Insilico Doses First Patient in AI-Driven Phase 3 IPF Trial

New York, New York, September 10, 2026

GENESIS-IPF-3 Begins Global Phase 3 Evaluation

The GENESIS-IPF-3 study is a randomized, double-blind, placebo-controlled Phase 3 trial designed to evaluate the efficacy and safety of ISM001-055 in patients with idiopathic pulmonary fibrosis. The global registrational program follows earlier clinical studies that provided evidence supporting continued development of the investigational therapy. IPF is a chronic and progressive fibrotic lung disease characterized by irreversible scarring of lung tissue. As fibrosis advances, lung function can progressively decline, resulting in increasing difficulty breathing and reduced exercise capacity. Despite available treatments that can slow disease progression, there remains a need for therapies with differentiated mechanisms and improved tolerability. ISM001-055 is an orally administered small molecule designed to inhibit fibroblast activation protein (FAP), a target associated with fibrotic processes. Insilico’s approach is intended to interfere with biological pathways involved in fibroblast activation and the accumulation of extracellular matrix that contributes to progressive lung scarring. The initiation of the Phase 3 program represents a major clinical-development milestone because the study is intended to generate the definitive evidence needed for potential regulatory submissions.

Generative AI Supports Drug Discovery

ISM001-055 is notable not only for its therapeutic target but also for the way the molecule was discovered and developed. Insilico used its generative AI-powered Pharma.AI platform to identify and prioritize potential drug targets, design novel molecules and support preclinical and clinical development. The company has described ISM001-055 as a first-in-class small-molecule inhibitor generated through its AI-enabled drug-discovery process. The candidate was designed to address FAP biology and was subsequently advanced through preclinical research and human clinical studies. The development of an AI-designed molecule into a Phase 3 clinical trial provides an important real-world test of whether computational approaches can accelerate and improve pharmaceutical research. Generative AI has increasingly been adopted across the industry for applications such as target identification, molecular design, biomarker discovery and clinical-trial optimization. However, advancing an AI-designed candidate through late-stage clinical testing remains a significant challenge because therapeutic efficacy and safety ultimately must be demonstrated in patients. The GENESIS-IPF-3 program therefore represents both a drug-development milestone and a validation point for AI-enabled pharmaceutical research.

Late-Stage Trial Targets Fibrosis and Lung Function

Earlier clinical studies of ISM001-055 provided data supporting the candidate’s continued evaluation in IPF. The Phase 3 program is now designed to determine whether the investigational therapy can deliver clinically meaningful benefits while maintaining an acceptable safety profile. The global trial will evaluate key measures of lung function and disease progression, together with safety and tolerability outcomes. Longer-term data will be particularly important in IPF because the disease progresses over time and treatment effects need to be sustained to provide meaningful patient benefit. Insilico expects GENESIS-IPF-3 to provide the pivotal evidence required to determine whether ISM001-055 can advance toward regulatory submissions. The company is also continuing development of its broader pipeline of AI-discovered and AI-designed therapeutic candidates across fibrosis, oncology, immunology and other disease areas. The first-patient dosing milestone does not establish that ISM001-055 is effective or safe for routine clinical use. The candidate remains investigational, and its future regulatory status will depend on the results of the Phase 3 program and subsequent review by health authorities. Nevertheless, the launch of GENESIS-IPF-3 marks an important point in the evolution of AI-driven drug discovery. A molecule originating from generative AI-supported research has now progressed into a global registrational study, providing an opportunity to evaluate whether the technology can successfully translate computationally generated drug concepts into clinically meaningful therapies. For patients with idiopathic pulmonary fibrosis, the study also represents continued pharmaceutical investment in new approaches to a disease associated with substantial morbidity and limited treatment options. Results from GENESIS-IPF-3 will be closely watched as Insilico seeks to demonstrate whether ISM001-055 can become a new targeted therapy for progressive pulmonary fibrosis.

Source: Insilico Medicine press release