Overview
- The Lancet published the HOPE‑3 Phase 3 paper that reports deramiocel slowed upper‑limb decline by 54% and found signals of cardiac benefit in subgroup analyses.
- FDA reviewers publicly challenged the trial, saying key cardiac data were limited and that Capricor changed statistical analyses after the study ended, which they say weakens the evidence.
- The FDA Cellular, Tissue, and Gene Therapies Advisory Committee voted 9–3 against the therapy’s cardiac benefit, with panelists noting missing scans, small subgroups, and uncertainty that heart measures reflect meaningful patient gain.
- Capricor defended the Lancet peer review and its prespecified Statistical Analysis Plan v3.0, but the panel’s vote triggered a nearly 60% drop in Capricor shares and keeps regulatory risk high.
- The final FDA decision remains set for August 22 and will determine whether deramiocel, a potential mutation‑agnostic cell therapy for advanced Duchenne, reaches patients who face progressive skeletal and cardiac decline.