Poolbeg Pharma (POLB) Study update summary

Program overview and objectives

• POLB 001 is being developed as a first-in-class oral preventative therapy for cancer immunotherapy-induced cytokine release syndrome (CRS), aiming to enable safer, at-home administration and broader use of immunotherapies for cancer patients.

• The drug targets p38 MAP kinase, a master regulator of inflammatory cytokines, and has shown broad cytokine inhibition compared to current standards like tocilizumab and JAK inhibitors.

• The TOPICAL trial is a single-arm, open-label study in relapsed/refractory multiple myeloma patients receiving teclistamab, focusing on safety and CRS prevention.

• The trial is designed for rapid execution, with approximately 30 patients recruited across leading UK sites, and allows for short-term monitoring (1–2 months per patient) to enable faster data collection.

• The ultimate goal is to partner the program, with a clear regulatory path to phase III and market entry, supported by recent FDA feedback and Orphan Drug Designation.

Interim study results and progress

• Interim data from the TOPICAL trial show positive safety outcomes, with the Safety Review Committee recommending continuation without protocol changes.

• POLB 001 has been well tolerated in a severely ill patient population, marking a significant milestone and de-risking event for the program.

• Recruitment is progressing rapidly, with strong engagement from investigators and additional sites being activated.

• The study is supported by a partnership with Johnson & Johnson for drug supply, further validating the approach.

• Further clinical data will be generated and shared with prospective partners as discussions continue.

Mechanism of action and clinical rationale

• POLB 001 is an oral p38 MAPK inhibitor that broadly suppresses pro-inflammatory cytokines without compromising the immune system.

• CRS is a severe, potentially life-threatening complication of cancer immunotherapies, often limiting their use to specialist centers.

• By preventing CRS, POLB 001 could reduce hospital stays, enable community-based treatment, and improve patient quality of life.

• POLB 001's safety profile and broad cytokine inhibition distinguish it from existing CRS management options.

• Bispecific antibodies are highlighted as increasingly important in cancer treatment, with POLB 001 aiming to address a critical side effect.