StockWatch: Capricor Plunges as FDA Panel, Staff Question Effectiveness of Lead Candidate Deramiocel

Capricor Therapeutics (CAPR) shares experienced a significant decline following a negative recommendation from the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee regarding its lead candidate Deramiocel. The advisory committee voted 9-3 against approving Deramiocel, citing a lack of "substantial evidence of effectiveness" in the Phase III HOPE-3 trial for treating cardiomyopathy in Duchenne muscular dystrophy (DMD).

The FDA set an August 22 decision date for the resubmitted biologics license application (BLA) under the Prescription Drug User Fee Act (PDUFA), typically adhering to the advice of its advisory committees. Capricor's Deramiocel is an allogeneic cardiosphere-derived cell (CDC) therapy, designed to secrete exosomes targeting macrophages to alter their expression profile for a healing effect.

The FDA's negative evaluation included concerns about whether Deramiocel achieved the primary and secondary endpoints, the high hypersensitivity rate among patients, and the absence of an updated statistical analysis plan. Capricor countered the FDA's decision, asserting that their results were based on the final analysis plan (SAP version 3.0) finalized prior to unblinding, and emphasizing the potential benefits for boys and young men with Duchenne muscular dystrophy who face significant unmet medical needs.

Despite the FDA's stance, Capricor presented updated data from HOPE-3 in The Lancet, reporting improvements in cardiac and skeletal muscle function and a possible slowing of muscle weakening and heart damage in advanced DMD patients. However, the study's key secondary endpoint of left ventricular ejection fraction (LVEF) did not reach statistical significance at 12 months.