• Total Revenue (2024): $560 million, representing 29% growth over 2023.
• Crysvita Revenue (2024): $410 million, with 25% growth over 2023.
• Dojolvi Revenue (2024): $88 million, representing 25% growth over 2023.
• Evkeeza Revenue (2024): $32 million.
• Mepsevii Revenue (2024): $30 million.
• Total Operating Expenses (2024): $1.1 billion.
• R&D Expenses (2024): $698 million.
• SG&A Expenses (2024): $322 million.
• Net Loss (2024): $569 million or $6.29 per share.
• Cash Equivalents and Marketable Securities (End of 2024): $745 million.
• Net Cash Used in Operations (2024): $414 million.
• 2025 Revenue Guidance: Expected between $640 million and $670 million, representing 14% to 20% growth over 2024.
• Crysvita Revenue Guidance (2025): Expected between $460 million and $480 million, representing 12% to 17% growth over 2024.
• Dojolvi Revenue Guidance (2025): Expected between $90 million and $100 million, representing 2% to 14% growth over 2024.

• Warning! GuruFocus has detected 4 Warning Signs with RARE.

Release Date: February 13, 2025

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

Positive Points

• Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) exceeded its revenue guidance for 2024, showcasing strong financial performance.
• The company successfully launched Evkeeza in Europe, Canada, and Japan, contributing to impressive international growth.
• Ultragenyx is advancing six late-stage programs in serious genetic conditions, with potential for significant value creation.
• The UX111 program for Sanfilippo syndrome shows promising clinical data, potentially leading to the company's first commercial gene therapy product.
• The company is on track for full-year GAAP profitability by 2027, supported by strong global revenue growth and multiple product launches.

Negative Points

• Ultragenyx reported a net loss of $569 million for 2024, highlighting ongoing financial challenges.
• Total operating expenses for the year were $1.1 billion, indicating high costs associated with R&D and SG&A.
• There is expected quarter-to-quarter revenue variability due to uneven ordering patterns for Crysvita in Latin America.
• The company faces risks and uncertainties related to regulatory approvals for its gene therapy programs.
• Ultragenyx's cash used in operations was $414 million in 2024, emphasizing the need for careful expense management.

Q & A Highlights

Q: Can you provide more details on your confidence in the second interim read for the OI study being sufficient to stop the study versus needing to go to the third interim? A: Emil Kakkis, CEO, stated that the second interim has a greater chance of hitting due to a 0.01 threshold and 12 months of treatment, allowing for group separation. He expressed confidence that the trial will be successful this year, whether at the second interim or final analysis, based on the positive progress of Phase 2 patients.

Q: If the second interim doesn't hit, what would be the reasons, and how long would we have to wait for the final analysis? A: Emil Kakkis explained that not hitting at the second interim could be due to variation in baseline fracture rates among patients. If it doesn't hit, they expect to release data by the end of the year on the final assessment.

Q: Once Orbit hits, will you immediately prepare to file, or will you wait for the Cosmic analysis to finish and add it to the application? A: Emil Kakkis confirmed that if Orbit hits, they will clean and lock the data quickly, allowing for a faster filing process. They will not wait for the Cosmic analysis to finish before filing.

Q: Can you clarify the interim analysis for the Cosmic study and its timing? A: Emil Kakkis stated that the interim analysis for Cosmic will be conducted in parallel with Orbit. If Orbit is positive, they will look at Cosmic data, both being evaluated at a 0.01 P-value. If Orbit doesn't stop, Cosmic will continue until final analysis.

Q: Regarding the UX111 gene therapy launch, how are you preparing for it, and what lessons are you applying from other programs? A: Emil Kakkis emphasized the importance of urgency in launching UX111, given the severe nature of the disease. They are working with payers to ensure a rapid review and approval process. Eric Crombez added that they are coordinating with treatment centers and leveraging their experience with other products to ensure a successful launch.

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