Overview
- The FDA staff briefing documents released Monday triggered about a 70% drop in Capricor (CAPR) stock after reviewers flagged serious problems in the company’s HOPE‑3 submission.
- Reviewers criticized post‑trial changes to how primary outcomes were measured, saying converting raw arm‑function scores to percent change and then back to raw values was not scientifically justified and reduces accuracy.
- The agency also noted Capricor moved from measuring left ventricular ejection fraction to a ranking approach for cardiac results and questioned whether enrolled patients had clear DMD‑related cardiomyopathy at baseline.
- FDA staff wrote that available data do not convincingly show therapeutically meaningful amounts of the intravenously delivered deramiocel reach heart tissue, which undermines the therapy’s claimed cardiac benefit.
- A Cellular, Tissue, and Gene Therapies Advisory Committee will review the HOPE‑3 record on July 29 and its recommendation is likely to shape whether regulators regard the trial as substantial evidence of effectiveness, affecting patients and investors.