QR Pharma commences clinical trial of Posiphen in early stage AD patients

QR Pharma, Inc. (QR), 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.

“Inhibiting APP is a novel target for a very serious disease, and I look forward to the further development of these promising, experimental drugs.”

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-β precursor protein (APP) which is cleaved into a number of toxic peptides. These peptides include amyloid-β₄₂ (Aβ42), 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 Aβ 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 (4x60 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 α and βAPP, Aβ 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.”