Biotechnology player Argenica Therapeutics has announced the latest positive preclinical data in hypoxic ischaemic encephalopathy (HIE) – which is when not enough oxygen or blood goes to a baby’s brain causing brain injury – with a single dose of the company’s ARG-007 drug has been shown to last out to four weeks.

ARG-007 has been shown to significantly reduce brain injury caused by both ischemia (reduced blood flow) and vasogenic oedema (brain swelling) in a full-term-equivalent animal model of HIE.

A 300nmol/kg dose of ARG-007 maintained over a 70% reduction in total brain injury from one week out to four weeks post injury and doses of 100 and 300 nmol/kg of ARG-007 also reduced vasogenic oedema (brain swelling) at 48 hours post injury by 35.4% and 32.9% respectively.

The prolonged effect of the drug is particularly important as HIE is an ongoing process with three waves of neuronal cell death occurring over hours to days after the initial event, so a neuroprotective drug must have a prolonged effect to give the brain the best chance of protection against the third wave of cell death.

A solid base to move into clinical trials

Argenica Therapeutics (ASX:AGN) is now in the process of completing preclinical studies required to initiate an Investigational New Drug Application with the US Food and Drug Administration (FDA).

MD Dr Liz Dallimore said the preclinical efficacy data is “extremely encouraging.”

“Whilst HIE is a rare paediatric condition, it has devastating outcomes for these babies, and a treatment is desperately needed,” she said.

“The fact that ARG-007’s effect in this preclinical model lasts out to four weeks indicates the treatment effect is sustained, protecting against the devastating third wave of brain cell death seen in HIE.

“This data, plus the additional data we will gather in larger animal studies, will provide a solid base to move into clinical trials for HIE.”

Argenica Therapeutics asx agn
Graph: Total Brain Injury (infarct volume from ischaemia and vasogenic oedema, mm3) in control (injury + saline) animals and treatment (ARG-007) animals. Following injury (ischaemia-hypoxia), treatment animals received ARG-007 at varying doses (nmol/kg). Following treatment, the 300 nmol/kg ARG-007 treated groups showed a significantly greater reduction in total brain injury compared with the saline control group (p=0.002).

Next trial phase planned in the US

AGN has engaged global contract research organisation Labcorp Drug Development’s (Labcorp) paediatric regulatory team to develop a regulatory and clinical trial strategy for ARG-007 in HIE in newborns.

And to meet requirements to undertake clinical trials in HIE in the US, AGN has initiated a preclinical juvenile toxicology study and preclinical efficacy studies in a large animal term model of HIE.

If these studies elicit positive results, then the company’s aim is to commence a Phase 1/2 trial in HIE in the US.

The results of the preclinical efficacy studies (funded by a grant from the Stan Perron Charitable Foundation) as well as the company’s engagement with the FDA, will be announced as they come to hand.





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


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