Sarepta presents new ELEVIDYS data in older ambulatory Duchenne patients

For best results when printing this announcement, please click on link below: Sarepta Therapeutics Presents New Data at 2026 World Muscle Society Annual Congress Demonstrating Safety and Clinically Meaningful Functional Benefit of ELEVIDYS (delandistrogene moxeparvovec) in Older Ambulatory Patients with Duchenne Patients treated with delandistrogene moxeparvovec at ages 8-12 demonstrated durable and clinically meaningful benefits across the North Star Ambulatory Assessment and timed function measures over 2 years compared to a well-matched external control group Separate findings in children treated at ages 2 to under 3 demonstrated robust delandistrogene moxeparvovec-dystrophin expression and transduction efficiency at 12 weeks In these analyses, the safety profile of delandistrogene moxeparvovec remained consistent and no new signals were identified Sarepta Therapeutics, Inc.

(NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today presented new data at the 31(st) Annual Congress of the World Muscle Society (WMS) that provide evidence of clinically meaningful functional benefit of ELEVIDYS (delandistrogene moxeparvovec) in older ambulatory patients with Duchenne muscular dystrophy. In ambulatory patients treated at ages 8 to 12, an age range in which functional decline becomes even more pronounced, treatment was associated with durable benefits across multiple measures over two years compared to a well-matched external control.

These findings suggest that the benefits previously observed in younger ambulatory patients are also being observed in older ambulatory patients. Sarepta also presented an analysis of patients treated at ages 2 and 3 that demonstrated robust delandistrogene moxeparvovec-dystrophin expression and sarcolemmal localization 12 weeks after infusion. Together, the analyses expand the evidence base across distinct ages and stages of ambulant Duchenne patients, while continued follow-up will be important to understand longer-term outcomes. , president, Research & Development and Technical Operations, Sarepta.

“Results presented at WMS 2026 fit within a broader and increasingly consistent body of evidence generated across the clinical development program. Seeing similar patterns emerge across ambulatory Duchenne populations reinforce that timely treatment with delandistrogene moxeparvovec has the potential to meaningfully change the trajectory of the disease. , professor and chair of the UC Davis Health Department of Physical Medicine and Rehabilitation, an investigator in the EMBARK and ENDEAVOR studies and an author on both presentations.

“In addition, the findings in younger children help us better understand the biological effects of treatment earlier in the disease course where preserving muscle may offer the greater opportunity for long-term benefit. 103LBP: Functional and safety outcomes in older ambulatory patients with Duchenne muscular dystrophy treated with delandistrogene moxeparvovec (C. ) This analysis used a prospectively specified approach to compare treated patients with similar patients from several well-characterized DMD natural history datasets.

Patients were matched based on the study eligibility criteria, and the groups were further balanced for key factors that can influence disease progression, with the aims of creating a reliable comparison and addressing common limitations of external control (EC) analyses. Patients from the EMBARK and ENDEAVOR studies who were aged 8-12 years at time of dosing (n=25), were compared to a well-matched EC group (n=99). Treatment with delandistrogene moxeparvovec demonstrated durable and clinically meaningful functional benefits over two years compared to EC.

0114 least squared means Clinically meaningful efficacy was observed across NSAA total score, RFF velocity and 10MWR velocity at 1 and 2 years post-infusion, with nominally statistically significant differences between delandistrogene moxeparvovec-treated and EC patients at Years 1 and 2. Results show a sustained treatment effect and slowing of disease progression and support the use of delandistrogene moxeparvovec in ambulatory patients with DMD aged 8-12 years.

Safety in this patient population was manageable and consistent with the known safety profile of delandistrogene moxeparvovec to date, and the most common treatment related adverse events were nausea and vomiting. 080P: Delandistrogene moxeparvovec micro-dystrophin expression and safety in 2—3-year-olds with Duchenne muscular dystrophy in ENDEAVOR and ENVOL studies (C. ) In this analysis, ambulatory patients with Duchenne aged ≥2 to 16 to 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.

, 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. , 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. , 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 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.

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Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “may,” “intends,” “prepares,” “looks,” “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 and business plans; the potential benefits of our technologies and scientific approaches; our clinical trials and our understanding of data to date; and ELEVIDYS, including its potential benefits in older patients. These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control.

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; success in preclinical and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful; our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events, or may cause undesirable side effects or adverse reactions, that result in significant negative consequences following any marketing approval; certain programs may never advance in the clinic or may be discontinued for a number of reasons, including regulators imposing a clinical hold and us suspending or terminating clinical research or trials; we may not be able to execute on our business plans, including meeting our expected or planned regulatory milestones and timelines, research and clinical development plans for various reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; 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.

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