AstraZeneca Amyloidosis Drug Fails Phase 3 Trials but Shows Potential in Subgroup

AstraZeneca's amyloidosis drug anselamimab failed to meet the primary endpoint in two late-stage (Phase 3) trials, which assessed improvement in survival and reduction of cardiovascular hospitalizations in patients with light chain (AL) amyloidosis153.

The trials enrolled 406 patients across two different Mayo stages of AL amyloidosis and were named the Cardiac Amyloid Reaching for Extended Survival (CARES) studies14.

Although the therapy missed its main goal in the overall trial population, AstraZeneca reported a clinically meaningful improvement in a prespecified—but undisclosed—subgroup of patients143.

AstraZeneca has not detailed which subgroup benefited but highlighted that the drug was well tolerated, with balanced adverse events between anselamimab and placebo arms15.

Anselamimab was originally developed by Caelum Biosciences, which AstraZeneca acquired in 2021, and is part of AstraZeneca's Alexion rare disease division13.

Despite the Phase 3 setback, AstraZeneca sees potential for anselamimab in addressing a critical treatment gap for certain patients with AL amyloidosis and plans to further analyze the efficacy and safety data to determine next steps34.

Sources:

1. https://www.biospace.com/drug-development/astrazenecas-amyloidosis-asset-stumbles-in-late-stage-trials

3. https://www.fiercebiotech.com/biotech/astrazenecas-al-amyloidosis-drug-fails-reduce-mortality-phase-3-trial

4. https://www.ainvest.com/news/astrazeneca-anselamimab-stumbles-al-amyloidosis-crossroads-rare-disease-innovation-2507/

5. https://www.biopharmadive.com/news/astrazeneca-anselamimab-phase-3-failure-results/753197/