Ultomiris to initiate a Phase III study to treat COVID-19.-Alexion Pharma

Alexion Pharmaceuticals, Inc.announced plans to initiate a global Phase III 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 FDA rapid review and acceptance of Alexion’s investigational new drug (IND) application for Ultomiris for severe COVID-19.

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 Solirfis (eculizumab) through our compassionate use program, which suggests that complement inhibition may improve coronaviral-mediated lung injury../p>

Independent investigators have expressed interest in studying the potential of C5 inhibition in severe COVID-19 pneumonia, and the company is aware of several ongoing or planned independent studies and anecdotal results from the use of its 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..