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AstraZeneca's amyloidosis treatment fails main goal in late-stage study
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AstraZeneca's amyloidosis treatment fails main goal in late-stage study
Jul 16, 2025 12:21 AM

July 16 (Reuters) - AstraZeneca ( AZN ) on Wednesday

said that its experimental therapy anselamimab did not meet the

main goal of a late-stage study for the treatment of AL

amyloidosis, a rare condition that causes a buildup of protein

deposits in the body.

The primary outcome of the study was being measured as a

combination of how long patients with advanced, heart-related

amyloidosis lived and how often they were hospitalised for

cardiovascular issues, the Anglo-Swedish drugmaker said.

While anselamimab did not achieve statistical significance

for the overall population in the study, the therapy showed

clinically meaningful improvement in a sub-group of patients

when compared with placebo, AstraZeneca ( AZN ) said.

"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," said Ashutosh Wechalekar, lead

principal investigator of the trial and a professor at

University College London.

AL amyloidosis, also referred to as light chain or primary

amyloidosis, leads to deposits of amyloid protein in the body

because of defective plasma cells.

If left untreated, the accumulation of these deposits,

particularly in the heart and kidneys, can cause organ damage

and dysfunction and lead to heart failure.

AstraZeneca ( AZN ) said it was evaluating the complete data on

anselamimab, which is being developed by its rare diseases

Alexion unit, adding that the company plans to share data with

health authorities and present them at an upcoming medical

conference, without specifying when.

(Reporting by Pushkala Aripaka in Bengaluru; Editing by Vijay

Kishore and Mrigank Dhaniwala)

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