Source record
A generative-AI-designed molecule in a phase 2a IPF trial
The source is Xu and colleagues, Nature Medicine, 3 June 2025 (doi:10.1038/s41591-025-03743-2), a 12-week randomized phase 2a trial of rentosertib in idiopathic pulmonary fibrosis. The molecule was designed on Insilico Medicine’s generative platform. Seventy-one patients were randomized. The primary endpoint was safety. A secondary forced-vital-capacity comparison at 60 mg once daily was +98.4 mL versus −20.3 mL on placebo, in small samples, and the authors said it needs a larger, longer trial.
- Source date
- Jun 3, 2025 · Year 2025
- Evidence label
- Published
- Source type
- Paper
Citation
Xu et al., Nature Medicine
Xu et al. Twelve-week randomized phase 2a trial of rentosertib (ISM001-055), a TNIK inhibitor designed with a generative platform, in idiopathic pulmonary fibrosis. Nature Medicine, 3 June 2025. doi:10.1038/s41591-025-03743-2. https://doi.org/10.1038/s41591-025-03743-2
Key public facts
- Insilico’s generative platform proposed TNIK and designed rentosertib (formerly ISM001-055) before the clinical trial.
- The trial was multicenter, double-blind, and placebo-controlled in adults with idiopathic pulmonary fibrosis at sites in China (NCT05938920).
- Seventy-one patients were randomized. The primary endpoint was safety over 12 weeks, not lung function.
- Treatment-emergent adverse events were about 71–83% across arms. Discussed discontinuations involved liver-test abnormalities or diarrhea.
- At 60 mg once daily, mean forced vital capacity changed by +98.4 mL versus −20.3 mL on placebo. That comparison was secondary, the samples were small, and the authors called for a larger, longer trial.
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