CAP-1002 is an investigational cardiac cell therapy, being developed by Capricor Therapeutics, for the treatment of heart conditions, including cardiomyopathy linked to Duchenne muscular dystrophy (DMD).

DMD is a genetic disorder caused by the absence of dystrophin – a protein that plays an essential role in maintaining muscle cells intact. 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. The condition is called cardiomyopathy, which is the leading cause of death in DMD patients.

How CAP-1002 works

CAP-1002 consists of cardiosphere-derived cells (CDCs), which are progenitor (or precursor/parent) cells capable of developing into mature heart cells, and modulate the immune system to promote heart repair.

The CDCs in CAP-1002 derive from the heart tissue of a healthy donor, 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 the ability of CDCs to improve heart function and exercise capacity, as well as to inhibit oxidative stress, inflammation, and scarring.

A Phase 1/2 clinical trial (NCT02485938) called HOPE-DMD is currently testing CAP-1002 in 25 male patients, ages 12 or more, with DMD-related cardiomyopathy. The open-label, multicenter study aims to evaluate the safety and effectiveness of CAP-1002. Participants are being randomized to receive either intracoronary infusions of the drug (total dose of 75 million CDCs) in three coronary arteries (the active group) or a usual care treatment (the control group). All heart measurements are done by MRI scans.

The first six-month results from the HOPE-DMD trial were announced by Capricor in April 2017. Overall, CAP-1002 was well-tolerated and safe. Participants in the active group demonstrated significant improvements in certain heart and arm function tests when compared to the control group. These clinical results are in line with the pre-clinical data.

Other information

CAP-1002 was granted orphan drug status by the U.S. Food & Drug and Administration (FDA) in April 2015 based on the positive results of preclinical studies.

On July 18, 2017, CAP-1002 was granted rare pediatric disease designation by the FDA for patients with DMD based on the positive six-month results from the HOPE-DMD trial. The designation provides Capricor with incentives to continue the development of CAP-1002 for DMD. The designation also makes the company eligible to receive priority review if the FDA approves marketing of the drug. HOPE-DMD’s 12-month results are expected by the end of 2017.

The company recently met with the FDA to discuss potential product registration strategies and plans to start a randomized, double-blind, placebo-controlled clinical trial to further evaluate the potential of intravenous, repeat-dose CAP-1002 in boys and young men with DMD.

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