iadademstat

Iadademstat is an orally available, highly selective, and potent small molecule inhibitor of lysine specific demethylase 1 (LSD1/KDM1A), an epigenetic enzyme involved in the regulation of gene expression. By covalently binding to the FAD cofactor in LSD1’s catalytic center, iadademstat blocks both the demethylating activity and scaffolding function of LSD1. This inhibition disrupts protein-protein interactions critical for maintaining stemness in cancer cells, particularly through uncoupling LSD1 from transcription factors like GFI-1. The result is forced differentiation of undifferentiated leukemic blasts, reduction in leukemic stem cell capacity and proliferation, and induction of tumor suppressor gene expression. Iadademstat has demonstrated preclinical antileukemic activity and has shown safety and preliminary efficacy as monotherapy or in combination with other agents (such as azacitidine or gilteritinib) in clinical trials for acute myeloid leukemia (AML) and small cell lung cancer (SCLC). It has received orphan drug designation for AML and SCLC[2][3][5][6][7].

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