Delandistrogene moxeparvovec

Approximately 25 non-ambulatory participants will receive sirolimus as part of the regimen in Cohort 8 of the ENDEAVOR study, which is expected to begin before the end of the year The enhanced immunosuppressive regimen is designed to mitigate the risk of acute liver injury (ALI) and acute liver failure (ALF) associated with AAV gene therapy Decisions regarding resuming commercial dosing for this population will be made in collaboration with FDA after reviewing study data CAMBRIDGE, Mass. --(BUSINESS WIRE)--Nov. 25, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved dosing in Cohort 8 of ENDEAVOR (Study 9001-103). The purpose of Cohort 8 is to evaluate the use of an enhanced immunosuppressive regimen as part of treatment with ELEVIDYS (delandistrogene moxeparvovec-rokl) for non-ambulant individuals with Duchenne muscular dystrophy. Data from Cohort 8 will be used to determine whether administering sirolimus prior to and after ELEVIDYS infusion can help reduce acute liver injury (ALI), a known risk associated with AAV gene therapy. The cohort will enroll approximately 25 participants in the U.S. who are non-ambulatory. The immunosuppression regimen will include 14 days of peri-infusion sirolimus dosing (prior to ELEVIDYS administration) and will continue for 12 weeks after ELEVIDYS administration. Primary endpoints include incidence of ALI and ELEVIDYS-dystrophin expression at 12 weeks. The approach is based on preclinical data and shaped by real-world clinical experience, including guidance from independent specialists in Duchenne and liver health. “We remain deeply committed to serving all individuals living with Duchenne, including those who have lost the ability to walk. Guided by real-world experience, external clinician expertise, and FDA input, Cohort 8 of the ENDEAVOR study will evaluate integrating sirolimus into our immunosuppression approach, with the goals of mitigating the risk of acute liver injury and restoring access for non-ambulant individuals living with Duchenne,” said Louise Rodino-Klapac , Ph.D., president of research & development and technical operations. “We plan to initiate Cohort 8 by the end of this year and pending enrollment, complete primary endpoint data collection in the second half of 2026.” ELEVIDYS is the only approved gene therapy for Duchenne. To date, ELEVIDYS has been administered to over 1,100 patients globally in clinical and real-world settings. Sarepta continues to work closely with the FDA to ensure that all regulatory decisions are grounded in science and the best interests of patients who face a rare, irreversibly progressive and ultimately fatal disease. ELEVIDYS remains available as appropriate to ambulatory individuals ages 4 and over per an updated label announced earlier this month. About ENDEAVOR (Study 9001-103) Study SRP-9001-103, also known as ENDEAVOR, is an open-label, Phase 1b study assessing the expression and safety of ELEVIDYS (delandistrogene moxeparvovec) in multiple cohorts of male patients with Duchenne. The study has enrolled 55 participants across 7 cohorts, and has dosed younger ambulatory individuals aged 2-7 at time of treatment, and older ambulant individuals and non-ambulant individuals. The primary endpoint in ENDEAVOR is the change from baseline in the quantity of ELEVIDYS micro-dystrophin protein expression measured by western blot at 12 weeks. Secondary outcome measures include change from baseline in micro-dystrophin expression measured by percent dystrophin positive fibers at 12 weeks. About ELEVIDYS (delandistrogene moxeparvovec-rokl) ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. ELEVIDYS is indicated for the treatment of ambulatory patients 4 years of age and older with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene. Limitations of Use ELEVIDYS is not recommended in patients with: Preexisting liver impairment (defined as gamma-glutamyl transferase [GGT] > 2 x upper limit of normal or total bilirubin > the upper limit of normal not due to Gilbert’s syndrome) or active hepatic viral infection due to the high risk of acute serious liver injury and acute liver failure. Recent vaccination (within 4 weeks of treatment) due to immunogenicity and potential safety concerns. Active or recent (within 4 weeks) infections due to safety concerns. IMPORTANT SAFETY INFORMATION BOXED WARNING: Acute Serious Liver Injury and Acute Liver Failure Acute serious liver injury, including life-threatening and fatal acute liver failure, has occurred. Patients with preexisting liver impairment may be at higher risk. Prior to infusion, assess liver function by clinical examination and laboratory testing. Administer systemic corticosteroids before and after ELEVIDYS infusion. Continue to monitor liver function weekly for the first 3 months after infusion and continue until results are unremarkable. Instruct patients to maintain proximity to an appropriate healthcare facility, as determined by the healthcare provider, for at least 2 months following ELEVIDYS infusion. Obtain prompt consultation with a specialist (e.g., gastroenterologist or hepatologist) if acute serious liver injury or impending acute liver failure is suspected. CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9, including a deletion of any portion or the entirety of these exons, in the DMD gene. WARNINGS AND PRECAUTIONS: Acute Serious Liver Injury and Acute Liver Failure See Boxed Warning. Acute serious liver injury marked by elevations of liver enzymes (e.g., GGT, ALT) and total bilirubin and acute liver failure has occurred with ELEVIDYS. Onset of the liver injury typically begins within 8 weeks of ELEVIDYS administration. In non-ambulatory patients treated with ELEVIDYS, acute liver failure with fatal outcome has occurred in the clinical and post-marketing settings. Life-threatening mesenteric vein thrombosis, complicated by bowel ischemia and necrosis, and portal hypertension have been reported following acute liver injury associated with ELEVIDYS in a non-ambulatory patient. Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury or acute liver failure. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled. Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. Serious Infections Increased susceptibility to serious infections may occur due to concomitant administration of corticosteroid regimen and additional immunosuppressants, and ELEVIDYS. Serious respiratory infections, including with fatal outcomes, have occurred in patients taking immunosuppressant corticosteroids required for ELEVIDYS administration. Monitor patients for signs and symptoms of infection before and after ELEVIDYS administration and treat appropriately. Administer immunizations according to best clinical practices and immunization guidelines prior to initiation of the corticosteroid regimen required before ELEVIDYS infusion. Avoid administration of ELEVIDYS to patients with active infections. Myocarditis Acute, serious, life-threatening myocarditis and troponin-I elevations have been observed within 24 hours to more than 1 year following ELEVIDYS infusion. If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated, until results return to near baseline levels or stabilize. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath. Advise patients to contact a physician immediately if they experience cardiac symptoms. Infusion-related Reactions Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate. ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available. Discontinue infusion for anaphylaxis. Immune-mediated Myositis Immune-mediated myositis, including serious and life-threatening events, has occurred approximately 1 month following ELEVIDYS infusion. Signs and symptoms include severe muscle weakness, including dysphagia, dyspnea, dysphonia, and hypophonia. Severe to life-threatening immune-mediated myositis has been reported in patients with deletions including portions of exons 1-17 and/or exons 59-71 of the DMD gene. Regardless of genetic mutation, advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, dysphonia, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment based on patient’s clinical presentation and medical history if these symptoms occur. Preexisting Immunity against AAVrh74 In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies. Perform baseline testing for the presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration. ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers ≥1:400. ADVERSE REACTIONS The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, thrombocytopenia, and troponin-I increased. Report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782). Please see the full Prescribing Information for ELEVIDYS, including Boxed Warning and Medication Guide. About Sarepta Therapeutics Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold a leadership position in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit www.sarepta.com or follow us on LinkedIn, X, Instagram and Facebook. Forward-Looking Statements This statement contains “forward-looking statements.” Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believe,” “anticipate,” “plan,” “expect,” “will,” “may,” “intend,” “prepare,” “look,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our future operations, ELEVIDYS, planned clinical trials and the timing of such trials, the potential benefits of an enhanced immunosuppression regimen, and ongoing interactions with FDA related to ELEVIDYS. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials are positive, the results of future research may not be consistent with past positive results, or may fail to meet regulatory approval requirements for the safety and efficacy of our products; our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; we may not be able to comply with all FDA requests in a timely manner or at all; the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business; and those risks identified under the heading “Risk Factors” in our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to review. Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained herein. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law. Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us. View source version on businesswire.com: https://www.businesswire.com/news/home/20251125916203/en/ Investor Contacts: Ian Estepan , 617-274-4052 iestepan@sarepta.com Ryan Wong , 617-800-4112 rwong@sarepta.com Media Contacts: Tracy Sorrentino , 617-301-8566 tsorrentino@sarepta.com Kara Hoeger , 617-710-3898 khoeger@sarepta.com Source: Sarepta Therapeutics, Inc.

Plus, news about Blue Lake, Bambusa, Inhibikase Therapeutics and Olema Oncology: 🛑 Sarepta terminates Elevidys trial with Hansa Biopharma’s imlifidase : The study was examining whether Hansa’s imlifidase, an IgG antibody-cleaving enzyme, could help reduce certain antibodies that prevent patients from receiving the Duchenne muscular dystrophy gene therapy. Sarepta axed the trial due to a “business decision,” according to an update this week to the federal clinical trials database. The study enrolled five patients, against an original target of six. “The study ended but the partnership remains — we’re working with Hansa on next steps for the program,” a Sarepta spokesperson told Endpoints News . The trial was being run in Europe, and was paused earlier this year along with other studies after Sarepta reported the first death of a patient receiving Elevidys. Hansa presented data from three patients on the trial in August, showing that they could receive Elevidys. However, expression levels of the shortened muscle protein expressed by Elevidys was lower than in other trials with the gene therapy. — Kyle LaHucik & Lei Lei Wu ✂️ Moderna cuts three programs: The company announced on Thursday that it is discontinuing two mid-stage vaccines for herpes simplex virus and varicella-zoster virus, as well as an early-stage candidate for glycogen storage disease type 1a. President Stephen Hoge didn’t go into detail on the decisions during an analyst event on Thursday, but said that “it didn’t feel like the commercial opportunity justified the level of investment necessarily.” — Nicole DeFeudis 🔵 Blue Lake gets partial FDA hold lifted on RSV vaccine for kids: The trial hold was placed more than a year ago after suspected cases of vaccine-associated enhanced respiratory disease sprung up in Moderna’s pediatric RSV study. The holds excluded companies from enrolling kids who tested negative for RSV exposure or who were under two years old. A spokesperson from Moderna said the company’s pediatric RSV studies remain on hold. — Max Bayer 💵 Bambusa nabs more funding: The bispecific antibody developer increased its total funding from about $90 million to approximately $140 million. This is with additional support from existing investors like RA Capital, Redmile and KKR’s Dawn Biopharma, CEO Shanshan Xu told Endpoints News . She started Bambusa after serving as a VP at BioNTech. The Boston biotech said it will use the money for its clinical-stage dermatology and respiratory drugs BBT001 and BBT002, and its earlier-stage candidates BBT003 and BBT004 for gastroenterology and rheumatology. — Kyle LaHucik 💰 Inhibikase Therapeutics prices $100M offering: The Delaware biotech priced the offering after saying Thursday night it would move IKT-001 into a global Phase 3 for pulmonary arterial hypertension. The company had $77.3 million at the end of September. The Phase 3 will begin in the first quarter of next year. The biotech’s share price $IKT was up about 16% in pre-market trading on Friday. — Kyle LaHucik 💰 Olema Oncology closes $218.5M offering: The San Francisco biotech is working on treatments for breast cancer and other conditions. The offering was priced at $19 per share $OLMA . The biotech’s shares closed up 13% to $22.01 on Thursday. — Kyle LaHucik