Nicotinic α6 program

Nicotinic acetylcholine (nAChRs) receptors are ligand-gated ion channels that are activated by acetylcholine under physiological conditions. The α6 subtype exhibits an extremely localized expression mainly confined to dopaminergic neurons in the area of the brain affected in Parkinson’s disease patients where they act as important regulators of dopamine signaling.

As a result of a focused screening campaign, Saniona has identified selective positive allosteric modulators (PAMs) of α6 containing receptors and furthermore demonstrated that these PAMs increase the affinity for acetylcholine. Given the restricted expression pattern of α6-containing nAChRs, selective PAMs could therefore provide a novel therapy to increase dopaminergic signaling in Parkinson’s disease patients. In addition, α6 selective PAMs have the potential to slow or stop neurodegeneration seen in Parkinson’s disease that could result in stabilization of symptoms and disease progression. The identified PAMs offer a novel approach to counteract degeneration of dopaminergic neurons in patients and could optimally be used as a disease modifying therapy against Parkinson’s disease.

Parkinson’s disease is a chronic and progressive neurological disorder that is characterized by well-known motor symptoms including tremors, stiffness of limbs, slowness of movements, and difficulties with posture and balance. In addition to motor symptoms, many Parkinson’s disease patients experience non-motor symptoms, including sleep disorders, sensory symptoms, depression and gastrointestinal symptoms. It is the second most common neurological disorder and more than five million people worldwide live with this disease.

In February 2016, The Michael J. Fox Foundation for Parkinson’s Research awarded Saniona a research grant of USD 590,700 to perform a chemical optimization of its nicotinic α6 modulators in order to identify compounds suitable to demonstrate activity in relevant animal models and to assess their potential neuroprotective effects. Saniona retains all rights to any potential products developed and commercialized from the program. The program is in the drug discovery phase.

As the world’s largest non-profit funder of Parkinson’s research, The Michael J. Fox Foundation (MJFF) is dedicated to accelerating a cure for Parkinson’s disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson’s patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding more than $800 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson’s research, the Foundation forges ground-breaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson’s disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson’s awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world.