Asceneuron: 2nd grant from Michael J. Fox Foundation for Parkinson therapeutics

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.”