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ALXN 1840 shows rapid and sustained improvement in copper mobilisation from tissues, potentially closing treatment gaps for Wilson disease community.

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Published:24th Jun 2022

Detailed results from the positive FoCus Phase III trial in Wilson disease showed that ALXN 1840, an investigational once-daily, oral medicine, met its primary endpoint demonstrating three-times greater copper mobilisation 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 ALXN 1840 experienced rapid copper mobilisation, with a response at four weeks and sustained through 48 weeks. Results from the trial will be presented on 23 June at the 2022 International Liver Congress (ILC) in London.

Wilson disease is a rare and progressive genetic condition in which the body’s pathway for removing excess copper is compromised. This may result in the accumulation of copper in a person’s liver, brain or other vital organs. Damage from excess copper build-up in tissues and organs may lead to symptoms of liver, neurological and psychiatric diseases, which may be irreversible. Even after SoC treatment is initiated, some patients experience worsening of disease, especially of neurologic symptoms. Change in neurological scale scores and clinician-reported functional assessments with ALXN 1840 treatment were also evaluated in a post-hoc analysis as secondary endpoints in the Phase III trial.

In patients who were symptomatic at baseline, there were greater improvements in neurological scores for those treated with ALXN 1840 compared to SoC (Unified Wilson Disease Rating Scale [UWDRS] part II symptomatic ALXN 1840 -1.7, SoC -0.8; UWDRS Part III symptomatic ALXN 1840 -2.91, SoC -1.31). However, there were no significant differences between treatment groups observed at 48 weeks.

Most patients in the trial had low symptom scores at baseline, so there was minimal room for total score improvement (UWDRS Part II ALXN 1840 -0.6, SoC -0.3; UWDRS Part III ALXN 1840 -2.20, SoC -1.02). As people with Wilson disease experience a highly varied degree of symptoms, this total score may not reflect the extent of disease severity. ALXN 1840 was well tolerated and the long-term safety and efficacy of ALXN 1840 is being assessed in an up to 60-month extension period.

Professor Karl Heinz Weiss, MD, Director of the Department of Internal Medicine at Salem Medical Centre Heidelberg and investigator in the FoCus Phase III trial, said: “These data from the largest global trial in Wilson disease to date show significant copper mobilisation from the tissues with ALXN 1840, even in patients who were on standard of care for over a decade on average. These results have the potential to reframe the way doctors can think about the disease given that current therapies focus on removing copper from the blood. We are also encouraged by initial neurological improvement with ALXN 1840 in those who were symptomatic and believe that assessing individual patient experiences may provide a better understanding of the impact on daily life.”

Condition: Wilson disease
Type: drug

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