1st U.S. Patient Enrolled in Phase 2a Trial Evaluating Vafidemstat for Alzheimer’s

1st U.S. Patient Enrolled in Phase 2a Trial Evaluating Vafidemstat for Alzheimer’s
A Phase 2a trial testing Oryzon’s investigational therapy vafidemstat for Alzheimer’s disease has enrolled its first patient in the U.S. The trial (NCT03867253), called ETHERAL-US, is an extension of the European trial (2017-004893-32), currently ongoing at 17 sites in the U.K., France, and Spain. Oryzon is planning to recruit up to 30 patients with mild to moderate Alzheimer’s at multiple sites in the U.S. for an expected total of at least 150 participants, including the European arm. ETHERAL-US is now recruiting participants in Florida and New Jersey. Information on contacts and locations is available here. Vafidemstat is an epigenetic therapy, meaning it targets the expression and activity of genes, and is capable of crossing the blood-brain barrier, a highly selective membrane that shields the central nervous system from general blood circulation. Specifically, vafidemstat inhibits two proteins, lysine-specific histone demethylase (LSD1) and monoamine oxidase B (MAOB), which regulate the expression of several genes by removing the epigenetic marks (methyl groups) that sit on top of the genes and work as “off-switch” signals. Preclinical studies on a mouse model of Alzheimer's and accelerated aging showed that vafidemstat restored memory, enhanced sociability, and decreased social avoidance. "Epigenetics plays a role in a broad range of CNS [central nervous system] disorders. LSD1 is as a fascinating target for
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