AstraZeneca's AL Amyloidosis Drug Misses Goal in Late-Stage Studies

AstraZeneca AZN reported disappointing results from two late-stage studies that evaluated its investigational light chain depleter antibody, anselamimab, in certain patients with a rare disease called light chain (AL) amyloidosis.

Both studies — part of the phase III CARES clinical program — were designed to assess anselamimab plus standard of care against placebo in patients with stage IIIa and stage IIIb AL amyloidosis, respectively. The primary endpoint of both studies was the same — a hierarchical combination of time to all-cause mortality (ACM) and frequency of cardiovascular hospitalizations (CVH). In other words, the studies were designed to determine whether the drug could help patients live longer and reduce heart-related hospitalizations.

Treatment with anselamimab failed to achieve statistical significance for the primary endpoint in either of the two studies. Though AstraZeneca mentioned that the drug showed clinically meaningful improvement in a prespecified patient subgroup, it did not disclose details of the same. Based on these findings, the company noted that anselamimab is the first fibril depleter to show potential benefit in AL amyloidosis. AZN is currently conducting a full analysis of the results and plans to share the findings at a future medical meeting.

AL amyloidosis is caused by deposits of amyloid protein in the body due to defective plasma cells. If left untreated, these deposits can cause progressive organ damage and may even lead to premature death, most commonly due to cardiac failure. Per AstraZeneca, around 74,000 people across the world are living with this disease.

AZN Stock Performance

Year to date, AstraZeneca’s shares have gained 7% against the industry’s 1% decline.

Another Setback in the AL Amyloidosis Space

AstraZeneca is not the first to report a setback in this space. In May, Prothena PRTA announced a similar setback with its own AL amyloidosis drug, birtamimab, in the phase III AFFIRM-AL study.

The AFFIRM-AL study failed to achieve its primary endpoint of time to ACM. Treatment with the Prothena drug also failed to meet any of its secondary endpoints. Based on this setback, PRTA ended the development of birtamimab and announced a restructuring program last month to curb cash burns, including a 63% workforce reduction.

AZN’s Zacks Rank

AstraZeneca currently carries a Zacks Rank #3 (Hold).

AstraZeneca PLC Price

 

AstraZeneca PLC price | AstraZeneca PLC Quote

Key Picks Among Biotech Stocks

Some better-ranked stocks from the sector are Akero Therapeutics AKRO and Agenus AGEN. While AKRO sports a Zacks Rank #1 (Strong Buy) at present, AGEN carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 90 days, loss per share estimates for Akero’s 2025 have improved from $4.08 to $3.96. Loss per share estimates for 2026 have narrowed from $4.35 to $4.27 during the same period. AKRO stock has surged 93% year to date.

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Agenus’ earnings beat estimates in two of the trailing four quarters and missed the mark on the other two occasions, delivering an average negative surprise of 22.71%.

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