How CAP-1002 works
DMD is a genetic disorder caused by the absence of dystrophin, a protein that plays an essential role in maintaining muscle cells. It is characterized by progressive muscle degeneration and weakness. The lack of dystrophin in the heart muscle causes it to become enlarged, thick, and rigid. This condition is called cardiomyopathy, which is the leading cause of death in DMD patients.
CAP-1002 consists of cardiosphere-derived cells (CDCs), which are progenitor (precursor/parent) cells capable of developing into mature heart cells. They modulate the immune system to promote heart repair.
The CDCs in CAP-1002 come from the heart tissue of a healthy donor. They are grown in a laboratory and stored until needed.
CAP-1002 is directly administered by infusion into one or more coronary arteries (the blood vessels in the heart) using regular cardiac catheterization methods.
CAP-1002 in clinical trials
Preclinical studies with CAP-1002 in mouse models of DMD showed that CDCs were able to improve heart function, skeletal muscle function, and exercise capacity, as well as to inhibit oxidative stress, inflammation, and scarring.
A Phase 1/2 clinical trial (NCT02485938) called HOPE-DMD tested CAP-1002 in 25 male patients age 12 or older who have DMD-related cardiomyopathy. The open-label, multicenter study aimed to evaluate the safety and effectiveness of a single-dose of CAP-1002. Participants were randomized to receive either intra-coronary infusions of CAP-1002 (total dose of 75 million CDCs) in three coronary arteries (the active group), or a usual care treatment (the control group). All heart measurements were done by MRI scans. The results of the trial were positive and showed that CAP-1002 was well-tolerated, safe, and effectively reduced scarring in the heart muscle tissue and improved its function.
A randomized, double-blind, placebo-controlled Phase 2 trial (NCT03406780) called HOPE-2 is investigating the safety and effectiveness of repeat-doses of CAP-1002 in a group of male patients age 10 and older in advanced stages of DMD who are pre-treated with steroids to avoid an allergic reaction to CAP-1002. Patients are selected randomly and injected either with an infusion of CAP-1002 (at a dose of 150 million cells per infusion), or a placebo, every three months for one year. Interim data from the trial showed that CAP-1002 improved muscle and lung function significantly. The trial is expected to be completed in late 2019 or early 2020.
CAP-1002 was granted orphan drug status by the U.S. Food and Drug Administration (FDA) in April 2015 based on positive results from preclinical studies.
CAP-1002 was granted rare pediatric disease designation by the FDA on July 18, 2017, based on the positive results from the HOPE-DMD trial. The designation provides Capricor with incentives to continue the development of CAP-1002 for DMD.
CAP-1002 was granted the regenerative medicine advanced therapy (RMAT) by the FDA in February 2018. This also was based on positive results from the HOPE-DMD trial, which showed that CAP-1002 significantly reduced scarring of the heart muscle and improved thickening of the heart’s interior wall.
RMAT designation allowed the company to meet with the FDA in December 2018 to review the development program of CAP-1002. The company would be eligible to receive a priority review if the FDA approves marketing of the drug.
Last updated: July 22, 2019
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