Sarepta completes enrollment in the EMERGENE study of SRP-9003

Sarepta (SRPT) Therapeutics announced that enrollment and dosing is complete in EMERGENE or Study SRP-9003-301 , a Phase 3 clinical trial of SRP-9003 or bidridistrogene xeboparvovec . SRP-9003 is an investigational gene therapy for the treatment of limb-girdle muscular dystrophy Type 2E/R4 or beta-sarcoglycanopathy. EMERGENE is a global study, and the primary endpoint is the biomarker expression of beta-sarcoglycan protein, the absence of which is the sole cause of LGMD2E/R4. “The completion of enrollment in the EMERGENE study marks a significant milestone to bring a potentially disease-modifying treatment to individuals living with LGMD2E, an ultra-rare form of LGMD with no treatments beyond symptom management. We are committed to securing approval of SRP-9003 as quickly as possible and are now closer to reaching that goal for patients and their families,” said Louise Rodino-Klapac, Ph.D., executive vice president, chief scientific officer and head of research and development, Sarepta Therapeutics. “The design of EMERGENE is an important precedent that informs development plans for Sarepta’s other LGMD pipeline programs, including our LGMD2D program which we just initiated and our LGMD2C program which we expect to initiate in the first quarter of 2025, and serves as a pathfinder for heterogenous neuromuscular gene therapies more broadly.” Data from EMERGENE are expected in the first half of 2025. Assuming a positive pre-Biologics License Application BLA meeting and supportive data from EMERGENE, Sarepta anticipates submitting a BLA to the U.S. Food and Drug Administration seeking accelerated approval for SRP-9003 in 2025.