Pegozafermin: This Novel Treatment Could Improve Liver Health in NASH

An estimated 25% of people in the United States have a condition called nonalcoholic fatty liver disease (NAFLD), a condition characterized by excess fat in the liver. Estimates vary but anywhere from 6-20% of people with NAFLD will develop nonalcoholic steatohepatitis (NASH). NASH, which sits under the larger umbrella of NAFLD, is characterized by liver fibrosis (scarring), inflammation, and damage. People with NASH may experience appetite and weight loss, fatigue, nausea and vomiting, yellowing of their skin and eyes, abdominal pain, and general weakness. 

Avoiding alcohol, controlling diabetes, losing weight, and practicing healthy lifestyle choices with diet and exercise are all standards-of-care. Despite the need for effective therapies, there are no FDA-approved treatments for NASH. Discovering new treatments could improve patient health and quality-of-life.

That is just what researchers from the University of California – San Diego’s School of Medicine are looking to do. According to an article in Medical XPress, the research team explored pegozafermin as a NASH treatment within a Phase 2b, multicenter, double-blinded, placebo-controlled clinical study. 

What is Pegozafermin?

Developed by 89bio, pegozafermin is a fibroblast growth factor 21 (FGF21) analog engineered using proprietary glycoPEGylation technology. FGF21 is naturally produced by our body, excreted by the liver, and plays a role in liver lipid (fat) metabolism, blood glucose and insulin regulation, and energy control. Pegozafermin works to not just sustain naturally produced FGF21, but to improve its efficacy in breaking down lipids. 

Improving NASH Treatment

Within the Phase 2b study, the research team explored the safety and efficacy of pegozafermin. Altogether, 222 people with NASH enrolled. Participants received either 15mg or 3mg subcutaneous pegozafermin weekly, 44mg pegozafermin bi-weekly, or a placebo. Primary endpoints for the study included improvements in fibrosis without disease progression and NASH resolution with no worsening scarring. 

Data published in The New England Journal of Medicine shared that the higher doses of pegozafermin were effective. 27% of these individuals saw some reduction in scarring, which was 20% higher than those receiving the placebo. The drug also reduced liver inflammation. There were side effects, but these were typically mild. 

Moving forward, researchers hope that a confirmatory Phase 3 trial could further highlight the benefits of pegozafermin and get a treatment option into the hands of patients in need.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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