Posiphen, a small orally active compound, has shown in cell cultures of normal mice, AD transgenic mice and down syndrome (DS) mice to reduce the synthesis of amyloid-ß precursor protein (APP). These peptides include amyloid-ß42 (Aß42), that attacks multiple pathways of neuronal cell life, inducing dysfunction, neuronal cell death and neuroinflammation, leading to cognitive impairment and neurodegeneration.
The trial will measure in the cerebrospinal fluid (CSF) and blood plasma of amnestic mild cognitively impaired (MCI) patients, the biochemical changes that are associated with AD, and correlate them with the pharmacokinetics of the drug and its metabolites.
Maria Maccecchini, CEO of QR Pharma, said: “The more we learn about Posiphen the more we see how it interferes with a basic pathway that leads to dementia in DS, AD and likely other conditions, such as head trauma and stroke. We have substantial data in a number of animal models all showing that Posiphen inhibits one of the major pathways leading to dementia and AD. We are very eager to see how the compound affects this pathway and other AD associated markers in patients with amnestic MCI.”