Poolbeg Pharma PLC has announced positive interim results for the first half of 2024, highlighting the progress of its lead development candidate, POLB 001, and its strategic focus on rare and orphan diseases. The company's efforts to develop innovative medicines for unmet medical needs are showing promise, with POLB 001 demonstrating efficacy in reducing cancer immunotherapy-induced Cytokine Release Syndrome (CRS) in preclinical studies.
POLB 001: A Potential Breakthrough for CRS
Cytokine Release Syndrome (CRS) is a systemic inflammatory response that can occur following cancer immunotherapy, particularly with T cell-engaging bispecific antibodies or CAR T-cell therapy. Affecting over 70% of patients receiving these treatments, CRS poses a significant clinical challenge, leading to increased healthcare costs and limiting access to specialized cancer centers. Poolbeg's POLB 001 is being developed as an orally delivered preventative therapy for cancer immunotherapy-induced CRS.
In vivo data has demonstrated POLB 001's efficacy in reducing cancer immunotherapy-induced CRS symptoms in an animal model. This positive data has strengthened and facilitated the expansion of patent applications for POLB 001 in this indication. Furthermore, the US Patent Office has granted Poolbeg's Immunomodulator II patent application, covering a class of drugs, including POLB 001, to treat or prevent hypercytokinemia (cytokine storm) induced in any disease indication.
Independent research commissioned by Poolbeg has confirmed a potential market opportunity of approximately US$10 billion for POLB 001 in Multiple Myeloma and Diffuse Large B-Cell Lymphoma alone. As cancer immunotherapies continue to advance across a broader range of hematological malignancies and solid tumors, the potential market for POLB 001 is expected to expand significantly.
Expanding into Rare and Orphan Diseases
Poolbeg is also increasing its focus on rare and orphan diseases. In April 2024, the company signed an exclusive 12-month option agreement with Silk Road Therapeutics Inc. to acquire tPTX, a novel, topical, muco-adherent formulation of Pentoxifylline for the treatment of oral ulcers in Behçet's Disease patients. Behçet's Disease, a chronic inflammatory condition with no cure, causes severe inflammation leading to debilitating symptoms, including painful oral ulcers.
tPTX has secured FDA Fast Track and Orphan Drug Designation, providing seven years of market exclusivity upon marketing authorization, and is potentially positioned for the expedited 505(b)(2) approval pathway in the US. Phase 2 trial data presented at the American College of Rheumatology meeting in 2019 demonstrated faster healing and pain reduction with tPTX compared to standard of care treatments.
Advancing the Oral GLP-1R Agonist Program
Poolbeg is also progressing its orally-delivered, glucagon-like peptide 1 receptor agonist (GLP-1R) program, with clinical trials expected to commence in late 2024. With approximately 42% of the US population affected by obesity, the economic impact of the condition is substantial. The GLP-1R market is projected to reach US$150 billion by 2031. Poolbeg's program aims to address the unmet need for effective oral GLP-1R options, utilizing a delivery system that enhances bioavailability and improves convenience.
Financial Position and Outlook
As of June 30, 2024, Poolbeg had a cash balance of £10.1 million. The company's strategy remains focused on partnering in-house programs to realize near-term value, while also targeting revenue generation and profitability from commercial-stage rare and orphan drugs. The addition of former Amryt Pharma team members, with proven experience in scaling a rare disease company, further strengthens Poolbeg's leadership team and its ability to execute on its growth strategy.
