Capricor Therapeutics has presented the 18-month results from its HOPE-2 open-label extension (OLE) study with the lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD) at the Muscular Dystrophy Association (MDA) conference in March 2023. Key results from the study include:

• Statistically significant differences in the Performance of the Upper Limb (PUL version 2.0) scale in the CAP-1002 original treatment group when compared to the original placebo group from HOPE-2 (p=0.02).
• Both groups experienced reduced disease progression once all patients began treatment in the OLE study.
• CAP-1002 treatment during the OLE portion of the study continues to yield a consistent 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 18-month results suggest that patients accumulate benefit over time with the preservation of skeletal muscle function, which underscores the potential long-term benefit of CAP-1002.

“There are thousands of boys and young men across the world who continue to face the devastating and immobilizing effects of DMD, and for whom few therapeutic options currently exist. Our 18-month results suggest that CAP-1002 has the potential to slow the decline of DMD progression and speaks to the long-term benefit for patients. We were pleased to present these results alongside other industry leaders who share Capricor’s mission of developing novel treatments for patients suffering from rare neuromuscular conditions.”

Dr. Linda Marbán, CEO of Capricor

Capricor is currently conducting the HOPE-3, Phase 3 trial, designed as a randomized, double-blind, placebo-controlled study with enrollment criteria similar to HOPE-2. The Phase 3 study is currently enrolling subjects in the United States.

See information about the clinical trial in our Clinical Trial Finder Tool: