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Capricor Therapeutics Announces Statistically Significant Clinical Benefits in Skeletal Muscle Function in Non-Ambulant Duchenne Muscular Dystrophy Patients Treated with CAP-1002 in HOPE-2 Open Label Extension Study
-Met Primary Endpoint of Performance of the Upper Limb PUL 2.0 (p=0.02)- -Results Suggest Disease Modification in DMD and Long-term Safety of CAP-1002-
About this update from Capricor Therapeutics, Inc.
[{"type":"text","content":"-Met Primary Endpoint of Performance of the Upper Limb PUL 2.0 (p=0.02)- -Results Suggest Disease Modification in DMD and Long-term Safety of CAP-1002- -Results were Presented at Late Breaking Session at PPMD’s Annual Conference on Saturday June 25, 2022- -Principal Investigator Dr. Craig McDonald and Capricor Management Will Host Conference Call and Webcast Today at 8:00 a.m. ET- SAN DIEGO, June 27, 2022 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases, today announced positive one-year results from its HOPE-2 open label extension study in non-ambulant patients with later-stage Duchenne muscular dystrophy (DMD). Data from the rollover open label extension (OLE) study show statistically significant improvements on the Performance of the Upper Limb (PUL version 2.0) scale for patients on CAP-1002 testing three different hypotheses of treatment benefit during the open label extension. Capricor presented these results at a late-breaking session at this year’s Parent Project Muscular Dystrophy (PPMD) Annual Conference. Topline Efficacy Results Difference in One Year Change from Baseline† Difference (95% Confidence Interval) CAP-1002 (n=20)1p-value Skeletal Muscle Function (Upper Limb) PUL Version 2.0 Open Label Extension Treatment Difference vs. HOPE-2 Treatment Difference3.8 (0.5, 7.0)0.023 Open Label Extension vs. HOPE-2 Placebo Group2.3 (0.5, 4.1)0.015 Open Label Extension vs. HOPE-2 Placebo Group off-treatment (Placebo GAP)2.8 (0.8, 4.8)0.006 †One year change from baseline for a phase refers to a subject's change in one year during that phase.1The linear mixed model uses all available data for all 20 subjects (12 completers). In the study, CAP-1002 was made available to all of the original 20 patients enrolled in the HOPE-2 study. Of those, 13 entered and 12 completed the first year of study follow-up. As in HOPE-2, CAP-1002 was administered quarterly and current results are from the one-year analysis. The breakdown of patients included seven from the original placebo group and six from the original CAP-1002 treatment group. “For patients with DMD, time is associated with loss of function. The progressive nature of the disease puts these patients ...