Top-line Ph III results for Ultomiris in adults with gMG meet primary goal

Positive top-line results from a Phase III study evaluating the safety and efficacy of Ultomiris (ravulizumab-cwvz) in adults with generalized myasthenia gravis (gMG) were announced yesterday by Alexion Pharmaceuticals (Nasdaq: ALXN), whose shares nevertheless dipped 3.3% to $180.51 by close of trading.

The study met, with high statistical significance, its primary endpoint of change from baseline in 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 III studies in paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).

Based on these results, Alexion plans to make regulatory filings in the USA, European Union and Japan in late 2021/early 2022.