Healx
AI is transforming drugs for rare diseases
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. 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.
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.
This is largely due to the high costs and low return of rare disease drug treatments. Developing them runs into the billions of dollars yet there is a comparatively small number of people buying them. This means that the more than 400 million people worldwide who are affected by these rare diseases either have found that treatments don’t exist or their cost is inflated to eye-watering levels.
Yet it doesn’t have to be this way.
Tim Guilliams and David Brown founded the Cambridge startup, Healx, that is turning to the data-crunching power of artificial intelligence (AI) to rewrite the economics of drug discovery for the world’s rarest diseases.
Whilst at Pfizer, Brown who is now Chairman of Healx, was named co-inventor on the patent for Viagra, and for 8 years he led the team that invented and developed Viagra through to proof of clinical efficacy in male impotence. The drug is also marketed for treatment of pulmonary hypertension under the trade name Revatio. He also had a pivotal role in the discovery of Relpax, a treatment for migraine. Together these drugs have achieved sales of over $40 billion.
Tim Guilliams is now CEO and says “It’s a huge problem and if you try to address it in the traditional way, you know two to three billion dollars per drug, 10 to 15 years, it just doesn’t work, it’s impossible,” Guilliams tells Verdict. “I think we found a sweet spot where we can basically maximise the work that’s already done by applying machine learning and finding shortcuts to this very long drug discovery process.”
While the technology underpinning Healx may be complex, the premise is simple. The company uses a range of algorithms to search for links between existing diseases for which there are treatments, and rare diseases for which there are not.
This process begins with a “deep data curation” phase, which sees Healx’s AI program look for gaps in existing medical data. Once identified, the biotech startup works with patient groups and academics to help fill them.
“The quality of what goes into your algorithm really relates to the quality that comes out,” explains Guilliams.