Argenica reports encouraging follow-up results from Phase II stroke study

• Argenica’s ARG-007 shows promising improvements in functional outcomes in Phase II stroke trial
• ARG-007 treated patients showed efficacy trend over placebo in exploratory functional endpoints
• On strength of data, Argenica plans to design and advance a targeted Phase 2b trial

Special Report: Perth-based biotech Argenica Therapeutics, which is developing treatments to reduce brain tissue death after stroke, plans to advance to a Phase 2b trial of its drug candidate ARG-007. 

Argenica Therapeutics (ASX:AGN) today reported positive improvements to functional outcomes after analysis of unblinded data of the Phase II trial of ARG-007 in acute ischemic stroke (AIS) patients.

The plan to advance to a Phase 2b trial of ARG-007 follows completion of the company’s Phase II study, which met primary safety endpoints but did not achieve efficacy measures.

The company first reported results from the 92-patient trial on September 3, but it is today’s update that should have investors feeling particularly encouraged.

The data pointed to consistent trends favouring ARG-007 treatment across multiple prespecified exploratory functional endpoints.

The primary endpoint of the Phase II trial was safety of a single ARG-007 dose in AIS participants.

Argenica said detailed analysis of data at days 30 and 90 has provided valuable insight into ARG-007’s potential benefit in ischaemic stroke, despite the small trial size and its design not being powered for statistical significance.

Post-stroke cognitive impairment

Cognitive impairment was assessed in trial participants using the validated Montreal Cognitive Assessment (MoCA) test.

The MoCA test showed that 58.3% of ARG-007 patients scored greater than 22 at Day 90, versus 35.3% of placebo patients.

This trend toward improved cognition suggests clinically meaningful benefits warranting confirmation in a larger study.

Independent daily living and quality of life

Patients were also assessed using the Barthel Index – a validated clinical scale used to measure a person’s functional independence in performing basic activities of daily living following neurological injury such as stroke.

The scale ranges from 0 to 100, with higher scores indicating greater independence and functional recovery.

Analysis showed functional independence (a score of 90 or more) from day 30 to 90 improved 13.3%, while placebo patients decreased 8.7%.

Furthermore, 51% of ARG-007 patients achieved functional independence at Day 90, compared with 43% of placebo.

On the Stroke Impact Scale (SIS), patients treated with ARG-007 showed a 10% improvement from day 30 to day 90, compared with just 2% in the placebo group. At day 90, the ARG-007 group recorded a median SIS score of 90%, versus 80% in the placebo group.

Patients also reported their overall health using the EQ-5D-5L visual analogue scale, which measures self-rated health on a scale from 0 (worst imaginable) to 100 (best imaginable).

ARG-007 treated patients reported a median score of 80 at day 90, compared with 72.5 for placebo patients.

Taken together, Argenica said these measures show a consistent positive trend favouring ARG-007 in patients recovering from acute ischaemic stroke.

Improvement in disability

The trial also assessed physical disability and functioning using the Modified Rankin Scale (mRS) – the standard measure of disability used in stroke trials, ranging from 0 (no symptoms) to 6 (death).

On the mRS 0–2 (functional independence) the number of ARG-007 patients improved by 6.6% versus a 4.3% reduction in placebo from Day 30 to Day 90.

While Day 90 mRS showed no meaningful difference, other functional measures highlight subtler, clinically important gains not captured by mRS which warrant pursuing a larger study powered for functional outcomes.

Watch: Liz Dallimore unpacks the Phase II trial results

Relevance of functional outcome measures

Argenica said functional outcomes, including cognition, disability, independence and quality of life at Day 90, are what matter most to patients and regulators, with imaging supporting but not defining efficacy.

Trends in ARG-007 data suggest preserved neuronal function, reduced secondary injury, and enhanced neuroplasticity, particularly in patients with prolonged ischemia risk.

Argenica said the combined functional outcome and imaging analyses will inform its Phase 2b trial design, focusing on endpoints where ARG-007 shows efficacy.

The trial protocol will be developed in consultation with clinicians, CROs, regulatory agencies, and potential pharmaceutical partners.

Argenica CEO and managing director Dr Liz Dallimore said the positive trends across multiple functional outcomes were “extremely encouraging” given the small Phase II cohort.

“On the strength of these data and signals in slow collateral patients, we plan to advance a targeted Phase 2b trial,” she said.

“We anticipate these results will be of interest to potential pharmaceutical partners.”

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

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.