Insilico AI-Designed Drug Shows Efficacy in Lung Disease Phase 2a Trial

Insilico Medicine, a biotech company with headquarters in Boston and Hong Kong, has announced that its AI-designed drug candidate, rentosertib, showed promising safety and efficacy in treating idiopathic pulmonary fibrosis (IPF) during a phase 2a clinical trial. This marks the first time an AI-created drug, developed through AI-identified targets, has passed a mid-stage clinical study, representing a significant milestone in AI-driven drug discovery. The 12-week trial involving 71 patients demonstrated improved lung function at higher doses, with rentosertib targeting a novel protein linked to lung scarring, as predicted by the AI model. While some patients experienced manageable liver-related side effects, the drug met its safety objectives, and Insilico plans larger trials to validate the findings and seek regulatory approval. The success of rentosertib highlights the transformative potential of AI in accelerating drug discovery and development, potentially revolutionizing treatments for diseases like IPF, which currently have limited therapeutic options. Insilico's CEO Alex Zhavoronkov described the results as among the most remarkable ever seen for this lung condition and emphasized the broader impact this breakthrough could have on healthcare innovation.