Madrigal Pharmaceuticals Inc. announced its first patient in a phase 2 clinical trial has been dosed with MGL-3196, a potential therapeutic for non-alcoholic steatohepatitis.
The multicenter study will comprise 117 adults with biopsy-proven NASH whom researchers will randomly assign MGL-3196 (Madrigal Pharmaceuticals) or placebo for 36 weeks, per a press release. They will measure efficacy at the end of 36 weeks via repeat MRI-based proton density fat fraction and conventional liver biopsy to examine histological evidence for the resolution of NASH.
“NASH is the leading liver disease in the United States with a growing prevalence for which no FDA approved treatment is yet available,” Stephen A. Harrison, MD, medical director for Pinnacle Clinical Research, San Antonio, Texas, said in the release. “We are hopeful that this novel approach, by targeting an underlying pathophysiologic mechanism of NASH, will eventually address a significant unmet need in this patient population.”
MGL-3196 is a liver-directed, thyroid hormone receptor (THR) beta-selective agonist drug that plays a role in controlling lipid metabolism, that can in turn affect levels of cholesterol and triglycerides and buildup of fat in the liver, per a press release.
“The lack of selectivity of older compounds limited their clinical utility, so we designed this study to provide clinical proof-of-concept for the role of highly-specific THR [beta] activation in regulating lipid metabolism in the liver to treat the cause of NASH,” Paul A. Friedman, MD, chairman and CEO of Madrigal, said in the release. “It will also allow us to confirm the value of assessing the early reduction of liver fat with imaging as a meaningful and predictive treatment endpoint.”
NASH is the fastest growing reason for liver transplants — affecting more than 15 million people — and is also associated with an increasing incidence of liver cancer, per the NIH.
Disclosures: Friedman is employed by Madrigal Pharmaceuticals. Healio.com/Hepatology was unable to confirm Harrison’s relevant financial disclosures at the time of publication.