LATE BREAKING NEWS E-POSTER PRESENTATION: LBP 5 HOPE-2 one-year results show clinically relevant improvements in upper limb &cardiac function in patients with later stage Duchenne Muscular Dystrophy

Abstract

HOPE-2 (Halt cardiOmyopathy ProgrEssion in Duchenne 2, NCT03406780) is a randomized, double blind, placebo controlled trial designed to evaluate whether CAP-1002, a cell therapy, improves skeletal muscle function, measured by the performance of the upper limb (PUL), and cardiac function measured by MRI in patients with later stage Duchenne Muscular Dystrophy (DMD). Efficacy of the active treatment, CAP-1002, was evaluated in 20 subjects against placebo treatment through functional assessment of upper limb performance, cardiac measurements, pulmonary tests, quality of life indicators, and biomarkers of inflammation and muscle damage. In the ITT Population, both the full PUL 1.2 and full PUL 2.0 showed significant and meaningful differences between CAP-1002 and Placebo groups in favor of the treatment (PUL 1.2: D=4.1; p=0.03; PUL 2.0: D=2.4; p=0.05). CAP-1002 at 12 months resulted in statistically significant differences versus Placebo by cardiac MRI in mean left ventricular (LV) ejection fraction (mean change from baseline: -0.33% versus -1.89%; D=1.56%; p=0.004), greater reductions in indexed LV end-diastolic volume (D=7.35 mL/m2; p=0.070), LV end-systolic volume (D=6.31 mL; p=0.0045), and indexed LV end-systolic volume (4.80 mL/m2; p=0.0122). LV end-systolic volume is often used as a surrogate for LV remodeling in clinical trials evaluating cardiac outcomes. There was a marked decrease in the CK-MB isozyme biomarker in CAP-1002-treated compared to the Placebo arm indicative of reduced cardiac muscle damage over the course of the study. The HOPE-2 study demonstrated that CAP-1002 therapy is safe and effective in treating deterioration of upper limb and cardiac function in DMD patients.