Rentosertib — first clinical proof-of-concept for an AI-discovered target
Insilico's TNIK inhibitor, with both target and molecule generated computationally, reported phase 2a results in idiopathic pulmonary fibrosis. Across 71 patients at 21 sites, the 60 mg once-daily arm showed a mean FVC change of +98.4 mL against −20.3 mL on placebo, with exploratory biomarker analysis supporting the proposed mechanism.
Novel-target programs live or die on phenotyped patient cohorts deep enough to validate mechanism and stratify responders — before a trial, not after it.