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A monoclonal antibody targeting cell surface nucleophosmin (csNPM1), developed as an immunotherapy candidate for treating acute myeloid leukemia (AML). csNPM1 is present on the surface of AML blasts and leukemic stem cells, but absent from normal hematopoietic stem cells. This antibody mediates immune destruction of csNPM1-expressing cells and has shown robust anti-tumor effects in preclinical AML models, including patient-derived xenografts, without observed toxicity to normal hematopoiesis. It is mutation-agnostic and potentially applicable to other tumor types where csNPM1 is expressed. The modality aims to selectively target cancer cells, potentially avoiding the cytopenias common with other AML therapies. This agent is preclinical and not yet tested in humans[1].
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