CAPR - Capricor Therapeutics Announces Positive Type-B Meeting with FDA for CAP-1002 Program for Duchenne Muscular Dystrophy | Benzinga
-Company Aligned with FDA on Demonstration of Non-Clinical Comparability; Allowing for Immediate Use of San Diego Manufacturing Facility-
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-Company Granted Subsequent Type-B Clinical Meeting with FDA in Second Quarter to Continue to Discuss Pathway to BLA-
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SAN DIEGO, April 24, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ:CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment and prevention of rare diseases, today announced an update from the Company's recent Type-B Chemistry, Manufacturing and Controls ("CMC") meeting with the U.S. Food and Drug Administration ("FDA") on next steps for the Biologics License Application ("BLA") submission with its lead asset CAP-1002 in treating Duchenne muscular dystrophy ("DMD").
The FDA has affirmed alignment with Capricor on the following topics:
Pre-BLA Meeting and Rolling BLA Submission
- The FDA advised Capricor to include discussion for a pre-BLA meeting and rolling BLA schedule in the upcoming Type-B meeting.
- Based on this feedback, Capricor has already been granted a subsequent Type-B meeting to be held in the second quarter of 2024 to discuss these topics, with the results of those discussions to potentially lead to an accelerated BLA filing.
- Capricor plans to share with FDA its HOPE-2 open label extension ("OLE") 3-year safety and efficacy data which is expected to be available in the second quarter of 2024 as part of Capricor's ISS and ISE strategy.
Establishment of Non-Clinical Comparability
- The FDA agreed that comparability between drug product manufactured at our two different facilities (Los Angeles and San Diego) has been demonstrated using the provided analytical comparability data.
- This allows for the use of CAP-1002 drug product manufactured at our San Diego manufacturing facility upon potential product approval.
- Data from Cohort B of the HOPE-3 clinical trial will not be necessary for FDA approval of the product.
"I am extremely pleased with our recent FDA interactions as we continue to work collaboratively with the agency to align on the most expeditious path towards registration of CAP-1002 for the treatment of DMD," said Linda Marbán, Ph.D., ...