WASHINGTON – Fibroblast growth factor 21 (FGF21), a nonmitogenic hormone, improved fibrosis, liver injury, and steatosis in patients with nonalcoholic steatohepatitis (NASH), according to a study presented at the American Association for the Study of Liver Disease’s annual meeting.
There is no drug therapy currently available for NASH, the most advanced form of nonalcoholic fatty liver disease (NAFLD), creating a strong need for effective treatments, according to
This treatment “relative to placebo was associated with improvements in biomarkers of fibrosis, metabolic parameters, and markers of hepatic injury,” said Dr. Sanyal. “These results suggest BMS-986036 [FGF21] has beneficial effects on steatosis, liver injury, and fibrosis in NASH.”
Investigators conducted a phase 2 multicenter, double-blind, placebo-controlled study of 74 NASH patients to test BMS-986036, a pegylated version of FGF21.
Patients were an average of 51 years old, most were women (64%), who were predominantly white (96%), with a mean hepatic fat fraction of 19%.
Patients received either a 10-mg treatment daily, a 20-mg treatment weekly, or placebo, over the course of 16 weeks, with patients distributed equally among the three arms.
Overall hepatic fat fraction among the daily and weekly treatment groups reduced by 6.8% and 5.2%, respectively, compared with the placebo group, which reduced by 1.3% (P less than .001).
Patients in the treatment arms also saw improvement in average adiponectin levels, growing 15.3% in the daily arm and 15.7% in the weekly arm. Meanwhile, adiponectin levels dropped by an average of 3.5% in the placebo group.
In investigating serum Pro-C3 levels, which are associated with fibrosis, patients in the daily and weekly treatment group saw an average drop of 29% and 19%, respectively, as opposed to an increase of 2% in the placebo group (P less than .0001).
Patients in the treatment groups saw no serious adverse effects, and no patients died during the study.
Dr. Sanyal received funding for this study from Bristol-Myers Squibb and reported receiving financial compensation from Pfizer, Nimbus, Novartis, AstraZeneca, and other similar companies.