image: Rentosertib stands as the first investigational drug in which both the biological target and the therapeutic compound were discovered using generative AI.
Credit: Insilico Medicine
Cambridge, MA – March 6, 2025 – Insilico Medicine("Insilico"), a clinical stage generative artificial intelligence (AI)-driven biotechnology company, recently announced that its breakthrough drug candidate for idiopathic pulmonary fibrosis (IPF) – Rentosertib (formerly known as ISM001-055) – has been granted an official generic name by the United States Adopted Names (USAN) Council. Rentosertib stands as the first investigational drug in which both the biological target and the therapeutic compound were discovered using generative AI.
Idiopathic Pulmonary Fibrosis (IPF) is a chronic, scary lung disease characterized by a progressive and irreversible decline in lung function. Affecting approximately 5 million people worldwide, IPF carries poor prognosis, with a median survival of 3 to 4 years. Current 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.
Insilico adopted a pioneering approach to anti-IPF research by leveraging its proprietary Pharma.AI platform to discover innovative therapeutics with the potential to halt or even reverse disease progression. The process began with PandaOmics, the platform’s biology engine, which analyzed vast omics and clinical datasets to identify TNIK (TRAF2 and NCK-interacting kinase) as a promising novel target for IPF. Building on this discovery, researchers used Chemistry42, the platform’s generative chemistry engine, to swiftly design and optimize new small-molecule compounds targeting TNIK, leading to the nomination of Rentosertib as the preclinical candidate.
This integrated AI-driven workflow drastically accelerated the development timeline, progressing from initial target identification to a preclinical candidate in just 18 months, as detailed in Insilico’s Nature Biotechnology paper published in March 2024. Notably, the name Rentosertib is partially derived from Feng Ren, PhD, Insilico’s co-CEO and Chief Scientific Officer, who was also the first author of the landmark publication.
“This is an important moment for the pharmaceutical industry and AI – Rentosertib is the first drug whose target and design were discovered by modern generative AI and now it has achieved an official name on the path to patients,” said Alex Zhavoronkov, Founder and CEO of Insilico Medicine. “The name Rentosertib is especially meaningful to us, as it not only honors Dr. Ren’s contributions, but also highlights the essential interplay between human scientific expertise and artificial intelligence in driving this innovative program forward. We hope that Rentosertib’s success in IPF will pave the way for faster and more cost-effective discoveries of lifesaving treatments for many other diseases using AI.”
"I am honored to have witnessed and led Rentosertib from target discovery to clinic development," said Feng Ren, PhD, co-CEO and Chief Scientific Officer of Insilico Medicine, “ An official generic name is typically assigned as a drug enters mid-stage development, signifying recognition of its potential as a new therapy. Rentosertib now joins the list of recognized drug candidates and will be referred to by this name in scientific literature and future clinical trials, replacing its laboratory code. We aim to rapidly advance the clinic development of this program, providing innovative options for patients while bringing solid validation for the AI-driven drug discovery industry.”
Currently, Rentosertib has successfully advanced through multiple clinical studies with encouraging results. In two Phase I trials conducted in New Zealand and China, Rentosertib was administered orally to healthy subjects in Phase I trials, yielding consistent results. The studies demonstrated favorable safety, tolerability, and pharmacokinetics (PK) profiles of Rentosertib, providing robust evidence to support its progression to Phase II clinical trials.
Building on this foundation, Insilico carried out a Phase IIa clinical trial in IPF patients to evaluate Rentosertib’s efficacy. In this 12-week Phase IIa study, Rentosertib met its primary endpoint of safety and tolerability across all dose levels. Positive results were also reported for the secondary efficacy endpoint, wherein a dose-dependent forced vital capacity (FVC) improvement was observed. Key findings from the Phase IIa trial include:
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Dose-dependent improvements in lung function: Patients who received Rentosertib showed greater improvements in lung capacity as measured by FVC. At the highest doses of 60mg QD, patients experienced a 98.4 mL mean improvement in FVC from baseline, compared to a mean decline in FVC change from baseline of -62.3 mL for patients in the placebo group.
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Additional improved clinical outcomes: A similar dose-related trend was observed in other measures – for instance, patients on the high dose showed a small improvement in percent predicted FVC (a normalization of lung capacity for age and size), while the placebo group declined. Patients taking Rentosertib also reported improvements in quality-of-life measures such as cough and overall respiratory symptoms at the highest dose.
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Strong safety and tolerability: Rentosertib continued to demonstrate a favorable safety profile in patients and the drug was well tolerated across all dosing groups. Most drug-related side effects were mild to moderate. No serious adverse events related to Rentosertib were reported, and safety findings were consistent with those seen in the earlier Phase I trials.
With the positive Phase IIa results and an official USAN name, Insilico plans to engage with global regulatory authorities and initiate larger pivotal trials to further evaluate Rentosertib’s efficacy in IPF. Insilico is dedicated to advancing Rentosertib, with the goal of making it the first AI-discovered therapy to reach patients, providing a critically needed new option for those affected by IPF and showcasing the transformative potential of generative AI in accelerating medical breakthroughs.
About Insilico Medicine
Insilico Medicine, a global clinical stage biotechnology company powered by generative AI, is connecting biology, chemistry, medicine and science research using next-generation AI systems. The company has developed AI platforms that utilize deep generative models, reinforcement learning, transformers, and other modern machine learning techniques for novel target discovery and the generation of novel molecular structures with desired properties. Insilico Medicine is developing breakthrough solutions to discover and develop innovative drugs for cancer, fibrosis, central nervous system diseases, infectious diseases, autoimmune diseases, and aging-related diseases.