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.

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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.

Furthermore, the drug provided a nearly three-fold increase in copper mobilisation versus SoC.

ALXN1840 was found to be generally well-tolerated with most adverse events reported to be mild to moderate in severity.

No neurological worsening associated with the treatment was noted in the trial, Alexion added.

Further analyses, which include specific patient-reported outcomes and clinician-reported functional evaluations, are underway.

Alexion CEO Marc Dunoyer said: “Where existing treatments remove copper from the blood, these 48-week Phase III results demonstrate ALXN1840’s significant impact in mobilising copper from tissues.

“As we advance this first innovation in Wilson disease treatment in more than 30 years, we will continue to follow these patients long term to further assess clinical impact on disease symptoms.”

The company is collaborating with health agencies globally and plans to submit these results for review soon.

Last week, Alexion announced plans to discontinue the international Phase III CHAMPION-ALS trial of its drug, Ultomiris (ravulizumab), for the treatment of amyotrophic lateral sclerosis.