Oryzon Genomics Readies Updates on ORY-2001, Being Tested in MS Patients in Phase 2 Trial

Oryzon Genomics will give updates on its leading investigational product ORY-2001, a brain-targeting epigenetic therapy now in a Phase 2 clinical trial recruiting multiple sclerosis patients, at a series of scientific conferences.

According to a press release, these include two conferences in the United States, the 2018 BIO International Convention, running from June 4 to 7 in Boston, and the Jefferies 2018 Global Healthcare Conference, in New York from June 5 to 8; and one in Europe, the 14th Spring European Midcap Event, in Paris from June 27 to 28.

ORY-2001 is classified as an epigenetic therapy, meaning it modulates the expression of genes without altering their DNA sequence. The therapy works by targeting and inhibiting, in a selective manner, the activity of two enzymes: the lysine (K)-specific demethylase 1A (LSD1) and monoamine oxidase B inhibitor (MAOB).

LSD1 (also known as KDM1A) is an epigenetic modulator, meaning it regulates the expression of several genes by removing epigenetic marks (methyl groups) that sit on top of the genes and work as “switch off” signals.

LSD1 regulates genes important in the onset and progression of neurodegenerative disorders, cancer, and viral infections. 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 was previously shown to reduce cognitive impairment and neuroinflammation in preclinical animal models of disease, including in a mouse model of MS — the experimental autoimmune encephalomyelitis (EAE) model.

In a Phase 1 clinical trial with healthy volunteers, ORY-2001 was found safe and capable of effectively reaching the brain.

Based on these positive results, the company launched its Phase 2a SATEEN clinical trial to test ORY-2001 in patients with relapsing-remitting or secondary progressive MS over a 36-week period, followed by an open-label extension.

The trial is ongoing at nine sites across Spain, and is currently enrolling patients. More information can be found on its EU trial document, under “sponsor information.”

Participants will be randomized to receive two doses of ORY-2001, administered orally, or to a placebo group.

The trial’s main objective (endpoint) is to evaluate the safety and tolerability of the two doses of ORY-2001 compared to the placebo. Additional (secondary) objectives include the effect of ORY-2001 on inflammation compared to placebo controls, measured by magnetic resonance imaging (MRI).

Enrolled patients will the be given the opportunity to continue or to switch to ORY-2001 – delivered in 0.6 or 1.2. mg doses five times per week – in an open-label extension phase of the study.

This is the first epigenetic therapy for MS and researchers hope that, if the results of the SATEEN trial are positive, it will increase the range of therapeutic options for the disease.

Oryzon is also testing ORY-2001 as a treatment for mild to moderate Alzheimer’s disease.