Rentosertib is Insilico Medicine’s most advanced candidate drug, with its novel target and molecular structure both discovered and designed through AI-driven processes. It holds first-in-class potential and is intended for the treatment of idiopathic pulmonary fibrosis. The upcoming Phase III clinical trial is a prospective, randomized, double-blind, placebo-controlled, parallel-group study, led by Professor Xu Zuojun from Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, as the Leading Principal Investigator (Leading PI), and co-led by Professor Zhong Nanshan, a renowned respiratory disease expert and academician of the Chinese Academy of Engineering, and Chen Chang, President of Shanghai Pulmonary Hospital, as Co-Leading PIs. The study is expected to enroll 320 patients with idiopathic pulmonary fibrosis (IPF) and aims to systematically evaluate the efficacy and safety of once-daily administration of Rentosertib over 52 weeks.
This study will use changes in forced vital capacity (FVC) as the primary endpoint, and will comprehensively incorporate multidimensional assessment indicators such as disease progression and patient quality of life. Notably, the study will also innovatively introduce validated AI algorithms to conduct quantitative analysis of factors such as patients’ fibrotic burden, aiming to further enhance the efficiency, stability, and precision of the study validation.
Dr. Ren Feng, Co-CEO and Chief Scientific Officer of Insilico Medicine, stated in his opening remarks: “We are deeply grateful for the long-standing trust and support from experts, research institutions, and industry partners. From nominating the candidate compound within 18 months to obtaining positive Phase IIa data, and now advancing to the preparatory stage of Phase III clinical research, we have always been confident in the unique advantages and clinical potential of Rentosertib. In the upcoming Phase III clinical study, we look forward to further validating the positive signals observed in the Phase IIa study, and through this pivotal study of the candidate drug, continuously testing the clinical application value of the AI-driven drug discovery pathway, witnessing together with all parties the tangible changes AI technology brings to patients.”
Professor Xu Zuojun, the Leading PI of this study from Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, said in his on-site speech: “Interstitial lung disease has always been a key focus in the field. From basic research to disease mechanisms and potential therapies, domestic research on this disease is continuously improving, and several encouraging clinical-stage results have been achieved. In the Phase IIa study of Rentosertib, we were pleased to observe a dose-dependent efficacy trend, and this positive result has garnered widespread attention and expectations from research institutions both domestically and internationally. For the larger-scale, longer-duration Phase III study, we look forward to strengthening collaboration among all parties in areas such as study standards, addressing potential challenges, and ensuring cross-center data consistency, to jointly promote the smooth progress of the study and form a more objective and comprehensive evaluation of Rentosertib, ultimately benefiting clinical patients.”
Chen Chang, President of Shanghai Pulmonary Hospital and Co-Leading PI of this study, said in his remote speech: “This will be a clinical study with industry benchmark significance. After seeing the potential of Rentosertib to control or even reverse the disease course of idiopathic pulmonary fibrosis (IPF), we are witnessing the transition of AI-driven drug discovery from concept to validation. By combining AI technology with Chinese innovation, we have the potential to achieve a leap from R&D to clinical application and ultimately to changing patients’ lives. As clinicians who understand patients’ struggles, participating in and driving this process holds great significance.”
Previously, Rentosertib completed the Phase IIa clinical trial GENESIS-IPF in September 2024, led by Professor Xu Zuojun as the principal investigator. The study results showed that the candidate drug had good safety and tolerability; among patients treated for 12 weeks, dose-dependent improvements were observed in multiple indicators, including forced vital capacity (FVC), showing a potential efficacy trend of reversing the disease course, and achieving the industry’s first clinical proof-of-concept for an AI drug.
Additionally, Insilico Medicine has established deep collaborative foundations with the research or medical institutions of the two newly joined Co-Leading PIs. In May 2025, research results on ISM3312, jointly advanced by Insilico Medicine and Guangzhou National Laboratory, were published in the globally authoritative academic journal Nature Communications, demonstrating broad-spectrum activity against multiple coronavirus infections and a low risk of drug resistance. In January 2026, Insilico Medicine and Shanghai Pulmonary Hospital announced the establishment of a joint laboratory and initiated the local deployment of Insilico Medicine’s self-developed hardware platform, PandaOmics Box, at Shanghai Pulmonary Hospital, to accelerate research on chest disease mechanisms and promote the translation of innovative achievements.
At the meeting, the Insilico Medicine team provided a detailed introduction to the AI-driven R&D journey of Rentosertib, shared previous preclinical and clinical validation results, and elaborated on the candidate drug’s characteristics and unique advantages, followed by a comprehensive overview of the clinical study plan. Based on core issues such as subject screening, risk prevention and control during the study, AI algorithm application, and data collection and analysis, the attending experts engaged in detailed discussions. Representatives from participating research institutions explained the subsequent study procedures and the application of related facilities from a practical perspective. All parties look forward to efficiently advancing this key study for large-scale validation of AI drugs through close and highly collaborative efforts.
About IPF
Idiopathic pulmonary fibrosis is a chronic, progressive fibrotic lung disease characterized by a progressive and irreversible decline in lung function. IPF affects approximately 5 million people worldwide, with a poor prognosis and a median survival of only 3 to 4 years. Current treatment options include antifibrotic drugs, which can slow disease progression but cannot stop or reverse the disease course, highlighting the urgent need for more effective disease-modifying therapies.
About Rentosertib
The early drug discovery and development of Rentosertib was completed in just 18 months, with fewer than 80 molecules synthesized and tested during the process. Supported by AI-enabled target identification and molecular design, its early discovery phase was significantly shortened compared to traditional drug R&D cycles. This AI-driven workflow was published in Nature Biotechnology in March 2024. Furthermore, Rentosertib achieved positive results in the GENESIS-IPF Phase IIa clinical trial, marking the world’s first proof-of-concept for efficacy in AI-driven drug discovery, with the relevant results published in Nature Medicine in June 2025.
About Insilico Medicine
Insilico Medicine is a global pioneer biotechnology company dedicated to integrating artificial intelligence and automation technologies to accelerate drug discovery and drive innovation in the life sciences, empowering humans to live longer and healthier lives. On December 30, 2025, the company was listed on the Main Board of the Hong Kong Stock Exchange under the stock code 03696.HK.
Using its self-developed Pharma.AI platform and advanced automated biology laboratory, Insilico Medicine is providing innovative drug solutions for unmet disease areas such as fibrosis, oncology, immunology, pain, obesity, and metabolic disorders. Additionally, Insilico Medicine continues to expand the application of Pharma.AI into diverse fields, including advanced materials, agriculture, nutritional products, and veterinary medicine. For more information, please visit the website www.insilico.com