Madrigal Pharmaceuticals (NASDAQ: MDGL) has announced promising results from its Phase 3 MAESTRO-NASH clinical trial of resmetirom, a potential treatment for nonalcoholic steatohepatitis (NASH).

The trial, which enrolled over 1,000 patients with biopsy-proven NASH, achieved its primary endpoints, showing liver histological improvement that the US Food and Drug Administration (FDA) considers likely to predict clinical benefit in NASH with liver fibrosis.

NASH is a serious liver disease characterized by the build-up of fat in the liver, leading to inflammation and scarring. If left untreated, it can progress to cirrhosis, a severe scarring of the liver, and can ultimately result in liver failure. There are currently no approved treatments for NASH, making the development of effective therapies a top priority.

The company says that In the MAESTRO-NASH trial, patients were given either resmetirom 80 mg, resmetirom 100 mg, or placebo once daily for 52 weeks. The trial found that both doses of resmetirom were safe and well-tolerated, with similar frequencies of serious adverse events across all treatment arms. The rate of study discontinuation due to adverse events was also low.

The primary efficacy analysis assessed histological response in 955 patients with biopsy-confirmed NASH and fibrosis after 52 weeks of treatment. The results showed that both doses of resmetirom significantly improved histological outcomes compared to placebo, with a higher percentage of patients achieving NASH resolution and a greater reduction in NAS scores.

In addition to the primary endpoints, the trial also evaluated several secondary endpoints, including liver stiffness, a measure of fibrosis. Both doses of resmetirom significantly reduced liver stiffness compared to placebo, indicating a potential improvement in fibrosis.

Madrigal is currently conducting four Phase 3 clinical trials of resmetirom for the treatment of NASH, including MAESTRO-NASH, MAESTRO-NAFLD-1, MAESTRO-NAFLD-OLE, and MAESTRO-NASH-OUTCOMES. The company plans to meet with the FDA to discuss the results of the MAESTRO-NASH trial and the potential for accelerated approval of resmetirom.

The company says that results of the MAESTRO-NASH trial are encouraging, as they suggest that resmetirom has the potential to be an effective treatment for NASH. If approved, it could provide a much-needed option for patients with this serious and progressive liver disease. It is worth noting that while the results are promising, the trial is still ongoing and more data will be needed to fully understand the safety and efficacy of resmetirom. Nevertheless, these initial results are a significant step forward in the search for effective treatments for NASH.

Becky Taub, M.D., Chief Medical Officer and President of Research & Development of Madrigal, stated, “These pivotal Phase 3 results demonstrate the potential for resmetirom to help patients achieve improvement in both the underlying steatohepatitis that drives this disease and the resulting fibrosis that is associated with progression to cirrhosis and its complications. The topline data also reinforce our confidence in the safety and tolerability profile of resmetirom. We believe the Phase 3 MAESTRO clinical development program, including the MAESTRO-NAFLD-1 and MAESTRO-NAFLD-OLE safety clinical trials and the recently initiated MAESTRO-NASH-OUTCOMES clinical trial, provides a strong foundation for our new drug application (NDA) and the potential accelerated approval of resmetirom for the treatment of non-cirrhotic NASH with liver fibrosis.”

Shares of Madrigal Pharmaceuticals trade on the NASDAQ under the ticker symbol MDGL. For more information visit www.madrigalpharma.com.