Mirum Pharmaceuticals (MIRM) 8th Annual Wolfe Research Healthcare Conference summary

Portfolio outlook

• Four medicines approved; five sNDA, NDA and BLA filings expected in 2027, with many more medicines and indications expected to be approved within 18 months.

• Significant revenue growth over the next 5+ years and substantial cash flow generation expected.

LIVMARLI and biliary atresia

• Alagille syndrome sales are growing QoQ, with treatment-eligible population penetration near 50%; PFIC is the largest growth driver and remains early in its ramp.

• PFIC growth reflects new patients, twice-daily dosing and older, heavier patients; sequential quarterly revenue increases of $10M-$15M are expected to continue for the foreseeable future.

• Genetic-testing education among adult hepatologists and GI doctors is finding new PFIC patients monthly; treatment is splitting the opportunity with a competitor.

• Biliary atresia patients are identified early through the Kasai procedure and overlap with existing pediatric hepatology contacts; ~1,000 U.S. patients, higher dosing and no expected competition make the opportunity at least Alagille-sized.

• The $10M-$15M sequential growth expectation covers the overall portfolio; annual revenue and specific 2027 guidance planned for early January.

Volixibat in PSC and PBC

• PSC discussions with FDA are progressing on plan for a H1 2027 submission; IBD analysis was part of the original study plan and showed no exacerbation.

• PBC data expected in Q1 2027; a placebo-adjusted effect in the ballpark of the interim result, which exceeded 2 points, would be a win, alongside reported sleep and fatigue improvements.

• PBC opportunity includes >80,000 patients; many have pruritus despite being controlled on UDCA, and PPAR therapies may not fully address itching.

• FDA breakthrough designation for PBC has enabled more interaction since 2024; management expressed greater confidence in recognition of the study as pivotal than in the PSC path.

• Estimated U.S. populations with pruritus: ~50,000 PBC patients and ~20,000 PSC patients; anticipated use across the spectrum of pruritus severity.