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.

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

• Portfolio peak revenue potential estimated at over $4B.

Commercial portfolio and pipeline

• Three approved rare disease medicines and five additional indications in development targeting high-need orphan indications.

• LIVMARLI, CHOLBAM, and CTEXLI are approved for various rare cholestatic and bile acid synthesis disorders.

• Pipeline includes volixibat for PSC and PBC, brelovitug for HDV, and MRM-3379 for Fragile X Syndrome, all with ongoing or planned registrational studies.

Product highlights and clinical data

• LIVMARLI demonstrated significant improvements in pruritus, serum bile acids, and transplant-free survival in ALGS and PFIC, with strong year-over-year sales growth (69% YoY in 2024).

• Volixibat showed rapid, statistically significant pruritus reduction in PSC and PBC, with positive interim Phase 2b results and FDA Breakthrough Therapy Designation.

• Brelovitug achieved high virologic response rates and ALT normalization in HDV, with favorable safety and ongoing Phase 3 trials.

• MRM-3379, a selective PDE4D inhibitor, is in Phase 2 for Fragile X Syndrome, showing efficacy in preclinical models and granted FDA Fast Track Designation.