Sarepta Reports Positive ELEVIDYS Gene Therapy Data for Duchenne Muscular Dystrophy

Sarepta Therapeutics reported positive topline three-year results from its Phase 3 EMBARK study evaluating its gene therapy ELEVIDYS in ambulatory patients with Duchenne muscular dystrophy. Patients treated with ELEVIDYS demonstrated statistically significant and clinically meaningful slowing of disease progression compared to an untreated external control group. The company reported no new treatment-related safety signals and said the safety profile remained consistent with previous data. Sarepta's shares rose more than 12% on the news.