Benzinga | Jan 31, 2022 07:05AM EST

Madrigal Pharmaceuticals Announces Topline Phase 3 MAESTRO-NAFLD-1 Data Demonstrate Resmetirom Was Safe, Well-Tolerated And Provided Statistically Significant Improvements In Key Measures Of Liver And Cardiovascular Health

Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL), today announced positive topline clinical data from the placebo-controlled, double-blind portion of its Phase 3 MAESTRO-NAFLD-1 (non-alcoholic fatty liver disease) safety study of resmetirom. The 52-week study demonstrated that resmetirom was safe and well-tolerated at 80 and 100 mg once a day dosing. Additionally, resmetirom helped patients with presumed non-alcoholic steatohepatitis (NASH) achieve significant, clinically relevant reductions in liver fat and atherogenic lipids.

"These positive results from the first of our two Phase 3 MAESTRO trials support our conviction that resmetirom has the potential to be the first medication approved for the treatment of patients with NASH and liver fibrosis. The blinded, placebo-controlled data from MAESTRO-NAFLD-1 reinforce previous positive Phase 2 and open-label Phase 3 safety findings in a much larger population of patients followed for 56 weeks. Rigorous safety evaluation is critical in NASH drug development because of the large numbers of patients with NASH that could be treated with a new medication once FDA approved. The large safety database we are generating through the MAESTRO trials supports our regulatory strategy under Subpart H and reflects our commitment to providing the data necessary for overall benefit-risk assessment," stated Paul Friedman, M.D., Chief Executive Officer of Madrigal.

Becky Taub, M.D., Chief Medical Officer and President of Research & Development of Madrigal added, "These positive topline MAESTRO-NAFLD-1 placebo-controlled data are highly encouraging and increase our confidence in the pivotal serial liver biopsy Phase 3 trial, MAESTRO-NASH, that will deliver safety and efficacy data later this year. MAESTRO-NAFLD-1 was conducted during the height of the COVID-19 pandemic. COVID-related visits and blister pack manufacturing interruptions were observed in the study. Despite this, MRI-PDFF reductions were robust, with nearly half of patients in the resmetirom 100 mg arm achieving a 50% PDFF reduction. Importantly, COVID-related issues or patient withdrawals have not had a significant impact in the MAESTRO-NASH serial liver biopsy study."

Stephen Harrison, M.D., Medical Director for Pinnacle Clinical Research, San Antonio, Texas, Visiting Professor of Hepatology, Oxford University, and Principal Investigator of the MAESTRO studies commented, "This positive readout from MAESTRO-NAFLD-1 is a significant milestone for the field. As the first Phase 3 study in NASH that does not rely on liver biopsy to identify patients and measure treatment response, MAESTRO-NAFLD-1 will help accelerate the role of non-invasive imaging and biomarkers in NASH drug development. We see a safety and tolerability profile for resmetirom in this study of nearly one thousand patients treated for 52 weeks that, similar to earlier studies, leads to very low adverse event discontinuation rates. We look forward to presenting additional analyses of safety and efficacy data from MAESTRO-NAFLD-1 at future scientific congresses."