Healx, a Cambridge-based AI-enabled biotech company, has initiated dosing in a Phase 2 clinical trial evaluating HLX-1502 for patients with neurofibromatosis type 1 (NF1), marking a significant milestone in rare disease treatment development.
Novel AI-Driven Approach to NF1 Treatment
The INSPIRE-NF1 trial represents a pioneering approach to treating NF1, a debilitating genetic disorder affecting approximately 1 in 2,500 people worldwide. The condition causes tumors to develop along nerve tissues, often beginning in childhood, leading to complications including pain, disfigurement, and potential malignancies.
"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. "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."
Trial Design and Objectives
The Phase 2, open-label, single-arm study will evaluate approximately 20 patients with plexiform neurofibromas (PNs) across U.S. centers. The trial's primary focus includes assessing tumor response rate, safety profiles, and pharmacokinetics of the oral investigational therapy.
HLX-1502, discovered using Healx's proprietary AI platform, has garnered significant regulatory support, receiving Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA. These designations underscore the urgent need for new treatment options in the NF1 community.
Expert Perspectives and Collaborative Efforts
Dr. Michael Fisher, director of the NF Program at Children's Hospital of Philadelphia and group chair of the NF1 Clinical Trials Consortium, expressed optimism about the trial: "Our consortium, consisting of 24 clinical centers across the United States and Australia, is committed to pushing NF research forward and finding new therapies for individuals with neurofibromatosis and schwannomatosis."
Tim Guilliams, Ph.D., co-founder and CEO of Healx, emphasized the broader implications: "This is much more than a company milestone—it represents a new hope for the NF1 community. If successful, this could mark the beginning of a paradigm shift in NF1 care."
Addressing an Urgent Unmet Need
NF1 affects approximately 3 million people worldwide, with limited treatment options available for various manifestations of the disease. The condition can cause multiple types of tumors, including plexiform neurofibromas that may result in severe disability and cutaneous neurofibromas leading to significant cosmetic and psychological distress.
The trial is being conducted in collaboration with the NF1 Clinical Trials Consortium, with operations centers at the University of Alabama at Birmingham and the Children's Hospital of Philadelphia. This collaborative approach, combined with innovative AI-driven drug discovery methods, represents a new frontier in developing treatments for rare genetic disorders.
