A Drug Designed By AI Is Starting Its First Human Trials

Four years ago, biotech company Insilico Medicine used AI to design a molecule targeting a protein involved in fibrosis in just 46 days. It was a proof of concept, as multiple effective drugs already existed for the protein, giving the company a wealth of data to train their AI with. This week the company started Phase 2 clinical trials in humans for a drug discovered and designed by AI. It’s a first for the pharmaceutical industry, and hopefully heralds a future where drug discovery is faster, cheaper, and better than it’s been for the last several decades.

Drug discovery has historically been a tedious, slow, expensive process. Researchers first have to figure out what causes a given disease, usually identifying a protein as the culprit. Of these, a few move on to further research, and fewer still make it to human clinical trials.

Out of more than a million screened molecules, on average just one makes it to late stage clinical trials and ends up getting approved for use.

Its drug candidate for idiopathic pulmonary fibrosis-a chronic condition where the lungs become scarred and it’s hard to breathe-took just a third of the usual time and a tenth of the usual cost to develop thanks to the company’s technology.

Together, the networks generate new objects like text or images-or in this case, chemical structures of small molecules.

Insilico’s platform also uses reinforcement learning, a type of machine learning that enables a system to learn by trial and error using feedback from its own actions.

Reinforcement learning has been central to recent advances in game-playing AI. One of the drugs the company developed with its platform is called INS018 055.

Insilico is recruiting 60 patients with idiopathic pulmonary fibrosis in China and the US, who will take a 12-week dose of the drug.

Insilico has used its technology to discover 12 pre-clinical drug candidates.

Three of these have advanced to clinical trials, but INS018 055 is the first to make it to Phase 2 trials. This is just the beginning-for Insilico and AI drug discovery generally.

According to a report put together by Morgan Stanley, AI tools could help develop 50 new drugs worth potentially $50 billion over the next decade.

“For Insilico, is the moment of truth,” the company’s founder and CEO, Alex Zhavoronkov, told the Financial Times.