Pheno Therapeutics Granted FDA IND Clearance For Lead Multiple Sclerosis Therapeutic Candidate PTD802

• US FDA clearance enables first-in-human study of PTD802, a selective GPR17 antagonist
• Small molecule therapeutic designed to promote remyelination in neurological diseases such as multiple sclerosis
• Milestone supports Pheno Therapeutics’ clinical development strategy

Pheno Therapeutics Limited. (Pheno), a clinical stage biotechnology company focused on the discovery and development of small molecule therapeutics for the treatment of neurological diseases, today announced that the US Food and Drug Administration (FDA) has cleared the  Investigational New Drug (IND) application for PTD802, the Company’s lead therapeutic candidate. The clearance allows Pheno to proceed with a first-in-human clinical trial of PTD802 in the US.

The announcement follows the Company’s clinical trial authorisation (CTA) from the UK’s MHRA in January 20251. The first-in-human study would evaluate the safety and tolerability of PTD802 in healthy volunteers. PTD802 is a novel small molecule therapeutic designed to promote remyelination, with an initial focus on multiple sclerosis (MS), a neurological disease with high unmet medical need.

Developed under an exclusive worldwide licence from UCB, PTD802 is a selective GPR17 (G protein-coupled receptor 17) antagonist, and is the first GPR17 programme to receive IND clearance, delivering a new class of neuroprotective therapeutics. This development also supports Pheno Therapeutics’ IP position in the US.

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

Fraser Murray, PhD, CEO, Pheno Therapeutics, said: “FDA IND clearance is an important milestone for our PTD802 programme, and a step further toward our ultimate goal of providing an effective treatment for neurological diseases associated with demyelination. As the first company to gain approval to begin clinical trials for a selective GPR17 antagonist, we are proud to be leading the way, and believe this approach has the potential to offer real patient benefit, in MS and beyond.”

About Pheno Therapeutics
Pheno Therapeutics is a biotechnology company, backed by leading biotech investors Advent Life Sciences and LifeArc, focused on the discovery and development of small molecule therapeutics that boost the function of human oligodendrocytes and their natural ability to promote remyelination, for the treatment of neurological diseases with high unmet medical need such as multiple sclerosis (MS).

Pheno combines world leading expertise in myelin biology, MS trials, patient selection and clinical cohorts. MS is a devastating chronic disease with significant individual and societal impact that often manifests in young adults and is associated with a wide range of neurological symptoms which can progress to total physical and cognitive disability. MS demyelination occurs when the immune system attacks and damages the myelin sheaths that insulate and nourish axons and nerve fibres in the central nervous system, leading to multifocal demyelination, axonal injury and neurodegeneration.

Pheno Therapeutics is seeking to promote remyelination and reverse the critical demyelination aspect of MS.

For more information, visit https://www.phenotherapeutics.com.