Alexion Updates on Phase 3 Study of ULTOMIRIS in Severe Covid

 – Independent data monitoring committee recommends pausing study enrollment due to lack of efficacy in pre-specified interim analysis –

– Company will conduct further analysis of trial data to determine next steps –

– No new safety findings were observed for ULTOMIRIS use in COVID-19 –

Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) today announced the decision to pause further enrollment in the global Phase 3 study of ULTOMIRIS® (ravulizumab-cwvz) in adults with severe COVID-19 requiring mechanical ventilation. This decision is based on the recommendation of an independent data monitoring committee (IDMC), following their review of data from a pre-specified interim analysis. The IDMC recommended that additional enrollment be paused, pending further analysis of the data, due to lack of efficacy when ULTOMIRIS was added to best supportive care, compared to best supportive care alone. There were no new safety findings observed. The study will continue for patients already enrolled, including completion of all study visits and planned ULTOMIRIS dosing according to the study protocol.

“We would like to thank the patients and their families, as well as investigators and healthcare professionals, who were essential to this study. We greatly value their contributions to help investigate potential ways to address this devastating pandemic,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “While initial anecdotal reports from compassionate use cases were promising, these results demonstrate the importance of conducting controlled clinical trials to fully evaluate the potential of new treatment approaches and generate the necessary evidence to make informed decisions. We are disappointed in this initial outcome, but plan to further analyze the data to identify potential subgroups who may benefit and to determine next steps. In addition, we remain fully committed to our efforts to serve the rare disease community and to continuing to provide ULTOMIRIS to the patients who currently rely on it.”

The IDMC’s recommendation was based on a pre-planned interim analysis of the primary endpoint – survival at Day 29 – once 122 patients completed the 29-day primary evaluation period. No secondary endpoints were analyzed as part of the interim analysis.

In the UK, the TACTIC-R platform study led by Cambridge University Hospitals NHS Foundation Trust, which includes an ULTOMIRIS cohort, is evaluating the potential of earlier immune modulatory treatment (hospitalized patients not requiring mechanical ventilation) in preventing progression of the virus, including reducing the need for ICU admission and ventilation. This independent study remains ongoing.

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