Alexion Pharmaceuticals has presented new results from the REGAIN study, a global, placebo-controlled Phase 3 registration trial of eculizumab (Soliris®) in patients with refractory generalized myasthenia gravis (gMG).

“Although the REGAIN study narrowly missed its primary endpoint, the additional data presented today suggest a magnitude of effect of eculizumab in refractory MG patients across four separate scales of disease severity that is unprecedented in my clinical investigation experience,” said James F. Howard Jr., M.D., Distinguished Professor of Neuromuscular Disease, Professor of Neurology, Medicine & Allied Health, and Chief, Neuromuscular Disorders Section, The University of North Carolina School of Medicine.

“These findings are particularly meaningful given the urgent need for a first-ever therapy with the potential to have a transformative impact on patients with refractory MG, who continue to face disabling limitations in their daily lives, including difficulty walking, talking, swallowing, and breathing normally.”