Resumen
IL3RA (alpha subunit of the interleukin 3 receptor) is a cell membrane protein frequently expressed in acute myeloid leukemia (AML) and Hodgkin lymphoma; therefore, it is a promising therapeutic target for cancer treatment. Here, we introduce BAY-943, a novel IL3RA-targeting antibody?drug conjugate that shows potent and selective efficacy in IL3RA-positive AML and Hodgkin lymphoma cell lines. In IL3RA-positive AML mouse models, BAY-943 improved survival and reduced tumor burden. Impressively, treatment with BAY-943 induced complete tumor remission in 12 out of 13 mice in an IL3RA-positive HL model. BAY-943 showed a favorable safety profile without any signs of toxicity in rats and monkeys. Overall, these preclinical results support the further development of BAY-943 for the treatment of IL3RA-positive hematologic malignancies.