Capricor Therapeutics, Inc. will present the positive 24-month results from its HOPE-2 open-label extension (OLE) study with lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD) at MDA Clinical and Scientific Conference which is taking place in Orlando, Florida from March 3-6, 2024. Key results from the study, include: CAP-1002 is an investigational cell therapy which aims to slow disease progression through immunomodulatory, anti-inflammatory and anti-fibrotic actions with the goal of potentially improving the rate of decline in skeletal and cardiac muscle function in patients with DMD. Skeletal muscle function as measured by the Performance of the Upper Limb (PUL v2.0) showed a mean PUL v2.0 decline after 24-months of treatment with CAP-1002 was 2.8 points versus a 7.7 point decline on average observed over 24-months in the placebo patient group.

Delta change=4.9 points; p=0.021. The average rate of decline in CAP-1002 treated patients showed an attenuation of disease progression by approximately 64%. CAP-1002 revealed clinically meaningful improvements in ameliorating cardiac function.

Cardiac function as measured by left ventricular ejection fraction (LVEF%) by MRI at the 24-month timepoint, improved in 67% of patients, compared to a steady decline in a comparable natural history population. CAP-1002 treatment during the OLE portion of the study continues to yield a consistent favorable safety profile and has been well-tolerated throughout the study. The HOPE-2 OLE study previously met the primary endpoint at the one-year time-point and these 24-month results suggest that patients accumulate benefit over time with preservation of skeletal muscle function, which underscore the potential long-term benefit of CAP-1002.

HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical study of Capricor?s lead investigational therapy, CAP-1002, in boys and young men who have DMD. Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months. Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point.

After the completion of the HOPE-2 study, all patients stopped treatment for approximately 392 days (mean, range [239, 567]), which is referred to as the gap phase. Then all eligible patients who wished to remain on treatment re-entered the OLE study where they received CAP-1002 (150 million cells per infusion) every three months over the course of 24-months. These 24-month results were presented previously at the 2023 Parent Project Muscular Dystrophy (PPMD) Annual Conference.