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Mirum Pharmaceuticals

A commercial-stage rare disease company focused on cholestatic liver diseases and related disorders of bile acid biology, with a portfolio of marketed medicines and a pipeline built around IBAT inhibition and liver-targeted development.

Headquarters and Global Presence

Mirum is headquartered in Foster City, California. The company operates a U.S.-centered commercial organization with regulatory and commercial activity across North America and Europe, including EU approvals for its lead cholestasis franchise.

Founding and History

Mirum was formed to develop and commercialize therapies for rare liver diseases. It is publicly traded on Nasdaq (MIRM). In January 2026, Mirum completed the acquisition of Bluejay Therapeutics, adding a late-stage antiviral liver disease program to its portfolio.

Therapy Areas and Focus

Mirum’s core focus is rare hepatology and bile acid–related disorders, including:

Pediatric cholestatic liver diseases with pruritus as a key morbidity (including Alagille syndrome and progressive familial intrahepatic cholestasis)
Inborn errors of bile acid metabolism
Genetic neurology linked to bile acid pathway dysfunction (cerebrotendinous xanthomatosis)

Technology Platforms and Modalities

Mirum is a small-molecule company with two main development pillars:

IBAT inhibition in cholestatic disease

LIVMARLI maralixibat, an oral, minimally absorbed IBAT inhibitor
Approved for cholestatic pruritus in Alagille syndrome and PFIC in major markets (with age cutoffs varying by region)
In clinical development for pruritus in additional rare cholestatic settings in a Phase III program

Next-generation IBAT inhibition

Volixibat, an investigational oral IBAT inhibitor being developed for cholestatic pruritus in primary sclerosing cholangitis
Phase IIb VISTAS study, with topline results guided for IIQ 2026

Bile acid replacement and related products

CHOLBAM cholic acid, for bile acid synthesis disorders due to single-enzyme defects and as adjunctive therapy in certain peroxisomal disorders with liver involvement
CTEXLI chenodiol, for adults with cerebrotendinous xanthomatosis

Liver viral disease (post-acquisition)

Brelovitug, an investigational therapy for chronic hepatitis delta virus, in a Phase III program with topline results expected in 2H 2026

Key Personnel

Chris Peetz, Chief Executive Officer
Eric Bjerkholt, Chief Financial Officer
Joanne Quan, MD, Chief Medical Officer
Lara Longpre, Chief Development Officer
Hassan Javanbakht, PhD, Chief Scientific Officer
Jean-Luc Girardet, PhD, Chief Technical Officer
Peter Radovich, President and Chief Operating Officer

Strategic Partnerships

Mirum has expanded its portfolio through targeted acquisitions in rare disease and hepatology, most recently with the completed acquisition of Bluejay Therapeutics in January 2026. Commercial execution is supported by market-access and patient-support infrastructure aligned to rare pediatric hepatology.

FAQ Section

Mirum develops and commercializes small molecules for rare liver diseases, with a core franchise in IBAT inhibition to reduce bile acid–driven pruritus and related cholestasis complications, complemented by bile acid replacement therapies for metabolic and neurologic disorders.

Mirum’s primary focus is rare hepatology, particularly pediatric cholestatic diseases such as Alagille syndrome and PFIC, as well as bile acid synthesis disorders and cerebrotendinous xanthomatosis. The company has expanded into viral liver disease through the Bluejay acquisition.

Key disclosed pipeline programs include:

Maralixibat in Phase III for pruritus in additional rare cholestatic settings
Volixibat in Phase IIb for cholestatic pruritus in primary sclerosing cholangitis
Brelovitug in Phase III for chronic hepatitis delta virus

January 2026: Mirum completed the acquisition of Bluejay Therapeutics, adding brelovitug and its Phase III HDV program
January 2026: Mirum provided preliminary unaudited 2025 results and 2026 outlook updates via its investor news flow
Late 2025: Mirum reported completion of enrollment in the Phase IIb VISTAS study of volixibat, with topline results expected in IIQ 2026

Public updates have been weighted toward operational milestones (trial enrollment completion, program progression, regulatory status and regional approvals). The next material readouts guided by the company include Phase IIb VISTAS topline data in IIQ 2026 and Phase III brelovitug topline data in 2H 2026.

Near-term milestones are execution-led: completion and reporting of the Phase IIb volixibat study in PSC pruritus, continued advancement of maralixibat’s Phase III program in additional cholestatic pruritus settings, and delivery of Phase III results for brelovitug in chronic HDV.

Mirum’s marketed products cluster around bile acid biology and rare liver disease care pathways: maralixibat for cholestatic pruritus, cholic acid replacement for bile acid synthesis disorders, and chenodiol for CTX. The company’s pipeline aims to expand pruritus indications and add a late-stage viral liver disease opportunity.

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