Asceneuron SA, an emerging leader in the development of innovative small molecules for neurodegenerative diseases, today announced it has been awarded a research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) for the further development of positive allosteric modulators of the M1 muscarinic acetylcholine receptor (M1 PAMs). M1 PAMs induce a change in the shape of the receptor, enhancing binding to the neurotransmitter acetylcholine. As a result, receptor activity is potentiated so that it can fulfil its signaling functions, critical for cognition, even in situations where acetylcholine levels are reduced, as observed in dementia.
The grant from the MJFF Therapeutic Pipeline Program will support studies for the optimization of molecules to provide preclinical proof of concept in a relevant laboratory model. Asceneuron has already identified suitable lead molecules and will use its expertise in the field of muscarinic acetylcholine receptors and CNS drug development to achieve this objective. As this important receptor is critically involved in learning and memory, M1 PAMs have the potential as novel and efficacious medications to treat cognitive deficits in Parkinson’s disease dementia patients.
Commenting on the award, Dirk Beher (PhD), chief executive officer and co-founder of Asceneuron, said: “We are very excited to advance this approach to treating an underserved need. We expect our understanding of the novel biological interactions between M1 PAMs and the M1 muscarinic acetylcholine receptor to yield a Parkinson’s dementia therapy with potentially greater selectivity, fewer side effects and longer durability of effect.”
Marco Baptista (PhD), director of research programs at MJFF, added: “M1 PAMs could provide a new treatment option for Parkinson’s dementia, a critical current unmet need. We believe Asceneuron is making promising progress toward this goal.”
About Parkinson’s Disease and Parkinson’s Disease Dementia (PDD)
Parkinson’s disease is a progressive, degenerative neurological movement disorder that affects approximately 6.3 million people worldwide. Although it typically develops after the age of 65, about 15% of people with the condition develop "young-onset" Parkinson's disease before reaching age 50.
As Parkinson's disease progresses, it becomes increasingly disabling, making daily activities like bathing or dressing difficult or impossible. Many of the symptoms of Parkinson's disease involve motor control, the ability to control your muscles and movement. Patients may also have problems with depression, sleep disruption and dementia. The cumulative prevalence of dementia can be as high as 78%, or 4.9 million sufferers globally, though it is most common in older patients. Neurochemically the most significant deficit in PDD is cholinergic which suggests that approaches focused on acetylcholine transmission and modulation may be effective treatment options.
About M1 PAMs
Positive allosteric modulators of the M1 muscarinic acetylcholine receptor (M1 PAMs) sensitize the receptor for its natural neurotransmitter acetylcholine. As a result, the M1 muscarinic receptor can still function in a situation where the release of acetylcholine is declining, as is observed in Parkinson’s and Alzheimer’s disease dementia. Since this receptor is critically involved in learning and memory, M1 PAMs have the potential to deliver novel and efficacious medications to treat cognitive deficits in Parkinson’s disease dementia patients as well as other dementia types including Dementia with Lewy Bodies (DLB) and Alzheimer’s disease.
Asceneuron is an emerging biotech company excelling in the development of orally bioavailable therapeutics for serious neurodegenerative disorders with high unmet medical needs such as orphan tauopathies, Alzheimer’s and Parkinson’s diseases. The lead product, an O-GlcNAcase inhibitor that in preclinical studies has been demonstrated to modulate tau pathology, is currently completing the critical regulatory studies to initiate human clinical testing. The O-GlcNAcase inhibitor is being developed for the orphan tauopathy progressive supranuclear palsy (PSP). Asceneuron is a privately held company financed by a strong syndicate of investors consisting of Sofinnova Partners, SR One, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), Kurma Partners and Merck Ventures. For more information, please visit www.asceneuron.com.
About The Michael J. Fox Foundation
As the world's largest nonprofit funder of Parkinson's research, The Michael J. Fox Foundation 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 $600 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 groundbreaking 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.