Longevity & AgingAI-Designed Drug Shows Lung Function Gains in Idiopathic Pulmonary Fibrosis Trial
Rentosertib (ISM001-055), a small-molecule TNIK inhibitor discovered and designed using generative AI by Insilico Medicine, completed a phase 2a randomized, double-blind, placebo-controlled trial in 71 IPF patients across 16 Chinese centers. Patients received 30 mg once daily, 30 mg twice daily, 60 mg once daily, or placebo for 12 weeks. The primary safety endpoint was met with adverse event rates comparable across all groups. Crucially, the 60 mg once-daily group showed a mean FVC increase of +98.4 ml versus a −20.3 ml decline in placebo — a clinically meaningful signal. The trial marks a milestone as one of the first AI-generated molecules to demonstrate efficacy signals in a controlled human trial, with the entire journey from target discovery to phase 2a completed in roughly four years.
