Positive topline results were announced from a phase 3 study evaluating the efficacy and safety of zilucoplan in adults with generalized myasthenia gravis.

Zilucoplan is a synthetic, macrocyclic peptide inhibitor of complement component 5 (C5) that binds to and inhibits the cleavage of C5 into C5a and C5b preventing 3 pathways of complement activation. The multicenter, randomized, double-blind, placebo-controlled RAISE study (ClinicalTrials.gov Identifier: NCT04115293) included 174 adults with gMG. Patients were randomly assigned 1:1 to receive zilucoplan or placebo subcutaneously once daily for 12 weeks. 

Findings showed that zilucoplan met the primary endpoint demonstrating a clinically meaningful and statistically significant improvement in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score at week 12 from baseline compared with placebo. Zilucoplan also met all key secondary endpoints, including statistically significant improvements in the Quantitative Myasthenia Gravis score, Myasthenia Gravis Composite score, and Myasthenia Gravis – Quality of Life revised score.

“gMG patients can experience varying and debilitating symptoms that impact their everyday lives in unique ways,” said James F. Howard, MD, Distinguished Professor of Neuromuscular Disease, Chief, Neuromuscular Disorders Section, University of North Carolina School of Medicine and lead investigator in the RAISE trial. “These exciting results give us additional reason to believe that zilucoplan can offer an important step forward in addressing the unmet needs of people living with gMG.”

The Food and Drug Administration (FDA) previously granted Orphan Drug designation to zilucoplan for generalized myasthenia gravis.

UCB is also developing rozanolixizumab, a monoclonal antibody, for the same indication. Results from the phase 3 MycarinG study (ClinicalTrials Identifier: NCT03971422) showed that treatment with rozanolixizumab was associated with a statistically significant and clinically meaningful change from baseline in the MG-ADL total score when compared with placebo.


UCB announces positive data in myasthenia gravis with zilucoplan phase 3 study results. News release. UCB, Inc. Accessed February 4, 2022. https://www.prnewswire.com/news-releases/ucb-announces-positive-data-in-myasthenia-gravis-with-zilucoplan-phase-3-study-results-301475451.html