Mirum Pharmaceuticals (MIRM) Goldman Sachs 45th Annual Global Healthcare Conference summary

Company overview and product portfolio

• Focuses on rare disease treatments, with LIVMARLI as the lead product approved for Alagille Syndrome and PFIC, expanding internationally and in Europe.

• Added Chenodal and Cholbam to the portfolio, targeting bile acid synthesis disorders and CTX, with ongoing label expansion opportunities.

• Plans to announce interim data for volixibat, targeting adult cholestatic diseases (PSC and PBC), with adaptive studies underway.

• Strong financial position with $310M-$320M revenue guidance for 2024, majority from LIVMARLI, and over $300M cash on hand.

• Strategy centers on overlooked rare disease opportunities and leveraging commercial and regulatory expertise.

Competitive landscape and market dynamics

• LIVMARLI maintains strong performance in Alagille Syndrome despite new competition, with favorable payer policies and first-line therapy status.

• PFIC label expansion in March is driving new patient starts and positive reimbursement trends.

• In Europe, LIVMARLI is the only approved product for Alagille Syndrome and recently received a positive CHMP opinion for PFIC.

• Differentiation from competitors is based on clinical impact, dosing advancements, and robust pivotal study results.

Pipeline and clinical development

• Volixibat is being developed for larger adult indications (PSC and PBC) due to different patient needs and market size.

• PSC has no approved therapies; volixibat's pivotal study uses pruritus as the primary endpoint, with an adaptive phase II-B design and interim analysis for dose selection.

• PBC trial (VANTAGE) includes both first and second-line patients, focusing on symptomatic relief and dose selection.

• Interim data for both PSC and PBC studies will inform timelines and pivotal data expectations, with pivotal readouts expected in 18-24 months.