Mirum Pharmaceuticals (MIRM) Corporate presentation summary

Commercial portfolio and financial outlook

• Four approved rare-disease medicines span rare liver and genetic diseases; five additional indications are in development, with portfolio peak revenue potential estimated above $4B.

• FY 2025 net product sales were $521M, including $360M for LIVMARLI and $161M for the bile acid portfolio; 2026 net product sales guidance is $715–720M.

• Cash balance was $501M as of September 30, 2026; cash flow positive is targeted for 2027.

• LIVMARLI sales reached $360M in 2025, up 69% YoY; quarterly total net product sales rose to $185.7M in Q3 2026 from $133.0M in Q3 2025.

LIVMARLI and volixibat

• EXPAND Phase 3 met its primary endpoint in pediatric cholestatic pruritus; placebo-adjusted ItchRO reduction was 0.98 points overall (p<0.0001) and 1.15 points in biliary atresia (p=0.0002); sNDA submission targeted for H1 2027.

• EXPAND showed a 69% reduction in serum bile acids; all secondary endpoints were statistically significant, and safety was generally consistent with known IBAT inhibitor effects.

• VISTAS Phase 2b met its primary endpoint in PSC: placebo-adjusted pruritus improvement was 1.64 points (p<0.0001); NDA submission targeted for H1 2027.

• VANTAGE Phase 2b interim analysis in PBC showed placebo-adjusted pruritus reductions of 2.4 points at 20 mg and 2.6 points at 80 mg; topline data expected Q1 2027.

• LIVMARLI label expansion in additional cholestatic settings targets an estimated 2,000 US/EU biliary atresia patients; IP coverage is presented through 2040+.

Brelovitug for HDV

• AZURE-1 and AZURE-4 Phase 3 studies met their Week 24 primary endpoint; combined virologic response plus ALT normalization was 56% and 45%, respectively, versus 0% in delayed-treatment arms.

• Brelovitug achieved virologic response rates of 86% and 85% in AZURE-1 and AZURE-4; BLA submission is targeted for H1 2027.

• EU registration-enabling AZURE-2 and AZURE-3 topline data are expected in H2 2027; brelovitug has FDA Breakthrough Therapy and EMA PRIME designations.