Poolbeg Pharma Plc CEO talked with Proactive about the company's recent achievement of orphan drug designation from the US FDA for POLB 001.
Jeremy Skillington explained that this designation supports the development of treatments for rare diseases and offers Poolbeg benefits, including seven years of market exclusivity in the US, tax credits, and exemption from FDA user fees, which could amount to approximately $2 million.
Skillington highlighted that the designation significantly enhances the drug’s commercial appeal, especially when engaging prospective partners. POLB 001 is being developed to address cytokine release syndrome (CRS), a common and serious side effect seen in around 70% of patients undergoing cancer immunotherapy with bispecific antibodies or CAR-T cell therapies.
Skillington noted that by helping reduce hospitalisation caused by CRS, POLB 001 could benefit patients, healthcare systems, and boost revenue for cancer immunotherapies.
He detailed a planned Phase IIa clinical trial in multiple myeloma patients, due to begin in the second half of 2025, with interim results expected in early 2026 and topline data by year-end. Poolbeg has received bispecific antibodies from big pharma partners free of charge for the trial, which Skillington said reflects industry interest.
Poolbeg recently raised £4.87 million, adding to its £6.2 million cash reserve, providing funding through 2027.
Proactive: Jeremy, it's great to see you again. How are you?
Jeremy Skillington: I'm very well, Steve. Very happy.
Proactive: I can imagine the company out with some pretty significant news today that you've been granted orphan drug designation for POLB 001. For those unfamiliar with how this works in the US, maybe you can explain what this designation means.
Jeremy Skillington: Yeah, absolutely. Very exciting news for Poolbeg this morning. We received orphan drug designation from the FDA for POLB 001. The FDA grants this status to support development of medicines for rare disorders that affect fewer than 200,000 people in the US. It's an incentive programme to encourage companies to develop treatments for rare diseases.
It gives Poolbeg clinical development and commercialisation benefits, which is obviously very exciting. You get the potential for seven years of US market exclusivity once the drug is approved. There are also other incentives like exemption from the $2 million prescription drug user fee and tax credits for clinical trials. So fantastic news for Poolbeg that came in late on Friday.
Proactive: This must be very positive for your discussions with prospective partners?
Jeremy Skillington: Absolutely. We’ve generated very interesting data for POLB 001. We have a clinical trial planned later this year and hope to generate more exciting data. This orphan drug designation significantly enhances the commercial appeal to partners. That seven-year exclusivity brings a lot of value. We’re very excited and looking forward to updating our partners.
Proactive: Can you talk about the broader patient context?
Jeremy Skillington: Patients receiving cancer immunotherapies, T-cell engagers, have to go into cancer centres for extended periods. These drugs are efficacious, but there’s a side effect issue: cytokine release syndrome (CRS). If it develops, patients have to stay in hospital longer. That’s a challenge for healthcare systems and slows patient throughput.
What we hope with POLB 001 is to reduce hospitalisation. CRS occurs in about 70% of patients receiving bispecific antibodies or CAR-T cell therapies. We’re aiming to address that. It’s a $10 billion market. Preventing CRS would reduce hospital stays and support revenue growth for cancer immunotherapies. So we’re facilitating on multiple fronts.
Proactive: Are there any inflexion points we should be watching for?
Jeremy Skillington: Yes, it’s a busy year. We’re starting a Phase IIa trial in the second half of this year in multiple myeloma patients. We’ll give POLB 001 prophylactically to prevent CRS before they get their bispecific antibody. It'll be an open-label, single-arm study, giving the drug twice a day. About 30 patients will be included.
We’ll get interim data in early 2026 and topline data by the end of that year. So a very quick trial with regular data flow. We’ve had strong interest from clinicians and big pharma, some of whom will provide bispecific antibodies free of charge for the trial. That’s a great endorsement.
Proactive: You also raised just under $5 million recently. Will that support this work?
Jeremy Skillington: Yes. That raise was on the back of the orphan drug designation. Last quarter was tough for fundraising, especially in biopharma. We raised £4.87 million – over $6 million – during challenging times. That will go towards the clinical trial, including background work. We also had £6.2 million in cash at the end of March, giving us runway into 2027.
Proactive: Anything else in the pipeline?
Jeremy Skillington: Yes, alongside oncology, we’ve got an oral encapsulated GLP-1 programme moving quickly. That’s entering the clinic this year with data expected in the first half of next year. It’s a 20-patient study run by Professor Carel Le Roux at University of Ulster.
With the orphan drug designation and the fundraise, we’re in good shape for an exciting 12–24 months ahead.
Proactive: Absolutely no time to chat, you’re too busy! Jeremy, thank you for your time.