Capricor Therapeutics announced an update on the Company?s positive Type-B clinical meeting with the U.S. Food and Drug Administration (?FDA?) on the design and execution of HOPE-3, Capricor?s pivotal Phase 3 trial with lead asset CAP-1002 in treating Duchenne muscular dystrophy (?DMD?). Feedback from the FDA on trial design and timeline confirms CAP-1002?s path towards a future Biologics License Application (?BLA?) submission. The FDA has affirmed alignment on the Phase 3 clinical trial?s design and timeline.

Key details for HOPE-3 are as follows: Primary endpoints remain unchanged. HOPE-3 will aim to enroll approximately 58 patients and enrollment is estimated to be completed in the fourth quarter of 2023. Capricor has treated 52 patients.

Topline data for HOPE-3 is expected in the fourth quarter of 2024. Capricor plans to submit a BLA for CAP-1002 in 2025, which will be supported by results using product manufactured at the Los Angeles site. Capricor and the FDA discussed the potential for alternative approval pathways and Capricor plans to further discuss these options with the FDA following the completion of enrollment.

CAP-1002 for the treatment of DMD has received Orphan Drug Designation and the regulatory pathway for CAP-1002 is supported RMAT (Regenerative Medicine Advanced Therapy Designation). In addition, if Capricor were to receive FDA marketing approval for CAP-1002 for the treatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (?PRV?) based on its previous receipt of a rare pediatric disease designation. Capricor retains full rights to the PRV, if received.

Duchenne muscular dystrophy (?DMD?) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles. Patients suffering from DMD typically lose their ability to walk in their teenage years and generally die of cardiac or respiratory complications by age 30. It occurs in approximately one in every 3,600 live male births across all races, cultures and countries.

DMD afflicts approximately 200,000 boys and young men around the world. Treatment options are limited and there is no cure. CAP-1002 consists of allogeneic cardiosphere-derived cells (?CDCs?), a population of stromal cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory, antifibrotic and regenerative actions in dystrophinopathy and heart failure.

CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile so that they adopt a healing, rather than a pro-inflammatory, phenotype. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 human subjects across several clinical trials.