AI-generated drug begins clinical trials in human patients

Alex Zhavoronkov (left), Founder and CEO of Insilico Medicine, and Feng Ren, Co-CEO and Chief Scientific Officer, at the company’s robotics lab in Suzhou, China.

Source: Insilico Medicine

The first drug created entirely by artificial intelligence entered clinical trials on human patients this week.

Insilico Medicine, a Hong Kong-based biotech startup with more than $400 million in funding, has developed drug INS018_055 to treat idiopathic pulmonary fibrosis, a chronic condition that causes scarring in the lungs. The condition, whose prevalence has increased in recent decades, currently affects about 100,000 people in the United States and can be fatal within two to five years if left untreated, according to the National Institutes of Health.

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

The discovery process for the new drug began in 2020 with hopes of developing a “moonshot” drug to overcome the challenges of current treatments for the condition, which mainly focus on slowing its progression and can cause uncomfortable side effects, he said Zhavoronkov.

He added that while Insilico chose to focus on IPF in part because of the disease’s impact on aging, the company has two other drugs that are made in part by AI in clinical stages. One is a Covid-19 drug in Phase 1 clinical trials, the other is a cancer drug, specifically a “USP1 inhibitor for the treatment of solid tumors” that recently received FDA approval to start with received clinical studies.

“When this company was founded, we focused on algorithms – developing the technology that can be used to discover and design new molecules,” Zhavoronkov said. “I never thought at the time that I would bring my own AI drugs to clinical trials with patients. But we realized that to validate our AI platform, we not only needed to develop a new drug for a new target, but also take it into clinical trials to prove our technology works.

The current study on the IPF drug is a randomized, double-blind, placebo-controlled study taking place over a period of 12 weeks in China. Insilico plans to expand the testing population to 60 subjects at 40 sites in the US and China. If the current phase 2 study is successful, it will proceed to another study with a larger cohort and then potentially reach phase 3 studies with hundreds of participants.

“We expect to receive results from the current phase II study within the next year,” Zhavoronkov said, adding that it is difficult to predict the exact timing of future phases, especially given that the disease is relatively rare and the patients are have to meet certain criteria. He added, “We are optimistic that within the next few years this drug will be ready to market and reach patients who could benefit.”