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Healx receives IND and Orphan Drug Designation for fragile X clinical trial

Healx has received Investigational New Drug (IND) approval from the US Food and Drug Administration (FDA) for the Phase 2a clinical study of the compound HLX-0201 for Fragile X syndrome. The clinical study will attempt to find at least one novel and effective combination therapy for Fragile X syndrome - the world’s leading inherited cause of autism and learning difficulties which affects roughly 1 in 4000 males and 1 in 8000 females across the globe.
8 November 2021   |  
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Cambridge, UK – 20 October 2021Healx, the AI-powered, patient-inspired technology company pioneering the next generation of drug discovery, is proud to announce that they have received Investigational New Drug (IND) approval from the US Food and Drug Administration (FDA) for the Phase 2a clinical study of HLX-0201. This is one of several compounds that are being developed by Healx to treat fragile X syndrome in the first stage of a combination therapy trial. The IND approval follows the receipt of Orphan Drug Designations in the EU in 2020, and in the US earlier this year. Together, these approvals represent a significant milestone for the company: validating their AI-driven drug discovery approach and formally marking their evolution into a clinical stage company. The team will begin recruitment for participants in the trial in the coming months.

Fragile X syndrome is the most common inherited cause of autism and learning difficulties in the world – affecting roughly 1 in 4,000 males and 1 in 8,000 females – but there are currently no effective treatments or cures. Healx’s adaptive clinical study will attempt to address this and the team’s ultimate aim is to bring at least one novel and effective combination therapy for the condition to market in the coming years. As experts in the field of AI-powered drug discovery for rare diseases, Healx has pioneered several proprietary discovery methods that have uncovered multiple candidates for the condition and the team plan to use the clinical study to find the best combination of these compounds with the strongest mode of action.

HLX-0201 – originally approved as a nonsteroidal anti-inflammatory drug – is the first compound to be studied in the trial. HLX-0201 was identified as a potential treatment by Healx’s novel omic-based drug matching methods, which compare the gene expression profile for a disease with the gene expression profiles from Healx’s curated drug database to find entirely novel connections and disease pathways between the two. HLX-0201 was then validated in preclinical models of fragile X, where it was found to modify several behaviours similar to those that are exhibited in patients with the condition. Notably, the therapeutic effect observed was found to act in a newly identified secondary mode of action.

Several other compounds identified by Healx’s AI methods are also progressing towards the clinic, with the ultimate aim of finding a combination with synergistic mechanisms of action. Combination therapies – where two or more drugs are used simultaneously – are particularly poised to help fragile X, which exhibits a broad range of symptoms including mild to severe intellectual disabilities, autism, attention deficit and hyperactivity, anxiety, speech delays, hypersensitivity to loud noises or bright light and, in some cases, epilepsy. By targeting different disease processes and pathways, a combination of drugs has the potential to improve a greater range of symptoms experienced by a patient. Promisingly, preclinical studies in a model of fragile X syndrome have already demonstrated that some of the compounds identified by Healx can work as an effective combination at a lower dosage than is required as a monotherapy.

Throughout this project, Healx has worked closely with the FRAXA Research Foundation, a leading research and support organisation for fragile X in the United States. Dr Mike Tranfaglia, Medical Director and Chief Scientific Officer at FRAXA, commented: “We are excited about the potential of HLX-0201: a proven drug with a long track record of effectiveness. It never would have occurred to us to investigate the compound for fragile X without Healx’s innovative technology pointing us in the right direction, but the preclinical studies have shown remarkable effectiveness. We also believe that combinations of available drugs can treat many rare diseases, and we are glad Healx is leading the way in devising optimal combinations to treat fragile X”.

Dr Anthony Hall, Healx’s Chief Medical Officer, also commented: “Receiving this IND approval is a critical step towards delivering effective treatments to fragile X patients around the world, and is a significant milestone in the development of Healx’s clinical pipeline. It is also a strong validation of our AI-driven drug discovery methods. We believe that HLX-0201 has strong potential to address the huge unmet needs for fragile X patients as part of a combination, and we are looking forward to launching the first phase of the study as soon as possible.

With their first clinical programme soon to start, Healx hopes to continue the momentum and grow their portfolio to 50 programmes by 2025. Healx is open to collaborating with industry, patient and academic groups to progress more novel rare disease treatments towards patients, and interested parties can find out more about partnering with the team here.


About Healx

Healx is a mission-driven technology company pioneering the next wave of drug discovery in order to bring novel, effective treatments to rare disease patients around the world. There are 7,000 known rare diseases that affect 400 million people across the globe, but only 5% of those conditions have an approved treatment. By combining frontier AI technology with deep drug discovery and development expertise, Healx can accelerate the pace, increase the scale and improve the chance of success of rare disease treatment development in order to meet this huge unmet need and have unprecedented patient impact.

Founded in Cambridge, UK, in 2014 by Dr Tim Guilliams (a Biochemical Engineer and tech entrepreneur) and Dr David Brown (co-inventor of Viagra and former Global Head of Drug Discovery at Roche), Healx has raised around $70 million to date, added nearly 20 projects to its risk-balanced therapeutic portfolio, and built a cross-functional team of over 100 tech and drug discovery experts. For more information, visit www.healx.ai or follow on Twitter and LinkedIn.

For more information about Healx or this clinical trial, please contact:

Charlotte Chorley

Director of Strategic Alignment and Communications, Healx


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