Capricor Therapeutics (CAPR) stock plunged more than 67% on Monday after U.S. Food and Drug Administration reviewers released briefing documents. The documents expressed concerns over whether its lead therapy Deramiocel has shown sufficient effectiveness to treat cardiomyopathy in patients with Duchenne muscular dystrophy. FDA reviewers stated the study failed to meet its pre-specified primary and secondary efficacy endpoints, finding no statistically significant difference between Deramiocel and placebo after 12 months. Duchenne muscular dystrophy is a rare inherited disorder that mainly affects boys, causing progressive muscle weakness, and there are no FDA-approved treatments specifically for the heart disease that often develops as the condition worsens. CAPR stock recorded its biggest ever single-day slide and is currently at its lowest levels since December 2025.
FDA Reviewers Report Deramiocel Study Failed to Meet Efficacy Endpoints
The FDA on Monday released briefing documents ahead of a July 29 meeting of its Cellular, Tissue, and Gene Therapies Advisory Committee. In the documents, FDA reviewers said the study failed to meet its pre-specified primary and secondary efficacy endpoints, finding no statistically significant difference between Deramiocel and placebo after 12 months. The advisory committee will review Capricor's Biologics License Application for the drug before the FDA makes its final decision by the Prescription Drug User Fee Act target date of Aug. 22, 2026. A PDUFA date is the deadline by which the FDA decides whether to approve a drug for marketing in the United States.
Reviewers Question Statistical Analysis Plan Changes
The documents show that reviewers raised concerns over changes made to the statistical analysis plan for Capricor's Phase 3 Hope-3 trial. In December 2025, Capricor stated that its Phase 3 Hope-3 trial met its primary endpoint of improving upper-limb function and its key secondary endpoint of preserving heart function, with both showing statistically significant results. However, the reviewers said multiple changes were made after the randomized portion of the study, including revisions to the primary and secondary endpoints, analytical methods, and data imputation strategy. They also noted that the final statistical analysis plan was not submitted for review before the company's Biologics License Application and that the study's pre-specified procedures for making such changes were not followed. The documents also highlighted a higher rate of hypersensitivity reactions in patients treated with Deramiocel, which it said may have increased the risk of functional unblinding during the trial.
Advisory Committee Review Scheduled for July 29
The advisory committee has been recommended to discuss whether data from the Hope-3 and Hope-2 studies show that Deramiocel can slow the decline in heart function in patients with Duchenne muscular dystrophy. The panel will also consider whether the combined results provide enough evidence that the therapy improves upper-limb function and is an effective treatment for the disease. The stock has shed more than 77% so far this year.
FAQ
What did FDA reviewers say about Capricor's Deramiocel study on Monday?
FDA reviewers said the study failed to meet its pre-specified primary and secondary efficacy endpoints, finding no statistically significant difference between Deramiocel and placebo after 12 months.
When will the advisory committee review Capricor's Biologics License Application?
An advisory committee will review Capricor's Biologics License Application on July 29, ahead of the FDA's final decision by the PDUFA target date of Aug. 22, 2026.
Why did FDA reviewers raise concerns over Capricor's trial?
Reviewers raised concerns over changes made to the statistical analysis plan after the randomized portion of the study, including revisions to endpoints and analytical methods, and noted that the final plan was not submitted for review before the company's application.
