Mirum Pharmaceuticals (MIRM) Corporate presentation summary

Commercial scale and portfolio

• Four approved rare-disease medicines; five additional indications in development, with estimated portfolio peak revenue potential of $4B+.

• 2025 net product sales were $521M; 2026 guidance is $715M–$720M. Livmarli sales reached $360M in 2025, up 69% YoY.

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

Rare liver disease pipeline

• EXPAND Phase 3 met its primary endpoint in pediatric cholestatic pruritus; placebo-adjusted itch reduction was 0.98 points in the overall population (p<0.0001); sNDA targeted H1 2027.

• VISTAS Phase 2b met its primary endpoint in PSC; placebo-adjusted itch reduction was 1.64 points (p<0.0001), and 56% had a ≥2-point reduction; NDA targeted H1 2027.

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

HDV program

• Brelovitug AZURE-1 Phase 3 met its primary endpoint at Week 24: response rates were 56% with 300 mg weekly and 45% with 900 mg every four weeks, vs 0% with delayed treatment.

• AZURE-4 topline data expected Q4 2026; AZURE-2 and AZURE-3 topline data expected H2 2027, supporting planned FDA and EMA submissions.