Capricor Therapeutics’ Deramiocel Fails FDA Phase 3 Trial in Duchenne Muscular Dystrophy

The U.S. Food and Drug Administration determined that Capricor Therapeutics’ experimental stem‑cell therapy deramiocel did not achieve the primary or secondary endpoints in its Phase 3 trial for Duchenne muscular dystrophy. Review documents showed no statistically significant improvement in upper‑arm muscle function or cardiac imaging compared with placebo, and the agency highlighted safety concerns including infusion‑related reactions and anaphylaxis.

The FDA also criticized post‑study modifications to the trial’s statistical analysis plan, stating that changes made after data collection could bias results. Capricor CEO Linda Marban contested the regulator’s characterization, claiming the agency relied on an early draft of the analysis plan. An FDA advisory committee is scheduled to meet on 29 July to discuss deramiocel’s potential approval, a key date that investors are watching closely.