Biotech begins human trials of drug designed by artificial intelligence

A biotech company backed by Chinese conglomerate Fosun Group and private equity giant Warburg Pincus has begun one of the first mid-stage human trials of a drug discovered and designed by artificial intelligence.

Insilico Medicine, which was founded by Latvian-born scientist Alex Zhavoronkov, said it had dosed a patient in China with a novel therapy to treat the chronic lung disease idiopathic pulmonary fibrosis.

The company said the drug, INS018_055, was the first entirely “AI-discovered and AI designed” drug to begin a phase 2 clinical trial and represented an important milestone for the industry.

“For Insilico, it is the moment of truth . . . but it is also a true test for AI and the entire industry should be watching,” said Zhavoronkov in an interview.

“Our company, and it’s a big, bold claim, can double the productivity of pretty much every big pharma company”.

Insilico is one of a new generation of biotechs, which have collectively raised billions of dollars to develop AI tools aimed at revolutionising drug development. It is part of a race by Big Pharma and investors to capitalise on a $50bn market opportunity for AI in the sector, according to a report by Morgan Stanley.

Zhavoronkov said Insilico’s AI platforms could potentially halve the time it took to discover drugs and slash the cost of bringing medicines to market — estimated by Deloitte at $2.3bn on average per therapy. Sanofi, Fosun and Johnson & Johnson were among several pharma companies that had signed partnership deals that provided access to Insilico’s technology, he said.   

AI platforms can crunch vast amounts of data to rapidly identify drug targets — proteins in the body associated with particular diseases — and molecules that can be made into medicines.

Several biotechs have recently announced drugs discovered or developed using AI and machine learning tools that have progressed to clinical trials, including Exscientia, Verge Genomics and Recursion Pharmaceuticals. Insilico uses generative AI to rapidly select novel drug targets and then design new molecules that can target a particular disease.

Zhavoronkov said Insilico’s AI could save two to four years in pre-clinical discovery depending on the novelty and complexity of the target. It did not save a lot of time in clinical development but improved the probability of success of a drug because of better chemistry and target choice. Insilico also used AI to recruit patients who were more likely to respond to the therapy, he said.

There are no guarantees AI-discovered drugs or the platforms that create them will be successful, and some critics warn the technology’s potential is overhyped. Last month Benevolent AI, a London-based biotech with an AI drug discovery platform, said it would lay off 180 staff, almost half its workforce, following the failure of its lead drug candidate.

Eric Topol, founder and director of the Scripps Research Translational Institute, said AI has already shown a lot of promise in reducing the time and cost of drug development, even though regulators had not yet approved a drug entirely developed using the technology.

“There is no shortage of interest. Every major pharma company has invested in partnerships with at least one, if not multiple, AI companies,” said Topol, author of Deep Medicine, a book exploring the potential of AI in healthcare.   

Insilico, which is dual headquartered in Hong Kong and New York, has raised more than $400mn from a range of investors mainly based in Asia and the US. It has used AI platforms to select 12 pre-clinical drug candidates, of which three have advanced to clinical trials.

Zhavoronkov said the company decided to take its IPF therapy into the clinic itself, rather than partner with a large pharma company, to retain control of its flagship programme and refine its own AI platforms.

“It’s extremely important to be able to generate huge amounts of data to ensure that your AI is reinforced but also to ensure that it’s constantly learning,” he said. “We are paving the way for new types of AI that can go end to end . . . So we span the entire pharmaceutical R&D lifecycle.”

Insilico conducted phase 1 trials on INS018_055 in New Zealand and China, which it said demonstrated favourable results that supported a phase 2 trial. This mid-stage trial will recruit 60 people with IPF in China and the US to assess the safety, tolerability and preliminary efficacy of the drug.