Capricor Therapeutics (NASDAQ:CAPR) saw its stock plummet by 53% on Friday after the U.S. Food and Drug Administration issued a Complete Response Letter (CRL), rejecting the company’s bid for approval of its Duchenne muscular dystrophy (DMD) therapy, Deramiocel.
The agency stated that the company’s Biologics License Application (BLA) lacked the required substantial evidence to demonstrate the drug’s effectiveness. It also pointed to unresolved issues in the Chemistry, Manufacturing, and Controls (CMC) section, despite Capricor’s belief that most concerns had been previously addressed.
CEO Linda Marbán expressed disappointment and said the outcome was unexpected, especially since the review had progressed without major setbacks. She highlighted that the process had included a successful pre-licensure inspection and mid-cycle review. The BLA had received Priority Review designation earlier this year in March.
Capricor plans to refile its application using data from its ongoing Phase 3 HOPE-3 trial, with topline results anticipated in the third quarter of 2025. The HOPE-3 study is a randomized, placebo-controlled trial involving 104 participants.
The FDA has invited Capricor to request a Type A meeting to outline a path forward. The company said it remains committed to developing Deramiocel for the treatment of cardiomyopathy in DMD patients.
The original submission was based on findings from the earlier HOPE-2 study, an open-label extension, and comparative natural history data, which the FDA ultimately deemed insufficient for approval.
Capricor Therapeutics stock price
This content is for informational purposes only and does not constitute financial, investment, or other professional advice. It should not be considered a recommendation to buy or sell any securities or financial instruments. All investments involve risk, including the potential loss of principal. Past performance is not indicative of future results. You should conduct your own research and consult with a qualified financial advisor before making any investment decisions.