AstraZeneca scores late-stage study success for Wilson disease drug

AstraZeneca's Alexion unit announced Thursday that a Phase III study of the experimental oral drug ALXN1840 in patients with Wilson disease met its primary endpoint, demonstrating a significant improvement in daily mean copper mobilisation from tissues versus standard-of-care treatments. The company said it plans to submit the data to regulators in the coming months.

The FoCus trial randomised 214 patients with Wilson disease aged 12 years and older to receive once-daily ALXN1840 or standard-of-care treatment, which consisted of chelation therapy with penicillamine or trientine, zinc therapy or a combination of both chelation and zinc therapy. Alexion noted that the study included 161 treatment-experienced patients and 53 treatment-naïve subjects.

3x greater copper mobilisation

According to top-line results, ALXN1840 demonstrated approximately three-times greater copper mobilisation from tissues than standard-of-care treatment as assessed by the daily mean Area Under the Effect Curve (AUEC) for directly measured non-ceruloplasmin-bound copper over 48 weeks. "Where existing treatments remove copper from the blood, these…results demonstrate ALXN1840's significant impact in mobilising copper from tissues," commented Marc Dunoyer, chief executive of Alexion.

The drugmaker said ALXN1840 was generally well tolerated in the study, with most reported adverse events considered mild-to-moderate in nature, while no neurological worsening upon initiation of treatment was observed. Alexion added that further analyses, including individual patient-reported outcomes and clinician-reported functional assessments, are ongoing and will be presented at an upcoming medical meeting.

Follows Ultomiris flop

Alexion acquired the drug, formerly known as WTX101, through its purchase of Wilson Therapeutics in 2018 for $855 million. Meanwhile, AstraZeneca completed its $39-billion takeover of Alexion last month and has so far reported late-stage results from one other drug gained through the deal. Last week, the company said that a Phase III study of the long-acting C5 complement inhibitor Ultomiris (ravulizumab) in adults with amyotrophic lateral sclerosis would be discontinued due to a lack of efficacy.

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