AstraZeneca division Alexion has reported that its drug, ALXN1840, met the primary goal in the Phase III FoCus clinical trial in patients with Wilson disease.

A rare and progressive genetic condition, Wilson disease occurs when the body’s pathway that removes additional copper is compromised.

ALXN1840 is a once-daily oral drug that can selectively attach to and eliminate copper from the body’s tissues and blood.

The pivotal, randomised, rater-blinded, multi-centre Phase III trial assessed the efficacy and safety of ALXN1840 versus standard-of-care (SoC) therapy in Wilson disease patients aged 12 years or above.

SoC included chelation treatment with penicillamine or trientine, zinc therapy or chelation along with zinc therapy.

FoCus enrolled a total of 214 subjects, comprising treatment-naïve patients and those who received SoC treatment for an average of ten years or more.

The primary goal evaluated copper mobilisation, assessed by the daily mean area under the effect curve (AUEC) for directly measured non-ceruloplasmin-bound copper (dNCC) over 48 weeks.

Positive high-level data from the trial showed that ALXN1840 offered a statistically significant improvement in daily mean copper mobilisation from tissues, indicating superiority over SoC.