FDA scientists flagged concerns about the efficacy of Capricor’s deramiocel ahead of an advisory committee meeting, according to Monday’s update. The agency said the stem-cell treatment for Duchenne muscular dystrophy did not meet objectives from a Phase 3 trial, contradicting Capricor’s earlier claims that the therapy met primary and secondary endpoints. Capricor said in December that deramiocel reached both primary and secondary endpoints in a randomized study, a finding the company had treated as a pivotal validation step for the therapy in a disease where development has been difficult. The FDA’s position sets up a higher-stakes forum for evaluating how clinical endpoints were achieved and interpreted. The stock reaction and the timing underscore the sensitivity of regulatory decisions in Duchenne, where differences in trial design, endpoint hierarchy, and event adjudication can materially change review outcomes. For the field, the episode is a reminder that even “positive” late-stage claims can come under intense scientific scrutiny when regulators challenge trial objective achievement.