Capricor to submit HOPE-3 data with complete response to FDA CRL

Capricor Therapeutics (CAPR) announced a regulatory update for its Biologics License Application for Deramiocel, the Company’s investigational cell therapy for the treatment of Duchenne muscular dystrophy. This update follows a recent Type A meeting with the U.S. Food and Drug Administration after the receipt of a Complete Response Letter in July 2025. The goal of the Type A meeting was to establish a path toward potential approval of Deramiocel for the treatment of DMD. Key outcomes included: The HOPE-3 clinical trial should serve as the “additional study” requested in the CRL. The HOPE-3 data can be submitted within the current BLA, maintaining PUL v2.0 as the primary efficacy endpoint and suggesting left ventricular ejection fraction as a key secondary endpoint, which Capricor intends to request for labeling consideration. Capricor plans to submit HOPE-3 data with its complete response to the CRL, with the goal of securing a label encompassing both cardiac and skeletal muscle function in DMD. In its meeting minutes, the FDA further emphasized its commitment, stating: “The FDA remains committed to collaborating with the applicant and will exercise further regulatory flexibility by reviewing data from the HOPE-3 trial.” Importantly, prior to issuance of the CRL, the majority of the BLA had undergone rigorous review with no significant deficiencies identified by the FDA during the mid-cycle review or pre-licensing inspections. All CMC items identified in the CRL have been addressed and communicated to the FDA. Capricor believes that the addition of HOPE-3 data will further strengthen the clinical package and support the broad potential of Deramiocel as a treatment for DMD. The Company also maintains a strong financial position to support the advancement of Deramiocel through regulatory review and toward potential launch.