By Dr Liz Dallimore, Neuroscientist, Nedlands
Acute stroke remains a leading cause of death and disability worldwide, where rapid intervention can mean the difference between recovery and permanent brain damage.
While thrombolysis and thrombectomy remain the foundation of ischaemic stroke treatment, a West Australian-developed neuroprotective drug may soon offer clinicians a new tool to preserve brain tissue in the crucial early hours as well as after treatment.
The current acute stroke workflow in emergency departments prioritises rapid recognition and imaging, with non-contrast CT and CT angiography used to assess eligibility for reperfusion therapies.
Patients with a confirmed large vessel occlusion (LVO) may receive intravenous thrombolysis within 4.5 hours of onset, and/or endovascular thrombectomy up to 24 hours of onset, per imaging criteria.
These treatments are highly time-sensitive and often inaccessible to patients in regional or remote areas, where delays in transfer may result in missed intervention windows. Even when performed promptly, reperfusion injury can contribute to ongoing neurological damage.
A new agent
A new neuroprotective peptide drug is currently being developed by a Perth biotechnology company, based on a decade of research from the Perron Institute and UWA.
Unlike existing therapies that target clot removal, this new drug is designed to protect brain cells from damage during and after ischaemia, offering a novel approach to stroke care.
Administered via intravenous infusion, the drug can be given prior to reperfusion therapy, potentially in emergency departments or even pre-hospital settings.
Its aim is two-fold:
- Preserve the ischaemic penumbra while awaiting reperfusion, potentially extending the time-window for current treatments
- Mitigate damage from post-treatment oxidative stress and inflammation.
This mechanism makes it complementary – not competitive – with existing standards of care, fitting ideally into current stroke treatment workflows.
Current and planned research
In April 2025 patient dosing in a Phase Two clinical trial of the peptide drug in acute ischaemic stroke patients was completed across multiple Australian stroke centres, including Sir Charles Gairdner Hospital and Fiona Stanley Hospital in WA.
The double-blind trial involved 92 AIS patients with LVO, each receiving the drug or placebo prior to undergoing thrombectomy. The key endpoints included:
- Safety – mortality, symptomatic intracranial haemorrhage, other serious adverse events
- Infarct volume reduction, assessed on imaging at 48 hours
- Functional outcomes, measured at 90-day follow-up
Trial data is currently blinded, however, all results are expected in September this year.
Following successful results from the Phase Two trial, the drug trials will progress to a Phase 2b/3 clinical trial. Preparation is underway for this trial, which is expected to recruit around 1000 patients across Australia, the US, Europe, Canada, and China, commencing in mid to late 2026.
RELATED: Perth stroke treatment hits phase 2
It will evaluate efficacy across a broader population and refine patient selection criteria. If proven successful, it could mark the world’s first clinically approved neuroprotective drug for acute stroke.
The development of this novel drug underscores WA’s leadership in translational neuroscience. The drug has already shown up to 70% reduction in brain cell death in preclinical studies.
The potential applications may extend beyond stroke, with ongoing preclinical research into traumatic brain injury (TBI), hypoxic ischaemic encephalopathy (HIE), and neurodegenerative conditions.
Key messages
- A new drug aims to extend the treatment window by protecting brain tissue until reperfusion can occur, as well as protect the brain from reperfusion injury post thrombectomy.
- As a pre-thrombectomy adjunct, the drug supports, not replaces, current ED stroke pathways
- Should Phase Two prove successful, a global Phase 2b/3 trial will commence with a focus on functional outcome measures.
Author competing interests – the author is the Managing Director of Argenica Therapeutics and is actively involved in the drug development and clinical research.
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