Madrigal Pharmaceuticals (MDGL) Surges On Positive Results From Highly-Anticipated Clinical Trial
2:30 pm Monday, December 19th, 2022
Madrigal Pharma (MDGL) is surging today after this clinical-stage biopharma announced some big news regarding its flagship drug in development. Madrigal announced positive topline results from its pivotal Phase 3 MAESTRO-NASH biopsy clinical trial of resmetirom, a liver-directed selective thyroid hormone receptor agonist.
The trial achieved both liver histological improvement endpoints that the FDA had proposed as being reasonably likely to predict clinical benefit to support accelerated approval for the treatment NASH with liver fibrosis. The company says these results demonstrate the potential for resmetirom to improve treatment for both the underlying steatohepatitis that drives this disease and the resulting fibrosis that is associated with progression to cirrhosis and its complications.
Resmetirom was also shown to be safe and well-tolerated at both the 80 mg and 100 mg doses. Madrigal now intends to file a NDA seeking accelerated approval of resmetirom for the treatment of non-cirrhotic NASH with liver fibrosis. This disease puts patients at risk of progressing to liver failure, liver cancer, need for liver transplant, and premature mortality.
Just to understand the potential numbers here. Nonalcoholic steatohepatitis (NASH) is a more advanced form of nonalcoholic fatty liver disease (NAFLD). According to the company, NAFLD afflicts more than 20% of adults globally, about 30% in the US. Of that population, 20% may have NASH. Once NASH progresses to significant liver fibrosis (stages F2 and F3) the risk increases dramatically. NASH is rapidly becoming the leading cause of liver transplantation in the US.
So why is the stock up so much? Because this appears to be good news all the way around. The trial achieved both liver histological improvement endpoints. This easily could have been a mixed result rather than a clean result on efficacy, which this appears to be. Also, it was important that it was shown to be safe and well-tolerated. Another factor here is that this trial was based on actual liver biopsies, which should aid Madrigal’s quest to get accelerated approval. There are currently no FDA-approved therapies for the treatment of NASH, so this is a big deal.
