Capricor announces intent for file BLA for full approval of Deramiocel

Capricor Therapeutics announced, following recent meetings with the U.S. Food and Drug Administration, its intent to file a Biologics License Application, BLA, based on existing cardiac and natural history data for deramiocel to treat all patients diagnosed with Duchenne muscular dystrophy cardiomyopathy. Following the FDA meetings: Capricor plans to commence the filing of a BLA in October of 2024 seeking full approval of deramiocel for the treatment of DMD-cardiomyopathy with full submission expected by year-end 2024. The BLA filing will be based on existing cardiac data from the Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials compared to natural history data provided by Vanderbilt University Medical Center and Cincinnati Children’s Hospital Medical Center. In order to support potential label expansion to treat DMD skeletal muscle myopathy, Capricor plans to combine Cohorts A and B of the Phase 3 HOPE-3 clinical trial to serve as a post-approval study and does not intend to unblind Cohort A at this time, which was expected to occur in the fourth quarter of 2024.