Alexion: Positive Topline Results in Phase 3 Myasthenia Gravis Study

 Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced positive topline results from a Phase 3 study evaluating the safety and efficacy of ULTOMIRIS® (ravulizumab-cwvz) in adults with generalized myasthenia gravis (gMG). The study met, with high statistical significance, its primary endpoint of change from baseline in the Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score, a patient-reported assessment, at Week 26, and for the subset of patients who have completed 26 weeks in the extension study to date, the positive treatment effect was maintained through a total of 52 weeks. ULTOMIRIS was well tolerated with a safety profile consistent with that observed in Phase 3 studies in paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Based on these results, Alexion plans to make regulatory filings in the U.S., European Union and Japan in late 2021/early 2022.

“The treatment landscape for people living with gMG has advanced and expanded rapidly in recent years, empowering both patients and caregivers. However, as a clinician and scientist, I know the work is not done,” said Professor James F. Howard, M.D., Department of Neurology at The University of North Carolina, Chapel Hill, USA, and lead primary investigator in the Phase 3 study. “These Phase 3 ULTOMIRIS results reinforce the critical role complement inhibition plays in treating gMG. I am encouraged by the opportunity this could provide for more patients to be treated early with a mechanism of action designed to preserve neuromuscular function.”

“The approval of SOLIRIS was a critically important first step in addressing the urgent need for a treatment for people with severe symptoms and complications of MG, and was the first new treatment for this devastating disease in more than 60 years. Today’s results demonstrate that ULTOMIRIS may help a broader range of patients than was studied in the SOLIRIS Phase 3 trial, including those with milder symptoms or earlier in their treatment journey, while still offering clinically meaningful benefits that were seen as early as Week 1 and maintained up to 52 weeks,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “These data provide confidence that ULTOMIRIS has the potential to become the new standard of care for gMG and may reduce patient burden with its less frequent dosing schedule, leading to better treatment adherence and patient satisfaction. We are working to prepare regulatory submissions in the U.S., EU and Japan as quickly as possible.”

https://www.businesswire.com/news/home/20210715006015/en/Alexion-Announces-Positive-Topline-Results-from-Phase-3-Study-of-ULTOMIRIS%C2%AE-ravulizumab-cwvz-in-Adults-with-Generalized-Myasthenia-Gravis-gMG