FDA staff raise concerns over Capricor’s Duchenne cell therapy as shares plunge; Legend CEO resigns, AstraZeneca reports pipeline woes

Capricor Therapeutics saw its shares tumble by nearly two‑thirds after FDA reviewers highlighted serious concerns about the data supporting its experimental Duchenne muscular dystrophy cell therapy, deramiocel. The agency noted that the therapy missed its original Phase 3 study goals and that the new application included statistical analyses the reviewers had not approved.

FDA scientists also warned that the study may have suffered from functional unblinding, calling the changes "unwarranted" based on the study design and original assumptions. Capricor has scheduled an advisory committee meeting for July 29 to address these issues.

Separately, Legend Biotech announced the resignation of CEO Ying Huang, contributing to a further slide in its stock price. AstraZeneca disclosed additional pipeline challenges while noting that a previously rejected drug finally secured regulatory approval.