Experimental fatty liver disease drug effective in clinical trial
Madrigal Pharmaceuticals’ experimental drug for fatty liver disease met both of the main goals in a highly anticipated late-stage study, it said on Monday, sending the company’s shares soaring over 200%.
The positive data sets the stage for the drug, resmetirom, to become the first approved treatment for NASH, or nonalcoholic steatohepatitis, a disease that affects about 5% of adults in the United States.
The data gives the company a timely boost after it raised “going concern” doubts in November citing lack of adequate cash.
Data from the trial shows a lower 80 milligram dose helped 26% of patients reach NASH resolution, compared to 30% for a higher 100 milligram dose, and 10% for placebo.
NASH is a form of nonalcoholic fatty liver disease, characterized by the organ developing fibrosis or scarring, which can progress to cirrhosis and liver failure.
The drug resmetirom also showed 1-stage improvement in fibrosis or scarring with no worsening of fatty liver disease in 24% of patients, compared to 26% for a higher 100 milligram dose, and 14% for placebo.
“With these unequivocally positive Phase 3 data in hand, our path forward is clear,” said Chief Executive Officer Paul Friedman.
The company plans to file a marketing application seeking accelerated approval for the drug in the first half of 2023.
Goldman Sachs analyst Andrea Tan has pegged peak global sales for the drug at $4.1 billion in 2033.
Madrigal’s shares more than tripled in premarket trading to $201.
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