Mirum Pharmaceuticals (MIRM) investor relations material

Financial performance and guidance

• Net product sales grew from $336M in 2022 to $521M in 2025, with 2026 guidance of $660–680M and a cash balance of $421M as of March 2026.

• LIVMARLI sales increased significantly, reaching $360M in 2025, with strong international and US growth.

• Portfolio peak revenue potential estimated at over $4B, driven by rare liver and genetic disease medicines.

• Cash flow positive expected in 2027, supported by expanding indications and new launches.

Product portfolio and pipeline

• Three approved rare disease medicines (LIVMARLI, Cholbam, Ctexli) and six additional indications in late-stage development.

• LIVMARLI approved for ALGS and PFIC, with label expansion opportunities in ultra-rare cholestatic pruritus and ongoing Phase 3 studies.

• Volixibat demonstrated efficacy in PSC and PBC, with positive Phase 2b results and NDA submission planned for H2 2026.

• Brelovitug showed 100% virologic response in HDV Phase 2b, with multiple Phase 3 trials supporting FDA/EMA submissions.

• Zilurgisertib for FOP met pivotal Phase 2 endpoints, with FDA Priority Review and PDUFA date set for September 2026.

• MRM-3379 for Fragile X Syndrome is in Phase 2, with topline data expected in 2027.

Clinical and commercial highlights

• LIVMARLI demonstrated significant improvements in pruritus, serum bile acids, and transplant-free survival in ALGS and PFIC.

• Volixibat reduced pruritus and improved fatigue and sleep in PSC and PBC, with a generally consistent safety profile.

• Brelovitug achieved rapid and sustained reductions in HDV RNA and ALT, with a favorable safety profile.

• Zilurgisertib reduced new HO lesions and flare activity in FOP, with no grade ≥3 TEAEs occurring in more than one patient.

• Bile acid portfolio (Cholbam, Ctexli) addresses multiple high-need genetic disorders, with growing sales and commercial synergies.