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Date of Report (Date of earliest event reported): January 18, 2023


Aceragen, Inc.

(Exact name of registrant as specified in its charter)



(State or Other

Jurisdiction of



(Commission File



(I.R.S. Employer

Identification No.)


505 Eagleview Blvd., Suite 212    
Exton, Pennsylvania   19341
(Address of Principal Executive Offices)   (Zip Code)


Registrant’s telephone number, including area code: (484) 348-1600


Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):


¨ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425).
¨ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12).
¨ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240-14d-2(b)).
¨ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240-13e-4(c)).


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


Title of each class   Trading
  Name of each
exchange on which registered
Common Stock, par value $0.001 per share   IDRA   Nasdaq Capital Market


Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):


Indicate by check mark whether the registrant is an emerging growth company as defined in as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).


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. ¨







Item 7.01Regulation FD Disclosure.


On January 18, 2023, Aceragen, Inc. (formerly known as Idera Pharmaceuticals, Inc.) (the “Company,” “we,” “us,” and “our”) uploaded a presentation to its website, www.aceragen.com, discussing the state of the Company. We may rely on all or part of this presentation any time we are discussing the current state of the Company in communications with investors or at conferences. A copy of the presentation is attached to this Current Report on Form 8-K as Exhibit 99.1 (the “Presentation”).


The information contained in the Presentation is summary information that is intended to be considered in the context of the Company’s Securities and Exchange Commission (“SEC”) filings and other public announcements that the Company may make, by press release or otherwise, from time to time. The Company undertakes no duty or obligation to publicly update or revise the information contained in this report, although it may do so from time to time as its management believes is warranted. Any such updating may be made through the filing of other reports or documents with the SEC, through press releases or through other public disclosure.


The information in this Current Report on Form 8-K, including Exhibit 99.1 attached hereto, shall not be deemed to be “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, and shall not be deemed to be incorporated by reference into any of the Company’s filings under the Securities Act of 1933, as amended, whether made before or after the date hereof and regardless of any general incorporation language in such filings, except to the extent expressly set forth by specific reference in such a filing.


The website address set forth above is included as an inactive textual reference only. The information contained on the website referenced herein is not incorporated into this Current Report on Form 8-K.


Item 9.01.Financial Statements and Exhibits.




Exhibit No.   Exhibit Name
99.1   Investor Presentation dated January 18, 2023.
104   Cover Page Interactive Data File (embedded within the Inline XBRL document).






Pursuant to the requirements of the Securities Exchange Act of 1934, the Registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.


  By: /s/ Bryant D. Lim
    Bryant D. Lim
    Chief Business Officer and General Counsel
Dated: January 18, 2023  




Exhibit 99.1


Therapeutics for Rare Pulmonary & Rheumatic Diseases Nasdaq: ACGN January 2023


Important Information The information provided in this presentation pertaining to Aceragen , Inc . (“ Aceragen ” or “Company”) is for informational purposes only to assist interested parties in making their own independent evaluation . Information contained in this presentation should not be relied upon as advice to buy or sell any securities of Aceragen . You should not construe the contents of this presentation as legal, tax, accounting or investment advice or a recommendation . You should consult your own counsel and tax and financial advisors as to legal and related matters concerning the matters described herein . No legally binding obligations will be created, implied, or inferred from this presentation or the information contained herein . The products outlined in this presentation are still under development . The features of any final product may be different from the features of any product under development described herein, and nothing should be construed as a commitment by Aceragen . While the information in this presentation is believed to be accurate, Aceragen and their respective agents, advisors, directors, officers, employees and stockholders make no representation or warranties, expressed or implied, as to the accuracy, completeness or reliability of such information . Neither Aceragen , nor any of its affiliates, agents, advisors, directors, officers, employees and stockholders shall have any liability whatsoever, under contract, tort, trust or otherwise, to you or any person resulting from the use of the information in this presentation by you or any of your representatives or for omissions from the information in this presentation . We reserve the right to amend or replace the information contained herein, in part or entirely, at any time, and undertakes no obligation to provide you with access to the amended information or to notify you thereof . The distribution of this presentation may also be restricted by law and persons into whose possession this presentation comes should inform themselves about and observe any such restrictions . The presentation is not directed to any person in any jurisdiction where (by reason of that person’s nationality, residence or otherwise) the publication or availability of the presentation is prohibited . Persons in respect of whom such prohibitions apply must not access the presentation . Forward - Looking Statements This presentation contains forward - looking statements within the meaning of Section 27 A of the Securities Act of 1933 , as amended, and Section 21 E of the Securities Exchange Act of 1934 , as amended . All statements, other than statements of historical fact, included or incorporated in this presentation, including, without limitation, statements regarding the Company's new development opportunities, clinical trials and studies, product designation and/or status, financial position, funding for continued operations, cash reserves, projected costs, prospects, clinical trials, plans, expectations, strategies, projections 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,” and "would" and similar expressions are intended to identify forward - looking statements, although not all forward - looking statements contain these identifying words . These forward - looking statements are predictions based on our current expectations and projections about future events and various assumptions . We cannot guarantee that we will 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 our actual results, performance, or achievements to differ materially from future results, performance, or achievements expressed or implied by such forward - looking statements . There are a number of important factors that could cause our actual results to differ materially from those indicated or implied by its forward - looking statements including, without limitation : whether we will be able to successfully integrate the acquired operations ; whether our cash resources will be sufficient to fund continuing operations ; and newly acquired operations, ; whether our products will advance into or through the clinical trial process when anticipated or at all or warrant submission for regulatory approval ; whether such products will receive approval from the U . S . Food and Drug Administration or equivalent foreign regulatory agencies ; whether our products receive approval, they will be successfully distributed and marketed ; and whether our collaborations will be successful . All forward - looking statements included in this press release are made as of the date hereof and are expressly qualified in their entirety by this cautionary notice, including, without limitation, those risks and uncertainties described in the Company’s Annual Report on Form 10 - K for the year ended December 31 , 2021 , and otherwise in the Company’s subsequent filings and reports filed with Securities and Exchange Commission . The forward - looking statements contained in this presentation are expressly qualified by this cautionary statement . Except as required by law, we undertake no obligation to update or revise publicly any forward - looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events . Readers are cautioned not to place undue reliance on forward - looking statements . This presentation does not constitute an offer to sell, or the solicitation of an offer to buy, any securities, nor shall there be any sale of securities in any states or jurisdictions in which such offer, solicitation or sale would be unlawful . 2 © 2023 ACERAGEN


Executive Leadership with Significant Rare Disease Experience John Taylor Chief Executive Officer Biopharma executive and with significant experience financing and building therapeutic companies primarily focused on products for orphan and rare diseases. Responsible for >$900M in executed transactions and investments over 25 - year career. Dan Salain Chief Operating Officer 30 years pharma leadership experience including product development, manufacturing, QA/QC, distribution, business development, and corporate operations. Developed and launched over 30 products globally. Carl Kraus Chief Medical Officer Infectious disease physician with 20+ years of clinical experience treating patients and 15 years CMO experience in related drug development. Former medical officer in the Office of Antimicrobial Products at FDA. Andy Jordan Chief Strategy Officer 35+ years executive experience in finance, accounting, and corporate governance including 20 - years at KPMG and as CFO of public and private biopharma companies. Led multiple IPOs and raised over $600M. Bryant Lim Chief Business Officer & General Counsel 25+ years legal and business experience across multiple small & large commercial - and development - stage public pharma companies. Former Chief Compliance Officer of Incyte Corp. and CBO/GC Idera. John Kirby Chief Financial Officer More than 25 years of public - company finance and business experience from his roles with small to large - sized public pharma companies, including ViroPharma and AstraZeneca, and in public accounting with KPMG. Responsible for raising over $175M. 3 © 2023 ACERAGEN


4 © 2023 ACERAGEN Finance and Equity ▪ Cash balance of ~$26.8M as of September 30, 2022 ▪ C ash available expected to provide capital runway for the Company into Q3 2023 ▪ Common shares outstanding as of 1/18/2023: ~8.5M ▪ Share count is post conversion of Series Z preferred and post 1 - for - 17 reverse split of the Company’s issued and outstanding shares


Creating Long - term Value Via Deal - Making Awarded $45M in funding for the development of ACG - 701 for the treatment of melioidosis from OCT 2021 Awarded $3.5M development award for ACG - 701 for the treatment of CF from JAN 2022 Aceragen merges with to form a Nasdaq - listed rare disease company SEPT 2022 Aceragen launches with $35M in product financing from and acquires worldwide rights to ACG - 801 from MARCH 2021 Aceragen acquires and ACG - 701 as a treatment for cystic fibrosis pulmonary exacerbations OCT 2021 5 © 2023 ACERAGEN


Rare Disease Company PULMONARY & RHEUMATIC Severe disease, efficient development Current portfolio includes Farber and cystic fibrosis MARKET POTENTIAL Estimated $850 million Concentrated commercial effort 6 © 2023 ACERAGEN INNOVATION GAP Potential value creation by meeting patient needs No competition in respective indications


Advanced Clinical Portfolio 2023 2025 2024 CF Ph2 data Melio Ph2 data 2023 2025 2024 Farber Ph2/3 data ACG - 701 (sodium fusidate) Cystic fibrosis ( acute exacerbations ) Melioidosis ACG - 801 (recombinant acid ceramidase) Farber disease REPRIEVE Study ADVANCE Study TERRA Study 7 © 2023 ACERAGEN


Target Product Profile Differentiated oral product for acute pulmonary exacerbations with anti - inflammatory, anti - infective, and mucin inhibitory activity Initial Indications Acute CF pulmonary exacerbations – Data expected 2H’2023 Melioidosis – Data expected 2H’2023 Regulatory Status / Designations NCE status, Orphan, Fast Track, and QIDP Estimated Market Potential >$500M ACG - 701 8 © 2023 ACERAGEN


CF Pulmonary Exacerbations (CF PEx) ▪ CF PEx are characterized as respiratory events accompanied by an acute decrease in lung function ▪ The 35,000 CF patients in the US experience ~1 - 2 exacerbations/year ▪ ~70% of the US CF population is at risk for MRSA infections ▪ Exacerbations and related complications account for nearly two - thirds of morbidity and mortality for CF patients ▪ No therapies FDA approved to treat acute CF PEx Peak sales potential in excess of $500M 9 © 2023 ACERAGEN 0 0.5 1 1.5 2 2.5 Before After Number of infection related visits in the 15 - weeks prior to and after Trikafta initiation per patient Infection - related Visits (N = 389) Source: Miller et. al. 2022 *Data in Miller et. al. converted to a per patient number by dividing total visits by number of patients


ACG - 701: Loading Dose Formulation Uniquely Suited for Severe Pulmonary Disease ▪ Loading dose achieves IV - like blood levels within 24 hours ▪ Dosing regimen enhances potency seen in prior clinical use ▪ 3X increase in potency for Staph/MRSA ▪ Extensive safety database (positive Phase 3) ▪ Potentially first product approved to treat acute PEx , addresses major symptoms ▪ Anti - inflammatory ▪ Anti - infective ▪ Mucin suppression ▪ Potential for 12 years of exclusivity from first approval in the US (NCE/Orphan Status/QIDP) and 10 years of exclusivity in Japan and EU (Orphan Status) 10 © 2023 ACERAGEN Source: Fernandes. Cold Spring Harbor 2016


ACG - 701: Patented Sodium F usidate F ormulation Phase 2 Phase 1 Phase 3 BE and Food Effects Multiple Dose Loading Dose Study DDI ABSSSI ABSSSI BJI PJI ABSSSI : Acute bacterial skin and skin structure infections PJI : Prosthetic Joint Infections BJI : Bone & joint infections ACG - 701 has demonstrated safety and efficacy in 650+ patients across multiple indications CF PEx Melioidosis/ CF PEx Sodium fusidate has unique activity at low pH 0.01 0.1 1 10 100 5 5.5 6 6.5 7 7.5 MIC (µg/mL) pH S. aureus B. anthracis F. tularensis Y. pestis B. mallei B. pseudomallei 11 © 2023 ACERAGEN


The REPRIEVE Study in CF PEx THE REPRIEVE STUDY DESIGN ▪ Randomized, double - blind, placebo - controlled study for newly diagnosed pulmonary exacerbations in CF patients ▪ Two - week oral BID treatment plus two - week follow - up ▪ Endpoints: CRISS, FEV1, and antimicrobial regimen changes through Day 14 in a single statistical measure (DOOR) 80 Adult CF pts ACG - 701 + OBT 25 sites Placebo + OBT 4Q 1Q 2Q 3Q 4Q FPI Phase 2 Enrollment 2022 2023 2024 CRISS: difficulty breathing, cough, cough up mucus, chest tightness, wheeze, feeling feverish, tired, and chills/sweats 12 © 2023 ACERAGEN DATA


The TERRA Study in Melioidosis is Underway 1Q 2022 2Q 3Q 4Q 1Q 2023 2Q 3Q 4Q FPI DATA DMC Review ▪ Randomized, double - blind, placebo - controlled study in hospitalized melioidosis patients ▪ Two - week BID dosing with two - week follow - up ▪ Endpoints: mortality, organ failure, sepsis and treatment modifications through Day 14 in a single statistical measure (DOOR) 125 Adult melioidosis pts ACG - 701 + SOC 12 sites Placebo + SOC The TERRA Study Design Phase 2 Enrollment 1Q 2024 SE Asia Monsoon Season SE Asia Monsoon Season 13 © 2023 ACERAGEN


ACG - 701: Key Points ▪ Compound has established efficacy and safety profile ▪ Loading dose designed to enhance performance in severe disease ▪ Extensive safety database for product ▪ Rare disease business model ▪ ~60,000 exacerbations/ yr in established CF market ▪ Total addressable market for acute exacerbations = multi - billion dollar potential ▪ 12 years of market exclusivity anticipated from approval 14 © 2023 ACERAGEN $500M sales potential in CF and melioidosis


ACG - 801 Target Product Profile Disease - modifying enzyme replacement therapy (ERT) addressing enzyme deficiency and ceramide accumulation in Farber disease patients Initial Indication Farber disease (monogenic loss of function) – Study start expected 1Q’2024 Regulatory Status / Designations Orphan, Fast Track, Biologic and Rare Pediatric Disease Annual Peak Sales Estimate >$350M 15 © 2023 ACERAGEN


Farber Disease ▪ Farber is an enzyme deficiency resulting in accumulation of ceramide, highly inflammatory lipid ▪ Disease severity is a spectrum; severe phenotype results in death before age 2 ▪ Respiratory failure common cause of death ▪ Current treatments relieve some pain but don’t impact disease progression ▪ Worldwide prevalence estimated to be 1,000 - 1,500 patients (similar to MPS VI )​ 16 © 2023 ACERAGEN


ACG - 801 for Farber Disease Farber mouse + ACG - 801 (resolution of macrophage infiltrates) Farber mouse untreated (black arrows: macrophage infiltrates) WT mouse (bone marrow) ACG - 801 treatment of Farber mice results in: x Reduction of ceramide deposits in target tissues​ x Reduction of inflammatory biomarkers/cell types to wild type levels​ x Amelioration of bone, joint, and soft tissue lesions (pathophysiology same as clinical endpoint) ​ x Additional 3 - week lifespan in newborn mice Source: ACG - 801 Investigator’s Brochure ACG - 801 EFFECTIVE IN FARBER MOUSE MODEL 17 © 2023 ACERAGEN


ACG - 801 for Farber Disease THE ADVANCE STUDY DESIGN ▪ Randomized, double - blind, placebo - controlled, first - in - human Phase 2/3 study in Farber disease patients ▪ Systemic IV infusion, every other week ▪ Endpoints are nodule changes and patient - specific disease burden improvement (e.g., pain, mobility, impact score) through week 28 15 Farber Ped & Adult Pts ACG - 801 7 sites Placebo 2023 2024 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q FPI 1Q 2025 FPI PIVOTAL (the ADVANCE study) 1Q 18 © 2023 ACERAGEN DATA


ACG - 801: Enzyme Replacement Therapy For Enzyme Deficiencies Have Good History Of Success Therapy (Disease) Pre - Clinical Effective Dose Aldurazyme (MPS I) 0.5 - 2mg/kg weekly (canine) Fabrazyme (Fabry) 0.3 - 3mg/kg bi - weekly (rodent) Naglazyme (MPS VI) 1 - 2mg/kg weekly (feline) Myozyme ( Pompe ) 10 - 100mg/kg bi - weekly (rodent) Kanuma (LALD) 0.35 - 3mg/kg bi - weekly (rodent) ACG - 801 (Farber) 1 - 10mg/kg bi - weekly (rodent) ▪ ACG - 801 has potential to address disease causing enzyme deficiency ▪ Historically, animal data have been highly predictive for ERT development in monogenic LSDs ▪ Global IP portfolio ▪ No known competitors PRECLINICAL EFFICACY HAS BEEN PREDICTIVE OF CLINICAL EFFICACY FOR ERTs >$350M in worldwide annual peak sales estimated for ACG - 801 for Farber disease 19 © 2023 ACERAGEN


20 © 2023 Aceragen ▪ 3 clinical programs with near - term inflection points, high unmet need ▪ Annual sales potential exceeding $850M ▪ Full commercial ownership of programs; centers of excellence model Late - Stage Clinical Portfolio Management Team with Proven Rare Disease Expertise ▪ Rare product leadership and commercialization ( Cinryze , Kanuma , Zenpep ) ▪ Collective experience that spans all aspects of product development Capital Efficient Development ▪ Strategic, non - dilutive funding ▪ NovaQuest, DoD and CF Foundation partnerships Key Takeaways