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Potential Liver Disease Treatment Identified in Trial

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Results from a new clinical trial have identified pegozafermin as a promising treatment for liver damage (fibrosis) related to non-alcoholic steatohepatitis (NASH). The research is published in the New England Journal of Medicine.

A potential new treatment for NASH

NASH is a type of non-alcoholic fatty liver disease (NAFLD). Approximately 24% of U.S. adults have NAFLD, according to the National Institutes of Health, while 1.5–6.5% have NASH.

NASH typically has very few symptoms, though it can lead to cirrhosis (severe liver scarring), liver cancer and even liver failure. Certain factors – such as type 2 diabetes, being overweight or having family members with the disease – can increase the risk of developing NASH, for which there are currently no FDA-approved therapies.

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In the current study, researchers carried out a clinical trial to assess the efficacy and safety of a new drug, pegozafermin, which mimics a hormone called fibroblast growth factor 12 (FGF21).

FGF21 is naturally produced by the liver. It has several roles, such as controlling energy use and lipid metabolism in the liver, and it has also been associated with reduced blood glucose and insulin levels as well as lowered body weight and liver fat.

“Identifying an effective drug for NASH is extremely promising for patients as currently there are no FDA-approved therapies for this condition,” said Dr. Rohit Loomba, lead author of the study and chief of the Division of Gastroenterology and Hepatology at UC San Diego School of Medicine. “NASH can adversely impact the quality of life in patients and can progress to cirrhosis. Its complications can lead to death or liver transplantation.”

Improvements in liver fibrosis

Researchers tested pegozafermin in a randomized, placebo-controlled, Phase 2b clinical trial (NCT04929483) of 219 NASH patients with moderate-to-severe liver fibrosis. The participants were assigned to different treatment groups and received varying regimens of pegozafermin injections. Patients received either 15 mg or 30 mg pegozafermin once weekly or 44 mg once every 2 weeks, while control groups received a placebo either weekly or every two weeks.

After 24 weeks, two treatment groups showed statistically significant improvements in liver fibrosis – 27% of patients in the 44 mg pegozafermin group and 26% in the 30 mg group showed improvements, compared to 7% in the pooled placebo groups.

Nausea and diarrhea were some of the most reported side effects and the highest number of side effects occurred in the 15 mg weekly group, in which 95% of patients reported adverse events. One severe adverse event considered related to the treatment was reported in a patient in the 44 mg pegozafermin group who developed acute pancreatitis.

“The study’s results show that the new potential treatment not only improves fibrosis but also improves inflammation and liver injury along with significant improvements across multiple non-invasive biomarkers of NASH activity and scarring,” said Loomba.

Study in further trials

The authors also state that future studies will assess the safety of the drug in more detail, including a larger, multi-center, international trial with a more diverse patient population over a longer treatment period.

“If successfully shown to be both safe and effective in a larger Phase 3 trial, this drug could be used to treat millions of patients with NASH, including our patients at UC San Diego Health,” Loomba added.

Reference: Loomba R, Sanyal AJ, Kowdley KV, et al. Randomized, controlled trial of the FGF21 analogue pegozafermin in NASH. N Engl J Med. 2023. doi: 10.1056/NEJMoa2304286

This article is a rework of a press release issued by the University of California, San Diego. Material has been edited for length and content.