Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy

Summary

The study will evaluate the efficacy and safety of the oral Factor D (FD) inhibitor ALXN2050 (ACH-0145228) monotherapy in patients with PNH that are treatment naïve, or patients currently treated with eculizumab who still experience anemia and reticulocytosis, or patients currently treated with ALXN2040 (danicopan) as monotherapy. After signing consent, participants will have periodic visits through Week 12, at which time the primary endpoint and key secondary assessments will be analyzed. Participants will continue on treatment past 12 weeks into a long-term extension portion of the trial.

Timeline

Start

2019-12-16

Primary completion

2023-04-27

Completion

2024-03-20

Publications

• Kulasekararaj A, Browett P, Risitano AM, Patriquin CJ, Yenerel MN, Marceau D, Sahin F, Algarra JL, Ogawa M, Yu J, Cross NB, Notaro R, Lee JW, Brodsky RA. Efficacy and safety of vemircopan as monotherapy in patients with paroxysmal nocturnal hemoglobinuria. Blood Adv. 2026 Feb 10;10(3):540-554. doi: 10.1182/bloodadvances.2025017731.

Drugs

Indications