Core Viewpoint - Ultragenyx Pharmaceutical reported a wider-than-expected loss in Q1 2025, despite a year-over-year revenue increase driven by higher product sales, but missed consensus estimates for revenue [1][3]. Financial Performance - The company reported a loss of $1.57 per share, compared to a loss of $2.03 per share in the same quarter last year [1]. - Total revenues for the quarter were $139.3 million, reflecting a 28% increase year over year, but falling short of the Zacks Consensus Estimate of $142 million [1]. - Operating expenses increased to $282.2 million, up 3% year over year, attributed to investments in late-stage pipeline programs and marketing costs [4]. Product Performance - Crysvita generated revenues of $102.9 million, a 25% increase year over year, with significant growth in Latin America and Turkey [2]. - Mepsevii revenues rose 27% year over year to $8.4 million, while Dojolvi revenues increased 4% to $17 million [3]. - Evkeeza recorded sales of $11 million, showing significant growth as the drug is launched in territories outside the U.S. [3]. Financial Guidance - Ultragenyx expects total revenues for 2025 to be between $640 million and $670 million, indicating a growth of approximately 14-20% compared to 2024 [6]. - Specific revenue expectations include Crysvita between $460-$480 million (up 12-17% year over year) and Dojolvi between $90 million and $100 million (up 2-14% year over year) [6]. Pipeline Developments - The company is developing UX143 for osteogenesis imperfecta in collaboration with Mereo BioPharma, with updates expected in mid-2025 [8]. - UX701, an investigational gene therapy for Wilson disease, is in a phase I/II/III study, and a BLA submission for DTX401 is planned for mid-2025 [10]. - The pivotal phase III Aspire study for GTX-102, targeting Angelman syndrome, is ongoing, with an additional open-label study planned for 2025 [11]. - The company submitted a BLA for UX111 for Sanfilippo syndrome type A, with a decision expected on August 18, 2025 [12].

For the quarter ended March 2025, Ultragenyx (RARE) reported revenue of $139.29 million, up 28% over the same period last year. EPS came in at -$1.57, compared to -$2.03 in the year-ago quarter. As these metrics influence top- and bottom-line performance, comparing them to the year-ago numbers and what analysts estimated helps investors project a stock's price performance more accurately. Here is how Ultragenyx performed in the just reported quarter in terms of the metrics most widely monitored and projecte ...

Corporate Presentation May 2025 Confidential and Proprietary Forward looking statements Cautionary note regarding forward-looking statements: This presentation contains forward-looking statements, including, but not limited to, statements regarding our expectations, estimates, assumptions, and projections regarding our future operating results and financial performance, including our expectations for profitability in 2027, anticipated cost or expense management, plans with respect to commercializing our pro ...

Core Insights - Ultragenyx Pharmaceutical Inc. reported total revenue of $139 million for Q1 2025, reflecting a 28% increase compared to Q1 2024, with Crysvita revenue at $103 million, a 25% increase year-over-year [4][8] - The company reaffirmed its 2025 financial guidance, projecting total revenue between $640 million to $670 million, with Crysvita revenue expected to be between $460 million to $480 million, and Dojolvi revenue between $90 million to $100 million [10][12] - The investigational treatment UX111 for Sanfilippo syndrome is on track for a Biologics License Application (BLA) action date set for August 18, 2025 [14] Financial Performance - Total revenues for Q1 2025 were $139 million, up from $108.8 million in Q1 2024, with significant contributions from Crysvita and Dojolvi [3][4] - Crysvita's product sales in Latin America and Türkiye reached $55 million, marking a 52% increase compared to the previous year [4][5] - Operating expenses totaled $282 million in Q1 2025, with research and development costs at $165.8 million [6][7] Net Loss and Cash Position - The net loss for Q1 2025 was $151 million, or $1.57 per share, an improvement from a net loss of $171 million, or $2.03 per share, in Q1 2024 [8][9] - As of March 31, 2025, the company had cash, cash equivalents, and marketable securities totaling $563 million, down from $745 million at the end of 2024 [9][29] Clinical Developments - The company is progressing with multiple clinical trials, including UX143 for osteogenesis imperfecta and GTX-102 for Angelman syndrome, with key interim analyses expected in mid-2025 [10][11] - The Phase 3 study for DTX401 is anticipated to submit a BLA in mid-2025, following positive results from the GlucoGene study [15][16] Strategic Focus - Ultragenyx aims to manage expenses while prioritizing investments in upcoming commercial launches and advancing multiple Phase 3 programs, which is expected to reduce net cash used in operations in 2025 compared to 2024 [10][12]