Asceneuron Awarded Second Grant from The Michael J. Fox Foundation for Accelerated Research into Novel Parkinson's Disease Therapies

LAUSANNE, Switzerland and SAN FRANCISCO, May 4, 2022 /PRNewswire/ -- Asceneuron SA, a clinical stage company dedicated to targeting the root causes of neurodegenerative diseases, today announces that it has been awarded a second grant from The Michael J. Fox Foundation for Parkinson's Research (MJFF) to accelerate the study of its clinical stage O-GlcNAcase (OGA) inhibitor ASN51 as a potential new treatment for Parkinson's disease (PD).

Parkinson's disease is caused by a loss of dopamine producing nerve cells in the substantia nigra. This leads to a reduction in the signalling mediated by the neurotransmitter dopamine in the brain causing the characteristic motoric dysfunction in PD. An estimated seven to 10 million people worldwide suffer from PD. Although there is no current cure for PD, Asceneuron is discovering and developing therapeutics for the high unmet medical needs of patients experiencing neurodegenerative disorders.

The grant will fund a preclinical proof-of-concept study to assess the disease-modifying properties of Asceneuron's OGA inhibitor ASN51 in a preclinical model of inherited PD. The genetic model is characterized by the overexpression of a-synuclein harbouring the A53T mutation known to cause early-onset, familial PD in humans. Aggregated forms of a–synuclein are the main component of the characteristic Lewy body pathology and thus thought to be causative of the loss of dopaminergic neurons in PD.

The aim of this study is to extend previously published findings demonstrating a reduction of motor impairment with Asceneuron's OGA inhibitors (e.g., Permanne et al., ACS Chem. Neurosci. 2022) to this alternative genetic disease model. The results will further interrogate the mechanism of action of OGA inhibitors with respect to a–synuclein toxicity and aggregation and provide support for the clinical exploration of ASN51 in PD patients. The project will begin immediately, with results expected in the fourth quarter of 2022.

Dirk Beher, Chief Executive Officer, Co-Founder of Asceneuron, commented: "We are pleased to have been awarded further funding support from The Michael J. Fox Foundation to progress research on our next generation O-GlcNAcase inhibitor ASN51. ASN51 has the potential to make a meaningful impact on patients suffering with Parkinson's disease to improve quality of life and slow progression. We are proud to receive the continued support from a foundation that contributes meaningfully towards development of improved therapies for those living with Parkinson's disease."

"The Michael J. Fox Foundation is dedicated to unlocking the complex biology of Parkinson's disease and unearth novel breakthrough treatments to improve patient lives and slow disease progression," said Luis Oliveira, PhD, Senior Associate Director of Research Programs, MJFF. "We are pleased to award Asceneuron a grant to support further research in the O-GlcNAcase inhibitor, ASN51, as a potential modifier of a-synuclein toxicity."

About Asceneuron

Asceneuron is a clinical stage biotech company focused on the development of orally bioavailable therapeutics for debilitating neurodegenerative disorders with high unmet medical need. The pipeline reflects our ambition to develop treatments for a wide a range of neurodegenerative diseases including orphan tauopathies, Alzheimer's and Parkinson's disease. Asceneuron has two clinical stage small molecule O-GlcNAcase inhibitors in development for the treatment of proteinopathies including one first in class for Parkinson's disease, one best in class in Alzheimer's disease and related disorders.

Asceneuron is a privately held company financed by a renowned syndicate of investors consisting of Sofinnova Partners, M Ventures, SR One, Johnson & Johnson Innovation – JJDC, Inc. (JJDC) and Kurma Partners. For more information, please visit www.asceneuron.com.

About The Michael J. Fox Foundation for Parkinson's Research

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 $1.5 billion 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; creates a robust open-access data set and biosample library to speed scientific breakthroughs and treatment with its landmark clinical study, PPMI; 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. For more information, visit us at www.michaeljfox.org, Facebook, Twitter, LinkedIn.

About ASN51

Asceneuron's best-in-class program ASN51, a next-generation O-GlcNAcase inhibitor, was awarded USD 2.2 million from the Alzheimer's Drug Discovery Foundation for a first in human Phase I study. The trial in progress recruits healthy volunteers and Alzheimer's disease patients at sites in Europe and Australia and began in Q2 2021. Asceneuron will be presenting results at the upcoming conferences on safety, tolerability, pharmacokinetics, and human target engagement in healthy volunteers.