Mirum's Breakthrough Liver Disease Treatments Show Promise at EASL 2026

Mirum Pharmaceuticals has unveiled new findings from its rare liver disease treatments at the European Association for the Study of the Liver (EASL) International Liver Congress 2026. The company presented late-breaking results from three key studies, including advancements in primary sclerosing cholangitis (PSC), chronic hepatitis delta virus (HDV), and progressive familial intrahepatic cholestasis (PFIC). In the Phase 2b VISTAS study, volixibat demonstrated a significant impact on patients with PSC. Those receiving the drug saw a mean reduction in serum bile acid (sBA) levels of 33.7 μmol/L, while the placebo group experienced a slight increase of 2.1 μmol/L. The treatment also led to a 2.72-point reduction in cholestatic pruritus, as measured by the Adult Itch Reported Outcome (ItchRO) scale—1.64 points better than placebo. Additionally, 56% of patients on volixibat achieved at least a 2-point reduction in itching, compared with 26% on placebo, and 37% saw a 3-point reduction versus 11% on placebo.

The Phase 2b AZURE-1 study of brelovitug in chronic HDV showed promising results at Week 24. Virologic response rates reached 45% in the 300 mg weekly arm and 35% in the 900 mg every-four-weeks arm, with no response in the delayed treatment group. Liver stiffness also decreased by 3.6 kPa and 2 kPa in the respective treatment arms. Data on LIVMARLI (maralixibat) in PFIC highlighted improved event-free survival (EFS) compared with real-world cases from the NAPPED database. The findings reinforce its role as an established therapy for this rare liver condition.

The presented data underscore the potential of Mirum’s treatments across multiple rare liver diseases. Volixibat’s reductions in bile acids and itching, brelovitug’s virologic and fibrosis improvements, and LIVMARLI’s survival benefits provide concrete evidence for further development. These results may shape future treatment options for patients with PSC, HDV, and PFIC.