Insilico Medicine Touts AI-Driven Drug Discovery

On June 3, the industry’s first proof-of-concept clinical validation of AI-driven drug discovery was published in Nature Medicine. Insilico Medicine, with twin headquarters in Boston and Hong Kong, reported promising safety and efficacy results from a Phase IIa trial of Rentosertib, a TNIK inhibitor developed using Insilico’s generative AI platform for idiopathic pulmonary fibrosis (IPF). Additionally, the exploratory analysis of biomarkers in the paper further validated the biological mechanism of TNIK inhibition, the novel target identified through a generative AI approach, supporting Rentosertib’s potential anti-fibrotic and anti-inflammatory effects.

Idiopathic Pulmonary Fibrosis (IPF) is a chronic lung disease characterized by a progressive and irreversible decline in lung function. Affecting approximately 5 million people worldwide, IPF carries a poor prognosis, with a median survival of 3 to 4 years. Current approved treatments can slow disease progression but do not stop or reverse it, leaving a significant unmet need for more effective, disease-modifying therapies.

Rentosertib is a potentially first-in-class small molecule targeting TNIK developed utilizing generative AI. In IPF, activation of TNIK drives pathological fibrosis in the lungs, contributing to the progressive decline in lung function. By inhibiting TNIK, Rentosertib aims to halt or reverse fibrotic processes, offering a disease-modifying treatment for patients with IPF.

By integrating advanced AI and automation technologies, Insilico Medicine has demonstrated significant efficiency improvements in practical applications, setting a benchmark for AI-driven drug research and development. Compared to the typical 2.5–4 years required in traditional drug discovery, Insilico’s 22 nominated candidate drugs from 2021 to 2024 took only 12–18 months on average to progress from project initiation to nomination of preclinical candidates (PCCs), with each project requiring synthesis and testing of only about 60–200 molecules. The success rate from PCC to IND-enabling stage reached 100%.

According to Dr. Zuojun Xu, Professor at the Peking Union Medical College and the lead investigator of the Phase IIa clinical trial of Rentosertib in IPF patients, “This study demonstrates that Rentosertib has the potential to provide meaningful clinical benefits for IPF patients…. However, the sample size in each patient group was relatively limited, and these findings will need to be validated in larger cohort studies.” In light of the encouraging study results, Insilico has begun discussions with regulatory authorities to facilitate the prospective evaluation of Rentosertib in larger cohorts of patients. (Source: Insilico Medicine Website, 03 June, 2025)