Capricor Therapeutics’ Duchenne muscular dystrophy therapy (DMD) appears to have slowed disease progression in older boys and young men who are in the later stages of their disease and unable to walk, setting the biotech up for a phase 3 trial.
CAP-1002 is being tested in a phase 2 study called HOPE-2 for advanced DMD patients. Capricor is ultimately trying to determine whether the therapy can slow disease progression using a measure of upper limb performance and other functions. About 20 patients were analyzed after receiving either CAP-1002 or placebo every three months.
The cell-based therapy met the main goal of the trial by improving upper limb and cardiac function, according to data to be presented today at the World Muscle Society Virtual Congress. This suggests “a clinically relevant slowing of disease progression,” the company said in a press release.
HOPE-2’s principal investigator Craig McDonald, M.D., noted statistically significant changes in both skeletal and cardiac function in the patients who have limited therapeutic options. McDonald is a professor at the University of California, Davis and chair of the Department of Physical Medicine and Rehabilitation.
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CAP-1002 was generally safe and well tolerated by the patients, although Capricor reported two “hypersensitivity events” that were treated with some medication.
“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,” said Capricor CEO Linda Marbán, Ph.D.
DMD is a rare, incurable disease caused by mutations in the DMD gene that reduce production of a protein called dystrophin, leading to progressive muscle degeneration. CAP-1002 is derived from donated heart muscle and is meant to regenerate skeletal and cardiac muscle cells. Each patient in HOPE-2 received 150 million cells per dose.
Capricor will now confirm the durability of the treatment beyond 12 months in extension studies in these older non-ambulatory patients. The company said further placebo-controlled trials will be needed to confirm any benefit for younger patients.
The results are the second piece of promising news for DMD patients this week after Solid Bio rolled out some limited data on a gene therapy that was previously sidelined by safety and manufacturing concerns.