Mirum Pharmaceuticals, Inc. Presents New Data from its Phase 2b VANTAGE Study at European Association for Study of Liver Meeting in Amsterdam, Netherlands

Published: 2025-05-09 08:30:00 ET MIRM

Published on 05/09/2025 at 08:30, updated on 05/09/2025 at 09:50

Mirum Pharmaceuticals, Inc. presented new data from its Phase 2b VANTAGE study at the European Association for the Study of the Liver (EASL) meeting in Amsterdam, the Netherlands. The VANTAGE study is evaluating volixibat in patients with cholestatic pruritus caused by primary biliary cholangitis (PBC). Data from the interim analysis of VANTAGE were highlighted with new analyses through week 28.

Patients were randomized to receive volixibat 20mg BID, 80mg BID, or placebo BID. The primary endpoint of the study was mean change in weekly averaged daily itch score, as measured by the adult ItchRO scale, from baseline to Week 17-Week 28. Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT).

Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (PSC) (VISTAS study), and primary biliary cholang inflammation (PBC) (VANTAGE study). In June, Mirum announced positive interim results from the Phase 2b VANTAGE Study showing statistically significant improvement in pruritus as well as reductions in serum bile acids and improvements in fatigue for patients treated with volixibat.

No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate. Volixibat has been granted breakthrough therapy designation for the treatment of PBC. LIVMARLI®?

(maralixibat) is an orally administered, ileal bile acid transport (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases, in both liquid and tablet formulations. It is approved for the treatment of cholestatic prurus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic prURitus.

LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects. LIVMARLI is available in oral solution or tablet formulations and is taken by mouth 30 minutes before a meal.

For Alagille syndrome, LIVMARLI is taken one time each day in the morning. Mirum has initiated the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholESTatic pruritus. CHOLBAM is FDA-approved for the treatment of cholESTatic prURitus.

CHOLBAM are FDA-approved for the treatment to help alter the inflammatory condition present in PBC.