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Pegbelfermin (BMS‐986036), PEGylated FGF21, in Patients with Obesity and Type 2 Diabetes: Results from a Randomized Phase 2 Study
Author(s) -
Charles Edgar D.,
NeuschwanderTetri Brent A.,
Pablo Frias Juan,
Kundu Sudeep,
Luo Yi,
Tirucherai Giridhar S.,
Christian Rose
Publication year - 2019
Publication title -
obesity
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.438
H-Index - 199
eISSN - 1930-739X
pISSN - 1930-7381
DOI - 10.1002/oby.22344
Subject(s) - medicine , adiponectin , nonalcoholic fatty liver disease , tolerability , gastroenterology , type 2 diabetes , fgf21 , placebo , fatty liver , endocrinology , adverse effect , insulin resistance , diabetes mellitus , steatosis , type 2 diabetes mellitus , obesity , disease , pathology , receptor , alternative medicine , fibroblast growth factor
Objective Obesity and type 2 diabetes mellitus (T2DM) are risk factors for nonalcoholic fatty liver disease, including nonalcoholic steatohepatitis. This study assessed pegbelfermin (BMS‐986036), recombinant PEGylated human fibroblast growth factor 21 (FGF21), in patients with obesity and T2DM predisposed to fatty liver. Methods In this randomized, double‐blind, placebo‐controlled study, patients with T2DM and BMI of 30 to 50 kg/m 2 received subcutaneous pegbelfermin (1, 5, or 20 mg daily or 20 mg weekly; n  = 96) or placebo ( n  = 24) for 12 weeks. Primary end points were safety, tolerability, and change in HbA1c. Additional end points included insulin sensitivity, lipids, adiponectin, and disease progression biomarkers. Results There were no significant effects of pegbelfermin versus placebo on HbA1c. Pegbelfermin 20 mg/d significantly improved high‐density lipoprotein cholesterol ( P  = 0.015) and triglycerides ( P  = 0.037). All pegbelfermin regimens significantly increased adiponectin levels; 20‐mg daily and weekly regimens decreased serum PRO‐C3. Most adverse events were mild; the most frequent adverse events were injection‐site bruising and diarrhea. Conclusions Twelve‐week pegbelfermin treatment did not impact HbA1c concentrations, but QW and higher daily doses were associated with improved metabolic parameters and fibrosis biomarkers in patients with obesity and T2DM predisposed to fatty liver. These results support evaluation of pegbelfermin in patients with obesity‐related metabolic diseases (e.g., nonalcoholic steatohepatitis).

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