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Ref Type Journal Article
PMID (29502954)
Authors Maes T, Mascaró C, Tirapu I, Estiarte A, Ciceri F, Lunardi S, Guibourt N, Perdones A, Lufino MMP, Somervaille TCP, Wiseman DH, Duy C, Melnick A, Willekens C, Ortega A, Martinell M, Valls N, Kurz G, Fyfe M, Castro-Palomino JC, Buesa C
Title ORY-1001, a Potent and Selective Covalent KDM1A Inhibitor, for the Treatment of Acute Leukemia.
Journal Cancer cell
Vol 33
Issue 3
Date 2018 Mar 12
Abstract Text The lysine-specific demethylase KDM1A is a key regulator of stem cell potential in acute myeloid leukemia (AML). ORY-1001 is a highly potent and selective KDM1A inhibitor that induces H3K4me2 accumulation on KDM1A target genes, blast differentiation, and reduction of leukemic stem cell capacity in AML. ORY-1001 exhibits potent synergy with standard-of-care drugs and selective epigenetic inhibitors, reduces growth of an AML xenograft model, and extends survival in a mouse PDX (patient-derived xenograft) model of T cell acute leukemia. Surrogate pharmacodynamic biomarkers developed based on expression changes in leukemia cell lines were translated to samples from patients treated with ORY-1001. ORY-1001 is a selective KDM1A inhibitor in clinical trials and is currently being evaluated in patients with leukemia and solid tumors.


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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
ORY-1001 ORY-1001 3 0
Drug Name Trade Name Synonyms Drug Classes Drug Description
ORY-1001 RO7051790|RO 7051790|RG6016|RG 6016|iadademstat KDM1A Inhibitor 12 ORY-1001 (iadademstat) binds to and inhibits KDM1A (LSD1), leading to increased H3K4 dimethylation and potentially resulting in increased differentiation and decreased growth of tumor cells (PMID: 29502954, PMID: 32191225).
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
Unknown unknown acute myeloid leukemia not applicable ORY-1001 Preclinical - Cell line xenograft Actionable In a preclinical study, ORY-1001 decreased colony forming ability in several acute myeloid leukemia (AML) cell lines and primary AML samples in culture, and reduced tumor growth in AML cell line xenograft model (PMID: 29502954). 29502954
Unknown unknown acute lymphoblastic leukemia not applicable ORY-1001 Preclinical - Pdx Actionable In a preclinical study, ORY-1001 reduced leukemic blast percentage and prolonged survival in a T-cell acute lymphoblastic leukemia patient-derived xenograft (PDX) model (PMID: 29502954). 29502954