A Study of Efruxifermin in Subjects With Histologically Confirmed Nonalcoholic Steatohepatitis (NASH)

Summary

This is a multi-center evaluation of efruxifermin (EFX) in a randomized, double-blind, placebo-controlled study administered for 16 weeks in subjects with biopsy proven F1 - F4 NASH.

Timeline

Start

2019-05-28

Primary completion

2021-02-10

Completion

2022-01-10

Outcome

Met primary endpoint

paper The primary endpoint-absolute change from baseline in HFF measured as magnetic resonance imaging-proton density fat fraction at week 12-was met. PMID 34239138 ↗

paper with statistically significant differences between efruxifermin groups and placebo (P < 0.0001 each). PMID 34239138 ↗

Publications

• Alkhouri N, Beyer C, Shumbayawonda E, Andersson A, Yale K, Rolph T, Chung RT, Vuppalanchi R, Cusi K, Loomba R, Pansini M, Dennis A. Decreases in cT1 and liver fat content reflect treatment-induced histological improvements in MASH. J Hepatol. 2025 Mar;82(3):438-445. doi: 10.1016/j.jhep.2024.08.031. Epub 2024 Sep 25.
• Harrison SA, Ruane PJ, Freilich B, Neff G, Patil R, Behling C, Hu C, Shringarpure R, de Temple B, Fong E, Tillman EJ, Rolph T, Cheng A, Yale K. A randomized, double-blind, placebo-controlled phase IIa trial of efruxifermin for patients with compensated NASH cirrhosis. JHEP Rep. 2022 Aug 23;5(1):100563. doi: 10.1016/j.jhepr.2022.100563. eCollection 2023 Jan.
• Harrison SA, Ruane PJ, Freilich BL, Neff G, Patil R, Behling CA, Hu C, Fong E, de Temple B, Tillman EJ, Rolph TP, Cheng A, Yale K. Efruxifermin in non-alcoholic steatohepatitis: a randomized, double-blind, placebo-controlled, phase 2a trial. Nat Med. 2021 Jul;27(7):1262-1271. doi: 10.1038/s41591-021-01425-3. Epub 2021 Jul 8.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Efruxifermin	Protein / enzyme biologic	28 mg	Subcutaneous
Subject	Efruxifermin	Protein / enzyme biologic	50 mg	Subcutaneous
Subject	Efruxifermin	Protein / enzyme biologic	70 mg	Subcutaneous

Indications