AZ’s new rare disease unit wins in Wilson disease

AstraZeneca’s new rare disease unit, formed through the $39bn purchase of Alexion Pharmaceuticals, had announced positive results from a phase 3 trial in Wilson disease.

Their candidate, ALXN1840, met its primary endpoint, removing three times more of the dangerous copper that builds up in the tissues of those with Wilson disease than standard-of-care treatment.

Wilson disease is a rare, progressive genetic condition in which the body’s pathway for removing excess copper is compromised. The resulting build-up of copper causes damage to tissues and organs, and leads to liver disease, psychiatric and/or neurological symptoms. The disease affects one in 30,000 live births in the US.

The data comes from the FoCus phase 3 trial, which tested ALXN1840 against the current standard of care for (SoC) patients with the rare condition, chelation and/or zinc therapy.

“It is encouraging to see the effect of ALXN1840 on both treatment-naïve patients and those who have been on SoC for an average of ten or more years,” said Yale University’s Michael Schilsky and principal investigator of the FoCus trial. “The phase 3 results provide evidence that tissue-bound copper remains built up in the organs even in patients who have been on SoC therapy for many years, and the potential for ALXN1840 to provide a new approach to mobilise and safely sequester copper from tissues.”

Alexion will work with health authorities worldwide and intends to submit the data for review in the coming months.

ALXN1840 was first discovered at Stockholm-based Wilson Therapeutics, a company named after the genetic disorder it sought to address, which was acquired by Alexion for $855m in 2018.

It is designed to be the first targeted de-coppering therapy that selectively and tightly binds to, and removes, copper from the body’s tissues and blood. The compound has orphan drug designation in the US and EU for the disease.

The FoCus trial saw 214 patients receive either ALXN1840 or SoC, including both treatment-naïve participants and those who have been on SoC therapy for an average of ten or more years.

Alexion/AstraZeneca Rare Disease is focused on novel molecules and targets in haematology, nephrology, neurology, metabolic disorders, cardiology and ophthalmology.