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AstraZeneca reports study success for rare disease drug acquired in Alexion deal

Should detailed study results bear out ALXN1480’s benefit and lead to an FDA approval, the experimental drug could become an important new option for people with Wilson disease.

“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,” said Alexion CEO Marc Dunoyer, who served as AstraZeneca’s CFO until a leadership reshuffle during the acquisition’s closing.

While other therapies remove copper from the blood, the new study results show ALXN1840 can clear copper from body tissue, a significant finding, he said. Detailed data supporting that claim aren’t yet available, but will be presented at an upcoming medical meeting, according to AstraZeneca.

There are few options for treatment of Wilson disease. Existing ones have been in the spotlight in recent years for sharp price hikes. In 2016, a U.S. Senate committee took the drugmaker Valeant to task for raising the prices of medicines including Syprine and Cuprimine for Wilson disease. The cost of a supply of Syprine had shot up to $21,627 from $652.

An eagerly awaited generic version of Syprine, meanwhile, did little to fix the problem for patients, as the New York Times reported in 2018. The cost for the new generic was still 28 times the price of Syprine in 2010, before Valeant’s price hikes.

AstraZeneca tested its new option, which is taken orally once a day, in 214 patients, about a quarter of whom had never been treated for the disease before. Patients who didn’t receive the experimental drug got Syprine, Cuprimine, zinc therapy or a combination of those medicines.

After 48 weeks, ALXN1840 showed a clear benefit in clearing copper from body tissue, AstraZeneca said. Trial participants then had the option to continue on for another five years as researchers evaluate long-term safety and efficacy for the drug. The company said the experimental medicine was generally well tolerated by patients, with most side effects classified as mild to moderate.