Mirum Pharmaceuticals (MIRM) Corporate presentation summary

Company overview and strategy

• Focuses on developing and commercializing medicines for rare diseases, with three approved products and three late-stage indications in high-need orphan indications.

• Demonstrates strong execution and efficiency, achieving global commercial reach since its founding in 2018.

• 2024 strategic goals include expanding product labels, leveraging expertise in IBAT inhibition, and growing the pipeline.

• Maintains a robust cash position of $303M as of March 31, 2024, supporting ongoing strategy execution.

• Projects 2024 net product sales guidance of $310–320M, reflecting continued commercial momentum.

Commercial portfolio and financial performance

• Three commercial medicines: LIVMARLI (maralixibat), CHOLBAM (cholic acid), and Chenodiol, targeting pediatric and adult rare liver diseases.

• Net product sales have grown from $75M in 2021 to $179M in 2023, with 2024 guidance of $310–320M.

• International expansion and broad partner/distributor network enhance global reach.

• Business model emphasizes patient support and engagement, especially in pediatric rare disease.

• Bile acid portfolio addresses multiple high-need settings, with consistent year-over-year growth.

Key products and clinical data

• LIVMARLI is approved for Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC), showing significant improvements in pruritus, serum bile acids, and transplant-free survival.

• Long-term data in ALGS: 84% experienced meaningful pruritus reduction, 83% had ≥20% reduction in serum bile acids, and 93% with >1-point itch reduction remained transplant-free at 6 years.

• In PFIC, 62% of patients had minimal to no itch after 26 weeks, with improvements across multiple subtypes.

• LIVMARLI safety profile is well-characterized, with most adverse events being mild/moderate GI symptoms; liver function monitoring is required.

• Bile acid replacement therapies (CHOLBAM, Chenodiol) address genetic disorders like CTX and PBD-ZSD, with positive Phase 3 data for CTX (RESTORE study).