Poolbeg Pharma (POLB) Study Update summary

Clinical program highlights

• Advancing POLB 001, a p38 MAP kinase inhibitor, as a prophylactic for cancer immunotherapy-induced CRS, with phase IIa trial preparations at an advanced stage and interim data expected in summer 2026.

• Received FDA orphan drug designation for POLB 001, providing market exclusivity, fee waivers, and regulatory advantages.

• Secured supply of GMP-grade POLB 001 and an approved bispecific antibody at no cost for the trial, reflecting strong pharma interest.

• The phase IIa open-label, single-arm trial (TOPICAL) will enroll about 30 relapsed/refractory multiple myeloma patients at The Christie NHS Foundation Trust and other UK cancer centers.

• Accelerating Clinical Trials Ltd (ACT) will conduct the trial, with Dr Emma Searle as Chief Investigator, and support rapid recruitment and high-quality data generation.

Scientific rationale and preclinical/clinical data

• POLB 001 inhibits p38 MAP kinase, reducing pro-inflammatory cytokines without suppressing the immune system, aiming to prevent CRS while preserving immunotherapy efficacy.

• Preclinical studies in humanized mouse models showed POLB 001 effectively prevented CRS and reduced key cytokines (IL-6, TNFα) in a dose-dependent manner.

• Phase 1B human challenge studies demonstrated favorable safety, tolerability, and dose-dependent reduction in inflammatory markers and heart rate.

• No further preclinical studies are required before the phase IIa trial.

Market opportunity and strategic positioning

• CRS affects up to 70% of patients receiving CAR-T or bispecific antibody therapies, creating a significant unmet need and healthcare burden.

• Effective CRS prevention could expand immunotherapy access beyond specialist centers, potentially transforming cancer care delivery and enabling treatment in community hospitals.

• The addressable market for POLB 001 is estimated at over $10 billion, with 500,000 eligible patients in the US and EU5 for key indications.

• Orphan drug status and strong IP position enhance partnering and monetization prospects.

• The company is focused on partnering opportunities based on positive trial data.