Capricor Therapeutics Prepares Full HOPE-3 Study Report For FDA After Phase 3 Results Show Promise In Duchenne Muscular Dystrophy

Capricor Therapeutics, a biotechnology company focused on developing innovative cell and exosome-based therapies for rare diseases, has provided an update on its Biologics License Application (BLA) for Deramiocel, its investigational first-in-class cell therapy for Duchenne muscular dystrophy (DMD). The company previously shared topline results from its Phase 3 HOPE-3 clinical study with the U.S. Food and Drug Administration (FDA) in late 2025. 

Following a review of these data, the FDA has formally requested the full HOPE-3 clinical study report (CSR) along with supporting materials to address the items outlined in the Complete Response Letter (CRL) issued to Capricor. Notably, the FDA did not request any additional clinical studies or new patient data as part of this request.

Capricor is actively preparing the HOPE-3 CSR and expects to submit the requested materials to the FDA in February 2026. The company anticipates that this submission will address the concerns raised in the CRL and enable continued review of the BLA, including the establishment of a new Prescription Drug User Fee Act (PDUFA) target action date.

Linda Marbán, Ph.D., Chief Executive Officer of Capricor, emphasized the company’s commitment to collaborating with the FDA to ensure an efficient review process. She highlighted that the request for the full HOPE-3 study report is an expected and appropriate next step following the FDA’s review of the topline data. Marbán noted that the HOPE-3 study demonstrated statistically significant and clinically meaningful improvements in both skeletal muscle and cardiac function, key factors that influence disease progression and long-term outcomes in Duchenne muscular dystrophy. 

She added that these results build on over a decade of consistent clinical evidence and reinforce Capricor’s confidence in Deramiocel’s potential. Marbán concluded that the company’s near-term priority is to address the FDA’s request and continue working collaboratively so that patients with late-stage DMD, who currently have limited treatment options, may gain access to Deramiocel as soon as possible.