Capricor Therapeutics announced today its intent to file a Biologics License Application (BLA) seeking full approval of deramiocel for the treatment of Duchenne-cardiomyopathy based on existing cardiac data from their Phase 2 trials of deramiocel (CAP-1002) compared to natural history data. Deramiocel is the company’s novel cell therapy for the treatment of patients diagnosed with Duchenne cardiomyopathy. 

According to Capricor, following recent meetings with the U.S. Food and Drug Administration (FDA) the company plans to commence the filing of a BLA in October of 2024 seeking full approval of deramiocel for the treatment of Duchenne-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 (OLE) trials compared to natural history data provided by Vanderbilt University Medical Center and Cincinnati Children’s Hospital Medical Center.

Capricor has indicated that it plans to combine Cohorts A and B of the Phase 3 HOPE-3 clinical trial to serve as a post-approval study in order to support potential label expansion to treat Duchenne skeletal muscle myopathy. Capricor does not intend to unblind Cohort A at this time, which was expected to occur in the fourth quarter of 2024.

PPMD has requested a webinar with Capricor and we will share additional information as it becomes available.

Read Capricor’s press release here.