Roche: Elevidys Trial In DMD Fails To Meet Primary Endpoint, But Shows Positive Efficacy Outcomes
Swiss drug major Roche Holding AG (RHHBY) announced Tuesday that EMBARK trial of Elevidys (delandistrogene moxeparvovec) in Duchenne muscular dystrophy or DMD failed to reach primary endpoint, but shows positive efficacy outcomes on all timed functional key endpoints.
In the global, randomised, double-blind Phase 3 EMBARK study, Elevidys-treated boys aged 4-7 years with DMD showed an increase on the North Star Ambulatory Assessment or NSAA, a measure of motor function, compared to placebo at 52 weeks.
In the study, Elevidys-treated patients improved 2.6 points on their NSAA total score 52 weeks after treatment, compared to 1.9 points in placebo-treated patients.
In all pre-specified, timed functional key secondary endpoints, time to rise from floor and 10 metre walk test, clinically meaningful and statistically significant improvements were observed, the company noted.
Both endpoints are prognostic factors for disease progression and loss of ability to walk.
Additionally, a clinically meaningful and statistically significant improvement was also observed for the pre-specified secondary endpoint stride velocity 95th centile. This novel digital endpoint, qualified by the European Medicines Agency (EMA), measures speed of walking via a wearable device (Syde).
Roche said it is analysinga all data, which will be discussed with health authorities to determine the path forward. Detailed results from the EMBARK study will be shared at an upcoming scientific congress and a medical journal publication will be pursued.
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