Zilucoplan Improvements Sustained for Generalized Myasthenia Gravis

In the RAISE study (NCT04115293) and RAISE XT open-label extension (NCT04225871), participants with generalized myasthenia gravis (gMG) treated with zilucoplan (RA101495; UCB, Atlanta, GA) continued to have significantly improved scores on the Myasthenia Gravis Activities of Daily Living (MG-ADL).

The double-blind RAISE study and the open-label RAISE-XT extension demonstrated favorable long-term safety results for zilucoplan for 12 and 24 weeks. Participants who previously received zilucoplan sustained improvement with lower MG-ADL scores (-6.30).Participants who switched from placebo to zilucoplan in the extension demonstrated improvements as early as week 1 and at 12 weeks had lower MG-ADL scores (-6.32) as well. 
 
Most participants in the RAISE trial who received zilucoplan achieved 3-point and 5-point improvements in MG-ADL and Quantitative Myasthenia Gravis (QMG) without rescue therapy vs placebo. Participants treated with zilucoplan demonstrated a clinically meaningful placebo-corrected mean improvement of 2.09 points (P<.001) in MG-ADL. Of those who took zilucoplan for all 24 weeks, 73.1% (P<.001) achieved a 3.0-point or more reduction in MG-ADL compared with 46.1% of those who receive placebo for the first 12 weeks. More than half of participants (58%) [P=.0012]) who received zilucoplan for 24 weeks achieved a 5.0-point or more reduction in QMG vs one-third (33.1%) who received placebo and switched to zilucoplan. 

The most common TEAEs were injection-site reactions (26.7% zilucoplan vs 14.8% placebo); all were nonserious, and mild in severity, except for 1 instance of injection-site pain of moderate severity in the zilucoplan group, and no participants discontinued due to an injection-site reaction.