Delandistrogene moxeparvovec

CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 21, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following update related to ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy. We have received feedback from the Medicines & Healthcare products Regulatory Agency (MHRA) in the United Kingdom (U.K.) that dosing may continue uninterrupted in ENVISION, study SRP-9001-303. ENVISION is a global, randomized, double-blind, placebo-controlled Phase 3 study of ELEVIDYS in non-ambulatory and older ambulatory individuals with Duchenne. 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. In the U.S., ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in the DMD gene For patients who are non-ambulatory and have a confirmed mutation in the DMD gene. The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). IMPORTANT SAFETY INFORMATION CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. WARNINGS AND PRECAUTIONS: 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 administration 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. Acute Serious Liver Injury: Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks. 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. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled. Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT, and total bilirubin levels return to near baseline levels). Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended. Immune-mediated Myositis: In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed. Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction. Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient’s clinical presentation and medical history if these symptoms occur. Myocarditis: Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials. 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. 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. 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 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 greater than or equal to 1:400. Adverse Reactions: The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia. 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). For further information, please see the full Prescribing Information. 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 leadership positions in Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs) 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. 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. 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, research and development programs, and clinical trials. 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: 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; and those risks identified under the heading “Risk Factors” in our most recent Annual Report on Form 10-K for the year ended December 31, 2024 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. View source version on businesswire.com: https://www.businesswire.com/news/home/20250521736806/en/ Investor Contact: Ian Estepan 617-274-4052 iestepan@sarepta.com Media Contacts: Tracy Sorrentino 617-301-8566 tsorrentino@sarepta.com Kara Hoeger 617-710-3898 KHoeger@sarepta.com Source: Sarepta Therapeutics, Inc.

iStock, Anton Vierietin Taking center stage at the American Society of Gene and Cell Therapy meeting was the first-ever reported case of a personalized i n vivo CRISPR editing therapy, which substantially eased the symptom burden in an infant. Over the weekend, the American Society of Gene and Cell Therapy held its annual meeting in New Orleans, hosting some of the space’s most cutting-edge research and spotlighting clinical findings that could have far-reaching implications on medical practice. BioSpace highlights some notable presentations here. A World First: Personalized CRISPR Therapy Arguably the biggest news out of ASGCT was the infant that has been treated with a “customized” base-editing therapy, which greatly eased the burden of the infant’s ultra-rare disease. The case, also published in the New England Journal of Medicine , focused on a male infant who presented with lethargy and respiratory distress within 48 hours of birth. He would eventually be diagnosed with severe carbamoyl-phosphate synthetase 1 (CSP-1) deficiency , a rare disease involving the toxic build-up of nitrogen in the body. Approximately half of babies diagnosed with the condition die “in early infancy,” according to the paper. However, after two infusions of a customized CRISPR-based gene editing therapy at 7 and 8 months of age, the patient was able to gradually increase dietary protein intake, which would otherwise have to be strongly restricted in patients with CSP-1 deficiency. Median blood ammonia levels dropped and doctors were also able to scale back nitrogen-scavenging medication. There were no severe safety concerns, according to the researchers, though they did detect “transient” elevations in liver enzymes as well as two viral infections within 4 weeks after the second dose. Rocket Shares ‘Encouraging’ Data for Cardiomyopathy Gene Therapy On Thursday, Rocket Pharmaceuticals revealed that its investigational gene therapy RP-A601 normalized right ventricle systolic function in patients with plakophilin-2 related arrhythmogenic cardiomyopathy. RP-A601 also suppressed or completely stabilized off-beat contractions of the ventricles. Aside from these clinical improvements, patients treated with RP-A601 likewise showed significantly better quality of life and functional performance. In a note to investors on Friday, BMO Capital Markets analysts called these findings “encouraging,” adding that they suggest “strong gene expression” alongside improvements in functional endpoints. On safety, the analysts noted that one patient developed a serious adverse event—Rocket did not specify what this toxicity was, revealing only that it “resolved without clinical sequelae within two months post-treatment”—though this safety signal is likely to be linked to immunomodulator use, according to BMO. While the serious safety event “can raise investor questions,” BMO argued that “the overall benefit/risk pro favorable given lack of approved disease-modifying treatments” in this space. Sarepta Provides More Cardio Color for Elevidys One of the most talked-about names at the ASGCT meeting was Elevidys, Sarepta Therapeutics ’ gene therapy for Duchenne muscular dystrophy. After all, the biologic was rocked by a patient death in March—a development that caused the biotech’s shares to crater by 22%. Sarepta sought to shore up confidence in Elevidys at ASGCT, presenting consolidated cardiovascular outcomes from four studies totaling 218 patients. Results showed that cardiac troponin levels, a marker of heart damage, fluctuated following treatment, though these were largely asymptomatic. Cardiac MRI analyses also showed that Elevidys did not result in meaningful left ventricular changes. The study also found two cases of myocarditis that arose “within days” of Elevidys treatment, though these resolved within three weeks. Overall, results showed that Elevidys had a manageable cardiac safety pro five years of follow-up. Also at ASGCT, Sarepta provided Elevidys data in ambulatory children aged 8- to 9-years—information that the space has been eagerly awaiting . These latest data show that Elevidys can elicit “ statistically significant and clinically meaningful ” functional improvements in older kids, indicating that the gene therapy can stabilize disease or slow its progression, according to Sarepta. Neurogene Unveils New Safety Protocol for Rett Syndrome Gene Therapy After Patient Death In November 2024, Neurogene reported that a young patient had died in its Phase I/II study for NGN-401, an investigational gene therapy for Rett Syndrome. A few days earlier, the patient, who received the 3E15 vg dose of NGN-401, was revealed to be in critical condition after showing signs of systemic hyperinflammation. At ASGCT, Neurogene detailed its new safety monitoring and treatment system that would allow it to detect and reverse this hyperinflammatory syndrome. In particular, the biotech conducted daily surveillance in the first week after treating patients, looking out for what it called the “three Fs:” fever, falling blood counts and elevated ferritin levels. Neurogene also implemented standard algorithms to treat systemic hyperinflammation in its trial, including high-dose corticosteroid intervention, followed by an IL-1 receptor agonist. Neurogene is moving forward with the lower, 1E15 vg dose of NGN-401, which is in Phase I/II development. The biotech “is not aware of any case of” systemic hyperinflammation at this dose level, as per a news release on Friday.