Insilico Medicine: first positive clinical results for a drug entirely designed by AI

The drug candidate ISM001-055, targeting idiopathic pulmonary fibrosis, successfully completes a Phase IIa study — a world first for a molecule both discovered and designed by generative artificial intelligence.

Insilico Medicine announced positive results for ISM001-055, a candidate for the treatment of idiopathic pulmonary fibrosis (IPF), both of which have been identified by generative artificial intelligence. The Phase IIa double-blind placebo trial involved 71 patients at 21 sites in China.

At the highest dose tested (60 mg once daily), patients experienced an average increase in their forced vital capacity of 98.4 mL over 12 weeks, compared with a decrease of 20.3 mL in the placebo group — a clinical signal considered encouraging. The overall safety profile remains favourable, with the most common side effects (diarrhoea, mild to moderate liver abnormalities) affecting approximately 15% of patients treated.