Capricor Therapeutics (NASDAQ: CAPR) announced that The Lancet has published results from its phase 3 HOPE-3 clinical trial evaluating deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD).

The randomized, double-blind, placebo-controlled trial enrolled 106 participants. According to the press release, the trial met its primary endpoint, with deramiocel slowing upper limb function decline by 54% compared to placebo, as measured by the Performance of Upper Limb 2.0 scale (p=0.03). The trial also reported clinically meaningful improvements in cardiac measures.

Craig McDonald, M.D., Distinguished Professor at UC Davis Health and lead author of the publication, said the trial is "the first Phase 3 trial to demonstrate a significant benefit on function in a largely non-ambulatory DMD population."

The publication followed independent expert peer review, which Capricor said validated the trial's design, statistical methodology, and findings. Results are based on the company's prespecified Statistical Analysis Plan version 3.0.

Capricor's Biologics License Application for deramiocel remains under active FDA review, with a PDUFA target action date of August 22, 2026. Deramiocel holds Orphan Drug Designation from both the FDA and the European Medicines Agency, as well as Regenerative Medicine Advanced Therapy designation in the U.S. and Rare Pediatric Disease Designation from the FDA.

DMD is a genetic disorder affecting approximately 15,000 individuals in the United States, primarily boys, and is characterized by progressive muscle degeneration. There is currently no cure for the disease.

Deramiocel remains an investigational candidate and has not been approved for commercial use in any indication.