The disease models are built with cells from patient donors, so they carry features of the disease itself. Drug developers can measure whether a candidate acts on those disease features in human tissue before it enters clinical trials. VivoSim offers efficacy testing alongside toxicology on its liver and gastrointestinal (GI) models.
The move builds on demonstrated disease model excellence already demonstrated. Its intestinal efficacy platform, built in part from cells of patients with ulcerative colitis or Crohn’s disease, received a "Poster of Distinction" award at Digestive Disease Week in May 2025, in work showing that the platform responds to clinically used therapeutics. VivoSim has also used its human disease models to identify new molecular targets that drive inflammatory bowel disease (IBD) and to explore how known drugs act.
Lack of efficacy is the most common reason drug candidates fail in clinical trials. In an analysis of more than 28,000 clinical trials stopped before reaching their endpoints, more than twice as many were stopped for negative results or lack of efficacy as for safety (Razuvayevskaya et al., Nature Genetics, 2024).
NAMkind™ Disease Models
- Liver (MASH): The NAMkind™ MASH disease model co-cultures healthy human liver cells with stellate, Kupffer, and endothelial cells from a donor with metabolic dysfunction-associated steatohepatitis (MASH), producing steatotic tissue with fibrosis. Compounds are dosed repeatedly over 14 or 21 days, with readouts for fibrosis and liver function. Cell-health markers measured in the same study separate an anti-fibrotic effect from cell damage.
- Intestine (IBD): The NAMkind™ IBD disease model is built from colon and small-intestine organoids derived from donors with ulcerative colitis and Crohn’s disease, with twelve donors currently available. It measures barrier integrity, inflammation, and fibrosis in the same well and reports results donor by donor. A healthy tissue comparator arm is available.
- Pharmacodynamic (PD) & Target Biomarker Profiling: The platform evaluates target engagement, gene expression panels, cytokine profiling, extracellular matrix turnover markers, and other relevant biomarkers across donor tissues, enabling sponsors to map mechanism-of-action and dose-response relationships alongside functional efficacy readouts.
In both models, each study runs the client’s compound alongside an approved or clinically validated reference compound in the same donor tissue, such as resmetirom in the MASH model or tofacitinib or upadacitinib in the IBD model. In results VivoSim has published for the IBD model, JAK inhibitors that look alike on paper had different effects on fibrosis, barrier function and inflammation, and methotrexate, a clinically used anti-inflammatory, reduced a fibrosis marker in a dose-dependent way in both Crohn’s patient models tested.