Mirum Pharmaceuticals (MIRM) Corporate presentation summary

Strategic positioning and growth

• Recognized as a leader in rare disease medicines with a global commercial footprint and three approved products, targeting high-need orphan indications and five additional pipeline indications.

• 2024 net product sales guidance raised to $330–335M, reflecting strong commercial execution and international expansion.

• Cash balance of $294M as of September 30, 2024, supports ongoing strategic initiatives and pipeline development.

• Proven track record of execution, efficiency, and financial self-sustainability.

Commercial portfolio and pipeline

• Three approved rare disease products: LIVMARLI (maralixibat), CHOLBAM (cholic acid), and chenodiol, with global approvals for key indications such as ALGS and PFIC.

• Five additional indications in development, including CTX (PDUFA Mar 28, 2025), PSC, PBC, and FXS.

• Pipeline includes IBAT inhibitors (volixibat) for adult cholestatic liver diseases and MRM-3379 for Fragile X Syndrome.

• Ongoing label expansion efforts for LIVMARLI and chenodiol to address broader patient populations.

Product performance and clinical data

• LIVMARLI demonstrates significant and sustained improvements in pruritus, serum bile acids, and transplant-free survival in ALGS and PFIC, with robust long-term safety data.

• CHOLBAM and chenodiol address bile acid synthesis disorders and CTX, with consistent year-over-year growth and positive Phase 3 data for CTX.

• Volixibat shows positive interim results in PSC and PBC, with rapid and statistically significant reductions in pruritus and serum bile acids, and has received FDA Breakthrough Therapy Designation for PBC.

• MRM-3379, a selective PDE4D inhibitor, is advancing to Phase 2 for FXS, targeting cognitive and functional improvements.