Cambridge, UK – 06 September 2023, 12:00 BST. Healx, the AI-powered, patient-inspired, techbio company pioneering the next generation of drug discovery has received Orphan Drug Designation from the US Food and Drug Administration (FDA) for its AI-discovered treatment of Neurofibromatosis Type 1 (NF1). Their team of expert scientists and technologists discovered HLX-1502 as a potential treatment for NF1 through the application of their innovative AI drug discovery platform.
Neurofibromatosis Type 1 is a rare genetic condition that causes tumours to grow along the nerves. The two most common types of tumours are plexiform and cutaneous. Currently, there is only one approved treatment for a sub-population of plexiform NF1 patients, a MEK inhibitor which has known side effects. There are no approved treatments for cutaneous NF1 patients. This represents a significant unmet need for the estimated 2 million people with the disease across the globe.
Currently, other companies are focused on the development of other MEK or kinase inhibitors for the treatment of NF1. Healx’s novel AI-informed approach discovered HLX-1502 which has both a first-in-class mechanism, as well as data which gives confidence that a good safety profile should be achievable.
Healx aims to develop HLX-1502 for both plexiform and cutaneous subtypes of NF1, thus enabling access to treatment for a wide range of NF1 patients. Healx plans to submit an Investigational New Drug (IND) application to take its wholly-owned HLX-1502 program into clinical trials in 2024.
Simone Manso, Head of Neurofibromatosis Therapy Development commented: