Sarepta Reports Positive Three-Year Data for Elevidys in DMD

Sarepta posted promising long-term Phase III results for its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys (delandistrogene moxeparvovec), despite a challenging 2025 marked by safety concerns. Topline data from part one of the EMBARK study (NCT05096221) showed statistically significant improvements in motor function versus an untreated external control group of patients aged 4–7.

Key outcomes included a 4.39-point increase in NSAA scores, a 2.70-second improvement in 10-meter walk/run (10MWR), and a 6.05-second reduction in time to rise (TTR). NSAA values remained above baseline at three years, while control patients declined. Disease progression was 70–73% slower in Elevidys-treated patients.

Safety remained consistent with prior studies, with no new adverse events. Following the readout, Sarepta’s stock rose nearly 4% to $21.96.

Despite the positive results, analysts remain cautious, noting that prior safety issues, a boxed warning, and limited non-ambulatory indications may limit near-term uptake. Sarepta is pursuing Phase Ib ENDEAVOR data to expand use in non-ambulatory patients, expected late 2026.

02-02-2026