Recombinant human acid ceramidase (Roivant Sciences)

Company completes preferred stock conversion, implements reverse stock split, and regains Nasdaq compliance Company positioned for success with late-stage rare disease portfolio and multiple clinical milestones in 2023 DURHAM, N.C. and EXTON, Pa., Jan. 17, 2023 (GLOBE NEWSWIRE) -- Idera Pharmaceuticals, Inc. (“Idera”) (Nasdaq: IDRA), a clinical-stage biopharmaceutical company committed to transforming the care of people living with rare pulmonary and rheumatic diseases, today announced the Company has changed its name and symbol to Aceragen, Inc. (“Aceragen,” the “Company,” “we,” “us,” or “our”) and “ACGN”. Additionally, the Company’s stockholders approved the conversion into common stock of the Series Z preferred shares resulting from the previously announced merger with Aceragen and authorized a reverse split of common stock. The Company’s Board of Directors has approved the reverse stock split at a ratio of 1-for-17 shares. As a result of these changes, which will be effective upon the market open on Wednesday, January 18, 2023, the Company will be in compliance with all applicable Nasdaq listing standards. Nasdaq has issued an approval letter confirming Aceragen’s listing. “We are excited to have completed the transformation of Aceragen via our merger with Idera and the subsequent adjustments to our stock and Nasdaq listing. We believe this transition strengthens our portfolio of late-stage clinical assets in cystic fibrosis and Farber disease and aligns with our goal of delivering important therapies for people living with rare diseases,” stated John Taylor, Aceragen’s Chief Executive Officer. “During the course of this year, we anticipate the achievement of significant clinical milestones that include two Phase 2 data read-outs for ACG-701, as well as lifting of the clinical hold and advancing toward the initiation of our Phase 2/3 trial in Farber disease for ACG-801.” “With the positive result of the stockholder vote behind us, I share in John’s excitement and optimism for Aceragen and look forward to the progress that this team will make for patients in need,” added Vincent J. Milano, Chair of Aceragen’s Board of Directors. Clinical Development Overview Aceragen has a portfolio of late-stage clinical assets in cystic fibrosis and Farber disease with clinical milestones anticipated in 2023. ACG-701 for Acute Pulmonary Exacerbations in Cystic Fibrosis ACG-701 is a proprietary oral, loading dose formulation of sodium fusidate being developed as a treatment for acute pulmonary exacerbations (“PEx”) associated with cystic fibrosis (“CF”), a major factor driving lung function decline in people living with CF. Sodium fusidate has an established clinical efficacy and safety pro 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 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. A Phase 2 trial of ACG-701 in CF PEx (the REPRIEVE study), a randomized double-blinded, placebo-controlled study, was initiated in December 2022 at clinical sites in the United States in collaboration with the CF Foundation’s Therapeutic Development Network (TDN). The CF Foundation has also provided funding of $3.5 million in support of the study. If approved, ACG-701 would represent the first product in the United States indicated for the treatment of newly diagnosed CF PEx patients. Data from the REPRIEVE study is expected in 2H 2023. The FDA has granted Orphan Drug Designation, Fast Track and Qualified Infectious Disease Product (QIDP) status to ACG-701 for CF PEx. ACG-701 for Melioidosis Aceragen has also executed an ~$50 million development partnership with the Department of Defense’s Defense Threat Reduction Agency (“DTRA”) to investigate ACG-701 as a potential medical countermeasure for melioidosis, a life-threatening infection caused by the B. pseudomallei pathogen. This program is centered around a Phase 2 trial, the TERRA study (NCT05105035), which is a randomized double-blind, placebo-controlled trial conducted in hospitalized melioidosis patients. TERRA was initiated in May 2022 and continues to actively enroll patients with an independent DMC review planned during Q1 2023 and a data read-out expected in 2H 2023. ACG-801 for Farber Disease ACG-801, recombinant human acid ceramidase, is an investigational biologic in development to be the first-ever enzyme replacement therapy for the treatment of Farber disease, a progressive, severe, and life-threatening lysosomal storage disorder that is caused by the monogenic deficiency of acid ceramidase. The biochemical hallmark of Farber disease is the loss of acid ceramidase enzyme activity leading to abnormal accumulation of ceramide, profound macrophage-driven inflammation and multi-organ disease affecting bone and joints, cartilage, the immune system, central nervous system, and the lungs. Complications of the disease are life threatening, with many patients dying in the first years of life. There are no ceramide-targeted medications currently available that can alter the disease natural history. The Company expects to initiate the ADVANCE clinical study for ACG-801 in Farber disease, a randomized, double-blind, placebo-controlled, first-in-human study, in the first quarter of 2024 with data expected in the first quarter of 2025. Due to the ultra-rare nature of Farber disease, if successful, this study has the potential to support registration of the product. The FDA has granted Orphan Drug, Fast Track, and Rare Pediatric Disease designations for ACG-801. Rare pediatric disease designation permits priority review voucher eligibility, upon FDA marketing authorization. Clinical Milestone Summary ACG-701 - REPRIEVE study in CF PEx, data expected 2H 2023 ACG-701 – TERRA study in melioidosis, data expected 2H 2023 ACG-801 – ADVANCE study in Farber disease, initiation expected in Q1 2024 Projected cash available is expected to provide the Company with capital runway into Q3’2023. About Aceragen, Inc. Aceragen is a clinical-stage biopharmaceutical company committed to transforming the care of people living with rare pulmonary and rheumatic diseases. Our portfolio includes late-stage programs based on well-established biological principles that we are developing to be innovative therapeutics capable of addressing the unmet medical needs of individuals living with rare diseases. To learn more about us and our programs, please visit Aceragen.com. Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements, other than statements of historical fact, included or incorporated in this press release, 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 Company does not assume any obligation to update any forward-looking statements and it disclaims 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. Please direct questions to: John Kirby Chief Financial Officer Aceragen, Inc. jkirby@aceragen.com

EXTON, Pa. and DURHAM, N.C., Nov. 14, 2022 (GLOBE NEWSWIRE) -- Idera Pharmaceuticals, Inc. (“Idera,” the “Company,” “we,” “us,” or “our”) (Nasdaq: IDRA) today reported its consolidated financial and operational results for the third quarter ended September 30, 2022. “We are pleased to have completed the merger of Idera and Aceragen at the end of the third quarter, better enabling us to deliver important therapies for people living with rare diseases,” stated John Taylor, Idera’s Chief Executive Officer. “Our resulting cash position is expected to provide runway into Q3 2023 and fund the advancement of our pipeline, including ACG-701 and ACG-801, through important anticipated 2023 clinical milestones. Our team continues to execute on the integration effort and diligently progress toward our near-term clinical milestones, which include initiation of our cystic fibrosis (CF) and Farber disease clinical trials and anticipated data from our melioidosis study.” Clinical Development Updates REPRIEVE Study in Cystic Fibrosis: Randomized, double-blind, placebo-controlled Phase 2 study evaluating treatment with ACG-701, a proprietary oral formulation of sodium fusidate, in newly diagnosed pulmonary exacerbations in CF patients. The REPRIEVE study is on track for initiation by the end of 2022, with data anticipated in Q3 2023 TERRA Study for Melioidosis: Randomized, double-blind, placebo-controlled Phase 2 study evaluating ACG-701 in hospitalized melioidosis patients. The independent Data Monitoring Committee (DMC) for the TERRA Study is expected to meet by the end of 2022 to recommend whether the study should continue as planned or, if efficacy and safety data are compelling, to be immediately unblinded for full analysis. This DMC assessment is intended to be in lieu of the interim analysis originally anticipated in Q1 2023. ADVANCE Study for Farber Disease: Randomized, double-blind, placebo-controlled, first-in-human, Phase 2 study of ACG-801 in patients with Farber disease. The ADVANCE study is on track for initiation in the second half of 2023. Corporate Updates Since June 30, 2022, the following corporate updates were announced: As previously disclosed, on September 28, 2022, the Company announced its acquisition, via a stock-for-stock transaction, of Aceragen, Inc., a private biotechnology company addressing severe, rare pulmonary and rheumatic diseases for which there is significant medical need and limited or no available treatments. The combined cash of the two companies is expected to provide runway into Q3 2023, funding the advancement of ACG-701 and ACG-801 through important 2023 clinical milestones. In connection with the acquisition, the Company announced several leadership and Board changes: John Taylor, the former Chief Executive Officer of Aceragen, was named Chief Executive Officer of the Company. Additional management team members are Carl Kraus, Aceragen’s former Chief Medical Officer, who now serves in that role for Idera; Bryant Lim, who continues in his role as Idera’s Chief Business Officer and General Counsel; Daniel Salain, Aceragen’s former Chief Operating Officer, who serves in that role for Idera; Andy Jordan, Aceragen’s former Chief Financial Officer, who was appointed Chief Strategy Officer for Idera; and John Kirby, who continues in his role as Idera’s Chief Financial Officer.Vincent Milano, Idera’s former Chief Executive Officer, was named Chair of the Board of Directors for the Company. Idera Board members Cristina Csimma, Pharm. D., M.H.P., James Geraghty, Maxine Gowen, Ph.D., and Michael Dougherty continue in their positions and are joined by John Taylor and Ron Wooten, Senior Founding Partner, NovaQuest Capital Management LLC. Mr. Taylor and Mr. Wooten previously served on Aceragen’s board. Third Quarter Financial Results The Company’s pro forma cash position as of September 30, 2022 was $26.8 million. Research and development expenses for the three months ended September 30, 2022 totaled $1.5 million, compared to $3.5 million for the same period in 2021. General and administrative expenses for the three months ended September 30, 2022 totaled $2.3 million, compared to $2.3 million for the same period in 2021. Restructuring costs for the three months ended September 30, 2022 totaled approximately $2.8 million, compared to $0.1 million for the same period in 2021, and relate to a reduction in force due to our acquisition of Aceragen. Merger related costs for the three months ended September 30, 2022 totaled $2.8 million. The Company recorded an income tax benefit of $6.0 million in the three months ended September 30, 2022. As a result of the factors above, net loss applicable to common stockholders for the three months ended September 30, 2022 was $3.1 million or $0.06 per basic and diluted share compared to net loss applicable to common stockholders of $6.0 million or $0.11 per basic and diluted share for the same period in 2021. About Idera PharmaceuticalsIdera is a clinical-stage biopharmaceutical company committed to transforming the care of people living with rare pulmonary and rheumatic diseases. Our portfolio includes late-stage programs that we are developing to be innovative therapeutics capable of addressing the unmet medical needs of these individuals. To learn more about Idera, visit IderaPharma.com. About ACG-701 for Acute Pulmonary ExacerbationsACG-701 is a proprietary oral formulation of sodium fusidate being developed as a potential treatment for acute pulmonary exacerbations (“PEx”) associated with cystic fibrosis (“CF”) and for melioidosis, a life-threatening infection caused by the B. pseudomallei pathogen. The Phase 2 trial of ACG-701 in CF PEx (the REPRIEVE study) is expected to initiate during Q4 2022 at clinical sites in the United States in collaboration with the CF Foundation’s Therapeutic Development Network (TDN). The CF Foundation has also graciously provided a funding award in support of the study. If approved, ACG-701 would represent the first product in the United States indicated for the treatment of CF PEx, a major factor driving lung function decline in people living with CF. Initial data from the REPRIEVE study is expected in Q3 2023. The active component of ACG-701, sodium fusidate, has the opportunity for new chemical entity (NCE) status in the US, as sodium fusidate has not been previously approved by the FDA. Despite this, the compound 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. The FDA has assigned Orphan, Fast Track, and Qualified Infectious Disease Product status to ACG-701 for CF PEx. The melioidosis clinical program for ACG-701 is supported by funding up to $49.7 million, of which $13.2 million has been received by the Company, from the Defense Threat Reduction Agency (“DTRA”) to investigate our product as a potential medical countermeasure for this disease. This trial, the TERRA study (NCT05105035), is currently enrolling, targeting review by a independent DMC during Q4 2022. This clinical study is distinct as it is the first randomized double-blinded, placebo-controlled trial to ever be run for melioidosis. Complete Phase 2 data are expected by Q2 2023. About ACG-801 for Farber DiseaseACG-801, recombinant human acid ceramidase, is an investigational biologic under development to be the first-ever enzyme replacement therapy for the treatment of acid ceramidase deficiency, also called Farber disease, a progressive, severe and life-threatening lysosomal storage disorder. The condition is hallmarked by loss of acid ceramidase enzyme activity leading to abnormal accumulation of ceramide, profound macrophage-driven inflammation and multi-organ disease affecting bone and joints, cartilage, the immune system, central nervous system, and the lungs. Many of these patients die from complications of the disease, with the most severely affected patients dying in the first years of life. There are no Farber disease-specific medications currently available that can alter the natural history of the disease. The Company expects to initiate the ADVANCE clinical study for ACG-801 in Farber disease in 2H 2023 with data expected in Q1 2024. Due to the ultra-rare nature of Farber disease, this study has the potential to be registrational. The FDA has granted Orphan, Fast Track, and Rare Pediatric Disease designations for ACG-801, which may be eligible for a priority review voucher (PRV) upon approval by FDA. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements, other than statements of historical fact, included or incorporated in this press release, 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 the Company’s current expectations and projections about future events and various assumptions. Idera cannot guarantee that it will achieve the plans, intentions, or expectations disclosed in its forward-looking statements and you should not place undue reliance on the Company's forward-looking statements. These forward-looking statements involve known and unknown risks, uncertainties, and other factors, which may be beyond Idera’s control, and which may cause the actual results, performance, or achievements of the Company 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 Idera's actual results to differ materially from those indicated or implied by its forward-looking statements including, without limitation: whether the Company will be able to successfully integrate the Aceragen operations; whether the Company’s stockholders approve the conversion of the Series Z Preferred Stock; whether the Company’s cash resources will be sufficient to fund the Company’s continuing operations and the newly acquired Aceragen operations, including the liabilities of Aceragen incurred in connection with the completion of the transactions; whether the Company’s 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, if the Company's products receive approval, they will be successfully distributed and marketed; whether the Company's collaborations will be successful; and whether the Company will be able to comply with the continued listing requirements of the Nasdaq Capital Market. 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 filings and reports filed with Securities and Exchange Commission. While Idera may elect to do so at some point in the future, the Company does not assume any obligation to update any forward-looking statements and it disclaims 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. Idera Pharmaceuticals, Inc. Statements of Operations(In thousands, except per share data) Three Months Ended Nine Months Ended September 30 September 30 2022 2021 2022 2021 Government contracts revenue$49 $- $49 $- Operating expenses: Research and development 1,470 3,507 5,960 14,271 General and administrative 2,268 2,331 7,325 7,959 Acquisition-related costs 2,836 - 2,836 Restructuring and other costs 2,802 130 2,802 1,322 Total operating expenses 9,376 5,968 18,923 23,552 Loss from operations (9,327) (5,968) (18,874) (23,552) Other income (expense) Warrant revaluation gain 116 - 116 6,983 Future tranche right revaluation gain - - - 118,803 Other income (expense), net 73 3 135 (24)(Loss) income before income tax benefit$(9,138) $(5,965) $(18,623) $102,210 Income tax benefit 6,039 - 6,039 - Net (loss) income$(3,099) $(5,965) $(12,584) $102,210 Undistributed earnings to preferred stockholders - - - - Net income (loss) applicable to common stockholders$(3,099) $(5,965) $(12,584) $102,210 Net income (loss) applicable to common stockholders — Basic$(3,099) $(5,965) $(12,584) $100,574 — Diluted$(3,099) $(5,965) $(12,584) $(23,576)Net income (loss) per share applicable to common stockholders — Basic$(0.06) $(0.11) $(0.24) $2.10 — Diluted$(0.06) $(0.11) $(0.24) $(0.46) Weighted-average number of common shares used in computing net income (loss) per share applicable to common stockholders — Basic 53,286 52,740 53,052 47,990 — Diluted 53,286 52,740 53,052 51,613 Idera Pharmaceuticals, Inc.Balance Sheet Data(In thousands) September 30, December 31, 2022 2021 Cash and cash equivalents$26,795 $32,545 Other assets 76,887 2,319 Total assets$103,682 $34,864 Total Redeemable$29,175 $- Total liabilities 53,739 5,411 Total stockholders' equity (deficit) 20,768 29,453 Total liabilities, convertible redeemable preferred stock and stockholders' equity (deficit)$103,682 $34,864