Capricor announces three-year safety, efficacy results from HOPE-2 OLE study

Capricor Therapeutics announced additional positive 3-year safety and efficacy results from its ongoing HOPE-2 open label extension study with its lead asset, deramiocel, for the treatment of Duchenne muscular dystrophy. Data from the HOPE-2 OLE study demonstrated improvements in multiple cardiac measures, including left ventricular ejection fraction, as well as indexed volumes. These are measures of cardiac function and are considered highly relevant in terms of predicting long-term outcomes. In addition, greater improvements in cardiac function were observed in those patients that had higher ejection fractions at the beginning of the HOPE-2 randomized trial. Published data supports the need for early intervention in order to maintain function and potentially slow the progression of the cardiomyopathy, one of the leading causes of death in patients with DMD. Currently, there is no approved treatment specifically for DMD cardiomyopathy, which underscores the need for additional therapies to treat DMD. Additionally, as previously reported, patients showed a statistically significant benefit in the PUL v2.0 total score when compared to an external comparator dataset of similar DMD patients. The HOPE-2 OLE study continues to show a favorable safety profile for long-term treatment of deramiocel. These data will be shared at this year’s Parent Project Muscular Dystrophy 30th Annual Conference being held in Orlando, Florida on June 29, 2024. “The results of the open label study are tremendously important for DMD patients, as they showed sustained skeletal and cardiac benefits after 3 years of continuous treatment with deramiocel, which underscores the potential long-term benefits this therapy can offer patients with DMD,” said Linda Marban, Ph.D., Capricor’s chief executive officer. “Based on our HOPE-2 OLE data and prior clinical results, we plan to discuss with the U.S. Food and Drug Administration (FDA) options to expedite our Biologics License Application filing. We continue to work closely with FDA with the goal of bringing deramiocel to patients as quickly as possible and look forward to sharing further updates as they become available. We thank the patients, their families and the broader Duchenne community for continuing to work with us throughout the development of this promising therapy.”