AI-Designed Drug Shows Early Promise for Lung Fibrosis Patients in Clinical Trial
- Researchers published results on June 4, 2025, from a 12-week phase 2a trial of rentosertib, an AI-designed drug for idiopathic pulmonary fibrosis in China.
- The trial followed a randomized, placebo-controlled design with 71 patients over 40 years old, excluding 57 for comorbidities or recent infections.
- Rentosertib, a TNIK inhibitor developed using AI, showed lung function improvements measured by forced vital capacity, especially at the 60 mg daily dose.
- The 60 mg dose group gained +98.4 ml in lung capacity, with similar adverse event rates across groups and manageable side effects reported during the trial.
- The study highlights AI's potential in drug discovery but notes limitations like small, homogeneous cohorts and short follow-up, requiring larger trials for validation.
12 Articles
12 Articles


AI-designed drug shows early promise for lung fibrosis patients in clinical trial
Researchers report that rentosertib, an AI-discovered TNIK inhibitor, showed promising safety and potential to improve lung function in patients with idiopathic pulmonary fibrosis in a 12-week phase 2a clinical trial. The highest dose group demonstrated a trend toward increased forced vital capacity, especially among patients not receiving standard antifibrotic therapy, supporting further clinical investigation.
A generative AI-discovered TNIK inhibitor for idiopathic pulmonary fibrosis: a randomized phase 2a trial
Despite substantial progress in artificial intelligence (AI) for generative chemistry, few novel AI-discovered or AI-designed drugs have reached human clinical trials. Here we present the results of the first phase 2a multicenter, double-blind, randomized, placebo-controlled trial testing the safety and efficacy of rentosertib (formerly ISM001-055), a first-in-class AI-generated small-molecule inhibitor of TNIK, a first-in-class target in idiopa…
Insilico’s rentosertib clears a phase 2a hurdle
An AI-designed drug, rentosertib, from Insilico Medicine improved lung function by 98.4 mL in a 71-patient idiopathic pulmonary fibrosis study published June 3 in Nature Medicine, marking what may be the first peer-reviewed phase 2a result for a molecule generated, with its target discovered, entirely by generative AI. In an email, Insilico CEO Alex Zhavoronkov,… The post Insilico’s AI-designed rentosertib shows promise in first phase 2a trial r…
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