Phase 2 N=128 Randomized Quadruple-blind Treatment

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

NASH - Nonalcoholic Steatohepatitis

Bottom Line

View on ClinicalTrials.gov: NCT04767529 ↗

Enrolled (actual)

128

Serious AEs

11.9%

Results posted

May 2025

Primary outcome: Primary: Effect of Efruxifermin (EFX) vs Placebo on Fibrosis Regression in Participants With Metabolic Dysfunction-associated Steatohepatitis (MASH)-Associated Stage 2 or 3 Fibrosis (F2 or F3) — 8; 15; 14 Participants — p=0.025

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Efruxifermin (Drug); Placebo (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Akero Therapeutics, Inc
Primary completion: May 2024

Outcome Measures

Outcome	Result	p-value
PRIMARY Effect of Efruxifermin (EFX) vs Placebo on Fibrosis Regression in Participants With Metabolic Dysfunction-associated Steatohepatitis (MASH)-Associated Stage 2 or 3 Fibrosis (F2 or F3)	8; 15; 14	0.025 sig
SECONDARY Proportion of Subjects Who Achieve Improvement in Liver Fibrosis ≥ 1 Stage and no Worsening of Steatohepatitis at Week 96	8; 12; 21	0.070
SECONDARY Proportion of Subjects Who Achieve Resolution of Steatohepatitis and no Worsening of Liver Fibrosis at Week 24 and Week 96	6; 18; 26; 8; 16; 16	0.002 sig
SECONDARY Proportion of Subjects Who Achieve Improvement in Liver Fibrosis ≥ 1 Stage at Week 24 and Week 96	9; 15; 14; 9; 12; 21	0.053
SECONDARY Change From Baseline in Hepatic Fat Fraction in EFX vs Placebo Groups	-1.9; -8.8; -11.2; -2.5; -6.3; -8.3	<0.001 sig
SECONDARY Change From Baseline in Lipoproteins at Week 24 and Week 96	10.5; -41.1; -44.7; 16.1; -26.9; -31.7	<0.001 sig
SECONDARY Change From Baseline of Hemoglobin A1c (Glycated Hemoglobin, HbA1c) at Week 24 and Week 96	-0.0; -0.3; -0.4; -0.0; 0.0; 0.2	—
SECONDARY Change From Baseline in C-peptide at Week 24 and Week 96	0.0; -0.8; -1.2; -0.2; -0.4; -1.0	—
SECONDARY Change From Baseline in Adiponectin at Week 24 and Week 96	296.4; 1411.5; 3088.8; 531.5; 975.3; 2018.3	—
SECONDARY Change From Baseline in Homeostatic Model Assessment of Insulin Resistance (HOMA-IR) at Week 24 and Week 96	0.7; -4.7; -5.6; -1.3; -4.1; -5.5	—
SECONDARY Change From Baseline in Enhanced Liver Fibrosis (ELF) Score at Week 24 and Week 96	0.1; -0.6; -0.7; -0.1; -0.7; -0.8	—
SECONDARY Change From Baseline in N-terminal Type III Collagen Propeptide (Pro-C3) at Week 24 and Week 96	-2.7; -51.1; -53.9; -17.2; -40.2; -50.5	—
SECONDARY Change From Baseline in Collagen III Neo-Peptide (C3M) at Week 24 and Week 96	0.2; -0.8; -1.1; 3.1; 3.3; 4.4	—
SECONDARY Change From Baseline in NIS4 Score at Week 24 and Week 96	-0.0; -0.3; -0.3; -0.1; -0.2; -0.3	—
SECONDARY Change in Body Weight at Week 24 and Week 96	-0.6; -0.2; -3.0; -1.5; -0.3; -3.5	0.750
SECONDARY Change From Baseline in Liver Stiffness (kPa) at Week 24 and Week 96	-0.8; -2.9; -4.2; -0.6; -4.0; -7.2	0.102

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.

Eligibility Criteria

Inclusion Criteria

Males and non-pregnant, non-lactating females between 18 - 75 years of age inclusive, based on the date of the screening visit.
Previous history or presence of 2 out of 4 components of metabolic syndrome (obesity, dyslipidemia, elevated blood pressure, elevated fasting glucose) or type 2 diabetes.
FibroScan measurement > 8.5 kPa [kilopascal].
Biopsy-proven NASH. Must have had a liver biopsy obtained ≤ 180 days prior to randomization with fibrosis stage 2 to 3 and a non-alcoholic fatty liver disease (NAFLD) activity score (NAS) of ≥ 4 with at least a score of 1 in each of the following NAS components:
Steatosis (scored 0 to 3),
Ballooning degeneration (scored 0 to 2), and
Lobular inflammation (scored 0 to 3).

Exclusion Criteria

Weight loss > 5% in the 3 months prior to screening until randomization or from the time of the diagnostic liver biopsy until randomization, whichever is longer.
Presence of cirrhosis on liver biopsy (stage 4 fibrosis).
Type 1 or uncontrolled Type 2 diabetes.

Other inclusion and exclusion criteria may apply.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04767529). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.