Clinical-stage biotechnology company Insilico Medicine (HKEX:3696) announced on Tuesday the initiation of the Phase III clinical trial for Rentosertib, its potentially first-in-class oral small-molecule inhibitor targeting TNIK for the treatment of idiopathic pulmonary fibrosis (IPF), a progressive, age-related fibrotic lung disease with high unmet medical need.

Rentosertib, formerly known as ISM001-055 / INS018_055, was discovered and designed through Insilico's Pharma.AI platform. The company says that the program combines a novel fibrosis target prioritised by Biology42: PandaOmics, Insilico's AI-powered biology engine, with a novel small molecule generated and optimised through Chemistry42, Insilico's generative chemistry platform. Insilico leverages PandaOmics for indication prioritisation and Medicine42's inClinico platform to predict and improve the program's clinical trial outcomes. The program's discovery-to-clinic path was published in Nature Biotechnology, while randomised Phase IIa clinical results were published in Nature Medicine and presented at the American Thoracic Society (ATS) 2025 International Conference.

According to Insilico, the launch of Rentosertib's Phase III clinical trial marks a major late-stage milestone for AI-driven drug discovery: a medicine whose target was identified with AI, whose chemical structure was designed with generative AI, and whose clinical development is aimed at a severe age-related disease in which current approved antifibrotic therapies can slow progression but do not reverse the degenerative course of disease.

To evaluate Rentosertib in this next stage of development, the upcoming Phase III clinical trial is a prospective, randomised, double-blind, placebo-controlled, parallel-group Phase III study in China. The study is expected to enrol 320 patients with IPF, and is designed to systematically evaluate the efficacy and safety of once-daily Rentosertib administered over 52 weeks.