Organovo

Organovo is a clinical-stage biotechnology company focused on developing FXR314, a potent, selective, orally administered non-bile acid FXR agonist, as a novel therapeutic approach for inflammatory bowel diseases (IBD), including ulcerative colitis and Crohn's disease. The company's proprietary technology enables the creation of three-dimensional (3D) human tissue models that mimic key aspects of native human tissue composition, architecture, function, and disease.

FXR314 has demonstrated promising results in Organovo's 3D human IBD models, improving epithelial barrier function and reducing fibrotic activity. preclinical data highlights FXR314's potential to address the critical need for novel treatments that go beyond modulating the immune response targeted by current IBD therapies. In initial clinical trials, FXR314 exhibited high potency, safety, tolerability, and the potential to avoid dose-limiting toxicity issues seen in other FXR-targeted compounds.

Organovo has received FDA clinical trial authorization for a Phase 2 trial of FXR314 in ulcerative colitis, with plans to begin enrollment in 2024 and targeted completion in 2025. The company also aims to investigate FXR314 for other IBDs, including Crohn's disease, as well as liver diseases like non-alcoholic steatohepatitis (NASH) and primary biliary cholangitis. Additionally, Organovo is planning to initiate medicinal chemistry efforts in 2023 to create a novel drug for an undisclosed target validated in its 3D Crohn's disease models, with an expected Investigational New Drug (IND) filing by the end of 2025.

In January 2024, Organovo presented findings at the Crohn's and Colitis Congress, highlighting FXR314's ability to improve epithelial barrier function and reduce fibrotic markers in its 3D models of Crohn's disease and ulcerative colitis. The company's 3D human IBD models are seen as a powerful tool to accurately predict the relevance of specific targets and assess the efficacy of therapeutic candidates, increasing the probability of success in clinical trials.