Sarepta Therapeutics, Inc. has announced positive topline results from Part 2 of EMBARK, the company’s Phase 3 clinical study of ELEVIDYS. ELEVIDYS is a micro-dystrophin gene therapy used for the treatment of individuals aged four years and older with Duchenne.
Results show that crossover-treated patients who received a placebo for 52 weeks in Part 1 and were then treated with ELEVIDYS in Part 2 showed increases on the North Star Ambulatory Assessment (NSAA) at 52 weeks. Crossover-treated patients also show statistically significant benefits of ELEVIDYS treatment on Time to Rise (TTR) and 10-meter walk/run (10MWR) compared to a prespecified external control cohort.
For patients treated with ELEVIDYS in Part 1, biopsy results indicate sustained expression at week 64, as well as sustained functional improvements on NSAA, TTR, 10MWR, and minimal muscle pathology progression two years after treatment as measured by MRI. Sarepta notes that these results indicate that the trajectory of disease in patients treated with ELEVIDYS is continuing to diverge from the natural history of Duchenne.
According to Sarepta, ELEVIDYS continues to be safe and well-tolerated. The company has reported that detailed results from Part 2 of the EMBARK study will be shared at future medical meetings.
PPMD is pleased to learn this news and we look forward to future updates from Sarepta.
Read Sarepta’s press release here.
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