RARE Daily

AstraZeneca Discontinues Wilson Disease Program, Takes $244 Million Write-Down

April 27, 2023

Rare Daily Staff

AstraZeneca said it discontinued development of its Alexion rare disease program ALXN1840 for Wilson disease and it will take a $244 million impairment charge.

The decision followed feedback from regulators following review of the data from the Wilson disease program, the company said including the phase 3 FoCus and phase 2 mechanistic trials despite the therapy reaching its endpoint in the phase 3 study. AstraZeneca acquired the program as part of its $39 billion acquisition of Alexion.

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. Although the disease is present at birth, the age of diagnosis occurs between five to 35 years. 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 standard-of-care treatment is initiated, some patients experience worsening of disease, especially of neurologic symptoms.

ALXN1840 is a potential new once-daily, oral medicine in development for the treatment of Wilson disease that is designed to selectively and tightly bind to and remove copper from the body’s tissues and blood.

On an earnings call with analysts, Marc Dunoyer, CEO of Alexion, AstraZeneca Rare Disease said that while the phase 3 study produced positive results, two subsequent mechanistic studies provided data that was less clear. “When you look at the totality of data, it was not possible for us to demonstrate clearly benefit/risk for the Wilson population and then we decided to discontinue it,” he said.

Photo: Marc Dunoyer is Chief Executive Officer of Alexion, AstraZeneca Rare Disease

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