Summary of Mirum Pharmaceuticals Conference Call Company Overview - **Company**: Mirum Pharmaceuticals (NasdaqGM:MIRM) - **Event**: 2026 Conference on March 11, 2026 Key Points Financial Guidance and Commercial Strategy - Mirum Pharmaceuticals reiterated its revenue guidance for 2026, projecting between **$630 million and $650 million** [4] - The company is focused on funding R&D programs while maintaining durable trends in commercial performance [4] Upcoming Data Events - Key data events scheduled for Q2 2026 include: - **VISTAS PSC** top-line data, pivotal for potential NDA filing by year-end [4] - **Brelovitug** Phase 2b interim readout from the AZURE-1 study [5] - **EXPAND** top-line readout in Q4 2026 [5] EXPAND Program - The EXPAND program targets ultra-rare cholestasis conditions, with a significant portion of patients expected to be pediatric [7][9] - The potential market for LIVMARLI is projected to reach **at least $1 billion**, with EXPAND contributing approximately one-third of that [9] Geographic Expansion - LIVMARLI is currently available in **low- to mid-30 countries**, with most international sales driven by Alagille syndrome [11] - Opportunities for growth exist in regions like the Middle East and Latin America, where sales have been limited [11] Adult PFIC Market - The adult PFIC market is still in early stages, with increased genetic testing among providers expected to drive growth [12] - The company anticipates continued patient diagnosis and treatment persistence, contributing to long-term growth [13] PSC and PBC Opportunities - Approximately **60%** of diagnosed PSC patients are reported to experience meaningful itch, indicating a significant market for antipruritic therapies [19] - The company plans to conduct market development work to raise awareness of pruritus among healthcare professionals and patients [20] Launch Timeline - Following the release of top-line results, Mirum plans to engage with the FDA for pre-submission interactions, aiming for an NDA filing in the second half of 2026 and potential approval in 2027 [24] - Significant revenue contributions from new products are expected to begin in **2028** [25] Brelovitug Market Development - The company is focused on shifting testing paradigms for hepatitis delta, with a goal to increase diagnosis rates through reflex testing [30][32] - The estimated diagnosed and insured population for delta is around **15,000**, with a full prevalence of approximately **40,000** [33] Competitive Positioning - Brelovitug is positioned as a highly active regimen with a strong safety profile, focusing on endpoints linked to improved patient outcomes [37] - The company anticipates a **$750 million** floor guidance for brelovitug, based on conservative assumptions regarding market penetration [39] Future Growth and Business Development - Mirum remains active in seeking new rare disease programs, maintaining a high bar for potential acquisitions to ensure financial strength [47][48] Additional Insights - The company emphasizes the importance of patient experience and education in driving treatment uptake and market growth [18][21] - Mirum's strategy includes leveraging existing teams and distributor networks to expand market access, particularly in regions with lower GDP [44]