Zinger Key Points
- Crossover-treated patients showed a 2.34-point NSAA improvement at 52 weeks vs. external controls despite an older average age.
- Part 1 patients sustained a +2.88 NSAA improvement two years post-treatment with consistent micro-dystrophin expression.
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On Monday, Sarepta Therapeutics Inc. SRPT released topline results from Part 2 of EMBARK (Study SRP-9001-301) Phase 3 clinical study of Elevidys (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne muscular dystrophy.
Crossover-treated patients who received a placebo in Part 1, crossed over at 52 weeks and were treated with Elevidys in Part 2 improved 2.34 points from baseline compared to matched external controls on the North Star Ambulatory Assessment (NSAA) 52 weeks after treatment. NSAA measures functional motor abilities in children with Duchenne.
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Despite being one year older (average age 7.18 years) than those treated in Part 1 (average age 5.98 years), crossover-treated patients showed clinically meaningful and statistically significant functional benefit for NSAA, Time to Rise (TTR) and 10-meter walk/run (10MWR) function tests compared with a pre-specified, propensity-weighted external control group (EC).
Individuals treated in part one of EMBARK (n=63) showed clinically meaningful and statistically significant improvements on the NSAA sustained two years after treatment with Elevidys. Functional motor improvements include:
- +2.88 points (improvement) on NSAA.
- -2.06 seconds (improvement) on Time to Rise.
- -1.36 seconds (improvement) on 10MWR.
Muscle biopsies from a subset of patients taken 64 weeks after dosing in part one showed consistent and sustained expression of micro-dystrophin. Muscle pathology on MRI continues to show minimal progression in underlying muscle pathology and remains highly consistent with the functional benefits shown.
No new safety signals were observed.
As part of a collaboration agreement signed in 2019, Sarepta is working with Roche Holdings AG RHHBY. Sarepta is responsible for regulatory approval and commercializing Elevidys in the U.S. and manufacturing.
Roche is responsible for regulatory approvals and bringing Elevidys to patients across the rest of the world.
Price Action: SRPT stock closed at $116.46 on Friday.
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