CONSHOHOCKEN, PA — Madrigal Pharmaceuticals (NASDAQ: MDGL) recently took a significant stride towards introducing the first treatment for Nonalcoholic Steatohepatitis (NASH) with liver fibrosis. The drug, resmetirom, has cleared a pivotal Phase 3 trial, MAESTRO-NASH, and its findings were recently published in the prestigious New England Journal of Medicine.
NASH is a critical contributor to liver-related mortality and is poised to burden healthcare systems worldwide. Resmetirom has received Breakthrough Therapy designation from the FDA and could be the first approved treatment for patients with NASH with liver fibrosis.
Dr. Stephen Harrison, the lead Principal Investigator of the MAESTRO studies, hailed MAESTRO-NASH as a groundbreaking study in a challenging field. He said the publication of detailed efficacy and safety data will provide clinicians with crucial information about the potential therapy.
The MAESTRO-NASH trial assessed resmetirom treatment against a placebo in NASH patients with significant fibrosis, a group facing heightened risk of progressing to cirrhosis and other adverse liver outcomes. A 52-week biopsy assessment was included to support accelerated approval and an ongoing 54-month outcomes evaluation designed to generate confirmatory data that, if positive, will affirm resmetirom’s clinical benefit and bolster full approval.
The trial exhibited outstanding results, achieving both primary endpoints proposed by the FDA as predictive of clinical benefit: NASH resolution without worsening fibrosis and fibrosis reduction without NAS worsening.
Madrigal’s Chief Medical Officer, Dr. Becky Taub, emphasized that patients with NASH with significant fibrosis are at an increased risk of progressing to cirrhosis, liver failure, liver cancer and premature death. As a liver-directed therapy shown to reverse fibrosis and resolve NASH in a critical Phase 3 trial, Taub believes resmetirom could usher in a new era of treatment for patients with NASH if it secures faster approval from the FDA.
Besides the two primary goals, the MAESTRO-NASH study also achieved multiple secondary endpoints, including a significant reduction from baseline in liver enzymes. Reductions in fibrosis biomarkers, atherogenic lipids and lipoproteins, and imaging tests were also noted in the resmetirom treatment groups compared with the placebo.
The incidence of serious adverse events across all treatment groups was similar, with transient diarrhea and nausea occurring more often with resmetirom at the therapy’s onset. However, no incidence of drug-induced liver injury, increases in bone fractures, fracture risk score with resmetirom, or adverse events related to thyroid hormone effects outside the liver were reported.
Madrigl’s CEO, Bill Sibold, said the unprecedented efficacy and safety results from the pivotal MAESTRO-NASH Phase 3 trial provide Madrigal with an extraordinary chance to establish resmetirom as the primary therapy for NASH with significant fibrosis, revolutionizing care for patients who currently have no approved treatment options.
The findings from this trial could enable the introduction of the first-ever approved treatment for NASH, vastly improving the treatment options for patients and potentially altering the future of liver disease management.
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