Mirum Pharmaceuticals, Inc., headquartered in Foster City, California, has scheduled the release of its first quarter 2024 financial results for May 8, 2024. In conjunction with this release, Mirum will conduct a conference call to discuss the financial outcomes and recent corporate advancements. The conference call is set for Wednesday, May 8, 2024, at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time). Participants in the United States can join toll-free at +1 833 470 1428, while international participants can dial +1 404 975 4839, using the passcode 479107. Additionally, the call can be accessed via a webcast on the company’s website under the Events & Presentations section, with a replay available for 30 days.
Mirum Pharmaceuticals is a biopharmaceutical company focused on the development and commercialization of treatments for rare diseases that affect both children and adults. Among Mirum’s approved therapies are LIVMARLI® (maralixibat) oral solution, CHOLBAM® (cholic acid) capsules, and CHENODAL® (chenodiol) tablets.
LIVMARLI, an ileal bile acid transporter (IBAT) inhibitor, is a key product in Mirum’s portfolio. It is approved for treating two specific rare liver conditions in both children and adults. In the United States, LIVMARLI is approved for treating cholestatic pruritus in patients with Alagille syndrome who are three months old or older. In Europe, the approval extends to patients who are at least two months old, with similar approvals in other global regions. Additionally, in the U.S., LIVMARLI is sanctioned for treating cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) who are at least five years old. Mirum has also submitted an application for approval in Europe for treating PFIC in patients as young as two months old.
CHOLBAM is approved by the U.S. Food and Drug Administration (FDA) for treating bile acid synthesis disorders due to single enzyme deficiencies and as an adjunctive treatment for peroxisomal disorders in patients exhibiting liver disease symptoms.
CHENODAL, another significant product, has been recognized by the FDA for its medical necessity in treating patients with cerebrotendinous xanthomatosis (CTX).
Mirum's developmental pipeline is robust, featuring late-stage investigational treatments for severe liver diseases. Volixibat, another IBAT inhibitor, is under evaluation in two potentially registrational studies. The Phase 2b VISTAS study is focused on primary sclerosing cholangitis, whereas the Phase 2b VANTAGE study targets primary biliary cholangitis. Furthermore, CHENODAL has been the subject of a Phase 3 clinical study, RESTORE, aimed at treating CTX patients, with promising topline results announced in 2023.
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