First AI-discovered drug enters Phase III trial

Insilico Medicine announced on Tuesday that it dosed the first patient with Rentosertib in GENESIS-IPF-3, marking the first time a drug candidate discovered and designed entirely by generative AI has entered a Phase III clinical trial. The dosing took place at Peking Union Medical College Hospital in Beijing, with Shanghai Pulmonary Hospital enrolling its first patient on the same day.insilico+2

Rentosertib, a small molecule inhibitor of TNIK, is being evaluated for the treatment of idiopathic pulmonary fibrosis, a chronic scarring lung disease that affects roughly 5 million people worldwide and carries a median survival of three to four years. Current approved antifibrotic treatments can slow disease progression but cannot stop or reverse it.insilico

A Trial Built on Phase IIa Results

The 52-week, randomized, double-blind, placebo-controlled study will enroll 320 patients across 47 centers in China. Its primary endpoint is the annual rate of decline in forced vital capacity (FVC), the standard registrational measure in IPF trials. In the earlier Phase IIa study, patients receiving 60 mg once daily showed a mean FVC change of +98.4 mL at 12 weeks, compared with -20.3 mL in the placebo group.insilico+2

The trial is led by Professor Zuojun Xu of Peking Union Medical College Hospital, with Academician Nanshan Zhong and President Chang Chen of Shanghai Pulmonary Hospital serving as co-leading principal investigators.insilico

"TNIK, the target driven by AI, had never previously been linked to fibrosis," Professor Xu said. "This perhaps indicates that AI is carving out a path distinct from traditional research paradigms in target discovery for complex diseases."insilico

From Discovery to Phase III

Rentosertib's journey from AI-identified target to Phase III has been documented across several peer-reviewed publications: its discovery process in Nature Biotechnology in 2024, Phase IIa results in Nature Medicine in 2025, and biological aging reversal data in Nature Biotechnology in 2026. The U.S. FDA granted the drug Orphan Drug Designation for IPF in February 2023, and China's Center for Drug Evaluation granted Breakthrough Therapy Designation in May 2025.allsci+1

Insilico, which listed on the Hong Kong Stock Exchange in December 2025, reported $106 million in revenue for the first half of 2026, a 287% year-over-year increase, and its first profitable half-year since listing. The company has also been advancing an inhaled formulation of Rentosertib in a parallel Phase I study following IND clearance in April 2026. Professor Xu estimated that obtaining final regulatory approval from the Phase III stage will take three to four years under favorable conditions.insilico+1