CHESHIRE, Conn.--(BUSINESS WIRE)--Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced that the Company’s study of eculizumab (Soliris®) in a small group of 14 patients with severe and refractory generalized myasthenia gravis, an ultra-rare and debilitating form of generalized myasthenia gravis (gMG), showed a strong disease improvement signal. The exploratory 14-patient study aimed to identify a clinically meaningful benefit of eculizumab in improving Quantitative Myasthenia Gravis disease severity score (QMG score) relative to placebo. The primary endpoint showed a clinically meaningful trend, and important secondary endpoints were achieved with statistical significance. Data from the Phase 2 study were presented today at the annual Scientific Session of the Myasthenia Gravis Foundation of America, Inc. (MGFA) in San Francisco. Alexion is now planning further investigation of eculizumab as a treatment for patients with severe and refractory gMG.
