Capricor Therapeutics, Inc. (CAPR) announced that the U.S. FDA has requested the full clinical study report from the Phase 3 HOPE-3 trial of Deramiocel as part of its Biologics License Application review for Duchenne muscular dystrophy.
The FDA's request follows its review of topline HOPE-3 data submitted in late 2025. Importantly, the agency did not ask for additional clinical trials or new patient data.
Capricor expects to submit the HOPE-3 CSR and supporting materials in February 2026, which it believes will address the items outlined in the Complete Response Letter (CRL) received in July 2025 and support continued FDA review, including assignment for a new PDUFA target action date.
The BLA was originally accepted with Priority Review in March 2025, and the FDA had assigned an initial PDUFA date of August 31, 2025, before issuing the CRL.
Duchenne muscular dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting skeletal, respiratory, and cardiac muscles. It impacts approximately 15,000 individuals in the United States, primarily boys, and currently has limited treatment options.
Deramiocel (CAP-1002) is an investigational allogeneic cell therapy derived from cardiosphere-derived cells (CDCs). These cells secrete exosomes that modulate immune responses and reduce fibrosis, aiming to preserve cardiac and skeletal muscle function. The therapy has been studied in more than 250 peer-reviewed publications and administered to over 250 patients across multiple trials.
The HOPE-3 Phase 3 trial enrolled 106 boys with DMD in a randomized, double-blind, placebo-controlled design. Participants received either Deramiocel or placebo every three months for four doses over 12 months. Results demonstrated statistically significant and clinically meaningful improvements in both skeletal muscle and cardiac function, key drivers of disease progression and long-term outcomes.
Deramiocel has received Orphan Drug Designation from both the FDA and the European Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.
Capricor has an exclusive commercialization and distribution agreement for Deramiocel in the U.S. and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval.
CAPR has traded between $4.30 and $40.37 over the past year. The stock is trading at $24.74, up 1.58 %.
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