Ultragenyx Forecasts $640M-$670M in Revenue for 2025 on Robust Product Growth

Insights from the Earnings Call: Ultragenyx Pharmaceutical Inc. (RARE) First Quarter 2025 Financial Update

Management View

• CEO Emil Kakkis highlighted substantial progress in both commercial and development pipelines, emphasizing revenue growth driven by international expansion and upcoming product launches globally. He pointed out the advancements in pivotal clinical programs such as UX143 for osteogenesis imperfecta, GTX-102 for Angelman syndrome, and UX701 for Wilson disease. The UX111 BLA review remains on track with a PDUFA date of August 18, 2025.
• Chief Financial Officer Howard Horn stated that the company saw revenues of $139 million in the first quarter of 2025, marking a 28% rise compared to the same period last year. This included contributions of $103 million from Crysvita, which showed strong expansion particularly in Latin America and Turkey. Additionally, Dojolvi brought in $17 million, while Evkeeza accounted for another $11 million. The operating costs amounted to $282 million, resulting in an overall net loss of $151 million.
• Chief Commercial Officer Erik Harris detailed the strong performance of Crysvita in Latin America, where approximately 40 new start forms were generated, adding 40 patients to reimbursed therapy. The total number of patients on commercial Crysvita in the region now stands at 775. Growth in the U.S. market was supported by increasing adult demand.

Outlook

• The revenue forecast for 2025 remains unchanged at $640 million to $670 million, indicating a year-over-year increase of 14% to 20%. It is projected that Crysvita will generate revenues of $460 million to $480 million, with Dojolvi’s estimated contribution falling within the range of $90 million to $100 million. Key factors driving this growth include deeper market penetration in Latin America along with the expanded presence of Evkeeza in both European markets and Japan.
• The management restated that the net cash utilized in operations should decline in the following quarters, which supports their plan for achieving full-year GAAP profitability by 2027.

Financial Results

• In the first quarter of 2025, revenues reached $139 million, reflecting a 28 percent rise compared to the same period in 2024. Crysvita was the top performer, contributing $103 million; this includes $41 million from North America along with $55 million sourced from Latin America and Turkey. Notably, sales in Latin America and Turkey saw an impressive 52 percent surge year-on-year.
• Net loss for the quarter was $151 million or $1.57 per share. Cash and cash equivalents stood at $563 million as of March 31, 2025. Operating cash use decreased compared to prior quarters, reflecting improved expense management.

Q&A

• Yaron Werber from TD Cowen raised concerns about the inconsistency and fluctuation in fracture rates observed in the UX143 Phase 3 trial. The company’s CEO, Kakkis, acknowledged that differences in how often fractures occur between participants might affect early outcomes. However, he remained optimistic about the study's ability to ultimately achieve positive results.
• Tazeen Ahmad from Bank of America queried about the chances of achieving success during the third interim analysis should the second one fail to meet expectations. In response, CEO Kakkis expressed strong faith in surpassing either the threshold set for the second interim or the ultimate analysis.
• Gena Wang, Barclays: Inquired about the impact of potential delayed placebo fracture rates due to bisphosphonate washout. CEO Kakkis stated that the effect of setrusumab is expected to outweigh any placebo group variations.

Sentiment Analysis

• Experts expressed reservations regarding inconsistencies within the clinical trial information and potential regulatory hurdles, adopting a wary stance. Nonetheless, they recognized the firm’s strong lineup of products under development along with their effective marketing strategies.
• Throughout, management retained an assured demeanor, highlighting favorable developments in clinical trials along with stringent fiscal practices. CEO Kakkis employed assertive phrasing like "our confidence remains strong" and "we anticipate" when addressing significant benchmarks and strategic directions.

Quarter-over-Quarter Comparison

• The revenue increased from $560 million in 2024 to an estimated yearly figure between $640 million and $670 million for 2025.
• Guidance language remained consistent, with reaffirmed targets for revenue and product-specific contributions.
• Analysts' focus shifted from general pipeline updates in Q4 2024 to specific trial variability concerns in Q1 2025.

Risks and Concerns

• Clinical trial variability, particularly in the UX143 study, was highlighted as a potential risk to interim success.
• Concerns were raised regarding regulatory changes and possible postponements in the review process forBLAs.
• Analysts questioned the durability and long-term market potential of products like setrusumab and GTX-102 amid competitive pressures.

Final Takeaway

Ultragenyx demonstrated strong revenue growth and pipeline advancement in Q1 2025, supported by key product contributions and international expansion. With reaffirmed revenue guidance of $640 million to $670 million for 2025, the company remains focused on progressing late-stage clinical programs and achieving GAAP profitability by 2027. While variability in clinical trial data and regulatory uncertainties pose challenges, management's confidence and strategic execution suggest continued momentum.

Read the full Earnings Call Transcript

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