Last updated Feb. 16, 2022, by Marisa Wexler, MS

✅ Fact-checked by José Lopes, PhD

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

The U.S. Food and Drug Administration has granted CAP-1002 regenerative medicine advanced therapy (RMAT), orphan drug, and rare pediatric disease designations, which are given to experimental therapies with the potential to improve care for serious or rare diseases.

How CAP-1002 works

DMD, a type of muscular dystrophy, 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 makes it weak and causes scarring, a condition called cardiomyopathy, 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. By releasing sacks of cellular material called exosomes, CDCs modulate immune cell activity 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 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. CAP-1002 also inhibited scarring, inflammation, and a type of cell damage called oxidative stress in the preclinical models.

A Phase 1/2 clinical trial (NCT02485938) called HOPE-Duchenne tested CAP-1002 in 25 male patients, ages 12 and up, with DMD-related cardiomyopathy. Most participants relied on a wheelchair and had substantial shoulder function impairment. All the participants in the open-label trial were given standard care, including corticosteroids, and 13 received one dose of CAP-1002 (75 million cells) administered directly into the heart.

Results from HOPE-Duchenne indicated that treatment with CAP-1002 reduced heart muscle scarring, and helped thicken the heart’s left ventricle, which is crucial for pumping oxygenated blood through the body. Benefits were still evident after six months and at one year. 

Capricor launched a double-blind Phase 2 trial (NCT03406780) called HOPE-2 in 2018. The study enrolled 20 boys and young men with relatively advanced DMD, 80% of whom were unable to walk. Participants were randomly assigned to receive a placebo or CAP-1002 (150 million cells per infusion), given via infusion into the bloodstream every three months for a year. All were given standard steroid treatment.

Results from HOPE-2 showed that, compared with a placebo, CAP-1002 significantly improved upper limb function, as assessed via a validated test called Performance of the Upper Limb (PUL) 1.2. It also improved several measures of lung and heart health. For example, left ventricle ejection fraction, which is an assessment of how much blood the heart pushes out to the body with each pump, was significantly higher, by 4% on average, among CAP-1002-treated patients.

Capricor is planning a potentially pivotal Phase 3 clinical trial called HOPE-3 (NCT05126758) to test the safety and effectiveness of CAP-1002 in DMD. The study, which is not yet recruiting, plans to enroll about 68 boys and young men with DMD, ages 10 and older, who have some difficulty walking. Participants will be given infusions of CAP-1002 (150 million cells per infusion) or a placebo every three months for a year. The study’s main goal is to assess the effect of treatment on upper limb function.

Other information

Potential allergic reactions to CAP-1002 may be managed with anti-inflammatory treatments prior to infusion of the therapy.


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