Sarepta reports positive ELEVIDYS protein expression data in young patients

Sarepta (SRPT) Therapeutics reported new results from its ENDEAVOR study, also known as Study 9001-103, of ELEVIDYS for Duchenne muscular dystrophy. In cohort 6, which included six participants aged 2 at treatment, ELEVIDYS demonstrated mean protein expression of 93.87%, as measured by western blot, and 79.9% dystrophin positive fibers, measured by immunofluorescence, at 12 weeks. The safety profile was consistent with prior studies. Common adverse events were nausea and vomiting; elevated liver enzymes seen in two patients resolved with steroid administration. Sarepta plans to discuss expanding the ELEVIDYS label to include younger patients with the FDA next month.