Monopar Therapeutics presents ALXN1840 data at EASL

Monopar Therapeutics (MNPR) is presenting data on the long-term efficacy and safety of its ALXN1840 drug candidate for Wilson disease at the European Association for the Study of the Liver International Liver Congress 2025. The poster supports the potential use of ALXN1840 as a therapeutic option for Wilson disease, a rare and progressive genetic condition in which the body’s pathway for removing excess copper is compromised, leading to damage from toxic copper build-up in tissues and organs such as the liver and brain. Efficacy data were pooled and analyzed from three clinical trials: Phase 2 WTX101-201, Phase 2 ALXN1840-WD-205, and Phase 3 WTX101-301. For safety analysis, data from the Phase 2 ALXN1840-WD-204 trial were also included. The median treatment duration with ALXN1840 was 961 days and 943.5 days for the efficacy and safety datasets, respectively. The data presented highlight the following: sustained improvements from baseline in the Unified Wilson Disease Rating Scale Part II and Part III; increased copper mobilization as evidenced by a sustained increase in dNCC; improvements on the Clinical Global Impression – Improvement scale for ALXN1840 compared to standard of care; improvement in the New Wilson Index for patients treated with ALXN1840; higher patient-reported convenience and effectiveness of ALXN1840 compared to standard of care, including those who transitioned from standard of care to ALXN1840 in the extension portion of the Phase 3 clinical trial and fewer than 5% of patients experienced a drug-related serious adverse event, with no cases of a drug-related renal or urinary system SAE.