Insilico Medicine, a clinical-stage generative AI-driven drug discovery company, announced positive preliminary results from its Phase IIa clinical trial evaluating ISM001-055, a first-in-class small molecule targeting TNIK (Traf2- and Nck-interacting kinase). ISM001-055 was designed using a unique AI platform to treat idiopathic pulmonary fibrosis (IPF).
ISM001-055’s Phase IIa study (NCT05938920) was a randomized, double-blind, placebo-controlled trial that enrolled 71 patients with IPF across 21 sites in China. Patients received either placebo, 30mg once daily (QD), 30mg twice daily (BID), or 60mg QD for 12 weeks. Patient enrollment began in April 2023, and the last subject’s follow-up visit was completed in August 2024. A parallel Phase IIa (NCT05975983) clinical trial in the U.S. is ongoing and actively enrolling patients.
In this 12-week Phase IIa study, ISM001-055 met its primary endpoint of safety and tolerability across all dose levels. Positive results were also reported for the secondary efficacy endpoint, wherein a dose-dependent FVC improvement was observed. Patients receiving 60mg QD of ISM001-055 demonstrated the largest improvement in FVC.
Following the positive results, Insilico Medicine will engage with regulators to discuss a Phase IIb study. The company aims to explore extended treatment durations and larger patient cohorts to further investigate ISM001-055’s therapeutic potential in IPF.
Source: Insilico Medicine reports positive Phase IIa results for ISM001-055, a novel first-in-class drug treatment for idiopathic pulmonary fibrosis (IPF) designed using generative AI
https://www.eurekalert.org/news-releases/1058384
A small-molecule TNIK inhibitor targets fibrosis in preclinical and clinical models
https://www.nature.com/articles/s41587-024-02143-0