
Zilucoplan Demonstrates Consistent Efficacy in Phase 3 RAISE Study of Generalized Myasthenia Gravis
Nearly half of patients treated with zilucoplan were considered responders on Myasthenia Gravis Activities of Daily Living (MG-ADL) at the first week, suggesting a rapid onset of efficacy.
A version of
In a recent analysis of
Among 174 patients with acetylcholine receptor antibody–positive generalized myasthenia gravis randomized to zilucoplan (n = 86) or placebo (n = 88), responder rates for the MG-ADL (73% vs 46%; P <.001) and Quantitative Myasthenia Gravis (QMG) score (58% vs 33%; P = .0012) were significantly higher for the zilucoplan group vs placebo at week 12. Notably at 1 week, 45% and 32% of zilucoplan-treated patients met the MG-ADL and QMG responder criteria, respectively, vs 30% and 8% of those on placebo. There were no meaningful differences observed in baseline disease characteristics of MG-ADL responders and nonresponders at week 12.
The data were presented at the
In November 2022, the
Presented at the
RAISE-XT, an ongoing study, featured 199 patients with MG who participated
At 24 months, 84.9% (n = 169) experienced a TEAE, and 23.1% (n = 46) documented serious AEs. Between both groups, the most common TEAEs were headache and worsening of MG, both occurring in 16.6% of patients. Throughout the study, 4 treatment-emergent deaths occurred, all in patients with multiple
MG-ADL rapidly improved throughout the OLE period among those who switched from placebo to zilucoplan. Similarly, the zilucoplan group demonstrated significant improvements on MG-ADL during the OLE (P = .0002). From the double-blind study baseline, zilucoplan-treated individuals achieved least square mean changes in MG-ADL score of –6.30 (95% CI, –7.44 to –5.15). These results were similar for the placebo-switch group, with score reductions of –6.32 (95% CI, –8.00 to –4.65). Scores on key secondary outcomes such as QMG score, MGC, and Myasthenia Gravis Quality of Life 15-item-revised were similar across both treatment groups as well.
References
1. Mantegazza R, Bresch S, Freimer M. Response to zilucoplan in the phase 3 RAISE study in patients with generalised myasthenia gravis. Presented at: EAN 2023; July 1-4, 2023; Budapest, Hungary. Abstract EPR-132.
2. UCB presents efficacy and safety results for zilucoplan and rozanolixizumab in generalized myasthenia gravis. News release. UCB Pharma. May 10, 2022. Accessed July 11, 2023. https://www.ucb-usa.com/stories-media/UCB-U-S-News/detail/article/ucb-presents-efficacy-and-safety-results-for-zilucoplan-and-rozanolixizumad-in-generalized-myasthenia-gravis
3. Genge A, Hussain Y, Kaminski HJ, et al. Safety and tolerability of zilucoplan in RAISE-XT: a multicenter, open-label extension study in patients with myasthenia gravis. Presented at: MDA 2023; February 19-22, 2023; Dallas, Texas. Abstract 145.
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