Argenica Therapeutics (ASX:AGN) AI reanalysis sharpens efficacy signal for ARG 007 in severe stroke cohort

Argenica Therapeutics has released an AI driven reinterpretation of its Phase 2 clinical dataset, producing the clearest evidence to date that ARG 007 delivers statistically significant and clinically meaningful benefit in patients presenting with more severe acute ischaemic stroke.

By applying the FDA cleared Brainomix 360 Stroke platform to standardise baseline severity through automated e ASPECTS scoring and volumetric assessment, the company has removed the variability inherent in manual stroke grading and revealed treatment effects that were previously obscured by an imbalance in baseline severity between study arms.

The analysis points decisively to a population with substantial early brain injury as the most responsive to neuroprotection.

Dr Liz Dallimore, Managing Director of Argenica Therapeutics, addresses key investor considerations arising from the new findings and outlines the implications for the company’s development trajectory.

How does the AI enabled reanalysis reshape the interpretation of the Phase 2 trial outcomes for ARG 007?

When we first presented the topline data and follow up functional data, the data analysis was completed using a statistical analysis model that included an assessment of stroke severity (APECTS) that was generated by the hospital sites. We now know that this classification by sites was significantly skewed in its accuracy, which resulted in the true treatment effect of ARG-007 being blurred.

Essentially, we were not comparing apples with apples.

The incorporation of Brainomix AI tools has materially enhanced our ability to interpret the Phase 2 dataset because it provides a standardised and highly reliable measure of baseline stroke severity, which can be inputed into the statistical analysis to determine the true treatment effect.

Manual ASPECTS scoring, although widely used, is known to produce variability between clinicians, and this variability can dilute treatment signals in neuroprotection studies that rely on precise assessment of infarct burden.

When stroke severity was reassessed using automated e ASPECTS and volumetric measures, it became clear that the ARG 007 arm had enrolled a higher proportion of patients with more severe strokes (lower ASPECTS and higher baseline infarcts), which inevitably influenced the topline results – skewing the data to show no treatment effect.

Once this imbalance was accounted for, the analysis revealed statistically significant improvements in functional outcomes and reductions in follow up infarct volume in patients with larger infarct cores.

This provides a far more accurate representation of ARG 007’s therapeutic impact and shows that the drug’s activity aligns with the level of early brain injury, which is fundamental to understanding neuroprotective potential.

ARG 007 demonstrates a treatment effect in severe large core LVO patients undergoing thrombectomy, highlighting its potential to become the first adjunctive neuroprotective therapy to improve outcomes in the highest need and highest value segment of the stroke market.

What magnitude of functional improvement was observed in severe stroke patients treated with ARG 007, and why is this particularly relevant?

The AI supported analysis allowed us to focus specifically on patients with more extensive infarct cores at baseline (i.e. when they first present to the emergency department prior to treatment), and in this group we observed statistically significant improvements at clinically meaningful intervals.

At 24 hours, ARG 007 treated patients demonstrated lower NIH Stroke Scale (NIHSS) scores than those receiving placebo, with a p value of 0.011. At 90 days, they also achieved significantly lower disability levels as measured by the modified Rankin Scale, with a p value of 0.005.

Subgroup analyses provided further granularity. Patients with e ASPECTS ≤6 experienced a statistically significant shift toward more favourable disability outcomes following treatment. A similar positive trend was seen in those with e ASPECTS ≤7.

These groups represent a substantial portion of the Phase 2 cohort, and they reflect the patients who typically experience the highest levels of disability and dependency despite successful reperfusion.

Functional outcomes measured by the NIH Stroke Scale at 24 hours and the modified Rankin Scale at 90 days were significantly improved compared to placebo.

The clinical relevance is straightforward: these are patients for whom current treatment options remain limited, and demonstrating improved independence after severe stroke addresses one of the largest unmet needs in modern stroke care.

What does the AI driven imaging analysis reveal about ARG 007’s capacity to reduce infarct progression?

The imaging analysis provides strong mechanistic reinforcement, showing that the magnitude of ARG 007’s effect increases in line with baseline infarct burden.

A statistically significant interaction was observed between baseline acute infarct volume and treatment effect, indicating that patients with larger initial cores, or more severe stroke on presentation to the emergency department, derived the greatest benefit. At an AIV of 30 ml, follow up infarct volume was significantly smaller in ARG 007 treated patients, with a p value of 0.025.

Corrected ischaemic lesion volumes showed a similar reduction, with a p value of 0.034.

Patients with minimal baseline core volumes did not demonstrate a treatment effect, which is consistent with clinical understanding that these patients often experience limited secondary injury once reperfusion is achieved.

The findings therefore align closely with the biological rationale for ARG 007, which targets secondary tissue injury that progresses despite vessel reopening.

ARG 007 treatment also resulted in significantly smaller final infarct volumes in patients with larger infarct cores at baseline.

These results provide a coherent picture in which functional benefit and imaging benefit reinforce one another.

What strategic implications arise from identifying severe stroke patients as the cohort with the strongest response?

Recognising that patients with severe acute ischaemic stroke experience the greatest therapeutic gain allows us to pursue a precision medicine strategy that is both clinically rational and commercially compelling.

Severe strokes represent a major global unmet need, and the Phase 2 dataset indicates that around 20% of all AIS patients fall into the segment where ARG 007 has demonstrated clear benefit.

This cohort also represents a high cost and high disability burden, which strengthens the health economic rationale for a therapy that can improve functional outcomes.

By enriching future studies for these patients, we can design a Phase 2b trial that maximises the probability of success while ensuring that the clinical findings translate directly into the group where treatment impact is most meaningful.

This approach aligns the program with a clearly defined, high value patient population supported by robust epidemiological and clinical evidence.

Analysis confirms ARG 007’s strongest benefit in more severe stroke patients who have the greatest need for neuroprotection, unlocking a significant commercial opportunity.

How will these new findings shape the next stage of ARG 007’s clinical development?

The Company is now advancing planning for a later stage clinical study that incorporates AI derived severity measures into screening and patient selection. The standardised ASPECTS as well as acute ischemic volume (AIV) analysis was only approved by the FDA in April 2025.

Therefore, the Company did not have the option to embed this into the execution of the Phase 2 trial. These AI tools are now being used more and more in clinical standard of care workflows, allowing Argenica to capitalise on their use to ensure greater accuracy in patient selection and data analysis in the future Phase 2b trial.

The alignment between imaging findings and functional outcomes provides the confidence needed to proceed with a more targeted Phase 2b design.

Current efforts are focused on assessing the specialised clinical, regulatory, operational and commercial capabilities required to execute a study of this scale in a high value patient group.

The integration of the Brainomix analysis will guide decisions around inclusion criteria, imaging endpoints and trial architecture, ensuring that the next stage of development is optimised for detecting the full therapeutic effect of ARG 007.

The company will continue to provide updates as preparations progress.

AI driven clarity strengthens the clinical and commercial pathway.

The AI based reinterpretation of the Phase 2 dataset has provided Argenica Therapeutics with a sharper, more precise understanding of where ARG 007 delivers the greatest therapeutic benefit and commercial value.

By revealing statistically significant improvements in neurological recovery, disability outcomes and infarct progression in patients with larger infarct cores, the analysis has confirmed a clear clinical rationale for progressing the drug into a more targeted late stage development program.

As Dr Liz Dallimore, Managing Director, notes, the convergence of functional and imaging outcomes offers a high degree of confidence as the company moves toward a precision designed Phase 2b study.

With a better defined target population and strengthened data foundations, Argenica is positioned to advance ARG 007 within a high need, high value segment of the global stroke market.Iscah

Please note the following valuable information before using this website. 

Independent Research 

Market Open Australia is intended to be used only for educational and informative purposes, and any information on this website should not be taken as investment advice or guidance. It is important to conduct your own research before making any investment decisions, which should be based on your own investment needs and personal circumstances. Any investment decisions based on information contained on this website should be taken in line with independent financial advice from a qualified professional or should be independently researched and verified.