Argenica Advances Stroke Trial and Secures FDA Orphan Status for ARG-006

Progress in Phase 2 Stroke Trial

Argenica Therapeutics Limited (ASX: AGN) has reported significant progress in its Phase 2 clinical trial of ARG-007, targeting acute ischaemic stroke (AIS) patients. By the end of December 2024, 74 out of 92 patients had been dosed, representing 80% of the trial cohort. Recruitment remains on track to complete dosing in early Q2 2025, with 79 patients dosed as of late January.

The trial’s Data Safety Monitoring Board (DSMB), comprising independent neurologists and a biostatistician, has reviewed safety data multiple times with no serious adverse events linked to ARG-007. The DSMB’s final meeting in January 2025 endorsed continuation of the study as per protocol, marking a key milestone in the trial’s progress.

Regulatory Milestones: FDA Designations for ARG-006

In parallel with clinical progress, Argenica secured Orphan Drug and Rare Pediatric Disease designations from the US Food and Drug Administration (FDA) for its second drug candidate, ARG-006. This candidate targets Hypoxic Ischaemic Encephalopathy (HIE) in term newborn infants, a serious neurological condition. These designations provide regulatory incentives similar to those granted for ARG-007, enhancing the company’s strategic options for clinical development in neonatal brain injury.

Argenica is also preparing an Investigational New Drug (IND) application for ARG-007 to enable future US clinical trials. The company is considering submitting a Fast Track application alongside the IND to expedite FDA review and facilitate early regulatory engagement.

Expanding Potential: Preclinical Data in Neurodegenerative Diseases

Beyond stroke and neonatal indications, Argenica continues to explore ARG-007’s therapeutic potential in neurodegenerative diseases. Recent preclinical studies in Alzheimer’s and Parkinson’s disease models have reinforced ARG-007’s multi-modal mechanism of action. The drug demonstrated neuroprotective effects, reduced inflammation, and inhibited pathological protein aggregation, key drivers of disease progression.

While encouraging, the Alzheimer’s disease in vivo studies revealed suboptimal dosing due to tissue hardening at injection sites, prompting plans to investigate alternative delivery routes such as oral and nasal administration to enhance brain bioavailability.

Financial Position and Leadership Strengthening

Argenica closed the quarter with $15.06 million in cash reserves, bolstered by a $2.75 million R&D tax incentive refund and positive operating cash flow. This funding position supports completion of the Phase 2 trial, ongoing preclinical research, and preparatory activities for a potential Phase 3 stroke trial.

The company also enhanced its leadership team with the appointment of Dr Jeannie Joughin as a Non-Executive Director, joining recent additions Dr Mark Etherton, Mr Rob Black, and Dr Stuart Gribble. These appointments bring deep expertise in neurology drug development, commercialization, and pharmaceutical operations, positioning Argenica for its next growth phase.

Outlook

With clinical milestones advancing and regulatory incentives secured, Argenica is well-positioned to progress ARG-007 and ARG-006 through critical development stages. The company’s expanding pipeline and robust cash position provide a solid foundation for future value creation in the competitive neurotherapeutics landscape.