Mirum Pharmaceuticals (MIRM) investor relations material

Key business updates and financial guidance

• Commercial guidance reiterated at $630–$650 million, with durable trends supporting R&D investment.

• Four pivotal clinical readouts expected over the next 18 months, including VISTAS PSC, brelovitug, and EXPAND studies.

• EXPAND population could represent a third of peak sales for LIVMARLI, with at least 500 pediatric patients in the U.S. and similar numbers in Europe.

• Geographic expansion ongoing, with sales in 30+ countries and further opportunities in the Middle East and Latin America.

• Adult PFIC market still in early stages, with increased genetic testing but more education needed for broader adoption.

Clinical development and launch timelines

• VISTAS PSC top-line data expected in Q2, potentially enabling NDA filing by year-end and approval in 2027.

• BreloVitug phase 2b interim readout in Q2, phase 3 top-line data in H2, and BLA filing in early 2027.

• EXPAND study top-line readout in Q4 2026; volixibat PBC data expected in H1 2025.

• Commercial launch for PSC anticipated in 2027, with top-line revenue impact in 2028.

• Commercial infrastructure for PSC and PBC launches will be highly synergistic, targeting the same prescriber base.

Market development and patient engagement

• Market development efforts include patient testimonials, peer programs, advocacy collaborations, and social media outreach.

• Raising awareness of pruritus burden is key, as many patients and caregivers underreport symptoms.

• Approximately 60% of PSC patients experience meaningful pruritus, though direct patient surveys suggest higher rates.

• Uptake of new therapies will depend on treatment experience and evolving physician-patient conversations.