Ultragenyx Reports Second Quarter 2025 Financial Results and Corporate Update
Ultragenyx (NASDAQ: RARE) reported Q2 2025 financial results with total revenue of $166 million, representing 13% growth year-over-year. Key revenue drivers include Crysvita at $120 million and Dojolvi at $23 million.
The company reaffirmed its 2025 guidance, expecting total revenue between $640-670 million. Despite a net loss of $115 million ($1.17 per share), Ultragenyx maintains its path to profitability target for 2027. Cash position stands at $539 million as of June 30, 2025.
Notable pipeline updates include: UX143 Phase 3 data for osteogenesis imperfecta expected by year-end, GTX-102 receiving FDA Breakthrough Therapy Designation for Angelman syndrome with Phase 3 fully enrolled, and planned BLA submission for DTX401 in Q4 2025.
Ultragenyx (NASDAQ: RARE) ha comunicato i risultati finanziari del secondo trimestre 2025 con un fatturato totale di 166 milioni di dollari, segnando una crescita del 13% rispetto all'anno precedente. I principali fattori di ricavo sono Crysvita con 120 milioni di dollari e Dojolvi con 23 milioni di dollari.
L'azienda ha confermato le previsioni per il 2025, aspettandosi un fatturato totale compreso tra 640 e 670 milioni di dollari. Nonostante una perdita netta di 115 milioni di dollari (1,17 dollari per azione), Ultragenyx mantiene l'obiettivo di raggiungere la redditività entro il 2027. La posizione di cassa al 30 giugno 2025 è di 539 milioni di dollari.
Tra gli aggiornamenti rilevanti della pipeline si segnalano: i dati di fase 3 di UX143 per l'osteogenesi imperfetta attesi entro fine anno, la designazione FDA Breakthrough Therapy per GTX-102 nella sindrome di Angelman con la fase 3 completamente arruolata, e la prevista presentazione della domanda BLA per DTX401 nel quarto trimestre 2025.
Ultragenyx (NASDAQ: RARE) informó los resultados financieros del segundo trimestre de 2025 con un ingreso total de 166 millones de dólares, lo que representa un crecimiento del 13% interanual. Los principales impulsores de ingresos incluyen Crysvita con 120 millones de dólares y Dojolvi con 23 millones de dólares.
La compañía reafirmó su guía para 2025, esperando un ingreso total entre 640 y 670 millones de dólares. A pesar de una pérdida neta de 115 millones de dólares (1.17 dólares por acción), Ultragenyx mantiene su objetivo de rentabilidad para 2027. La posición de efectivo al 30 de junio de 2025 es de 539 millones de dólares.
Actualizaciones destacadas de la cartera incluyen: datos de fase 3 de UX143 para osteogénesis imperfecta esperados para fin de año, GTX-102 recibió la designación de Terapia Innovadora de la FDA para el síndrome de Angelman con la fase 3 completamente reclutada, y la presentación planificada de BLA para DTX401 en el cuarto trimestre de 2025.
Ultragenyx (NASDAQ: RARE)는 2025년 2분기 재무 실적을 발표하며 총 매출 1억 6600만 달러로 전년 대비 13% 성장했다고 밝혔습니다. 주요 매출원은 Crysvita가 1억 2000만 달러, Dojolvi가 2300만 달러입니다.
회사는 2025년 가이던스를 재확인하며 총 매출 6억 4000만~6억 7000만 달러를 예상하고 있습니다. 순손실은 1억 1500만 달러(주당 1.17달러)였으나, Ultragenyx는 2027년 수익성 달성 목표를 유지하고 있습니다. 2025년 6월 30일 기준 현금 보유액은 5억 3900만 달러입니다.
주요 파이프라인 업데이트로는 연말까지 골형성 부전증을 위한 UX143 3상 데이터 발표 예정, 엔젤만 증후군 치료제 GTX-102가 FDA 혁신 치료제 지정 획득 및 3상 완전 등록, 2025년 4분기 DTX401의 BLA 제출 계획이 포함됩니다.
Ultragenyx (NASDAQ : RARE) a publié ses résultats financiers du deuxième trimestre 2025 avec un chiffre d'affaires total de 166 millions de dollars, soit une croissance de 13 % par rapport à l'année précédente. Les principaux moteurs de revenus sont Crysvita avec 120 millions de dollars et Dojolvi avec 23 millions de dollars.
L'entreprise a confirmé ses prévisions pour 2025, prévoyant un chiffre d'affaires total compris entre 640 et 670 millions de dollars. Malgré une perte nette de 115 millions de dollars (1,17 dollar par action), Ultragenyx maintient son objectif de rentabilité pour 2027. La position de trésorerie s'élève à 539 millions de dollars au 30 juin 2025.
Parmi les mises à jour notables du pipeline : les données de phase 3 de l’UX143 pour l'ostéogenèse imparfaite attendues d’ici la fin de l’année, la désignation Breakthrough Therapy de la FDA pour le GTX-102 dans le syndrome d'Angelman avec la phase 3 entièrement recrutée, et la soumission prévue du BLA pour le DTX401 au quatrième trimestre 2025.
Ultragenyx (NASDAQ: RARE) meldete die Finanzergebnisse für das zweite Quartal 2025 mit einem Gesamtumsatz von 166 Millionen US-Dollar, was einem Wachstum von 13 % gegenüber dem Vorjahr entspricht. Die wichtigsten Umsatztreiber sind Crysvita mit 120 Millionen US-Dollar und Dojolvi mit 23 Millionen US-Dollar.
Das Unternehmen bestätigte seine Prognose für 2025 und erwartet einen Gesamtumsatz zwischen 640 und 670 Millionen US-Dollar. Trotz eines Nettoverlusts von 115 Millionen US-Dollar (1,17 US-Dollar pro Aktie) hält Ultragenyx an seinem Ziel fest, bis 2027 profitabel zu sein. Die Barbestände beliefen sich zum 30. Juni 2025 auf 539 Millionen US-Dollar.
Wichtige Pipeline-Updates umfassen: Phase-3-Daten von UX143 für Osteogenesis imperfecta werden bis Jahresende erwartet, GTX-102 erhielt die FDA Breakthrough Therapy Designation für das Angelman-Syndrom mit vollständig eingeschriebener Phase 3, und die geplante BLA-Einreichung für DTX401 im vierten Quartal 2025.
- Revenue growth of 13% year-over-year to $166 million in Q2 2025
- Net loss improved to $115 million from $132 million in Q2 2024
- GTX-102 received FDA Breakthrough Therapy Designation for Angelman syndrome
- Phase 3 Aspire study for GTX-102 completed enrollment ahead of schedule
- UX143 demonstrated acceptable safety profile in Phase 3 interim analysis
- Operating expenses increased to $274 million from $263 million year-over-year
- Net cash used in operations increased to $275 million for H1 2025
- FDA issued Complete Response Letter for UX111 BLA requiring additional CMC information
- Net loss continues at $115 million despite revenue growth
- Increased cash burn expected compared to 2024 due to program delays
Insights
Ultragenyx shows strong revenue growth while advancing key pipeline assets, though profitability remains a 2027 target.
Ultragenyx delivered $166 million in Q2 revenue, representing 13% year-over-year growth. The company's flagship product Crysvita generated $120 million (72% of total revenue), while Dojolvi contributed $23 million. Despite this growth, the company still posted a $115 million net loss ($1.17 per share), though this represents an improvement from the $132 million loss in Q2 2024.
The quarterly cash burn rate of $108 million remains substantial, leaving $539 million in cash reserves. At the current burn rate, this provides approximately 5 quarters of runway, though management has reaffirmed their path to profitability by 2027 through revenue growth and spending prioritization.
The company's financial guidance remains unchanged, projecting full-year revenue between $640-670 million (14-20% growth over 2024). However, they've indicated that net cash used in operations will modestly increase compared to their previous guidance due to timing delays affecting several programs.
The critical value drivers remain in the pipeline. Two particularly important catalysts are approaching: Phase 3 data for UX143 in osteogenesis imperfecta expected around year-end, and the full enrollment of the Phase 3 study for GTX-102 in Angelman syndrome, which received Breakthrough Therapy Designation from the FDA. The FDA's Complete Response Letter for UX111 (Sanfilippo syndrome) represents a regulatory setback, though management characterizes the issues as "readily addressable" manufacturing concerns rather than clinical efficacy problems.
The 20% revenue growth in the first half compared to 2024 demonstrates commercial momentum, but the pathway to profitability will require both continued top-line expansion and disciplined cost management as multiple late-stage programs advance toward potential commercialization.
Ultragenyx's key pipeline assets reach critical stages with UX143 Phase 3 readouts approaching and GTX-102 earning Breakthrough status.
Ultragenyx's clinical pipeline is reaching several inflection points that could significantly transform the company's trajectory in rare disease therapeutics. The Phase 3 Orbit and Cosmic studies for UX143 in osteogenesis imperfecta (OI) have progressed well, with a Data Monitoring Committee interim analysis confirming an acceptable safety profile. Final analysis data expected around year-end 2025 will be pivotal, as positive results could position UX143 as the first approved therapy specifically targeting bone quality in OI patients.
The GTX-102 program for Angelman syndrome has gained significant momentum with two important developments: FDA Breakthrough Therapy Designation and completion of enrollment in the Phase 3 Aspire study. The Breakthrough designation acknowledges the preliminary evidence showing consistent developmental gains across multiple symptom domains in patients treated for up to 3 years. With 129 patients now enrolled across 28 global sites, the Phase 3 study is well-positioned for its data readout in H2 2026.
The regulatory setback for UX111 in Sanfilippo syndrome represents a delay rather than a program failure. The FDA's Complete Response Letter focused on chemistry, manufacturing, and controls issues rather than clinical efficacy concerns. The agency acknowledged the robustness of the neurodevelopmental outcome data, suggesting the clinical package is compelling despite the manufacturing issues.
The company is also advancing DTX401 for Glycogen Storage Disease Type Ia toward a BLA submission in Q4 2025, supported by strong 96-week data showing substantial reductions in daily cornstarch requirements (60-64%). Meanwhile, enrollment continues in the fourth cohort of the UX701 study for Wilson Disease.
Collectively, these programs represent potential breakthroughs for patients with devastating rare diseases that currently have limited or no treatment options, particularly UX143 for OI and GTX-102 for Angelman syndrome, which could become transformative therapies if their Phase 3 studies succeed.
Second quarter total revenue of
Crysvita® revenue of
Reaffirm 2025 Revenue Guidance: Total revenue between
UX143 for osteogenesis imperfecta Phase 3 data from Orbit and Cosmic studies expected around the end of the year
GTX-102 for Angelman syndrome received Breakthrough Therapy Designation from FDA;
Phase 3 Aspire study fully enrolled
NOVATO, Calif., Aug. 05, 2025 (GLOBE NEWSWIRE) -- 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, today reported its financial results for the quarter ended June 30, 2025.
“In the first half of the year, we delivered
Second Quarter 2025 Selected Financial Data Tables and Financial Results
| Revenues (dollars in thousands), (unaudited) | ||||||||||||||||
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| Crysvita | ||||||||||||||||
| Product sales – Latin America and Türkiye | $ | 34,727 | $ | 40,449 | $ | 89,807 | $ | 76,690 | ||||||||
| Royalty revenue – U.S. and Canada | 79,083 | 67,045 | 119,936 | 107,447 | ||||||||||||
| Royalty revenue – Europe | 6,596 | 6,176 | 13,528 | 12,118 | ||||||||||||
| Total Crysvita Revenue | 120,406 | 113,670 | 223,271 | 196,255 | ||||||||||||
| Dojolvi | 23,207 | 19,355 | 40,216 | 35,717 | ||||||||||||
| Evkeeza | 14,573 | 7,856 | 25,604 | 11,131 | ||||||||||||
| Mepsevii | 8,310 | 6,145 | 16,697 | 12,756 | ||||||||||||
| Total revenues | $ | 166,496 | $ | 147,026 | $ | 305,788 | $ | 255,859 | ||||||||
Total Revenues
Ultragenyx reported
| Selected Financial Data (dollars in thousands, except per share amounts), (unaudited) | ||||||||||||||||
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| Total revenues | $ | 166,496 | $ | 147,026 | $ | 305,788 | $ | 255,859 | ||||||||
| Operating expenses: | ||||||||||||||||
| Cost of sales | 23,002 | 21,280 | 51,664 | 38,813 | ||||||||||||
| Research and development | 164,736 | 161,503 | 330,508 | 339,990 | ||||||||||||
| Selling, general and administrative | 86,646 | 80,604 | 174,443 | 158,764 | ||||||||||||
| Total operating expenses | 274,384 | 263,387 | 556,615 | 537,567 | ||||||||||||
| Net loss | $ | (114,951 | ) | $ | (131,598 | ) | $ | (266,031 | ) | $ | (302,282 | ) | ||||
| Net loss per share, basic and diluted | $ | (1.17 | ) | $ | (1.52 | ) | $ | (2.73 | ) | $ | (3.54 | ) | ||||
Operating Expenses
Total operating expenses for the second quarter of 2025 were
Net Loss
For the second quarter of 2025, Ultragenyx reported net loss of
Cash Balance and Net Cash Used in Operations
Cash, cash equivalents, and marketable debt securities were
2025 Financial Guidance
Ultragenyx reaffirmed its revenue guidance for 2025. Total revenues are expected to grow approximately 14
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
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 their final analyses around the end of 2025. The randomized, placebo-controlled Phase 3 portion of the Orbit study was evaluated by the Data Monitoring Committee at an interim analysis in July 2025 and they informed the company that UX143 demonstrated an acceptable safety profile and that the study should continue to the final analysis. Conduct of the study is going well and patient safety in the Phase 3 is consistent with the Phase 2.
Data from the Cosmic study were not analyzed at the interim timepoint, consistent with the statistical analysis plan. Study conduct is going well and safety in this younger patient population is consistent with the safety profile in the other studies.
Patients will continue dosing in the ongoing Phase 3 Orbit and Cosmic clinical studies with the final analyses to be conducted after patients have been on therapy for at least 18-months. The threshold for the Phase 3 Orbit final analysis is p<0.04 and for the Phase 3 Cosmic final analysis is p<0.05.
GTX-102 an antisense oligonucleotide for Angelman syndrome: Phase 3 study fully enrolled; Phase 3 data expected in the second half of 2026
In June 2025, Breakthrough Therapy Designation (BTD) was granted by the FDA for GTX-102 as a treatment for Angelman syndrome. The FDA’s decision was based on preliminary clinical evidence including positive data from the Phase 1/2 study in 74 patients (4-17 years of age) with a full maternal UBE3A gene deletion, that showed participants have made consistent developmental gains with rapid, sustained and continuing improvements across multiple symptom domains when treated for up to 3 years. BTD aims to expedite the development and review of drugs that are intended to treat serious or life-threatening diseases and whose preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on one or more clinically significant endpoints over existing therapies.
In July 2025, enrollment of the global Phase 3 Aspire study was completed, ahead of plan due to patient and investigator interest, 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. The primary endpoint is improvement in cognition assessed by Bayley-4 cognitive raw score, and the key secondary endpoint (with a
The Phase 2/3 Aurora study, which will evaluate GTX-102 in other Angelman syndrome genotypes and ages, is expected to initiate in the second half of 2025.
UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA): Working with FDA to resolve observations from Complete Response Letter (CRL)
In July 2025, the FDA issued a CRL for the Biologics License Application (BLA) for UX111 requesting additional information and improvements related to specific aspects of chemistry, manufacturing and controls (CMC) procedures and validation as well as observations from the recently completed manufacturing facility inspections. The company believes the observations are readily addressable and many have been addressed. The company will work with the FDA through a Type A meeting to agree on the planned resolution of the observations. Once agreement on the contents of a filing have been reached, the company expects to resubmit the BLA and anticipates up to a 6-month review period to follow the resubmission.
Clinical review had been ongoing and the FDA has acknowledged that the neurodevelopmental outcome data provided to date are robust and the biomarker data provide additional supportive evidence. The CRL did not note any review issues related to the clinical data package nor clinical inspections and specified that updated clinical data for particular endpoints from the current patients be included in the resubmission.
DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): BLA submission expected in the fourth quarter of 2025
A BLA for DTX401 for the treatment of GSDIa is planned to be submitted in the fourth quarter of 2025. The BLA will include data from the randomized, placebo controlled Phase 3 study and the previously disclosed 96-week data that demonstrated patients had even greater reductions in total daily cornstarch at their last visit compared to baseline in both the ongoing DTX401 group (-
UX701 AAV gene therapy for Wilson Disease: Phase 1/2/3 study ongoing; Cohort 4 enrollment ongoing, completion expected in second half of 2025
Enrollment is ongoing in 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. The company is on track to enroll five patients in Cohort 4 who will receive 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. Enrollment in Cohort 4 is expected to complete in the second half of 2025.
Conference Call and Webcast Information
Ultragenyx will host a conference call today, Tuesday, August 5, 2025, at 2 p.m. PT/5 p.m. ET to discuss the second 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 https://ir.ultragenyx.com/events-presentations. The replay of the call will be available for three months.
About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.
Forward-Looking Statements and Use of Digital Media
Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, the components and timing of regulatory submissions, the company’s ability to provide the requested documentation and address the comments in the CRL to the satisfaction of the FDA, the timing of resubmission of the BLA and the timing of FDA review of any such resubmission, the timing and outcome of any FDA inspections related to UX111, the timing of future regulatory interactions related to UX111 are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause the company’s clinical development programs, commercial success of its products and product candidates, continued collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, risks related to serious or undesirable side effects of our product candidates, the company’s ability to achieve its projected development goals in its expected timeframes, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, our limited experience in generating revenue from product sales, risks related to product liability lawsuits, our dependence on Kyowa Kirin for the commercialization of Crysvita in certain major markets, including the U.S. and Canada, and for our commercial supply of Crysvita in those markets, fluctuations in buying or distribution patterns from distributors and specialty pharmacies, smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, our ability to successfully manage the expansion of our company, competition from other therapies or products, regulatory scrutiny of the company’s products and product candidates, the company’s limited experience as a company in operating its own manufacturing facility, market acceptance of our products, uncertainty related to insurance coverage and reimbursement, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company’s future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx’s products and drug candidate. Ultragenyx undertakes no obligation to update or revise any forward-looking statements.
For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 7, 2025, and its subsequent periodic reports filed with the SEC.
In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/).
| Ultragenyx Pharmaceutical Inc. | ||||||||||||||||
| Selected Statement of Operations Financial Data | ||||||||||||||||
| (in thousands, except share and per share amounts) | ||||||||||||||||
| (unaudited) | ||||||||||||||||
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| Statement of Operations Data: | ||||||||||||||||
| Revenues: | ||||||||||||||||
| Product sales | $ | 80,817 | $ | 73,805 | $ | 172,324 | $ | 136,294 | ||||||||
| Royalty revenue | 85,679 | 73,221 | 133,464 | 119,565 | ||||||||||||
| Total revenues | 166,496 | 147,026 | 305,788 | 255,859 | ||||||||||||
| Operating expenses: | ||||||||||||||||
| Cost of sales | 23,002 | 21,280 | 51,664 | 38,813 | ||||||||||||
| Research and development | 164,736 | 161,503 | 330,508 | 339,990 | ||||||||||||
| Selling, general and administrative | 86,646 | 80,604 | 174,443 | 158,764 | ||||||||||||
| Total operating expenses | 274,384 | 263,387 | 556,615 | 537,567 | ||||||||||||
| Loss from operations | (107,888 | ) | (116,361 | ) | (250,827 | ) | (281,708 | ) | ||||||||
| Change in fair value of equity investments | (9 | ) | (3,991 | ) | (166 | ) | (245 | ) | ||||||||
| Non-cash interest expense on liabilities for sales of future royalties | (14,041 | ) | (15,960 | ) | (28,383 | ) | (31,807 | ) | ||||||||
| Other income, net | 7,934 | 5,572 | 15,602 | 12,791 | ||||||||||||
| Loss before income taxes | (114,004 | ) | (130,740 | ) | (263,774 | ) | (300,969 | ) | ||||||||
| Provision for income taxes | (947 | ) | (858 | ) | (2,257 | ) | (1,313 | ) | ||||||||
| Net loss | $ | (114,951 | ) | $ | (131,598 | ) | $ | (266,031 | ) | $ | (302,282 | ) | ||||
| Net loss per share, basic and diluted | $ | (1.17 | ) | $ | (1.52 | ) | $ | (2.73 | ) | $ | (3.54 | ) | ||||
| Shares used in computing net loss per share, basic and diluted | 98,460,445 | 86,580,516 | 97,381,745 | 85,433,443 | ||||||||||||
| Ultragenyx Pharmaceutical Inc. Selected Activity included in Operating Expenses (in thousands) (unaudited) | ||||||||||||||||
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| Non-cash stock-based compensation | $ | 38,615 | $ | 39,363 | $ | 78,525 | $ | 76,297 | ||||||||
| Ultragenyx Pharmaceutical Inc. | ||||||||
| Selected Balance Sheet Financial Data | ||||||||
| (in thousands) | ||||||||
| (unaudited) | ||||||||
| June 30, | December 31, | |||||||
| 2025 | 2024 | |||||||
| Balance Sheet Data: | ||||||||
| Cash, cash equivalents, and marketable debt securities | $ | 539,039 | $ | 745,029 | ||||
| Working capital | 426,544 | 472,970 | ||||||
| Total assets | 1,306,265 | 1,503,456 | ||||||
| Total stockholders' equity | 151,286 | 255,297 | ||||||
Contacts Ultragenyx Pharmaceutical Inc.
Investors
Joshua Higa
ir@ultragenyx.com
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