Mirum Pharmaceuticals (MIRM) Corporate presentation summary

Strategic overview and financial performance

• Focused on delivering high-impact medicines for rare liver and genetic diseases, with a portfolio targeting multi-billion dollar revenue potential and a 2026 net product sales guidance of $680–700M.

• Five approved indications and four in late-stage development, with five FDA Breakthrough Therapy Designations and over $4B estimated peak revenue potential.

• Commercial portfolio includes Livmarli, Ctexli, Cholbam, and ATEBRIOZ, with significant year-over-year sales growth and global commercial reach.

• Cash flow positive expected in 2027, supported by a $561M cash balance as of June 2026.

Pipeline and clinical development

• Livmarli is approved for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), with label expansion opportunities in ultra-rare cholestatic pruritus.

• Volixibat demonstrated positive Phase 2b results in primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), with NDA submissions targeted for 2027.

• Brelovitug showed efficacy and favorable safety in chronic hepatitis delta virus (HDV), meeting primary endpoints in Phase 3 and supporting FDA/EMA submissions.

• ATEBRIOZ (zilurgisertib) received FDA approval for fibrodysplasia ossificans progressiva (FOP), with ongoing studies in younger patients.

• MRM-3379, a PDE4D inhibitor for Fragile X syndrome (FXS), is in Phase 2 with topline data expected in 2027.

Market opportunity and patient impact

• Addressable markets include rare liver diseases (ALGS, PFIC, PSC, PBC, HDV) and rare genetic diseases (CTX, BASD, FOP, FXS), with significant unmet needs and high symptom burden.

• Livmarli demonstrated substantial improvements in pruritus, serum bile acids, and transplant-free survival in ALGS and PFIC patients.

• Volixibat and brelovitug address pruritus and disease progression in PSC, PBC, and HDV, with strong clinical data supporting efficacy and safety.

• Bile acid replacement therapies (Cholbam, Ctexli) address multiple high-need genetic settings, with growing commercial synergies.