Sarepta’s EMBARK study data shows continuing benefits to ambulatory individuals treated with their microdystrophin gene therapy, Elevidys

duchenne sarepta 2025

Sarepta Therapeutics shared positive topline results from Part 2 of the EMBARK study, showing that treatment with the microdystrophin gene therapy, Elevidys, is associated with sustained benefits and disease stabilization in ambulatory individuals.   EMBARK is a Phase 3 study of Elevidys (in individuals aged 4-7 years) with a crossover design, in which individuals treated with placebo in Part 1 “cross over” at 52 weeks and then receive Elevidys in Part 2. 

Sarepta reported that Part 2 crossover individuals showed significantly functional benefits in NSAA, Time to rise (TTR), and the 10 meter walk/run (10MWR) test after1 year, compared to an external control group.  They also reported additional data in individuals treated in Part 1.  Those individuals showed continued expression of Elevidys in biopsies taken at week 64, and sustained functional improvements on NSAA, TTR, and 10MWR at 2 years.  MRI results in these individuals 2 years after treatment show they have less progressing of the muscle pathology changes observed in non-treated individuals.   

PRESS RELEASE:  https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-results-part-2-embark-study

Roche PRESS RELEASE: https://www.roche.com/media/releases/med-cor-2025-01-27

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