Scientific Publications by Oryzon

At ORYZON we do cutting edge research in the field of epigenetics, with a particular focus in oncology and CNS. We share our findings with the scientific community through peer-reviewed publications in leading scientific journals, as well as through communications in the form of posters and oral presentations in international scientific conferences. Below you can find a list of our most relevant scientific publications.

CNS
  • PORTICO – Double-blind, randomized placebo-controlled, adaptive phase IIb trial with vafidemstat in borderline personality disorder – aggregated baseline characteristics, demographics and safety. Ropacki M et al, 36th European College of Neuropsychopharmacology congress, ECNP 2023, poster communication
  • Safety and efficacy data from SATEEN Trial In Multiple Sclerosis. Rodríguez Acevedo B et al, 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis, ECTRIMS 2021, poster communication
  • ESCAPE trial: Preliminary data on the effect of vafidemstat treatment in the COVID-19 induced immune response in hospitalized patients. Arévalo M et al, 31st European Congress of Clinical Microbiology and Infectious Diseases, ECCMID-2021, poster communication
  • REIMAGINE-AD: vafidemstat shows efficacy in Alzheimer-related agitation&aggression after 12 months. Boada M et al, 15th International Conference on Alzheimer’s and Parkinson’s Diseases AD/PD 2021, poster communication
  • Topline ETHERAL Phase II trial data. Bullock R et al, 15th International Conference on Alzheimer’s and Parkinson’s Diseases AD/PD 2021, poster communication
  • First-in-Human Randomized Trial to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of the KDM1A Inhibitor Vafidemstat. Antonijoan RM, et al. CNS Drugs, 2021, https://doi.org/10.1007/s40263-021-00797-x
  • Modulation of KDM1A with vafidemstat rescues memory deficit and behavioral alterations. Maes T, et al. PLOS ONE, 2020, doi: 10.1371/journal.pone.0233468.
  • Vafidemstat in mild to moderate Alzheimer’s disease: The ETHERAL study European cohort interim analysis. Bullock R, et al. AAIC 2020, oral communication.
  • Vafidemstat reduces aggressiveness in three different psychiatric disorders. Final data from the REIMAGINE trial. Bullock R, et al. 28th European Congress of Psychiatry, EPA 2020, poster communication.
  • Vafidemstat safety and efficacy in Alzheimer-related agitation & aggression: Phase II REIMAGINE-AD 6-month data. Bullock R, et al. Advances in Alzheimer’s and Parkinson’s Therapies congress, AAT-AD/PD 2020, poster communication.
  • Vafidemstat in Alzheimer’s disease: initial 6-month ETHERAL data. Bullock R, et al. Advances in Alzheimer’s and Parkinson’s Therapies congress, AAT-AD/PD 2020, oral communication.
  • Vafidemstat: An epigenetic drug with emerging therapeutic potential, composite data from three psychiatric disorders from the REIMAGINE trial. Ramos-Quiroga JA, et al. International College of Neuropsychopharmacology International meeting, CINP 2019, poster communication.
  • Vafidemstat improves aggression scores in Autism: REIMAGINE, third cohort clinical data. Ropacki M, et al. 32th European College of Neuropsychopharmacology Congress, ECNP 2019, poster communication.
  • Safety Evaluation of Vafidemstat on Mild to Moderate Alzheimer’s Subjects. Bullock R, et al. Alzheimer’s Association International Conference, AAIC 2019, poster communication.
  • Vafidemstat is safe and effective in treating adult ADHD patients: Phase II REIMAGINE clinical trial. Bullock R, et al. 7th World Federation ADHD meeting 2019, poster communication.
  • Vafidemstat, a new approach to treat aggression in central nervous systems disorders: clinical study REIMAGINE. Report in a cohort of Borderline Personality patients. Ferrer M, et al. 27th European Congress of Psychiatry, EPA 2019, poster communication.
  • ETHERAL: Vafidemstat in Alzheimer’s disease: Design and baseline data. Bullock R, et al. 14th International Conference on Alzheimer’s and Parkinson’s Diseases, ADPD 2019, poster communication.
  • Understanding Epigenetic Alterations in Alzheimer's and Parkinson's Disease: Towards Targeted Biomarkers and Therapies. Ciceri F, et al. Curr Pharm Des. 2017; 23(5):839-857. doi: 10.2174/1381612823666170124121140. Review. PMID: 28120717
  • First-in-man Clinical Trial to Assess Safety, Tolerability and Pharmacokinetics of ORY-2001, a novel epigenetic drug for treatment of neurodegenerative diseases. Molinero C, et al. 13th International Conference on Alzheimer’s and Parkinson’s Diseases, ADPD2017, poster communication.
  • KDM1 histone lysine demethylases as targets for treatments of oncological and neurodegenerative disease Maes T, et al. Epigenomics, 2015 Jun;7(4):609-26. doi: 10.2217/epi.15.9 Review.
Oncology
  • Iadademstat in combination with azacitidine in patients with newly diagnosed acute myeloid leukaemia (ALICE): an open-label, phase 2a dose-finding study. Salamero O et al, The Lancet Haematology, 2024, https://doi.org/10.1016/S2352-3026(24)00132-7
  • Iadademstat in combination with gilteritinib for patients with mutated FLT3 relapsed/refractory acute myeloid leukemia. Fahti A et al, American Society of Clinical Oncology (ASCO) 2023 Annual Meeting, poster communication
  • Iadademstat Combination with Azacitidine Is a Safe and Effective Treatment in First Line Acute Myeloid Leukemia. Final Results of the ALICE Trial. Salamero O et al, 64th American Society of Hematology (ASH) Annual Meeting, 2022, oral communication
  • Iadademstat and gilteritinib synergistically abrogate viability of both treatment-naïve and drug-resistant AML cells. Sacilotto N et al, 34th EORTC-NCI-AACR Symposium, 2022, poster communication
  • Comprehensive in Vitro Characterization of the LSD1 Small Molecule Inhibitor Class in Oncology. Sacilotto N, et al. ACS Pharmacology & Translational Science, 2021, https://doi.org/10.1021/acsptsci.1c00223
  • Robust efficacy signals in elderly AML patients treated with iadademstat in combination with azacitidine (ALICE Phase IIa Trial). Salamero O, et al. ASH 2020, e-poster communication.
  • A First-in-Human Phase 1 Study of iadademstat (ORY-1001), a First-in-Class Lysine-Specific Histone Demethylase 1A Inhibitor, in relapsed or refractory Acute Myeloid Leukemia. Salamero O, et al., J Clin Oncol 2020, 38(36): 4260-4273, doi: 10.1200/JCO.19.03250
  • Final safety and efficacy data from CLEPSIDRA trial in 2L ED-SCLC. Navarro A, et al. European Society for Medical Oncology Congress, ESMO 2020, poster communication.
  • Iadademstat Shows Efficacy in Combination with Azacitidine in Elderly AML Patients. ALICE Trial. Salamero O, et al. 25th Congress of the European Hematology Association, EHA 2020, poster communication.
  • Pre-clinical activity of combined LSD1 and mTORC1 inhibition in MLL-translocated acute myeloid leukaemia, Deb G et al. Leukemia. 2019 Nov 28. doi: 10.1038/s41375-019-0659-6
  • CLEPSIDRA: a pilot, biomarker-guided study to assess safety, tolerability, dose finding and efficacy of iadademstat in combination with platinum-etoposide in patients with relapsed, extensive-stage small cell lung cancer. Navarro A, et al. European Society for Medical Oncology Congress, ESMO 2019, poster communication.
  • Iadademstat shows preliminary efficacy signals in relapsed ED-SCLC patients: A case report within CLEPSIDRA, a Phase II trial of iadademstat in combination with platinum-etoposide in biomarker-positive patients. Xaus J, et al. 20th World Conference on Lung Cancer, IASLC 2019, poster communication.
  • ALICE: An AML study with LSD1i in Combination with azacitidine in the Elderly. Montesinos P, et al. 24th Congress of the European Hematology Association, EHA 2019, poster communication.
  • ORY-1001, a Potent and Selective Covalent KDM1A Inhibitor, for the Treatment of Acute Leukemia. Maes T, et al. Cancer Cell. 2018 Mar 12;33(3):495-511.e12. doi: 10.1016/j.ccell.2018.02.002. Epub 2018 Mar 1. PMID: 29502954
  • Safety, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Activity in Acute Leukemia of ORY-1001, a First-in-Class Inhibitor of Lysine-Specific Histone Demethylase 1A (LSD1/KDM1A): Initial Results from a First-in-Human Phase 1 Study. Somervaille T, et al. Americal Society of Hematology Annual meeting, ASH 2016, poster communication.
  • KDM1 histone lysine demethylases as targets for treatments of oncological and neurodegenerative disease Maes T, et al. Epigenomics, 2015 Jun;7(4):609-26. doi: 10.2217/epi.15.9 Review.
  • Advances in the development of histone lysine demethylase inhibitors. Maes T, et al. Curr Opin Pharmacol. 2015 Jun 5;23:52-60. doi: 10.1016/j.coph.2015.05.009. Review. PMID: 26057211
Other targets, diseases & Technology
  • ORY-4001, a novel potent and selective oxadiazole-bBased HDAC6 inhibitor shows pre-clinical therapeutic efficacy in CMT1A. Sacilotto N et al, Peripheral Nerve Society (PNS) Annual Meeting 2023, poster communication
  • Discovery of a novel series of selective non-hydroxamic HDAC6 inhibitors. Salas J, et al. American Chemical Society meeting, ACS 2020, e-poster.
  • Direct Measurement of KDM1A Target Engagement Using Chemoprobe-based Immunoassays. Mascaró C, et al. J Vis Exp. 2019 Jun 13;(148). doi: 10.3791/59390.
  • Chemoprobe-based assays of histone lysine demethylase 1A target occupation enable in vivo pharmacokinetics and -dynamics studies of KDM1A inhibitors. Mascaró C, et al. J. Biol. Chem. 2019 doi: 10.1074/jbc.RA118.006980. Epub 2019 Feb 25. PMID: 30804215
  • Oral administration of the LSD1 inhibitor ORY-3001 increases fetal hemoglobin in sickle cell mice and baboons. Rivers A, et al. Exp Hematol. 2018 Aug 17. pii: S0301-472X(18)30750-1. doi: 10.1016/j.exphem.2018.08.003. [Epub ahead of print] PMID: 30125603