This Phase III study aims to evaluate the efficacy and safety of iptacopan (LNP023) in patients with anti-acetylcholine receptor antibody-positive (AChR+) generalized myasthenia gravis (gMG) on stable standard of care (SOC) treatment.

gMG is a rare, chronic autoimmune disorder caused by autoantibodies against neuromuscular junction components, leading to debilitating fatigable muscle weakness and other clinical consequences. Current therapies for the treatment of gMG have limitations, including infections, systemic side effects, and often require parenteral administration. Iptacopan (LNP023) is an oral, potent, and selective inhibitor of factor B in the alternative complement pathway, currently in clinical development for the treatment of gMG.

This ongoing randomized, double-blind, placebo-controlled, multicenter Phase III study (NCT06517758) is enrolling patients aged 18-75 years with Class II-IV MG (according to the Myasthenia Gravis Foundation of America) who test positive for AChR+ antibodies and are on SOC treatment. Participants must have a baseline MG-ADL score ≥6, with ≥50% due to non-ocular symptoms, and inadequate disease control on one or more non-steroidal immunosuppressant treatments (NSIST) or frequent plasmapheresis/plasma exchange/IVIG therapy. Eligible participants will be randomized (1:1) to receive either iptacopan or placebo during the 6-month double-blind core period, followed by iptacopan administration in the  open-label extension (up to 60 months). The primary endpoint is the change in Myasthenia Gravis Activity of Daily Living (MG-ADL) score from baseline to Month 6. Key secondary endpoints include assessments of Quantitative MG (QMG), and MG Composite (MGC), among others.

The study will enroll approximately 146 eligible participants.

This study will investigate the efficacy and safety of iptacopan versus placebo in adult patients with AChR+ gMG.