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Capricor Therapeutics Announces Positive 3-Year Efficacy Results from HOPE-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular Dystrophy
--Results in Performance of the Upper Limb (PUL 2.0) Continue to Show Benefits in Skeletal Muscle Function After 3 Years of CAP-1002 Treatment (p
About this update from Capricor Therapeutics, Inc.
[{"type":"text","content":"--Results in Performance of the Upper Limb (PUL 2.0) Continue to Show Benefits in Skeletal Muscle Function After 3 Years of CAP-1002 Treatment (p --Stabilization in Left Ventricular Ejection Fraction (LVEF) Suggests Preservation of Cardiac Function-- --Results Recently Shared with FDA at Type-B Meeting Held in May 2024-- SAN DIEGO, June 04, 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 positive 3-year results from the ongoing HOPE-2 open-label extension (OLE) study with CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD). Patients treated with CAP-1002 continue to show positive benefits after 3 years of treatment in both the PUL 2.0 and LVEF measures when compared to an external comparator dataset of similar DMD patients (dataset provided by Cincinnati Children’s Hospital Medical Center (CCHMC)). Further, CAP-1002 treatment during the HOPE-2-OLE study continues to have a consistently well-tolerated safety profile throughout the study. The Company plans to present these results in more detail at the upcoming Parent Project Muscular Dystrophy (PPMD) 30th Annual Conference being held June 27-29, 2024. 3-Year HOPE-2 OLE Topline Study Results Primary Endpoint3-Year Timepoint Change from Baseline*Delta Changep-valueSkeletal-Muscle (Upper Limb Function)Full Performance of Upper Limb (PUL 2.0)CAP-1002 (n=12)External Comparator (n=32)-4.1 points-7.8 points+3.7 pointsp *Baseline is referred to as start of HOPE-2 OLE study, changes in means are shown “I am extremely encouraged by these positive results from our HOPE-2 OLE study which continue to support the long-term impact of CAP-1002 for the treatment of DMD,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “Importantly, the external comparator of the disease shows a steady state of decline in both skeletal and cardiac function; however over the course of 3-years, we observed a statistically significant reduction in PUL decline as well as stabilization in LVEF in CAP-1002 treated patients suggesting sustained disease attenuation. Furthermore, at our recent Type-B meeting with the U.S. Food and Drug Administration (FDA), we shared these results with the FDA to support what we expect to see in the HOPE-3 pivotal...