AstraZeneca drug fails to meet main goal in late-stage amyloidosis trial

(Sharecast News) - An investigational AstraZeneca treatment for amyloidosis has failed to show a statistical significance in late-stage trials, the blue chip pharmaceutical group said on Wednesday. The blue chip said that high-level results from a phase III clinical programme for anselamimab in light chain (AL) amyloidosis "did not achieve statistical significant for the primary endpoint when compared to placebo in patients with specific stages of the disease".

The endpoint was defined as a hierarchical combination of time to all-cause mortality and frequency of cardiovascular hospitalisations.

AL amyloidosis is a rare, progressive disorder caused by defective plasma cells in bone marrow.

Anselamimab, an investigational monoclonal antibody, is intended to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients.

However, AstraZeneca flagged that despite the lack of overall statistical significance in the overall patient population, the treatment did show a "highly clinically meaningful improvement" in a prespecified sub-group of sufferers when compared to placebo.

Ashutosh Wechalekar, the lead principle investigator of the programme and professor of medicine and haematology at University College Hospital, said: "While the study did not meet the primary endpoint in the overall potential population, results from a pre-defined subgroup suggest that anselamimab, by targeting and clearing amyloid deposits, may address a leading cause of organ damage and functional impairment in these patients."

Marc Dunoyer, chief executive of AstraZeneca's specialist rare disease unit Alexion, added: "Anselamimab is the first and only investigational fibril depleter to show clinical benefits in AL amyloidosis, and these results underscore its potential to address a critical treatment gap in a prespecified subgroup of patients."