First fully AI-generated drug enters clinical trials

The first drug fully discovered and designed by artificial intelligence (AI) has entered clinical trials with human patients this week. 

Insilico Medicine, a biotech startup that has received over $400 million in funding, announced that they have successfully administered the first dose of their drug, INS018_055, to patients in the Phase II clinical trial. 

The drug is an anti-fibrotic small molecule inhibitor used in the treatment of idiopathic pulmonary fibrosis (IPF), a condition marked by scarring in the lungs and difficulty breathing. While there are other drugs on the market for this condition, INS018_055 is first-in-class, meaning the mechanisms through which it treats IPF differs from what’s currently on the market. 

“With demonstrated potential against both fibrosis and inflammation, INS018_055 could offer another option for patients worldwide,” said Feng Ren, PhD, co-CEO and Chief Scientific Officer of Insilico Medicine in the announcment. “The achievement of the first dose for INS018_055 in the Phase II clinical trial is not only an important step for Insilico, but also a milestone for AI-driven drug discovery and development. Together, we are expecting more achievements powered by AI for global unmet medical needs.”

Pioneers of drug discovery through generative AI

“When this company was launched, we were focused on algorithms — developing the technology that could discover and design new molecules,” Zhavoronkov explained. “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 has developed and validated various methodologies and functionalities for its generative adversarial network (GAN)-based AI platform. These advancements have been successfully integrated into the commercially accessible Pharma.AI platform, a “drug discovery engine” that can be used to “discover signatures of diseases and identify the most promising targets for billions of molecules that already exist or can be generated.”

Currently, the company has successfully nominated 12 preclinical candidates for internal drug development programs since 2021. Notably, three of these candidates have progressed into human clinical trials, demonstrating the platform’s potential in accelerating the journey from discovery to clinical evaluation.

“When we started developing generative AI for drug discovery, I never expected to see the clinical and preclinical results we have today,” said Alex Zhavoronkov, PhD, Founder and co-CEO of Insilico Medicine said in the announcement. 

“Initiating Phase II trials with this novel inhibitor for IPF represents a major milestone for deep generative reinforcement learning in drug discovery. We will explore the efficacy for patients of AI-discovered and designed treatments in clinical trials, which is a true validation of our generative AI platform. We are eager to continue to advance this potentially first-in-class therapy forward to help patients in need and show the value of generative AI in drug discovery and development.”