On November 4, 2025 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, reported its financial results for the quarter ended September 30, 2025.
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"We are a global commercial company with multiple products generating meaningful growth that is expected to accelerate from anticipated launches from our late-stage clinical pipeline," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "We announced today that we bolstered our balance sheet with a royalty financing ahead of pivotal milestones expected over the next year to support multiple late-stage data readouts, multiple regulatory submissions, and multiple launches. This includes the highly anticipated phase 3 study readouts for UX143 in osteogenesis imperfecta around the end of the year."
Third Quarter 2025 Selected Financial Data Tables and Financial Results
Revenues (dollars in thousands), (unaudited)
Three Months Ended September 30, Nine Months Ended September 30,
2025 2024 2025 2024
Crysvita
Product sales – Latin America and Türkiye $ 47,003 $ 35,604 $ 136,810 $ 112,294
Royalty revenue – U.S. and Canada 57,186 55,985 177,122 163,432
Royalty revenue – Europe 7,754 6,258 21,282 18,376
Total Crysvita Revenue 111,943 97,847 335,214 294,102
Dojolvi 24,275 21,374 64,491 57,091
Evkeeza 16,717 10,657 42,321 21,788
Mepsevii 6,998 9,616 23,695 22,372
Total revenues $ 159,933 $ 139,494 $ 465,721 $ 395,353
Total Revenues
Ultragenyx reported $160 million in total revenue for the third quarter of 2025, which represents 15% growth compared to the same period in 2024. Crysvita revenue in the third quarter 2025 was $112 million, which includes product sales of $47 million from Latin America and Türkiye. Dojolvi revenue in the third quarter 2025 was $24 million. Evkeeza revenue in the third quarter 2025 was $17 million as we continue to launch in the Ultragenyx territories outside of the United States.
Selected Financial Data (dollars in thousands, except per share amounts), (unaudited)
Three Months Ended September 30, Nine Months Ended September 30,
2025 2024 2025 2024
Total revenues $ 159,933 $ 139,494 $ 465,721 $ 395,353
Operating expenses:
Cost of sales 27,991 21,021 79,655 59,834
Research and development 216,212 170,109 546,720 510,099
Selling, general and administrative 86,620 80,351 261,063 239,115
Total operating expenses 330,823 271,481 887,438 809,048
Net loss $ (180,413 ) $ (133,516 ) $ (446,444 ) $ (435,798 )
Net loss per share, basic and diluted $ (1.81 ) $ (1.40 ) $ (4.55 ) $ (4.91 )
Operating Expenses
Total operating expenses for the third quarter of 2025 were $331 million, including non-cash stock-based compensation of $37 million.
Net Loss
For the third quarter of 2025, Ultragenyx reported net loss of $180 million, or $1.81 per share basic and diluted, compared with a net loss for the third quarter of 2024 of $134 million, or $1.40 per share basic and diluted.
Cash Balance and Net Cash Used in Operations
Cash, cash equivalents, and marketable debt securities were $447 million as of September 30, 2025.
The company announced today that it received $400 million through the sale of an additional 25% of its royalty interest on the future sales of Crysvita in the United States and Canada, to OMERS. Payments to OMERS will begin in January of 2028. OMERS will also continue to receive 30% of Crysvita net sales in the U.S. and Canada following the achievement of the 2022 royalty purchase agreement transaction’s cap of 1.45 times the purchase price. Total payments to OMERS pursuant to the new agreement are capped at 1.55 times the 2025 purchase price.
For the three months ended September 30, 2025, net cash used in operations was $91 million and for the nine months ended September 30, 2025 was $366 million.
2025 Financial Guidance
Ultragenyx reaffirmed its revenue guidance for 2025. Total revenues are expected to grow approximately 14-20% compared to 2024.
Reaffirm for the full year 2025:
Total revenue to be in the range of $640 million to $670 million
Crysvita revenue to be in the range of $460 million to $480 million
Dojolvi revenue to be in the range of $90 million to $100 million
Ultragenyx also reaffirmed its net cash used in operations guidance for 2025, which is expected to modestly increase compared to 2024 and its path to full year GAAP profitability in 2027.
Recent Updates and Clinical Milestones
UX143 (setrusumab) monoclonal antibody for osteogenesis imperfecta (OI): Final analysis for Phase 3 Orbit and Cosmic studies around the end of 2025
The Phase 3 Orbit and Cosmic studies, which evaluate setrusumab in pediatric and young adult patients with OI, are progressing towards final analyses at which time patients will have been on therapy for at least 18 months. Data from these studies are expected around the end of 2025.
GTX-102 an antisense oligonucleotide for Angelman syndrome: Phase 3 Aspire study fully enrolled; Phase 3 data expected in the second half of 2026
In July 2025, enrollment of the global Phase 3 Aspire study was completed with 129 patients screened and randomized across 28 global sites. Participants are randomized 1:1 to receive GTX-102 by intrathecal injection via lumbar puncture or to the sham comparator group during the 48-week primary efficacy analysis period. Data from this study are expected in the second half of 2026.
Enrollment has begun in the supportive Phase 2/3 Aurora study, which is evaluating GTX-102 in other Angelman syndrome genotypes and ages.
UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA): expect to resubmit Biologics License Application (BLA) early in 2026
Following receipt of a Complete Response Letter (CRL), the company has had constructive formal and informal discussions with the FDA. The additional clinical data requested by the agency, and that will be included in the BLA, continues to show a durable treatment effect across multiple biomarkers and further clinical separation from natural history, while maintaining an acceptable safety profile.
The company plans to resubmit the BLA early in 2026 and will be followed by an up to 6-month review per FDA regulations.
DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): BLA rolling submission underway, expect to complete in the fourth quarter of 2025
Rolling submission of a BLA for DTX401 for the treatment of GSDIa began in August 2025. The BLA will include data from the randomized, placebo-controlled Phase 3 study that demonstrated statistically significant and clinically meaningful reductions in daily cornstarch intake compared with placebo at Week 48. It will also include longer-term data that was announced in September 2025 that demonstrated patients showed an even greater reduction in mean daily cornstarch intake in the 48-week crossover period. Both the originally treated DTX401 group (n=20) and the crossover group (n=19) who received DTX401 at Week 48 had a mean reduction in daily cornstarch intake of 61% at Week 96. Quality of life improved following treatment with DTX401 for patients in both groups as measured by the Patient Global Impression of Change (PGIC). At Week 96, improvements in disease management were reported by 83% (10/12) of patients in the DTX401 group and 95% (18/19) of patients in the crossover group.
Rolling submission of the BLA is expected to complete in the fourth quarter of 2025.
UX701 AAV gene therapy for Wilson Disease: Cohort 4 enrollment complete, data expected in the first half of 2026
In September 2025, the company completed enrollment of the fourth cohort evaluating a 4.0e13 GC/kg dose in the ongoing, dose-finding, stage of the pivotal Cyprus2+ study of UX701 for the treatment of Wilson disease. A total of five patients were enrolled in Cohort 4. These patients received immunomodulation therapy with rituximab and tacrolimus, in addition to the prophylactic oral corticosteroid regimen patients in Cohorts 1 through 3 received, prior to being dosed with UX701. Data from this study are expected in the first half of 2026.
Conference Call and Webcast Information
Ultragenyx will host a conference call today, Tuesday, November 4, 2025, at 2 p.m. PT/5 p.m. ET to discuss the third quarter 2025 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source The replay of the call will be available for three months.
(Press release, Ultragenyx Pharmaceutical, NOV 4, 2025, View Source [SID1234659374])