Organovo Presents FXR314 Phase 2 MASH Data at The Liver Meeting

SAN DIEGO, Nov. 20, 2024 -- Organovo Holdings, Inc. (Nasdaq:ONVO), a clinical-stage biotech firm concentrating on innovative treatments for inflammatory bowel disease (IBD) such as ulcerative colitis, announced the presentation of their leading clinical-stage drug FXR314. Dr. Eric Lawitz from the Texas Liver Institute and the University of Texas Health San Antonio delivered the presentation at The Liver Meeting, organized by the American Association for the Study of Liver Diseases (AASLD), held from November 15-19, 2024, in San Diego, California.

The presentation, titled “Pharmacokinetics, Safety and Efficacy of the Novel Non-bile Acid FXR Agonist FXR314 in Patients with Metabolic Dysfunction-Associated Steatohepatitis: Results from a Phase 2 Study,” took place on Sunday, November 17, during the MASLD and MASH – New therapies session. Dr. Lawitz outlined the comprehensive findings of a 16-week randomized, placebo-controlled, multi-center Phase 2 trial involving FXR314 for MASH patients. In this study, 214 participants were randomized equally into three groups: 3 mg FXR314, 6 mg FXR314, or placebo.

The outcomes indicated a notable decrease in liver fat content from the baseline in those treated with FXR314 compared to the placebo. The drug's safety profile showed significantly lower rates of pruritus than other FXR agonists. Specifically, patients on 3 mg FXR314 had an LS mean liver fat reduction of 22.8% (p=0.0010) and those on 6 mg saw a 17.5% decrease (p=0.0267), compared to a 6.1% reduction in the placebo group. Moreover, 29.2% of the subjects on 3 mg and 32.2% on 6 mg of FXR314 achieved over 30% reduction in MRI-PDFF, against 9.5% in the placebo group.

The study also showed improvements in liver function and hepatocellular damage without any signs of worsening liver fibrosis. FXR314 was well tolerated, with most treatment-emergent adverse events being mild to moderate. The frequency of drug-related treatment discontinuation was low and comparable across different groups. Common issues associated with FXR class drugs, such as pruritus (3 mg 2.8%, 6 mg 4.2%, and placebo 2.8%) and changes in LDL-C levels (1.5%, 4.5%, and -3.6% for the 3 mg, 6 mg, and placebo groups respectively), were not significantly noted.

“These results are promising, as FXR314 treatment led to liver fat reduction without the expected toxicities associated with this class of drugs,” noted Dr. Lawitz. “This unique profile makes further evaluation of FXR314 for MASH treatment very exciting. The specificity of its intestinal activation is particularly noteworthy.”

Organovo is a clinical-stage biotech company engaged in developing drugs that exhibit efficacy in three-dimensional (3D) human tissues, which are used as potential candidates for drug development. The company's flagship molecule, FXR314, is progressing toward Phase 2 trials for inflammatory bowel disease and has potential applications in metabolic liver disease and oncology. Organovo utilizes proprietary technology to create 3D human tissues that replicate essential aspects of native human tissue structure, function, and disease characteristics.