pinometostat

Pinometostat is a first-in-class, small molecule inhibitor of the histone lysine-methyltransferase DOT1L (disruptor of telomeric silencing 1-like). It specifically blocks DOT1L activity, thereby inhibiting methylation of nucleosomal histone H3 on lysine 79 (H3K79), which is critical for the expression of leukemogenic genes in mixed lineage leukemia (MLL) rearranged leukemias. By preventing this methylation, pinometostat suppresses the expression of MLL fusion protein target genes and induces apoptosis in leukemic cells with MLL gene translocations. Pinometostat has been primarily developed for acute leukemias with MLL rearrangements and has orphan drug status for precursor cell lymphoblastic leukemia/lymphoma and acute myeloid leukemia. It is administered as a continuous intravenous infusion[1][3][4][5][7].

Other names

pinometostat anhydrous