Madrigal Pharmaceuticals is a clinical-stage biopharmaceutical company pursuing novel therapeutics that target a specific thyroid hormone pathway in the liver, which is a key regulatory mechanism common to a spectrum of cardio-metabolic and fatty liver diseases with high unmet medical need.
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. Seth Baum, M.D. AASLD 2019 Presentation