Study reveals POLB 001 could be a $10 billion game changer for cancer patients

Poolbeg Pharma (AIM: POLB, OTCQB: POLBF, 'Poolbeg' or the 'Company'), a biopharmaceutical company focused on the development and commercialisation of innovative medicines targeting diseases with a high unmet medical need, today announces that independent research[i] [ii] conducted on behalf of Poolbeg confirms a >$10 billion market opportunity for POLB 001 in Cancer Immunotherapy-Induced CRS as an orally delivered preventative therapy. Cancer immunotherapies have been approved in rare and orphan blood cancers and so the Company can see potential for POLB 001 in one or more of these cancer types. POLB 001 in cancer immunotherapy-induced CRS will be developed alongside Poolbeg's existing portfolio of assets including its influenza programme; its AI drug discovery programmes; and oral delivery of GLP-1 for obesity and other metabolic diseases.

Rare and orphan diseases represent a significant value creation opportunity for the Company and its shareholders as they are underserved and there remains a substantial opportunity for innovative medicines to treat these severe and often chronic conditions. It is anticipated that acquiring and commercialising rare and orphan drugs could potentially lead to attractive revenue generation opportunities. Poolbeg will draw on the expertise of the wider team, including the individuals of the former Amryt team, who have a proven track record of successfully building and commercialising orphan drugs. Regulatory authorities offer incentives to companies developing orphan drugs, such as reduced filing fees, tax breaks and market exclusivity post approval and the Company Directors believe that Poolbeg is well positioned to benefit from these opportunities.

The Company also announced further detail on the significant market opportunity for POLB 001 CRS induced by CAR-T and bispecific antibody treatments (BsAb), where CRS can affect the majority of patients and can lead to extended hospital stays, discontinuation of treatment and mortality risk. There are currently few approved therapies for the management of acute CRS and no preventative therapies indicated for CRS.

The size and attractiveness of this market has encouraged the Company, as the field of cancer immunotherapies, including CAR-T and BsAb, is burgeoning and is expected to grow to US$100 billion - US$140 billion[iii] by 2030. CRS occurs in the majority of CAR-T and BsAb treatments frequently impacting >70%[iv] of patients and cannot be predicted. All grades of CRS can lead to extended hospital stays, causing an estimated US$5.5 billion[v] of hospitalisation costs to healthcare systems, and further indirect costs through patient treatment and follow up.

An effective oral preventative therapy for cancer immunotherapy-induced CRS has the potential to significantly reduce direct costs in the healthcare system and improve patient access to these therapeutics, potentially including access to these therapies outside of specialist centres, while further augmenting wider market uptake.

Poolbeg recently announced promising in vivo results for POLB 001 in addressing cancer immunotherapy-induced CRS, where CRS clinical symptoms were significantly improved by administration of POLB 001. Dose dependent reductions were seen in all serum proinflammatory cytokines measured.

Jeremy Skillington, Chief Executive Officer, Poolbeg Pharma plc said: "Rare and Orphan disease medicines fit Poolbeg's efficient capital model particularly well. Clinical development is typically more cost efficient and the market opportunity remains high because of the high unmet medical need. There is also a very active M&A market in this sector which Amryt took advantage of. We see this strategic approach as highly complementary to our existing programmes. The former Amryt team that have joined Poolbeg have an exceptional track record, have hit the ground running and are aligned with our vision of rapidly generating revenues and creating value for shareholders."


  • [i] Grand View Research. CAR T-Cell Therapy Market Analysis 2023-2030; Bispecific Antibodies Market Size, Share & Trends Analysis Report.
  • [ii] Datamonitor Healthcare. Forecast: Diffuse Large B-Cell Lymphoma and Multiple Myeloma, 2023.
  • [iii] Grand View Research. CAR T-Cell Therapy Market Analysis 2023-2030; Bispecific Antibodies Market Size, Share & Trends Analysis Report.
  • [iv] Average rate from Summary of Product Characteristics (SmPCs) for Yescarta, Tecartus, Abecma, Kymriah, Carvykti, Breyanzi, Elrexfio, Columvi, Epkinly, Tecvayli and Talvey
  • [v] Adapted from 1. Datamonitor Healthcare. Forecast: Diffuse Large B-Cell Lymphoma and Multiple Myeloma, 2023 2. Abramson JS et al. Cytokine release syndrome and neurological event costs in lisocabtagene maraleucel-treated patients in the TRANSCEND NHL 001 trial. Blood Adv. 2021 Mar 23;5(6):1695-1705.

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