AstraZeneca's Experimental Therapy Stumbles In Late-Stage Study For Rare Protein Deposit Condition

On Wednesday, AstraZeneca Plc (NASDAQ:AZN) released high-level results from the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 program.

The trial showed that anselamimab, a light chain depleter antibody, did not achieve statistical significance for the primary endpoint compared to placebo in patients with Mayo stages IIIa and IIIb light chain (AL) amyloidosis.

The primary endpoint was a hierarchical combination of time to all-cause mortality (ACM) and frequency of cardiovascular hospitalizations (CVH). All patients in the clinical program received a background standard of care for plasma cell dyscrasia.

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Anselamimab showed highly clinically meaningful improvement in time to ACM and frequency of CVH in a prespecified subgroup of patients, compared to placebo.

Light chain (AL) amyloidosis is a systemic and progressive type of amyloidosis in which immunoglobulin light chain proteins are abnormally produced by defective plasma cells in the bone marrow.

These abnormal proteins misfold, aggregate and form amyloid fibrils that deposit and accumulate in tissues or organs, particularly in the heart and kidneys. Worldwide, an estimated 74,000 patients are living with AL amyloidosis.

Anselamimab was well tolerated, with most events balanced between the anselamimab treatment arm and the placebo arm. Evaluation of full results is ongoing to further characterise the efficacy and safety of anselamimab.

In other clinical news this week, AstraZeneca also announced positive topline data on Monday, July 14, from the BaxHTN Phase 3 trial for baxdrostat.

This investigational oral aldosterone synthase inhibitor is being developed for patients with uncontrolled or treatment-resistant hypertension.

The trial demonstrated that baxdrostat, at both 2mg and 1mg doses, achieved a statistically significant and clinically meaningful reduction in mean seated systolic blood pressure (SBP) compared to placebo at 12 weeks.

This success is particularly noteworthy as uncontrolled and resistant hypertension remain significant public health challenges, with many patients struggling to achieve blood pressure goals despite multiple medications.

Baxdrostat aims to address this unmet need by targeting aldosterone dysregulation, a key contributor to hard-to-control hypertension.

Further solidifying its research and development pipeline, AstraZeneca entered a strategic research collaboration in June with Shijiazhuang City-based CSPC Pharmaceuticals Group Limited.

This partnership aims to leverage CSPC’s AI-driven drug discovery platform to accelerate the discovery and development of novel oral candidates for multiple high-priority targets.

The collaboration specifically focuses on chronic indications, including a preclinical small-molecule oral therapy for immunological diseases.

Price Action: AZN stock is trading lower by 0.30% to $70.11 at last check Wednesday.

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