Poolbeg Pharma (POLB) , a clinical-stage biopharmaceutical company focused on infectious diseases, announced that Accelerating Clinical Trials (ACT), a specialist blood cancer organisation, will conduct its upcoming POLB 001 Phase 2a trial. Poolbeg also confirmed the supply of a bispecific antibody for the study at no cost to POLB.
POLB 001 is Poolbeg's flagship anti-inflammatory oral candidate proved effective in reducing Cytokine Release Syndrome (CRS) associated with certain cancer immunotherapies. The Phase 2a trial is titled TOPICAL (Trial of Prevention of ImmunoCytokine Adverse events in Myeloma) and will further investigate the safety and efficacy of POLB 001 in reducing the incidence of CRS.
Poolbeg believes POLB 001 has the potential to transform the cancer immunotherapy field by expanding administration from centralised specialist centres into community hospitals by making the treatments safer through the prevention of life-threatening CRS. As such, POLB 001 could meaningfully increase the number of patients that can receive these treatments.
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Poolbeg announces key steps toward the Phase 2a trial of its flagship candidate POLB 001. Accelerating Clinical Trials (ACT) has been contracted to conduct the study, with an approved antibody already secured at no cost to POLB. Poolbeg confirmed sufficient supply of GMP-grade POLB 001 for the study, which is at an advanced stage of preparation, with interim data expected in mid-2026. In terms of funding, Poolbeg's forecast cash runway extends into 2027, providing ample funding to develop POLB 001.
POLB 001 has already shown significant promise in treating CRS - a toxicity that occurs frequently following certain cancer immunotherapies. Due to CRS risk, administration of such immunotherapies is currently restricted to specialist centres, which limits their uptake. As an oral therapy to prevent or treat CRS, POLB 001 has the potential to enable broader use of the immunotherapies in an outpatient setting.
Earlier in 2025, Poolbeg presented data related to POLB 001 from a pre-clinical in vivo study, which evaluated the effect of POLB 001 on CRS compared to Adalimumab, the gold standard CRS inhibitor. In short, POLB 001 effectively reduced CRS and demonstrated superior cytokine inhibition compared to Adalimumab. Delivering an effective oral drug for CRS will be a significant value inflection point for Poolbeg, with POLB 001 having a 3rd party-estimated market potential of US$10bn.
The demand for effective CRS prevention is driven by the rapid growth of CRS-inducing cancer immunotherapies, with more than 70% of patients receiving such treatments affected. With the >US$10bn opportunity, there is strong potential for partnering on positive data from the Phase 2a trial. In May 2025, POLB 001 was granted an Orphan Drug Designation (ODD) by the FDA, further validating its value and increasing the appeal to prospective partners.
The FDA nod marked a significant milestone for POLB 001, as the ODD gives Poolbeg substantial clinical development and commercialisation benefits, including the potential for a 7-year market exclusivity period in the US, potential waiver exemption of Prescription Drug User Fee Act application fees, and tax credits for qualifying clinical trials.
POLB shares jumped 11% on today's announcement.
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