RARE Daily

Capricor Reports Positive Results from Phase 2 Study of Regenerative Therapy in DMD

May 13, 2020

Rare Daily Staff

Capricor Therapeutics reported positive top-line results from a phase 2 study of CAP-1002, its cell therapy to treat patients in advanced stages of the rare, progressive, and fatal neuromuscular condition Duchenne muscular dystrophy.

Duchenne muscular dystrophy (DMD) causes muscle degeneration and leads to death, generally before the age of 30, most commonly from heart failure. It is characterized by progressive weakness and chronic inflammation of the skeletal, heart, and respiratory muscles. Boys and young men typically lose their ability to walk in their teens and generally die of cardiac or respiratory complications by the third decade of life. Treatment options are limited, and there is no cure.

CAP-1002 consists of allogeneic “off-the-shelf” cardiosphere-derived cells, or CDCs, a type of cardiac cell therapy that has been shown in pre-clinical and clinical studies to exert potent immunomodulatory activity. It is being investigated for its potential to modify the immune system’s activity to encourage cellular regeneration. The cells function by releasing exosomes that are taken up largely by macrophages and T-cells and begin a cycle of repair.

HOPE-2 is a randomized, double-blind, placebo-controlled, phase 2 clinical trial of CAP-1002, the company’s lead experimental therapy, in boys and young men who are in advanced stages of DMD. Study patients were treated via intravenous delivery with either CAP-1002 or placebo every 3 months. Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point in the intent to treat (ITT) population. Approximately 80 percent of the patients were non-ambulant, and all patients were on a stable regimen of steroids. Demographic and baseline characteristics were similar between the two treatment groups.

The 12-month data from HOPE-2 showed statistically meaningful improvements in upper limb, cardiac and respiratory function. With the exception of steroids, preservation of function in DMD is uncommon. The placebo patients declined consistent with natural history, but in the treated group, most patients were stable or improved throughout the one-year treatment period.

There was also a reduction in the biomarker CK-MB, an enzyme that is only released when there is cardiac muscle cell damage. In normal human subjects, there is typically no CK-MB measurable in the blood. It is well accepted that continuous muscle cell damage in DMD leads to pathologically high enzyme levels associated with cardiac muscle cell loss.  HOPE-2 demonstrated a reduction in CK-MB levels as compared to placebo. This is the first ever study in DMD that correlates cardiac functional stabilization with reduction of a biomarker of cell damage.

“To date, there are no therapies to treat the cardiomyopathy associated with DMD,” said Linda Marbán, president and CEO of Capricor. “Based on the statistically and clinically meaningful improvements in these and other measures of skeletal, cardiac, and respiratory performance, we have requested an End-of-Phase 2 meeting with FDA to discuss next steps and a pathway to approval of a Biologics License Application for CAP-1002 in DMD.”


Photo: Linda Marbán, president and CEO of Capricor

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