Item 9A. Controls and Procedures
Item 9A. Controls and Procedures
Evaluation of Disclosure Controls and Procedures
Management carried out an evaluation, under the supervision and with the participation of our Principal Executive Officer and our Principal Financial Officer, of the effectiveness of our “disclosure controls and procedures” as of the end of the period covered by this Annual Report, pursuant to Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, or the Exchange Act. In connection with that evaluation, our Principal Executive Officer and our Principal Financial Officer concluded that our disclosure controls and procedures were effective and designed to provide reasonable assurance that the information required to be disclosed is recorded, processed, summarized, and reported within the time periods specified in the SEC rules and forms as of December 31, 2025. For the purpose of this review, disclosure controls and procedures means controls and procedures designed to ensure that information required to be disclosed by us in the reports that we file or submit is recorded, processed, summarized and reported within the time periods specified in the SEC’s rules and forms. These disclosure controls and procedures include, without limitation, controls and procedures designed to ensure that information required to be disclosed by us in the reports that we file or submit is accumulated and communicated to management, including our Principal Executive Officer and our Principal Financial Officer, as appropriate to allow timely decisions regarding required disclosure. In designing and evaluating the disclosure controls and procedures, our management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving the desired control objectives, and our management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures.
Management’s Annual Report on Internal Control Over Financial Reporting
Management is responsible for establishing and maintaining adequate internal control over financial reporting as defined in Rules 13a-15(f) and 15d-15(f) of the Exchange Act. Our management used the Committee of Sponsoring Organizations of the Treadway Commission Internal Control - Integrated Framework issued by the Committee of Sponsoring Organizations of the Treadway Commission (2013 framework), or the COSO framework, to evaluate the effectiveness of internal control over financial reporting. Management believes that the COSO framework is a suitable framework for its evaluation of financial reporting because it is free from bias, permits reasonably consistent qualitative and quantitative measurements of our internal control over financial reporting, is sufficiently complete so that those relevant factors that would alter a conclusion about the effectiveness of our internal control over financial reporting are not omitted and is relevant to an evaluation of internal control over financial reporting.
Management has assessed the effectiveness of our internal control over financial reporting as of December 31, 2025, and has concluded that as of such date, our internal control over financial reporting was effective.
Our independent registered public accounting firm, Ernst & Young LLP, has audited the financial statements included in this Annual Report and has issued a report on the effectiveness of our internal control over financial reporting. The report of Ernst & Young LLP is included below.
Changes in Internal Control over Financial Reporting
There have been no changes in our internal control over financial reporting (as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act) during our fourth quarter ended December 31, 2025, that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.
76
Report of Independent Registered Public Accounting Firm
To the Stockholders' and the Board of Directors of Ultragenyx Pharmaceutical Inc.
Opinion on Internal Control Over Financial Reporting
We have audited Ultragenyx Pharmaceutical Inc.’s internal control over financial reporting as of December 31, 2025, based on criteria established in Internal Control—Integrated Framework issued by the Committee of Sponsoring Organizations of the Treadway Commission ( 2013 framework) (the COSO criteria). In our opinion, Ultragenyx Pharmaceutical Inc. (the Company) maintained, in all material respects, effective internal control over financial reporting as of December 31, 2025, based on the COSO criteria.
We also have audited, in accordance with the standards of the Public Company Accounting Oversight Board (United States) (PCAOB), the consolidated balance sheets of the Company as of December 31, 2025 and 2024, the related consolidated statements of operations, comprehensive loss, stockholders’ equity and cash flows for each of the three years in the period ended December 31, 2025, and the related notes and our report dated February 18, 2026 expressed an unqualified opinion thereon.
Basis for Opinion
The Company’s management is responsible for maintaining effective internal control over financial reporting and for its assessment of the effectiveness of internal control over financial reporting included in the accompanying Management’s Annual Report on Internal Control over Financial Reporting. Our responsibility is to express an opinion on the Company’s internal control over financial reporting based on our audit. We are a public accounting firm registered with the PCAOB and are required to be independent with respect to the Company in accordance with the U.S. federal securities laws and the applicable rules and regulations of the Securities and Exchange Commission and the PCAOB.
We conducted our audit in accordance with the standards of the PCAOB. Those standards require that we plan and perform the audit to obtain reasonable assurance about whether effective internal control over financial reporting was maintained in all material respects.
Our audit included obtaining an understanding of internal control over financial reporting, assessing the risk that a material weakness exists, testing and evaluating the design and operating effectiveness of internal control based on the assessed risk, and performing such other procedures as we considered necessary in the circumstances. We believe that our audit provides a reasonable basis for our opinion.
Definition and Limitations of Internal Control Over Financial Reporting
A company’s internal control over financial reporting is a process designed to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles. A company’s internal control over financial reporting includes those policies and procedures that (1) pertain to the maintenance of records that, in reasonable detail, accurately and fairly reflect the transactions and dispositions of the assets of the company; (2) provide reasonable assurance that transactions are recorded as necessary to permit preparation of financial statements in accordance with generally accepted accounting principles, and that receipts and expenditures of the company are being made only in accordance with authorizations of management and directors of the company; and (3) provide reasonable assurance regarding prevention or timely detection of unauthorized acquisition, use, or disposition of the company’s assets that could have a material effect on the financial statements.
Because of its inherent limitations, internal control over financial reporting may not prevent or detect misstatements. Also, projections of any evaluation of effectiveness to future periods are subject to the risk that controls may become inadequate because of changes in conditions, or that the degree of compliance with the policies or procedures may deteriorate.
/s/ Ernst & Young LLP
San Mateo, California
February 18, 2026
I tem 9B. Other Information
During the three months ended December 31, 2025, none of our directors and officers adopted a Rule 10b5-1 trading arrangement intended to satisfy the affirmative defense conditions of Rule 10b5-1(c).
77
Item 9C. Disclosure Regarding Foreign Jurisdictions that Prevent Inspections
Not applicable.
78
P ART III
Item 10. Di rectors, Executive Officers and Corporate Governance
Except as set forth below, the information required by this Item is incorporated herein by reference to information in the proxy statement for our 2026 Annual Meeting of Stockholders, which we will file with the SEC within 120 days of the end of the fiscal year to which this Annual Report relates, or the “2026 Proxy Statement”, including under the headings “Nominees and Incumbent Directors,” “Executive Officers,” ““Board of Directors and Committees,” “Corporate Governance” and, as applicable, “Delinquent Section 16(a) Beneficial Ownership Reports.”
We have adopted a Global Code of Conduct that applies to all of our directors, officers and employees, including our principal executive, principal financial and principal accounting officers, or persons performing similar functions. Our Global Code of Conduct is posted on our website located at https://ir.ultragenyx.com/ under “Corporate Governance”. We intend to disclose future amendments to certain provisions of the Global Code of Conduct, and waivers of the Global Code of Conduct granted to executive officers and directors, on the website within four business days following the date of the amendment or waiver.
I tem 11. Executive Compensation
The information required by this Item is incorporated herein by reference to information in the 2026 Proxy Statement, including under the headings “Executive Compensation,” “Director Compensation,” and “Board of Directors and Committees.”
Item 12. Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters
The information required by this Item is incorporated herein by reference to information in the 2026 Proxy Statement, including under the headings “Security Ownership of Certain Beneficial Owners and Management” and “Equity Compensation Plan Information.”
Item 13. Certain Relationships and Related Transactions, and Director Independence
The information required by this Item is incorporated herein by reference to information in the 2026 Proxy Statement, including under the headings “Certain Relationships and Related-Person Transactions,” “Corporate Governance,” and “Board of Directors and Committees.”
Item 14. Principal Accountant Fees and Services
The information required by this Item is incorporated herein by reference to information in the 2026 Proxy Statement, including under the heading “Ratification of the Selection of Independent Registered Public Accounting Firm.”
79
PA RT IV
Item 15. Ex hibits and Financial Statement Schedules
(a) The following documents are filed as part of this Annual Report.
(1) Consolidated Financial Statements
Consolidated Financial Statements—See Index to Consolidated Financial Statements at page F-1 of this Annual Report.
(2) Consolidated Financial Statement Schedules
Consolidated Financial Statement schedules have been omitted in this Annual Report because they are not applicable, not required under the instructions, or the information requested is set forth in the Consolidated Financial Statements or related notes thereto.
(b) Exhibits
Exhibit
Incorporated by Reference
Filed
Number
Exhibit Description
Form
Date
Number
Herewith
3.1
Amended and Restated Certificate of Incorporation
8-K
2/5/2014
3.1
3.2
Second Amended and Restated Bylaws
8-K
12/21/2023
3.1
4.1
Form of Common Stock Certificate
S-1
11/8/2013
4.2
4.2
Form of Indenture
S-3 ASR
2/21/2024
4.2
4.3
Form of Pre-Funded Warrant
8-K
10/23/2023
4.1
4.4
Form of Pre-Funded Warrant
8-K
6/17/2024
4.1
4.5
Description of Common Stock
10-K
2/14/2020
4.3
10.1*
Collaboration and License Agreement, effective as of August 29, 2013, between Ultragenyx Pharmaceutical Inc. and Kyowa Hakko Kirin Co., Ltd.
S-1/A
12/23/2013
10.1
10.2
Amendment No. 1 to Collaboration and License Agreement, effective as of August 24, 2015, between Ultragenyx Pharmaceutical Inc. and Kyowa Hakko Kirin Co., Ltd.
10-Q
11/10/2015
10.2
10.3
Amendment No. 2 to Collaboration and License Agreement, effective as of November 28, 2016, between Ultragenyx Pharmaceutical Inc. and Kyowa Hakko Kirin Co., Ltd.
10-K
2/21/2018
10.3
10.4*
Amendment No. 3 to Collaboration and License Agreement, effective September 29, 2017, between Ultragenyx Pharmaceutical Inc. and Kyowa Hakko Kirin Co., Ltd.
10-K
2/21/2018
10.4
10.5*
Amendment No. 4 to Collaboration and License Agreement, effective as of January 29, 2018, between Ultragenyx Pharmaceutical Inc. and Kyowa Hakko Kirin Co., Ltd.
10-K
2/21/2018
10.5
10.6*
Amendment No. 5 to Collaboration and License Agreement, effective as of April 30, 2018, between Ultragenyx Pharmaceutical Inc. and Kyowa Hakko Kirin Co., Ltd.
10-Q
8/3/2018
10.1
10.7*
Amendment No. 6 to Collaboration and License Agreement, effective as of February 1, 2019, between Ultragenyx Pharmaceutical Inc. and Kyowa Hakko Kirin Co., Ltd.
10-Q
5/7/2019
10.2
80
10.8*
Amendment No. 7 to Collaboration and License Agreement, effective as of December 5, 2018, between Ultragenyx Pharmaceutical Inc. and Kyowa Hakko Kirin Co., Ltd.
10-Q
5/7/2019
10.3
10.9*
Amendment No. 8 to Collaboration and License Agreement, effective as of July 4, 2019, between Ultragenyx Pharmaceutical Inc. and Kyowa Kirin Co., Ltd. (formerly, Kyowa Hakko Kirin Co., Ltd.)
10-Q
8/2/2019
10.1
10.10*
Amendment No. 9 to Collaboration and License Agreement, effective December 23, 2019, between Ultragenyx Pharmaceutical Inc. and Kyowa Kirin Co., Ltd.
10-K
2/14/2020
10.10
10.11*
Amendment No. 10 to Collaboration and License Agreement, effective as of April 1, 2020, between Ultragenyx Pharmaceutical Inc. and Kyowa Kirin Co., Ltd.
10-Q
5/7/2020
10.2
10.12*
Amendment No. 11 to Collaboration and License Agreement, effective as of December 17, 2021 between Ultragenyx Pharmaceutical Inc. and Kyowa Kirin Co., Ltd.
10-K
2/16/2022
10.13
10.13*
Amendment No. 12 to Collaboration and License Agreement, effective as of September 29, 2022, between Ultragenyx Pharmaceutical Inc. and Kyowa Kirin Co., Ltd.
10-Q
11/3/2022
10.1
10.14*
Amendment No. 13 to Collaboration and License Agreement, effective as of May 16, 2023, between Ultragenyx Pharmaceutical Inc. and Kyowa Kirin Co., Ltd.
10-Q
8/3/2023
10.1
10.15*
Supply Agreement, effective as of November 18, 2020, between Ultragenyx Pharmaceutical Inc. and Kyowa Kirin Inc.
10-K
2/19/2025
10.15
10.16*
Amendment No. 1, effective as of September 13, 2024, to the Supply Agreement between Ultragenyx Pharmaceutical Inc. and Kyowa Kirin, Inc.
10-K
2/19/2025
10.16
10.17*
Unit Purchase Agreement, dated as of July 15, 2022, by and among Ultragenyx Pharmaceutical Inc., GeneTx Biotherapeutics LLC, the Unitholders and Deborah A. Guagliardo
10-Q
7/29/2022
10.2
10.18*
Royalty Purchase Agreement, dated as of December 17, 2019, between Ultragenyx Pharmaceutical Inc. and RPI Finance Trust
10-K
2/14/2020
10.25
10.19*
Royalty Purchase Agreement, dated as of July 14, 2022, by and among Rare Delaware Inc., Ultragenyx Pharmaceutical Inc. and OCM LS23 Holdings LP
10-Q
7/29/2022
10.1
10.20*
Royalty Purchase Agreement, dated as of November 3, 2025, by and among Rare Delaware Inc., Ultragenyx Pharmaceutical Inc. and OCM LS23 Holding LP
X
10.21#
2014 Incentive Plan (as amended)
10-K
2/17/2017
10.20
10.22#
Form of Incentive Stock Option Agreement (2014 Plan)
S-1/A
1/17/2014
10.14
10.23#
Form of Non Statutory Stock Option Agreement (Employees) (2014 Plan)
S-1/A
1/17/2014
10.15
10.24#
Form of Restricted Stock Unit Agreement (Employees) (2014 Plan )
10-Q
5/10/2016
10.1
10.25#
Form of Non-Statutory Stock Option Agreement (Annual Grant for Directors) (2014 Plan)
10-Q
8/3/2021
10.2
81
10.26#
Form of Restricted Stock Unit Agreement (Annual Grant for Directors ) (2014 Plan)
10-Q
8/3/2021
10.3
10.27#
Form of Non-Statutory Stock Option Agreement (Grant for New Directors) (2014 Plan)
10-Q
8/3/2021
10.4
10.28#
Form of Restricted Stock Unit Agreement (Grant for New Directors) (2014 Plan)
10-Q
8/3/2021
10.5
10.29#
Amended and Restated 2023 Incentive Plan
S-8
7/12/2024
4.4
10.30#
Form of Incentive Stock Option Agreement (2023 Plan)
10-K
2/21/2024
10.30
10.31#
Form of Non Statutory Stock Option Agreement (Employees)(2023 Plan)
10-K
2/21/2024
10.31
10.32#
Form of Restricted Stock Unit Agreement (Employees) (2023 Plan)
10-K
2/21/2024
10.32
10.33#
Form of Non-Statutory Stock Option Agreement (Annual Grant for Directors) (2023 Plan)
10-K
2/21/2024
10.33
10.34#
Form of Restricted Stock Unit Agreement (Annual Grant for Directors) (2023 Plan)
10-K
2/21/2024
10.34
10.35#
Form of Non-Statutory Stock Option Agreement (Grant for New Directors) (2023 Plan)
10-K
2/21/2024
10.35
10.36#
Form of Restricted Stock Unit Agreement (Grant for New Directors) (2023 Plan)
10-K
2/21/2024
10.36
10.37#
Form of Performance Stock Unit Agreement (2023)
10-Q
5/4/2023
10.1
10.38#
Form of Performance Stock Unit Agreement (2024)
10-K
2/19/2025
10.38
10.39#
Form of Performance Stock Unit Agreement (2025)
10-Q
5/6/2025
10.1
10.40#
Amended and Restated 2014 Employee Stock Purchase Plan
S-8
6/8/2023
4.5
10.41#
Corporate Bonus Plan
S-1/A
1/17/2014
10.27
10.42#
Employment Inducement Plan
10-K
2/12/2021
10.43
10.43#
First Amendment to Employment Inducement Plan
S-8
6/8/2023
4.7
10.44#
Second Amendment to Employment Inducement Plan
S-8
7/12/2024
4.7
10.45#
Third Amendment to Employment Inducement Plan
S-8
7/18/2025
4.8
10.46#
Form of Non Statutory Stock Option Agreement (Inducement Plan)
10-K
2/12/2021
10.44
10.47#
Form of Non Statutory Stock Option Agreement (Inducement Plan) (ex-US)
10-K
2/12/2021
10.45
10.48#
Form of Restricted Stock Unit Agreement (Inducement Plan)
10-K
2/12/2021
10.46
10.49#
Form of Restricted Stock Unit Agreement (Inducement Plan)(ex-US)
10-K
2/12/2021
10.47
10.50#
Ultragenyx Pharmaceutical Inc. Deferred Compensation Plan
10-Q
8/3/2021
10.1
10.51#
Amendment No. 1 to the Ultragenyx Pharmaceutical Inc. Deferred Compensation Plan
10-Q
11/3/2021
10.1
10.52#
Executive Employment Agreement, dated as of June 15, 2011, between Ultragenyx Pharmaceutical Inc. and Emil D. Kakkis, M.D., Ph.D.
S-1
11/8/2013
10.18
82
10.53#
Amendment No. 1 to Executive Employment Agreement, dated August 8, 2014, between Ultragenyx Pharmaceutical Inc. and Emil D. Kakkis, M.D., Ph.D.
10-Q
8/11/2014
10.2
10.54#
Amendment No. 2, dated September 13, 2022, to Executive Employment Agreement between Ultragenyx Pharmaceutical Inc. and Emil D. Kakkis, M.D., Ph.D.
10-Q
11/3/2022
10.2
10.55#
Offer Letter, dated as of April 26, 2016, between Ultragenyx Pharmaceutical Inc. and Karah Parschauer
10-Q
8/9/2016
10.3
10.56#
Amendment, dated September 13, 2022, to Offer Letter between Ultragenyx Pharmaceutical Inc. and Karah Parschauer
10-Q
11/3/2022
10.6
10.57#
Offer Letter, dated as of February 20, 2015, between Ultragenyx Pharmaceutical Inc. and Dennis Huang
10-K
2/17/2017
10.36
10.58#
Amendment, dated September 13, 2022, to Offer Letter between Ultragenyx Pharmaceutical Inc. and Dennis Huang
10-Q
11/3/2022
10.7
10.59#
Offer Letter, dated as of June 11, 2015, between Ultragenyx Pharmaceutical Inc. and John R. Pinion II
10-K
2/17/2017
10.37
10.60#
Amendment, dated September 13, 2022, to Offer Letter between Ultragenyx Pharmaceutical Inc. and John R. Pinion II
10-Q
11/3/2022
10.9
10.61#
Amended and Restated Offer Letter, dated March 31, 2023, between Ultragenyx Pharmaceutical Inc. and Eric Crombez, M.D.
10-Q
5/4/2023
10.2
10.62#
Offer Letter, dated June 2, 2023, between Ultragenyx Pharmaceutical Inc. and Howard Horn
8-K
7/12/2023
10.1
10.63#
Amendment, dated September 6, 2023, to the Offer Letter between Ultragenyx Pharmaceutical Inc. and Howard Horn
8-K
9/8/2023
10.1
10.64#
Offer Letter, dated May 16, 2017, between Ultragenyx Pharmaceutical Inc. and Erik Harris
10-Q
8/2/2019
10.4
10.65#
Addendum #1, dated August 8, 2017, to Offer Letter dated May 16, 2017 between Ultragenyx Pharmaceutical Inc. and Erik Harris
10-Q
8/2/2019
10.5
10.66#
Addendum #2, dated June 19, 2019, to Offer Letter dated May 16, 2017 between Ultragenyx Pharmaceutical Inc. and Erik Harris
10-Q
8/2/2019
10.6
10.67#
Amendment No. 3, dated September 13, 2022, to Offer Letter between Ultragenyx Pharmaceutical Inc. and Erik Harris
10-Q
11/3/2022
10.8
10.68#
Form of Indemnification Agreement
10-K
3/24/2014
10.23
10.69
Standard Lease, dated as of July 5, 2011, between Ultragenyx Pharmaceutical Inc. and Condiotti Enterprises, Inc.
S-1
11/8/2013
10.22
10.70
Addendum One to Standard Lease, dated as of July 5, 2011, between Ultragenyx Pharmaceutical Inc. and Condiotti Enterprises, Inc.
10-K
2/26/2016
10.34
83
10.71
Addendum Two to Standard Lease, dated as of March 7, 2012, between Ultragenyx Pharmaceutical Inc. and Condiotti Enterprises, Inc.
10-K
2/26/2016
10.35
10.72
Addendum #3 to Standard Lease, effective as of February 12, 2014, between Ultragenyx Pharmaceutical Inc. and Condiotti Enterprises, Inc.
8-K
2/25/2014
10.1
10.73
Addendum #4 to Standard Lease, effective as of March 9, 2015, between Ultragenyx Pharmaceutical Inc. and Condiotti Enterprises, Inc.
8-K
3/13/2015
10.1
10.74
Addendum #5 to Standard Lease, effective as of April 7, 2015, between Ultragenyx Pharmaceutical Inc. and Condiotti Enterprises, Inc.
10-K
2/26/2016
10.38
10.75
Addendum #6 to Standard Lease, effective as of April 29, 2019, between Ultragenyx Pharmaceutical Inc. and Condiotti Enterprises, Inc.
10-Q
8/2/2019
10.3
10.76
Addendum #7 to Standard Lease, effective as of November 22, 2024, between Ultragenyx Pharmaceutical Inc. and Condiotti Enterprises, Inc.
10-K
2/19/2025
10.77
10.77
Lease Agreement, dated as of December 8, 2015, between Marina Boulevard Property, LLC and Ultragenyx Pharmaceutical Inc.
10-K
2/26/2016
10.43
10.78
Lease Agreement, dated November 2, 2015, between Dimension Therapeutics, Inc. and ARE-MA Region No. 20, LLC, and Consent to Assignment to Ultragenyx Pharmaceutical Inc.
10-K
2/21/2018
10.66
10.79
First Amendment to Lease Agreement, dated March 20, 2018, between Ultragenyx Pharmaceutical Inc. and ARE-MA Region No. 20, LLC
10-Q
5/8/2018
10.6
10.80
Second Amendment to Lease Agreement, dated July 1, 2018, between Ultragenyx Pharmaceutical Inc. and ARE-MA Region No. 20, LLC
10-Q
8/3/2018
10.3
10.81
Third Amendment to the Lease Agreement, dated July 29, 2019, between Ultragenyx Pharmaceutical Inc. and ARE-MA Region No., LLC.
10-Q
7/30/2020
10.2
10.82
Amended and Restated Fourth Amendment, dated August 4, 2020, to the Lease Agreement between Ultragenyx Pharmaceutical Inc. and ARE-MA Region No., LLC.
10-Q
10/27/2020
10.5
10.83
Fifth Amendment, dated November 25, 2024, to the Lease Agreement between Ultragenyx Pharmaceutical Inc. and ARE-MA Region No. 20, LLC
10-K
2/19/2025
10.84
10.84
Lease Agreement, dated December 15, 2019, between Ultragenyx Pharmaceutical Inc. and ARE-San Francisco No. 17, LLC.
10-K
2/12/2021
10.81
10.85
First Amendment, dated September 20, 2020, to the Lease Agreement between Ultragenyx Pharmaceutical Inc. and ARE-San Francisco No. 17, LLC.
10-K
2/12/2021
10.82
10.86
Second Amendment, dated October 21, 2020, to the Lease Agreement between Ultragenyx Pharmaceutical Inc. and ARE-San Francisco No. 17, LLC.
10-K
2/12/2021
10.83
84
10.87
Third Amendment, dated July 27, 2022, to the Lease Agreement between Ultragenyx Pharmaceutical Inc. and ARE-San Francisco No. 17, LLC
10-K
2/16/2023
10.92
10.88
Fourth Amendment, dated December 13, 2024, to the Lease Agreement between Ultragenyx Pharmaceutical Inc. and GI ETS Shoreline LLC (as successor-in-interest to ARE-San Francisco No. 17, LLC)
10-K
2/19/2025
10.89
10.89
Office Lease, dated April 19, 2019, between Ultragenyx Pharmaceutical Inc. and Woburn MCB II, LLC
10-K
2/14/2020
10.70
10.90
First Amendment, dated December 9, 2025, to the Lease Agreement between Ultragenyx Pharmaceutical Inc. and Woburn MCB II, LLC
X
10.91
Lease, dated August 18, 2022, between Ultragenyx Pharmaceutical Inc. and Brickbottom I QOZB L.P.
10-K
2/17/2023
10.95
10.92
First Amendment, dated March 12, 2024, between Ultragenyx Pharmaceutical Inc. and Brickbottom I QOZB L.P.
10-K
2/19/2025
10.93
10.93
Commercial Lease, dated July 2, 2018, between Ultragenyx Pharmaceutical Inc. and 32 Leveroni LLC
10-K
2/14/2020
10.71
19.1
Ultragenyx Insider Trading Policy
X
21.1
Subsidiaries of Ultragenyx Pharmaceutical Inc.
X
23.1
Consent of Independent Registered Public Accounting Firm
X
24.1
Power of Attorney (included on the signature page of this report)
31.1
Certification of Principal Executive Officer of Ultragenyx Pharmaceutical Inc., as required by Rule 13a-14(a) or Rule 15d-14(a) of the Exchange Act as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002
X
31.2
Certification of Principal Financial Officer of Ultragenyx Pharmaceutical Inc., as required by Rule 13a-14(a) or Rule 15d-14(a) of the Exchange Act as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002
X
32.1§
Certification by the Principal Executive Officer and Principal Financial Officer, as required by Rule 13a-14(b) or Rule 15d-14(b) and Section 1350 of Chapter 36 of Title 18 of the United States Code (18 U.S.C. §1350)
X
97.1
Ultragenyx Pharmaceutical Inc. Clawback Policy
2/21/2024
97.1
101.INS
XBRL Instance Document, formatted in Inline XBRL
X
101.SCH
Inline XBRL Taxonomy Extension Schema Document
X
104
The cover page from this Annual Report on Form 10-K, formatted in Inline XBRL and contained in Exhibit 101
X
* Certain identified information has been omitted by means of marking such information with asterisks in reliance on Item 601(b)(10)(iv) of Regulation S-K because it is both (i) not material and (ii) the type that the registrant treats as private or confidential.
# Indicates management contract or compensatory plan.
85
§ The certification attached as Exhibit 32.1 that accompanies this Annual Report is not deemed filed with the SEC and is not to be incorporated by reference into any filing of Ultragenyx Pharmaceutical Inc. under the Securities Act or the Exchange Act, whether made before or after the date of this Annual Report, irrespective of any general incorporation language contained in such filing.
Item 16. Form 10-K Summary
None.
86
SIGN ATURES
Pursuant to the requirements of Section 13 or 15(d) of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
U LTRAGENYX P HARMACEUTICAL I nc .
By:
/s/ Emil D. Kakkis
Emil D. Kakkis, M.D., Ph.D.
President and Chief Executive Officer
(Principal Executive Officer)
Date: February 18, 2026
PO WER OF ATTORNEY
KNOW ALL PERSONS BY THESE PRESENTS, that each person whose signature appears below constitutes and appoints Emil D. Kakkis, M.D., Ph.D. and Howard Horn, and each of them, as his or her true and lawful attorneys-in-fact and agents, with full power of substitution for him or her, and in his or her name in any and all capacities, to sign any and all amendments to this Annual Report, and to file the same, with exhibits thereto and other documents in connection therewith, with the Securities and Exchange Commission, granting unto said attorneys-in-fact and agents, and each of them, full power and authority to do and perform each and every act and thing requisite and necessary to be done therewith, as fully to all intents and purposes as he or she might or could do in person, hereby ratifying and confirming all that said attorneys-in-fact and agents, and any of them, his or her substitute or substitutes, may lawfully do or cause to be done by virtue hereof.
Pursuant to the requirements of the Securities Exchange Act of 1934, this report has been signed below by the following persons on behalf of the registrant and in the capacities and on the dates indicated.
Signature
Title
Date
/s/ Emil D. Kakkis
Emil D. Kakkis, M.D., Ph.D.
President and Chief Executive Officer and Director
(Principal Executive Officer)
February 18, 2026
/s/ Howard Horn
Howard Horn
Executive Vice President, Chief Financial Officer, Corporate Strategy
(Principal Financial Officer)
February 18, 2026
/s/ Theodore A. Huizenga
Theodore A. Huizenga
Senior Vice President and Chief Accounting Officer
(Principal Accounting Officer)
February 18, 2026
/s/ Daniel G. Welch
Daniel G. Welch
Chairman of the Board
February 18, 2026
/s/ Deborah Dunsire
Deborah Dunsire, M.D.
Director
February 18, 2026
/s/ Matthew K. Fust
Matthew K. Fust
Director
February 18, 2026
/s/ Michael Narachi
Michael Narachi
Director
February 18, 2026
/s/ Amrit Ray
Director
February 18, 2026
Amrit Ray, M.D.
/s/ Corsee D. Sanders
Director
February 18, 2026
Corsee D. Sanders, Ph.D.
/s/ Shehnaaz Suliman
Director
February 18, 2026
Shehnaaz Suliman, M.D.
87
Ultragenyx Pharmaceutical Inc.
INDEX TO FINANCIAL STATEMENTS
Page
Report of Independent Registered Public Accounting Firm (PCAOB ID: 42 )
F- 2
Consolidated Financial Statements:
Consolidated Balance Sheets
F- 4
Consolidated Statements of Operations
F- 5
Consolidated Statements of Comprehensive Loss
F- 5
Consolidated Statements of Stockholders’ Equity (Deficit)
F- 6
Consolidated Statements of Cash Flows
F- 7
Notes to Consolidated Financial Statements
F- 9
F- 1
Report of Independent R egistered Public Accounting Firm
To the Stockholders and the Board of Directors of Ultragenyx Pharmaceutical Inc.
Opinion on the Financial Statements
We have audited the accompanying consolidated balance sheets of Ultragenyx Pharmaceutical Inc. (the Company) as of December 31, 2025 and 2024, the related consolidated statements of operations, comprehensive loss, stockholders' equity and cash flows for each of the three years in the period ended December 31, 2025, and the related notes (collectively referred to as the “consolidated financial statements”). In our opinion, the consolidated financial statements present fairly, in all material respects, the financial position of the Company at December 31, 2025 and 2024, and the results of its operations and its cash flows for each of the three years in the period ended December 31, 2025, in conformity with U.S. generally accepted accounting principles.
We also have audited, in accordance with the standards of the Public Company Accounting Oversight Board (United States) (PCAOB), the Company's internal control over financial reporting as of December 31, 2025, based on criteria established in Internal Control— Integrated Framework issued by the Committee of Sponsoring Organizations of the Treadway Commission (2013 framework), and our report dated February 18, 2026 expressed an unqualified opinion thereon.
Basis for Opinion
These financial statements are the responsibility of the Company's management. Our responsibility is to express an opinion on the Company’s financial statements based on our audits. We are a public accounting firm registered with the PCAOB and are required to be independent with respect to the Company in accordance with the U.S. federal securities laws and the applicable rules and regulations of the Securities and Exchange Commission and the PCAOB.
We conducted our audits in accordance with the standards of the PCAOB. Those standards require that we plan and perform the audit to obtain reasonable assurance about whether the financial statements are free of material misstatement, whether due to error or fraud. Our audits included performing procedures to assess the risks of material misstatement of the financial statements, whether due to error or fraud, and performing procedures that respond to those risks. Such procedures included examining, on a test basis, evidence regarding the amounts and disclosures in the financial statements. Our audits also included evaluating the accounting principles used and significant estimates made by management, as well as evaluating the overall presentation of the financial statements. We believe that our audits provide a reasonable basis for our opinion.
Critical Audit Matter
The critical audit matter communicated below is a matter arising from the current period audit of the financial statements that was communicated or required to be communicated to the audit committee and that: (1) relates to accounts or disclosures that are material to the financial statements and (2) involved our especially challenging, subjective or complex judgments. The communication of the critical audit matter does not alter in any way our opinion on the consolidated financial statements, taken as a whole, and we are not, by communicating the critical audit matter below, providing a separate opinion on the critical audit matter or on the account or disclosure to which it relates.
Liabilities for sales of future royalties
Description of the Matter
As discussed in Note 10, the Company has entered into three royalty purchase agreements under which the Company sold its rights to receive royalty payments arising from the net sales of Crysvita in the European and North American markets in exchange for $320 million, $500 million and $400 million.
The proceeds from each transaction were recorded as liabilities and are being amortized using the effective interest method over the estimated lives of the respective arrangements. To determine the amortization of the liabilities, the Company is required to estimate the total amount of future royalty payments to be paid to the respective counterparties, subject to capped amount. The Company estimates an imputed interest on the unamortized portion of the liability and records non-cash interest expense relating to the transaction.
Auditing the Company’s liabilities related to the sale of future royalties was challenging because it required significant judgment to evaluate management’s forecasts of expected royalty payments, which are dependent on forward-looking assumptions.
F- 2
How We Addressed the Matter in Our Audit
We obtained an understanding, evaluated the design and tested the operating effectiveness of controls over the Company’s process of accounting for the liabilities related to the sale of future royalties, including controls over the Company’s estimates of projected sales of Crysvita in the European and North American markets.
To test management’s estimates of future royalties and the imputed effective interest rates, we performed audit procedures that included, among others, evaluating the reasonableness of management’s assumptions related to forecasted royalty growth rates. We compared management’s forecasted royalty payments to historical trends and to analyst expectations for underlying Crysvita sales. We also performed a sensitivity analysis over the estimated future royalties to assess the impact of changes in key assumptions on the interest expense recorded for the year ended December 31, 2025.
/s/ Ernst & Young LLP
We have served as the Company’s auditor since 2012.
San Mateo, California
February 18, 2026
F- 3
ULTRAGENYX PHARMACEUTICAL INC.
CONSOLIDATED BA LANCE SHEETS
(in millions, except per share amounts)
December 31,
2025
2024
ASSETS
Current assets:
Cash and cash equivalents
$
421
$
174
Marketable securities
259
436
Accounts receivable, net
158
122
Inventory
52
45
Prepaid expenses and other assets
61
40
Total current assets
951
817
Property, plant, and equipment, net
244
266
Marketable securities
57
135
Intangible assets, net
176
178
Goodwill
44
44
Other assets
60
63
Total assets
$
1,532
$
1,503
LIABILITIES AND STOCKHOLDERS' EQUITY (DEFICIT)
Current liabilities:
Accounts payable
$
31
$
39
Accrued liabilities
265
241
Lease liabilities
12
10
Liabilities for sales of future royalties
69
50
Other liabilities
7
4
Total current liabilities
384
344
Lease liabilities
24
30
Deferred tax liabilities
30
30
Liabilities for sales of future royalties
1,147
820
Other liabilities
20
17
Total liabilities
1,605
1,241
Commitments and contingencies (Note 15)
Noncontrolling interest
7
7
Stockholders’ equity (deficit):
Preferred stock, par value of $ 0.001 per share— 25.0 shares authorized; nil
outstanding in 2025 and in 2024
—
—
Common stock, par value of $ 0.001 per share— 250.0 shares authorized;
outstanding— 96.6 in 2025 and 92.5 in 2024
—
—
Treasury stock, at cost, 0.2 shares in 2025 and 0.1 shares in 2024
( 8
)
( 4
)
Deferred compensation obligation
8
4
Additional paid-in capital
4,451
4,213
Accumulated other comprehensive income (loss)
1
( 1
)
Accumulated deficit
( 4,532
)
( 3,957
)
Total stockholders’ equity (deficit)
( 80
)
255
Total liabilities, noncontrolling interest and stockholders’ equity (deficit)
$
1,532
$
1,503
See accompanying notes.
F- 4
ULTRAGENYX PHARMACEUTICAL INC.
CONSOLIDATED ST ATEMENTS OF OPERATIONS
(in millions, except per share amounts)
Year Ended December 31,
2025
2024
2023
Revenues:
Product sales
$
369
$
285
$
180
Royalty revenue
304
275
183
Collaboration and license
—
—
71
Total revenues
673
560
434
Operating expenses:
Cost of sales
109
77
45
Research and development
750
698
648
Selling, general and administrative
349
321
310
Total operating expenses
1,208
1,096
1,003
Loss from operations
( 535
)
( 536
)
( 569
)
Interest income
25
37
27
Non-cash interest expense on liabilities for sales of future royalties
( 62
)
( 63
)
( 66
)
Other income (expense)
1
( 5
)
( 1
)
Loss before income taxes
( 571
)
( 567
)
( 609
)
(Provision for) benefit from income taxes
( 4
)
( 2
)
2
Net loss
$
( 575
)
$
( 569
)
$
( 607
)
Net loss per share, basic and diluted
$
( 5.83
)
$
( 6.29
)
$
( 8.25
)
Shares used in computing net loss per share, basic and diluted
98.6
90.5
73.5
See accompanying notes.
ULTRAGENYX PHARMACEUTICAL INC.
CONSOLIDATED STATEM ENTS OF COMPREHENSIVE LOSS
(in millions)
Year Ended December 31,
2025
2024
2023
Net loss
$
( 575
)
$
( 569
)
$
( 607
)
Other comprehensive income (loss):
Foreign currency translation adjustments
3
( 2
)
—
Change in unrealized gain (loss) on available-for-sale securities
( 1
)
—
8
Other comprehensive income (loss):
2
( 2
)
8
Total comprehensive loss
$
( 573
)
$
( 571
)
$
( 599
)
See accompanying notes.
F- 5
ULTRAGENYX PHARMACEUTICAL INC.
CONSOLIDATED STATE MENTS OF STOCKHOLDERS’ EQUITY (DEFICIT)
(In millions)
Common Stock
Additional
Paid-In
Accumulated
Other
Comprehensive
Accumulated
Treasury
Deferred Compensation
Total
Stockholders'
Shares
Capital
Income (Loss)
Deficit
Stock
Obligation
Equity (Deficit)
Balance as of December 31, 2022
70.2
$
3,140
$
( 7
)
$
( 2,781
)
$
—
$
—
$
352
Issuance of common stock and pre-funded warrants in connection with underwritten
public offering, net of issuance costs
9.8
326
—
—
—
—
326
Issuance of common stock in connection with
at-the-market offering, net of issuance costs
1.2
53
—
—
—
—
53
Stock-based compensation
—
135
—
—
—
—
135
Issuance of common stock under
equity plan awards, net of tax
1.1
8
—
—
—
—
8
Other comprehensive income
—
—
8
—
—
—
8
Net loss
—
—
—
( 607
)
—
—
( 607
)
Balance as of December 31, 2023
82.3
$
3,662
$
1
$
( 3,388
)
$
—
$
—
$
275
Issuance of common stock and pre-funded warrants in connection with underwritten
public offering, net of issuance costs
8.8
381
—
—
—
—
381
Stock-based compensation
—
159
—
—
—
—
159
Issuance of common stock under
equity plan awards, net of tax
1.4
11
—
—
—
—
11
Deferred compensation
—
—
—
—
( 4
)
4
—
Other comprehensive loss
—
—
( 2
)
—
—
—
( 2
)
Net loss
—
—
—
( 569
)
—
—
( 569
)
Balance as of December 31, 2024
92.5
$
4,213
$
( 1
)
$
( 3,957
)
$
( 4
)
$
4
$
255
Issuance of common stock in connection with
at-the-market offering, net
2.2
80
—
—
—
—
80
Stock-based compensation
—
152
—
—
—
—
152
Issuance of common stock under
equity plan awards, net of tax
1.9
6
—
—
—
—
6
Deferred compensation
—
—
—
—
( 4
)
4
—
Other comprehensive income
—
—
2
—
—
2
Net loss
—
—
—
( 575
)
—
—
( 575
)
Balance as of December 31, 2025
96.6
$
4,451
$
1
$
( 4,532
)
$
( 8
)
$
8
$
( 80
)
See accompanying notes.
F- 6
ULTRAGENYX PHARMACEUTICAL INC.
CONSOLIDATED STA TEMENTS OF CASH FLOWS
(in millions)
Year Ended December 31,
2025
2024
2023
Operating activities:
Net loss
$
( 575
)
$
( 569
)
$
( 607
)
Adjustments to reconcile net loss to net cash used in operating activities:
Stock-based compensation
153
158
135
Amortization of discount on marketable securities, net
( 4
)
( 13
)
( 13
)
Depreciation and amortization
35
36
26
Non-cash royalty revenue
( 112
)
( 101
)
( 69
)
Non-cash interest expense on liabilities for sales of future royalties
62
63
66
Other
( 2
)
( 2
)
3
Changes in operating assets and liabilities:
Accounts receivable
( 31
)
( 34
)
( 23
)
Inventory
( 6
)
( 11
)
( 7
)
Prepaid expenses and other assets
( 10
)
12
15
Accounts payable, accrued, and other liabilities
24
47
( 1
)
Net cash used in operating activities
( 466
)
( 414
)
( 475
)
Investing activities:
Purchase of property, plant, and equipment
( 6
)
( 7
)
( 44
)
Purchase of marketable securities
( 186
)
( 409
)
( 526
)
Proceeds from sale of marketable securities
—
3
51
Proceeds from maturities of marketable securities
444
410
696
Payments for intangible asset
( 15
)
( 13
)
( 3
)
Other
( 1
)
( 2
)
( 6
)
Net cash provided by (used in) investing activities
236
( 18
)
168
Financing activities:
Proceeds from the sale of future royalties, net
392
—
—
Proceeds from the issuance of common stock and pre-funded warrants in connection with underwritten
public offerings, net
—
381
326
Proceeds from the issuance of common stock in connection with at-the-market
offering, net
80
—
53
Proceeds from the issuance of common stock under equity plans, net
6
11
8
Proceeds from issuance of equity from noncontrolling interest
—
7
—
Other
—
—
1
Net cash provided by financing activities
478
399
388
Effect of exchange rate changes on cash
4
( 2
)
—
Net increase (decrease) in cash, cash equivalents, and restricted cash
252
( 35
)
81
Cash, cash equivalents, and restricted cash at beginning of year
184
219
138
Cash, cash equivalents, and restricted cash at end of year
$
436
$
184
$
219
See accompanying notes.
F- 7
ULTRAGENYX PHARMACEUTICAL INC.
CONSOLIDATED STATEMENTS OF CASH FLOWS
(in millions)
Year Ended December 31,
2025
2024
2023
Supplemental disclosures of non-cash information:
Acquired lease liabilities arising from obtaining right-of-use assets and property, plant, and equipment
$
6
$
10
$
22
Non-cash interest expense on liabilities for sales of future royalties capitalized during the year into ending property, plant and equipment
$
—
$
—
$
9
Costs of intangible assets included in accounts payable, accrued and other liabilities
$
5
$
15
$
10
See accompanying notes.
F- 8
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements
1. Organizat ion and Basis of Presentation
Ultragenyx Pharmaceutical Inc., or the Company, is a biopharmaceutical company incorporated in Delaware.
The Company is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultra-rare genetic diseases. The Company operates as one reportable segment and has four commercially approved products.
Crysvita® (burosumab) is approved in the United States, or U.S., the European Union, or EU, and certain other regions for the treatment of X-linked hypophosphatemia, or XLH, in adult and pediatric patients one year of age and older. Crysvita is also approved in the U.S. and certain other regions for the treatment of fibroblast growth factor 23, or FGF23-related hypophosphatemia in tumor-induced osteomalacia, or TIO, associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adults and pediatric patients 2 years of age and older.
Mepsevii® (vestronidase alfa) is approved in the U.S., the EU and certain other regions, as the first medicine for the treatment of children and adults with mucopolysaccharidosis VII, or MPS VII, also known as Sly syndrome.
Dojolvi® (triheptanoin) is approved in the U.S. and certain other regions for the treatment of pediatric and adult patients severely affected by long-chain fatty acid oxidation disorders, or LC-FAOD.
Evkeeza® (evinacumab) is approved in the U.S. and the European Economic Area, or EEA, and Japan for the treatment of homozygous familial hypercholesterolemia, or HoFH. The Company has exclusive rights to commercialize Evkeeza® (evinacumab) outside of the U.S.
In addition to the approved products, the Company has the following ongoing clinical development programs:
• UX111 (formerly ABO-102) is an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease;
• DTX401 is an adeno-associated virus 8, or AAV8, gene therapy product candidate for the treatment of patients with glycogen storage disease type Ia, or GSDIa;
• GTX-102 is an antisense oligonucleotide, or ASO for the treatment of Angelman syndrome, a debilitating and rare neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene;
• DTX301 is an AAV8 gene therapy product candidate in development for the treatment of patients with ornithine transcarbamylase, or OTC deficiency, the most common urea cycle disorder;
• UX701 is an adeno-associated virus 9, or AAV9, gene therapy designed to deliver stable expression of a truncated version of the ATP7B copper transporter following a single intravenous infusion to improve copper distribution and excretion from the body and reverse pathological findings of Wilson liver disease; and
• UX143 which is subject to the Company ’ s collaboration agreement with Mereo BioPharma 3, or Mereo, is a fully human monoclonal antibody that inhibits sclerostin, a protein that acts on a key bone-signaling pathway and inhibits the activity of bone-forming cells for the treatment of patients with Osteogenesis Imperfecta, or OI.
The Company has sustained operating losses and expects such annual losses to continue in the near term. The Company’s ultimate success depends on the outcome of its research and development and commercialization activities. Through December 31, 2025 , the Company has relied primarily on the sale of equity securities, revenues from commercial products, the sale of certain future royalties, and strategic collaboration arrangements to finance its operations. The Company may need to raise additional capital to fully implement its business plans through the issuance of equity, borrowings, or strategic alliances with partner companies. However, if such financing is not available at adequate levels, the Company would need to reevaluate its operating plans.
2. Summary of Significant Accounting Policies
Basis of Consolidation
The Consolidated Financial Statements include the accounts of the Company and its wholly-owned subsidiaries. The Company cons olidates any variable interest entity, or VIE, for which it is the primary beneficiary. Certain reclassifications have been made to prior periods in the consolidated financial statements and accompanying notes to conform with the current presentation.
F- 9
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
Segment Reporting
The Company operates as one reportable segment relating to the research, development and commercialization of its products. The segment derives its current revenues from its four commercially approved products.
The Company’s Chief Operating Decision Maker, or CODM, is the Company's Chief Executive Officer . The CODM manages the Company’s operations on an integrated basis for the purpose of allocating resources. When evaluating the Company’s financial performance, the CODM regularly reviews total revenues and total expenses and makes decisions using this information on a consolidated basis.
Use of Estimates
The accompanying Consolidated Financial Statements have been prepared in accordance with U.S. generally accepted accounting principles, or GAAP. The preparation of the Consolidated Financial Statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent liabilities and the reported amounts of expenses in the Consolidated Financial Statements and the accompanying notes. On an ongoing basis, management evaluates its estimates, including those related to clinical trial accruals, fair value of assets and liabilities, income taxes, stock-based compensation, revenue recognition, and the liabilities for sales of future royalties. Management bases its estimates on historical experience and on various other market-specific and relevant assumptions that management believes to be reasonable under the circumstances. Actual results could differ from those estimates.
Cash, Cash Equivalents, and Restricted Cash
The Company considers all highly liquid investments with original maturities of three months or less from the date of purchase to be cash equivalents. Cash equivalents consist primarily of amounts invested in money market accounts.
Restricted cash primarily consists of money market accounts used as collateral for the Company’s obligations under its facility leases and to guarantee the fulfillment of certain sales orders to certain government-sponsored customers.
The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Consolidated Statements of Cash Flows (in millions):
December 31,
2025
2024
2023
Cash and cash equivalents
$
421
$
174
$
214
Restricted cash included in other current assets
13
7
2
Restricted cash included in other non-current assets
2
3
3
Total cash, cash equivalents, and restricted cash
shown in the statements of cash flows
$
436
$
184
$
219
Marketable Securities
All marketable securities have been classified as “available-for-sale” and are carried at estimated fair value as determined based upon quoted market prices or pricing models for similar securities. Management determines the appropriate classification of its investments at the time of purchase and reevaluates such designation as of each balance sheet date. Investments with a maturity of one year or less from the balance sheet date are reported as current marketable securities and investments with a maturity of greater than one year from the balance sheet date are reported as non-current marketable securities. Unrealized gains and losses are excluded from earnings and are reported as a component of comprehensive loss. Realized gains and losses and declines in fair value judged to be other than temporary, if any, on available-for-sale securities are included in other expense. The cost of securities sold is based on the specific-identification method. Interest on investments is included in interest income.
Equity Investments
The Company records investments in equity securities, other than equity method investments, at fair market value, using market quotes when readily determinable. Equity securities with no readily determinable fair values are recorded using the measurement alternative of cost adjusted for observable price changes in orderly transactions for identical or similar investments of the same issuer less impairment, if any. Investments in equity securities are recorded in other assets on the Company's Consolidated Balance Sheets. Unrealized gains and losses are reported in change in fair value of equity investments on the Company’s Consolidated Statements of Operations. The Company regularly reviews its non-marketable equity securities for indicators of impairment.
F- 10
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
Concentration of Credit Risk, Credit Losses, and Other Risks and Uncertainties
Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash, cash equivalents, and investments. The Company’s cash, cash equivalents, and investments are held by financial institutions that management believes are of high credit quality. The Company’s investment policy limits investments to fixed income securities denominated and payable in U.S. dollars such as U.S. government obligations, money market instruments and funds, corporate bonds, commercial paper, and asset-backed securities and places restrictions on maturities and concentrations by type and issuer. Such deposits may, at times, exceed federally insured limits. The Company has not experienced any losses on its deposits of cash and cash equivalents and its accounts are monitored by management to mitigate risk. The Company is exposed to credit risk in the event of default by the financial institutions holding its cash and cash equivalents, corporate issuers, and other financial instruments, to the extent recorded in the Consolidated Balance Sheets.
The Company is exposed to credit losses primarily through receivables from customers and collaborators and through its available-for-sale debt securities. For trade receivables and other financial instruments, the Company uses a forward-looking expected loss model that recognizes a current period charge for losses that are expected to be incurred over the life of the financial instrument.
The Company’s expected loss allowance methodology for the receivables is developed using historical collection experience, current and future economic market conditions, a review of the current aging status and financial condition of the entities. Specific allowance amounts are established to record the appropriate allowance for customers that have a higher probability of default. Balances are written off when determined to be uncollectible. The Company’s expected loss allowance methodology for the debt securities is developed by reviewing the extent of the unrealized loss, the size, term, geographical location, and industry of the issuer, the issuers’ credit ratings and any changes in those ratings, as well as reviewing current and future economic market conditions and the issuers’ current status and financial condition. There were no material credit losses recorded for receivables and available-for-sale debt securities which were attributable to credit risk for the years ended December 31, 2025 and 2024.
For available-for-sale debt securities with unrealized losses, the losses are recognized as allowances rather than as reductions in the amortized cost of the securities. There was no allowance for losses on available-for-sale debt securities which were attributable to credit risk for the years ended December 31, 2025 and 2024.
The Company is dependent on third-party manufacturers to supply products for research and development activities in its programs. In particular, the Company relies and expects to continue to rely on a small number of manufacturers to supply it with its requirements for the active pharmaceutical ingredients and formulated drugs related to these programs. These programs could be adversely affected by a significant interruption in the supply of active pharmaceutical ingredients and formulated drugs.
Inventory
The Company values inventory at the lower of cost and net realizable value and determines the cost of inventory using the average cost method. The Company expenses costs associated with the manufacture of product candidates prior to regulatory approval. Inventories consist of currently approved products. The Company periodically reviews its inventories for excess amounts or obsolescence and writes down obsolete or otherwise unmarketable inventory to its estimated net realizable value. Management determines excess inventory based on expected future demand. Estimates related to future demand are sensitive to significant inputs and assumptions such as acceptance by patients and physicians and the availability of formulary coverage and adequate reimbursement from private third-party payers for the product.
Property, Plant, and Equipment
Property, plant, and equipment are stated at cost, less accumulated depreciation and amortization. Depreciation and amortization is computed using the straight-line method over the estimated useful lives of the respective assets. Depreciation and amortization begins when the asset is placed in service. Interest costs incurred during the construction of major capital projects are capitalized until the underlying asset is ready to be placed in service. Maintenance and repairs are charged to operations as incurred. Upon sale or retirement of assets, the cost and related accumulated depreciation or amortization are removed from the balance sheet and the resulting gain or loss, if any, is reflected in operations. See “Note 4. Balance Sheet Components” for further disclosure on the useful lives of property, plant, and equipment.
F- 11
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
Intangible Assets
Finite-lived intangibles consist of contractual payments made in conjunction with license agreements and collaboration agreements. The contractual payments are recorded as intangible assets and are amortized over their estimated useful lives. The Company reviews its definite-lived intangible assets when events or circumstances may indicate that the carrying value of these assets is not recoverable and exceeds their fair value. The Company measures fair value based on the estimated future undiscounted cash flows associated with these assets in addition to other assumptions and projections that the Company deems to be reasonable and supportable.
Indefinite-lived intangibles consist of acquired in-process research and development, or IPR&D. IPR&D assets represent capitalized incomplete research projects that the Company acquired through business combinations. Such assets are initially measured at their acquisition date fair values and are tested for impairment annually and whenever events or changes in circumstances indicate that the carrying amount may not be recoverable. When development of the project is complete, which generally occurs when regulatory approval to market a product is obtained, the associated assets will be deemed finite-lived and will be amortized over a period that best reflects the economic benefits provided by these assets.
If it is determined that an intangible asset becomes impaired, the carrying value is written down to its fair value with the related impairment charge recognized in Consolidated Statements of Operations in the period in which the impairment occurs. The Company has not recorded any impairments of intangible assets to date.
Goodwill
Goodwill represents the excess of purchase price over fair value of net assets acquired in a business combination and is not amortized. Goodwill is subject to impairment testing at least annually during the fourth quarter or when a triggering event occurs that could indicate a potential impairment. If it is determined that the goodwill becomes impaired, the carrying value is written down to its fair value with the related impairment charge recognized in Consolidated Statements of Operations in the period in which the impairment occurs. The Company has not recorded any impairments of goodwill.
Impairment of Long-Lived Assets
The Company evaluates its long-lived assets, including property and equipment, for impairment whenever events or changes in circumstances indicate that the carrying value of these assets may not be recoverable. Recoverability of these assets is measured by comparison of the carrying amount of each asset to the future undiscounted cash flows expected to result from the use of the asset and its eventual disposition. If the asset is considered to be impaired, the amount of any impairment is measured as the difference between the carrying value and the fair value of the impaired asset. The Company has not recorded material impairment of any long-lived assets.
Accruals of Research and Development Costs
The Company records accruals for estimated costs of research, preclinical and clinical studies and manufacturing development. These costs are a significant component of the Company’s research and development expenses. A substantial portion of the Company’s ongoing research and development activities are conducted by third-party service providers, including contract research organizations. The Company accrues the costs incurred under its agreements with these third parties based on actual work completed in accordance with agreements established with these third parties. The Company determines the actual costs through obtaining information from external service providers as to the progress or stage of completion of the services and the agreed-upon fee to be paid for such services.
Liabilities for Sales of Future Royalties
The Company sold the right to receive certain royalty payments from net sales of Crysvita in certain territories to RPI Finance Trust, or RPI, an affiliate of Royalty Pharma, and to OCM LS23 Holdings LP, an investment vehicle for Ontario Municipal Employees Retirement System, or OMERS, as further described in “Note 10. Liabilities for Sales of Future Royalties.” At inception, the Company recorded a liability based upon estimated future cash flows discounted at a market rate. The liability is amortized using the effective interest method over the estimated life of the applicable arrangement. To determine the amortization of the liability, the Company estimates the total amount of future royalty payments to be received by the Company and paid to RPI and OMERS. Any estimated royalty payments in excess of the initial liability are recorded as non-cash interest expense. Consequently, the Company estimates imputed interest on the unamortized portion of the liabilities and records interest expense based on the estimated term of the arrangements.
F- 12
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
The Company periodically assesses the expected royalty payments using a combination of historical results, internal projections and forecasts from external sources. To the extent such payments are greater or less than the Company’s initial estimates or the timing of such payments is materially different than its original estimates, the Company employs the prospective method to adjust the amortization of the liabilities and the effective interest rate.
Revenue Recognition
Product Sales
The Company sells its approved products through a limited number of distributors. Under Accounting Standards Codification, or ASC, 606, Revenue from Contracts with Customers, revenue from product sales is recognized at the point in time when control is transferred to these distributors. The Company also recognizes revenue from sales of certain products on a “named patient” basis, which are allowed in certain countries prior to the commercial approval of the product. Prior to recognizing revenue, the Company makes estimates of the transaction price, including any variable consideration that is subject to a constraint. Amounts of variable consideration are included in the transaction price to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur and when the uncertainty associated with the variable consideration is subsequently resolved. Product sales are recorded net of estimated government-mandated rebates and chargebacks, estimated product returns, and other deductions.
Provisions for returns and other adjustments are provided for in the period the related revenue is recorded, as estimated by management. These reserves are based on estimates of the amounts earned or to be claimed on the related sales and are reviewed periodically and adjusted as necessary. The Company’s estimates of government mandated rebates, chargebacks, estimated product returns, and other deductions depend on the identification of key customer contract terms and conditions, as well as estimates of sales volumes to different classes of payors. If actual results vary, the Company may need to adjust these estimates, which could have a material effect on earnings in the period of the adjustment.
Collaboration, License, and Royalty Revenue
The Company has certain license and collaboration agreements that are within the scope of ASC 808, Collaborative Agreements , which provides guidance on the presentation and disclosure of collaborative arrangements. Generally, the classification of the transactions under the collaborative arrangements is determined based on the nature of contractual terms of the arrangement, along with the nature of the operations of the participants. The Company records its share of collaboration revenue, net of transfer pricing related to net sales in the period in which such sales occur, if the Company is considered as an agent in the arrangement. The Company is considered an agent when the collaboration partner controls the product before transfer to the customers and has the ability to direct the use of and obtain substantially all of the remaining benefits from the product. Funding received related to research and development services and commercialization costs is generally classified as a reduction of research and development expenses and selling, general and administrative expenses, respectively, in the Consolidated Statements of Operations, because the provision of such services for collaborative partners are not considered to be part of the Company’s ongoing major or central operations.
The Company utilizes certain information from its collaboration partners to record collaboration revenue, including revenue from the sale of the product, associated reserves on revenue, and costs incurred for development and sales activities. For the periods covered in the financial statements presented, there have been no material changes to prior period estimates of revenues and expenses. The Company also records royalty revenues under certain of the Company’s license or collaboration agreements in exchange for the license of intellectual property.
As described in “Note 10. Liabilities for Sales of Future Royalties”, for certain royalty payments from net sales of Crysvita in applicable territories that were sold to RPI and OMERS, the Company records the non-cash royalty revenue on a prospective basis as royalty revenue in the Consolidated Statements of Operations over the term of the applicable arrangement.
The terms of the Company’s collaboration and license agreements may contain multiple performance obligations, which may include licenses and research and development activities. The Company evaluates these agreements under ASC 606, Revenue from Contracts with Customers , to determine the distinct performance obligations. The Company analogizes to ASC 606 for the accounting for distinct performance obligations for which there is a customer relationship. Prior to recognizing revenue, the Company makes estimates of the transaction price, including variable consideration that is subject to a constraint. Amounts of variable consideration are included in the transaction price to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur and when the uncertainty associated with the variable consideration is subsequently resolved. Total consideration may include nonrefundable upfront license fees, payments for research and
F- 13
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
development activities, reimbursement of certain third-party costs, payments based upon the achievement of specified milestones, and royalty payments based on product sales derived from the collaboration.
If there are multiple distinct performance obligations, the Company allocates the transaction price to each distinct performance obligation based on its relative standalone selling price. The standalone selling price is generally determined based on the prices charged to customers or using expected cost-plus margin. The Company estimates the efforts needed to complete the performance obligations and recognizes revenue by measuring the progress towards complete satisfaction of the performance obligations using input measures.
Deferred Compensation Plan
The Company maintains a nonqualified deferred compensation plan whereby certain employees and members of the board of directors are able to defer certain equity awards and other compensation. Amounts deferred are invested into shares of the Company's common stock and corporate-owned life insurance. The plan complies with the provisions of Section 409A of the Internal Revenue Code. All the investments held in the plan are recorded in other non-current assets in the Consolidated Balance Sheets. The short-term portion of the corresponding liability for the plan is included in accrued expenses. The long-term portion of the liability is included in other non-current liabilities in the Consolidated Balance Sheets. Changes in the value of the deferred compensation assets and liabilities are recorded in earnings as they occur. Certain equity awards deferred under the plan are required to be settled through the issuance of Company stock. These awards are recorded as treasury stock and deferred compensation obligation within stockholders’ equity (deficit).
Leases
Lease agreements are evaluated to determine whether an arrangement is or contains a lease in accordance with ASC 842, Leases . The Company determines if an arrangement includes a lease at inception. Right-of-use lease assets and lease liabilities are recognized based on the present value of the future minimum lease payments over the lease term at the commencement date. The right-of-use lease asset includes any lease payments made and excludes lease incentives. Incremental borrowing rate is used in determining the present value of future payments. The Company applies a portfolio approach to the property leases to apply an incremental borrowing rate to leases with similar lease terms. The lease terms may include options to extend or terminate the lease. The Company recognizes the options to extend the lease as part of the right-of-use lease assets and lease liabilities only if it is reasonably certain that the option would be exercised. Lease expense for minimum lease payments is recognized on a straight-line basis over the non-cancelable lease term. The Company has elected to not separate lease and non-lease components. See “Note 9. Leases” for further disclosure.
Comprehensive Loss
Comprehensive loss is the change in stockholders’ equity (deficit) from transactions and other events and circumstances other than those resulting from investments by stockholders and distributions to stockholders. The Company’s other comprehensive loss is comprised of unrealized gains and losses on investments in available-for-sale securities and foreign currency translation adjustments.
Research and Development
Research and development costs are expensed as incurred and consist of salaries and benefits, stock-based compensation expense, lab supplies and facility costs, as well as fees paid to other nonemployees and entities that conduct certain research and development activities on the Company’s behalf. Amounts incurred in connection with license agreements are also included in research and development expense. Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are deferred. The deferred amounts are expensed as the related goods are delivered or the services are performed.
Stock-Based Compensation
Stock-based awards issued to employees, including stock options, performance stock options, or PSOs, restricted stock units, or RSUs, and performance stock units, or PSUs are recorded at fair value as of the grant date and recognized as expense on a straight-line basis over the employee’s requisite service period (generally the vesting period). PSOs and PSUs vest only if certain specified criteria are achieved and the employees’ continued service requirements are met; therefore, the expense recognition occurs when the likelihood of the PSOs and PSUs being earned is deemed probable. Stock compensation expense on awards expected to vest is recognized net of estimated forfeitures.
F- 14
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
Income Taxes
The Company uses the liability method of accounting for income taxes. Under this method, deferred tax assets and liabilities are determined based on the differences between the financial reporting and the tax bases of assets and liabilities and are measured using the enacted tax rates and laws that will be in effect when the differences are expected to reverse. The Company must then assess the likelihood that the resulting deferred tax assets will be realized. A valuation allowance is provided when it is more likely than not that some portion or all of a deferred tax asset will not be realized. Due to the Company’s lack of earnings history, the net deferred tax assets have been fully offset by a valuation allowance.
In conjunction with the acquisition of Dimension Therapeutics, Inc., or Dimension, a deferred tax liability was recorded reflecting the tax impact of the difference between the book basis and tax basis of acquired IPR&D. Such deferred income tax liability is not used to offset deferred tax assets when analyzing the Company’s valuation allowance as the acquired IPR&D is considered to have an indefinite life until the Company completes or abandons development of the acquired IPR&D.
The Company recognizes benefits of uncertain tax positions if it is more likely than not that such positions will be sustained upon examination based solely on their technical merits, as the largest amount of benefit that is more likely than not to be realized upon the ultimate settlement. The Company’s policy is to recognize interest and penalties related to the underpayment of income taxes as a component of income tax expense or benefit. To date, there have been no interest or penalties charged in relation to the unrecognized tax benefits.
Foreign Currency
Assets and liabilities of non-U.S. subsidiaries that operate in a local currency environment, where the local currency is the functional currency, are translated to U.S. dollars at exchange rates in effect at the balance sheet date, with the resulting translation adjustments directly recorded to a separate component of accumulated other comprehensive loss. Income and expense accounts are translated at average exchange rates for the period. Transactions which are not in the functional currency of the entity are remeasured into the functional currency and gains or losses resulting from the remeasurement recorded in other expense.
Net Loss per Share
Basic net loss per share is calculated by dividing the net loss by the weighted-average number of shares of common stock outstanding during the period, without consideration for common stock equivalents. Shares of common stock into which pre-funded warrants may be exercised are considered outstanding for the purposes of computing basic net loss per share because the shares may be issued for little or no consideration, are fully vested and are exercisable after the original issuance date. Diluted net loss per share is the same as basic net loss per share, since the effects of potentially dilutive securities are antidilutive. In periods when we have incurred a net loss, options and warrants to purchase common stock are considered common stock equivalents, but have been excluded from the calculation of diluted net loss per share, as their effect is antidilutive.
Recent Accounting Pronouncements
In November 2024, the FASB issued ASU 2024-03, Income Statement-Reporting Comprehensive Income-Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses , requiring public entities to disclose additional information about specific expense categories in the notes to the financial statements on an interim and annual basis. ASU 2024-03 is effective for fiscal years beginning after December 15, 2026, and for interim periods beginning after December 15, 2027, with early adoption permitted. The Company is currently evaluating the impact of adopting ASU 2024-03.
In December 2023, the FASB issued ASU 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures requiring public entities to disclose disaggregated information about their effective tax rate reconciliation and additional information about income taxes paid. Disclosure requirements will be applied on a prospective basis. ASU 2023-09 is effective for fiscal years beginning after December 15, 2024. The Company adopted ASU 2023-09 during the year ended December 31, 2025.
F- 15
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
3. Fair Value Measurements
Certain financial assets and liabilities are recorded at fair value. The carrying amount of certain financial instruments, including cash and cash equivalents, accounts receivable, accounts payable and accrued liabilities approximate fair value due to their relatively short maturities. The carrying amounts of liabilities for the sales of future royalties also approximate their fair value. Assets and liabilities recorded at fair value on a recurring basis in the balance sheets are categorized based upon the level of judgment associated with the inputs used to measure their fair values. Fair value is defined as the exchange price that would be received for an asset or an exit price that would be paid to transfer a liability in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. The authoritative guidance on fair value measurements establishes a three-tier fair value hierarchy for disclosure of fair value measurements as follows:
Level 1 —Inputs are unadjusted, quoted prices in active markets for identical assets or liabilities at the measurement date;
Level 2 —Inputs are observable, unadjusted quoted prices in active markets for similar assets or liabilities, unadjusted quoted prices for identical or similar assets or liabilities in markets that are not active, or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the related assets or liabilities; and
Level 3 —Unobservable inputs that are significant to the measurement of the fair value of the assets or liabilities that are supported by little or no market data.
The Company’s financial instruments consist of Level 1, Level 2, and Level 3 assets. Where quoted prices are available in an active market, securities are classified as Level 1. Money market funds and U.S. Government treasury bills are classified as Level 1. Level 2 assets consist primarily of corporate bonds, asset backed securities, commercial paper, U.S. Government Treasury and agency securities, and debt securities in government-sponsored entities based upon quoted market prices for similar movements in active markets, quoted prices for identical or similar instruments in markets that are not active and model-based valuation techniques for which all significant inputs are observable in the market or can be corroborated by observable market data for substantially the full term of the assets. Where applicable these models project future cash flows and discount the future amounts to a present value using market-based observable inputs obtained from various third-party data providers, including but not limited to, benchmark yields, interest rate curves, reported trades, broker/dealer quotes and reference data.
The Company determines the fair value of its equity investment in Solid Biosciences, Inc., or Solid, by using the quoted market prices, which are Level 1 fair value measurements.
The following tables set forth the fair value of the Company’s financial assets and liabilities remeasured on a recurring basis based on the three-tier fair value hierarchy (in millions):
December 31, 2025
Level 1
Level 2
Level 3
Total
Financial Assets:
Money market funds
$
320
$
—
$
—
$
320
Time deposits
—
10
—
10
Corporate bonds
—
263
—
263
Commercial paper
—
37
—
37
U.S. Government Treasury and agency securities
12
23
—
35
Investment in Solid common stock
3
—
—
3
Deferred compensation assets
—
19
—
19
Total financial assets
$
335
$
352
$
—
$
687
Financial Liabilities:
Deferred compensation liabilities
$
—
$
19
$
—
$
19
F- 16
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
December 31, 2024
Level 1
Level 2
Level 3
Total
Financial Assets:
Money market funds
$
114
$
—
$
—
$
114
Time deposits
—
10
—
10
Corporate bonds
—
392
—
392
Commercial paper
—
21
—
21
U.S. Government Treasury and agency securities
—
159
—
159
Investment in Solid common stock
2
—
—
2
Deferred compensation assets
—
15
—
15
Total financial assets
$
116
$
597
$
—
$
713
Financial Liabilities:
Deferred compensation liabilities
$
—
$
16
$
—
$
16
Deferred compensation liabilities consist of short-term liabilities of $ 1 million and $ 1 million as of December 31, 2025 and 2024 , respectively, included in accrued liabilities on the Consolidated Balance Sheets, and long-term liabilities of $ 18 million and $ 15 million as of December 31, 2025 and 2024, respectively, included in other non-current liabilities on the Consolidated Balance Sheets. There have been no material net gains or losses on deferred compensation assets or liabilities for the periods presented.
4. Balance Sheet Components
Cash Equivalents and Marketable Securities
The fair values of cash equivalents and marketable securities classified as available-for-sale securities consist of the following (in millions):
December 31, 2025
Amortized
Gross Unrealized
Estimated
Cost
Gains
Losses
Fair Value
Money market funds
$
320
$
—
$
—
$
320
Time deposits
10
—
—
10
Corporate bonds
263
—
—
263
Commercial paper
37
—
—
37
U.S. Government Treasury and agency securities
35
—
—
35
Total
$
665
$
—
$
—
$
665
December 31, 2024
Amortized
Gross Unrealized
Estimated
Cost
Gains
Losses
Fair Value
Money market funds
$
114
$
—
$
—
$
114
Time deposits
10
—
—
10
Corporate bonds
391
1
392
Commercial paper
21
—
—
21
U.S. Government Treasury and agency securities
159
—
—
159
Total
$
695
$
1
$
—
$
696
At December 31, 2025 , the remaining contractual maturities of available-for-sale securities were less than two years . The cost of securities sold is based on the specific-identification method. There have been no significant realized gains or losses on available-for-sale securities for the periods presented. All marketable securities with unrealized losses at December 31, 2025 have been in a loss position for less than 12 months or the loss is not material and is temporary in nature.
F- 17
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
Inventory
Inventory consists of the following (in millions):
December 31,
2025
2024
Work-in-process
$
27
$
22
Finished goods
25
23
Total inventory
$
52
$
45
Property, Plant, and Equipment, net
Property, plant, and equipment, net consists of the following (in millions):
December 31,
Useful life (years)
2025
2024
Building
20 - 30
$
182
$
182
Leasehold improvements
Shorter of lease term or estimated useful life
55
58
Research and development equipment
5
65
60
Furniture and office equipment
5
7
6
Computer equipment and software
3 - 5
16
16
Manufacturing equipment
5 - 15
37
37
Land
Not applicable
17
17
Other
Varies by asset
1
2
Property, plant, and equipment, gross
380
378
Less: accumulated depreciation
( 136
)
( 112
)
Property, plant, and equipment, net
$
244
$
266
Depreciation expense for the years ended December 31, 2025, 2024, and 2023 was $ 27 million, $ 30 million and $ 22 million, respectively. Amortization of leasehold improvements and software is included in depreciation expense.
Accrued Liabilities
Accrued liabilities consist of the following (in millions):
December 31,
2025
2024
Research and clinical study expenses
$
23
$
24
Payroll and related expenses
89
94
Revenue related reserves
57
33
Manufacturing and related expenses
67
25
Commercial and development milestones
5
45
Other
24
20
Total accrued liabilities
$
265
$
241
5. Intangible Assets, net
Indefinite-lived Intangibles
As a result of the accounting for our acquisition of Dimension Therapeutics, Inc. in November 2017, the Company has IPR&D assets of $ 129 million as of December 31, 2025 and 2024. IPR&D assets represent the fair value of acquired programs to develop an AAV gene therapy for OTC deficiency and to develop an AAV gene therapy for glycogen storage disease type Ia. IPR&D assets are considered to be indefinite-life until the completion or abandonment of the associated research and development efforts.
F- 18
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
Finite-lived Intangibles
Subsequent to the FDA approval of Dojolvi for the treatment of LC-FAOD in 2020, the Company recorded $ 5 million for the attainment of various development and commercial milestones as finite-lived intangible assets which are amortized over a weighted-average total useful life of 6 years.
In January 2022, the Company entered into a collaboration with Regeneron to commercialize Evkeeza for HoFH outside of the U.S. Pursuant to the collaboration agreement, the Company has incurred an upfront payment and regulatory and sales milestones to date totaling $ 63 million. As these payments are for the Company’s use of intellectual property for Evkeeza for HoFH, they were recorded as intangible assets , which are amortized over a weighted-average total useful life of 9 years.
The Company's intangible assets were as follows (in millions, except for years):
December 31, 2025
Gross Carrying Amount
Weighted-Average Life (Years)
Accumulated Amortization
Net Carrying Amount
Indefinite-lived intangibles
$
129
—
$
—
$
129
Finite-lived intangibles
68
9
( 21
)
47
Total intangible assets
$
197
—
$
( 21
)
$
176
December 31, 2024
Gross Carrying Amount
Weighted-Average Life (Years)
Accumulated Amortization
Net Carrying Amount
Indefinite-lived intangibles
$
129
—
$
—
$
129
Finite-lived intangibles
62
9
( 13
)
49
Total intangible assets
$
191
—
$
( 13
)
$
178
The Company recorded costs of sales of $ 8 million, $ 6 million and $ 4 million for the years ended December 31, 2025, 2024, and 2023, respectively, related to the amortization of intangible assets.
The expected amortization of intangible assets, as of December 31, 2025, for each of the next five years and thereafter is as follows (in millions):
2026
$
8
2027
7
2028
7
2029
7
2030
7
Thereafter
11
Total
$
47
F- 19
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
6. Revenue
The following table disaggregates total revenues from external customers by product sales, royalty revenue, and collaboration and license revenue (in millions):
Year Ended December 31,
2025
2024
2023
Product sales:
Crysvita
$
177
$
135
$
75
Dojolvi
96
88
71
Evkeeza
59
32
4
Mepsevii
37
30
30
Total product sales
369
285
180
Crysvita royalty revenue
304
275
183
Collaboration and license revenue:
Crysvita collaboration revenue in Profit-Share Territory
—
—
70
Other
—
—
1
Total collaboration and license revenue
—
—
71
Total revenues
$
673
$
560
$
434
The following table disaggregates total revenues based on geographic location (in millions):
Year Ended December 31,
2025
2024
2023
North America
$
377
$
340
$
307
Latin America
170
131
77
Europe, Middle East, and Africa
108
80
48
Asia-Pacific
18
9
2
Total revenues
$
673
$
560
$
434
The following table presents the activity and ending balances for product sales related accruals and allowances (in millions):
Year Ended December 31,
2025
2024
2023
Balance of product sales reserve at beginning of year
$
33
$
17
$
11
Provisions
50
38
19
Payments
( 30
)
( 21
)
( 13
)
Adjustments
4
( 1
)
—
Balance of product sales reserve at end of year
$
57
$
33
$
17
The Company’s largest accounts receivable balance was from a collaboration partner, KKC, and was 62 % and 70 % of the total accounts receivable balance as of December 31, 2025 and 2024 , respectively.
7. Investment in Amlogenyx Inc.
In July 2024, the Company contributed certain intellectual property rights to Amlogenyx Inc., or Amlogenyx, a subsidiary of the Company, and received 9.0 million shares of common stock of Amlogenyx. A third-party investor along with one of its affiliated entities, and the Company, each contributed $ 7 million to Amlogenyx and in exchange, each received approximately 1.6 million shares of series seed preferred stock of Amlogenyx. The purpose of Amlogenyx is to pursue the application of the Company’s novel adeno-associated virus, or AAV, gene therapy to treat beta-amyloid disorders and related neurodegenerative diseases.
Amlogenyx was determined to be a VIE and the Company is the primary beneficiary as it has the power to direct the activities that would most significantly impact the economic performance of Amlogenyx, including the performance of R&D activities relating to its sole product candidate. As the primary beneficiary, the Company has consolidated the financial position, results of operations and cash flows of Amlogenyx in its financial statements and all intercompany balances have been eliminated in consolidation. Upon initial consolidation, the non-controlling interest of the third-party investor was recorded at its estimated fair value of $ 7.0 million, which is equal to their original investment.
F- 20
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
As of December 31, 2025 and 2024 , total assets included on the Consolidated Balance Sheets for Amlogenyx were $ 5 million and $ 14 million, respectively. The assets primarily consisted of cash and cash equivalents which may only be used to settle obligations of Amlogenyx.
Noncontrolling interest related to the third-party investment in Amlogenyx is reported on the Consolidated Balance Sheets in mezzanine equity.
Changes in the carrying value of noncontrolling interest for the year ended December 31, 2025, were as follows (in millions):
Noncontrolling Interest
As of December 31, 2023
$
—
Issuance of equity from noncontrolling interest
7
As of December 31, 2024
$
7
Changes and adjustments
—
As of December 31, 2025
$
7
8. License and Research Agreements
Kyowa Kirin Co., Ltd.
In August 2013, the Company entered into a collaboration and license agreement with Kyowa Kirin Co., Ltd., or KKC. Under the terms of this collaboration and license agreement, as amended, the Company and KKC collaborate on the development and commercialization of Crysvita in the field of orphan diseases in the U.S. and Canada, or the Profit-Share Territory, and in the European Union, UK, and Switzerland, or the European Territory, and the Company has the right to develop and commercialize such products in the field of orphan diseases in Mexico and Central and South America, or Latin America.
The collaboration and license agreements are within the scope of ASC 808, which provides guidance on the presentation and disclosure of collaborative arrangements.
Product Sales Revenue for Latin America and Türkiye
The Company is responsible for commercializing Crysvita in Latin America and Türkiye. The Company is considered the principal in these territories as the Company controls the product before it is transferred to the customer. Accordingly, the Company records revenue on a gross basis for the sale of Crysvita once the product is delivered and the risk and title of the product is transferred to the distributor. In Türkiye, KKC has the option to assume responsibility for commercialization efforts.
Transfer Price and Royalties on Product Sales Revenue
Under the collaboration agreement, KKC manufactures and supplies Crysvita, which is purchased by the Company for sales in Latin America and Türkiye. KKC charges the Company a transfer price of 30 % of net sales for supply of product to Latin America. The Company also pays to KKC a low single-digit royalty on net sales in Latin America.
Collaboration and Royalty Revenue for Sales in the Profit-Share Territory
The Company and KKC shared commercial responsibilities and profits in the Profit-Share Territory until April 2023. Under the collaboration agreement, KKC manufactured and supplied Crysvita for commercial use in the Profit-Share Territory and charged the Company a transfer price of 30 % of net sales in 2023. The remaining profit or loss after supply costs from commercializing products in the Profit-Share Territory was shared between the Company and KKC on a 50 / 50 basis until April 2023. In April 2023, commercialization responsibilities for Crysvita in the Profit-Share Territory transitioned to KKC. Thereafter, the Company is entitled to receive a tiered double-digit revenue share from the mid- 20 % range up to a maximum rate of 30 %.
During the prior profit-share period, as KKC was the principal in the sale transaction with the customer, the Company recognized a pro-rata share of collaboration revenue, net of transfer pricing, in the period the sale occurred. The Company concluded that its portion of KKC’s sales in the Profit-Share Territory prior to April 2023 was analogous to a royalty and therefore recorded its share as collaboration revenue, similar to a royalty. Starting in April 2023, the Company began to record as royalty revenue in the period the underlying sales occurred.
F- 21
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
In July 2022, the Company sold to OMERS its right to receive 30 % of the future royalty payments, beginning in April 2023, that are due to the Company based on net sales of Crysvita in the U.S. and Canada, subject to a cap. In November 2025, the Company sold to OMERS its right to an additional 25 % of the future royalty payments, beginning in January 2028, that are due to the Company based on net sales of Crysvita in the U.S. and Canada, subject to a cap. These agreements are further described in “Note 10. Liabilities for Sales of Future Royalties.”
Royalty Revenue for Sales in the European Territory
KKC has commercial responsibility for Crysvita in the European Territory. Under the collaboration agreement, the Company is entitled to receive royalties of up to 10 % of net sales in the European Territory, which are recorded as royalty revenue as the underlying sales occur. In December 2019, the Company sold its right to receive these royalty payments to Royalty Pharma, effective January 1, 2020, as further described in “Note 10. Liabilities for Sales of Future Royalties.”
Total Crysvita revenue was as follows (in millions):
Year Ended December 31,
2025
2024
2023
Product sales
$
177
$
135
$
75
Revenue in Profit-Share Territory:
Royalty revenue
192
174
113
Non-cash royalty revenue
83
75
49
Collaboration revenue
—
—
70
Total revenue in Profit-Share Territory
275
249
232
Non-cash royalty revenue in European Territory
29
26
21
Total Crysvita revenue
$
481
$
410
$
328
Collaboration Cost Sharing and Payments
Under the collaboration agreement, KKC and the Company share certain development and commercialization costs, and as a result, the Company was reimbursed for these costs and operating expenses were reduced. KKC also receives a transfer price and royalty on net product sales revenue which is recorded in cost of sales. These amounts were recognized in the Company’s Consolidated Statements of Operations in connection with the collaboration agreement with KKC as follows (in millions):
Year Ended December 31,
2025
2024
2023
Research and development
$
( 5
)
$
( 4
)
$
( 7
)
Selling, general and administrative
$
—
$
( 4
)
$
( 17
)
Cost of sales
$
61
$
46
$
18
Collaboration Receivable and Payable
The Company had accounts receivable from KKC in the amount of $ 98 million and $ 85 million from royalties, other receivables of $ 1 million and $ 2 million recorded in other current assets, and accrued liabilities of $ 12 million and $ 7 million from amounts owed for transfer price and royalties as well as commercial and development activity reimbursements, as of December 31, 2025 and 2024, respectively.
Regeneron
In January 2022, the Company entered into a collaboration with Regeneron to commercialize Evkeeza for HoFH outside of the U.S. Pursuant to the terms of the agreement, the Company received the rights to develop, commercialize and distribute the product for HoFH in countries outside of the U.S. The Company paid Regeneron a $ 30 million upfront payment. As of December 31, 2025 , the Company has recognized an aggregate of $ 33 million for regulatory and sales milestones under the agreement. As these payments are for the Company’s use of intellectual property for Evkeeza for HoFH, they were recorded as intangible assets. See "Note 5. Intangible Assets, net" for additional details. The Company is required to pay up to an aggregate of $ 31 million in the future contingent upon the achievement of certain regulatory and sales milestones. The Company may share in certain costs for global trials led by Regeneron. Additionally, the Company pays Regeneron a product purchase price and royalties on certain revenues.
F- 22
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
The collaboration agreement is within the scope of ASC 808 which provides guidance on the presentation and disclosure of collaborative arrangements. As the Company is the principal in sales transactions with the customer, the Company recognizes product sales and cost of sales in the period the related sales occur and the related revenue recognition criteria are met. Under the collaboration agreement, Regeneron supplies the product and charges the Company a product purchase price from the low 20 % range up to 40 % on net sales, which is recognized as cost of sales in the Company’s Consolidated Statement of Operations.
Under the collaboration agreement, Regeneron and the Company share certain development and commercialization costs. Regeneron also receives a product purchase price and royalty on net product sales revenue which is recorded in cost of sales. These amounts were recognized in the Company’s Consolidated Statements of Operations in connection with the collaboration agreement with Regeneron as follows (in millions):
Year Ended December 31,
2025
2024
2023
Research and development
$
1
$
( 3
)
$
8
Cost of sales
$
16
$
8
$
1
The Company had collaboration payables for this arrangement included in accrued liabilities on the Consolidated Balance Sheets of $ 9 million and $ 18 million as of December 31, 2025 and 2024, respectively.
Saint Louis University
In November 2010, the Company entered into a license agreement with Saint Louis University, or SLU. Under the terms of this license agreement, SLU granted the Company an exclusive worldwide license to make, have made, use, import, offer for sale, and sell therapeutics related to SLU’s beta-glucuronidase product for use in the treatment of human diseases.
Under the license agreement, the Company is obligated to pay to SLU a low single-digit royalty on net sales of the licensed products in Europe and Japan, subject to certain potential deductions. The Company's obligation to pay royalties to SLU in these territories continues until the expiration of any orphan drug exclusivity.
Abeona
In May 2022, the Company announced an exclusive License Agreement for the AAV gene therapy for UX111 with Abeona for the treatment of MPS IIIA. Under the terms of the agreement, the Company assumed responsibility for the UX111 program and in return, the Company is obligated to pay tiered royalties of up to 10 % on net sales and commercial milestone payments of up to $ 30.0 million contingent upon regulatory approval of the product. Additionally, the Company entered into an Assignment and Assumption Agreement with Abeona to transfer and assign to the Company the exclusive license agreement between Nationwide Children’s Hospital, or NCH, and Abeona for certain rights related to UX111. Under this agreement, the Company is obligated to pay up to $ 1 million contingent upon achievement of development and regulatory milestones as well as royalties in the low single-digits of net sales.
Mereo
In December 2020, the Company entered into a License and Collaboration Agreement with Mereo to collaborate on the development of setrusumab. Under the terms of the agreement, as amended, the Company will lead future global development of setrusumab in both pediatric and adult patients with OI. The Company was granted an exclusive license to develop and commercialize setrusumab in the U.S., Türkiye, and the rest of the world, or the Ultragenyx Territory, excluding the EEA, UK, and Switzerland, or the Mereo Territory, where Mereo retains commercial rights. Each party will be responsible for post-marketing
F- 23
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
commitments in their respective territories and Ultragenyx will be responsible for commercial supply in both the Ultragenyx Territory and Mereo Territory.
Upon the closing of the transactions under the License and Collaboration Agreement with Mereo in January 2021, the Company made a payment of $ 50 million to Mereo. As of December 31, 2025, the Company has made payments totaling $ 9 million for regulatory milestones achieved. The Company is obligated to pay Mereo up to $ 245 million in future milestone payments, contingent upon the achievement of certain regulatory and commercial milestones. The Company pays for all global development costs and will pay a tiered double-digit percentage royalties to Mereo on net sales in the Ultragenyx Territory. Mereo will pay the Company a fixed double-digit percentage royalty on net sales in the Mereo Territory. If the Company receives and resells an FDA priority review voucher, or PRV, in connection with a new drug application approval, Mereo is entitled to receive a portion of proceeds from the sale of the PRV or a cash payment from the Company, in the event the Company chooses to retain the PRV.
In December 2024, the Company entered into a manufacturing and supply agreement with Mereo where it is responsible for the supply of setrusumab to Mereo in the Mereo Territory. Mereo is responsible for reimbursing us for a portion of the manufacturing process development costs as well as future commercial supply costs.
Although Mereo is a VIE, the Company is not the primary beneficiary as it does not have the power to direct the activities that would most significantly impact the economic performance of Mereo. Prior to the achievement of certain development milestones, all consideration paid to Mereo represents rights to potential future benefits associated with Mereo’s in-process research and development activities, which have not reached technological feasibility and have no alternative future use.
For the years ended December 31, 2025 and 2024 , the Company recorded offsets to research and development expense of $ 5 million and $ 1 million, respectively. For the year ended December 31, 2023, the Company recorded development costs of $ 9 million for the achievement of a clinical milestone recorded in research and development expense.
As of December 31, 2025 and 2024, t he Company had receivables from Mereo in the amount of $ 1 million and $ 1 million, respectively, recorded in other current assets for development activity reimbursements.
GeneTx Biotherapeutics LLC
In August 2019, the Company entered into a Program Agreement and a Unitholder Option Agreement with GeneTx Biotherapeutics LLC, or GeneTx, as subsequently amended, or the Option Agreement, to collaborate on the development of GeneTx’s GTX-102, an ASO for the treatment of Angelman syndrome. In July 2022, the Company exercised its option to acquire GeneTx, pursuant to the terms of the Option Agreement. During the year ended December 31, 2024, the Company achieved a $ 30 million regulatory milestone upon the initiation of the Phase 3 Aspire clinical study for GTX-102. The Company is obligated to pay up to $ 85 million in additional regulatory approval milestones for the achievement of U.S. and EU product approvals, and up to $ 75 million in commercial milestone payments based on annual worldwide net product sales, contingent upon the achievement of the milestones. The Company will also pay tiered mid- to high single-digit percentage royalties based on licensed product annual net sales. If the Company receives and resells an FDA priority review voucher, or PRV, in connection with a new drug application approval, GeneTx unitholders are entitled to receive a portion of proceeds from the sale or a cash payment from the Company if the Company chooses to retain the PRV.
As part of the Company's acquisition of GeneTx, the Company assumed a License Agreement with Texas A&M University, or TAMU. The Company recognized an aggregate of $ 1 million for clinical milestones under the TAMU agreement, and has future obligations up to $ 23 million for various milestones and a nominal annual license fee that may increase up to a maximum of $ 2 million. The Company will also pay mid-single-digit percentage royalties based on licensed product annual net sales. As of December 31, 2025 and 2024 , the Company had nil and $ 1 million, respectively, in collaboration payables under this arrangement.
Prior to the achievement of certain development and regulatory milestones, amounts paid towards the milestones are classified as in-process research and development expense, as the acquired in-process research and development intangible asset has not yet reached technological feasibility and has no alternative future use.
University of Pennsylvania
The Company has a research, collaboration, and license agreement with University of Pennsylvania School of Medicine, or Penn, which provides the terms for the Company and Penn to collaborate with respect to the pre-clinical development of gene therapy products for the treatment of certain indications. Under the agreement, Penn granted the Company an exclusive, worldwide license to certain patent rights arising out of the research program, subject to certain retained rights, and a non-exclusive, worldwide license to certain Penn intellectual property, in each case to research, develop, make, have made, use, sell, offer for sale, commercialize and import licensed products in each indication for the term of the agreement. The Company will fund the cost of the research program in accordance with a mutually agreed-upon research budget and will be responsible for clinical development,
F- 24
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
manufacturing and commercialization of each indication. The Company is obligated to make milestone payments of up to $ 5 million for each indication, if certain development milestones are achieved. The Company is also obligated to make milestone payments of up to $ 25 million per approved product, if certain commercial milestones are achieved, as well as low to mid- single-digit royalties on net sales of each licensed product.
REGENXBIO, Inc.
The Company has a license agreement with REGENXBIO, Inc., or REGENX, for an exclusive, sublicensable, worldwide commercial license under certain intellectual property for preclinical and clinical research and development, and commercialization of drug therapies using REGENX's licensed patents for the treatment of OTC deficiency and GSD1a. The Company will pay an annual fee and certain milestone fees per disease indication, low to mid- single-digit royalty percentages on net sales of licensed products, and milestone and sublicense fees owed by REGENX to its licensors, which are contingent upon the attainment of certain development activities as outlined in the agreement. This license agreement was terminated for certain indications in November 2025.
The Company also had an option and license agreement with REGENX under which the Company had an exclusive, sublicensable, worldwide license to make, have made, use, import, sell, and offer for sale licensed products to treat Wilson disease and CDKL5 deficiency . This option and license agreement was terminated in November 2025.
9. Leases
The Company leases office space and research, testing and manufacturing laboratory space in various facilities in Novato and Brisbane, California, in Somerville and Woburn, Massachusetts, and in certain foreign countries, under operating agreements expiring at various dates through 2031 . Certain lease agreements include options for the Company to extend the lease for multiple renewal periods and provide for annual minimum increases in rent, usually based on a consumer price index or annual minimum increases. None of these optional periods have been considered in the determination of the right-of-use lease asset or the lease liability for the leases as the Company did not consider it reasonably certain that it would exercise any such options. The Company recognizes lease expense on a straight-line basis over the non-cancelable term of its operating leases. The variable lease expense primarily consists of common area maintenance and other operating costs.
The components of lease expense were as follows (in millions):
Year Ended December 31,
2025
2024
2023
Operating lease expense
$
12
$
12
$
13
Variable lease expense
5
6
5
Total
$
17
$
18
$
18
Cash paid for amounts included in the measurement of operating lease liabilities for the years ended December 31, 2025, 2024, and 2023 was $ 14 million, $ 16 million, and $ 13 million, respectively, and was included in net cash used in operating activities in the Consolidated Statements of Cash Flows.
Right-of-use lease assets were $ 23 million and $ 26 million as of December 31, 2025 and 2024, respectively, and were included in other non-current assets on the Consolidated Balance Sheets.
The following table summarizes maturities of lease liabilities and the reconciliation of lease liabilities as of December 31, 2025 (in millions):
Year Ending December 31,
Operating
2026
$
15
2027
10
2028
8
2029
7
2030
2
Thereafter
1
Total future lease payments
43
Less: Amount representing interest
( 7
)
Present value of future lease payments
36
Less: Lease liabilities, current
( 12
)
Lease liabilities, non-current
$
24
F- 25
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
For the years ended December 31, 2025 and 2024, the weighted-average remaining operating lease terms and the weighted-average discount rates used to determine the lease liability were as follows:
Year Ended December 31,
2025
2024
Weighted-average remaining lease term (in years)
4
4
Weighted-average discount rate
10.0
%
10.1
%
10. Liabilities for Sales of Future Royalties
In December 2019, the Company entered into a Royalty Purchase Agreement with RPI. Pursuant to the agreement, RPI paid $ 320 million to the Company in consideration for the right to receive royalty payments effective January 1, 2020, arising from the net sales of Crysvita in the EU, the U.K., and Switzerland under the terms of the Company’s Collaboration and License Agreement with KKC dated August 29, 2013, as amended, or the KKC Collaboration Agreement. The agreement with RPI will automatically terminate, and the payment of royalties to RPI will cease, in the event aggregate royalty payments received by RPI are equal to or greater than $ 608.0 million prior to December 31, 2030, or in the event aggregate royalty payments received by RPI are less than $ 608 million prior to December 31, 2030, or when aggregate royalty payments received by RPI are equal to $ 800 million.
In July 2022, the Company entered into a Royalty Purchase Agreement with OMERS, or the 2022 OMERS Agreement. Pursuant to the agreement, OMERS paid $ 500 million to the Company in consideration for the right to receive 30 % of the future royalty payments due to the Company from KKC based on net sales of Crysvita in the U.S. and Canada under the terms of the KKC Collaboration Agreement. The calculation of royalty payments to OMERS is based on net sales of Crysvita beginning in April 2023 and will expire upon the earlier of the date on which aggregate payments received by OMERS equals $ 725 million or on the date the final royalty payment is made to the Company under the KKC Collaboration Agreement.
In November 2025, the Company entered into a second Royalty Purchase Agreement with OMERS, or the 2025 OMERS Agreement. Pursuant to the agreement, OMERS paid $ 400 million to the Company in exchange for the right to receive an additional 25 % of future royalty payments, starting on January 1, 2028, on net sales of Crysvita in the U.S. and Canada. OMERS will also continue to receive 30 % of Crysvita net sales in the U.S. and Canada following the achievement of $ 725 million in aggregate payments under the 2022 OMERS Agreement. The 2025 OMERS Agreement will expire upon the earlier of the date on which aggregate payments received by OMERS under this transaction equal $ 620 million or on the date the final royalty payment is made to the Company under the KKC Collaboration Agreeme nt. Under the agreement, OMERS granted the Company an option to repurchase the entire purchased interest, that expires in November 2027 , for 1.35 times the purchase price, or $ 540 million .
Proceeds from these transactions were recorded as liabilities for sales of future royalties on the Consolidated Balance Sheets. Upon inception of the respective arrangements, the Company recorded liabilities of $ 320 million, $ 500 million, and $ 400 million, net of transaction costs of $ 6 million, $ 9 million, and $ 8 million, for the RPI Agreement, the 2022 OMERS Agreement, and the 2025 OMERS Agreement, respectively. The Company records the royalty revenue arising from the net sales of Crysvita in the applicable territories as royalty revenue in the Consolidated Statements of Operations over the term of the arrangements. Royalties earned under the RPI Agreement, the 2022 OMERS Agreement, and the 2025 OMERS Agreement from inception to December 31, 2025 have been $ 128 million, $ 206 million, and nil , respectively. The Company’s effective annual interest rates were 5.5 %, 7.3 %, and 9 .2 % for the RPI Agreement, the 2022 OMERS Agreement, and the 2025 OMERS Agreement, respectively, as of December 31, 2025.
There are a number of factors that could materially affect the amount and timing of royalty payments from KKC in the applicable territories, most of which are not within the Company’s control. Such factors include, but are not limited to, the success of KKC’s sales and promotion of Crysvita, changing standards of care, macroeconomic and inflationary pressures, the introduction of competing products, pricing for reimbursement in various territories, manufacturing or other delays, intellectual property matters, adverse events that result in governmental health authority imposed restrictions on the use of Crysvita, significant changes in foreign exchange rates as the royalty payments are made in U.S. dollars, or USD, while significant portions of the underlying sales of Crysvita are made in currencies other than USD, and other events or circumstances that could result in reduced royalty payments from sales of Crysvita, all of which would result in a reduction of royalty revenue and the non-cash interest expense over the life of the arrangement. Conversely, if sales of Crysvita in the relevant territories are more than expected, the royalty revenue and the non-cash interest expense recorded by the Company would be greater over the term of the arrangements.
The following table shows the activity within the liability account (in millions):
F- 26
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
Liabilities for Sales of Future Royalties
RPI
OMERS 2022
OMERS 2025
Total
December 31, 2023
$
377
$
515
$
—
$
892
Royalty revenue
( 26
)
( 59
)
—
( 85
)
Non-cash interest expense
24
39
—
63
December 31, 2024
375
495
—
870
Net proceeds from sale of future royalties
—
—
392
392
Royalty revenue
( 29
)
( 79
)
—
( 108
)
Non-cash interest expense
20
36
6
62
December 31, 2025
$
366
$
452
$
398
$
1,216
11. Equity
At-the-Market Offerings
In February 2024, the Company entered into a Sales Agreement with Cowen and Company, LLC, or Cowen, pursuant to which the Company may offer and sell shares of the Company’s common stock having an aggregate offering proceeds of up to $ 350 million, from time to time, in at-the-market, or ATM, offerings through Cowen. The Company sold 2.2 million shares under the ATM for net proceeds of $ 80 million during the year ended December 31, 2025.
In May 2021, the Company entered into an Open Market Sale Agreement with Jefferies LLC, or Jefferies, pursuant to which the Company may offer and sell shares of the Company’s common stock having an aggregate offering proceeds of up to $ 350 million, from time to time, in ATM offerings through Jefferies. During the year ended December 31, 2023 , there were 1.2 million shares sold under the ATM resulting in net proceeds of $ 53 million.
Underwritten Public Offering
In June 2024, the Company completed an underwritten public offering in which 8.8 million shares of common stock were sold, including the exercise in full by the underwriters of their option to purchase an additional 1.3 million shares, at a public offering price of $ 39.00 per share. In connection with the offering, the Company sold to certain investors pre-funded warrants, in lieu of common stock, to purchase 1.5 million shares of common stock at a purchase price of $ 38.999 per pre-funded warrant, which equals the public offering price per share of common stock less the $ 0.001 exercise price per share of each pre-funded warrant. The total proceeds that the Company received from the offering were $ 381 million, net of underwriting discounts and commissions.
In October 2023, the Company completed an underwritten public offering in which 9.8 million shares of common stock were sold, including the exercise in full by the underwriters of their option to purchase an additional 1.5 million shares, at a public offering price of $ 30.00 per share. In connection with the offering, the Company sold to certain investors pre-funded warrants, in lieu of common stock, to purchase 1.7 million shares of common stock at a purchase price of $ 29.999 per pre-funded warrant, which equals the public offering price per share of common stock less the $ 0.001 exercise price per share of each pre-funded warrant. The total proceeds that the Company received from the offering were $ 327 million, net of underwriting discounts and commissions.
The pre-funded warrants were classified as a component of permanent equity in the Company's Consolidated Balance Sheets as they are freestanding financial instruments that are immediately exercisable, do not embody an obligation for the Company to repurchase its own shares and permit the holders to receive a fixed number of shares of common stock upon exercise. All of the shares underlying the pre-funded warrants have been included in the weighted-average number of shares of common stock used to calculate net loss per share, basic and diluted, attributable to common stockholders because the shares may be issued for little or no consideration, are fully vested, and are exercisable after the original issuance date of the pre-funded warrants. As of December 31, 2025 , no pre-funded warrants had been exercised.
The table below summarizes pre-funded warrants activity:
Pre-funded warrants
(millions)
As of December 31, 2023
1.7
Issuance of pre-funded warrants
1.5
As of December 31, 2024
3.2
Changes and adjustments
—
As of December 31, 2025
3.2
F- 27
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
12. Stock-Based Awards
Equity Plan Awards
Under the terms of the Company's 2023 Incentive Plan, or 2023 Plan, and Employment Inducement Plan, or Inducement Plan, awards may be granted at an exercise price not less than fair market value. The exercise price of an option may not be less than the fair market value. The term of an award granted under the 2023 Plan and Inducement Plan may not exceed ten years . Typically, the vesting schedule for option grants to employees provides that 1/4 of the grant vests upon the first anniversary of the date of grant, with the remainder of the shares vesting monthly thereafter at a rate of 1/48 of the total shares subject to the option. Typically, the vesting schedule for RSU grants provides that 1/4 of the grant vests upon the annual anniversary of the date of grant over the period of four years .
Under the 2014 Employee Stock Purchase Plan, or ESPP, eligible employees may purchase common stock at 85 % of the lesser of the fair market value of common stock on the offering date or the purchase date with a six-month look-back feature. ESPP purchases are settled with common stock from the ESPP’s previously authorized and available pool of shares. During the year ended December 31, 2025 , the Company issued 0.2 million shares of common stock under the ESPP.
The table below summarizes the Company's equity plans as of December 31, 2025:
Plan
Year of Adoption
Expiration Date, as Amended
Maximum Number of Shares Authorized
(millions)
Shares Available for Future Issuance
(millions)
Employment Inducement Plan
2021
February 3, 2031
1.7
0.3
2023 Incentive Plan (1)
2023
June 7, 2033
15.5
7.9
2014 Employee Stock Purchase Plan
2014
June 7, 2033
7.3
6.2
(1) Maximum number of shares authorized and shares available for future issuance under the 2023 Incentive Plan includes 4.0 million shares subject to the 2014 Incentive Plan cancelled after June 7, 2023 .
Stock Option Activity
The following table summarizes activity under the Company’s stock option plans and related information:
Options Outstanding
Number of Options
(millions)
Weighted-Average Exercise Price
Weighted-Average Remaining Contractual Term (Years)
Aggregate Intrinsic Value
(In millions)
Outstanding — December 31, 2024
9.4
$
65.54
6
$
2
Options granted
1.2
41.75
Options exercised
—
38.57
Options cancelled
( 1.7
)
73.53
Outstanding — December 31, 2025
8.9
60.87
6
—
Vested and exercisable — December 31, 2025
6.5
66.31
5
—
Vested and expected to vest — December 31, 2025
8.7
61.24
6
—
F- 28
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
The following table summarizes the Company's options exercised and vested for each of the periods indicated (in millions except for weighted-average estimated fair value of options granted):
Year Ended December 31,
2025
2024
2023
Intrinsic value of options exercised
$
—
$
1
$
5
Cash received from the exercise of options
$
—
$
6
$
3
Weighted-average estimated fair value of options granted
$
22.92
$
29.88
$
25.53
Estimated fair value of options vested
$
46
$
54
$
60
The aggregate intrinsic values of options outstanding, vested and exercisable, and vested and expected to vest were calculated as the difference between the exercise price of the options and the fair value of the Company’s common stock.
Performance Stock Options
The following table summarizes activity under the Company’s Performance Stock Option, or PSO, plans and related information:
PSOs Outstanding
Number of Options
(millions)
Weighted-Average Exercise Price
Weighted-Average Remaining Contractual Term (Years)
Aggregate Intrinsic Value
Outstanding — December 31, 2024
1.2
$
67.37
2
$
—
PSOs cancelled
( 0.4
)
67.37
Outstanding — December 31, 2025
0.8
67.37
1
—
Vested and exercisable — December 31, 2025
0.8
67.37
1
—
Vested and expected to vest — December 31, 2025
0.8
67.37
1
—
During the year ended December 31, 2022, PSOs were granted to certain nonexecutive employees. PSOs are subject to vest only if specified operational milestones are achieved and the employees’ continued service with the Company. The Company uses the Black-Scholes method to calculate the fair value at the grant date and is recognizing stock-based compensation expense for the PSOs that are expected to vest. Stock-based compensation for PSOs is recognized over the service period, beginning in the period the Company determines it is probable that a milestone will be achieved. Forfeitures of PSOs are recognized as they occur. The Company reassesses the probability of the performance condition at each reporting period and adjusts the compensation cost based on the probability assessment. As of December 31, 2025, certain operational milestones had been achieved. The aggregate intrinsic values of PSOs outstanding, vested and exercisable, and vested and expected to vest were calculated as the difference between the exercise price of the PSOs and the fair value of the Company’s common stock. The total estimated grant date fair value of PSOs vested during the years ended December 31, 2025, 2024, and 2023 was $ 14 million, $ 10 million, and $ 3 million, respectively.
Restricted Stock Units
The following table summarizes activity under the Company’s Restricted Stock Units, or RSU, plans and related information:
RSUs Outstanding
Number of Shares
(millions)
Weighted-Average Grant Date Fair Value
Unvested — December 31, 2024
5.2
$
53.22
RSUs granted
2.9
41.04
RSUs vested
( 1.5
)
56.39
RSUs cancelled
( 0.7
)
49.78
Unvested — December 31, 2025
5.9
46.23
F- 29
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
The fair value of the RSUs is determined on the grant date based on the fair value of the Company’s common stock. The fair value of the RSUs is recognized as expense ratably over the vesting period of one to four years . The total grant date fair value of the RSUs vested during the years ended December 31, 2025, 2024, and 2023 was $ 85 million, $ 63 million, and $ 55 million, respectively. The aggregate intrinsic value of the shares of the RSUs vested during the years ended December 31, 2025, 2024, and 2023 was $ 62 million, $ 54 million, and $ 33 million, respectively.
Performance Stock Units
The following table summarizes activity under the Company’s Performance Stock Units, or PSUs and related information:
PSUs Outstanding
Number of Shares
(millions)
Weighted-Average Grant Date Fair Value
Unvested — December 31, 2024
0.6
$
59.39
PSUs granted
0.4
50.77
PSUs vested
( 0.2
)
53.44
PSUs cancelled
( 0.1
)
49.95
Unvested — December 31, 2025
0.7
57.48
The fair value of the PSUs is determined on the grant date based on the fair value of the Company’s common stock, except for certain PSUs with a market vesting condition, for which fair value is estimated using a Monte Carlo simulation model. PSUs are subject to vest only if certain specified criteria are achieved and the employees’ continued service with the Company. For certain PSUs, the number of PSUs that may vest are also subject to the achievement of certain specified criteria, including both performance conditions and market conditions. As of December 31, 2025, certain specified criteria were deemed probable of achievement or already achieved. Stock-based compensation for PSUs is recognized over the service period beginning in the period the Company determines it is probable that the performance criteria will be achieved. The total grant date fair value of the PSUs vested during the years ended December 31, 2025, 2024, and 2023 was $ 12 million, $ 3 million, and $ 4 million, respectively, with an aggregate intrinsic value of the shares of $ 9 million, $ 2 million and $ 1 million, respectively.
Stock-Based Compensation Expense
Total stock-based compensation expense recognized was as follows (in millions):
Year Ended December 31,
2025
2024
2023
Cost of sales
$
2
$
1
$
1
Research and development
84
87
75
Selling, general and administrative
67
70
59
Total stock-based compensation expense
$
153
$
158
$
135
Stock-based compensation of $ 2 million, $ 3 million, and $ 2 million was capitalized into inventory for the years ended December 31, 2025, 2024, and 2023, respectively. Capitalized stock-based compensation is recognized as cost of sales when the related product is sold.
As of December 31, 2025 , the total unrecognized compensation expense related to unvested equity awards, net of estimated forfeitures, was $ 232 million, which the Company expects to recognize over an estimated weighted-average period of 2 years. In determining the estimated fair value of the stock options, PSOs and ESPP, the Company uses the Black-Scholes option-pricing model and assumptions discussed below. Each of these inputs is subjective and generally requires significant judgment to determine.
Expected Term —The Company’s expected term represents the period that the Company’s stock-based awards are expected to be outstanding and is determined using the simplified method (based on the mid-point between the vesting date and the end of the contractual term).
Expected Volatility —The Company’s expected volatility is based on historical volatility over the look-back period corresponding to the expected term.
Risk-Free Interest Rate —The risk-free interest rate is based on the U.S. Treasury zero coupon issues in effect at the time of grant for periods corresponding with the expected term of option.
Expected Dividend —The Company has never paid dividends on its common stock and has no plans to pay dividends on its common stock. Therefore, the Company used an expected dividend yield of zero.
F- 30
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
Strike price for options awards and PSOs is equal to the closing market value of our common stock on the date of grant.
The fair value of stock option awards granted was estimated at the date of grant using a Black-Scholes option-pricing model with the following weighted-average assumptions:
Year Ended December 31,
2025
2024
2023
Expected term (years)
6
6
6
Expected volatility
53 %
55 %
55 %
Risk-free interest rate
4.1 %
4.2 %
4.2 %
Expected dividend rate
0.0 %
0.0 %
0.0 %
13. Defined Contribution Plan
The Company sponsors a retirement plan in which substantially all of its full-time employees in the U.S. and certain other foreign countries are eligible to participate. Eligible participants may contribute a percentage of their annual compensation to this plan, subject to statutory limitations. The Company recorded $ 10 million, $ 10 million, and $ 10 million as expense related to the plan for the years ended December 31, 2025, 2024, and 2023 , respectively.
14. Income Taxes
The components of the Company’s loss before income taxes were as follows (in millions):
Year Ended December 31,
2025
2024
2023
Domestic
$
565
$
563
$
609
Foreign
6
4
—
Total loss before income taxes
$
571
$
567
$
609
The components of the Company’s income tax provision (benefit) were as follows (in millions):
Year Ended December 31,
2025
2024
2023
Current provision for income taxes:
Federal
$
—
$
—
$
—
State
—
—
( 3
)
International
4
3
3
Total current tax provision
4
3
—
Deferred tax provision:
Federal
—
—
—
State
—
—
( 2
)
International
—
( 1
)
—
Total deferred tax benefit
—
( 1
)
( 2
)
Total provision for (benefit from) income taxes
$
4
$
2
$
( 2
)
The Company has incurred net operating losses since inception. The Company has not reflected any benefit of such net operating loss carryforwards in the accompanying financial statements. The Company has established a full valuation allowance against its deferred tax assets due to the uncertainty surrounding the realization of such assets.
For the year ended December 31, 2023, the Company recognized an income tax benefit of $ 5 million attributable to modifications in its state apportionment methodology, offset by an income tax expense of $ 3 million from foreign jurisdictions. For the year ended December 31, 2024, the Company recognized income tax expense of $ 1 million from foreign jurisdictions alongside a nominal amount of state tax. For the year ended December 31, 2025, the Company recognized income tax expense of $ 4 million from foreign jurisdictions and a nominal amount of state tax. The Company realized no benefit for current year losses due to a full valuation allowance against the U.S. net deferred tax assets.
F- 31
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
The table below provides the updated requirements of ASU 2023-09 for the year ended December 31, 2025.
The effective tax rate of our provision for income taxes differs from the federal statutory rate as follows (in millions except percentage amounts):
Year Ended December 31,
2025
Federal statutory income tax rate
$
( 120
)
21.0
%
State income taxes, net of federal benefit (1)
—
—
Foreign tax effects
5
( 0.9
)
Effect of changes in tax laws or rates enacted in the current period
—
—
Effect of cross-border tax laws
5
( 0.9
)
Tax credits
—
—
Research tax credit
( 12
)
2.1
Orphan drug credit
( 32
)
5.6
Change in valuation allowance
145
( 25.4
)
Nontaxable or nondeductible items
Share-based payment awards
14
( 2.5
)
Other
( 1
)
0.3
Uncertain tax positions
—
—
Other Adjustments
—
—
Provision for income taxes
$
4
( 0.7
)
%
(1) Includes a nominal amount of state tax. Illinois State tax made up the majority (greater than 50 percent) of the tax effect in this category.
As previously disclosed for the years ended December 31, 2024 and 2023, prior to the adoption of ASU 2023-09, the effective income tax rate differs from the statutory federal income tax rate as follows:
Year Ended December 31,
2024
2023
Federal statutory income tax
21.0
%
21.0
%
State income taxes, net of federal benefit
—
0.8
Federal tax credits
10.8
7.3
Other
0.1
( 0.7
)
Nondeductible permanent items
( 1.1
)
( 0.3
)
Stock-based compensation
( 1.6
)
( 1.8
)
Uncertain tax positions
( 2.0
)
( 1.4
)
Change in valuation allowance
( 27.1
)
( 24.1
)
Foreign rate differential
( 0.4
)
( 0.5
)
Provision for income taxes
( 0.3
)
%
0.3
%
F- 32
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
Income taxes paid ( net of refunds) for the year ended December 31, 2025 is disaggregated as follows (in millions):
Year Ended December 31,
2025
Federal income taxes paid (net of refunds)
$
—
State income taxes paid (net of refunds)
—
Foreign income taxes paid (net of refunds)
Brazil
1
Mexico
1
Other (1)
2
Total income taxes paid (net of refunds)
$
4
(1) There was a nominal amount of foreign taxes paid (net of refunds) to Canada, Switzerland, France, Japan, and the Netherlands.
The tax effect of temporary differences that give rise to significant portions of the deferred tax assets is presented below (in millions):
Year Ended December 31,
2025
2024
Deferred tax assets:
Loss carryforwards
$
511
$
309
Tax credits
426
370
Stock options
46
52
Accruals and reserves
34
32
Fixed assets and intangibles
26
32
Liabilities for sales of future royalties
276
197
Basis difference in equity investments
8
9
Capitalized research and development costs
34
212
Other
—
1
Gross deferred tax assets
1,361
1,214
Valuation allowance
( 1,355
)
( 1,207
)
Total deferred tax assets
6
7
Deferred tax liabilities:
In-process research and development
( 30
)
( 30
)
Right-of-use lease assets
( 5
)
( 6
)
Gross deferred tax liabilities
( 35
)
( 36
)
Net deferred tax liabilities
$
( 29
)
$
( 29
)
As of December 31, 2025 and 2024 , the Company had approximately $ 2,104 million and $ 1,191 million, respectively, of federal net operating loss carryforwards available to reduce future taxable income that will begin to expire in 2031 . As of December 31, 2025 and 2024 , the Company had approximately $ 936 million and $ 744 million, respectively, of state net operating loss carryforwards available to reduce future taxable income that will begin to expire in 2031 .
As of December 31, 2025 and 2024 , the Company had federal research tax credit carryforwards of approximately $ 60 million and $ 45 million, respectively, available to reduce future tax liabilities that will begin to expire in 2031 . As of December 31, 2025 and 2024 , the Company had state research credit carryforwards of $ 110 million and $ 92 million, respectively, available to reduce future tax liabilities. The majority of these credits will be carried forward indefinitely, and others will expire at various dates in the future.
As of December 31, 2025 and 2024 , the Company had federal Orphan Drug Credits of approximately $ 379 million and $ 339 million, respectively, available to reduce future tax liabilities that will begin to expire in 2031 .
F- 33
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
The Company’s ability to use net operating loss and tax credit carryforwards to reduce future taxable income and liabilities may be subject to annual limitations pursuant to Internal Revenue Code Sections 382 and 383 as a result of ownership changes in the past and future. As a result of ownership changes in 2012 and 2011, $ 4 million of federal net operating loss carryforwards, $ 4 million of state net operating loss carryforwards, and a nominal amount of federal tax credits are permanently limited. Deferred tax assets for net operating losses and tax credits have been reduced and a corresponding adjustment to the valuation allowance has been recorded.
The valuation allowance increased by $ 148 million and $ 171 million during the years ended December 31, 2025 and 2024, respectively.
The Company recorded unrecognized tax benefits for uncertainties in income taxes. A reconciliation of the Company’s unrecognized tax benefits follows (in millions):
December 31,
2025
2024
2023
Balance at beginning of year
$
97
$
80
$
67
Additions based on tax positions related to current
year
15
15
12
Additions for tax positions of prior years
—
2
1
Reductions for tax positions of prior years
( 1
)
—
—
Balance at end of year
$
111
$
97
$
80
Approximately $ 1 million in unrecognized tax benefits would impact the Company’s effective tax rate if recognized. The Company has elected to include interest and penalties as a component of tax expense. For the years ended December 31, 2025, 2024 and 2023, the Company recognized nominal amounts for accrued interest and penalties as a component of income tax expense. The Company does not anticipate that the amount of existing unrecognized tax benefits will significantly increase or decrease during the next year.
It is the Company’s intention to reinvest the earnings of its non-U.S. subsidiaries in their operations. As of December 31, 2025 , the Company had not made a provision for any incremental foreign withholding taxes on approximately $ 17 million of the excess of the amount of net income for financial reporting over the tax basis of investments in foreign subsidiaries that are essentially permanent in duration. If these earnings were repatriated to the U.S., the deferred tax liability associated with these temporary differences would result in a nominal amount of withholding taxes.
The Company files income tax returns in the U.S. federal, 40 state tax jurisdictions, and ten foreign countries. The federal and state income tax returns from inception to December 31, 2025 remain subject to examination.
15. Commitments and Contingencies
The Company has various manufacturing, clinical, research, and other contracts with vendors in the conduct of the normal course of its business. Other than as noted below, contracts are terminable, with varying provisions regarding termination. If a contract with a specific vendor were to be terminated, the Company would only be obligated for the products or services that the Company had received at the time the termination became effective.
Manufacturing and service contract obligations primarily relate to manufacturing of product for our clinical stage pipeline, the majority of which are due in the next 12 month s .
The terms of certain of the Company’s licenses, royalties, development and collaboration agreements, as well as other research and development activities, require the Company to pay potential future milestone payments based on product development success. The amount and timing of such obligations are unknown or uncertain. These potential obligations are further described in “Note 8. License and Research Agreements.”
See “Note 9. Leases” for lease commitments.
Contingencies
In the ordinary course of business, the Company may become party to various claims and complaints. See “Item 3. Legal Proceedings” for material legal proceedings the Company is aware of. The process of resolving matters through litigation or other means is inherently uncertain, however management does not believe that any ultimate liability resulting from any of these potential claims will have a material adverse effect on its results of operations, financial position, or liquidity.
F- 34
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
Guarantees and Indemnifications
The Company indemnifies each of its directors and officers for certain events or occurrences, subject to certain limits, while the director or officer is or was serving at the Company’s request in such capacity, as permitted under Delaware law and in accordance with its certificate of incorporation and bylaws. The term of the indemnification period lasts as long as a director or officer may be subject to any proceeding arising out of acts or omissions of such director and officer in such capacity. The maximum amount of potential future indemnification is unlimited; however, the Company currently holds director liability insurance. This insurance allows the transfer of risk associated with the Company’s exposure and may enable it to recover a portion of any future amounts paid. The Company believes that the fair value of these indemnification obligations is minimal. Accordingly, it has not recognized any liabilities relating to these obligations for any period presented.
16. Related Party Transaction
In July 2022, the Company entered into an agreement with a non-profit foundation in which two members of the Company’s board of directors, including the Company’s Chief Executive Officer, at the time also served as board members of the foundation, whereby an aggregate $ 1 million contribution is being paid to the foundation over a four-year period, beginning in the third quarter of 2022, to support rare disease education and awareness. A total of $ 1 million has been recorded as research and development expense for this agreement to date. There are no further obligations under this agreement.
17. Net Loss per Share
The following table sets forth the computation of the basic and diluted net loss per share during the years ended December 31, 2025, 2024, and 2023 (in millions, except per share data):
Year Ended December 31,
2025
2024
2023
Numerator:
Net loss
$
( 575
)
$
( 569
)
$
( 607
)
Denominator:
Weighted-average shares used to compute net loss per
share, basic and diluted
98.6
90.5
73.5
Net loss per share, basic and diluted
$
( 5.83
)
$
( 6.29
)
$
( 8.25
)
The following weighted-average outstanding common stock equivalents were excluded from the computation of diluted net loss per share for the periods presented because including them would have been antidilutive (in millions):
Year Ended December 31,
2025
2024
2023
Options to purchase common stock,
restricted stock units, and performance stock units
16.8
16.3
14.2
18. Accumulated Other Comprehensive Income (Loss)
Total accumulated other comprehensive income (loss) consisted of the following (in millions):
Year Ended December 31,
2025
2024
Cumulative foreign currency translation adjustment
$
1
$
( 2
)
Unrealized gain on securities available-for-sale
—
1
Total accumulated other comprehensive income (loss)
$
1
$
( 1
)
19. Subsequent Events
In February 2026, the Company’s Board of Directors approved a strategic restructuring plan that includes a reduction of approximately 10 % of the Company’s workforce, or the Restructuring Plan. As a result of this reduction in force, the Company estimates that it will incur restructuring charges of approximately $ 50 million in total in connection with employee related termination and severance costs, charges for the termination of UX143 manufacturing agreements, and other related activities,
F- 35
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Consolidated Financial Statements (continued)
substantially all of which are expected to be recognized during the first half of 2026. Cash payments related to these costs are expected to be made over the same period.
The Restructuring Plan is intended to reduce the Company's headcount and expenses by focusing resources on the Company’s largest value drivers. Management’s estimates are based on information available as of the approval date of the Restructuring Plan and are subject to change as the plan is implemented.
F- 36