The US FDA has cleared PTD802 for a first-in-human trial, allowing Pheno Therapeutics to move the experimental MS treatment into clinical testing.
The Investigational New Drug clearance allows the company to proceed with a clinical trial in the US.
The first study would assess the safety and tolerability of the treatment in healthy volunteers.
Fraser Murray, PhD, chief executive of Pheno Therapeutics, said: “FDA IND clearance is an important milestone for our PTD802 programme, and a step further towards 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.”
The announcement follows clinical trial authorisation granted by the UK’s Medicines and Healthcare products Regulatory Agency in January 2025.
PTD802 is a novel small-molecule treatment designed to promote remyelination, the process of restoring the protective myelin coating around nerves.
It is initially being developed for multiple sclerosis, a neurological condition for which Pheno said there remains a high unmet medical need.
The treatment was developed under an exclusive worldwide licence from pharmaceutical company UCB.
It is a selective GPR17 antagonist, designed to block G protein-coupled receptor 17, and is the first GPR17 programme to receive Investigational New Drug clearance.
Pheno said the programme could provide a new class of treatments aimed at protecting nerve cells. The development also supports the company’s intellectual property position in the US.
In multiple sclerosis, the immune system attacks and damages the myelin sheaths that insulate and nourish axons and nerve fibres in the central nervous system.
This can cause myelin loss in several areas, damage to axons and neurodegeneration, the progressive loss of nerve cell structure or function.
The condition can cause a wide range of neurological symptoms. Although existing drugs can control inflammation, it can progress to total physical and cognitive disability.
