Phase 2 RCT | FGF21 analogue Pegozafermin treatment leads to improvement in NASH fibrosis

Summary: In a phase 2b, multicenter, double-blind, randomized, placebo-controlled trial, the fibroblast growth factor 21 (FGF21) analogue pegozafermin was examined for its efficacy and safety in treating patients with biopsy-confirmed noncirrhotic nonalcoholic steatohepatitis (NASH). A total of 222 patients with moderate or severe fibrosis (stage F2 or F3) were assigned to receive either subcutaneous pegozafermin at varying doses or a placebo. The primary endpoints were an improvement in fibrosis and resolution of NASH without worsening of fibrosis at 24 weeks.

The results showed that a larger percentage of patients receiving pegozafermin met the criteria for fibrosis improvement and NASH resolution compared to those receiving placebo. The most significant improvements were observed in the 44-mg pegozafermin group, with 27% showing fibrosis improvement and 26% meeting NASH resolution criteria, compared to 7% and 2% in the placebo group, respectively. Adverse events were mainly gastrointestinal and included nausea and diarrhea. These findings support the progression of pegozafermin into phase 3 development.

Article: Randomized, Controlled Trial of the FGF21 Analogue Pegozafermin in NASH – New England Journal of Medicine (link to abstract – $ for full-text)

Commentary on Twitter

Late breaking at #EASLCongress: In this phase 2b, placebo-controlled trial involving patients with nonalcoholic steatohepatitis, pegozafermin, a long-acting glycopegylated FGF21 analogue, reduced fibrosis at 24 weeks. Full results by @drloomba et al.: https://t.co/mfRRrV2yX2

— NEJM (@NEJM) June 24, 2023