A drug that New Haven-born Alexion Pharmaceuticals acquired in an $855 million buyout of a Swedish biopharma three years ago could pay dividends for new Alexion parent AstraZeneca.
The UK-based pharma giant, which acquired the drug, ALXN1840, as part of its $39 billion Alexion takeover last month, said the experimental treatment aced a late-stage trial for patients with Wilson disease.
Wilson disease, a rare genetic condition, prevents the body from eliminating copper properly. As a result, copper accumulates in the brain and other organs and can lead to liver disease and psychiatric or neurological symptoms.
Trial data announced Thursday showed the drug removed three times as much excess copper from body tissues than standard of care treatments.
Based on the results, AstraZeneca said it plans to seek regulatory approval for the once-daily oral medicine “in the coming months.”
In a statement, Marc Dunoyer, Alexion’s new CEO since the merger, said the drug would be the first new treatment for the condition in more than 30 years.
“While existing treatments remove copper from the blood, these 48-week Phase 3 results demonstrate ALXN1840’s significant impact in mobilizing copper from tissues,” he said.
The trial enrolled 214 patients, including one group who had never been treated for the disease and another cohort who had been on the standard treatment for an average of 10 years or more.
Dr. Michael Schilsky, medical director of adult liver transplant at Yale-New Haven Transplantation Center and the trial’s principal investigator, said it was “encouraging” to see the drug’s effect on both sets of patients.
He said the results “provide evidence that tissue-bound copper remains built up in the organs even in patients who have been on [standard therapy] for many years” and that Alexion’s drug has the potential to provide a new approach to removing it.
Alexion obtained the drug through its acquisition of Wilson Therapeutics in 2018 under former CEO Ludwig Hantson, who at the time was pushing to diversify Alexion’s drug portfolio and lessen the company’s dependence on its blockbuster drug Soliris.
The good news came less than a week after AstraZeneca nixed a trial for Ultomiris, another Alexion drug, in patients with amyotrophic lateral sclerosis, or ALS, due to “lack of efficacy.”
Alexion employs more than 500 people at its New Haven research facility at 100 College St.
Contact Natalie Missakian at news@newhavenbiz.com
