Capricor completed the HOPE-2 clinical trial for Capricor’s lead investigational therapy, CAP-1002. The clinical trial evaluated the safety and efficacy of repeat, intravenous (IV) doses of allogeneic cardiosphere-derived cells (CDCs), or CAP-1002, in boys and young men in advanced stages of Duchenne muscular dystrophy, a fatal genetic disorder for which there are limited treatment options.
While there are many clinical initiatives in Duchenne muscular dystrophy, HOPE-2 is one of the very few to focus on non-ambulant patients. These boys and young men are looking to maintain what function they have in their arms and hands, and Capricor’s previous study of a single intracoronary dose of CAP-1002 indicates the therapy may be able to help them retain those functions or to slow their loss of those functions.
For HOPE-2, the primary efficacy endpoint was the relative change in patients’ abilities to perform manual tasks that relate to activities of daily living and are important to their quality of life. These abilities will be measured through a validated test for skeletal muscle function in Duchenne muscular dystrophy, the Performance of the Upper Limb (PUL) test.
The FDA has granted Capricor’s CAP-1002 RMAT and Orphan Drug Designation, and the FDA has also granted a Rare Pediatric Disease Designation to CAP-1002 for DMD. The Rare Pediatric Disease Designation, as well as the Orphan Drug Designation previously granted, covers the broad treatment of DMD. If Capricor were to receive market approval for CAP-1002 by the FDA, Capricor would be eligible to receive a Priority Review Voucher.
Please visit ClinicalTrials.gov for more information regarding the HOPE-2 clinical trial.
Watch patient testimonial from the HOPE-1 clinical trial here
Watch a video on DMD and CAP-1002’s focus here
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