AstraZeneca says rare disease drug anselamimab fails late-stage trial

AstraZeneca PLC said its experimental drug for a rare disease failed to meet the main goal of a late-stage study, a setback for its growing rare disease business.

The drug, anselamimab, did not achieve a statistically significant improvement in the overall patient population for treating light chain amyloidosis, a progressive disorder where abnormal proteins build up in organs, the company said on Wednesday.

The primary goal was a combination of reducing death from any cause and the frequency of cardiovascular hospitalizations compared to a placebo.

However, AstraZeneca said the treatment showed a "highly clinically meaningful improvement" in survival and hospitalizations in a pre-specified subgroup of patients.

"Anselamimab is the first and only investigational fibril depleter to show clinical benefit in AL amyloidosis, and these results underscore its potential to address a critical treatment gap in a prespecified subgroup of patients," said Marc Dunoyer, Chief Executive Officer of Alexion, AstraZeneca's rare disease unit.

The company said the drug was well tolerated and that it plans to share the full data with global health authorities.

The trial failure comes as AstraZeneca has been bolstering its rare disease pipeline, including the recent completion of its acquisition of Amolyt Pharma for up to $1.05 billion.