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 is the first in a series which navigates the reactions of eight panelists, including Michael Betel, Scott Friedman, Kris Kowdley, Mazen Noureddin, Rachel Zayas and co-surfers Jörn Schattenberg, Louise Campbell and Roger Green. As each recalls their various reactions to the news, the group addresses several points. Mazen describes his relief that we now have a Phase 3 trial that achieves efficacy and safety goals. Scott follows up with his anticipation of increased enthusiasm and energy for the entire set of stakeholders. Michael, the patient advocate representative among the group, reacts with a question: are we ready? Comments from Kris and Jörn drive one interesting outlook: this is the second positive Phase 3 trial in terms of efficacy. Such results might clear the field for a simpler approval process. Additionally, Jörn notes an approval would be in consideration in the EU where EMA requires both NAS improvement and regression of fibrosis.
From the entrepreneur’s perspective, Rachel recalls her decision a couple of years ago to pivot her company from hepatocellular carcinoma to NASH as what she calls a “bet on science.” This statement aligns with Roger’s persistent belief that this study will open the doors for more investment. Jörn and Mazen discuss some of the specific elements of patient screening and population that demonstrate the power of this study. As the session winds down, Roger shares an interesting observation: while the researchers have begun by talking about patients, in an earlier, separate conversation Donna Cryer began by talking about investors.