AS-99 TFA is a first-in-class, potent, and selective ASH1L histone methyltransferase inhibitor with anti-leukemic activity. It blocks cell proliferation, induces apoptosis and differentiation, downregulates MLL fusion target genes, and reduces the leukemia burden in vivo. The compound has an IC50 of 0.79 μM and a Kd of 0.89 μM for ASH1L.
- First-in-class, potent, and selective ASH1L histone methyltransferase inhibitor.
- Exhibits anti-leukemic activity.
- Blocks cell proliferation.
- Induces apoptosis and differentiation.
- Downregulates MLL fusion target genes.
- Reduces leukemia burden in vivo.
- Selective for ASH1L, with no significant inhibition observed at 50 μM on a panel of 20 other histone methyltransferases.