A Study of Efruxifermin in Non-Cirrhotic 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 in non-cirrhotic subjects with biopsy-proven F2 - F3 NASH.

Timeline

Start

2021-02-16

Primary completion

2024-05-02

Completion

2024-05-02

Outcome

Met primary endpoint

registry Placebo vs Efruxifermin 28 mg; p = 0.025; percent difference 22.6 (95% CI 3.8 to 41.4) NCT04767529 ↗

registry Placebo vs Efruxifermin 50 mg; p = 0.036; percent difference 22.5 (95% CI 1.6 to 43.3) NCT04767529 ↗

Publications

• Background Harrison SA, Frias JP, Neff G, Abrams GA, Lucas KJ, Sanchez W, Gogia S, Sheikh MY, Behling C, Bedossa P, Shao L, Chan D, Fong E, de Temple B, Shringarpure R, Tillman EJ, Rolph T, Cheng A, Yale K; HARMONY Study Group. Safety and efficacy of once-weekly efruxifermin versus placebo in non-alcoholic steatohepatitis (HARMONY): a multicentre, randomised, double-blind, placebo-controlled, phase 2b trial. Lancet Gastroenterol Hepatol. 2023 Dec;8(12):1080-1093. doi: 10.1016/S2468-1253(23)00272-8. Epub 2023 Oct 3.
• Noureddin M, Frias JP, Neff GW, Lucas KJ, Behling C, Bedossa P, Dubourg J, Chan D, Burch M, Fong E, de Temple B, Minerva M, Barrett K, Shringarpure R, Tillman EJ, Rolph T, Cheng A, Yale K. Safety and efficacy of once-weekly efruxifermin versus placebo in metabolic dysfunction-associated steatohepatitis (HARMONY): 96-week results from a multicentre, randomised, double-blind, placebo-controlled, phase 2b trial. Lancet. 2025 Aug 16;406(10504):719-730. doi: 10.1016/S0140-6736(25)01073-6.

Drugs

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

Indications