Capricor Faces FDA Hurdle for Deramiocel Approval, Raises $144.8 Million

Capricor Therapeutics (CAPR) received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application (BLA) for Deramiocel, a cell therapy for Duchenne muscular dystrophy. The FDA requires further clinical data, and Capricor intends to provide evidence from its ongoing Phase 3 HOPE-3 trial. The company's cash position is approximately $144.8 million, and analysts forecast an average target price of $34.60 with a potential upside of 203.51%.

Capricor Therapeutics (CAPR) received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application (BLA) for Deramiocel, a cell therapy aimed at treating cardiomyopathy associated with Duchenne muscular dystrophy (DMD). The FDA cited insufficient evidence of effectiveness and the need for additional clinical data, leading to the CRL. Capricor plans to resubmit the BLA with data from its ongoing Phase 3 HOPE-3 trial, with topline results expected in Q3 2025. The company's cash position is approximately $144.8 million, and analysts forecast an average target price of $34.60 with a potential upside of 203.51%.

The FDA's CRL indicated that the current application does not meet the statutory requirement for substantial evidence of effectiveness. Capricor has received several regulatory designations for Deramiocel, including Orphan Drug, Regenerative Medicine Advanced Therapy (RMAT), and Rare Pediatric Disease Designation, which could qualify it for a Priority Review Voucher once approved. Despite this setback, Capricor remains committed to addressing the FDA's concerns and pursuing approval for Deramiocel.

Capricor's BLA for Deramiocel was granted Priority Review in March 2025, supported by data from the HOPE-2 trial, its open-label extension (OLE), and natural history comparisons from FDA-funded datasets. The company has secured a Type A meeting with the FDA to discuss the next steps. The HOPE-3 trial is a randomized, double-blind, placebo-controlled clinical trial involving 104 patients, designed to provide the necessary additional evidence of effectiveness.

The delay in approval represents a significant setback for Capricor, which had previously secured a Priority Review for the application. However, the company's strong cash position and analyst ratings suggest a resilient financial outlook. Analysts have issued buy and outperform ratings on CAPR stock, with a median price target of $34.60 and a potential upside of 203.51%.

Capricor's commitment to the DMD community and ongoing efforts to provide effective treatment options underscore its dedication to addressing an unmet medical need. The company's proactive approach to addressing the FDA's concerns, including the resubmission of the BLA with HOPE-3 trial data, indicates a collaborative and determined effort to secure approval for Deramiocel.

References:
[1] https://www.gurufocus.com/news/2971512/capricor-therapeutics-provides-regulatory-update-on-deramiocel-bla-for-duchenne-muscular-dystrophy-capr-stock-news
[2] https://www.nasdaq.com/articles/capricor-therapeutics-resubmit-bla-deramiocel-following-fda-complete-response-letter-plans