Capricor Reports Positive Final Data From its Phase 2 DMD Trial of Experimental Cell Therapy

Capricor Therapeutics reported its experimental cell therapy CAP-1002 for the rare neuromuscular condition Duchenne muscular dystrophy met its primary endpoint and various secondary endpoints suggesting clinically relevant slowing of disease progression in its phase 2 trial.

Photo: Linda Marbán, CEO of Capricor

Duchenne muscular dystrophy is a genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart, and respiratory muscles. Patients suffering from DMD typically lose their ability to walk in their teenage years and generally die of cardiac or respiratory complications by age 30. It occurs in one in every 3,600 live male births across all races, cultures, and countries. DMD afflicts approximately 200,000 boys and young men around the world. Treatment options are limited, and there is no cure.

CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a type of progenitor cell that has been shown in preclinical and clinical studies to exert potent immunomodulatory activity and is being investigated for its potential to modify the immune system’s activity to encourage cellular regeneration. The therapy has been shown to regenerate skeletal and cardiac muscle cells. This final data will be presented today at this year’s World Muscle Society Virtual Congress.

“This groundbreaking study is extremely exciting as we saw statistically significant changes of CAP-1002 in both skeletal and cardiac function,” said Craig McDonald, the national principal investigator for the HOPE-2 clinical trial and UC Davis professor and chair of the Department of Physical Medicine and Rehabilitation. “For these older boys who have limited therapeutic options, these data support the belief that CAP-1002 may become an important therapeutic option and possibly slow the progression of DMD.”

HOPE-2 was a randomized, double-blind, placebo-controlled, phase 2 clinical trial of the company’s lead investigational therapy, CAP-1002, in boys and young men who have DMD and are non-ambulant, the later stage of the disease process. The trial was conducted at nine sites across the United States. Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every three 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 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. Final data analysis demonstrated that young men in the advanced stages of DMD experienced improvements in skeletal and cardiac measurements after receiving four doses of CAP-1002 over the course of one year.

Subjects in the trial were evaluated using the Performance of the Upper Limb (PUL), a validated tool specifically designed for assessing high (shoulder), mid (elbow) and distal (wrist and hand) function, with a conceptual framework reflecting the progression of weakness in upper limb function.

CAP-1002 was generally safe and well tolerated throughout the study. With the exception of two hypersensitivity reactions early in the clinical trial, which were mitigated with a common pre-medication regimen, there were no serious safety signals identified by the HOPE-2 Data and Safety Monitoring Board (DSMB).

“The data suggests that CAP-1002 slowed the decline of DMD in patients for whom few options currently exist,” said Linda Marbán, CEO of Capricor. “Now that we have clarity from the FDA and based on the strength of this data set, we are poised to embark on the HOPE-3 pivotal trial once we have secured an appropriate partner that can help drive CAP-1002 forward towards commercialization.”

This is the second clinical trial investigating CAP-1002 showing similar results in the treatment of DMD patients. Capricor completed the HOPE-Duchenne (phase 1/2) trial in 2019, the results of which were published in Neurology, the medical journal of the American Academy of Neurology. The company has initiated a technology transfer with Lonza, a leading global CMO to prepare for commercial manufacturing of CAP-1002.

Author: Rare Daily Staff