Spain-based clinical stage biopharmaceutical company Oryzon Genomics has dosed the first subject in a phase I study to investigate the safety, pharmacokinetics (PK) and pharmacodynamics (PD) of its oral LSD1-MAOB dual selective inhibitor ORY-2001.
It is a randomised, double-blind, placebo-controlled single and multiple ascending dose programme study.
Being conducted in Spain, the phase I study is under a clinical trial application (CTA).
Following a positive study result, ORY-2001 is expected to move into a phase II study in Alzheimer’s patients in the first half of next year.
ORY-2001 is a highly selective dual LSD1-MAOB inhibitor.
Oryzon further said that the molecule, which focuses on cognitive decline and memory loss, has a good safety profile and therapeutic index in preclinical trials.
In non-transgenic AD mouse models, long-term treatments with the ORY-2001 drug demonstrated a clear cognitive improvement.
LSD1 is an epigenetic modulator that regulates histone methylation.
Epigenetic approaches to alter progression of several neurodegenerative diseases focus on making changes in patterns of gene expression in neurons and also in glia cells and are of interest to the pharma industry.
Oryzon is also exploring the potential of ORY-2001 in other neurodegenerative diseases, including Huntington’s disease, Parkinson’s disease and other forms of dementia.
Oryzon chief medical officer Cesar Molinero said: "With our clinical trials on LSD1 inhibitors, we are gaining very valuable information of the potential of Histone Demethylase inhibitors in human diseases beyond oncology and we are particularly interested in the potential of ORY-2001 in neurodegenerative and neuroinflammatory diseases."
Alzheimer’s is said to be the most common form of dementia found in adults and is estimated to impact around 5.3 million people in the US and more than 30 million people worldwide.
In January, Oryzon received approval for its clinical trial application from the Spanish Drug Agency (AEMPS) to initiate the phase I clinical trial for ORY-2001 for treatment of Alzheimer’s.