Mirum Pharmaceuticals (MIRM) Corporate presentation summary

Financial performance and guidance

• Net product sales guidance for 2025 is $490–510M, with positive cash flow expected in 2025.

• Cash balance reported at $322M as of Q2 2025.

• Consistent year-over-year growth in bile acid portfolio sales, with $123M in 2022 and $109M in 2023.

• Portfolio estimated to have multi-billion dollar revenue potential across key products and pipeline.

Commercial portfolio and pipeline

• Three approved ultra-rare disease medicines: LIVMARLI, CHOLBAM, and CTEXLI.

• Four additional indications in development, including high-need orphan settings.

• LIVMARLI approved for ALGS and PFIC in both US and EU, with label expansion opportunities in ultra-rare cholestatic pruritus.

• Volixibat advancing in Phase 2b for PSC and PBC, both with positive interim analyses and FDA Breakthrough Therapy Designation for PBC.

• MRM-3379, a PDE4D inhibitor, entering Phase 2 for Fragile X Syndrome in Q4 2025.

Product efficacy and safety

• LIVMARLI demonstrates significant reductions in pruritus and serum bile acids in ALGS and PFIC, improving transplant-free survival.

• Well-characterized safety profile for LIVMARLI, with most adverse events being mild or moderate; liver function monitoring recommended.

• CHOLBAM and CTEXLI address bile acid synthesis disorders and CTX, with consistent sales and established safety profiles.