 31, 2023.

Item 3. Legal Proceedings.

None.

Item 4. Mine Safety Disclosures.

Not applicable.

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PART II.

Item 5.

Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities.

Market Information

Our common stock is listed under the symbol “~~IDRA~~ACGN” on the Nasdaq.

Holders of Record

As of March 431, ~~2022~~2023, we had ~~approximately~~27 51 common stockholders of record registered on our books, excluding shares held through banks and brokers.

Dividends

We have never declared or paid cash dividends on our common stock, and we do not expect to pay any cash dividends on our common stock in the foreseeable future. The declaration and payment of dividends in the future, of which there can be no assurance, will be determined by our ~~Board~~board of ~~Directors~~directors in light of conditions then existing, including earnings, financial condition, capital requirements, and other factors.

Recent Sales of Unregistered Securities

WePursuant ~~did~~to ~~not~~the ~~issue~~Merger ~~any~~Agreement ~~unregistered~~(as ~~equity~~defined ~~securities~~below), ~~during~~on September 28, 2022, the ~~year~~Company ~~ended~~issued ~~December~~451,608 31shares of Common Stock and 80,656 ~~2021~~Series Z Preferred Stock to the common stockholders of Legacy Aceragen and 5 shares of Series X Preferred Stock to NovaQuest. Under the terms of the Merger Agreement, all options to purchase or acquire shares of Legacy Aceragen held by Continuing Employees (as defined in the Merger Agreement) and all issued and outstanding warrants to purchase shares of Legacy Aceragen’s capital stock were assumed by the Company and converted into options or warrants to purchase shares of Common Stock and Series Z Preferred Stock on the same terms and conditions as applied to such options and warrants immediately prior to the Merger (but with such changes as the Company in good faith determined were necessary to reflect such assumption and conversion).

~~Issuer~~Such ~~Purchases~~issuances were exempt from registration under the Securities Act, in reliance on Section 4(a)(2) thereof, and Regulation D promulgated thereunder. Each of ~~Equity~~the common stockholders of Legacy Aceragen and NovaQuest represented that it was an “accredited investor,” as defined in Regulation D, and is acquiring the securities for investment only and not with a view towards, or for resale in connection with, the public sale or distribution thereof. The securities have not been registered under the Securities Act and such securities may not be offered or sold in the United States absent registration or an exemption from registration under the Securities Act and any applicable state securities laws.

WeAs ~~did~~provided for the Series Z Certificate of Designation, following stockholder approval of the Conversion Proposal (as defined below), each share of Series Z Preferred Stock then outstanding automatically converted into a number of shares of Common Stock equal to the Conversion Ratio (as defined in the Series Z Certificate of Designation). Such conversion was subject to certain limitations, including that the Company was not ~~repurchase~~permitted to effect any conversion of ~~our~~shares ~~equity~~of ~~securities~~Series ~~during~~Z ~~the~~Preferred ~~year~~Stock ~~ended~~into ~~December~~shares 31of Common Stock if, ~~2021~~as a result of such conversion, such holder, together with its affiliates, would beneficially own more 19.99% of the total number of shares of Common Stock issued and outstanding immediately after giving effect to such conversion.

Issuer Purchases of Equity Securities

We did not repurchase any of our equity securities during the year ended December 31, 2022.

Item 6. Reserved.

Item 6.

[Reserved.]

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Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations.

The following discussion and analysis of our financial condition and results of operations should be read intogether ~~conjunction~~ with our consolidated audited financial statements and ~~the~~accompanying ~~related~~ notes appearing elsewhere in this Form 10-K. In addition to historical information, this discussion and analysis includes forward-looking statements that are subject to risks and uncertainties, including those discussed in the section titled “Risk Factors,” set forth in Part I, Item 1A of this Form 10-K, that could cause actual results to differ materially from historical results or anticipated results.

Prior to January 17, 2023, we were known as Idera Pharmaceuticals, Inc. On September 28, 2022, we completed the Aceragen Acquisition, whereby we acquired all of the outstanding equity interests in Legacy Aceragen. In connection with the Aceragen Acquisition and related transactions, we changed our name to Aceragen, Inc. Unless the context indicates otherwise, references in this section to the “Company,” “Aceragen,” “Idera,” “we,” “us,” “our” and similar terms refer to Aceragen, Inc. (f/k/a Idera Pharmaceuticals, Inc.) and our consolidated subsidiaries. References to “Legacy Aceragen” refer to Aceragen, Inc. prior to the consummation of the Aceragen Acquisition.

Overview

We are a clinical-stage biopharmaceutical company with a business strategy focused on the clinical development, and ultimately the commercialization, of ~~drug~~product candidates for rare disease indications characterized by small, well-defined patient populations with serious unmet medical needs. Our current focus is to ~~identify~~develop and ~~potentially~~optimize ~~acquire~~commercial ~~rights~~value toof ~~novel~~ACG-701 ~~development~~(patented ~~and~~formulation ~~commercial~~of ~~stage~~sodium ~~rare~~fusidate) ~~disease~~and ~~programs~~ACG-801 ~~through~~(recombinant ~~new~~human ~~business~~acid ~~development~~ceramidase ~~opportunities,~~(rhAC)) ~~including~~for ~~additional~~appropriate ~~strategic alternatives~~patients. We have in the past and may in the future explore collaborative alliances to support development and commercialization of any of our drug candidates. We may also seek to identify and potentially acquire rights to novel development and commercial-stage rare disease programs through new business development opportunities, including additional strategic alternatives.

Until ~~May~~December 2021, we were developing tilsotolimod, via intratumoral injection, for the treatment of ~~anti-PD1~~solid ~~refractory~~tumors ~~metastatic melanoma~~ in combination with ~~ipilimumab~~nivolumab, an anti-~~CTLA4~~PD1 antibody marketed as ~~Yervoy~~Opdivo® by Bristol Myers Squibb Company (“BMS”), inand/or aipilimumab, an anti-CTLA4 antibody marketed as Yervoy® by BMS. Due to Phase 3 ~~registration~~results ~~trial.~~in ~~During~~anti-PD-1 ~~the~~refractory ~~first~~advanced ~~quarter~~melanoma of(ILLUMINATE-301), reported in March 2021, wewhich ~~announced~~showed ~~that~~the study failed to meet its primary endpoint, as well as a decision in December 2021 to discontinue enrollment in ILLUMINATE-~~301~~206, ~~the~~our ~~Company’s~~Phase ~~pivotal~~2 ~~registration~~study ~~trial~~in solid tumors, Company-sponsored development of tilsotolimod in ~~combination~~oncology was discontinued. Although clinical trials with ~~ipilimumab~~tilsotolimod ~~versus~~have ~~ipilimumab~~not ~~alone~~yet intranslated ~~patients~~into ~~with~~a ~~anti-PD-1~~new ~~refractory~~treatment ~~advanced~~alternative ~~melanoma~~for patients, ~~did~~we ~~not~~believe ~~meet~~that ~~its~~data ~~primary~~supporting ~~endpoint~~tilsotolimod’s mechanism of ~~Objective~~action ~~Response~~and ~~Rate~~encouraging ~~(“ORR”). Based~~safety onprofile ~~subsequent~~from ~~evaluation~~across the array of ~~the~~pre-clinical ~~full~~and ~~data~~clinical ~~set~~work to date, together with its intellectual property protection, are noteworthy. As a result, in ~~May~~December 2021, we announced that we would ~~not~~consider an out-licensing arrangement so that tilsotolimod’s full potential might continue ~~the~~to ~~trial~~be explored on behalf of patients who did not respond to ~~its~~traditional ~~Overall~~immunotherapy, ~~Survival~~together ~~(“OS”)~~with ~~primary~~other ~~endpoint~~alternatives.

~~Through~~In ~~December~~September ~~2021~~2022, we ~~were~~acquired ~~also~~Legacy ~~evaluating~~Aceragen, ~~intratumoral~~a ~~tilsotolimod~~privately-held inbiotechnology ~~combination~~company ~~with~~addressing ~~nivolumab~~rare, anorphan ~~anti-PD1~~pulmonary, ~~antibody~~and ~~marketed~~rheumatic asdiseases ~~Opdivo®~~for bywhich ~~BMS,~~there ~~and~~are ~~ipilimumab~~limited ~~for~~or no available treatments. Legacy Aceragen owned or controlled the ~~treatment~~intellectual property related to ACG-701 (patented formulation of ~~multiple~~sodium ~~solid~~fusidate) ~~tumors~~and inACG-801 a(recombinant ~~multicohort~~human ~~Phase~~acid 2ceramidase ~~trial~~(rhAC)). InFollowing ~~December~~the ~~2021~~Aceragen Acquisition, weour ~~announced~~business ~~that~~strategy ~~preliminary~~is ~~data~~to ~~from~~develop ~~the~~and ~~second~~optimize 10commercial ~~patients~~value ~~dosed~~of inACG-701 ~~the~~and ~~safety~~ACG-801 ~~cohort~~for ofappropriate ~~ILLUMINATE~~patients. Accordingly, we are developing ACG-~~206~~701 ~~showed~~to atreat ~~safety~~cystic ~~profile~~fibrosis ~~consistent~~(“CF”) ~~with~~pulmonary ~~the~~exacerbations ~~first~~(“PEx”) 10and ~~patients~~melioidosis, ina ~~ILLUMINATE~~severe, life-~~206~~threatening infection, and ~~with~~ACG-801 ~~prior~~to ~~studies.~~treat Nopatients ~~further~~suffering ~~enrollment~~from a genetic mutation in ~~ILLUMINATE-206~~the isASAH ~~planned~~1 atgene, ~~this~~also ~~time~~known as Farber disease.

~~While~~Recent our clinical trials with tilsotolimod have not yet translated into a new treatment alternative for patients, data supporting tilsotolimod’s mechanism of action and encouraging safety profile from across the array of pre-clinical and clinical work to date, together with its intellectual property protection, are noteworthy. As a result, in December 2021, we announced that we will consider an out-licensing arrangement so that its full potential may continue to be explored on behalf of patients who do not respond to traditional immunotherapy. Developments

~~Historically,~~Business substantially all our revenues have been from collaboration and license agreements, although we did not generate any such revenue in 2021, and we have received no revenues from the sale of commercial products. Going forward, we plan to continue to invest in research and development. Accordingly, we anticipate a significant portion of our operating expenses will continue to be related to research and development in 2022 and beyond. See additional information below under the headings “Results of Operations” regarding research and development expenses to date and “Financial Condition, Liquidity and Capital Resources” regarding our future funding requirements. Acquisition

~~Recent~~On ~~Events~~the ~~and~~Acquisition ~~Updates~~Date, in accordance with the Merger Agreement, we acquired 100% of the outstanding security interests of Legacy Aceragen in a “stock-for-stock” transaction, whereby all Legacy Aceragen outstanding equity interests were exchanged for a combination of shares of our common stock, shares of Series Z Preferred Stock,

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and shares of the newly-designated Series X Preferred Stock. Under the terms of the Merger Agreement, Legacy Aceragen stockholders received (i) 451,608 shares of our common stock (inclusive of shares subject to repurchase), (ii) 80,656 shares of Series Z Preferred Stock (inclusive of shares subject to repurchase), and (iii) five shares of Series X Preferred Stock. In addition, all outstanding options and warrants to purchase Legacy Aceragen common stock were converted into stock options and warrants to purchase shares of our common stock and Series Z Preferred Stock on terms substantially identical to those in effect prior to the Aceragen Acquisition, except for adjustments to the underlying number of shares and the exercise price based on the Merger Agreement exchange ratio.

Pursuant to the Merger Agreement, we held the Special Meeting at which our stockholders approved, among other matters: the Conversion Proposal and (ii) the Reverse Stock Split Proposal. The transactions following the approval of the Merger Agreement Meeting Proposals are discussed in further detail below.

Reverse Stock Split and Corporate Name Change

Following approval of the Reverse Stock Proposal, our board of directors approved a one-for-seventeen (1:17) reverse split of our issued and outstanding shares of common stock (the “Reverse Stock Split”). On January 17, 2023, we filed with the Secretary of State of the State of Delaware a Certificate of Amendment to our Restated Certificate of Incorporation (the “Certificate of Amendment”) to effect the Reverse Stock Split and the Company Name Change (as defined below). The Reverse Stock Split became effective as of 4:59 p.m. Eastern Time on January 17, 2023. As a result of the effectiveness of the Reverse Stock Split, every 17 shares of our issued and outstanding common stock were automatically combined, converted, and changed into one share of our common stock, without any change in the number of authorized shares or the par value per share. In addition, a proportionate adjustment was made to the per share exercise price and the number of shares issuable upon the exercise of all outstanding stock options, restricted stock units, and warrants to purchase shares of common stock and the number of shares reserved for issuance pursuant to our equity incentive compensation plans.

Also on January 17, 2023, and in connection with the previously-announced merger between Legacy Aceragen and us, our board of directors approved a change in name from “Idera Pharmaceuticals, Inc.” to “Aceragen, Inc.” (the “Company Name Change”), as reflected in the Certificate of Amendment. The Company Name Change became effective as of 4:59 p.m. Eastern Time on January 17, 2023.

Conversion of Series Z Non-Voting Convertible Preferred Stock

Following approval of the Conversion Proposal, effective January 17, 2023 at 5:00 p.m. Eastern Time, all 80,656 outstanding shares of our Series Z Preferred Stock were automatically converted into 4,744,467 shares of our common stock pursuant to the terms of the Series Z Preferred Stock (the “Series Z Preferred Stock Conversion”).

Nasdaq Compliance

As previously disclosed by Current Reports on Form 8-K filed with the SEC on December 1, 2021, May 27, 2022, and November 23, 2022, the Company received deficiency letters from the Nasdaq Listing Qualifications Department (the “Staff”) of The Nasdaq Stock Market, LLC (“Nasdaq”), notifying the Company that it was not in compliance with Nasdaq Listing Rule 5550(a)(2), which requires the Company to maintain a minimum closing bid price of $1.00 per share for continued listing on The Nasdaq Capital Market. In response to the Staff’s letter, in November 2022, the Company timely requested a hearing before a Nasdaq Hearing Panel (the “Panel”), which was held on January 5, 2023. By decision dated January 10, 2023, the Panel granted the Company an extension until January 20, 2023, to complete the Special Meeting and attendant transactions, including the Reverse Stock Split, and thereby evidence compliance with all applicable criteria for continued listing on The Nasdaq Capital Market, including the initial listing $4.00 bid price requirement applicable due to the Series Z Preferred Stock Conversion, which constituted a “change of control” (as that term is defined by Nasdaq), requiring the Company to meet all criteria for initial listing on The Nasdaq Capital Market at that time. Following the Reverse Stock Split, which was effective upon the market open on Wednesday, January 18, 2023, the Company was in compliance with all applicable Nasdaq listing standards and Nasdaq issued an approval letter confirming Aceragen’s listing.

Reduction-in-Force

In April 2021, following the announcement that the ILLUMINATE-301 trial did not meet its primary endpoint of ORR, we implemented a reduction-in-force which affected approximately 50% of our workforce through September 30, 2021, primarily in the area of research and development. The decision was made in order to align our workforce with our needs in light of the outcome of ILLUMINATE-301’s ORR endpoint, our ongoing ILLUMINATE development program, and other business development activities focused on identifying new portfolio opportunities. 60

In connection with these actions, we incurred and paid termination costs for the reduction in workforce, which includes severance, benefits and related costs, of approximately $1.3 million during the year ended December 31, 2021.

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Cost-reduction Plan Implementation

~~Nasdaq~~On ~~Compliance~~April 13, 2023, the Board approved certain cost-cutting measures with a view to preserving capital to support our continuing operations. As part of this plan, we have commenced the furlough of 12 employees, representing approximately 46% of our workforce. Additionally, certain of our employees and executive officers will defer portions of their respective base salaries in amounts that exceed $200,000, with such deferrals having a retroactive effective date of April 5, 2023. We will continue to review operations for other opportunities to reduce costs and pursue financing opportunities. For more information, please see Item 9B of this Form 10-K.

As previously disclosed, on November 26, 2021, we received a deficiency letter (the “Nasdaq Letter”) from the Nasdaq Listing Qualifications Department, notifying us that the Company is not in compliance with Nasdaq Listing Rule 5550(a)(2), which requires the Company to maintain a minimum bid price of at least $1 per share for continued listing on The Nasdaq Capital Market (the “Minimum Bid Requirement”). The Company’s failure to comply with the Minimum Bid Requirement was based on the Company’s common stock per share price being below the $1 threshold for a period of 30 consecutive business days. In accordance with Nasdaq Listing Rule 5810(c)(3)(A) (the “Compliance Period Rule”), the Company has been provided an initial period of 180 calendar days (the “Compliance Date”), to regain compliance with the Minimum Bid Requirement. If, at any time before the Compliance Date, the bid price for the Company’s common stock closes at $1.00 or more per share for a minimum of 10 consecutive business days, as required under Nasdaq requirements, the Staff will provide written notification to the Company that it complies with the Minimum Bid Requirement, unless the Staff exercises its discretion to extend this 10-day period pursuant to Nasdaq Listing Rule 5810(c)(3)(H).

Results of Operations

The following is a discussion of results of operations for fiscal ~~2021~~2022 compared to fiscal ~~2020~~2021. For a discussion of results of operations for fiscal ~~2020~~2021 compared to fiscal ~~2019~~2020, please refer to Item 7—Management’s Discussion and Analysis of Financial Condition and Results of Operations in the Company’s Annual Report on Form 10-K for the year ended December 31, ~~2020~~2021, filed with the SEC on March 131, ~~2021~~2022.

Years ended December 31, 2022 and 2021

~~Years ended December 31, 2021 and 2020~~Overview

During each of the years ended December 31, 2022 and 2021 our loss from operations totaled $27.8 million.

Prior to the Aceragen Acquisition, all our revenues had been from collaboration and license agreements, although we did not generate any such revenue in 2021 and have received no revenues from the sale of commercial products. Additionally, research and development expenses historically comprised the majority of our total operating expenses until we terminated our ILLUMINATE development program in December 2021, resulting in general and administrative expenses, acquisition-related costs and restructuring costs to comprise the majority of our total operating expenses for the year ended December 31, 2022. Following the Aceragen Acquisition, we expect to generate revenues from certain U.S. government contracts and, assuming adequate funding, for research and development expenses to comprise the majority of our total operating expenses, including in 2023 and beyond.

The following summarizes the components of loss from operations, as discussed further below:



	Year Ended December 31,
($ in thousands)	2022		2021		$ Change		% Change
Government contracts revenue	$	4,862	$	—	$	4,862	100%
Operating expenses:
Research and development		12,188		16,375		(4,187)	(26)%
General and administrative		12,213		9,976		2,237	22%
Acquisition-related costs		4,566		—		4,566	100%
Restructuring and other costs		3,713		1,322		2,391	181%
Total operating expenses		32,680		27,673		5,007	18%
Loss from operations	$	(27,818)	$	(27,673)	$	(145)	1%

Government Contracts Revenue

In connection with the Aceragen Acquisition, we assumed certain U.S. government contracts. Revenues from reimbursable contracts are recognized as costs are incurred, generally based on allowable direct costs incurred during the period, plus allocable overheads together with any recognizable earned fee.

Government contracts revenue for the year ended December 31, 2022 totaled $4.9 million of which $4.6 million related to a contract assumed in the Aceragen Acquisition funded by the Department of Defense’s DTRA to develop ACG-701 as a potential medical countermeasure against the pathogen that causes melioidosis, B. Pseudomallei. This contract is expected to further fund clinical and regulatory development of ACG-701 up to an additional $30.0 million. Our other government contracts are not currently significant to our operations and not expected to

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be material in the future. We did not generate any government contracts revenue for the fiscal year ended December 31, 2021.

Research and Development Expenses

For each of our research and development programs, we incur both direct and indirect expenses. We track direct research and development expenses by program, which may include internal personnel costs and other third-party costs such as contract research, consulting and clinical trial and manufacturing costs. We do not allocate indirect research and development expenses, which may include regulatory, laboratory (equipment and supplies), personnel, facility, and other overhead costs (including depreciation and amortization), to specific programs.

During the fiscal year ended December 31, 2022, our overall research and development expenses declined by 26%, as compared to 2021, primarily due to decreases in external development costs associated with tilsotolimod (IMO-2125) and other drug development expenses. These decreases are primarily attributed to (i) lower costs incurred with contract research organizations supporting our ILLUMINATE development program as a result of our decision to discontinue development of tilsotolimod, (ii) lower costs incurred with drug manufacturing activities, and (iii) lower expenses incurred in connection with the Scriptr Agreement.

We also expect that our research and development costs will increase in future periods as we proceed with the development of ACG-701 and ACG-801, however, such development will depend on our ability to raise capital, as discussed further below under the caption “Liquidity and Capital Resources.”

In the table below, research and development expenses are set forth in the following categories: (i) ACG-701 (REPRIEVE Study (CF PEx)) external development expense, (ii) ACG-701 (TERRA Study (Melioidosis)) external development expense, (iii) ACG-801 (Farber disease) external development expense, (iv) Tilsotolimod (IMO-2125), and (v) other drug development expenses.



	Year Ended December 31,
($ in thousands)	2022		2021		$ Change		% Change
ACG-701 development expense
REPRIEVE Study (CF PEx)	$	673	$	—	$	673	100%
TERRA Study (Melioidosis)		3,128		—		3,128	100%
Subtotal		3,801		—		3,801	100%
ACG-801 development expense (Farber disease)		1,684		—		1,684	100%
Tilsotolimod (IMO-2125) development expense		3,234		9,247		(6,013)	(65)%
Other drug development expense		3,469		7,128		(3,659)	(51)%
Total research and development expenses	$	12,188	$	16,375		(4,187)	(26)%

During the fiscal year ended December 31, 2021, our overall research and development expenses declined by 34% as compared to 2020, primarily due to decreases in external development costs associated with tilsotolimod (IMOACG-~~2125).~~701 ~~This~~Development decrease is primarily related to: (i) costs incurred with contract research organizations during the year ended December 31, 2021 to support our ILLUMINATE-301 trial, which was discontinued by the Company in the second quarter of 2021; and (ii) lower costs incurred with drug manufacturing activities. The decrease of the research and development expenses is offset by the costs associated with ILLUMINATE-206 and the Scriptr Agreement.Expenses

~~Tilsotolimod~~These ~~(IMO~~expenses are comprised of expenses we incurred in connection with the development of ACG-~~2125)~~701 for CF PEx and Melioidosis, including our ongoing REPRIEVE and TERRA Studies, and include external development expenses asincurred ~~well~~with ascontract ~~expenses~~research ~~related~~organizations, tocontract ~~the~~development ~~Scriptr~~and ~~Agreement~~manufacturing ~~(as~~organizations, ~~more~~subcontractors, ~~fully~~and ~~described~~other ~~under~~third-party ~~the~~vendors. ~~heading~~In ~~“Collaborative~~addition, ~~Alliances”~~these ~~above~~expenses ininclude ~~Item~~salary ~~1.)~~costs, ~~will~~but ~~continue~~exclude toother beinternal apersonnel-related ~~significant~~costs, ~~portion~~such ofas ~~our~~stock-based ~~total~~compensation ~~research~~ and ~~development~~other ~~spending~~benefits, inand ~~2022~~overhead expenses.

41We acquired the ACG-701 development program in connection with the Aceragen Acquisition and began to incur program-related expenses following the Acquisition Date. We expect to continue to incur significant expenses related to the development of ACG-701 in 2023 and beyond, subject to adequate financing.

ACG-801 Development Expenses

These expenses are comprised of expenses we incurred in connection with the development of ACG-801 for Farber disease, including our anticipated ADVANCE Study, which we expect to initiate clinical activities for in the first half of 2024, subject to funding. Such expenses include external development expenses incurred with contract research organizations, contract development and manufacturing organizations, subcontractors, and other third-

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Inparty ~~the~~vendors. ~~table~~In ~~below~~addition, ~~research~~these ~~and~~expenses ~~development~~include ~~expenses~~salary ~~are~~costs, ~~set~~but ~~forth~~exclude inother ~~the~~internal ~~following~~personnel-related ~~categories:~~costs, ~~Tilsotolimod~~such ~~(IMO~~as stock-~~2125)~~based compensation and other ~~drug~~benefits, ~~development~~and overhead expenses.


	Year Ended December 31,				% Change
($ in thousands)	2021		2020		2021 vs 2020
Tilsotolimod (IMO-2125) external development expense	$	9,247	$	16,707	(45)%
Other drug development expense		7,128		8,065	(12)%
Total research and development expenses	$	16,375	$	24,772	(34)%

~~Tilsotolimod~~We ~~(IMO~~acquired the ACG-~~2125)~~801 ~~External~~development ~~Development~~program ~~Expenses~~in connection with the Aceragen Acquisition and began to incur program-related expenses following the Acquisition Date. We expect to continue to incur significant expenses related to the development of ACG-801 in 2023 and beyond, subject to adequate financing.

~~These~~Tilsotolimod ~~expenses include external expenses we have incurred in connection with the development of tilsotolimod as part of our immuno~~(IMO-~~oncology~~2125) ~~program.~~Development These external expenses include payments to independent contractors and vendors for drug development activities conducted after the initiation of tilsotolimod clinical development in immuno-oncology, but exclude internal costs such as payroll and overhead expenses. Expenses

WeThese ~~commenced~~expenses ~~clinical~~include external expenses we have incurred in connection with the development of tilsotolimod, as part of our ~~immuno-oncology~~ILLUMINATE development program, inwhich ~~July~~we ~~2015,~~commenced ~~and~~clinical ~~from~~development of in July 2015 ~~through~~but ~~December~~was ~~31,~~discontinued ~~2021,~~in weDecember ~~incurred approximately $91~~2021.1 ~~million~~These ~~in tilsotolimod~~ external ~~development~~ expenses, ~~including~~include ~~costs~~payments ~~associated~~to ~~with~~independent ~~the~~contractors ~~preparation~~and vendors for ~~and~~drug ~~conduct~~development ofactivities ~~ILLUMINATE-204,~~conducted ~~ILLUMINATE-101,~~after ~~ILLUMINATE-301, ILLUMINATE-206, and~~ the ~~manufacture~~initiation of ~~additional~~tilsotolimod ~~drug~~clinical ~~substance~~development ~~for~~in ~~use~~immuno-oncology inbut ~~our~~exclude ~~clinical~~internal ~~trials~~costs such as salaries and ~~additional~~other ~~nonclinical~~personnel-related ~~studies~~costs and overhead expenses.

Following the announcement that all Company-sponsored development of tilsotolimod was discontinued in December 2021, all significant study-related activities concluded. As such, we do not anticipate incurring significant additional expenses related to tilsotolimod in 2023 and beyond.

Other Drug Development Expenses

These expenses include internal costs, such as ~~payroll~~salary and other personnel-related costs and overhead expenses, ~~associated~~not ~~with~~allocated ~~all~~to ~~our~~a ~~clinical~~specific development ~~programs~~program. In addition, these expenses include costs incurred related to our research collaboration with Scriptr and other external expenses, such as payments to contract vendors, associated with compounds that were previously being developed but are not currently being developed, other than tilsotolimod. WeFor the years ended December 31, 2022 and 2021, we incurred $23.5 million and 7.1 million, respectively, of other drug development expenses. ~~within~~The decrease in other drug development expenses ~~related~~during 2022, as compared to ~~our~~2021, ~~research~~was ~~collaboration~~primarily ~~with~~due to (i) lower expenses incurred pursuant to the Scriptr inAgreement of $1.6 million, and (ii) lower personnel-related costs resulting from the April 2021 reduction-in-force (discussed below), which primarily impacted research and development personnel as we were focused in 2022 on strategic alternatives, which resulted in the Aceragen Acquisition.

General and Administrative Expenses

General and administrative expenses consist primarily of payroll, stock-based compensation expense, consulting fees, and professional legal fees associated with our patent applications and maintenance, our corporate regulatory filing requirements, our corporate legal matters, and our business development initiatives. ~~For the years ended December 31, 2021 and 2020, general and administrative expenses totaled $10.0 million and $11.9 million, respectively.~~

~~General~~For the year ended December 31, 2022, general and administrative expenses ~~decreased~~totaled ~~by approximately~~ $112.92 million, ora ~~16.3~~22% increase, inas compared to $10.0 million for the year ended December 31, 2021. The increase in general and administrative expenses during 2022, as compared to ~~2020~~2021, was primarily due to ~~lower~~increases ~~salary,~~in: ~~bonus,~~(i) ~~stock~~personnel ~~compensation~~costs ~~expense, employee-~~related ~~expense~~to ~~related~~the toacquisition ~~terminated~~of ~~employees~~Legacy asAceragen ~~part~~employees of(including ~~our~~salaries, ~~reduction~~stock-~~in-force~~based compensation and bonuses), (ii) professional and ~~commercial~~consulting ~~research~~fees ~~support~~(including accounting and legal costs), ~~partially~~and ~~offset~~(iii) byother ~~increased~~overhead ~~consulting expenses~~costs.

~~Restructuring~~Acquisition-related Costs

In April 2021, following the announcement that the ILLUMINATE-301 trial did not meet its primary endpoint of ORR, we implemented a reduction-in-force which affected approximately 50% of the workforce through December 31, 2021, primarily in the area of research and development. The decision was made in order to align our workforce with its needs in light of the outcome of ILLUMINATE-301’s ORR endpoint, its ongoing ILLUMINATE development program and other business development activities focused on identifying new portfolio opportunities.

~~Restructuring~~Acquisition-related costs ~~for~~consist ~~the~~of ~~year~~charges ~~ended~~for ~~December 31~~transaction, ~~2021~~integration-related ~~totaled~~professional ~~approximately~~fees, ~~$1.3~~retention ~~million~~bonuses and isother ~~comprised~~incremental ~~of termination~~ costs ~~including~~directly ~~severance, benefits and~~ related ~~costs. No~~to ~~such~~these ~~costs were incurred during the year ended December 31, 2020~~activities.

42Acquisition-related costs for the year ended December 31, 2022 was $4.6 million. All acquisition-related costs related to the Aceragen Acquisition and primarily consisted of legal and transaction related fees, and retention bonuses to certain employees. No such costs were incurred during 2021.

Restructuring and Other Costs

In April 2021, following the announcement that the ILLUMINATE-301 trial did not meet its primary endpoint of ORR, we implemented a reduction-in-force, which affected approximately 50% of our workforce through December 31,

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~~Interest~~2021, ~~Income~~primarily in the area of research and development. The decision was made in order to align our workforce with its needs in light of the outcome of ILLUMINATE-301’s ORR endpoint, its ongoing ILLUMINATE development program, and other business development activities focused on identifying new portfolio opportunities. In September 2022, in connection with the Aceragen Acquisition, we restructured our operations and implemented a reduction-in-force which affected approximately 54% of our pre-Aceragen Acquisition workforce.

~~Interest~~For ~~income for~~ the ~~years~~year ended December 31, ~~2021~~2022, restructuring and ~~2020~~other costs totaled $03.17 million, ~~and~~a 185% increase, compared to $01.23 million for the year ended December 31, ~~respectively~~2021. The ~~decrease~~increase in ~~2021~~restructuring costs during 2022, as compared to ~~2020~~2021, was primarily due to ~~lower~~increases ~~interest~~in ~~rates~~severance and ~~decrease~~related inbenefits ~~average~~resulting ~~short~~from the composition of the workforce effected from the reduction-~~term~~in-force ~~investment~~implemented ~~and~~in ~~cash~~September ~~balances~~2022, ~~and~~which ~~yields~~included several executives, in connection with the Aceragen Acquisition.

~~Amounts~~Interest ~~may~~Income ~~fluctuate from period to period due to changes in average investment balances~~(Expense), ~~including commercial paper and money market funds classified as cash equivalents, and composition of investments.~~net

~~Warrant~~Interest ~~Revaluation~~income, ~~Gain~~net orof ~~Loss~~interest expense (which included non-cash charges for accretion of discounts on the Acquisition Obligation of approximately $0.1 million), for the year ended December 31, 2022 totaled $0.2 million. Interest income, net of interest expense, for the year ended December 31, 2021 was not material. The increase in 2022, as compared to 2021, was primarily due to higher interest rates.

~~During~~Amounts ~~the~~may ~~years~~fluctuate ~~ended~~from ~~December~~period ~~31,~~to ~~2021~~period ~~and~~due ~~2020,~~to wechanges ~~recorded~~in aaverage ~~non-cash~~investment ~~warrant~~balances, ~~revaluation~~money ~~gain~~market ~~(loss)~~funds ofclassified ~~approximately $7.0~~as ~~million~~cash equivalents, and ~~$(3.7)~~composition ~~million,~~of ~~respectively~~investments.

~~The~~Warrant ~~non-cash~~Revaluation gain for the fiscal year ended December 31, 2021 relates to the derecognition of the warrant liability in the first quarter of 2021 due to the termination of such liability-classified warrants that were issued in connection with our December 2019 Private Placement. Gain

~~The~~During ~~non-cash loss for~~ the ~~fiscal~~years ~~year~~ ended December 31, ~~2020~~2022 ~~relate to the revaluation of our liability-classified warrants issued in connection with the December 2019 Private Placement. Due to the nature of~~ and ~~inputs in the model used to assess the fair value of our outstanding warrants~~2021, itwe isrecorded ~~not abnormal to experience significant fluctuations during each remeasurement period. These fluctuations may be due to~~ a ~~variety~~non-cash ofwarrant ~~factors,~~revaluation ~~including~~gain ~~changes in our stock price and changes in estimated stock price volatility over the remaining life~~ of ~~the warrants~~approximately $0.4 ~~Warrant~~million ~~revaluation~~and ~~loss for 2020 was driven primarily by an increase in our stock price during each period~~$7.0 ~~More specifically~~million, ~~the significant warrant revaluation loss for the 2020 period was primarily due to the approximate 102% increase in our stock price during the period January 1, 2020 to December 31, 2020~~respectively.

The non-cash ~~warrant~~gain ~~revaluation~~for ~~gain~~the ~~(loss)~~fiscal ~~mentioned~~year ~~above~~ended ~~are~~December ~~fully~~31, ~~described~~2022 related to the change in ~~Note~~fair 7value of our liability classified warrants assumed in connection with the ~~Notes~~Aceragen Acquisition in September 2022. Due to ~~Financial~~the ~~Statements~~nature ~~appearing~~of ~~elsewhere~~and inputs in ~~this~~the ~~Form~~model ~~10-K~~used to assess the fair value of our outstanding warrants, it is not abnormal to experience significant fluctuations during each remeasurement period. These fluctuations may be due to a variety of factors, including changes in our stock price and changes in estimated stock price volatility over the remaining life of the warrants. Warrant revaluation loss for 2022 was driven primarily by a decrease in our stock price during the period.

The non-cash gain for the fiscal year ended December 31, 2021 related to the derecognition of the warrant liability in the first quarter of 2021 due to the termination of such liability-classified warrants that were issued pursuant to the December 2019 Securities Purchase Agreement as more fully described in Note 9 of the notes to condensed consolidated financial statements appearing elsewhere in this Form 10-K .

Series X Preferred Stock Liability Loss

During the year ended December 31, 2022, we recorded a non-cash Series X Preferred Stock liability loss of approximately $2.4 million. No such gain or loss was recorded during the year ended December 31, 2021.

The non-cash loss for the year ended December 31, 2022 related to the change in fair value of our liability-classified Series X Preferred Stock, which was issued in connection with the Aceragen Acquisition in September 2022.

Future Tranche Right Revaluation Gain or Loss

During the ~~years~~year ended December 31, 2021 ~~and 2020~~, we recorded a non-cash future tranche right revaluation gain ~~(loss)~~ of approximately $118.8 million. ~~and~~No ~~$(72.4)~~such ~~million~~gain or loss was recorded during the year ended December 31, ~~respectively~~2022.

The non-cash gain for the ~~fiscal~~ year ended December 31, 2021 ~~relates~~related to the derecognition of the future tranche right liability induring the first quarter of 2021 ~~due~~associated with the future tranche rights issued pursuant to the ~~termination~~December 2019 Securities Purchase Agreement, as more fully described in Note 9 of the ~~future~~notes ~~tranche~~to ~~rights~~condensed ~~that~~consolidated ~~were~~financial ~~issued~~statements appearing elsewhere in ~~connection~~this ~~with~~Form ~~our~~10-K, ~~December~~due ~~2019~~to ~~Private~~the ~~Placement~~termination of the future tranche rights.

The non-cash loss for the fiscal year ended December 31, 2020 relates to the revaluation of our liability-classified tranche rights liability issued in connection with the December 2019 Private Placement. Due to the nature of and inputs in the model used to assess the fair value of the future tranche rights, it is not abnormal to experience significant fluctuations during each remeasurement period. These fluctuations may be due to a variety of factors, including changes in our stock price and changes in estimated stock price volatility over the remaining estimated lives of the future tranche rights. Changes in the fair value of the future tranche right liability and resulting future tranche right revaluation loss for 2020 was driven primarily by an increase in our stock price during the periods. More specifically, the significant future tranche right revaluation loss for the 2020 period was primarily due to the approximate 102% increase in our stock price during the period January 1, 2020 to December 31, 2020.64

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Foreign Currency Exchange and Other Gain (Loss), net

~~Both~~During ~~non-cash~~each ~~future~~of ~~tranche~~the ~~right~~years ~~revaluation~~ended ~~gain~~December ~~(loss)~~31, ~~for~~2022 ~~the~~and ~~respective~~2021, ~~periods~~we ~~are~~recorded ~~fully~~a ~~described~~net inforeign ~~Note~~currency 7exchange and other loss of ~~the~~less ~~Notes~~than $0.1 million. Such gains and losses, net, are not material to ~~Financial~~our ~~Statements~~business ~~appearing~~and ~~elsewhere~~not expected to be material in ~~this~~the ~~Form~~foreseeable ~~10-K~~future.

Income Tax Benefit

During the year ended December 31, 2022, we recorded $6.3 million in non-cash income tax benefit related to our evaluation of the realizability of our deferred tax assets that we determined that the valuation allowance should be decreased in consideration of positive and negative evidence bearing upon our ability to realize certain of our deferred tax assets.

There was no income tax benefit or expense recorded during the year ended December 31, 2021.

Net Income or Loss to Common Stockholders

As a result of the factors discussed above, ~~our~~we ~~net~~incurred ~~income~~a (net loss) ~~was~~of $9823.14 million ~~and $(112.7) million~~ for the ~~years~~year ended December 31, ~~2021 and 2020~~2022, ~~respectively.~~compared ~~See~~to ~~Note~~net 16income of ~~the~~$98.1 ~~Notes~~million tofor ~~Financial~~the ~~Statements~~year ~~appearing~~ended ~~elsewhere~~December 31, ~~in this Annual Report on Form 10-K for additional details~~2021.

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Basic net loss applicable to common stockholders for the year ended December 31, 2022 was $23.4 million, as compared to basic net income applicable to common stockholders for the year ended December 31, 2021 of $96.9 million. Excluding the non-cash warrant revaluation gain of $7.0 million and future tranche right revaluation gain of $118.8 million for the year ended December 31, 2021, basic net loss applicable to common stockholders was $28.8 million.

Diluted net loss applicable to common stockholders for the year ended December 31, 2022 was $23.4 million, as compared to diluted net loss applicable to common stockholders for the year ended December 31, 2021 of $28.8 million.

Net Operating Loss Carryforwards

WeAs ~~have~~of ~~completed~~December 31, ~~several~~2022, ~~financings~~the ~~since~~Company ~~the~~had ~~effective~~cumulative ~~date~~federal, ofvarious ~~the~~state, ~~Tax~~and ~~Reform~~Switzerland ~~Act~~net operating loss carryforwards (“NOLs”) of ~~1986~~approximately $355.8 million, ~~which~~$362.7 asmillion, ofand ~~December 31~~$0.9 million, ~~2021~~respectively, ~~have~~available ~~resulted~~to inreduce ~~ownership~~federal ~~changes~~and ~~that~~state ~~will~~taxable ~~significantly~~income, ~~limit~~respectively. ~~our~~As ~~ability~~a toresult ~~utilize~~of ~~our~~the Tax Cuts and Jobs Act of 2017, federal net operating ~~loss~~losses ~~carryforwards~~incurred ~~(“NOLs”)~~for ~~and~~taxable ~~tax~~years ~~credit~~beginning ~~carryforwards.~~after InJanuary ~~December 2017~~1, we2018 ~~completed~~have aan ~~study~~unlimited ~~which~~carryforward ~~determined~~period, ~~that~~but ~~ownership~~can ~~changes~~only ~~had~~be ~~occurred.~~utilized ~~The~~to ~~federal~~offset ~~and~~80% ~~state~~of ~~net~~taxable ~~operating~~income ~~loss~~in ~~and~~future ~~tax~~taxable ~~credit~~periods. ~~carryforwards~~Of ~~and~~the ~~related~~$355.8 ~~deferred~~million ~~tax~~of ~~assets~~federal ~~discussed~~NOLs, ~~below~~$158.4 ~~and~~million ~~included~~have inan ~~Note~~unlimited 13carryforward toand the ~~financial~~remaining ~~statements~~NOLs ~~appearing~~are ~~elsewhere~~subject into ~~this~~expiration ~~Form~~through ~~10-K~~2037. ~~have~~In ~~been~~addition, ~~adjusted~~at toDecember 31, ~~reflect~~2022, the ~~limitations~~Company ~~that~~had ~~resulted~~cumulative ~~from~~federal ~~this~~and ~~study~~state tax credit carryforwards of $28.3 ~~The~~million ~~Company~~and ~~continues~~$1.9 tomillion, ~~monitor~~respectively. ~~equity~~The ~~activity~~federal credits expire through 2042 and ~~potential~~the ~~ownership~~state ~~changes~~credits expire through 2033.

AsSections of382 ~~December 31,~~and ~~2021,~~383 weof ~~had~~the ~~cumulative~~Internal ~~federal~~Revenue ~~and~~Code ~~state NOLs~~ of ~~approximately~~1986, ~~$327.5~~as ~~million~~amended, ~~and~~prescribe ~~$322.0~~limitations ~~million~~on ~~available~~the toamount ~~reduce~~of ~~federal~~NOLs and ~~state~~tax ~~taxable~~credit ~~income,~~carryforwards ~~respectively.~~that Asmay abe ~~result~~utilized ofin ~~the~~any ~~Tax~~one ~~Cuts~~year. ~~and~~Under ~~Jobs~~Internal ~~Act~~Revenue ofCode ~~2017~~Section 382, ~~federal~~if ~~net~~a ~~operating~~corporation ~~losses~~undergoes ~~incurred~~an ~~for~~“ownership ~~taxable~~change,” ~~years~~the ~~beginning~~corporation’s ~~after~~ability ~~January~~to 1,use ~~2018~~its ~~have~~pre-change anNOL ~~unlimited~~carryforwards ~~carryforward~~and ~~period,~~other ~~but~~pre-change ~~can~~tax ~~only~~attributes ~~be utilized~~ to offset ~~80%~~its ofpost-change ~~taxable~~ income inmay ~~future~~be ~~taxable periods~~limited. OfIn December 2017, the ~~$327.5~~Company ~~million~~completed ofa ~~federal~~study ~~NOLs,~~which ~~$130~~determined that ownership changes had occurred.1 ~~million~~The ownership changes have anand ~~unlimited~~will ~~carryforward~~continue ~~and~~to subject the ~~remaining~~Company’s ~~NOLs~~pre-ownership ~~are~~change ~~still~~NOL ~~subject~~carryforwards to ~~expiration~~an ~~through~~annual ~~2037.~~limitation, ~~State~~which ~~NOLs~~will ~~are~~significantly ~~still~~restrict ~~subject~~the Company’s ability to ~~expiration~~use ~~according~~them to ~~the~~offset ~~laws~~taxable ofincome ~~each~~in ~~respective~~periods ~~jurisdiction~~following the ownership change. The ~~Company~~federal ~~files~~and state ~~tax~~net ~~returns~~operating inloss ~~Massachusetts~~and tax credit carryforwards and ~~Pennsylvania~~related ~~whereby~~deferred ~~both~~tax ~~jurisdictions~~assets ~~impose~~discussed aabove ~~20-year~~and ~~carryforward~~included ~~period.~~in ~~All~~Note ~~$322.0~~16 ~~million~~to ofthe ~~state~~consolidated ~~NOLs~~financial ~~expire~~statements ~~through~~appearing ~~2041,~~elsewhere ~~with~~in ~~the~~this ~~first~~Form ~~year~~10-K ofhave ~~expiration~~been ~~being~~adjusted ~~2032~~to ~~for~~reflect ~~$23.4~~the ~~million~~limitations ofthat ~~Massachusetts~~resulted ~~NOLs~~from this study. InAs ~~addition,~~no atstudy ~~December 31,~~has ~~2021,~~been ~~the~~completed ~~Company~~subsequent ~~had~~to ~~cumulative~~2017, ~~federal~~additional ~~and~~ownership ~~state~~change ~~tax~~limitations ~~credit~~may ~~carryforwards~~result offrom ~~$26.7~~ownership ~~million~~changes ~~and~~that ~~$1.9~~have ~~million~~occurred, ~~respectively,~~or ~~available~~may tooccur ~~reduce~~in ~~federal~~the ~~and~~future. ~~state~~In ~~income~~conjunction ~~taxes,~~with ~~respectively,~~the ~~which~~Aceragen ~~expire~~Acquisition, ~~through~~the ~~2041~~Company ~~and~~acquired ~~2033,~~Legacy ~~respectively,~~Aceragen’s ~~for~~ federal, ~~and~~various state ~~purposes~~, ~~other~~and ~~than~~Switzerland ~~those~~NOL’s ~~that~~of ~~have~~$8.1 million, an$19.1 ~~unlimited~~million, ~~carryforward~~and ~~period~~$0.8 million, respectively.

4465

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Financial Condition, Liquidity and Capital Resources

Financial Condition

As of December 31, ~~2021~~2022, we had an accumulated deficit of $~~735~~758.58 million. ToAs of the date of this Form 10-K, substantially all our revenues have been from collaboration and license agreements and wea ~~have~~contract ~~received~~with nothe ~~revenues~~U.S. ~~from~~government that we assumed in the ~~sale~~Aceragen ofAcquisition, ~~commercial~~and ~~products.~~we Wehave ~~generated~~received no ~~revenue~~revenues ~~for~~from the ~~fiscal~~sale ~~year~~of ~~ended~~commercial ~~December 31, 2021~~products.

We have devoted substantially all our efforts to research and development, including clinical trials, and we have not completed development of any commercial products. Our research and development activities, together with our general and administrative expenses, are expected to continue to result in substantial operating losses for the foreseeable future. These losses, among other things, have had and will continue to have an adverse effect on our stockholders’ equity, total assets, and working capital. ~~Because~~Due ofto the numerous risks and uncertainties associated with developing drug candidates, and if approved, commercial products, we are unable to predict the extent of any future losses, whether or when any of our drug candidates will become commercially available or when we will become profitable, if at all.

Liquidity and Capital Resources

Overview

We require cash to fund our operating expenses and to make capital expenditures. Historically, we have funded our cash requirements primarily through the following:

(i) sale of common stock, preferred stock and future tranche rights, and warrants (including pre-funded warrants);

(ii)

exercise of warrants;

(iii)

debt financing, including capital leases;

(iv) license fees, research funding and milestone payments under collaborative and license agreements, and clinical funding arrangements, including reimbursements under U.S. Government funded programs; and

(v)

interest income.

We filed a shelf registration statement on Form S-3 on August 4, 2020, which was declared effective on September 2, 2020, relating to the sale, from time to time, in one or more transactions, up to $150.0 million of common stock, preferred stock, depository shares and warrants.SVB

On March 10, 2023, Silicon Valley Bank (“SVB”), at which we maintain cash and cash equivalents in multiple accounts, was closed by the California Department of Financial Protection and Innovation, which appointed the Federal Deposit Insurance Corporation (FDIC) as receiver. The failure of SVB exposed us to liquidity and credit risk prior to the completion of the FDIC resolution of SVB in a manner that fully protects all depositors. As a result, we do not anticipate any losses with respect to our funds that had been deposited with SVB.

LPC Purchase Agreement

On March 4, 2019, we entered into a Purchase Agreement with Lincoln Park Capital Fund, LLC (“Lincoln Park”), pursuant to which, upon the terms and subject to the conditions and limitations set forth therein, Lincoln Park has committed to purchase an aggregate of $35.0 million of shares of Company common stock from time to time at our sole discretion (the “LPC Purchase Agreement”).

During the years ended December 31, 2021 and 2020, we sold 800,000 and 750,000 shares of common stock, respectively, pursuant to the LPC Purchase Agreement, resulting in net proceeds of $4.2 million and $1.7 million, respectively. The LPC Purchase Agreement had a 36-month term which expired on March 4, 2022. Accordingly, we no longer have access to capital under the LPC Purchase Agreement.

ATM Agreement

In November 2018, we entered into an Equity Distribution Agreement (the “ATM Agreement”) with JMP Securities LLC (“JMP”) pursuant to which we may issue and sell shares of ~~its~~our common stock having an aggregate offering price of up to $50.0 million through JMP as ~~its~~our agent.

45During the year ended December 31, 2021, we sold 301,021 shares of common stock pursuant to the ATM Agreement resulting in net proceeds, after deduction of commissions and other offering expenses, of $15.3 million. No shares were sold during the year ended December 31, 2022. As of March 31, 2023, we may sell up to an additional $19.5 million of shares under the ATM Agreement

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During the years ended December 31, 2021 and 2020, we sold 5,117,357 and 3,608,713 shares of common stock, respectively, pursuant to the ATM Agreement, resulting in net proceeds, after deduction of commissions and other offering expenses, of $15.3 million and $12.3 million, respectively. As of March 31, 2022, we may sell up to an additional $19.5 million of shares under the ATM Agreement.

~~The LPC Purchase Agreement and ATM Agreement are more fully described in Note 8 of the notes to our financial statements included elsewhere in this Form 10-K.~~66

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Funding Requirements

We had cash and cash equivalents of approximately $3212.50 million at December 31, ~~2021~~2022. We believe based on our current operating plan, our existing cash, and cash equivalents on hand as of December 31, ~~2021~~2022 will enable us to fund our operations ~~through~~into ~~the~~May ~~one-year~~2023. ~~period~~Accordingly, ~~subsequent~~we need to raise additional capital to continue to fund our operations and service our obligations in the ~~filing~~future. ~~date~~If ofwe ~~this~~are ~~Form~~unable ~~10-K~~to raise additional capital when needed, we will not be able to continue as a going concern. ~~Specifically~~We do not currently have any products approved for sale and do not generate any revenue from product sales. Accordingly, we ~~believe~~expect to rely primarily on equity and/or debt financings and our ~~available~~clinical ~~funds~~funding ~~will~~arrangements be(e.g., ~~sufficient~~contract with Department of Defense’s DTRA) to ~~enable~~fund usour continued operations. We are seeking and expect to ~~perform~~continue to seek additional funding through financings of equity and/or debt securities, strategic research and development collaborations, clinical funding arrangements, or the ~~following:~~sale or license of technology assets. We may also engage in strategic alternative conversations of mergers, acquisition, or sale of the company or assets.

(i) fund business development related activities, such as identifying and potentially acquiring rights to novel development and commercial stage rare disease programs, including additional strategic alternatives;

~~(ii)~~Financing ~~conclude~~may ~~our~~not ~~current~~be ~~Low-Dose~~available to us when we need it, ~~High-Frequency~~or ~~Cohort~~on favorable or acceptable terms, or at all. We could be required to seek funds through collaborative alliances or through other means that may require us to relinquish rights to some of our ~~Phase~~technologies, 2drug ~~study~~candidates or drugs that we would otherwise pursue on our own. In addition, if we raise additional funds by issuing equity securities, our then existing stockholders may experience dilution. The terms of ~~tilsotolimod~~any infinancing ~~combination~~may ~~with~~adversely ~~nivolumab~~affect the holdings or the rights of existing stockholders. An equity financing that involves existing stockholders may cause a concentration of ownership. Debt financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends, and ~~ipilimumab~~are ~~for~~likely to include rights that are senior to the ~~treatment~~holders of ~~MSS~~our common stock. Any additional debt or equity financing may contain terms which are not favorable to us or to our stockholders, such as liquidation and other preferences, or liens or other restrictions on our assets. As discussed in Note 16 to the consolidated financial statements included elsewhere in this Form 10-~~CRC~~K, ~~(ILLUMINATE~~additional equity financings may also result in cumulative changes in ownership over a three-~~206);~~year period in excess of 50% which would limit the amount of net operating loss and tax credit carryforwards that we may utilize in any one year.

~~(iii)~~If ~~conclude~~we ~~our~~are ~~Phase~~unable 3to ~~clinical~~raise ~~trial~~additional ofcapital ~~tilsotolimod~~when inrequired ~~combination~~or ~~with~~on ~~ipilimumab~~acceptable ~~for~~terms, ~~the~~we ~~treatment~~may ofbe ~~anti-PD1~~required ~~refractory metastatic melanoma (ILLUMINATE-301);~~to:

●

significantly delay, scale back, or discontinue the development or commercialization of our product candidates;

●

dispose of technology assets, including current product candidates, or relinquish or license on unfavorable terms, our rights to technologies or any of our product candidates that we otherwise would seek to develop or commercialize ourselves;

●

pursue the sale of our company to a third party at a price that may result in a loss on investment for our stockholders; or

●

file for bankruptcy or cease operations altogether.

~~(iv)~~Any ~~fund~~of ~~certain~~these ~~research~~events ~~including~~could ~~investigator~~have ~~initiated~~a ~~clinical~~material ~~trials~~adverse ofeffect ~~tilsotolimod~~on ~~and~~our ~~the~~business, ~~Scriptr~~operating ~~Agreement;~~results, and prospects.

~~(v)~~We ~~maintain~~believe athe ~~level~~key offactors ~~general~~which ~~and~~will ~~administrative~~affect ~~expenses~~our ability to ~~support~~obtain ~~the~~funding ~~business.~~are:

●

the receptivity of the capital markets to financings by biotechnology companies generally and companies with drug candidates and technologies similar to ours specifically;

●

the receptivity of the capital markets to any in-licensing, product acquisition or other transaction we may enter into or attempt to enter into;

●

the results of our clinical development activities in our drug candidates we develop on the timelines anticipated;

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●

competitive and potentially competitive products and technologies and investors’ receptivity to our drug candidates we develop and the technology underlying them in light of competitive products and technologies;

●

the cost, timing, and outcome of regulatory reviews; and

●

our ability to enter into additional collaborations with biotechnology and pharmaceutical companies and the success of such collaborations.

In addition, weincreases ~~are~~in ~~seeking~~expenses ~~and~~or ~~expect~~delays toin ~~continue~~clinical todevelopment ~~seek~~may ~~additional~~adversely ~~funding~~impact ~~through~~our ~~collaborations,~~cash ~~the~~position ~~sale~~and orrequire ~~license~~additional offunds ~~assets~~ or ~~financings~~cost ~~of equity or debt securities~~reductions. ~~We believe the key factors which will affect our ability to obtain funding are:~~

~~(i)~~Cash ~~the receptivity of the capital markets to any in-licensing, product acquisition or other transaction we may enter into;~~Flows

~~(ii)~~The ~~the~~following ~~receptivity~~table presents a summary of the ~~capital~~primary ~~markets~~sources toand ~~financings~~uses byof ~~biotechnology~~cash ~~companies~~for ~~generally~~the ~~and~~years ~~companies~~ended ~~with~~December ~~drug~~31, ~~candidates~~2022 and ~~technologies similar to ours specifically;~~2021:



	Year Ended December 31,
(in thousands)	2022		2021
Net cash provided by (used in):
Operating activities	$	(24,495)	$	(24,597)
Investing activities		5,482		4,500
Financing activities		(1,488)		19,413
Decrease in cash and cash equivalents	$	(20,501)	$	(684)

~~(iii)~~Operating Activities. The net cash used in operating activities for the ~~results~~periods presented consisted primarily of our ~~clinical~~net ~~development~~(loss) ~~activities~~income adjusted for non-cash charges/gains and changes in ~~our~~components ~~drug~~of ~~candidates~~working wecapital. ~~develop~~The onchange in our operating cash outflows for the ~~timelines~~year ~~anticipated;~~ended December 31, 2022, as compared to 2021, was nominal.

~~(iv)~~Investing ~~competitive~~Activities. Net ~~and~~cash ~~potentially~~provided ~~competitive~~by ~~products~~investing ~~and~~activities ~~technologies~~for ~~and~~the ~~investors'~~year ~~receptivity~~ended toDecember ~~our~~31, ~~drug~~2022 ~~candidates~~consisted weof ~~develop~~$5.5 ~~and~~million in cash acquired in connection with the ~~technology~~Aceragen ~~underlying~~Acquisition. ~~them~~Net incash ~~light~~provided by investing activities for the year ended December 31, 2021 consisted of ~~competitive~~$4.5 ~~products~~million ~~and~~in ~~technologies;~~proceeds from the maturity of available-for-sale securities.

~~(v)~~Financing Activities. Net cash used in financing activities for the ~~cost~~year ended December 31, ~~timing~~2022 consisted of $1.5 million in repayments of outstanding debt assumed in connection with the Aceragen Acquisition, offset in part by proceeds from employee stock purchases and ~~outcome~~exercise of ~~regulatory~~common ~~reviews;~~stock warrants. Net cash provided by financing activities for the year ended December 31, 2021 included aggregate net proceeds of $19.5 million from financing arrangements consisting of $4.2 million received pursuant to the LPC Purchase Agreement (as defined below) and $15.3 million received under the ATM Agreement, plus $0.3 million received from the exercise of stock options and warrants, partially offset by $0.4 million in payments related to our short-term insurance premium financing arrangement.

~~(vi)~~Material ~~our~~Cash ~~ability to enter into additional collaborations with biotechnology and pharmaceutical companies and the success of such collaborations; and~~Requirements

~~(vii)~~Aceragen ~~the~~Acquisition ~~impact of the COVID-19 pandemic to global economy and capital markets, and to our business and our financial results.~~Obligation

InAs ~~addition~~of December 31, ~~increases~~2022, we had a material debt obligation in ~~expenses~~the oramount ~~delays~~of $6.1 million related to a $7.5 million debt obligation we assumed in ~~clinical~~connection ~~development~~with ~~may~~the ~~adversely~~Aceragen ~~impact~~Acquisition, ~~our~~of ~~cash~~which ~~position~~approximately $1.5 million was paid in October 2022. On January 31, 2023, we issued 12% convertible unsecured promissory notes (the “Convertible Notes”) to certain of the debtholders in an aggregate amount of approximately $5.9 million. The Convertible Notes bear annual interest at 12%, beginning on April 1, 2023, and ~~require~~may ~~additional~~be ~~funds~~converted orinto ~~cost~~shares ~~reductions~~of Company’s common stock. The terms of the Convertible Notes also provide the noteholders with customary registration rights covering the common stock issued following any conversion of the Convertible Notes.

See Note 7 of the notes to our consolidated financial statements appearing elsewhere in this Form 10-K for additional information.

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Cash Flows

~~The~~Lease ~~following table presents a summary of the primary sources and uses of cash for the years ended December 31, 2021 and 2020:~~Obligations



	Year Ended December 31,
(in thousands)	2021		2020
Net cash provided by (used in):
Operating activities	$	(24,597)	$	(33,772)
Investing activities		4,500		(1,687)
Financing activities		19,413		28,669
Decrease in cash and cash equivalents	$	(684)	$	(6,790)

~~Operating~~As ~~Activities. The~~of ~~net~~December 31, ~~cash~~2022, ~~used~~we inhad ~~operating~~a ~~activities~~material ~~for~~lease ~~all~~commitment ~~periods~~in ~~presented~~an ~~consists~~aggregate ~~primarily~~amount of ~~our~~$0.6 ~~net~~million ~~income~~relating ~~(losses)~~to ~~adjusted~~our ~~for~~facility ~~non-cash charges/gains and changes~~ in ~~components~~Exton, ~~of working capital~~Pennsylvania. ~~The~~This ~~decrease~~lease inexpires ~~cash~~on ~~outflow~~May ~~for~~31, ~~the~~2025. ~~year~~See ~~ended~~Note ~~December~~15 ~~31,~~of ~~2021,~~the asnotes ~~compared~~ to ~~2020,~~our ~~related~~consolidated ~~primarily~~financial tostatements ~~lower~~appearing ~~costs~~elsewhere ~~incurred~~in ~~for~~this ~~our~~Form ~~tilsotolimod~~10-K ~~development~~for ~~program~~additional ~~during 2021~~information.

Investing Activities. Net cash provided by (used in) investing activities primarily consisted of the following amounts relating to our investments in available-for-sale securities and purchases and disposals of property and equipment:

●Critical ~~For~~Accounting ~~the~~Estimates ~~year ended December 31, 2021, proceeds from the maturity of available-for-sale securities were $4.5 million; and~~

● For the year ended December 31, 2020, proceeds from the maturity of available-for-sale securities of $10.5 million, substantially offset by the purchase of $12.2 million of available-for-sale securities.

~~Financing~~This ~~Activities~~management’s discussion and analysis of financial condition and results of operations is based on our financial statements, which have been prepared in accordance with accounting principles generally accepted in the United States. ~~Net~~ ~~cash~~The ~~provided~~preparation byof ~~financing~~these ~~activities~~financial ~~primarily~~statements ~~consisted~~requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and the ~~following~~disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts ~~raised~~of inrevenues ~~connection~~and ~~with~~expenses during the ~~following~~reporting ~~transactions:~~periods. On an ongoing basis, management evaluates its estimates and judgments, including those related to (i) indefinite-lived intangible assets, (ii) warrants and Series X Preferred Stock liabilities and related revaluation gains (losses), (iii) research and development prepayments, accruals and related expenses, and (iv) stock-based compensation. Management bases its estimates and judgments on historical experience and on various other factors that are believed to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions.

●We ~~For~~regard ~~the~~an ~~year~~accounting ~~ended~~estimate ~~December~~or ~~31,~~assumption ~~2021,~~underlying ~~aggregate~~our ~~net~~financial ~~proceeds~~statements ofas ~~$19.5~~a ~~million~~“critical ~~from~~accounting ~~financing~~estimate” ~~arrangements~~if: consisting of $4.2 million received pursuant to the LPC Purchase Agreement and $15.3 million received under the ATM Agreement, plus $0.3 million received from the exercise of stock options and warrants, partially offset by $0.4 million in payments related to our short-term insurance premium financing arrangement; and

● ~~For~~ the ~~year~~nature ~~ended December 31, 2020, aggregate net proceeds~~ of ~~$28.8~~the ~~million~~estimate ~~from~~or ~~financing~~assumption ~~arrangements~~is ~~consisting~~material ofdue ~~$14.8 million received pursuant~~ to the ~~April~~level ~~2020~~of subjectivity and ~~July~~judgment ~~2020~~necessary ~~Securities Purchase Agreements, $1.7 million received pursuant~~ to ~~the~~account ~~LPC~~for ~~Purchase~~highly ~~Agreement~~uncertain ~~and~~matters ~~$12.3~~or ~~million received under~~ the ~~ATM~~susceptibility ~~Agreement,~~of ~~plus~~such anmatters ~~additional $0.1 million in proceeds from employee stock purchases under our 2017 Employee Stock Purchase Plan, partially offset by $0.2 million in payments related~~ to ~~our~~change; ~~short-term insurance premium financing arrangement.~~and

●

the impact of the estimates and assumptions on financial condition or operating performance is material.

~~Material~~While ~~Cash~~our ~~Requirements~~significant accounting policies are described in more detail in Note 2 to our financial statements appearing elsewhere in this Form 10-K, we believe the following accounting policies to be the most critical to the judgments and estimates used in the preparation of our financial statements.

As of December 31, 2021, we had a material lease commitment in an aggregate amount of $0.8 million relating to our facility in Exton, Pennsylvania. This lease expires on May 31, 2025. See Note 12 of the Notes to Financial Statements in this Form 10Indefinite-Klived ~~for~~Intangible ~~additional information.~~Assets

47Indefinite-lived intangible assets consist of In-Process Research & Development (“IPR&D”). The fair values of IPR&D project assets acquired in business combinations are capitalized. We generally utilize the Multi-Period Excess Earning Method to determine the estimated fair value of the IPR&D assets acquired in a business combination. The projections used in this valuation approach are based on many factors, such as relevant market size, the estimated probability of regulatory success rates, anticipated patent protection, expected pricing, expected treated population and estimated payments (e.g., royalty). The estimated future net cash flows are then discounted to the present value using an appropriate discount rate. These assets are treated as indefinite-lived intangible assets until completion or abandonment of the projects, at which time the assets are amortized over the remaining useful life or written off, as appropriate.

Intangible assets with indefinite lives, including IPR&D, are tested for impairment if impairment indicators arise and, at a minimum, annually. However, an entity is permitted to first assess qualitative factors to determine if a quantitative impairment test is necessary. Further testing is only required if the entity determines, based on the qualitative assessment, that it is more likely than not that an indefinite-lived intangible asset’s fair value is less than its carrying amount. Otherwise, no further impairment testing is required. The indefinite-lived intangible asset impairment test consists of a one-step analysis that compares the fair value of the intangible asset with its carrying amount. If the carrying amount of an intangible asset exceeds its fair value, an impairment loss is recognized in an amount equal to that excess. We consider many factors in evaluating whether the value of our intangible assets with indefinite lives may not be recoverable, including, but not limited to, expected growth rates, the cost of equity and debt capital, general economic conditions, our outlook and market performance of our industry and recent and forecasted financial performance.

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Critical Accounting Policies and Estimates

~~This~~Warrant ~~management’s discussion~~ and ~~analysis~~Series ofX ~~financial~~Preferred ~~condition~~Stock ~~and~~Liabilities results of operations is based on our financial statements, which have been prepared in accordance with accounting principles generally accepted in the United States. The preparation of these financial statements requires management to make estimates and ~~assumptions~~Related ~~that~~Revaluation ~~affect~~Gain the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of revenues and expenses during the reporting period. On an ongoing basis, management evaluates its estimates and judgments, including those related to warrant and future tranche right liabilities and related revaluation gains (~~losses~~Loss), research and development prepayments, accruals and related expenses, and stock-based compensation. Management bases its estimates and judgments on historical experience and on various other factors that are believed to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions.

Warrant Liability. In connection with the Aceragen Acquisition, a portion of the consideration paid to Legacy Aceragen warrant holders was in the form of warrants to purchase shares of Series Z Preferred Stock (the “Series Z Warrants”). The Series Z Warrants are classified as liabilities because the underlying Series Z Preferred Stock is contingently redeemable. We ~~regard~~use ana ~~accounting~~Black-Scholes ~~estimate~~option orpricing ~~assumption~~model to value our liability-classified warrants, which incorporates assumptions and estimates, including (i) the remaining contractual term of the warrants, (ii) risk-free interest rate, (iii) expected dividend yield, and (iv) expected volatility of the price of the underlying shares of Series Z Preferred Stock. The estimated the expected stock volatility is based on the historical volatility of our ~~financial~~common ~~statements~~stock asfor a ~~“critical~~term ~~accounting~~equal ~~estimate”~~to ~~where:~~the remaining contractual term of the warrants. The risk-free interest rate was determined by reference to the U.S. Treasury yield curve for time periods approximately equal to the remaining contractual term of the warrants. Expected dividend yield was determined based on the fact that we had never paid cash dividends and did not expect to pay any cash dividends in the foreseeable future. Due to the nature of and inputs in the model used to assess the fair value of the warrants, it is not abnormal to experience significant fluctuations during each remeasurement period.

●Series X Preferred Stock Liability. In conjunction with the ~~nature~~Aceragen ofAcquisition, we determined that the ~~estimate~~newly orissued ~~assumption~~Series isX ~~material~~Preferred ~~due~~Stock torepresents ~~the~~a ~~level~~sale of ~~subjectivity~~future revenues and ~~judgment~~is ~~necessary~~classified as a liability under Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) 470, Debt, and have elected to account for ~~highly~~the ~~uncertain~~Series ~~matters~~X orPreferred Stock liability under the ~~susceptibility~~fair value option. The fair value of the Series X Preferred Stock liability represents the present value of estimated future payments, including royalty payments, as well as estimated payments that are contingent upon the achievement of specified milestones. The fair value of the Series X Preferred Stock liability is based on the cumulative probability of the various estimated payments. The fair value measurement is based on significant Level 3 unobservable inputs such ~~matters~~as estimated sales proceeds related to ~~change;~~the PRV, the relevant market size, the estimated probability of regulatory success rates, anticipated patent protection, expected pricing, expected treated population, sales by region, estimated royalty payments and discount rate. Any changes in the fair value of the liability in the reporting periods are recognized in the consolidated statement of operations until it is settled.

● the impact of the estimates and assumptions on financial condition or operating performance is material.

While our significant accounting policies are described in more detail in Note 2 to our financial statements appearing elsewhere in this Form 10-K, we believe the following accounting policies to be most critical to the judgments and estimates used in the preparation of our financial statements.

Research and Development Prepayments, Accruals and Related Expenses

As part of the process of preparing our financial statements, we are required to estimate our accrued and prepaid expenses for research and development activities performed by third parties, including ~~Clinical Research Organizations (“~~CROs”) clinical investigators and our research collaboration partners. These estimates are made as of the reporting date of the work completed over the life of the individual study in accordance with agreements established with CROs and clinical trial sites. Some CROs invoice us on a monthly basis, while others invoice upon achievement of milestones and the expense is recorded as services are rendered. We determine the estimates of research and development activities incurred at the end of each reporting period through discussion with internal personnel and outside service providers and research collaboration partners as to the progress or stage of completion of trials or services, as of the end of each reporting period, pursuant to contracts with clinical trial centers and CROs and the agreed upon fee to be paid for such services. We periodically confirm the accuracy of our estimates with the service providers and make adjustments if necessary. Clinical trial site costs related to patient enrollments are recorded as patients are entered into the trial.

Stock-Based Compensation

We recognize all share-based payments to employees and directors as expense in our statements of operations based on their fair values. We record compensation expense over an award’s requisite service period, or vesting period, based on the award’s fair value at the date of grant. Our policy is to charge the fair value of stock options as an expense, adjusted for forfeitures, on a straight-line basis over the vesting period, which is generally four years for employees and one year for directors.

We use the Black-Scholes option pricing model to estimate the fair value of stock option grants. The Black-Scholes option pricing model relies on a number of key assumptions to calculate estimated fair values, including assumptions as to average risk-free interest rate, expected dividend yield, expected life, and expected volatility. For the assumed risk-free interest rate, we use the U.S. Treasury security rate with a term equal to the expected life of the option. Our assumed dividend yield of zero is based on the fact that we have never paid cash dividends to common stockholders and have no present intention to pay cash dividends. We use an expected option life based on actual experience. Our assumption for expected volatility is based on the actual stock-price volatility over a period equal to the expected life of the option.

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common stockholders and have no present intention to pay cash dividends. We use an expected option life based on actual experience. Our assumption for expected volatility is based on the actual stock-price volatility over a period equal to the expected life of the option.

If factors change and we employ different assumptions for estimating stock-based compensation expense in future periods, or if we decide to use a different valuation model, the stock-based compensation expense we recognize in future periods may differ significantly from what we have recorded in the current period and could materially affect our loss from operations, net income (loss) and earnings (loss) per share. It may also result in a lack of comparability with other companies that use different models, methods, and assumptions. The Black-Scholes option pricing model was developed for use in estimating the fair value of traded options that have no vesting restrictions and are fully transferable. These characteristics are not present in our option grants. Although the Black-Scholes option pricing model is widely used, existing valuation models, including the Black-Scholes option pricing model, may not provide reliable measures of the fair values of our stock-based compensation.

Warrant and Future Tranche Right Liabilities and Related Revaluation Gain (Loss)

We entered into the December 2019 Securities Purchase Agreement, as more fully described in Note 7 of the Notes to Financial Statements appearing elsewhere in this Form 10-K, pursuant to which we issued shares of convertible preferred stock with detachable warrants. Additionally, the December 2019 Securities Purchase Agreement contains call options on redeemable preferred shares with warrants (conditionally exercisable for shares that are puttable), which we refer to as future tranche rights.

We determined that these warrants and future tranche rights represent freestanding financial instruments and account for both the warrants and future tranche rights as liabilities, which requires the measurement of the fair value of the liability at the time of issuance and recording changes as a charge to current earnings at each reporting period, which is included in Warrant Liability Revaluation Expense and/or Future Tranche Right Liability Revaluation Expense in our statements of operations.

Warrant Liability. We use an option pricing model to value our liability-classified warrants. Inherent in the valuation model are assumptions related to volatility, risk-free interest rate, expected term, dividend rate, and other scenarios (i.e. probability of complex features of the warrants being triggered). Due to the nature of and inputs in the model used to assess the fair value of the warrants, it is not abnormal to experience significant fluctuations during each remeasurement period.

Future Tranche Right Liability. We use both a lattice model and a Monte Carlo simulation to value the future tranche rights. We selected these models as we believe they are reflective of all significant assumptions that market participants would likely consider in negotiating the transfer of the future tranche rights. Such assumptions include, among other inputs, stock price volatility, risk-free rates, and expected terms inclusive of early exercise and cancellation assumptions. Due to the nature of and inputs in the model used to assess the fair value of the future tranche rights, it is not abnormal to experience significant fluctuations during each remeasurement period.

All outstanding warrants and future tranche rights previously issued pursuant to the December 2019 Securities Purchase Agreement were terminated during the three months ended March 31, 2021. Accordingly, we are no longer eligible to receive additional proceeds pursuant to the December 2019 Securities Purchase Agreement and the related warrant liability and future tranche right liability were derecognized during the three months ended March 31, 2021.

New Accounting Pronouncements

New accounting pronouncements are discussed in Note 2 of the ~~Notes~~notes to ~~Financial~~our ~~Statements~~consolidated financial statements appearing elsewhere in this Form 10-K.

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Item 7A.

Quantitative and Qualitative Disclosures about Market Risk.

As of December 31, ~~2021~~2022, all material assets and liabilities are in U.S. dollars, which is our functional currency. Although our foreign subsidiary is included in our consolidated financial statements, operations at this entity are not material and there are currently no significant financial assets or liabilities denominated in a foreign currency in which exchange rate fluctuations would result in a material adverse impact to our results of operations, financial position, or cash flows.

We maintain investments in accordance with our investment policy. The primary objectives of our investment activities are to preserve principal, maintain proper liquidity to meet operating needs, and maximize yields. Although our investments are subject to credit risk, our investment policy specifies credit quality standards for our investments and limits the amount of credit exposure from any single issue, issuer, or type of investment. We regularly review our investment holdings in light of the then current economic environment. At December 31, ~~2021~~2022, all our invested funds were invested in money market funds classified in cash and cash equivalents on the accompanying balance sheet.

Based on a hypothetical ten percent adverse movement in interest rates, the potential losses in future earnings, fair value of risk sensitive financial instruments, and cash flows are immaterial to our earnings, although the actual effects may differ materially from the hypothetical analysis.

Item 8.

Financial Statements and Supplementary Data.

All financial statements required to be filed hereunder are filed as listed under Item 15(a) of this Form 10-K and are incorporated herein by ~~this~~ reference.

~~There~~We ~~have~~are ~~been~~a nosmaller ~~retrospective~~reporting ~~changes~~company, toas ~~our~~defined ~~statements~~by ofRule ~~operations~~12b-2 ~~for any~~ of the ~~quarters~~Exchange ~~within~~Act, ~~the~~and ~~two~~therefore ~~years~~we inare ~~the~~permitted ~~period~~to ~~ended~~provide ~~December~~scaled ~~31,~~Item ~~2021~~8 disclosure.

There have been no retrospective changes to our consolidated statements of operations for any of the quarters within the two years ended December 31, 2022, except for the retroactive effect of the 1-for-17 reverse stock split as described in Note 19 to the consolidated financial statements appearing elsewhere in this Form 10-K.

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Item 9.

Changes in and Disagreements with Accountants on Accounting and Financial Disclosure.

None.

Item 9A. Controls and Procedures.

Disclosure Controls and Procedures

Our management, with the participation of our principal executive officer and principal financial officer, evaluated the effectiveness of our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act) as of December 31, ~~2021~~2022. In designing and evaluating our disclosure controls and procedures, management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving their objectives and our management necessarily applied its judgment in evaluating the cost-benefit relationship of possible controls and procedures. Based on this evaluation, our principal executive officer and principal financial officer concluded that, as of December 31, ~~2021~~2022, our disclosure controls and procedures were (1) designed to ensure that material information relating to us is made known to our principal executive officer and principal financial officer by others, particularly during the period in which this report was prepared, and (2) effective, in that they provide reasonable assurance that information required to be disclosed by us in the reports we file or submit under the Exchange Act is recorded, processed, summarized and reported within the time periods specified in the applicable SEC’s rules and forms.

Internal Control over Financial Reporting

a)	Management’s Annual Report on Internal Control over Financial Reporting

Our management, with the participation of our principal executive officer and principal financial officer, is responsible for establishing and maintaining adequate internal control over financial reporting. Internal control over financial reporting is defined in Rule 13a-15(f) and 15d-15(f) promulgated under the Exchange Act as a process designed by, or under the supervision of, the Company’s principal executive and principal financial officers and effected by the Company’s Board of Directors, management, and other personnel, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles and includes those policies and procedures that:

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assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles and includes those policies and procedures that:

●

Pertain to the maintenance of records that in reasonable detail accurately and fairly reflect the transactions and dispositions of the assets of the Company;

●

Provide reasonable assurance that transactions are recorded as necessary to permit preparation of financial statements in accordance with generally accepted accounting principles, and that receipts and expenditures of the Company are being made only in accordance with authorizations of management and directors of the Company; and

●

Provide reasonable assurance regarding prevention or timely detection of unauthorized acquisition, use, or disposition of the Company’s assets that could have a material effect on the financial statements.

● Provide reasonable assurance regarding prevention or timely detection of unauthorized acquisition, use or disposition of the Company’s assets that could have a material effect on the financial statements.

Because of its inherent limitations, internal control over financial reporting may not prevent or detect misstatements. Therefore, even those systems determined to be effective can provide only reasonable assurance with respect to financial statement preparation and presentation. Projections of any evaluation of effectiveness to future periods are subject to the risk that controls may become inadequate because of changes in conditions, or that the degree of compliance with the policies or procedures may deteriorate.

Management assessed the effectiveness of our internal control over financial reporting as of December 31, ~~2021~~2022. In making this assessment, management used the criteria set forth by the Committee of Sponsoring Organizations of the Treadway Commission in Internal Control — Integrated Framework (2013).

~~Based on its assessment, management believes that, as of December 31, 2021, the Company’s internal control over financial reporting is effective based on those criteria.~~ 72

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We acquired Legacy Aceragen in September 2022. Due to the timing of the Aceragen Acquisition and as allowed under SEC guidance, management’s assessment of and conclusion regarding the design and effectiveness of internal control over financial reporting excluded the internal control over financial reporting of Legacy Aceragen, which is relevant to our 2022 consolidated financial statements as of and for the year ended December 31, 2022.

Based on its assessment, management believes that, as of December 31, 2022, the Company’s internal control over financial reporting was effective based on those criteria.

b)	Attestation Report of the Registered Public Accounting Firm

Not Applicable.

c)	Changes in Internal Control over Financial Reporting.

No change in our internal control over financial reporting occurred during the fourth quarter of the fiscal year ended December 31, ~~2021~~2022 that has materially affected, or is reasonably likely to materially affect, our internal control over financial reporting. However, we are in the process of integrating Legacy Aceragen into our system of internal control over financial reporting which may result in future changes to our internal control environment.

Item 9B.

Other Information.

~~None~~The information included in this Item 9B is provided, at our option, in lieu of filing such information under Item 5.02 and Item 8.01 of Current Report on Form 8-K.

On April 13, 2023, the Board approved certain cost-cutting measures with a view to preserving capital to support our continuing operations. As part of this plan, we have commenced the furlough of 12 employees (including an executive officer), representing approximately 46% of our workforce. Additionally, certain of our employees and executive officers, including our principal executive officer, John Taylor, and our principal financial officer, John Kirby, will have a portion of their salaries deferred in amounts that exceed $200,000, with such deferrals having a retroactive effective date of April 5, 2023. Such deferrals are expected to continue until such time as the Company is able to secure sufficient additional financing, as mutually agreed upon. If such financing is obtained, the impacted employees and executive officers, including Mr. Taylor and Mr. Kirby, will receive lump-sum payments equal to their respective aggregate deferred base salary amounts.

Item 9C. Disclosure Regarding Foreign Jurisdictions that Prevent Inspections.

Not applicable.

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PART III.

Item 10.

Directors, Executive Officers, and Corporate Governance.

~~The~~Information ~~information~~about ~~required by this item is incorporated by reference to~~ our ~~Proxy~~Directors ~~Statement for the 2022 Annual Meeting of Stockholders to be filed with the SEC within 120 days of the fiscal year ended December 31, 2021.~~

Set forth below is information about each member of our board of directors, including (a) the year in which each director first became a director, (b) their age as of March 30, 2023, (c) their positions and offices with our Company, (d) their principal occupations and business experience during at least the past five years and (e) the names of other public companies for which they currently serve, or have served within the past five years, as a director. We have also included information about each director's specific experience, qualifications, attributes, or skills that led our board of directors to conclude that such individual should serve as one of our directors. We also believe that all of our directors have a reputation for integrity, honesty and adherence to high ethical standards. They each have demonstrated business acumen and an ability to exercise sound judgment, as well as a commitment of service to our Company and our board of directors.

John Taylor, our Chief Executive Officer and a member of our board, is the son-in-law of Andy Jordan, our Chief Strategy Officer. With the exception of the foregoing, there are no family relationships among our directors and executive officers.

WeThe ~~have~~following ~~adopted~~table aprovides ~~written~~information ~~code~~about ofthose ~~business conduct and ethics that applies to our principal executive officer, principal financial officer, principal accounting officer or controller, or~~ persons ~~performing~~who ~~similar~~currently ~~functions.~~serve Weas ~~have~~directors ~~posted a current copy~~ of the ~~Code of Business Conduct and Ethics in the “Investors — Corporate Governance” section of our website, which is located at www~~Company.iderapharma.com. We intend to satisfy the disclosure requirements under Item 5.05 of Form 8-K regarding an amendment to, or waiver from, a provision of our code of business conduct and ethics by posting such information on our website at www.iderapharma.com.

Committee Memberships (1)

Name

Age

Relationship

Audit

Comp

N&CG

Class – Election Year

Vincent J. Milano

Chair of the Board

Class I – 2023

Dr. Cristina Csimma

Director

Class I – 2023

Michael Dougherty

Director

Class I – 2023

Dr. Maxine Gowen

Director

Class II – 2024

Ronald Wooten

Director

Class III – 2025

John C. Taylor

Director

Class III – 2025

•

“C” indicates Chair of applicable committee.

Vincent J. Milano was appointed Chair of our board of directors in 2022, having previously served as our President and Chief Executive Officer and as a member of our board of directors since 2014. Prior to joining us, Mr. Milano served as Chairman, President, and Chief Executive Officer of ViroPharma Incorporated (“ViroPharma”), a pharmaceutical company that was acquired by Shire plc in 2014, from 2008 to 2014, as its Vice President, Chief Financial Officer, and Chief Operating Officer from 2006 to 2008, and as its Vice President, Chief Financial Officer, and Treasurer from 1996 to 2005. Mr. Milano also served on the board of directors of ViroPharma from 2008 to 2014. Prior to joining ViroPharma, Mr. Milano served in increasingly senior roles, most recently senior manager, at KPMG LLP, an independent registered public accounting firm, from 1985 to 1996.

Mr. Milano currently serves on the board of directors of Aclaris Therapeutics, Inc. (Nasdaq: ACRS) and Biocryst Pharmaceuticals, Inc. (Nasdaq: BCRX), since 2020 and 2021, respectively. Mr. Milano previously served as a director of Spark Therapeutics, Inc., Vanda Pharmaceuticals Inc., and privately held VenatoRx Pharmaceuticals, Inc. from 2014 to 2019, from 2010 to 2019, and from 2013 to 2022, respectively. Mr. Milano holds a Bachelor of Science degree in Accounting from Rider College.

We believe that Mr. Milano’s qualifications to sit on our board of directors include his significant public company management and board experience and knowledge of our industry.

Dr. Cristina Csimma, PharmD, MHP is a biopharmaceutical leader and strategic advisor with decades of experience in biotechnology, large pharma, and venture capital. Dr. Csimma currently serves on the board of directors of Syncona Partners, LLP (LON: SYNC), having been elected to its board of directors in February 2022. She also serves as a board director and a member of the compensation committee of Palisade Bio, Inc. (Nasdaq: PALI), having been elected to its board of directors in 2017. She also serves as the chair of the board of directors of Caraway Therapeutics, Inc. since 2019 (executive chair in 2019). Dr. Csimma also serves on advisory boards,

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including: the Muscular Dystrophy Association Venture Philanthropy Scientific Advisory Committee since 2006; the Harvard and Brigham and Women’s Hospital MRCT Center External Advisory Board since 2015; the TREAT-NMD Advisory Committee for Therapeutics (TACT) since 2009; and the Executive Oversight Board to the National Institutes of Health (NIH) NeuroNext Network since 2013.

Dr. Csimma previously served as chair of the board of directors of Forendo Pharma between 2020 and 2021 (executive chair in 2021) when it was acquired by Organon & Co. Dr. Csimma also previously served as a director on the boards of Seneca Biopharma, Inc. (formerly Neuralstem Inc., from 2017 until 2021 when it merged with Leading BioSciences Inc. to form Palisade Bio), Juniper Pharmaceuticals, Inc. (from 2010 until its acquisition by Catalent, Inc. in 2018), and Vtesse Inc. (from 2014 until its acquisition by Sucampo Pharmaceuticals, Inc. in 2017). Dr. Csimma was the executive chair and a senior advisor of Exonics Therapeutics, Inc. (from 2016 to 2017), and was President, founding Chief Executive Officer, and board director of Cydan Inc. from 2012 to 2014. She also served on the board of directors of T1D Exchange (non-profit Type 1 Diabetes) from 2018 to 2020 and the NIH Blueprint Neurotherapeutics Network External Oversight Committee from 2014 to 2018, was Vice President of Drug Development at Virdante Pharmaceuticals Inc. from 2009 to 2011, Principal at Clarus Ventures LLC (now Blackstone Life Science), and held roles of increasing responsibility in Clinical Development and Translational Research at Wyeth (now Pfizer Inc.), Genetics Institute, and Dana Farber Cancer Institute. Dr. Csimma holds both a Doctor of Pharmacy and a Bachelor of Science in Pharmacy from the Massachusetts College of Pharmacy and Allied Health Sciences, as well as a Master of Health Professions from Northeastern University.

We believe that Dr. Csimma’s qualifications to sit on our board of directors include her significant public company management and board experience and knowledge of our industry.

Michael Dougherty was appointed as a member of our board of directors in 2019 and served as the board’s chair from 2021 to 2022. Mr. Dougherty currently serves on the board of directors of Trevena, Inc. (Nasdaq: TRVN). Mr. Dougherty was executive chairman of Celator Pharmaceuticals, Inc., or Celator, from 2015 until its acquisition by Jazz Pharmaceuticals plc in 2016; he also served as a director of Celator from 2013 to 2016. Mr. Dougherty previously served in a variety of senior positions in the biopharmaceutical industry, including as Chief Executive Officer at Kalidex Phamaceuticals, Inc.; President and Chief Executive Officer at Adolor Corporation; President and Chief Operating Officer at Genomics Collaborative, Inc.; President and Chief Executive Officer at Genaera Corporation; and Chief Financial Officer at Centocor, Inc. He also previously served as a member of the board of directors of and Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), Foundation Medicine, Inc., Adolor Corporation, Genaera Corporation, Aviragen Therapeutics, Inc., Cempra, Inc., and ViroPharma Incorporated. Mr. Dougherty received a Bachelor of Science in Accounting from Villanova University.

We believe that Mr. Dougherty’s qualifications to sit on our board of directors include his significant public company management and board experience and knowledge of our industry.

Dr. Maxine Gowen served as the Chief Executive Officer and a board director of TamuroBio Inc., a privately held drug development company, from 2019 to 2021, and she remains on the board of directors. She was the founding President and Chief Executive Officer of Trevena, Inc. (“Trevena”) (Nasdaq: TRVN), a publicly traded biopharmaceutical company, from 2007 until her retirement in 2018; she remained a member of its board of directors until 2021. Prior to joining Trevena, Dr. Gowen was Senior Vice President for the Center of Excellence for External Drug Discovery at GlaxoSmithKline plc (“GSK”), where she held a variety of leadership positions in research and development (“R&D”), venture capital, and business development during her 15-year tenure. Before GSK, Dr. Gowen was Senior Lecturer and Head, Bone Cell Biology Group, Department of Bone and Joint Medicine, of the University of Bath, U.K. Dr. Gowen has served as a director of Aclaris Therapeutics, Inc. (Nasdaq: ACRS) since 2019, Passage Bio, Inc. (Nasdaq: PASG), and as its Chair since 2021, and Merus NV (Nasdaq: MRUS) since 2021. She previously held board seats in the state biotechnology industry association, Life Sciences of Pennsylvania (from 2015 to 2021) and in the national biotechnology industry association (“BIO”) (from 2008 to 2018). Dr. Gowen previously served as a director of Human Genome Sciences, Inc. (from 2008 to 2012) and Akebia Therapeutics, Inc. (from 2014 to 2021), both publicly traded companies, as well as Panorama Medicine, a privately held biotechnology company (from 2020 to 2021). She received her Ph.D. from the University of Sheffield, U.K., an M.B.A. with academic honors from The Wharton School of the University of Pennsylvania, and a B.Sc. with Honors in Biochemistry from the University of Bristol, U.K.

We believe that Dr. Gowen’s qualifications to sit on our board of directors include her significant public company management and board experience and knowledge of our industry.

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Ronald Wooten was appointed to our Board of Directors in connection with the closing of the Aceragen Acquisition. Prior to the closing of the Aceragen Acquisition, Mr. Wooten served as a member of the board of directors of Legacy Aceragen since May 2021. Mr. Wooten has been a partner of NovaQuest Capital Management, L.L.C., an investment firm that focuses on the biopharmaceutical sector, since its inception in 2010. Since 2010, Mr. Wooten has been a member of the investment committee of NovaQuest Pharma Opportunities Fund III, NovaQuest Pharma Opportunities Fund IV, NovaQuest Pharma Opportunities Fund V, NovaQuest Private Equity Fund I, and NovaQuest Animal Health Fund I. From 2000 to 2010, he was President of the NovaQuest business unit of Quintiles Inc., a contract research company (“Quintiles”). Mr. Wooten was previously Executive Vice President at Quintiles and served on its board of directors from 2008 to 2010. Mr. Wooten’s previous experience includes nine years with First Union Securities, where he served as a Managing Director of Investment Banking. Mr. Wooten holds a B.A. degree in Chemistry from the University of North Carolina at Chapel Hill and an M.B.A. from Boston University.

We believe that Mr. Wooten’s qualifications to sit on our board of directors include his significant public company management and board experience and knowledge of our industry.

John C. Taylor was appointed as our Chief Executive Officer and to our board of directors in connection with the closing of the Aceragen Acquisition. Mr. Taylor was a co-founder and previously served as the Chief Executive Officer of Legacy Aceragen since its founding in 2021. Since 2018, Mr. Taylor has served as the Entrepreneur In Residence for the North Carolina Biotechnology Center. From 2013 to 2018, Mr. Taylor served as the Chief Executive Officer for Spyryx Biosciences, Inc., a company focused on developing therapies targeting novel regulation of ENaC for lung complications associated with cystic fibrosis. Prior to this, Mr. Taylor served as the Vice President, Corporate Development for Synageva BioPharma Corp. from 2009 to 2013 and Vice President, Business Development for Javelin Pharmaceuticals, Inc. from 2008 to 2009. Mr. Taylor holds a Bachelor of Science in Biological Sciences from Clemson University and a Master of Science in Technology Management from the University of Pennsylvania.

We believe that Mr. Taylor’s qualifications to sit on our board of directors include his significant public company management and board experience and knowledge of our industry.

Information about our Executive Officers

Our currently-serving executive officers and their respective ages and positions are described below. Our executive officers serve until they resign, or the board terminates their position.


Name	Age	Position
John Taylor*	53	President and Chief Executive Officer
John J. Kirby	51	Chief Financial Officer
Andy Jordan	75	Chief Strategy Officer
Dr. Carl Kraus	54	Chief Medical Officer
Daniel Salain	55	Chief Operating Officer

* Mr. Taylor is a member of our board of directors. See "Information about our Directors" above for more information about Mr. Taylor.

John J. Kirby has served as the Chief Financial Officer of Idera since 2019. Mr. Kirby joined the Company in 2015 as our Vice President of Corporate Accounting. He served as Vice President of Finance from 2018 to 2019 and has served as Senior Vice President and Chief Financial Officer since 2019 (and as principal financial officer and principal accounting officer since 2018). Prior to joining us, Mr. Kirby served as Assistant Controller at Endo Pharmaceuticals, Inc. from 2014 to 2015. From 2012 to 2014, Mr. Kirby served as Vice President, Chief Accounting Officer and Corporate Controller at ViroPharma Incorporated, which was acquired by Shire Plc in 2014. Mr. Kirby began his career at KPMG, LLP in its Healthcare and Life Science Practice and served as a Regional Audit Director at AstraZeneca Pharmaceuticals L.P. prior to joining ViroPharma Incorporated. Mr. Kirby received his B.S. in Accountancy from Villanova University and is a licensed certified public accountant in the Commonwealth of Pennsylvania. Mr. Kirby has also served on the board of trustees of the Delaware Museum of Nature & Science (formerly the Delaware Museum of Natural History) since 2018.

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Andy Jordan was appointed as the Chief Strategy Officer of the Company in connection with the closing of the Aceragen Acquisition. Previously, Mr. Jordan served as the Chief Financial Officer of Legacy Aceragen from 2021 to September 2022. Prior to joining Legacy Aceragen, Mr. Jordan founded AR Jordan Consulting, working primarily with biotechnology companies, focusing on bringing to market drugs to treat orphan/rare diseases, serving as its principal from 2008 to 2021. Prior to this, Mr. Jordan held executive-level positions with Guilford Pharmaceuticals, Inc., Odyssey Pharmaceuticals, Inc., and InfraReDx, Inc. Mr. Jordan served as a member of the board of directors for Gemin X BioPharmaceuticals, Inc. from 2003 to 2011 and Spyryx Biosciences, Inc. from 2013 to 2018. Mr. Jordan holds a Bachelor of Arts in Liberal Arts from Rutgers University and an M.B.A. in Professional Accounting from Rutgers University — Newark.

Dr. Carl Kraus was appointed as the Chief Medical Officer of the Company in connection with the closing of the Aceragen Acquisition. Prior to this appointment, Dr. Kraus served as the Chief Medical Officer of Legacy Aceragen from 2021 to September 2022. Prior to this, in 2017, Dr. Kraus founded Arrevus, Inc. (“Arrevus”), a clinical-stage biotechnology company developing novel therapies for orphan diseases, and served as its Chief Executive Officer from 2017 to 2020. Prior to founding Arrevus, Dr. Kraus served as the Chief Medical Officer of Nanotherapeutics, Inc. from 2013 to 2017, the Vice President, Medical Affairs, Risk Management/REMS of Medscape, LLC from 2010 to 2013, and the Vice President, Infectious Diseases, Scientific Affairs of PRA International from 2008 to 2010. Prior to joining industry, Dr. Kraus was a medical officer in the Center for Drug Evaluation and Research at the Food & Drug Administration from 2002 to 2005. Dr. Kraus holds a Bachelor of Arts in Biology from Washington University in St. Louis and received his M.D. in Medicine from Washington University School of Medicine in St. Louis.

Daniel Salain was appointed as the Chief Operating Officer of the Company in connection with the closing of the Aceragen Acquisition. Prior to this appointment, Mr. Salain served as Chief Operating Officer of Legacy Aceragen from 2020 to September 2022. Previously, Mr. Salain was the Chief Operating Officer of Graybug Vision, Inc., a clinical-stage biopharmaceutical company focused on developing medicines for ocular diseases, from 2017 to 2020, and was the Senior Vice President, Global Head of Manufacturing & Supply Chain at Ophthotech, a clinical-stage biotechnology company, from 2015 to 2017. Prior to working at Ophthotech, Mr. Salain was the Vice President of Global Operations, Manufacturing & Supply Chain at Aptalis Pharmaceuticals Inc., a company that focused on developing products to treat gastrointestinal diseases and disorders, from 1999 to 2014. Mr. Salain has a Bachelor of Science degree in Chemistry and Marketing from the University of Indianapolis.

Code of Business Conduct and Ethics

We have adopted a written code of business conduct and ethics that applies to our principal executive officer, principal financial officer, principal accounting officer or controller, or persons performing similar functions. We have posted a current copy of the Code of Business Conduct and Ethics in the “Investors — Corporate Governance” section of our website, which is located at www.aceragen.com. We intend to satisfy the disclosure requirements under Item 5.05 of Form 8-K regarding an amendment to, or waiver from, a provision of our code of business conduct and ethics by posting such information on our website at www.aceragen.com

Audit Committee

Our board has established a formal standing audit committee. The current members of our audit committee are Mr. Dougherty (Chair), Dr. Gowen, and Mr. Wooten. Our board has determined that Mr. Dougherty is an “audit committee financial expert” within the meaning of SEC rules and regulations. Each member of the audit committee is independent as defined under applicable rules of the Nasdaq, including the independence requirements contemplated by Rule 10A-3 under the Exchange Act.

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Item 11.

Executive Compensation.

~~The~~In ~~information~~accordance ~~required~~with byItem ~~this~~402(l) ~~item~~of isRegulation ~~incorporated~~S-K, ~~by reference to our Proxy Statement for~~ the ~~2022~~Company ~~Annual~~has ~~Meeting~~elected ~~of Stockholders~~ to beavail ~~filed~~itself ~~with~~of the ~~SEC~~scaled ~~within~~disclosure ~~120~~requirements ~~days~~available ofto ~~the~~smaller ~~fiscal~~reporting ~~year ended December 31, 2021~~companies.

This section discusses the material components of the Company’s executive compensation program for our named executive officers (“NEOs”) for the fiscal year ended December 31, 2022:

●

John C. Taylor, President and Chief Executive Officer⁽¹⁾

●

John J. Kirby, Senior Vice President and Chief Financial Officer

●

Bryant D. Lim, former Chief Business Officer, General Counsel and Secretary⁽²⁾

●

Vincent J. Milano, former President and Chief Executive Officer⁽³⁾

●

Daniel B. Soland, former Senior Vice President and Chief Operating Officer⁽⁴⁾

(1)

Mr. Taylor was appointed as the Company’s President and Chief Executive Officer on September 28, 2022 in connection with the Aceragen Acquisition.

(2)

Mr. Lim was an executive officer for the entire fiscal year ended December 31, 2022. On February 17, 2023, Mr. Lim resigned as the Company’s Senior Vice President, Chief Business Officer and General Counsel.

(3)

Mr. Milano served as the Company’s President and Chief Executive Officer until September 28, 2022.

(4)

Mr. Soland served as the Company’s Senior Vice President and Chief Operating Officer until September 28, 2022.

Summary Compensation Table

The following table sets forth information concerning the compensation of our named executive officers during the fiscal years ended December 31, 2021 and 2022:


NAME AND PRINCIPAL POSITION	YEAR	SALARY ($)	STOCK AWARDS ($)(1)	OPTION AWARDS ($)(2)	NON-EQUITY INCENTIVE PLAN COMPENSATION ($)		ALL OTHER COMPENSATION ($)(3)	TOTAL ($)
John C. Taylor	2022	77,629	—	—	134,541		9,019	221,189
Chief Executive Officer

John J. Kirby	2022	374,015	—	45,670	143,423		295,633	858,741
Chief Financial Officer	2021	365,000	—	182,575	73,000		39,408	659,983

Bryant D. Lim	2022	374,015	—	45,670	143,423		304,028	867,136
Former Chief Business Officer,	2021	365,000	—	182,575	73,000		42,965	663,540
General Counsel, and Secretary

Vincent J. Milano	2022	460,953	—	84,444	—		883,348	1,428,745
Former Chief Executive Officer	2021	600,000	—	340,189	150,000	⁽⁴⁾	44,244	1,134,433

Daniel B. Soland	2022	318,750	—	45,670	—

Filing Compare · Aceragen, Inc.