Ultragenyx (NASDAQ: RARE) boosts 2025 revenue and cuts 10% of staff
Rhea-AI Filing Summary
Ultragenyx Pharmaceutical reported strong 2025 revenue growth but continued heavy losses while launching a major cost-cutting plan. Total revenue reached $673 million, up 20% year over year, led by Crysvita at $481 million and Dojolvi at $96 million. Fourth-quarter revenue was $207 million, up 25% from 2024. Despite this, the company posted a 2025 net loss of $575 million, or $5.83 per share, and operating expenses of $1.2 billion, including $153 million of stock-based compensation.
Ultragenyx initiated a strategic restructuring with a 10% workforce reduction, affecting about 130 employees, and expects about $50 million in restructuring and related charges, mainly from severance and terminating UX143 manufacturing agreements. Cash, cash equivalents and marketable securities were $737 million at year-end 2025. For 2026, the company guides total revenue from current products of $730–$760 million, Crysvita revenue of $500–$520 million, and Dojolvi revenue of $100–$110 million, and reiterates a path to profitability in 2027 with a planned 38% reduction in R&D spending versus 2025 and at least a 15% combined R&D and SG&A reduction.
Positive
- Robust top-line growth: 2025 revenue rose to $673 million, up 20% year over year, with fourth-quarter revenue of $207 million increasing 25% over 2024, driven by Crysvita, Dojolvi, and Evkeeza.
- Clear profitability roadmap: Management guides 2026 revenue from current products to $730–$760 million and targets 2027 profitability with a planned 38% reduction in R&D versus 2025 and at least a 15% cut in combined R&D and SG&A.
- Strong late-stage pipeline catalysts: 2026 milestones include an anticipated DTX401 PDUFA action date in Q3 2026, pivotal Phase 3 Aspire data for GTX-102 in Angelman syndrome in the second half of 2026, and key UX701 Cyprus2+ dose-finding data.
Negative
- Persistently large losses and cash burn: Despite revenue growth, Ultragenyx recorded a 2025 net loss of $575 million and used $466 million in net cash for operations, with $1.2 billion in operating expenses.
- Workforce reduction and restructuring costs: The 10% headcount reduction affecting about 130 employees and roughly $50 million in restructuring and related charges, including UX143 manufacturing terminations, signal operational retrenchment and near-term expense overhang.
- Balance sheet pressure: Year-end 2025 cash, cash equivalents, and marketable securities of $737 million sit alongside total stockholders’ equity of negative $80 million, highlighting accumulated deficits and leverage on the capital structure.
Insights
Ultragenyx pairs strong revenue growth with deep losses and a sizable restructuring aimed at 2027 profitability.
Ultragenyx delivered 2025 revenue of
The company is responding with a strategic restructuring, including a 10% workforce reduction of roughly 130 employees and about
Management guides 2026 revenue from current products to
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
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Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
Securities registered pursuant to Section 12(b) of the Act:
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Item 2.02 Results of Operations and Financial Condition.
On February 12, 2026, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release announcing its financial results for the three months ended December 31, 2025 and for the year ended December 31, 2025 (the “Press Release”). A copy of the Press Release is furnished herewith as Exhibit 99.1.
The information set forth under Item 2.02 and in Exhibit 99.1 shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, except as shall be expressly set forth by specific reference in such filing.
Item 2.05 Costs Associated with Exit or Disposal Activities.
On February 12, 2026, the Company began implementation of a strategic restructuring plan to reduce expenses (the “Restructuring”). As part of the Restructuring, the Company is implementing a 10% workforce reduction of approximately 130 employees across the Company (the “RIF”). The Company began notifying affected employees on February 12, 2026, and expects this RIF to be substantially completed in the first half of 2026.
The Company estimates that it will incur approximately $50 million in total restructuring and restructuring-related charges, consisting primarily of (i) approximately $10 million in total for employee severance payments and other employee-related costs and (ii) approximately $40 million in total for charges related to the termination of UX143 manufacturing agreements and other related activities. The Company anticipates that substantially all of these charges will be recognized during the first half of 2026. Cash payments related to the costs are expected to be made over the same period.
The restructuring charges and the timing of the charges that the Company expects to incur in connection with the Restructuring are subject to a number of estimates and assumptions, and actual results may differ materially. The Company may also incur additional costs or charges not currently contemplated due to events that may occur as a result of, or that are associated with, the Restructuring.
Forward-Looking Statements
Except for the factual statements made herein, information contained in this Current Report on Form 8-K consists of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that involve risks, uncertainties and assumptions that are difficult to predict. Words such as “anticipates”, intends,” “expects,” “plans” and similar expressions, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. Such forward-looking statements are not guarantees of performance and actual actions or events could differ materially from those contained in such statements. These forward-looking statements include, but are not limited to, statements about the percentage reduction in organizational headcount; the timing of completion of the RIF; the Company’s expectations regarding the expected results and anticipated benefits of the Restructuring; and the Company’s estimates regarding the amount, timing and nature of the Restructuring charges. There can be no assurance that the Restructuring or the RIF will have the intended effect on the Company’s business plans and financial results, or that any anticipated charges and any anticipated cost savings associated with the Restructuring will achieve its intended benefits. In addition, the Company’s workforce reduction may be greater or less than anticipated and the workforce reduction may have an adverse impact on the Company’s business and results of operations. 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 the Company in general, see the Company’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 4, 2025, and its subsequent periodic reports filed with the SEC.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits
Exhibit No. |
Description |
99.1 |
Press Release, dated February 12, 2026. |
104 |
The cover page from the Company’s Current Report on Form 8-K dated February 12, 2026 formatted in Inline XBRL. |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
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Ultragenyx, Pharmaceutical, Inc. |
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Date: |
February 12, 2026 |
By: |
/s/ Howard Horn |
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Howard Horn |
Exhibit 99.1
Contacts Ultragenyx Pharmaceutical Inc.
Investors
Joshua Higa
ir@ultragenyx.com
Media
Jess Rowlands
media@ultragenyx.com
Ultragenyx Reports Fourth Quarter and Full Year 2025 Financial Results and Corporate Update
2025 total revenue of $673 million,
Crysvita® revenue of $481 million and Dojolvi® revenue of $96 million
2026 total revenue from current products expected to be between $730 million to $760 million
Initiated a strategic restructuring plan to significantly reduce and focus expenses and headcount, reiterate path to profitability in 2027
2026 catalysts include two potential approvals and expected pivotal Phase 3 data from the GTX-102 Phase 3 Aspire study for Angelman syndrome
NOVATO, Calif. – February 12, 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, today reported its financial results for the quarter and year ended December 31, 2025.
“The year ahead marks an important turning point for the company, as we approach two potential product launches and a pivotal data readout that, together, could significantly accelerate our commercial revenue trajectory,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “We are implementing a strategic restructuring plan to reduce our operating expenses and ensure our resources are squarely aligned with our highest-impact opportunities, while leading the future of rare disease with multiple first ever treatments.”
Fourth Quarter 2025 Selected Financial Data Tables and Financial Results
Revenues (dollars in millions), (unaudited) |
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Three Months Ended December 31, |
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Year Ended December 31, |
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2025 |
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2024 |
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2025 |
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2024 |
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Crysvita |
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Product sales - Latin America and Türkiye |
$ |
40 |
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$ |
22 |
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$ |
177 |
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$ |
135 |
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Royalty revenue - U.S. and Canada |
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97 |
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87 |
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275 |
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249 |
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Royalty revenue - Europe |
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8 |
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7 |
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29 |
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26 |
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Total Crysvita Revenue |
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145 |
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116 |
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481 |
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410 |
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Dojolvi |
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32 |
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31 |
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96 |
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88 |
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Evkeeza |
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17 |
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10 |
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59 |
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32 |
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Mepsevii |
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13 |
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8 |
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37 |
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30 |
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Total revenues |
$ |
207 |
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$ |
165 |
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$ |
673 |
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$ |
560 |
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Revenues
Ultragenyx reported $207 million in total revenue for the fourth quarter 2025, which represents 25% growth compared to the same period in 2024. Crysvita revenue in the fourth quarter 2025 was $145 million, which includes product sales of $40 million from Latin America and Türkiye. Dojolvi revenue in the fourth quarter 2025 was $32 million. Evkeeza revenue in the fourth quarter 2025 was $17 million.
Total revenue for the year ended December 31, 2025 was $673 million, which represents 20% growth compared to the prior year. Full year 2025 Crysvita revenue was $481 million, which represents 17% growth compared to the prior year. This includes product sales of $177 million from Latin America and Türkiye, which represents 31% growth compared to the prior year. Dojolvi revenue in 2025 was $96 million, which represents 9% growth compared to the prior year. Evkeeza revenue in 2025 was $59 million, which represents 84% growth compared to the prior year, as demand continues to build following launches in the company’s territories outside of the United States.
2
Selected Financial Data (dollars in millions, except per share amounts), (unaudited) |
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Three Months Ended December 31, |
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Year Ended December 31, |
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2025 |
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2024 |
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2025 |
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2024 |
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Total revenues |
$ |
207 |
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$ |
165 |
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$ |
673 |
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$ |
560 |
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Operating expenses: |
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Cost of sales |
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29 |
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17 |
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109 |
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77 |
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Research and development |
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203 |
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188 |
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750 |
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698 |
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Selling, general and administrative |
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89 |
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82 |
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349 |
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321 |
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Total operating expenses |
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321 |
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287 |
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1,208 |
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1,096 |
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Net loss |
$ |
(129 |
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$ |
(133 |
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$ |
(575 |
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$ |
(569 |
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Net loss per share, basic and diluted |
$ |
(1.29 |
) |
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$ |
(1.39 |
) |
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$ |
(5.83 |
) |
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$ |
(6.29 |
) |
Operating Expenses
Total operating expenses for the fourth quarter 2025 were $321 million. Total operating expenses for the year ended December 31, 2025 were $1.2 billion, including $153 million of non-cash stock-based compensation.
Net Loss
Net loss for the fourth quarter 2025 was $129 million, or $1.29 per share basic and diluted, compared with a net loss for the fourth quarter 2024 of $133 million, or $1.39 per share basic and diluted. Net loss for the year ended December 31, 2025 was $575 million, or $5.83 per share basic and diluted, compared with a net loss the prior year of $569 million, or $6.29 per share, basic and diluted.
Cash Balance and Net Cash Used in Operations
Cash, cash equivalents, and marketable securities were $737 million as of December 31, 2025. For the three months ended December 31, 2025, net cash used in operations was $100 million and for the year ended December 31, 2025 was $466 million.
Financial Guidance and Strategic Restructuring Plan
2026 revenue guidance
3
Strategic restructuring plan and path to profitability in 2027
Ultragenyx has initiated a strategic restructuring plan designed to reduce its headcount and expenses and focus resources on its largest value drivers. The significant reduction and partial reinvestment of expenses, and the planned growth in revenue from current and new product launches, are designed to keep the company on its path to profitability in 2027.
Today, in connection with the restructuring plan, the company announced a 10% workforce reduction, impacting approximately 130 employees.
Based on the progression of the business and the reductions from the restructuring plan:
2026 Clinical and Regulatory Catalysts
Earlier today the company received an Incomplete Response Letter (IRL) regarding its resubmitted BLA. The IRL requests additional supportive documentation related to its CRL CMC responses, which the company will provide in a resubmission.
4
Conference Call and Webcast Information
Ultragenyx will host a conference call today, Thursday, February 12, 2026, at 2 p.m. PT/5 p.m. ET to discuss the fourth quarter and full year 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, including the company’s expectations for profitability in 2027, anticipated cost or expense reductions, including the company’s expectations related to benefits and savings from the strategic restructuring plan, 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 for UX111 to the satisfaction of the FDA, the timing of FDA review of the company’s BLA submissions, the timing and outcome of any FDA inspections related to UX111 or other clinical product candidates, the timing of future regulatory interactions related to the company’s clinical product candidates are forward-looking
5
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, delays or unexpected costs and other adverse effects related to the strategic restructuring plan, 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 November 5, 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-/).
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6
Ultragenyx Pharmaceutical Inc.
Selected Statement of Operations Financial Data
(in millions, except per share amounts)
(unaudited)
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Three Months Ended December 31, |
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Year Ended December 31, |
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2025 |
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2024 |
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2025 |
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2024 |
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Statement of Operations Data: |
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Revenues: |
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Product sales |
$ |
102 |
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$ |
72 |
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$ |
369 |
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$ |
285 |
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Royalty revenue |
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105 |
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93 |
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304 |
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275 |
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Total revenues |
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207 |
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165 |
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673 |
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560 |
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Operating expenses: |
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Cost of sales |
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29 |
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17 |
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109 |
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77 |
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Research and development |
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203 |
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188 |
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750 |
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698 |
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Selling, general and administrative |
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89 |
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82 |
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349 |
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321 |
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Total operating expenses |
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321 |
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287 |
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1,208 |
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1,096 |
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Loss from operations |
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(114 |
) |
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(122 |
) |
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(535 |
) |
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(536 |
) |
Non-cash interest expense on liabilities for sales of future royalties |
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(19 |
) |
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(15 |
) |
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(62 |
) |
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(63 |
) |
Other income, net |
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5 |
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4 |
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26 |
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32 |
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Loss before income taxes |
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(128 |
) |
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(133 |
) |
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(571 |
) |
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(567 |
) |
Provision for income taxes |
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(1 |
) |
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— |
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(4 |
) |
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(2 |
) |
Net loss |
$ |
(129 |
) |
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$ |
(133 |
) |
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$ |
(575 |
) |
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$ |
(569 |
) |
Net loss per share, basic and diluted |
$ |
(1.29 |
) |
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$ |
(1.39 |
) |
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$ |
(5.83 |
) |
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$ |
(6.29 |
) |
Shares used in computing net loss per share, basic and diluted |
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99.9 |
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95.7 |
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98.6 |
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90.5 |
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Ultragenyx Pharmaceutical Inc.
Selected Activity included in Operating Expenses
(in millions)
(unaudited)
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Three Months Ended December 31, |
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Year Ended December 31, |
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2025 |
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2024 |
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2025 |
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2024 |
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Non-cash stock based compensation |
$ |
38 |
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$ |
40 |
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$ |
153 |
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$ |
158 |
|
GTX-102 clinical milestone |
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— |
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$ |
30 |
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— |
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$ |
30 |
|
7
Ultragenyx Pharmaceutical Inc.
Selected Balance Sheet Financial Data
(in millions)
(unaudited)
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December 31, |
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December 31, |
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2025 |
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2024 |
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Balance Sheet Data: |
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Cash, cash equivalents, and marketable securities |
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$ |
737 |
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$ |
745 |
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Working capital |
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567 |
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|
473 |
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Total assets |
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1,532 |
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1,503 |
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Total stockholders' equity (deficit) |
|
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(80 |
) |
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255 |
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8
FAQ
How did Ultragenyx (RARE) perform financially in 2025?
What restructuring actions is Ultragenyx (RARE) taking in 2026?
What 2026 revenue guidance did Ultragenyx (RARE) provide?
What is Ultragenyx’s (RARE) path to profitability?
What were Ultragenyx’s (RARE) cash and operating expense levels at year-end 2025?
What key clinical and regulatory milestones does Ultragenyx (RARE) expect in 2026?
Filing Exhibits & Attachments
2 documentsPress Releases
