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Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis

Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in... Key PointsQuestionWhat are the clinical effects of zilucoplan, a subcutaneously self-administered macrocyclic peptide inhibitor of complement component 5, in a broad population of patients with moderate to severe acetylcholine receptor autoantibody–positive generalized myasthenia gravis? FindingsIn a randomized, double-blind, placebo-controlled, multicenter phase 2 trial, zilucoplan yielded rapid, clinically meaningful, statistically significant, and sustained improvements in the primary and key secondary end points. Near-complete complement inhibition was associated with a faster onset and greater magnitude of benefit than submaximal complement inhibition, and favorable safety and tolerability were observed. MeaningThe findings support a potential therapeutic role for zilucoplan in generalized myasthenia gravis and further evaluation in a phase 3 study. http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png JAMA Neurology American Medical Association

Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis

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References (73)

Publisher
American Medical Association
Copyright
Copyright 2020 Howard JF Jr et al. JAMA Neurology.
ISSN
2168-6149
eISSN
2168-6157
DOI
10.1001/jamaneurol.2019.5125
Publisher site
See Article on Publisher Site

Abstract

Key PointsQuestionWhat are the clinical effects of zilucoplan, a subcutaneously self-administered macrocyclic peptide inhibitor of complement component 5, in a broad population of patients with moderate to severe acetylcholine receptor autoantibody–positive generalized myasthenia gravis? FindingsIn a randomized, double-blind, placebo-controlled, multicenter phase 2 trial, zilucoplan yielded rapid, clinically meaningful, statistically significant, and sustained improvements in the primary and key secondary end points. Near-complete complement inhibition was associated with a faster onset and greater magnitude of benefit than submaximal complement inhibition, and favorable safety and tolerability were observed. MeaningThe findings support a potential therapeutic role for zilucoplan in generalized myasthenia gravis and further evaluation in a phase 3 study.

Journal

JAMA NeurologyAmerican Medical Association

Published: May 17, 2020

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