Mirum Pharmaceuticals (MIRM) investor relations material

Financial outlook

• 2026 revenue guidance is $715M-$720M, with growth expected across all four approved medicines and significant 2027 contributions from recently approved ATEBRIOZ in FOP.

• Cash-flow-positive guidance remains for 2027; cash flow was positive in 2025 and H1 2026, while H2 2026 includes elevated R&D and CMC investment for brelovitug and volixibat.

Volixibat in PSC and PBC

• PSC filing is planned for H1 2027 after further FDA engagement; the FDA recommended Phase III at the July pre-NDA meeting but said filing could proceed at discretion, and plans remain on track.

• A pre-planned PSC analysis found no IBD exacerbation signal, reassuring for the ~70% of PSC patients with IBD.

• Estimated PSC population is ~50,000 in the U.S., with ~60% having significant itch warranting treatment; a similar number is estimated outside the U.S.

• PBC data are expected in Q1 2027; 2024 interim results showed >2-point pruritus improvement plus sleep and fatigue improvements, supporting breakthrough designation and increased FDA dialogue.

• PBC population is estimated at 80,000-100,000; ~1/3 are controlled on UDCA but still have itch, while volixibat may also address patients with inadequate control on PPARs.

Brelovitug and HDV

• AZURE-1 and AZURE-4 Phase III studies met their 24-week primary endpoint versus delayed therapy, with rapid viral suppression and ALT normalization; responses continued to deepen in follow-up through 96 weeks.

• Phase IIa extension data through 108 weeks showed 100% ALT normalization, 100% virologic response and >70% TND; tolerability was also described as favorable.

• Management raised brelovitug’s projected opportunity to >$1B from $750M, driven by a higher-than-assumed Hepcludex price; the estimate includes competitive assumptions, with market share still uncertain.

• Claims data identified ~15,000 diagnosed, insured patients under physician care as a baseline; broader reflex testing could expand identification beyond that pool.

• The planned sales-force expansion includes ~40 representatives for adult PFIC, brelovitug and volixibat, expected to be in place by year-end 2026.