Key results from the study, include:
- CAP-1002 is an investigational cell therapy which aims to slow disease progression through immunomodulatory, anti-inflammatory and anti-fibrotic actions with the goal of potentially improving the rate of decline in skeletal and cardiac muscle function in patients with DMD.
- Skeletal muscle function as measured by the Performance of the Upper Limb (PUL v2.0) showed a mean PUL v2.0 decline after 24-months of treatment with CAP-1002 was 2.8 points versus a 7.7 point decline on average observed over 24-months in the placebo patient group.
- Delta change=4.9 points; p=0.021
- The average rate of decline in CAP-1002 treated patients showed an attenuation of disease progression by approximately 64%
- CAP-1002 revealed clinically meaningful improvements in ameliorating cardiac function.
- Cardiac function as measured by left ventricular ejection fraction (LVEF%) by MRI at the 24-month timepoint, improved in 67% of patients, compared to a steady decline in a comparable natural history population
- CAP-1002 treatment during the OLE portion of the study continues to yield a consistent favorable safety profile and has been well-tolerated throughout the study.
- The HOPE-2 OLE study previously met the primary endpoint at the one-year time-point and these 24-month results suggest that patients accumulate benefit over time with preservation of skeletal muscle function, which underscore the potential long-term benefit of CAP-1002.
“We are pleased to be presenting our positive long-term open label extension data at this year’s
Podium Presentation in Clinical Trial Updates Session
Title: | 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 |
Date: | |
For more information on the MDA conference, please visit www.mdaconference.org.
About the HOPE-2 Open Label Extension (OLE) Study
HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical study of Capricor’s lead investigational therapy, CAP-1002, in boys and young men who have DMD. Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months. Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point and the results were published in
About
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For more information, please contact:
Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755
Source:
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