Alexion Pharmaceuticals, Inc. announced plans to initiate a global Phase 3 study to investigate ULTOMIRIS (ravulizumab-cwvz) in a subset of adults with COVID-19 – those who are 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 has been in close contact with physicians and global health authorities in an effort to rapidly evaluate the potential of C5 inhibition in treating patients with severe COVID-19,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “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. 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.”
The decision to begin this trial is based on a) published preclinical data suggesting that inhibition of terminal complement can lower cytokine and chemokine levels and significantly reduce lung inflammation and pathology in animal models of viral pneumoniai, and b) elevated complement biomarkers and promising preliminary clinical evidence from patients who have accessed SOLIRIS® (eculizumab) through our compassionate use program, which suggests that complement inhibition may improve coronaviral-mediated lung injury.
Independent investigators have expressed interest in studying the potential of C5 inhibition in severe COVID-19 pneumonia, and we are aware of several ongoing or planned independent studies and anecdotal results from the use of our C5 inhibitors in patients with COVID-19. While these healthcare professionals continue to aggregate data regarding the potential of terminal complement inhibition in COVID-19 pneumonia from the approximately 100 patients who have been treated so far, Alexion believes that the outcomes reported to date warrant conducting a controlled clinical program to explore the impact of C5 inhibition with ULTOMIRIS and establish clinical evidence supporting the role of terminal complement in coronaviral pneumonia. We believe ULTOMIRIS represents the future of C5 inhibition, with its weight-based dosing, reduced burden on hospital systems due to less frequent dosing and it can be manufactured at a higher capacity, providing the opportunity to better meet future supply demands.