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AAN 2023 | Advancing myasthenia gravis treatment: RAISE trial of zilucoplan

James F. Howard, Jr., MD, FAAN, University of North Carolina School of Medicine, Chapel Hill, NC, presents findings on RAISE, a randomized, Phase III trial (NCT04115293) of zilucoplan in generalized myasthenia gravis (MG) patients. Zilucoplan is a complement inhibitor that specifically targets C5, but it possesses unique characteristics that set it apart from other inhibitors. Unlike full-size antibodies, zilucoplan is a macrocyclic 14 amino acid peptide, allowing it to adopt binding conformations that enhance its efficacy in targeting C5. It also targets C5b, providing a second chance to capture any “escaped” molecules from the terminal complement cascade. Notably, zilucoplan is administered through daily subcutaneous injections, which are rapid and involve a small volume of slightly more than half a cubic centimeter of an aqueous solution. Patients with MG have reported good tolerability with this delivery method. The efficacy of zilucoplan has been demonstrated in the RAISE trial, showing rapid, robust, and sustained responses. These positive outcomes were observed not only during the 12-week blinded trial but also in the subsequent open-label extension. Additionally, zilucoplan’s unique characteristic of being stable at room temperature allows patients to self-administer it at home, eliminating the need for visits to infusion centers. It can also be easily transported, enabling patients to travel without concerns about refrigeration. Prof. Howard, Jr. highlights the affordability of zilucoplan as another advantage, making it accessible to a wider patient population. These results offer new possibilities for patients with MG with improved convenience, effectiveness, and quality of life. This interview took place at the American Academy of Neurology Annual Meeting 2023 in Boston, MA.

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Research support paid to institution: Alexion Pharmaceuticals, Argenx, Cartesian Therapeutics, The Centers for Disease Control and Prevention, Millennium/Takeda Pharmaceuticals, National Institutes of Health, PCORI, Ra Pharmaceuticals (now UCB)
Honoraria: Alexion Pharmaceuticals, Argenx, Biologix Pharma, Ra Pharmaceuticals (now UCB), F. Hoffmann-LaRoche Ltd, Immunovant, Merck EMB Serono, NMD Pharma, Novartis Pharma, Regeneron Pharmaceuticals, Sanofi US, Toleranzia AB, Horizon Therapeutics, Zai Labs
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