Six of six critically ill COVID-19 patients survived after being treated as compassionate care cases with Capricor Therapeutics Inc.’s lead candidate, an off-the-shelf cardiac cell therapy. The success prompted the FDA to review the data and approve the company’s expanded access protocol for treating as many as 20 more COVID-19 patients.
The market liked all of it, as the stock (NASDAQ:CAPR) closed 252.7% higher at $8.50 per share on April 29.
The five male and one female patients, ages 19 to 75, were treated at Cedars-Sinai Medical Center in Los Angeles with Capricor’s CAP-1002, a cardiac cell therapy made up of allogeneic cardiosphere-derived cells that exert immunomodulatory activity, according to preclinical and clinical studies. While it has an IND, CAP-1002 is not approved for any indications.
In one month, the six patients, all suffering from acute respiratory distress syndrome received I.V. infusions of 150 million allogeneic cardiosphere-derived cells. Of the five who were on ventilators, four no longer needed them within one to four days following the infusion, the company said. The fifth remains on mechanical ventilation, and the sixth patient is receiving supplemental oxygen and is listed as clinically stable. Four of the six have been discharged from Cedars-Sinai.
Laboratory biomarkers correlated with poor outcomes were measured in all six patients. Following the infusion, several patients showed improvements in biomarkers such as ferritin, absolute lymphocyte counts and C-reactive protein. No adverse events related to the administration of CAP-1002 were observed.
Capricor said CAP-1002’s immune-modulatory properties could be at the center of reining in an overstimulated immune system by modulating various inflammatory cytokines and regulating the immune response. It’s possible that the body’s overreaction to COVID-19, rather than the virus itself, can lead to death, the company added.
Los Angeles-based Capricor also plans a randomized, placebo-controlled trial for treating patients with moderate and severe COVID-19. Capricor also said it submitted an expanded access IND to investigate use of CAP-1002 in certain COVID-19 patients.
The company also expects top-line results from its interventional phase II HOPE-2 study of CAP-1002 in treating Duchenne muscular dystrophy (DMD) in mid-May. Interim data from the prespecified interim analysis showed that teens and young men with advanced DMD saw skeletal, pulmonary and cardiac improvements after receiving multiple doses of CAP-1002. The patients showed improvements in the Performance of the Upper Limb, a tool for assessing high (shoulder), mid (elbow) and distal (wrist and hand) function, with a conceptual framework reflecting the progression of weakness in ambulant and non-ambulant patients.
HOPE-2 is a randomized, double-blind, placebo-controlled trial in which participants receive CAP-1002 or a placebo in a 1-to-1 ratio. The study began in April 2018 and was set to be completed in April 2020.