Potentially first-in-class oral TNIK inhibitor advances into late-stage development after milestones marked by vital peer-reviewed papers: the discovery-to-clinic journey in Nature Biotechnology, and positive Phase IIa clinical results in Nature Medicine
Rentosertib represents a novel mechanism, novel AI-identified target, and novel AI-empowered molecule emerging from Insilico's aging-biology-driven Pharma.AI platform
The GENESIS-IPF Phase IIa study showed manageable safety and tolerability, with the 60 mg once-daily arm demonstrating mean forced vital capacity improvement of +98.4 mL at 12 weeks
CAMBRIDGE, Mass., July 7, 2026 /PRNewswire/ -- Insilico Medicine ("Insilico," HKEX: 3696), a clinical-stage biotechnology company powered by generative AI, today announced 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 program combines a novel fibrosis target prioritized by Biology42: PandaOmics, Insilico's AI-powered biology engine, with a novel small molecule generated and optimized through Chemistry42, Insilico's generative chemistry platform. Insilico leverages PandaOmics for indication prioritization 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 randomized Phase IIa clinical results were published in Nature Medicine and presented at the American Thoracic Society (ATS) 2025 International Conference.
The initiation 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, randomized, double-blind, placebo-controlled, parallel-group Phase III study. It will be led by Professor Zuojun Xu of Peking Union Medical College Hospital, Chinese Academy of Medical Sciences as the Leading Principal Investigator (Leading PI), with Academician Nanshan Zhong of the Chinese Academy of Engineering, a renowned expert in respiratory medicine, and President Chang Chen of Shanghai Pulmonary Hospital serving as Co-Leading Principal Investigators (Co-Leading PIs). The study is expected to enroll 320 patients with idiopathic pulmonary fibrosis (IPF) and is designed to systematically evaluate the efficacy and safety of once-daily Rentosertib administered over 52 weeks.