SOURCE: Oryzon Genomics

Oryzon Genomics

September 12, 2016 08:00 ET

Oryzon to Present ORY-2001 Preclinical Efficacy Data in Multiple Sclerosis Animal Model at ECTRIMS-2016 Annual Meeting

World Renowned Key Opinion Leader Dr. Xavier Montalban to Join Oryzon's Scientific Advisory Board

BARCELONA, SPAIN and CAMBRIDGE, MA--(Marketwired - September 12, 2016) - Oryzon Genomics (MAD: ORY) (ISIN Code: ES0167733015), a public clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with strong unmet medical need, announced today it will present efficacy data on its proprietary oral epigenetic drug ORY-2001 in a preclinical multiple sclerosis (MS) animal model.

The data will be presented at the 32nd congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) to be held in London (UK) on September 14-17 in the Poster entitled "LSD1 inhibition, a potential epigenetic therapeutic approach for the treatment of multiple sclerosis." The ECTRIMS congress is considered the world's largest annual international conference devoted to basic and clinical research in multiple sclerosis.

ORY-2001 is a highly selective dual LSD1-MAOB inhibitor. Previously, in Alzheimer's Disease (AD) mouse models, long-term treatments with the drug demonstrated a robust and marked cognitive improvement. ORY-2001 has a good safety profile and high therapeutic index in animals and is currently in Phase I clinical study in healthy volunteers. Biomarkers identified in the AD mouse model led Oryzon to test the compound in a neuroinflammatory model.

In a widely used MS model, the Experimental Autoimmune Encephalomyelitis (EAE) mice model, treatment with ORY-2001 during the effector phase greatly inhibited the development of EAE and reduced disease incidence and severity. This represents the first report of the benefit of epigenetic modulation of histone demethylation in an autoimmune disorder and points at a potential of ORY-2001 for the treatment of MS.

LSD1 is an epigenetic modulator, which regulates histone methylation. Epigenetic approaches to modify the progression of various neurodegenerative diseases focus on producing changes in patterns of gene expression in neurons and also in glia cells and are of interest for the pharmaceutical industry. Dr. Tamara Maes, Oryzon's VP and CSO, said "the positive results obtained in different animal models of neurodegenerative and neuroinflammatory disorders suggest that common components controlled by epigenetic mechanisms may contribute to different diseases and suggests a way to explore novel therapies."

The world renowned Key Opinion Leader in MS, Dr. Xavier Montalban has also joined the Scientific Advisory Board of the Company to help Oryzon's scientists to explore the clinical potential of LSD1 inhibitors in the treatment of neuroinflammatory disorders like MS.

Oryzon has a highly competitive and productive Epigenetics Platform, centered around the development of LSD1 inhibitors, with a first drug currently in Phase IIA in acute leukemia that validates the platform scientifically and clinically. This Platform has so far produced two compounds in clinical development with multiple indication opportunities for both drugs. Oryzon's LSD1 drugs are pioneering the Histone demethylases field with its forerunner oncologic drug, ORY-1001, licensed to Roche in 2014, and ORY-2001 in Phase I in neurodegenerative disorders. In addition, Oryzon has nominated recently a third epigenetic compound, also against LSD1, as preclinical candidate for a yet undisclosed non-oncological orphan indication.

About Multiple Sclerosis

Multiple Sclerosis is a primary autoimmune mechanism involving recurrent inflammatory attacks on the central nervous system (CNS) due to a malfunctioning of immune system that leads to neurological disability. This attack, performed by lymphocyte and macrophage infiltrates and glial cell activation, produces the loss of the myelin of oligodendrocytes. These cells recover the axons of the neighboring neurons with sheets of myelin. Myelin is crucial for saltatory conduction of nerve impulses. Inflammation is the core contributor to the degeneration associated with MS. However the exact underlying cause of the inflammatory autoimmune process is poorly understood. MS appears to involve a complex combination of genetic susceptibility and a non-genetic trigger. The World Health Organization (WHO) reports that the median estimated global prevalence is 30 cases per 100,000 population and the estimated incidence is approximately 2.5 cases per 100,000 population. Globally, MS has an average age at onset of 30 years and occurs about twice as frequently in women as in men. MS affects approximately 2.5 million people worldwide each year and is the most debilitating neurological disease in young adults. There are several forms of MS, relapse-remitting and progressive MS. The progressive forms are still a strong unmet medical need. The overall MS market in the U.S. and EU5 is expected to grow to $20.0 billion in 2024.

About Oryzon

Founded in 2000 in Barcelona, Spain, Oryzon (ISIN Code: ES0167733015) is a clinical stage biopharmaceutical company considered as the European champion in Epigenetics. The company has one of the strongest portfolios in the field and a clinical asset already partnered with Roche. Oryzon's LSD1 program is currently covered by 20 patent families and has rendered two compounds in clinical trials. In addition, Oryzon has ongoing programs for developing inhibitors against other epigenetic targets. The company has a strong technological platform for biomarker identification and performs biomarker and target validation for a variety of malignant and neurodegenerative diseases. Oryzon's strategy is to develop first in class compounds against novel epigenetic targets through Phase II clinical trials, at which point it is decided on a case-by-case basis to either keep the development in-house or to partner or outlicense the compound for late stage development and commercialization. The company has offices in Barcelona and Cambridge, Massachusetts. For more information, visit


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