DEF 14A of Ultragenyx Pharmaceutical Inc., Apr 26, 2024

DEF 14A of Ultragenyx Pharmaceutical Inc.

Filed Apr 26, 2024 · FY20230001308179-24-000609

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 | | | | | | | | Deborah Dunsire, M.D. | 61 | Board member and advisor to various life science companies | 2017 | 2024 | II | | | Michael Narachi | 64 | Board member and advisor to various life science companies | 2015 | 2024 | II | | | Corsee D. Sanders, Ph.D. | 67 | Board member and advisor to various life science companies | 2021 | 2024 | II | | | Continuing Directors | | | | | | | | Matthew K. Fust | 59 | Board member and advisor to various life science companies | 2014 | 2025 | III | | | Amrit Ray, M.D. | 51 | Board member and advisor to various life science companies | 2022 | 2025 | III | | | Emil D. Kakkis, M.D., Ph.D. | 63 | President and Chief Executive Officer, Ultragenyx | 2010 | 2026 | I | | | Shehnaaz Suliman, M.D. | 52 | Chief Executive Officer of ReCode Therapeutics | 2019 | 2026 | I | | | Daniel G. Welch | 66 | Board member and advisor to various life science companies | 2015 | 2026 | I | |

  3. Board of Directors

    | Board Composition Highlights | | | | Age | Tenure | Diversity | | | | |

  4. Board Diversity

    Board Diversity Our Nominating and Corporate Governance Committee places significant emphasis on diversity and actively considers whether potential candidates would assist in achieving a mix of Board members that represents a diversity of talents, skills, backgrounds, and expertise. The current composition of our Board reflects the importance of diversity to the Board. Currently, three of our directors (38%) self-identify as Asian, one (13%) self-identifies as LGBTQ+ and three (38%) are women. We also have a female chairperson of our Nominating and Corporate Governance Committee. In accordance with Nasdaq’s board diversity listing standards, below is the aggregate statistical information about our Board’s self-identified gender and racial characteristics and LGBTQ+ status as voluntarily confirmed to us by each of our directors.

  5. Board Diversity

    | Board Diversity Matrix (as of the date of this Proxy Statement) | | | | | | Total Number of Directors: | 8 | | | | | | Female | Male | Non-Binary | Did not Disclose Gender | | Gender | 3 | 5 | — | — | | Number of Directors who identify in any of the categories below: | | | | | | African American or Black | — | — | — | — | | Alaskan Native or Native American | — | — | — | — | | Asian | 2 | 1 | — | — | | Hispanic or Latinx | — | — | — | — | | Native Hawaiian or Pacific Islander | — | — | — | — | | White | 1 | 4 | — | — | | Two or More Races or Ethnicities | — | — | — | — | | LGBTQ+ | — | 1 | — | — | | Did not disclose demographic background | — | — | — | — |

  6. Board Diversity, Skills and Experience

    Board Diversity, Skills and Experience Our Nominating and Corporate Governance Committee also considers numerous other skills, qualities and experiences when evaluating director nominees, 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. In response to stockholder feedback, we also provide below enhanced disclosure regarding the diversity of our Board.

  7. Board Diversity, Skills and Experience

    | | 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 | | | | | | | | | | Diversity | | | | | | | | | | Gender | | | | | | | | | | Male | | | | | | | | | | Female | | | | | | | | | | Race/Ethnicity | | | | | | | | | | Asian | | | | | | | | | | White | | | | | | | | | | LGBTQ+ | | | | | | | | |

  8. 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.

  9. 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 at least 90% of board and committee meetings of which the director was a member in 2023 | | • | 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 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 | | • | Corporate Governance Guidelines and robust Global Code of Conduct |

  10. Election of Class II Directors

    Election of Class II 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 II directors (whose terms expire at the Annual Meeting) are Deborah Dunsire M.D., Michael Narachi and Corsee D. Sanders, Ph.D. Dr. Dunsire and Mr. Narachi were most recently elected by stockholders at the 2021 Annual Meeting; Dr. Sanders was recommended by a third party search firm and appointed to the Board after the 2021 Annual Meeting. The Class III directors (whose terms expire at the 2025 Annual Meeting) are Matthew K. Fust and Amrit Ray, M.D. 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 elected at the Annual Meeting will hold office until the 2027 annual meeting of stockholders 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.

  11. 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 – Board Diversity, Skills and Experience”, the Nominating and Corporate Governance Committee also considers each potential nominee’s contribution to the Board’s diversity of talents, skills, backgrounds, including with respect to age, gender, national origin, sexual orientation and identification, race, ethnicity and culture, and expertise. In 2023, the Board adopted a “Rooney Rule” 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 includes women and minorities in the pool of candidates (and instructs any search firm the Nominating and Corporate Governance Committee engages to do so) and interviews at least one woman or one minority candidate. 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 titled “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”.

  12. 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.

  13. Class II Directors Nominated for Election

    Class II Directors Nominated for Election

  14. Class II Directors Nominated for Election

    | Deborah Dunsire, M.D. | | | Board member and advisor to various life science companies | | | Age: 61 Director since: 2017 INDEPENDENT Committees: 2 Other Public Directorships: 1 | 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 Millenium: The Takeda Oncology Company after the acquisition between 2008 and 2013. Prior to Millenium, Dr. Dunsire held various roles of increasing responsibility at Novartis Pharma AG between 1988 and 2005. Dr. Dunsire currently serves on the board of Syros Pharmaceuticals, a publicly traded biotechnology company, and previously served as a director of Allergan, Inc., a publicly traded pharmaceutical company, from December 2006 until April 2015 and a director of Alexion Pharmaceuticals Inc., a publicly traded biopharmaceutical 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. |

  15. Class II Directors Nominated for Election

    | Michael Narachi | | | Board member and advisor to various life science companies | | | Age: 64 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 an officer and 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 at Davis. He also holds an M.B.A. from the Anderson Graduate School of Management at 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. |

  16. Class II Directors Nominated for Election

    | Corsee D. Sanders, Ph.D. | | | Board member and adviser to various life science companies | | | Age: 67 Director since: 2021 INDEPENDENT Committees: 2 Other Public Directorships: 3 | Dr. Sanders currently serves as 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, and AltruBio Inc., a private biopharmaceutical 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. |

  17. Class III Directors Continuing in Office Until 2025

    Class III Directors Continuing in Office Until 2025

  18. Class III Directors Continuing in Office Until 2025

    | Matthew Fust | | | Board member and advisor to various life science companies | | | Age: 59 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 Sunesis Pharmaceuticals, Inc., a biotechnology company, from May 2005 to May 2017, the board 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. |

  19. Class III Directors Continuing in Office Until 2025

    | Amrit Ray, M.D. | | | Board member and advisor to various life science companies | | | Age: 51 Director since: 2022 INDEPENDENT Committees: 1 Other Public Directorships: 1 | Dr. Ray currently serves as a board member and advisor to life science companies. Previously, Dr. Ray served as Chief Patient Officer at Biohaven Pharmaceuticals, 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 on the board of directors of Fortrea, a publicly traded, global clinical research organization, and on the board of directors of several privately owned life science companies. 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. |

  20. Class I Directors Continuing in Office until 2026

    Class I Directors Continuing in Office until 2026

  21. Class I Directors Continuing in Office until 2026

    | Emil D. Kakkis, M.D., Ph.D. | | | Founder, President and CEO | | | Age: 63 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. |

  22. Class I Directors Continuing in Office until 2026

    | Shehnaaz Suliman, M.D., M.Phil., M.B.A | | | Chief Executive Officer of ReCode Therapeutics | | | Age: 52 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. |

  23. Class I Directors Continuing in Office until 2026

    | Daniel G. Welch | | | Board member and advisor to various life science companies | | | Age: 66 Director since: 2015 INDEPENDENT CHAIRMAN Committees: 2 Other Public Directorships: 3 | Mr. Welch is a board member and advisor to life science companies. Mr. Welch currently serves on the boards of Structure Therapeutics, Nuvation Bio Inc. and Prothena Corporation, each of which are publicly traded biotechnology companies. 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 AveXis, Inc., a publicly traded biotechnology company, from January 2016 through its acquisition in May 2018, and on the boards of directors of Intercept Pharmaceuticals, Inc from November 2015 to June 2021 and Seagen 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. |

  24. Class I Directors Continuing in Office until 2026

    The three nominees who receive the greatest number of affirmative votes will be elected as Class II 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.

  25. 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 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.

  26. Compensation Highlights

    | • | Modest base salary adjustments: Base salary increases for our current executives (excluding Mr. Horn who joined the Company in October 2023) were 3.5%. Salary increases were generally based on competitive market positioning and individual performance and were aligned to increases provided to all employees in good performance standing. | | • | Annual bonuses linked to pre-determined milestone performance goals and individual accomplishments. Our Chief Executive Officer received a below target bonus of 75% of his base salary based solely on corporate performance. Annual bonuses for our other NEOs ranged from 47% to 55% of their base salary when the impact of individual performance is factored in. | | • | Our equity mix included performance-based equity: We maintained our equity mix at one-third of each PSUs, RSUs and stock options for NEOs other than our Chief Executive Officer and for our Chief Executive Officer, we increased the percentage of PSUs to 60% for 2023 annual grants. | | • | PSU payouts linked to performance: Our relative TSR-based 2021 PSUs paid out below target, at 87%. We did not achieve the threshold level of performance under our revenue-based 2022 PSUs and as such, such PSUs were forfeited with no payout. | | • | Pay mix is highly “at risk”: The percentage of pay that is “at risk” for our CEO and NEOs (excluding Mr. Horn who joined us in October 2023) is 94% and 86%, respectively, helping us align pay with performance. |

  27. 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.

  28. 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 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 2023” and “– Fiscal 2024 Compensation – Equity Grants.” | | | | | We hold an annual say-on-pay vote for stockholders. | | |

  29. 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 2023, 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 $237,000. In addition, in 2023, management also engaged Aon to provide survey data relating to broad-based compensation matters. The aggregate cost of such other consulting services provided in 2023 by Aon and other affiliates of Aon (not related to Aon’s executive compensation, director compensation and equity consulting services provided to the Compensation Committee) was approximately $27,000. 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.

  30. 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 2023, 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:

  31. Defining and Comparing Compensation to Peer Group Benchmarks

    | • | companies with emphasis on orphan pharmaceutical products; | | • | companies with comparable market capitalizations (i.e., in the range of $2 billion to $16 billion); | | • | companies with revenue of between $200 million and $1 billion; and | | • | companies with headcounts between 300 to 3,000 employees. |

  32. Defining and Comparing Compensation to Peer Group Benchmarks

    Our 2023 peer group is comprised of the following 21 companies in the pharmaceutical and biotechnology industries, reflecting the addition of Alkermes plc, Apellis Pharmaceuticals, Inc., BioCryst Pharmaceuticals, Inc., Corcept Therapeutics Inc. and the removal of Acceleron Pharma, Inc., Agios Pharmaceuticals and Nektar Therapeutics from our 2022 peer group.

  33. Defining and Comparing Compensation to Peer Group Benchmarks

    | ACADIA Pharmaceuticals Inc. | Biomarin Pharmaceutical Inc. | Insmed Incorporated | | Alkermes plc | Blueprint Medicines Corporation | Ionis Pharmaceuticals, Inc. | | Alnylam Pharmaceuticals Inc. | Corcept Therapeutics Inc. | Jazz Pharmaceuticals plc | | Amicus Therapeutics | Exelixis Inc. | Neurocrine Biosciences, Inc. | | Apellis Pharmaceuticals, Inc. | FibroGen, Inc. | PTC Therapeutics, Inc. | | BioCryst Pharmaceuticals, Inc. | Global Blood Therapeutics, Inc. | Sarepta Therapeutics, Inc. | | Biohaven Pharmaceutical Holding Company Ltd. | Halozyme Therapeutics | United Therapeutics Corporation |

  34. Defining and Comparing Compensation to Peer Group Benchmarks

    We believe that the compensation practices of our 2023 peer group provided us with appropriate benchmarks for evaluating the compensation of our named executive officers for 2023 because of the developmental, market and organizational characteristics we shared with our peer group. At the time that we selected our 2023 peer group we were at approximately the 40th percentile of the peer group in terms of market capitalization, the 79th percentile in terms of employees headcount and the 31st percentile in terms of revenue.

  35. 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.

  36. 2023 Base Salaries

    2023 Base Salaries The Compensation Committee engaged Aon to conduct a competitive review and analysis of our current executive compensation program relative to our 2023 peer group. Aon prepared an Executive Compensation Assessment report in December 2022 that provided a competitive assessment of our executive compensation program as compared to the 2023 peer group data for base salaries, target total cash compensation, and equity compensation. For 2023, increases in base salaries for our named executive officers, were 3.5% annualized, excluding Mr. Horn who joined the Company in October 2023. Mr. Horn’s base salary was established in connection with his appointment. The overall 2023 merit budget was based on an Aon trend report regarding projected market merit spends for 2023. Individual increases in base salaries were also based on achievement of 2022 individual goals. The following table shows the increases in base salaries for our named executive officers between fiscal 2022 and fiscal 2023. Mr. Horn joined the Company in October 2023 and as such, did not have a fiscal 2022 base salary.

  37. Annual Bonus

    Annual Bonus

  38. Overview

    Overview Our annual incentive program provides an opportunity for cash bonus awards based upon the attainment of annual performance goals. For all executive officers, except the Chief Executive Officer, the goals relate to both corporate and individual performance. Corporate performance goals include business, financial, and operational measures or objectives. Individual performance goals focus on individual contributions that drive achievement of the corporate goals and provide leadership for the executive officers’ respective functions. The Chief Executive Officer’s bonus is based solely on corporate performance. The individual goals are adopted at the beginning of each performance year by the Chief Executive Officer and communicated to each executive officer. The Compensation Committee considers the individual performance of each executive officer (other than our Chief Executive Officer) and our overall corporate performance for the preceding fiscal year in deciding whether to award a bonus and, if one is to be awarded, the amount of the bonus. All executive officers are assigned annual bonus targets, expressed as a percent of base salary, based on each executive officer’s accountability, scope of responsibilities, and potential impact on performance, as well as peer group competitive data for similarly situated positions. In 2023, the Company modified its bonus structure for all employees, including executive officers other than the Chief Executive Officer, such that the bonus payout for employees (excluding the Chief Executive Officer) is based on the employee’s annual target bonus percentage multiplied by the corporate performance achievement multiplied by the individual performance achievement. Prior to the change, the annual bonus was weighted 75% for corporate performance and 25% for individual performance for executives other than the Chief Executive Officer. The annual bonus for the Chief Executive Officer continues to be weighted 100% for corporate performance. With respect to the corporate component of the bonus, the maximum payout is 150% of target. With respect to the individual component of the bonus, the minimum payout is 0% and the maximum payout is 133% of the Company achievement. Actual payouts are based upon achievement with respect to established goals (for corporate performance) and for performance in three categories (for individual performance) – achievement of individual goals, key deliverables of the role and demonstration of the Company’s values and behaviors. Generally, after the end of each fiscal year, the Compensation Committee assesses corporate performance, and determines an overall percentage of goal achievement, for such year. This corporate performance score determines the size of the bonus pool applicable to the corporate component of the bonus plan. A score at or below 50% results in the corporate component of the bonus pool not being funded. 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.

  39. Fiscal 2023 Bonuses

    Fiscal 2023 Bonuses Annual corporate goals for fiscal 2023 were proposed by our executive officers and approved by our Compensation Committee in early 2022. Individual objectives for our executive officers for 2023 were proposed by each executive officer, with review, input and confirmation from our Chief Executive Officer. The target bonuses, as a percentage of base salary, for the named executive officers for fiscal 2023 are set forth in the following table. The target bonus for Dr. Kakkis increased from 75% of base salary in 2022 to 80% of base salary in 2023 to align with market practices.

  40. Fiscal 2023 Bonuses

    | Name | Target Bonus for Fiscal 2023 (% of Base Salary) | | Emil D. Kakkis, M.D., Ph.D. | 80 | | Howard Horn* | 50 | | Erik Harris | 50 | | John R. Pinion II | 50 | | Karah Parschauer | 50 |

  41. Fiscal 2023 Bonuses

    | * As Mr. Horn commenced employment with the Company after September 30, 2023, he was not eligible to receive an annual bonus for fiscal year 2023. |

  42. Fiscal 2023 Bonuses

    In February 2024, the Compensation Committee assessed our overall 2023 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 2023 fiscal year:

  43. Fiscal 2023 Bonuses

    | Goal | Weighting | Achievement Against Goal | Weighted Achievement | | 1. Commercial Products • Achieve targets for global revenue in our territories and number of patients on products in target territories | 35% | 91% | 32% | | 2. Research & Development • Achieve enrollment objectives for pivotal trials for specified product candidates | 52% | 89% | 46% | | 3. Gene Therapy Manufacturing • Complete engineering runs and GMP runs for drug product and drug supply | 5% | 150% | 8% | | 4. General Corporate • Maintaining operating cash usage within 5% of budget • Retaining over 80% of employees rated as “meets expectations” or above • Achieve threshold scores in annual employee engagement survey: overall engagement score of 80%+, inclusion and diversity score of 75%+ and employee well-being score of 75%+ • Execute all goals while maintaining ethical and compliant culture with no adverse regulatory actions | 8.5% | 50% | 4% |

  44. Fiscal 2023 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 2023 corporate goals, including the successful analyst day hosted by the Company in October 2023, completion of a financing transaction and successful execution of a complex clinical pipeline which involved foregoing achievement on some corporate goals to prioritize the Company’s key clinical programs. For all goals combined, including additional achievements by the Company not previously defined, the Compensation Committee determined an overall 94% achievement for fiscal year 2023. In February 2024, 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 (other than Mr. Horn) 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.

  45. Fiscal 2023 Bonuses

    | Named Executive Officer | Key 2023 Achievements | | Erik Harris Chief Commercial Officer and EVP | • Achieved continued growth in product sales to support achievement of global revenue targets • Achieved operating margin and operating expenses for global commercial team within specified variance • Executed commercial planning for launch readiness for pipeline programs • Submission of initial country dossiers for Evkeeza in specified countries | | John R. Pinion II Chief Quality Officer and EVP, Translational Sciences | • Led and sponsored non-clinical activities to enable development and advancement of translational science portfolio • Successfully executed IND enabling studies for specified pre-clinical programs • Sponsored and hosted successful R&D day • Leadership support for Company’s planned spinout of the Alzheimer’s program • Executive sponsor for Company’s DEIB action committee | | Karah Parschauer Chief Legal Officer & Corporate Affairs and EVP | • Successfully managed and/or resolved litigation and investigations • Supported successful analyst day and subsequent financing • Assumed additional responsibilities with patient advocacy team • Successful execution of government affairs matters related to newborn screening, state biomarker testing and other rare disease legislative initiatives |

  46. Fiscal 2023 Bonuses

    Dr. Kakkis evaluated the performance of Mr. Harris, Mr. Pinion and Ms. Parschauer 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 2023.

  47. Achievement of Goals and Relationship to Compensation Awarded

    | Name | Title | Corporate Component Score (100% for CEO) | Individual Component Score | Total Fiscal 2023 Bonus | Bonus Achieved (as % of Base Salary) | | Emil D. Kakkis, M.D., Ph.D. | President and Chief Executive Officer | 94 | — | $622,700 | 75% | | Erik Harris | Chief Commercial Officer and Executive Vice President | 94 | 101 | $279,600 | 47% | | John R. Pinion II | Chief Quality Officer and Executive Vice President of Translational Sciences | 94 | 113 | $294,800 | 53% | | Karah Parschauer | Chief Legal Officer & Corporate Affairs and Executive Vice President | 94 | 118 | $298,400 | 55% |

  48. Equity Compensation

    Equity Compensation

  49. 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 Incentive 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. We do not have any program, plan or obligation that requires us to grant equity awards on specified dates, although we make annual equity grants in or around March of each year to allow management and the Compensation Committee to review all elements of compensation at the same point in the year. We also do not have any program, plan or practice to time award dates of stock option grants to our executive officers in coordination with the release of material nonpublic information and typically our annual grants occur during an open trading window. Equity awards may occasionally be granted following a significant change in job responsibilities or to meet special retention or performance objectives. 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.

  50. Annual Equity Grants in Fiscal 2023

    Annual Equity Grants in Fiscal 2023 In March 2023, 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, and Mr. Horn, who received new hire grants of options and RSUs upon the commencement of his employment. 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 2023 (2023 PSUs) consist of a revenue portion (50% of the PSUs) with a two-year performance period, a relative total stockholder return (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 revenue-based 2023 PSUs will vest based upon achievement of revenue-based targets 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. 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 2023 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, 2023 and ending December 31, 2025, 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, 2026. The relative TSR-based PSUs will be earned as follows based on our percentile ranking among the Nasdaq Biotechnology Index during the performance period:

  51. Annual Equity Grants in Fiscal 2023

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

  52. Annual Equity Grants in Fiscal 2023

    The strategic performance based 2023 PSUs will vest 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. 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 2023 PSUs for below threshold performance.

  53. Annual Equity Grants in Fiscal 2023

    | 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% |

  54. Annual Equity Grants in Fiscal 2023

    The table below sets forth all equity awards granted in fiscal 2023 to our named executive officers.

  55. Annual Equity Grants in Fiscal 2023

    | Name | Date of Grant | Number of Options | Number of RSUs | Target Number of PSUs | | Emil D. Kakkis, M.D., Ph.D. | 3/1/2023 | 83,500 | 46,200 | 138,700 | | Howard Horn(1) | 10/9/2023 | 140,469 | 80,336 | — | | Erik Harris | 3/1/2023 | 39,400 | 22,000 | 22,000 | | John R. Pinion II | 3/1/2023 | 39,400 | 22,000 | 22,000 | | Karah Parschauer | 3/1/2023 | 39,400 | 22,000 | 22,000 |

  56. Annual Equity Grants in Fiscal 2023

    | (1) Mr. Horn received a new hire sign-on equity award consisting of options and RSUs upon commencement of his employment with the Company. |

  57. 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). The revenue-based 2022 PSUs vested based upon achievement of the following revenue-based targets (and application of linear interpolation) during the period beginning January 1, 2022 and ending December 31, 2023.

  58. Performance of 2022 PSUs

    | Level of Performance | Aggregate Revenue | Earned Revenue-Based PSU Percentage | | Actual | $798M | 0% | | Threshold | $815M | 50% | | Target | $907M | 100% | | Maximum | $1,075M | 200% |

  59. Performance of 2022 PSUs

    In February 2024, based on the Company’s 2022 and 2023 revenue performance measured in accordance with U.S. 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 performance period for the relative TSR-based 2022 PSUs extends through December 31, 2024, and if earned, such portion will vest in early 2025.

  60. Performance of 2021 PSUs

    Performance of 2021 PSUs The PSU awards granted to our executive officers in March 2021 (2021 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 2023, based on the Company’s 2021 and 2022 revenue performance measured in accordance with U.S. GAAP, the Compensation Committee certified that the revenue-based 2021 PSUs vested at 60.5% of target, and such earned 2021 PSUs vested on March 1, 2023, as described in our 2023 proxy statement. The relative TSR-based 2021 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, 2021 and ending December 31, 2023, 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, 2024. The relative TSR-based PSUs was earned as follows based on our percentile ranking among the Nasdaq Biotechnology Index during the performance period:

  61. Performance of 2021 PSUs

    | Level of Performance | TSR Percentile | Earned TSR PSUs | | Threshold | 25th | 25% | | Actual | 46th | 87% | | Target | 50th | 100% | | Stretch | 75th | 150% | | Maximum | 90th | 200% |

  62. Performance of 2021 PSUs

    Based on the Company’s ranking relative to the TSR of the companies in the Nasdaq Biotechnology Index of the 46th percentile during the performance period, the Compensation Committee certified on March 1, 2024 that the relative TSR-based 2021 PSUs were earned at 87% of target, and such earned 2021 PSUs vested on such certification date.

  63. 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 2024, referred to as our 2024 peer group, by balancing the following criteria:

  64. Peer Group

    | • | companies with emphasis on orphan pharmaceutical products; | | • | companies with comparable market capitalizations (i.e., in the range of $2 billion to $16 billion); | | • | companies with revenue of between $200 million and $1.4 billion; and | | • | companies with headcounts between 300 to 4,000 employees. |

  65. Peer Group

    Our 2024 peer group is comprised of the following 20 companies in the pharmaceutical and biotechnology industries, reflecting the addition of BridgeBio Pharma, Inc. Intra-Cellular Therapies Inc. and Sage Therapeutics, Inc. and the removal of Alnylam Pharmaceuticals Inc., Biohaven Pharmaceutical Holding Company, Ltd., Global Blood Therapeutics, Inc. and United Therapeutics Corporation from our 2023 peer group.

  66. Peer Group

    | 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. | |

  67. Peer Group

    We believe that the compensation practices of our 2024 peer group provided us with appropriate compensation benchmarks for evaluating the compensation of our named executive officers for 2024.

  68. Annual Bonuses

    Annual Bonuses For 2024, 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 other significant changes were made to the bonus plan for 2024.

  69. Equity Compensation

    Equity Compensation For 2024, the equity grants to our executive officers, other than our Chief Executive Officer, continued to reflect an equal value split among options, RSUs and PSUs. 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 2024 (2024 PSUs) consist of a revenue portion with a two-year performance period (1/3 of the 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 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 relative TSR-based 2024 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, 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 strategic-based 2024 PSU awards will vest based on achievement of specified strategic goals during the period beginning January 1, 2024 and ending December 31, 2026, 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, 2027. 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.

  70. Risk Management and Mitigation

    Risk Management and Mitigation In reviewing the compensation structure in fiscal 2023, 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 2023, our corporate goals were reviewed and approved by the Board in early 2023, 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.

  71. Summary Compensation Table

    Summary Compensation Table The following table sets forth the compensation earned during the years ended December 31, 2023, 2022, and 2021 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.

  72. Summary Compensation Table

    | Name and Principal Position | Year | Salary | Bonus(1) | Stock Awards(2) | Option Awards(3) | Non-Equity Incentive Plan Compensation(4) | All Other Compensation(5) | Total | | Emil D. Kakkis, M.D., Ph.D. President and Chief Executive Officer | 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 | | | 2021 | 768,846 | — | 5,410,512 | 2,835,580 | 499,100 | 8,700 | 9,522,738 | | Howard Horn(6) Chief Financial Officer, Corporate Strategy and Executive Vice President | 2023 | 124,808 | 60,000 | 2,867,459 | 2,888,773 | — | — | 5,941,040 | | Erik Harris Chief Commercial Officer and Executive Vice President | 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 | | | 2021 | 543,846 | — | 1,653,212 | 866,427 | 245,025 | 8,700 | 3,317,210 | | John R. Pinion II, Chief Quality Officer and Executive Vice President, Translational Sciences | 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 | | | 2021 | 509,939 | — | 1,653,212 | 866,427 | 224,942 | 8,700 | 3,263,220 | | Karah Parschauer Chief Legal Officer & Corporate Affairs and Executive Vice President | 2023 | 535,169 | — | 2,169,750 | 1,020,050 | 298,375 | 13,200 | 4,036,544 |

  73. Summary Compensation Table

    | (1) The amount reported in this column represents a sign-on bonus under Mr. Horn’s offer letter. | | (2) 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 2023, assuming achievement of the maximum performance level, was as follows: $14,695,265 for Dr. Kakkis and $2,330,900 for each Mr. Harris, Mr. Pinion and Ms. Parschauer. Mr. Horn was not granted any PSUs in 2023. | | (3) 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. 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 named executive officers from the options. | | (4) 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. | | (5) Amounts reported in this column for the 2023 fiscal year reflects 401(k) matching contributions. | | (6) Mr. Horn joined the Company as Chief Financial Officer, Corporate Strategy and Executive Vice President in October 2023. |

  74. 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 2023.

  75. 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 or Units Granted (#)(3) | Option Awards: Number of Securities Underlying Options Granted (#) | Exercise Price of Option Awards ($/Share) | Grant Date Fair Value of Stock and Option Awards ($)(4) | | Name | Grant Date | Threshold ($) | Target ($) | Maximum ($) | Threshold (#) | Target (#) | Maximum (#) | | | | | | Emil D. Kakkis, M.D., Ph.D. | | 331,200 | 662,400 | 993,600 | — | — | — | — | — | — | — | | | 3/1/2023 | — | — | — | — | — | — | — | 83,500 | 45.65 | 2,161,782 | | | 3/1/2023 | — | — | — | — | — | — | 46,200 | — | — | 2,109,030 | | | 3/1/2023 | — | — | — | 60,681 | 138,700 | 277,400 | — | — | — | 7,347,633 | | Howard Horn(5) | | | | | | | | | | | | | | 10/9/2023 | — | — | — | — | — | — | — | 140,469 | 35.68 | 2,888,773 | | | 10/9/2023 | — | — | — | — | — | — | 80,366 | — | — | 2,867,459 | | Erik Harris | | — | 294,600 | 587,727 | — | — | — | — | — | — | — | | | 3/1/2023 | — | — | — | — | — | — | — | 39,400 | 45.65 | 1,020,050 | | | 3/1/2023 | — | — | — | — | — | — | 22,000 | — | — | 1,004,300 | | | 3/1/2023 | — | — | — | 9,625 | 22,000 | 44,000 | — | — | — | 1,165,450 | | John R. Pinion II | | — | 277,500 | 553,613 | — | — | — | — | — | — | — | | | 3/1/2023 | — | — | — | — | — | — | — | 39,400 | 45.65 | 1,020,050 | | | 3/1/2023 | — | — | — | — | — | — | 22,000 | — | — | 1,004,300 | | | 3/1/2023 | — | — | — | 9,625 | 22,000 | 44,000 | — | — | — | 1,165,450 | | Karah Parschauer | | — | 269,000 | 536,655 | — | — | — | — | — | — | — | | | 3/1/2023 | — | — | — | — | — | — | — | 39,400 | 45.65 | 1,020,050 | | | 3/1/2023 | — | — | — | — | — | — | 22,000 | — | — | 1,004,300 | | | 3/1/2023 | — | — | — | 9,625 | 22,000 | 44,000 | — | — | — | 1,165,450 |

  76. 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 2023 annual incentive program as established by the Board and described in “Compensation Discussion and Analysis” above. Actual payments made for fiscal 2023 are provided in the Summary Compensation Table. | | (2) The amounts in these columns represent the threshold, target and maximum level of achievement for the 2023 PSUs granted under our 2014 Incentive Plan. The PSU awards consist of a revenue portion (50% of the PSUs) with a two-year performance period, a relative total stockholder return (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. See ” – 2023 Annual Equity Grants in Fiscal 2023” above for a description of the 2023 PSUs. | | (3) The amounts in this column represents the RSUs granted under our 2014 Incentive Plan and our 2023 Incentive Plan during 2023. | | (4) This column reflects the aggregate grant date fair value of equity awards granted in 2023 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 12 to our financial statements included in our Annual Report for fiscal 2023. | | (5) Represents the new hire option and RSU awards made to Mr. Horn upon his commencement of employment with the Company. As Mr. Horn commenced employment with the Company after September 30, 2023, he was not eligible to receive a cash payment under the 2023 annual non-equity incentive program. |

  77. Outstanding Equity Awards at December 31, 2023

    Outstanding Equity Awards at December 31, 2023 The following table sets forth information concerning the outstanding equity awards held by each of the named executive officers as of December 31, 2023.

  78. Outstanding Equity Awards at December 31, 2023

    | | | Option Awards(1) | | | | Stock Awards | | | | | Name | Grant Date | Number of Securities Underlying Unexercised Options (#) Exercisable | Number of Securities Underlying Unexercised Options (#) Unexercisable | Option Exercise Price ($) | Option Expiration Date | Number of Shares or Units of Stock That Have Not Vested (#)(2) | Market Value of Shares or Units of Stock That Have Not Vested ($) | Equity Incentive Plan Awards: Number of Unearned Shares, Units or Other Rights That Have Not Vested (#)(3) | 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. | 3/1/2023 | — | 83,500 | 45.65 | 3/1/2033 | 46,200 | 2,209,284 | 138,700 | 6,632,634 | | | 3/1/2022 | 40,408 | 51,952 | 67.37 | 3/1/2032 | 39,030 | 1,866,415 | 10,408 | 497,711 | | | 3/1/2021 | 24,750 | 11,250 | 142.47 | 3/1/2031 | 12,132 | 580,152 | — | — | | | 3/1/2020 | 52,594 | 3,506 | 56.08 | 3/1/2030 | 4,250 | 203,235 | — | — | | | 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 | 10/9/2023 | — | 140,469 | 35.68 | 10/9/2033 | 80,366 | 3,843,102 | — | — | | Erik Harris | 3/1/2023 | — | 39,400 | 45.65 | 3/1/2033 | 22,000 | 1,052,040 | 22,000 | 1,052,040 | | | 3/1/2022 | 10,894 | 14,006 | 67.37 | 3/1/2032 | 10,500 | 502,110 | 2,800 | 133,896 | | | 3/1/2021 | 7,563 | 3,437 | 142.47 | 3/1/2031 | 3,707 | 177,269 | — | — | | | 3/1/2020 | 20,625 | 1,375 | 56.08 | 3/1/2030 | 1,950 | 93,249 | — | — | | | 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 | — | — | — | — | | John R. Pinion II | 3/1/2023 | — | 39,400 | 45.65 | 3/1/2033 | 22,000 | 1,052,040 | 22,000 | 1,052,040 | | | 3/1/2022 | 10,894 | 14,006 | 67.37 | 3/1/2032 | 10,500 | 502,110 | 2,800 | 133,896 | | | 3/1/2021 | 7,563 | 3,437 | 142.47 | 3/1/2031 | 3,707 | 177,269 | — | — | | | 3/1/2020 | 20,625 | 1,375 | 56.08 | 3/1/2030 | 1,950 | 93,249 | — | — | | | 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 | — | — | — | — | | Karah Parschauer | 3/1/2023 | — | 39,400 | 45.65 | 3/1/2033 | 22,000 | 1,052,040 | 22,000 | 1,052,040 | | | 3/1/2022 | 10,894 | 14,006 | 67.37 | 3/1/2032 | 10,500 | 502,110 | 2,800 | 133,896 | | | 3/1/2021 | 7,563 | 3,437 | 142.47 | 3/1/2031 | 3,707 | 177,269 | — | — | | | 3/1/2020 | 20,625 | 1,375 | 56.08 | 3/1/2030 | 1,950 | 93,249 | — | — | | | 3/1/2019 | 21,500 | — | 67.55 | 3/1/2029 | — | — | — | — | | | 3/1/2018 | 17,000 | — | 48.43 | 3/1/2028 | — | — | — | — | | | 3/1/2017 | 12,000 | — | 88.80 | 3/1/2027 | — | — | — | — | | | 6/20/2016 | 17,664 | — | 54.50 | 6/20/2026 | — | — | — | — |

  79. Outstanding Equity Awards at December 31, 2023

    | (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) 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, 3,132 of the total earned PSUs listed in the March 1, 2021 row vested on March 1, 2024 and for (ii) each of Mr. Harris, Mr. Pinion and Ms. Parschauer, 957 of the total earned PSUs listed in the March 1, 2021 row vested on March 1, 2024. Please see the section entitled “—Narrative Disclosure to Summary Compensation Table—Covered Transaction” for accelerated vesting provisions that apply on certain terminations of employment. | | (3) 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. |

  80. Option Exercises and Stock Vested

    Option Exercises and Stock Vested The following table sets forth certain information concerning the option awards exercised and stock awards vested for our named executive officers during fiscal 2023.

  81. Option Exercises and Stock Vested

    | | Option Awards | | Stock Awards | | | Name | Number of Shares Acquired on Exercise (#) | Value Realized on Exercise ($)(1) | Number of Shares Acquired on Vesting (#) | Value Realized on Vesting ($)(2) | | Emil D. Kakkis, M.D., Ph.D. | 47,853 | 1,272,193 | 35,477 | 1,619,525 | | Howard Horn | — | — | — | — | | Erik Harris | — | — | 11,363 | 521,022 | | John R. Pinion II | — | — | 14,488 | 661,377 | | Karah Parschauer | — | — | 11,238 | 513,015 |

  82. Option Exercises and Stock Vested

    | (1) Value realized does not represent proceeds from any sale of any common stock acquired upon exercise, but is determined by multiplying the number of shares acquired upon exercise by the difference between the exercise price of the option and the closing price of our common stock on The Nasdaq Global Select Market on each exercise date. | | (2) 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 stock awards that vested. |

  83. 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 2023.

  84. 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 2023. 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 lump sum, 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:

  85. Nonqualified Deferred Compensation

    | Fund | 2023 Rate of Return (%) | | PIMCO VIT Short-Term Admin | 5.9 | | Vanguard VIF Total Bond Market Index | 5.6 | | Western Asset Core Plus Vit I | 6.8 | | DFA VIT Inflation-Protected Secs Instl. | 4.0 | | Empower Multi-Sector Bond Investor (MXLMX) | 7.9 | | American Funds IS® Capital World Bond 2 | 6.1 | | Vanguard VIF Equity Income | 8.1 | | Fidelity® VIP Index 500 Initial | 26.1 | | Invesco VI Equally Wtd S&P 500 I | 13.7 | | T. Rowe Price Blue Chip Growth Port | 49.3 | | American Century Vp Mid Cap Value I (AVIPX) | 6.1 | | Empower S&P Mid Cap 400® Index Inv (MXMDX) | 15.8 | | MFS® VIT Mid Cap Growth Init | 21.3 | | DFA VA US Targeted Value | 20.0 | | Empower S&P Smallcap 600® Index Inv (MXISX) | 15.5 | | Clearbridge Variable Small Cap Growth I (QLMSIX) | 8.4 | | Vanguard VIF Total Intl Stock Market Index | 15.5 | | Vanguard VIF International | 14.7 | | American Funds IS® New World 2 | 16.0 | | MFS® VIT III Global Real Estate Initial | 11.5 |

  86. Nonqualified Deferred Compensation

    For fiscal year 2023, other than Mr. Pinion, none of our named executive officers participated in the Deferred Compensation Plan.

  87. 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 | — | — | — | — | — | | John R. Pinion II, Chief Quality Officer and Executive Vice President, Translational Sciences | 183,239(1) | — | 34,125 | — | 217,365 | | Karah Parschauer, Chief Legal Officer & Corporate Affairs and Executive Vice President | — | — | — | — | — |

  88. Nonqualified Deferred Compensation

    | (1) Represents the value of 4,014 RSUs as of the applicable vesting date that were deferred into the Deferred Compensation Plan upon the partial vesting of such RSUs. |

  89. 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, 2023. The closing price of our common stock on The Nasdaq Global Select Market as of December 29, 2023, the last trading day of 2023, was $47.82, 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, 2023 by the difference between the closing price of our common stock as of December 31, 2023 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, 2023. 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, 2023 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 2023 and the applicable COBRA premiums for December 2023.

  90. Dr. Emil Kakkis

    | Potential Payments Upon Termination or Change of Control | Qualifying Termination | Qualifying Termination following a Covered Transaction | | Base Salary | $1,656,000 | $1,656,000 | | Bonus | 662,400 | 1,324,800 | | Acceleration of equity awards | — | 12,170,625 | | COBRA reimbursements | 55,969 | 55,969 | | TOTAL | $2,374,369 | $15,207,394 |

  91. 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 | — | 5,548,396 | | COBRA reimbursements | 405 | 607 | | TOTAL | $885,405 | $6,876,503 |

  92. Erik Harris

    | Potential Payments Upon Termination or Change of Control | Qualifying Termination | Qualifying Termination following a Covered Transaction | | Base Salary | $589,000 | $883,500 | | Bonus | 294,500 | 441,750 | | Acceleration of equity awards | — | 3,096,102 | | COBRA reimbursements | 8,621 | 12,931 | | TOTAL | $892,121 | $4,434,283 |

  93. John R. Pinion II

    | Potential Payments Upon Termination or Change of Control | Qualifying Termination | Qualifying Termination following a Covered Transaction | | Base Salary | $555,000 | $832,500 | | Bonus | 277,500 | 416,250 | | Acceleration of equity awards | — | 3,096,102 | | COBRA reimbursements | 38,552 | 57,828 | | TOTAL | $871,052 | $4,402,680 |

  94. Karah Parschauer

    | Potential Payments Upon Termination or Change of Control | Qualifying Termination | Qualifying Termination following a Covered Transaction | | Base Salary | $538,000 | $807,000 | | Bonus | 269,000 | 403,500 | | Acceleration of equity awards | — | 3,096,102 | | COBRA reimbursements | 27,985 | 41,977 | | TOTAL | $834,985 | $4,348,579 |

  95. 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 2023 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 2023. A summary of the non-employee director cash compensation arrangements for fiscal 2023 is set forth below:

  96. 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 | $10,000 | | Non-Chairman members | $5,000 | | Research and Development Committee: | | | Chairman | $14,000 | | Non-Chairman members | $7,000 |

  97. Director Compensation

    In 2024, upon recommendation of the Compensation Committee, the Board increased the annual cash compensation for certain committee members as follows: from $10,000 to $12,000 for the Chairperson of the Nominating & Corporate Governance Committee, from $5,000 to $6,000 for the other members of the Nominating & Corporate Governance Committee, from $14,000 to $15,000 for the Chairman of the Research and Development Committee and from $7,000 to $7,500 for the other members of the Research and Development Committee. Under the non-employee director compensation policy for fiscal 2023, each non-employee director who was initially appointed or elected to the Board received an equity award with a target value of $800,000, 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 2023, 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 2024, upon recommendation of the Compensation Committee, the Board maintained the target values of annual awards for non-employee directors but decreased the target values of awards for newly appointed directors from $800,000 to $600,000. 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 2023 by our non-employee directors.

  98. Director Compensation

    | Name | Fees Earned in Fiscal 2023 | Stock Awards(1) | Option Awards(2) | Total | | Daniel G. Welch | $100,000 | $200,025 | $199,867 | $499,892 | | Deborah Dunsire, M.D. | 60,000 | 200,025 | 199,867 | 459,892 | | Lars Ekman, M.D., Ph.D.(3) | 32,000 | — | — | 32,000 | | Matthew K. Fust | 80,000 | 200,025 | 199,867 | 479,892 | | Michael Narachi | 82,500 | 200,025 | 199,867 | 482,392 | | Amrit Ray, M.D. | 60,500 | 200,025 | 199,867 | 460,392 | | Corsee D. Sanders, Ph.D. | 69,500 | 200,025 | 199,867 | 469,392 | | Shehnaaz Suliman, M.D. | 67,000 | 200,025 | 199,867 | 466,892 |

  99. 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 the fiscal year, 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, 2023, our then non-employee directors had the following outstanding RSUs: Mr. Welch – 3,860; Dr. Dunsire – 3,860; Dr. Ekman - 0; Mr. Fust – 3,860; Mr. Narachi – 3,860; Dr. Ray – 7,273; Dr. Sanders – 5,158; and Dr. Suliman – 3,860. | | (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 2023, 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, 2023, our then non-employee directors had the following outstanding options: Mr. Welch – 64,230; Dr. Dunsire – 50,480; Dr. Ekman - 0; Mr. Fust – 46,730; Mr. Narachi – 64,230; Dr. Ray – 22,880; Dr. Sanders – 20,610; and Dr. Suliman – 46,730. | | (3) Dr. Ekman retired from the Board effective immediately after the 2023 Annual Meeting on June 7, 2023. |

  100. 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 2023, the annual total compensation for Dr. Emil Kakkis, our Chief Executive Officer and President, was $13,077,993 and for our median employee was $353,878, resulting in a pay ratio of 37:1. 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 2023 on the calculation date, (B) the target bonus for 2023, and (C) the accounting value of any equity awards granted during 2023, 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, 2023, whether employed on a full-time, part-time, or seasonal basis. For any permanent employees who were only employed for part of the 2023 fiscal year, we annualized their compensation to present a more accurate representation of their comparative annual compensation. On October 1, 2023, we had 1,300 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.

  101. 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.”

  102. Pay versus Performance

    | | | | Average Summary Compensation Table Total for Non-PEO NEOs(3) | | Value of Initial Fixed $100 Investment Based On: | | | | | Year | Summary Compensation Table Total for PEO(1) | Compensation Actually Paid to PEO(2) | | 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) | | 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 |

  103. 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 and 2023. |

  104. 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: |

  105. Pay versus Performance

    | Compensation Actually Paid to PEO | 2023 | 2022 | 2021 | 2020 | | Summary Compensation Table Total | $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 | (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 | | (6,264,762) | 4,152,033 | 10,163,438 | | Plus (less), year over year change in fair value of outstanding and unvested equity awards granted in prior years | (1,167,400) | (2,934,552) | (5,753,632) | 9,346,926 | | Plus (less), year over year change in fair value of equity awards granted in prior years that vested in the year | (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 | $11,293,489 | $3,311,432 | $(2,152,804) | $22,726,743 |

  106. 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 2023, Mr. Horn, Mr. Harris, Mr. Pinion and Ms. Parschauer, (ii) for 2022 and 2021, Dr. Camille Bedrosian, Mr. Harris, Mr. Pinion and Mardi Dier; and (ii) for 2020, Dr. Bedrosian, Ms. Dier, Mr. Harris, Mr. Pinion, and Shalini Sharp. |

  107. 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: |

  108. Pay versus Performance

    | Compensation Actually Paid to Non-PEO NEOs | 2023 | 2022 | 2021 | 2020 | | Summary Compensation Table Total | $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 | (3,831,408) | (3,130,468) | (2,519,639) | (2,027,088) | | Plus, year end fair value of outstanding and unvested equity awards granted during the year | 4,476,201 | 1,264,910 | 1,268,695 | 4,150,071 | | Plus (less), year over year change in fair value of outstanding and unvested equity awards granted in prior years | (237,537) | (784,234) | (2,359,539) | 2,748,610 | | Plus (less), year over year change in fair value of equity awards granted in prior years that vested in the year | (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 | $4,829,669 | $(175,590) | $(1,211,638) | $8,015,000 |

  109. 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. | | (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. | | (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. | | (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. |

  110. Proposal No. 1 — Election of directors

    Proposal No. 1 — Election of directors Directors are elected by a plurality of the votes cast, with the three 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 II 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 must promptly tender his or her resignation to the Board following certification of the election results.