A Fully AI-Generated Drug Is Being Tested On Humans For The First Time Ever

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A Hong Kong-based biotech startup has brought its fully developed by artificial intelligence drug into clinical trials with human patients. The company states that its drug, INS0_055, demonstrates the power of its AI platform in drug discovery and development.

Insilico Medicine is a biotech startup focused on developing new drugs with the aid of AI. It states on its website that there are few "truly novel drugs on the market," and that the number of those is dropping. Insilico points to a number of obstacles that are keeping new drug discoveries from advancing, with one being the complexity of bringing one to market. The startup says that by using AI, there is a major opportunity to bring "new speed and efficiency to the process." It has utilized all of this AI-infused technology to get its novel treatment for idiopathic pulmonary fibrosis into clinical trials with human patients.

"It's the first fully generative AI drug to reach human clinical trials, and specifically Phase II trials with patients," noted Alex Zhavoronkov, founder and CEO of Insilico Medicine, in an interview with CNBC. "While there are other AI-designed drugs in trials, ours is the first drug with both a novel AI-discovered target and a novel AI-generated design."

Insilico began its process of bringing its fully generated by AI drug in 2020. The company hoped that it could create a "moonshot" medicine that could overcome the problems of other treatments that mainly focus on slowing down idiopathic pulmonary fibrosis, along with the fact that other drugs also have very uncomfortable side effects, according to Zhavoronkov.

The CEO added that the company focused on idiopathic pulmonary fibrosis treatment due to it having great implications for the process of aging.

Zhavornkov said that when the company launched, it was mainly focused on algorithms. He continued by remarking, "I never imagined in those early days that I would be taking my own AI drugs into clinical trials with patients. But we realized that in order to validate our AI platform, we needed to not only design a new drug for a new target, but bring it into clinical trials to prove that our technology worked."

Insilico is also working on two other drugs, one for COVID-19 and the other being a cancer drug. The cancer treatment is a "USP1 inhibitor for the treatment of solid tumors," according to Zhavoronkov.

As of right now, the drug treatment for idiopathic pulmonary fibrosis is a randomized, double-blind, placebo-controlled trial taking place over a 12-week period. The company plans to expand the trial to 60 patients at 40 different sites, including sites in the US and China. If successful, it will proceed on to another study, and potentially reach phase three where hundreds of participants could join.

Insilico expects to have the results of Phase II by next year. Zhavoronkov added, "We are optimistic that this drug will be ready for market, and reach patients who may benefit from it, in the next few years."