ziftomenib + cytarabine

Ziftomenib + cytarabine is a combination regimen under investigation for the treatment of acute myeloid leukemia (AML) in patients with nucleophosmin 1 mutation (NPM1-m) or lysine(K)-specific methyltransferase 2A rearrangement (KMT2A-r)[1][3][4][5]. Ziftomenib is a potent, selective small molecule inhibitor of menin, a cofactor that interacts with MLL/KMT2A and NPM1-mutated oncoproteins, thereby blocking oncogenic gene expression programs fundamental to AML pathogenesis[1][3]. Cytarabine is a cytotoxic chemotherapy agent that inhibits DNA synthesis by acting as an antimetabolite. The combination is administered as part of intensive induction (often with daunorubicin as 7+3 regimen), consolidation with cytarabine, and sometimes as maintenance with ziftomenib monotherapy[1][3][4][5]. The combination has demonstrated high rates of composite complete remission (CRc), including measurable residual disease (MRD)-negative remissions, in both NPM1-m and KMT2A-r AML, with an acceptable tolerability profile and no additive myelosuppression observed[4][5]. The principal developer is Kura Oncology, partnered with Kyowa Kirin[2][5]. Clinical development is ongoing in phase 3 trials, both in intensive and non-intensive settings[1][5].

Other names

ziftomenib + cytarabine