Capricor Locks in Type A Meeting With the FDA to Discuss the Future of DMD Cardiomyopathy Cell Therapy Candidate Deramiocel

Capricor Therapeutics has scheduled a Type A meeting with the FDA to discuss future regulatory moves for Deramiocel (also known as CAP-1002), an investigational allogeneic cardiosphere-derived cell therapy intended for the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy.¹

The company noted that as a result of the meeting’s scheduling, which will focus on the pathway to resubmission of a biologics license application (BLA) for Deramiocel, it has rescheduled its release of financial results for the second quarter to August 11, 2025. Capricor will additionally hold a webcast and conference call on that date in which it will provide a corporate update.

Capricor’s initial BLA for Deramiocel completed its rolling submission in January 2025 and was accepted for review by the FDA in March 2025.^2,3 The BLA was supported by results from the completed HOPE-2 clinical trial (NCT03406780) and HOPE-2’s ongoing open-label extension (OLE) study (HOPE-2-OLE; NCT04428476), with data from these studies being compared to an FDA funded natural history dataset.

In July 2025, however, Capricor’s BLA was met with a complete response letter (CRL) from the FDA in which the agency stated that the statutory requirement for “substantial evidence of effectiveness” was not met and that more clinical data will be needed.⁴ Furthermore, the FDA also drew attention to outstanding parts of the BLA’s Chemistry, Manufacturing, and Controls (CMC) section, that were not reviewed by the agency because of the CRL’s timing; although, Capricor stated that most of these CMC items had been addressed previously in its interactions with the FDA.

At the time, the company pointed out that it planned a resubmission of the BLA, with additional data to be included from the ongoing phase 3 HOPE-3 clinical trial (NCT05126758), for 2025’s third quarter. HOPE-3 data had notably been absent from the original BLA.

“We are surprised by this decision by the FDA,” Linda Marbán, PhD, the chief executive officer of Capricor, said in a statement at the time the CRL was announced.¹ “We have followed their guidance throughout the process. Prior to the CRL, the review had advanced without major issues, including a successful prelicensure inspection and completion of the midcycle review. Capricor plans to submit data from the phase 3 HOPE-3 clinical trial to provide additional evidence of effectiveness from an adequate and well-controlled study. The HOPE-3 trial is a randomized, double-blind, placebo-controlled clinical trial of 104 patients, with topline results expected in the third quarter of 2025. We believe these data, if positive, along with our existing long-term clinical results showing cardiac stabilization, preservation of skeletal muscle function, and a consistent safety profile, could support efforts to resolve the questions raised by the FDA for the treatment of cardiomyopathy associated with DMD. While this was an unexpected decision by the FDA, we remain committed to the DMD community to get deramiocel through the approval process.”

REFERENCES
1. Capricor Therapeutics announces type A meeting scheduled with U.S. FDA. News release. Capricor Therapeutics. August 8, 2025. Accessed August 8, 2025. https://www.capricor.com/investors/news-events/press-releases/detail/322/capricor-therapeutics-announces-type-a-meeting-scheduled
2. Capricor Therapeutics completes submission of biologics license application to the U.S. FDA for Deramiocel for the treatment of Duchenne muscular dystrophy. News release. Capricor Therapeutics. January 2, 2025. Accessed August 8, 2025. https://www.capricor.com/investors/news-events/press-releases/detail/303/capricor-therapeutics-completes-submission-of-biologics
3. Capricor Therapeutics announces FDA acceptance and priority review of its biologics license application for Deramiocel to treat Duchenne muscular dystrophy. News release. Capricor Therapeutics. March 4, 2025. Accessed August 8, 2025. https://www.capricor.com/investors/news-events/press-releases/detail/305/capricor-therapeutics-announces-fda-acceptance-and-priority
4. Capricor Therapeutics provides regulatory update on Deramiocel BLA for Duchenne muscular dystrophy. News release. Capricor Therapeutics. July 11, 2025. Accessed August 8, 2025. https://www.capricor.com/investors/news-events/press-releases/detail/319/capricor-therapeutics-provides-regulatory-update-on