Mirum Pharmaceuticals (MIRM) Corporate presentation summary

Financial performance and guidance

• Net product sales guidance for 2025 is $420–435M, reflecting strong year-over-year growth and positive cash flow in 2024.

• Cash, cash equivalents, and investments total $293M, supporting ongoing operations and pipeline development.

• Commercial portfolio includes three approved ultra-rare disease medicines with expanding indications.

Commercial portfolio and pipeline

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

• LIVMARLI is approved for Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) in both the US and EU.

• Pipeline includes volixibat for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), and MRM-3379 for Fragile X Syndrome (FXS).

• Volixibat and MRM-3379 each have multi-billion dollar revenue potential.

Product highlights and clinical data

• 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 and manageable.

• CHOLBAM and CTEXLI address bile acid synthesis disorders and cerebrotendinous xanthomatosis, showing consistent sales growth.