Argenica Therapeutics (ASX:AGN) delivers promising functional recovery trends in ARG-007 Phase 2 stroke trial

Argenica Therapeutics (ASX:AGN) has reported encouraging results from its Phase 2 clinical trial of ARG-007, a neuroprotective therapy in development to improve recovery outcomes for patients following acute ischaemic stroke.

The study, which primarily evaluated the safety of a single dose of ARG-007, also incorporated exploratory functional endpoints that measured cognitive performance, independence in daily living, and quality of life.

Although the trial was not powered for statistical significance in these functional outcome measures due to its small sample size, the data showed consistent and positive trends across all major functional measures, with ARG-007 patients demonstrating stronger recovery trajectories than those in the placebo group.

Dr Liz Dallimore, Managing Director of Argenica Therapeutics, expands further on the findings and their implications for the company’s next phase of clinical development, including the design of a targeted Phase 2b trial focused on confirming efficacy across key functional endpoints.

What were the most important findings from the Phase 2 clinical trial of ARG-007?

The Phase 2 trial was primarily a safety study. Demonstrating safety in neurology is critical – it is essentially a go/no-go decision in neurology drug development. The fact that we have shown ARG-007 is well tolerated in stroke patients allows us to move into pivotal efficacy studies. However, we also wanted to determine whether the drug could change the functional outcomes of stroke patients, and very pleasingly the trial demonstrated encouraging functional improvements in patients treated with ARG-007 compared to those who received a placebo.

These improvements trends were evident across cognition, daily living independence, and overall quality of life all of which are critical and validated indicators of stroke recovery.

At day 90, 58.3 percent of ARG-007 patients achieved normal cognitive function on the Montreal Cognitive Assessment (MoCA-30), compared with 35.3 percent in the placebo group, suggesting a clinically relevant effect.

This test evaluates memory, executive function, and language, with scores above 22 representing preserved or improved cognition.

When assessed using the Barthel Index, which measures independence in daily activities such as walking, dressing, and feeding, the proportion of ARG-007 patients achieving functional independence (score >90) increased by 13.3 percent from day 30 to day 90.

In contrast, the placebo group declined by 8.7 percent over the same period.

Patients also reported a higher perceived quality of life, with ARG-007 recipients scoring a median of 80 on the EQ-5D-5L scale at day 90, compared to 72.5 for placebo.

While the study was small and exploratory in nature, these consistent trends indicate a positive treatment effect that warrants further evaluation in a larger, adequately powered study.

Why were functional outcomes considered more relevant than imaging results such as infarct volume?

Functional outcomes are the measures that most directly reflect a patient’s real-world recovery – patient reported outcomes are what matters most.

They assess how well an individual can think, move, and live independently after a stroke, which provides a far clearer picture of clinical benefit than imaging data alone.

“Functional outcomes are the measures that most directly reflect a patient’s real-world recovery. They assess the ability to think clearly, move independently, and return to daily activities, outcomes that matter most to patients and clinicians.”

While imaging studies such as infarct volume remain useful as supportive evidence, they cannot fully capture how a patient functions in daily life.

Whilst infarct volume is a good predictor of functional outcomes, it is possible for two individuals with similar infarct sizes to have vastly different outcomes in cognition and mobility. This is because the brain injury can affect different regions of the brain which will lead to different outcomes. Further, stroke patients can be significantly impacted by secondary injury as a result of inflammation and deterioration of the penumbra in the brain, also leading to worse functional outcomes. A neuroprotective drug that reduces inflammation or promotes survival in the penumbra will not impact infarct volume, but will impact functional outcomes.

Therefore, regulatory agencies, including the FDA, prioritise validated, patient-centred functional endpoints at day 90 as the primary criteria for assessing stroke therapies.

By focusing on measures like the MoCA, Barthel Index, Modified Rankin Scale, and EQ-5D-5L, Argenica is aligning its approach with global clinical and regulatory expectations that value meaningful improvements in patient outcomes over radiographic changes.

How do these results shape the design of the upcoming Phase 2b trial?

The results from this study provide a strong foundation for shaping the next phase of development.

The upcoming Phase 2b trial will be specifically designed to detect measurable improvements in functional outcomes, particularly cognitive performance and independence in daily living, the areas where ARG-007 demonstrated the most pronounced benefit. We will work closely with the FDA to ensure these endpoints are appropriately designed.

The new trial will incorporate insights from subgroup analyses, including findings in patients with slow collateral blood flow, where encouraging signals of efficacy were observed. As well as enhancing patient selection and stratification through the use of automated imaging during patient recruitment.

Argenica will work closely with its global Clinical Advisory Group, research partners, and regulators to ensure that the Phase 2b study design is both clinically relevant and operationally feasible.

By concentrating on endpoints that reflect tangible improvements in recovery, the trial aims to confirm ARG-007’s impact in a larger population and provide the data required for future regulatory discussions.

What do these findings mean for potential pharmaceutical partnerships?

These results demonstrate that ARG-007 is producing human clinical data showing trends toward better recovery outcomes, an important step that increases its attractiveness to potential pharmaceutical partners.

Even as a smaller, exploratory study, the consistent positive direction across all functional endpoints, including cognition, independence, and quality of life indicates a meaningful biological effect.

This aligns well with what larger pharmaceutical companies seek in early-stage neuroprotective assets: strong safety data, measurable patient benefit, and a clear pathway toward a registrational study.

The upcoming Brainomix imaging analysis, which will complement the existing functional data, is expected to add further value for partners looking for well-supported clinical candidates in stroke and neuroprotection.

What are the next steps and expected timelines for the program?

Argenica’s immediate next step is to complete the Brainomix analysis of patient brain imaging data, expected by the end of this calendar year, as well as finalising the clinical study report.

This analysis will provide deeper insight into how ARG-007 may be influencing neurological recovery.

Following this, the company will finalise the Phase 2b trial protocol in collaboration with clinicians, regulatory agencies, and prospective pharmaceutical partners.

The upcoming study will target specific functional endpoints, ensuring it captures the clearest and most relevant measures of patient benefit.

Once the design is approved, Argenica plans to progress swiftly into trial commencement, with the aim of confirming the promising efficacy signals observed in the current dataset and laying the groundwork for a potential registrational Phase 3 trial.

Looking Ahead: Advancing ARG-007 Toward Broader Clinical Validation

Dr Liz Dallimore, Managing Director of Argenica Therapeutics, said the company’s latest findings mark an important step in the ongoing development of its lead neuroprotective candidate.

“These positive trends in functional outcomes, that appear to be clinically meaningful and across all assessments, are extremely exciting given the small numbers of patients in this Phase 2 trial.”

“On the strength of these data, as well as the signal we see in slow collateral patients, we plan to advance a targeted Phase 2b.”

“We anticipate that this Phase 2 data will be of interest to potential pharmaceutical partners.”

Argenica is now focused on using these results to refine the design of its next trial and to further validate ARG-007’s impact on recovery outcomes.

The company believes that its emphasis on functional improvement, cognition, independence, and quality of life, provides the most meaningful pathway to demonstrate the drug’s value both clinically and commercially.

With strong safety data, consistent functional improvement trends, and continued collaboration with global experts, Argenica is well positioned to progress toward a new generation of stroke recovery therapies that improve real-world outcomes for patients.

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