Early this week, Intercept Pharmaceuticals announced success from Phase 3 clinical trials for obeticholic acid (OCA). This is an experimental medicine designed to treat fatty liver disease known as nonalcoholic steatohepatitis (NASH). This means, of course, that we may be on our way to the first drug available to patients with NASH; and the drug company could be on their way to a big time commercial success.
Intercept President and Chief Executive Officer, Mark Pruzanski, MD comments, “We are thrilled to report the first positive registrational Phase 3 study results in patients with NASH, a devastating disease that is on track to become a leading cause of liver transplant in coming years.”
This Phase 3 clinical trial involved 933 patients who have been diagnosed with, at least, Stage 2 liver fibrosis (due to NASH), as medically confirmed form a liver biopsy. Within this population, all of the patients were randomized into three treatment groups for 18 months: one dose of OCA, two doses of OCA, or a placebo. The data shows that the highest dose improved liver fibrosis without worsening NASH at twice the rate of placebo: about 23% compared to 12%.
It should be noted that diet and exercise, together, can be very effective at reversing the early stage of fatty liver disease. Unfortunately, obesity rates in the US are on a quick rise and NASH prevalence is certainly following suit. Thus, it may be important to investigate medications like this to help patients who are not successful at stemming the condition in its early stages.
That in mind, Zobair M. Younossi, M.D., adds that “Patients with significant fibrosis due to NASH are at the greatest risk of progression to severe liver-related complications, such as liver failure and death, and fibrosis is considered the strongest predictor of liver-related mortality in this population.”
This highlights, of course, the importance of not only making the necessary lifestyle changes to reduce risk, but also addressing health factors early with effective treatments, like what we could find from OCA.
Younossi is a Professor and Chairman of the Department of Medicine at Inova Fairfax Medical Campus and at Virginia Commonwealth University (Inova Campus), as well as the Chair of the trial’s Steering Committee.
At the end of the day, Pruzanski goes on to say “The topline REGENERATE data we are reporting today support our belief that OCA will become the first approved medicine…a much-needed therapeutic option to address the enormous unmet medical need in this population.”