Pheno Therapeutics receives clinical trial authorization for novel multiple sclerosis therapeutic

Pheno Therapeutics Limited., a biotechnology company focused on the discovery and development of small molecule therapeutics for the treatment of neurological diseases, announced today it has received clinical trial authorization (CTA) from the UK’s MHRA (Medicines and Healthcare products Regulatory Agency) for its lead candidate, PTD802.

A selective GPR17 (G protein-coupled receptor 17) antagonist, PTD802 is a novel small molecule therapeutic designed to promote remyelination. Developed under an exclusive worldwide license agreement with UCB, the programme is targeted toward treatment of neurological diseases with high unmet medical need, with an initial focus on multiple sclerosis (MS).

Demyelination in MS occurs when the immune system attacks and damages the myelin sheaths that insulate and nourish axons and nerve fibers in the central nervous system, leading to multifocal demyelination, axonal injury, and neurodegeneration. MS is a chronic disease caused by demyelination, often associated with a wide range of neurological symptoms, which despite the ability of existing drugs to control the inflammatory component of the disease, can progress to total physical and cognitive disability. 

Current treatments for MS focus mainly on the immune aspects of the disease, reducing severity and frequency of relapses. There is an urgent and unmet need for effective therapeutics that limit disability progression in MS, with remyelination offering a promising neuroprotective treatment. Whilst GPR17 antagonists have potential utility beyond MS, PTD802 is a hugely promising first-in-class oral remyelination agent which we believe will be the next step in devising combinatorial approaches to preventing MS progression.”

Professor Siddharthan Chandran, Co-Founder, Pheno Therapeutics

“We are delighted to have received approval from the MHRA to progress our PTD802 programme to a Phase 1 trial, a major milestone, marking our transition to a clinical stage organization. As the first company to carry out dosing of a selective GPR17 antagonist in healthy humans we are leading the way in the race to develop GPR17-targeting remyelination therapeutics,” added Fraser Murray, PhD, Chief Executive Officer of Pheno Therapeutics. “With this first-in-human programme we are moving closer to our goal of delivering transformational drugs for the treatment of neurological diseases associated with demyelination.”