Capricor Therapeutics announces Type-B meeting with FDA for CAP-1002

Capricor Therapeutics announced an update on the Company’s positive Type-B clinical meeting with the U.S. Food and Drug Administration on the design and execution of HOPE-3, Capricor’s pivotal Phase 3 trial with lead asset CAP-1002 in treating Duchenne muscular dystrophy. Feedback from the FDA on trial design and timeline confirms CAP-1002’s path towards a future Biologics License Application submission. “We are pleased to have reached an important regulatory milestone that further defines the path towards registration of CAP-1002 for DMD and brings us potentially one step closer to addressing the great unmet medical need for these patients,” said Linda Marban, Ph.D., Capricor’s CEO. “During recent meetings with the FDA, we aligned on key features of HOPE-3, which as currently designed, and if successful, is expected to provide sufficient evidence of effectiveness to support our BLA submission and significantly expedites our path towards potential approval of CAP-1002. In addition, although product from our San Diego site would not be required to support registration of CAP-1002, we do plan to enroll a separate cohort with product manufactured from our San Diego site, with a view toward meeting potential increased commercial demand following initial registration. Looking ahead, we remain on track to complete HOPE-3 enrollment and expect to report the outcome of an interim analysis in the fourth quarter of 2023. We look forward to continuing to deliver important clinical and regulatory milestones, including the submission of a BLA for CAP-1002 for the treatment of DMD.” 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. As of today, 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.