First Phase III trial of a generative-AI-discovered drug doses first patient (Insilico's rentosertib)
On 2026-09-10 Insilico Medicine dosed the first patients in GENESIS-IPF-3, billed as the world's first Phase III trial of a drug whose target and molecule were discovered with generative AI: rentosertib, a TNIK inhibitor for idiopathic pulmonary fibrosis, tested in 320 patients at 47 Chinese centers over 52 weeks.
Key facts
- First patients dosed 2026-09-10 at Peking Union Medical College Hospital and Shanghai Pulmonary Hospital
- Randomized, double-blind, placebo-controlled; 320 participants; 47 centers in China; once daily for 52 weeks
- Primary endpoint: annual rate of FVC decline over 52 weeks; key secondary: time to first disease-progression event
- Phase IIa (Nature Medicine, 2025): 60 mg QD arm showed mean FVC +98.4 mL at 12 weeks, dose-dependent trend
- Mechanism: TNIK inhibition (target also identified by Insilico's AI platform)
Science result
- Field
- medicine / drug discovery / pulmonary fibrosis
- Problem
- Idiopathic pulmonary fibrosis (IPF) therapy via a novel target
- Result
- AI-identified target (TNIK) and AI-designed molecule (rentosertib) reached Phase III after a Phase IIa in Nature Medicine showing +98.4 mL mean FVC at 12 weeks on 60 mg vs a decline on placebo.
- AI system
- Insilico Pharma.AI (PandaOmics, Chemistry42)
- Human role
- AI-assisted: AI proposed target and molecule; human chemists, clinicians and regulators ran development and trials
- Verification
- Phase IIa peer-reviewed in Nature Medicine (June 2025); Phase III ongoing
- Status
- pending
- Why surprising
- The first drug with both target and molecule discovered by generative AI entered Phase III about five years after target discovery.
What happened
Insilico's rentosertib is the furthest-advanced drug in which both the target and the molecule came from generative AI. Phase III is the final stage before regulatory approval.
Why it matters
If positive (results likely 2027+), it would be the first approved generative-AI-discovered drug — the key proof point for AI drug discovery's promise to cut time and cost.
Changelog
- 2026-09-29: created
- 2026-09-29: added science block (science & math tab)
Related events
Sources (4)
- officialInsilico: first patient dosed in GENESIS-IPF-3
- officialPR Newswire: Insilico initiates Phase III trial for rentosertib
- pressEurekAlert: Nature Medicine publishes rentosertib Phase IIa results (June 2025)
- pressDrug Target Review: Insilico begins Phase III of AI-designed drug
id: 2026-09-10-insilico-rentosertib-phase-3 · updated 2026-09-29 · open in the interactive timeline