• Total Net Product Sales (Q4 2024): $99.4 million, a 43% year-over-year increase from $69.5 million in Q4 2023.
• Total Net Product Sales (Full Year 2024): $336.4 million, up from $178.9 million in 2023.
• LIVMARLI Net Product Sales (Q4 2024): $64.1 million.
• LIVMARLI Net Product Sales (Full Year 2024): $213.3 million, a 50% increase compared to 2023.
• Bile Acid Medicines Net Product Sales (Q4 2024): $35 million, approximately 25% growth over Q4 2023.
• Bile Acid Medicines Net Product Sales (Full Year 2024): $123.1 million.
• Total Operating Expense (2024): $424.5 million.
• R&D Expense (2024): $140.6 million.
• SG&A Expense (2024): $202.2 million.
• Cost of Sales (2024): $81.6 million.
• Non-Cash Stock-Based Compensation Expense (2024): $48.4 million.
• Intangible Amortization and Other Non-Cash Items (2024): $31 million.
• Cash, Cash Equivalents, and Investments (End of 2024): $293 million, an increase of approximately $7 million from the start of the year.
• 2025 Net Product Sales Guidance: $420 million to $435 million.

• Warning! GuruFocus has detected 6 Warning Signs with MDXG.

Release Date: February 26, 2025

For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Positive Points

• Mirum Pharmaceuticals Inc (NASDAQ:MIRM) reported total net product sales of $336.4 million for 2024, exceeding the upper end of their revised guidance range.
• The company achieved a 43% year-over-year increase in net product sales in the fourth quarter of 2024.
• LIVMARLI's net product sales increased by 50% compared to 2023, driven by strong demand in Alagille syndrome and PFIC.
• The FDA approved CTEXLI for cerebrotendinous xanthomatosis (CTX) in adults, providing 7 years of market exclusivity.
• Mirum Pharmaceuticals Inc (NASDAQ:MIRM) is cash flow positive and expects to maintain this status in 2025, with projected net product sales between $420 million and $435 million.

Negative Points

• Total operating expenses for 2024 were $424.5 million, which includes significant R&D and SG&A expenses.
• The company faces risks and uncertainties that may cause actual results to differ materially from forward-looking statements.
• The convertibles mature in four years, which could impact future financial planning.
• The company is still in the early stages of its journey, indicating potential challenges in achieving long-term strategic goals.
• Enrollment for some clinical studies, such as the VANTAGE study, is not expected to complete until 2026, delaying potential market entry.

Q & A Highlights

Q: How does the strong stock performance and earnings impact Mirum's business development and capital allocation strategies? A: Christopher Peetz, CEO, explained that Mirum's strong financial position allows the company to pursue acquisitions and roll up rare disease products, creating value. Eric Bjerkholt, CFO, added that the convertible notes maturing in four years do not significantly impact current plans, and the company has flexibility for business development opportunities.

Q: What assumptions support the $1 billion revenue potential for MRM-3379 in Fragile X syndrome? A: Christopher Peetz, CEO, stated that the market potential is based on a 50,000-patient market for male Fragile X patients in the U.S., with substantial upside beyond $1 billion. Joanne Quan, CMO, added that PDE4D's expression in the brain suggests potential applications in other intellectual disability conditions.

Q: What is the current market penetration for LIVMARLI in Alagille syndrome, and what drives future growth? A: Christopher Peetz, CEO, noted that penetration is around 40% for eligible U.S. patients, with growth driven by further penetration, new diagnoses, and weight-based dosing adjustments over time.

Q: How will the recent approval of CTEXLI for CTX affect sales? A: Christopher Peetz, CEO, explained that the approval allows for converting existing chenodiol patients to CTEXLI and aims to increase diagnosis rates, gradually improving sales by reaching more patients earlier.

Q: What is the patient mix for LIVMARLI between Alagille syndrome and PFIC, and how will it evolve? A: Christopher Peetz, CEO, stated that most LIVMARLI patients are currently Alagille syndrome patients, but there has been a significant increase in PFIC patient starts, contributing to new patient growth.

Q: How will the commercial team expand for the potential label expansion of LIVMARLI in cholestatic pruritus? A: Christopher Peetz, CEO, indicated that no expansion is needed as the same prescribers for Alagille syndrome and PFIC will handle the expanded indication.

Q: How does the VANTAGE study account for prior OCA use in PBC patients? A: Joanne Quan, CMO, mentioned that prior OCA use has not been a significant issue, and enrollment has increased since interim results. Most VANTAGE study patients are first-line, before OCA or new PPARs use.

Q: What is the expected timeline for top-line data from the volixibat study in PSC? A: Christopher Peetz, CEO, stated that top-line data is expected approximately six months after enrollment completion, which is on track for the second half of the year.

For the complete transcript of the earnings call, please refer to the full earnings call transcript.