Ultragenyx Reports Fourth Quarter and Full Year 2024 Financial Results and Corporate Update
Ultragenyx Pharmaceutical (NASDAQ: RARE) reported strong financial results for Q4 and full year 2024, with total revenue of $560 million, representing 29% growth compared to 2023. Key revenue drivers included Crysvita at $410 million (+25% YoY) and Dojolvi at $88 million (+25% YoY).
The company provided 2025 guidance projecting total revenue between $640-670 million, with Crysvita revenue of $460-480 million and Dojolvi revenue of $90-100 million. Despite revenue growth, Ultragenyx reported a net loss of $569 million ($6.29 per share) for 2024, though improved from $607 million loss in 2023.
The company ended 2024 with $745 million in cash and equivalents, with net cash used in operations of $414 million for the year. Multiple clinical milestones are expected in 2025, including a PDUFA decision for Sanfilippo syndrome gene therapy and Phase 3 results in osteogenesis imperfecta.
Ultragenyx Pharmaceutical (NASDAQ: RARE) ha riportato risultati finanziari solidi per il quarto trimestre e per l'intero anno 2024, con un fatturato totale di 560 milioni di dollari, che rappresenta una crescita del 29% rispetto al 2023. I principali motori di fatturato includevano Crysvita con 410 milioni di dollari (+25% rispetto all'anno precedente) e Dojolvi con 88 milioni di dollari (+25% rispetto all'anno precedente).
L'azienda ha fornito indicazioni per il 2025, prevedendo un fatturato totale compreso tra 640-670 milioni di dollari, con un fatturato per Crysvita di 460-480 milioni di dollari e un fatturato per Dojolvi di 90-100 milioni di dollari. Nonostante la crescita dei ricavi, Ultragenyx ha riportato una perdita netta di 569 milioni di dollari (6,29 dollari per azione) per il 2024, sebbene sia migliorata rispetto alla perdita di 607 milioni di dollari nel 2023.
L'azienda ha chiuso il 2024 con 745 milioni di dollari in contante e equivalenti, con un uso netto di contante nelle operazioni di 414 milioni di dollari per l'anno. Si prevedono molteplici traguardi clinici nel 2025, inclusa una decisione PDUFA per la terapia genica della sindrome di Sanfilippo e risultati di fase 3 nell'osteogenesi imperfetta.
Ultragenyx Pharmaceutical (NASDAQ: RARE) reportó resultados financieros sólidos para el cuarto trimestre y el año completo 2024, con ingresos totales de 560 millones de dólares, lo que representa un crecimiento del 29% en comparación con 2023. Los principales impulsores de ingresos incluyeron Crysvita con 410 millones de dólares (+25% interanual) y Dojolvi con 88 millones de dólares (+25% interanual).
La compañía proporcionó orientación para 2025 proyectando ingresos totales entre 640-670 millones de dólares, con ingresos de Crysvita de 460-480 millones de dólares y de Dojolvi de 90-100 millones de dólares. A pesar del crecimiento de los ingresos, Ultragenyx reportó una pérdida neta de 569 millones de dólares (6,29 dólares por acción) para 2024, aunque mejoró respecto a la pérdida de 607 millones de dólares en 2023.
La compañía terminó 2024 con 745 millones de dólares en efectivo y equivalentes, con un uso neto de efectivo en operaciones de 414 millones de dólares para el año. Se esperan múltiples hitos clínicos en 2025, incluida una decisión PDUFA para la terapia génica de la síndrome de Sanfilippo y resultados de fase 3 en osteogénesis imperfecta.
Ultragenyx Pharmaceutical (NASDAQ: RARE)는 2024년 4분기 및 연간 강력한 재무 결과를 보고했으며, 총 수익은 5억 6천만 달러로 2023년 대비 29% 성장했습니다. 주요 수익원은 Crysvita가 4억 1천만 달러(+25% 전년 대비)와 Dojolvi가 8천 8백만 달러(+25% 전년 대비)를 포함했습니다.
회사는 2025년 가이던스를 제공하며 총 수익을 6억 4천-6억 7천만 달러로 예상하고, Crysvita의 수익은 4억 6천-4억 8천만 달러, Dojolvi의 수익은 9천-1억 달러로 예측했습니다. 수익 성장에도 불구하고, Ultragenyx는 2024년에 5억 6천 9백만 달러(주당 6.29달러)의 순손실을 기록했지만, 이는 2023년의 6억 6070만 달러 손실보다 개선된 수치입니다.
회사는 2024년을 7억 4천 5백만 달러의 현금 및 현금성 자산으로 마감했으며, 연간 운영에서 사용한 순현금은 4억 1천 4백만 달러입니다. 2025년에는 산필리포 증후군 유전자 치료에 대한 PDUFA 결정 및 골형성 부전증에 대한 3상 결과를 포함한 여러 임상 이정표가 예상됩니다.
Ultragenyx Pharmaceutical (NASDAQ: RARE) a annoncé de solides résultats financiers pour le quatrième trimestre et l'année entière 2024, avec un chiffre d'affaires total de 560 millions de dollars, représentant une croissance de 29 % par rapport à 2023. Les principaux moteurs de revenus comprenaient Crysvita avec 410 millions de dollars (+25 % en glissement annuel) et Dojolvi avec 88 millions de dollars (+25 % en glissement annuel).
L'entreprise a fourni des prévisions pour 2025, projetant un chiffre d'affaires total entre 640-670 millions de dollars, avec des revenus de Crysvita de 460-480 millions de dollars et des revenus de Dojolvi de 90-100 millions de dollars. Malgré la croissance des revenus, Ultragenyx a enregistré une perte nette de 569 millions de dollars (6,29 dollars par action) pour 2024, bien que cela soit une amélioration par rapport à une perte de 607 millions de dollars en 2023.
L'entreprise a terminé 2024 avec 745 millions de dollars en liquidités et équivalents, avec un usage net de liquidités dans les opérations de 414 millions de dollars pour l'année. Plusieurs jalons cliniques sont attendus en 2025, y compris une décision PDUFA pour la thérapie génique de la syndrome de Sanfilippo et des résultats de phase 3 dans l'ostéogenèse imparfaite.
Ultragenyx Pharmaceutical (NASDAQ: RARE) hat starke finanzielle Ergebnisse für das vierte Quartal und das gesamte Jahr 2024 gemeldet, mit einem Gesamtumsatz von 560 Millionen US-Dollar, was einem Wachstum von 29 % im Vergleich zu 2023 entspricht. Zu den wichtigsten Umsatztreibern gehörten Crysvita mit 410 Millionen US-Dollar (+25 % im Jahresvergleich) und Dojolvi mit 88 Millionen US-Dollar (+25 % im Jahresvergleich).
Das Unternehmen gab eine Prognose für 2025 ab, die einen Gesamtumsatz zwischen 640-670 Millionen US-Dollar projiziert, mit einem Umsatz von Crysvita von 460-480 Millionen US-Dollar und einem Umsatz von Dojolvi von 90-100 Millionen US-Dollar. Trotz des Umsatzwachstums meldete Ultragenyx einen Nettoverlust von 569 Millionen US-Dollar (6,29 US-Dollar pro Aktie) für 2024, was jedoch eine Verbesserung gegenüber einem Verlust von 607 Millionen US-Dollar im Jahr 2023 darstellt.
Das Unternehmen schloss das Jahr 2024 mit 745 Millionen US-Dollar an Bargeld und Äquivalenten ab, wobei im Laufe des Jahres netto 414 Millionen US-Dollar für den Betrieb verwendet wurden. Im Jahr 2025 werden mehrere klinische Meilensteine erwartet, darunter eine PDUFA-Entscheidung für die Gentherapie bei Sanfilippo-Syndrom und Ergebnisse der Phase 3 bei Osteogenesis imperfecta.
- Total revenue grew 29% YoY to $560 million in 2024
- Crysvita revenue increased 25% to $410 million
- Dojolvi revenue grew 25% to $88 million
- Strong 2025 guidance projecting 14-20% revenue growth
- Cash position remains solid at $745 million
- Net loss of $569 million in 2024
- Operating expenses increased to $1.096 billion in 2024
- Significant cash burn with $414 million used in operations
Insights
The financial results reveal a company executing well on its commercial strategy while maintaining a strong development pipeline. The 29% year-over-year revenue growth to
Several key metrics warrant attention:
- Gross margin remains healthy at
86% ($76.7 million cost of sales on$560 million revenue) - R&D expenses of
$697.9 million reflect significant investment in the pipeline, representing125% of revenue - Operating loss improved slightly, but the company still faces substantial cash burn with
$414 million used in operations during 2024
The 2025 guidance of
The pipeline progress, including potential approvals in Sanfilippo syndrome and GSDIa, could significantly impact the financial trajectory. These catalysts, combined with geographic expansion of existing products, suggest potential for accelerated revenue growth and improved operational leverage in the medium term.
The reduction in net loss per share from
The clinical pipeline demonstrates remarkable breadth and strategic positioning in rare diseases, with multiple potential value-driving catalysts in 2025:
- UX111 for Sanfilippo syndrome represents the nearest-term opportunity, with PDUFA decision expected in H2 2025. The recently presented data showing statistically significant improvements in Bayley-III scores with corresponding biomarker changes suggests a strong probability of approval.
- The Phase 3 Orbit study for UX143 in osteogenesis imperfecta approaches a critical interim analysis in mid-2025. The dual-track approach with both Orbit and Cosmic studies provides comprehensive data in different patient populations.
- GTX-102's Phase 3 program in Angelman syndrome employs a sophisticated trial design with the Multi-domain Responder Index across five domains, potentially setting a new standard for CNS disorder clinical trials.
The gene therapy portfolio shows particular promise:
- DTX401 for GSDIa achieved its primary endpoint with meaningful cornstarch reduction, demonstrating the platform's ability to address metabolic disorders
- DTX301 for OTC deficiency completed Phase 3 enrollment with a well-defined endpoint strategy
- UX701 for Wilson Disease continues optimization with an innovative approach to immunomodulation
The company's strategic focus on rare and ultra-rare diseases, combined with a mix of modalities (antibodies, antisense oligonucleotides, and gene therapy), creates multiple shots on goal while maintaining leadership in specific disease areas. The emphasis on biomarker-driven development and innovative trial designs suggests a sophisticated approach to rare disease drug development.
2024 Total Revenue of
Crysvita® revenue of
2025 Financial Guidance: Total Revenue between
NOVATO, Calif., Feb. 13, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today reported its financial results for the quarter and full year ended December 31, 2024 and shared financial guidance for 2025.
“We have created a next-generation rare disease company on a pathway to profitability with meaningful revenue growth from multiple global products and a series of potential new drug approvals,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “The major regulatory and clinical catalysts ahead of us this year are the pending PDUFA decision for our gene therapy to treat Sanfilippo syndrome, submission of our second gene therapy biologics license application in Glycogen Storage Disease Type Ia, readout of our pivotal Phase 3 results in osteogenesis imperfecta, and completion of enrollment in our Phase 3 trial in Angelman syndrome."
Fourth Quarter and Full Year 2024 Selected Financial Data Tables and Financial Results
| Revenues (dollars in thousands), (unaudited) | |||||||||||||||
| Three Months Ended December 31, | Year Ended December 31, | ||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||
| Crysvita | |||||||||||||||
| Product sales | $ | 22,415 | $ | 18,379 | $ | 134,709 | $ | 75,697 | |||||||
| Revenue in Profit-Share Territory | 85,534 | 70,124 | 248,966 | 231,574 | |||||||||||
| Royalty revenue in European Territory | 7,473 | 5,612 | 25,849 | 20,783 | |||||||||||
| Total Crysvita Revenue | 115,422 | 94,115 | 409,524 | 328,054 | |||||||||||
| Dojolvi | 31,103 | 23,286 | 88,194 | 70,633 | |||||||||||
| Evkeeza | 10,374 | 2,102 | 32,162 | 3,642 | |||||||||||
| Mepsevii | 7,978 | 7,889 | 30,350 | 30,441 | |||||||||||
| Other | — | — | — | 1,479 | |||||||||||
| Total revenues | $ | 164,877 | $ | 127,392 | $ | 560,230 | $ | 434,249 | |||||||
Total Revenues
Ultragenyx reported
Total revenue for the year ended December 31, 2024 was
| Selected Financial Data (dollars in thousands, except per share amounts), (unaudited) | |||||||||||||||
| Three Months Ended December 31, | Year Ended December 31, | ||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||
| Total revenues | $ | 164,877 | $ | 127,392 | $ | 560,230 | $ | 434,249 | |||||||
| Operating expenses: | |||||||||||||||
| Cost of sales | 16,894 | 12,051 | 76,728 | 45,209 | |||||||||||
| Research and development | 187,766 | 160,557 | 697,865 | 648,449 | |||||||||||
| Selling, general and administrative | 82,495 | 76,833 | 321,610 | 309,799 | |||||||||||
| Total operating expenses | 287,155 | 249,441 | 1,096,203 | 1,003,457 | |||||||||||
| Net loss | $ | (133,385 | ) | $ | (123,190 | ) | $ | (569,183 | ) | $ | (606,639 | ) | |||
| Net loss per share, basic and diluted | $ | (1.39 | ) | $ | (1.52 | ) | $ | (6.29 | ) | $ | (8.25 | ) | |||
Operating Expenses
Total operating expenses for the fourth quarter of 2024 were
Net Loss
For the fourth quarter of 2024, Ultragenyx reported net loss of
Cash Balance and Net Cash Used in Operations
Cash, cash equivalents, and marketable debt securities were
2025 Financial Guidance
Revenues are expected to grow approximately 14
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): Phase 3 Orbit study progressing to second interim analysis (IA2) expected in mid-2025
Patients continue dosing in the ongoing Phase 3 Orbit and Cosmic clinical trials, which evaluate setrusumab in pediatric and young adult patients with OI. The randomized, placebo-controlled Phase 3 portion of the Orbit study is progressing towards the second interim analysis in mid-2025 or a final analysis in the fourth quarter 2025. Conduct of the study is going well and patient safety in the Phase 3 is consistent with the Phase 2. Patients in the Cosmic study also are continuing to be treated with either setrusumab or intravenous bisphosphonates (IV-BP) therapy and will be evaluated in parallel with the Orbit interim and final analyses.
GTX-102 an antisense oligonucleotide for Angelman syndrome: Phase 3 study enrolling; expect enrollment completion in second half of 2025
Enrollment in the global Phase 3 Aspire study began in December 2024 and is expected to enroll approximately 120 children ages four to 17 with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. Participants will be 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 will be 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 2025.
UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA): Biologics license application (BLA) submitted; expect Prescription Drug User Fee Act (PDUFA) decision on the application and potential launch in second half of 2025
In December 2024, Ultragenyx submitted a BLA to the U.S. Food and Drug Administration for UX111 supported by the available data, including from the ongoing pivotal Transpher A study. New clinical data were recently presented at WORLDSymposium™ 2025, that demonstrated treatment with UX111 led to a statistically significant improvement in the Bayley-III raw scores for the subdomains of cognition, receptive communication and expressive communication in patients with MPS IIIA compared to natural history data from untreated patients. These clinical endpoints were correlated with substantial and sustained reduction in levels of heparan sulfate in cerebrospinal fluid. A PDUFA decision and potential launch are expected in the second half of 2025.
DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): BLA filing expected in mid-2025
Ultragenyx previously announced positive topline results from the Phase 3 GlucoGene study for the treatment of patients aged eight years and older. The study achieved its primary endpoint, demonstrating that treatment with DTX401 resulted in a statistically significant and clinically meaningful reduction in daily cornstarch intake compared with placebo at Week 48.
After the 48-week primary efficacy analysis period, crossover patients (previously treated with placebo) were eligible to receive DTX401. These patients were able to titrate cornstarch much more rapidly once they were confirmed to have been treated and had timely direct access to their glucose levels. Patients from the original DTX401 treatment arm who have reached 78 weeks also continued to reduce their daily cornstarch intake, while maintaining glycemic control. DTX401 has demonstrated a consistent and acceptable safety profile with no new safety concerns identified as of the data cut-off.
These results have been discussed with regulatory authorities in a pre-BLA meeting and will be included as part of a BLA submission in mid-2025.
DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency: Enrollment in Phase 3 study complete
Enrollment in the Phase 3 Enh3ance study has been completed with a total of 37 patients. The pivotal study enrolled participants 12 years of age and older, randomized 1:1 to DTX301 or placebo. The primary endpoints are response as measured by change in 24-hour ammonia levels and removal of ammonia-scavenger medications and protein-restricted diet. Based on the recently amended protocol, the change in 24-hour ammonia levels will be measured through Week 36 after which the study would unblind and patients will be followed for a total of up to 64 weeks to determine the complete responders able to remove safely both ammonia-scavenger medications and protein-restricted diet control.
UX701 AAV gene therapy for Wilson Disease: Phase 1/2/3 study ongoing; expect Cohort 4 enrollment completion in second half of 2025
In Stage 1 of the Phase 1/2/3 Cyprus2+ study, 15 patients across three sequential dose cohorts were enrolled and demonstrated clinical activity as well as improvements in copper metabolism. Multiple responders completely tapered off their standard-of-care treatment with responses seen in all three dose cohorts.
The company expects to enroll a fourth cohort in Stage 1 at a moderately increased dose and with an optimized immunomodulation regimen to enhance the efficiency and efficacy of the gene therapy, with the objective of having the majority of patients come off standard-of-care treatment before selecting a dose for the randomized placebo-controlled stage of the study. 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, Thursday, February 13, 2025, at 2 p.m. PT/5 p.m. ET to discuss the fourth quarter and full year 2024 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 ultrarare 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, and the components and timing of regulatory submissions 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 commercial supply of Crysvita, fluctuations in buying or distribution patterns from distributors and specialty pharmacies, the transition back to Kyowa Kirin of our exclusive rights to promote Crysvita in the United States and Canada and unexpected costs, delays, difficulties or adverse impact to revenue related to such transition, smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, competition from other therapies or products, 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 November 6, 2024, 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 December 31, | Year Ended December 31, | ||||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||||
| Statement of Operations Data: | |||||||||||||||||
| Revenues: | |||||||||||||||||
| Product sales | $ | 71,870 | $ | 51,656 | $ | 285,415 | $ | 180,413 | |||||||||
| Royalty revenue | 93,007 | 75,736 | 274,815 | 182,652 | |||||||||||||
| Collaboration and license | — | — | — | 71,184 | |||||||||||||
| Total revenues | 164,877 | 127,392 | 560,230 | 434,249 | |||||||||||||
| Operating expenses: | |||||||||||||||||
| Cost of sales | 16,894 | 12,051 | 76,728 | 45,209 | |||||||||||||
| Research and development | 187,766 | 160,557 | 697,865 | 648,449 | |||||||||||||
| Selling, general and administrative | 82,495 | 76,833 | 321,610 | 309,799 | |||||||||||||
| Total operating expenses | 287,155 | 249,441 | 1,096,203 | 1,003,457 | |||||||||||||
| Loss from operations | (122,278 | ) | (122,049 | ) | (535,973 | ) | (569,208 | ) | |||||||||
| Change in fair value of equity investments | (1,548 | ) | 1,889 | (1,115 | ) | 397 | |||||||||||
| Non-cash interest expense on liabilities for sales of future royalties | (15,522 | ) | (17,328 | ) | (63,041 | ) | (66,004 | ) | |||||||||
| Other income, net | 5,944 | 10,596 | 32,543 | 26,351 | |||||||||||||
| Loss before income taxes | (133,404 | ) | (126,892 | ) | (567,586 | ) | (608,464 | ) | |||||||||
| Benefit from (provision for) income taxes | 19 | 3,702 | (1,597 | ) | 1,825 | ||||||||||||
| Net loss | $ | (133,385 | ) | $ | (123,190 | ) | $ | (569,183 | ) | $ | (606,639 | ) | |||||
| Net loss per share, basic and diluted | $ | (1.39 | ) | $ | (1.52 | ) | $ | (6.29 | ) | $ | (8.25 | ) | |||||
| Shares used in computing net loss per share, basic and diluted | 95,681,451 | 81,118,873 | 90,538,118 | 73,543,862 | |||||||||||||
| Ultragenyx Pharmaceutical Inc. Selected Activity included in Operating Expense (in thousands) (unaudited) | |||||||||||||||
| Three Months Ended December 31, | Year Ended December 31, | ||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||
| Non-cash stock based compensation | $ | 40,190 | $ | 33,744 | $ | 158,056 | $ | 135,213 | |||||||
| GTX-102 clinical milestone | $ | 30,000 | — | $ | 30,000 | — | |||||||||
| UX143 clinical milestone | — | — | — | $ | 9,000 | ||||||||||
| Ultragenyx Pharmaceutical Inc. | ||||||||
| Selected Balance Sheet Financial Data | ||||||||
| (in thousands) | ||||||||
| (unaudited) | ||||||||
| December 31, | December 31, | |||||||
| 2024 | 2023 | |||||||
| Balance Sheet Data: | ||||||||
| Cash, cash equivalents, and marketable debt securities | $ | 745,029 | $ | 777,110 | ||||
| Working capital | 472,970 | 451,747 | ||||||
| Total assets | 1,503,456 | 1,491,013 | ||||||
| Total stockholders' equity | 255,297 | 275,414 | ||||||
Contacts Ultragenyx Pharmaceutical Inc.
Investors
Joshua Higa
ir@ultragenyx.com
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