Mirum Pharmaceuticals, Inc. Q3 FY2024 earnings call

• Commercial execution: Strong growth in all three commercial medicines, with Q3 net product sales of $90.3 million.
• Pipeline progress: Volixibat received breakthrough therapy designation for PBC based on Phase 2b VANTAGE study interim results; Phase 3 EXPAND study for LIVMARLI in Cholestatic Pruritus underway; CHENODAL's NDA for CTX granted priority review with PDUFA date of December 28, 2024; added MRM-3379 for Fragile X syndrome with plan to start Phase 2 study next year.
• Financials: Achieved positive operating cash flow in Q3; net loss for the quarter ended September 30 was $15 million or $0.32 per share; cash, cash equivalents, and investments as of September 30 were $293.8 million.

In the third quarter of 2024, Mirum Pharmaceuticals saw strong performance across its commercial segments. LIVMARLI had total global net product sales of $59.1 million, with U.S. sales at $43.5 million and international sales at $15.6 million. CHOLBAM and CHENODAL had net product sales of $31.2 million in the third quarter. Total net product revenue for the third quarter was $90.3 million, which is an 89% increase from the third quarter of 2023. For the full year, the company increased its guidance to $330 to $335 million in net product revenue.

• Increased full-year net product revenue guidance to $330 to $335 million.
• Phase 3 EXPAND study for LIVMARLI in Cholestatic Pruritus is underway.
• CHENODAL's NDA for CTX has a PDUFA date of December 28, 2024.
• Plan to start Phase 2 study for MRM-3379 in Fragile X syndrome next year.

• Forward-looking statements involve risks and uncertainties that may cause actual results to differ materially from projections. Refer to the risk factors in the latest form 10-Q and subsequent SEC filings for more information.

Q: Details on Fragile X registrational endpoints and engagement with regulators.

A: Joanne Quan stated they can use the NIH toolbox as a registrational endpoint and will engage with the FDA early next year. Chris Peetz added the NIH toolbox endpoint is a patient-reported outcome.

Q: Volixibat data at AASLD, LIVMARLI growth, BD strategy.

A: Chris Peetz mentioned Volixibat data will be presented at AASLD; Peter Radovich discussed LIVMARLI growth due to faster reimbursement; Chris Peetz said BD focuses on overlooked rare disease value.

Q: MRM-3379 deal terms, OpEx impact, PFIC growth drivers.

A: Eric Bjerkholt said upfront for MRM-3379 was $7.5 million with a $7.5 million Phase 3 start milestone; Peter Radovich noted the program is an efficient early stage investment; Chris Peetz said PFIC growth is due to faster reimbursement, de novo demand, and some switches.

Q: EXPAND study timeline, PDE4D strategy.

A: Joanne Quan said EXPAND study sites are opening and expect to start screening patients soon; Chris Peetz said PDE4D focus is on Fragile X first with plan to evaluate broader indications later.

Q: BD strategy comparison, CHENODAL market opportunity.

A: Chris Peetz said BD looks for overlooked value across commercial to Phase 1/2 ready; Chris Peetz mentioned CHENODAL has ~1-2k prevalent patients in the U.S. with potential sales impact from label approval.

Q: PFIC US/EU provider reach, EXPAND study enrollment, PDE target scope.

A: Chris Peetz said substantially reached US PFIC providers; Chris Peetz noted EXPAND study sites are opening but too early for enrollment details; Chris Peetz said PDE target includes rare genetic diseases.