Healx, an AI-enabled, clinical-stage biotech company dedicated to rare diseases, today announced the first patient has been dosed in INSPIRE-NF1, a Phase 2 trial evaluating the safety and efficacy of HLX-1502, an oral investigational therapy, in patients with neurofibromatosis type 1 (NF1).

NF1 is a debilitating rare genetic disorder that causes tumours to form along nerves, often manifesting in childhood. It affects approximately one in 2,500 individuals worldwide and can lead to severe complications, including pain, disfigurement, and malignancies. Despite its impact, treatment options remain limited, with many patients facing invasive surgeries or therapies with severe side effects.

A Potential Breakthrough for NF1 Patients

“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.”

HLX-1502 was discovered using Healx’s proprietary AI platform, which accelerates the identification of potential drug candidates. The therapy has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration, underscoring its potential significance for the NF1 community.

Driving a New Era in NF1 Treatment Innovation

“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.”

Partnering for Patient Impact

“We are excited about the launch of this trial and what it could mean for those affected by NF1 worldwide,” said 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. “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. Healx’s innovative approach to treatment discovery gives us renewed hope for continued meaningful progress.”

The NF1 Clinical Trials Consortium has an Operations Center based at the University of Alabama at Birmingham (UAB), led by Girish Dhall, M.D., and Karen Cole-Plourde, and a Data Coordinating Center at CHOP led by Richard Aplenc, M.D., Ph.D.

About the INSPIRE-NF1 Study

INSPIRE-NF1 is a Phase 2, open-label, single-arm study evaluating the safety and efficacy of HLX-1502 in NF1 patients with plexiform neurofibromas (PNs). These aggressive tumours can lead to severe complications, including functional impairments and malignant transformation. The trial is enrolling approximately 20 patients in the U.S., focusing on key outcome measures such as tumour response rate, safety and tolerability, and pharmacokinetics. Additional information is available at clinicaltrials.gov (NCT06541847).

About Neurofibromatosis Type 1 (NF1)

NF1 is a rare genetic disorder that predisposes individuals to develop multiple tumours, affecting roughly 3 million people worldwide. Plexiform neurofibromas can cause severe disability, while cutaneous neurofibromas—benign but often numerous tumours—result in significant cosmetic and psychological distress. Treatment options are extremely limited, highlighting an urgent unmet need.

About Healx

Healx is a generative AI-enabled, clinical-stage biotech pioneering the next generation of drug discovery for rare diseases. With over 10,000 rare diseases affecting 400 million people worldwide, yet only 5% having an approved treatment, Healx is on a mission to change that. By combining AI-driven insights with deep drug development expertise, Healx accelerates the discovery and development of life-changing therapies.

Founded in Cambridge, UK, by biochemical engineer and entrepreneur Tim Guilliams, Ph.D., and David Brown, Ph.D., co-inventor of Viagra and former Global Head of Drug Discovery at Roche, Healx has raised approximately $125 million to date. For more information, visit www.healx.ai or follow us on LinkedIn and X.

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