Rentosertib
Rentosertib is an investigational new drug that is being evaluated for the treatment of idiopathic pulmonary fibrosis. It targets TNIK. It is asserted to be the first drug generated entirely by generative artificial intelligence to reach mid-stage human clinical trials, and the first to target a novel AI-discovered biological pathway.
Development
Rentosertib was developed using a generative AI platform designed to identify novel disease-associated targets and rapidly design optimized small-molecule inhibitors. TNIK was identified as a central regulator of fibrosis and inflammation in IPF. Rentosertib selectively inhibits TNIK, and progressed from target discovery through phase 0 and 1 clinical trials in under 30 months.Clinical trials
A multicenter, double-blind, placebo-controlled, randomized phase 2a trial was conducted in China, testing the drug in 71 IPF patients from July 2023 to June 2024, encompassing an administration period of 12 weeks. Participants were randomly assigned to receive 30 mg once daily, 30 mg twice daily, 60 mg QD, or placebo.The trial's primary endpoint was the incidence of treatment-emergent adverse events.