Positive Phase 3 Topline Results Announced for Zilucoplan in Generalized Myasthenia Gravis


In the RAISE trial, patients treated with zilucoplan exhibited clinically meaningful and statistically significant improvement from baseline in Myasthenia Gravis-Activities of Daily Living Profile total score at week 12, when compared with those who received placebo.

James F. Howard, MD

James F. Howard, MD

Positive topline results were announced by UCB for the phase 3 RAISE trial (NCT04115293) of zilucoplan, a self-administered, subcutaneous peptide inhibitor of complement component 5, in patients with generalized myasthenia gravis (gMG). The trial met its primary end point in demonstrating a clinically meaningful and statistically significant improvement in Myasthenia Gravis-Activities of Daily Living Profile total score from baseline to week 12 for those treated with zilucoplan vs placebo.1

The trial also met all secondary end points, including statistically significant improvements from baseline in Quantitative Myasthenia Gravis score, Myasthenia Gravis Composite score, and Myasthenia Gravis Quality of Life 15-item Scale score for those treated with zilucoplan, compared with those treated with placebo. Considering the positive results, UCB anticipates progressing with regulatory filing for the treatment in gMG in the US, Europe, and Japan later in 2022.

“gMG patients can experience varying and debilitating symptoms that impact their everyday lives in unique ways,” lead trial investigatorJames F. Howard, MD, Distinguished Professor of Neuromuscular Disease, chief, Neuromuscular Disorders Section, University of North Carolina School of Medicine, said in a statement.1 “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. As we strive to improve the management of this complex and unpredictable disease, any new medicines will be welcomed by physicians to help us realize our goal of offering effective and flexible treatment approaches in gMG which are tailored to the needs of individual patients.”

There were no unexpected safety findings for zilucoplan, and the treatment was well-tolerated, in line with previous studies of zilucoplan. Between the zilucoplan group and the placebo group, similar incidence of serious treatment emergent adverse events was observed.

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The multicenter, randomized, double-blind, placebo-controlled study evaluated zilucoplan in approximately 174 adults with gMG. Patients were randomized 1:1 to receive subcutaneous doses of zilucoplan or placebo over the course of 12 weeks. In addition to determining clinical benefit, investigators sought to determine if complement inhibition was effective across patients with acetylcholine receptor antibody positive MG, regardless of prior treatment or response to other previous therapies or disease duration.

The results follow an additional announcement from UCB about positive topline data from the phase 3 MycarinG study (NCT03971422), which investigated the subcutaneous-infused monoclonal antibody, rozanolixizumab, targeting the neonatal Fc receptor in patients with gMG. The treatment is being developed for the same indication as zilucoplan, with the trial meeting both its primary and secondary end points with statistical significance.2

“Today’s results represent another significant milestone in UCB’s efforts to bring transformational outcomes to those living with myasthenia gravis. Positive results for zilucoplan and rozanolixizumab—each with a different mechanism of action—bring us one step closer to achieving our ambition of delivering choice and flexibility for a broad range of patients and physicians at each step of their treatment journey, addressing significant unmet needs and offering unique patient value,” Iris Loew-Friedrich, executive vice president and chief medical officer, UCB, said in a statement.1 “We thank the MG community for their continued insights, partnership and participation in this study.”

1. UCB announces positive data in myasthenia gravis with zilucoplan phase3 study results. News release. UCB. February 4, 2022. Accessed February 4, 2022. https://www.ucb-usa.com/stories-media/UCB-U-S-News/detail/article/ucb-announces-positive-data-in-myasthenia-gravis-with-zilucoplan-phase-3-study-results
2. UCB announces positive phase 3 results for rozanolixizumab in generalized myasthenia gravis. News release. UCB. December 10, 2021. Accessed Februrary 4, 2022. https://www.ucb-usa.com/stories-media/UCB-U-S-News/detail/article/ucb-announces-positive-phase-3-results-rozanolixizumab
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