Sarepta Reports Positive Phase 3 EMBARK Data For Elevidys In Duchenne Muscular Dystrophy

Sarepta Therapeutics

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Kaufen / Verkaufen

(RTTNews) - Sarepta Therapeutics, Inc. (SRPT) Monday reported positive topline results from Part 2 of the Phase 3 study, dubbed EMBARK, of its gene therapy Elevidys approved for the treatment of Duchenne muscular dystrophy.

Results from the study showed that treatment with Elevidys brought sustained benefit and disease stabilization in ambulatory individuals with Duchenne Muscular Dystrophy.

Crossover-treated patients, those who received a placebo in Part 1 and crossed over at 52 weeks and were treated with Elevidys in Part 2, improved 2.34 points from baseline on the North Star Ambulatory Assessment (NSAA) 52 weeks after treatment. NSAA is a standardized clinical scale used to measure the motor function of individuals with Duchenne Muscular Dystrophy (DMD).

It was also noted that crossover-treated patients with average age of 7.18 years showed clinically meaningful and statistically significant functional benefit for NSAA, Time to Rise (TTR), and 10-meter walk/run (10MWR) function tests compared to those treated in Part 1 with average age of 5.98 years.