AstraZeneca’s Wilson Disease Drug Boasts Positive Results in Phase 3 Study

AstraZeneca (AZ) said that its once-daily oral investigational drug, ALXN1840, performed well in phase 3 clinical trial for patients with Wilson disease, a rare, genetic condition, removing excess copper from tissues approximately three times more effectively than standard of care treatments. 

The progressive disease affects the body’s ability to remove excess copper, leading to damage from toxic copper build-up in tissues and organs, liver disease, psychiatric and/or neurological symptoms.

The trial enrolled 214 patients, including treatment-naïve participants and those who have been on the standard of care therapy for an average of 10 or more years. AZ said its analysis is ongoing and that results will be presented at an upcoming medical meeting.

The company gained access to ALXN1840 in its $39 billion purchase of Boston, Mass.-based Alexion Pharmaceuticals.