ALXN-1840 for Wilson Disease

Monopar’s late-breaker poster presentation is available at the following link: https://www.monopartx.com/pipeline/ALXN 1840/EASL-poster-may-2025.

The poster supports the potential use of ALXN 1840 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 II WTX101-201, Phase II ALXN1840-WD-205, and Phase III WTX101-301 (n=255). For safety analysis, data from the Phase II ALXN1840-WD-204 trial were also included (n=266). The median treatment duration with ALXN 1840 was 961 days (2.63 years) and 943.5 days (2.58 years) for the efficacy and safety datasets, respectively

. The data presented highlight the following:  i. Sustained improvements from baseline in the Unified Wilson Disease Rating Scale (“UWDRS”) Part II (patient-reported symptoms) and Part III (clinician-assessed symptoms) ii. Increased copper mobilization as evidenced by a sustained increase in dNCC (directly measured non-ceruloplasmin-bound copper); iii. Improvements on the Clinical Global Impression –iv. Improvement (“CGI-I”) scale for ALXN 1840 compared to standard of care; v . Improvement in the New Wilson Index (based on bilirubin, AST, INR, leukocytes, and albumin) for patients treated with ALXN 1840; . vi. Higher patient-reported convenience and effectiveness of ALXN 1840 compared to standard of care, including those who transitioned from standard of care to ALXN 1840 in the extension portion of the Phase III clinical trial; and vii. Fewer than 5% of patients experienced a drug-related serious adverse event (“SAE”), with no cases of a drug-related renal or urinary system SAE. 

“These data show that the long-term efficacy, safety, and convenience profile of ALXN1840 are very encouraging and that ALXN1840 has the potential to provide a meaningful benefit to Wilson disease patients’ daily lives,” said Dr. Karl Weiss, Medical Director of Salem Medical Center Heidelberg, and lead author of the presentation at EASL.