Poolbeg Pharma PLC - POLB 001 Phase 2a Trial Update

Poolbeg Pharma plc 

ACT appointed for POLB 001 Phase 2a Trial and supply of approved bispecific antibody drug secured at no cost

Trial to take place at The Christie NHS Foundation Trust and other leading UK specialist cancer centres

25 September 2025 - Poolbeg Pharma (AIM: POLB, 'Poolbeg' or the 'Company'), a clinical-stage biopharmaceutical company with a core focus on transforming the cancer immunotherapy field, announces that the Company has signed an agreement for a specialist blood cancer trials organisation, Accelerating Clinical Trials Limited ("ACT"), to conduct the upcoming POLB 001 Phase 2a trial and has also secured the supply of an approved bispecific antibody drug for the trial at no cost to the Company.

The trial will be led by Dr Emma Searle, Consultant Haematologist at The Christie NHS Foundation Trust, Honorary Senior Lecturer at the University of Manchester, as well as Chair of the British Society of Haematology Research and Grants Committee. The Christie, a leading cancer research institute based in Manchester, and a number of other leading UK specialist cancer centres will participate.

The Phase 2a first-in-patient open-label, single-arm trial is titled TOPICAL (Trial of Prevention of ImmunoCytokine Adverse events in Myeloma). The study aims to investigate the safety and efficacy of POLB 001, in particular its ability to reduce the incidence of Cytokine Release Syndrome ("CRS") in approximately 30 relapsed/refractory multiple myeloma patients receiving an approved bispecific antibody.

The Company already has sufficient supply of Good Manufacturing Practice ("GMP") grade POLB 001 and this agreement represents an advanced stage of trial preparation, with interim data anticipated in summer 2026. The Company's forecast cash runway extends into 2027, providing more than adequate funding to achieve key clinical development milestones.

The Directors believe that POLB 001 has the potential to transform the cancer immunotherapy field by expanding administration from centralised specialist cancer centres into community hospitals by making the treatments safer through the prevention of the life-threatening side effect, CRS. As such, POLB 001 could increase the number of patients that can receive these life-saving treatments, thereby increasing the market opportunity.

Jeremy Skillington, PhD, Chief Executive Officer of Poolbeg Pharma, said: "The signing of this agreement, and the supply of the approved bispecific antibody at no cost to the Company, are major milestones for the upcoming POLB 001 Phase 2a trial. The trial is at an advanced stage of preparation and remains on track to deliver data in 2026, we look forward to sharing the data with prospective partners. With a cash runway into 2027, and multiple near-term clinical inflection points, we believe that we are well positioned to deliver value for our shareholders."

"We are optimistic about the potential of POLB 001 to transform the delivery of cancer immunotherapies by expanding administration away from specialist centralised cancer centres and into the community setting, closer to patients' homes. This shift could make these life-saving treatments more widely accessible. POLB 001 has the potential to make cancer immunotherapies safer by reducing the incidence of potentially life-threatening cancer immunotherapy-induced cytokine release syndrome."  

Dr Emma Searle, MBChB MA MRCP FRCPath PhD, Consultant Haematologist at The Christie NHS Foundation Trust, and Chief Investigator of the forthcoming POLB 001 trial said: "I have seen first-hand the challenges that CRS presents to the delivery of cancer immunotherapies, requiring many of our patients to be hospitalised for treatment. These transformative therapies will continue to be restricted until there is a way to administer them more safely. POLB 001 holds great promise in tackling this issue; potentially leading to improved patient wellbeing, reducing the strain on healthcare systems while making these treatments more accessible to a broader patient population."

Dr Paul Sherrington, CEO of Accelerating Clinical Trials Ltd said: "At ACT we work hand-in-hand with the haematological community to accelerate the delivery of clinical trials of new therapies, leveraging our wide network of research centres in the UK. We look forward to completing this study with POLB 001 which holds great potential to transform the cancer immunotherapy field and address this critical unmet medical need."

Market Opportunity

The demand for effective CRS prevention solutions is driven by the rapid growth of CRS-inducing cancer immunotherapies with more than 70% of patients receiving certain cancer immunotherapies affected. Independent research has identified a market opportunity of >US$10 billion for POLB 001 and as such, the Company sees strong potential for partnering on positive data from the forthcoming trial. The recent FDA Orphan Drug Designation further validates POLB 001's scientific rationale and enhances its commercial appeal for prospective partners.

Investor presentation

Poolbeg Pharma will provide a live presentation via the Investor Meet Company platform on Monday 29 September 2025 at 5pm.

The presentation is open to analysts and investors, those who already follow Poolbeg on the Investor Meet Company platform will automatically be invited. Investors can sign up to Investor Meet for free and add Poolbeg Pharma plc to their company dashboard here.

Enquiries 

About Poolbeg Pharma plc 

Poolbeg Pharma plc (AIM: POLB) is a clinical-stage biopharmaceutical company with a core focus on transforming the cancer immunotherapy field. The Company's lead asset, POLB 001, has the potential to expand administration of cancer immunotherapies from centralised specialist cancer centres into community hospitals by making the treatments safer through the prevention of the life-threatening side effect, Cytokine Release Syndrome (CRS). As such, POLB 001 could increase the number of patients that can receive these life-saving treatments, thereby increasing the market opportunity. Poolbeg is also advancing the development of a patient-friendly therapy for obesity with an oral encapsulated GLP-1, offering a differentiated approach within one of the world's largest markets. With multiple near-term clinical value inflection points, and an experienced team with a proven track record, Poolbeg is focussed on partnering its high value programmes that are targeting large markets and addressing critical unmet medical needs. 

Stay updated: Website | Sign up for RNS alerts | Presentation | X | LinkedIn

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.