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Poolbeg Pharma plc announces exclusive option agreement to acquire Orphan Drug Candidate for Behçet's Disease
30 April 2024 - Poolbeg Pharma (AIM: POLB, OTCQB: POLBF, 'Poolbeg' or the 'Company'), a biopharmaceutical company focussed on the development and commercialisation of innovative medicines targeting diseases with a high unmet medical need, announces that it has entered into an exclusive 12-month option agreement with Silk Road Therapeutics Inc, for a nominal fee, to acquire a novel topical muco-adherent formulation of Pentoxifylline (tPTX) for the treatment of oral ulcers in patient's suffering from Behçet's Disease.
Key highlights
The topical formulation of PTX, a small molecule with broad anti-inflammatory activity, has successfully demonstrated accelerated oral ulcer healing and decreased pain compared to standard of care in patients with Behçet's Disease in a Phase 2 clinical trial.
The drug is positioned for a potential 505(b)(2) approval pathway in the U.S. which can result in a much less expensive and much faster route to approval and commercialisation. tPTX has successfully secured FDA Fast Track Designation, designed to expedite development and review of certain medicines. It has also received Orphan Drug Designation from the FDA, providing seven years of market exclusivity upon marketing authorisation.
Jeremy Skillington, PhD, Chief Executive Officer of Poolbeg Pharma, said: "The foundation of any pharma company is a strong pipeline of assets, and we believe that topical PTX could fit well into our pipeline alongside our other assets as we increase our focus on rare and orphan diseases.
"We are delighted to enter this exclusive option agreement with Silk Road Therapeutics and Dr Timothy Coté, whose extensive experience, including as Director of the FDA Office of Orphan Products Development, adds invaluable insights into the regulatory pathway and ultimate approval and commercialisation of the drug.
"tPTX has the potential to transform the lives of patients suffering from Behçet's Disease, who currently lack effective and safe long term treatment options. Under this exclusive option agreement, we will advance our due diligence process and engage with Silk Road Therapeutics to further understand the clinical pathway to approval, and we look forward to updating the market in due course."
Timothy R. Coté, M.D., M.P.H., Chief Executive Officer of Silk Road Therapeutics, said: "Poolbeg Pharma shares our commitment to advancing healthcare innovation to support rare disease patients. With Fast Track Designation and Orphan Drug Designation secured, topical PTX has the potential to swiftly reach patients and offer a potentially transformative solution for those suffering from Behçet's Disease."
Investor presentation
Poolbeg Pharma CEO, Jeremy Skillington, PhD, and CBO David Allmond, along with Dr Timothy Coté, CEO of Silk Road Therapeutics, will provide a live presentation via the Investor Meet Company platform on Tuesday 30 April 2024 at 6pm BST.
The presentation is open to analysts and investors, those who already follow Poolbeg on the Investor Meet Company platform will automatically be invited. Investors can sign up to Investor Meet for free and add Poolbeg Pharma plc to their company dashboard.
[1] Hatemi G, Yurttas B, Kutlubay Z, Cote T, Derkunt S, Yazici Y, Yazici H. Pentoxifylline Gel for Oral Ulcers in Patients with Behçet's Syndrome [abstract]. Arthritis Rheumatol. 2019; 71 (suppl 10). https://acrabstracts.org/abstract/pentoxifylline-gel-for-oral-ulcers-in-patients-with-behcets-syndrome/. Accessed April 17, 2024.
[2] Ali S, Nagieb CS, Fayed HL. Effect of Behcet's disease-associated oral ulcers on oral health related quality of life. Spec Care Dentist. 2023 Jul-Aug;43(4):435-442. doi: 10.1111/scd.12782. Epub 2022 Sep 20. PMID: 36127751.
[3] behcetsuk.org
Key highlights
- 12-month option agreement with Silk Road Therapeutics to acquire novel topical muco-adherent formulation of Pentoxifylline (tPTX), a treatment for oral ulcers in patient's suffering from Behçet's Disease which currently has no cure
- Phase 2 trial successfully completed, demonstrating superiority over standard of care
- Secured Orphan Drug Designation and Fast Track Designation from the FDA
- Positioned for a potential 505(b)(2) approval pathway in the U.S.
- Poolbeg will continue due diligence throughout the term of the exclusive option agreement which will include engagement with Silk Road Therapeutics to ascertain the clinical approval pathway
The topical formulation of PTX, a small molecule with broad anti-inflammatory activity, has successfully demonstrated accelerated oral ulcer healing and decreased pain compared to standard of care in patients with Behçet's Disease in a Phase 2 clinical trial.
The drug is positioned for a potential 505(b)(2) approval pathway in the U.S. which can result in a much less expensive and much faster route to approval and commercialisation. tPTX has successfully secured FDA Fast Track Designation, designed to expedite development and review of certain medicines. It has also received Orphan Drug Designation from the FDA, providing seven years of market exclusivity upon marketing authorisation.
Jeremy Skillington, PhD, Chief Executive Officer of Poolbeg Pharma, said: "The foundation of any pharma company is a strong pipeline of assets, and we believe that topical PTX could fit well into our pipeline alongside our other assets as we increase our focus on rare and orphan diseases.
"We are delighted to enter this exclusive option agreement with Silk Road Therapeutics and Dr Timothy Coté, whose extensive experience, including as Director of the FDA Office of Orphan Products Development, adds invaluable insights into the regulatory pathway and ultimate approval and commercialisation of the drug.
"tPTX has the potential to transform the lives of patients suffering from Behçet's Disease, who currently lack effective and safe long term treatment options. Under this exclusive option agreement, we will advance our due diligence process and engage with Silk Road Therapeutics to further understand the clinical pathway to approval, and we look forward to updating the market in due course."
Timothy R. Coté, M.D., M.P.H., Chief Executive Officer of Silk Road Therapeutics, said: "Poolbeg Pharma shares our commitment to advancing healthcare innovation to support rare disease patients. With Fast Track Designation and Orphan Drug Designation secured, topical PTX has the potential to swiftly reach patients and offer a potentially transformative solution for those suffering from Behçet's Disease."
Investor presentation
Poolbeg Pharma CEO, Jeremy Skillington, PhD, and CBO David Allmond, along with Dr Timothy Coté, CEO of Silk Road Therapeutics, will provide a live presentation via the Investor Meet Company platform on Tuesday 30 April 2024 at 6pm BST.
The presentation is open to analysts and investors, those who already follow Poolbeg on the Investor Meet Company platform will automatically be invited. Investors can sign up to Investor Meet for free and add Poolbeg Pharma plc to their company dashboard.
[1] Hatemi G, Yurttas B, Kutlubay Z, Cote T, Derkunt S, Yazici Y, Yazici H. Pentoxifylline Gel for Oral Ulcers in Patients with Behçet's Syndrome [abstract]. Arthritis Rheumatol. 2019; 71 (suppl 10). https://acrabstracts.org/abstract/pentoxifylline-gel-for-oral-ulcers-in-patients-with-behcets-syndrome/. Accessed April 17, 2024.
[2] Ali S, Nagieb CS, Fayed HL. Effect of Behcet's disease-associated oral ulcers on oral health related quality of life. Spec Care Dentist. 2023 Jul-Aug;43(4):435-442. doi: 10.1111/scd.12782. Epub 2022 Sep 20. PMID: 36127751.
[3] behcetsuk.org