Company Overview - Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing and commercializing novel therapies for serious rare and ultra-rare genetic diseases [3] - The company has established a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical needs [3] Management and Strategy - The management team of Ultragenyx is experienced in the development and commercialization of rare disease therapeutics [4] - The company's strategy emphasizes time- and cost-efficient drug development to deliver safe and effective therapies to patients urgently [4] Upcoming Events - Howard Horn, the chief financial officer and executive vice president of Ultragenyx, will participate in a fireside chat at Bank of America's 2025 Healthcare Conference on May 13, 2025, at 8:00 a.m. PT [1] - A live and archived webcast of the panel will be available on the company's website [2]

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 ...

Company Performance - Ultragenyx reported a quarterly loss of $1.57 per share, slightly worse than the Zacks Consensus Estimate of a loss of $1.54, but an improvement from a loss of $2.03 per share a year ago, indicating a year-over-year improvement in loss figures [1] - The company posted revenues of $139.29 million for the quarter ended March 2025, missing the Zacks Consensus Estimate by 1.90%, but showing an increase from $108.83 million in the same quarter last year [2] - Over the last four quarters, Ultragenyx has surpassed consensus EPS estimates two times and topped consensus revenue estimates three times [2] Stock Outlook - Ultragenyx shares have declined approximately 7.6% since the beginning of the year, compared to a decline of 3.9% for the S&P 500 [3] - The current consensus EPS estimate for the upcoming quarter is -$1.27 on revenues of $160.05 million, and for the current fiscal year, it is -$5.14 on revenues of $653.73 million [7] - The estimate revisions trend for Ultragenyx is mixed, resulting in a Zacks Rank 3 (Hold), suggesting the stock is expected to perform in line with the market in the near future [6] Industry Context - The Medical - Biomedical and Genetics industry, to which Ultragenyx belongs, is currently ranked in the top 31% of over 250 Zacks industries, indicating a favorable industry outlook [8] - Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can impact Ultragenyx's stock performance [5]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the quarterly period ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the transition period from to . Commission File No. 001-36276 ULTRAGENYX PHARMACEUTICAL INC. (Exact name of registrant as specified in its charter) Delaware 27-2546083 (State or other j ...

Ultragenyx Pharmaceutical (RARE) Q1 2025 Earnings Call May 06, 2025 05:00 PM ET Speaker0 Good afternoon, and welcome to the Ultragenyx First Quarter twenty twenty five Financial Results Conference Call. At this time, all participants are in a listen only mode. At the end of the prepared remarks, you will have an opportunity to ask questions during the Q and A portion of the call. It is now my pleasure to turn the call over to Joshua Higa, Vice President of Investor Relations. Speaker1 Thank you. We have iss ...

Financial Data and Key Metrics Changes - In Q1 2025, Ultragenyx reported revenue of $139 million, representing a 28% increase compared to Q1 2024 [18] - The net loss for the quarter was $151 million, or $1.57 per share, with total operating expenses of $282 million [20] - Cash, cash equivalents, and marketable securities stood at $563 million as of March 31, 2025 [20] Business Line Data and Key Metrics Changes - Crysvita generated $103 million in revenue, with contributions of $41 million from North America, $55 million from Latin America and Turkey, and $7 million from Europe, marking a 25% growth over 2024 [18] - DeJolvi contributed $17 million, consistent with its expected growth trajectory, while EVKYSA generated $11 million as demand builds [19] - Mepsevii contributed $8 million as the company continues to treat patients in this ultra-rare indication [19] Market Data and Key Metrics Changes - In Latin America and Turkey, Crysvita revenue grew by 52% over 2024, driven by successful reimbursement negotiations [18] - The U.S. market for Crysvita is expected to continue growing as the company identifies new pediatric and adult patients [11] - The EMEA region saw over 260 patients treated with DERJOVY under named patient sales, with strong demand noted particularly in France [13] Company Strategy and Development Direction - The company is focused on expanding its commercial operations globally while preparing for new product launches in the U.S. and worldwide [5] - Ultragenyx is advancing multiple late-stage programs, including UX143 for osteogenesis imperfecta and GTX-102 for Angelman syndrome, with data expected in 2026 [7][26] - The company is on track to submit two Biologics License Applications (BLAs) in 2025, with one currently under review and another expected mid-year [8][9] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence that 2025 will be a transformational year, with expectations of significant revenue growth and multiple clinical data readouts [32] - The company is optimistic about the demand for its products, particularly in Latin America and the pediatric and adult XLH markets in the U.S. [21] - Management acknowledged the evolving tariff landscape but does not expect material exposure for any products, including Crysvita [23] Other Important Information - The company is actively monitoring regulatory interactions and has scheduled inspections of manufacturing facilities and clinical sites [9] - The company expects net cash used in operations to decrease in the remaining quarters of 2025, aiming for full-year GAAP profitability by 2027 [21] Q&A Session Summary Question: Insights on setrusumab and the second interim analysis - Management discussed the variability in fracture rates among patients and the statistical methods used to analyze data, emphasizing confidence in achieving significant results [35][39] Question: Likelihood of success if the study moves to a third interim read - Management expressed confidence in hitting the p-value threshold based on observed data trends and the size of the study [46][49] Question: Impact of recent CBER nomination on the rare disease space - Management noted concerns about the nomination but emphasized the importance of maintaining accelerated approval pathways for rare diseases [51][54] Question: Enrollment status for the Angelman program - Management confirmed that all global sites for the Angelman program are active and screening patients [66][68] Question: Clarification on the timing of interim data release - Management indicated that data cleaning and analysis for the interim results would take approximately eight weeks, with a commitment to timely communication of results [90][92]

Exhibit 99.1 Contacts Ultragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Ultragenyx Reports First Quarter 2025 Financial Results and Corporate Update First quarter total revenue of $139 million, Crysvita® revenue of $103 million and Dojolvi® revenue of $17 million Reaffirm 2025 Financial Guidance: Total revenue between $640 million to $670 million, Crysvita revenue of $460 million to $480 million, and Dojolvi revenue of $90 million to $100 million UX111, an investigational treatment for ...

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]