Capricor Therapeutics Announces Positive Type-B Meeting with FDA for CAP-1002 Program for Duchenne Muscular Dystrophy

-Company Aligned with FDA on Demonstration of Non-Clinical Comparability; Allowing for Immediate Use of San Diego Manufacturing Facility-

-FDA Feedback Supports Requests for a Pre-BLA Meeting and Subsequent Rolling BLA Submission Following Upcoming Q2 Type-B FDA Meeting-

-Company Granted Subsequent Type-B Clinical Meeting with FDA in Second Quarter to Continue to Discuss Pathway to BLA-

-Capricor Management to Host Virtual Investor Webcast to Discuss Latest Program Updates on Monday, April 29 at 8:30 a.m. ET-

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.