Mirum Pharmaceuticals Inc Dossier
Qualitative Analysis
Business overview
Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) is a global, commercial-stage biopharmaceutical company dedicated to identifying, acquiring, developing, and commercializing breakthrough therapies for debilitating rare and orphan diseases, with a primary focus on rare liver and rare genetic conditions. The company's commercial portfolio features three approved medicines: LIVMARLI (maralixibat) oral solution, CHOLBAM (cholic acid) capsules, and CTEXLI (chenodiol) tablets. LIVMARLI is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). CHOLBAM is indicated for bile acid synthesis disorders due to single enzyme defects and peroxisomal disorders, while CTEXLI is utilized for cerebrotendinous xanthomatosis (CTX). Mirum is leveraging its established commercial infrastructure to expand into adult cholestatic liver diseases and other ultra-rare genetic disorders.
Research as of 19 Jun 2026
Strategic Initiatives
Growth programs, investments, and their expected impact
Driving market penetration and global sales growth for LIVMARLI (maralixibat) in Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), alongside bile acid medicines CHOLBAM and CTEXLI.
Expected impact: Expected to generate $660 million to $680 million in net product sales for FY 2026.
Advancing volixibat through late-stage development for PSC and PBC, and executing the Phase 3 AZURE program for brelovitug in chronic HDV.
Expected impact: Positions Mirum to transition into a multi-asset, blockbuster-potential rare disease leader.
Mergers, Acquisitions & Partnerships
Recent deals and strategic collaborations
Recent Acquisitions
To acquire worldwide rights to brelovitug, a late-stage, fully human monoclonal antibody for chronic hepatitis delta virus (HDV), adding a high-potential Phase 3 asset to Mirum's rare liver disease pipeline.
Financial impact: Upfront consideration consisted of $250 million in cash (offset by $56.6 million of Bluejay's net cash acquired) and $370 million in Mirum common stock, plus up to $200 million in potential sales-based milestones. One-time acquisition-related IPR&D expenses of $726.3 million heavily impacted Q1 2026 GAAP profitability.
Strategic Partnerships
In-licensed exclusive worldwide rights to develop and commercialize zilurgisertib, an oral ALK2 inhibitor for fibrodysplasia ossificans progressiva (FOP), adding an active NDA-stage asset with Priority Review to Mirum's rare genetic disease pipeline.
Terms: Incyte received an upfront payment of $16 million and is eligible for additional development and regulatory milestones, plus royalties on future sales.