Poolbeg Pharma plc - Interim results for the six months to 30 June 2024

Lead development candidate (POLB 001) on track with positive data demonstrating efficacy

Independent research endorses potential US$10bn market opportunity for POLB 001 in cancer immunotherapy-induced CRS

Patent portfolio significantly strengthened & expanded

Continued focus on high value programmes & partnerships to develop & commercialise assets

Strong Balance Sheet - cash balance of £10.1m

Experienced leadership team with track record of delivering significant value

18 September 2024- Poolbeg Pharma (AIM: POLB, 'Poolbeg' or the 'Company'), a clinical-stage biopharmaceutical company focussed on the development and commercialisation of innovative medicines targeting diseases with a high unmet medical need, announces its unaudited interim results for the six months to 30 June 2024 ("H1 24").

Highlights, Business Update & Outlook

Jeremy Skillington, PhD, Chief Executive Officer of Poolbeg, commented: "The value and attractiveness of POLB 001 to Pharma partners has been greatly enhanced following the independent confirmation of a potential market opportunity of c.US$10 billion as an oral preventative therapy for cancer immunotherapy-induced CRS. During the period, we also strengthened our patent and overall intellectual property portfolio with positive in vivo data. As Pharma companies seek to enhance the safety and market reach of their cancer immunotherapies, we believe that POLB 001 is well placed to generate value for shareholders while addressing a critical unmet medical need for patients.

"Our increasing focus on rare and orphan diseases is exemplified by the exclusive option agreement we signed to acquire tPTX, which comes with robust clinical data, FDA Fast Track Designation, Orphan Drug Designation and the potential for a 505(b)(2) approval pathway in the US. tPTX has the potential to reach patients rapidly and offers a transformative solution for those suffering from Behçet's Disease.

"We continue to pursue our efforts to maximise the value of our development pipeline and are seeing significant interest from potential partners. Our experienced team, bolstered by the addition of a number of the former Amryt leadership team, is well-positioned to execute on our strategy of acquiring, developing, partnering, and commercialising innovative medicines that will help improve the lives of patients with rare and orphan diseases, and where there is a high unmet medical need."

References

1. Independent research commissioned by Poolbeg

Enquiries 

About Poolbeg Pharma plc 

Poolbeg Pharma plc is a clinical-stage biopharmaceutical company focussed on acquiring, developing and commercialising innovative medicines that will help improve the lives of patients with rare and orphan diseases and where there is a high unmet medical need. Poolbeg comprises a strong and growing portfolio of development assets. For more information, please go to www.poolbegpharma.com or follow us on Twitter and LinkedIn @PoolbegPharma.

Forward-Looking Statements

This announcement may contain forward-looking statements and the words "expect", "anticipate", "intends", "plan", "estimate", "aim", "forecast", "project" and similar expressions (or their negative) identify certain of these forward-looking statements. The forward-looking statements in this announcement are based on numerous assumptions and Poolbeg's present and future business strategies and the environment in which Poolbeg expects to operate in the future. Forward-looking statements involve inherent known and unknown risks, uncertainties and contingencies because they relate to events and depend on circumstances that may or may not occur in the future and may cause the actual results, performance or achievements to be materially different from those expressed or implied by such forward-looking statements. These statements are not guarantees of future performance or the ability to identify and consummate investments. Many of these risks and uncertainties relate to factors that are beyond Poolbeg's ability to control or estimate precisely, such as future market conditions, currency fluctuations, the behaviour of other market participants, the outcome of clinical trials, the actions of regulators and other factors such as Poolbeg's ability to obtain financing, changes in the political, social and regulatory framework in which Poolbeg operates or in economic, technological or consumer trends or conditions. Past performance should not be taken as an indication or guarantee of future results, and no representation or warranty, express or implied, is made regarding future performance. No person is under any obligation to update or keep current the information contained in this announcement or to provide the recipient of it with access to any additional relevant information.

CEO Statement

I am delighted to present the unaudited Interim Financial Statements of Poolbeg Pharma plc for the six months to 30 June 2024.

A Growing Focus on Rare and Orphan Diseases

We are actively increasing our focus on rare and orphan diseases, leveraging both the expertise of the key former Amryt Pharma plc executives that joined Poolbeg in early 2024, and the potential of POLB 001 to address cancer immunotherapy-induced Cytokine Release Syndrome (CRS). We believe that there is potential for POLB 001 to be a rare and orphan therapy, as the patients receiving T cell engaging bispecific antibodies and CAR T-cell therapy are often suffering from rare or orphan blood cancers. Rare diseases affect a small percentage of the population, and due to the high unmet medical need, regulatory authorities offer significant incentives for developing orphan drugs. The orphan drug market is expected to grow to US$368 billion by 2030², with orphan drug sales due to account for 20% of global prescription drug sales by 2026³. With our Leadership Team's extensive expertise and proven track record in rare and orphan diseases, we believe Poolbeg is well-positioned to capitalise on these opportunities.

In April 2024, we signed an exclusive 12-month option agreement with Silk Road Therapeutics Inc. to acquire a novel, topical, muco-adherent formulation of Pentoxifylline (tPTX) for the treatment of oral ulcers in Behçet's Disease patients. This disease, which has no cure, causes severe inflammation leading to debilitating symptoms, the most common being painful oral ulcers which significantly impact essential functions like eating, drinking and speaking. tPTX has shown promising results in a Phase 2 trial, as presented at the American College of Rheumatology meeting in 2019, demonstrating faster healing and pain reduction compared to standard of care treatments. tPTX has secured FDA Fast Track and Orphan Drug Designation, providing seven years of market exclusivity upon marketing authorisation, and is potentially positioned for the expedited route to approval and commercialisation available via the 505(b)(2) pathway in the U.S.

Strong Progress Across our Pipeline of Assets

POLB 001 - A breakthrough preventative therapy for cancer immunotherapy-induced CRS, as well as a promising treatment for severe influenza. CRS affects over 70% of patients receiving T cell-engaging bispecific antibodies or CAR T-cell therapy.⁴ With the cancer immunotherapy market expected to grow to US$120 billion by 2030^{5, 6, 7}, the need for effective CRS management is critical, as the condition currently leads to significant healthcare costs and restricts access to treatment at specialist cancer centres. Earlier this year, independent research commissioned by Poolbeg confirmed a market potential for POLB 001 of c.US$10 billion in Multiple Myeloma and Diffuse Large B-Cell Lymphoma alone due to the significant advances in bispecific antibody and CAR T-cell therapies for these indications.⁸ Cancer immunotherapies are being widely developed across a broader range of haematological malignancies (including many rare or orphan cancers) and solid tumours, which we believe will expand the opportunity for POLB 001 far beyond the estimate of US$10 billion.

In January 2024, we announced positive in vivo results which demonstrated POLB 001's efficacy in reducing cancer immunotherapy-induced CRS symptoms in an animal model. The data strengthened and facilitated the expansion of patent applications for POLB 001 in cancer immunotherapy-induced CRS. We also convened an Independent Advisory Board with international Key Opinion Leaders, healthcare payers and clinical trial experts, which endorsed the attractiveness of POLB 001's Target Product Profile (TPP) and its potential as an oral therapy to address the significant unmet medical need of cancer immunotherapy-induced CRS.

In May 2024, we received the fully granted patent from the US Patent Office for our Immunomodulator II patent application, covering a class of drugs (including POLB 001) for treating hypercytokinemia (cytokine storm) and for preventing hypercytokinemia in a patient after an immune response has been triggered. This encompasses cytokine storm that is induced in any disease indication. Further patent applications have been filed and have complementary coverage as we continue to expand our existing patent portfolio covering POLB 001.

As an oral therapy to prevent or treat CRS, POLB 001 has the potential to enable broader use of cancer immunotherapies in an outpatient setting to make these life-saving therapeutics more accessible to patients. With robust data and intellectual property, and interest from scientific, clinical, and commercial partners to advance its development, we are excited by the potential of POLB 001 to have a meaningful impact on patients' lives while generating value for our shareholders.

Oral GLP-1R Agonist - With approximately 42% of the US population affected by obesity⁹, the economic impact of the condition on US businesses and employees reached an estimated US$347.5 billion in 2023.¹⁰ This has driven the growth of prescription weight loss drugs, particularly glucagon-like peptide 1 receptor agonists (GLP-1R), a market projected to reach US$150 billion by 2031.¹¹ Despite the demand, oral GLP-1R options are limited, with only one available drug offering just 1% bioavailability.¹² Our Oral GLP-1R agonist programme aims to address this unmet need using a delivery system that utilises Generally Regarded as Safe (GRAS) components to encapsulate API's (active pharmaceutical ingredients), such as GLP-1R agonist, for oral delivery to specific areas of the gut and into systemic circulation with the aim of enhancing bioavailability and improving convenience. We are progressing towards the initiation of a proof-of-technology clinical trial in late 2024 to demonstrate the successful delivery of an oral GLP-1R agonist in humans.

Artificial Intelligence - As part of our AI-led programmes, we have successfully identified valuable novel drug targets for influenza and new potential drug candidates for the treatment of Respiratory Syncytial Virus (RSV). Using our data-first approach, these novel targets are based on the host response to stop or slow disease progression. This strategy is less likely to be impacted by viral resistance versus traditional vaccines and antivirals, which target the virus itself. AI-led solutions typically enable faster target identification, at lower cost, reduced risk, and potentially increased likelihood of success. Having successfully prioritised candidates from both programmes late last year, and with continued global interest in AI-led drug discovery, we are continuing to actively discuss the exciting outputs from our AI-led drug discovery programmes with prospective partners.

Financial

Poolbeg had a cash balance of £10.1 million as at 30 June 2024. The loss for the period amounted to £2.3 million (H1 23: £1.8 million) comprised of R&D expenses of £0.7 million (H1 23: £0.9 million), administrative expenses £2.1 million (H1 23: £1.4 million), and tax rebates and other income & charges of £0.6 million (H1 23: £0.5 million).

Outlook

As we build on the momentum from H1 24, our focus remains on strategically partnering our in-house programmes to unlock near-term value, while also targeting future revenue generation and profitability from commercial-stage rare and orphan drugs. We are excited by the results of in vivo data demonstrating POLB 001's efficacy in reducing cancer immunotherapy-induced CRS and the potential for this market opportunity to generate value for shareholders while addressing a critical unmet medical need.

The option agreement signed with Silk Road Therapeutics underscores our commitment to strategic cash management by providing us the opportunity to evaluate the potential of the asset whilst preserving capital. This approach aligns with our disciplined financial strategy, ensuring that we continue to manage our resources effectively as we explore opportunities that could enhance our pipeline and deliver value to our shareholders. The addition of former Amryt Pharma team members, with proven experience in scaling a rare disease company and establishing commercial infrastructure in the US and globally, further strengthens our Leadership Team and our ability to execute on this growth strategy.

Jeremy Skillington, PhD                                                                                     

CEO

17 September 2024

References

2. Fortune Business Insights, July 2023

3. European Pharmaceutical Review, May 2022

4. Average rate from Summary of Product Characteristics (SmPCs) for Yescarta, Tecartus, Abecma, Kymriah, Carvykti, Breyanzi, Elrexfio, Columvi, Epkinly, Tecvayli and Talvey

5. Grand View Research. CAR T-Cell Therapy Market Analysis 2023-2030

6. Grand View Research. Bispecific Antibodies Market Size, Share & Trends Analysis Report

7. Datamonitor Healthcare. Forecast: Diffuse Large B-Cell Lymphoma and Multiple Myeloma, 2023

8. Independent research commissioned by Poolbeg

9. Stierman B, Afful J, Carroll MD, et al. National Health and Nutrition Examination Survey 2017-March 2020 prepandemic data files development of files and prevalence estimates for selected health outcomes. Natl Health Stat Report. 2021;158.

10. Global Data, Assessing the Economic Impact of Obesity and Overweight on Employers, Feb 2024

11. The Economist, March 2023

12. EMA Product information 2020