Long Term Safety and Efficacy of CAP-1002 in late-stage patients with DMD: A New Treatment Approach to Target Skeletal and Cardiac Muscle Pathogenesis

Topic:

Clinical Trials

Poster Number: M155

Author(s):

Craig McDonald, MD, UC Davis Health, Suzanne Hendrix, PhD, Pentara Corporation, Michelle Eagle, PT, PhD, ATOM International, Matthew Harmelink, MD, Children's Wisconsin, Arun Varadhachary, MD, PhD, Washington University School of Medicine, Cuixia Tian, MD, Cincinnati Children’s Hospital Medical Center, University of Cincinnati College of Medicine, Susan Apkon, MD, Children's Hospital Colorado, Chet Villa, MD, Cincinnati Children’s Hospital Medical Center, Michael Taylor, MD, University of Texas, Austin, Kan Hor, MD, Nationwide Children Hospital, Ohio State University, Mitchell Wassom, MS, Pentara Corporation, Paige Desai, Capricor, Mark Awadalla, Capricor Inc., Linda Marbán, PhD, Capricor Inc.

CAP-1002 are cardiosphere-derived cells (CDCs) with immunomodulatory, anti-inflammatory, and anti-fibrotic properties. Long-term treatment of CAP-1002 in patients with DMD shows attenuation of disease progression as measured by the Performance of the Upper Limb (PUL v2.0).
After completing the 12-month, double-blind, placebo-controlled HOPE-2 study in DMD; patients entered a subsequent open-label extension study to receive CAP-1002. Patients were off treatment for an average of one year (“Gap Phase”) prior to entering HOPE-2-OLE. In the ongoing HOPE-2-OLE study, 12 patients completed 24-months of treatment of CAP-1002 (8 infusions).
The mean PUL 2.0 decline after 24 months of treatment in HOPE-2-OLE is 2.8 points when compared to the decline observed in the patients that went untreated for 24 months (placebo patients in HOPE-2 + Gap Phase). These patients observed an average decline of approximately 7.7 points in this 24-month period. (Δ=4.9 points, p=0.021). The average rate of decline in CAP-1002 treated patients showed an attenuation of disease progression by 64%.
Cardiomyopathy is prevalent in DMD and is a leading cause of death. Stabilization or improvement of cardiac function can be considered as a result of intervention. Measurement of cardiac function (left ventricular ejection fraction; EF%) by MRI in the HOPE2-OLE at the 24-month timepoint, showed benefit in EF% in 67% of patients when compared to end of HOPE-2 (~3 years).
CAP-1002 maintains a favorable safety profile in HOPE-2-OLE and continues to demonstrate cumulative, clinically meaningful benefit in preserving skeletal and cardiac muscle function by potentially modifying the underlying disease.