Sarepta releases mixed top-line results from EMBARK study of Elevidys

31 October 2023

US biotech Sarepta Therapeutics (Nasdaq: SRPT) unveiled mixed results late Monday for its gene therapy Elevidys (delandistrogene moxeparvovec-rokl) in patients with Duchenne muscular dystrophy between the ages of four through seven years. The stock was halted in late trading.

In the study, Elevidys-treated patients improved 2.6 points on their North Star Ambulatory Assessment (NSAA) total score 52 weeks after treatment compared to 1.9 points in placebo-treated patients.

However, the trial missed the primary North Star Ambulatory Assessment/NSAA endpoint, with a treatment difference of 0.65-points (p=0.2441).

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