Capricor Therapeutics to file BLA for deramiocel in DMD cardiomyopathy; rolling submission in Oct 2024

CAPRICOR THERAPEUTICS, INC.

Plans to file BLA for deramiocel to treat DMD cardiomyopathy based on existing cardiac and natural history data; rolling submission begins Oct 2024 with full submission by year-end 2024.
BLA supported by Phase 2 HOPE-2 and HOPE-2 OLE data vs natural history from Vanderbilt and Cincinnati Children's.
Will combine Cohorts A and B of Phase 3 HOPE-3 as a post-approval study; no unblinding of Cohort A as previously planned for Q4 2024.
Deramiocel has FDA Orphan Drug, RMAT, and Rare Pediatric Disease designations (PRV eligible upon approval).
No approved therapies for DMD cardiomyopathy; deramiocel targets leading cause of death in Duchenne.

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