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 Louise Campbell commenting on two specific benefits. First to mention is the fact that the multiple metabolic measurements “check a lot of boxes,” as she puts it, in terms of liver disease. Second, she notes there is great benefit for patients now that caregivers can tell them “if diet and exercise does not work, there is something else we can do.” Mazen Noureddin agrees, but notes that for F3 patients, drug therapy should start on the first or second visit. He also discusses the value of Hispanic patients and ways to discuss efficacy. Jörn Schattenberg asks whether the relatively similar results at two dose levels build or defeat the case for two dose levels. Kris Kowdley states confidently that having two dose levels is a benefit that these results demonstrate.
Louise asks how we can drill down into the patients that did not succeed, and Scott Friedman raises issues around payer response and precision therapy. He notes that it may also redefine use of placebo in clinical trials. Michael Betel asks what role bariatric therapies – notably surgery – might play in liver therapy and how we evaluate it. Through the rest of this session, Kris, Mazen and Roger discuss ways that the scope of data in this study and the rest of the Madrigal program will enrich scientific knowledge for years to come.