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Investing.com -- Capricor Therapeutics (NASDAQ:CAPR) stock plunged 53% after the company received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) rejecting its application for Deramiocel, a cell therapy for Duchenne muscular dystrophy (DMD).
The FDA stated that the Biologics License Application (BLA) "does not meet the statutory requirement for substantial evidence of effectiveness" and requires additional clinical data. The agency also noted outstanding Chemistry, Manufacturing, and Controls (CMC) issues, though Capricor believes it had addressed most of these in prior communications.
Capricor CEO Linda Marbán expressed surprise at the decision, noting that "prior to the CRL, the review had advanced without major issues, including a successful pre-licensure inspection and completion of the mid-cycle review." The company had been granted Priority Review for the application in March 2025.
The biotechnology company now plans to resubmit its BLA with data from its ongoing Phase 3 HOPE-3 trial, which is expected to produce topline results in Q3 2025. The HOPE-3 study is a randomized, double-blind, placebo-controlled trial involving 104 patients.
The FDA has offered Capricor the opportunity to request a Type A meeting to discuss next steps toward potential approval. The company remains focused on pursuing the indication for treating cardiomyopathy associated with DMD.
Capricor’s original submission included data from the HOPE-2 trial, its open-label extension, and natural history comparisons from FDA-funded datasets, which ultimately proved insufficient for approval.
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