Sarepta Therapeutics’ Investigational Gene Therapy SRP-9001 for Duchenne Muscular Dystrophy Demonstrates Significant Functional Improvements Across Multiple Studies

• Sarepta and its partner Roche present new results and analyses at the International Congress on Neuromuscular Diseases (ICNMD), which demonstrate that SRP-9001 shows consistent, statistically significant functional benefits in individuals with Duchenne versus a propensity-weighted external control that continue to positively diverge from natural history disease course
• In the 20-patient Cohort 1 of SRP-9001-103 (ENDEAVOR), SRP-9001-treated participants improved 4 points from their pre-therapy baselines, and 3.8 points (unadjusted means) and 3.2 points (least squared means) at 52 weeks on the North Star Ambulatory Assessment (NSAA) compared to a propensity-weighted external control group (p=<0.0001)
  • The results from ENDEAVOR, using commercially representative SRP-9001, provide further support to reinforce confidence in our ongoing Phase 3 Study SRP-9001-301, EMBARK
• In the 4-patient Study SRP-9001-101, at 4 years, patients – currently on average over 9 years old and in the predicted steep decline phase of disease – did not decline but showed a 7-point increase above their pre-treatment baselines on NSAA, and 9.9 point (unadjusted means) and a 9.4 point (least squared means) improvement versus a propensity-weighted external control (p=0.0125)
• In a 52-patient integrated analysis across Studies 101, 102, and 103 at target dose, at one year, SRP-9001 treated patients improved 3.1 points (unadjusted means) and 2.4 points (least squared means) on NSAA versus propensity-weighted external control (p=<0.0001)
• Safety and tolerability profile for SRP-9001 remains consistent across treated patients

CAMBRIDGE, Mass., July 06, 2022 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared new functional data across multiple studies from the clinical development program for SRP-9001 (delandistrogene moxeparvovec) for the treatment of Duchenne muscular dystrophy. SRP-9001 is an investigational gene therapy for Duchenne being developed in partnership with Roche. Data are being presented this week at the 17th International Congress on Neuromuscular Diseases (ICNMD 2022) in Brussels.