DEF 14A of Ultragenyx Pharmaceutical Inc., Mar 28, 2025

DEF 14A of Ultragenyx Pharmaceutical Inc.

Filed Mar 28, 2025 · FY20240001308179-25-000296

Open on EDGAR

  1. Board of Directors

    Board of Directors The following table provides summary information about each nominee for director at the Annual Meeting and our continuing directors, as of the date of this Proxy Statement.

  2. Board of Directors

    | Nominee/Director Name | Age | Principal Occupation | Director Since | Year Current Term Expires | Current Director Class | Independent | | Director Nominees | | | | | | | | Matthew K. Fust | 60 | Board member and advisor to various life science companies | 2014 | 2025 | III | | | Amrit Ray, M.D. | 52 | Board member and advisor to various life science companies | 2022 | 2025 | III | | | Continuing Directors | | | | | | | | Emil D. Kakkis, M.D., Ph.D. | 64 | Founder, President and CEO, Ultragenyx | 2010 | 2026 | I | | | Shehnaaz Suliman, M.D. | 53 | Chief Executive Officer of ReCode Therapeutics | 2019 | 2026 | I | | | Daniel G. Welch | 67 | Board member and advisor to various life science companies | 2015 | 2026 | I | | | Deborah Dunsire, M.D. | 62 | Board member and advisor to various life science companies | 2017 | 2027 | II | | | Michael Narachi | 65 | Board member and advisor to various life science companies | 2015 | 2027 | II | | | Corsee D. Sanders, Ph.D. | 68 | Board member and advisor to various life science companies | 2021 | 2027 | II | |

  3. Board of Directors

    | Board Composition Highlights | | | | |

  4. Building the Right Board

    Building the Right Board Our Nominating and Corporate Governance Committee is responsible for reviewing with the entire Board from time to time the appropriate skills and characteristics required of directors in the context of the current Board composition and the anticipated needs of the Board and the Company. Our Nominating and Corporate Governance Committee actively considers whether potential candidates would assist in achieving a mix of Board members that represents a broad range of knowledge, skills, and experience, including whether the nominee has specific strengths that would augment existing skills and experiences of the Board and in a manner that is aligned with the Company’s strategic direction. The following matrix highlights each director’s primary skills or knowledge in these areas as identified by the Nominating and Corporate Governance Committee. The matrix does not encompass all of the knowledge, skills, experiences or attributes of our directors, and the fact that a particular knowledge, skill, experience or attribute is not listed does not mean that a director does not possess it. In addition, the absence of a particular knowledge, skill, experience or attribute with respect to any of our directors does not mean the director in question is unable to contribute to the decision-making process in that area. The type and degree of knowledge, skill and experience listed below may vary among the members of the Board.

  5. Building the Right Board

    | | Welch | Kakkis | Dunsire | Fust | Narachi | Ray | Sanders | Suliman | | Skills and Experience | | | | | | | | | | Core Board Capabilities | | | | | | | | | | Biopharma C-level leadership | | | | | | | | | | Scientific and research leadership | | | | | | | | | | Clinical development leadership | | | | | | | | | | Regulatory leadership | | | | | | | | | | Rare disease commercial experience | | | | | | | | | | Biopharma commercial leadership | | | | | | | | | | Global access, pricing and reimbursement | | | | | | | | | | Global business operations | | | | | | | | | | Finance and capital markets | | | | | | | | | | Corporate strategy and/or business development leadership | | | | | | | | | | Corporate governance and board experience | | | | | | | | | | Relevant / Advisory Capabilities | | | | | | | | | | Compliance (GXP, commercial) | | | | | | | | | | Corporate legal and IP | | | | | | | | | | Government affairs and policy | | | | | | | | | | Human resources and organizational development | | | | | | | | | | Manufacturing/supply chain | | | | | | | | | | Background | | | | | | | | | | Gender | | | | | | | | | | Male | | | | | | | | | | Female | | | | | | | | | | Race/Ethnicity | | | | | | | | | | Asian | | | | | | | | | | White | | | | | | | | | | LGBTQ+ | | | | | | | | |

  6. Corporate Governance Overview

    Corporate Governance Overview We are committed to maintaining good corporate governance practices and we periodically review our practices. We believe that good corporate governance, including the practices listed below, promotes the long term interests of our stockholders.

  7. Corporate Governance Overview

    | • | All of our directors are independent, other than our President and Chief Executive Officer | | • | Director Resignation Policy that applies when a director fails to receive majority support in an uncontested election | | • | Completely independent Audit Committee, Nominating and Corporate Governance Committee and Compensation Committee | | • | Effective and active independent Chairman | | • | Annual Board and committee self-evaluations | | • | Active stockholder engagement program | | • | All our current directors attended 100% of the Board and committee meetings of which the director was a member in 2024 | | • | Minimum stock ownership requirements for named executive officers and directors | | • | Director Overboarding Policy limiting the total number of public company boards that a director may serve to five total public company boards and public company CEO directors to three total public company boards | | • | Board and committees are authorized to engage outside advisors independently of management | | • | Clawback Policy that complies with Rule 10D-1 under the Securities Exchange Act of 1934, as amended (Exchange Act) and also includes a discretionary recoupment provision that permits recovery of all incentive compensation (including time-based and performance-based equity awards) in the event of fraud or intentional misconduct (see section entitled “Clawback Policy” of this Proxy Statement for additional details) | | • | Prohibition against hedging and pledging transactions by our directors and employees, including our executive officers | | • | Mixture of short, medium and long-tenured directors | | • | Corporate Governance Guidelines and robust Global Code of Conduct |

  8. PROPOSAL 1 Election of Class III Directors

    PROPOSAL 1 Election of Class III Directors Our Amended and Restated Certificate of Incorporation provides that the Board is to be divided into three classes as nearly equal in number as reasonably possible, with directors in each class generally serving three-year terms. The total Board size is currently fixed at eight directors. The Class III directors (whose terms expire at the Annual Meeting) are Matthew K. Fust and Amrit Ray, M.D. Mr. Fust and Dr. Ray were most recently elected by stockholders at the 2022 Annual Meeting. The Class I directors (whose terms expire at the 2026 Annual Meeting) are Emil D. Kakkis, M.D., Ph.D., Shehnaaz Suliman, M.D., and Daniel G. Welch. The Class II directors (whose terms expire at the 2027 Annual Meeting) are Deborah Dunsire, M.D., Michael Narachi and Corsee D. Sanders, Ph.D. The Class III directors elected at the Annual Meeting will hold office until the 2028 Annual Meeting or until their successors are elected and qualified, unless they resign or their seats become vacant due to death, removal, or other cause in accordance with our Amended and Restated Bylaws (bylaws). As described below, the Board, upon the recommendation of the Nominating and Corporate Governance Committee, has nominated the director nominees listed above for election as directors at the Annual Meeting. They have indicated their willingness and ability to serve if elected. Should any of them become unable or, for good cause, unwilling to serve, the persons named on the enclosed proxy card as proxy holders may vote all proxies given in response to this solicitation for the election of a substitute nominee(s) chosen by the Board or the Board may reduce the size of the Board.

  9. Nomination of Directors

    Nomination of Directors The Nominating and Corporate Governance Committee reviews and recommends to the Board potential nominees for election to the Board. In reviewing potential nominees, the Nominating and Corporate Governance Committee considers the qualifications of each potential nominee in light of the Board’s existing and desired mix of experience and expertise. Specifically, the Nominating and Corporate Governance Committee considers each potential nominee’s personal and professional ethics, integrity, values, experience, interest in the Company, and commitment to the representation of the long-term interests of the stockholders. As described more fully above under “Proxy Statement Overview – Building the Right Board”, the Nominating and Corporate Governance Committee also considers each potential nominee’s contribution to the Board’s composition as whole with in achieving a mix of knowledge, skills, experience, and background. The Board has adopted a policy in the Company’s Corporate Governance Guidelines, which provides that as part of the search process for each new director, the Nominating and Corporate Governance Committee will add diverse candidates into the pool of candidates (and instructs any search firm the Nominating and Corporate Governance Committee engages to do so). Directors ultimately are selected based on the skills, experiences and qualifications that best support the Company in the context of the Board as a whole. Additionally, the Nominating and Corporate Governance Committee considers whether a nominee will be able to dedicate sufficient time to, and focus on, his or her duties as a member of the Board. The Board membership criteria are set forth in our Corporate Governance Guidelines, a copy of which is available on our website at www.ultragenyx.com in the “Corporate Governance” subsection of the “Investors” tab. The Nominating and Corporate Governance Committee assesses its effectiveness in balancing these considerations in connection with its annual evaluation of the composition of the Board. After reviewing the qualifications of potential Board candidates, the Nominating and Corporate Governance Committee presents its recommendations to the Board, which selects the final director nominees. We did not pay any fees to any third party to identify or assist in identifying or evaluating nominees for the Annual Meeting. The Nominating and Corporate Governance Committee considers stockholder-recommended director nominees using the same criteria set forth above and in the same manner as director candidates recommended by other sources. Stockholders who wish to recommend a potential nominee to the Nominating and Corporate Governance Committee for consideration for election at a future annual meeting of stockholders must submit such recommendation to the Nominating and Corporate Governance Committee as described in the section entitled “Stockholder Communications” and provide the Nominating and Corporate Governance Committee with the same information that would be required to nominate a director candidate in accordance with the process and within the deadline for nominating director candidates set forth below under the question “When are other proposals and director nominations for next year’s annual meeting due?” in the section entitled “Additional Information”.

  10. Nominees and Incumbent Directors

    Nominees and Incumbent Directors Information regarding our director nominees and our current directors, including their respective age as of the date of this Proxy Statement and their principal occupation, is set forth below.

  11. Class III Directors Nominated for Election

    Class III Directors Nominated for Election

  12. Class III Directors Nominated for Election

    | Matthew K. Fust | | | Board member and advisor to various life science companies | | | Age: 60 Director since: 2014 INDEPENDENT Committees: 2 Other Public Directorships: 3 | Mr. Fust is a board member and advisor to life science companies. Mr. Fust currently serves on the board of directors of Atara Biotherapeutics, Inc., Crinetics Pharmaceuticals, Inc. and Neumora Therapeutics Inc., all of which are publicly traded biopharmaceutical companies. Mr. Fust also previously served on the board of directors of Dermira, Inc., a biopharmaceutical company, from August 2014 to February 2020 and the board of MacroGenics, Inc., a biotechnology company, from March 2014 to May 2020. He retired as Executive Vice President of Onyx Pharmaceuticals, Inc., a biopharmaceutical company, where he served from January 2009 to January 2014. From May 2003 to December 2008, Mr. Fust served as Chief Financial Officer at Jazz Pharmaceuticals, Inc., a specialty pharmaceutical company. From 2002 to 2003, Mr. Fust served as Chief Financial Officer at Perlegen Sciences, Inc., a biopharmaceutical company. Previously, he was Senior Vice President and Chief Financial Officer at ALZA Corporation, a pharmaceutical company, where he was an executive from 1996 to 2002. From 1991 to 1996, Mr. Fust was a manager in the healthcare strategy practice at Andersen Consulting, a consulting company. Mr. Fust holds a B.A. in Accounting from the University of Minnesota and an M.B.A. from the Stanford Graduate School of Business. Skills and Qualifications specific to Ultragenyx: We believe that Mr. Fust is qualified to serve on our Board due to his extensive experience in the life sciences industry, his financial experience and ability to be our “audit committee financial expert,” and his service as a director of other public biopharmaceutical companies. |

  13. Class III Directors Nominated for Election

    | Amrit Ray, M.D., M.B.A. | | | Board member and advisor to various life science companies | | | Age: 52 Director since: 2022 INDEPENDENT Committees: 1 Other Public Directorships: 1 | Dr. Ray is a board member and advisor to life science companies. Dr. Ray currently serves on the board of directors of Fortrea Holdings Inc., a publicly traded, global clinical research organization, and on the board of directors of several privately owned life science companies. Previously, Dr. Ray served as Chief Patient Officer at Biohaven Ltd. a public biopharmaceutical company, from March 2022 to December 2022 when acquired by Pfizer. Prior to his role at Biohaven, he served as Senior Adviser to Bain Capital Life Sciences, an investment company, from February 2021 to March 2022. Prior to Bain Capital, Dr. Ray served as Global President, Head of R&D and Medical, for Pfizer Essential Health and subsequently the Pfizer Upjohn division at Pfizer, Inc., a public pharmaceutical company, from 2017 to January 2021. Prior to Pfizer, he held positions of increasing responsibility at Johnson & Johnson, a public pharmaceutical company, including serving as Senior Vice President, External Affairs (Science and Medicine) in 2017, Senior Vice President, Chief Medical Officer of Janssen from 2012 to 2017 and Senior Vice President, Chief Safety Officer from 2009 to 2012. Dr. Ray currently serves as a Trustee at the Board of the Hastings Center for Bioethics, and as a Visiting Professor of Practice, Faculty of Medical Sciences at Newcastle University in the United Kingdom. Dr. Ray holds a B.S., with Honours, in Immunology and a M.D. (M.B., Ch.B.) from the University of Edinburgh. He also holds an M.B.A. from the Tuck School of Business at Dartmouth College. Skills and Qualifications specific to Ultragenyx: We believe that Dr. Ray is qualified to serve on our Board due to his extensive experience in the life sciences industry, and particularly his research and development expertise. |

  14. Class I Directors Continuing in Office until 2026

    Class I Directors Continuing in Office until 2026

  15. Class I Directors Continuing in Office until 2026

    | Emil D. Kakkis, M.D., Ph.D. | | | Founder, President and CEO | | | Age: 64 Director since: 2010 NOT INDEPENDENT Committees: 1 Other Public Directorships: 0 | Dr. Kakkis is our founder and has served as our President and Chief Executive Officer and as a member of our Board since our inception in April 2010. Dr. Kakkis served as our interim principal financial officer following the departure of the Company’s Chief Financial Officer in November 2022 until the appointment of Mr. Horn as the Company’s Chief Financial Officer in October 2023. Prior to Ultragenyx, from September 1998 to February 2009, Dr. Kakkis served in various executive capacities, and ultimately as Chief Medical Officer, at BioMarin Pharmaceutical Inc., a biopharmaceutical company. Dr. Kakkis then served as a development consultant to BioMarin from 2009 to 2010. Dr. Kakkis is also Founder of EveryLife Foundation for Rare Diseases, a non-profit organization he started in 2009 to accelerate biotechnology innovation for rare diseases. Dr. Kakkis received the Termeer Visionary Leadership Award in 2019 and the Leadership Award from the California Life Sciences Association in 2021. Dr. Kakkis is board certified in Medical Genetics and was board certified in Pediatrics. He holds a B.A. in Biology from Pomona College and combined M.D. and Ph.D. degrees from the UCLA School of Medicine’s Medical Scientist Training Program where he received the Bogen prize for his research. Skills and Qualifications specific to Ultragenyx: We believe that Dr. Kakkis possesses specific expert knowledge of genetics and rare diseases and operational experience in the life sciences sector that qualify him to serve on our Board. |

  16. Class I Directors Continuing in Office until 2026

    | Shehnaaz Suliman, M.D., M.Phil., M.B.A | | | Chief Executive Officer of ReCode Therapeutics | | | Age: 53 Director since: 2019 INDEPENDENT Committees: 2 Other Public Directorships: 1 | Dr. Suliman has served as Chief Executive Officer of ReCode Therapeutics, a privately-held, integrated genetic medicines company, since January 2022. Prior to joining ReCode Therapeutics, Dr. Suliman served as President and Chief Operating Officer of Alector, Inc., a clinical stage biotechnology company, from December 2019 to December 2021 and previously served as Senior Vice President, Corporate Development and Strategy of Theravance Biopharma, Inc., a biopharmaceutical company, from July 2017 to March 2019. Prior to her position at Theravance, Dr. Suliman worked for Genentech, Inc., a biopharmaceutical company, as Group Leader and Project Team Leader in the R&D Portfolio Management and Operations Group from September 2010 to May 2015 and then as Vice President and Global Therapeutic Head, Roche Partnering from June 2015 to July 2017. Prior to Genentech, Dr. Suliman held various management roles of increasing responsibility at Gilead Sciences, Inc., a biopharmaceutical company, from January 2005 and September 2010. Prior to Gilead, Dr. Suliman was an investment banker with Lehman Brothers and Petkevich & Partners, advising public and private companies on buy- and sell-side transactions. She is a member of the board of directors of 10X Genomics, Inc., a publicly traded life science technology company. Dr. Suliman received her M.D. at the University of Cape Town Medical School, South Africa, and holds an M.B.A, with distinction, and M.Phil. in Development Studies from Oxford University, where she was a Rhodes Scholar. Skills and Qualifications specific to Ultragenyx: We believe that Dr. Suliman is qualified to serve on our Board due to her extensive operational experience with global biopharmaceutical companies, and particularly her expertise in business development, corporate strategy and clinical drug development. |

  17. Class I Directors Continuing in Office until 2026

    | Daniel G. Welch | | | Board member and advisor to various life science companies | | | Age: 67 Director since: 2015 INDEPENDENT CHAIRMAN Committees: 2 Other Public Directorships: 2 | Mr. Welch is a board member and advisor to life science companies. Mr. Welch currently serves as chairman on the boards of Structure Therapeutics, Inc. and Prothena Corporation plc, each a publicly traded biotechnology company. Between January 2015 and January 2018, he was an Executive Partner at Sofinnova Ventures, a venture capital firm. Prior to Sofinnova, Mr. Welch served as Chairman, Chief Executive Officer, and President of InterMune, Inc., a biotechnology company, from May 2008 to October 2014 and served as President and Chief Executive Officer of InterMune and a member of its board of directors from September 2003 to May 2008. From August 2002 to January 2003, Mr. Welch served as Chairman and Chief Executive Officer of Triangle Pharmaceuticals, Inc., a pharmaceutical company. From October 2000 to June 2002, Mr. Welch served as President of the pharmaceutical division of Elan Corporation, plc. Mr. Welch previously served as chairman of the board of directors of Nuvation Bio from July 2020 to September 2024 and as a director on the boards of directors of Intercept Pharmaceuticals, Inc from November 2015 to June 2021 and Seagan Inc. from July 2007 to December 2023. Mr. Welch holds a B.S. from the University of Miami and an M.B.A. from the University of North Carolina. Skills and Qualifications specific to Ultragenyx: We believe that Mr. Welch is a strong operating executive with operational and strategic expertise in the global pharmaceutical market, whose experience contributes valuable insight to the Board. |

  18. Class II Directors Continuing in Office until 2027

    Class II Directors Continuing in Office until 2027

  19. Class II Directors Continuing in Office until 2027

    | Deborah Dunsire, M.D. | | | Board member and advisor to various life science companies | | | Age: 62 Director since: 2017 INDEPENDENT Committees: 2 Other Public Directorships: 1 | Dr. Dunsire is a board member and advisor to life science companies. Dr. Dunsire served as President and Chief Executive Officer of H. Lundbeck A/S, a pharmaceutical company, from September 2018 until August 2023. She previously served as President and Chief Executive Officer and as a director of Xtuit Pharmaceuticals, Inc., a private biopharmaceutical company, from January 2017 to March 2018. Prior to her position at Xtuit, she served as President and Chief Executive Officer and a director of FORUM Pharmaceuticals Inc., a private pharmaceutical company, from July 2013 to May 2016. Prior to FORUM, Dr. Dunsire worked for Takeda Pharmaceutical Company Limited, a publicly traded pharmaceutical company, as a corporate officer from June 2010 to June 2011 and a director from June 2011 to June 2013. She served as President and Chief Executive Officer and as a director of Millennium Pharmaceuticals, Inc., a publicly traded biopharmaceutical company, between 2005 and 2008, when it was acquired by Takeda, and then as President and Chief Executive Officer of Millennium: The Takeda Oncology Company after the acquisition between 2008 and 2013. Prior to Millennium, Dr. Dunsire held various roles of increasing responsibility at Novartis Pharma AG between 1988 and 2005. Dr. Dunsire currently serves on the board of McKesson Corporation, a publicly traded healthcare services company. She previously served on the board of directors of Syros Pharmaceuticals, a publicly traded biotech company, from September 2021 to November 2024 and of Alexion Pharmaceuticals Inc., a publicly traded biotech company, from January 2018 until July 2021 when it was acquired by AstraZeneca. She obtained an MBBCh from the University of the Witwatersrand. Skills and Qualifications specific to Ultragenyx: We believe that Dr. Dunsire is qualified to serve on our Board due to her extensive experience in the biotechnology and pharmaceutical sectors, including service as the chief executive officer of various pharmaceutical companies, which gives her the skills to provide us with operational and strategic insights. |

  20. Class II Directors Continuing in Office until 2027

    | Michael Narachi | | | Board member and advisor to various life science companies | | | Age: 65 Director since: 2015 INDEPENDENT Committees: 2 Other Public Directorships: 0 | Mr. Narachi previously served as President and Chief Executive Officer of CODA Biotherapeutics, Inc., a private biotherapeutics company, from August 2018 through October 2022 and served as a director of CODA Biotherapeutics until the company’s sale in March 2023. Between March 2009 and July 2018, Mr. Narachi served as President, Chief Executive Officer and director of Orexigen Therapeutics, Inc., a biotechnology company. Orexigen filed for reorganization under Chapter 11 of the U.S. Bankruptcy Code in March 2018. Previously, Mr. Narachi served as Chairman, Chief Executive Officer, and President of Ren Pharmaceuticals, Inc., a private biotechnology company, from November 2006 to March 2009. In 2004, Mr. Narachi retired as Vice President of Amgen Inc., a leading therapeutics company, where he served as General Manager of Amgen’s Anemia Business from 1999 to 2003, until his retirement in 2004. Mr. Narachi joined Amgen in 1984 and held various senior positions throughout the organization over a 20-year career including global development leader for Neupogen/Neulasta, Vice President of development and representative director for Amgen Japan; head of corporate strategic planning; Chief Operations Officer of Amgen Colorado; and vice president, licensing and business development. Mr. Narachi previously served on the board of directors of BIO, the Biotechnology Innovation Organization, as a member of the board of directors of PhRMA, the Pharmaceutical Research and Manufacturers of America, and as the chairman of the board of directors of Celladon Corporation, a publicly traded gene therapy company, from October 2013 to March 2016, and as a director of AMAG Pharmaceuticals, Inc., a publicly traded specialty pharmaceutical company, from November 2006 to April 2014. Mr. Narachi holds a B. S. in Biology and an M. A. in Biology and Genetics from the University of California, Davis. He also holds an M.B.A. from the Anderson Graduate School of Management at the University of California, Los Angeles. Skills and Qualifications specific to Ultragenyx: We believe that Mr. Narachi is qualified to serve on our Board due to his extensive experience in the life sciences industry, his service as the chief executive officer of various biotechnology companies, and his membership on various boards of directors in the biotechnology and pharmaceutical sectors, all of which give him the skills to provide us with operational and strategic insights. |

  21. Class II Directors Continuing in Office until 2027

    | Corsee D. Sanders, Ph.D. | | | Board member and adviser to various life science companies | | | Age: 68 Director since: 2021 INDEPENDENT Committees: 2 Other Public Directorships: 3 | Dr. Sanders is a board member and advisor to life science companies. Dr. Sanders currently serves as a member of the board of directors of Beigene Ltd., Molecular Templates Inc. and Legend Biotech Corporation, each a publicly traded biotechnology company. She currently serves as a co-chair of the Board of Advisors, and Chair of the Science and Technology Advisory Committee for the Fred Hutchinson Cancer Center. She most recently served as Executive Vice President at Juno Therapeutics, Inc., a biopharmaceutical company, from 2017 to 2018 and strategic advisor to the Chief Medical Officer of Celgene Corporation, a biopharmaceutical company, from 2018 to 2019 following Juno’s acquisition by Celgene. Following the acquisition of Celgene by Bristol Myers Squibb, Dr. Sanders served as Transition Advisor to the Clinical Development team at BMS from 2019 to 2020. Prior to her role at BMS, she held positions of increasing responsibility at Genentech/Roche, a biotechnology company, from 1994 to 2017, including serving as Senior Vice President, Global Head of Clinical Operations and Industry Collaboration, from 2012 to 2017. Dr. Sanders holds a B.S. and M.S. in Statistics, magna cum laude, from the University of the Philippines. She also holds an M.A. and Ph.D. in Statistics from the Wharton Doctoral Program at the University of Pennsylvania. Skills and Qualifications specific to Ultragenyx: We believe that Dr. Sanders is qualified to serve on our Board due to her established and extensive experience in global clinical development and her role as an advisor and director to various companies in the biotechnology and pharmaceutical sectors, all of which give her the skills to provide us with operational and strategic insights. |

  22. Class II Directors Continuing in Office until 2027

    The two nominees who receive the greatest number of affirmative votes will be elected as Class III directors. Shares as to which a stockholder withholds voting authority and broker non-votes, if any, are not considered votes cast and therefore will have no effect on the vote outcome.

  23. Limits on Non-Employee Director Compensation

    Limits on Non-Employee Director Compensation The aggregate dollar value of equity-based (based on the grant date fair value of equity-based awards determined for financial reporting purposes) and cash compensation granted under the Second A&R 2023 Plan or otherwise to any one non-employee director during any fiscal year will not exceed $900,000, with up to $1,500,000 to be permitted for a non-employee director in the fiscal year he or she first joins our Board or is first designated as Chairman of our Board or Lead Director.

  24. Outcomes from Engagement

    | Feedback Received | | Action Taken | | More of executive’s compensation should be based on performance and “at risk” | | We have increased the percentage of PSUs from 33% to 50% for our executive officers (excluding our Chief Executive Officer) for the 2025 equity awards. For our Chief Executive Officer, we maintained the percentage of PSUs of 60% for 2024 and 2025 annual grants. We have also incorporated a “cap” on the portion of the PSUs subject to relative TSR performance for 2025 such that no more than the target number of PSUs may be earned if our absolute TSR is negative, regardless of our relative TSR performance. | | Greater transparency related to corporate goals and goals achievement | | We have included a more detailed summary regarding our bonus program and the impact of corporate goals and individual goals achievements on the bonus payout. We have also included additional detailed disclosure related to achievement of our corporate goals, including the achievement of each goal category. See the section below entitled “Annual Bonuses”. |

  25. Compensation Highlights

    | • | Increasing emphasis on performance-based awards: We maintained our equity mix at one-third of each PSUs, RSUs and stock options for named executive officers other than our Chief Executive Officer for 2024 annual grants but we increased the percentage of PSUs to 50% for the 2025 annual grant for our named executive officers (other than our Chief Executive Officer) without increasing the size of the pay package. For our Chief Executive Officer, PSUs were 60% of his annual grants in 2024 and 2025, consistent with the allocation implemented in 2023. |

  26. Compensation Highlights

    | • | Lengthening the time horizon for strategic performance portion of the PSUs: We lengthened the performance period for the strategic performance portion of the PSUs from two years to three years and, in doing so, we will require sustained performance over a longer-term timeframe. |

  27. Compensation Highlights

    | • | Increasing emphasis on relative TSR portion and the strategic performance portion of PSU awards: We shifted the PSU award mix to 1/3 for each of the revenue portion (decrease), strategic performance portion (increase), and relative TSR portion (increase) to add rigor and complexity to our PSU program. |

  28. Compensation Highlights

    | • | Further limit PSU upside: Beginning in 2025, the relative TSR portion of the PSUs is subject to a “cap” such that no more than the target number of PSUs may be earned if our absolute TSR is negative, regardless of our relative TSR performance. |

  29. Compensation Highlights

    | • | PSU payouts linked to performance: Our revenue portion of the 2023 PSUs paid out below target, at 74%. |

  30. CEO Realizable Compensation

    CEO Realizable Compensation To ensure ongoing alignment to the compensation philosophy, in 2024 our Compensation Committee evaluated the relationship between the target and realizable value of the compensation granted to our CEO from 2022 through 2024. The average realizable pay of our CEO over 2022 through 2024 is lower than the average target compensation for such three-year period, primarily as a result of the decrease in value of in-flight equity awards resulting from our stock performance as of the end of 2024 and the payouts from PSUs issued in 2022 and 2023 that paid out below target, as reflected in the following chart and described below.

  31. CEO Realizable Compensation

    | (1) Target value represents the base salary rate in effect in each year, target annual bonus, and the grant date fair value of long-term incentive awards (PSUs and RSUs). | | (2) Realizable value is calculated as the sum of the compensation deliverable in each year, including actual salary paid, annual bonus earned, and the value of long-term incentive plan components (PSUs, RSUs, and stock options) as valued on December 31, 2024 using our year-end stock price of $42.07 per share. The realizable value for PSUs granted in 2022 was adjusted to reflect the recent payout at 25% of target. The realizable value for PSUs granted in 2023 was adjusted to reflect recent payouts based on Revenue and Strategic performance, and the Relative TSR payout was assumed to be at 100% of target. The realizable value for PSUs granted in 2024 (for which the performance period has not ended) assumes payout at 100%. |

  32. Compensation Philosophy and Objectives

    | • | align compensation elements with our annual goals and long-term business strategies and objectives; |

  33. Compensation Philosophy and Objectives

    | • | promote the achievement of key strategic and financial performance measures by linking short-term and long-term cash and equity incentives to the achievement of measurable corporate and individual performance goals and objectives; and |

  34. Compensation Philosophy and Objectives

    The Compensation Committee has historically compensated executive officers with three primary compensation components: a base salary, an annual bonus opportunity, and equity-based compensation. The Compensation Committee believes that cash compensation in the form of base salary and an annual bonus opportunity provides our executive officers with short-term rewards for success in achieving annual goals and objectives, and that long-term compensation through the award of stock options, RSUs and PSUs aligns the objectives of management with those of our stockholders with respect to long-term performance and success of the Company. In setting compensation levels for our executive officers, the Compensation Committee does not formulaically benchmark against any one specific reference point. Instead, it considers a variety of factors, including peer group survey data, tenure, role, responsibilities, performance, and competitive market practices. Compensation paid to our named executive officers is delivered primarily through at-risk pay, based on both short-term and long-term incentives, including the achievement of corporate and individual goals and objectives. In addition to our compensation elements, the following compensation program features are designed to align our executive team’s interests with stockholder interests and market best practices.

  35. Compensation Philosophy and Objectives

    | What We Do | | What We Don’t Do | | | | Our Compensation Committee is comprised solely of independent directors. | | We do not offer any tax gross-up payments to our executive team for any change-of-control payments. | | | Our Compensation Committee engages its own independent, external compensation consultant to assist the committee in its review of executive and director compensation practices. | | As discussed above under “—Corporate Governance”, we prohibit our employees, including our executive officers, from hedging our securities. | | | We proactively engage with our stockholders throughout the year. | | We prohibit our employees, including our executive team, from pledging our securities. | | | We have a clawback policy that permits recovery of all incentive compensation (including time-based and performance-based equity awards) in the event of fraud or intentional misconduct by our current or former executive officers; see “—Clawback Policy”. | | We do not offer our executive team any substantially enhanced benefits or perquisites when compared with our overall employee population. | | | We require our executive officers and directors to hold shares of our common stock in order to align their long-term interests with those of our stockholders; see “—Minimum Stock Ownership Requirements”. | | We prohibit the repricing of outstanding stock options without stockholder approval. | | | We have double-trigger vesting of outstanding equity awards following covered transactions under our employment arrangements with our executive officers. See “Summary Compensation Table—Narrative Disclosure to Summary Compensation Table—Covered Transaction”. | | The Second A&R 2023 Plan does not include an “evergreen” feature. | | | We have established a long-term incentive program applicable to all current employees, including our executive officers, to further tie compensation to performance and focus employee efforts on corporate goals and objectives; see “—Equity Compensation—Annual Equity Grants in Fiscal 2024” and “—Fiscal 2025 Compensation – Equity Grants.” | | | | | We hold an annual say-on-pay vote for stockholders. | | |

  36. Compensation Consultant

    Compensation Consultant The Compensation Committee retains the services of an independent, external compensation consultant, Aon’s Human Capital Solutions Practice, a division of Aon plc (Aon). The mandate of the consultant is to assist the Compensation Committee in its review of executive and director compensation practices, including the competitiveness of pay levels, executive compensation design, benchmarking with our peers in the industry, and other technical considerations, including tax- and accounting-related matters. The Compensation Committee annually evaluates Aon’s performance and determines whether to engage Aon or another compensation consultant and has the final authority to engage and terminate Aon’s services. In 2024, the cost of Aon’s consulting services related to executive compensation, director compensation and equity plan design and considerations provided to the Compensation Committee was approximately $208,597. In addition, in 2024, management also engaged Aon to provide survey data relating to broad-based compensation matters. The aggregate cost of the survey data provided by Aon in 2024 was approximately $33,205. No other consulting services were provided by Aon or its affiliates to the Company in 2024. Our Compensation Committee has assessed the independence of Aon consistent with Nasdaq listing standards and has concluded that the engagement of Aon does not raise any conflict of interest.

  37. Defining and Comparing Compensation to Peer Group Benchmarks

    Defining and Comparing Compensation to Peer Group Benchmarks While we do not establish compensation levels based solely on benchmarking, pay practices at other companies are an important factor that the Compensation Committee considers in assessing the reasonableness of compensation and ensuring that our compensation practices are competitive in the marketplace. Market data is one element considered by the Compensation Committee when making executive compensation decisions, but the Compensation Committee does not set compensation levels based solely on market data. Rather, the Compensation Committee reviews the 25th, 50th and 75th percentiles of relevant market data as one frame of reference in making its executive compensation decisions. Final executive compensation decisions reflect a variety of factors, including each executive’s experience, performance rating, the relative importance of the executive’s role within the organization, as well as where each executive’s pay level falls relative to the market data. In order to evaluate the level of compensation for our named executive officers for 2024, our Compensation Committee, using information provided by Aon, established a peer group of publicly traded, national, and regional companies in the biopharmaceutical and biotechnology industries based on a balance of the following criteria:

  38. Defining and Comparing Compensation to Peer Group Benchmarks

    | • | companies with emphasis on orphan pharmaceutical products; |

  39. Defining and Comparing Compensation to Peer Group Benchmarks

    | • | companies with comparable market capitalizations (i.e., in the range of $2 billion to $16 billion); |

  40. Defining and Comparing Compensation to Peer Group Benchmarks

    | • | companies with revenue of between $200 million and $1.4 billion; and |

  41. Defining and Comparing Compensation to Peer Group Benchmarks

    | • | companies with headcounts between 300 to 4,000 employees. |

  42. Defining and Comparing Compensation to Peer Group Benchmarks

    Our 2024 peer group is comprised of the following 20 companies in the pharmaceutical and biotechnology industries, reflecting the removal of Alnylam Pharmaceuticals Inc., Biohaven Pharmaceutical Holding Company, Ltd., Global Blood Therapeutics, Inc. and United Therapeutics Corporation from our peer group used to evaluate compensation for our named executive officers in 2023 in order to add BridgeBio Pharma, Inc. Intra-Cellular Therapies Inc. and Sage Therapeutics, Inc. who our Compensation Committee determined better fit the balanced criteria described above.

  43. Defining and Comparing Compensation to Peer Group Benchmarks

    | ACADIA Pharmaceuticals Inc. | BridgeBio Pharma, Inc. | Ionis Pharmaceuticals, Inc. | | Alkermes plc | Corcept Therapeutics Inc. | Jazz Pharmaceuticals plc | | Amicus Therapeutics | Exelixis Inc. | Neurocrine Biosciences, Inc. | | Apellis Pharmaceuticals, Inc. | FibroGen, Inc. | PTC Therapeutics, Inc. | | BioCryst Pharmaceuticals, Inc. | Halozyme Therapeutics | Sage Therapeutics, Inc. | | BioMarin Pharmaceutical Inc. | Insmed Incorporated | Sarepta Therapeutics, Inc. | | Blueprint Medicines Corporation | Intra-Cellular Therapies Inc. | |

  44. Defining and Comparing Compensation to Peer Group Benchmarks

    We believe that the compensation practices of our 2024 peer group provided us with appropriate benchmarks for evaluating the compensation of our named executive officers for 2024 because of the developmental, market and organizational characteristics we shared with our peer group. At the time that we selected our 2024 peer group we were at approximately the 26th percentile of the peer group in terms of market capitalization, the 81st percentile in terms of employee headcount and the 44th percentile in terms of revenue.

  45. Annual Performance Reviews

    Annual Performance Reviews Our Compensation Committee conducts an annual performance review of our named executive officers and approves their compensation. By the end of the first quarter of each year, base salaries and equity awards for the fiscal year are approved and, for purposes of determining potential payments under our corporate bonus plan (the bonus plan), target bonuses, annual corporate goals and individual performance objectives are established and set forth in writing. After the end of each year, our Compensation Committee determines the amounts that will be paid to our executive officers under our bonus plan after carefully (1) reviewing overall corporate performance; (2) evaluating each named executive officer’s annual performance against established corporate goals; and (3) in the case of executive officers other than our Chief Executive Officer, reviewing the achievement of individual performance objectives. At its first regularly scheduled meeting each year, our Compensation Committee, with input from the Board, evaluates our Chief Executive Officer’s individual performance, determines whether to adjust his base salary, and determines the amount of equity awards and his bonus, if any, under our bonus plan. Our Compensation Committee may also review and adjust the compensation of our executive officers throughout the course of the year.

  46. 2024 Base Salaries

    2024 Base Salaries The Compensation Committee engaged Aon to conduct a competitive review and analysis of our current executive compensation program relative to our 2024 peer group. Aon prepared an Executive Compensation Assessment report in February 2024 that provided a competitive assessment of our executive compensation program as compared to the 2024 peer group data for base salaries, target total cash compensation, and equity compensation. For 2024, base salary increases for our Chief Executive Officer was 4% annualized and approximately 3% annualized for Mr. Harris and Mr. Pinion. Mr. Horn who joined our company in October 2023 did not receive a base salary increase for 2024. Dr. Crombez received a base salary increase of 15% following an independent compensation market analysis, which identified that his prior base salary was below the median for similar roles at peer companies. This adjustment brought his salary into line with market norms for the position while ensuring competitiveness in attracting and retaining talent. The overall 2024 merit budget was based on an Aon trend report regarding projected market merit spends for 2024. Individual increases in base salaries were also based on achievement of 2023 individual goals. The following table shows the increases in base salaries for our named executive officers between fiscal 2023 and fiscal 2024. Mr. Horn joined the Company in October 2023 and as such, did not receive an increase in his base salary for fiscal 2024.

  47. Annual Bonus

    Annual Bonus Our annual incentive program provides the opportunity for cash bonus awards based on the achievement of annual performance goals. For executive officers, other than the Chief Executive Officer, bonus awards are based on both corporate and individual performance, with corporate performance serving as the primary driver.

  48. Annual Bonus

    | • | Corporate performance goals focus on business, financial, and operational measures or objectives. | | • | Individual performance goals reflect an executive’s contributions toward achieving corporate goals and leadership within their respective function. | | • | The Chief Executive Officer’s bonus is entirely based on corporate performance. |

  49. Annual Bonus

    At the beginning of each performance year, the Chief Executive Officer sets and communicates individual goals to each executive officer. At the end of the year, the Compensation Committee evaluates corporate performance and each executive officer’s contributions (excluding the Chief Executive Officer) to determine whether a bonus will be awarded and, if so, the amount. The annual bonus is not guaranteed and is entirely at risk. The Chief Executive Officer’s annual bonus award is based 100% on corporate performance achievement. For executive officers other than the Chief Executive Officer, the annual bonus is calculated as follows: While individual performance is a key factor, corporate performance is the primary driver in the formula. If corporate goals are not met, an executive may not receive an annual bonus award, even if the executive performed well individually. Similarly, if corporate goals are met, an executive with low individual performance may receive a reduced award or no bonus award. The corporate performance achievement percentage is capped at 150% and the individual performance achievement percentage is capped at 133%, resulting in a maximum bonus of 150% of target for the Chief Executive Officer and 200% of target for the other executive officers. The corporate performance score determines the overall bonus pool funding. If corporate performance achievement is at or below 50%, the corporate component of the bonus is not funded and no bonuses will be awarded, regardless of individual performance. Actual payouts are at risk and contingent on both corporate and individual performance. Corporate performance payouts are based on the achievement of predefined goals, while individual performance is assessed across three key areas: attainment of individual goals, execution of key role deliverables, and demonstration of the Company’s values and behaviors. Subject to the rights contained in any agreement between the Company and the executive officer, an executive officer must be employed by the Company on the bonus payment date to be eligible to receive a bonus payment.

  50. Fiscal 2024 Bonuses

    Fiscal 2024 Bonuses Annual corporate goals for fiscal 2024 were proposed by our executive officers and approved by our Compensation Committee in early 2024. Individual objectives for our executive officers, other than the Chief Executive Officer, for 2024 were proposed by each executive officer, with review, input and confirmation from our Chief Executive Officer. Each executive officer’s annual target bonus is based on their role, responsibilities, accountability, potential impact on corporate performance and peer group benchmarks. The target bonuses, as a percentage of base salary, for the named executive officers for fiscal 2024 are set forth in the following table.

  51. Fiscal 2024 Bonuses

    | Name | Target Bonus for Fiscal 2024 (% of Base Salary) | | Emil D. Kakkis, M.D., Ph.D. | 80 | | Howard Horn | 50 | | Erik Harris | 50 | | Eric Crombez, M.D. | 50 | | John R. Pinion II | 50 |

  52. Fiscal 2024 Bonuses

    In February 2025, the Compensation Committee assessed our overall 2024 performance against the achievement of the corporate goals to determine a total percentage of achievement between 0% and 150%. The Compensation Committee considered the following performance goals, as well as the relative weighting of these goals, in assessing overall performance for the 2024 fiscal year:

  53. Fiscal 2024 Bonuses

    | Goal (Weighting) | Key Results | Achievement Against Goal | Weighted Achievement | | Commercial (30%) Revenue goals for commercial assets | Exceeded. Achieved revenue targets between base and outperform | 123% | 37% | | Development (60%) Clinical program-based goals focused on clinical development and enrollment-targets and milestone, including enrollment of the phase 3 portion of our study for UX143 and the BLA filing for UX111 by specified timelines. Highest weight was placed on goals related to GTX-102, UX143, UX701 and DTX401. | Met. Overall achievement of base target for development goals | 100% | 60% | | People and Governance (10%) | Met most. As summarized below, met or exceeded goals related to culture and governance. | 70% | 7.0% | | • Budget: Maintaining operating cash usage within 5% of budget (5%) | • Did not meet. Minimum target goal was not achieved primarily due to expenses from acceleration of select manufacturing and development activities as well as timing of certain cash flows (0% achievement) | | | | • Culture: Maintain high retention of employees in good performance standing (1.5%) | • Exceeded. Retention rate exceeded outperform target (150% achievement) | | | | • Culture: Goals related to maintaining and improving employee engagement, inclusion and well being (each 0.5%, together 1.5%) | • Employee engagement: Met base target for goal (100% achievement) • Inclusion: Exceeded outperform goal (150% achievement) • Well-being: Met base target for goal (100% achievement) | | | | • Governance: Goals related to continuous compliant and ethical practices (2%) | • Exceeded. Achieved outperform target goals (150% achievement) | | |

  54. Fiscal 2024 Bonuses

    In establishing these goals, the Compensation Committee selected performance goals that it considered aggressive, meaning that they are goals that were considered achievable, but only with a high degree of diligence and success in execution. In assessing performance against these goals, the Compensation Committee reviewed each goal and determined whether or not it was achieved. The Compensation Committee then referred to the relative importance of the goals, based on the previously established weightings of each goal. The Compensation Committee also considered additional key corporate achievements that were not represented in the 2024 corporate goals, including completion of a successful financing transaction and significant work to gain alignment with the FDA to support the submission of the BLA for UX111 for accelerated approval. For all goals combined, including additional achievements by the Company not previously defined, the Compensation Committee determined an overall 110% achievement for fiscal year 2024. In February 2025, in addition to assessing the foregoing corporate goals, the Compensation Committee (after consultation with our Chief Executive Officer) assessed the individual accomplishments of our named executive officers for purposes of determining the individual component of their annual bonus, other than our Chief Executive Officer whose annual bonus is based solely on the corporate goals described above. Key individual achievements for these named executive officers are summarized below.

  55. Fiscal 2024 Bonuses

    | Named Executive Officer | Key 2024 Achievements | | Howard Horn Chief Financial Officer and EVP, Corporate Strategy | • Successful execution of follow on equity offering for gross proceeds of $403 million • Informed decision making and drove execution of business development transactions, 2024 strategy and budget and long-range plan • Execution of defined operational excellence projects in collaboration with business teams | | Erik Harris Chief Commercial Officer and EVP | • Achieved continued growth in product sales to support achievement of global revenue targets, including exceeding top range of revenue guidance • Executed commercial planning for launch readiness for late-stage programs • Retention of key contributors for commercial leadership team | | Eric Crombez, M.D. Chief Medical Officer and EVP | • Led development team in achievement of high priority clinical milestones • Effective resource planning and right-sizing of development teams to achieve milestones • Continued close collaboration with cross-functional executive team to elevate development team performance and drive the business | | John R. Pinion II Chief Quality Officer and Executive Vice President, Translational Sciences | • Led and sponsored non-clinical activities to enable development and advancement of translational science portfolio • Successfully led start-up and advancement of Amlogenyx operations and other specified pre-clinical development, per plan • Led reliable quality supply across commercial and clinical products to support patient needs, revenue growth and clinical program success |

  56. Fiscal 2024 Bonuses

    Dr. Kakkis evaluated the performance of Mr. Horn, Mr. Harris, Dr. Crombez and Mr. Pinion after considering the above achievements and provided a proposed bonus amount for each such officer to the Compensation Committee in light of such officer’s achievements during 2024.

  57. Achievement of Goals and Relationship to Compensation Awarded

    | Name | Title | Corporate Component Score (%) (100% for CEO) | Individual Component Score (%) | Total Fiscal 2024 Bonus | Bonus Achieved (as % of Base Salary) | | Emil D. Kakkis, M.D., Ph.D. | President and Chief Executive Officer | 110 | — | $757,768 | 88% | | Howard Horn | Chief Financial Officer and Executive Vice President, Corporate Strategy | 110 | 106 | $343,970 | 58% | | Erik Harris | Chief Commercial Officer and Executive Vice President | 110 | 110 | $367,235 | 61% | | Eric Crombez, M.D. | Chief Medical Officer and Executive Vice President | 110 | 115 | $382,030 | 63% | | John R. Pinion II | Chief Quality Officer and Executive Vice President, Translational Sciences | 110 | 108 | $339,768 | 59% |

  58. Equity Compensation

    Equity Compensation

  59. Overview

    Overview Stock Options, Restricted Stock Units and Performance Stock Units. Executive officers are eligible to receive equity compensation in the form of stock options, RSUs and/or PSUs. The Compensation Committee grants stock options, RSUs and/or PSUs annually to executive officers to recognize their contributions to the achievement of corporate objectives, to align their interests with those of our stockholders by creating value tied to the performance of our stock price, and for retention purposes. In determining the form and value of an annual grant, the Compensation Committee considers the contributions and responsibilities of each executive officer, appropriate incentives for the achievement of our long-term growth, the size and value of grants made to other executives at peer companies holding comparable positions, individual achievement of designated performance goals, and our overall performance relative to corporate objectives. The Compensation Committee also grants stock options and RSUs to new executive officer hires. Under the terms of our 2023 Plan, pursuant to which all equity grants are currently made, the exercise price of any stock options awarded must be equal to at least 100% of the fair market value of our common stock (the closing sales price on The Nasdaq Global Select Market) on the date of grant. Authority to make equity grants to executive officers rests with the Compensation Committee. Recommendations for equity grants are made by Aon based on grant values for similarly situated executive positions in our peer group companies and accounting for dilution constraints and management of our burn rate. Our CEO recommends grants for individual executives within those guidelines. The Compensation Committee then reviews and considers our CEO’s recommendation and approves the final grant amounts. The Compensation Committee also determines and approves the final grant amounts to our CEO. We believe that annual equity awards serve as a useful performance recognition mechanism, encouraging the retention of executive officers by maintaining their focus on our long-term performance, as well as on the achievement of specific performance goals. Our typical option awards to executive officers (including our named executive officers) have a term of 10 years and vest and become exercisable over a period of four years, with 25% of the underlying shares vesting on the first anniversary of the grant date and the remainder monthly over the next three years. Our typical RSU awards to executive officers (including our named executive officers) vest and become exercisable over a period of four years, with 25% of the underlying shares vesting on each anniversary of the grant date. In addition to the new hire and annual equity awards described above, the Compensation Committee considers grants of other equity awards, as needed, to align business strategy with our compensation practices.

  60. Annual Equity Grants in Fiscal 2024

    Annual Equity Grants in Fiscal 2024

  61. Grants of Ultragenyx Awards

    Grants of Ultragenyx Awards In March 2024, the Compensation Committee approved equity grants that reflected an equal value split among options, RSUs and PSUs for our named executive officers other than our Chief Executive Officer, who instead received 60% of his grant in the form of PSUs, with the remaining portion split equally among options and RSUs. The increased portion of our Chief Executive Officer’s annual equity grant that is performance-based is intended to enhance the alignment with stockholder interests. The PSU awards granted to our executive officers in March 2024 (2024 PSUs) consist of a revenue portion (1/3 of the 2024 PSUs, which reflects a decrease from 50% of the 2023 PSUs) with a two-year performance period, a relative TSR portion (1/3 of the 2024 PSUs, which reflects an increase compared to 25% of the 2023 PSUs) with a three-year performance period and a strategic performance portion (1/3 of the 2024 PSUs, which reflects an increase compared to 25% of the 2023 PSUs) with a three-year performance period, which is longer than the two-year performance period for the strategic portion of the 2023 PSUs. The increased emphasis on relative TSR portion and the strategic performance portion of the PSU awards reduces the overall portion of our incentive compensation program that is focused on revenue and creates a more balanced mix of performance goals that are key to creation of long-term success. The increased performance period for the strategic performance portion of the 2024 PSUs results in additional program rigor due to a sustained performance requirement over a longer timeframe. The revenue-based 2024 PSUs will vest based upon achievement of revenue-based targets during the period beginning January 1, 2024 and ending December 31, 2025, with all of the earned revenue-based PSUs vesting on the later of (i) the date in which the Compensation Committee certifies such achievement and (ii) March 1, 2026. The revenue-based PSUs will be earned at 50% of target for threshold performance and 200% of target for maximum performance, with no PSUs being earned for below threshold performance. The relative TSR-based 2024 PSUs will vest based upon our TSR performance relative to the TSR of the companies in the Nasdaq Biotechnology Index during the period beginning January 1, 2024 and ending December 31, 2026, with all of the earned relative TSR-based PSUs vesting on the later of (i) the date in which the Compensation Committee certifies such achievement and (ii) March 1, 2027. The relative TSR-based PSUs will be earned as follows based on our percentile ranking among the Nasdaq Biotechnology Index during the performance period:

  62. Grants of Ultragenyx Awards

    | Level of Performance | TSR Percentile | Earned TSR PSUs | | Threshold | 25th | 25% | | Target | 50th | 100% | | Stretch | 75th | 150% | | Maximum | 90th | 200% |

  63. Grants of Ultragenyx Awards

    The strategic performance based 2024 PSUs will vest upon achievement of specified strategic performance objectives during the period between January 1, 2024 and ending December 31, 2026, with all of the earned strategic performance based PSUs vesting on the later of (i) the date in which the Compensation Committee certifies such achievement and (ii) March 1, 2027. The strategic performance based PSUs will be earned as follows based on the number of strategic goals achieved during the performance period. No PSUs are earned under the strategic performance based 2024 PSUs for below threshold performance.

  64. Grants of Ultragenyx Awards

    | Level of Performance | Number of Strategic Goals Achieved | Earned Strategic PSUs | | Threshold | 2 out of 5 | 50% | | Target | 3 out of 5 | 100% | | Stretch | 4 out of 5 | 150% | | Maximum | 5 out of 5 | 200% |

  65. Grants of Ultragenyx Awards

    | Name | Date of Grant | Number of Options | Number of RSUs | Target Number of PSUs | | Emil D. Kakkis, M.D., Ph.D. | 3/1/2024 | 70,094 | 38,428 | 115,284 | | Howard Horn | 3/1/2024 | 34,200 | 19,400 | 19,400 | | Erik Harris | 3/1/2024 | 34,200 | 19,400 | 19,400 | | Eric Crombez, M.D. | 3/1/2024 | 34,200 | 19,400 | 19,400 | | John R. Pinion II | 3/1/2024 | 31,400 | 17,800 | 17,800 |

  66. Grants of Performance Stock Options (PSOs) in Amlogenyx Inc. (Amlogenyx)

    Grants of Performance Stock Options (PSOs) in Amlogenyx Inc. (Amlogenyx) In addition to grants of Ultragenyx options, RSUs and PSUs in fiscal 2024 as described above, in recognition of their contributions to Amlogenyx, Dr. Kakkis, Mr. Horn and Mr. Pinion also received grants of PSOs from Amlogenyx, a privately-held subsidiary of Ultragenyx in which Ultragenyx currently holds a majority interest, during fiscal 2024. The Amlogenyx PSOs will vest upon achievement of specified performance criteria related to clinical milestones, with each criteria weighted 25%. If the performance criteria are not achieved by the end of the 18 month performance period on April 21, 2026, the unvested portion of the Amlogenyx PSOs will be forfeited.

  67. Grants of Performance Stock Options (PSOs) in Amlogenyx Inc. (Amlogenyx)

    | Name | Date of Grant | Number of Amlogenyx PSOs | | Emil D. Kakkis, M.D., Ph.D. | 10/21/2024 | 350,000 | | Howard Horn | 10/21/2024 | 22,500 | | John R. Pinion II | 10/21/2024 | 100,000 |

  68. Performance of 2023 PSUs

    Performance of 2023 PSUs The PSU awards granted to our executive officers in March 2023 (2023 PSUs) consisted of a revenue portion with a two-year performance period (50% of the PSUs), a relative TSR portion (25% of the PSUs) with a three-year performance period and a strategic performance portion (25% of the PSUs) with a two-year performance period. The performance periods of the revenue portion and strategic performance portion of the 2023 PSUs ended on December 31, 2024 and the revenue portion and strategic performance portion of the 2023 PSUs vested on March 1, 2025, as described below. The performance period for the relative TSR-based 2023 PSUs extends through December 31, 2025, and if earned, such portion will vest in early 2026.

  69. Achievement of Revenue Portion of 2023 PSUs

    Achievement of Revenue Portion of 2023 PSUs The revenue-based 2023 PSUs vested based upon achievement of the following revenue-based targets (and application of linear interpolation) during the period beginning January 1, 2023 and ending December 31, 2024, with all of the earned revenue-based PSUs vesting on the later of (i) the date in which the Compensation Committee certifies such achievement and (ii) March 1, 2025.

  70. Achievement of Revenue Portion of 2023 PSUs

    | Level of Performance | Aggregate Revenue | Earned Revenue-Based PSU Percentage | | Threshold | $945M | 50% | | Actual | $995M | 74% | | Target | $1,050M | 100% | | Maximum | $1,260M | 200% |

  71. Achievement of Revenue Portion of 2023 PSUs

    In February 2025, based on the Company’s 2023 and 2024 revenue performance measured in accordance with U.S. GAAP (GAAP), the Compensation Committee certified that the revenue based 2023 PSUs were earned at 74% of target, and such earned 2023 PSUs vested on March 1, 2025.

  72. Achievement of Strategic Performance Portion of 2023 PSUs

    Achievement of Strategic Performance Portion of 2023 PSUs The strategic performance based 2023 PSUs vested upon achievement of specified strategic performance objectives during the period between January 1, 2023 and ending December 31, 2024, with all of the earned strategic performance based PSUs vesting on the later of (i) the date in which the Compensation Committee certifies such achievement and (ii) March 1, 2025.

  73. Achievement of Strategic Performance Portion of 2023 PSUs

    | Level of Performance | Number of Strategic Goals Achieved | Earned Strategic PSUs | | Threshold | 2 out of 5 | 50% | | Target | 3 out of 5 | 100% | | Actual | 3.5 out of 5 | 125% | | Stretch | 4 out of 5 | 150% | | Maximum | 5 out of 5 | 200% |

  74. Achievement of Strategic Performance Portion of 2023 PSUs

    In February 2025, based on the Company’s achievement of 3.5 out of 5 strategic performance objectives, the Compensation Committee certified that the strategic performance portion of 2023 PSUs were earned at 125% of target, and such earned 2023 PSUs vested on March 1, 2025.

  75. Performance of 2022 PSUs

    Performance of 2022 PSUs The PSU awards granted to our executive officers in March 2022 (2022 PSUs) consisted of a revenue portion with a two-year performance period (80% of the PSUs) and a relative TSR portion with a three-year performance period (20% of the PSUs). In February 2024, based on the Company’s 2022 and 2023 revenue performance measured in accordance with GAAP, the Compensation Committee certified that the threshold level of revenue-based 2022 PSUs had not been achieved and as a result, the revenue-based portion of the 2022 PSUs were forfeited. The relative TSR-based 2022 PSUs vested based upon our TSR performance relative to the TSR of the companies in the Nasdaq Biotechnology Index during the period beginning January 1, 2022 and ending December 31, 2024, with all of the earned relative TSR-based PSUs vesting on the later of (i) the date in which the Compensation Committee certifies such achievement and (ii) March 1, 2025. The relative TSR-based PSUs was earned as follows based on our percentile ranking among the Nasdaq Biotechnology Index during the performance period:

  76. Performance of 2022 PSUs

    | Level of Performance | TSR Percentile | Earned TSR PSUs | | Threshold | 25th | 25% | | Target | 50th | 100% | | Actual | 62nd | 124% | | Stretch | 75th | 150% | | Maximum | 90th | 200% |

  77. Performance of 2022 PSUs

    Based on the Company’s ranking relative to the TSR of the companies in the Nasdaq Biotechnology Index of the 62nd percentile during the performance period, in February 2025, the Compensation Committee certified that the relative TSR-based 2022 PSUs were earned at 124% of target, and such earned 2022 PSUs vested on March 1, 2025.

  78. Peer Group

    Peer Group The Compensation Committee reviews our list of peer companies annually to determine if revisions are needed to reflect changes in our development status, market capitalization, changes in individual peer companies, and other factors. The Compensation Committee engaged Aon to assist in reviewing our peer group and in suggesting revisions, as appropriate. Based on Aon’s assessment and recommendations, the Compensation Committee selected 20 publicly traded companies in the pharmaceutical and biotechnology industries to serve as our new list of peer companies for 2025, referred to as our 2025 peer group, by balancing the following criteria:

  79. Peer Group

    | • | companies with emphasis on orphan pharmaceutical products; | | • | companies with comparable market capitalizations (i.e., in the range of $2 billion to $14 billion); | | • | companies with revenue of between $250 million and $1.6 billion; and | | • | companies with headcounts between 300 to 4,000 employees. |

  80. Peer Group

    Our 2025 peer group is comprised of the following 19 companies in the pharmaceutical and biotechnology industries, reflecting the addition of Axsome Therapeutics, Inc., Denali Therapeutics Inc. and Vericel Corporation and the removal of BioCryst Biopharmaceuticals, Inc., FibroGen, Inc. and Sage Therapeutics, Inc. from our 2024 peer group due to misalignment with our market capitalization and the removal of BioMarin Biopharmaceutical Inc. who exceeded our market capitalization and revenue ranges.

  81. Peer Group

    | ACADIA Pharmaceuticals Inc. | Corcept Therapeutics Inc. | Jazz Pharmaceuticals plc | | Alkermes plc | Denali Therapeutics Inc. | Neurocrine Biosciences, Inc. | | Amicus Therapeutics | Exelixis Inc. | PTC Therapeutics, Inc. | | Apellis Pharmaceuticals, Inc. | Halozyme Therapeutics | Sarepta Therapeutics, Inc. | | Axsome Therapeutics, Inc. | Insmed Incorporated | Vericel Corporation | | Blueprint Medicines Corporation | Intra-Cellular Therapies Inc. | | | BridgeBio Pharma, Inc. | Ionis Pharmaceuticals, Inc. | |

  82. Peer Group

    We believe that the compensation practices of our 2025 peer group provided us with appropriate compensation benchmarks for evaluating the compensation of our named executive officers for 2025.

  83. Annual Bonuses

    Annual Bonuses For 2025, the Compensation Committee maintained the target annual bonus of 80% for our Chief Executive Officer and maintained the target bonus amount of 50% for all other named executive officers. No significant changes were made to the bonus plan for 2025.

  84. Equity Compensation

    Equity Compensation For 2025, the Compensation Committee determined to increase the PSU component of the annual equity grants to our executive officers, other than our Chief Executive Officer, from 33% to 50%, with the remaining portion of the grant equally split at 25% options and 25% RSUs. For our Chief Executive Officer, the Compensation Committee maintained the equity split of 60% PSUs, 20% options and 20% RSUs. The PSU awards granted to our executive officers in 2025 (2025 PSUs) consist of a revenue portion with a two-year performance period (1/3 of the 2025 PSUs), a relative TSR portion with a three-year performance period (1/3 of PSUs) and a strategic performance portion (1/3 of the PSUs) with a three-year performance period. The revenue-based 2025 PSUs will vest based upon achievement of revenue-based targets during the period beginning January 1, 2025 and ending December 31, 2026, with all of the earned revenue-based PSUs vesting on the later of (i) the date in which the Compensation Committee certifies such achievement and (ii) March 1, 2027. The relative TSR-based 2025 PSU awards will vest based upon our TSR performance relative to the TSR of the companies in the Nasdaq Biotechnology Index during the period beginning January 1, 2025 and ending December 31, 2027, with a maximum payout of 100% of target in the event our absolute TSR is negative, with all of the earned relative TSR-based PSUs vesting on the later of (i) the date in which the Compensation Committee certifies such achievement and (ii) March 1, 2028. The strategic-based 2025 PSU awards will vest based on achievement of specified strategic goals during the period beginning January 1, 2025 and ending December 31, 2027, with all of the earned strategic based PSUs vesting on the later of (i) the date in which the Compensation Committee certifies such achievement and (ii) March 1, 2028. The PSUs will be earned at 50% of target for threshold performance (or 25% of target for the relative TSR-based PSUs) and 200% of target for maximum performance, with no PSUs becoming earning for below threshold performance.

  85. Equity Grant Timing

    Equity Grant Timing Generally, the Compensation Committee grants annual equity awards, including stock options, on March 1 of each year following the filing of our Annual Report on Form 10-K and grants new hire equity awards, including stock options, on the 16th of each month following the individual’s start date. The Compensation Committee may also grant equity awards at other times during the year, such as in connection with special retention awards, performance recognition, or in the event of a significant change in job responsibilities. Under the 2014 Employee Stock Purchase Plan, eligible employees may purchase shares at a discount, with purchase dates generally on April 30 and October 31 of each year, using payroll deductions accumulated during the prior six-month period. The Compensation Committee did not take material nonpublic information into account when determining the timing and terms of equity awards, including stock options, during 2024, and we did not time the disclosure of material nonpublic information for the purpose of affecting the value of executive compensation. During 2024, no stock options were granted to the named executive officers in the period beginning four business days before and ending one business day after the filing or furnishing of any Form 10-Q, Form 10-K or Form 8-K that disclosed material nonpublic information.

  86. Risk Management and Mitigation

    Risk Management and Mitigation In reviewing the compensation structure in fiscal 2024, the Compensation Committee also considered how our compensation policies may affect our risk profile and how compensation policies may be used to mitigate risks facing us. More specifically, the Compensation Committee considered the general design philosophy of our policies for employees whose conduct would be most affected by incentives established by compensation policies. In considering these issues, the Compensation Committee concluded that the use of performance-based bonuses and long-term equity awards did not appear to create undue risks for us or encourage excessive risk-taking behavior on the part of employees. With respect to bonus awards, the amount of an individual’s award depends principally (exclusively, in the case of our Chief Executive Officer) on our overall performance, which reduces the ability and incentive for an individual to take undue risks in an effort to increase the amount of his or her bonus award for a particular year. For fiscal 2024, our corporate goals were reviewed and approved by the Board in early 2024, upon the recommendation of the Compensation Committee, and are considered to be generally of the nature that would not encourage or reward excessive risk taking. Additionally, the Compensation Committee monitors our performance throughout the year and has the ability to intervene in instances where our actions vis-à-vis our performance goal attainment would be considered unduly risky, so that the Compensation Committee may act to prevent or penalize such actions. With respect to equity awards, these awards typically vest over several years, meaning that long-term value creation, contrasted with short-term gain, presents the best opportunity for employees to benefit from these awards.

  87. Summary Compensation Table

    Summary Compensation Table The following table sets forth the compensation earned during the years ended December 31, 2024, 2023, and 2022 by our Chief Executive Officer, our Chief Financial Officer and our next three highest-paid executives. We refer to these officers as our named executive officers.

  88. Summary Compensation Table

    | Name and Principal Position | Year | Salary | Bonus | Stock Award(1) | Option Award(2) | Non-Equity Incentive Plan Compensation(3) | All Other Compensation(4) | Total | | Emil D. Kakkis, M.D., Ph.D. President and Chief Executive Officer | 2024 | $856,008 | $— | $9,374,511 | $2,715,600 | $757,768 | $13,800 | $13,717,687 | | | 2023 | 823,692 | — | 9,456,663 | 2,161,782 | 622,656 | 13,200 | 13,077,993 | | | 2022 | 796,154 | — | 7,463,295 | 3,310,681 | 558,000 | 12,200 | 12,140,330 | | Howard Horn Chief Financial Officer, Corporate Strategy and Executive Vice President | 2024 | 590,000 | — | 2,271,944 | 1,076,335 | 343,970 | 13,800 | 4,296,049 | | | 2023 | 124,808 | 60,000 | 2,867,459 | 2,888,773 | — | — | 5,941,040 | | Erik Harris Chief Commercial Officer and Executive Vice President | 2024 | 604,231 | — | 2,271,944 | 1,038,603 | 367,235 | 13,800 | 4,295,813 | | | 2023 | 585,969 | — | 2,169,750 | 1,020,050 | 279,598 | 13,200 | 4,068,567 | | | 2022 | 566,331 | — | 2,007,768 | 892,550 | 269,706 | 12,200 | 3,748,555 | | Eric Crombez, M.D. Chief Medical Officer and Executive Vice President | 2024 | 591,846 | — | 2,271,944 | 1,038,603 | 382,030 | 13,800 | 4,298,223 | | John R. Pinion II Chief Quality Officer and Executive Vice President, Translational Sciences | 2024 | 570,366 | — | 2,084,548 | 1,121,271 | 339,768 | 13,800 | 4,129,753 | | | 2023 | 552,092 | — | 2,169,750 | 1,020,050 | 294,761 | 13,200 | 4,049,853 | | | 2022 | 533,315 | — | 2,007,768 | 892,550 | 260,678 | 12,200 | 3,706,511 |

  89. Summary Compensation Table

    | (1) The amounts reported in this column for a fiscal year represent the grant date fair value of the RSUs and PSUs granted to our named executive officers during the fiscal year, as computed in accordance with ASC Topic 718, not including any estimates of forfeitures, and, with respect to the PSUs, assuming the most probable outcome of the performance conditions as of the grant date. The assumptions used in calculating the grant date fair value of the RSUs and PSUs reported in this column are set forth in the notes to our financial statements included in our Annual Report. The amounts reported in this column reflect the accounting cost for these RSUs and PSUs and do not correspond to the actual economic value that may be realized by the named executive officers from the RSUs and the PSUs. The value of the PSUs reported in this column for 2024, assuming achievement of the maximum performance level, was as follows: $14,622,623 for Dr. Kakkis, $2,460,715 for each of Mr. Horn, Dr. Crombez and Mr. Harris and $2,257,733 for Mr. Pinion. | | (2) The amounts reported in this column for a fiscal year represent the grant date fair value of the stock options granted to our named executive officers during the fiscal year, as computed in accordance with ASC Topic 718, not including any estimates of forfeitures. Amounts included in this column for Dr. Kakkis, Mr. Horn and Mr. Pinion also include the grant date fair value of the PSOs granted from Amlogenyx, a privately-held subsidiary of Ultragenyx in which Ultragenyx currently holds a majority interest, as described above under “ —Annual Equity Grants in Fiscal 2024”. The grant date fair value of the Amlogenyx PSOs is as follows: $586,950 for Dr. Kakkis, $37,733 for Mr. Horn and $167,700 for Mr. Pinion. The assumptions used in calculating the grant date fair value of the Amlogenyx PSOs reported in this column were as follows: expected term of 5.75 years, risk free rate of 3.98%, volatility of 85% and exercise price of $2.31. The amounts reported in this column reflect the accounting cost for these stock options, and do not correspond to the actual economic value that may be received by the named executive officers from the options. | | (3) Amounts for a fiscal year represent annual cash bonuses earned in that fiscal year and paid in the subsequent fiscal year based on achievement of performance goals and other factors deemed relevant by our Compensation Committee under our annual incentive program. | | (4) Amounts reported in this column for the 2024 fiscal year reflects 401(k) matching contributions. |

  90. Grants of Plan-Based Awards

    Grants of Plan-Based Awards The following table sets forth certain information regarding grants of plan-based awards to the named executive officers during fiscal 2024.

  91. Grants of Plan-Based Awards

    | | | Estimated Future Payouts Under Non-Equity Incentive Plan Awards(1) | | | Estimated Future Payouts Under Equity Incentive Plan Awards(2) | | | Stock Awards: Number of Shares of Stock | Option Awards: Number of Securities Underlying | Exercise Price of | Grant Date Fair Value of Stock | | Name | Grant Date | Threshold ($) | Target ($) | Maximum ($) | Threshold (#) | Target ($) | Maximum ($) | or Units Granted (#)(3) | Options Granted (#) | Option Awards ($/Share) | and Option Awards ($)(4) | | Emil D. Kakkis, | | 344,400 | 688,800 | 1,033,200 | — | — | — | — | — | — | — | | M.D., Ph.D. | 3/1/2024 | — | — | — | — | — | — | — | 70,094 | 53.69 | 2,128,650 | | | 3/1/2024 | — | — | — | — | — | — | 38,428 | — | — | 2,063,199 | | | 3/1/2024 | — | — | — | 48,045 | 115,284 | 230,568 | — | — | — | 7,311,311 | | | 10/21/2024(5) | — | — | — | — | 350,000 | — | — | — | 2.31 | 586,950 | | Howard Horn | | — | 295,000 | 588,525 | — | — | — | — | — | — | — | | | 3/1/2024 | — | — | — | — | — | — | — | 34,200 | 53.69 | 1,038,603 | | | 3/1/2024 | — | — | — | — | — | — | 19,400 | — | — | 1,041,586 | | | 3/1/2024 | — | — | — | 8,085 | 19,400 | 38,800 | — | — | — | 1,230,358 | | | 10/21/2024(5) | — | — | — | — | 22,500 | — | — | — | 2.31 | 37,733 | | Erik Harris | | — | 303,500 | 605,483 | — | — | — | — | — | — | — | | | 3/1/2024 | — | — | — | — | — | — | — | 34,200 | 53.69 | 1,038,603 | | | 3/1/2024 | — | — | — | — | — | — | 19,400 | — | — | 1,041,586 | | | 3/1/2024 | — | — | — | 8,085 | 19,400 | 38,800 | — | — | — | 1,230,358 | | Eric Crombez., | | — | 302,000 | 602,490 | — | — | — | — | — | — | — | | M.D. | 3/1/2024 | — | — | — | — | — | — | — | 34,200 | 53.69 | 1,038,603 | | | 3/1/2024 | — | — | — | — | — | — | 19,400 | — | — | 1,041,586 | | | 3/1/2024 | — | — | — | 8,085 | 19,400 | 38,800 | — | — | — | 1,230,358 | | John R. Pinion II | | — | 286,000 | 570,570 | — | — | — | — | — | — | — | | | 3/1/2024 | — | — | — | — | — | — | — | 31,400 | 53.69 | 953,571 | | | 3/1/2024 | — | — | — | — | — | — | 17,800 | — | — | 955,682 | | | 3/1/2024 | — | — | — | 7,418 | 17,800 | 35,600 | — | — | — | 1,128,866 | | | 10/21/2024(5) | — | — | — | — | 100,000 | — | — | — | 2.31 | 167,700 |

  92. Grants of Plan-Based Awards

    | (1) The amounts in these columns represent the threshold (for Dr. Kakkis only), target and maximum amount of each named executive officer’s cash payments under our 2024 annual incentive program as established by the Board and described in “Compensation Discussion and Analysis” above. Actual payments made for fiscal 2024 are provided in the Summary Compensation Table. | | (2) The amounts in these columns represent the threshold, target and maximum level of achievement for the 2024 PSUs granted under our 2023 Incentive Plan. The PSU awards consist of a revenue portion (33.4% of the PSUs) with a two-year performance period, a relative total stockholder return (TSR) portion (33.3% of the PSUs) with a three-year performance period and a strategic performance portion (33.3% of the PSUs) with a three-year performance period. See “—Annual Equity Grants in Fiscal 2024” above for a description of the 2024 PSUs. | | (3) The amounts in this column represents the RSUs granted under our 2023 Plan during 2024. | | (4) This column reflects the aggregate grant date fair value of equity awards granted in 2024 as computed in accordance with ASC Topic 718, not including any estimates of forfeitures. The assumptions used in calculating the grant date fair value of the stock and option awards reported in this column are set forth in Note 13 to our financial statements included in our Annual Report for fiscal 2024. | | (5) Reflects PSOs granted by Amlogenyx, a privately-held subsidiary of Ultragenyx in which Ultragenyx currently holds a majority interest, as described above under “—Annual Equity Grants in Fiscal 2024”. The Amlogenyx PSOs will vest upon achievement of specified clinical performance criteria, with each criteria weighted 25%. If the performance criteria are not achieved during the 18-month performance period ending April 21, 2026, any unvested portion of the Amlogenyx PSOs will be forfeited. |

  93. Outstanding Equity Awards at December 31, 2024

    Outstanding Equity Awards at December 31, 2024 The following table sets forth information concerning the outstanding equity awards held by each of the named executive officers as of December 31, 2024.

  94. Outstanding Equity Awards at December 31, 2024

    | | | Option Awards(1) | | | | | Stock Awards | | | | | Name | Grant Date | Number of Securities Underlying Unexercised Options (#) Exercisable | Number of Securities Underlying Unexercised Options (#) Unexercisable | Equity Incentive Plan Awards: Number of Securities Underlying Unexercised Unearned Options (#)(2) | Option Exercise Price ($) | Option Expiration Date | Number of Shares or Units of Stock That Have Not Vested (#)(3) | Market Value of Shares or Units of Stock That Have Not Vested ($) | Equity Incentive Plan Awards: Number of Unearned Shares, Units or Other Rights That Have Not Vested (#)(4) | Equity Incentive Plan Awards: Market or Payout Value of Unearned Shares, Units or Other Rights That Have Not Vested ($) | | Emil D. Kakkis, M.D., Ph.D. | 10/21/2024 | — | — | 350,000 | 2.31 | 10/21/2034 | — | — | — | — | | | 3/1/2024 | — | 70,094 | — | 53.69 | 3/1/2034 | 38,428 | 1,616,666 | 115,284 | 4,849,998 | | | 3/1/2023 | 36,531 | 46,969 | — | 45.65 | 3/1/2033 | 129,036 | 5,428,545 | 44,314 | 1,864,290 | | | 3/1/2022 | 63,498 | 28,862 | — | 67.37 | 3/1/2032 | 38,926 | 1,637,617 | — | — | | | 3/1/2021 | 33,750 | 2,250 | — | 142.47 | 3/1/2031 | 4,500 | 189,315 | — | — | | | 3/1/2020 | 56,100 | — | — | 56.08 | 3/1/2030 | — | — | — | — | | | 3/1/2019 | 66,000 | — | — | 67.55 | 3/1/2029 | — | — | — | — | | | 3/1/2018 | 94,500 | — | — | 48.43 | 3/1/2028 | — | — | — | — | | | 3/1/2017 | 78,000 | — | — | 88.80 | 3/1/2027 | — | — | — | — | | | 6/1/2016 | 63,700 | — | — | 70.57 | 6/1/2026 | — | — | — | — | | | 5/21/2015 | 68,300 | — | — | 84.89 | 5/21/2025 | — | — | — | — | | Howard Horn | 10/21/2024 | — | — | 22,500 | 2.31 | 10/21/2034 | — | — | — | — | | | 3/1/2024 | — | 34,200 | — | 53.69 | 3/1/2034 | 19,400 | 816,158 | 19,400 | 816,158 | | | 10/9/2023 | 40,971 | 99,498 | — | 35.68 | 10/9/2033 | 60,274 | 2,535,727 | — | — | | Erik Harris | 3/1/2024 | — | 34,200 | — | 53.69 | 3/1/2034 | 19,400 | 816,158 | 19,400 | 816,158 | | | 3/1/2023 | 17,238 | 22,162 | — | 45.65 | 3/1/2033 | 31,471 | 1,323,985 | 7,029 | 295,710 | | | 3/1/2022 | 17,119 | 7,781 | — | 67.37 | 3/1/2032 | 10,472 | 440,557 | — | — | | | 3/1/2021 | 10,313 | 687 | — | 142.47 | 3/1/2031 | 1,375 | 57,846 | — | — | | | 3/1/2020 | 22,000 | — | — | 56.08 | 3/1/2030 | — | — | — | — | | | 6/19/2019 | 12,000 | — | — | 63.27 | 6/19/2029 | — | — | — | — | | | 3/1/2019 | 13,000 | — | — | 67.55 | 3/1/2029 | — | — | — | — | | | 3/1/2018 | 3,900 | — | — | 48.43 | 3/1/2028 | — | — | — | — | | | 7/6/2017 | 30,000 | — | — | 63.28 | 7/6/2027 | — | — | — | — | | Eric Crombez, M.D. | 3/1/2024 | — | 34,200 | — | 53.69 | 3/1/2034 | 19,400 | 816,158 | 19,400 | 816,158 | | | 5/1/2023 | 3,310 | 5,052 | — | 44.03 | 5/1/2033 | 18,464 | 776,780 | 7,029 | 295,710 | | | 3/1/2023 | 4,308 | 5,537 | — | 45.65 | 3/1/2033 | 5,538 | 232,984 | — | — | | | 3/1/2022 | 7,491 | 8,249 | — | 67.37 | 3/1/2032 | 1,587 | 66,765 | — | — | | | 4/16/2021 | — | — | — | — | | 500 | 21,035 | — | — | | | 3/1/2021 | 4,041 | 269 | — | 142.47 | 3/1/2031 | 360 | 15,145 | — | — | | | 3/1/2020 | 13,000 | — | — | 56.08 | 3/1/2030 | — | — | — | — | | | 3/1/2019 | 8,000 | — | — | 67.55 | 3/1/2029 | — | — | — | — | | | 11/17/2017 | 7,500 | — | | 48.11 | 11/17/2027 | — | — | — | — |

  95. Outstanding Equity Awards at December 31, 2024

    | | | Option Awards(1) | | | | | Stock Awards | | | | | Name | Grant Date | Number of Securities Underlying Unexercised Options (#) Exercisable | Number of Securities Underlying Unexercised Options (#) Unexercisable | Equity Incentive Plan Awards: Number of Securities Underlying Unexercised Unearned Options (#)(2) | Option Exercise Price ($) | Option Expiration Date | Number of Shares or Units of Stock That Have Not Vested (#)(3) | Market Value of Shares or Units of Stock That Have Not Vested ($) | Equity Incentive Plan Awards: Number of Unearned Shares, Units or Other Rights That Have Not Vested (#)(4) | Equity Incentive Plan Awards: Market or Payout Value of Unearned Shares, Units or Other Rights That Have Not Vested ($) | | John R. Pinion II | 10/21/2024 | — | — | 100,000 | 2.31 | 10/21/2034 | — | — | — | — | | | 3/1/2024 | — | 31,400 | — | 53.69 | 3/1/2034 | 17,800 | 748,846 | 17,800 | 748,846 | | | 3/1/2023 | 17,238 | 22,162 | — | 45.65 | 3/1/2033 | 31,471 | 1,323,985 | 7,029 | 295,710 | | | 3/1/2022 | 17,119 | 7,781 | — | 67.37 | 3/1/2032 | 10,472 | 440,557 | — | — | | | 3/1/2021 | 10,313 | 687 | — | 142.47 | 3/1/2031 | 1,375 | 57,846 | — | — | | | 3/1/2020 | 22,000 | — | — | 56.08 | 3/1/2030 | — | — | — | — | | | 3/1/2019 | 23,000 | — | — | 67.55 | 3/1/2029 | — | — | — | — | | | 3/1/2018 | 27,000 | — | — | 48.43 | 3/1/2028 | — | — | — | — | | | 3/1/2017 | 18,000 | — | — | 88.80 | 3/1/2027 | — | — | — | — | | | 6/3/2016 | 11,000 | — | — | 69.53 | 6/3/2026 | — | — | — | — | | | 6/1/2016 | 17,800 | — | — | 70.57 | 6/1/2026 | — | — | — | — | | | 7/16/2015 | 90,000 | — | — | 124.87 | 7/16/2025 | — | — | — | — |

  96. Outstanding Equity Awards at December 31, 2024

    | (1) The options vest with respect to 1/4th of the shares underlying the option on the one-year anniversary of the applicable grant date, and with respect to 1/48th of the shares underlying the option, on each monthly anniversary thereafter, subject to the holder’s continued service to us through each such vesting date. Please see the section entitled “—Narrative Disclosure to Summary Compensation Table—Covered Transaction” for accelerated vesting provisions that apply on certain terminations of employment. | | (2) Reflects PSOs from Amlogenyx, a privately-held subsidiary of Ultragenyx in which Ultragenyx currently holds a majority interest. The Amlogenyx PSOs will vest upon achievement of specified clinical performance criteria, with each criteria weighted 25%. If the performance criteria are not achieved during the 18-month performance period ending April 21, 2026, any unvested portion of the Amlogenyx PSOs will be forfeited. | | (3) Amounts in this column reflects unvested RSUs as well as earned but unvested PSUs. The RSUs vest with respect to 1/4th of the underlying shares on each anniversary of the grant date over a four-year period. For (i) Dr. Kakkis, 12,906 of the total earned PSUs listed in the March 1, 2022 row and 94,386 of the total earned PSUs listed in the March 1, 2023 row vested on March 1, 2025 and for (ii) each of Mr. Harris and Mr. Pinion, 3,472 of the total earned PSUs listed in the March 1, 2022 row, for Mr. Harris and Mr. Pinion, 14,971 of the total earned PSUs listed in the March 1, 2023 row vested on March 1, 2025 and for Dr. Crombez, 14,971 of the of the total earned PSUs listed in the May 1, 2023 row vested on March 1, 2025. Please see the section entitled “—Narrative Disclosure to Summary Compensation Table—Covered Transaction” for accelerated vesting provisions that apply on certain terminations of employment. | | (4) The PSUs set forth in the table are reported at target achievement. The PSUs vest as described above under “Compensation Discussion and Analysis—Equity Compensation”. Please see the section entitled “—Narrative Disclosure to Summary Compensation Table—Covered Transaction” for accelerated vesting provisions that apply on certain terminations of employment. |

  97. Option Exercises and Stock Vested

    Option Exercises and Stock Vested The following table sets forth certain information concerning the stock awards vested for our named executive officers during fiscal 2024. No named executive officers exercised any option awards during fiscal 2024.

  98. Option Exercises and Stock Vested

    | | Stock Awards | | | Name | Number of Shares Acquired on Vesting (#) | Value Realized on Vesting ($)(1) | | Emil D. Kakkis, M.D., Ph.D. | 36,442 | 1,956,571 | | Howard Horn | 20,092 | 1,072,511 | | Erik Harris | 13,282 | 713,111 | | Eric Crombez, M.D. | 5,781 | 292,588 | | John R. Pinion II | 11,620 | 623,878 |

  99. Option Exercises and Stock Vested

    | (1) Value realized is equal to the closing price of our common stock on The Nasdaq Global Select Market on each vesting date multiplied by the number of shares of stock that vested on such date. |

  100. Pension Benefits

    Pension Benefits We do not have a defined benefit plan. Our named executive officers did not participate in, or otherwise receive any special benefits under, any pension or defined benefit retirement plan sponsored by us during fiscal 2024.

  101. Nonqualified Deferred Compensation

    Nonqualified Deferred Compensation In June 2021, we adopted a non-qualified deferred compensation plan (Deferred Compensation Plan). Our Deferred Compensation Plan permits highly-compensated U.S. based employees, including our named executive officers, as well as non-employee members of the Board to defer up to 75% of their base salary and up to 100% of director compensation and other types of compensation, including annual cash bonuses, RSUs and PSUs awarded. Generally, a deferral election must be made no later than December 31 of the previous year and is irrevocable. Deferrals with respect to salary are deducted from the participant’s salary in equal installments for the period of January 1 to December 31 of each year. These deferral elections are for the salary earned by the participant for the particular salary pay period during that year, which would otherwise be payable to the participant in such pay period. The election to defer salary under the Deferred Compensation Plan is in addition to any deferral election made by the participant under our 401(k) Plan. Deferrals for performance-based annual bonuses are for those bonuses earned during the year for which the election applies, which are payable the following year. The Deferred Compensation Plan is intended to provide participants with a tax deferral opportunity for compensation paid by us. The deferred amounts are not subject to income tax or income tax withholding when earned and deferred, but are fully taxable (and withheld appropriately) when distributed. The Deferred Compensation Plan authorizes the Company to make matching contributions at our sole discretion. The participant is 100% vested at all times in his or her deferred cash account (including any company matching contributions), and deferrals of any compensation subject to vesting (such as RSUs and PSUs) shall vest in accordance with the provisions of the underlying award. No matching contributions were made by the Company in 2024. The Deferred Compensation Plan provides for distribution of deferred compensation and earnings thereon upon a participant’s separation from service with us, his or her retirement, a date specified by the participant in his or her compensation deferral agreement, the death of a participant (in such a case, to the designated beneficiary) or a “change in control.” Payment distributions can be made in a lum psum, annual installments of up to five years at the participant’s election or for “specified date accounts only”, installments of up to 15 years, at the participant’s election. The Deferred Compensation Plan credits gains and losses to deferred amounts based upon “deemed” investments in mutual funds investing in equity instruments or debt securities chosen by each participant (which the participant may change at any time) from a “menu” of fund options provided by us. The investment returns credited to participants’ accounts in the Deferred Compensation Plan correspond to actual returns of the chosen funds. The performance of the mutual funds fluctuates with the conditions of the capital markets and the economy generally, and is affected by prevailing interest rates and credit risks. The investment options under the Deferred Compensation Plan include:

  102. Nonqualified Deferred Compensation

    | Fund | 2024 Rate of Return (%) | | PIMCO VIT Short-Term Admin | 6.1 | | Vanguard VIF Total Bond Market Index | 1.2 | | Western Asset Core Plus Vit I | (0.4) | | DFA VIT Inflation-Protected Secs Instl. | 1.9 | | Empower Multi-Sector Bond Investor (MXLMX) | 5.1 | | American Funds IS® Capital World Bond 2 | (3.0) | | Vanguard VIF Equity Income | 15.1 | | Fidelity® VIP Index 500 Initial | 24.9 | | Invesco VI Equally Wtd S&P 500 I | 12.7 | | T. Rowe Price Blue Chip Growth Port | 35.5 | | American Century Vp Mid Cap Value I (AVIPX) | 8.7 | | Empower S&P Mid Cap 400® Index Inv (MXMDX) | 13.3 | | MFS® VIT Mid Cap Growth Init | 14.7 | | Dimensional VA US Targeted Value | 8.1 | | Empower S&P Smallcap 600® Index Inv (MXISX) | 7.9 | | Clearbridge Variable Small Cap Growth I (QLMSIX) | 4.5 | | Vanguard VIF Total Intl Stock Market Index | 5.1 | | Vanguard VIF International | 9.0 | | American Funds IS® New World 2 | 6.6 | | MFS® VIT III Global Real Estate Initial | (2.7) |

  103. Nonqualified Deferred Compensation

    For fiscal year 2024, other than Mr. Pinion, none of our named executive officers, participated in the Deferred Compensation Plan.

  104. Nonqualified Deferred Compensation

    | Name | Executive Contributions in Last FY ($) | Company Contributions in Last FY ($) | Aggregate Earnings in Last FY ($) | Aggregate Withdrawals/ Distributions ($) | Aggregate Balance at Last FYE ($) | | Emil D. Kakkis, M.D., Ph.D., President and Chief Executive Officer | — | — | — | — | — | | Howard Horn Chief Financial Officer, Corporate Strategy and Executive Vice President | — | — | — | — | — | | Erik Harris, Chief Commercial Officer and Executive Vice President | — | — | — | — | — | | Eric Crombez, M.D., Chief Medical Officer and Executive Vice President | — | — | — | — | — | | John R. Pinion II, Chief Quality Officer and Executive Vice President, Translational Sciences | 86,011(1) | — | 29,798 | — | 333,174 |

  105. Nonqualified Deferred Compensation

    | (1) Represents the value of 1,602 RSUs as of the applicable vesting date that were deferred into the Deferred Compensation Plan upon the partial vesting of such RSUs. |

  106. Potential Payments Upon Termination or Change of Control

    Potential Payments Upon Termination or Change of Control The amount of compensation and benefits payable to each named executive officer in various termination and change in control situations, as described above under “—Narrative Disclosure to Summary Compensation Table—Involuntary Termination of Employment” and “—Narrative Disclosure to Summary Compensation Table—Covered Transaction, has been estimated in the tables below. The tables below do not include the values of any amounts that a named executive officer may receive under the Deferred Plan as a result of a termination of employment or a change in control, as all amounts under the Deferred Plan are fully vested benefits. The value of the option, RSU, and PSU vesting acceleration was calculated for each of the tables below based on the assumption that the change in control and executive’s employment termination occurred on December 31, 2024. The closing price of our common stock on The Nasdaq Global Select Market as of December 31, 2024 was $42.07, which was used as the value of our common stock for purposes of the following tables. The value of the option vesting acceleration was calculated by multiplying the number of unvested option shares subject to vesting acceleration as of December 31, 2024 by the difference between the closing price of our common stock as of December 31, 2024 and the exercise price for such unvested option shares. No value is attributed to unvested options subject to acceleration which have exercise prices above the closing market price of our common stock as of December 31, 2024. The value of the RSU and PSU vesting acceleration was calculated by multiplying the number of unvested RSUs, earned but unvested PSUs and unearned PSUs (based on an assumed target level of performance) subject to vesting acceleration as of December 31, 2024 by the closing price of our common stock as of the last trading day of 2023. The value of COBRA reimbursements was calculated for each of the tables below based on the elections for each named executive officer in effect in December 2024 and the applicable COBRA premiums for December 2024.

  107. Dr. Emil Kakkis

    | Potential Payments Upon Termination or Change of Control | Qualifying Termination | Qualifying Termination following a Covered Transaction | | Base Salary | $1,722,000 | $1,722,000 | | Bonus | 688,800 | 1,377,600 | | Acceleration of equity awards | — | 15,586,430 | | COBRA reimbursements | 40,086 | 40,086 | | TOTAL | $2,450,886 | $18,726,116 |

  108. Howard Horn

    | Potential Payments Upon Termination or Change of Control | Qualifying Termination | Qualifying Termination following a Covered Transaction | | Base Salary | $590,000 | $885,000 | | Bonus | 295,000 | 442,500 | | Acceleration of equity awards | — | 4,803,835 | | COBRA reimbursements | 26,378 | 39,567 | | TOTAL | $911,378 | $6,170,902 |

  109. Erik Harris

    | Potential Payments Upon Termination or Change of Control | Qualifying Termination | Qualifying Termination following a Covered Transaction | | Base Salary | $607,000 | $910,500 | | Bonus | 303,500 | 455,250 | | Acceleration of equity awards | — | 3,750,414 | | COBRA reimbursements | 8,935 | 13,402 | | TOTAL | $919,435 | $5,129,566 |

  110. Eric Crombez, M.D.

    | Potential Payments Upon Termination or Change of Control | Qualifying Termination | Qualifying Termination following a Covered Transaction | | Base Salary | $604,000 | $906,000 | | Bonus | 302,000 | 453,000 | | Acceleration of equity awards | — | 3,040,735 | | COBRA reimbursements | 20,043 | 30,064 | | TOTAL | $926,043 | $4,429,799 |

  111. John R. Pinion II

    | Potential Payments Upon Termination or Change of Control | Qualifying Termination | Qualifying Termination following a Covered Transaction | | Base Salary | $572,000 | $858,000 | | Bonus | 286,000 | 429,000 | | Acceleration of equity awards | — | 3,615,790 | | COBRA reimbursements | 39,974 | 59,961 | | TOTAL | $897,974 | $4,962,751 |

  112. Director Compensation

    Director Compensation Our Board has adopted a non-employee director compensation policy that is designed to provide a total compensation package that enables us to attract and retain, on a long-term basis, high caliber non-employee directors. A Board compensation review prepared by Aon in February 2024 provided a competitive assessment of our compensation practices for non-employee directors in connection with the Compensation Committee’s evaluation of the level of compensation for our non-employee directors for 2024. A summary of the non-employee director cash compensation arrangements for fiscal 2024 is set forth below:

  113. Director Compensation

    | | Annual Retainer | | Board of Directors: | | | Chairman | $85,000 | | Non-Chairman members | $50,000 | | Audit Committee: | | | Chairman | $25,000 | | Non-Chairman members | $12,500 | | Compensation Committee: | | | Chairman | $20,000 | | Non-Chairman members | $10,000 | | Nominating and Corporate Governance Committee: | | | Chairman | $12,000 | | Non-Chairman members | $6,000 | | Research and Development Committee: | | | Chairman | $15,000 | | Non-Chairman members | $7,500 |

  114. Director Compensation

    In 2025, upon recommendation of the Compensation Committee, the Board maintained the annual cash compensation levels under the non-employee director compensation policy. Under the non-employee director compensation policy for fiscal 2024, each non-employee director who was initially appointed or elected to the Board received an equity award with a target value of $600,000, comprised 50% of options to purchase shares of our common stock and 50% of RSUs on the date he or she first becomes a non-employee director. The options vest monthly over a three-year period and the RSUs vest annually in equal amounts over a three-year period, in each case subject to the director’s continued service to us through each such vesting date. In addition, for fiscal year 2024, on the date of the annual meeting of stockholders, each continuing non-employee director received an annual equity award at a target value of $400,000, comprised of 50% options to purchase shares of our common stock and 50% RSUs, each of which would vest in full upon the earlier of (1) our subsequent annual meeting of stockholders and (2) the first anniversary of the date of grant, subject to the director’s continued service to us through such vesting date. The exercise price of all of the foregoing options was equal to the fair market value of a share of our common stock on the date of grant. In 2025, upon recommendation of the Compensation Committee, the Board maintained the target values of annual awards for non-employee directors and the target annual awards for newly appointed directors. Dr. Kakkis, our President and Chief Executive Officer, receives no compensation for his service as a director. The following table shows the compensation earned in fiscal 2024 by our non-employee directors.

  115. Director Compensation

    | Name | Fees Earned or Paid in Cash in Fiscal 2024 | Stock Awards(1) | Option Awards(2) | Total | | Daniel G. Welch | $100,750 | $200,010 | $199,374 | $500,134 | | Deborah Dunsire, M.D. | 67,375 | 200,010 | 199,374 | 466,759 | | Matthew K. Fust | 80,750 | 200,010 | 199,374 | 480,134 | | Michael Narachi | 82,500 | 200,010 | 199,374 | 481,884 | | Amrit Ray, M.D. | 64,750 | 200,010 | 199,374 | 464,134 | | Corsee D. Sanders, Ph.D. | 69,875 | 200,010 | 199,374 | 469,259 | | Shehnaaz Suliman, M.D. | 68,875 | 200,010 | 199,374 | 468,259 |

  116. Director Compensation

    | (1) The amounts reported in this column for a fiscal year represent the grant date fair value of the RSUs granted to our non-employee directors during fiscal 2024, as computed in accordance with ASC Topic 718, not including any estimates of forfeitures. The assumptions used in calculating the grant date fair value of the RSUs reported in this column are set forth in the notes to our financial statements included in our Annual Report. The amounts reported in this column reflect the accounting cost for these RSUs, and do not correspond to the actual economic value that may be received by the non-employee directors from the RSUs. As of December 31, 2024, each of our non-employee directors had 5,345 outstanding RSUs. | | (2) The amounts reported in this column represent the grant date fair value of the stock options granted to our non-employee directors during fiscal 2024, as computed in accordance with ASC Topic 718, not including any estimates of forfeitures. The assumptions used in calculating the grant date fair value of the stock options reported in this column are set forth in the notes to our financial statements included in our Annual Report. The amounts reported in this column reflect the accounting cost for these stock options, and do not correspond to the actual economic value that may be received by the non-employee directors from the options. As of December 31, 2024, our then non-employee directors had the following outstanding options: Mr. Welch – 74,130; Dr. Dunsire – 60,380; Mr. Fust – 56,630; Mr. Narachi – 74,130; Dr. Ray – 32,780; Dr. Sanders – 30,510; and Dr. Suliman – 56,630. |

  117. CEO Pay Ratio

    CEO Pay Ratio We are required by SEC rules adopted under the Dodd-Frank Act to disclose the ratio of our median employee’s annual total compensation to the annual total compensation of our principal executive officer. This disclosure provides a measure of the equitability of pay within our company. We believe our compensation philosophy and process yield an equitable result for all of our employees. For 2024, the annual total compensation for Dr. Emil Kakkis, our Chief Executive Officer and President, was $13,717,687 and for our median employee was $325,295, resulting in a pay ratio of 42:1. In accordance with Item 402(u) of Regulation S-K, we identified the median employee by (i) aggregating for each applicable employee (A) base salary for 2024 on the calculation date, (B) the target bonus for 2024, and (C) the accounting value of any equity awards granted during 2024, and (ii) ranking this annual compensation measure for our employees from lowest to highest. This calculation encompasses individuals, excluding our CEO, employed by us on October 1, 2024, whether employed on a full-time, part-time, or seasonal basis. For any permanent employees who were only employed for part of the 2024 fiscal year, we annualized their compensation to present a more accurate representation of their comparative annual compensation. On October 1, 2024, we had 1,291 employees. The total compensation of our identified median employee using the same methodology we use for our named executive officers as set forth in the Summary Compensation Table. The SEC’s rules for identifying the median employee and calculating the pay ratio based on that employee’s annual total compensation allow companies to adopt a variety of methodologies, to apply certain exclusions, and to make reasonable estimates and assumptions that reflect their employee populations and compensation practices. As a result, the pay ratio reported by other companies may not be comparable to the pay ratio reported above, as other companies have different employee populations and compensation practices and may utilize different methodologies, exclusions, estimates and assumptions in calculating their own pay ratios.

  118. Pay versus Performance

    Pay versus Performance As required by Section 953(a) of the Dodd-Frank Wall Street Reform and Consumer Protection Act, and Item 402(v) of Regulation S-K, we are providing the following information about the relationship between Compensation Actually Paid (CAP) and certain financial performance of the Company. For further information concerning the Company’s pay for performance philosophy and how the Company’s aligns executive compensation with the Company’s performance, refer to “Executive Compensation – Compensation Discussion and Analysis.”

  119. Pay versus Performance

    | | | | Average | | Value of Initial Fixed $100 Investment Based On: | | | | | Year | Summary Compensation Table Total for PEO(1) | Compensation Actually Paid to PEO(2) | Summary Compensation Table Total for Non-PEO NEOs(3) | Average Compensation Actually Paid to Non-PEO NEOs(4) | Total Shareholder Return(5) | Peer Group Total Shareholder Return(6) | Net Income (Loss) (in thousands)(7) | Revenue (in thousands)(8) | | 2024 | $13,717,687 | $8,186,261 | $4,254,959 | $2,790,240 | $99 | $114 | $(569,183) | $560,230 | | 2023 | $13,077,993 | $11,293,489 | $4,524,001 | $4,829,669 | $112 | $115 | $(606,639) | $434,249 | | 2022 | $12,140,330 | $3,311,432 | $3,911,773 | $(175,590) | $108 | $111 | $(707,421) | $363,329 | | 2021 | $9,522,738 | $(2,152,804) | $3,314,256 | $(1,211,638) | $197 | $125 | $(454,025) | $351,406 | | 2020 | $5,144,220 | $22,726,743 | $2,706,102 | $8,015,000 | $324 | $126 | $(186,566) | $271,030 |

  120. Pay versus Performance

    | (1) The dollar amounts reported are the amounts in the “Total” column of the Summary Compensation Table in each applicable year for Dr. Kakkis, who served as our Chief Executive Officer during 2020, 2021, 2022, 2023 and 2024. |

  121. Pay versus Performance

    | (2) The dollar amounts reported represent the amount of CAP, as computed in accordance with SEC rules. The dollar amounts do not reflect the actual amount of compensation earned by or paid during the applicable year. In accordance with SEC rules, the following adjustments were made to total compensation to determine the CAP: |

  122. Pay versus Performance

    | Compensation Actually Paid to PEO | 2024 | 2023 | 2022 | 2021 | 2020 | | Summary Compensation Table Total | $13,717,687 | $13,077,993 | $12,140,330 | $9,522,738 | $5,144,220 | | Less, value of “Stock Awards” and “Option Awards” reported in Summary Compensation Table | (12,090,111) | (11,618,444) | (10,773,976) | (8,246,092) | (3,709,589) | | Plus, year end fair value of outstanding and unvested equity awards granted during the year | 8,562,675 | 11,288,072 | (6,264,762) | 4,152,033 | 10,163,438 | | Plus (less), change in fair value from prior year end to current year end of outstanding and unvested equity awards granted in prior years | (2,033,745) | (1,167,400) | (2,934,552) | (5,753,632) | 9,346,926 | | Plus (less), change in fair value from prior year end to vesting date of equity awards granted in prior years that vested in the year | 29,755 | (286,732) | (1,385,132) | (1,827,851) | 1,781,748 | | Less, prior year-end fair value of any equity awards that failed to meet vesting conditions in the year | — | — | — | — | — | | Compensation Actually Paid to Dr. Kakkis | $8,186,261 | $11,293,489 | $3,311,432 | $(2,152,804) | $22,726,743 |

  123. Pay versus Performance

    | (3) The dollar amounts reported represent the average of the amounts reported for the Company’s named executive officers (NEOs) as a group (excluding our CEO) in the “Total” column of the Summary Compensation Table in each applicable year. The names of each of the NEOs (excluding our CEO) included for purposes of calculating the average amounts in each applicable year are as follows: (i) for 2024, Mr. Horn, Erik Harris, Dr. Crombez and Mr. Pinion, (ii) for 2023, Mr. Horn, Mr. Harris, Mr. Pinion and Ms. Parschauer, (iii) for 2022 and 2021, Dr. Camille Bedrosian, Mr. Harris, Mr. Pinion and Mardi Dier; and (iv) for 2020, Dr. Bedrosian, Ms. Dier, Mr. Harris, Mr. Pinion, and Shalini Sharp. |

  124. Pay versus Performance

    | (4) The dollar amounts reported represent the average amount of CAP to the NEOs as a group (excluding our CEO), as computed in accordance with SEC rules. The dollar amounts do not reflect the actual average amount of compensation earned by or paid to the NEOs as a group (excluding our CEO) during the applicable year. In accordance with the SEC rules, the following adjustments were made to average total compensation for the NEOs as a group (excluding our CEO) for each year to determine the compensation actually paid, using the same methodology described above in Note 2: |

  125. Pay versus Performance

    | Compensation Actually Paid to Non-PEO NEOs | 2024 | 2023 | 2022 | 2021 | 2020 | | Summary Compensation Table Total | $4,254,959 | $4,524,001 | $3,911,773 | $3,314,256 | $2,706,102 | | Less, value of “Stock Awards” and “Option Awards” reported in Summary Compensation Table | (3,293,798) | (3,831,408) | (3,130,468) | (2,519,639) | (2,027,088) | | Plus, year end fair value of outstanding and unvested equity awards granted during the year | 2,412,702 | 4,476,201 | 1,264,910 | 1,268,695 | 4,150,071 | | Plus (less), change in fair value from prior year end to current year end of outstanding and unvested equity awards granted in prior years | (607,133) | (237,537) | (784,234) | (2,359,539) | 2,748,610 | | Plus (less), change in fair value from prior year end to vesting date of equity awards granted in prior years that vested in the year | 23,510 | (101,588) | (573,020) | (915,411) | 437,305 | | Less, prior year-end fair value of any equity awards that failed to meet vesting conditions in the year | — | — | (864,551) | — | — | | Compensation Actually Paid to Non-PEO NEOs | $2,790,240 | $4,829,669 | $(175,590) | $(1,211,638) | $8,015,000 |

  126. Pay versus Performance

    | (5) Cumulative TSR is calculated by dividing (a) the sum of (i) the cumulative amount of dividends for the measurement period, assuming dividend reinvestment, and (ii) the difference between the Company’s share price at the end and the beginning of the measurement period by (b) the Company’s share price at the beginning of the measurement period. The beginning of the measurement period for each year in the table is December 31, 2019. |

  127. Pay versus Performance

    | (6) Represents the weighted peer group TSR, weighted according to the respective companies’ stock market capitalization at the beginning of each period for which a return is indicated. The peer group used for this purpose is the following published industry index: Nasdaq Biotechnology Index. |

  128. Pay versus Performance

    | (7) The dollar amounts reported represent the amount of net income calculated in accordance with GAAP reflected in the Company’s audited financial statements for the applicable year. |

  129. Pay versus Performance

    | (8) The dollar amounts reported represent the amount of revenue calculated in accordance with GAAP reflected in the Company’s audited financial statements for the applicable year. |

  130. Proposal No. 1 — Election of directors

    Proposal No. 1 — Election of directors Directors are elected by a plurality of the votes cast, with the two nominees obtaining the greatest number of affirmative votes being elected as directors. Shares as to which a stockholder withholds voting authority and broker non-votes, if any, are not considered votes cast and therefore will have no effect on the vote outcome. As described above under “Proposal No. 1 – Election of Class III Directors,” any nominee for director who receives a greater number of “withhold” votes for his or her election than votes “for” his or her election is expected to promptly tender his or her resignation to the Board following certification of the election results.