Oral Factor D Inhibition Falls Short in AChR-Positive Generalized Myasthenia Gravis: What the Vemircopan Phase 2 Trial Means for Complement-Targeted Therapy
Highlights
In this phase 2, double-blind randomized clinical trial, oral vemircopan, a selective factor D inhibitor, did not outperform placebo in acetylcholine receptor antibody-positive generalized myasthenia gravis.
The primary responder endpoint was numerically similar across groups: 64% with placebo, 57% with vemircopan 180 mg twice daily, and 57% with vemircopan 120 mg twice daily.
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