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 U.S. Food and Drug Administration (FDA) has approved its investigational new drug (IND) application for its lead therapeutic candidate, PTD802. This approval allows Pheno to proceed with first-in-human clinical trials of PTD802 in the United States.
This announcement follows the company’s Clinical Trial Authorization (CTA) from the UK’s MHRA in January 2025. The first-in-human study will 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 license from UCB, PTD802 is a selective GPR17 (G protein-coupled receptor 17) antagonist and the first GPR17 program to receive IND approval, providing a new class of neuroprotective therapeutics. This development also supports Pheno Therapeutics’ intellectual property status in the United States.
In MS, the immune system attacks and damages the myelin sheath that insulates and nourishes axons and nerve fibers in the central nervous system, causing multifocal demyelination, axonal damage, and neurodegeneration. The disease is often associated with a wide range of neurological symptoms and can progress to complete physical and cognitive disability, despite the ability of existing drugs to control the inflammatory component.
The FDA IND approval is an important milestone for our PTD802 program and represents another step toward our ultimate goal of providing effective treatments for neurological diseases associated with demyelination. As the first company to receive approval to begin clinical trials of a selective GPR17 antagonist, we are proud to be leading the way and believe this approach has the potential to deliver real benefits for patients with MS and beyond. ”
Dr. Fraser Murray, Pheno Therapeutics CEO
