Mirum Pharmaceuticals (MIRM) Corporate presentation summary

Commercial performance and financial outlook

• Net product sales grew from $75M in 2022 to $521M in 2025, with 2026 guidance of $630–$650M and a CAGR of 91% from 2022–2025.

• Portfolio peak revenue potential estimated at over $4B, with multi-billion dollar opportunities in key products and pipeline assets.

• Cash flow positive expected in 2027, supported by a $391M cash balance at the end of 2025.

• Quarterly net product sales show consistent growth, with LIVMARLI and bile acid portfolio as main contributors.

Product portfolio and pipeline

• Three approved rare disease medicines and five additional indications in development targeting high-need orphan indications.

• LIVMARLI (maralixibat) approved for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), with label expansion opportunities in ultra-rare cholestatic pruritus.

• Bile acid replacement therapies (CHOLBAM and CTEXLI) address rare genetic diseases such as CTX and BASD.

• Volixibat, an IBAT inhibitor, is in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), both with significant unmet need.

• Brelovitug (for HDV) and MRM-3379 (for FXS) are advancing in clinical trials, with both receiving FDA breakthrough or fast track designations.

Clinical development and regulatory milestones

• Four potentially registrational topline readouts expected in the next 18 months, including VISTAS (PSC), VANTAGE (PBC), AZURE (HDV), and EXPAND (ultra-rare cholestatic pruritus).

• Volixibat demonstrated positive interim results in both PSC and PBC, with significant reductions in pruritus and serum bile acids.

• Brelovitug showed high virologic response rates and favorable safety in HDV, with multiple phase 3 trials ongoing to support FDA and EMA submissions.

• MRM-3379 is in phase 2 for FXS, with preclinical and early clinical data supporting efficacy and safety.