Insilico Medicine's AI-Designed Drug Clears Phase 2a for Idiopathic Pulmonary Fibrosis
Somewhere in China, across 22 clinical sites, 71 patients with a disease called idiopathic pulmonary fibrosis just took part in something that had never happened before: a drug where both the target and the molecule itself were designed by generative AI, not a human research team.
The drug is called rentosertib, though you might see it under its earlier name, ISM001-055. It's a small-molecule TNIK inhibitor, and the trial, GENESIS-IPF, was randomized, double-blind, and placebo-controlled — the gold standard for proving a drug actually works, not just that it seems to.
It passed its main safety goal. Side effects looked similar across all dosing groups. Then came the part worth paying attention to: at the highest dose, 60 mg once daily, patients' lung function improved. Forced vital capacity rose by an average of 98.4 mL over 12 weeks. The placebo group's lung function fell by 20.3 mL over the same period. Two key markers tied to lung scarring, COL1A1 and MMP10, dropped too, exactly what you'd expect if the drug's proposed mechanism is real.
The results were published in Nature Medicine and presented at ATS 2025. Rentosertib has since moved into a phase III trial.
As of this writing, no AI-discovered medicine has been approved by a major regulator anywhere in the world. Rentosertib is one of the furthest along in trying to be first, which is exactly why its phase III trial is worth keeping an eye on.