FDA feedback gives Argenica clear path to lift clinical hold on brain drug

• FDA provides clear path for Argenica to lift clinical hold on its ARG-007 stroke drug
• Further clarification on safety of dose in humans and three additional in vitro lab studies requested
• Company confident studies are straightforward and can be completed quickly

Special Report: Argenica Therapeutics now has a clear path forward to clear the clinical hold currently in place on its investigational new drug application that will enable clinical trials for its lead drug ARG-007.

In its full letter, the US Food and Drug Administration (FDA) requested additional information to provide assurance that proposed dosing for a US trial of ARG-007 in acute ischaemic stroke can be achieved safely in humans.

The FDA has also requested that Argenica Therapeutics (ASX:AGN) conduct three additional in vitro cell culture studies with clinical research organisations to address identified gaps in data.

AGN plans to use safety data from its Phase 2 acute ischaemic stroke trial, which will be available in September, as part of its response to the FDA’s request for safety assurance.

It adds that three additional studies are small ones that can be completed quickly and build on existing data it has already generated.

“We are pleased to have received the clarification from the FDA as to the additional information required to progress the IND application,” managing director Dr Liz Dallimore said.

“Importantly from a timing and cost perspective, the requested additional in vitro assays are standard assays which are straightforward and efficient to perform.

“Argenica will work with the FDA to ensure the proposed approach to providing the additional information is adequate to lift the clinical hold.”

Preventing brain injury

During acute neurological events such as stroke, traumatic brain injury (TBI), and hypoxic brain damage, around 1.9 million brain cells die for every minute that blood flow is halted.

AGN’s ARG-007 is a synthetic peptide designed to protect brain cells from dying in the critical minutes and hours by protecting brain tissue during and after these events.

The company noted it was targeting acute ischaemic stroke as many patients that experienced it had a thrombectomy, where a radiologist inserts a catheter through the wrist or groin and guides it up into the brain to physically remove a clot.

However, this process has the risk of reperfusion injury, which occurs when blood flow rapidly returns to previously oxygen-deprived brain tissue after a clot is removed and potentially causes additional damage.

Dosing of patients in a double-blinded, randomised, placebo-controlled Phase 2 trial of ARG-007 in acute ischaemic stroke patients was completed in April.

This involved 92 patients presenting to eight emergency departments around Australia with data-read out due in September with safety the main endpoint.

The secondary endpoint for the trial will examine efficacy, with a brain scan taken 48 hours post administration of either the placebo or ARG-007.

ARG-007 would be the first neuroprotective drug on the global market if approved, tapping into a large addressable global market for stroke and potentially other indications.

The global stroke management market was valued at $36.1 billion in 2022, and is projected to reach $74 bn by 2032, driven by several factors including a growing ageing population.

There are more than 45,000 strokes annually in Australia and 795,000 in the US – the world’s largest healthcare markets.

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.