Poolbeg Pharma PLC (AIM:POLB) CEO Jeremy Skillington joined Steve Darling from Proactive to announce a significant milestone in the company’s clinical development strategy: the U.S. Food and Drug Administration has granted Orphan Drug Designation to POLB 001, Poolbeg’s innovative oral therapy aimed at preventing Cytokine Release Syndrome caused by T-cell engager bispecific antibody immunotherapies.
POLB 001, an oral small molecule inhibitor of the IL-1 receptor, is designed to modulate the inflammatory response without suppressing the underlying anti-cancer immune activation. The FDA’s Orphan Drug Designation recognizes the potential of POLB 001 to address a rare but serious medical condition, affecting fewer than 200,000 individuals annually in the United States. This designation confers a range of developmental and commercial incentives including seven years of U.S. market exclusivity post-approval, waiver of certain FDA fees, such as the Prescription Drug User Fee Act or PDUFA application fees and others.
The company plans to initiate a Phase 2a clinical trial in the second half of 2025, marking the next step in evaluating POLB 001’s efficacy and safety in a controlled setting. Poolbeg anticipates completing interim analysis in the first half of 2026, with topline data expected in the second half of 2026.
With the Orphan Drug Designation in hand, Poolbeg is now well-positioned to advance discussions with potential partners and stakeholders as it progresses toward its clinical milestones.
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