Argenica Completes Phase 2 Dosing for ARG-007 Stroke Trial, Eyes FDA Fast Track

Phase 2 Trial Milestone Achieved

Argenica Therapeutics Limited (ASX: AGN) has reached a significant milestone in its development of ARG-007, a novel neuroprotective peptide aimed at reducing brain tissue death after stroke. The company announced the completion of patient dosing in its Phase 2 clinical trial for acute ischaemic stroke (AIS) patients, with a total of 92 participants enrolled across Australian hospitals. This trial focuses on patients with large vessel occlusion strokes undergoing endovascular thrombectomy, a critical subgroup with high unmet medical need.

The trial remains double-blinded, with half the patients receiving ARG-007 and the other half a saline placebo. The final 90-day functional assessments are underway, after which the data will be unblinded and analysed. Argenica anticipates releasing topline results in the third quarter of calendar year 2025, which will provide early insights into the safety and preliminary efficacy of ARG-007 in this patient population.

Safety Endorsed by Independent Board

Throughout the trial, an independent Data Safety Monitoring Board (DSMB) has overseen patient safety and study progress. The DSMB recently conducted its final review of safety data from the first 76 patients dosed, representing 83% of the trial cohort, and recommended that the study continue without any modifications to the protocol. This endorsement is a positive signal that ARG-007 is well tolerated in the acute stroke setting, a critical hurdle for any neuroprotective agent.

Expanding ARG-007’s Potential Beyond Stroke

In parallel with the clinical trial, Argenica has advanced preclinical research exploring ARG-007’s efficacy in other neurological conditions, notably traumatic brain injury (TBI). Recent data from a larger rat model study demonstrated that ARG-007 effectively prevents axonal injury and neuroinflammation, key drivers of both acute and chronic brain damage following TBI. These findings corroborate earlier pilot studies and suggest ARG-007’s neuroprotective effects may extend beyond stroke, potentially broadening its therapeutic scope.

Regulatory Pathway and Financial Position

Looking ahead, Argenica is preparing to submit an Investigational New Drug (IND) application to the US Food & Drug Administration (FDA) in early May 2025. This submission, accompanied by a Fast Track designation request, aims to facilitate expedited development and regulatory review in the US market. Securing FDA approval for clinical trials stateside would be a pivotal step toward global development and commercialization.

Financially, Argenica reported cash reserves of $12.9 million as of 31 March 2025, sufficient to fund the completion of the Phase 2 trial and the regulatory submission process. The company’s quarterly operating cash outflow was $2.154 million, reflecting ongoing investment in research, clinical activities, and corporate functions. Non-dilutive grants exceeding $4 million have also supported the company’s neurological research programs.

Outlook

With patient dosing complete and safety affirmed, all eyes will now be on the upcoming topline data expected in Q3 2025. Positive results could validate ARG-007’s potential as a first-in-class neuroprotective therapy for stroke and possibly other brain injuries. Meanwhile, the FDA IND submission and Fast Track application mark critical regulatory milestones that could accelerate ARG-007’s path to market.