AI-generated drug begins clinical trials in human patients

The primary drug fully generated by artificial intelligence entered clinical trials with human patients this week.

Insilico Medicine, a Hong Kong-based biotech startup with greater than $400 million in funding, created the drug, INS018_055, as a treatment for idiopathic pulmonary fibrosis, a chronic disease that causes scarring within the lungs. The condition, which has increased in prevalence in recent a long time, currently affects about 100,000 people within the U.S. and might result in death inside two to 5 years if untreated, in response to the National Institutes of Health. 

“It’s the primary fully generative AI drug to succeed in human clinical trials, and specifically Phase II trials with patients,” Alex Zhavoronkov, founder and CEO of Insilico Medicine, told CNBC. “While there are other AI-designed drugs in trials, ours is the primary drug with each a novel AI-discovered goal and a novel AI-generated design.” 

The invention process for the brand new drug began in 2020, with hopes to create a “moonshot” medicine to beat challenges with current treatments for the condition, which mostly give attention to slowing progression and may cause uncomfortable unintended effects, Zhavoronkov said.

He added that Insilico has chosen to give attention to IPF partially due to condition’s implications in aging, but the corporate has two other drugs partially generated by AI within the clinical stage. One is a Covid-19 drug in phase one clinical trials, and the opposite is a cancer drug, specifically a “USP1 inhibitor for the treatment of solid tumors,” that recently received FDA approval to initiate clinical trials.

“When this company was launched, we were focused on algorithms — developing the technology that might discover and design latest molecules,” Zhavoronkov said. “I never imagined in those early days that I can be taking my very own AI drugs into clinical trials with patients. But we realized that with a view to validate our AI platform, we wanted to not only design a latest drug for a latest goal, but bring it into clinical trials to prove that our technology worked.” 

The IPF drug’s current study is a randomized, double-blind, placebo-controlled trial going down over 12 weeks in China, and Insilico has plans to expand the testing population to 60 subjects at 40 sites within the U.S. and China. If the present phase two study is successful, it would go on to a different study with a bigger cohort, after which potentially reach phase three studies with tons of of participants. 

“We expect to have results from the present Phase II trial next 12 months,” Zhavoronkov said, adding that it’s difficult to predict exact timing for future phases, especially for the reason that disease is comparatively rare and patients must fulfill specific criteria. He added, “We’re optimistic that this drug will likely be ready for market, and reach patients who may profit from it, in the subsequent few years.”