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A tiny biotech's experimental treatment for a silent disease that strikes millions just cleared a key hurdle and the stock is up more than 200%

Dec 20, 2022, 01:55 IST
Business Insider
Madrigal Pharmaceuticals is hoping to succeed where others have failed in treating NASH, a serious liver disease.Pramote Polyamate/Getty Images
  • Madrigal Pharmaceuticals reported positive results in its phase 3 trial for a drug to treat NASH.
  • As a result, its stock has increased by more than 200%.
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Madrigal Pharmaceuticals' stock jumped more than 200% on Monday after the biotech released positive results for its drug to treat NASH.

NASH, or nonalcoholic steatohepatitis, is a buildup of fat in the liver that is not caused by alcohol abuse. It can lead to liver scarring and liver failure. Many people don't even know they have it, which is why some people call it a "silent" disease.

Madrigal said in a news release Monday that its drug, called resmetirom, was safe and effective compared to the placebo group. In a trial of more than 950 patients, 26% of patients taking 80mg and 30% of patients taking 100mg of the drug showed that NASH activity like swelling had been reduced. The other main criteria was at least a one-stage improvement in liver damage, and both dosage groups met it with 24% and 26% of patients showing improvement.

The placebo groups showed a 10% and 14% improvement for these two main criteria.

"These unprecedented results from the MAESTRO-NASH clinical trial signal a major turning point for the field," Dr. Stephen Harrison, who led the studies, said in a news release.

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Madrigal is aiming to file for accelerated approval for resmetirom in the first half of 2023.

Pharma hasn't found a way to treat NASH yet

The pharma industry has been watching NASH for a while.

Intercept Pharmaceuticals is aiming to be the first to get FDA approval for its NASH drug but a faiure in a late-stage trial in September casts doubt on its ability to do so.

It's not alone in having issues on the path to approval—Gilead Science has also stumbled in recent years. Attempts by pharma giants like Bristol-Myers Squibb have put their efforts to develop treatments for NASH on the shelf.

There is also currently no approved treatment specifically for NASH. It is now the leading cause of liver transplants in patients 65 years old or older.

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