Mirum Pharmaceuticals to Host Investor Call on Volixibat Study Results June 17, 2024

Mirum Pharmaceuticals, Inc., based in Foster City, California, has announced an upcoming investor call to discuss interim results from two of its Phase 2b clinical studies. The call is scheduled for Monday, June 17, at 8:30 a.m. Eastern Time. The studies in question are the Volixibat VANTAGE study, focusing on primary biliary cholangitis (PBC), and the Volixibat VISTAS study, focusing on primary sclerosing cholangitis (PSC).

Mirum Pharmaceuticals is a biopharmaceutical company committed to improving the lives of patients with rare diseases. The company's portfolio includes three approved drugs: LIVMARLI® (maralixibat) oral solution, CHOLBAM® (cholic acid) capsules, and CHENODAL® (chenodiol) tablets.

LIVMARLI is an ileal bile acid transporter (IBAT) inhibitor that has been approved for treating rare liver diseases in both children and adults. In the United States, it is approved for treating cholestatic pruritus in patients with Alagille syndrome who are three months or older. In Europe, it is approved for patients aged two months and older. LIVMARLI is also approved in the U.S. to treat cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) who are five years of age and older. The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has given a positive opinion for the treatment of PFIC in patients as young as three months, with a final decision from the European Commission expected by the third quarter of 2024.

CHOLBAM is approved by the U.S. Food and Drug Administration (FDA) for treating bile acid synthesis disorders caused by single enzyme deficiencies and as an adjunctive treatment for peroxisomal disorders in patients exhibiting signs or symptoms of liver disease.

CHENODAL has been recognized by the FDA for medical necessity in treating patients with cerebrotendinous xanthomatosis (CTX), a rare genetic disorder.

Mirum is also advancing its late-stage pipeline with two investigational treatments aimed at debilitating liver diseases. Volixibat, an IBAT inhibitor, is currently under evaluation in two potentially registrational Phase 2b studies: the VISTAS study for PSC and the VANTAGE study for PBC. Additionally, CHENODAL has shown positive results in the Phase 3 RESTORE study, which is aimed at treating patients with CTX. The topline results from this study were reported in 2023.

The upcoming investor call will provide insights into the interim analysis results from the Volixibat studies, which are crucial for advancing these investigational treatments. Participants can join the call via a dial-in number or by accessing a webcast on Mirum’s website. A replay of the webcast will be available for 30 days following the call.

Mirum Pharmaceuticals continues to focus on transforming the treatment landscape for rare diseases, particularly those affecting the liver. With a robust pipeline and several approved medications, the company is making significant strides in providing effective treatments for patients with limited options.