ALXN1840 FoCus Phase III trial in Wilson disease hits goal

Positive high-level results from the FoCus Phase III trial in Wilson disease showed ALXN1840 met the primary endpoint with a statistically-significant improvement in daily mean copper mobilization from tissues, demonstrating superiority compared with standard-of-care (SoC) treatments, according to UK pharma major AstraZeneca’s Alexion rare disease unit that was created following the 2021 $39 billion acquisition of Alexion Pharmaceuticals.

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 mobilized 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.1