QR Pharma to Present at 10th Annual World Pharma Congress on Parkinson’s Disease

About Posiphen®

QR’s lead compound, Posiphen®, is a small orally active compound with high blood brain barrier permeability, which lowers amyloid precursor protein (APP) levels. It targets the mRNA of a number of proteins that are overexpressed in several neurological disorders such as Alzheimer’s disease, Parkinson’s disease and Down syndrome. It is in clinical development as an oral treatment for Alzheimer’s disease. Posiphen reduces the rate of synthesis of APP in cell cultures, normal, transgenic and trisomic mice as well as in humans. APP is cleaved into a number of toxic peptides, including Aβ42, which is cleaved from the middle while the others are cleaved from the N- and C- terminal ends. These peptides attack multiple pathways of neuronal cell life leading to synaptic loss and nerve cell death. This degeneration of the brain induces dysfunction, neuroinflammation and leads to cognitive impairment and neurodegeneration. QR conducted a trial in patients with mild cognitive impairment to confirm Posiphen’s mechanism of action in humans and correlate it with the pharmacokinetics of the compound and its metabolites in CSF and plasma. Posiphen lowers levels of APP, tau and inflammatory markers by about 40%, approaching the levels found in healthy volunteers.