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ON1232580 (also known as HX301) is a novel, potent small molecule dual kinase inhibitor targeting Colony-Stimulating Factor 1 Receptor (CSF1R) and FMS-like Tyrosine Kinase 3 (FLT3). It is currently in preclinical development for the treatment of acute myeloid leukemia (AML). The compound exhibits high potency with IC50 values of approximately 0.7 nM for CSF1R and 7 nM for FLT3. By inhibiting these two key receptor tyrosine kinases, ON1232580 aims to disrupt the survival and proliferation pathways of leukemic cells, particularly in AML populations characterized by FLT3-ITD mutations or CSF1R expression. Preclinical studies have demonstrated significant anti-leukemic activity in both AML cell lines and patient-derived xenograft (PDX) models.
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