Efruxifermin for compensated cirrhosis due to MASH
phase 3drug program · high · Wed Sep 04 2024 00:00:00 GMT+0000 (Coordinated Universal Time)
Treat compensated cirrhosis caused by NASH/MASH and improve fibrosis-related liver pathology.
Randomized, double-blind, placebo-controlled clinical evaluation of efruxifermin in subjects with compensated cirrhosis due to NASH/MASH, including the SYMMETRY phase 2b trial and subsequent phase 3 study.
A phase 3 compensated cirrhosis study was published on ClinicalTrials.gov in 2024.
SYMMETRY-related 96-week analyses reported that efruxifermin improved fibrosis and reduced fibrosis and septa area by quantitative digital pathology in participants with compensated cirrhosis due to MASH.
Efruxifermin for MASH
phase 3drug program · high · Fri Dec 01 2023 00:00:00 GMT+0000 (Coordinated Universal Time)
Develop a once-weekly FGF21 analogue treatment for metabolic dysfunction-associated steatohepatitis, including fibrosis improvement and MASH resolution.
Subcutaneous efruxifermin, a bivalent fibroblast growth factor 21 analogue, evaluated in randomized, double-blind, placebo-controlled clinical trials and phase 3 studies.
Phase 3 trials are evaluating efruxifermin in non-cirrhotic MASH with F2/F3 fibrosis and in broader non-invasively diagnosed MASLD/MASH populations.
In the HARMONY phase 2b trial, 96-week data showed greater fibrosis improvement with efruxifermin than placebo, with no reported drug-induced liver injury or deaths; gastrointestinal adverse events were more common with efruxifermin.
SYNCHRONY Real-World Trial
phase 3drug program · high · Fri Nov 10 2023 00:00:00 GMT+0000 (Coordinated Universal Time)
Assess safety and tolerability of efruxifermin compared with placebo in subjects with non-invasively diagnosed NASH/MASH and NAFLD/MASLD.
Phase 3 randomized placebo-controlled real-world trial design using non-invasive diagnosis in MASLD or MASH participants.
Study design, rationale, and baseline characteristics were presented in 2026.
HARMONY phase 2b efruxifermin study
phase 2drug program · high · Tue Feb 16 2021 00:00:00 GMT+0000 (Coordinated Universal Time)
Evaluate efruxifermin in non-cirrhotic subjects with biopsy-confirmed NASH/MASH and F2-F3 fibrosis.
Multicenter, randomized, double-blind, placebo-controlled phase 2b trial of once-weekly subcutaneous efruxifermin versus placebo.
96-week HARMONY results were reported in 2025, with additional AI-powered histology and genotype analyses reported in 2025-2026.
At week 96, efruxifermin produced greater fibrosis improvement than placebo, especially at 50 mg; publications also report consistent efficacy across PNPLA3 genotypes and histology changes in liver microarchitecture.
BALANCED phase 2a efruxifermin study
phase 2drug program · medium · Tue May 28 2019 00:00:00 GMT+0000 (Coordinated Universal Time)
Evaluate efruxifermin in subjects with biopsy-proven F1-F4 NASH over 16 weeks.
Multicenter, randomized, double-blind, placebo-controlled clinical study of efruxifermin administered for 16 weeks.
ClinicalTrials.gov record published for a 16-week biopsy-proven NASH study.
Efruxifermin digital pathology analyses
exploratorybiomarker · high
Use digital pathology and noninvasive fibrosis measures to characterize efruxifermin-driven changes in liver fibrosis, septa area, and liver microarchitecture in MASH trials.
AI-powered histology analysis and quantitative digital pathology applied to HARMONY and SYMMETRY phase 2b trial samples.
2026 analysis reported digital pathology and noninvasive fibrosis measures across HARMONY and SYMMETRY phase 2b trials.
Analyses reported improvements in digital pathology and noninvasive fibrosis measures, including reduced fibrosis and septa area in compensated cirrhosis and liver microarchitecture changes in F2/F3 MASH.
FGF21 receptor agonism mechanism research
exploratoryresearch program · medium
Investigate how FGF21 receptor agonism affects steatosis, free fatty acid handling, mitochondrial oxidation, and mitochondrial reactive oxygen species.
Mechanistic research examining FGF21 receptor agonism and its effects on directing free fatty acids to mitochondria and increasing oxidation without raising mitochondrial reactive oxygen species.
2026 EASL presentation on FGF21 receptor agonism reversing steatosis.
The supplied title reports that FGF21 receptor agonism reverses steatosis by directing free fatty acids to mitochondria and increasing their oxidation without raising mitochondrial reactive oxygen species.
