Chemistry:Rentosertib
Rentosertib (also known as ISM001‑055[1] and INS018_055) is an investigational new drug that is being evaluated for the treatment of idiopathic pulmonary fibrosis (IPF). It targets TNIK (TRAF2 and NCK-interacting protein kinase).[1] It is asserted to be the first drug generated entirely by generative artificial intelligence to reach mid-stage human clinical trials, and the first to target a novel AI-discovered biological pathway.[2][3][4]
Development
Rentosertib was developed using a generative AI platform designed to identify novel disease-associated targets and rapidly design optimized small-molecule inhibitors.[1][5][6] TNIK was identified as a central regulator of fibrosis and inflammation in IPF. Rentosertib selectively inhibits TNIK, and progressed from target discovery through phase 0 and 1 clinical trials in under 30 months.[1]
Clinical trials
A multicenter, double-blind, placebo-controlled, randomized phase 2a trial was conducted in China, testing the drug in 71 IPF patients over a period of 12 weeks. Participants were randomly assigned to receive 30 mg once daily (QD), 30 mg twice daily (BID), 60 mg QD, or placebo.[1]
The trial's primary endpoint was the incidence of treatment-emergent adverse events (TEAEs).[1]
Nomenclature
In March 2025, the United States Adopted Names (USAN) Council approved "Rentosertib" as the official nonproprietary name for ISM001-055.[5]
References
- ↑ 1.0 1.1 1.2 1.3 1.4 1.5 Cite error: Invalid
<ref>tag; no text was provided for refs namedXu_2025 - ↑ Cite error: Invalid
<ref>tag; no text was provided for refs namedField_2023 - ↑ "World's first AI-developed drug for deadly lung disease enters landmark clinical trials in China, US". South China Morning Post. 13 March 2024. https://www.scmp.com/news/china/science/article/3255232/worlds-first-ai-developed-drug-deadly-lung-disease-enters-landmark-clinical-trials-china-us.
- ↑ "Artificial Intelligence and Machine Learning in Acute Respiratory Distress Syndrome Management: Recent Advances.". Frontiers in Medicine 12. July 2025. doi:10.3389/fmed.2025.1597556. PMID 40740956.
- ↑ 5.0 5.1 "First AI-designed drug, Rentosertib, officially named by USAN". March 14, 2025. https://www.drugtargetreview.com/news/157365/first-ai-designed-drug-rentosertib-named-by-usan/.
- ↑ "Insilico Medicine Announces Developmental Candidate Benchmarks and Timelines for Novel Therapeutics Discovered Using Generative AI". EurekAlert! (American Association for the Advancement of Science). February 11, 2025. https://www.eurekalert.org/news-releases/1073344.
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