Capricor Therapeutics IncFDA briefing document questions efficacy and safety of deramiocel, with negative advisory committee findings.

Capricor Therapeutics shares fell about 67% in Monday morning trading after the FDA released a briefing document ahead of a July 29 advisory committee meeting that questioned the efficacy and safety of its Duchenne muscular dystrophy candidate deramiocel. The document, prepared for the Cellular, Tissue, and Gene Therapies Advisory Committee, noted that the phase 3 HOPE-3 study showed no evidence of effectiveness on skeletal or cardiac function and that pre-specified primary and secondary endpoints were not met. FDA scientists also highlighted that changes were made to the statistical analysis plan after the double-blind and open-label extension phases, including modifications to endpoint definitions and analytical methods, and that a final version of the plan was not submitted for FDA review prior to the BLA resubmission. Additionally, 42% of patients in the treatment group experienced hypersensitivity reactions compared to 15% in the placebo group. Despite the decline, Oppenheimer reiterated its outperform rating, expecting the panel to vote yes on the single voting question and viewing the drop as a buying opportunity.
Capricor Therapeutics IncFDA briefing document questions efficacy and safety of deramiocel, with negative advisory committee findings.
SAP SE