After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.
The benefits of taking Elevidys, Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy, continue three years post treatment, the company said Monday.
Sarepta announced year three data from the Phase III EMBARK trial testing Elevidys in ambulatory patients with DMD, marking a positive step after a discouraging 2025 marred by patient deaths and a plummeting stock price.
Elevidys appeared to stabilize the symptoms of DMD by a number of measures, according to the data. Patients receiving the drug saw improvements in motor function as measured by a commons scale of the disease’s effect on walking and muscle-use ability. Patients also had a slowing of disease progression by 73% in time-to-rise and 70% in the 10-meter walk-run measurement, compared to an external control. The results in all three measurements were statistically significant, Sarepta said.
“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.”
Sarepta’s shares rose 9% to $23.09 on Monday morning, compared to a previous closing price of $21.13 on Friday.
Sarepta has been tracking patients in EMBARK every year for the past three years and will continue logging data out to five years. The first year of the trial was used to support Elevidys’ full approval in June 2024, according to Jefferies, after which patients in the treatment and placebo arms were switched, while keeping the trial blinded.
Crystal Proud, a pediatric neurologist who specializes in DMD, said on Sarepta’s Monday call that the numerical benefits in the trial “mirror my observations” of her patients, and that the benefits of Elevidys have been “tangible and measurable.”
Sarepta reported that four patients in year three of the trial experienced adverse events , though none of the safety signals were new. Sarepta did not say what the adverse events were.
Monday’s readout comes after a tumultuous 2025 for Elevidys. In March, the gene therapy was implicated in the death of a young boy with DMD who developed acute liver injury leading to liver failure. This was followed by another patient death in June 2025, likewise linked to liver damage.
In an investor call to discuss the second mortality, CEO Doug Ingram insisted that “liver injury is a known risk of AAV therapies.”
Still, the deaths ushered in months of regulatory difficulties and business realignment efforts for Sarepta. In July 2025, the biotech laid off around 500 employees in a pivot that included moving away from gene therapies and leaning into its siRNA pipeline.
Sarepta at the time also added a black box warning to Elevidys for acute liver injury, liver failure and the potential for death. The FDA in November 2025 officially imposed the boxed warning on Elevidys and also narrowed its approval to only ambulatory patients 4 years and up.
The FDA paused dosing of Elevidys for non-ambulatory patients after the deaths last year. Sarepta is planning on releasing data on a combination of Elevidys and the immunosuppressant medication rapamycin by the end of 2026, that, according to Jefferies, could push the FDA to resume Elevidys shipments in non-ambulatory patients, who make up half of the DMD patient population.
