This is a multi-center evaluation of efruxifermin (EFX) in a randomized, double-blind, placebo-controlled study in subjects with non-cirrhotic NASH/MASH and fibrosis stage 2 or 3 (F2 or F3).
The study will enroll subjects in two cohorts for a total samples size of 1650 subjects.
Males and non-pregnant, non-lactating females between 18 - 80 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.
Steatosis (scored 0 to 3),
Ballooning degeneration (scored 0 to 2), and
Other causes of liver disease based on medical history and/or liver histology and/or central laboratory results.
Presence of cirrhosis on liver biopsy (fibrosis stage 4).
Type 1 or uncontrolled Type 2 diabetes.
Parallel
Efruxifermin
DrugAdministered by subcutaneous injection
Placebo
DrugAdministered by subcutaneous injection
Cohort 1 Only: Resolution of NASH/MASH and a ≥ 1 stage improvement in fibrosis
Based on NAS (scored by 0-3 for steatosis, 0-3 for inflammation, and 0-2 for ballooning) and NASH CRN fibrosis score (scored by a fibrosis score of 0-4, where 0 = no fibrosis, 1 = perisinusoidal or periportal fibrosis, 2 = perisinusoidal and portal/periportal fibrosis, 3 = bridging fibrosis, 4 = cirrhosis)
Event-free survival
Based on time from randomization to the first clinical event including evidence of disease progression, liver decompensation events, liver transplantation or eligibility for liver transplantation, and all-cause mortality.
Cohort 1 Only: Resolution of NASH/MASH and no worsening of fibrosis
Based on NAS (scored by 0-3 for steatosis, 0-3 for inflammation, and 0-2 for ballooning) and NASH CRN fibrosis score (scored by a fibrosis score of 0-4, where 0 = no fibrosis, 1 = perisinusoidal or periportal fibrosis, 2 = perisinusoidal and portal/periportal fibrosis, 3 = bridging fibrosis, 4 = cirrhosis)
Cohort 1 Only: ≥ 1 stage improvement in fibrosis and no worsening of steatohepatitis
Based on NAS (scored by 0-3 for steatosis, 0-3 for inflammation, and 0-2 for ballooning) and NASH CRN fibrosis score (scored by a fibrosis score of 0-4, where 0 = no fibrosis, 1 = perisinusoidal or periportal fibrosis, 2 = perisinusoidal and portal/periportal fibrosis, 3 = bridging fibrosis, 4 = cirrhosis)
Change from baseline of non-invasive markers of liver fibrosis: ELF score
The ELF score measures the severity of liver fibrosis and estimates the risk of disease progression to cirrhosis or liver-related events, with higher scores indicating higher liver fibrosis level and higher risk of disease progression.
Change from baseline of non-invasive markers of liver fibrosis: ELF score components (TIMP-1, HA, PIIINP, Pro-C3)
Tissue inhibitor of metalloproteinase-1 \[TIMP-1\], hyaluronic acid \[HA\], amino terminal pro-peptide of type 3 procollagen \[PIIINP\]), and propeptide of type 3 procollagen (Pro-C3)
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