Capricor reports positive news on the path to approval for Duchenne cell therapy

Capricor Therapeutics shared a regulatory update for its BLA for deramiocel, the company’s lead cell therapy candidate for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).

The update follows a recent Type A meeting with the U.S. FDA after the company was hit with a surprise CRL in July 2025. In the CRL, the FDA said that the BLA does not meet the statutory requirement for substantial evidence of effectiveness and cited the need for additional clinical data. The CRL also referenced certain outstanding items in the chemistry, manufacturing and controls section of the application, but, according to a press release from Capricor, the company believes it has addressed these issues in prior communications with the FDA.

Now, Capricor says the CMC items identified in the CRL have been addressed and communicated to the FDA. According to the company, 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. The trial is a randomized, double-blind, placebo-controlled clinical trial of 104 patients, with topline results expected mid-Q4 2025 to support BLA resubmission.

Deramiocel consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in dystrophiopathies, such as DMD.