Mirum Pharmaceuticals (MIRM) Corporate presentation summary

Financial performance and guidance

• 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.

• Peak revenue potential for the portfolio is estimated at over $4B.

• Cash flow positive expected in 2027, with a $391M cash balance reported.

Commercial portfolio and pipeline

• Three approved rare disease medicines: LIVMARLI, CHOLBAM, and CTEXLI, with five additional indications in development.

• LIVMARLI is approved for Alagille syndrome (ALGS) and PFIC, with label expansion opportunities in ultra-rare cholestatic pruritus.

• Volixibat is in late-stage development for PSC and PBC, both with significant unmet needs and FDA Breakthrough Therapy Designation.

• Brelovitug is advancing in HDV, showing 100% virologic response in Phase 2b and granted FDA Breakthrough and EU PRIME designations.

• MRM-3379, a PDE4D inhibitor for Fragile X Syndrome, is in Phase 2 with FDA Fast Track Designation.

Clinical and regulatory milestones

• Four potentially registrational topline readouts expected in the next 18 months: VISTAS (PSC), AZURE-1 & 4 (HDV), EXPAND (ultra-rare cholestatic pruritus), and VANTAGE (PBC).

• Volixibat and brelovitug are advancing toward regulatory submissions and potential launches in 2026–2027.

• LIVMARLI and pipeline assets have robust IP protection in the US and Europe, with coverage extending to 2040+.