Mirum Pharmaceuticals (MIRM) Corporate presentation summary

Commercial and financial overview

• Four approved rare-disease medicines and five additional indications in development; estimated portfolio peak revenue potential exceeds $4B.

• Annual net product sales rose from $336M in 2022 to $521.3M in 2025; 2026 guidance is $680M–$700M.

• Livmarli sales reached $360M in 2025, including $244.7M U.S. and $115.3M international sales; Q2 2026 total sales were $128.7M.

• Cash, cash equivalents and investments were $561M as of June 30, 2026; cash flow positive in 2027 is a stated target.

Rare liver disease programs

• EXPAND Phase 3 met its primary endpoint: pruritus score change was -1.81 with maralixibat versus -0.83 with placebo; sNDA submission targeted for H1 2027.

• VISTAS Phase 2b met its primary endpoint in PSC: pruritus score change was -2.72 with volixibat versus -1.08 with placebo; NDA submission targeted for H1 2027, pending FDA discussion.

• VANTAGE interim analysis in PBC showed placebo-adjusted pruritus responses of -2.4 at 20 mg and -2.6 at 80 mg; topline data expected Q1 2027.

• Brelovitug AZURE-1 Phase 3 met its primary endpoint: response was 56% with weekly dosing and 45% with monthly dosing versus 0% for delayed treatment; AZURE-4 topline expected Q4 2026.

• AZURE-2 and AZURE-3 topline data are expected H2 2027; the programs support planned EU registration.

Rare genetic disease programs

• Atebrioz (zilurgisertib) received FDA approval to reduce total new heterotopic ossification in adults and children aged 12 years and older with FOP.

• In PROGRESS Cohort 1, zilurgisertib showed an 81% reduction in the proportion of patients with new lesions at Week 24; younger-patient cohorts are in development.

• MRM-3379 is in a Phase 2 study for Fragile X syndrome; topline data are expected in 2027.

• Cholbam and Ctexli address bile acid synthesis disorders and cerebrotendinous xanthomatosis; combined bile acid portfolio sales were $161.3M in 2025.