Argenica Therapeutics’ ARG-007 (R18D) pre-clinical data shows 84% reduction in cellular uptake of Alpha-Synuclein (α-syn), a key hallmark linked to neurodegenerative diseases such as Parkinson’s and Alzheimer’s.

Argenica Therapeutics (ASX:AGN) also confirmed an inhibitory effect of ARG-007 on α-syn aggregation in a cell-free assay, with the inhibitory effect strengthening with increasing doses, resulting in the reduction of α-syn aggregation by an impressive 90%.

Data published in the scientific journal Biomedicines, from the laboratory of Argenica’s chief scientific officer Prof. Bruno Meloni, confirms ARG-007 significantly reduces the cellular uptake of α-syn protein aggregates into enteroendocrine cells, which are a key site of α-syn misfolding and aggregation.

Without the ARG-007 treatment, the paper confirms that the α-syn protein aggregates are readily taken up into cells.

Additional data recently generated in Prof Meloni’s laboratory also confirms a dose-dependent inhibitory effect of ARG-007 on α-syn aggregation in a cell-free assay.

The study tested three different concentrations of Argenica’s therapeutic to reduce human α-syn aggregation in an in-vitro model.

The study demonstrated that as the dose level of ARG-007 increased, so too did the inhibitory effect on α-syn increase, with the highest level (20 μM concentration) of ARG-007 tested showing a reduction of α-syn protein aggregation by 90% compared to untreated controls.


ARG-007 inhibits α-synuclein protein aggregation in cell free assay. Pic: Supplied (AGN).


Parkinson’s hallmarks

One of the key hallmarks of Parkinson’s Disease is the accumulation of aggregates of α-syn (known as Lewy bodies) in neurons. Its immunoreactivity is a highly specific biomarker for Parkinson’s Disease, confirming ARG-007 reduces both cellular uptake and aggregation of α-syn.

Argenica MD Dr Liz Dallimore said the new data is even more encouraging when you consider it with the company’s Abeta data previously announced.

“The scientific community now understands that neurodegenerative diseases are extremely complex, however the aggregation and accumulation of several proteins in the brain appear to be an important contributor,” Dallimore says.

“The ability of a therapy such as ARG-007 to work on a number of these protein aggregates is very important from a scientific perspective, and we look forward to progressing preclinical studies in this area further.”

Argenica says it will continue to progress pre-clinical studies of ARG-007 in Alzheimer’s Disease, with data from a mouse model expected early next year.

“Further consideration will now also be given to potential Parkinson’s Disease animal studies that Prof. Meloni can initiate,” the company stated.



This article was developed in collaboration with Argenica Therapeutics, a Stockhead advertiser at the time of publishing.


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