Poolbeg Pharma has been granted Orphan Drug Designation (ODD) by the US Food and Drug Administration for POLB 001, an oral therapy being developed to prevent cytokine release syndrome (CRS) caused by T-cell engager bispecific antibodies.

CRS is a potentially life-threatening immune response that affects more than 70 percent of patients receiving certain immunotherapies. There are currently no approved therapies that prevent this condition, which may lead to multi-organ failure or death in severe cases. POLB 001 aims to address this unmet need by acting as a p38 MAP kinase inhibitor to modulate the inflammatory response.

“POLB 001 is potentially a breakthrough, orally delivered, preventative therapy for cancer immunotherapy-induced CRS which could significantly impact patients’ lives,” said Jeremy Skillington, CEO at Poolbeg Pharma.

The FDA’s Orphan Drug Designation is granted to treatments for rare diseases affecting fewer than 200,000 people in the US. The designation provides incentives such as tax credits on clinical trial costs, potential waivers of certain user fees, and up to seven years of market exclusivity following regulatory approval.

Poolbeg expects to begin a Phase 2a clinical trial in the second half of 2025, with the first patient scheduled to be dosed later this year. Interim data are expected in the first half of 2026, and topline results in the second half. The company has noted interest from larger pharmaceutical companies willing to provide bispecific antibodies to support the study, which could represent a significant cost-saving and signal broader industry engagement.

“Orphan Drug Designation from the FDA underscores the urgency and importance of developing innovative therapies for this critical unmet medical need,” said Professor Brendan Buckley, non-executive director and scientific advisory board member.

“We look forward to progressing POLB 001 in our upcoming Phase 2a trial and working with regulatory agencies and potential partners.”

Skillington added that the designation enhances the therapy’s commercial potential, stating: “We believe this will help bring POLB 001 to market faster. If approved, it has the potential to improve quality of life for patients, reduce pressure on healthcare systems, and expand access to cancer immunotherapies.”

Poolbeg sees POLB 001 as a key part of its pipeline, offering a preventative approach to a serious side effect of modern cancer treatment. The next clinical phase will be essential in determining its role in future immuno-oncology protocols.