Ultragenyx Pharmaceutical Inc (RARE) Q1 2025 Earnings Call Highlights: Strong Revenue Growth ...

GuruFocus News

Wed, May 7, 2025, 1:23 AM 4 min read

Revenue: $139 million in Q1 2025, representing 28% growth over Q1 2024.
Crysvita Revenue: $103 million, with 25% growth over 2024; $41 million from North America, $55 million from Latin America and Turkey, $7 million from Europe.
Dojolvi Revenue: $17 million, consistent with steady growth.
Evkeeza Revenue: $11 million, with increasing demand following launches outside the US.
Mepsevii Revenue: $8 million, treating patients in ultra-rare indications.
Operating Expenses: $282 million, including R&D expenses of $166 million, SG&A expenses of $88 million, and cost of sales of $29 million.
Net Loss: $151 million, or $1.57 per share.
Cash Position: $563 million in cash, cash equivalents, and marketable securities as of March 31, 2025.
Net Cash Used in Operations: $166 million in Q1 2025.
2025 Revenue Guidance: Expected between $640 million and $670 million, representing 14% to 20% growth over 2024.
Crysvita Revenue Guidance: Expected between $460 million and $480 million, representing 12% to 17% growth over 2024.
Dojolvi Revenue Guidance: 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: May 06, 2025

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

Positive Points

Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) reported a 28% revenue growth in Q1 2025 compared to Q1 2024, driven by strong performance across its product portfolio.
The company has made significant progress in its late-stage clinical programs, including UX143 for osteogenesis imperfecta and GTX102 for Angelman syndrome, with promising data expected in 2026.
Ultragenyx is actively expanding its global footprint, with successful commercialization efforts for Crysvita in Latin America and Evkeeza in Europe and Japan.
The company is on track to submit two BLAs in 2025, with UX111 for Sanfilippo syndrome under FDA review and DTX401 for GSDIa expected to be submitted mid-year.
Ultragenyx has a strong cash position with $563 million in cash, cash equivalents, and marketable securities, providing financial stability for ongoing and future projects.

Negative Points

Ultragenyx reported a net loss of $151 million for Q1 2025, highlighting ongoing financial challenges despite revenue growth.
The company faces variability in revenue due to uneven ordering patterns for Crysvita in Latin America, which could impact financial predictability.
There are uncertainties related to regulatory approvals and potential delays in clinical trial readouts, which could affect the timeline for product launches.
Operating expenses remain high at $282 million for the quarter, driven by significant R&D and SG&A costs, which could pressure profitability.
The company is navigating complex pricing negotiations and reimbursement challenges in various international markets, which could impact revenue growth.

Q & A Highlights

Q: Can you explain the significance of dispersion in the setrusumab study and provide an update on the fracture reduction observed? A: Emil Kakkis, CEO, explained that variation in the annualized fracture rate at baseline is a factor, with patients having different fracture rates. The study stratifies patients to manage this variation. The Phase 2 data showed a 67% reduction in fracture frequency, which remained consistent at 14 months with a p-value of 0.01. The separation of fracture rates over time suggests a reasonable chance of success in the second interim analysis.

Q: If the study moves to a third interim read, what is the likelihood of success? A: Emil Kakkis stated that the next assessment would be the final one with a p-value threshold of 0.04, making it easier to achieve. He expressed confidence in hitting the target, citing the significant bone mineral density gains and fracture rate effects observed within two to three months of treatment.

Q: How do you view the potential impact of FDA leadership changes on rare disease approvals? A: Emil Kakkis noted that while there are concerns about FDA leadership changes, the agency has emphasized the importance of rare disease approvals. Ultragenyx is less concerned due to its strong clinical data, but the broader industry may face challenges. The company remains optimistic about the FDA's commitment to advancing rare disease treatments.

Q: How will you communicate the outcome of the interim analysis for setrusumab to the market? A: Emil Kakkis explained that if the interim analysis is positive, they will inform the market promptly. If the study continues to the final assessment, they will also communicate that. The company plans to release top-line data relatively soon after the analysis, especially if it leads to a BLA filing.

Q: What is the status of the Angelman syndrome program enrollment and global site activation? A: Eric Crombez, CMO, reported that the Angelman syndrome study is on track to fully enroll this year. Global sites, including those in the U.S., Europe, and Japan, are active and screening patients. The company is leveraging its experience to efficiently enroll this large pivotal trial.

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

This article first appeared on GuruFocus.

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