Poolbeg Pharma PLC (AIM:POLB) has secured a key regulatory boost from the US authorities for a drug it is developing to prevent a dangerous complication linked to advanced cancer treatments.
The London-listed biotech group said on Tuesday that the Food and Drug Administration had granted Orphan Drug Designation to POLB 001, an experimental treatment designed to protect patients from a condition known as cytokine release syndrome (CRS).
A frequent and potentially life-threatening side-effect of some cancer immunotherapies, the condition can cause a surge of immune activity, leading to high fever, organ failure and even death.
Under US rules, orphan status is reserved for medicines targeting diseases that affect fewer than 200,000 people in the country.
The designation gives companies a range of incentives, including tax breaks, a waiver of certain regulatory fees, and seven years of market exclusivity in the United States if the product is approved.
Jeremy Skillington, Poolbeg’s chief executive, 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."
POLB 001 is a tablet designed to block a protein called p38 MAP kinase, which plays a key role in triggering the inflammatory response linked to CRS. The company is preparing to start a mid-stage clinical trial in the second half of this year, with early results expected in the first half of 2026 and headline data by the end of that year.
Poolbeg said it had seen strong interest from larger pharmaceutical companies willing to provide the type of antibody used in the cancer treatment that causes CRS.
These antibodies are central to the trial and offering them free of charge would help reduce costs and demonstrate confidence in the approach, the company said.
There are currently no approved medicines that can be taken in advance to prevent CRS, and Poolbeg believes its product could improve access to cutting-edge cancer therapies by making them safer and easier to administer outside of hospitals.