ILA secures Marburg data for its FDA approval pathway

Our 2025 Biotech Pick of The Year Island Pharmaceuticals (ASX: ILA) just secured a key data package for its Marburg drug - Galidesivir.

ILA is looking to get FDA approvals for the drug using the US FDA's "Animal Rule" pathway.

The Animal Rule pathway allows drugs to be approved on animal data and human safety data alone.

Marburg is a Category A bioterrorism threat with up to 88% fatality, and the only Category A biothreat with no vaccine or approved treatment.

ILA’s drug already has ILA’s drug already has human safety trials completed and the results showed a 100% primate survival at 24-48 hour injection versus 0% for placebo) (source)

🎓We have covered the Animal Rule process in detail before here: The journey for ILA to get a drug to market in under 12 months

Today ILA signed a Letter of Authorisation giving it the right to cross-reference a detailed Marburg-Angola natural history study in its submissions to the FDA.

(source)

Natural history data shows how the disease progresses in the absence of treatment.

Basically a way for ILA to define a baseline against which a drug's efficacy is measured against.

It’s also required to support a potential New Drug Application (NDA).

This update directly addresses prior feedback from the FDA, which had said the published data on ILA's proposed animal model wasn't detailed enough for its review. (source)

With the new data acquired ILA shouldn’t have to build its own baseline model to support its NDA.

That work would need specialised Biosafety Level 4 labs and primate studies, so this saves ILA time and cash in otherwise needing to redo a study on it.

ILA can instead focus on the two dose optimisation studies.

The US Army (USAMRIID) study is expected to start next quarter, with the Texas Biomed study to follow in Q1 2027.

Should the trials be successful, ILA would be eligible for a Priority Review Voucher (PRV), which is an incentive set up to encourage work on drugs to treat diseases such as this.

The interesting part about this is that it can knock a few months off the FDA wait time to get through the approval process.

Those few months may not be of much value to ILA, but they are highly sought after by big pharma, we have seen them this year go for ~US$200M:

(source)(source)(source)

Also, in a recent announcement ILA also mentioned that Animal Rule countermeasures have averaged US$467M in US stockpile contracts:

(source)

What else is ILA up to?

The current Ebola outbreak in Africa continues to spread at a much faster rate than prior outbreaks, mostly in The DRC currently.

(source)

Unfortunately it appears inevitable that this outbreak is going to get a LOT worse over the coming months.

Simply because there are NO vaccines or treatments for this particular strain of Ebola that are proven to be effective.

During the last major Ebola outbreak, US government agencies deployed over US$70M in funding to develop and test a drug called Galidesivir against viral diseases.

Including against Ebola.

ILA’s same drug Galidesivir ALREADY has Phase 1 safety data (in humans) and effectiveness (in animals) against Ebola, Marburg and 20+ other viral diseases.

Our 2025 Biotech Pick of The Year, Island Pharmaceuticals (ASX:ILA), acquired Galidesivir in 2025.

(after the prior owner shifted strategic focus to other diseases).

So while ILA decided to develop the drug for the Marburg virus first, there presents a unique opportunity to progress the drug against Ebola also.

Recently ILA signed an agreement with neighbouring Uganda which has had cases recently.

As a part of this ILA’s drug will be able to treat humans so should it be used, it will also be able to collect the data from it.

This means that ILA is paying for the drug supply only, so should it be used it effectively gets an in human trial done for no additional cost.

We covered this more in our recent article here: ILA ramping up drug manufacturing to help fight “worst ever” Ebola outbreak

What's next for ILA?

🔄 Ebola (Uganda deployment):

1. ✅ Government + regulatory approvals for compassionate use in Uganda
2. 🔄 Drug supply / manufacturing scale-up (additional supply commissioned) (source)
3. 🔲 First patients dosed (due within CY2026) - (source)
4. 🔲 First human efficacy, safety and virological data from the field
5. 🔲 Any expansion of deployment beyond Uganda (DRC?)

🔄 Animal rule approvals process for Marburg disease

Here are the two next major milestones for ILA’s Galidesivir drug:

• Stage 1 - Optimisation study - Working out the optimal dose and the best time to administer it.
• Stage 2 - Pivotal confirmatory study - The big one - this will determine how effective Galidesivir really is. Fingers crossed it improves on that 94% survival rate.

Here are the milestones we are tracking for the trials:

Stage 1 - Optimisation study:

• ✅FDA confirmed Animal Rule eligibility
• ✅FDA confirmed staged approach for approvals
• ✅CRADA (Cooperative Research and Development Agreement) signed with USAMRIID (US Army’s premier infectious disease research institute) and Geneva Foundation (highly influential non-profit that manages nearly US$383M in military medical research funding)
• 🔄Optimisation study (commencing soon)

Stage 2 - Pivotal study:

• 🔲 Pivotal study design completed
• 🔲 Pivotal study commences
• 🔲 Pivotal study results
• 🔲 FDA submission (NDA)

Here is an indicative timeline on when to expect all of the above from ILA’s recent presentation:

(source)