FDA says Capricor's DMD cell therapy failed to meet Phase 3 endpoints

The FDA briefing documents released before an advisory committee meeting state that the Phase 3 trial of Capricor's deramiocel did not achieve its pre-specified primary and secondary efficacy endpoints.

The company had previously reported in December that the HOPE-3 trial met both primary and secondary endpoints, but FDA reviewers noted no statistically significant difference between deramiocel and placebo at 12 months.

Reviewers also highlighted that after the double-blind portion, the statistical analysis plan was altered multiple times during the open-label extension, changing endpoint definitions and analytical methods.