Efruxifermin for NASH with Cirrhosis

This trial explores whether a drug called efruxifermin can treat a liver condition known as NASH with cirrhosis. NASH, or non-alcoholic steatohepatitis, is a serious liver disease often linked to obesity and diabetes. Participants will receive either efruxifermin or a placebo (a look-alike with no active drug) to evaluate the treatment's effectiveness. This trial may suit those diagnosed with cirrhosis due to NASH or those with type 2 diabetes using specific diabetes medications. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of people.

The trial information does not specify if you need to stop taking your current medications. However, if you are in Cohort D, you must have been using a GLP-1R agonist (a type of diabetes medication) for at least 90 days before joining the study.

Research has shown that efruxifermin is generally safe and well-tolerated. One study found that participants experienced improvements in liver health indicators, suggesting the treatment might benefit the liver without causing significant harm.

Unlike the standard treatments for NASH with cirrhosis, such as lifestyle changes and medications like vitamin E and pioglitazone, Efruxifermin is unique because it targets the underlying causes of liver damage through a novel mechanism. Efruxifermin is a fusion protein designed to activate the fibroblast growth factor 21 (FGF21) pathway, which plays a crucial role in regulating metabolism and reducing liver fibrosis. Researchers are excited about Efruxifermin because it has the potential to not only halt disease progression but also reverse liver damage, offering hope for improved liver health and function in patients with NASH and cirrhosis.

Research has shown that efruxifermin (EFX), one of the treatments studied in this trial, may help treat non-alcoholic steatohepatitis (NASH) with liver scarring. In earlier studies, patients taking efruxifermin experienced improvements in liver health and scarring. These improvements suggest the drug might aid in liver healing and better function. Although efruxifermin did not meet some early goals in reducing scarring, its potential benefits for liver health remain promising. Overall, efruxifermin could offer hope for those with liver issues related to NASH. Participants in this trial may receive either efruxifermin or a placebo.¹³⁴⁶⁷