Capricor Therapeutics announced the publication of final one-year results from its Phase 3 HOPE-3 trial for Deramiocel in Duchenne muscular dystrophy in The Lancet, confirming the primary endpoint with a 54% slowing of upper limb function decline (p=0.03). However, an updated statistical model for the key secondary cardiac endpoint, left ventricular ejection fraction, yielded a p-value of 0.09 compared to the previously reported 0.04. Concurrently, the FDA issued a Form 483 following a Bioresearch Monitoring inspection citing documentation and vendor oversight issues, though management asserts data integrity remains intact. The company maintains its PDUFA target action date for Deramiocel of August 22, 2026. Capricor Therapeutics is a biotechnology company developing cell and exosome-based therapeutics for rare diseases.
