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iadademstat + gilteritinib is a combination therapy under clinical investigation for the treatment of relapsed/refractory acute myeloid leukemia (AML) with FLT3 mutations. Iadademstat (ORY-1001) is a highly selective, potent, oral covalent inhibitor of the epigenetic enzyme lysine-specific demethylase 1 (LSD1/KDM1A), modulating gene expression to promote differentiation and reduce leukemic stem cell survival. Gilteritinib is a small molecule tyrosine kinase inhibitor targeting FLT3 and has FDA approval for relapsed/refractory FLT3-mutated AML. The FRIDA trial (Phase Ib) investigates this combination due to preclinical and emerging clinical data showing synergistic anti-leukemic activity, potentially overcoming resistance to FLT3 inhibitors alone. Preliminary data show notable bone marrow blast clearance and remission rates, with an acceptable safety profile[1][3][4][5][6][7].
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