ALXN1840 shows promise in potentially closing treatment gaps for Wilson disease community

Detailed results from the positive FoCus Phase III trial in Wilson disease showed that ALXN1840, an investigational once-daily, oral medicine, met its primary endpoint demonstrating three-times greater copper mobilization from tissues compared to the standard of care (SoC) arm (Least Square Mean Difference [LSM Diff] 2.18 µmol/L; p< 0.0001), 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.

Results from the trial were presented today at the 2022 International Liver Congress (ILC) in London.