Capricor's Lead Duchenne Therapy Hits FDA Regulatory Roadblock

Core Viewpoint - Capricor Therapeutics Inc. received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application (BLA) for Deramiocel, indicating that the application cannot be approved in its current form due to insufficient evidence of effectiveness and the need for additional clinical data [1][2]. Group 1: FDA Response and Requirements - The FDA stated that the BLA does not meet the statutory requirement for substantial evidence of effectiveness and that additional clinical data is needed [2]. - The CRL referenced outstanding items in the Chemistry, Manufacturing and Controls (CMC) section, which Capricor claims to have addressed in prior communications, but the FDA did not review these materials due to the timing of the CRL issuance [3]. Group 2: Next Steps and Company Plans - The FDA confirmed that it will restart the review clock upon resubmission and offered Capricor the opportunity to request a Type A meeting to discuss the path forward [4]. - Capricor plans to submit data from the Phase 3 HOPE-3 clinical trial to provide additional evidence of effectiveness, with topline results expected in the third quarter of 2025 [5]. Group 3: Market Reaction - Following the news, CAPR stock is down 37.4% at $7.14 during the premarket session [6].