Radnor, PA, February 10, 2010 QR Pharma, Inc., a developer of novel drugs to treat Alzheimer’s disease (AD), announced today that it began a clinical trial of its lead compound, Posiphen®, in early stage AD patients.
Posiphen®, a small orally active compound, has been shown in cell cultures of normal mice, AD transgenic mice and Down Syndrome (DS) mice to reduce the synthesis of amyloid-b precursor protein (APP) which is cleaved into a number of toxic peptides. These peptides include amyloid-b42 (Ab42), that attacks multiple pathways of neuronal cell life, inducing dysfunction, neuronal cell death and neuroinflammation, and 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.
Recent reports suggest high endogenous variability of Aß peptides between subjects. Therefore, the company decided to use subjects as their own controls. Amnestic MCI patients will receive Posiphen® at 240 mg/ day for 10 days (4×60 mg was found in a multiple dose safety study to be well tolerated in elderly healthy volunteers). Serial CSF samples will be collected via indwelling lumbar catheter for 12 hours one day before the start of dosing and after the last dose. Plasma samples will be taken at the same sampling times. CSF and plasma/serum samples will be analyzed for Posiphen®, metabolites and soluble a and ßAPP, Aß 40/42 and other AD associated markers.
“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,” said Maria Maccecchini, CEO of QR Pharma.
This study will show that Posiphen® goes into the brain and further elucidate what the drug does in the brain leading to proof of mechanism and optimization of dose finding.
Hal Broderson, QR Pharma Board Member commented: “Inhibiting APP is a novel target for a very serious disease, and I look forward to the further development of these promising, experimental drugs”.
About QR Pharma, Inc
About QR Pharma, Inc.: Headquartered in Radnor, Pennsylvania, QR Pharma, Inc. is a clinical-stage specialty pharmaceutical company committed to developing therapeutics with novel approaches for the treatment of cognitive impairment and Alzheimer’s disease (AD). QR currently has two product development programs based on oral small-molecule, blood-brain barrier passable therapeutics that target two distinct pathways for the treatment of AD.
Contact: Maria L. Maccecchini, PhD
Tel: 484 253 2296
Fax: 484 253 2288