Capricor to File BLA for Full Approval of Deramiocel for Duchenne Muscular Dystrophy Cardiomyopathy

Capricor Therapeutics, a biotechnology firm renowned for developing cell and exosome-based treatments for rare diseases, has announced its intention to file a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA). This decision follows recent discussions with the FDA and is based on existing cardiac and natural history data. The application, aimed at obtaining approval for deramiocel to treat cardiomyopathy in patients with Duchenne muscular dystrophy (DMD), will begin in October 2024.

The planned BLA submission will rely on cardiac data from the Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials, compared with natural history data from Vanderbilt University Medical Center and Cincinnati Children’s Hospital Medical Center. In an effort to potentially expand the treatment indication to include DMD skeletal muscle myopathy, Capricor intends to use Cohorts A and B of the Phase 3 HOPE-3 clinical trial as a post-approval study, without unblinding Cohort A as initially planned for the fourth quarter of 2024.

Linda Marbán, Ph.D., CEO of Capricor, emphasized the urgent need for approved therapies for DMD cardiomyopathy, a leading cause of death in DMD patients. She highlighted the strong cardiac data from their studies and the FDA's dedication to advancing treatments for rare diseases as vital factors in seeking approval. Marbán also mentioned the intention to expand the label for skeletal muscle myopathy after initial approval.

Deramiocel has demonstrated significant effectiveness in reducing cardiac issues associated with DMD across multiple clinical trials. Marbán believes that the safety and efficacy data support their expedited path to potential approval, aiming to bring this novel treatment to patients swiftly. She acknowledged the support from patients, families, and advocates, as well as the FDA's commitment to accelerating DMD treatments.

Deramiocel, which consists of allogeneic cardiosphere-derived cells (CDCs), has received the FDA Orphan Drug Designation and benefits from the RMAT (Regenerative Medicine Advanced Therapy) Designation. Should Capricor receive FDA marketing approval for deramiocel for DMD treatment, they would qualify for a Priority Review Voucher (PRV) due to their previous rare pediatric disease designation.

The Phase 2 HOPE-2 and HOPE-2 OLE trials provided the cardiac data foundational to the BLA submission. These studies demonstrated deramiocel’s potential to mitigate cardiac symptoms in DMD patients significantly. The historical data comparison involved collaborators from Vanderbilt University Medical Center and Cincinnati Children’s Hospital Medical Center.

Capricor’s strategic approach involves leveraging their rigorous clinical and preclinical data to navigate the regulatory pathway efficiently. Their lead product, deramiocel, not only shows promise in treating DMD cardiomyopathy but also holds potential for broader applications within muscular dystrophies and heart diseases, thanks to its immunomodulatory, antifibrotic, and regenerative properties.

Capricor's commitment to advancing its therapeutic pipeline is further evidenced by their internal GMP (Good Manufacturing Practice) manufacturing capabilities, ensuring the necessary support for the BLA and commercialization phases.

The company scheduled a webcast to discuss these updates, providing stakeholders with insights into their progress and future plans. This transparency underscores Capricor's dedication to engaging with the community and keeping them informed about critical developments.

In summary, Capricor Therapeutics is poised to make significant strides in treating DMD cardiomyopathy through their upcoming BLA submission for deramiocel, leveraging robust clinical data and strong regulatory support. Their innovative approach and commitment to addressing unmet medical needs position them as a key player in the landscape of rare disease therapeutics.