STOCK TITAN

Ultragenyx expects approximately 20% workforce cut

The planned approximately 20% workforce reduction is expected to be substantially complete by year-end 2026, with approximately $25 million in charges.

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(High)

Sentiment and the balance of points

Rhea-AI Sentiment reads the wording of the document, how positive or negative its language is on a 1 to 5 scale. The balance of points shown with the takes weighs what the document actually discloses, so the two can disagree, for example when a trial that missed its main goal is described in upbeat language.

Form Type
8-K

Rhea-AI Filing Summary

Ultragenyx Pharmaceutical Inc. (RARE) reported third-quarter 2026 revenue of $173 million, compared with $160 million a year earlier, and a net loss of $118 million, versus $180 million. CRYSVITA revenue was $109 million and DOJOLVI revenue was $30 million. The company reaffirmed 2026 total revenue guidance of $730 million to $760 million, excluding revenue from new product launches. Cash, cash equivalents and marketable securities were $401 million as of September 30, 2026.

Ultragenyx completed the GENGLYCOS priority review voucher sale for $210 million in gross proceeds; 20% of the gross proceeds will be paid to NIH. Agreements for both vouchers provide a combined sale price of $413 million; the $203 million FAYUVI sale is expected to close later in November, and net proceeds from both sales are expected to be approximately $350 million. The company expects a workforce reduction of approximately 20%, approximately $25 million in restructuring charges, and substantial completion by year-end 2026. It expects 2027 R&D expenses below $350 million and SG&A expenses below $375 million, and reaffirmed full-year GAAP profitability in 2027.

4 points · 1 major

How this balance works

Rhea-AI gives every point it takes from this document a weight. Minor counts 1, Moderate 3 and Major 9, so one Major point outweighs several Minor ones. The bar adds up the weights on each side, and when neither side holds more than 65% of the total the balance reads Mixed.

It reads the document as published, with the same rules for every company, and it does not look at what the market expected or at how the stock traded, so a point can be objectively good on a day the stock falls.

Rhea-AI Sentiment measures something else, the tone of the wording.

0 major · 1 point

Hollow bars mark forward-looking points. How the balance works

Positive

  • Major point. Forward-looking: it has not happened yet and may not happen.Ultragenyx expects at least a 50% reduction in 2027 R&D expenses versus 2025.
  • Moderate pointThird-quarter revenue reached $173 million, versus $160 million in 2025.
  • Moderate pointQuarterly net loss narrowed to $118 million from $180 million.
  • Moderate point. Forward-looking: it has not happened yet and may not happen.Ultragenyx reaffirmed expected GAAP profitability in 2027.

Negative

  • Moderate point. Forward-looking: it has not happened yet and may not happen.Expected restructuring includes an approximately 20% workforce reduction and approximately $25 million in charges. 1.6% of market cap

Filing Explained

Substantially all restructuring charges are expected to be recognized in Q4 2026, with cash payments generally expected over the same period; actual costs may differ, and additional charges may arise.

Item 2.01 Completion of Acquisition or Disposition of Assets Financial
The company completed a significant acquisition or sale of business assets.
Item 2.02 Results of Operations and Financial Condition Financial
Disclosure of earnings results, typically an earnings press release or preliminary financials.
Item 2.05 Costs Associated with Exit or Disposal Activities Financial
The company committed to an exit plan involving layoffs, facility closures, or restructuring charges.
Item 9.01 Financial Statements and Exhibits Exhibits
Financial statements, pro forma financial information, or exhibit attachments filed with this report.
Third-quarter total revenue $173 million Three months ended September 30, 2026; $160 million in 2025
Third-quarter net loss $118 million loss Three months ended September 30, 2026; $180 million loss in 2025
Cash, cash equivalents, and marketable securities $401 million As of September 30, 2026
GENGLYCOS voucher gross proceeds $210 million Sale completed October 30, 2026
Combined price for two voucher sales $413 million Agreements for the GENGLYCOS and FAYUVI vouchers
Estimated restructuring and related charges Approximately $25 million Substantially all expected to be recognized in the fourth quarter of 2026
Expected workforce reduction Approximately 20% of employees Expected to be substantially complete by the end of 2026
2027 R&D expenses Less than $350 million Company expectation for 2027
Priority Review Voucher regulatory
"Rare Pediatric Disease Priority Review Voucher"
A priority review voucher is a transferable regulatory incentive that lets a company move a future drug or device application to the front of the review line, shortening the review period by several months. For investors it matters because the voucher can speed up market access for a high-value product or be sold to other companies for significant cash, acting like a tradable fast-pass that can accelerate revenue or create immediate financial upside.
accelerated approval regulatory
"FDA granted accelerated approval for GENGLYCOS"
Accelerated approval is a process that allows new medical treatments to be approved more quickly than usual if they address serious or life-threatening conditions and show promising early results. For investors, it signals that a treatment may reach the market sooner, potentially boosting a company's prospects, but it also involves some uncertainty since full evidence of effectiveness is still being gathered.
non-cash stock-based compensation financial
"including $34 million of non-cash stock-based compensation"
Non-cash stock-based compensation is pay given to employees or directors in the form of company shares or rights to buy shares instead of cash; it shows up on financial statements as an accounting charge even though no cash leaves the company. It matters to investors because it can lower reported profits and increase the number of shares outstanding—like paying with coupons instead of cash—affecting earnings per share and ownership dilution even though the company keeps its cash.
placebo-controlled study medical
"Phase 3 Enh3ance placebo-controlled study"
A placebo-controlled study is a clinical trial where one group of participants receives the experimental treatment and another group receives a placebo — an inactive pill or procedure that looks the same but has no therapeutic effect. Comparing results between the two groups helps show whether the treatment itself causes the observed effects rather than expectations or chance; investors view these trials as stronger evidence of a drug’s effectiveness, similar to comparing a new tool against a blank control in an experiment.
Total revenue $173 million Compared with $160 million in the third quarter of 2025
Net loss $118 million loss Compared with $180 million loss in the third quarter of 2025
Research and development expenses $154 million Compared with $216 million in the third quarter of 2025
Total operating expenses $283 million Compared with $331 million in the third quarter of 2025
Guidance

Reaffirmed 2026 total revenue guidance of $730 million to $760 million, excluding revenue from new product launches; 2026 CRYSVITA revenue guidance of $500 million to $520 million and DOJOLVI revenue guidance of $100 million to $110 million. Ultragenyx expects 2027 R&D expenses below $350 million, SG&A expenses below $375 million, and full-year GAAP profitability in 2027.

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What were RARE's third-quarter 2026 revenue and net loss?

Ultragenyx reported revenue of $173 million and a net loss of $118 million for the three months ended September 30, 2026. In the comparable 2025 quarter, revenue was $160 million and net loss was $180 million.

How much did RARE receive from the GENGLYCOS priority review voucher sale?

Ultragenyx received $210 million in gross proceeds when the GENGLYCOS voucher sale closed on October 30, 2026. Under the patent license agreement with NIH, 20% of the gross proceeds will be paid to NIH.

When will RARE complete its workforce reduction, and what charges are expected?

Ultragenyx expects the reduction, which covers approximately 20% of employees, to be substantially complete by the end of 2026. It estimates approximately $25 million in restructuring and related charges, primarily severance and other employee-related costs, with substantially all expected to be recognized in the fourth quarter of 2026.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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false000151567300015156732026-10-302026-10-30

 

 

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

Date of Report (Date of earliest event reported): October 30, 2026

 

 

Ultragenyx Pharmaceutical Inc.

(Exact name of Registrant as Specified in Its Charter)

 

 

Delaware

001-36276

27-2546083

(State or Other Jurisdiction
of Incorporation)

(Commission File Number)

(IRS Employer
Identification No.)

 

 

 

 

 

60 Leveroni Court

 

Novato, California

 

94949

(Address of Principal Executive Offices)

 

(Zip Code)

 

Registrant’s Telephone Number, Including Area Code: 415 483-8800

 

 

(Former Name or Former Address, if Changed Since Last Report)

 

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:

☐Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
☐Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
☐Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
☐Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:


Title of each class

 

Trading
Symbol(s)

 


Name of each exchange on which registered

Common Stock, $0.001 par value

 

RARE

 

Nasdaq Global Select Market

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 2.01 Completion of Acquisition or Disposition of Assets.

On October 30, 2026, Ultragenyx Pharmaceutical Inc. (the “Company”) completed the previously disclosed sale (the “PRV Sale”) of its Rare Pediatric Disease Priority Review Voucher (“PRV”) to the buyer. The Company was awarded the PRV by the U.S. Food and Drug Administration (“FDA”) on August 19, 2026 upon approval of GENGLYCOS™ (pariglasgene brecaparvovec-opnr), also known as DTX401, in adult and pediatric patients eight years and older with glycogen storage disease type Ia (GSDIa).

 

The PRV Sale was completed pursuant to the terms of an asset purchase agreement dated October 6, 2026 (the “PRV Asset Purchase Agreement”). Pursuant to the PRV Asset Purchase Agreement, the Company received gross proceeds of $210 million from the buyer upon the closing of the PRV Sale. Pursuant to the terms of a patent license agreement with the U.S. Department of Health (the “NIH”) dated December 10, 2018, 20% of the gross proceeds from the PRV Sale will be paid by the Company to the NIH.

The foregoing description of the PRV Asset Purchase Agreement does not purport to be complete and is qualified in its entirety by the full text of the PRV Asset Purchase Agreement, a copy of which will be filed with the Company’s Annual Report on Form 10-K for the year ended December 31, 2026.

 

Item 2.02 Results of Operations and Financial Condition.

 

On November 3, 2026, the Company issued a press release announcing its financial results for the three months ended September 30, 2026 (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 November 3, 2026, the Company announced a restructuring plan to reduce expenses (the “Restructuring”). As part of the Restructuring, the Company is implementing a workforce reduction of approximately 20% of its employees (the “RIF”). The Company communicated the RIF on November 3, 2026 and expects the RIF to be substantially completed by the end of 2026.

The Company estimates that it will incur approximately $25 million in total restructuring and restructuring-related charges, consisting primarily of employee severance payments and other employee-related costs. The Company anticipates that substantially all of these charges will be recognized during the fourth quarter 2026. Cash payments related to the costs are generally 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 August 5, 2026 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 November 3, 2026.

104

The cover page from the Company’s Current Report on Form 8-K dated October 30, 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.

 

 

 

Ultragenyx Pharmaceutical Inc.

 

 

 

 

Date:

November 3, 2026

By:

/s/ Howard Horn

 

 

 

Howard Horn
Executive Vice President, Chief Financial Officer, Corporate Strategy

 

 


Exhibit 99.1

img45776771_0.jpg

Contacts Ultragenyx Pharmaceutical Inc.

Investors

Joshua Higa

ir@ultragenyx.com

 

Media
Jess Rowlands
media@ultragenyx.com

 

Ultragenyx Reports Third Quarter 2026 Financial Results, Announces Substantial Expense Reductions, and Reaffirms Profitability in 2027

Third quarter total revenue of $173 million,

CRYSVITA® revenue of $109 million and DOJOLVI® revenue of $30 million

 

GENGLYCOS™ and FAYUVI™ BLAs approved by FDA in the third quarter

Two Priority Review Vouchers (PRVs) under agreement for combined sales price of $413 million

 

Substantial reduction of expenses and headcount underway, expected to drive at least a 50% reduction in 2027 R&D expenses vs 2025

 

NOVATO, Calif. – November 03, 2026 – 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 September 30, 2026, announced substantial expense reductions, and reaffirmed its guidance for 2026 revenue and 2027 profitability.

 

“We received two BLA approvals from the FDA within one month for our first gene therapy treatments and the launches are progressing well with growing demand for these products,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “With substantial reductions in headcount and expenses, combined with two launches and continued growth in our base business, we expect meaningful profitability in 2027 and going forward.”

 

Third Quarter 2026 Revenue Highlights and 2026 Revenue Guidance

•
Total revenue in the third quarter 2026 was $173 million. The company reaffirms its full year 2026 total revenue guidance of $730 million to $760 million, which excludes revenue from new product launches.
•
CRYSVITA revenue in the third quarter 2026 was $109 million, consistent with expected ordering patterns. The company reaffirms its full year 2026 Crysvita revenue guidance of $500 million to $520 million.
•
DOJOLVI revenue in the third quarter 2026 was $30 million. The company reaffirms its full year 2026 Dojolvi revenue guidance of $100 million to $110 million.
•
EVKEEZA® revenue in the third quarter 2026 was $24 million, driven by increased demand from new country launches and early access.
•
MEPSEVII® revenue in the third quarter 2026 was $10 million.

 

 


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Summary of Third Quarter 2026 Financial Results

Selected Financial Data (dollars in millions, except per share amounts), (unaudited)

 

 

Three Months Ended September 30,

 

Nine Months Ended September 30,

 

 

2026

 

 

2025

 

2026

 

 

2025

 

Total revenues

$

173

 

 

$

160

 

$

523

 

 

$

466

 

Operating expenses:

 

 

 

 

 

 

 

 

 

 

Cost of sales

 

32

 

 

 

28

 

 

96

 

 

 

80

 

Research and development

 

154

 

 

 

216

 

 

508

 

 

 

547

 

Selling, general and administrative

 

97

 

 

 

87

 

 

273

 

 

 

261

 

Total operating expenses

 

283

 

 

 

331

 

 

877

 

 

 

888

 

Net loss

$

(118

)

 

$

(180

)

$

(395

)

 

$

(446

)

Net loss per share, basic and diluted

$

(1.16

)

 

$

(1.81

)

$

(3.89

)

 

$

(4.55

)

Operating Expenses

Total operating expenses for the third quarter 2026 were $283 million, including $34 million of non-cash stock-based compensation.
 

Net Loss

Net loss for the third quarter 2026 was $118 million, or $1.16 per share basic and diluted, compared with a net loss for the third quarter 2025 of $180 million, or $1.81 per share basic and diluted.

 

Cash Balance and Net Cash Used in Operations

Cash, cash equivalents, and marketable securities were $401 million as of September 30, 2026. For the three months ended September 30, 2026, net cash used in operations was $43 million.

 

In October 2026, the Company entered into two definitive agreements to sell the PRVs received upon the approval of GENGLYCOS and FAYUVI for a combined price of $413 million. The sale of the GENGLYCOS PRV closed last week and the FAYUVI PRV sale is expected to close later this month. Net proceeds from both sales are expected to be approximately $350 million.

 

Initiation of substantial expense reductions and reaffirming profitability in 2027
 

With the recent completion of late-stage development programs, including first-ever FDA approvals in glycogen storage disease type Ia (GSDIa) and Sanfilippo syndrome type A, Ultragenyx is entering a new stage focused on commercial execution and a smaller, more targeted R&D footprint. To support this transition, the company has initiated substantial headcount and expense reductions and expects to reduce 2027 R&D expenses to less than $350 million and 2027 SG&A expenses to less than $375 million. Savings actions include:

•
Reducing headcount by approximately 20%, resulting in a revised workforce of approximately 1,000 employees
•
Completing wind-down of all ongoing studies for GTX-102 for Angelman syndrome
•
Terminating UX701 for Wilson disease based on not achieving study goals
•
Reducing expenses across the company, including reduction of real estate footprint

 

In addition to executing these critical expense reductions, the company is focused on driving successful launches for GENGLYCOS and FAYUVI and continuing to grow current product revenues. R&D investments will be focused on delivering primary data from the responder endpoint of the ongoing Phase 3 study for DTX301 for ornithine

2

 


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transcarbamylase deficiency, determining if there is a viable path forward for UX143 (setrusumab) for osteogenesis imperfecta, and measured spending to progress early-stage programs for future INDs.

 

Fourth quarter 2026 financial results are expected to include approximately $25 million of severance and other non-recurring restructuring charges, and the reduction in force is expected to be substantially completed by the end of 2026. The company continues to anticipate achieving full-year GAAP profitability in 2027 and going forward.

 

Recent Corporate Updates and Upcoming Milestones

•
GENGLYCOS (pariglasgene brecaparvovec) AAV8 gene therapy for the treatment of glycogen storage disease type Ia (GSDIa): In August 2026, the U.S. Food and Drug Administration (FDA) granted accelerated approval for GENGLYCOS as a treatment for adult and pediatric patients eight years and older with GSDIa. The company received a PRV upon this approval and sold it for $210 million.

 

•
FAYUVI (rebisufligene etisparvovec) AAV9 gene therapy for the treatment of Sanfilippo syndrome type A (MPS IIIA): In September 2026, the FDA granted standard full approval for FAYUVI as a treatment of pediatric patients with Sanfilippo syndrome Type A. The company received a PRV upon this approval and, in October 2026, entered into a definitive agreement to sell the PRV for $203 million.

 

•
UX701 (rivunatpagene miziparvovec) AAV9 gene therapy for the treatment of Wilson disease: Additional data in the dose-finding stage of the Cyprus2+ study of UX701 for the treatment of Wilson disease, including data from the fourth dosing cohort, demonstrated clinical activity in some patients, but did not meet the criteria that the majority of patients achieve biochemical response and elimination of standard of care, to advance the program to the pivotal Phase 3, stage of development. UX701 demonstrated a favorable safety profile across all cohorts dosed. Data from the completed study is expected to be presented at an upcoming medical or scientific congress.

 

The company will terminate further development of this program and is working with clinical sites and patient advocacy groups to support the patient community through this transition.

 

•
UX016 novel prodrug for sialic acid used as a substrate replacement therapy for the treatment of GNE myopathy: The Phase 1/2 study is evaluating safety and dose of UX016 and its impact on muscle strength in adults with GNEM. The UX016 program is fully funded through clinical proof-of-concept by an external venture philanthropy agreement with a patient group, which supports all costs for the Phase 1/2 study and dosing will be initiated soon.

 

•
DTX301 (avalotcagene ontaparvovec) AAV8 gene therapy for the treatment of Ornithine Transcarbamylase, or OTC, deficiency: The Phase 3 Enh3ance placebo-controlled study showed substantial improvement in ammonia control and reduction in the need for scavenger medicines along with profound improvement in global impression scale. The study continues with patients in both treatment and cross-over groups progressing through 64 weeks of follow-up to evaluate the second primary endpoint of the reduction in the use of ammonia scavengers and dietary management. Data are expected in the first half of 2027.

 

•
UX143 (setrusumab) monoclonal antibody for the treatment of osteogenesis imperfecta: Phase 3 data with pre- and post-hoc analyses were presented at the American Society for Bone and Mineral Research 2026 Annual Meeting showing a meaningful benefit on bone mineral density, fractures, and other endpoints in the two Phase 3 studies. The company will continue to engage regulatory agencies to determine the necessary data to support a regulatory filing and if there is a potential path forward for UX143.

 

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Conference Call and Webcast Information

Ultragenyx will host a conference call today, Tuesday, November 3, 2026, at 2 p.m. PT/5 p.m. ET to discuss the third quarter 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.

 

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 regarding Ultragenyx’s expectations and projections concerning its future operating results and financial performance, including its 2026 revenue guidance for total revenue, Crysvita and Dojolvi, its anticipated R&D and SG&A expenses in 2027, the expected timing of closing and net proceeds from the sales of its PRVs, the anticipated scope, timing, costs, benefits and savings of its headcount and expense reductions, including expected severance and other restructuring charges and the expected timing of completion of the reduction in force, and the timing and sustainability of profitability, including expected full-year GAAP profitability in 2027 and going forward; the timing, progress, results and plans for its clinical programs and studies, including the wind-down of the GTX-102 studies, the termination of the UX701 program, the anticipated timing of data from the Phase 3 Enh3ance study of DTX301, engagement with regulatory agencies regarding UX143 and whether there is a potential path forward for UX143, and initiation of dosing in the UX016 study; Ultragenyx’s ability to successfully launch and commercialize GENGLYCOS and FAYUVI, demand for these products, and Ultragenyx’s ability to satisfy post-marketing requirements, including confirmatory study requirements associated with the accelerated approval of GENGLYCOS; and the potential patient impact, commercial opportunity and growth associated with Ultragenyx’s products and product candidates, 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, the risk that the FDA may withdraw the accelerated approval of GENGLYCOS if confirmatory studies do not verify clinical benefit, risks related to serious or undesirable side effects of our products and 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 launching and commercializing gene therapy products, 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, the risk that the PRV sales may not close on the expected timeline or at all, delays or unexpected costs and other adverse effects related to the headcount and expense reductions, including disruption to our operations and our ability to retain key employees, competition from other therapies or products, regulatory scrutiny of the company’s products and product candidates, the

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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 marketable securities 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 product candidates. 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 August 5, 2026, 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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Ultragenyx Pharmaceutical Inc.

Selected Revenue Data

(in millions)

(unaudited)

 

 

Three Months Ended September 30,

 

Nine Months Ended September 30,

 

 

2026

 

 

2025

 

2026

 

 

2025

 

CRYSVITA

 

 

 

 

 

 

 

 

 

 

Product sales - Latin America and Türkiye

$

41

 

 

$

47

 

$

141

 

 

$

137

 

Royalty revenue - U.S. and Canada

 

61

 

 

 

57

 

 

194

 

 

 

177

 

Royalty revenue - Europe

 

7

 

 

 

8

 

 

23

 

 

 

22

 

Total CRYSVITA Revenue

 

109

 

 

 

112

 

 

358

 

 

 

336

 

DOJOLVI

 

30

 

 

 

24

 

 

75

 

 

 

64

 

EVKEEZA

 

24

 

 

 

17

 

 

63

 

 

 

42

 

MEPSEVII

 

10

 

 

 

7

 

 

27

 

 

 

24

 

Total revenues

$

173

 

 

$

160

 

$

523

 

 

$

466

 

 

Ultragenyx Pharmaceutical Inc.

Selected Statement of Operations Financial Data

(in millions, except per share amounts)

(unaudited)

 

 

Three Months Ended September 30,

 

Nine Months Ended September 30,

 

 

2026

 

 

2025

 

2026

 

 

2025

 

Statement of Operations Data:

 

 

 

 

 

 

 

 

 

 

Revenues:

 

 

 

 

 

 

 

 

 

 

Product sales

$

105

 

 

$

95

 

$

306

 

 

$

267

 

Royalty revenue

 

68

 

 

 

65

 

 

217

 

 

 

199

 

Total revenues

 

173

 

 

 

160

 

 

523

 

 

 

466

 

Operating expenses:

 

 

 

 

 

 

 

 

 

 

Cost of sales

 

32

 

 

 

28

 

 

96

 

 

 

80

 

Research and development

 

154

 

 

 

216

 

 

508

 

 

 

547

 

Selling, general and administrative

 

97

 

 

 

87

 

 

273

 

 

 

261

 

Total operating expenses

 

283

 

 

 

331

 

 

877

 

 

 

888

 

Loss from operations

 

(110

)

 

 

(171

)

 

(354

)

 

 

(422

)

Non-cash interest expense on liabilities for sales of future royalties

 

(20

)

 

 

(14

)

 

(63

)

 

 

(42

)

Other income, net

 

3

 

 

 

6

 

 

15

 

 

 

21

 

Loss before income taxes

 

(127

)

 

 

(179

)

 

(402

)

 

 

(443

)

Benefit from (provision for) income taxes

 

9

 

 

 

(1

)

 

7

 

 

 

(3

)

Net loss

$

(118

)

 

$

(180

)

$

(395

)

 

$

(446

)

Net loss per share, basic and diluted

$

(1.16

)

 

$

(1.81

)

$

(3.89

)

 

$

(4.55

)

Shares used in computing net loss per share, basic and diluted

 

102.1

 

 

 

99.8

 

 

101.6

 

 

 

98.2

 

 

6

 


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Ultragenyx Pharmaceutical Inc.

Selected Activity included in Operating Expenses

(in millions)

(unaudited)

 

Three Months Ended September 30,

 

Nine Months Ended September 30,

 

 

2026

 

 

2025

 

2026

 

 

2025

 

Non-cash stock-based compensation

$

34

 

 

$

37

 

$

98

 

 

$

116

 

Restructuring expense

 

—

 

 

 

—

 

 

30

 

 

 

—

 

 

 

Ultragenyx Pharmaceutical Inc.

Selected Balance Sheet Financial Data

(in millions)

(unaudited)

 

September 30,

 

 

December 31,

 

 

2026

 

 

2025

 

Balance Sheet Data:

 

 

 

 

 

Cash, cash equivalents, and marketable securities

$

401

 

 

$

737

 

Working capital

 

155

 

 

 

567

 

Total assets

 

1,187

 

 

 

1,532

 

Total stockholders' deficit

 

(376

)

 

 

(80

)

7

 


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