Insilico found that its new small-molecule drug candidate, rentosertib, designed with its AI platform Chemistry42, was “generally safe and potentially effective” for treating idiopathic pulmonary fibrosis (IPF), according to its phase 2A clinical trial results published in the journal Nature Medicine on Tuesday.
IPF is a chronic disease that causes lung tissue to thicken and stiffen, leading to breathing difficulties. During a 12-week study of rentosertib, which enrolled 71 patients in China, Insilico observed an improvement in lung function for those who received a higher dose, according to the paper.
While adverse reactions related to liver toxicity occurred in some patients who were also taking another IPF drug, rentosertib met its safety objectives for the trial, Insilico said.
The study showed the “transformative potential of AI in drug discovery and development”, paving the way for faster and more innovative therapeutic advancements, Insilico founder and CEO Alex Zhavoronkov said in a statement.

Insilico was founded in 2014 in Baltimore, Maryland, as part of the Emerging Technology Centre at Johns Hopkins University. The company announced last year that it had moved its global headquarters to Boston. It has a significant presence in mainland China, with a lab in Suzhou, and Hong Kong, where it has an office at Science Park.