Sarepta Plunges 30.24% on Gene Therapy Trial Halt

On June 16, 2025, Sarepta's stock experienced a significant drop of 30.24% in pre-market trading, reflecting investor concerns over recent developments in the company's gene therapy trials.

Sarepta Therapeutics has temporarily halted dosing in its Envision study, a Phase 3 confirmatory trial required under the FDA's accelerated approval pathway. This decision comes after the company reported a second fatality from acute liver failure in a patient treated with its gene therapy, Elevidys, designed for Duchenne muscular dystrophy.

In response to these safety concerns, Sarepta has suspended shipments of Elevidys for non-ambulatory patients and is considering strengthening the immunosuppressive regimen. The company is consulting with a panel of multi-disciplinary clinical experts and engaging with regulatory authorities to evaluate an enhanced regimen that includes sirolimus, aimed at mitigating the risk of acute liver failure by moderating liver enzyme elevations.

Sarepta remains committed to ensuring patient safety and will provide further updates as developments occur. The company will host an investor call on June 16, 2025, at 8:00 am Eastern time, available via webcast on Sarepta's investor relations website.