The REGAIN study was a placebo-controlled Phase 3 study assessing the efficacy and safety eculizumab (Soliris) in patients with refractory generalized myasthenia gravis. Last month, we reported that the trial failed to meet its primary endpoint. At the 2016 International Congress on Neuromuscular Diseases (ICNMD) meeting in Toronto, Canada, further analysis of the REGAIN study was presented by James Howard Jr, MD of the Department of Neurology at the University of North Carolina School of Medicine and showed that the totally of the data seems to indicate the drug is effective for treating patients with refractory generalized myasthenia gravis.
Howard J, Utsugisawa K, Benatar M, et al. REGAIN: a randomized, double-blind, placebo-controlled, multi-center phase 3 study of the safety and efficacy of Eculizumab in subjects with refractory generalized myasthenia gravis. Poster presented at ICNMD 2016; Toronto, Canada; July 5-9, 2016. Abstract #416.