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AstraZeneca PLC on Thursday reported strong results from phase III clinical trials for ALXN1840.
The Cambridge-based pharmaceutical company said the FoCus phase III trial of its once-daily, investigational oral medicine ALXN1840 met its primary endpoint in the treatment of Wilson disease.
Wilson disease is an inherited condition which results in excess copper levels in the liver, brain and other organs. This leads to damage which can greatly impact those with the disease.
ALXN1840 showed copper mobilisation from tissues three-times greater than the standard of care arm.
Patients experienced rapid copper mobilisation, with a response at four weeks, which was sustained through 48 weeks.
ALXN1840 is a novel molecule designed to bind to copper and remove it from the body's tissue and blood.
Shares in AZ were up 0.6% to 10,368.00 pence each in London on Thursday afternoon.
Two mechanistic trials in Wilson disease are underway, and AZ's rare disease group Alexion is working with global health authorities to submit the data for review.
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