Organovo provides timing for release of FXR314 Phase 2 NASH results

Organovo Holdings will release final and complete data from a Phase 2a trial of FXR314 in non-alcoholic steatohepatitis patients by April 2024. The Company anticipates presentations at scientific meetings as well as publication in peer-reviewed journals. The release of this data will be the first public release of the completed clinical trial data. In an already reported interim analysis of about 60 patients after 16 weeks of treatment, FXR314 lowered liver fat content as demonstrated by a reduction in the median MRI-PDFF score of 28.6% in the 3 mg cohort and 26.9% in the 6 mg group compared with a reduction of only 1.5% in the placebo group. Post-hoc comparative assessment of relative liver fat reduction in the interim cohort found the decrease with 3 mg to be statistically significant compared to placebo. In a measure of activity in individual patients, FXR314 achieved greater than 30 percent liver fat reduction in 47 percent of patients in the 3 mg cohort and 35 percent in the 6 mg cohort, compared with 12 percent in the placebo arm. FXR314 was generally well-tolerated in this NASH population, with no treatment-related serious adverse events. All treatment-related AEs were mild-moderate with no apparent dose relationship. Mild-moderate pruritus was reported in one patient in the 3 mg cohort and one patient in the 6 mg cohort. No pruritus-related treatment discontinuations occurred. Organovo’s current development program for FXR314 focuses on inflammatory bowel disease, where the drug’s differentiated mechanism of action, as highlighted in Organovo’s recently provided mechanism of action video, provides substantial promise that the drug’s impact will strongly complement the biology of other successful drugs in ulcerative colitis and Crohn’s disease. Organovo plans to begin enrollment for a proof-of-concept Phase 2 ulcerative colitis study in 1H 2024, with targeted completion in 1H 2025. The drug’s additional promise in liver fibrosis and NASH makes it a strong candidate for development in that area through partnership collaborations with Organovo.