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Patient Information

Oryzon has currently two drugs in clinical development, ORY-1001, a selective LSD1 inhibitor for oncology, and ORY-2001, a dual LSD1-MAOB inhibitor for neurodegenerative diseases.

Oryzon has completed a first clinical trial with ORY-1001 in patients with acute leukemia, and especially in those with alteration in the MLL gene and other specific genomic alterations.

With ORY-2001, Oryzon has completed one Phase I clinical trial in healthy volunteers; and has initiated twoPhase IIa clinical trials in multiple sclerosis and Alzheimer’s disease, both trials currently recruiting patients.

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 of 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 Quiron, Madrid
  • 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 ORY-2001 compared to placebo in patients with mild-moderate Alzheimer´s Disease.

This study, conducted in Spain, France and UK, is already approved in Spain and France, and approval in UK is expected shortly. The centers currently active and recruting are:

  • Hospital Vall d’Hebrón, Barcelona
  • Fundación ACE, Barcelona
  • Hospital Santa Maria, Lleida

Status: currently recruiting patients

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

 

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.