Mirum Pharmaceuticals (MIRM) Corporate presentation summary

Strategic focus and portfolio overview

• Focused on delivering high-impact medicines for rare liver and genetic diseases, with a portfolio of three approved medicines and six additional indications in late-stage development.

• Five FDA Breakthrough Therapy designations and a peak revenue potential exceeding $4 billion.

• 2026 net product sales guidance of $680–700M, with expectations to be cash flow positive in 2027.

• Global commercial reach with products addressing both pediatric and adult rare disease populations.

Key products and clinical programs

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

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

• Brelovitug, a monoclonal antibody for chronic hepatitis delta virus (HDV), showed 100% virologic response in Phase 2b and is advancing through multiple Phase 3 trials.

• Zilurgisertib, an ALK2 inhibitor for fibrodysplasia ossificans progressiva (FOP), achieved significant reductions in new lesion formation and flare activity in pivotal Phase 2, with FDA priority review and PDUFA date in September 2026.

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

Financial performance and guidance

• Annual net product sales grew from $336M in 2022 to $521M in 2025, with projected $680–700M in 2026.

• Quarterly net product sales reached $176.2M in Q2 2026, with strong growth in both US and international markets.

• Operating income showed improvement, with a focus on achieving cash flow positivity in 2027.

• Cash balance of $561M as of June 30, 2026.