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

Phase 2 Terminated 29 enrolled Alexion Pharmaceuticals, Inc.

Non-randomizedSingle-groupOpen-labelTreatment

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

Outcome

Met primary endpoint

Stopped: “Did not appropriately control IVH; significantly increased rates of BTH and LDH excursions (LDH excursions is defined by LDH values \> X2ULN) compared to ravulizumab.”

paper This trial evaluating vemircopan monotherapy met its primary efficacy endpoint; PMID 41124654 ↗

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

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