Efruxifermin misses mark in MASH-related compensated cirrhosis

Treatment with the bivalent fibroblast growth factor 21 (FGF21) analogue efruxifermin falls short of improving fibrosis in patients with compensated cirrhosis caused by metabolic dysfunction–associated steatohepatitis (MASH), according to a phase 2b study.

The study included 181 patients with MASH who had biopsy-confirmed compensated cirrhosis (stage 4 fibrosis). These patients were randomly assigned to receive subcutaneous efruxifermin at a dose of 28 or 50 mg or placebo. Treatment was administered subcutaneously, once weekly.

The primary outcome was a reduction of at least one stage of fibrosis without worsening of MASH at week 36. The same criterion was evaluated at week 96 as the secondary outcome.

Of the patients, 154 underwent liver biopsy at week 36 and 134 at week 96. The number of patients who met the primary outcome did not significantly differ between the efruxifermin and placebo groups. At week 36, fibrosis reduction without worsening of MASH occurred in 18 percent of patients in the 28-mg efruxifermin group, 19 percent in the 50-mg efruxifermin group, and 13 percent in the placebo group (p=0.62 and p=0.52, respectively).

At week 96, 21 percent of patients in the 28-mg efruxifermin group, 29 percent in the 50-mg efruxifermin group, and 11 percent in the placebo group had a reduction in fibrosis without worsening of MASH.

In terms of safety, more patients on efruxifermin vs placebo experienced gastrointestinal adverse events, with most events being mild or moderate in severity.