(RTTNews) - Sarepta Therapeutics Inc. (SRPT), on Monday, announced positive topline three-year results from its EMBARK study, showing that ELEVIDYS significantly slowed disease progression across key functional measures in ambulatory patients with Duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) is a severe, progressive neuromuscular disorder caused by mutations in the DMD gene, leading to the absence of dystrophin and gradual loss of muscle function. Children with Duchenne often experience worsening mobility between ages 7 and 10, with declines in walking, rising from the floor, and other daily movements.
The three-year data come from Part 1-treated patients in EMBARK, Sarepta's global, randomized, placebo-controlled Phase 3 trial evaluating ELEVIDYS in ambulatory boys aged 4-7 at the time of dosing. The study's primary endpoint was change from baseline in the North Star Ambulatory Assessment (NSAA) at Week 52, with long-term follow-up extending through multiple years.
Three-Year Results Show Strong and Growing Treatment Effect
Across all key functional measures, ELEVIDYS-treated patients demonstrated clinically meaningful and durable benefit compared with a pre-specified, propensity-weighted external control group:
• NSAA: Mean score remained above baseline at Year 3, while the external control group declined.
• Time to Rise (TTR): 73% slowing of disease progression.
• 10-meter walk/run (10MWR): 70% slowing of disease progression.
Notably, the treatment effect widened between Years 2 and 3, suggesting increasing divergence from the expected disease trajectory.
The company also noted that the safety profile remained consistent with prior experience in ambulatory patients treated with ELEVIDYS.
Study Design and Long-Term Follow-Up
EMBARK is a two-part, crossover Phase 3 study in which participants received either ELEVIDYS or a placebo in Part 1, then crossed over in Part 2. Following completion of EMBARK, patients were eligible to enroll in EXPEDITION, a long-term follow-up study. Of the 64 patients treated with ELEVIDYS in Part 1, 52 continue to be followed three years after treatment.
ELEVIDYS: The Only Approved Gene Therapy for Duchenne
ELEVIDYS is a single-dose AAV-based gene therapy designed to deliver a micro-dystrophin transgene to skeletal muscle. It is currently the only approved gene therapy for Duchenne, available to ambulatory individuals aged 4 and older under an updated FDA label issued at the end of 2025.
Sarepta continues to collaborate with Roche to expand global access, with Roche responsible for regulatory approvals and commercialization outside the United States. ELEVIDYS has now been administered to more than 1,200 patients worldwide across clinical and real-world settings.
Next Steps
Sarepta plans to present the full three-year dataset at upcoming medical meetings and publish additional analyses. Two-year EMBARK results were recently published in Neurology and Therapy, and one-year results appeared in Nature Medicine.
SRPT has traded between $10.42 and $120.05 over the past year. The stock is currently trading at $22.38, up 5.92%.
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