James F. Howard, Jr., MD, The University of North Carolina at Chapel Hill, discusses the interim analysis of the RAISE-XT extension study for myasthenia gravis (MG).
MG is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. The condition results from a defect in the transmission of nerve impulses to muscles, which is due to the presence of antibodies against the acetylcholine receptor. The exact reason this occurs is not known.
The RAISE-XT extension study is an ongoing, multicenter, open-label extension of a phase 3 study evaluating the long-term safety, tolerability, and efficacy of zilucoplan in patients with AChR+ generalized MG. Zilucoplan is a C5 complement inhibitor approved by the U.S. Food and Drug Administration (FDA) for the treatment of MG.
The 200 patients enrolled in the open-label extension and received daily, self-administered subcutaneous 0.3 mg/kg of zilucoplan. The primary endpoint was incidence of treatment-emergent adverse events (TEAEs) and secondary endpoints included change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score.
94% of patients experienced a TEAE, the most common being MG worsening and COVID-19. However, improvements to MG-ADL scores were observed in those who received zilucoplan in the parent study and those who switched from placebo in the parent study to zilucoplan in the extension. Overall, the treatment demonstrated a favorable long-term safety profile and tolerability as well as sustained efficacy.
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To learn more about MG and other rare autoimmune conditions, visit https://checkrare.com/diseases/autoimmune-auto-inflammatory-disorders/