Insilico Medicine

Idiopathic pulmonary fibrosis (IPF) is a chronic, progressive lung disease affecting ~100,000 people in the U.S. with a prognosis of death within 2–5 years if untreated. Existing treatments only slow disease progression and cause uncomfortable side effects. Insilico sought a 'moonshot' molecule targeting a novel biological mechanism rather than incrementally improving existing therapies.

Insilico Medicine applied its proprietary generative AI platform to simultaneously discover a novel disease target and design a novel small-molecule drug candidate (INS018_055) from scratch. The AI performed both target identification and molecular generation — making it the first drug with both an AI-discovered target and an AI-generated molecular design. Discovery work began in 2020 and the compound was advanced into a randomized, double-blind, placebo-controlled Phase II trial.

INS018_055 became the first fully generative AI drug to reach Phase II human clinical trials, enrolling patients in China with plans to expand to 60 subjects across 40 sites in the U.S. and China. The Phase II readout is expected in 2024. Insilico also has two additional AI-assisted compounds in the clinical stage: a COVID-19 drug in Phase I and an FDA-cleared cancer drug (USP1 inhibitor for solid tumors).

• End-to-end generative AI can compress the drug discovery timeline from target identification through clinical candidate nomination, bypassing years of traditional screening.
• Validating an AI drug-discovery platform requires advancing compounds all the way into human trials — in-silico results alone are insufficient proof.
• A single AI platform can be applied across multiple disease areas simultaneously, enabling a diversified clinical pipeline from one technology investment.