Ra Pharmaceuticals, Inc. announced that full results from its Phase 2 clinical trial of zilucoplan in patients with generalized myasthenia gravis (gMG) were published online in JAMA Neurology. As reported in the JAMA Neurology article, “Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients with Moderate to Severe Generalized Myasthenia Gravis: Results of a Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Trial,” the study demonstrated that zilucoplan resulted in rapid, clinically meaningful, statistically significant, and sustained improvements in the primary and key secondary endpoints, namely, change from baseline to week 12 in Quantitative Myasthenia Gravis (QMG) and MG Activities of Daily Living (MG-ADL) scores, respectively. Further, near-complete complement inhibition was associated with a faster onset and greater magnitude of benefit than submaximal complement inhibition, with a favorable safety and tolerability profile. Ra Pharma is currently evaluating zilucoplan in the RAISE study, a single, global, pivotal, randomized, double-blind, placebo-controlled, Phase 3 clinical trial, for the treatment of gMG. The trial, which incorporates feedback from the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), is designed to evaluate the efficacy of a once-daily, subcutaneously (SC) self-administered dose of 0.3 mg/kg of zilucoplan versus placebo. Top-line results from the RAISE study are expected in early 2021.