Island Pharma closes in on FDA feedback for potential Ebola and Marburg treatment

• Island Pharma advances Galidesivir toward FDA review
• US FDA feedback due in November as Marburg threat looms
• Island aims to turn potential into impact

Outbreaks like Marburg don’t give second chances.

When they hit, they hit fast, spreading through communities with deadly efficiency, leaving little room for trial and error.

It’s a rare but highly lethal virus that causes severe haemorrhagic fever with fatality rates that can reach up to 90%.

And as new viruses continue to push the limits of global preparedness, the search for a proven antiviral isn’t just about science, it’s about time.

One drug quietly building momentum on that front is Galidesivir, a broad-spectrum antiviral designed to stop RNA viruses (the kind behind Ebola, Zika and Marburg) from replicating.

Originally developed with strong US government support, Galidesivir already carries an impressive résumé: human safety data from early-stage clinical work, and remarkable survival results in animal models of deadly diseases.

That’s what makes it such a strong candidate for the next phase, and why one Australian biotech has been steadily laying the groundwork to bring it to the world stage.

The Animal Rule advantage

Island Pharmaceuticals (ASX:ILA) has been working to progress Galidesivir’s regulatory pathway in the US, engaging with the FDA under what’s known as the Animal Rule.

This rule allows drugs for deadly infections to be approved based on animal efficacy studies when human trials aren’t ethical or possible.

For a disease like Marburg or Ebola, where human infection studies are obviously out of the question, the FDA’s Animal Rule is the key to getting life-saving treatments to market faster.

Island Pharma CEO Dr David Foster acknowledged the Animal Rule has been used sparingly.

He explained that to qualify, the program must meet certain requirements:

1. It must be aiming to treat a serious or life-threatening condition caused by exposure to lethal and permanently disabling toxic biological substances.

2. Definitive human studies cannot be conducted as it would be unethical to deliberately expose healthy human volunteers to a lethal or permanently disabling toxic biologic substance.

“In the case of our Galidesivir program, we believe that Marburg, a fatal hemorrhagic virus, qualifies as a serious or life threatening condition caused by exposure to a lethal and permanently disabling toxic biological substance,” said Dr Foster.

“Therefore, it would be unethical to conduct human clinical trials.

“The FDA feedback will be pivotal in that it will clarify the regulatory path forward for Galidesivir,” he added.

What Island is asking of the FDA

Foster explained that Island has asked the FDA for guidance on three key fronts.

First, the company has submitted questions to the FDA regarding the viability of using the Animal Rule pathway for Galidesivir’s development.

Second, it has asked whether Galidesivir, as a program targeting filoviruses and pursuing approval via the Animal Rule, could qualify for a Priority Review Voucher (PRV) – read more below.

“And finally, requesting guidance on design of the next animal study we believe will be required for approval,” he said.

The company hopes to get a definitive steer from the agency next month.

“We are hoping that they will confirm that the program is eligible for an Animal Rule approval and that it is eligible for a PRV.

“We also hope to get clarity on the design of the animal study.

“We believe we have a strong rationale in support of the Animal Rule and PRV potential, but of course the FDA is the final decision maker in this regard.”

“Looking at other products that have been pursued via the Animal Rule, they include countermeasures against similar threats including anthrax, botulism toxin and smallpox virus,” Foster added.

Island has now taken the next step by submitting briefing materials to the FDA under its open Investigational New Drug (IND) application.

The submission supports an upcoming Type C meeting, which gives Island the opportunity to seek the FDA’s feedback (not approval).

The opportunities that come with a Priority Review Voucher

A PRV is a valuable incentive that can significantly accelerate review times, or be traded for serious value later down the track.

“Obtaining a Priority Review Voucher and contract for supplying the strategic national stockpile are vital aspects of our commercial strategy,” Foster said.

“We believe that the Galidesivir program is eligible for a Tropical Disease Priority Review Voucher.

“If so, and if we are awarded a PRV upon Galidesivir’s approval, we would expect to sell the PRV. Recent PRV sales have ranged from US$150 million to US$160 million. This would certainly be a company changing event.”

He noted that a potential stockpile contract could be even bigger.

“Moreover, the stockpile contracts are potentially more significant.

“With proceeds from a PRV and a government stockpile contract, it is not unreasonable to see the possibility of significant revenue upon approval of Galidesivir, which again, may require one additional successful animal study.”

The stakes couldn’t be higher

It’s worth remembering what’s at stake here.

Marburg is part of the same viral family as Ebola – highly contagious, often deadly, and capable of sparking outbreaks that test even the best-prepared health systems.

With no approved treatment currently available, the need for an effective antiviral is real.

The next few months will show whether Galidesivir can move from promising antiviral to a serious contender in the global fight against emerging viral threats.

And as Island Pharma waits for that all-important letter from the FDA in November, one thing’s clear – this small Aussie company wants to position itself to make a global impact.

At Stockhead we tell it like it is. While Island Pharmaceuticals is a Stockhead advertiser, it did not sponsor this article.