I have always been fascinated with how new technologies such as artificial intelligence (AI) and machine learning could be used to accelerate the discovery and development of disease treatments.
However, it wasn’t until Healx co-founder Dr David Brown (co-inventor of Viagra) and I met with rare disease parent, Nick Sireau, who was tirelessly trying to repurpose a drug called nitisinone as a treatment for his children’s condition, that we realised just how great the therapeutic unmet need was for rare disease patients – and how our AI drug discovery technology might be able to help.
The challenge of rare disease drug discovery
There are over 7,000 rare diseases that affect 400 million people worldwide. Of these diseases, only 5% of them have an approved treatment. This lack of approved treatments is due, in part, to limitations of the traditional model of drug discovery whereby the expense ($2-3 billion), timelines (12-14 years) and failure rates (95%) associated with bringing a new drug to market mean pharma companies often struggle to justify investment in treatments for smaller populations.
And that’s where Healx comes in.
AI-powered and patient-inspired treatments
At Healx, we use AI to accelerate the discovery and development of repurposed rare disease treatments. By combining our AI technology with patient insights and drug discovery expertise, we have been able to predict new treatments which are around 80% faster and 90% cheaper than the typical drug discovery process currently allows.
But whilst technology is integral to our work, we recognise that without the support of patients themselves, we could not have achieved as much as we have done so far – they are the real rare disease experts after all.
Putting it into practice: the FRAXA case study
The effectiveness of this AI-powered and patient-inspired approach to drug discovery can already be seen through our success in progressing a number of new treatments for fragile X syndrome – the most common genetic cause of autism and learning disabilities.
Working in close collaboration with the US-based patient group, FRAXA Research Foundation, we were able to take a number of novel therapies from drug prediction stage to the point of being clinically ready for testing – within the space of just 24 months.
The success of this partnership inspired us to scale up and replicate this model – across multiple disease areas and in collaboration with multiple patient groups worldwide – through the recent launch of our Rare Treatment Accelerator. This partnering programme gives patient groups and Healx the opportunity to work together, to quickly discover and develop repurposed treatments for rare diseases using AI, moving us yet another step closer to achieving our mission to take 100 rare disease treatments towards the clinic by 2025.
Working together with selected patient groups, we will invest up to the value of $1 million in AI and drug discovery resources per partnership. To give you an idea of our commitment to these partnerships, we have pledged a total of $20 million – from the $56 million raised in our recent Series B finance round – over the next two years towards finding new treatments.
The future of AI in healthcare
Over the course of the last decade, it’s been fascinating to watch how AI has been steadily transforming the healthcare industry. From its use in improving the accuracy of breast cancer screening to helping reduce the number of uncancelled missed appointments, we’re seeing ever more innovative examples of AI being used to enhance health and patient care.
As for what 2020 holds in store for Healx, this year will see the start of clinical trials for our AI-derived fragile X treatments and we will continue our pioneering work developing the most advanced AI to predict new treatments for rare disease patients. Looking further into the future, we can see our approach to treating small populations of patients evolving to offer precision medicine solutions. After all, every single one of us is a rare combination of lifestyle choices and inherited biology.