Capricor Therapeutics reports Phase 3 HOPE-3 data showing Deramiocel improves upper limb function and slows progression in Duchenne muscular dystrophy, with FDA review underway.
Written By: Karthik Teja Macharla, PharmD
Reviewed By: Pharmacally Editorial Team
Capricor Therapeutics reported Phase 3 HOPE-3 trial results for Deramiocel in Duchenne muscular dystrophy at the American Academy of Neurology Annual Meeting 2026, with findings presented by Aravindhan Veerapandiyan, Associate Professor and Director of the Comprehensive Neuromuscular Program at Arkansas Children’s Hospital.
The study demonstrated a statistically and clinically meaningful benefit in upper limb function as measured by the Performance of the Upper Limb (PUL v2.0) scale, a primary endpoint in Duchenne trials where prior therapies have not shown significant improvement.
These results were supported by the Duchenne Video Assessment, a home-based measure that indicated slower disease progression in tasks such as self-feeding, reflecting preserved patient independence.
Dr. Veerapandiyan indicated that the consistency across clinical and real-world measures suggests a meaningful treatment effect in slowing functional decline.
Linda Marbán, Chief Executive Officer of Capricor, stated that the data demonstrate preserved function, delayed disease progression, and tangible impact on daily activities, supporting the potential of Deramiocel as a disease-modifying therapy.
The company’s biologics license application is currently under review by the U.S. Food and Drug Administration, with a target action date of August 22, 2026.
Deramiocel (CAP-1002) is an allogeneic cell therapy composed of cardiosphere-derived cells that exert immunomodulatory and anti-fibrotic effects through the release of exosomes, which promote a reparative, anti-inflammatory macrophage response.
The therapy has been evaluated across more than 250 studies and administered to over 250 patients. Duchenne muscular dystrophy is a severe X-linked disorder caused by the absence of dystrophin, leading to progressive muscle degeneration and eventual cardiomyopathy and heart failure, the primary causes of mortality.
Deramiocel has received multiple regulatory designations, including Orphan Drug status in the U.S. and Europe, as well as Regenerative Medicine Advanced Therapy, Advanced Therapy Medicinal Product, and Rare Pediatric Disease designations.
The findings add to prior evidence suggesting Deramiocel’s potential to preserve both skeletal and cardiac muscle function in Duchenne muscular dystrophy. The data also support ongoing regulatory review, with the company’s biologics license application currently under evaluation by the U.S. Food and Drug Administration.
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About the Writer
Karthik Teja Macharla, PharmD is a Pharm.D. graduate with a strong interest in clinical research, pharmacovigilance, and medical writing. In his words, he is passionate about converting complex medical information into clear, evidence-based scientific communication, committed to contributing to patient safety and advancing healthcare through accurate and impactful medical content.