Capricor Therapeutics Announces Plans to File BLA for Deramiocel in the Treatment of Duchenne Muscular Dystrophy Cardiomyopathy, Shares Close Up 52%

Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on cell and exosome-based therapies, has announced its intent to file a Biologics License Application (BLA) for deramiocel (CAP-1002) for the treatment of cardiomyopathy in Duchenne muscular dystrophy (DMD) patients. The BLA submission is expected to begin in October 2024 and will be supported by positive cardiac data from Phase 2 HOPE-2 trials and natural history data. Full submission is anticipated by the end of 2024. Following the announcement, Capricor’s stock surged, closing up 52.43%.

Key Highlights:

• Label Expansion Potential: The initial approval would target cardiomyopathy in DMD patients, with potential for label expansion to include skeletal muscle myopathy pending positive results from the ongoing Phase 3 HOPE-3 trial.
• Significant Market Opportunity: The DMD drugs market is projected to reach $5.5 billion by 2032. Approximately 25-59% of DMD patients develop cardiomyopathy, positioning deramiocel to meet a significant unmet need.
• Regulatory Path: Deramiocel has FDA Orphan Drug Designation and RMAT designation. If approved, Capricor would be eligible for a Priority Review Voucher, potentially worth over $100 million.