Products

ORY-1001 is a selective inhibitor of Lysine Specific Demethylase-1 (LSD1, aka KDM1), an epigenetic modulator that regulates gene expression by demethylating specifically some lysines in the histones. LSD1 forms part of protein complexes involved in transcriptional regulation, and misregulation of these transcriptional complexes may result in disease. This orally-active subnanomolar specific LSD1 inhibitor reduces leukemic stem cell potential, potently inhibits colony formation, overcomes the differentiation block in AML cell lines, and induces apoptosis / inhibits proliferation at sub-nanomolar concentrations in selected AML cell lines.ORY-1001 has successfully passed the regulatory toxicology studies and is now entering into a Phase 1 clinical study on acute leukemia patients.

* On 9-11 July 2013, the Committee for Orphan Medicinal Products (COMP) has recommended the granting of an orphan designation for trans-N1-((1R,2S)-2-phenylcyclopropyl)cyclohexane-1,4-diamine bis-hydrochloride for treatment of acute myeloid leukaemia.