ILA starts manufacturing for Marburg disease study and FDA submission

Our 2025 biotech pick of the year Island Pharmaceuticals (ASX: ILA) just kicked off manufacturing of its marburg disease drug Galidesivir.

ILA also confirmed trial partners for the dose optimisation study are set to kick off imminently.

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Right now, ILA already has enough drug supply in inventories to run its planned Marburg dose optimisation trials.

Today’s manufacturing news is more about securing supply for future work ILA will need to do AND relates to the Ebola outbreak:

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We are mainly Invested in ILA for the Marburg disease trials. (more on why in a second)

BUT we also know ILA’s drug has shown effectiveness against more than 20 RNA viruses including Ebola…

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ILA’s drug has had over US$70M spent on it - funded mostly by the US government (including the Department of War) - for various diseases including Marburg and Ebola and has gone through phase 1 human safety trials (with no safety issues).

The interesting part is that the last time a major ebola outbreak occurred - the US government started funding research using ILA’s drug.

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The work Biocryst originally did on ILA’s drug was:

“substantially funded with Federal funds from the National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health and the Department of Health and Human Services and the Biomedical Advanced Research and Development Authority (BARDA)” (source)

And right now it looks like the Ebola outbreak in the DRC and Uganda is starting to become a big area of concern for the rest of the world…

The WHO escalated to a “Public Health Emergency of International Concern” (PHEIC) - the organisation’s highest level of global health alert.

And the headlines just keep getting worse every week:

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Today’s news is an early indication, ILA may be getting itself prepared (with excess drug supply) to put its drug forward as a potential research candidate for a treatment for the current ebola outbreak.

So while today’s news relates primarily to the trials ILA plans to run for Marburg this year… It could also be ILA getting prepared for a major global pivot to searching for an ebola treatment.

We covered why we think ILA’s drug could be an option in detail in our last note here: ILA: World Health Organisation declares “Health Emergency of International Concern” as harder to detect strain of Ebola with no approved vaccine kills 80, spreads to major city.

ILA’s most advanced target is still Marburg disease

ILA’s current focus and most advanced treatment is for Marburg disease.

Marburg is the only Category A bioterrorism threat (the highest level) with NO current vaccine or FDA-approved treatment - which also makes it the biggest biowarfare risk.

That also means the US has no stockpiles of any Marburg treatments or vaccines.

Making an FDA-approved Marburg drug a strong candidate for US government stockpile contracts.

Which range between $US100M to US$1.2BN in lifetime sales (source)

The US tries to maintain stockpiles of treatments against all kinds of nasty diseases that can be weaponised.

But it has nothing to counter Marburg disease.

We Invested in ILA as we think it has a very good chance of filling this gap, and it can be paid handsomely for it.

ILA’s Marburg treatment has qualified for the FDA’s Animal Rule - a special “urgency” approval ruling given by the US FDA to bypass human trials that can cut 10 to 15 years off US FDA approval for a drug.

ILA is also eligible for a Priority Review (PRV) voucher.

These PRVs are a tool the FDA uses to incentivise companies to develop treatments for rare paediatric diseases, tropical diseases, or medical countermeasures…

... and these vouchers are tradeable on the open market.

IF the FDA approves ILA’s drug it could receive one of these tradeable vouchers.

Here are some previous sales - including the most recent for ~US$200M:

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To get approved under the Animal Rule process, ILA now needs to complete the following two step trial process:

• The first stage is an optimisation study - to work out the optimal dose and when to administer that dose
• The second pivotal confirmatory study will follow right after - The pivotal study will be when we see how effective ILA’s Marburg drug really is. Fingers crossed it improves on the previous studies, which had survival rates averaging 94%.

What's next for ILA?

🔄 Marburg disease animal rule approval pathway

Next we want to see ILA execute the following:

1. 17th November: ILA confirms Animal Rule eligibility for its Marburg drug ✅
2. 4th February: staged approach for FDA approvals confirmed ✅
3. NEXT: We want to see ILA start “optimisation studies” ahead of a pivotal study later this year.
4. Ongoing 🔄: We want to see ILA sign more agreements with Biosecurity Level 4 (BSL4) facilities that are able to run animal studies - more sites means the studies can be completed quicker.
5. THEN 🔲, we want to see ILA start animal trials (pivotal trial) for Marburg disease. (this is the big one)

Assuming the clinical trial results are positive, ILA will then submit to the FDA for an Investigational New Drug Application (IND) of its drug (typically a 6-month review timeframe).

And all of this happens inside the next 12 months. ILA is targeting regulatory submissions in Q1-2027:

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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)

IF the pivotal study results are positive, ILA could then pursue:

🔲 FDA approval of Galidesivir for Marburg

🔲 Priority Review Voucher (~US$200M based on recent sales)

🔲 US Government Strategic National Stockpile contract (potentially worth hundreds of millions in revenue)

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

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