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Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA Following FDA Review of HOPE-3 Topline Data
FDA has requested the HOPE-3 clinical study report (CSR) as part of the BLA review process Com...
About this update from Caprock Mining Corp
[{"type":"text","content":"Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA Following FDA Review of HOPE-3 Topline Data\nFDA has requested the HOPE-3 clinical study report (CSR) as part of the BLA review processCompany expects to submit updates to the BLA in February 2026 to support continued FDA review SAN DIEGO, Jan. 20, 2026 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today provided a regulatory update regarding its Biologics License Application (BLA) for Deramiocel, the Company’s investigational first-in-class cell therapy for the treatment of Duchenne muscular dystrophy (DMD). As previously disclosed, the Company provided topline results from its Phase 3 HOPE-3 clinical study to the U.S. Food and Drug Administration (FDA) in late 2025. Following its review of these data, the FDA has formally requested the full HOPE-3 clinical study report (CSR) and supporting data to address the Complete Response Letter (CRL). The FDA did not request any additional clinical studies or new patient data as part of this request. Preparation of the HOPE-3 CSR is well underway, and the Company plans to submit the requested materials to the FDA in February 2026. The Company expects that this submission will address the items outlined in the CRL and support continued review of the BLA, including the assignment of a new Prescription Drug User Fee Act (PDUFA) target action date. “We are actively engaging with the FDA in order to facilitate an efficient review of the HOPE-3 data that directly address the issues raised in the CRL we received in July 2025. We were pleased that the FDA requested the HOPE-3 clinical study report, as this is an expected and appropriate next step following their initial review of the topline data,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “The HOPE-3 results demonstrated statistically significant and clinically meaningful improvements in both skeletal muscle and cardiac function—key drivers of disease progression and long-term outcomes in Duchenne. These findings build on more than a decade of consistent clinical evidence and reinforce our confidence in Deramiocel’s potential. Our near-term priority is to address the FDA’s ...