FDA Accepts BLA for Capricor’s DMD Cardiomyopathy Cell Therapy Deramiocel With Priority Review

The FDA has accepted the biologics license application (BLA) that Capricor Therapeutics submitted for Deramiocel (also known as CAP-1002), an investigational allogeneic cardiosphere-derived cell therapy intended to treat Duchenne muscular dystrophy (DMD) cardiomyopathy, with priority review.¹ The agency has set the Prescription Drug User Fee Act (PDUFA) action date for the BLA as August 31, 2025.

According to Capricor, the FDA indicated it had not yet made a decision regarding whether an Advisory Committee meeting will be necessary during the review process, but that no potential review issues were noted by the agency. The BLA is 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.

"We are thrilled to announce the acceptance of our BLA bringing us one step closer to providing this first-in-class treatment for Duchenne-cardiomyopathy, a condition for which there are no approved therapies" Linda Marbán, PhD, chief executive officer of Capricor Therapeutics, said in a statement.¹ “If our application is successful, we expect deramiocel to be a lifelong treatment, administered quarterly, with the potential to be widely adopted across the DMD-cardiomyopathy treatment landscape. We want to extend our appreciation to the patients, their families and advocates who continue to work with Capricor and to the FDA for its commitment to accelerating treatments for DMD.”

Capricor completed the submission of its rolling BLA for Deramiocel in January 2025.² At the time, the company noted that the submission made it eligible to receive a $10 million milestone payment from its partner Nippon Shinyaku under a United States Commercialization and Distribution Agreement.

According to 24-month data from HOPE-2-OLE that Capricor reported in June 2023, 6 of 9 patients who reached 24 months of follow-up showed improvement in left ventricular ejection fraction (LVEF) as measured by cardiac magnetic resonance imaging, in comparison to when they were assessed at the conclusion of HOPE-2.³ In addition to the LVEF results, the company also reported that at 24 months of follow-up patients demonstrated statistically significant benefit (P = .021) on the Performance of the Upper Limb (PUL version 2.0) scale in comparison to the rate of decline seen in the placebo group in HOPE-2 at 1 year of follow-up. With regard to safety, Capricor characterized the cell therapy as “well-tolerated” and deemed its safety profile favorable in the study.

“Deramiocel has shown in multiple clinical trials attenuation of DMD-cardiomyopathy, which is currently one of the leading causes of death in those with DMD,” HOPE-2-OLE national principal investigator Craig McDonald, MD, a professor in the Department of Physical Medicine and the Rehabilitation Chair at University of California, Davis, added to the statement.¹ “Based on the totality of the safety and efficacy data deramiocel has shown, this potential approval offers patients a first-in-class therapeutic for DMD-cardiomyopathy.”

Notably, a phase 3 clinical trial dubbed HOPE-3 is also evaluating Deramiocel in DMD. According to the clinicaltrials.gov page for HOPE-3, which was most recently updated on February 4, 2025, the study is active, with an actual enrollment of 104 patients, and no longer recruiting new participants. In September 202, Capricor reported that it had participated in a Type-B meeting with the FDA in which the company and the agency aligned on several aspects of the trial, including its primary end point: the mean change from baseline in the full PUL version 2.0, measured at 12 months posttreatment.⁴

REFERENCES
1. 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 March 4, 2025. https://www.capricor.com/investors/news-events/press-releases/detail/305/capricor-therapeutics-announces-fda-acceptance-and-priority
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 March 4, 2025.https://www.capricor.com/investors/news-events/press-releases/detail/303/capricor-therapeutics-completes-submission-of-biologics
3. Capricor Therapeutics announces positive 24-month results from ongoing HOPE-2 open label extension study of CAP-1002 in Duchenne muscular dystrophy. News release. Capricor Therapeutics. June 30, 2023. Accessed March 4, 2025. https://www.capricor.com/investors/news-events/press-releases/detail/255/capricor-therapeutics-announces-positive-24-month-results
4. Capricor Therapeutics announces positive Type-B meeting with the FDA to discuss pathway to BLA for CAP-1002 in Duchenne muscular dystrophy. News release. Capricor Therapeutics. September 29, 2023. Accessed March 4, 2025. https://www.capricor.com/investors/news-events/press-releases/detail/261/capricor-therapeutics-announces-positive-type-b-meeting