ORY-1001 is a highly potent and selective covalent LSD1 inhibitor.246 A phase I study on the safety, pharmacodynamics, and pharmacokinetics of ORY-1001 in relapsed or refractory AML sponsored by Oryzon Genomics has been successfully completed in 2016 (EudraCT 2013-002447-29). Here, KDM1A is linked to acute myeloid leukemia.