On August 1, 2024 Healx, an AI-enabled, clinical-stage biotech company specializing in rare diseases, reported it has raised $47 million in a Series C round (Press release, Healx, AUG 1, 2024, View Source [SID1234645286]). The Series C round was co-led by Silicon Valley-based R42 Group and Atomico, one of Europe’s largest venture capital firms, with participation from new and existing investors including Balderton, Jonathan Milner, Global Brain, btov, Ayana Capital, o2h and VU Venture Partners. Proceeds of the financing will be used to advance the company’s pipeline of medicines in rare oncology, renal and neurodevelopmental disorders, including advancing its lead program HLX-1502 through a Phase 2 clinical trial for the treatment of neurofibromatosis Type 1 (NF1). In conjunction with the financing, Stanford Medicine Adjunct Professor Ronjon Nag, Ph.D., founder of R42 Group and 2024 Silicon Valley Hall of Fame AI inductee, joins the board of Healx.
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"It is truly a pivotal time at Healx," said Tim Guilliams, Ph.D., co-founder and CEO of Healx. "By December 2024, we will advance HLX-1502 into a Phase 2 clinical trial in patients with NF1 both for plexiform neurofibroma, a genetic condition with limited treatment options and for cutaneous neurofibroma, for which there are no FDA approved options. We are also generating important preclinical data for multiple additional compounds identified using our novel generative AI drug discovery engine. Those compounds target rare diseases with significant unmet needs, including additional nerve-related tumor disorders, autosomal dominant polycystic kidney disease and neurodevelopmental disorders such as Angelman syndrome.
We are also generating important preclinical data for multiple additional compounds identified using our novel generative AI drug discovery engine. Those compounds target rare diseases with significant unmet needs, including additional nerve-related tumor disorders, autosomal dominant polycystic kidney disease and neurodevelopmental disorders such as Angelman syndrome.
The Healx drug discovery pipeline is powered by Healnet, an AI-driven discovery platform designed to identify clinically de-risked therapeutic opportunities for rare diseases. Healnet incorporates recent advances in generative AI to find connections between biological and chemical entities that could be turned into new treatments.
Healx was co-founded in Cambridge (UK) by its chairman and Viagra co-inventor David Brown, Ph.D., and Tim Guilliams, Ph.D., with a mission to apply emerging technologies to speed the discovery of rare disease treatments.
U.S. FDA IND Clearance
Healx also announced that it has received clearance from the U.S. Food and Drug Administration (FDA) to proceed with its Phase 2 clinical trial of HLX-1502. This trial will focus on treating adults with NF1 and inoperable plexiform neurofibroma.
"This IND approval marks another significant milestone in our efforts to harness powerful AI to develop a new treatment for NF1-associated plexiform neurofibroma," said Tim Guilliams, Ph.D., CEO of Healx. "We are committed to advancing this promising investigational treatment through clinical development and bringing it one step closer to patients in need."
HLX-1502 is a tablet taken orally that works differently than other treatments and offers a new and differentiated investigational treatment option for patients with NF1.
NF1 is a rare genetic disorder associated with predisposition to develop multiple benign and malignant tumors. It affects approximately 1 in 2,500 individuals. Two notable types of tumors associated with NF1 are plexiform neurofibromas and cutaneous neurofibromas. Plexiform neurofibromas are complex tumors growing aggressively along nerves, which often leads to significant morbidity and risk of malignant transformation. These tumors can affect various parts of the body, causing functional impairments, disfigurement and pain and requiring multidisciplinary management. Currently, there is only one treatment option available for some children with plexiform neurofibromas which is, however, associated with tolerability and safety concerns including gastrointestinal, heart, eye and skin toxicity.
Cutaneous neurofibromas are benign tumors that often cause significant symptoms, leading to impairment in quality of life, and also result in considerable cosmetic concerns. There are no approved treatments for NF1-associated cutaneous neurofibromas, leaving a major unmet need for the estimated 3 million people with NF1 worldwide.
HLX-1502 has received Orphan Drug and Rare Pediatric Disease designations from the FDA for treating NF1. These FDA designations provide several benefits to encourage the development of treatments for rare diseases and further highlight HLX-1502’s potential to significantly improve the lives of NF1 patients.
"I could not be more excited to start this trial soon. Our treatment – with its novel mechanism of action and potential for a compelling and differentiated safety profile – represents a significant advancement in the NF1 field and a much needed hope for the NF1 community. It has the potential to greatly improve NF1 patients’ lives, which is by far what matters the most and what drives us," said Simone Manso, Healx head of neurofibromatosis therapy development.
Healx has an investment agreement with its long-term research partner, Children’s Tumor Foundation (CTF), whereby Healx will receive milestone-driven payments from CTF to support the advance of Healx’s NF programs including its lead candidate, HLX-1502. CTF has been a longtime partner of Healx and actively supports its mission to deliver much-needed therapies to this patient group.
About Healnet
Behind advances in our therapeutic pipeline lies Healnet, a unique AI-driven drug discovery engine that is tailored to discover treatments for rare diseases. The platform uses disease multi-omics signature reversal and AI to identify novel therapeutic hits. Using Healx lab-generated phenotypic and transcriptomics profiling data, novel disease biology, mechanisms and targets are identified, which enables the design of second-generation compounds using virtual screening, predictive modeling and phenomics.
Healnet is now incorporating modern advances in generative AI, increasing the quantity and quality of its data sources, creating intelligent AI workflows and enabling natural language reasoning over its biological and chemical predictions. To better design optimized novel compounds for our programs, Healx will develop chemically-aware large language models (LLMs). Moving beyond general text-based large language models like ChatGPT from OpenAI, Healx is exploring how models trained on raw biological data that speak the language of the cell can identify and exploit targets and result in greater understanding of rare disease biology. These technological capabilities empower Healx’s scientists to operate with enhanced expertise, efficiency and accuracy in order to uncover previously undiscoverable treatments.