Poolbeg Pharma Regulatory News (POLB)

Poolbeg Pharma plc

POLB 001 LPS human challenge clinical trial successfully completed

No serious adverse events reported

Results of full data analysis expected in Q2 2023

12 December 2022 - Poolbeg Pharma (AIM: POLB, OTCQB: POLBF, 'Poolbeg' or the 'Company'), a leading infectious disease focused biopharmaceutical company, announces that, further to its announcement in July 2022, it has now received the initial results having completed the Lipopolysaccharide (LPS) human challenge clinical trial for POLB 001, a viral strain agnostic, small molecule immunomodulator being developed to address the unmet medical need for severe influenza.

Key highlights from the study:

· No further clinical activity is required to complete the objectives of the trial, bringing the recruitment and clinical phase to a close on schedule

· No serious adverse events reported. POLB 001 was found to be safe and well tolerated

· Data analysis has commenced and full data read-out is expected in Q2 2023

· The comprehensive dataset is expected to facilitate progression of the product to the next clinical phase for potential partners

This LPS challenge trial was designed to evaluate the effect of POLB 001 on inflammatory responses in 36 healthy volunteers between 18 and 55 years of age following an intradermal and an intravenous LPS challenge. LPS acts as a surrogate for the hyperinflammatory response associated with severe influenza and other diseases.

POLB 001 is a unique potential treatment for viruses such as severe influenza as it is strain agnostic, unlike other flu treatments and prophylactics. This means that it can be effective regardless of which strain of influenza is dominant in any particular season or geography. As a shelf-stable, oral drug it could also be ideal as a stockpiling candidate for both seasonal outbreaks and pandemic preparedness.

Jeremy Skillington, PhD, CEO of Poolbeg Pharma, said:

"We have completed our first trial on schedule, achieving a key milestone for Poolbeg, thanks to the committed efforts of the team. We are actively planning the next steps for POLB 001 and are engaging with industry on potential partnering opportunities.

"The threat of influenza shouldn't be underestimated. Cases are on the rise - the WHO estimates that there are 3-5 million severe influenza cases globally per year, with real pandemic potential. Severe influenza can also lead to other major complications such as pneumonia and stroke. There is a significant unmet need for severe influenza treatments, so we eagerly anticipate the full results of this study next year."

Matthijs Moerland, PhD, Principal Investigator at CHDR, said:

"LPS human challenge trials are a highly reliable way of measuring the efficacy of anti-inflammatory drugs and these initial data are an important first step. We are pleased to be involved in the development of a potential treatment for such an unmet need as severe influenza and we look forward to the vital insights that the full analysis will bring."

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About Poolbeg Pharma

Poolbeg Pharma specialises in the development of innovative medicines to address the unmet need in prevalent and emerging infectious diseases. Poolbeg has a disciplined portfolio approach to mitigate risk, accelerate drug development and enhance investor returns. The Company simultaneously advances multiple programmes in cost-effective clinical trials, rapidly generating early human safety and efficacy data to enable early partnering / out-licensing, with the funds generated reinvested in the pipeline. Poolbeg also uses AI to interrogate human challenge trial data sets to quickly identify new targets and drugs, and in-license near or in the clinic medicines, leading to faster development and greater commercial appeal.

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 hVIVO plc (formerly 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 has completed its LPS human challenge trial with full data read-out expected in Q2 2023; a first-in-class, intranasally administered RNA-based immunotherapy for respiratory virus infections (POLB 002); and a vaccine candidate for Melioidosis (POLB 003). The Company is also developing an Oral Vaccine Platform and is progressing two Artificial Intelligence (AI) Programmes to accelerate the power of its human challenge model data and biobank, with results from the first programme expected by year end. 

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

Further detail on POLB 001

This trial is a randomised, double-blind, placebo-controlled, multiple dose, LPS human challenge trial in 36 healthy volunteers to assess the potential efficacy of POLB 001 in treating the hyperinflammatory responses associated with severe influenza. As part of the trial, researchers stimulated 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 received 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, which will produce a broad-ranging dataset.

Given POLB 001's mode of action, it is viral strain agnostic as 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 influenza 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.

The data collected in the previous Phase I study demonstrated that POLB 001 administration produces a potent and long-lasting inhibition of p38 MAP Kinase activity in humans. The study showed that after administration of single doses up to 600 mg and repeated doses up to 150 mg there were neither serious nor limiting adverse events to POLB 001, and that after a twice daily dose of 150mg an inhibition of LPS-induced TNFα of between 70% and 90% was achieved. 

About Influenza

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. Complications include a 6-fold increased risk of heart attack, an 8-fold increased risk of Pneumonia, and an 8-fold increased risk of stroke. This leads to an enormous compound cost of treatment - influenza contributes to an estimated economic burden of $11.2bn every year in the US alone.