INSILICO (03696): The company's leading AI-driven candidate innovative drug Rentosertib has completed the enrollment of its first patient in a Phase III clinical trial for idiopathic pulmonary fibrosis.

INSILICO (03696) announced that the leading AI-driven candidate innovative drug Rentosertib (also known as ISM001-055/INS018_055) has completed the enrollment and administration of the first patient in the Phase III clinical trial (GENESIS-IPF-3) at Peking Union Medical College Hospital; on the same day, Shanghai Pulmonary Hospital also completed its first patient enrollment in this trial. GENESIS-IPF-3 is a Phase III clinical trial (NCT07687459, CTR20262475) evaluating Rentosertib in patients with idiopathic pulmonary fibrosis (IPF). This 52-week, prospective, randomized, multi-center, double-blind, placebo-controlled, parallel-group study targeting IPF patients is expected to recruit a total of 320 subjects at 47 clinical centers in China. The primary endpoint is the annualized rate of decline in forced vital capacity (FVC) over 52 weeks; a key secondary endpoint is the time to the first occurrence of a disease progression event. This trial aims to assess whether Rentosertib can provide clinically meaningful benefits that cover a broader patient population and longer treatment duration compared to the Phase IIa study. The Phase III clinical trial is led by Professor Xu Zuojun from Peking Union Medical College Hospital, with renowned respiratory disease experts, Academician Zhong Nanshan from the Chinese Academy of Engineering, and Dean Chen Chang from Shanghai Pulmonary Hospital serving as co-principal investigators. Rentosertib is a potential "first-in-class" small molecule inhibitor targeting TNIK, powered by INSILICO's proprietary generative AI platform, Pharma.AI, for discovery and development. In IPF, the activation of TNIK drives pathological fibrosis in the lungs, leading to progressive declines in lung function. By inhibiting TNIK, Rentosertib aims to block or reverse the fibrosis process, providing a treatment option with disease improvement potential for IPF patients. In the GENESIS-IPF Phase IIa clinical trial, Rentosertib demonstrated good safety profiles and a dose-dependent trend towards efficacy, marking a milestone as the industry's first validated concept of AI-driven new drug development. The findings of this research were published in Nature Medicine in June 2025. IPF is a chronic, progressive pulmonary fibrosis disease characterized by a progressively irreversible decline in lung function. IPF affects approximately 5 million people globally and has a poor prognosis, with a median survival of only 3 to 4 years. Currently approved treatments include antifibrotic drugs that can slow disease progression but cannot stop or reverse the disease process, creating an urgent need for more effective treatment methods to improve the disease.