Alzheimer's disease (AD), a very debilitating and the most common dementing disorder of the elderly, represents the sixth leading cause of death in the United States and continues to rise in incidence (Stone et al., 2011). Its major hallmarks are neurodegeneration, synaptic loss, brain shrinkage and abnormal protein deposition, particularly of amyloid plaques and neurofibrillary tangles that derive from amyloid-ß peptide (Aß) and phosphorylated tau, respectively (Sambamurti et al., 2002, 2006; Selkoe 2011). AD neurochemical changes result in severe cognitive decline, a reduced quality of life and ultimately death. Currently approved AD drugs provide symptomatic relief and temporary slow cognitive decline, but do not halt or modify disease progression (Popp & Arlt, 2011).
QR Pharma is developing two clinical compounds: Posiphen inhibits the translation of amyloid precursor protein (APP) and α-Synuclein (aSyn) and has the potential to stop or slow the progression of the disease; it is going into phase II human efficacy trials. Bisnorcymserine (BNC), due to its mechanism of action of inhibiting butyrylcholinesterase, has the potential to prolong symptomatic efficacy in moderate to severe AD patients, upon the decline in effectiveness of existing acetylcholinesterase inhibitor drugs.
| Posiphen® | APP/Aß and aSyn translational inhibition for early AD and PD Phase II clinical stage |
| BNC | Butyrylcholinesterase inhibition for moderate to severe AD IND Phase I clinical stage |
To further the existing pipeline, QR Pharma is partnering with the NIH laboratory of Dr. Nigel Greig, the founder of the present technology at NIA/NIH, and with Dr. Jack Rogers, Massachusetts General Hospital, on new approaches to AD, PD and other neurological diseases. The company will also consider other candidate approaches or compounds to fill its pipeline.