Capricor Therapeutics’ deramiocel delivered a measurable slowing of muscle decline in advanced Duchenne muscular dystrophy in the Phase 3 HOPE-3 trial, with results published in The Lancet. In the randomized, double-blind study (106 boys and young men aged 10–22), deramiocel preserved upper-arm and elbow movement compared with placebo and showed a potential signal for heart-function preservation in participants with baseline cardiac disease. At the same time, FDA reviewers sharply criticized the trial’s statistical interpretation ahead of an advisory committee meeting, according to separate coverage ahead of the FDA’s Cellular, Tissue and Gene Therapies panel. The agency challenged whether the treatment separated from placebo on prespecified endpoints and faulted changes to the statistical analysis plan after it was completed. Capricor framed deramiocel as a bloodstream-delivered allogeneic, heart-derived cardiosphere-derived cell therapy and positioned HOPE-3 as a pivotal dataset for patients who have already lost independent ambulation. The discord between company and regulators keeps the approval pathway highly contested, even with a positive topline readout.