AstraZeneca PLC (LSE:AZN) announced that a phase III trial of its ALXN1840 medicine for treatment of Wilson disease has met its primary endpoint.
The FoCus Phase III trial of ALXN1840 showed a statistically significant improvement in daily mean copper mobilisation from tissues, demonstrating superiority compared with standard-of-care treatments, the company said.
Wilson disease is a rare and progressive genetic condition in which the body's ability to remove excess copper is compromised. Damage from toxic copper build-up in tissues and organs leads to liver disease, psychiatric and/or neurological symptoms.
ALXN1840, a potential new once-daily, oral medicine, demonstrated three times greater copper mobilisation than standard treatments, AstraZeneca said.
Marc Dunoyer, the chief executive of AstraZeneca’s rare disease subsidiary Alexion, said: "Where existing treatments remove copper from the blood, these 48-week Phase III results demonstrate ALXN1840's significant impact in mobilising copper from tissues.
“As we advance this first innovation in Wilson disease treatment in more than 30 years, we will continue to follow these patients long term to further assess clinical impact on disease symptoms. We look forward to learning more about how we can evolve the treatment of this progressive and devastating disease."
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