Healx Begins Phase 2 Trial Dosing for HLX-1502 in Neurofibromatosis Type 1
Healx, an AI-driven, clinical-stage biotechnology company focused on developing treatments for rare diseases, has announced a major milestone in its efforts to provide new therapeutic options for patients with neurofibromatosis type 1 (NF1). The company has successfully dosed the first patient in INSPIRE-NF1, a Phase 2 clinical trial evaluating the safety and efficacy of HLX-1502, an investigational oral therapy, for individuals suffering from this debilitating genetic disorder.
Understanding Neurofibromatosis Type 1 (NF1)
Neurofibromatosis type 1 is a complex genetic condition that affects approximately one in 2,500 individuals worldwide. The disorder is characterized by the formation of benign tumors along nerves throughout the body, often beginning in childhood. These tumors, known as plexiform neurofibromas, can cause severe pain, disfigurement, and, in some cases, malignant transformations leading to aggressive cancers. NF1 is also associated with a range of additional symptoms, including learning disabilities, skeletal abnormalities, cardiovascular issues, and vision impairment, which further complicate patient care and management.
Despite the significant impact NF1 has on those diagnosed, treatment options remain limited. Currently, the primary approaches involve surgical removal of tumors or the use of MEK inhibitors—medications that have shown efficacy in reducing tumor size but are often associated with significant side effects, including severe fatigue, gastrointestinal issues, and skin rashes. These limitations underscore the urgent need for novel and better-tolerated therapeutic alternatives.
HLX-1502: A Potential Breakthrough
HLX-1502, the investigational therapy at the center of the Phase 2 INSPIRE-NF1 trial, was discovered using Healx’s proprietary artificial intelligence (AI) platform. This platform is designed to accelerate the identification of promising drug candidates by analyzing vast datasets and predicting potential treatments more efficiently than traditional drug discovery methods.
“This trial represents an important step in determining the potential of HLX-1502 as a treatment option for patients,” said Simone Manso, head of neurofibromatosis therapy development at Healx. “There are no treatments for many NF1 symptoms; current treatments for NF1 plexiform neurofibromas, such as surgery and MEK inhibitors, are helpful but come with their own set of trade-offs, in terms of lasting complications and side effects. There is a need for new treatments for the NF1 community that balance effectiveness and safety. Evaluating HLX-1502 in this context is a key focus of our research efforts. We are grateful for the support from the Children’s Tumor Foundation, a vital partner in this journey, and to the Neurofibromatosis Clinical Trials Consortium for making this clinical trial a reality.”
The investigational therapy has already received multiple regulatory designations from the U.S. Food and Drug Administration (FDA), including Fast Track, Orphan Drug, and Rare Pediatric Disease designations. These designations highlight the urgency of developing treatments for NF1 and recognize the potential of HLX-1502 to meet the critical unmet medical needs of this patient population.
The Significance of the INSPIRE-NF1 Trial
The Phase 2 INSPIRE-NF1 trial is designed to assess the safety, tolerability, and therapeutic efficacy of HLX-1502 in patients diagnosed with NF1. Conducted in collaboration with the Neurofibromatosis Clinical Trials Consortium, the study involves multiple clinical centers across the United States and Australia.
The trial’s primary goal is to evaluate the ability of HLX-1502 to target and reduce the growth of plexiform neurofibromas while maintaining a favorable safety profile. Secondary objectives include assessing improvements in pain management, overall quality of life, and functional outcomes in patients undergoing treatment.
“This is much more than a company milestone—it represents a new hope for the NF1 community,” said Tim Guilliams, Ph.D., co-founder and CEO of Healx. “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.”
The Role of AI in Drug Discovery
The use of artificial intelligence in pharmaceutical research has been rapidly transforming the drug development landscape, particularly for rare diseases, which often receive less investment due to the small patient population. Healx’s AI-driven approach aims to significantly shorten the time it takes to bring promising therapies from the lab to the clinic.
AI enables researchers to analyze large-scale biomedical datasets, identify potential drug repurposing opportunities, and predict how compounds interact with disease mechanisms. This capability is particularly crucial for rare diseases like NF1, where traditional drug discovery methods can be slow and costly.
Healx’s innovative methodology has already demonstrated success in identifying promising candidates for clinical evaluation, and HLX-1502 represents one of the most advanced examples of AI-driven drug discovery reaching the clinical stage.
Collaboration for a Cure
A major driving force behind the advancement of HLX-1502 into clinical trials is the collaboration between Healx and leading institutions dedicated to NF1 research and treatment. The NF1 Clinical Trials Consortium, a network of 24 clinical centers across the United States and Australia, plays a crucial role in advancing research for individuals affected by neurofibromatosis and related conditions.
Michael Fisher, M.D., director of the NF Program and chief of the Neuro-Oncology Section at the Children’s Hospital of Philadelphia, and the group chair of the NF1 Clinical Trials Consortium, expressed optimism about the trial’s potential impact.
“We are excited about the launch of this trial and what it could mean for thoseHealx affected by NF1 worldwide,” said Dr. Fisher. Healx “Our consortium, consisting of leading researchers Healx and clinicians, is committed to pushing NF research forward and finding new therapies for individuals with neurofibromatosis and schwannomatosis. Healx’s innovative approach to treatment discovery gives us renewed hope for continued meaningful progress.”
The NF1 Clinical Trials Consortium operates with an Operations Center based at the University of Alabama at Birmingham (UAB), led by Girish Dhall, M.D., and Karen Cole-Plourde. Additionally, the consortium’s Data Coordinating Center at the Children’s Hospital of Philadelphia is managed by Richard Aplenc, M.D., Ph.D.
