Healx Secures $47M Series C, FDA Approves Phase 2 IND for Neurofibromatosis 1 Trial

Healx, a biotech company based in Cambridge, UK, that leverages AI to develop treatments for rare diseases, has successfully raised $47 million in a Series C funding round. The financing was co-led by R42 Group from Silicon Valley and Atomico, a major European venture capital firm. Additional participants included both new and existing investors such as Balderton, Jonathan Milner, Global Brain, btov, Ayana Capital, o2h, and VU Venture Partners. This funding will be used primarily to advance Healx's portfolio of treatments for rare conditions, including their lead compound, HLX-1502, which is set to enter a Phase 2 clinical trial for neurofibromatosis Type 1 (NF1).

Ronjon Nag, Ph.D., founder of R42 Group and an adjunct professor at Stanford Medicine, will join Healx's board as part of the financing agreement. Healx CEO and co-founder Tim Guilliams, Ph.D., expressed excitement about the coming advancements, noting that HLX-1502 will soon progress to a Phase 2 clinical trial targeting both plexiform and cutaneous neurofibromas in NF1 patients. The company is also working on preclinical data for several other compounds identified through their proprietary AI drug discovery engine, which are aimed at rare diseases with significant unmet medical needs.

Healx's AI-driven platform, Healnet, plays a crucial role in their drug discovery efforts. The platform uses advanced generative AI to uncover potential therapeutic connections between biological and chemical entities, thus accelerating the identification of new treatments for rare diseases. 

Founded by Tim Guilliams, Ph.D., and David Brown, Ph.D., in Cambridge, UK, Healx has been committed to using technology to expedite the discovery of treatments for rare diseases. Brown, one of the co-inventors of Viagra, brings extensive experience in drug discovery to the company.

The U.S. Food and Drug Administration (FDA) has cleared Healx to proceed with a Phase 2 clinical trial of HLX-1502, which aims to treat adults with NF1 and inoperable plexiform neurofibromas. This approval marks a significant milestone for Healx in their use of AI to develop new treatments for complex conditions. NF1 is a genetic disorder that affects approximately 1 in 2,500 individuals and is characterized by the development of multiple benign and malignant tumors. Current treatments for some children with plexiform neurofibromas come with significant safety concerns, including gastrointestinal, heart, eye, and skin toxicity, highlighting the need for new therapeutic options.

HLX-1502, which has been granted Orphan Drug and Rare Pediatric Disease designations by the FDA, offers a new potential treatment for patients with NF1. These designations provide benefits aimed at encouraging the development of treatments for rare diseases and underscore the potential impact of HLX-1502 on NF1 patients.

Simone Manso, the head of neurofibromatosis therapy development at Healx, expressed enthusiasm about the upcoming clinical trial, emphasizing that HLX-1502's novel mechanism of action and favorable safety profile could offer significant advancements for NF1 patients. 

Healx has a strategic partnership with the Children’s Tumor Foundation (CTF), which will provide milestone-driven payments to support the progress of Healx's NF programs, including HLX-1502. This partnership reflects a shared commitment to developing much-needed therapies for NF1 patients.

The backbone of Healx’s drug discovery is Healnet, a sophisticated AI-driven platform that integrates disease multi-omics signature reversal and AI to discover new treatments. The platform leverages phenotypic and transcriptomics profiling data to identify novel disease biology, mechanisms, and targets. It utilizes modern advancements in generative AI to enhance its data sources, develop intelligent AI workflows, and enable natural language reasoning over biological and chemical predictions. These capabilities empower Healx’s scientists to create optimized compounds and uncover previously undiscovered treatments.

Founded to address the significant gap in treatment options for rare diseases, Healx combines generative AI with drug discovery expertise to accelerate the development of effective treatments. With 10,000 known rare diseases affecting 400 million people globally, the company's mission is to ensure every rare disease patient has access to effective treatment options.

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