Becky Taub and Paul Friedman, are leading the efforts of Madrigal Pharmaceuticals to develop the first treatment for NAFLD/NASH. That drug is called Resmetirom and is currently in phase 3 trials. Many observers think they will be the first to have a successful drug for fatty liver disease.
Madrigal Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company pursuing novel therapeutics that target a specific thyroid hormone receptor pathway in the liver, which is a key regulatory mechanism common to a spectrum of fatty liver and cardio-metabolic diseases with high unmet medical need. Madrigal’s lead candidate, resmetirom, is a first-in-class, orally administered, small-molecule, liver-directed, thyroid hormone receptor (THR)-β selective agonist that is in currently in two Phase 3 clinical studies, MAESTRO-NASH and MAESTRO- NAFLD-1, designed to demonstrate multiple benefits in NASH (non-alcoholic steatohepatitis) patients.
Madrigal recognizes that compounds with greater selectivity for thyroid hormone receptor (THR)-β and liver uptake has the greatest potential to overcome challenges faced by prior, less selective compounds and deliver the full therapeutic potential of THR-β agonism. The Company believes that resmetirom, its lead product candidate, is the first orally administered, small-molecule, liver-directed, truly β-selective THR agonist.
Resmetirom has demonstrated the potential for a broad array of therapeutically beneficial effects, not only by improving the non-alcoholic steatohepatitis (NASH) phenotype through reducing liver fat and liver inflammation, and by restoring hepatocytes to normal function, but also by decreasing levels of multiple atherogenic lipids. These pleiotropic actions, coupled with an excellent safety profile, suggest that resmetirom could be the preferred option for treating NASH and for addressing the heightened cardiovascular risk in non-alcoholic fatty liver disease (NAFLD)/NASH patients.