Poolbeg Pharma PLC Announces Option Agreement to Acquire Orphan Drug Candidate

Exclusive Option Agreement to Acquire Orphan Drug Candidate for Behçet's Disease

Novel therapeutic with Fast Track and Orphan Designation

LONDON, UK / ACCESSWIRE / April 30, 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
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

There is a clear unmet medical need for an effective treatment for this rare disease, which has no cure. Behçet's Disease causes inflammation of blood vessels and tissues, resulting in debilitating symptoms, the most common being oral ulcers which impact essential functions like eating, drinking and speaking.^[1] Patients also suffer from reduced psychosocial quality of life^[2]. Current standard of care is inadequate with safety concerns and there is a clear need for alternative treatment options.

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.