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

• Resmetirom is the first investigational medicine for NASH to achieve fibrosis improvement and NASH resolution primary endpoints in a Phase 3 trial
• Detailed analyses reinforce the safety profile of resmetirom
• Resmetirom has the potential to become the first and only medicine approved for NASH; PDUFA date is March 14, 2024
  

CONSHOHOCKEN, Pa., Feb. 08, 2024 (GLOBE NEWSWIRE) -- Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL), a clinical-stage biopharmaceutical company pursuing novel therapeutics for nonalcoholic steatohepatitis (NASH), today announced the publication of the pivotal Phase 3 MAESTRO-NASH trial of resmetirom in the New England Journal of Medicine.

NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally. Resmetirom received Breakthrough Therapy designation from the FDA and is under review to become the first medicine approved to treat patients with NASH with liver fibrosis. The FDA granted resmetirom Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) date of March 14, 2024, the target date by which FDA intends to complete its review.

Stephen Harrison, M.D., Chairman for both Pinnacle Clinical Research and Summit Clinical Research, San Antonio, Texas, Visiting Professor of Hepatology, Oxford University, and lead Principal Investigator of the MAESTRO studies, commented, “MAESTRO-NASH is a landmark study in a disease that has historically been very challenging for drug development. The publication of detailed efficacy and safety data in the New England Journal of Medicine will provide clinicians with valuable information about the medication that may soon become the first approved therapy for patients with NASH.”

The MAESTRO-NASH trial evaluates resmetirom treatment vs. placebo in patients with NASH with significant fibrosis (consistent with fibrosis stages 2 and 3), a population at elevated risk of progressing to cirrhosis and other adverse liver outcomes. The study includes a 52-week biopsy assessment to support accelerated approval and an ongoing 54-month outcomes study designed to generate confirmatory data that, if positive, will help verify resmetirom’s clinical benefit and support full approval.

Based on the results of the 52-week biopsy portion of the trial, MAESTRO-NASH is the only Phase 3 study in NASH to achieve both primary endpoints that FDA proposed as reasonably likely to predict clinical benefit: NASH resolution with no worsening of fibrosis and fibrosis reduction with no worsening of NAFLD activity score (NAS). Approximately 50% of patients treated with resmetirom 100 mg with biopsies at Week 52 showed either NASH resolution or fibrosis improvement. More than 80% of patients with biopsies at Week 52 had either fibrosis reversal or no progression of fibrosis.