Rare disease cell therapy developer Capricor Therapeutics, Inc. (NASDAQ:CAPR) said Thursday that it received the official minutes of the recent meeting with the U.S. Food and Drug Administration regarding the company's CAP-1002, its off-the-shelf CDC (cardiosphere-derived cells) treatment for the genetic disorder Duchenne muscular dystrophy.
Investors are applauding the news today for a couple reasons. First, the minutes suggest that the FDA sees enough pre-clinical safety and efficacy data to support an investigational new drug application seeking to commence clinical studies evaluating repeated infusions of CAP-1002.
Second, the regulatory agency also seems okay with Capricor's proposal to use the Performance of the Upper Limb (PUL) instrument as the metric for the primary endpoint in clinical trials that Capricor hopes will lead to a biologics license application one day for the therapy.
The Los Angeles-based company is currently conducting a phase 1/2 clinical trial of CAP-1002 in boys and young men with Duchenne with cardiac involvement. Six-month data to date indicates a statistically-significant improvement versus standard of care in the 25 patients enrolled and treated with CAP-1002 as measured by PUL and specific assessments of heart function.
Cardiomyopathy is the number one cause of death in patients with Duchenne.
Dubbed the HOPE (Halt cardiomyOPathy progrEssion in Duchenne) study and partially funded by the California Institute for Regenerative Medicine, this clinical look at CAP-1002 has further shown the treatment to be generally safe and well tolerated. Patients are treated with CAP-1002 through a non-stop flow technique in all three coronary arteries.
In short, the opinion of the FDA paves the way for Capricor to initiate a phase 2 study of repeated, IV doses of CAP-1002, which is now expected by the end of the year. Given the gross need for novel therapies to treat the disease that kills most patients in their twenties, the investment community is rallying behind shares of the stock.
After closing Wednesday at $1.01, shares have popped as high as $1.64 in Thursday trading, currently holding at $1.24 with about two hours left in the session.
Shares are still down over 50% so far in 2017, though, underpinned by poor data in May from a separate phase 2 trial of CAP-1002 for helping patients that experienced a large heart attack with residual cardiac dysfunction.
