A Multiple Ascending Dose Study of Pegozafermin in Participants With Biopsy Confirmed Nonalcoholic Steatohepatitis (NASH) or Nonalcoholic Fatty Liver Disease (NAFLD) and at High Risk of NASH

Summary

Part 1: This is a multi-center evaluation of pegozafermin (administered weekly or every other week) in a randomized, double-blind, placebo-controlled study administered for 12 weeks in participants with NASH and NAFLD at high risk of NASH, including a pre-defined number of participants with biopsy confirmed NASH and fibrosis stages F1-F3 to be enrolled. Part 2: This is a multi-center, open label evaluation of pegozafermin at 27 mg administered weekly for 20 weeks in participants with biopsy-proven NASH (NAS ≥4, fibrosis stage F2 or F3).

Timeline

Start

2019-07-29

Primary completion

2020-08-28

Completion

2022-01-19

Outcome

Met primary endpoint

paper Twelve subjects (63%) met the primary endpoint of ≥2-point improvement in NAFLD activity score PMID 37718721 ↗

Publications

• Alkhouri N, Lazas D, Loomba R, Frias JP, Feng S, Tseng L, Balic K, Agollah GD, Kwan T, Iyer JS, Morrow L, Mansbach H, Margalit M, Harrison SA. Clinical trial: Effects of pegozafermin on the liver and on metabolic comorbidities in subjects with biopsy-confirmed nonalcoholic steatohepatitis. Aliment Pharmacol Ther. 2023 Nov;58(10):1005-1015. doi: 10.1111/apt.17709. Epub 2023 Sep 18.
• Tseng CL, Balic K, Charlton RW, Margalit M, Mansbach H, Savic RM. Population Pharmacokinetics and Pharmacodynamics of Pegozafermin in Patients with Nonalcoholic Steatohepatitis. Clin Pharmacol Ther. 2023 Dec;114(6):1323-1331. doi: 10.1002/cpt.3046. Epub 2023 Oct 2.
• Loomba R, Lawitz EJ, Frias JP, Ortiz-Lasanta G, Johansson L, Franey BB, Morrow L, Rosenstock M, Hartsfield CL, Chen CY, Tseng L, Charlton RW, Mansbach H, Margalit M. Safety, pharmacokinetics, and pharmacodynamics of pegozafermin in patients with non-alcoholic steatohepatitis: a randomised, double-blind, placebo-controlled, phase 1b/2a multiple-ascending-dose study. Lancet Gastroenterol Hepatol. 2023 Feb;8(2):120-132. doi: 10.1016/S2468-1253(22)00347-8. Epub 2022 Dec 12.

Drugs

Indications