Insilico Medicine Doses First Patient in GENESIS-IPF-3, the World's First Phase III Trial of a Generative AI-Driven Innovative Drug

CAMBRIDGE, Mass., Sept. 9, 2026 /PRNewswire/ -- Insilico Medicine ("Insilico," HKEX: 3696), a clinical-stage biotechnology company powered by generative AI, today announced that it has dosed the first patient with Rentosertib (known as ISM001-055 / INS018_055) in GENESIS-IPF-3, a Phase III clinical trial (NCT07687459, CTR20262475) at Peking Union Medical College Hospital, while Shanghai Pulmonary Hospital has also enrolled its first patient on the same day.

The Phase III clinical trial is a prospective, multi-center, randomized, double-blind, placebo-controlled, parallel-group study designed to systematically evaluate the efficacy and safety of once-daily Rentosertib administered over 52 weeks. The study is 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).

As Leading PI of the study, Professor Zuojun Xu from Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, commented: "TNIK, the target driven by AI, had never previously been linked to fibrosis. This perhaps indicates that AI is carving out a path distinct from traditional research paradigms in target discovery for complex diseases. Dosing the first patient marks another key clinical milestone for Rentosertib; after preliminary findings in the 12 week duration phase IIa study showed promising results, including improvements in lung function among patients with idiopathic pulmonary fibrosis (IPF). This Phase III study is designed to validate the phase IIa study findings in a larger cohort of idiopathic pulmonary fibrosis patients treated for a longer duration of 52 weeks. From initiating Phase III to obtaining final regulatory approval, it will take three to four years under favorable conditions. We look forward to Rentosertib achieving its primary endpoints and successfully securing market approval to benefit IPF patients."

Till today, Insilico has published multiple peer-reviewed papers to document the milestones of Rentosertib along the R&D journey, all the way from target identification to Phase IIa positive results. In 2024, a Nature Biotechnology publication demonstrated the AI-driven early discovery process of Rentosertib. In 2025, a Nature Medicine study reported Phase IIa results of Rentosertib, demonstrating a promising dose-dependent efficacy trend. Most recently, in 2026, a Nature Biotechnology study revealed a consistent reduction in biological age after Rentosertib dosage, across six independent biological aging clocks.

"I believe Rentosertib will be transformative to patient care in the field of IPF, and AI has enabled this breakthrough with higher speed and reduced cost, which could make a lot of difference in the future of drug discovery," said Carol Satler, MD, PhD, Senior Vice President for Clinical Development, Non-Oncology at Insilico Medicine. "With the first patient dosed, we hope to see real-world improvements and further validate the favorable findings observed in Phase IIa study, and to witness the rapid translation of a scientific concept into potential patient benefit in record time."

Insilico's dual-purpose therapeutic strategy is also proving its viability as a sustainable commercial model. Recently, Insilico reported total revenue of approximately $106 million in the first half of 2026, a 287% year-over-year increase, and achieved its first profitable half-year since listing, with an adjusted net profit exceeding $51 million. This milestone was driven by a series of out-licensing, co-development, and R&D collaborations with global partners, including Eli Lilly, Servier, Takeda, SK Biopharmaceuticals, Qilu Pharmaceutical, Hygtia Therapeutics, CMS, and Tenacia. As of the latest practicable date, the total contract value of transactions announced by Insilico in 2026 reached approximately $7.3 billion, pushing the cumulative contract value of its major collaborations since 2021 approximately $11 billion.

On the AI-driven R&D front, Insilico nominated nine development candidates within nine months of 2026 as of late August, setting a new company record for annual pipeline productivity and achieving eight clinical milestones across its proprietary and co-developed programs. Leading this progress is rentosertib (ISM001-055), the world's first drug candidate discovered and developed using generative AI, which has advanced to a Phase III trial evaluating for idiopathic pulmonary fibrosis (IPF). In addition, Insilico Medicine launched a comprehensive set of benchmarks that allow foundation models to be evaluated in all tasks needed for drug discovery and launched state of the art (SOTA) foundation models outperforming other models and even internal tools in benchmarks. Using this new capability Insilico hopes to expand and accelerate longevity drug discovery and development both in terms of scale, therapeutic modalities, and indications.

About the Phase III Clinical Trial

The Phase III clinical trial will evaluate Rentosertib in patients with idiopathic pulmonary fibrosis. The 52-week prospective, randomized, multi-center, double-blind, placebo-controlled, parallel-group Phase III study in patients with idiopathic pulmonary fibrosis is expected to enroll a total of 320 participants across 47 centers in China. The primary endpoint is the annual rate of decline in forced vital capacity (FVC) over 52 weeks. The Key secondary endpoint is time to first occurrence of any disease progression event. The trial is intended to assess whether Rentosertib can provide clinically meaningful benefit in a larger patient population and over a longer treatment period than the Phase IIa study. While the initiation of the Phase III trial represents an important milestone in the development of Rentosertib, the drug remains investigational and has not been approved by any regulatory authority.

About Rentosertib

Rentosertib is a potentially first-in-class small molecule targeting TNIK developed utilizing generative AI. In IPF, the activation of TNIK drives pathological fibrosis in the lungs, contributing to the progressive decline in lung function. By inhibiting TNIK, Rentosertib aims to halt or reverse fibrotic processes, offering a disease-modifying treatment for patients with IPF. The U.S. Food and Drug Administration granted Orphan Drug Designation to Rentosertib for IPF in February 2023.

The program's discovery-to-clinic development was published in Nature Biotechnology, its Phase IIa clinical results were published in Nature Medicine, and its medicinal chemistry was published in the Journal of Medicinal Chemistry. Recently, Rentosertib has demonstrated potential to reverse biological age, with the data and analysis results supported by six different aging clock models published in Nature Biotechnology.

About Idiopathic Pulmonary Fibrosis (IPF)

Idiopathic Pulmonary Fibrosis (IPF) is a chronic, scarring lung disease characterized by a progressive and irreversible decline in lung function. Affecting approximately 5 million people worldwide, IPF carries a poor prognosis, with a median survival of 3 to 4 years. Current approved treatments, including antifibrotic drugs, can slow disease progression but do not stop or reverse it, leaving a significant unmet need for more effective, disease-modifying therapies.

About Insilico Medicine

Insilico Medicine is a pioneering global biotechnology company dedicated to integrating artificial intelligence and automation technologies to accelerate drug discovery, drive innovation in the life sciences, and extend healthy longevity to people on the planet. The company was listed on the Main Board of the Hong Kong Stock Exchange on December 30, 2025, under the stock code 03696.HK.

By integrating AI and automation technologies and deep in-house drug discovery capabilities, Insilico is delivering innovative drug solutions for unmet needs including fibrosis, oncology, immunology, pain, and obesity and metabolic disorders. Additionally, Insilico extends the reach of Pharma. AI across diverse industries, such as advanced materials, agriculture, nutritional products and veterinary medicine.

For more information, please visit www.insilico.com

SOURCE Insilico Medicine