AstraZeneca's Drug Shows Promise In Copper Build-Up Genetic Disease

AstraZeneca plc AZN reported high-level results from the FoCus Phase 3 trial evaluating ALXN1840 in Wilson disease.
The study met the primary goal of improving the daily mean copper mobilized from tissues over 48 weeks. Wilson disease is a genetic condition in which the body's ability to remove excess copper is compromised.
The late-stage trial showed ALXN1840 was three times more successful in helping remove deposits from tissues compared with standard care. The drug candidate was added to the AZN pipeline via the recent acquisition of Alexion.
The experimental oral medicine taken once daily could provide a less invasive option for patients typically given treatments that involve injecting a metal removing solution into the blood or zinc therapy.
Copper build-up can cause liver disease and psychiatric or neurological symptoms, such as personality changes, tremors, and difficulty walking, swallowing, or talking.
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