• Net Product Sales: $90.3 million, an 89% increase from Q3 2023.
• Full Year Guidance: Increased to $330 to $335 million in net product revenue.
• Operating Cash Flow: Achieved positive operating cash flow for the first time in Q3 2024.
• Total Operating Expenses: $103.9 million, including R&D expense of $31.7 million and SG&A expense of $50.5 million.
• Net Loss: $15 million or $0.32 per share.
• Cash, Cash Equivalents, and Investments: $293.8 million as of September 30, 2024.
• Livmarli Global Net Product Sales: $59.1 million, with US sales at $43.5 million and international sales at $15.6 million.
• Kolbam and Chenodal Net Product Sales: $31.2 million in Q3 2024.

• Warning! GuruFocus has detected 3 Warning Sign with MIRM.

Release Date: November 12, 2024

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

Positive Points

• Mirum Pharmaceuticals Inc (NASDAQ:MIRM) reported a significant increase in net product sales, reaching $90.3 million in Q3 2024, an 89% increase from the same quarter last year.
• The company has raised its full-year 2024 guidance to $330 to $335 million in net product revenue, reflecting strong commercial execution.
• Mirum Pharmaceuticals Inc (NASDAQ:MIRM) received breakthrough therapy designation for its drug candidate in treating cholestatic pruritus in PBC, highlighting its potential effectiveness.
• The company achieved positive operating cash flow for the first time in Q3 2024, indicating improved financial health.
• Mirum Pharmaceuticals Inc (NASDAQ:MIRM) expanded its pipeline with the addition of MRM 3,379, a candidate for treating Fragile X syndrome, aligning with its strategy in rare genetic neurology.

Negative Points

• Despite strong sales growth, Mirum Pharmaceuticals Inc (NASDAQ:MIRM) reported a net loss of $15 million for Q3 2024.
• The company's total operating expenses were high at $103.9 million, which includes significant R&D and SG&A expenses.
• Mirum Pharmaceuticals Inc (NASDAQ:MIRM) faces challenges in pricing and reimbursement negotiations in Europe for its products.
• The company has a substantial milestone payment obligation, including a $10 million payment to Takeda and future payments related to MRM 3,379.
• There is uncertainty regarding the regulatory path and endpoints for the newly acquired Fragile X syndrome program, which may impact future development timelines.

Q & A Highlights

Q: Can you explain the current registrational endpoint in Fragile X and any opportunity for an accelerated path? A: Joanne Quan, Chief Medical Officer, explained that they plan to use the NIH toolbox as a registrational endpoint. Although they haven't had further discussions with the FDA yet, they believe this is a viable path forward. The molecule's selectivity and high brain penetration provide advantages, and they are excited about moving forward with the FDA.

Q: Are you planning to engage with regulators before initiating the phase two study for Fragile X? A: Joanne Quan confirmed that they plan to engage with the FDA early next year regarding the program.

Q: Can you provide more detail on the data expected from the late-breaking presentation at AASLD for Veli? A: Christopher Peetz, CEO, mentioned that the top-line data is already out, showing a strong response. They are excited to share the update soon, focusing on the registrational endpoint they are pursuing.

Q: What is driving the acceleration in PFIC sales, and what does it mean for growth moving forward? A: Peter Radovich, President and COO, stated that reimbursement has been a key driver, with coverage secured sooner than expected. They are seeing demand from IBAT-naive patients and some switches, contributing to growth.

Q: How does the recent in-licensing of MRM 3,379 fit into your broader business development strategy? A: Christopher Peetz explained that the addition of MRM 3,379 aligns with their focus on rare genetic diseases. They see it as an efficient early-stage program with targeted investment to achieve proof of concept, fitting well with their capabilities in rare disease.

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