Mirum Pharmaceuticals, Inc. Q4 FY2024 earnings call

• Strategic priorities for 2025 include global growth of commercial medicines, advancing pipeline, selectively pursuing product acquisitions, and maintaining financial discipline.
• 2024 was a big year with total net product sales exceeding revised guidance, LIVMARLI's strong performance, FDA approval of CTEXLI, and pipeline progress like Volixibat's positive interim results and MRM-3379's program initiation.
• Joanne Quan discussed pipeline progress in Volixibat studies for PSC and PBC, and MRM-3379's Phase 2 study preparation.
• Eric Bjerkholt noted the business was cash flow positive in 2024, ended 2024 with $293 million in cash, etc., and guided for net product sales of $420-435 million in 2025 with positive cash flow.

Total net product sales in 2024 were $336.4 million. LIVMARLI had total net product sales of $213 million in 2024, with approximately $155 million from the US business and $59 million internationally. The bile acid medicine portfolio had total net product sales of $123 million in 2024. In the fourth quarter of 2024, total net product sales were $99.4 million, a 43% year-over-year increase. LIVMARLI's fourth quarter net product sales were $64.1 million, and its 2024 total was $213.3 million, a 50% increase from 2023. The bile acid medicines portfolio had fourth quarter 2024 net product sales of $35 million, a ~25% growth from the prior year's fourth quarter.

• Expect net product sales between $420 million and $435 million in 2025.
• Anticipate positive cash flow again in 2025.

Forward-looking statements involve risks and uncertainties; refer to risk factors in latest form 10-K and subsequent SEC filings for more information.

Q: Congrats on the great progress. Just a strategic question about BD and capital allocation given stock trading above conversion price.

A: Chris Peetz mentioned leveraging financial position for rare disease product acquisitions, and Eric Bjerkholt noted convert matures in four years and financial flexibility for BD alternatives.

Q: Talk about underlying assumptions for MRM-3379's $1B revenue potential in Fragile X and other indications.

A: Chris Peetz discussed market sizing for Fragile X (50,000-patient market in US), and Joanne Quan mentioned PDE4D's potential in other intellectual disability-related conditions.

Q: On Alagille market penetration and US growth drivers.

A: Chris Peetz said Alagille syndrome eligible treatment population in US is ~40% penetrated, with growth from prevalent patient penetration, new diagnoses, and dose adjustments.

Q: Elaborate on CTX approval's impact on sales trajectory.

A: Chris Peetz said plan is to have diagnosed CTX patients convert to CTEXLI, aiming to impact diagnosis and treatment earlier.

Q: Patient mix for LIVMARLI in 2024 Q4 and 2025 expectations.

A: Chris Peetz said majority of LIVMARLI contribution is from Alagille syndrome patients, with step-up in PFIC patient starts contributing to new patient starts.

Q: Commercial team expansion for expanded pruritus pending EXPAND study.

A: Chris Peetz said planning for LIVMARLI's label expansion with EXPAND leverages existing prescribers, no major field team expansion needed.

Q: VANTAGE study's consideration of prior OCA use and enrollment.

A: Joanne Quan said enrollment picked up nicely since interim results, not seeing issues with OCA use.

Q: Label outlook for EXPAND study and patient number estimation.

A: Chris Peetz said label wording to be ironed out, patient number estimated as PFIC-sized or larger based on compassionate use and physician interest.

Q: Volixibat study in PSC timeline and EXPAND study patient experience.

A: Chris Peetz said Volixibat study in PSC has ~6 months after enrollment completion for topline data; Joanne Quan mentioned positive patient experiences with biliary atresia patients in EXPAND study.