Capricor Recruiting Duchenne Patients for Phase 2 Trial of CAP-1002

Capricor Therapeutics’ Phase 2 clinical trial to evaluate the safety and effectiveness of CAP-1002 in boys and young men with Duchenne muscular dystrophy (DMD) has started recruiting participants.

The HOPE-2 trial (NCT03406780) is planned to enroll up to 84 participants ages 10 or older with advanced stages of the disease who have been treated with standard therapies, including glucocorticoids.

From 12 to 15 clinical centers are expected to take part in the study; the University of California Davis Medical Center is the first site currently recruiting and treating patients.

Study participants will randomly receive either the investigative therapy or a placebo, administrated via intravenous injection, every three months for a total of four treatment cycles over a 12-month period.

Investigators will evaluate the potential of the treatment to improve the physical function of Duchenne patients. This will include improvements in upper limb and finger movements, which are essential for the patients to be able to perform manual tasks related to daily living activities.

“We are very pleased to begin this important clinical trial of CAP-1002,” Craig McDonald, MD, the national principal investigator for the HOPE-2 clinical trial and UC Davis professor, said in a press release.

“The HOPE-2 trial will study whether CAP-1002 can maintain or improve cardiac and skeletal muscle function. Because many of the participants are non-ambulatory, the study will focus primarily on the impact on arm mobility,” he said.

If the trial shows a positive balance between the effects of the treatment and its safety, the patients may be enrolled in an extension study during which they may continue treatment with CAP-1002.

CAP-1002 is a cell therapy that is composed of cardiosphere-derived cells from donated tissue. But these engineered cells do not work as conventional stem cells do. Instead of trying to restore dystrophin levels — the protein lacking in DMD — they release tiny vesicles containing factors that will prevent inflammation and balance the immune response.

Recent data collected from mouse models of DMD revealed that additional administrations of CAP-1002 could significantly improve the animals’ physical response to exercise.

In addition, treated mice showed improvement in absolute force in the lower limbs and in the diaphragm, as well as reduced scarring in the skeletal and cardiac muscles. This finding suggests that repetitive dosing of the treatment may help sustain its beneficial effects for longer periods of time.

These preclinical results were presented at the 11th Annual Neuromuscular Translational Research Conference in Cambridge, England.

The poster was titled, “Allogeneic CDCs have a low immunogenic profile and repeat-dosing showed additional improvement when compared with single treatment in a Duchenne muscular dystrophy model.”

Results from the previous Phase 1/2 HOPE-Duchenne trial (NCT02485938) showed that a single dose of CAP-1002 cells could effectively improve muscle wall thickening of the heart, a feature required for proper blood pumping throughout the body.

The investigative treatment was also found to improve skeletal muscle function in eight of the nine treated boys, demonstrated by a range of hand and arm function tests.

“We are thrilled to begin enrolling participants in HOPE-2 because we have seen the potential for improvements in muscle function in both pre-clinical studies and in our earlier HOPE-Duchenne trial,” said Linda Marbán, PhD, president and CEO of Capricor.

“We are hopeful that HOPE-2 may potentially be a registration trial,” being one of the few studies to focus on helping Duchenne patients with seriously impaired walking capacity, she added.

The U.S. Food and Drug Administration has granted CAP-1002 orphan drug status, Rare Pediatric Disease designation, and Regenerative Medicine Advanced Therapy designation, all of which are expected to expedite approval and delivery of this cell therapy to DMD patients.