CNM-Au8 is an investigational therapy being developed by Clene Nanomedicine for Parkinson’s disease and other conditions, including amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS).

The U.S. Food and Drug Administration (FDA) granted CNM-Au8 orphan drug status for the treatment of ALS.

How does CNM-Au8 work?

In Parkinson’s disease, dopaminergic neurons in areas of the brain called the striatum and substantia nigra start to die when mitochondria — the energy powerhouses of the cell — fail to provide enough energy for their activity. Another factor contributing to the death of dopaminergic neurons is oxidative stress caused when cells are unable to detoxify the harmful free oxygen radicals produced during metabolic reactions in which mitochondria play an important role.

CNM-Au8 is a concentrated suspension of gold (Au) nanocrystals that helps to catalyze or drive various biological processes. CNM-Au8 catalyzes the conversion of nicotinamide adenine dinucleotide (NADH) to its oxidized form (NAD+), which is required for the production of a key energy molecule used by mitochondria called adenosine triphosphate (ATP). CNM-Au8 can also protect cells from oxidative stress, thanks to its antioxidant properties.

These characteristics of CNM-Au8 can help protect dopaminergic neurons and improve their survival, thereby slowing the progression of Parkinson’s disease.

CNM-Au8 in clinical trials for Parkinson’s disease

Pre-clinical studies in rat models of Parkinson’s disease showed that CNM-Au8 improved the survival of dopaminergic neurons, lowered intracellular oxidative stress, and improved mitochondrial function.

A Phase 1 clinical trial (NCT02755870) to determine the safety and tolerability of single and multiple doses of CNM-Au8 in 86 healthy male and female volunteers was completed in October 2016. The results showed that CNM-Au was safe and well-tolerated across all tested doses with no serious adverse events.

A Phase 2 open-label clinical trial (NCT03815916) called REPAIR-PD is currently recruiting about 24 participants with Parkinson’s disease in Texas. The aim of the trial is to study the safety, pharmacokinetics (movement in the body), and metabolic effects of CNM-Au8 in the brain. Clene Nanomedicine announced in December 2019 that the dosing of the first participant had begun. The trial is expected to be completed in July.

The company is currently pursuing funding to start another Phase 2 clinical trial, RESCUE-PD, in 2020 to further test CNM-Au8.

 

Last updated: Feb. 20, 2020

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Özge has a MSc. in Molecular Genetics from the University of Leicester and a PhD in Developmental Biology from Queen Mary University of London. She worked as a Post-doctoral Research Associate at the University of Leicester for six years in the field of Behavioural Neurology before moving into science communication. She worked as the Research Communication Officer at a London based charity for almost two years.