Madrigal Pharmaceuticals Announces Publication of the Phase 3 MAESTRO-NASH Trial of Resmetirom in the New England Journal of Medicine - Seite 2

Becky Taub, M.D., Chief Medical Officer and President of Research & Development of Madrigal, stated, “Patients with NASH with significant fibrosis are at increased risk of progressing to cirrhosis, liver failure, liver cancer and premature death. Additionally, NASH is the leading cause of liver transplant among women in the U.S. and may soon be the leading cause overall. Despite its serious impact on patients and the health system, there are no approved treatments for the disease. As a liver-directed therapy that has demonstrated efficacy in both reversing fibrosis and resolving NASH in a pivotal Phase 3 clinical trial, we believe resmetirom will change the treatment paradigm for patients with NASH with significant fibrosis if it receives accelerated approval from the FDA.”

In addition to the two primary endpoints, multiple secondary endpoints were achieved in the MAESTRO-NASH study, including statistically significant reduction from baseline in liver enzymes (ALT, AST and GGT). Reductions in atherogenic lipids and lipoproteins, fibrosis biomarkers and imaging tests (MRI-PDFF, CAP and liver stiffness measures) were observed in resmetirom treatment arms as compared with placebo. MAESTRO-NASH also included many biomarker and imaging assessments that may be used in real world clinical practice to identify appropriate patients for treatment and monitor response to resmetirom, if approved.

The incidence of serious adverse events was similar across the treatment groups, 10.9%, 12.7%, and 11.5% in 80 mg, 100 mg and placebo groups respectively. Transient diarrhea and nausea were more frequent with resmetirom at the beginning of therapy. No increase in the incidence of diarrhea and nausea was noted among resmetirom-treated patients relative to placebo-treated patients after the first few weeks of treatment. There was no incidence of drug-induced liver injury. There were no increases in bone fractures, or fracture risk score with resmetirom or increase in adverse events related to thyroid hormone effects outside the liver such as heart rate changes or sex hormone abnormalities.

Bill Sibold, Chief Executive Officer of Madrigal, stated, “The unprecedented efficacy and safety results from the pivotal MAESTRO-NASH Phase 3 trial provide Madrigal with a unique opportunity to establish resmetirom as the foundational therapy for NASH with significant fibrosis and transform care for patients who currently have no approved treatment options. Resmetirom is the only investigational medication to achieve both fibrosis improvement and NASH resolution endpoints in Phase 3, and we intend to build on our leadership position in NASH drug development with two ongoing outcomes trials that carry the potential to confirm clinical benefit and expand the eligible patient population for resmetirom to include patients with more advanced disease.”