Organovo Unveils FXR314 and Tofacitinib Combo Data at DDW2024 for IBD

Organovo Holdings, Inc. (Nasdaq:ONVO), a clinical stage biotechnology company specializing in innovative treatments for inflammatory bowel disease (IBD) such as ulcerative colitis, has unveiled promising data from a poster presentation at Digestive Disease Week (DDW2024) in Washington, D.C. This data illustrates the potential effectiveness of the company's primary clinical-stage compound, FXR314, in combination with tofacitinib for treating IBD, employing both 3D models and preclinical studies.

The poster, titled “Combination therapy of the FXR agonist FXR314 with tofacitinib in models of inflammatory bowel disease,” was showcased on May 18, 2024, during the “Translational application of intestinal stem cells and organoid models” session.

Dr. Fabrice Piu, Vice President of Research and Development, commented on the findings, emphasizing the synergistic benefits of integrating Organovo’s non-steroidal, non-bile acid FXR agonist with the JAK inhibitor tofacitinib. This combination supports the use of FXR314 both as a standalone therapy and as part of a combination treatment in IBD. Crucial results demonstrated that the duo of FXR314 and tofacitinib improved various parameters related to intestinal barrier function, inflammation, and fibrosis at optimal concentrations. Moreover, when used together at sub-efficacious concentrations, the combination further enhanced these measures. Additionally, FXR314 alone showed improvements in colon health and histopathology on par with established IBD treatments such as tofacitinib and IL-12/23 antagonists.

Researchers arrived at these conclusions through two distinct methodologies. The first involved Organovo’s unique 3D IBD model, which co-cultures multiple cell types sourced from IBD patients suffering from either Crohn’s disease or ulcerative colitis. In this model, the team assessed target engagement, epithelial barrier integrity, inflammation, and fibrosis using a range of functional assays. The second approach employed the adoptive T-Cell transfer model, a recognized and predictive preclinical model of colitis, to evaluate the effects of combining FXR314 and tofacitinib within the treatment regime.

Keith Murphy, Organovo’s Executive Chairman, expressed enthusiasm about the results, confirming the company’s strong belief in FXR314’s potential in monotherapy for IBD. He noted these initial combination therapy results are particularly promising. Murphy highlighted that these findings not only reinforce the potential of FXR314 in combination therapies but also in monotherapy, providing valuable data that could influence future development and application. The results presented at Digestive Disease Week suggest that FXR314, when used alongside an already prescribed biologic, could enhance patient outcomes compared to the biologic alone.

Organovo is committed to advancing drugs effective in three-dimensional (3D) human tissues, which are considered promising candidates for drug development. FXR314, the company’s leading compound, is progressing towards Phase 2 clinical trials for IBD and may also have applications in metabolic liver disease and oncology. Organovo utilizes proprietary technology to create 3D human tissues that replicate critical aspects of native human tissue composition, structure, function, and disease.

In summary, Organovo’s recent findings presented at DDW2024 highlight significant advancements in the treatment of IBD, particularly through the combination of FXR314 and tofacitinib. These promising results pave the way for future clinical studies and potential new treatment paradigms for IBD patients.