Healx Doses First Patient in Phase 2 Trial of HLX-1502 for Neurofibromatosis Type 1
Healx, a clinical-stage biotech company utilizing AI to advance treatments for rare diseases, has announced that the first patient has been dosed in its Phase 2 trial, INSPIRE-NF1. This trial is evaluating the safety and efficacy of HLX-1502, an oral investigational therapy, for treating neurofibromatosis type 1 (NF1).
Neurofibromatosis Type 1 (NF1) is a rare genetic disorder that causes tumors to form along the nerves, often starting in childhood. Affecting approximately one in 2,500 individuals worldwide, it can lead to debilitating complications, including pain, disfigurement, and malignancies. Despite its severe impact on patients’ lives, treatment options are limited, leaving many individuals with few choices but invasive surgeries or therapies that carry significant side effects. This stark reality has prompted the need for innovative solutions, and Healx, a clinical-stage biotech company specializing in rare diseases, is addressing this challenge with its ongoing Phase 2 trial of HLX-1502.
A Breakthrough in NF1 Treatment
The Phase 2 trial, named INSPIRE-NF1, is designed to assess the safety and efficacy of HLX-1502, an oral investigational therapy, in patients with NF1. The trial marks a crucial step forward for patients who currently lack effective treatment options. “This trial represents an important step in determining the potential of HLX-1502 as a treatment option for patients,” said Simone Manso, Healx’s head of neurofibromatosis therapy development. “There are no treatments for many NF1 symptoms; current therapies, such as surgery and MEK inhibitors, are helpful but come with their own set of complications and side effects. We are focused on finding new treatments for the NF1 community that balance safety and effectiveness. HLX-1502 holds promise in addressing this need.”
The therapy was discovered using Healx’s AI-driven platform, which accelerates the identification of potential drug candidates. The U.S. Food and Drug Administration (FDA) has granted HLX-1502 Fast Track, Orphan Drug, and Rare Pediatric Disease designations, recognizing its potential to significantly impact the NF1 community. These designations provide a streamlined path for HLX-1502’s clinical development and future approval.
HLX-1502’s Promise for the NF1 Community
Healx’s CEO, Tim Guilliams, Ph.D., emphasizes the transformative potential of HLX-1502. “This is much more than just a company milestone; it represents renewed hope for the NF1 community,” Guilliams stated. “For too long, patients have had limited treatment options, many of which come with severe side effects. HLX-1502 is being evaluated as an alternative to existing MEK inhibitors, which are effective but carry known risks. If successful, HLX-1502 could mark a paradigm shift in NF1 care—offering patients a treatment option that doesn’t compromise their quality of life.”
Current treatments, such as MEK inhibitors, provide relief but also come with complications. The aim of HLX-1502 is to offer a safer, more effective option for managing NF1, particularly in patients suffering from plexiform neurofibromas (PNs), a type of tumor commonly associated with NF1.
Support from Key Partners
Healx’s innovative approach has garnered support from critical partners in the field. “We are excited about the launch of this trial and the potential impact on those affected by NF1,” said Michael Fisher, M.D., director of the NF Program at the Children’s Hospital of Philadelphia, and the group chair of the NF1 Clinical Trials Consortium. “Our consortium is dedicated to advancing NF research and discovering new therapies for individuals with NF1 and other neurofibromatosis-related disorders. Healx’s novel approach to treatment discovery gives us renewed hope for progress.”
The NF1 Clinical Trials Consortium, which consists of 24 clinical centers across the United States and Australia, plays an essential role in pushing forward the research for NF1 treatments. Healx’s collaboration with this consortium strengthens its position as a leader in developing effective therapies for rare diseases.
The INSPIRE-NF1 Study: Key Details
INSPIRE-NF1 is an open-label, single-arm Phase 2 study enrolling approximately 20 patients in the U.S. The study focuses on patients with NF1 and PNs, aggressive tumors that can lead to severe complications such as functional impairments and malignant transformation. The primary endpoints include tumor response rate, safety, tolerability, and pharmacokinetics of HLX-1502. The trial also aims to assess the overall potential of HLX-1502 as a long-term treatment option for NF1 patients.
NF1: An Urgent Need for Treatment
NF1 is a genetic disorder that causes individuals to develop multiple tumors throughout their bodies. In addition to causing pain and disfigurement, NF1 can lead to a range of complications such as cognitive impairments, muscle weakness, and cardiovascular issues. Plexiform neurofibromas, which affect nerve fibers, can lead to functional impairments and require immediate attention.
With an estimated 3 million people affected worldwide, the need for effective treatments for NF1 is urgent. Most current therapies only provide partial relief or come with debilitating side effects, underscoring the importance of new treatment options that address the root causes of the disease.
Healx’s Mission
Healx’s mission is to harness AI to accelerate the discovery of novel treatments for rare diseases. With over 10,000 rare diseases impacting millions of people globally, yet only 5% of these diseases having an approved treatment, Healx is determined to change the landscape of rare disease therapy development. By combining deep AI insights with expert drug development knowledge, Healx is fast-tracking the discovery of therapies that could change the lives of patients suffering from rare and under-treated conditions like NF1.
Founded in Cambridge, UK, by Tim Guilliams, Ph.D., and David Brown, Ph.D., Healx has raised approximately $125 million to fund its groundbreaking work in rare disease drug discovery. The company’s innovative platform is at the forefront of a new era of treatment development, one that could offer hope to the millions affected by rare diseases.
Healx’s work on HLX-1502 is just one example of its commitment to transforming the treatment of rare diseases. As the Phase 2 trial progresses, it holds the promise of not only improving the lives of NF1 patients but also laying the foundation for the future of rare disease therapies.
