Capricor Therapeutics is amending its Biologics License Application to pursue a narrower upper limb skeletal muscle indication for deramiocel after an FDA advisory committee voted 3 to 9 against the cardiomyopathy indication in Duchenne muscular dystrophy. The HOPE-3 trial's primary endpoint showed a statistically significant slowing of upper limb disease progression, with a 4.5% mean difference favoring the drug and a p-value of 0.029. However, the company's cash and marketable securities fell to $237.9 million as of June 30, 2026, from $318.1 million at the end of 2025, and second-quarter net loss widened to $40.7 million, or $0.70 per share, from $25.9 million, or $0.57 per share, a year earlier. The left ventricular ejection fraction result across all patients was revised under the prespecified statistical model from a 2.4 percentage point difference at p=0.04 down to 1.8 percentage points at p=0.09, weakening a key secondary measure. Short interest stands at 33.72% of float, and the FDA has indicated it will review the coming BLA amendment and extend the PDUFA date accordingly.
Capricor Therapeutics IncFDA panel rejection and narrower indication amendment weaken regulatory prospects.
