Capricor Reports 4-Year Data For Deramiocel, Shows Sustained Benefit In Duchenne Patients

Core Insights - Capricor Therapeutics released four-year safety and efficacy results from the HOPE-2 Open-Label Extension study of Deramiocel, showing a median change of -0.5 points in treated patients compared to baseline [1] - A subgroup analysis indicated that patients with baseline Left Ventricular Ejection Fraction (LVEF) >45% experienced greater clinical benefits, suggesting early intervention may preserve cardiac function [2] - The treatment also slowed skeletal muscle disease progression, with a smaller average decline in performance of the upper limb (PUL v2.0) in the fourth year (0.6 points) compared to the first year (1.8 points) [3] - Extended treatment with Deramiocel may help attenuate the progression of Duchenne Muscular Dystrophy (DMD) over time, maintaining a favorable safety profile throughout the study [4] - CEO Linda Marbán emphasized the strength and durability of Deramiocel's clinical benefits and the company's focus on moving toward potential approval with their Biologics License Application (BLA) under priority review [5] Summary by Category Efficacy Results - After four years of treatment, Deramiocel-treated patients showed a median change of -0.5 points compared to baseline [1] - Patients with baseline LVEF >45% showed greater clinical benefits, supporting early intervention [2] - The treatment slowed skeletal muscle disease progression, with a decline of 0.6 points in the fourth year compared to 1.8 points in the first year [3] Safety Profile - Deramiocel maintained a favorable safety profile throughout the study, indicating its potential for long-term use [4] Regulatory and Market Impact - The company is executing with urgency towards potential approval, with their BLA under priority review [5] - CAPR stock experienced a decline of 31.75%, trading at $8.16 at the time of publication [5]