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AstraZeneca’s FoCus Phase III trial met primary endpoint

Marc Dunoyer

Positive results from the trial in Wilson disease showed that ALXN1840 met the primary endpoint with a statistically significant improvement in daily mean copper mobilization from tissues, demonstrating superiority compared with standard-of-care (SoC) treatments.

The primary endpoint gauged the daily mean Area Under the Effect Curve (AUEC) for directly measured non-ceruloplasmin-bound copper (dNCC) over 48 weeks. This novel measure assesses the daily mean copper mobilised from tissues, reflecting the underlying burden of the copper accumulation.


ALXN1840, a potential new once-daily, oral medicine, demonstrated approximately three times greater copper mobilisation than SoC. The trial enrolled 214 patients, including treatment-naïve participants and those who have been on SoC therapy for an average of ten or more years. Additional analyses, including individual patient-reported outcomes and clinician-reported functional assessments, are ongoing.

“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. We look forward to learning more about how we can evolve the treatment of this progressive and devastating disease,” says Marc Dunoyer, Chief Executive Officer, Alexion.

ALXN1840 was generally well-tolerated with most reported adverse events considered mild to moderate, and no neurological worsening upon initiation of treatment was observed. In the ALXN1840 treatment group, the most frequently reported adverse event was a reversible increase in transaminase levels.

Alexion is working closely with health authorities worldwide and intends to submit these data for review in the coming months.

Photo of Marc Dunoyer: AstraZeneca