Volixibat - (VISTAS)

May 30, 2026: Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) reported new rare liver disease data presented at the EASL International Liver Congress 2026, including Phase 2b readouts for volixibat (PSC) and brelovitug (chronic HDV), plus an outcomes analysis for LIVMARLI (maralixibat) in PFIC.

Volixibat (Phase 2b VISTAS; primary sclerosing cholangitis pruritus): Met the primary endpoint on Adult ItchRO with a 2.72-point reduction from baseline and a 1.64-point placebo-adjusted reduction (p<0.0001). Responder rates favored volixibat (≥2-point: 56% vs 26%, p=0.0019; ≥3-point: 37% vs 11%, p=0.0011). Serum bile acids decreased (placebo-adjusted −35.8 µmol/L, p=0.0324). GI AEs and liver test elevations were more frequent vs placebo. Upcoming: pre-NDA meeting with FDA in summer 2026; planned NDA submission in 2H 2026.

Brelovitug (Phase 2b AZURE-1; chronic HDV): Week 24 virologic response rates were 100% (300 mg QW) and 75% (900 mg Q4W), with ALT normalization 45% and 40%, respectively; delayed-treatment arm was 0% for both. Composite endpoint achieved in 45% and 35% vs 0%. Phase 3 AZURE-1/AZURE-4 topline data expected 2H 2026; potential BLA and U.S. launch in 2027.

LIVMARLI (PFIC): Versus an external NAPPED cohort, maralixibat-treated patients (n=41) showed improved event-free survival (HR 0.29; p=0.0001) over up to 4.8 years, including reduced risk of surgical biliary diversion (HR 0.05; p=0.0036) and liver transplant/death (HR 0.44; p=0.02).