AstraZeneca looking to add to small group of Wilson’s disease drugs

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An experiment Astra Zeneca medicine for Wilson’s disease achieve its goal in a phase III study in which the drug, ALXN1840, was able to achieve copper mobilization three times the standard of care.

In people with Wilson’s disease, excessive copper accumulation in organs and tissues caused by the loss of the protein ATP7B needed to transport copper can lead to liver disease, as well as neurological and psychiatric symptoms. It is estimated that approximately 9,000 people in the United States have been diagnosed with the disease.

There are only a handful of drugs approved by the FDA to treat this genetic condition, including Syprine, a drug that has been around since the 1960s, and the Cuvrior more recently approved, developed by the French company Orphalon. Based on data from the FoCus Phase III study, AstraZeneca hopes to join this small group of drugs approved by the US Food and Drug Administration.

In the Phase III study, which was billed as the largest global Wilson’s disease trial to date, ALXN1840 met its primary endpoint demonstrating three times the mobilization of copper at standard of care , including patients who have already been treated for at least 10 years. Even after standard treatment, some patients continue to see progression of their disease and worsening of neurological symptoms, AstraZeneca said.

Data from the trials showed that patients experienced copper mobilization, which is the filtering out of copper buildup, within four weeks and for 48 weeks, the company said. In patients who had symptoms of Wilson’s disease at baseline, treatment with ALXN1840 generated greater improvements in neurological scores than standard of care. These data were a secondary endpoint of the study. However, AstraZeneca noted that in this subgroup there were no significant differences between the treatment groups seen at 48 weeks.

AstraZeneca noted that most patients in the trial had low symptom scores at baseline, which it said indicated a minimal margin for improvement in the total score. Because people with Wilson’s disease have a widely varying degree of symptoms, this total score may not reflect the extent of disease severity, AstraZeneca said.

ALXN1840 was well tolerated in the FoCus study, and long-term safety and efficacy are being evaluated for an extension period of up to 60 months. AstraZeneca intends to present full Focus Phase III study data today at the 2022 International Liver Congress in London.

ALXN1840, which was developed by AstraZeneca’s rare disease subsidiary company Alexion, is an experimental molecule designed to bind and remove copper from the body. ALXN1840 has received orphan drug designation in the United States and orphan drug designation in the European Union for Wilson’s disease.

Marc Dunoyer, chief executive of Alexion, noted that many patients with Wilson’s disease may continue to experience symptoms even after years of using available therapies. He said this demonstrates a significant need for reassessment of the standard of care used to treat this genetic disease.

“Applying our 30 years of experience in rare disease clinical development, Alexion has conducted rigorous scientific research to bring new thinking to Wilson’s disease around the importance of copper mobilization from tissue. These data reinforce our efforts to potentially introduce a new treatment for patients who have gone decades without meaningful innovation,” Dunoyer said in a statement.

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