Efruxifermin in Compensated Liver Cirrhosis Caused by MASH

Area of Science:

• Hepatology
• Pharmacology
• Clinical Trials

Background:

• Metabolic dysfunction-associated steatohepatitis (MASH) can lead to advanced liver fibrosis, including compensated cirrhosis (stage 4).
• Efruxifermin, a fibroblast growth factor 21 (FGF21) analogue, has shown promise in reducing fibrosis in earlier stages of MASH.
• Efficacy and safety data are needed for efruxifermin in patients with MASH-induced compensated cirrhosis.

Purpose of the Study:

• To evaluate the efficacy and safety of efruxifermin in patients with biopsy-confirmed compensated cirrhosis caused by MASH.
• To assess fibrosis reduction and MASH status at 36 and 96 weeks of treatment.

Main Methods:

• A phase 2b, randomized, placebo-controlled, double-blind trial was conducted.
• Patients with MASH and compensated cirrhosis received subcutaneous efruxifermin (28 mg or 50 mg daily) or placebo.
• The primary outcome was at least one stage of fibrosis reduction without MASH worsening at 36 weeks.

Main Results:

• At 36 weeks, no significant difference in fibrosis reduction was observed between efruxifermin groups and placebo.
• At 96 weeks, a higher proportion of patients in the 50 mg efruxifermin group achieved fibrosis reduction (29%) compared to placebo (11%).
• Gastrointestinal adverse events were more frequent with efruxifermin, mostly mild to moderate.

Conclusions:

• Efruxifermin did not demonstrate significant fibrosis reduction at 36 weeks in patients with MASH-related compensated cirrhosis.
• The 50 mg dose showed a potential benefit at 96 weeks, suggesting a possible longer-term therapeutic effect.
• Further research is needed to confirm the efficacy and safety profile of efruxifermin in this patient population.