Pharmaceutical Business review

QR Pharma Receives US Patent To Treat Down Syndrome

The patent expands upon the original Posiphen and analog patent that claims composition of matter and use of compounds to lower amyloid-ß precursor protein (APP) and thereby treat dementia and alzheimer’s disease (AD).

Posiphen, a small orally active compound, has been shown in cell cultures as well as in normal, transgenic (model of AD) and trisomic (model of DS) mice to reduce the synthesis of APP which is cleaved into a number of toxic peptides, including amyloid-ß42 (Aß42). These peptides, independent of each other, attack multiple pathways of neuronal cell life inducing dysfunction, neuronal cell death and inflammation and leading to cognitive impairment.

Trisomic mice treated with Posiphen show preservation of specific brain regions that consistently degenerate without treatment. Posiphen can normalize APP levels and preserve neuronal cells which opens a window for hope in DS.

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. Posiphen is presently in a clinical trial in AD patients and we are very hopeful to see the results.”