Healx has secured the US Food and Drug Administration (FDA) clearance for an investigational new drug application (IND) to commence a Phase II clinical trial of HLX-1502, targeting adults with neurofibromatosis Type 1 (NF1) and inoperable plexiform neurofibroma.
An orally administered tablet, HLX-1502 is a new treatment approach for NF1 patients, offering a differentiated investigational option.
Go deeper with GlobalData
Healx CEO Tim Guilliams said: “This IND approval marks another significant milestone in our efforts to harness powerful AI to develop a new treatment for NF1-associated plexiform neurofibroma.”
The investigational treatment received orphan drug and rare pediatric disease designations from the FDA for NF1 treatment.
These designations provide incentives to promote the development of therapies for rare diseases.
Healx’s research partner, Children’s Tumor Foundation (CTF), supports the advancement of the company’s NF programmes, including HLX-1502, through milestone-driven payments.
How well do you really know your competitors?
Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.
Thank you!
Your download email will arrive shortly
Not ready to buy yet? Download a free sample
We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form
By GlobalDataHealx’s drug discovery process is powered by Healnet, an AI-driven platform that utilises generative AI to identify new therapeutic opportunities for rare diseases.
This platform seeks to find connections between biological and chemical entities that could lead to new treatments.
The company’s latest $47m Series C funding round, co-led by R42 Group and Atomico, will further support the development of medicines for rare oncology, renal, and neurodevelopmental disorders.
The funding will also enable the progression of HLX-1502 through the Phase II clinical trial for NF1.
As part of the investment, R42 Group founder Ronjon Nag will join the board of Healx.
Guilliams added: “By December 2024, we will advance HLX-1502 into a Phase II clinical trial in patients with NF1 both for plexiform neurofibroma, a genetic condition with limited treatment options and for cutaneous neurofibroma, for which there are no FDA-approved options.
“We are also generating important preclinical data for multiple additional compounds identified using our novel generative AI drug discovery engine. Those compounds target rare diseases with significant unmet needs, including additional nerve-related tumour disorders, autosomal dominant polycystic kidney disease and neurodevelopmental disorders such as Angelman syndrome.”
