• US FDA confirmed Phase 2 trial protocol for AGN-007 is acceptable
  • The treatment has been shown to improve outcomes in pre-clinical stroke models
  • A Phase 2 trial is planned with patient dosing scheduled for Q1 CY24


The US Food and Drug Administration (FDA) has given Argenica Therapeutics positive feedback for its clinical development program of stroke treatment ARG-007 under a pre-investigational new drug type B meeting request (pre-IND meeting).

The company’s lead candidate ARG-007, a neuroprotective treatment for acute ischaemic stroke, has been successfully demonstrated to improve outcomes in pre-clinical stroke models, traumatic brain injury (TBI) and hypoxic ischaemic encephalopathy (HIE).

A Phase 1 clinical trial was recently completed in healthy human volunteers to assess the safety and tolerability of a single dose of ARG-007, and Argenica Therapeutics (ASX:AGN) sought feedback and guidance from the FDA on the appropriateness of the chemistry, manufacturing and controls (CMC), as well as feedback and guidance on the completed preclinical studies and proposed Phase 2 trial protocol.

The feedback was positive and confirmed the Phase 2 trial protocol was acceptable to assess preliminary efficacy (proof-of-concept) of ARG-007.


Confidence to pursue Phase 2 trial

The feedback provides Argenica further confidence as it progresses ARG-007 into a Phase 2 clinical trial in acute ischemic stroke patients, and ultimately into a Phase 3 trial in the US.

“The positive feedback we’ve received from the FDA is extremely encouraging and provides the company with great confidence as we progress ARG-007 into a Phase 2 clinical trial in acute ischaemic stroke patients at the end of this calendar year,” AGN MD Dr Liz Dallimore said.

“The feedback provides the company with a confirmed path for clinical development of ARG-007.”


Reducing brain tissue death after stroke

The company is focused on developing novel therapeutics to reduce brain tissue death after stroke and other types of brain injury and neurodegenerative diseases to improve patient outcomes.

For the Phase 2 trial, only patients with a diagnosed large vessel occlusion (LVO) stroke that are eligible for endovascular thrombectomy (mechanical removal of a clot in the brain) will be eligible to be enrolled.

LVO strokes account for close to 40% of all acute ischaemic strokes, however, are responsible for 60% of post-stroke dependency and 90% of mortalities after stroke, and therefore are considered the most devastating type of stroke.


Patient dosing Q1 CY24

The ethics application for Phase 2 has been submitted, and the drug-manufacturing process was initiated with vials expected to arrive at trial sites in Australia by mid-December 2023, ready for administration to patients as recruitment and dosing of patients commences in Q1 CY2024.

The company is also continuing to generate preclinical data in other neurological conditions, including in TBI, HIE and Alzheimer’s Disease.

In fact, ARG-007 has also been shown to prevent aggregation of key neurodegenerative protein linked to Parkinson’s and Alzheimer’s.

Argenica will continue to progress pre-clinical studies of ARG-007 in Alzheimer’s Disease, with data from a mouse model expected early next year.



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.