AS-99 TFA is a first-in-class, potent, and selective ASH1L histone methyltransferase inhibitor (IC50 of 0.79 μM, Kd of 0.89 μM) with anti-leukemic activity. It functions by blocking cell proliferation, inducing apoptosis and differentiation, downregulating MLL fusion target genes, and reducing the leukemia burden in vivo.
- Potent and selective ASH1L histone methyltransferase inhibitor
- Demonstrates anti-leukemic activity
- Blocks cell proliferation
- Induces apoptosis and differentiation
- Downregulates MLL fusion target genes
- Reduces leukemia burden in vivo
- Exhibits over 100-fold selectivity towards ASH1L over 20 other histone methyltransferases
- Suppresses MLL fusion driven transcriptional programs
- Leads to dose-dependent downregulation of canonical MLL fusion target genes