FDA disputes Capricor's claims of Phase 3 success for DMD cell therapy, deramiocel

The FDA has publicly disputed Capricor Therapeutics’ claim that its Phase 3 HOPE-3 trial for the Duchenne muscular dystrophy (DMD) cell therapy deramiocel met its primary endpoints, stating the study "did not meet its pre-specified primary and secondary efficacy endpoints" and showed no statistically significant difference from placebo at 12 months.

FDA briefing documents ahead of a July 29, 2026, advisory committee meeting highlighted that Capricor made multiple changes to the statistical analysis plan (SAP) after the randomized portion of the trial, including redefining endpoints and data‑imputation methods, which the agency views as undermining the validity of the success claim.

The FDA raised concerns about blinding in HOPE‑3, noting a marked difference in hypersensitivity‑reaction rates between deramiocel‑treated and placebo‑treated patients, which may have unmasked treatment assignment.

An FDA advisory committee voted 9 to 3 against deramiocel, agreeing with staff that the evidence for efficacy in DMD‑associated cardiomyopathy is insufficient, setting the stage for a potential regulatory rejection by the August 22, 2026, PDUFA date.

Capricor disputes the FDA’s interpretation, maintaining that final SAP version 3.0 before unblinding supports a statistically significant benefit on the primary upper‑limb performance endpoint and supportive benefits in cardiac function, and argues that the FDA analyses are based on an obsolete draft of the SAP.