AstraZeneca (AZ) announced on 16 July that its investigational drug anselamimab failed to meet the primary composite endpoint of time to all-cause mortality (ACM) and cardiovascular hospitalisation (CVH) frequency in the Phase III CARES trial for amyloid light-chain (AL) amyloidosis. The study evaluated anselamimab, a first-in-class anti-amyloid fibril mAb, in patients with Mayo stage IIIa and IIIb AL amyloidosis. However, a prespecified patient subgroup showed clinically meaningful improvements in ACM and CVH.

Anselamimab targets misfolded amyloid fibrils to promote clearance of toxic deposits in organs. AL amyloidosis is a rare, progressive disease where abnormal light chains form amyloid fibrils, damaging vital organs like the heart and kidneys. Despite the overall trial outcome, researchers highlighted its potential to address organ dysfunction in specific subgroups. AZ's rare disease unit Alexion emphasised anselamimab's novel mechanism as the only fibril-clearing therapy demonstrating clinical benefit in AL amyloidosis.

According to PharmCube's NextBiopharm® database, anselamimab is one of 14 clinical-stage candidates looking to enter the AL amyloidosis market (which currently holds 23 launched products), and the only amyloid-targeting mAb directed at this indicaiton. Click here to request a free trial for NextBiopharm®.