Healx has dosed the first subject in the open-label Phase II INSPIRE-NF1 trial to assess the efficacy and safety of HLX-1502 in individuals with neurofibromatosis type 1 (NF1), a rare genetic disorder that causes tumours to form along nerves.
The single-arm trial focuses on oral investigational therapy’s efficacy and safety in NF1 subjects with plexiform neurofibromas (PNs), which are aggressive tumours that can lead to complications such as malignant transformation and functional impairments.
Nearly 20 subjects in the US are being enrolled for the trial, which will concentrate on important outcome measures such as tolerability, pharmacokinetics, safety as well as tumour response rate.
Last August, Healx received the US Food and Drug Administration (FDA) clearance for its investigational new drug application (IND) to begin the Phase II trial of HLX-1502.
Healx CEO and co-founder Tim Guilliams said: “For too long, patients have had limited treatment options that come with serious trade-offs. HLX-1502 is being evaluated as a potential treatment for NF1, with the goal of providing an alternative to existing MEK inhibitors, which have known side effects.
“If successful, this could mark the beginning of a paradigm shift in NF1 care—one that not only improves patient lives today but also paves the way toward a future where patients no longer have to choose between treatment effectiveness and quality of life.”
Discovered using the company’s AI platform, HLX-1502 has gained an orphan drug, rare paediatric disease, and fast-track designations from the FDA.
In April 2024, Healx signed an investment agreement with its long-term partner Children’s Tumor Foundation (CTF), which funds research aimed at developing treatments for individuals living with NF.
CTF’s milestone-driven payments will support the progression of Healx’s neurofibromatosis programme, including HLX-1502.
