Overview
- The FDA staff released briefing materials Monday that said the pivotal HOPE-3 trial for deramiocel showed methodological and evidentiary problems and that the disclosure sent Capricor’s stock tumbling about 70 percent.
- Reviewers said Capricor changed how the trial measured its main outcomes after the study ended, converting raw scores to percent changes and altering cardiac assessments in ways the agency called scientifically unjustified.
- Federal reviewers reported limited evidence that the intravenously given cells reached heart tissue in meaningful amounts and noted that enrolled patients had near‑normal average heart‑pumping function at baseline, raising questions about whether they had measurable DMD cardiomyopathy.
- Capricor formally disputed the staff analysis, saying its results follow a final analysis plan (SAP v3.0) that it says was fixed before unblinding and that the Phase 3 HOPE-3 data show a statistically significant benefit on the trial’s primary endpoint.
- The Cellular, Tissue, and Gene Therapies advisory committee will give nonbinding advice on July 29 and its recommendation could shape the FDA’s decision, investor confidence in cell therapies for rare diseases, and the prospects for patients with Duchenne‑related heart disease.