Sarepta presents EMBARK study data at ASGCT conference

Sarepta (SRPT) Therapeutics presented new data from Part 2 of the EMBARK study that continue to support the clinical benefits of Elevidys, the only approved gene therapy for patients with Duchenne muscular dystrophy. These data are among other Elevidys data from Sarepta’s portfolio presented during the 28th annual meeting of the American Society of Gene & Cell Therapy Conference. In the recent analysis of Part 2 of the EMBARK study, participants with Duchenne muscular dystrophy who had received a placebo in Part 1 and were aged 8 to 9 years at crossover were included. At one year post Elevidys treatment, there were between-group differences on all key endpoints that were statistically significant, including 4.75 points on North Star Ambulatory Assessment, 6.87 seconds in time-to-rise from the floor, and 4.76 seconds in 10-meter walk/run compared to a well-matched external control cohort. The results presented at ASGCT are from the ongoing analysis of results from Part 2 of EMBARK, which compared two-year outcomes from 63 participants against data from an external control group of untreated individuals with Duchenne. Results at two years post-treatment showed that individuals treated with Elevidys had better outcomes in multiple motor function measures, compared to a well-matched external control group. Additionally, no new safety signals were observed in the EMBARK study over the two-year duration and, in a subset of patients, micro-dystrophin expression and sarcolemmal localization was sustained from Week 12 to Week 64.