The use of 50 mg Efruxifermin for 16 weeks in patients with compensated nonalcoholic steatohepatitis (NASH) cirrhosis was associated with promising improvements in indicators of hepatic damage, fibrosis, and lipid and glucose metabolism, as deciphered from a recent randomized phase IIa trial. The goal was to determine Efruxifermin's safety and tolerability in NASH people with compensated cirrhosis who are at elevated risk of developing decompensation and hepatic failure.

A total of 30 NASH patients with stage 4 fibrosis were segregated to get Efruxifermin 50 mg (n = 20) or placebo (n = 10) once weekly for 16 weeks. Tolerability and Safety profile of Efruxifermin were the main outcomes ascertained. In a sample of patients who agreed to a liver biopsy, secondary and exploratory objectives included noninvasive indicators of hepatic damage and fibrosis, lipid and glucose metabolism, and alterations in histology. Majority of adverse effects linked with Efruxifermin were grade 2 (n=19, 63.3%) or grade 1 (n=7, 23.3%), making it safe and well-tolerated.

The most typically noted adverse effects were gastrointestinal, including nausea and/or transient, mild to moderate diarrhea. Key indicators of liver damage (alanine transaminase [ALT]), as well as glucose and lipid metabolism, showed notable improvements. N-terminal type III collagen propeptide (Pro-C3) (LS mean alteration from baseline -9 μg/L Efruxifermin vs -3.4 μg/L placebo) and Enhanced Liver Fibrosis (ELF) Score (-0.4 Efruxifermin vs +0.4 placebo) were considerably reduced after a 16-week course of Efruxifermin, and there was also a trend towards decreased liver stiffness (LS mean alteration from baseline - 5.7 kPa Efruxifermin vs -1.1 kPa placebo).

Notably, 33% (4 out of 12) of Efruxifermin-treated people with liver biopsy following 16 weeks experienced an improvement in fibrosis of at least one stage without NASH deterioration. An additional 3 (25%) experienced NASH resolution in comparison to 0 of 5 placebo recipients.

Thus, Efruxifermin treatment shows promising therapeutic benefits and warrants additional research on Efruxifermin as a therapy for compensated cirrhosis caused by NASH in adequately-powered studies with longer durations.