Poolbeg Pharma granted FDA orphan drug designation
27 May 2025 - Poolbeg Pharma (AIM: POLB, 'Poolbeg' or the 'Company'), a clinical-stage biopharmaceutical company focussed on the development of innovative medicines to address unmet medical needs, announces that the US Food and Drug Administration ("FDA") has granted Orphan Drug Designation ("ODD") to POLB 001 as an oral preventative therapy for T-cell engager bispecific antibody-induced Cytokine Release Syndrome ("CRS").
The FDA grants orphan status to support the development of medicines for rare disorders affecting less than 200,000 people in the US. ODD provides Poolbeg with clinical development and commercialisation benefits including the potential for a seven-year period of US market exclusivity following regulatory approval of POLB 001, potential waiver exemption of Prescription Drug User Fee Act application fees, and the potential for tax credits for qualifying clinical trials.
POLB 001 is a Phase 2 ready oral p38 MAP kinase inhibitor to prevent or treat cancer immunotherapy-induced CRS, a severe side-effect that occurs in more than 70%1 of patients and which may lead to multi-organ failure and death. There are currently no approved preventative therapies for CRS. This ODD further validates POLB 001's scientific rationale and data which, if approved, has the potential to revolutionise cancer immunotherapy delivery by making it safer, enabling outpatient administration, reducing healthcare burden, and ultimately expanding patient access to these breakthrough immunotherapies.
Poolbeg expects the first patient to be dosed in its proposed Phase 2a in H2 2025, with interim analysis expected in H1 2026 and Phase 2a 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 which represents a significant validation of the industry interest in POLB 001 and its potential. ODD strengthens POLB 001's commercial appeal for prospective partners and the Company sees potential for partnering on positive data from its Phase 2a trial.
Jeremy Skillington, PhD, Chief Executive Officer of Poolbeg Pharma, said:
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. If approved, we believe POLB 001 has the potential to improve quality of life for patients, reduce pressure on healthcare systems, and expand access to cancer immunotherapies.
Professor Brendan Buckley, Non-Executive Director and Scientific Advisory Board Member added:
Orphan Drug Designation from the FDA underscores the urgency and importance of developing innovative therapies for this critical unmet medical need. We look forward to progressing POLB 001 in our upcoming Phase 2a clinical trial and working closely with prospective partners and regulatory agencies to bring this potential therapy to patients as quickly as possible.
