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Healx to receive investment from Children’s Tumor Foundation to advance Neurofibromatosis Type 1 treatments

11 April 2024   |  
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  • Milestone payments from CTF will support advancement of Healx’s lead compound, HLX-1502, to clinical trials and broader Neurofibromatosis Type 1 programme
  • Investment reinforces long-term, patient-focused partnership combining Healx’s proprietary AI-powered drug discovery platform with CTF’s preclinical and clinical research and patient community knowledge

Cambridge, UK – 11 April 2024, 07:00 BST. Healx, the AI-powered, patient-inspired, techbio company pioneering the next generation of drug discovery, today announced that it has signed an investment agreement with its long-term partner, Children’s Tumor Foundation (CTF). Milestone-driven payments from CTF will support the advance of Healx’s AI-powered neurofibromatosis programme including its lead candidate, HLX-1502, which is on course to enter a Phase 2 clinical trial in the coming months.

Healx has partnered with CTF since 2020 to combine its cutting-edge AI platform and drug discovery expertise in identifying novel treatments for rare diseases such as Neurofibromatosis Type 1 (NF1) with CTF’s preclinical and clinical research and patient knowledge.

NF1 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, a MEK inhibitor, for a subset of patients with plexiform neurofibromas. There are no approved pharmacological treatments for patients with cutaneous neurofibromas, representing a significant unmet need for the estimated 3 million people with the disease across the globe.

Identified using Healx’s AI-informed approach, HLX-1502 has a novel mechanism of action, and is supported by data that suggest a favourable safety profile. Healx plans to develop HLX-1502 for both plexiform and cutaneous subtypes of NF1. Financial details are not disclosed.

Dr Tim Guilliams, co-founder and CEO, Healx said: “CTF is a long-standing partner of Healx, and this investment underscores their confidence in the potential of Healx’s neurofibromatosis programme to bring novel treatments to patients with unmet need. Over the years, Healx has been able to appreciate CTF’s expertise not only in terms of patient understanding, but also in the broader approach to developing treatments for this highly debilitating disease. Healx is pleased that CTF has decided to deepen its commitment to our AI-enabled research journey to deliver much-needed therapies to this patient group.”

Dr Annette Bakker, President, CTF said: “Patient-centric research holds a unique position in the R&D ecosystem and the Children’s Tumor Foundation is pleased to provide this approach to support Healx’s AI-driven search for new neurofibromatosis treatments. We operate as a strategic partner to drug discovery and development pioneers such as Healx, and we hope our commitment will encourage others to invest in such organisations who offer a real chance of improving the lives of the millions of NF patients waiting for treatments.”


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 10,000 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 by Dr Tim Guilliams (a Biochemical Engineer and techbio entrepreneur) and Dr David Brown (co-inventor of Viagra and former Global Head of Drug Discovery at Roche), Healx has raised over $100 million to date and added a dozen projects to its risk-balanced therapeutic portfolio. For more information, visit www.healx.ai or follow on X and LinkedIn.

About Children’s Tumor Foundation

The Children’s Tumor Foundation is the world’s leading organization dedicated to funding and driving innovative research that will result in effective treatments for the millions of people worldwide living with NF, a group of genetic conditions that causes tumors to grow on nerves throughout the body. As a trusted partner to pharmaceutical, biotech, and other industry partners, we collaborate closely to accelerate research and development efforts, ensuring that promising treatments reach those who need them. One in every 2,000 people is born with some type of neurofibromatosis or schwannomatosis, which may lead to blindness, deafness, bone abnormalities, disfigurement, learning disabilities, disabling pain, or cancer. NF affects all populations equally, and while there is no cure yet, the Children’s Tumor Foundation mission of driving research, expanding knowledge, and advancing care for the NF community fosters our vision of one day ending NF.  CTF would like to acknowledge Goodwin for its continuous pro bono support. For more information, please visit: ctf.org or follow us on LinkedIn.

Healx Media contact


Healx Partnership contact

Simone Manso

Head, Neurofibromatosis Therapy Development


CTF Media contact

Simon Vukelj

Chief Marketing Officer


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