AstraZeneca's ALXN1840 Shows Promising Action Against Copper Metabolism Disorder

AstraZeneca Plc AZN announced detailed results from the positive FoCus Phase 3 trial of ALXN1840 in Wilson's disease, an inherited condition in which the body's pathway for removing excess copper is compromised.
The data showed that ALXN1840 met its primary endpoint demonstrating three-times greater copper mobilization from tissues compared to the standard of care (SoC) arm, including in patients who had been treated previously for an average of 10 years.
In the trial, people taking ALXN1840 experienced rapid copper mobilization, with a response at four weeks and sustained through 48 weeks.
In patients who were symptomatic at baseline, there were greater improvements in neurological scores for those treated with ALXN1840 compared to SoC. However, there were no significant differences between treatment groups observed at 48 weeks.1
Most trial patients had low baseline symptom scores, so there was minimal room for total score improvement. As people with Wilson's disease experience a highly varied degree of symptoms, this total score may not reflect the extent of disease severity.
ALXN1840 was well tolerated, and the long-term safety and efficacy of ALXN1840 are being assessed in an up to 60-month extension period.
Price Action: AZN shares are up 1.19% at $64.31 during the market session on the last check Thursday.