Capricor Therapeutics, 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). – Read More: What is Deramiocel? What Does CAP-1002 Do? –
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. – Learn More: Capricor Therapeutics Announces Completion of Mid-Cycle Review Meeting with FDA on Deramiocel –
“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.”
“These four-year data reinforce the strength and durability of Deramiocel’s clinical benefit and favorable safety profile across both cardiac and skeletal muscle function,” said Linda Marbán, Ph.D., CEO of Capricor Therapeutics. “With our BLA under priority review and several key regulatory steps now completed, we are executing with focus and urgency as we move toward potential approval. Our continued dialogue with the FDA remains on track with no evidence of any delays. We thank the patients, families, and clinicians who have been instrumental in advancing this program.”
