AstraZeneca’s Anselamimab Falls Short in AL Amyloidosis Phase III Trials

AstraZeneca’s rare disease unit, Alexion, reported that anselamimab, an investigational antibody targeting light chains in AL amyloidosis, did not meet its primary endpoints in two global Phase III CARES trials. The trials, involving 406 patients with Mayo stage IIIa and IIIb disease, failed to show significant improvements in combined all-cause mortality and cardiovascular hospitalisation rates. However, a prespecified subgroup did show clinically meaningful benefit, though details remain undisclosed. Despite this setback, anselamimab was well-tolerated, with adverse events comparable between treatment and placebo groups. AL amyloidosis is a rare, life-threatening condition where misfolded light chain proteins deposit as fibrils in organs, leading to dysfunction, especially in the heart. AstraZeneca will continue analysing trial data and engage with regulatory bodies. This marks a second failure for monoclonal antibody therapies in AL amyloidosis, following Prothena’s birtamimab. The only FDA-approved therapy remains Darzalex Faspro, while other candidates like NXC-201 continue to advance in early-stage trials.

27-07-2025