Mirum Pharmaceuticals (MIRM) Corporate presentation summary

Strategic focus and portfolio overview

• Focused on high-impact medicines for rare liver and genetic diseases, with a purpose-built rare disease model and global commercial reach.

• Portfolio includes three approved rare disease medicines and six additional indications in development, targeting both rare liver and genetic diseases.

• Four FDA Breakthrough Therapy designations and multiple late-stage clinical programs.

• Estimated peak revenue potential exceeds $4 billion, with 2026 net product sales guidance of $660–680 million and cash flow positivity expected in 2027.

Key products and clinical progress

• LIVMARLI (maralixibat) is approved for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), showing strong year-over-year sales growth and label expansion opportunities.

• Volixibat, an IBAT inhibitor, demonstrated statistically significant reductions in pruritus in primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), with NDA submission for PSC expected in H2 2026.

• Brelovitug, a monoclonal antibody for chronic hepatitis delta virus (HDV), met primary endpoints in Phase 2b and is advancing through multiple Phase 3 trials for FDA and EMA submissions.

• Zilurgisertib (ALK2 inhibitor) for fibrodysplasia ossificans progressiva (FOP) has an NDA filed with PDUFA date set for September 26, 2026.

• MRM-3379 (PDE4D inhibitor) for fragile X syndrome (FXS) is in Phase 2, with topline results expected in 2027.

Financial performance and guidance

• Annual net product sales grew from $336M in 2022 to $521M in 2025, with guidance of $660–680M for 2026.

• LIVMARLI sales increased from $75M in 2022 to $360M in 2025, reflecting strong adoption and international expansion.

• Bile acid portfolio (Cholbam and Ctexli) generated $161M in 2025, addressing multiple high-need rare genetic disease settings.

• Cash balance of $421M as of March 31, 2026, supporting ongoing development and commercialization.