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Capricor Therapeutics Announces Long-Term Benefit of Deramiocel (CAP-1002) in Both Skeletal Muscle and Cardiac Function in the HOPE-2 OLE Study in Duchenne Muscular Dystrophy
-Results in Performance of the Upper Limb (PUL v2.0) Continue to Show Slowing of Disease Progression in Later-Stage DMD Patients- -Improvements Seen in
About this update from Capricor Therapeutics, Inc.
[{"type":"text","content":"-Results in Performance of the Upper Limb (PUL v2.0) Continue to Show Slowing of Disease Progression in Later-Stage DMD Patients- -Improvements Seen in Multiple Cardiac Endpoints Demonstrating Stabilization of Cardiac Function Over 3 Years of Treatment- -Pre-BLA Meeting with FDA Scheduled for Q3 2024 to Discuss Options to Expedite BLA Filing- -Results to be Presented at PPMD Annual Conference on June 29 in Session titled “Approaches to Alter Progression”- SAN DIEGO, June 28, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced additional positive 3-year safety and efficacy results from its ongoing HOPE-2 open label extension (OLE) study with its lead asset, deramiocel (CAP-1002), for the treatment of Duchenne muscular dystrophy (DMD). Data from the HOPE-2 OLE study demonstrated improvements in multiple cardiac measures, including left ventricular ejection fraction (LVEF), as well as indexed volumes (left ventricular end systolic volume (LVESV) and left ventricular end diastolic volume (LVEDV)). 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 (> 45%) 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 (+3.7 points, p 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 (PPMD) 30th Annual Conference being held in Orlando, Florida on June 29, 2024. 3-Year HOPE-2 OLE Study Results Primary endpointSkeletal muscle (upper-limb function)3-year timepoint C...