Capricor Says FDA Briefing Relies On Obsolete Analysis Plan Ahead Of Deramiocel Advisory Committee Meeting For Duchenne Muscular Dystrophy BLA

Capricor Therapeutics (NASDAQ:CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today provided an update ahead of the Company's upcoming FDA Advisory Committee meeting, at which an independent advisory committee will discuss Capricor's Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD), as part of the FDA's ongoing review of the application.

"Capricor has engaged fully and transparently with the FDA throughout the review process for our Biologics License Application for Deramiocel and has been responsive to every request from the FDA. Our results are governed by the final analysis plan, SAP version 3.0, which was finalized prior to unblinding. It is critical to understand that the post-hoc analyses in the FDA’s briefing materials rely on SAP version 1.1, an unsigned incomplete internal draft which became obsolete with the addition of cohort B and did not include content specifically requested by FDA. The Phase 3 HOPE-3 results demonstrate a statistically significant benefit on the primary endpoint, PUL 2.0, with supportive benefits in cardiac function, and we believe Deramiocel offers a meaningful treatment option for boys and young men living with Duchenne, who continue to face a significant unmet medical need. Ahead of the meeting, we have posted a portion of our slides to accurately frame the discussion for stakeholders on our website," said Linda Marbán, Ph.D., Chief Executive Officer of Capricor Therapeutics.

These materials will be available in the Investors section of the Company’s website shortly.