Poolbeg Pharma advances POLB 001 into UK trial – ICYMI Proactive uses images sourced from Shutterstock
Poolbeg Pharma PLC (AIM:POLB, OTC:POLBF, FRA:POLBF) CEO, Jeremy Skillington, talked with Proactive about recent progress alongside Principal Scientist Liam Tremble, highlighting key regulatory and commercial developments for POLB 001.
Tremble explained that the company has secured full regulatory approval from the MHRA, alongside HRA and ARSAC clearances, marking a major milestone that allows the POLB 001 clinical trial to proceed in the UK.
He noted that this is “the last regulatory hurdle before we progress to opening up the sites and getting patients screened and into the study,” positioning the company to begin recruitment shortly. With six clinical sites now confirmed, including NHS Lothian and Royal Stoke University Hospital, the study is expected to generate interim data later this summer.
The trial is focused on cytokine release syndrome (CRS), an acute condition associated with certain immunotherapies. Tremble highlighted that the risk window occurs early in treatment, meaning data can be gathered quickly.
Skillington also outlined insights from independent US payer research, conducted to support discussions with potential partners. The findings confirmed a strong value proposition for POLB 001, with insurers recognising the significant cost burden associated with CRS. He stated that “the findings highlight pricing levels [and] demonstrate that 001 is a multi-billion dollar peak sale potential in the US,” underlining the scale of the opportunity.
The company sees POLB 001 as a potential preventative solution in a market with a clear unmet need, strengthening its position ahead of anticipated clinical data and future partnership discussions.
Proactive: Jeremy, Liam, very good to speak with you. I’ll start with you Liam, you’ve announced some significant updates for the POLB 001 topical trial. Please can you explain to investors why clinical trial authorization from the MHRA is such a significant milestone?
Liam Tremble: I am really delighted today to be able to announce that we have these regulatory approvals in place. This basically means that the trial is able to proceed in the United Kingdom. For these approvals, we submitted all of our preclinical, clinical and manufacturing data. They review them to make sure that the drug is safe to give to humans and that the protocol is designed properly.
Along with the MHRA approval, this normally comes with Health Research Authority approval and ARSAC approval, which is the radiation approval. All of those are now in place, meaning we now have all regulatory approvals to proceed with the study. This is the last regulatory hurdle before we progress to opening up the sites and getting patients screened and into the study.
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Proactive: You say you’re on track to deliver interim data this summer. Can you tell us more about the next steps and how the expansion to additional sites will impact the trial?
Liam Tremble: We’re also delighted to announce that we do have two more sites that have come on board for the trial, bringing us up to six. The additional sites are NHS Lothian in Edinburgh and the Royal Stoke University Hospital. These are leading research centres. It’s important that these sites have experience with bispecific antibodies and the management of cytokine release syndrome so that we collect high-quality data and ensure the drug is used appropriately.
We have site initiation visits scheduled and, all going well, we’re hoping to have patients screened and recruited very soon. Cytokine release syndrome is an acute condition that occurs early in treatment. The risk period is within the first couple of weeks, so we’re monitoring patients closely during that time. This means data will accumulate quickly, keeping us on track to deliver data later this summer.
Proactive: Jeremy, you also shared insights from independent US payer research. Can you share the rationale for conducting this research?
Jeremy Skillington: We conducted the research to support the value proposition for potential partners as we continue discussions. This is a relatively new therapeutic area, so it’s important to get feedback from US payers. These were independently conducted interviews with commercial insurers, Medicare and Medicaid providers, covering over 75 million lives.
We wanted to better understand the economic burden of cytokine release syndrome and the potential value of a preventative solution. It was important to confirm there is an unmet need and a significant market, which we were pleased to see.
Proactive: What were the key findings from the research and what do they mean for POLB 001?
Jeremy Skillington: The research confirmed a strong and clearly defined value proposition across these payers. Cytokine release syndrome occurs in many CAR-T and bispecific antibody treatments, and there’s no way to predict who will develop it or how severe it will be.
When CRS develops, treatment often has to be delayed or stopped, which impacts patient outcomes and increases hospital stays and costs. Prevention could reduce these issues significantly.
The insurers recognised the cost burden and assessed what they would be willing to pay to prevent CRS. The findings highlight pricing levels and demonstrate that POLB 001 has multi-billion-dollar peak sales potential in the US. There is a significant market opportunity, and this is compelling for potential partners.
Following this, we expect positive data from the clinical trial, and partnership discussions are likely to progress. This represents a strong commercial opportunity alongside the clinical data.
Proactive: Well, congratulations on this latest milestone. I hope you’ll keep us updated with your progress.