MHRA grants CTA for the POLB 001 TOPICAL trial

MHRA grants Clinical Trial Authorisation for the TOPICAL trial and independent research confirms significant commercial opportunity for POLB 001

Trial on track to deliver interim data this summer

15 April 2026- 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 UK Medicines and Healthcare products Regulatory Agency ("MHRA") has granted Clinical Trial Authorisation ("CTA") for the POLB 001 TOPICAL trial. With all required regulatory clearances now in place and site initiation visits scheduled, the trial remains on track to deliver interim data this summer.

The Company also announces positive findings from new independent US focused payer research conducted by Acumetis Global indicates multi-billion-dollar peak sales potential in the United States.

POLB 001 on track to deliver interim data this summer

POLB 001 is being developed as a potential first approved preventative therapy for cancer immunotherapy-induced Cytokine Release Syndrome ("CRS"). The TOPICAL trial will investigate POLB 001 in approximately 30 relapsed/refractory multiple myeloma patients receiving the approved bispecific antibody teclistamab, which has been supplied by Johnson & Johnson at no cost to Poolbeg.

The study now spans six sites across the UK, with NHS Lothian in Edinburgh and the Royal Stoke University Hospital joining existing sites at the NHS Trusts at The Christie, The Royal Marsden, University College London Hospitals, and University Hospitals Birmingham. Site initiation visits are scheduled with patient screening set to commence shortly.

Unlike many oncology studies, given that CRS is an acute condition which occurs within days or weeks of initiation of a bispecific antibody, the design of the TOPICAL trial will allow rapid read out.

Independent US payer research reconfirms compelling commercial opportunity

The research, which engaged current payers covering approximately 75 million lives across a mix of commercial insurance providers, Medicare and Medicaid, confirms the strong, clearly defined value proposition for POLB 001. In particular, findings underscore POLB 001's potential to play an important role in the management of CRS by reducing the significant hospital costs associated with the condition and enabling a meaningful shift in care away from specialist centres of excellence toward outpatient and community settings.

The report also noted that CRS occurs in the majority of CAR T and bispecific antibody treatments, with no established diagnostic method currently available to predict either the likelihood or severity of CRS in individual patients. Dosing of bispecific antibodies must be withheld until CRS resolves, often delaying patients from achieving therapeutically effective dosing levels and prolonging hospitalisation, while those who experience high-grade CRS must permanently discontinue treatment, meaning that these patients can no longer receive the potentially life-saving treatment.

The research reconfirmed the significant cost burden associated with managing CRS and that an effective preventative treatment would represent a compelling value proposition for healthcare systems.

The findings highlight pricing levels that demonstrate POLB 001's multi-billion-dollar peak sales potential, providing increased confidence in the Company's anticipated market opportunity and further supports POLB 001's future value and appeal to potential partners.

Jeremy Skillington, PhD, Chief Executive Officer of Poolbeg Pharma, said: "We have made excellent progress on the TOPICAL clinical trial, with all regulatory approvals now received, additional trial sites added to the study, and site initiation visits scheduled, we are on track to achieve interim data this summer. We believe POLB 001 has the potential to transform the cancer immunotherapy field through the prevention of CRS, improving patient quality of life and expanding the number of patients that can receive these life-saving cancer immunotherapies.

"In addition, the findings from the independent research report offer important external validation of both the clinical relevance and commercial opportunity of POLB 001. The clear willingness for payers to address the significant burden associated with CRS, reinforces our confidence in POLB 001's ability to deliver meaningful value to healthcare systems and its potential to achieve multi-billion-dollar peak sales in the US."

Chris Grimes Crompton, Partner, Value Strategy, Acumetis Global said: "We interviewed payers across the commercial insurance, Medicare and Medicaid landscape to explore the burden of CRS and their attitudes towards novel treatment approaches. The research confirmed that CRS represents a significant and costly challenge in the cancer immunotherapy field as well as limiting the utilisation of new immunotherapy treatments due to the requirement for specialised centres to administer care.

"This research validates both the scale of that unmet need and the appetite among payers for an effective preventative approach. We observed a willingness to pay at commercially meaningful price points, driven by the potential to reduce hospitalisation costs, potentially allowing for care to be decentralised and ultimately delivering better outcomes for some of the sickest patients. This positions POLB 001 as a compelling CRS solution with significant market potential."

*TOPICAL: Trial of Prevention of ImmunoCytokine Adverse events in Myeloma.