Core Insights - Sarepta Therapeutics is conducting a conference call to discuss its strategic restructure and updates regarding the ELEVIDYS label [2][3] Company Overview - Douglas S. Ingram, the CEO, is leading the discussion and will provide insights into the company's future direction and developments [3]

Core Viewpoint - Shares of Sarepta Therapeutics experienced a significant decline of over 30% due to concerns regarding the future of its approved gene therapy treatment, Elevidys, following FDA intervention [1][2]. Group 1: FDA Actions - The FDA is requesting Sarepta Therapeutics to voluntarily halt all shipments of Elevidys, indicating potential regulatory challenges for the product [1]. - FDA Commissioner Marty Makary mentioned that the agency is evaluating whether Elevidys should remain on the market [2]. Group 2: Patient Safety Concerns - The FDA is investigating two patient deaths associated with Elevidys, raising serious safety concerns about the gene therapy [2]. - Additionally, Sarepta reported a third death linked to a different experimental gene therapy, further complicating the company's situation [2].

Shares of Sarepta Therapeutics plummeted Friday following a report that a third patient has died during a clinical trial for one of its medications. https://t.co/b4EzEu4AXX ...

Summary of Sarepta Therapeutics (SRPT) Conference Call - July 18, 2025 Company Overview - **Company**: Sarepta Therapeutics (SRPT) - **Focus**: Gene therapy for Limb Girdle Muscular Dystrophy (LGMD) and related conditions Key Points Industry and Company Context - The call addressed recent developments in the LGMD program, particularly concerning the investigational candidate SRP-9004 for LGMD type 2D [3][4] - The company is transitioning focus from gene therapy to its siRNA platform due to risk-adjusted net present values (NPVs) and the need to manage expenses [4][10] Core Issues Discussed - A serious adverse event (fatality) occurred in a trial for SRP-9004, which was not deemed material to the overall discussions on the previous call [3][4] - The decision to discontinue other LGMD programs was based on risk-adjusted NPVs rather than safety events [10][18] - The company has paused dosing of Elevitus in non-ambulatory patients while developing a protocol for sirolimus as prophylactic immunosuppression [5][51] Safety and Regulatory Considerations - The event in question involved a 51-year-old non-ambulatory patient and was reported to the FDA, which has not changed its position regarding Elevitus [6][21][94] - The company maintains that the risk of acute liver failure (ALI) is a known risk associated with AAV-mediated therapies, particularly in non-ambulatory patients [52][83] - Approximately 30-40% of patients experience elevated liver enzymes, with a subset requiring hospitalization [47][88] Patient Population and Clinical Trials - Between 35 and 40 patients have been dosed across LGMD trials, with a roughly equal split between ambulatory and non-ambulatory patients [56] - The company has conducted three clinical trials for LGMD2E, with no fatalities reported in other gene therapy trials [32][41] Future Directions and Market Considerations - The company plans to continue pursuing the LGMD2E program (SRP-9003) despite the challenges, as it is an ultra-rare disease with significant unmet needs [60] - Discussions with the FDA regarding the use of sirolimus and its potential impact on the label for SRP-9003 are ongoing [41][51] Communication and Transparency - The company emphasizes its commitment to transparency with investors, patients, and physicians regarding safety signals and clinical trial results [18][100] - Ongoing communication with the physician community about the risks associated with Elevitus and LGMD therapies is prioritized [98] Additional Important Points - The company has a history of being transparent about safety signals and has communicated the risks of elevated liver enzymes to the physician community [18][98] - The manufacturing processes for different therapies (e.g., Elevitus vs. LGMD) are distinct, which may influence safety profiles [32][29] - The company is evaluating the potential need for additional data to support the Biologics License Application (BLA) for LGMD2E [41][68] This summary captures the essential points discussed during the conference call, focusing on the company's strategic direction, safety considerations, and ongoing communication efforts.

About this content About Emily Jarvie Emily began her career as a political journalist for Australian Community Media in Hobart, Tasmania. After she relocated to Toronto, Canada, she reported on business, legal, and scientific developments in the emerging psychedelics sector before joining Proactive in 2022. She brings a strong journalism background with her work featured in newspapers, magazines, and digital publications across Australia, Europe, and North America, including The Examiner, The Advocate, ...

Core Viewpoint - A class action securities lawsuit has been filed against Sarepta Therapeutics, Inc. alleging securities fraud related to the company's gene therapy product, ELEVIDYS, which is intended for treating Duchenne muscular dystrophy [1][2]. Group 1: Lawsuit Details - The lawsuit seeks to recover losses for Sarepta investors who were affected by alleged securities fraud between June 22, 2023, and June 24, 2025 [1]. - The complaint claims that Sarepta made false statements regarding ELEVIDYS, including significant safety risks, failure to detect severe side effects, and the potential halting of trials due to adverse events [2]. Group 2: Next Steps for Investors - Investors who suffered losses during the specified timeframe have until August 25, 2025, to request to be appointed as lead plaintiff, although participation in any recovery does not require this [3]. - Class members may be entitled to compensation without any out-of-pocket costs or fees [3]. Group 3: Firm Background - Levi & Korsinsky, LLP has a history of securing hundreds of millions of dollars for shareholders and is recognized as one of the top securities litigation firms in the United States [4].

Core Viewpoint - The article discusses a class action lawsuit against Sarepta Therapeutics, Inc. related to the safety of its gene therapy ELEVIDYS, alleging that the company and its executives made misleading statements regarding the therapy's risks and trial protocols [1][3]. Company Overview - Sarepta Therapeutics, Inc. is a commercial-stage biopharmaceutical company focused on developing therapies for Duchenne muscular dystrophy, including the gene therapy ELEVIDYS [2]. Allegations of the Lawsuit - The lawsuit claims that Sarepta failed to disclose significant safety risks associated with ELEVIDYS, including severe side effects that were not detected during clinical trials [3]. - It is alleged that the severity of adverse events from ELEVIDYS treatment led to the halting of recruitment and dosing in clinical trials, attracting regulatory scrutiny and increasing risks around the therapy's approvals [3]. Key Events Impacting Stock Price - On March 18, 2025, Sarepta reported a patient death due to acute liver failure after ELEVIDYS treatment, causing the stock price to drop over 27% [4]. - Following an April 4, 2025 disclosure that EU authorities requested a review of the death, Sarepta's stock fell more than 7% [5]. - On June 15, 2025, a second patient death was reported, leading to a 42% decline in stock price after Sarepta suspended shipments and paused dosing in clinical studies [6]. - On June 24, 2025, the FDA announced an investigation into the risk of acute liver failure associated with ELEVIDYS, resulting in an 8% drop in stock price [7]. Legal Process - Investors who purchased Sarepta securities during the class period can seek appointment as lead plaintiff in the lawsuit, which allows them to represent the interests of all class members [8].

Sarepta said another patient has died from acute liver failure after receiving one of its gene therapies, putting additional pressure on the biotech company https://t.co/DSR52u0ort ...

Core Viewpoint - The article discusses a class action lawsuit against Sarepta Therapeutics, alleging violations of the Securities Exchange Act of 1934 related to the company's gene therapy product, ELEVIDYS, which has been linked to severe safety risks and adverse events [1][3]. Group 1: Lawsuit Details - The class action lawsuit is titled Dolgicer v. Sarepta Therapeutics, Inc., and it involves purchasers of Sarepta securities from June 22, 2023, to June 24, 2025 [1]. - Sarepta is accused of making false or misleading statements regarding the safety of ELEVIDYS, which is intended to treat Duchenne muscular dystrophy [2][3]. - The lawsuit claims that Sarepta failed to disclose significant safety risks associated with ELEVIDYS, including severe side effects that were not detected during clinical trials [3]. Group 2: Impact on Stock Price - Following the disclosure of a patient's death due to acute liver failure after treatment with ELEVIDYS on March 18, 2025, Sarepta's stock price fell by more than 27% [4]. - On April 4, 2025, after the company announced that EU authorities requested a review of the death, the stock price dropped by over 7% [5]. - A second patient death was reported on June 15, 2025, leading to a further decline in stock price by more than 42% [6]. - On June 24, 2025, the FDA issued a Safety Communication regarding the investigation into the deaths, resulting in an additional stock price drop of over 8% [7]. Group 3: Legal Process and Representation - Investors who purchased Sarepta securities during the class period can seek appointment as lead plaintiff in the lawsuit, which allows them to act on behalf of other class members [8]. - The lead plaintiff can choose a law firm to represent the class, and participation as lead plaintiff does not affect the ability to share in any potential recovery [8].

Company Restructuring - Sarepta Therapeutics' shares surged over 20% in morning trading [1] - The company will reduce its workforce as part of a restructuring plan [1] - Sarepta Therapeutics is pausing several developmental projects [1]