ADDF Invests $300,000 to Support Phase 2 Clinical Trial of Epigenetic Therapy

April 24, 2017

Category: New Grants

The Alzheimer’s Drug Discovery Foundation (ADDF) has approved a grant of $300,000 to Oryzon Genomics to continue support for its groundbreaking epigenetic drug ORY-2001.

Oryzon is now completing a Phase 1 clinical trial of ORY-2001, which is the first epigenetic treatment for any neurodegenerative disorder to reach human trials. The ADDF began funding the development of Oryzon's promising new approach for treating Alzheimer's in 2010. With a successful Phase 1 outcome, ORY-2001 is expected to proceed to a Phase 2 study in Alzheimer’s disease patients in 2017. This new funding from the ADDF will support the development of biomarkers for the Phase 2 study to monitor patients' response to the drug and its correlation with clinical outcomes.  

Howard Fillit, MD, Founding Executive Director and Chief Science Officer of the ADDF, says: “Our continued investment in Oryzon reflects the ADDF’s belief in the promise of epigenetic therapies and Oryzon’s approach. The companion biomarker program is important for advancing ORY-2001 into later-stage clinical trials, and we are pleased to support its development.”

ORY-2001 is a highly selective dual LSD1-MAOB inhibitor that is pioneering the histone demethylases field in neurodegenerative disorders. In preclinical testing, the molecule restored cognitive function and memory loss and changed gene expression on the hippocampus, increasing memory-related genes and decreasing neuroinflammation genes. The company has also recently presented data that supports the potential application of this experimental drug in other neuroinflammatory disorders such as multiple sclerosis, with ORY-2001 providing efficient protection against demyelination in the spinal cord .

Oryzon Genomics is a leader is epigenetic therapy development and is also developing drug programs for acute leukemia, small-cell lung cancer, and an as yet undisclosed non-oncological orphan indication.