Pheno Therapeutics Receives Approval for Multiple Sclerosis Drug Trial PTD802

EDINBURGH, UK I January 14, 2025 I Pheno Therapeutics Limited, a biotechnology firm concentrating on the development of small molecule therapies for neurological ailments, has announced the approval of a clinical trial authorisation (CTA) by the UK Medicines and Healthcare products Regulatory Agency (MHRA) for its leading therapy, PTD802.

PTD802 is a new small molecule therapeutic that acts as a selective antagonist of GPR17, a G protein-coupled receptor. This innovative compound is designed to encourage remyelination, a process aimed at restoring the protective myelin sheath around nerve fibers that is damaged in conditions such as multiple sclerosis (MS). The development of PTD802 is part of an exclusive global licensing collaboration with UCB, which focuses on tackling neurological diseases that currently lack effective treatments. The initial target of this programme is MS.

Multiple sclerosis is a chronic, progressive condition characterized by the immune system mistakenly attacking the myelin sheath covering nerve fibers in the central nervous system. This immune-mediated damage results in multifocal demyelination, injury to axons, and eventual neurodegeneration. Despite existing therapies that manage the inflammatory aspects of MS, the disease can still advance to stages of severe physical and cognitive impairment.

Professor Siddharthan Chandran, Co-Founder of Pheno Therapeutics, emphasized the limitations of current MS treatments, which primarily address immune responses to reduce the severity and frequency of disease relapses. He pointed out the critical need for effective therapies that can prevent the progression of disability in MS patients, highlighting remyelination as a promising neuroprotective strategy. While GPR17 antagonists could be useful for other conditions, PTD802 is particularly promising as a pioneering oral agent for remyelination, potentially leading to novel combinatorial methods to halt MS progression.

Fraser Murray, PhD, CEO of Pheno Therapeutics, expressed the company's excitement upon receiving the MHRA's approval to advance PTD802 to a Phase 1 clinical trial. This progression marks a significant achievement for Pheno Therapeutics, transitioning it into a clinical-stage organization. Dr. Murray noted that Pheno Therapeutics is pioneering the administration of a selective GPR17 antagonist in healthy volunteers, setting a precedent in the quest to develop remyelination therapies targeting GPR17.

The initiation of this first-in-human programme signifies a crucial step towards Pheno Therapeutics’ aspiration to deliver breakthrough treatments for neurological diseases linked to demyelination. This advancement not only underscores the potential of PTD802 but also positions the company at the forefront of scientific efforts to develop new therapeutic options for individuals with neurological conditions.