Spain’s Oryzon Genomics will offer new data on the preclinical efficacy of ORY-2001, an epigenetic modulator it is developing to treat multiple sclerosis (MS). Its presentation is set for Oct. 26 at MSParis2017, the joint international meeting of the European and Americas Committees for Treatment and Research in Multiple Sclerosis (ECTRIMS/ACTRIMS).
Dr. Tamara Maes, Oryzon’s chief scientific officer, will present the poster, “Characterization of the efficacy of ORY-2001, a novel epigenetic drug for the treatment of multiple sclerosis, during the effector phase of the EAE model,” which has already been shortlisted for best poster award at MSParis2017.
ORY-2001 is a highly selective inhibitor of two enzymes: lysine (K)-specific demethylase 1A (LSD1) and monoamine oxidase B inhibitor (LSD1-MAOB). The small molecule reduces cognitive impairment, memory loss and neuroinflammation.
LSD1 (also known as KDM1A) is defined as an epigenetic modulator, because it works by removing epigenetic marks (methyl groups) from several genes, thereby affecting their expression. Genes important in the onset and progression of neurodegenerative disorders, but also cancer and viral infections, are among those regulated by LSD1. By blocking LSD1 activity, ORY-2001 can modify gene expression.
MAOB plays an important role in the breakdown of neurotransmitters, or chemical messengers, like dopamine. ORY-2001 can also block the action of this enzyme.
In previous studies with several preclinical models of MS, ORY-2001 showed strong and durable efficacy. Its neuroprotective effects were also detected in experiments with mouse models of Alzheimer’s disease.
In a recent Phase 1 clinical trial with healthy volunteers, ORY-2001 was found safe and capable of effectively reaching the brain. The company plans to start a Phase 2a study soon.
In 2008, the World Health Organization (WHO) estimated the global prevalence of MS at 30 cases per 100,000 inhabitants. In a 2013 update, WHO put the number of people with MS worldwide at 2.3 million, up from 2.1 million in 2008.
The joint ECTRIMS/ACTRIMS Congress will offer a comprehensive scientific and teaching program consisting of courses, plenary lectures and various poster sessions.
Themes covered include recent advances in neurobiology and neuro-immunology; new diagnostic criteria; MS biomarkers; MS management and insight into MS borders; clinical trials, and emerging strategies to boost repair. The meeting will include a special section for MS nurses and symposia sponsored by the European Charcot Foundation and other partners.