Poolbeg Pharma (POLB) Investor Update summary

Strategic and Clinical Program Updates

• Lead program POLB001 received FDA orphan drug designation for preventing cytokine release syndrome (CRS) in cancer immunotherapy, providing significant regulatory and commercial incentives.

• Phase 2A clinical trial for POLB001 in multiple myeloma patients is set to begin in H2 2025, with interim data expected in H1 2026 and top-line results in H2 2026.

• Recent fundraise of £4.865 million, combined with a cash balance of £6.2 million, extends cash runway into 2027, supporting clinical execution and milestone achievement.

• Pharma partners have shown strong interest, with indications of providing bispecific antibodies free of charge for the trial.

• The company maintains independence in trial funding to maximize future partnering leverage.

Scientific and Market Rationale

• POLB001 targets a $10 billion+ market by addressing CRS, a severe side effect affecting up to 70% of patients on bispecific or CAR T-cell therapies.

• No approved therapies currently exist for CRS prevention; POLB001 offers a novel, orally available prophylactic option.

• Preclinical models and a phase 1B LPS challenge study demonstrated POLB001’s efficacy in reducing key cytokines and clinical symptoms.

• Oral delivery could ease healthcare system burdens by reducing hospital stays and enabling at-home treatment.

• Key opinion leaders and myeloma experts have strongly endorsed the program.

Regulatory and Commercial Advantages

• Orphan drug designation provides fee waivers, tax credits, and seven years of U.S. market exclusivity, protecting against competitors.

• The designation validates the scientific rationale and increases attractiveness to potential pharma partners.

• The company’s strategy is to remain agnostic to specific pharma partners until clinical data is available, maximizing deal potential.

• Market opportunity is expected to grow as the use of bispecifics and CAR Ts expands, with 500,000 patients projected in the U.S. and EU5 by 2030.

• Orphan drug pricing and reimbursement models support commercial viability even for rare disease indications.