Insilico Medicine reports positive results for idiopathic pulmonary fibrosis therapy

ISM001-055 shows promise in improving lung function among patients

Insilico Medicine, a clinical-stage generative AI-driven drug discovery company, has announced positive preliminary results from its Phase 2a clinical trial of ISM001-055.

This first-in-class small molecule targets TNIK (Traf2- and Nck-interacting kinase) and was designed using generative AI to treat idiopathic pulmonary fibrosis (IPF).

The study met its primary endpoint of safety and secondary efficacy endpoints, showing a dose-dependent response in forced vital capacity (FVC), a critical measure of lung function in IPF patients.

Insilico’s proprietary AI platform played a crucial role in identifying the target and designing the molecule. The development of ISM001-055 was detailed in a March 2024 Nature Biotechnology paper, which highlighted TNIK as a novel therapeutic target in IPF and described the preclinical and early clinical studies that supported its potential as a disease-modifying agent.

The Phase IIa study (NCT05938920) was a randomised, double-blind, placebo-controlled trial involving 71 IPF patients across 21 sites in China.

Participants were randomised to receive either a placebo, 30mg once daily (QD), 30mg twice daily (BID), or 60mg QD for 12 weeks.

Enrollment began in April 2023, and the last follow-up visit was completed in August 2024. A parallel Phase 2a trial (NCT05975983) in the US is ongoing and actively enrolling patients.

In this 12-week study, ISM001-055 demonstrated a favorable safety profile and tolerability across all dose levels. Additionally, a dose-dependent improvement in FVC was observed, with the 60mg QD dose showing the most significant improvement.

Complete topline data will be presented at an upcoming medical conference, and the clinical trial results will be submitted for publication in a peer-reviewed journal.

The positive Phase 2a results represent a proof-of-concept success for AI-driven drug discovery and offer hope for a new therapeutic option for IPF patients.