Press release
Sarepta Announces Positive Topline Three-Year EMBARK Results Showing ELEVIDYS Significantly Slows Disease Progression on Key Functional Measures in Ambulatory Duchenne Patients
At a mean age of 9 years old, ELEVIDYS-treated patients achieved mean North Star Ambulatory Assessment (NSAA) scores above baseline three years after
About this update from Sarepta Therapeutics, Inc.
[{"type":"text","content":"\n\nAt a mean age of 9 years old, ELEVIDYS-treated patients achieved mean North Star Ambulatory Assessment (NSAA) scores above baseline three years after treatment (n=52)\n\n\n\nELEVIDYS gene therapy demonstrated 70% or greater reduction in the rate of decline relative to the propensity-weighted external control group, as measured by Time to Rise (TTR) and 10-meter walk/run (10MWR). ELEVIDYS-treated patients showed an increasing treatment effect over time, with the functional gap versus the external control group significantly widening between Year 2 and Year 3\n\n\n\nNo new treatment-related safety signals were observed, consistent with the manageable safety profile observed with ELEVIDYS in ambulatory patients to-date\n\n\n\nInvestor webcast to be held today at 8:30 a.m. ET\n\n\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nSarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced positive topline three-year functional results from Part 1-treated patients in EMBARK (Study SRP-9001-301), the global, randomized placebo-controlled Phase 3 study evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory individuals with Duchenne muscular dystrophy who were aged four to seven at time of treatment and at time of last assessment were on average over nine years of age.\n\n\nThree years after treatment, patients who received ELEVIDYS in Part 1 of EMBARK demonstrated statistically significant, clinically meaningful and durable efficacy across all key motor function measures, North Star Ambulatory Assessment (NSAA), Time to Rise (TTR) and 10-meter walk/run (10MWR), when compared to a pre-specified propensity-weighted untreated external control group (EC)*. The mean NSAA score remained above baseline at Year 3 for the ELEVIDYS-treated group (n=52) while the EC group (n=73) continued to show the expected age-related decline below their baseline score. The ELEVIDYS group showed a 73% slowing of disease progression as measured by TTR and 70% slowing of disease progression as measured by 10MWR when compared to the EC group.**\n\n\nPatients treated with ELEVIDYS in Part 1 maintained significantly higher levels of motor function three years after treatment compared to the EC group. Topline efficacy results are summarized in the table below:\n\n\n\n\nFunctional Outcomes\n\n\n\n\n\n...