Latest news with #ELEVIDYS
Yahoo
4 days ago
- Business
- Yahoo
Sarepta Therapeutics (NasdaqGS:SRPT) SRP-9003 Gene Therapy Earns FDA Platform Technology Designation
Sarepta Therapeutics recently announced that its SRP-9003 gene therapy received a platform technology designation from the FDA, and updated safety protocols for ELEVIDYS in the UK. Despite these developments, the company's shares fell 43% last month. This decline may have been influenced by market volatility amid geopolitical tensions and weak economic data. While these broader market factors likely added weight to the movement, Sarepta's price plunge suggests specific investor concerns, potentially around the challenges and risks disclosed in their recent product updates, affected sentiment. Be aware that Sarepta Therapeutics is showing 2 risks in our investment analysis and 1 of those is significant. Outshine the giants: these 26 early-stage AI stocks could fund your retirement. The recent FDA platform technology designation for SRP-9003 and updated safety protocols for ELEVIDYS in the UK illuminate the company's focus on addressing safety concerns and operational intricacies, potentially positively affecting Sarepta's future revenue and earnings prospects. These actions aim to bolster patient and physician confidence, fostering smoother administrative processes and treatment uptakes. Nevertheless, the broad 43% share price decline last month amid the geopolitical and economic backdrop highlights the market's sensitivity towards perceived risks and uncertainties, as reflected in Sarepta's significant price fluctuations. Over the past three years, Sarepta's total shareholder returns, including dividends, have decreased by 68.73%. This underperformance is further emphasized by a comparison to the one-year return, where Sarepta lagged behind both the US Biotechs industry, which decreased by 9.6%, and the broader US market, which increased by 10.9%. This historical performance context underscores the challenges Sarepta faces in aligning its operational improvements with shareholder value recovery. In terms of future prospects, these strategic updates and anticipated product data releases could influence revenue and earnings forecasts, potentially validating analysts' growth expectations. Analysts project a price target of US$147.33, 68.3% above the current share price of US$46.75. Achieving this target hinges on ramping up administrative efficiencies, expanding therapeutic offerings, and realizing long-term revenue drivers, assuming favorable patient and regulatory responses in the wake of recent developments. The analysis detailed in our Sarepta Therapeutics valuation report hints at an deflated share price compared to its estimated value. This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Companies discussed in this article include NasdaqGS:SRPT. This article was originally published by Simply Wall St. Have feedback on this article? Concerned about the content? with us directly. Alternatively, email editorial-team@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
4 days ago
- Business
- Business Wire
SRPT BREAKING NEWS: Sarepta Therapeutics, Inc. is being Investigated for Securities Fraud after Second Elevidys Death; Investors with Losses are Notified to Contact BFA Law
NEW YORK--(BUSINESS WIRE)--Leading securities law firm Bleichmar Fonti & Auld LLP announces an investigation into Sarepta Therapeutics, Inc. (NASDAQ: SRPT) for potential violations of the federal securities laws. Leading securities law firm Bleichmar Fonti & Auld LLP announces an investigation into Sarepta Therapeutics, Inc. (NASDAQ: SRPT) for potential violations of the federal securities laws. Share If you invested in Sarepta, you are encouraged to obtain additional information by visiting: Why is Sarepta being Investigated? Sarepta is a biopharmaceutical company focused on developing treatments for rare diseases. Sarepta's most important product is Elevidys, a therapy for the treatment of Duchenne muscular dystrophy. During the relevant period, Sarepta repeatedly touted the safety profile of Elevidys and told investors that the benefits of the treatment outweighed its risks. In truth, Elevidys causes fatal acute liver failure in some patients. The Stock Declines as the Truth is Revealed On March 18, 2025, Sarepta announced that a patient that had been treated with Elevidys died after suffering acute liver failure. On this news, the price of Sarepta stock fell $27.81 per share, or over 27%, from $101.35 per share on March 17, 2025, to $73.54 per share on March 18, 2025. Nevertheless, on the same day, Sarepta assured investors that 'the benefit-risk of ELEVIDYS remains positive.' Next, on June 15, 2025, Sarepta announced that a second patient treated with Elevidys had died from acute liver failure. The company further announced that because of the second death, it was suspending certain shipments of Elevidys and paused dosing in an ongoing clinical trial of the treatment. On this news, the price of Sarepta stock fell $15.24 per share, or more than 42%, from $36.18 per share on June 13, 2025, to $20.94 per share on June 16, 2025. Click here for more information: . What Can You Do? If you invested in Sarepta you may have legal options and are encouraged to submit your information to the firm. All representation is on a contingency fee basis, there is no cost to you. Shareholders are not responsible for any court costs or expenses of litigation. The firm will seek court approval for any potential fees and expenses. Why Bleichmar Fonti & Auld LLP? Bleichmar Fonti & Auld LLP is a leading international law firm representing plaintiffs in securities class actions and shareholder litigation. It was named among the Top 5 plaintiff law firms by ISS SCAS in 2023 and its attorneys have been named Titans of the Plaintiffs' Bar by Law360 and SuperLawyers by Thompson Reuters. Among its recent notable successes, BFA recovered over $900 million in value from Tesla, Inc.'s Board of Directors, as well as $420 million from Teva Pharmaceutical Ind. Ltd. For more information about BFA and its attorneys, please visit Attorney advertising. Past results do not guarantee future outcomes.
Yahoo
5 days ago
- Business
- Yahoo
Why Sarepta Therapeutics (SRPT) Stock Is Down Today
Shares of biotech company Sarepta Therapeutics (NASDAQ:SRPT) fell 44.4% in the afternoon session after the company reported a second death linked to its Duchenne muscular dystrophy gene therapy. Elevidys. In response, the company temporarily halted shipments of Elevidys for patients who are unable to walk and paused an associated clinical trial. The stock market overreacts to news, and big price drops can present good opportunities to buy high-quality stocks. Is now the time to buy Sarepta Therapeutics? Access our full analysis report here, it's free. Sarepta Therapeutics's shares are very volatile and have had 25 moves greater than 5% over the last year. But moves this big are rare even for Sarepta Therapeutics and indicate this news significantly impacted the market's perception of the business. The biggest move we wrote about over the last year was 3 months ago when the stock dropped 24.9% on the news that the company reported the death of a Duchenne muscular dystrophy patient following treatment with its gene therapy, ELEVIDYS. The patient reportedly suffered acute liver failure, a known risk of AAV-mediated gene therapies. However, Sarepta suggested that a recent cytomegalovirus (CMV) infection might have worsened the condition. Despite this, the company maintained that ELEVIDYS still had a favorable benefit-risk profile. It planned to update the prescribing information and inform regulators, clinical investigators, and prescribing physicians of the incident. Markets were likely reacting negatively due to concerns that the event could increase regulatory scrutiny, make doctors more hesitant to prescribe the therapy, or slow adoption overall. ELEVIDYS had been a big piece of Sarepta's growth story as it contributed more than 50% of sales in the Q4'2024 quarter. Any threat to its continued adoption could put pressure on the stock. Sarepta Therapeutics is down 84.1% since the beginning of the year, and at $19.70 per share, it is trading 88% below its 52-week high of $163.85 from June 2024. Investors who bought $1,000 worth of Sarepta Therapeutics's shares 5 years ago would now be looking at an investment worth $122.96. Here at StockStory, we certainly understand the potential of thematic investing. Diverse winners from Microsoft (MSFT) to Alphabet (GOOG), Coca-Cola (KO) to Monster Beverage (MNST) could all have been identified as promising growth stories with a megatrend driving the growth. So, in that spirit, we've identified a relatively under-the-radar profitable growth stock benefiting from the rise of AI, available to you FREE via this link. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
6 days ago
- Health
- Yahoo
Sarepta Therapeutics Presents Data at the American Society of Gene & Cell Therapy Conference, Including Statistically Significant Functional Outcomes for 8- and 9-Year-Old Patients in New Data Analysis of EMBARK Part 2
Significant functional benefits for 8- and 9-year-olds with Duchenne in Part 2 of the EMBARK study, contributing to the evidence of stabilization or slowing of disease progression in later childhood when muscle weakness typically progresses Statistically significant differences were observed on all key endpoints including 4.75 points (P=0.0026) on North Star Ambulatory Assessment (NSAA), 6.87 seconds in time-to-rise (TTR) from the floor (P=0.0010), and 4.76 seconds in 10-meter walk/run (10MWR) (P=0.0097) compared to a well-matched external control cohort Five abstracts, including two oral presentations at American Society of Gene & Cell Therapy Conference, span Sarepta's portfolio of approved and pipeline therapies across Duchenne and limb-girdle muscular dystrophy CAMBRIDGE, Mass., May 16, 2025--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today presented new data from Part 2 of the EMBARK study that continue to support the clinical benefits of ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy. These data are among other ELEVIDYS data from Sarepta's portfolio presented during the 28th annual meeting of the American Society of Gene & Cell Therapy (ASGCT) Conference. In the recent analysis of Part 2 of the EMBARK study, participants with Duchenne muscular dystrophy who had received a placebo in Part 1 and were aged 8 to 9 years (n=14) at crossover were included. At one year post ELEVIDYS treatment, there were between-group differences (least square means) on all key endpoints that were statistically significant, including 4.75 points (P=0.0026) on North Star Ambulatory Assessment (NSAA), 6.87 seconds in time-to-rise (TTR) from the floor (P=0.0010), and 4.76 seconds in 10-meter walk/run (10MWR) (P=0.0097) compared to a well-matched external control cohort. "The latest data from the EMBARK study highlighting motor function improvements in 8- and 9-year-old boys is encouraging and adds to the growing body of evidence supporting ELEVIDYS," said Aravindhan Veerapandiyan, M.D., Associate Professor of Pediatrics at the University of Arkansas for Medical Sciences and Arkansas Children's Hospital. "What stands out is that these patients were treated at an age when motor decline is typically expected in those with Duchenne. Yet, those who received ELEVIDYS demonstrated statistically significant and clinically meaningful functional improvements compared to external controls." The results presented at ASGCT are from the ongoing analysis of results from Part 2 of EMBARK, which compared two-year outcomes from 63 participants against data from an external control group of untreated individuals with Duchenne. Results at two years post-treatment showed that individuals treated with ELEVIDYS had better outcomes in multiple motor function measures, compared to a well-matched external control group. Additionally, no new safety signals were observed in the EMBARK study over the two-year duration and, in a subset of patients (n=16), micro-dystrophin expression and sarcolemmal localization was sustained from Week 12 to Week 64. "This has been a significant year for our neuromuscular portfolio, with multiple, ongoing analyses and longer-term data on efficacy and safety presented for ELEVIDYS," said Louise Rodino-Klapac, Ph.D., chief scientific officer and head of research and development, Sarepta Therapeutics. "Building on the topline EMBARK Part 2 data from earlier this year, we're committed to sharing ongoing analyses as fast as possible. The one-year results of patients treated with ELEVIDYS at 8 to 9 years old provide evidence that those treated with gene therapy outperform those who don't receive it at a critical point when more dramatic functional decline is expected." A full listing of Sarepta's presentations at ASGCT are below. Abstracts can be found at Data from presentations are embargoed until 6:00 AM CT on the presentation day for oral abstracts and until 6:00 AM CT on May 13, 2025 for poster abstracts. Oral Presentations (*Previously presented at MDA 2025 and supplemented with additional data) Title Date, Time Long-term Functional Outcomes and Safety Following Delandistrogene Moxeparvovec Treatment in DMD: EMBARK 2-Year Results* May 164:30 – 4:45 p.m. CSTRoom 393-396 Cardiovascular Investigation of SRP-9005 ( in Non-Human Primates: A Gene Therapy for Limb-Girdle Muscular Dystrophy 2C/R5 May 145 – 5:15 p.m. CSTNew Orleans Theater B Poster Presentations (*Denotes encore presentation) Poster # Title #1350 3-Year Functional Outcomes of Patients with Duchenne Muscular Dystrophy: Pooled Delandistrogene Moxeparvovec Clinical Trial Data vs. External Controls* #1353 Assessment of Cardiac Outcomes in Delandistrogene Moxeparvovec Clinical Trials for Duchenne Muscular Dystrophy* #1422 In Situ Biodistribution and Localization of Bidridistrogene Xeboparvovec (SRP-9003) in LGMD2E/R4 Mice After 1 Year of Follow-up About EMBARK, Study SRP-9001-301 Study SRP-9001-301, also known as EMBARK, is a multinational, phase 3, randomized, two-part crossover, placebo-controlled study of ELEVIDYS in individuals with Duchenne muscular dystrophy between the ages of 4 to 7 years. The primary endpoint is change from baseline in NSAA Total Score at Week 52 following treatment. Eligible participants received a single dose of ELEVIDYS during either Part 1 or Part 2 of the study. In Part 1, participants (n=125) were randomized according to age (≥4 to <8 years) or NSAA Total Score at screening (>16 to <29) and received either 1.33 x1014 vg/kg of ELEVIDYS or placebo with a follow-up period for 52 weeks. In Part 2, participants cross over - meaning, those who were previously treated with placebo in Part 1 receive ELEVIDYS and participants who were previously treated with ELEVIDYS receive placebo, with a follow-up period for 52 weeks. All patients remained blinded through Part 1 and Part 2. Secondary outcome measures in EMBARK include the quantity of micro-dystrophin produced by ELEVIDYS at week 12 (in a subset of participants) as measured by western blot, timed function tests, stride velocity and validated patient reported outcome measures for mobility and upper limb function. One-year results from the Part 1 placebo-controlled period of the EMBARK study were published in Nature Medicine in October 2024 and quantitative muscle MR (magnetic resonance) outcomes from part 1 of EMBARK were published in JAMA Neurology in May 2025. 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 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 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 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 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, clinical trials and the potential benefits and risks of 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, these data may not be sufficient to support approval by the FDA or other global regulatory authorities; success in clinical trials does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results; 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 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 Contacts Investor Contact: Ian Estepan617-274-4052iestepan@ Media Contacts: Tracy Sorrentino617-301-8566tsorrentino@ Kara Hoeger617-710-3898KHoeger@
Yahoo
6 days ago
- Business
- Yahoo
Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne
- The Company is developing an enhanced immunosuppressive regimen in consultation with a panel of multi-disciplinary clinical experts and engaging with regulators - Shipments of ELEVIDYS for infusions in non-ambulatory patients in commercial setting are suspended until enhanced regimen is approved and in place - ENVISION study is paused while seeking a protocol amendment to incorporate additional immunosuppression - Sarepta to host investor call on June 16, 2025, at 8:00 am Eastern time CAMBRIDGE, Mass., June 15, 2025--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety update regarding ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy, and steps the Company is taking to strengthen the safety profile in non-ambulatory patients. These steps follow a second reported case of acute liver failure (ALF) resulting in death. The cases of ALF to date have both occurred in non-ambulatory individuals with Duchenne. Sarepta extends its deepest sympathies to the affected families and care teams. Key Safety Initiatives Evaluating and Enhancing Immunosuppressive Regimen: As part of a comprehensive review of safety data, Sarepta is taking proactive steps to mitigate the risk of acute liver failure in non-ambulatory patients. Sarepta is working to immediately convene an independent group of leading experts in Duchenne and liver health to consider an enhanced immunosuppression regimen for ELEVIDYS. This panel will evaluate data and assess our proposed regimen, which includes sirolimus and is supported by preclinical data demonstrating the effectiveness of additional immunosuppression in moderating liver enzyme elevations, a key factor in mitigating potential safety events. Sarepta will share the panel's recommendations with the U.S. Food & Drug Administration (FDA), and implementation of any new regimen will be subject to FDA guidance and allowance. Suspending Shipments of ELEVIDYS for Non-Ambulatory Patients: Sarepta is temporarily suspending shipments of ELEVIDYS for non-ambulatory patients while an enhanced immunosuppressive regimen is evaluated, discussed with regulatory bodies, and put in place. For ambulatory patients, no treatment changes are being proposed and the current practice of administering corticosteroids before and after ELEVIDYS infusion, along with post-treatment monitoring, remains the same. ENVISION Study Paused: Sarepta has voluntarily paused dosing in the ENVISION clinical study (also known as Study SRP-9001-303). FDA concurs with this action. The pause will allow for the evaluation of a protocol amendment to incorporate an enhanced immunosuppressive regimen for the non-ambulatory patient cohort and incorporate any additional feedback from the FDA. Regulatory alignment is needed before screening and dosing in ENVISION may resume. ENVISION is a global, randomized, double-blind, placebo-controlled trial evaluating ELEVIDYS in older ambulatory and non-ambulatory individuals living with Duchenne muscular dystrophy. In the U.S., it serves as the confirmatory trial required under the FDA's accelerated approval pathway for non-ambulatory patients. "Our paramount priority is the safety and well-being of the patients we serve. We are taking immediate, decisive steps to better understand and mitigate the risk of acute liver failure, including enhancing the immunosuppressive regimen, for those with Duchenne who are non-ambulatory," said Louise Rodino-Klapac, Ph.D., chief scientific officer and head of research & development, Sarepta. "We are deeply saddened by the loss of a second patient and extend our heartfelt condolences to the patient's family and his care team during this incredibly difficult time. Duchenne muscular dystrophy is a devastating disease that profoundly affects lives and often cuts them far too short. With more than 900 individuals treated to-date, we know how much hope families place in new treatment options like ELEVIDYS – and we are committed to honoring that hope by acting swiftly, guided by scientific rigor and the insights of leading experts, to strengthen safety for all future patients." Commitment to Long-Term Safety and Understanding Sarepta remains committed to a thorough approach and the highest standards of patient safety and scientific rigor. The event has been reported to FDA and global health authorities and will inform ongoing discussions around a potential label update to reflect the risk of severe ALF and additional immune management strategies for non-ambulatory patients. While elevated liver enzymes are a known class effect of all AAV-based gene therapies, the exact mechanism behind AAV-related liver toxicity remains unclear. Current evidence suggests it is likely driven by an adaptive immune response. The Company will provide additional updates as appropriate. Investor Conference Call Details Sarepta will be hosting a conference call and webcast to discuss this update and provide an update on the Company's business on Monday, June 16, 2025, at 8:00 am Eastern time. The event will be webcast live under the investor relations section of Sarepta's website at: and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone. 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 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 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 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 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, clinical trials, ELEVIDYS, the potential benefits of an enhanced immunosuppression regimen in dosing in non-ambulatory patients, and expected plans and milestones, including providing additional updates as appropriate and engaging with regulators on an enhanced immunosuppressive regimen for dosing in non-ambulatory patients. 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, these data may not be sufficient to support approval by the FDA or other global regulatory authorities; success in clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or with advisory committee recommendations, or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; 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. View source version on Contacts Investor Contact: Ian Estepan617-274-4052iestepan@ Media Contacts: Tracy Sorrentino617-301-8566tsorrentino@ Kara Hoeger617-710-3898KHoeger@
