Sarepta Therapeutics Announces Positive Three-Year EMBARK Data of AAVrh74 Gene Therapy in Ambulatory DMD Patients

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Jan. 26, 2026–

Sarepta Therapeutics announced positive three-year functional results from Part 1 of the EMBARK Phase 3 study (SRP-9001-301), demonstrating that ambulatory Duchenne muscular dystrophy (DMD) patients treated with the AAV-based gene therapy ELEVIDYS (delandistrogene moxeparvovec-rokl) — at a mean age of 9 years — maintained North Star Ambulatory Assessment (NSAA) scores above baseline (n=52), outperforming a propensity-weighted external control group on key tasks like jumping, standing on one leg, rising from the floor, and 10-meter walk/run. The therapy achieved a 73% reduction in the rate of decline on Time to Rise (TTR) and 70% on 10MWR versus controls, with statistically significant improvements (NSAA +4.39 points, p=0.0002; TTR -6.05 seconds, p<0.0001; 10MWR -2.70 seconds, p=0.0039) and a widening treatment effect between Years 2 and 3, indicating growing separation from natural disease progression.

CEO Douglas Ingram emphasized that the long-term data not only confirm earlier benefits but also highlight the therapy’s ability to alter Duchenne’s course when started early, preventing irreparable muscle damage in this progressive neuromuscular disease affecting young boys. Pediatric neurologist Crystal Proud, M.D., an EMBARK investigator, noted the meaningful impact on everyday movements. No new treatment-related safety signals emerged, consistent with ELEVIDYS’ profile in ambulatory patients. Despite heightened scrutiny following two non-ambulatory teen deaths linked to acute liver failure, Sarepta reported no new issues. Oppenheimer analysts suggested the data could improve physician/patient perceptions of the benefit-risk profile and accelerate commercial uptake, despite skepticism over external controls. ELEVIDYS, the only approved AAV-based gene therapy for DMD (using AAVrh74 for micro-dystrophin delivery), is indicated for ambulatory patients aged 4 and older per the 2025 FDA label update and has treated over 1,200 patients globally.