University of Edinburgh biotechnology spinout Pheno Therapeutics has announced its lead therapeutic candidate has been cleared as an Investigational New Drug (IND) by the US Food and Drug Administration (FDA), allowing it to move to healthy volunteer studies.
Its candidate is PTD802 - 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.
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.
The announcement follows the Company’s clinical trial authorisation from the UK’s MHRA. The first-in-human study will evaluate the safety and tolerability of PTD802 in healthy volunteers.
Supported by Edinburgh Innovations, Pheno Therapeutics spun out of the College of Medicine and Veterinary Medicine in 2019, co-founded by phenotypic screening pioneer Professor Neil Carragher of the University’s Institute of Genetics and Cancer and Professor Siddharthan Chandran, of the Institute for Neuroscience and Cardiovascular Research.
The spinout was built on Professor Carragher’s Phenomics Drug Discovery platform, combined with Professor Chandran’s human induced pluripotent stem cell expertise and clinical regenerative neurology specialism. The company has been funded by Advent Life Sciences and LifeArc Ventures.
Edinburgh Innovations (EI) supported the translation of research and spin out process, and Old College Capital manages the University’s shareholding.
Dr Susan Bodie, EI’s Director of Innovation Development and Licensing and Pheno Therapeutics Board Observer, said:
Congratulations to the Pheno team on this important stage in the long journey from lab to clinic. The company is built on decades of research, clinical experience and cutting-edge science that ultimately offers hope for patients with the devastating condition of MS. ”
Dr Fraser Murray, CEO of 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. ”
