technology

For Patient

Patient Information

Oryzon has two drugs in clinical development, Iadademstat (also known as ORY-1001), a selective LSD1 inhibitor for oncology, and Vafidemstat (also known as ORY-2001), a dual LSD1-MAOB inhibitor for central nervous system (CNS) disorders.

Oryzon has completed one Phase I clinical trial with Iadademstat (ORY-1001) in patients with acute leukemia, and especially in those with alteration in the MLL gene and other specific genomic alterations. Recently, Oryzon has received the approval to start two Phase IIa studies with Iadademstat: the ALICE study will evaluate the safety, tolerability, dose finding and efficacy of Iadademstat in combination with standard treatment with Azacitidine in newly diagnosed patients with Acute Myeloid Leukemia (AML) not eligible for intensive chemotherapy, whereas the CLEPSIDRA study will evaluate the safety, tolerability, dose finding and efficacy of Iadademstat in combination with platinum-etoposide in patients with small cell lung cancer.

With Vafidemstat (ORY-2001), Oryzon has completed one Phase I clinical trial in healthy volunteers and is currently conducting and recruiting patients in two Phase IIa studies in multiple sclerosis and in Alzheimer’s disease. In addition, Oryzon has recently received the approval to start the REIMAGINE study, a Phase IIa study that will assess the safety, tolerability and effect of Vafidemstat on aggressiveness in patients with neurodegenerative disorders (Lewy Body Dementia (LBD) and Alzheimer's Disease (AD)) or psychiatric disorders (Autism Spectrum Disorder (ASD), Borderline Personality Disorder (BPD) and adult Attention Deficit Hyperactivity Syndrome (ADHD)).

Both Oryzon’s experimental drugs target the Lysine-specific demethylase LSD1, a critical enzyme involved in the regulation of histone and chromatin remodeling. By doing this, they regulate important genes involved in the control of cellular differentiation, survival and proliferation.

Oryzon’s compounds are investigational medicines, meaning that they are being evaluated in clinical trials and are not yet available for use outside of a clinical trial setting. Approval by regulatory authorities such as the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA) at the end of their clinical development is the way to make our medicines available to the patients in need of these medicines.

In the meantime, our priority is to advance in the clinical trials in order to obtain the data required for the review and approval of our medicaments by the regulatory authorities. We provide below a summary of the clinical trials sponsored by Oryzon.

 

Ongoing studies

SATEEN study (SAfety, Tolerability and Efficacy in an EPIGENETIC approach to treat Multiple Sclerosis)

“Randomized, double-blind, placebo-controlled, 3-arm, 36 weeks parallel-group study to evaluate the safety and tolerability of ORY-2001 in patients with Relapsing-Remitting Multiple Sclerosis (RRMS) and Secondary Progressive Multiple Sclerosis (SPMS)”

Phase IIa study to evaluate the safety and tolerability and clinical effect of Vafidemstat (ORY-2001) compared to placebo in patients with Multiple Sclerosis.

This study is conducted in Spain and the centers participating are:

  • CEMCAT - Hospital Vall d’Hebron, Barcelona
  • Hospital Virgen de la Macarena, Sevilla
  • Hospital Josep Trueta, Girona
  • Hospital Universitario Germans Trias i Pujol, Badalona
  • Hospital Universitario y Politécnico de La Fe, Valencia
  • Hospital Regional Universitario Carlos Haya, Malaga
  • Hospital Puerta de Hierro, Madrid
  • Hospital del Mar, Barcelona

Status: currently recruiting patients

For more information related to this clinical trial see the EU Clinical Trials Register.

 

ETHERAL study (Epigenetic THERapy in ALzheimer’s Disease)

“A multicentre, multinational, randomised, double-blind, placebo-controlled, 3-arm, 24-week parallel-group study to evaluate the safety, tolerability and preliminary efficacy of ORY-2001 in patients with mild-moderate Alzheimer's Disease”

International and multicenter Phase IIa study to evaluate the safety, tolerability and preliminary efficacy of Vafidemstat (ORY-2001) compared to placebo in patients with mild or moderate Alzheimer´s Disease.

The study is conducted in Spain, France and UK and the centers currently initiated and recruiting patients are:

  • Hospital Vall d’Hebrón, Barcelona,ES
  • Fundación ACE, Barcelona,ES
  • Hospital Santa Maria, Lleida,ES
  • CSM Alamedilla, Salamanca,ES
  • Quirón Salud Policlínica Guipúzcoa,ES
  • Sant Pancras Clinical Research, London,UK
  • Re:Cognition Health London,UK
  • Re:Cognition Health Guildford,UK
  • Re:Cognition Health Birmingham,UK
  • Re:Cognition Health Plymouth,UK
  • Kingshill Research, Avon & Wiltshire Mental Health Partnership NHS Trust, Victoria Centre, Great Western Site, Swindon,UK
  • Neurology Cognitiv Center - GH Lariboisière - Fernand Widal. Paris,FR
  • IM2A (Institut de la Mémoire et de la Maladie d'Alzheimer) Hopital de la Salpêtrière. Paris,FR

Status: currently recruiting patients

For more information related to this clinical trial see the EU Clinical Trials Register.

 

REIMAGINE study

“An unicenter, open-label, 1-arm, 8-week study to evaluate the efficacy, safety and tolerability of ORY-2001 in aggression in adult population with Alzheimer’s Disease (AD), Lewy Body Dementia (LBD), Adult attention deficit hyperactivity disorder (ADHD), Borderline Personality Disorder (BPD), Autism Spectrum Disorder (ASD)”

Phase IIa study to evaluate the safety, tolerability and efficacy of Vafidemstat (ORY-2001) on aggressiveness in patients with neurodegenerative disorders (Lewy Body Dementia (LBD) and Alzheimer's Disease (AD)) and psychiatric disorders (Autism Spectrum Disorder (ASD), Borderline Personality Disorder (BPD) and adult Attention Deficit Hyperactivity Disorder (ADHD)).

This study is conducted in Spain at Hospital Vall d’Hebrón in Barcelona.

Status: currently recruiting patients

For more information related to this clinical trial see the EU Clinical Trials Register.

 

ALICE study (An AML trial with LSD1i in Combination with azacitidine in the Elderly)

“A pilot study to assess the safety, tolerability, dose finding and efficacy of ORY-1001 in combination with azacitidine in older patients with AML in first line therapy”

Phase IIa study to evaluate the safety, tolerability, dose finding and efficacy of Iadademstat (ORY-1001) in combination with standard treatment with Azacitidine for newly diagnosed patients with Acute Myeloid Leukemia (AML) not eligible for intensive chemotherapy.

This study is conducted in Spain and the centers participating are:

  • Hospital Universitario Vall d´Hebrón, Barcelona,ES
  • Hospital Universitario y Politécnico La Fe, Valencia ,ES

Status: pending to start recruitment

For more information related to this clinical trial see the EU Clinical Trials Register.

 

CLEPSIDRA study (A Combination trial of LSD1 and Etop-Platinum in Small Cell Lung Cancer in Biomarker-ID Relapsed pAtients)

“A pilot study to assess the safety, tolerability, dose finding and efficacy of ORY-1001 in combination with platinum-etoposide chemotherapy in patients with relapsed, extensive-stage disease small cell lung cancer”

Phase IIa study to evaluate the safety, tolerability, dose finding and efficacy of Iadademstat (ORY-1001) in combination with platinum-etoposide chemotherapy in patients with relapsed, extensive-stage disease small cell lung cancer which are candidate to re-challenge with platinum-etoposide chemotherapy.

This study is conducted in Spain and the centers participating are:

  • Centro Oncológico Clara Campal, Madrid,ES
  • Hospital Universitario y Politécnico La Fe, Valencia,ES
  • Hospital Universitario 12 de Octubre, Madrid,ES
  • Instituto Oncológico Dr. Rosell, Barcelona,ES

Status: pending to start recruitment

 

Completed studies

“A phase I study of Human Pharmacokinetics and Safety of ORY-1001, an LSD1 inhibitor, in relapsed or refractory acute leukemia”.

This is a study to establish the pharmacokinetics (how the human body processes the drug) and safety of the new anticancer drug ORY-1001 in cancer of blood and bone marrow after treatment or remission. This study included an extension arm on specific leukemia population for a preliminary efficacy evaluation. This clinical trial, which was conducted in 10 hospitals in Spain, France and United Kingdom, has finalized and preliminary results were presented at the American Society of Hematology (ASH) conference in December 2016. The publication of the final results in a scientific journal is in preparation.

For more information related to this clinical trial see the EU Clinical Trials Register.

 

"A Study to Assess the Safety, Tolerability and Pharmacokinetic of Single and Multiple Oral Doses of ORY-2001 in Healthy Male, Female Subjects and Elderly Population".

This Phase I study was performed at the Hospital de St. Pau in Barcelona in healthy volunteers to assess the safety and pharmacokinetics of the new drug ORY-2001. The study has been completed and the final results were presented at the Alzheimer Association International Conference (AAIC) in July 2017. The corresponding publication in a scientific journal is in preparation.