Shares in Capricor Therapeutics dropped sharply after the U.S. Food and Drug Administration raised concerns about its experimental therapy for Duchenne muscular dystrophy. The company’s drug, called deramiocel, is being evaluated for treating heart complications that arise in patients with the rare muscle condition. Reviewers at the FDA questioned how the company analyzed data from its Phase 3 trial, particularly changes made to how results were measured.
The agency noted that a shift from raw scores to percentage change in arm function was not scientifically justified. They also questioned how heart function was assessed, pointing out that the method used was less accurate than originally planned. The FDA staff expressed doubt about whether patients in the trial actually had the heart condition the therapy was meant to treat.
Many of the participants had normal heart-pumping ability at the start of the study, which raised concerns about how well the treatment could be tested. Additional concerns were raised about whether enough of the therapy reached the heart to make a difference. Capricor had based its application on results from multiple trials, including HOPE-2 and HOPE-3.
The company had previously claimed that its Phase 3 trial showed significant results for the main outcome and related measures. Despite this, the FDA reviewers said that no meaningful difference was seen between patients who received deramiocel and those who got a placebo. The company responded by saying the FDA’s review was based on an outdated internal draft of its analysis.
The company’s CEO said the FDA had looked at a version of the data that was not yet finalized. The advisory committee will meet on July 29 to discuss whether the therapy should be approved for use in patients. The final decision is expected by August 22, which is the FDA’s target date for a ruling on the application.
Duchenne muscular dystrophy is a rare condition that mostly affects boys and causes muscle weakness over time. There are currently no treatments approved specifically for the heart problems that often come with this disease. Other treatments for Duchenne focus on the genetic cause of the illness rather than heart-related symptoms.
The company has said it still believes its evidence supports the therapy and that it could be a first-in-class treatment. The stock price of Capricor Therapeutics fell to its lowest point since December 2025 after the FDA’s comments. The situation has brought attention to how clinical data is interpreted and reviewed for new therapies.
Experts are watching closely as the advisory committee prepares to weigh in on whether the drug should be approved. The outcome of the meeting could have a major impact on future treatment options for patients with Duchenne muscular dystrophy.
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