Poolbeg Pharma (POLB) , a clinical-stage biopharma company focused on infectious diseases, said the US Food and Drug Administration had granted Orphan Drug Designation (ODD) to POLB 001 - Poolbeg's flagship anti-inflammatory candidate. The asset is a Phase 2-ready oral drug, proved effective in reducing Cytokine Release Syndrome (CRS) associated with certain cancer immunotherapies.
Poolbeg said the FDA had granted orphan status to POLB 001 as an oral preventative therapy for "T-cell engager bispecific antibody-induced CRS". The FDA grants this status to support the development of medicines for rare disorders affecting less than 200,000 people in the US.
The FDA nod marks 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 15% on the news.
Jeremy Skillington, CEO of Poolbeg, commenting: "POLB 001 is potentially a breakthrough, orally delivered, preventative therapy for cancer immunotherapy-induced CRS which could significantly impact patients' lives. We were delighted to receive Orphan Drug Designation from the FDA, which is a significant development for Poolbeg and for POLB 001, one that we believe will enhance the commercial appeal for prospective partners and help bring POLB 001 to the market faster."
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Poolbeg Pharma shares climbed 15% on the announcement that the US FDA had granted the company's flagship asset POLB 001 orphan status in the US. The status comes with significant commercial benefits and brings the anti-inflammatory candidate one step closer to commercial production. The status should also significantly help POLB's partnering discussions on the asset.
The Phase 2-ready POLB 001 has 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.
POLB 001 is approaching Phase 2a trials, with the first patient expected to be dosed in H2 2025. Interim analysis results are expected in H1 2026, followed by topline data in H2 2026. There are strong indications from Big Pharma to provide the necessary bispecific antibody, free of charge, to support the Phase 2a trial. This represents significant validation of the industry's interest in POLB 001, with the ODD further strengthening POLB 001's commercial appeal, and further validation expected from the Phase 2a trial.
Poolbeg recently presented excellent 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.
In terms of balance sheet, Poolbeg had a comfortable £6.2m cash at the end of March 2025. This will be materially bolstered by a proposed £4.1m fundraising to support the POLB 001 Phase 2a trial, and the proof-of-concept trial for POLB's oral GLP-1 programme. The increased cash balance will give POLB ample runway to develop its pipeline and ongoing negotiations with prospective partners into FY26 and beyond.
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