Capricor Therapeutics Announces Positive 4-Year Data from HOPE-2 Open-Label Extension Study of Deramiocel in Duchenne Muscular Dystrophy

• Four-year data show preservation of cardiac function, including LVEF
• Skeletal muscle disease progression continues to slow with extended treatment (PUL v2.0)
• Deramiocel’s long-term safety profile remains favorable
• Results presented at PPMD’s 2025 Annual Conference

SAN DIEGO, June 20, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today announced positive four-year safety and efficacy results from its ongoing HOPE-2 Open-Label Extension (OLE) study of Deramiocel, the Company’s lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD). The data will be featured in the session titled “Therapies that Slow Progression” at the Parent Project Muscular Dystrophy (PPMD) 2025 Annual Conference, taking place June 21, 2025, in Las Vegas, Nevada.

After four years of continuous treatment, Deramiocel-treated patients showed a median change of -0.5 points compared to baseline. Further, a subgroup analysis of patients with baseline LVEF >45% showed an even greater clinical benefit, supporting early intervention with Deramiocel to potentially preserve cardiac function.

Additionally, treatment continued to slow skeletal muscle disease progression, as measured by Performance of the Upper Limb (PUL v2.0), with patients experiencing a smaller average decline in the fourth year (0.6 points) compared to the first year (1.8 points). Together, these findings suggest that extended treatment with Deramiocel may help attenuate the progression of DMD over time. Deramiocel continues to maintain a favorable safety profile throughout the study.

“Cardiomyopathy remains one of the leading causes of mortality in Duchenne and addressing this aspect of the disease is critical to improving outcomes,” said Pat Furlong, Founding President and CEO of PPMD. “The long-term data from the HOPE-2 OLE study are encouraging, particularly in demonstrating cardiac stabilization over four years. Deramiocel represents an important therapeutic approach and we support continued progress through the regulatory pathway to ensure that treatments targeting both heart and muscle function are available to our community as quickly as possible.”