In a monumental moment in the never-ending pursuit of putting a major dent in Fatty Liver disease, Madrigal Pharmaceuticals announced positive topline results from the pivotal Phase 3 MAESTRO-NASH clinical trial of resmetirom for the treatment of NASH and liver fibrosis. Madrigal’s leadership intends to file for resmetirom’s accelerated approval with the FDA in the first half of 2023. If approved, the drug may become the first NASH medication introduced to market, depending on the outcome and timing of Intercept Pharmaceuticals’ refiling of obeticholic acid.
To recognize this special occasion, Surfing the NASH Tsunami hosts its largest ever suite of panelists who react to the enthusiasm, synergy and implications surrounding the news. This conversation starts with Jörn Schattenberg investigating the impact that the use of a broad set of NITs in this study will have on NIT adoption and use in clinical patient treatment. Mazen Noureddin comments on how the use of NITs in clinical trials can reduce recruitment time and costs associated with the trials. Scott Friedman points out that as we strive to learn how to identify drug failures faster, FDA might require broad NIT use as part of that effort.
As the conversation continues, Rachel Zayas and Scott jump into more basic scientific issues. Rachel asks whether there is synergistic effect between LDL lowering and the primary endpoints and what that means for the disease. Scott asks what the results tell us on two fronts: first, what does it tell us about the ability of fat lowering to regress fibrosis if THR beta has no direct antifibrotic effect. Second, what about this drug converts fat lowering into fibrosis regression when other drugs that can lower fat and maintain lower fat levels do not demonstrate antifibrotic effects? After more comments about the science of the trial, Michael Betel asks how quickly focus will shift from monotherapy to combination therapy given how many patients did not succeed with resmetirom monotherapy. Mazen notes that other therapies were administered to patients in this trial. As the session ends, Louise Campbell asks questions about semaglutide design and Mazen discusses benefits and trial opportunities for this drug.