Sarepta Therapeutics (NASDAQ: SRPT) suffered a devastating blow on Monday, June 16 when its stock plummeted 42% following news of a second patient death linked to its Duchenne muscular dystrophy gene therapy, Elevidys. The biotechnology company and its global partner Roche immediately suspended commercial and clinical use of the treatment in non-ambulatory patients pending a comprehensive safety review.
Sarepta Therapeutics (SRPT) sold off more than 40% after suspending its Elevidys Duchenne muscular dystrophy drug, which was linked to two fatal liver failure cases. Diane King Hall break takes a closer look into the report.
Sarepta's shares sink as second Elevidys-linked death triggers safety concerns, dosing halts and trial pauses.
Sarepta Therapeutics Inc (NASDAQ:SRPT) shares plunged almost 45% after the company disclosed the death of a second patient from acute liver failure linked with its experimental gene therapy Elevidys for Duchenne muscular dystrophy (DMD). In response, the company is convening an independent expert panel to evaluate and develop an enhanced immunosuppressive regimen, including sirolimus, to reduce the risk of acute liver failure in non-ambulatory DMD patients treated with Elevidys, with any new protocol subject to Food and Drug Administration (FDA) review and approval.
Sarepta's stock has plunged due to safety concerns with ELEVIDYS, including two deaths in non-ambulatory DMD patients and regulatory uncertainty. Despite these setbacks, ELEVIDYS remains effective for ambulatory patients, and Sarepta is proactively addressing safety via enhanced immunosuppression and trial amendments. Valuation appears depressed, with the market pricing in a worst-case scenario, but historical precedent (ZOLGENSMA) suggests recovery is possible if risks are managed.
A second patient receiving its experimental gene therapy Elevidys has died from liver failure, raising serious questions on the drug's outlook.
Shares of Sarepta Therapeutics plunged 34% in premarket trading on Monday, a day after the company disclosed a second case of patient death due to acute liver failure after receiving its gene therapy for a rare form of muscular dystrophy.
Sarepta's stock collapsed due to ELEVIDYS safety concerns, regulatory setbacks, and reduced revenue guidance, but I see potential upside at current prices. ELEVIDYS's safety profile, especially in non-ambulatory patients, led to shipment suspension and a significant reduction in peak sales estimates. Despite setbacks, Sarepta's first-mover advantage, deep pipeline, and gene therapy leadership support a high-risk, high-reward investment thesis.
Sarepta Therapeutics on Sunday said there had been a second reported case of acute liver failure resulting in death after a patient received the company's gene therapy for a rare form of muscular dystrophy.
Sarepta Therapeutics (SRPT) reported earnings 30 days ago. What's next for the stock?
SRPT reports mixed first-quarter 2025 results. The stock plunges after the company significantly lowers its 2025 sales guidance.
Sarepta Therapeutics, Inc. (NASDAQ:SRPT ) Q1 2025 Earnings Conference Call May 6, 2025 4:30 PM ET Company Participants Mary Jenkins - Associate Director, IR Doug Ingram - President and CEO Dallan Murray - EVP, CCO Louise Rodino-Klapac - EVP, Head of R&D, CSO Ian Estepan - EVP, CFO Conference Call Participants Tazeen Ahmad - Bank of America Securities Ritu Baral - TD Cowen Louise Chen - Scotiabank Andrew Tsai - Jefferies Eliana Merle - UBS Brian Abrahams - RBC Capital Markets Debjit - Guggenheim Securities Salveen Richter - Goldman Sachs Brian Skorney - Baird Michael Ulz - Morgan Stanley Gil Blum - Needham & Company Joe Schwartz - Leerink Partners Yanan Zhu - Wells Fargo Kostas Biliouris - BMO Capital Markets Anupam Rama - JPMorgan Biren Amin - Piper Sandler David Hoang - Deutsche Bank Rick Miller - Cantor Fitzgerald Andreas Argyrides - Oppenheimer & Co. Gena Wang - Barclays Operator Good afternoon, and welcome to the Sarepta Therapeutics First Quarter 2025 Financial Results Conference Call. As a reminder, today's program is being recorded.