QR Pharma and MUSC receive $220,000 SBIR grant to study Posiphen in AD transgenic mice

QR Pharma, Inc. (QR), a clinical-stage specialty pharmaceutical company committed to developing therapeutics with novel approaches for the treatment of cognitive impairment and Alzheimer's disease and the Medical University of South Carolina (MUSC) announced today that they received a Small Business Innovation Research (SBIR) phase I grant in the amount of $220,000 to study Posiphen® and its metabolites in cell culture and in transgenic Alzheimer mice.

“Posiphen, an APP-lowering compound, should target all the toxic fragments cleaved from APP and therefore provide benefits beyond those realized by compounds that target Aβ”

Posiphen, a small orally active compound, has been shown in cell cultures and in normal mice, AD transgenic mice and Down syndrome (DS) mice as well as in humans to reduce the synthesis of amyloid-β precursor protein (APP), which is cleaved into a number of toxic peptides. These peptides include N- and C-terminal fragments and amyloid-beta 42 (Aβ42) that is widely believed to be responsible for Alzheimer disease pathogenesis.

Posiphen was found to be metabolized into three major metabolites. The primary activity is APP inhibition; it is exhibited by Posiphen as well as all three metabolites. The other two activities, acetylcholinesterase inhibition and neuro-regeneration seem to be properties of one or another metabolite, but not of Posiphen. This grant will help evaluate the absolute and relative activities of Posiphen and metabolites as well as test their activities in transgenic mice.

"APP mutations and duplications cause Familial Alzheimer's disease. APP triplication causes Down syndrome and high levels of APP after head trauma, stroke and brain injury lead to neurodegeneration," said Kumar Sambamurti, PhD, Professor of Neurosciences at MUSC. "Posiphen, an APP-lowering compound, should target all the toxic fragments cleaved from APP and therefore provide benefits beyond those realized by compounds that target Aβ".

"Finding these two additional activities of Posiphen and better defining them will help QR Pharma to better understand the efficacy of the compound in humans," said Maria Maccecchini, PhD, CEO of QR Pharma. "The original APP inhibitory activity should give us disease modification, while acetylcholinesterase inhibition should lead to better short term improvements in cognition and neuro-regeneration could lead to a halt in the cognitive decline."