Poolbeg Pharma plc

Major milestone reached as POLB 001 human challenge clinical trial ready to commence

Regulatory approval gives green light for trial to proceed

20 July 2022 - Poolbeg Pharma (AIM: POLB, OTCQB: POLBF, 'Poolbeg' or the 'Company'), a clinical stage infectious disease pharmaceutical company with a unique capital light clinical model, has received ethics and competent authority approval to commence its Lipopolysaccharide (LPS) human challenge clinical trial for POLB 001, a strain agnostic, small molecule immunomodulator which aims to address the unmet medical need for a treatment for severe influenza.

Key Highlights:

Jeremy Skillington, PhD, CEO of Poolbeg Pharma, said: "This is a major milestone for Poolbeg as we are ready to commence the LPS human challenge trial of POLB 001, our severe influenza treatment. Significant preparatory work has been completed in order to reach this stage with the completion of GMP* manufacturing, formulation, trial protocol design amongst other vital regulatory requirements. We look forward to receiving the valuable, broad-ranging data and insights that this trial is expected to generate by the end of 2022. We stated at our IPO in July 2021 that we intended to bring POLB 001 into the clinic for this LPS trial in the summer of 2022 and are happy to have achieved this stated objective and demonstrate our ability to effectively execute on our strategy.

"The unmet need for new treatments for severe influenza presents a significant opportunity to improve outcomes for patients as well as to potentially rapidly monetise our lead asset. The unique mode of action of POLB 001 to reduce hyperinflammatory response in the body means that it is agnostic to the strain of virus and has the potential to expand to other disease indications and as such, could provide further opportunities to create significant value beyond severe influenza for the Company and our shareholders."

*Good Manufacturing Practice

The information contained within this announcement is deemed by the Company to constitute inside information as stipulated under the Market Abuse Regulations (EU) No. 596/2014 (as implemented into English law) ("MAR"). With the publication of this announcement via a Regulatory Information Service, this inside information is now considered to be in the public domain.

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About Poolbeg Pharma

Poolbeg Pharma is a clinical stage infectious disease pharmaceutical company, with a capital light clinical model which aims to develop multiple products faster and more cost effectively than the conventional biotech model. The Company, headquartered in London, is led by a team with a track record of creation and delivery of shareholder value and aspires to become a "one-stop shop" for Big Pharma seeking mid-stage products to license or acquire.

The Company is targeting the growing infectious disease market. In the wake of the COVID-19 pandemic, infectious disease has become one of the fastest growing pharma markets and is expected to exceed $250bn by 2025.

With its initial assets from Open Orphan plc, an industry leading infectious disease and human challenge trials business, Poolbeg has access to knowledge, experience, and clinical data from over 20 years of human challenge trials. The Company is using these insights to acquire new assets as well as reposition clinical stage products, reducing spend and risk. Amongst its portfolio of exciting assets, Poolbeg has a small molecule immunomodulator for severe influenza (POLB 001) which will commence its LPS human challenge trial imminently, with results expected by year end 2022. A first-in-class, intranasally administered RNA-based immunotherapy for respiratory virus infections. (POLB 002); and a vaccine for Melioidosis (POLB 003). The Company is also developing an oral vaccine delivery platform and is progressing two artificial intelligence (AI) drug discovery programmes to accelerate the power of its human challenge model data and biobank.

For more information, please go to www.poolbegpharma.com or follow us on Twitter and LinkedIn @PoolbegPharma

Further detail on the LPS trial, POLB 001 and the Influenza opportunity and beyond

Rigorous randomised, double-blind, placebo-controlled, multiple dose, LPS human challenge trial in 36 healthy volunteers to assess efficacy of POLB 001 in treating the dangerous effects of severe influenza. As part of the trial, researchers will stimulate the immune systems of healthy volunteers with LPS across three cohorts. LPS triggers a robust immune response and acts as a surrogate for the hyperinflammatory effects associated with severe influenza infection, as well as other diseases. Each cohort will receive escalating doses of POLB 001 to evaluate its effectiveness in suppressing the body's harmful inflammatory response to both intradermal (a shallow injection) and intravenous (an injection in a vein) administered LPS.

The Principal Investigator is Dr. Matthijs Moerland and the trial will be conducted at the Centre for Human Drug Research in The Netherlands and has received all relevant regulatory and ethics approvals. POLB 001 has previously been proven to be safe and well tolerated in a successful Phase I clinical trial. The first results of this LPS human challenge trial are expected before the end of the year, and the Company expects to be in a position to rapidly monetise POLB 001 thereafter by partnering or out-licensing to Pharma for further development and commercialisation.

Influenza is a viral pathogen that infects approximately one-eighth of the world's population each year, an estimated 1 billion people infected globally, attacking the respiratory system leading to between 5 and 10 million hospitalisations and as many as 500,000 influenza related deaths. Survivors of severe influenza can suffer organ damage, leading to chronic and life-changing injuries. Given POLB 001's mode of action, it is strain agnostic meaning that it treats the body's reaction to infection rather than targeting the virus directly and, as such, POLB 001 is unaffected by the seasonal variants of influenza that arise each year which is a significant advantage over treatments available on the market. Therefore, POLB 001 has the potential to be a transformational treatment for patients and to become a leading severe influenza treatment. POLB 001 has patent protection until 2038 and is a shelf-stable oral drug which makes it ideal as a stockpiling candidate for both seasonal outbreaks and pandemic preparedness.

POLB 001 also has potential therapeutic applications beyond severe influenza, due to its mode of action of reducing hyperinflammation (cytokine storm). This is when harmful inflammation occurs in different areas of the body, such as the heart and lungs, causing organ damage which is linked with many diseases. POLB 001 has the potential to block this by interrupting the positive feedback loop of inflammatory mediators. Poolbeg is currently investigating new potential uses and in due course hopes to expand its IP around this asset to cover new disease areas thereby increasing the value of the asset.