ORY-1001 free base

ORY-1001 is a KDM1A inhibitor (IC50 <20nM) with high selectivity against related FAD dependent aminoxidases (MAO-A/B, IL4I1, KDM1B >100uM, SMOX 7uM) . Treatment of THP-1 (MLL-AF9) cells with ORY-1001, results in a time/dose dependent me2H3K4 accumulation at KDM1A target genes and concomitant induction of differentiation markers (EC50 me2H3K4 and FACS CD11b <1nM) . ORY-1001 induces apoptosis in THP-1 and inhibits proliferation and colony formation of MV (4;11) (MLL-AF4) cells (EC50 <1nM) . ORY-1001 does not inhibit non-related histone modifiers.