A randomized, controlled trial of the pan-PPAR agonist lanifibranor in NASH

New England Journal of MedicineFrancque SM, Bedossa P, Ratziu V, et al. | October 25, 2021


In view of the benefit conferred by 1,200-mg dose of lanifibranor (a pan-peroxisome proliferator–activated receptor agonist) vs placebo in active nonalcoholic steatohepatitis (NASH) patients in this trial, further evaluation of lanifibranor in phase 3 trials is supported.

  • This phase 2b, double-blind, randomized, placebo-controlled trial comprised 247 patients with noncirrhotic, highly active NASH randomized to receive 1,200 mg or 800 mg of lanifibranor or placebo once daily for 24 weeks.

  • Treatment with 1,200-mg dose of lanifibranor vs placebo conferred reduction of at least 2 points in the SAF-A score (the activity part of the Steatosis, Activity, Fibrosis scoring system that includes scores for ballooning and inflammation), without worsening of fibrosis, in a significantly higher percentage of patients.

  • Compared with placebo, both the 1,200-mg and 800-mg doses of lanifibranor were superior for resolution of NASH without worsening of fibrosis, improvement in fibrosis stage of at least 1 without worsening of NASH, and resolution of NASH plus improvement in fibrosis stage of at least 1.

  • The lanifibranor groups exhibited reduction in liver enzyme levels and improvement in the levels of the majority of lipid, inflammatory, and fibrosis biomarkers.

  • For adverse events, the dropout rate was less than 5% and was similar across the trial groups.

  • Lanifibranor, vs placebo, was more frequently associated with diarrhea, nausea, peripheral edema, anemia, and weight gain.

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