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Ref Type
Authors Francis Burrows, Tao Wu, Linda Kessler, Shuangwei Li, Jingchuan Zhang, Patrick Zarrinkar, Liansheng Li, Tomasz Cierpicki, Jolanta Grembecka, Pingda Ren and Yi Liu
Title A novel small molecule menin-MLL inhibitor for potential treatment of MLL-rearranged leukemias and NPM1/DNMT3A-mutant AML
Journal Mol Cancer Ther
Abstract Text Mol Cancer Ther 2018;17(1 Suppl):Abstract nr LB-A27


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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
KO-539 KO-539 1 1
Drug Name Trade Name Synonyms Drug Classes Drug Description
KO-539 KO539|KO 539 MEN1-KMT2A Inhibitor 5 KO-539 is a small molecule inhibitor of MEN1 (menin)-KMT2A (MLL1) complex, which may lead to growth inhibition of KMT2A (MLL1)-positive tumor cells (Mol Cancer Ther 2018;17(1 Suppl):Abstract nr LB-A27).
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
KMT2A rearrange acute myeloid leukemia predicted - sensitive KO-539 Preclinical - Cell line xenograft Actionable In a preclinical study, KO-539 inhibited growth of KMT2A (MLL1)-rearranged acute myeloid leukemia cells in culture, and prolonged survival in cell-line xenograft models (AACR; Mol Cancer Ther 2018;17(1 Suppl):Abstract nr LB-A27). detail...