The trial assessed the safety, tolerability, pharmacokinetics, and preliminary efficacy of 12-week oral ISM001-055 to treat IPF. Credit: Jo Panuwat D / Shutterstock.

Insilico Medicine has announced positive preliminary results from its Phase IIa clinical trial of ISM001-055, a small molecule drug designed using generative AI, to treat idiopathic pulmonary fibrosis (IPF).

The randomised, double-blind, placebo-controlled trial was conducted across 21 sites in China, enrolling 71 patients with IPF.

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It assessed the safety, tolerability, pharmacokinetics, and preliminary efficacy of 12-week oral ISM001-055.

The trial included four cohorts: placebo, 30mg once daily (QD), 30mg twice daily (BID), and 60mg QD.

Patient enrolment commenced in April 2023 and concluded in August 2024. The study’s positive outcomes will aid the company in holding talks with the regulatory authorities regarding a Phase IIb study design.

According to the findings, the trial met its primary safety endpoint and secondary efficacy endpoints, indicating a dose-dependent improvement in lung function among IPF patients.

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The 12-week study also demonstrated the drug’s safety and tolerability across all dosage levels, with the 60mg QD dose showing the most significant improvement in forced vital capacity (FVC), a key measure of lung health in IPF patients.

Parallel to the Chinese study, a Phase IIa trial is actively enrolling patients in the US.

Insilico Medicine co-CEO Alex Zhavoronkov said: “This study result represents a critical milestone in AI-powered drug discovery and my life to date. 

“While we expected the drug to be safe, we did not expect to see such a clear dose-dependent efficacy signal after such a short dosing period. IPF is a very diverse disease, and it is very rare to see improvement in FVC. With our novel TNIK inhibitor, we attempted to go after what we think is a common mechanism in fibrotic diseases and in ageing to maximise indication expansion potential.”