Capricor Therapeutics shares tumble after FDA reviewers raise concerns over Duchenne therapy

Health Written by: Emily Jarvie 10:50 Mon 27 Jul 2026 --> Disclaimer No investment advice About this content Editorial Standards & Policies Share article About this content × About Emily Jarvie Emily began her career as a political journalist for Australian Community Media in Hobart, Tasmania. After she relocated to Toronto, Canada, she reported on business, legal, and scientific developments in the emerging psychedelics sector before joining Proactive in 2022. She brings a strong journalism background with her work featured in newspapers, magazines, and digital publications across Australia, Europe, and North America, including The Examiner, The Advocate, The Canberra Times, and... Read more About the publisher Proactive financial news and online broadcast teams provide fast, accessible, informative and actionable business and finance news content to a global investment audience. All our content is produced independently by our experienced and qualified teams of news journalists. Proactive news team spans the world’s key finance and investing hubs with bureaus and studios in London, New York, Toronto, Vancouver, Sydney and Perth. We are experts in medium and small-cap markets, we also keep our community up to date with blue-chip companies, commodities and broader investment stories. This is content that excites and engages motivated private investors. The team delivers news and unique insights across the market including but not confined to: biotech and pharma, mining and natural resources, battery metals, oil and gas, crypto and emerging digital and EV technologies. Use of technology Proactive has always been a forward looking and enthusiastic technology adopter. Our human content creators are equipped with many decades of valuable expertise and experience. The team also has access to and use technologies to assist and enhance workflows. Proactive will on occasion use automation and software tools, including generative AI. Nevertheless, all content published by Proactive is edited and authored by humans, in line with best practice in regard to content production and search engine optimisation. Capricor Therapeutics shares tumble after FDA reviewers raise concerns over Duchenne therapy Published: 10:50 27 Jul 2026 EDT Capricor Therapeutics (NASDAQ:CAPR) shares fell about 67% on Monday after US Food and Drug Administration (FDA) staff reviewers raised concerns about the evidence supporting the effectiveness of the company's experimental cell therapy, deramiocel, for Duchenne muscular dystrophy (DMD)-associated cardiomyopathy. The FDA released briefing documents ahead of a July 29 meeting of its Cellular, Tissue, and Gene Therapies Advisory Committee, which will review the therapy's efficacy and overall risk-benefit profile before the agency's August 22 Prescription Drug User Fee Act (PDUFA) target action date. FDA reviewers questioned changes Capricor made to its analysis after completion of its late-stage trial, noting the company switched its primary assessment of arm function from raw scores on a 42-point scale to percentage change. The agency wrote that it did not consider the conversion "scientifically justified," saying it added complexity and reduced accuracy. The reviewers also highlighted changes to the analysis of heart function, which shifted from measuring changes in left ventricular ejection fraction to ranking patients' results. In addition, FDA staff questioned whether patients enrolled in the study had DMD-related cardiomyopathy, noting they had normal heart-pumping function on average at baseline. The reviewers also said evidence was limited that enough of the intravenously administered therapy reached the heart to produce a meaningful benefit. Deramiocel is being reviewed under a biologics license application supported by data from the Phase 2 HOPE-2 trial, its long-term extension study, and the Phase 3 HOPE-3 trial. In announcing the advisory committee meeting last month, Capricor said the application was supported by statistically significant results on the Phase 3 trial's primary endpoint, key secondary cardiac endpoint and other controlled secondary endpoints. The company said it remained confident in the overall evidence supporting deramiocel and its potential as a first-in-class treatment for Duchenne muscular dystrophy. There are currently no FDA-approved therapies specifically for DMD-associated cardiomyopathy. Existing Duchenne treatments, including Sarepta Therapeutics' gene therapy Elevidys and exon-skipping drugs, target the underlying genetic cause of the disease rather than the heart complications that develop as it progresses. Continue reading