Insilico Medicine’s AI-designed drug for idiopathic pulmonary fibrosis (IPF), Rentosertib, has been granted an official name by USAN. This is the first drug where both the target and compound were discovered using generative AI, marking a major milestone in AI-driven drug development.
AI or Artificial intelligence is being used to develop new drugs, diagnose diseases, and provide personalized care to patients 3d rendering
Insilico Medicine, a leading biotechnology company leveraging generative artificial intelligence (AI) for drug discovery, has announced that its drug candidate for idiopathic pulmonary fibrosis (IPF), previously known as ISM001-055, has been granted the official generic name Rentosertib by the United States Adopted Names (USAN) Council. Rentosertib is 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 severe, chronic lung disease that progressively impairs lung function and affects approximately five million people worldwide. With a median survival of only three to four years, current treatments can only slow its progression, failing to halt or reverse the disease. This significant unmet medical need has driven researchers to explore novel approaches to treatment.
AI-powered drug discovery revolution
Insilico Medicine’s pioneering use of its proprietary Pharma.AI platform has enabled the rapid discovery and development of Rentosertib. The research began with PandaOmics, an AI-powered biology engine that analysed vast datasets to identify TNIK (TRAF2 and NCK-interacting kinase) as a promising target for IPF treatment. Following this, Chemistry42, the platform’s generative chemistry engine, was utilised to design and optimise small-molecule compounds targeting TNIK, leading to the nomination of Rentosertib as a preclinical candidate.
This AI-driven approach significantly accelerated the drug discovery process, reducing the timeline from target identification to preclinical candidate selection to just 18 months. These findings mark a major advancement in the efficiency of pharmaceutical research.
Graphic of lungs
Idiopathic Pulmonary Fibrosis (IPF) is a chronic and progressive lung disease that causes scarring of lung tissue, making it increasingly difficult for the lungs to function properly. This leads to worsening breathlessness and reduced oxygen supply to the body, significantly affecting patients’ quality of life and life expectancy.
Clinical progress and promising results
Rentosertib has progressed through multiple clinical trials with encouraging outcomes. In two Phase I trials conducted in New Zealand and China, the drug was administered orally to healthy participants and demonstrated a favourable safety, tolerability, and pharmacokinetics (PK) profile. These results supported its transition into Phase II trials.
A subsequent Phase IIa clinical trial focused on IPF patients over a 12-week period. Rentosertib met its primary endpoint of safety and tolerability at all dosage levels. Furthermore, positive results were observed for the secondary efficacy endpoint, showing a dose-dependent improvement in lung function. Patients receiving the highest dose of 60mg QD exhibited a mean improvement of 98.4 mL in forced vital capacity (FVC) from baseline, while the placebo group experienced a mean decline of -62.3 mL. Improvements were also recorded in quality-of-life measures such as cough reduction and overall respiratory symptoms at the highest dose.
Rentosertib is the first drug whose target and design were discovered by modern generative 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,” stated Alex Zhavoronkov, Founder and CEO of Insilico Medicine. “The name Rentosertib is especially meaningful to us, as it not only honours Dr Ren’s contributions, but also highlights the essential interplay between human scientific expertise and artificial intelligence in driving this innovative programme forward.”
Next steps and future prospects
With the positive Phase IIa results and official naming by USAN, Insilico Medicine is now engaging with global regulatory authorities to initiate larger pivotal trials. The company aims to further evaluate Rentosertib’s efficacy and expedite its path to regulatory approval. If successful, Rentosertib could become the first AI-discovered therapy to reach patients, potentially transforming the treatment landscape for IPF.