UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

SCHEDULE 14A

Proxy Statement Pursuant to Section 14(a) of the Securities
Exchange Act of 1934 (Amendment No.)

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ULTRAGENYX PHARMACEUTICAL INC.

(Name of Registrant as Specified In Its Charter)

(Name of Person(s) Filing Proxy Statement, if other than the Registrant)

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60 Leveroni Court
Novato, California 94949

Notice of Annual Meeting of Stockholders

We are pleased to make use of the U.S. Securities and Exchange Commission (SEC) rules that allow companies to furnish proxy materials to their stockholders via the Internet. We believe the ability to deliver proxy materials electronically allows us to provide our stockholders with the information they need, while lowering the costs of delivery and reducing the environmental impact from the distribution of our Annual Meeting materials.

We look forward to speaking with you at the Annual Meeting.

Sincerely,

Emil D. Kakkis, M.D., Ph.D.

President and Chief Executive Officer

April 26, 2024

WHETHER OR NOT YOU EXPECT TO ATTEND THE ANNUAL MEETING, PLEASE VOTE VIA THE INTERNET AS INSTRUCTED IN THE NOTICE OF INTERNET AVAILABILITY OR, IF YOU REQUESTED AND RECEIVED A PRINTED COPY OF THE PROXY MATERIALS, COMPLETE, DATE, SIGN, AND RETURN THE ENCLOSED PROXY CARD USING THE ENCLOSED RETURN ENVELOPE OR VOTING INSTRUCTION FORM PROVIDED WITH THE PRINTED PROXY MATERIALS, AS PROMPTLY AS POSSIBLE SO THAT YOUR SHARES MAY BE REPRESENTED AT THE ANNUAL MEETING. YOU MAY ALSO VOTE AT THE VIRTUAL ANNUAL MEETING

VOTING METHODS
If you are an owner of record as of the record date, you may vote via any of the following methods:		INTERNET Visit the website at: www.proxyvote.com		TELEPHONE If you requested and received a proxy card, call toll-free at 1-800-690-6903		MAIL If you requested and received a proxy card, sign, date and mail the proxy card in the enclosed envelope		AT THE MEETING: Vote at the meeting by going to: www.virtualshareholdermeeting.com/RARE2024

If you are a beneficial owner of shares held through a broker, bank or other owner of record, you must follow the voting instructions you receive from the owner of record to vote your shares.

Proxy Statement Overview

This overview highlights certain information contained elsewhere in this Proxy Statement and does not contain all of the information that you should consider. You should read the entire Proxy Statement carefully before voting. For more complete information regarding our business and 2023 performance, please review our 2023 Annual Report on Form 10-K as filed with the U.S. Securities and Exchange Commission (SEC) in February 2024.

Meeting and Voting Information

Meeting Date and Meeting Time		Record Date		Location
June 18, 2024		April 23, 2024		Live via audio webcast at
9:30 a.m. Pacific Time				www.virtualshareholdermeeting.com/RARE2024

We intend to mail the Notice Regarding the Availability of Proxy Materials containing instructions on how to access this Proxy Statement and our 2023 Annual Report on Form 10-K beginning on or about April 26, 2024 to all stockholders entitled to vote at the Annual Meeting.

Business Highlights

In 2023, we generated significant momentum across our clinical and commercial programs to position our company for what we expect to be a catalyst-rich 2024.

Our key financial and operational accomplishments in 2023 included:

ULTRAGENYX PHARMACEUTICAL INC. • 2024 Proxy Statement     4

Program Highlights and Other Achievements

We hosted a Company Analyst Day in October 2023 where we announced meaningful updates and data from three of our key clinical programs: UX143 (setrusumab) monoclonal antibody for the treatment of Osteogenesis Imperfecta, GTX-102 antisense oligonucleotide (ASO) for the treatment of Angelman syndrome and UX701 for the treatment of Wilson Disease. We also continued to make important progress on our other clinical pipeline programs.

See page 40 for more information about our achievements in 2023.

Corporate Responsibility Highlights

At Ultragenyx, we are committed to bringing novel products to patients for the treatment of rare and ultrarare diseases, with a focus on serious, debilitating genetic diseases. Our purpose is to lead the future of rare disease medicine as we seek to treat individuals afflicted by diseases with limited or no treatment options. We recognize that their lives and well-being are dependent upon our efforts to develop new therapies. For this reason, we are passionate about developing these therapies with the utmost urgency and care. For more information about our Corporate Responsibility efforts and initiatives, please see our Corporate Responsibility Report for fiscal year 2023 (2023 Corporate Responsibility Report), which will be available on our website at www.ultragenyx.com under “Ultra-Committed – Corporate Responsibility”. Website references throughout this document are provided for convenience only, and the content on the referenced websites is not incorporated by reference into this document.

See page 31 for more information about our Corporate Responsibility highlights in 2023.

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.

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

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Board Composition Highlights
Age		Tenure		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.

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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.

	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+

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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.

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PROPOSAL 1

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.

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”.

ULTRAGENYX PHARMACEUTICAL INC. • 2024 Proxy Statement     9

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.

Class II Directors Nominated for Election

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Class III Directors Continuing in Office Until 2025

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Class I Directors Continuing in Office until 2026

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Vote Required

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.

Director Resignation Policy

We have a Director Resignation Policy, which is set forth in our Corporate Governance Guidelines, a copy of which is available on our website in the “Corporate Governance” subsection of the “Investors” tab. The policy establishes that any director nominee who receives more “withhold” votes than “for” votes in an uncontested election of directors is required to tender his or her resignation promptly following the certification of the election results. Broker non-votes, if any, are not counted as either a “withhold” or “for” vote.

The Nominating and Corporate Governance Committee will promptly consider the tendered resignation and make a recommendation to the Board. The Board will act on the recommendation of the Nominating and Corporate Governance Committee no later than 90 days following the certification of the election results. The Board will promptly publicly disclose its decision and, if applicable, the reasons for rejecting the tendered resignation.

THE BOARD OF DIRECTORS RECOMMENDS A VOTE “FOR” EACH OF THE DIRECTOR NOMINEES IDENTIFIED ABOVE.

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PROPOSAL 2

Approval of the Ultragenyx Pharmaceutical Inc. Amended and Restated 2023 Incentive Plan

On April 20, 2024, the Board approved an amendment and restatement of the Ultragenyx Pharmaceutical Inc. 2023 Incentive Plan (the 2023 Plan and as amended and restated, the A&R 2023 Plan), subject to stockholder approval at the Annual Meeting. The A&R 2023 Plan provides for an additional 4.0 million shares of our common stock to be available for issuance thereunder. We believe that our equity compensation policies enable us to attract and retain a highly-skilled team of executives and aligns our executives’ interests with those of our stockholders by rewarding short-term and long-term performance and tying compensation to increases in stockholder value. If the A&R 2023 Plan is approved by our stockholders, we intend to file a Form S-8 with the SEC following the Annual Meeting during the second or third quarter that covers the additional shares reserved for issuance under the A&R 2023 Plan.

Share Reserve Under the A&R 2023 Plan

The maximum number of shares of our common stock authorized for issuance under the A&R 2023 Plan is (i) 8.5 million shares plus (ii) the number of shares subject to any award outstanding under the 2014 Incentive Plan (2014 Plan) or the 2011 Incentive Plan (2011 Plan and together with the 2014 Plan, the Prior Plans) as of June 7, 2023 that after June 7, 2023 are not issued because such award is forfeited, canceled, terminates, expires or otherwise lapses without being exercised, or is settled in cash. Based on the recent range of our stock price, our current compensation practices, our anticipated future awards, as well as our three-year burn rate, we believe the A&R 2023 Plan share reserve will be sufficient for us to grant equity awards for approximately one year based on our stock price and compensation philosophy and policies.

As part of the Compensation Committee’s recommendation to the Board to approve the A&R 2023 Plan and the additional shares available for issuance under the A&R 2023 Plan, the Compensation Committee considered advice from Aon, its independent compensation consultant. The Compensation Committee also carefully analyzed our historical burn rate, anticipated future equity award needs to help drive our long-term strategic plan, and the dilutive impact of the A&R 2023 Plan’s share reserve.

Reasons for Seeking Stockholder Approval

We believe the following are important considerations for stockholders in determining whether to approve the A&R 2023 Plan:

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Key Features and Governance Best Practices

The A&R 2023 Plan reflects a number of provisions that protect stockholders and reflect corporate governance best practices, including the following:

Shares Remaining Under the Prior Plans

As of March 28, 2024, a total of 83,094,037 shares of our common stock were outstanding and the fair market value of our common stock was $46.69 based on the closing sale price of our common stock on the Nasdaq Global Select Market as of March 28, 2024. The following table sets forth information regarding outstanding equity awards and shares available for future equity awards under the 2023 Plan (without giving effect to approval of the A&R 2023 Plan), the Prior Plans, our Employment Inducement Plan (the “Inducement Plan), the Dimension Therapeutics, Inc. 2015 Stock Option and Incentive Plan (the “DT 2015 Plan), the Dimension Therapeutics, Inc. 2013 Stock Plan (the “DT 2013 Plan”), and the shares available for future equity awards under our Amended and Restated 2014 Employee Stock Purchase Plan (ESPP) as of March 28, 2024.

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Dilution

Dilution is commonly measured by “overhang,” which generally refers to the total number of equity awards outstanding plus the total number of shares available for grant under our equity plans, divided by the sum of the total common stock outstanding, the number of equity awards outstanding and the total number of shares available for grant under our equity plans. If the A&R 2023 Plan is approved, our overhang will be approximately 22% as of March 28, 2024; however, many of the stock options attributing to our overhang are underwater. As shown above, we had approximately 11,255,498 stock options outstanding, with a weighted average exercise price of $65.84 as of March 28, 2024. That included 6,322,256 options currently exercisable, with a weighted average exercise price of $71.44, and 4,933,242 options not yet vested, with a weighted average exercise price of $58.66. That compares to a closing stock price on March 28, 2024, of $46.69. The 9,155,384 stock options currently underwater account for 9% of our overhang if the A&R 2023 Plan is approved.

Historical Burn Rate

Our equity plan share usage over 2021, 2022 and 2023 represented a three-year average burn rate of 4.95%, as described in the table below.

Summary of the A&R 2023 Plan

The following summary describes the material terms of the A&R 2023 Plan. This summary of the A&R 2023 Plan is not a complete description of all provisions of the A&R 2023 Plan and is qualified in its entirety by reference to the A&R 2023 Plan, which is attached hereto as Appendix A. Stockholders are encouraged to read the A&R 2023 Plan in its entirety.

Purpose

The purpose of the A&R 2023 Plan is to advance the Company’s interests by providing for the grant to participants of equity and other incentive awards.

Plan Administration

The Compensation Committee serves as the primary administrator of the A&R 2023 Plan, except that the Compensation Committee may, subject to applicable law, delegate authority to one or more members of the Board or one or more of our officers or employees (the Compensation Committee or such delegee, the Plan Administrator).

The Plan Administrator has the authority to, among other things, (i) interpret and construe the A&R 2023 Plan, any rules and regulations under the A&R 2023 Plan and the terms and conditions of any award granted under the A&R 2023 Plan, (ii) determine eligibility for and grant awards under the A&R 2023 Plan, (iii) determine, modify or waive the terms and conditions of awards under the A&R 2023 Plan, (iv) prescribe forms, rules and procedures relating to the A&R 2023 Plan, (v) establish and verify the extent of satisfaction of any performance goals or other conditions applicable to the grant, issuance, retention, vesting, exercisability or settlement of any award under the A&R 2023 Plan, and (vi) otherwise do all things necessary or appropriate to carry out the purposes of the A&R 2023 Plan. The Plan Administrator’s determinations under the A&R 2023 Plan are conclusive and binding.

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Authorized Shares

Subject to adjustment as described below, the maximum number of shares of our common stock that may be delivered in satisfaction of awards under the A&R 2023 Plan is (i) 8.5 million, plus (ii) the number of shares of common stock subject to any award under a Prior Plan as of June 7, 2023 that become available as a result of the termination, cancellation or forfeiture of such awards under such Prior Plan after June 7, 2023.

Shares of our common stock to be issued under the A&R 2023 Plan may be authorized but unissued shares of our common stock or previously issued shares acquired by us. If any shares of our common stock underlying awards that are settled in cash, canceled, forfeited or otherwise expire or lapse without being exercised (to the extent applicable), the shares of common stock allocable to the terminated portion of such award will again be available for issuance under the A&R 2023 Plan.

However, notwithstanding the foregoing, the following shares of common stock will not be available for issuance under the Plan: (a) shares withheld from an award under the A&R 2023 Plan or a Prior Plan to satisfy the tax withholding obligations with respect to such award, (b) shares withheld from an award under the A&R 2023 Plan or a Prior Plan in payment of the exercise price of an award requiring exercise, (c) shares repurchased on the open market by us using proceeds from the exercise price paid with respect to awards under the A&R 2023 Plan or a Prior Plan, or (d) gross shares subject to an SAR granted under the A&R 2023 Plan or a Prior Plan that are not issued in connection with the stock-settlement of such SAR.

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.

Eligibility

The Plan Administrator will select participants from among our, and our affiliates’, executives, employees in good performance standing, directors, consultants and advisors who are in a position to contribute significantly to our success and the success of our affiliates. Eligibility for options intended to be incentive stock options, or ISOs, is limited to our employees or the employees of certain of our affiliates. As of March 28, 2024, there were approximately 1,269 employees (including eight executive officers) and seven non-employee directors who would be eligible to participate in the A&R 2023 Plan. As of March 28, 2024, no consultants or advisors would be eligible to participate in the A&R 2023 Plan.

Awards Under the A&R 2023 Plan

The A&R 2023 Plan provides for grants of stock options, SARs, restricted and unrestricted stock, stock units (including RSUs), performance awards, cash awards and other awards convertible into or otherwise based on shares of our common stock. Dividend equivalents may also be provided in connection with an award under the A&R 2023 Plan (other than stock options and SARs), provided that such dividend equivalents will be subject to the same limits or restrictions as the awards to which they relate, and will not be payable until such awards vest.

Stock Options

A stock option is an award that entitles the participant to receive, upon exercise, shares of our common stock upon payment of the exercise price. The exercise price of an option may not be less than the fair market value (or, in the case of an ISO granted to a ten percent shareholder, 110% of the fair market value) of a share of our common stock on the date of grant. The Plan Administrator will determine the time or times at which stock options become exercisable and the terms on which they remain exercisable. The maximum term of a stock option is 10 years (or, in the case of an ISO granted to a ten percent shareholder, five years). Reload stock options are prohibited under the A&R 2023 Plan.

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SARs

SARs entitle the participant to receive, upon exercise, shares of our common stock or cash equal to the excess of the value of the shares subject to the SAR over the exercise price. The exercise price of an SAR may not be less than the fair market value of a share of our common stock on the date of grant. The Plan Administrator will determine the time or times at which SARs become exercisable and the terms on which they remain exercisable. The maximum term of an SAR is 10 years.

Restricted and Unrestricted Stock

A restricted stock award is an award of our common stock subject to restrictions requiring that it be redelivered or offered for sale to the Company if specified conditions are not satisfied, while an unrestricted stock award is an award of our common stock that is not subject to any restrictions. The Plan Administrator will determine the terms of awards of restricted and unrestricted stock.

Stock Units

A stock unit award is denominated in shares of our common stock and entitles the participant to receive stock or cash measured by the value of the shares in the future. An RSU is a stock unit award that is subject to the satisfaction of performance conditions or other vesting conditions. The Plan Administrator will determine the terms of awards of stock units, including RSUs.

Performance Awards

A performance award is an award under the A&R 2023 Plan where the vesting, settlement or exercisability is subject to specified performance criteria.

Cash Awards

Cash awards are awards under the A&R 2023 Plan that are denominated in cash.

Termination of Employment

The Plan Administrator will determine the effect of termination of employment or service on an award under the A&R 2023 Plan. Unless otherwise expressly provided by the Plan Administrator, upon a termination of a participant’s employment all unvested options then held by the participant and other awards requiring exercise will terminate and all other unvested awards will be forfeited and all vested stock options and SARs then held by the participant will remain outstanding for three months, or one year in the case of death, or, in each case, until the applicable expiration date, if earlier. All stock options and SARs held by a participant immediately prior to the participant’s termination of employment will immediately terminate upon termination of employment if the termination is for cause or occurs in circumstances that, in the determination of the Plan Administrator, would have constituted grounds for the participant’s employment to be terminated for cause.

Transferability

Awards under the A&R 2023 Plan may not be transferred except by will or by the laws of descent and distribution. For awards other than ISOs, the Plan Administrator may permit the transfer not for value to any transferee eligible to be covered by the provisions of Form S-8.

Clawback; Recovery of Compensation

Awards granted under the A&R 2023 Plan are subject to forfeiture, termination and rescission, and a participant will be obligated to return to us the value received with respect to awards, to the extent provided by the Plan Administrator in an award agreement, pursuant to Company policy relating to the recovery of erroneously-paid incentive compensation (including our Clawback Policy), or as otherwise required by law or applicable stock exchange listing standards.

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Covered Transaction

In the event of a covered transaction (as defined in the A&R 2023 Plan), the Plan Administrator may, among other things, provide for (i) continuation or assumption of outstanding awards, (ii) new grants in substitution of outstanding awards, (iii) the accelerated vesting or delivery of shares under awards or for a cash-out of outstanding awards, in each case on such terms and with such restrictions as it deems appropriate. However, if the award is not continued or assumed or a new grant is not substituted for the award, then stock options and SARs will become fully exercisable and all other awards shall become vested (with any performance based on target or actual performance as determined by the Plan Administrator).

Prohibition Against Repricing

We will not, without stockholder approval (except in the case of a change in our capitalization, as described below), (i) reduce the exercise price of a stock option or SAR, (ii) other than in the case of covered transaction, at any time when the exercise price of a stock option or SAR is above the fair market value of a share of stock, cancel and re-grant or exchange such stock option or SAR for cash or a new award having a lower (or no) exercise price, or (iii) take any other action with respect to an award that would be treated as a repricing under generally accepted accounting principles.

Adjustment

In the event of a stock dividend, stock split or combination of shares including a reverse stock split, recapitalization or other change in our capital structure that constitutes an equity restructuring within the meaning of the Financial Accounting Standards Board, Accounting Standards Codification Topic 718, Compensation — Stock Compensation, the Plan Administrator will make appropriate adjustments to the maximum number of shares that may be delivered under the A&R 2023 Plan, and will also make appropriate adjustments to the number and kind of shares of stock or securities subject to awards, the exercise prices of such awards or any other terms of awards affected by such change. The Plan Administrator will also make the types of adjustments described above to take into account distributions and other events other than those listed above if it determines that such adjustments are appropriate to avoid distortion and preserve the value of awards.

Amendment and Termination

The Plan Administrator will be able to amend the A&R 2023 Plan or outstanding awards for any purpose which may at the time be permitted by law, or terminate the A&R 2023 Plan as to future grants of awards, except that the Plan Administrator will not be able to alter the terms of an award if it would affect materially and adversely a participant’s rights under the award without the participant’s consent (unless expressly provided in the A&R 2023 Plan or the right to alter the terms of an award was expressly reserved by the Plan Administrator at the time the award was granted). Stockholder approval will be required for any amendment to the A&R 2023 Plan to the extent such approval is required by law, including the Internal Revenue Code of 1986, as amended (the “Code”), or applicable stock exchange requirements. No awards may be made under the A&R 2023 Plan after 10 years from June 7, 2023.

New Plan Benefits

As described above, the selection of participants who will receive awards under the A&R 2023 Plan and the size and types of awards will be determined by the Plan Administrator in its discretion. Therefore, the amount of any future awards under the A&R 2023 Plan is not yet determinable and it is not possible to predict the benefits or amounts that will be received by, or allocated to, particular individuals or groups of employees.

Awards Granted Under the 2023 Plan

No awards made under the 2023 Plan prior to the date of the Annual Meeting were granted subject to stockholder approval of the A&R 2023 Plan. Pursuant to SEC rules, the following table sets forth information with respect to awards that have been granted under the 2023 Plan to the groups named below as of March 28, 2024, with PSUs based on achievement of target performance. No associate of any director, executive officer or director nominee has received awards under the 2023 Plan, and no person has received more than 5% of all awards under the 2023 Plan.

ULTRAGENYX PHARMACEUTICAL INC. • 2024 Proxy Statement     19

U.S. Federal Income Tax Consequences

The following is a summary of the U.S. federal income tax treatment applicable to us and the participants who receive awards under the A&R 2023 Plan based on the federal income tax laws in effect on the date of this Proxy Statement. This summary is not intended to be exhaustive and does not address all matters relevant to a particular participant based on their specific circumstances. The summary expressly does not discuss the income tax laws of any state, municipality, or non-U.S. taxing jurisdiction, or the gift, estate, excise (including the rules applicable to deferred compensation under Section 409A of the Code), or other tax laws other than U.S. federal income tax law. Because individual circumstances may vary, we recommend that all participants consult their own tax advisor concerning the tax implications of awards granted under the A&R 2023 Plan.

Stock Option Grants

Stock options granted under the A&R 2023 Plan may be either ISOs, which satisfy the requirements of Section 422 of the Code, or non-statutory stock options (NSOs), which are not intended to meet such requirements. The U.S. federal income tax treatment for the two types of options differs as follows:

Incentive Stock Options

No taxable income is recognized by the participant at the time of the grant of an ISO, and no taxable income is recognized for ordinary income tax purposes at the time the ISO is exercised, although taxable income may arise at that time for alternative minimum tax purposes. Unless there is a disqualifying disposition, as described below, the participant will recognize long-term capital gain in an amount equal to the excess of (i) the amount realized upon the sale or other disposition of the purchased shares over (ii) the exercise price paid for the shares.

A disqualifying disposition occurs if the disposition is less than two years after the date of grant or less than one year after the exercise date. If there is a disqualifying disposition of the shares, then the excess of (i) the fair market value of those shares on the exercise date or (if less) the amount realized upon such sale or disposition over (ii) the exercise price paid for the shares will be taxable as ordinary income to the participant. Any additional gain or loss recognized upon the disposition will be a capital gain or loss.

If the participant makes a disqualifying disposition of the purchased shares, then we will be entitled to an income tax deduction, for the taxable year in which such disposition occurs, equal to the amount of ordinary income recognized by the participant as a result of the disposition. We will not be entitled to any income tax deduction if the participant makes a qualifying disposition of the shares.

Non-Statutory Stock Options

No taxable income is recognized by a participant upon the grant of an NSO. The participant in general will recognize ordinary income, in the year in which the NSO is exercised, equal to the excess of the fair market value of the purchased shares on the exercise date over the exercise price paid for the shares, and the participant will be required to satisfy the tax withholding requirements applicable to such income. We will be entitled to an income tax deduction equal to the amount of ordinary income recognized by the participant with respect to the exercised NSO.

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SARs

No taxable income is recognized upon receipt of an SAR. The holder will recognize ordinary income in the year in which the SAR is exercised, in an amount equal to the excess of (i) the fair market value of the underlying shares of common stock on the exercise date over (ii) the base price in effect for the exercised right, and the holder will be required to satisfy the tax withholding requirements applicable to such income. We will be entitled to an income tax deduction equal to the amount of ordinary income recognized by the holder in connection with the exercise of the SAR.

Stock Awards

Participants will recognize ordinary income at the time unrestricted stock awards are granted in an amount equal to the excess of (i) the fair market value of the shares on the grant date over (ii) the cash consideration (if any) paid for the shares. The holder will be required to satisfy the tax withholding requirements applicable to the income.

No taxable income is recognized at the time restricted stock awards are issued, but the participant will have to report as ordinary income, as and when those shares subsequently vest, an amount equal to the excess of (i) the fair market value of the shares on the vesting date over (ii) the cash consideration (if any) paid for the shares. The participant may, however, elect under Section 83(b) of the Code to include as ordinary income in the year the unvested shares are issued an amount equal to the excess of (a) the fair market value of those shares on the issue date over (b) the cash consideration (if any) paid for such shares. If the Section 83(b) election is made, the participant will not recognize any additional income as and when the shares subsequently vest.

We will be entitled to an income tax deduction equal to the amount of ordinary income recognized by the participant at the time such ordinary income is recognized by the participant.

Other Awards

Generally, no taxable income is recognized upon receipt of stock units (including RSUs), performance awards or cash awards. The holder will recognize ordinary income in the year in which the shares subject to the award are actually issued or in the year in which the award is settled in cash. The amount of that income will be equal to the fair market value of the shares on the date of issuance or the amount of the cash paid in settlement of the award, and the holder will be required to satisfy the tax withholding requirements applicable to the income.

We will be entitled to an income tax deduction equal to the amount of ordinary income recognized by the holder at the time the shares are issued or the cash amount is paid.

Deductibility of Executive Compensation

Section 162(m) of the Code limits the deductibility for federal income tax purposes of certain compensation paid to any “covered employee” in excess of $1 million. For purposes of Section 162(m), the term “covered employee” includes any individual who serves as chief executive officer, chief financial officer or one of the other three most highly compensated executive officers for 2017 or any subsequent calendar year. It is expected that compensation deductions for any covered employee with respect to awards under the A&R 2023 Plan will be subject to the $1 million annual deduction limitation.

Vote Required

Approval of the A&R 2023 Plan requires the affirmative vote of a majority of the votes cast on this proposal. Because abstentions and broker non-votes, if any, are not counted as votes cast for or against this resolution, they will have no effect on the outcome of the vote.

THE BOARD OF DIRECTORS RECOMMENDS A VOTE “FOR” PROPOSAL NO. 2.

ULTRAGENYX PHARMACEUTICAL INC. • 2024 Proxy Statement     21

PROPOSAL 3

Ratification of the Selection of Independent Registered Public Accounting Firm

Our Audit Committee has selected Ernst & Young LLP as our independent registered public accounting firm for the fiscal year ending December 31, 2024, and has further directed that we submit the selection of Ernst & Young LLP for ratification by our stockholders at the Annual Meeting.

We are not required to submit the selection of our independent registered public accounting firm for stockholder approval, but are submitting our selection of Ernst & Young LLP for stockholder ratification as a matter of good corporate governance. If the stockholders do not ratify this selection, the Audit Committee will reconsider its selection of Ernst & Young LLP. Even if the selection is ratified, our Audit Committee may direct the appointment of a different independent registered public accounting firm at any time during the year if the Audit Committee determines that the change would be in our best interests.

The Audit Committee reviews and pre-approves all audit and non-audit services performed by our independent registered public accounting firm. The Audit Committee may delegate its pre-approval authority to one or more of its members and has delegated such authority to the Chairman of the Audit Committee; any pre-approval decisions made by the Chairman are reported to the Audit Committee at the next scheduled committee meeting. The Audit Committee may pre-approve specified audit-related services (assurance and related services that are reasonably related to the performance of the audit or review of our financial statements and that are traditionally performed by the independent auditor) as well as specified tax services that the Audit Committee believes would not impair the independence of the independent auditor, and that are consistent with rules on auditor independence established by the SEC and the Public Company Accounting Oversight Board (PCAOB). The Audit Committee may also pre-approve those permissible non-audit services classified as “all other services” that it believes are routine and recurring services and would not impair the independence of the independent auditor and are consistent with SEC and PCAOB rules on auditor independence. All requests or applications for services to be provided by the independent auditor will be submitted to the Chief Financial Officer and must include a detailed description of the services to be rendered. The Chief Financial Officer or the Principal Accounting Officer, as the case may be, will authorize those services that have been pre-approved by the Audit Committee. If there is any question as to whether a proposed service fits within the pre-approved categories of services, the Chairman of the Audit Committee is to be consulted for a determination. For services that have not been pre-approved by the Audit Committee, requests or applications to provide services will be submitted to the Audit Committee by both the independent auditor and the Chief Financial Officer, and must include a joint oral or written statement as to whether, in their view, the request or application is consistent with the SEC’s and PCAOB’s rules on auditor independence.

All services rendered by Ernst & Young LLP in fiscal 2023 were approved in accordance with these policies. In its review of non-audit services, the Audit Committee considers, among other things, the possible impact of the performance of such services on the independent registered public accounting firm’s independence. The Audit Committee has determined that the non-audit services performed by Ernst & Young LLP in the fiscal year ended December 31, 2023 were compatible with maintaining the independent registered public accounting firm’s independence. Additional information concerning the Audit Committee and its activities can be found in the following sections of this Proxy Statement: “Board of Directors and Committees—Board Committees” and “Report of the Audit Committee.”

Ernst & Young LLP has audited our financial statements since our inception. Representatives of Ernst & Young LLP are expected to be present at the Annual Meeting, will have the opportunity to make a statement if they desire to do so, and are expected to be available to respond to appropriate stockholder questions.

Fees for Independent Registered Public Accounting Firm

The following is a summary of the aggregate audit fees billed or expected to be billed by Ernst & Young LLP for the indicated fiscal years and the fees billed by Ernst & Young LLP for all other services rendered during the indicated fiscal years.

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Vote Required

Ratification of the selection of the independent registered public accounting firm requires the affirmative vote of a majority of the votes cast. Because abstentions and broker non-votes, if any, are not counted as votes cast for or against this proposal, they will have no effect on the outcome of the vote.

THE BOARD OF DIRECTORS RECOMMENDS A VOTE “FOR” PROPOSAL NO. 3.

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Report of the Audit Committee

The Audit Committee evaluates auditor performance, manages relations with our independent registered public accounting firm, and evaluates policies and procedures relating to internal control systems. The Audit Committee operates under a written Audit Committee Charter that has been adopted by the Board, a copy of which is available on our website at www.ultragenyx.com. All members of the Audit Committee currently meet the independence and qualification standards for Audit Committee membership set forth in the listing standards and rules of Nasdaq and the SEC.

No member of the Audit Committee is a professional accountant or auditor. The members’ functions are not intended to duplicate or to certify the activities of management and the independent registered public accounting firm. The Audit Committee serves a board-level oversight role in which it provides advice, counsel, and direction to management and the auditors on the basis of the information it receives, discussions with management and the auditors, and the experience of the Audit Committee’s members in business, financial, and accounting matters.

The Audit Committee oversees our financial reporting process on behalf of the Board. Our management has the primary responsibility for the financial statements and reporting process, including our system of internal controls over financial reporting. In fulfilling its oversight responsibilities, the Audit Committee reviewed and discussed with management the audited financial statements included in the Annual Report on Form 10-K for the fiscal year ended December 31, 2023. This review included a discussion of the quality and the acceptability of our financial reporting, including the nature and extent of disclosures in the financial statements and the accompanying notes.

The Audit Committee discussed with our independent registered public accounting firm, which is responsible for expressing an opinion on the conformity of the audited financial statements with generally accepted accounting principles in the United States of America, their judgments as to the quality and the acceptability of our financial reporting and such other matters as are required to be discussed with the Committee pursuant to applicable rules of the PCAOB and the SEC. The Audit Committee has received the written disclosures and the letter from the independent registered public accounting firm required by the applicable rules of the PCAOB regarding the independent accountant’s communications with the Audit Committee concerning independence. The Audit Committee discussed with the independent registered public accounting firm their independence.

In addition to the matters specified above, the Audit Committee discussed with our independent registered public accounting firm the overall scope, plans, and estimated costs of their audit. The Audit Committee met with the independent registered public accounting firm periodically, with and without management present, to discuss the results of the independent registered public accounting firm’s examinations, the overall quality of our financial reporting, and the independent registered public accounting firm’s reviews of the quarterly financial statements and drafts of the quarterly and annual reports.

Based on the reviews and discussions referred to above, the Audit Committee recommended to the Board that our audited financial statements should be included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2023.

Submitted by the Audit Committee of the Board of Directors

Matthew Fust, Chairperson
Corsee D. Sanders, Ph.D.
Michael Narachi

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PROPOSAL 4

Advisory (Non-Binding) Vote to Approve the Compensation of our Named Executive Officers

Background

Section 14A of the Securities Exchange Act of 1934, as amended (Exchange Act), as adopted pursuant to the Dodd-Frank Wall Street Reform and Consumer Protection Act (Dodd-Frank Act) requires that stockholders have the opportunity to cast an advisory (non-binding) vote to approve the compensation of our named executive officers (say-on-pay vote).

The say-on-pay vote is a non-binding vote on the compensation of our “named executive officers,” as described in the Compensation Discussion and Analysis section, the tabular disclosure regarding such compensation, and the accompanying narrative disclosure, set forth in this Proxy Statement. The say-on-pay vote is not a vote on our general compensation policies, compensation of our Board of Directors, our compensation policies as they relate to risk management, or our pay ratio.

Our philosophy in setting compensation policies for executive officers has two fundamental objectives: (1) to attract and retain a highly-skilled team of talented executives and (2) to align our executives’ interests with those of our stockholders by rewarding short-term and long-term performance and tying compensation to increases in stockholder value. The Compensation Committee believes that executive compensation should be directly linked to performance, including continuous improvements in corporate performance and accomplishments of our strategic plan that are expected to increase stockholder value. The Compensation Discussion and Analysis section starting on page 39 provides a more detailed discussion of the executive compensation program and compensation philosophy.

The vote under this Proposal No. 4 is advisory and therefore not binding on us, the Board, or our Compensation Committee. However, our Board, including our Compensation Committee, values the opinions of our stockholders and, to the extent there is any significant vote against this proposal, we will consider our stockholders’ concerns and evaluate what actions may be appropriate to address those concerns. The Dodd-Frank Act requires us to hold the say-on-pay vote at least once every three years, and we have determined to hold a say-on-pay vote every year. Unless the Board modifies its policy on the frequency of holding say-on-pay advisory votes, the next say-on-pay vote will occur in 2025.

Stockholders will be asked at the Annual Meeting to approve the following resolution pursuant to this Proposal No. 4:

RESOLVED, that the stockholders of Ultragenyx Pharmaceutical Inc. approve, on an advisory basis, the compensation of the Company’s “named executive officers” (as defined in the Company’s definitive proxy statement for the 2024 Annual Meeting of Stockholders (the “Proxy Statement”)), as such compensation is described in the Compensation Discussion and Analysis section, the tabular disclosure regarding such compensation, and the accompanying narrative disclosure, set forth in the Company’s Proxy Statement.

Vote Required

Approval of this resolution requires the affirmative vote of a majority of the votes cast on this proposal. Because abstentions and broker non-votes, if any, are not counted as votes cast for or against this resolution, they will have no effect on the outcome of the vote.

THE BOARD OF DIRECTORS RECOMMENDS A VOTE “FOR” PROPOSAL NO. 4.

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Corporate Governance

Director Independence

Our Board currently consists of eight members. Our Board has determined that Dr. Dunsire, Mr. Fust, Mr. Narachi, Dr. Ray, Dr. Sanders, Dr. Suliman, and Mr. Welch qualify as “independent” directors in accordance with Nasdaq listing requirements and rules. Dr. Kakkis is not considered independent because he is an employee of the Company. Former director Lars Ekman, M.D., Ph.D., whose term expired at the 2023 Annual Meeting of Stockholders (2023 Annual Meeting), was independent during the period he served on the Board. Under Nasdaq rules, the Board’s determination of a director’s independence considers objective tests, such as whether the director is, or has been within the last three years, an employee of the Company and whether the director or any of his or her family members has engaged in certain types of business dealings with us. Under Nasdaq rules, our Board also evaluates whether any relationships exist that, in the opinion of our Board, would interfere with the exercise of independent judgment in carrying out the responsibilities of a director. In making these independence determinations, our Board reviewed and discussed information provided by the directors and us with regard to each director’s business and personal activities and relationships as they may relate to us and our management. There are no family relationships among any of our directors or executive officers.

Director Overboarding Policy

Our Board believes that all members of the Board must have sufficient time to focus on his or her Board duties. Our Corporate Governance Guidelines limit the total number of public company boards that a director may serve as follows:

All of our directors are currently in compliance with our policy.

Global Code of Conduct

We have adopted a Global Code of Conduct that applies to all of our employees, officers, and directors, including our principal executive officer, principal financial officer, principal accounting officer or controller, or persons performing similar functions. Our Global Code of Conduct is available on our website, www.ultragenyx.com, under the “Corporate Governance” subsection of the “Investors” tab. We intend to promptly disclose on our website any future changes or amendments to, or waivers of, to the Global Code of Conduct that we are required to disclose under applicable rules. Our Board is responsible for applying and interpreting the Global Code of Conduct in situations where questions are presented to it.

Anti-Hedging and Anti-Pledging Policy

Our insider trading policy prohibits our directors and employees, including our executive officers, from engaging in hedging transactions that are designed to hedge, offset or transfer with respect to equity compensation received by a director or employee or other equity securities of the Company held (directly or indirectly) by a director or employee, all or a portion of the risk of a decline in the market price of shares of our stock. Our insider trading policy also prohibits our director and employees, including our executive officers, from pledging Company securities as collateral for a loan.

Stockholder Communications

Generally, stockholders who have questions or concerns regarding the Company should contact our Investor Relations department at (844) 280-7681. However, any stockholders who wish to address questions regarding our business or affairs directly with the Board, or any individual director, should direct his or her questions in writing to the Chairman of the Board, c/o Ultragenyx Pharmaceutical Inc., 60 Leveroni Court, Novato, California 94949. At the request of the Chairman of the Board, the Corporate Secretary reviews all correspondence addressed to the Chairman, organizes the correspondence, and provides it to the Chairman or to individual directors, as appropriate. Our independent directors have requested that certain items that are inappropriate or unrelated to the Board’s duties, such as spam, junk mail, mass mailings, solicitations, resumes, and job inquiries not be provided to directors.

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Board of Directors and Committees

During fiscal 2023, our Board met five times. Each of our current directors attended at least 90% of the aggregate meetings of the Board and meetings of the committees of which the director was a member in fiscal 2023 held during the period when the director served on the Board or the committees, as applicable.

The Board has a standing Audit Committee, Compensation Committee, Nominating and Corporate Governance Committee, and Research and Development Committee. All members of the Audit, Compensation, and Nominating and Corporate Governance Committees are non-employee directors whom the Board has determined are independent under applicable independence standards (including the heightened independence standards that apply to Audit Committee and Compensation Committee members).

Eight of the directors serving at the time of the 2023 Annual Meeting attended such annual meeting. Each director who is up for election at an annual meeting of stockholders or who has a term that continues after such annual meeting is encouraged to attend the annual meeting of stockholders.

Board Leadership Structure

We currently separate the positions of Chairman of the Board and Chief Executive Officer, which allows our Chief Executive Officer, Dr. Kakkis, to focus on our day-to-day business, while allowing the Chairman of the Board, Mr. Welch, to lead the Board in its fundamental role of providing advice to and independent oversight of management. Independent oversight of management is an important goal of the Board, which is why our Corporate Governance Guidelines provide that a lead independent director will be appointed by the Board if the Chairman is not independent. Additionally, our Board recognizes the time, effort, and energy that the Chief Executive Officer is required to devote to his position in the current business environment, as well as the commitment required to serve as our Chairman. Our Board also believes that the separation of the Chairman and Chief Executive Officer positions fosters a greater role for the independent directors in the oversight of our Company and active participation of the independent directors in setting agendas and establishing priorities and procedures for the work of our Board. The benefits of the separated Chairman and Chief Executive Officer positions are augmented by the independence of eight of our nine current directors, including our Chairman, and our independent Board committees that provide appropriate oversight in the areas described below. At executive sessions of independent directors, these directors can speak candidly on any matter of interest. The independent directors of the Board regularly meet in executive sessions, and met four times in 2023, and the Chairman presides at these sessions. We believe this structure provides effective oversight of our management and the Company.

The Board believes that its programs for risk oversight, as described under “Role of the Board in Risk Oversight” below, would be effective under a variety of leadership frameworks. Accordingly, the Board’s risk oversight function did not significantly impact its selection of the current leadership structure.

Role of the Board in Risk Oversight

The Board has overall responsibility for the oversight of our risk management process, which is designed to support the achievement of organizational objectives, including strategic objectives, to improve long-term organizational performance, and enhance stockholder value. Risk management includes not only understanding company-specific risks and the steps management implements to manage those risks, but also assessing what level of risk is acceptable and appropriate for us, taking into account the immediacy of any such risks, and evaluating risks and circumstances across various timeframes, including the short, medium and long term. Management is responsible for establishing our business strategy, identifying and assessing the related risks, and implementing appropriate risk management practices. The Board periodically reviews our business strategy and management’s assessment of the related risk and discusses with management the appropriate level of risk for us. The Board also periodically evaluates and discusses potential emerging risks with members of senior management as well as third-party advisors and experts. In 2023, the Board and the committees, as appropriate, reviewed with management the various risks and mitigation strategies related to the macroeconomic environment, cost containment strategies, prioritization of the Company’s pipeline programs, cybersecurity risks and the Company’s initiatives related to Corporate Responsibility and sustainability matters. The Board also delegates to Board committees oversight of selected elements of risk as set forth below.

Board Committees

Our Board currently has a standing Audit Committee, Compensation Committee, Nominating and Governance Committee and Research and Development Committee. Each of these committees operates under a written charter which sets forth the functions and responsibilities of the committee, a copy of which is available on our website at www.ultragenyx.com under the “Corporate Governance” subsection of the “Investors” tab.

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ULTRAGENYX PHARMACEUTICAL INC. • 2024 Proxy Statement     28

ULTRAGENYX PHARMACEUTICAL INC. • 2024 Proxy Statement     29

Compensation Committee Interlocks and Insider Participation

During fiscal 2023, the Compensation Committee consisted of Dr. Dunsire and Messrs. Narachi and Welch. None of the members of our Compensation Committee has at any time during the prior three years been one of our officers or employees. None of our executive officers currently serves, or in the past fiscal year has served, as a member of the board of directors or compensation committee of any entity that has one or more executive officers serving on our Board or Compensation Committee.

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Corporate Responsibility Matters*

At Ultragenyx, our purpose is to lead the future of rare disease medicine as we seek to treat individuals afflicted by diseases with limited or no treatment options, and we recognize that their lives and well-being are dependent upon our collective efforts to develop new therapies. For this reason, we are passionate about developing these therapies with the utmost urgency and care. Our strong commitment to ethics, integrity and corporate responsibility is centered around partnering with the rare disease community, improving access to treatments, maintaining a people-first culture, and investing in innovation.

Our 2023 Corporate Responsibility report will be available in digital format on our website at www.ultragenyx.com under “Ultra-Committed – Corporate Responsibility”. Website references throughout this document are provided for convenience only, and the content on the referenced websites is not incorporated by reference into this document. Our 2023 Corporate Responsibility Report is based on our Corporate Responsibility framework, which centers on six pillars: Innovation, Patients, People, Communities, Planet and Governance. The contents of our Corporate Responsibility Reports are not deemed to be part of this Proxy Statement or incorporated by reference herein.

Innovation

We are committed to delivering novel, disease modifying treatments with speed and urgency to rare disease communities with limited or no treatment options. In the coming years, we anticipate developing six to seven clinical therapies with the potential to reach up to 150,000 patients.

In 2023, we advanced key clinical programs including our programs for UX143 in osteogenesis imperfecta (OI), GTX-102 for in Angelman syndrome and UX701 for Wilson disease. See “Executive Compensation – Compensation and Discussion Analysis - Business Highlights” for a description of the advances we made in 2023 in these programs.

We are committed to helping the rare disease community by sharing our science and expertise to advance future drug development, whether by us our others. We host a recurring Rare Bootcamp, where we share our knowledge, expertise, insights and connections to help patient families, foundations and other organizations seeking to develop novel treatments for rare diseases. Our bootcamp is designed for incredibly determined patient families and advocates who have started funding their own rare disease research and are looking to better coordinate and build structure around their efforts. Since 2017, we have held eight bootcamps with over 135 individuals from more than 100 organizations attending.

Patients

We are committed to supporting the broader rare disease community through our efforts to develop novel therapies, share our science and expertise, achieve broad access to screening and treatment, and partner with policy makers for meaningful change. We believe that the greatest impact we can have on the lives of individuals with rare disease is to make our treatments accessible and affordable, and to engage and support rare disease families along their journey.

We believe that pricing should not be a barrier to accessing treatment and we are committed to pricing our medicines responsibly. We set our drug prices with global pricing in mind and in the U.S., we use our best efforts to help make it possible that no patient foregoes treatment for financial reasons. In 2023, we continued to align U.S. price increases consistent with the Consumer Price Index for the commercial products where we lead promotion activities.

We believe policy and regulation are critical to the cost-effective and timely development and commercialization of treatments for individuals living with rare diseases. Our Global Policy Committee identifies priority areas for our engagement and advocacy, aiming to educate, inspire and influence key decision-makers in the rare disease field. The committee’s objective is to enable all individuals with a rare disease to receive an accurate diagnosis, quality care and access to available therapy as quickly as possible.

We are a founding member of the Rare Disease Company Coalition (RDCC), a coalition of 23 companies that educates policymakers on the distinct considerations of life science companies operating in the rare disease space and focuses on three priorities: supporting robust development and innovation, promoting accessibility, and enabling earlier diagnosis.

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People

We are committed to working to maintain a diverse, inclusive, safe and healthy environment. We are also committed to fair and equitable compensation practices that are transparent and free from bias. We strive to provide employees with a workplace and work environment where they can do their best work and where they want to stay long term. Our voluntary employee turnover rate in 2023 was 10.5%, which is below the U.S. and global averages for our industry according to benchmark reports from Aon/Radford.

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We are committed to fair and equitable compensation practices within a pay-for-performance framework and believe these principles are critical in supporting our culture and achieving our mission. As part of this commitment, we regularly conduct thorough pay analyses designed to assess and uphold equitable compensation practices. These assessments take into account various factors such as performance, experience, level, tenure and location to identify any potential pay disparities among employees in similar roles. To strengthen our endeavors, we leverage software that enhances our capacity to effectively monitor and manage pay equity practices. This includes utilizing data analytics, which enable a more comprehensive examination of compensation across roles, departments and demographic groups. With real-time monitoring capabilities, we track adherence to our pay equity principles throughout the hiring process and employment lifecycle, facilitating prompt identification of any emerging issues.

Communities

We are committed to supporting initiatives that provide (1) impactful resources for the rare disease community, (2) public health and access to care, (3) Science, Technology, Engineering, the Arts and Mathematics (STEAM) education, and (4) local, at-risk communities. In 2023, we approved over $2.5 million in charitable donations, independent medical education and health-related grants across 21 countries. We offer employees the opportunity to take two paid volunteer days each year (16 hours), so that they can spend time giving back to our communities and contribute to local initiatives. In 2023, we introduced our Ultragenyx Inaugural Days of Service where more than 50% of our employees worldwide registered to be a part of numerous impactful volunteer activities.

In addition to our philanthropic efforts, we support the rare disease community through educational initiatives, patient advocacy, research and access to information. Our specific focus is on patient and professional organizations that provide vital support to the rare disease community.

Planet

We are committed to developing an environmental strategy that minimizes our environmental footprint across our business. In 2023, we continued to implement improvements to reduce our environmental footprint and to develop our environmental strategy.

A few of our initiatives are described below:

Governance

We are committed to maintaining responsible corporate governance practices to promote the long term interest of our stockholders and other stakeholders. A summary of our corporate governance practices is described in this Proxy Statement under “– Corporate Governance.”

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Executive Officers

Our current executive officers, their respective ages as of the date of this Proxy Statement, and positions are set forth in the following table. Biographical information regarding each executive officer (other than Dr. Kakkis) is set forth following the table. Biographical information for Dr. Kakkis is set forth above under Proposal No. 1 (Election of Class II Directors). Mr. Crombez was appointed as our Chief Medical Officer and Executive Vice President effective March 2023 and Mr. Horn joined the Company as our Chief Financial Officer, Corporate Strategy and Executive Vice President effective October 2023.

Dr. Eric Crombez has served as our Chief Medical Officer, Executive Vice President since March 2023. Dr. Crombez previously served as our Chief Medical Officer for gene therapy and inborn errors of metabolism, overseeing global clinical development and execution for the Company’s gene therapy programs, after he joined the Company following the acquisition of Dimension Therapeutics, a biotechnology company, in November 2017. At Dimension Therapeutics, Dr. Crombez served as Chief Medical Officer from December 2014 to November 2017 where he led the clinical development efforts for clinical gene therapy programs in hemophilia B, hemophilia A, ornithine transcarbamylase (OTC) deficiency and glycogen storage disease type Ia (GSDIa). Previously, he worked at Shire plc, a biopharmaceutical company, in its Human Genetics Therapy business unit. Dr. Crombez is an appointed industry representative on the FDA Cellular, Tissue and Gene Therapies Advisory Committee. He previously served as assistant professor, Department of Pediatrics, Division of Medical Genetics at the David Geffen School of Medicine at the University of California, Los Angeles (UCLA). Dr. Crombez is a board-certified clinical geneticist and completed residencies in pediatrics and medical genetics and a fellowship in clinical biochemical genetics at the UCLA School of Medicine. Dr. Crombez obtained his B.S. in biology from the University of Michigan, Ann Arbor, and his M.D. from Wayne State University School of Medicine, Detroit.

Erik Harris has served as our Chief Commercial Officer and Executive Vice President since June 2019. Prior to his appointment as our Chief Commercial Officer, Erik served as our Senior Vice President and Head of North American Commercial Operations from July 2017 to June 2019. Prior to Ultragenyx, Mr. Harris spent six years at Crescendo Bioscience, Inc., a molecular diagnostic company, most recently as Vice President of Commercial. Earlier in his career, Mr. Harris served as Vice President of Marketing at InterMune, Inc., a biotechnology company, and also held positions in the commercial organizations at Elan Pharmaceuticals, Inc., Genentech, Inc., and Bristol-Myers Squibb Company. At the start of his professional career, Mr. Harris served as a Lieutenant Commander in Naval Aviation and Congressional Fellow for the United States Navy. Mr. Harris currently serves on the board of directors of Denali Therapeutics Inc., a public biopharmaceutical company. Mr. Harris holds a B.S. from the United States Naval Academy and an M.B.A. from the Wharton School of Business.

Howard Horn has served as our Chief Financial Officer, Corporate Strategy and Executive Vice President since October 2023. He previously served as Chief Financial Officer and as a member of the founding management team at Vir Biotechnology, a public biotech company from March 2017 to April 2023, where he led the accounting, finance, investor relations and facilities functions. Prior to Vir Biotechnology, Mr. Horn was at Biogen, a biotechnology company, where he served first as Vice President, Strategic Corporate Finance and led Biogen’s corporate capital allocation processes, and then as Vice President, Business Planning and led Biogen’s resource allocation processes across all functions and regions. Previously, he held positions of increasing responsibility as a consultant in the Pharmaceutical and Medical Products Practice at McKinsey & Company, and as an equity research analyst in the Life Sciences group at UBS Group AG. Mr. Horn received his B.A. in Economics from Princeton University and his M.B.A. from the Wharton School of the University of Pennsylvania.

Dennis Huang has served as our Executive Vice President since January 2016, our Chief Technical Operations Officer since May 2015 and as our Chief Technical Operations Officer and Gene Therapy Operations since December 2021. From May 2015 to January 2016, he served as our Senior Vice President. Prior to Ultragenyx, Mr. Huang served as Senior Vice President of Manufacturing and Supply Chain at InterMune, Inc., a biotechnology company, from August 2013 to March 2015. Prior to InterMune, Mr. Huang served as Vice President of Biologic Manufacturing and Development at Allergan, Inc., a global pharmaceutical company, from May 2006 to August 2013. Mr. Huang currently serves on the board of directors of CytoDel, Inc., a private biopharmaceutical company. Mr. Huang holds a B.A. in Chemistry from Knox College in Galesburg, Illinois.

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Thomas Kassberg has served as our Executive Vice President since January 2016 and our Chief Business Officer since November 2011. From November 2011 to January 2016, he served as our Senior Vice President. Prior to Ultragenyx, Mr. Kassberg worked as Vice President of Business Development at Corium International, Inc., a biotechnology company, from July 2010 to October 2011. Prior to his work at Corium International, Inc., Mr. Kassberg worked as an independent consultant in corporate development and business strategy and consulted with a number of companies from March 2009 to June 2010, including Corium International, Inc., a biopharmaceutical company, and Rib-X Pharmaceuticals, Inc., a pharmaceutical company focused on the development of novel antibiotics. Before becoming a consultant, Mr. Kassberg worked at Proteolix, Inc., a biotechnology company subsequently acquired by Onyx Pharmaceuticals, from January 2008 to February 2009, where he served as Senior Vice President of Corporate Development. Mr. Kassberg holds a B.A. in Business Administration from Gustavus Adolphus College and an M.B.A. from Northwestern University.

Karah Parschauer has served as our Chief Legal Officer & Corporate Affairs, Executive Vice President since February 2023, as our Chief Legal Officer and Executive Vice President since December 2021 and as our General Counsel and Executive Vice President since June 2016. Prior to Ultragenyx, Ms. Parschauer served in various executive capacities, and most recently as Vice President, Associate General Counsel, at Allergan plc, a pharmaceutical company, from June 2005 to June 2016. Prior to Allergan, Ms. Parschauer was an attorney at Latham & Watkins LLP, where she practiced in the areas of mergers and acquisitions, securities offerings and corporate governance. Ms. Parschauer currently serves on the board of directors of Evolus, Inc., a publicly traded performance beauty company, and the board of directors of Tenaya Therapeutics, a publicly traded biotechnology company. Ms. Parschauer holds a B.A. in Biology from Miami University and a J.D. from Harvard Law School.

John R. Pinion II has served as our Chief Quality Officer and Executive Vice President of Translational Sciences since September 2017. From January 2016 to September 2017, he served as our Executive Vice President of Analytical Sciences and Research, and from July 2015 to September 2017, as our Chief Quality Operations Officer. From July 2015 to January 2016, he served as our Senior Vice President of Analytical Sciences and Research. Prior to Ultragenyx, Mr. Pinion served in various roles with increasing responsibilities at Genentech, a pharmaceutical company, from 2005 to June 2015, including his most recent position as the Senior Vice President and Global Head of Quality and Compliance for Roche/Genentech Pharma Technical Operations from October 2009 to July 2015. Mr. Pinion currently serves on the board of directors of Aroa Biosurgery, a soft tissue regeneration company listed on the Australian Securities Exchange. Mr. Pinion holds a B.S. of Science in Mechanical Engineering from the University of West Virginia.

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Certain Relationships and Related-Person Transactions

Related-Person Transactions

Since January 1, 2023, we have not become, and are not currently proposed to be, a participant in any transactions required to be disclosed in the Proxy Statement under SEC rules with any “related persons,” which are generally considered to be our directors and executive officers, nominees for director, holders of more than 5% of our outstanding common stock, and members of their immediate families.

Procedures for Related-Person Transactions

We have adopted a written related-person transactions policy that governs the review, approval, and/or ratification of transactions with a related person where the amount involved exceeds $100,000 and in which any related person has or will have a direct or indirect interest. Under the policy, a “related person” is defined as any person described in Item 404(a) of Regulation S-K and includes any director, nominee for director, or executive officer of the Company; a beneficial owner of more than five percent of any class of our voting securities; and a person who is an immediate family member of any such director, nominee for director, executive officer, or more-than-five percent beneficial owner (the term “immediate family member” included any child, stepchild, parent, stepparent, spouse, sibling, mother-in-law, father-in-law, son-in-law, daughter-in-law, brother-in-law, or sister-in-law and any person (other than a tenant or employee) sharing the household of any such director, nominee for director, executive officer, or more-than-five percent beneficial owner).

Pursuant to this policy, prior to entering into a transaction with a related person, our Chief Financial Officer (or Chief Legal Officer, in the case where the Chief Financial Officer has a direct or indirect interest in the transaction) will review the proposed transaction to determine if such transaction qualifies as a related-person transaction. If the Chief Financial Officer (or Chief Legal Officer, if applicable) determines that the proposed transaction is a related-person transaction, then the proposed transaction will be submitted to the Audit Committee for consideration at the next Audit Committee meeting; provided, however, that if the Chief Financial Officer (or Chief Legal Officer, if applicable), in consultation with the Chief Executive Officer, determines that it is not practicable or desirable to wait until the next meeting of the Audit Committee, then the Chief Financial Officer (or Chief Legal Officer, if applicable) shall submit the proposed transaction to the chairperson of the Audit Committee (who possesses delegated authority to act between meetings of the Audit Committee to pre-approve or ratify, as applicable, any related-person transaction in which the aggregate amount involved is expected to be less than $1 million).

In the event that our Chief Executive Officer or Chief Financial Officer (or Chief Legal Officer, if applicable) becomes aware of a related-person transaction that has not been previously approved or previously ratified under our related-person transaction policy, the transaction, if pending or ongoing, will be promptly submitted to the Audit Committee or the chairperson of the Audit Committee for consideration. If the transaction is already completed, the Audit Committee or the chairperson of the Audit Committee shall evaluate the transaction to determine if rescission of the transaction and/or any disciplinary action is appropriate.

In evaluating these transactions, the Audit Committee or the chairperson of the Audit Committee, as applicable, will consider all of the relevant facts and circumstances available, including (if applicable) but not limited to: the benefits to us; the impact on a director’s independence in the event the related person is a director, an immediate family member of a director, or an entity in which a director has a position or relationship; the availability of other sources for comparable products or services; the terms of the transaction; and the terms available to unrelated third parties or to employees generally. The Audit Committee or the Chairperson of the Audit Committee, as applicable, will only approve related-person transactions that are in, or are not inconsistent with, the best interests of the Company and its stockholders, as the Audit Committee or the Chairperson of the Audit Committee determines in good faith.

No member of the Audit Committee shall participate in any review, consideration or approval of any related-person transaction with respect to which such member or any of his or her immediate family members is the related person.

Delinquent Section 16(a) Reports

Under Section 16(a) of the Exchange Act and SEC rules, our directors, executive officers and beneficial owners of more than 10% of any class of equity security are required to file periodic reports of their ownership, and changes in that ownership, with the SEC. To our knowledge, based solely on a review of copies of the reports filed with the SEC and written representations from our directors and executive officers that no Form 5 was required, other than one Form 4 to report one transaction with respect to Theodore Huizenga, which were not timely filed due to administrative error, all reports required to be filed by our executive officers, directors and beneficial owners of more than 10% of our common stock were timely filed in fiscal 2023.

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Security Ownership of Certain Beneficial Owners and Management

The following table sets forth information relating to the beneficial ownership of our common stock as of April 12, 2024 (unless otherwise indicated), by:

The number of shares beneficially owned by each entity, person, director or executive officer is determined in accordance with the rules of the SEC, and the information is not necessarily indicative of beneficial ownership for any other purpose. Under such rules, beneficial ownership includes any shares over which the person has sole or shared voting power or investment power as well as any shares that the person has the right to acquire within 60 days through the exercise of any stock options, warrants or other rights. We believe, based on the information furnished to us, that except as otherwise indicated, and subject to applicable community property laws, the persons named in the table below have sole voting and investment power with respect to all shares of common stock held by that person.

The percentage of shares beneficially owned is computed on the basis of 83,103,882 shares of our common stock outstanding as of April 12, 2024. Shares of our common stock that a person has the right to acquire within 60 days are deemed outstanding for purposes of computing the percentage ownership of the person holding such rights, but are not deemed outstanding for purposes of computing the percentage ownership of any other person, except with respect to the percentage ownership of all directors and executive officers as a group. Unless otherwise indicated below, the address for each beneficial owner listed is c/o Ultragenyx Pharmaceutical Inc., 60 Leveroni Court, Novato, California 94949.

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Executive Compensation

Compensation Discussion and Analysis

The following compensation discussion and analysis describes the material elements of compensation earned in fiscal 2023 by each of the executive officers identified below, who are referred to collectively as our “named executive officers.” Our named executive officers with respect to the fiscal year that ended on December 31, 2023 are:

These persons constitute our principal executive officer, our principal financial offer and our three other most highly paid executive officers serving during fiscal 2023. Mr. Horn joined the Company as Chief Financial Officer and principal financial officer effective October 16, 2023. Dr. Kakkis served as interim principal financial officer in 2023 prior to Mr. Horn’s appointment.

Business Highlights

In 2023, we generated significant momentum across our clinical and commercial programs to position our company for what we expect to be a catalyst-rich 2024.

Revenue and Commercial Highlights

We achieved $434 million in total revenue in 2023 compared to total revenue of $363 million in 2022, which represented 20% growth. We achieved continued strong growth in product sales in 2023 compared to the prior year. Revenue from product sales of Crysvita^® in countries where we lead commercialization activities increased from $43 million to $76 million, or by 77%, compared to 2022, largely due to sales in Latin America. Total revenue from Crysvita^® sales, including royalty revenue we received from our partner, Kyowa Kirin, from sales of the product in North America (following the transition of our commercialization responsibilities in North America to Kyowa Kirin in 2023) and in the European territory increased by 17% from $279 million in 2022 to $328 million in 2023. Global revenue from Dojolvi^® increased by 27% from $56 million in the prior year to $71 million in 2023 and global revenue from Mepsevii^® increased by 48% from $21 million in 2022 to $30 million in 2023.

In 2023, we continued to progress international regulatory and reimbursement negotiations across our marketed products and continued to add new approvals and positive reimbursement decisions, specifically in certain launch markets for Evkeeza. For instance, in December 2023, we received approval from the European Commission for an expanded indication for Evkeeza in children aged five years and older with homozygous familial hypercholesterolemia (HoFH) and in January 2024, we announced that we had achieved marketing authorization for Evkeeza in Japan for HoFH.

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Program Highlights

Meaningful Data Announcements. We hosted a company Analyst Day in October 2023 where we announced meaningful updates and data from three of our key clinical programs:

Continued Progress on Other Clinical Programs. In addition to our meaningful progress on UX143, GTX-102 and UX701 as described above, we continued to make important progress on our other clinical programs:

Other Corporate Achievements

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Stockholder Outreach

Stockholder Advisory Vote on Executive Compensation

Each year, our stockholders are provided the opportunity to cast an advisory vote on the compensation of our named executive officers, or the “say-on-pay” vote, and the Compensation Committee considers the outcome of the prior year’s say-on-pay vote when making decisions relating to the compensation of our named executive officers and our executive compensation programs. We received 95% support for our say-on-pay proposal at our 2023 Annual Meeting, representing strong support of our executive compensation programs and an increase over the 88% support we received at our 2022 Annual Meeting. We did not make any changes to our executive compensation programs as a result of the say-on-pay vote.

Stockholder Engagement

We regularly engage with our stockholders to solicit their feedback on a variety of topics, including executive compensation, corporate governance, corporate responsibility and other topics in order to gain a better understanding of their perspective on these issues. Stockholder feedback is important, and the information we glean from these engagements is highly valued. Our stockholder engagement team has consisted of certain independent directors and members of our legal, investor relations and human resources expertise areas. Stockholders also regularly meet with members of our senior management team to discuss our strategy and review our business, goals and performance. In 2023, we engaged with several shareholders who owned, in aggregate, a meaningful percentage of outstanding shares. The feedback we received from those conversations were conveyed to the rest of our senior management team and the board and relevant board committees, as appropriate.

Compensation Highlights

As described below under “Compensation Philosophy and Objectives”, our Compensation Committee believes that executive compensation should be directly linked to short-term and long-term performance. A few of the key decisions made by the Compensation Committee aligned with such philosophy are as follows:

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Compensation Philosophy and Objectives

Our philosophy in setting compensation policies for executive officers has two fundamental objectives: (1) to attract and retain a highly-skilled team of executives and (2) to align our executives’ interests with those of our stockholders by rewarding short-term and long-term performance and tying compensation to increases in stockholder value. The Compensation Committee believes that executive compensation should be directly linked to both continuous improvements in corporate performance (pay for performance) and accomplishments that are expected to increase stockholder value. In furtherance of this goal, the Compensation Committee has adhered to the following guidelines as a foundation for decisions that affect the levels of compensation:

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.

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.

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Roles in Determining Compensation

Compensation Committee

The Board has delegated to the Compensation Committee the responsibility to ensure that total compensation paid to our executive officers, including our named executive officers, is consistent with our compensation policy and objectives. The Compensation Committee oversees and approves all compensation arrangements and actions for our executive officers, including our named executive officers. While the Compensation Committee draws on a number of resources, including input from the Board, the Chief Executive Officer, and its independent compensation consultants, to make decisions regarding our executive compensation program, ultimate decision-making authority rests with the Compensation Committee. The Compensation Committee retains discretion over base salaries, annual bonuses, and equity compensation for executive officers. The Compensation Committee relies upon the judgment of its members in making compensation decisions, after reviewing our corporate performance and carefully evaluating an executive’s performance during the year against established goals, operational performance, and business responsibilities. In addition, the Compensation Committee utilizes discretion in the assessment process to respond to and adjust to a dynamic business environment. The Compensation Committee may delegate its authority to one or more subcommittees. The Compensation Committee may also delegate authority to review and approve the compensation of our employees to certain of our executive officers.

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.

Chief Executive Officer

The Chief Executive Officer and other executive officers, attends Compensation Committee meetings and works with the Compensation Committee Chairman and Aon to develop compensation recommendations for the executive officers (excluding the Chief Executive Officer), based upon individual experience and breadth of knowledge, internal considerations, individual performance during the fiscal year, competitive market considerations, and other factors deemed relevant by the Compensation Committee. The recommendations are then submitted to the Compensation Committee for review and consideration. The Compensation Committee works directly with Aon and the other non-employee directors of the Board to evaluate the performance of the Chief Executive Officer and determine compensation actions for the Chief Executive Officer and the Chief Executive Officer does not participate in the portions of meetings in which his compensation is discussed and determined.

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 25^th, 50^th and 75^th 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:

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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.

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 40^th percentile of the peer group in terms of market capitalization, the 79^th percentile in terms of employees headcount and the 31^st percentile in terms of revenue.

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.

Base Salary

Overview

The Compensation Committee believes it is important to provide adequate fixed compensation to our executive officers working in a highly volatile and competitive industry. The Compensation Committee’s choice of actual pay levels versus our competitive market reflects consideration of our stockholders’ interests in paying what is necessary to achieve our corporate goals, while setting competitive levels which are essential to retain these key executives. In determining appropriate base salary levels for a given executive officer, the Compensation Committee considers the following factors:

The effective date of annual merit increases to base salary is March 1.

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.

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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.

Annual Bonus

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.

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.

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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.

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:

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.

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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.

Achievement of Goals and Relationship to Compensation Awarded

The overall 94% achievement score for the 2023 corporate goals, combined with Dr. Kakkis’ assessment of the individual performance and achievement of Mr. Harris, Mr. Pinion and Ms. Parschauer during fiscal 2023 resulted in the Compensation Committee approving bonus awards for performance in 2023 as set forth in the following table.

The bonuses awarded under our 2023 annual incentive program were paid in March 2024.

Equity Compensation

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.

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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.

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:

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.

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

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.

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.

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:

Based on the Company’s ranking relative to the TSR of the companies in the Nasdaq Biotechnology Index of the 46^th 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.

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Fiscal 2024 Compensation

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:

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.

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.

Base Salaries

For 2024, increases in base salaries for our named executive officers, were 4% annualized for Dr. Kakkis to align to the external market peer group, 3% annualized for Mr. Harris and Mr. Pinion to align to the market and 6% annualized for Ms. Parschauer for assuming additional responsibilities and to align to the market. Mr. Horn did not receive an increase in base salary for 2024 as he had joined the Company in October 2023. The overall 2024 merit budget was based on an Aon trend report regarding projected market merit budgets and spend for 2024.

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.

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

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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.

Employee Benefits Program

Executive officers are eligible to participate in all of our employee benefit plans, including medical, dental, vision, group life, disability, and accidental death and dismemberment insurance. In each case, participation is on the same basis as other employees, subject to applicable law. We also provide vacation and other paid holidays to all employees, including executive officers, all of which we believe to be comparable to those provided at peer companies. These benefit programs are designed to enable us to attract and retain our workforce in a competitive marketplace. Reliable and competitive health, welfare and vacation benefits ensure that we have a productive and focused workforce.

In addition, our named executive officers are eligible to participate in a retirement savings plan (401(k) Plan), which is a tax-qualified defined contribution plan pursuant that allows participants to contribute certain amounts of their annual compensation, subject to limits prescribed by the Internal Revenue Service. All of our employees are eligible to participate in the 401(k) Plan on the same terms as the named executive officers.

Our named executive officers, along with our other highly-compensated U.S. based employees and non-employee directors of our Board, are also eligible to participate in our nonqualified deferred compensation plan. Please see ” -Nonqualified Deferred Compensation Plan” below for a summary of the plan.

Clawback Policy

Our Board has adopted a Clawback Policy intended to comply with the listing standards from Nasdaq implementing Exchange Act Rule 10D-1. In the event the Company is required to prepare an accounting restatement of the Company’s financial statements due to material non-compliance with any financial reporting requirement under the federal securities laws, the Company will recover, on a reasonably prompt basis, the excess incentive-based compensation received by any covered executive officer, including the named executive officers, during the prior three fiscal years that exceeds the amount that the named executive officer otherwise would have received had the incentive-based compensation been determined based on the restated financial statements. The Clawback Policy also permits recoupment of all incentive compensation, including time-based and performance-based equity awards and bonuses, in the event of fraud or intentional misconduct by a current or former executive officer, including the named executive officers.

Minimum Stock Ownership Requirements

We maintain stock ownership guidelines in order to align the long-term interests of our executive officers and directors with those of our stockholders. The guidelines require holding shares of our common stock with value equivalent to 3x the annual retainer for Board members, 3x base salary for our Chief Executive Officer and 1x base salary for the other named executive officers. Shares that count toward satisfaction of these guidelines include shares owned outright by the individual (including RSUs that have vested), shares retained after an option exercise or issuance under another type of equity award granted under the company’s equity incentive plans, shares retained after purchase under the ESPP and shares held in trust for the benefit of the individual. Unexercised options and unvested and unearned RSUs and PSUs do not count towards satisfaction of these guidelines. These guidelines were required to be achieved by the end of 2022 for our named executive officers and Board members who have been named executive officers and Board members as of the date the policy was implemented and within five years of appointment for newly appointed named executive officers and Board members. As of December 31, 2023, each of our Board members and named executive officers who were required to be in compliance with the guidelines by the end of 2023 had met the ownership guidelines.

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

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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.

Compensation Committee Report

The Compensation Committee has reviewed and discussed with management the Compensation Discussion and Analysis required by Item 402(b) of Regulation S-K. Based on this review and discussion, the Compensation Committee recommended to the Board of Directors that the foregoing Compensation Discussion and Analysis be included in this Proxy Statement and incorporated by reference in our Annual Report on Form 10-K for the fiscal year ended December 31, 2023.

Submitted by the Compensation Committee of the Board of Directors

Michael Narachi, Chairman

Deborah Dunsire, M.D.

Daniel G. Welch

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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.

Narrative Disclosure to Summary Compensation Table

Employment Arrangements with Our Named Executive Officers

Dr. Kakkis, our Chief Executive Officer, is party to an employment agreement with us that provides for base salary and participation in our employee benefit plans, subject to the terms of those plans. Pursuant to the terms of the employment agreement, the employment of Dr. Kakkis is at will; we may terminate his employment at any time, without advance notice, for any reason or for no reason at all, and Dr. Kakkis may terminate his employment at any time, upon four weeks’ prior written notice, for any reason or for no reason at all.

Each of our other named executive officers is party to an offer letter with us that provides for base salary, an annual bonus opportunity, and an initial grant of equity. They are eligible to participate in our employee benefit plans, subject to the terms of those plans. Pursuant to the terms of the offer letters, their employment is at will and may be terminated either by us or by them, with or without advance notice, for any reason or for no reason at all. Pursuant to Mr. Horn’s offer letter, he received a $60,000 sign-on bonus in 2023, which is subject to repayment in the event his employment is terminated for “cause” (as defined below) or as a result of his resignation that is not a “constructive termination” (as defined below) within 12 months of his start date.

Each of these employment arrangements and offer letters, as amended, also contain provisions that provide for certain payments and benefits in the event of an involuntary termination of employment. In addition, the named executive officers may be entitled to accelerated vesting of their outstanding and unvested awards in certain circumstances. The information below describes certain compensation that may become due and payable as a result of certain events as reflected in the amended arrangements.

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Involuntary Termination of Employment

Pursuant to their employment arrangements or offer letters, each named executive officer is eligible to receive certain payments and benefits in the event of certain qualifying terminations, including termination of his or her employment by us without “cause” (as defined below) or resignation of his or her employment with “good reason” or because of a “constructive termination” (each, as defined below). Certain benefits are enhanced in the event the qualifying termination occurs within a specified period of time (12 months for Dr. Kakkis, and 18 months for Mr. Horn, Mr. Harris, Mr. Pinion and Ms. Parschauer) after the consummation of a “covered transaction” (as defined below), which we refer to as the covered transaction protection period. Upon the timely execution of a general release of claims, each named executive officer is eligible to receive the following payments and benefits:

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Definitions

For purposes of Dr. Kakkis’ employment agreement, “cause” means his:

For purposes of each of the offer letters with Mr. Horn, Mr. Harris, Mr. Pinion and Ms. Parschauer “cause” means the named executive officer’s:

For purposes of Dr. Kakkis’ employment agreement, “good reason” means any of the following events without his consent, subject to standard notice and cure requirements:

For purposes of each of the offer letters with Mr. Horn, Mr. Harris, Mr. Pinion and Ms. Parschauer, “constructive termination” means the occurrence of any of the following events without the named executive officer’s consent, subject to standard notice and cure requirements:

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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.

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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.