Alexion Pharmaceuticals Inc. announced today it would start a late-stage study of its rare blood-disorder drug, Ultomiris, in COVID-19 patients hospitalized with severe pneumonia or acute respiratory distress syndrome (ARDS).
The study is expected to enroll approximately 270 patients across countries with high numbers of diagnosed cases, beginning in May, and will evaluate the impact of Ultomiris, a biologic medicine, on survival, duration of mechanical ventilation, and hospital stay compared to best supportive care.
This follows the U.S. Food and Drug Administration’s (FDA) rapid review and acceptance of Alexion’s investigational new drug (IND) application for Ultomiris for severe COVID-19.
Alexion’s drug works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells.
“Based on early anecdotal information available from compassionate use cases in multiple countries, we are launching a controlled clinical trial to evaluate the potential of ULTOMIRIS in mitigating the severe pneumonia and lung injury caused by the virus,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “As we move quickly to initiate this program, we also remain committed to serving the patients who currently rely on our medicines and providing continuous supply to these patients.”
Preclinical data suggests that inhibition of terminal complement can lower cytokine and chemokine levels and significantly reduce lung inflammation and pathology in animal models of viral pneumonia.