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Healx receives US Orphan Drug Designation for its AI-discovered treatment of Neurofibromatosis Type 1 (NF1)

FDA grants HLX-1502 Orphan Drug Designation (ODD) for the treatment of Neurofibromatosis Type 1 (NF1):
  • Neurofibromatosis Type 1 is a rare genetic condition that causes tumour growth on the nerves, affecting over 2 million people across the globe.
  • HLX-1502 was discovered by Healx’s proprietary AI drug discovery platform, and has a first in class mechanism for the treatment of Neurofibromatosis Type 1.
6 September 2023   |  
X minutes
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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:

We are proud to receive the Orphan Drug Designation for HLX-1502, it marks a significant milestone for Healx. This designation not only recognizes the innovative potential of our AI-driven drug discovery platform, but also underscores our commitment to addressing the unmet medical needs of NF1 patients and the value of working together with our partner, The Children’s Tumor Foundation, and the NF community. Together, we have a unique opportunity to make a profound impact on the lives of people living with NF1, which is what matters the most.”

The FDA grants Orphan Drug Designation to potential new medicines intended for the treatment, diagnosis or prevention of rare diseases, or disorders, that affect fewer than 200,000 people in the United States. As well as granting HLX-1502 eligibility for seven years of market exclusivity, the designation will provide a number of other pre-marketing approval benefits. These include tax credits for running related clinical research in the United States, waiver of the Prescription Drug User Fee Act, and regulatory guidance from the FDA.

About Healx

Healx is a mission-driven techbio company pioneering the next generation of drug discovery in order to bring novel, effective treatments to rare disease patients around the world. There are 7,000 known rare diseases that affect 400 million people across the globe, but only 5% of those conditions have an approved treatment. By combining frontier AI technology with deep drug discovery and development expertise, Healx can accelerate the pace, increase the scale and improve the chance of success of rare disease treatment development in order to meet this huge unmet need and have unprecedented patient impact.

Founded in Cambridge, UK, in 2014 by Dr Tim Guilliams (a Biochemical Engineer and tech entrepreneur) and Dr David Brown (co-inventor of Viagra and former Global Head of Drug Discovery at Roche), Healx has raised around $90 million to date, added 16 projects to its risk-balanced therapeutic portfolio. For more information, visit www.healx.ai or follow on X and LinkedIn.

For media interviews and questions and more information about Healx or our programs, please contact:

Nick Street

Head of Operations, People and Technology


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