CAPR Stock Is Crashing Today – FDA Briefing Casts Doubt On Duchenne Cell Therapy Before Key Vote

• 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 documents show that reviewers raised concerns over changes made to the statistical analysis plan for Capricor’s Phase 3 Hope-3 trial.
• An advisory committee will review Capricor’s Biologics License Application on July 29.

Shares of Capricor Therapeutics (CAPR) tanked more than 67% on Monday after U.S. Food and Drug Administration (FDA) reviewers expressed concerns in briefing documents over whether its lead therapy Deramiocel has shown sufficient effectiveness to treat cardiomyopathy in patients with Duchenne muscular dystrophy (DMD).

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 also recorded its biggest ever single-day slide. It is currently at its lowest levels since December 2025.

Reviewers Say Deramiocel Study Fails to Meet Primary, Secondary 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 (BLA) for the drug before the FDA makes its final decision by the Prescription Drug User Fee Act (PDUFA) 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 Changes To Trial Analysis

The documents show that reviewers raised concerns over changes made to the statistical analysis plan (SAP) 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 SAP was not submitted for review before the company’s BLA 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.

What’s Next?

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.

Retail’s Take On CAPR

Despite the slump, retail sentiment on Stocktwits turned ‘extremely bullish’ from ‘bullish’ a day earlier, amid a 320% jump in message volumes. CAPR was also among the top trending stocks on the platform at the time of writing.

One user said there’s a “60% chance that they will have to redo the Adequate and controlled cardiac primary trial.”

However, another user was confident that Capricor will be able to answer whatever questions the committee will have with “proven data and results.”

The stock has shed more than 77% so far this year.

Also read: FBRX Stock On Track For Highest Levels In Over 4.5 Years As ArgenX Scoops Up Autoimmune Drug Maker

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