Capricor reports a 76% slower PUL 2.0 decline after switching to deramiocel in the HOPE-3 extension study in Duchenne muscular dystrophy.
Written By: Aasritha Thippavajjala, PharmD
Reviewed By: Pharmacally Editorial Team
Capricor Therapeutics reported new results on October 5, 2026, from the open-label extension (OLE) of HOPE-3, its Phase 3 trial of deramiocel (CAP-1002), an investigational allogeneic cardiosphere-derived cell therapy for Duchenne muscular dystrophy (DMD). The data, presented in a late-breaking poster at the 31st Annual Congress of the World Muscle Society in Hiroshima, Japan, showed that patients who switched from placebo to deramiocel had a 76% reduction in the rate of decline on the PUL 2.0 total score compared with their own prior year on placebo.
HOPE-3 Design and Open-Label Extension
HOPE-3 is a randomized, double-blind, placebo-controlled Phase 3 trial that enrolled 106 patients with DMD. Participants received deramiocel or placebo intravenously every three months for 12 months. One-year results from the randomized period were published in The Lancet in July 2026.
Among patients completing the randomized portion, 98 entered the OLE, including 49 from each original treatment group. The extension provides data through 24 months, with analyses of upper limb function based on the Performance of the Upper Limb 2.0 (PUL 2.0) total score.
Year 1 analyses included 52 patients in the original placebo group and 54 in the deramiocel group. Year 2 analyses included 49 patients in each group, while observed Month 24 data were available from 42 and 40 patients, respectively.
Slower PUL 2.0 Decline After Treatment Switch
In the original placebo group, the least-squares mean change in PUL 2.0 total score was −2.05 during Year 1 on placebo and −0.49 during Year 2 after switching to deramiocel. The difference was 1.56 points, with a 95% confidence interval of 0.47 to 2.64.
Capricor calculated this difference as a 76% reduction in the rate of decline, relative to the group’s decline during its preceding year on placebo. The comparison therefore reflects a within-group, year-over-year change after treatment initiation rather than a randomized placebo-controlled comparison during Year 2.
In the original deramiocel group, which received deramiocel during both years, the least-squares mean change was −0.95 in Year 1 and −0.89 in Year 2. The difference was 0.05 points, with a 95% confidence interval of −1.03 to 1.14, which includes zero.
Craig McDonald, MD, principal investigator of HOPE-3 at UC Davis Health, said the similar rate of decline across the two treatment years was consistent with the possibility that the slower decline followed treatment initiation rather than simply the passage of time. The company also said the crossover findings were consistent with observations from the randomized, placebo-controlled period.
Because these were open-label extension analyses, no alpha was allocated to the comparisons.
Exploratory Natural-History Analysis
Capricor also compared observed Month 24 changes with predictions from a natural-history model developed using baseline PUL 2.0 score, age and ambulatory status. These analyses were descriptive and were not matched or statistically tested. The OLE was not powered for Month 24 comparisons, and the observed values were unadjusted mean changes.
After 12 months of deramiocel treatment, the original placebo group had an observed Month 24 change of −3.76 points compared with a predicted decline of −5.35 points. The original deramiocel group, after 24 months of treatment, had an observed change of −3.42 points compared with a predicted −5.88 points.
During the untreated first year, the placebo group had an observed decline of −2.7 points compared with a predicted −2.9 points, indicating that its initial trajectory was broadly consistent with the natural-history model.
FDA Review and Next Steps
Capricor said the FDA has received the HOPE-3 OLE data as part of a major amendment to the deramiocel Biologics License Application, together with sensitivity and robustness analyses supporting the randomized trial results. The FDA’s PDUFA target action date is November 22, 2026.
Capricor also scheduled a webinar with Parent Project Muscular Dystrophy for October 7 to discuss the HOPE-3 findings.
Deramiocel remains investigational and has not been approved for commercial use in any indication. The findings reported from the OLE are company-reported and should be interpreted in the context of the open-label design and exploratory nature of the extension analyses.
Reference
Capricor Therapeutics Presents Positive HOPE-3 Open-Label Extension Data on Upper Limb Function in Duchenne Muscular Dystrophy at 2026 World Muscle Society Congress, Capricor Therapeutics, 05 October 2026
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy (HOPE-3), ClinicalTrials.gov ID NCT05126758
About the Writer
Aasritha Thippavajjala (Linkedin) is a pharm D student and aspiring medical writer with medical writing, clinical training experience and a strong interest in clinical research and patient safety She has hands-on exposure to clinical pharmacy activities including medication profile assessment, clinical case review, identification of potential drug-related problems, ADR awareness, patient counselling, and clinical documentation. She has also gained experience in medical literature review, evidence synthesis, and scientific communication through her published review article on digital twin-based patient simulation and her poster presentation at the 74th Indian Pharmaceutical Congress. Certified in ICH Good Clinical Practice (E6(R3)) and Scientific Writing in Health Research, she is passionate about applying her clinical knowledge and research skills to medical writing ,clinical research and contributing to accurate, evidence-based healthcare.