Zilucoplan Improved Activities of Daily Living for Individuals with Myasthenia Gravis

The phase 3 clinical trial, RAISE (NCT04115293) of subcutaneous zilucoplan (RA101495; UCB, Atlanta, GA) demonstrated improvement in activities of daily living for adults with generalized myasthenia gravis (gMG). Participants treated with zilucoplan vs placebo had clinically meaningful and statistically significant improvement from baseline in their Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total scores at week 12. Scores on the Quantitative Myasthenia Gravis (QMG) score, Myasthenia Gravis Composite (MGC) score and MG-QoL15r score also improved from baseline. 

“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. 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.”

Zilucoplan was well-tolerated with no major unexpected safety findings. Participants treated with zilucoplan or placebo had similar rates of serious treatment emergent adverse events (TEAEs). The safety and efficacy of zilucoplan, a peptide inhibitor of complement component 5 (C5), have not been established nor approved for use in any indication by any regulatory authority worldwide.