Sarepta Therapeutics announced that its gene therapy Elevidys continues to show benefits for Duchenne muscular dystrophy (DMD) patients three years after treatment. The company released new data from the Phase III EMBARK trial, which is studying Elevidys in ambulatory DMD patients.
According to Sarepta, the therapy helped stabilize DMD symptoms based on several measures. Patients treated with Elevidys showed improvements in motor function and experienced a 73% slowing in disease progression for time-to-rise and a 70% slowing in the 10-meter walk-run test compared to an external control group. Sarepta stated that all results were statistically significant.
“We’re encouraged by the concordance of these functional measures,” analysts at Jefferies wrote Monday morning after the data announcement, “especially as physical function deteriorates after age.”
Crystal Proud, a pediatric neurologist specializing in DMD, said during Sarepta’s call that the numerical benefits seen in the trial “mirror my observations” of her patients and described Elevidys’ benefits as “tangible and measurable.”
The company reported four adverse events among participants during year three of the trial but did not specify their nature. Sarepta noted that none of these safety signals were new.
Sarepta’s stock price increased by 9% following the release of this data.
In 2025, two patient deaths linked to liver damage led to regulatory challenges for Sarepta. In response, CEO Doug Ingram stated: “liver injury is a known risk of AAV therapies.” The company subsequently laid off about 500 employees and shifted focus away from gene therapies toward its siRNA pipeline. A black box warning was added to Elevidys for acute liver injury, liver failure, and potential death; this warning was officially imposed by the FDA in November 2025 when it also limited approval to ambulatory patients aged four years and older.
The FDA paused dosing of Elevidys for non-ambulatory patients after last year’s deaths. Sarepta plans to release data by late 2026 on combining Elevidys with rapamycin—an immunosuppressant—which could potentially lead regulators to allow use again in non-ambulatory patients.
Sarepta will continue monitoring EMBARK trial participants through five years post-treatment.
