Healx – Accelerating the Discovery and Development of Rare Disease Treatments

Artificial Intelligence (AI) is an exciting, growing field. Due to the high and growing number of data, the comprehensive evaluation of information behind data makes AI tools indispensable. In Drug Discovery and Development the application of AI has become important to accelerate progress and enhance decision making in many fields and disciplines of medicinal chemistry, molecular and cell biology, pharmacology, pharmacokinetics, formulation development, and toxicology. In clinical testing AI has high importance in increasing success rates by enhancing trial design, selection of the target patient population, patient stratification and evaluation of patient samples. The increasing relevance of AI in drug discovery and development is reflected by the growing number of start-up companies specialized in this field, the growing number of collaborations from Pharma with AI platforms.

Healx is a mission-driven technology company pioneering the next generation 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.

Pioneering the Next Generation of Drug Discovery

It isn’t effective to start with a single target in mind. Rare diseases are often not well studied and there is a limited understanding of many of the aspects necessary to support a drug discovery programme. Healx's AI platform, Healnet, overcomes these challenges by analyzing millions of drug and disease data points to find novel connections that could be turned into new treatment opportunities. By applying frontier technologies across the discovery and development pipeline, they can run multiple stages in parallel and at scale. One disease, one target, one drug: it's an overly simple model, yet it's the one used by nearly all pharmaceutical companies. The next generation of drug discovery is AI-powered, parallel and hypothesis-free.

IMPACT-FXS Clinical Trial

Fragile X syndrome (FXS) is a rare neurodevelopmental condition and the most common genetic cause of learning difficulties in the world, but there are currently no effective or approved therapies for the condition available. Healx is sponsoring a Phase 2 clinical trial (clinicaltrials.gov identifier NCT04823052) to explore the safety and efficacy of several potential medications for fragile X syndrome. The IMPACT-FXS study (which stands for Investigating Multiple Pathways And Combined Treatments for FXS) is the world’s first umbrella trial for the investigation of therapies for FXS, and will evaluate several compounds first as monotherapies and then as combination therapies in order to identify the best medication. This first phase of this research study will test two investigational medications (HLX-0201 and HLX-0206) in 13- to 40- year-old boys and men with fragile X syndrome (FXS). The purpose of this study is to test the safety and tolerability of the medications, and to see if they reduce symptoms of FXS.

This research will examine candidate medications that could be new treatments for males with FXS to see if the medications are safe and have the intended effects. It is Healx’s hope that this study will lead to a better understanding of these study medications as potential treatments for FXS. The information gained in this study may be useful in treating other people diagnosed with FXS in the future.

The Visionary Leader Upfront

Dr. Tim Guilliams is a Co-founder and the Chief Executive Officer of Healx. He is a tech entrepreneur from the Cambridge Cluster, passionate about using big data and AI to accelerate treatments for rare diseases. In 2019, Healx raised $56 million in Series B funding and was listed in Tech Nation’s Future Fifty cohort. In 2020, the company won 5 prizes at the CogX Awards including Grand Jury Prize and Best AI Product and was listed in CB Insights Digital Health 150.

As CEO of Healx, Tim has been listed in The Sunday Times Maserati 100 list of entrepreneurs in 2019, was was named as ‘One to watch’ in The Telegraph’s Top 50 Most Ambitious Business Leaders in 2020, and was selected as member of the Forbes Technology Council in 2022.

Tim is also the Co-Founder and Trustee of the Cambridge Rare Disease Network (CRDN). Prior to Healx and CRDN, he obtained his PhD at the University of Cambridge in the field of Biophysics and Neuroscience, developing nanobody technology for Parkinson's disease. Before moving to Cambridge UK, Mr. Guilliams obtained a MEng in Bio and Chemical Engineering from the University of Brussels.

"The next-generation AI platform we built to find novel connections between drugs and diseases. We combine these predictions with in-house expertise to accelerate new treatments through our pipeline to clinical trials and on to patients."