Ultragenyx Pharmaceutical Inc 8-K, filed Nov 3, 2026
Ultragenyx Pharmaceutical Inc. (RARE)
About this 8-K
Ultragenyx Pharmaceutical Inc filed this 8-K on November 3, 2026, reporting on events of October 30, 2026.
Items reported
- Item 2.01: Completed an acquisition or sale of assets
- Item 2.02: Financial results
- Item 2.05: Exit or restructuring costs
- Item 9.01: Financial statements and exhibits
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 2.01 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 |
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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.
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Ultragenyx Pharmaceutical Inc. |
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Date: |
November 3, 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 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
Summary of Third Quarter 2026 Financial Results
Selected Financial Data (dollars in millions, except per share amounts), (unaudited)
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Three Months Ended September 30, |
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Nine Months Ended September 30, |
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2026 |
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2025 |
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2026 |
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2025 |
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Total revenues |
$ |
173 |
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$ |
160 |
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$ |
523 |
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$ |
466 |
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Operating expenses: |
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Cost of sales |
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32 |
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28 |
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96 |
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80 |
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Research and development |
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154 |
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216 |
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508 |
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547 |
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Selling, general and administrative |
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97 |
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87 |
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273 |
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261 |
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Total operating expenses |
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283 |
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331 |
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877 |
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888 |
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Net loss |
$ |
(118 |
) |
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$ |
(180 |
) |
$ |
(395 |
) |
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$ |
(446 |
) |
Net loss per share, basic and diluted |
$ |
(1.16 |
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$ |
(1.81 |
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$ |
(3.89 |
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$ |
(4.55 |
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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:
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
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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
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.
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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)
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Three Months Ended September 30, |
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Nine Months Ended September 30, |
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2026 |
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2025 |
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2026 |
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2025 |
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CRYSVITA |
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Product sales - Latin America and Türkiye |
$ |
41 |
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$ |
47 |
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$ |
141 |
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$ |
137 |
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Royalty revenue - U.S. and Canada |
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61 |
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57 |
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194 |
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177 |
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Royalty revenue - Europe |
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7 |
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8 |
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23 |
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22 |
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Total CRYSVITA Revenue |
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109 |
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112 |
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358 |
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336 |
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DOJOLVI |
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30 |
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24 |
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75 |
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64 |
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EVKEEZA |
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24 |
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17 |
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63 |
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42 |
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MEPSEVII |
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10 |
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7 |
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27 |
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24 |
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Total revenues |
$ |
173 |
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$ |
160 |
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$ |
523 |
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$ |
466 |
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Ultragenyx Pharmaceutical Inc.
Selected Statement of Operations Financial Data
(in millions, except per share amounts)
(unaudited)
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Three Months Ended September 30, |
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Nine Months Ended September 30, |
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2026 |
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2025 |
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2026 |
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2025 |
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Statement of Operations Data: |
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Revenues: |
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Product sales |
$ |
105 |
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$ |
95 |
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$ |
306 |
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$ |
267 |
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Royalty revenue |
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68 |
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65 |
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217 |
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199 |
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Total revenues |
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173 |
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160 |
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523 |
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466 |
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Operating expenses: |
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Cost of sales |
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32 |
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28 |
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96 |
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80 |
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Research and development |
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154 |
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216 |
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508 |
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547 |
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Selling, general and administrative |
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97 |
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87 |
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273 |
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261 |
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Total operating expenses |
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283 |
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331 |
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|
877 |
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888 |
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Loss from operations |
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(110 |
) |
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(171 |
) |
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(354 |
) |
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(422 |
) |
Non-cash interest expense on liabilities for sales of future royalties |
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(20 |
) |
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(14 |
) |
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(63 |
) |
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(42 |
) |
Other income, net |
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3 |
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6 |
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15 |
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21 |
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Loss before income taxes |
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(127 |
) |
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(179 |
) |
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(402 |
) |
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(443 |
) |
Benefit from (provision for) income taxes |
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9 |
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(1 |
) |
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7 |
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(3 |
) |
Net loss |
$ |
(118 |
) |
|
$ |
(180 |
) |
$ |
(395 |
) |
|
$ |
(446 |
) |
Net loss per share, basic and diluted |
$ |
(1.16 |
) |
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$ |
(1.81 |
) |
$ |
(3.89 |
) |
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$ |
(4.55 |
) |
Shares used in computing net loss per share, basic and diluted |
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102.1 |
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99.8 |
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101.6 |
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98.2 |
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Ultragenyx Pharmaceutical Inc.
Selected Activity included in Operating Expenses
(in millions)
(unaudited)
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Three Months Ended September 30, |
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Nine Months Ended September 30, |
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2026 |
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2025 |
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2026 |
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2025 |
|
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Non-cash stock-based compensation |
$ |
34 |
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$ |
37 |
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$ |
98 |
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$ |
116 |
|
Restructuring expense |
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— |
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— |
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30 |
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— |
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Ultragenyx Pharmaceutical Inc.
Selected Balance Sheet Financial Data
(in millions)
(unaudited)
|
September 30, |
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December 31, |
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2026 |
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2025 |
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Balance Sheet Data: |
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Cash, cash equivalents, and marketable securities |
$ |
401 |
|
|
$ |
737 |
|
Working capital |
|
155 |
|
|
|
567 |
|
Total assets |
|
1,187 |
|
|
|
1,532 |
|
Total stockholders' deficit |
|
(376 |
) |
|
|
(80 |
) |
7