Elafibranor, an investigational therapy, demonstrated a favorable safety profile and dose-dependent efficacy over 12 weeks in people living with primary sclerosing cholangitis (PSC), a rare liver disease with no approved treatment options.

Patients treated with elafibranor showed significant improvements in liver biochemical markers, including alkaline phosphatase (ALP), compared with placebo. Stabilization of non-invasive markers of liver fibrosis was also observed, alongside a significant improvement in pruritus for those on the higher 120 mg dose.

The results come from the Phase 2 Elmwood trial, conducted by Ipsen, which will be presented at the European Association for the Study of the Liver (EASL) Congress on May 10.

PSC is a progressive liver disease that leads to bile duct scarring and liver failure, with liver transplantation currently the only effective intervention. No therapies have yet been approved by the FDA or EMA for the condition.

The 12-week study randomized 68 patients to receive either elafibranor 80 mg, 120 mg, or placebo. The primary endpoint assessed safety and tolerability. Treatment-emergent adverse events were similar across groups, and serious adverse events occurred only in the placebo group.

Efficacy findings showed dose-dependent reductions in ALP at week 12, with significant changes observed as early as week 4. Reductions in other liver enzymes, including alanine aminotransferase (ALT) and gamma-glutamyl transferase (GGT), were also reported.

Markers of liver fibrosis remained stable in patients treated with elafibranor compared with deterioration seen in the placebo group. In addition, patients receiving the 120 mg dose reported a meaningful improvement in pruritus, an important symptom for PSC patients.

Researchers said the findings support the further evaluation of elafibranor in larger and longer-term trials.