Capricor tumbles after FDA panel rejects Duchenne heart therapy

• The FDA previously rejected the therapy in 2023 and requested additional data after determining it did not meet efficacy requirements
• Panel members recommended a larger, longer trial with different endpoints to provide clearer evidence of whether the therapy preserves heart function
• Analysts expect the FDA will likely issue another Complete Response Letter (rejection) rather than approve deramiocel by the August 22 decision deadline

Summary: Capricor Tumbles After FDA Panel Rejects Duchenne Heart Therapy

Market Impact:

Capricor Therapeutics (CAPR.O) plunged nearly 60% in premarket trading Thursday following an FDA advisory panel's rejection of its Duchenne muscular dystrophy heart therapy.

Key Decision:

The FDA panel voted 9-3 against deramiocel's effectiveness data on Wednesday, aligning with FDA staff concerns about insufficient evidence of therapeutic benefit. Panel members cited unconvincing cardiac-benefit evidence, missing data, and uncertainty over whether the study's heart-function measurements reflected meaningful patient outcomes.

Background:

This marks the second setback for the therapy. In 2023, the FDA declined approval and requested additional data, stating the treatment failed to meet efficacy requirements. Analysts noted concerns about changes made to key analyses after the late-stage study's completion.

Analyst Perspectives:

• Piper Sandler analyst Edward Tenthoff expects the FDA will likely issue another Complete Response Letter and reject approval
• Cantor Fitzgerald analyst Kristen Kluska observed that FDA staff appeared "extremely negative during the entire conversation" and entered the meeting predisposed to reject the application
• Analysts suggest a larger, longer trial with different endpoints may be necessary to demonstrate clear evidence of functional preservation

Timeline:

The FDA is expected to make its final decision on deramiocel by August 22. While the agency isn't required to follow advisory panel recommendations, it typically considers their input when making approval decisions.

Indication:

Deramiocel is a cell therapy designed to treat cardiomyopathy associated with Duchenne muscular dystrophy, a rare genetic disorder.