Mirum Pharmaceuticals (MIRM) Corporate presentation summary

Strategic positioning and growth

• Recognized as a leader in rare disease medicine development and commercialization, with a global commercial footprint and three approved medicines targeting high-need orphan indications.

• 2024 net product sales guidance is $310–320M, reflecting strong year-over-year growth and financial self-sustainability with a $295M cash balance as of June 30, 2024.

• Strategic goals include expanding pipeline, leveraging expertise in IBAT inhibition, and pursuing label expansions for key products.

Commercial portfolio and pipeline

• Three approved rare disease products: LIVMARLI (maralixibat), CHOLBAM (cholic acid), and chenodiol, with four additional late-stage indications in development.

• LIVMARLI is approved for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC) in both the US and EU, with ongoing international launches and robust evidence supporting long-term benefits.

• Pipeline includes volixibat for adult cholestatic liver diseases (PSC and PBC), both in Phase 2b with positive interim analyses and FDA Breakthrough Therapy Designation for PBC.

Product performance and clinical data

• LIVMARLI demonstrated significant reductions in pruritus and serum bile acids, improving transplant-free survival in ALGS and PFIC patients.

• Bile acid portfolio (CHOLBAM and chenodiol) addresses multiple genetic disorders, with chenodiol showing positive Phase 3 results in CTX and an NDA submitted.

• Volixibat showed rapid, statistically significant improvements in pruritus and serum bile acids in PSC and PBC, with favorable safety profiles.