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 authorization (CTA) from the UK's MHRA in January 2025. 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 program 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 fibers 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.
FDA IND clearance is an important milestone for our PTD802 program, 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."
Fraser Murray, PhD, CEO, Pheno Therapeutics