US investment boosts Healx in start to transformational 2024
The company is to receive unspecified investment from long-term partner the Children’s Tumor Foundation - a prominent patient group based in New York - to advance Neurofibromatosis Type 1 treatments.
Milestone payments from CTF will support the advance of Healx’s lead compound, HLX-1502, to clinical trials and broader Neurofibromatosis Type 1 programme.
The compound is on course to enter a Phase 2 clinical trial in the coming months.
The fresh backing from the Big Apple reinforces a 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.
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 aidded: “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 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.”
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 per cent 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 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.
Dr Guilliams said: "2024 is going to be a pivotal year for Healx as we further validate the ability of our AI-platform to identify potentially novel treatments for rare diseases, as evidenced by the advance of HLX-1502 towards clinical trials for neurofibromatosis type 1.
"We have a growing number of partners showing great confidence in our approach to identifying new therapies to treat rare diseases and we look forward to sharing more news in the coming months."
