Efstathios Kastritis, MD, discusses the background of birtamimab and the rationale for investigating the agent in combination with standard-of-care chemotherapy in newly diagnosed patients with Mayo Stage IV amyloid light chain amyloidosis in the phase 3 AFFIRM-AL trial.
Efstathios Kastritis, MD, Division of Clinical Therapeutics, University of Athens School of Medicine, discusses the background of birtamimab (formerly NEOD001) and the rationale for investigating the agent in combination with standard-of-care chemotherapy in newly diagnosed patients with Mayo Stage IV amyloid light chain (AL) amyloidosis in the phase 3 AFFIRM-AL trial (NCT04973137).
Birtamimab is a monoclonal antibody designed to specifically and selectively target and clear the amyloid that accumulates and results in organ dysfunction and failure in patients with AL amyloidosis.
Birtamimab was previously investigated in the phase 3 VITAL study (NCT02312206) in patients with newly diagnosed AL amyloidosis. The study was terminated due to futility when the monoclonal antibody plus standard of care did not demonstrate a statistically significant difference in time to all-cause mortality or time to cardiac hospitalization vs placebo in the overall population.
However, a post-hoc analysis from VITAL showed that birtamimab elicited a significant survival benefit compared with placebo in newly diagnosed patients with Mayo Stage IV AL amyloidosis, Kastritis continues. The 9-month overall survival rate was 74% in patients with Mayo Stage IV AL amyloidosis treated with birtamimab, compared with 49% for patients treated with placebo.
Although these data were promising, the findings for patients with Mayo Stage IV AL amyloidosis were from a post-hoc analysis, and a prospective trial is needed to further investigate the efficacy signal in this patient population, Kastritis adds. The ongoing AFFIRM-AL trial is evaluating birtamimab in combination with chemotherapy vs chemotherapy alone in patients with Mayo Stage IV AL amyloidosis.
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