Mirum Pharmaceuticals (MIRM) investor relations material

Financial and operational highlights

• Achieved $133 million in Q3 revenue and reached positive net income for the first time.

• Three approved products in ultra-rare genetic diseases and a robust pipeline with three pivotal readouts expected in the next 18 months.

• Continued strong growth in core products, with high patient retention and steady new patient additions.

• Tablet formulation of LIVMARLI launched, improving compliance and persistence among older patients.

• Gradual pricing tailwind expected as patient base matures and dose adjustments increase over time.

Product and clinical development updates

• Volixibat pivotal readout for PSC expected in Q2 next year; PBC study enrollment ongoing, with label expansion for LIVMARLI also progressing.

• Interim analyses show Volixibat has strong impact on fatigue, sleep, and pruritus in PBC and PSC.

• Expand study targets ultra-rare cholestatic pruritus, with high demand from compassionate use cases; at least 500 U.S. patients identified.

• Fragile X program advancing, leveraging higher CNS exposure for potential cognitive benefits.

Market dynamics and growth drivers

• LIVMARLI has about 50% penetration in Alagille syndrome in the U.S., with high capture of new diagnoses and long-term patient retention.

• PFIC launch exceeded expectations, driven by strong clinical data and increased genetic testing, revealing a larger market than initially estimated.

• Competitive landscape in PFIC and Alagille is favorable, with LIVMARLI holding over 80% share in Alagille and approaching half in PFIC.

• Volixibat positioned as potential first and only approved therapy for PSC, with significant opportunity in both PSC and PBC.