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BN104 is a novel, potent and highly selective oral small molecule menin inhibitor developed by BioNova Pharmaceuticals for the treatment of hematological malignancies. Its primary mechanism is inhibition of menin (MEN1), which disrupts the interaction between menin and MLL1 (KMT2A), leading to downregulation of leukemogenic genes such as HOXA and MEIS1 and upregulation of differentiation-associated genes like CD11b[1][3][4]. BN104 has demonstrated efficacy in preclinical models for acute myeloid leukemia (AML) with mixed lineage leukemia rearranged (MLLr) or nucleophosmin mutant (NPM1c). It has shown a favorable safety profile with low risk for QTc prolongation due to weak hERG inhibition[4][5][8]. The drug has received Orphan Drug Designation from the US FDA for AML and is currently in Phase 1/2 clinical trials in China for AML and precursor B-cell lymphoblastic leukemia/lymphoma[3][4].
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