As its latest trials move into next phase, founder and A HealthTech firm using AI to speed up the creation of treatments for rare diseases has moved into the next phase of its most recent clinical trials.

Cambridge-based Healx has created Healnet, an AI drug discovery platform which uses a suite of complex AI algorithms to quickly and accurately predict potential new drug-disease relationships from data.

The firm’s clinical trials are currently focused on treatments for ‘fragile x’, a genetic disorder characterised by mild-to-moderate intellectual disability.

These trials will be moved into the next phase – Phase 2a – this year, said founder and CEO Dr Tim Guilliams.

“Our mission remains the same: to take as many rare disease treatments towards the clinic as possible, ultimately providing hope to the 400 million people living with rare conditions worldwide,” Guilliams told BusinessCloud.

As the healthcare sector faces major strains on resources, the CEO says he welcomes any new technology which could successfully speed up and diversify treatment.

The biggest challenge facing rare disease patients is that of the more than 7,000 rare diseases discovered to date, only 5% have any form of approved treatment.

Traditional drug discovery methods have a timeline of between 12 and 14 years cost up to $3m, and have an estimated failure rate of 95%.

“The traditional approach to drug discovery, with its lengthy timelines, high associated costs and failure rates, means pharma companies tend to prioritise blockbuster drugs for larger disease populations. Unfortunately, this commercial model is not viable for rare disease patients,” he said.

“At Healx, we are using the latest in AI and machine learning (ML) technologies to disrupt the traditional process of drug discovery – making it faster, cheaper and more effective.

“Our AI-powered approach is able to cut the discovery-to-clinic timeline to as little as 24 months – along with the associated costs and failure rates.”

Since founding the firm in 2014 alongside Dr David Brown, co-creator of Viagra, it has now grown to a team of more than 50, including drug discoverers, AI engineers, and bioinformaticians, who combine IT and data with healthcare expertise.

The firm has also welcomed Dr Anthony Hall as Chief Medical Officer and Dr Bruce Bloom as Chief Collaboration Officer in the last six months.

“With a huge range of rare disease knowledge, drug discovery experience, and a wealth of industry relationships, both Tony and Bruce are leveraging their skills to help us progress treatments faster,” said Guilliams.

Last year the firm secured $56m in Series B funding.

Over the course of the next two years, the firm has committed $20m of this funding towards its Rare Treatment Accelerator programme, which allows it to work alongside patient groups to progress new rare disease treatments.

The funding will also see it invest up to $1m in AI and drug discovery resources per project.

“There will always be challenges associated with bringing effective treatments to market – from recruiting the right patients to funding clinical trials – however, by harnessing AI in this way we are at least able to fast-track those parts of the drug discovery process within our control,” he said.

“While technology is an integral part of our work, we are committed to keeping rare disease patients at the very heart of what we do – they are the real rare disease experts after all.”