Non-alcoholic steatohepatitis (NASH), the most common chronic liver disease in the United States, puts patients at increased risk of developing cirrhosis and hepatocellular carcinoma. Patients with NASH are also at an increased risk of experiencing cardiovascular events and overall mortality. Pegbelfermin (BMS-986036) is a PEGylated human fibroblast growth factor 21 (FGF21) that has been previously shown to improve metabolic markers, such as insulin sensitivity and serum adiponectin concentration in obese individuals with type 2 diabetes. This multicenter, randomized, double-blind phase 2a trial aimed to investigate the safety and efficacy of pegbelfermin. Patients between the ages of 21 and 75 with biopsy-proven NASH and a hepatic fat fraction of at least 10% (n=75) were randomized to receive 16 weeks of 10 mg of pegbelfermin once a day, 20 mg of pegbelfermin once a week, or placebo once a day. Researchers found that the absolute hepatic fat fraction was significantly decreased in the two groups receiving pegbelfermin as compared to placebo, with a mean change of -6.8% in the group receiving pegbelfermin daily versus the -1.3% mean change seen in the placebo group (p=0.0004). In the group receiving weekly pegbelfermin, a significant difference was also noted with a mean change of -5.2% in the intervention group compared to -1.3% in the placebo group (p=0.008). No deaths, treatment-related serious adverse events, or discontinuations due to adverse events were observed. Investigators therefore concluded that treatment with 16 weeks of subcutaneous pegbelfermin was well tolerated and resulted in a significant reduction in hepatic fat fraction in patients with NASH.
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