Pemvidutide for Non-Alcoholic Fatty Liver Disease

(PERFORMA Trial)

This trial tests a new drug called pemvidutide for individuals with metabolic dysfunction-associated steatohepatitis (MASH), a type of non-alcoholic fatty liver disease. The researchers aim to evaluate the effectiveness and safety of pemvidutide compared to a placebo (a substance with no active drug). The trial includes two groups: one based on liver biopsy results and the other on non-invasive tests. Participants may qualify if they have a MASH diagnosis, type 2 diabetes or metabolic syndrome, and have not experienced significant weight changes recently. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Research has shown that pemvidutide is generally safe for people. In a study with patients, after 48 weeks of treatment, most tolerated the drug well, and only a few stopped due to side effects. In one group, no participants stopped because of side effects, while in another group, only 1% did. This indicates that most people can take pemvidutide without major issues. Earlier research also found that discontinuation due to side effects was rare, with only a few cases across different dose groups. Overall, these findings suggest that pemvidutide is safe for most people, with only minor side effects for some.¹²³⁴⁵

Pemvidutide is unique because it targets both the GLP-1 and glucagon receptors, which is different from many existing treatments for non-alcoholic fatty liver disease (NAFLD) that primarily focus on metabolic pathways or liver fat reduction. Researchers are excited about pemvidutide because this dual-action mechanism could potentially improve liver health and aid in weight loss simultaneously, addressing multiple aspects of NAFLD. Moreover, the option of different dosing (1.8 mg and 2.4 mg) allows for flexibility in treatment, which could tailor therapy to individual patient needs and enhance outcomes.

Research has shown that pemvidutide can significantly reduce liver fat and inflammation in individuals with metabolic dysfunction-associated steatohepatitis (MASH). One study revealed that patients taking 1.8 mg of pemvidutide experienced a 54.7% decrease in liver fat. Another study found that more than half of the patients taking the drug saw improvement in their MASH, compared to only 20% of those on a placebo. Pemvidutide also improved cholesterol levels, weight, and waist size. These findings suggest that pemvidutide might effectively treat liver issues related to MASH. Participants in this trial will receive either 1.8 mg or 2.4 mg of pemvidutide, or a placebo, to further evaluate its effectiveness.⁶⁷⁸⁹¹⁰