Capricor Gains 3-Month FDA Extension For Deramiocel, After Filing Upper-Limb Data Amendment

Capricor Therapeutics said the FDA granted a three-month extension for its review of deramiocel, moving the action date from Aug. 22 to Nov. 22 after the company submitted additional data and asked the agency to consider a narrower proposed indication.

The amendment to the biologics license application includes 24-month open-label extension data from the Phase 3 HOPE-3 study and requests review of existing and new data in support of a refined proposed indication focused on upper limb function. That matters because the late-stage review had become entangled with debate over cardiac data, even though upper limb function was the study’s primary endpoint.

The road here

Late last month, advisers voted 9-3 against recommending approval for deramiocel. During that meeting, FDA advisers and reviewers spent substantial time on left ventricular ejection fraction, or LVEF, a cardiac-related secondary endpoint tied to Duchenne muscular dystrophy cardiomyopathy.

Capricor CEO Linda Marbán had previously told BioSpace that the company requested in a pre-BLA meeting last August to switch the HOPE-3 primary efficacy endpoint to LVEF from change from baseline in upper limb function. Capricor submitted a protocol amendment to designate LVEF as the primary efficacy endpoint, with upper limb performance as a pre-specified secondary endpoint, but no such change was ultimately made.

That left Capricor arguing that the advisory committee’s emphasis was misplaced. Marbán said the focus on LVEF was flawed because it remained a secondary endpoint and was calculated across the full Duchenne muscular dystrophy population rather than in patients with established cardiomyopathy, the population addressed by the proposed indication.

What changed

The latest filing appears designed to simplify that argument. Marbán said Monday that HOPE-3 met its primary endpoint, demonstrating a statistically significant benefit in upper limb function, and that the extra year of follow-up strengthens the case for a refined label built around that measure.

The FDA’s designation of the package as a major amendment is procedurally important. It delays the decision, but it also signals the agency is reviewing the new material rather than closing the file on the original timetable. For Capricor, that is a better outcome than forcing a decision on the original record immediately after an unfavorable advisory committee meeting.

Capricor’s stock rose as much as 16% Monday morning and was $7.05 per share at the time of publication. The company is also facing pressure from shareholder Kaos Capital, which recently objected to Capricor’s plan to halt all other pipeline work until it gets further regulatory clarity on deramiocel.