Rare Disease Report

Encouraging Results for Duchenne Patients Seen in Capricor Study

APRIL 26, 2017
Jim Radke
Young men with Duchenne muscular dystrophy often develop cardiomyopathy as the disease progresses.
Small biotech, Capricor Therapeutics has developed allogenic cardiosphere-derived cells (CAP-1002) that can be infused into the main coronary arteries of Duchenne patient to strengthen the heart in these boys. Until recently, that strengthening was just theory based on animal models but the company just announced interim results from their ongoing Phase 1/2 trial and the muscle strengthening is not just a theory anymore.
These boys’ hearts seem to be getting stronger.
The HOPE (Halt cardiomyOPathy progrEssion in Duchenne) Trial is a 12-month study evaluating the safety and exploratory efficacy of CAP-1002 in 25 patients, 12 years and older, with DMD who had cardiomyopathy, or heart disease, secondary to DMD. Patients received either a single dose of CAP-1002 (13 patients) or usual care (12 patients). CAP-1002 was infused into each of the 3 main coronary arteries at a total dose of 75 million cells.
All cardiac assessments were performed by MRI.
While the study was designed to measures safety, and the drug was well tolerated with no patient discontinuing, the company and the Duchenne community were excited by the preliminary efficacy results that were shared by the company during their webcast.
For example, scar reduction was observed in the patients given CAP-1002 (see figure 1) as was systolic wall thickening (see figure 2).

Figure 1: Cardiac Scar Reduction

Figure 2: Systolic Wall Thickening

In addition to cardiac muscle improvement, the study found skeletal muscle strength to improve, as measured the Performance of the Upper Limb (PUL) test, in patients given CAP-1002 compared to controls (P = .045).
Finally, quality of life scores assessed at 3 months showed the patients given CAP-1002 had statically significant higher quality of life scores compared to controls but that difference was not observed at 6 months (figure 3).

Figure 3: Quality of Life Scores

KOLs Thrilled with the Preliminary Results

"In HOPE, we saw potential effects in both the heart and skeletal muscle that appear quite compelling in an exploratory trial,” John L. Jefferies, M.D., Professor of Pediatric Cardiology and Adult Cardiovascular Diseases at the University of Cincinnati and Director, Advanced Heart Failure and Cardiomyopathy, and Principal Investigator of the HOPE Trial, said via news release.
“These results clearly support the conduct of a confirmatory clinical trial in DMD to further evaluate the potential of CAP-1002. We look forward to an effective medication becoming available for people with this progressive and fatal disease, one that is poorly met by current options,” he continued.
Joao A. C. Lima, M.D., Professor of Medicine and Director of the Magnetic Resonance Imaging, or MRI, Core Lab at The Johns Hopkins University School of Medicine, added, "The observed signal in global cardiac scar reduction and the increase in the thickening of the left ventricle during contraction are very encouraging. The population treated in HOPE had very advanced cardiac involvement, and to see such positive results following just a single dose of CAP-1002 is remarkable. Cardiac disease is the most common cause of mortality among those with DMD."

Talks with the FDA Planned

Linda Marbán, Ph.D., Capricor's president and chief executive officer said the company hopes to meet with the FDA shortly to discuss the results and the company’s plans for future studies and FDA applications. Dr Marbán said, “We believe the interim HOPE results may enable us to pursue one of the FDA's Expedited Programs for Serious Conditions, and we will apply for either or both of the Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations for CAP-1002."

About Duchenne Muscular Dystrophy (DMD)

DMD is caused by lack of a functional dystrophin protein, a protein that helps keep muscle cells intact. Patients with progressive muscle disorder experience symptoms in early childhood, losing the ability to walk as early as age 10. These patients, mostly boys, experience life-threatening heart and lung complications in their late teens and twenties.

Currently, the only drugs approved for DMD are Exondys 51 (eteplirsen) which is an exon-skipping therapy that is effective in about 13% of DMD patients and Emflaza (deflazacort) which is a corticosteroid approved for all DMD patients. 

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