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GNS396 is a small molecule FMS-like tyrosine kinase 3 (FLT3) inhibitor being developed by Genoscience Pharma for the treatment of acute myeloid leukemia (AML). The drug is designed to target cancer stem cells by acting as a lysosomotropic agent that disrupts lysosomal functions and inhibits autophagy pathways, thereby depriving cancer cells of essential nutrients. GNS396 was intended to be combined with chemotherapy drugs like cytarabine to simultaneously target both tumorigenic and non-tumorigenic leukemia cell populations, with the goal of overcoming cellular heterogeneity and plasticity in AML to potentially increase remission time and reduce relapse frequency.
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