Study of ALXN2050 in Adult Participants With Generalized Myasthenia Gravis

Phase 2 Terminated 70 enrolled Alexion Pharmaceuticals, Inc.

RandomizedParallel-groupTriple-blindTreatment

Summary

This study will evaluate the efficacy and safety of ALXN2050 (120 milligrams \[mg\], 180 mg) in participants with generalized myasthenia gravis (gMG). Safety will be monitored throughout the study.

Timeline

Start: 2022-04-27
Primary completion: 2023-11-30
Completion: 2024-04-03

Outcome

Missed primary endpoint

Stopped (Business): “Sponsor decision”

registry analysis (other test); Group 1: ALXN2050 180 mg BID vs Group 3: Placebo; p = 0.6797; Difference -7.1 (90% CI -28.8 to 15.0); Barnard's Unconditional Exact Test NCT05218096 ↗

paper The proportion of participants achieving the primary end point did not differ significantly between placebo (18 of 28 [64%]; 90% CI, 47%-79%) and either vemircopan group PMID 42043793 ↗

Publications

Sacca F, Gialdini G, Howard JF Jr, Vu TH, Meisel A, Peric S, Lunemann JD, Sgarzi M, Shaibani A, Bril V, Yeh JH, Reyes-Garrido V, Liao S, Metais C, van der Valk RJP; ALXN2050-MG-201 Investigators. Efficacy and Safety of Vemircopan in Generalized Myasthenia Gravis: A Randomized Clinical Trial. JAMA Neurol. 2026 Jun 1;83(6):544-553. doi: 10.1001/jamaneurol.2026.0902.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Vemircopan	Small molecule	120 mg	Oral
Subject	Vemircopan	Small molecule	180 mg	Oral