ALXN 1840 demonstrated approximately three times greater copper mobilisation from tissues

The primary endpoint gauged the daily mean Area Under the Effect Curve (AUEC) for directly measured non-ceruloplasmin-bound copper (dNCC) over 48 weeks. This novel measure assesses the daily mean copper mobilised from tissues, reflecting the underlying burden of the copper accumulation.

Wilson disease is a rare and progressive genetic condition in which the body’s pathway for removing excess copper is compromised. Damage from toxic copper build-up in tissues and organs leads to liver disease, psychiatric and/or neurological symptoms. ALXN 1840, a potential new once-daily, oral medicine, demonstrated approximately three times greater copper mobilisation than SoC. The trial enrolled 214 patients, including treatment-naïve participants and those who have been on SoC therapy for an average of ten or more years. Additional analyses, including individual patient-reported outcomes and clinician-reported functional assessments, are ongoing and will be presented at an upcoming medical meeting.