DEF 14A of Rhythm Pharmaceuticals, Inc., Apr 29, 2026

DEF 14A of Rhythm Pharmaceuticals, Inc.

Filed Apr 29, 2026 · FY20250001628280-26-028375

Open on EDGAR

  1. PROPOSAL NO. 1—ELECTION OF DIRECTORS

    PROPOSAL NO. 1—ELECTION OF DIRECTORS In accordance with Rhythm’s certificate of incorporation and bylaws, each as currently amended and in effect, the Board is divided into three classes of directors of approximately equal size. The members of each class of directors are elected to serve a three-year term with the term of office of each class ending in successive years. David W.J. McGirr and David P. Meeker, M.D. are the Class III directors whose terms expire at the Annual Meeting. Each of David W.J. McGirr and David P. Meeker, M.D. has been nominated for, and has agreed to stand for, election to the Board to serve as a Class III director of Rhythm for three years until the 2029 Annual Meeting of Stockholders and until his successor is duly elected and qualified or until his earlier death, incapacity, resignation or removal. It is intended that, unless you give contrary instructions, shares represented by proxies will be voted for the election of each of the two nominees listed above as director nominees. Rhythm has no reason to believe that any nominee will be unable to serve. In the event that one or more nominees is unexpectedly not available to serve, proxies may be voted for another person nominated as a substitute by the Board, or the Board may reduce the number of directors to be elected at the Annual Meeting. Information relating to each nominee for election as director and for each continuing director, including his or her period of service as a director of Rhythm, principal occupation and other biographical material, is included under “Director Biographies” below. Based on voluntary self-identification by our directors, our Board consists of three female and four male directors.

  2. THE BOARD OF DIRECTORS UNANIMOUSLY RECOMMENDS THAT YOU VOTE “FOR”

    THE BOARD OF DIRECTORS UNANIMOUSLY RECOMMENDS THAT YOU VOTE “FOR” EACH OF THE CLASS III DIRECTOR NOMINEES NAMED IN THIS PROXY STATEMENT.

  3. BOARD OF DIRECTORS

    BOARD OF DIRECTORS

  4. Board Composition and Structure

    Board Composition and Structure Our certificate of incorporation and bylaws, each as currently amended and in effect, state that our Board shall consist of a number of directors that shall be fixed exclusively by the Board from time to time in accordance with the bylaws of the Company. Each director holds office until his or her successor is duly elected and qualified or until his or her death, incapacity, resignation or removal. Our certificate of incorporation provides that our directors may be removed only for cause by the affirmative vote of the holders of at least 75% of the voting power of the outstanding shares of capital stock of Rhythm entitled to vote in the election of directors, voting together as a single class, at a meeting of the stockholders called for that purpose. Any vacancy in the Board, including a vacancy that results from an increase in the number of directors, may be filled only by the vote of a majority of the remaining directors then in office, although less than a quorum, or by the sole remaining director. Our certificate of incorporation, as currently amended and in effect, provides that our Board is divided into three classes of directors, with the classes as nearly equal in number as possible. Each of our directors identified below serves in the class indicated. Subject to any earlier resignation or removal in accordance with the terms of our certificate of incorporation and bylaws, our current Class I directors will serve until the 2027 Annual Meeting of Stockholders; our current Class II directors, will serve until the 2028 Annual Meeting of Stockholders; and our current Class III directors, if elected at the Annual Meeting, will serve until the 2029 Annual Meeting of Stockholders. Any additional directorships resulting from an increase in the number of directors will be apportioned by our Board among the three classes as equally as possible. Below is a list of the names, ages as of April 27, 2026 and classification of the individuals who currently serve as our directors, two of whom are also nominees to be re-elected at the Annual Meeting.

  5. Board Composition and Structure

    | Name | Age | Position(s) | Class | Term Expires | | Stuart A. Arbuckle(1)(3) | 60 | Director | I | 2027 | | Jennifer Good(2) | 61 | Director | II | 2028 | | Christophe R. Jean(2)(3) | 70 | Director | I | 2027 | | David W. J. McGirr(2)(3) | 71 | Director | III | 2026 | | David P. Meeker, M.D. | 71 | Director, Chairman of the Board | III | 2026 | | Kimberly J. Popovits(1) | 67 | Director | II | 2028 | | Lynn A. Tetrault, J.D.(1) | 63 | Lead Director | I | 2027 |

  6. Board Composition and Structure

    _______________________________________________________ (1)Member of the Compensation & Management Development Committee. (2)Member of the Audit Committee. (3)Member of the Governance & Nominating Committee. In consultation with the Governance & Nominating Committee, the Board has determined that the classified board structure remains appropriate for us at this time. A classified board provides for stability, continuity and experience among our Board. Further, the Board believes that building a cohesive board is an important goal. In our industry in particular, long-term focus is critical. The time horizon required for successful development of pharmaceuticals makes it vital that we have a board that understands the implications of this process and has the ability to develop and implement long-term strategies while benefiting from an in-depth knowledge of Rhythm’s business and operations. A classified board structure helps to ensure that there will be the continuity and stability of leadership required to navigate a challenging economic environment while resisting the pressure to focus on short-term results at the expense of our long-term value and success. The future success of Rhythm depends in significant part on the ability to attract and retain capable and experienced directors. In this regard, we believe that longer terms for our directors will enhance director independence from both management and stockholder special interest groups.

  7. Director Biographies

    Director Biographies Information concerning our continuing directors and director nominees is set forth below. The biographical description of each continuing director and director nominee includes the specific experience, qualifications, attributes and skills that led the Board to conclude that such person should serve as a director.

  8. Class III Directors (Nominees Standing for Re-Election)

    Class III Directors (Nominees Standing for Re-Election)

  9. David W.J. McGirr

    David W.J. McGirr Mr. McGirr has served as a member of our Board since 2015. Mr. McGirr also serves as a director of Insmed Incorporated, a pharmaceutical company devoted to the treatment of rare diseases. Previously, Mr. McGirr held various roles at Cubist Pharmaceuticals, including Senior Advisor to the chief executive officer and Senior Vice President and Chief Financial Officer. Prior to joining Cubist, Mr. McGirr was the President and Chief Operating Officer of Hippo Inc., an internet technology, venture-financed company. Before that, he held roles of increasing responsibility at GAB Robins North America, Inc., a risk management company, including as Chief Executive Officer. Earlier in his career, Mr. McGirr served in various positions within the S.G. Warburg Group, ultimately as Chief Financial Officer, Chief Administrative Officer and Managing Director of S.G. Warburg & Co., Inc. Mr. McGirr received a B.Sc. in Civil Engineering from the University of Glasgow and received an M.B.A. from The Wharton School at the University of Pennsylvania. Mr. McGirr has been designated an audit committee financial expert as defined in applicable SEC rules. In concluding that he is qualified to serve as a director, the Board considered Mr. McGirr’s deep experience in corporate finance and financial reporting, as well as his knowledge, perspective and corporate governance expertise. The Board also considered that his expertise in financial and accounting matters, his prior experience as Chief Financial Officer of a leading biotechnology company, and his service on other public company boards and committees would significantly benefit the Company. David P. Meeker, M.D. Dr. Meeker has served as a member of our Board since 2015 and became Chairman of the Board in April 2017. Dr. Meeker has served as our President and Chief Executive Officer since July 2020. Previously, Dr. Meeker served as CEO of KSQ Therapeutics, Inc., a biotechnology company, from October 2017 to July 2020. From October 2011 until June 2017, Dr. Meeker served as President and CEO of Genzyme Corporation and subsequently as EVP, Head Sanofi Genzyme, a unit of Sanofi S.A., a global pharmaceutical company. Dr. Meeker oversaw the company’s specialty business units—Rare Diseases, Multiple Sclerosis, Oncology and Immunology. As an Executive Vice President of Sanofi S.A., he was a member of Sanofi S.A.’s Executive Committee. Dr. Meeker joined Genzyme Corporation in 1994 as Medical Director to work on the Cystic Fibrosis Gene Therapy program. Subsequently, as Vice President, Medical Affairs, he was responsible for the development of rare disease therapies that today represent transformative and life-saving advancements in medicine for patients. Prior to Genzyme Corporation’s merger with Sanofi S.A. in 2011, Dr. Meeker was Genzyme Corporation’s Chief Operating Officer, responsible for its commercial organization, overseeing its business units, country management organization and global market access functions. He played an important role in the integration with Sanofi S.A. Prior to joining Genzyme Corporation, Dr. Meeker was the director of the Pulmonary Critical Care Fellowship at the Cleveland Clinic and an assistant professor of medicine at Ohio State University. Dr. Meeker is currently Chairman of the board of directors of Pharvaris GmbH and the Chairman of the board of directors of Trevi Therapeutics, Inc. He has authored more than 40 articles and multiple book chapters. Dr. Meeker received his M.D. from the University of Vermont Medical School. He completed the Advanced Management Program at Harvard Business School in 2000. In concluding that he is qualified to serve as a director, the Board considered Dr. Meeker’s deep experience as a senior executive at leading global pharmaceutical companies, including his experience serving as our President and Chief Executive Officer, and his involvement in the development and commercialization of pharmaceutical product candidates for the treatment of rare and ultra-rare diseases. The Board also considered the integral role he played in the growth of Genzyme during its period of rapid growth. The Board also noted Dr. Meeker’s extensive scientific and clinical experience, both in academia and as part of one of the world’s leading medical institutions.

  10. Continuing Directors Not Standing for Re-Election at the 2026 Annual Meeting

    Continuing Directors Not Standing for Re-Election at the 2026 Annual Meeting

  11. Stuart A. Arbuckle

    Stuart A. Arbuckle Mr. Arbuckle has served as a member of our Board since July 2019. From August 2021 to July 2025, Mr. Arbuckle was Executive Vice President and Chief Operating Officer at Vertex Pharmaceuticals Incorporated, a biopharmaceutical company, where he was responsible for the company’s global commercial team, including reimbursement and access, sales, marketing, patient support, market research and other activities that supported the approved use of Vertex’s cystic fibrosis medicines. Mr. Arbuckle also oversaw the Human Resources, Corporate Communications and Program and Portfolio Management functions. Prior to assuming that role, he served as Executive Vice President and Chief Commercial Officer at Vertex from September 2012 to March 2021. Prior to joining Vertex, Mr. Arbuckle held several commercial leadership positions at Amgen Inc., including leading sales and marketing efforts for Amgen Inc.’s portfolio of cancer medicines, such as Aranesp®, Neulasta® injection and NEUPOGEN®, and leading the successful launches of XGEVA® injection and Nplate® injection. Most recently, he served as Vice President and Regional General Manager at Amgen Inc., where he led efforts to expand the company’s presence in Asia, the Middle East, and Africa. Prior to these roles, Mr. Arbuckle spent 15 years at GlaxoSmithKline plc, where he held sales and marketing roles for medicines aimed at treating metabolic, respiratory, musculoskeletal, cardiovascular and other diseases. Mr. Arbuckle serves on the boards of Alnylam Pharmaceuticals, Inc. and Praxis Precision Medicines, Inc. Mr. Arbuckle previously served as a board member of ImmunoGen, Inc. from January 2019 to February 2024. Mr. Arbuckle holds a BSc in Pharmacology and Physiology from the University of Leeds in the United Kingdom. The Board considered Mr. Arbuckle’s senior executive roles at publicly-held life sciences companies, as well as his service on the boards of directors of biotechnology companies and organizations, in concluding that he is qualified to serve as a director.

  12. Jennifer Good

    Jennifer Good Ms. Good has served as a member of our Board since June 2019. She is a co-founder of Trevi Therapeutics, Inc., a clinical-stage biopharmaceutical company, and has served as a member of the board of directors and as the President and Chief Executive Officer of Trevi Therapeutics, Inc. since its inception in 2011. Previously, Ms. Good served at Penwest Pharmaceuticals Co. from 1997 to 2010, where she held various positions including President and Chief Executive Officer, Chief Operating Officer and Chief Financial Officer. Ms. Good received a Bachelor of Business Administration degree with a concentration in accounting from Pacific Lutheran University in 1987 and is a Certified Public Accountant (inactive). The Board considered the role Ms. Good played in the founding and growth of Trevi Therapeutics, Inc. and her senior executive roles at publicly traded life sciences companies, as well as her service on the boards of directors of biotechnology companies and other organizations, in concluding that she is qualified to serve as a director.

  13. Christophe R. Jean

    Christophe R. Jean Mr. Jean has served as a member of our Board since 2015. He is currently a Strategic Advisor for Oraxys S.A., a private equity firm, a role he has held since June 2019 and an External Advisor to Bain & Company, a management consulting firm, since January 2025. Mr. Jean previously served as a Senior Advisor for JSB Partners, LLC, a specialized biotechnology and pharmaceutical investment banking and advisory services firm, from March 2018 until December 2019. Previously, Mr. Jean served as Executive Vice President of Corporate Strategy, Business Development, Alliances and M&A for Ipsen S.A., a pharmaceutical company, from 2013 until his retirement in July 2018, after serving for 11 years in the position of Executive Vice President, Chief Operating Officer, with responsibility for all commercial operations and medical affairs worldwide as well as Ipsen S.A.’s therapeutic area franchises. Prior to joining Ipsen S.A., Mr. Jean was President and CEO for the pharmaceutical activities of the Pierre Fabre Group and Head of Europe, Middle East, and Africa for Novartis Pharma AG’s Pharmaceutical Division. Prior to the merger of Ciba-Geigy AG and Sandoz Inc. that formed Novartis Pharma AG, he held a number of marketing and management positions in Europe and Latin America for Ciba-Geigy AG, culminating as Head of Finance and IT Worldwide and Member of the Pharma Executive Committee. Mr. Jean also serves on the boards of directors of private companies. He holds an M.B.A. from Harvard Business School. The Board considered Mr. Jean’s international business experience and executive leadership at several of the world’s leading pharmaceutical companies and his long career in commercial leadership roles in concluding that he is qualified to serve as a director. The Board also considered Mr. Jean’s extensive experience in commercializing therapeutics outside of the United States.

  14. Kimberly J. Popovits

    Kimberly J. Popovits Kimberly J. Popovits has served on our board of directors since April 2026. Ms. Popovits has served as a Senior Advisor to Blackstone Life Sciences since 2020. She served as President and Chief Executive Officer of Genomic Health, Inc., a publicly traded healthcare company, from January 2009, and as Chairman of the Board of Genomic Health, Inc. from March 2012, until the acquisition of Genomic Health, Inc. by Exact Sciences Corporation in November 2019. Ms. Popovits previously served as President and Chief Operating Officer of Genomic Health from February 2002 to January 2009. Prior to leading Genomic Health, Ms. Popovits served as Senior Vice President, Marketing and Sales at Genentech, Inc. (“Genentech”). During her 15 years at Genentech, Ms. Popovits led the successful commercialization of 14 new therapies. Ms. Popovits currently serves on the board of directors of 10x Genomics, Inc. and Kiniksa Pharmaceuticals International, plc. Ms. Popovits served on the board of directors of Talis Biomedical Corporation, a publicly-traded biotechnology company, from 2020 to 2025, where she also served as Interim Chief Executive Officer from August 2021 to November 2021. She previously served on the board of directors of MyoKardia, Inc., a publicly-traded biopharmaceutical company that was subsequently acquired by Bristol Myers Squibb and Exact Sciences. She holds a B.A. in Business from Michigan State University. The Board considered Ms. Popovits's senior executive roles in publicly traded life sciences companies, her extensive experience and strong background in executive leadership at commercial-stage companies, as well as her service on the boards of directors of numerous publicly held biotechnology companies, in concluding that she is qualified to serve as a director. Lynn A. Tetrault, J.D. Ms. Tetrault has served as a member of our Board since December 2020. Ms. Tetrault currently serves as a board member and Independent Chairperson of NeoGenomics, Inc., a publicly held oncology diagnostics company, and served as Interim Chief Executive Officer of NeoGenomics from March 2022 to August 2022. She joined the board of NeoGenomics in 2015. Ms. Tetrault serves on the board of directors of Alumis, Inc., previously Acelyrin, Inc., a publicly held biotechnology company, since December 2023. Ms. Tetrault spent most of her career at AstraZeneca PLC serving in a variety of senior positions, including most recently as Executive Vice President of Human Resources and Corporate Affairs from May 2007 to March 2014. In May 2017, Ms. Tetrault founded Anahata Leadership, Inc., an advisory firm focused on supporting the leadership and development of executive women, and she continues to serve as its principal. Ms. Tetrault began her career as a lawyer in private healthcare practice in Boston. Ms. Tetrault holds a B.A. from Princeton University and a J.D. from the University of Virginia. In concluding that she is qualified to serve as a director, the Board considered Ms. Tetrault’s significant experience as a senior executive at a global biopharmaceutical company, her strong background in executive leadership, and her service on the boards of several publicly held healthcare companies.

  15. DIRECTOR COMPENSATION

    DIRECTOR COMPENSATION We maintain a non-employee director compensation program that is intended to provide a total compensation package that enables us to attract and retain qualified and experienced individuals to serve as directors and to align our directors’ interests with those of our stockholders. Our Compensation & Management Development Committee periodically reviews the compensation of our non-employee directors and, as applicable, makes recommendations to the Board for its consideration of changes to our non-employee director compensation program. Our Compensation & Management Development Committee compares the compensation of our Board to compensation paid to non-employee directors of similarly sized public companies. Our Compensation & Management Development Committee also considers the responsibilities we ask of the members of our Board along with the amount of time required to perform those responsibilities. Under our non-employee director compensation program, we provide our non-employee directors with cash retainers and equity awards in the form of stock options and restricted stock units (“RSUs”). We do not pay any compensation to our President and Chief Executive Officer in connection with his service on our Board. The compensation that we pay to our President and Chief Executive Officer is discussed in the “Executive Compensation” section of this Proxy Statement. Following an analysis of director compensation levels prepared by the Compensation & Management Development Committee’s compensation consultant, Pay Governance, in June 2025 the Compensation & Management Development Committee recommended and the Board approved an updated director compensation program, which became effective on June 24, 2025. The annual equity awards for non-employee directors are granted on the date of the Company’s annual meeting of stockholders. For 2025, the Board equity mix was updated to improve alignment between the executive team and the non-employee directors. The mix of long-term incentive awards for the non-employee directors was changed to 50% options and 50% RSUs (from two-thirds options and one-third RSUs previously), based on the fair values of such awards. In addition, the Board elected to continue to impose a grant value cap of $600,000, for the annual equity award and $1,200,000 for any initial awards. This cap is intended to apply if the value of the fixed number of options and RSUs that is granted annually to the Board reached the upper quartile of the Company's peer group and was put into place to avoid oversized director compensation in the event of exceptional stock price performance. As a result, for 2025 annual awards to our non-employee directors, the option grant was reduced from 10,000 options to 7,037 options and the RSU grant was reduced from 6,667 RSUs to 4,712 RSUs. Pursuant to our non-employee director compensation program, for 2025, each non-employee director received an annual retainer fee of $50,000 paid in arrears, in addition to other annual retainers, as applicable depending on their roles within the Board and its committees. Below is a list of all annual retainers for non-employee directors:

  16. DIRECTOR COMPENSATION

    | Non-Employee Director | Annual Fee | | Annual Cash Retainer | $50,000 | | Lead Director | $35,000 | | Non-Executive Chair | $30,000 | | Chair of the Audit Committee | $20,000 | | Member of the Audit Committee (other than chair) | $10,000 | | Chair of the Compensation & Management Development Committee | $20,000 | | Member of the Compensation & Management Development Committee (other than chair) | $10,000 | | Chair of the Governance & Nominating Committee | $10,000 | | Member of the Governance & Nominating Committee (other than chair) | $5,000 |

  17. DIRECTOR COMPENSATION

    ________________________________________________________ We also reimburse our non-employee directors for reasonable travel and out-of-pocket expenses incurred in connection with attending Board and committee meetings. Under our non-employee director compensation program, each individual initially appointed or elected to the Board as a non-employee director is eligible to receive for initial appointments or elections, an initial option award to purchase 20,000 shares of our Common Stock and an initial award of 13,334 RSUs. The awards are granted under our 2017 Equity Incentive Plan on the date he or she first becomes a non-employee director. These initial awards vest annually over a three-year period from the date of grant, subject to continued service as a non-employee director through the vesting date. Our director compensation program includes a limit on the grant date fair value of the initial equity awards granted to newly appointed non-employee directors of $1,200,000. If the grant date fair value of the initial equity award would exceed this limit, an award equal to 50% of the limit will be paid in options and an award equal to 50% will be paid in RSUs, based on the grant date fair values of such options and RSUs. During 2025, no non-employee directors were initially appointed or elected to the Board. On June 24, 2025, the date of our 2025 annual meeting of stockholders, each continuing non-employee director who served on the Board for a minimum of six months was eligible to receive an option to purchase 10,000 shares of our Common Stock and 6,667 RSUs (which amounts were reduced to 7,037 options and 4,712 RSUs as a result of the previously described value cap of $600,000); the options and RSUs will vest in full upon the earlier of the first anniversary of the date of grant or the date of our 2026 annual meeting of stockholders. The exercise price for each of the option grants is equal to the fair market value of our Common Stock on the date of grant. All outstanding equity awards held by our non-employee directors vest in full immediately prior to the occurrence of a change in control. Our director compensation program includes a limit on the grant date fair value of the annual equity awards granted to continuing non-employee directors of $600,000. If the grant date fair value of an annual equity award would otherwise exceed this limit, an award equal to 50% of the limit will be paid in options and an award equal to 50% will be paid in RSUs, based on the grant date fair values of such options and RSUs.

  18. 2025 Director Compensation Table

    2025 Director Compensation Table The following table sets forth information regarding compensation earned by our non-employee directors during fiscal year 2025. Dr. Meeker did not receive any compensation for his service on our Board while employed as our President and Chief Executive Officer during 2025.

  19. 2025 Director Compensation Table

    | Name | Fees Earned or Paid in Cash ($)(1) | Stock Awards ($)(2) | Option Awards ($)(2) | Total ($) | | Stuart Arbuckle | $60,000 | $299,966 | $300,015 | $659,981 | | Camille Bedrosian, M.D. (3) | $65,000 | $299,966 | $300,015 | $664,981 | | Jennifer Good | $60,000 | $299,966 | $300,015 | $659,981 | | Christophe R. Jean | $60,000 | $299,966 | $300,015 | $659,981 | | Edward T. Mathers | $95,000 | $299,966 | $300,015 | $694,981 | | David W. J. McGirr | $75,000 | $299,966 | $300,015 | $674,981 | | Lynn A. Tetrault | $70,000 | $299,966 | $300,015 | $669,981 |

  20. 2025 Director Compensation Table

    ________________________________________________________ (1)Amounts represent annual cash compensation earned for services rendered by each member of the Board. (2)Amounts reflect the full grant-date fair value of stock options and RSUs granted during 2025 computed in accordance with ASC Topic 718, rather than the amounts paid to or realized by the named individual. We provide information regarding the assumptions used to calculate the value of option and stock awards made to our directors in Note 8 to the consolidated financial statements included in our Annual Report on Form 10-K for the year ended December 31, 2025. (3)Dr. Bedrosian resigned from the Board effective December 16, 2025. The following table sets forth, as of December 31, 2025, the aggregate numbers of option awards (exercisable and unexercisable) and unvested stock awards held by each non-employee director at that time.

  21. 2025 Director Compensation Table

    | Name | Option Awards Outstanding at 2025 Fiscal Year End | Unvested Stock Awards Outstanding at 2025 Fiscal Year End | | Stuart Arbuckle | 118,537 | 4,712 | | Camille Bedrosian, M.D. (1) | 100,000 | 0 | | Jennifer Good | 35,037 | 4,712 | | Christophe R. Jean | 113,537 | 4,712 | | Edward T. Mathers | 113,537 | 4,712 | | David W. J. McGirr | 135,347 | 4,712 | | Lynn A. Tetrault | 72,037 | 4,712 |

  22. 2025 Director Compensation Table

    (1)Dr. Bedrosian resigned from the Board effective December 16, 2025.

  23. 2025 Stockholder Engagement and Stockholder Feedback and Our Responses

    | What We Heard | How We Responded | Why It Matters | | Stockholders want more transparency on how we determine CEO pay and ensure its alignment with company performance. | Within this CD&A we have enhanced disclosure regarding the determination of CEO pay and the resulting strong pay‑for‑performance alignment. Our three-year total stockholder returns are in the 84th percentile when compared to our executive compensation peer group. Our CEO pay for that same period is in the 79th percentile indicating clear pay for performance alignment and confirming that our executive compensation program designs are working as intended. In response to stockholder feedback requesting greater emphasis on linking payouts to the strategic and financial achievements of each year, the CEO’s 2026 bonus award will be calculated based on 100% corporate achievement with no individual performance component. | Demonstrates alignment of CEO pay with stockholder value creation and improves transparency. | | Stockholders want to know what metrics are used to select Rhythm's peer group. | We believe that our investors understand that peer group selection for a biopharmaceutical company like Rhythm is unique compared to other industries. Most high-growth biopharmaceutical companies, in addition to considering stage (clinical or commercial) and scope (domestic or global), utilize market capitalization as one of the primary determinants for peer group selection, as market capitalization appropriately reflects the market’s expectations for and value ascribed to the Company's pipeline. At the time the peer group used for 2025 compensation decisions was approved in September 2024, Rhythm’s market capitalization of $2.9B was closely aligned with the peer median of $2.6B supporting comparisons with similarly sized, high growth biopharmaceutical companies. | Ensures compensation decisions reflect an appropriate competitive set of biopharma companies for comparability. |

  24. 2025 Stockholder Engagement and Stockholder Feedback and Our Responses

    | What We Heard | How We Responded | Why It Matters | | Stockholders would like more detailed disclosures on the annual bonus plan goals and calculation of bonus payments thereunder. | We have provided enhanced disclosure of the 2025 Corporate Objectives Scorecard and how Company performance was assessed against the objectives. Rhythm had a strong 2025, which was reflected in the Board approving a 120% corporate achievement factor for the bonus plan for 2025. The 2025 annual bonus plan was tied to corporate objectives that reflect a balance of financial, pipeline, and other key operational metrics that support Rhythm’s sustained growth and drive progress toward long-term strategic achievements. In response to stockholder feedback, we have provided details as to how our 2025 annual bonus awards were determined in the CD&A table below entitled “2025 Rhythm Pharmaceuticals Corporate Objectives Scorecard”. | Helps investors understand how annual performance outcomes are assessed and align with company achievements. | | Stockholders would like to see a greater emphasis on performance-based long-term incentives as part of the overall executive compensation package. | Our CEO and other executive officers are eligible for long-term incentives that reflect a mix of 50% stock options and 50% restricted stock units ("RSUs"), based on the fair value of such awards. We consider stock options to be performance-based, in that value is only realized if there is growth in the Company’s stock price following the grant. We believe that our program is highly aligned with our pay for performance philosophy as our executives cannot realize any actual compensation from their stock options unless the share price increases, providing a strong incentive link to stockholder value creation. Further, we have heard stockholder requests for the introduction of performance-based awards to our executive long-term incentive grant program. The Compensation & Management Development Committee will take this feedback under advisement and consider the integration of such awards into future program design. | Supports long‑term stockholder alignment and ensures balanced incentive design. |

  25. 2025 Stockholder Engagement and Stockholder Feedback and Our Responses

    | What We Heard | How We Responded | Why It Matters | | Stockholders requested more details on the special PSU grants with a 2024-2026 performance period. | In 2024, the Compensation & Management Development Committee approved special 3- year performance stock unit (“PSU”) grants in order to drive executive focus and performance at a critical inflection point in Rhythm’s growth trajectory. The PSU grants were intended as a special incentive grant and were not intended to be incorporated into the executive annual long-term grant mix which consists of 50% stock options and 50% RSUs. While the investors we met with were generally supportive of the rationale we provided for the special PSU grants, they requested more disclosure for such grants and future grants of PSUs. In response to stockholder feedback, we have provided enhanced details as to the rationale behind the special PSU grants and the metrics selected in the CD&A section below entitled “2024-2026 Special PSU Grants and Determination of Performance.” | Increases transparency regarding the purpose of the special PSU grants and respond to stockholder disclosure expectations. | | Stockholders questioned whether the classified board structure remains appropriate for Rhythm. They also would like more transparency regarding succession planning. | Some stockholders expressed concern regarding our corporate governance structure, in particular sunsetting our classified board structure. Considering the longer-term time horizons for the successful execution of the Company’s strategy, we have determined that a classified board remains appropriate at this time to ensure the necessary continuity and stability of leadership. Stockholders also requested greater transparency regarding succession planning. Succession planning for the CEO and executive officers is a key priority for the Compensation & Management Development Committee and the Board of Directors and is discussed regularly. We are committed to ongoing consideration of these and other governance topics. | Ensures governance supports long‑term strategy and oversight continuity. |

  26. Compensation Governance and Best Practices

    | What We Do | | What We Do Not Do | | | ✓ | Emphasize performance-based, at-risk compensation. | X | Do not grant guaranteed salary, bonus or equity compensation. | | ✓ | Emphasize the use of equity compensation to promote executive alignment with stockholders and reward long-term value creation. | X | Do not provide excessive perquisites. | | ✓ | Weight the overall pay mix towards incentive compensation for senior executives. | X | Do not provide any tax gross-ups for change-in-control payments. | | ✓ | Engage an independent compensation consultant to advise our Compensation & Management Development Committee. | X | Do not reprice stock option awards issued under our 2017 Equity Incentive Plan without stockholder approval. | | ✓ | Engage with stockholders to understand views on executive compensation and respond to concerns. | X | Do not provide supplemental executive retirement plans. | | ✓ | Maintain a NASDAQ-compliant clawback policy. | X | Do not provide excessive severance payments. | | ✓ | Maintain an insider trading policy, including anti-hedging and anti-pledging provisions. | | | | ✓ | Maintain an independent Compensation & Management Development Committee. | | | | ✓ | Set challenging corporate performance goals and provide our stockholders with detailed disclosure around our goal setting process and achievement. Maintain an industry-specific and size-appropriate compensation peer group. | | | | ✓ | Maintain an industry-specific and size-appropriate compensation peer group. | | | | ✓ | Conduct annual compensation risk assessment | | | | ✓ | Utilize an annual incentive plan with pre-set corporate goals and capped payouts | | | | ✓ | Maintain an annual say-on-pay vote frequency | | | | ✓ | Maintain a stock ownership policy | | |

  27. Role of Compensation Consultant

    Role of Compensation Consultant In order to design a competitive executive compensation program that will continue to attract top executive talent and reflect our compensation philosophy, our Compensation & Management Development Committee retains an independent compensation consultant to provide executive compensation advisory services, help evaluate our compensation philosophy and objectives and provide guidance in designing and administering our executive compensation program. During 2025, the Compensation & Management Development Committee continued to engage Pay Governance LLC (“Pay Governance”) as its executive compensation advisor. The Compensation & Management Development Committee has evaluated the independence of Pay Governance pursuant to the requirements of Nasdaq and SEC rules and has determined that Pay Governance does not have any conflicts of interest in advising the Compensation & Management Development Committee. Pay Governance, as an independent executive advisory firm, did not provide any other services to the Company in 2025 for which the amount of fees exceeded $120,000.

  28. Peer Group

    Peer Group In September 2024, we reviewed and updated our peer group to ensure it remained aligned with our company size and stage of development. At that time, Pay Governance proposed a group of peer companies based on the following parameters: •Operates in the biopharmaceutical industry and, to the degree possible, having a rare disease focus; •Comparable in terms of clinical and/or commercial stage of development, importance of pipeline to overall value, headcount, and revenue; and •Comparable in terms of market capitalization (as noted above, at the time the peer group used for 2025 compensation decisions was approved in September 2024, Rhythm’s market capitalization of $2.9B was closely aligned with the peer median of $2.6B, supporting comparisons with similarly sized, high growth biopharma companies). We believe that investors understand peer group selection for a biopharmaceutical company like Rhythm is unique compared to other industries, as stockholders put a premium on the long-term value of our pipeline as well as near-term financial performance. Most high-growth biopharmaceutical companies use market capitalization as one of the primary determinants for peer group selection as it appropriately reflects the market’s expectations for and value ascribed to the Company's pipeline. This holds true for Rhythm as the value of our pipeline is reflected in the Company’s market capitalization, which best represents the total value that our team is delivering for stockholders and for patients. In addition, as a high growth commercial company, market capitalization is a leading indicator of Rhythm’s potential future stockholder value creation. Accordingly, market capitalization is appropriately weighted more heavily than revenue when comparing ourselves to other biopharmaceutical companies of similar size and stage for purposes of executive compensation comparisons. Based on the above criteria and considerations, the following peer group was approved by our Compensation & Management Development Committee, which peer group was used to make 2025 compensation decisions.

  29. Peer Group

    | Agios Pharmaceuticals | Amicus Therapeutics | Apellis Pharmaceuticals | Ardelyx | | BioCryst Pharmaceuticals | Blueprint Medicines Corporation | BridgeBio Pharma, Inc. | Catalyst Pharmaceuticals, Inc. | | Insmed Incorporated | Intra-Cellular Therapies | Ironwood Pharmaceuticals | Krystal Biotech, Inc. | | Mirum Pharmaceuticals, Inc. | Ocular Therapeutix | PTC Therapeutics, Inc. | SpringWorks Therapeutics | | TG Therapeutics | Travere Therapeutics | Ultragenyx Pharmaceuticals | Vericel Corporation |

  30. Peer Group

    For 2025, the Compensation & Management Development Committee used Pay Governance’s analysis to help structure a competitive executive compensation program, position executive compensation by considering market data, and make individual compensation decisions based on comparable positions at companies with which we compete for talent. While the Compensation & Management Development Committee does not establish compensation levels solely based on a review of competitive data, such data is a useful tool in its deliberations as our compensation policies and practices must be competitive in the marketplace for us to be able to attract, motivate and retain qualified executive officers.

  31. Base Salary

    Base Salary The base salaries of our NEOs are an important part of their total compensation package, and are intended to reflect their respective positions, duties, performance, experience and responsibilities. Base salary is a visible and stable fixed component of our compensation program. Base salaries provide our NEOs with a reasonable degree of financial certainty and stability. Our Compensation & Management Development Committee, and with respect to our Chief Executive Officer, the Board, annually reviews and determines the base salaries of our executives and evaluates the base salaries of new hires at the time of hire. In February 2025, our Compensation & Management Development Committee, and with respect to our Chief Executive Officer, the Board, approved base salary increases for the NEOs to be effective as of February 24, 2025 for our US-based NEOs and March 1, 2025 for Mr. Mazabraud to align with payroll calendars. Such increases were intended to position our NEOs’ base salaries closer to the market median of our peer group companies, as well as to reflect additional considerations set forth above under “Determination of Executive Compensation.” Following such determinations, our NEO’s base salaries, which were within +/- 5% of the market median for their roles based on peer group analysis, were approved as set forth below:

  32. Cash Incentive Compensation

    Cash Incentive Compensation We consider annual cash incentive bonuses to be an important component of our total compensation program that provides incentives necessary to retain executive officers and drive company performance in alignment with short and long-term strategic goals. Each NEO is eligible to receive an annual performance-based cash bonus based on a specified target annual bonus award amount expressed as a percentage of the NEO’s base salary. Target annual bonus percentages are assessed annually and aligned with market median for similar roles within Rhythm's peer group. Actual bonuses are calculated by multiplying the target bonus amounts by a weighted achievement score determined by the Compensation & Management Development Committee and the Board based on overall achievement of the annual corporate goals and each named executive officer’s achievement of his or her individual goals. In 2025, achievement of the corporate goals accounted for 70% of the overall target achievement score, and achievement of each named executive officer’s individual goals accounted for 30% of the overall target achievement score. Beginning in 2026, achievement of the corporate goals accounts for 100% of the CEO’s overall target achievement score. At the beginning of each year, the Compensation & Management Development Committee and the Board review and approve the annual corporate goals. There is no minimum bonus percentage or amount established for the named executive officers and, as a result, the actual bonus amounts vary from year to year based on corporate and individual performance. For the 2025 annual bonus program, the corporate goals were designed to measure our commercial growth and the development of our product candidates, including the progression and enrollment of our clinical programs, announcement of clinical trial results, achievement of product candidate development and regulatory milestones, expansion of genetic disease and patient identification programs, and progression of our commercial capabilities through establishment of infrastructure and facilities. The individual goals were designed to measure each officer’s individual contribution to the corporate goals and his or her leadership performance. Each year, the Compensation & Management Development Committee and the Board also consider achievements that go beyond the corporate objectives explicitly identified prior to the start of the year, in order to recognize opportunistic achievements that drive our business forward and which may not have been anticipated when the Corporate Objectives Scorecard was developed and approved the prior year. For example, the Board recognized that for 2025, Rhythm delivered outsized stockholder returns of 91% based on the change in stock price over the course of 2025; completed successful financing initiatives to provide Rhythm with a cash runway that we believe will be sufficient to fund our operations for at least 24 months from February 26, 2026, the date we filed our Annual Report on Form 10-K; added a Part D arm to our RM-718 trial for patients with Prader Willi syndrome; completed important technical operations improvements and initiatives to ensure clinical and commercial drug supply to support our rapid growth and expanded our operations globally, including the addition of an operational affiliate in Japan and significant growth in headcount in anticipation of the launch of IMCIVREE for patients with acquired HO. Actual achievement of the 2025 corporate goals was 99.7% and the Board, on recommendation of the Compensation & Management Development Committee, unanimously approved awarding a performance payout of 120% for the corporate achievement component of the bonus plan due to the previously described significant, value-creating achievements beyond those specifically enumerated at the beginning of 2025. As our business is growing and scaling rapidly and management identifies additional strategic opportunities as they arise, the Board determined that it was appropriate to take into account and reward actual achievements looking back at the year as a whole and not solely those items identified at the beginning of the year. For 2025, the corporate performance goals as well as their relative weightings and levels of actual achievement are set out in the table below. Each goal allowed for payouts exceeding 100% of target, contingent on achieving enhanced or accelerated milestones. Maximum opportunities are listed in the chart below by category. The maximum payout opportunity for the 2025 corporate bonus program was capped at 148%.

  33. Cash Incentive Compensation

    | 2025 Rhythm Pharmaceuticals Corporate Objectives Scorecard | | | | | Objective Category | Category Weight | Maximum Opportunity | Achievement | | Global | | | | | Total Net Revenue - Target of $185M Actual achieved - $191M at constant currency | 30.0% | 60.0% | 33.2% | | Global HO Readiness - Refine and execute market access strategy | 5.0% | 5.0% | 5.0% | | North America | | | | | HO Readiness - Ensure field team readiness for launch | 10.0% | 10.0% | 10.0% | | International | | | | | HO Readiness - Accelerate cross-functional plan for Japan; Scientific Communications and Congresses Plan; French and Italian early access programs | 5.0% | 5.0% | 5.0% | | Pipeline | | | | | Advance HO - Setmelanotide HO Phase 3; Regulatory Submissions; Bivamelagon HO Phase 2 Advance Setmelanotide Program - PWS, Congenital HO Advance CMC for RM-718 and Bivamelagon Advance CHI Program and New Research | 40.0% | 57.5% | 36.5% | | Operational Excellence | | | | | Continuous Improvement Goals | 5.0% | 5.0% | 5.0% | | People and Organization | | | | | Cultivating a High-Performing Culture | 5.0% | 5.0% | 5.0% | | Objective Category Sub-Totals | 100.0% | 147.5% | 99.7% | | Additional Achievements Outside of Above | | | | | Financing and Cash Runway; Pipeline Advancement (Part D for RM-718 for PWS); People and Organization (Japan expansion; #6 on Boston Globe Top Places to Work) | N.A. | N.A. | 20.0% | | TOTAL (as determined by the Board): | | | 120.0% |

  34. Cash Incentive Compensation

    The 2025 individual goals for each NEO were aligned with and based on each NEO’s ability to influence the company’s attainment of the corporate objectives. Individual goals represent 30% of each NEOs annual target bonus opportunity. Our NEOs’ 2025 target bonuses as a percentage of base salary were as follows:

  35. Cash Incentive Compensation

    | Name | 2025 Target Bonus (as a percentage of base salary) | | David P. Meeker | 75% | | Hunter C. Smith | 50% | | Jennifer Lee | 45% | | Yann Mazabraud | 45% | | Alastair Garfield | 45% |

  36. Cash Incentive Compensation

    After the end of 2025, the Compensation & Management Development Committee and the Board determined that the Company had achieved the 2025 corporate goals at 120% of target, reflecting the significant progress on key objectives made during 2025. The average individual performance modifier for all NEOs for 2025 was 153%. The table below shows each NEO’s target bonus and actual payout under the 2025 annual bonus program. The annual bonus payout formula is as follows: Actual Payout = ((Salary x Target Bonus % x 70% x Corporate Achievement Multiplier) + (Salary x Target Bonus % x 30% x Individual Achievement Multiplier)) * Time in Role Proration.

  37. Cash Incentive Compensation

    | Named Executive Officer | 2025 Target Bonus | Actual 2025 Bonus Payout | | David P. Meeker | $588,750 | $759,488 | | Hunter C. Smith | $262,100 | $338,000 | | Jennifer Lee | $232,425 | $293,000 | | Yann Mazabraud(1) | $252,760 | $344,659 | | Alastair Garfield | $222,750 | $287,000 |

  38. Cash Incentive Compensation

    ________________________________________________________ (1)These amounts were converted from Euros to U.S. dollars based on an exchange rate of 1.17631 as of December 31, 2025.

  39. Equity-Based Long-Term Incentive Awards

    Equity-Based Long-Term Incentive Awards We maintain the 2017 Equity Incentive Plan (the “2017 Plan”), under which we may grant equity incentive awards to directors, employees and consultants of our Company and our affiliates, to enable us to attract and retain services, skills and experience of these individuals, which we believe are essential to our long-term success. We do not currently have any formal policy for determining the number of equity-based awards to grant to NEOs.

  40. Annual Equity Awards

    Annual Equity Awards In 2025, our Compensation & Management Development Committee approved grants of stock options and RSUs to our NEOs. Our NEOs were awarded a blend of stock options and RSUs similar to other commercial stage life sciences companies in the market. Annual equity awards are designed as long-term incentive and retention tools, determined by the Compensation & Management Development Committee and based on a number of factors, including number of shares as a percent of the company’s common shares outstanding, equity value, equity as a portion of total direct compensation, and relevant external market data and recommendations provided by the Compensation & Management Development Committee’s independent compensation consultant. The stock options are scheduled to vest and become exercisable in sixteen substantially equal quarterly installments following the applicable vesting commencement date. The RSUs are scheduled to vest in four equal annual installments following the applicable vesting commencement dates. Vesting of the stock options and RSUs is subject to continued service through each applicable vesting date. The following table contains a summary of grants of stock options and RSUs to our NEOs in 2025:

  41. Annual Equity Awards

    | Name | Number of Shares Underlying Stock Options | Number of RSUs | | David P. Meeker | 200,000 | 133,350 | | Hunter C. Smith | 62,500 | 41,650 | | Jennifer Lee | 55,000 | 36,650 | | Yann Mazabraud | 60,000 | 40,000 | | Alastair Garfield | 40,000 | 26,650 |

  42. 2024-2026 Special PSU Grants and Determination of Performance

    2024-2026 Special PSU Grants and Determination of Performance In 2024 the Committee granted PSU awards on a special, one-time basis to our NEOs with long-term performance criteria designed to drive stockholder value and align executive pay with compensation performance. The PSUs granted in 2024 vest upon the achievement of key strategic financial, commercial and clinical milestone performance goals over a three-year period. Shares will vest, if any, following the conclusion of the performance period based on the Committee’s certification of performance results relative to the original goals. While a portion of the PSUs are earned based on annual net product revenue achievements for each year during the performance period, these PSUs do not vest until the end of the performance period. Given the rapidly evolving nature of our business and revenue base, setting annual targets was deemed more appropriate than basing achievement on a pre-defined longer term revenue goal. Payouts may range from 0% to 140% of target depending on performance and are calculated by multiplying the sum of the achievement percentages for all goals over the performance period by the target number of PSUs subject to an award. The performance metrics for the PSUs granted in 2024 are set forth in the below table:

  43. 2024-2026 Special PSU Grants and Determination of Performance

    | 2024-2026 Rhythm Pharmaceuticals PSU Program Scorecard | | | | Milestone | Metric | Achievement Percentage | | 1.2024 Net Product Revenue | Threshold: $104 million | 0.00% | | | Tier 1 Target: $110 million | 6.66% | | | Tier 2 Target: $122 million | 13.33% | | | Maximum: $159 million | 26.66% | | 2.2025 Net Product Revenue | Threshold: $130 million | 0.00% | | | Tier 1 Target: $157 million | 6.66% | | | Tier 2 Target: $185 million | 13.33% | | | Maximum: $240.5 million | 26.66% | | 3.2026 Net Product Revenue | Threshold: | 0.00% | | | Target: | 13.34% | | | Maximum: | 26.68% | | 4.Hypothalamic Obesity Readout | Positive top line readout in hypothalamic obesity phase 3 clinical trial no later than June 30, 2025 | 20.00% | | 5.Hypothalamic Obesity Approval | FDA approval for New Drug Application for Setmelanotide for the treatment of hypothalamic obesity no later than June 30, 2026 | 20.00% | | 6.Progress Next Generation Programs | Completion phase 1/2 RM-718 study no later than June 30, 2025 | 10.00% | | | Completion of phase 2 LB54640 hypothalamic obesity study no later than June 30, 2025 | 10.00% |

  44. 2024-2026 Special PSU Grants and Determination of Performance

    When the Compensation & Management Development Committee approved the PSU awards in April 2024, it elected to defer finalization of the 2025 Net Product Revenue and 2026 Net Product Revenue milestones. As a result and consistent with Financial Accounting Standards Codification Topic 718 (“ASC Topic 718”), a portion of the PSU awards was deemed for accounting purposes to have been granted in February 2025 when the 2025 Net Product Revenue milestones were established. The table below provides the target number of PSUs deemed granted to each of our NEOs.

  45. 2024-2026 Special PSU Grants and Determination of Performance

    | Name | Total Target Performance Units from FY 2024 Award | Number of Target Performance Units Granted in FY2024 | Number of Target Performance Units related to FY25 Revenue Goal(1) | Target Performance Units related to FY26 Revenue Goal(2) | | David P. Meeker | 50,000 | 36,665 | 6,665 | 6,670 | | Hunter C. Smith | 40,000 | 29,332 | 5,332 | 5,336 | | Jennifer Lee | 40,000 | 29,332 | 5,332 | 5,336 | | Yann Mazabraud | 40,000 | 29,332 | 5,332 | 5,336 | | Alastair Garfield | 40,000 | 29,332 | 5,332 | 5,336 |

  46. 2024-2026 Special PSU Grants and Determination of Performance

    (1)Represents the target number of PSU awards that, consistent with ASC 718, were deemed granted in February 2025 when the 2025 Net Product Revenue milestones were established. (2)Represents the target number of PSU awards that, consistent with ASC 718, are expected to be deemed granted upon achievement of the 2026 Net Product Revenue target approved in February 2026 by the Board of Directors. In February 2025, the Board reviewed the Company’s achievement against the 2024 Net Product Revenue milestone of $110 million. Based on actual 2024 net product revenue of $130.1 million, the Compensation & Management Development Committee recommended and the Board approved an achievement level of 121.9% of target, resulting in an achievement percentage of 16.25% for the 2024 Net Product Revenue Milestone (versus the target weight of 13.3%). In February 2026, the Board reviewed the Company’s achievement against the 2025 Net Product Revenue milestone of $185 million. Based on actual 2025 net product revenue of $191.0 million (at constant currency), the Compensation & Management Development Committee recommended and the Board approved an achievement level of 110.7% of target, resulting in a weighted achievement percentage of 14.8% for the 2025 Net Product Revenue Milestone (versus the target weight of 13.3%). Based on stockholder feedback, the Compensation & Management Development Committee will evaluate whether to grant additional performance-based equity awards as part of the annual long-term incentive program following completion of the performance period for the special 2024 PSUs.

  47. Severance and Change in Control Arrangements

    Severance and Change in Control Arrangements We are party to employment letter agreements with each of our NEOs. The employment letter agreements for our NEOs that are based in the United States provide for severance benefits and payments upon a termination without cause or for good reason, whereas Mr. Mazabraud’s employment letter agreement provides for a three-month notice period in the event of a termination of his employment other than a termination due to serious or gross misconduct plus severance benefits and payments per French law and mandatory industry-wide collective bargaining agreement provisions for the pharmaceutical industry in France. Our Compensation & Management Development Committee believes that these types of arrangements are necessary to attract and retain executive talent and are a customary component of executive compensation. In particular, such arrangements can mitigate a potential disincentive for our NEOs when they are evaluating a potential acquisition of the Company and can encourage retention through the conclusion of the transaction. The payments and benefits provided under our severance and change in control arrangements are designed to be competitive with market practices. A description of these arrangements, as well as information on the estimated payments and benefits that our NEOs would have been eligible to receive as of December 31, 2025, are set forth in “Potential Payments Upon Termination or Change in Control” below.

  48. Rhythm Non-Qualified Deferred Compensation Plan

    Rhythm Non-Qualified Deferred Compensation Plan In January 2026, Rhythm implemented a Non-Qualified Deferred Compensation Plan (NQDCP) for executives in order to attract and retain key employees. Under the NQDCP, participating executive employees have the opportunity to defer receipt of a portion of their salary, bonus, and other specified compensation, including equity awards.

  49. “Golden Parachute” Payments

    “Golden Parachute” Payments Sections 280G and 4999 of the Code provide that certain executive officers and other service providers who are highly compensated or hold significant equity interests may be subject to an excise tax if they receive payments or benefits in connection with a change in control of the Company that exceeds certain prescribed limits, and that we, or a successor, may forfeit a tax deduction on the amounts subject to this additional tax. While the Compensation & Management Development Committee may take the potential forfeiture of such tax deduction into account when making compensation decisions, it will award compensation that it determines to be consistent with the goals of our executive compensation program even if such compensation is not deductible by us. We do not provide any tax gross-ups to cover excise taxes under Section 4999 in connection with a change in control.

  50. Accounting for Share-Based Compensation

    Accounting for Share-Based Compensation We follow ASC Topic 718, for our share-based compensation awards. ASC Topic 718 requires companies to measure the compensation expense for all share-based payment awards made to employees and directors, including stock options and RSUs, based on the grant date “fair value” of these awards. This calculation is performed for accounting purposes and reported in the compensation tables below, even though our NEOs may never realize any value from their awards.

  51. 2025 Summary Compensation Table

    2025 Summary Compensation Table The following table contains information about the compensation earned by each of our NEOs during the fiscal years indicated.

  52. 2025 Summary Compensation Table

    | Name and Principal Position | Year | Salary ($) | Stock Awards ($)(1) | Option Awards ($)(2) | Non-Equity Incentive Plan Compensation ($)(3) | All Other Compensation ($)(4) | Total ($) | | David P. Meeker | 2025 | 807,692 | 8,274,785 | 7,688,560 | 759,488 | 14,000 | 17,544,525 | | Chief Executive Officer | 2024 | 740,472 | 4,382,955 | 8,784,115 | 744,135 | 12,065 | 14,663,742 | | | 2023 | 704,715 | 1,545,275 | 4,334,521 | 634,053 | 11,893 | 7,230,457 | | Hunter C. Smith | 2025 | 540,477 | 2,933,277 | 2,402,675 | 338,000 | 14,000 | 6,228,429 | | Chief Financial Officer | 2024 | 499,385 | 1,458,419 | 2,726,806 | 352,000 | 13,800 | 5,050,410 | | | 2023 | 476,154 | 538,795 | 1,011,892 | 261,000 | 20,166 | 2,308,007 | | Jennifer Lee | 2025 | 531,635 | 2,649,827 | 2,114,354 | 293,000 | 14,000 | 5,602,816 | | Executive Vice President, | 2024 | 488,266 | 1,241,807 | 2,272,339 | 302,000 | 13,800 | 4,318,212 | | Head of North America | 2023 | 464,731 | 493,668 | 885,405 | 246,000 | 16,406 | 2,106,210 | | Yann Mazabraud(5) | 2025 | 557,231 | 2,839,739 | 2,306,568 | 344,659 | 70,258(6) | 6,118,455 | | Executive Vice President, | 2024 | 467,224 | 1,999,709 | 2,272,339 | 297,075 | 58,923(7) | 5,095,270 | | Head of International | 2023 | 448,848 | 805,100 | — | 238,688 | 51,621(7) | 1,544,257 | | Alastair Garfield Chief Scientific Officer | 2025 | 505,385 | 2,082,927 | 1,537,712 | 287,000 | 14,000 | 4,427,024 |

  53. 2025 Summary Compensation Table

    ________________________________________________________ (1)Amounts reflect the full grant-date fair value of stock awards granted during fiscal 2025 computed in accordance with ASC Topic 718, rather than the amounts paid to or realized by the named individual. We provide information regarding the assumptions used to calculate the value of all stock awards made to our NEOs in Note 8 to the consolidated financial statements included in our Annual Report on Form 10-K for the year ended December 31, 2025. The amounts shown in the table also include the portion of PSUs granted in 2024 that vest based on achievement of 2025 Net Product Revenue milestones that were established at the beginning of 2025. Consistent with ASC Topic 718, those PSUs were deemed for accounting purposes to have been granted in February 2025 when the milestones were established. As required pursuant to SEC disclosure rules, the grant-date fair values of the PSUs were computed based on the probable outcomes of the performance conditions as of the grant date. Assuming maximum achievement of the performance conditions, the value of the PSUs as of the grant date is $755,678 for Dr. Meeker, and $604,542 for Mr. Smith, Ms. Lee, Mr. Mazabraud, and Mr. Garfield. (2)Amounts reflect the full grant-date fair value of stock options granted during fiscal 2025 computed in accordance with ASC Topic 718, rather than the amounts paid to or realized by the named individual. We provide information regarding the assumptions used to calculate the value of all stock options granted to our NEOs in Note 8 to the consolidated financial statements included in our Annual Report on Form 10-K for the year ended December 31, 2025. (3)Amounts for fiscal 2025 represent payments earned by our NEOs based upon the achievement of certain company and individual performance objectives for fiscal 2025. Please see the description of the annual bonus program under “Cash Incentive Compensation” in the CD&A above. (4)Other than for Mr. Mazabraud, amounts for fiscal 2025 represent employer matching contributions to our 401(k) plan. (5)Amounts for 2025 shown for Mr. Mazabraud have been converted from Euros to U.S. dollars based on an exchange rate of 1.17631 as of December 31, 2025. (6)Amount includes the aggregate amount of payments made in respect of Mr. Mazabraud’s work-from-home allowance of $9,411 and employer contributions to statutory pension on his behalf of $60,847. (7)The amounts shown in the “All Other Compensation” column for fiscal years 2024 and 2023, and the corresponding “Total” columns for Mr. Mazabraud for such years, have been updated to reflect the removal of non-compensatory social insurance contributions in the amount of $526,353 in 2024 and $197,752 in 2023. Grants of Plan-Based Awards in Fiscal 2025 The following table provides supplemental information relating to grants of plan-based awards made during fiscal 2025 to help explain information provided above in our Summary Compensation Table. This table presents information regarding all grants of plan-based awards occurring during fiscal year 2025.

  54. 2025 Summary Compensation Table

    | | | Estimated Future Payouts Under Non-Equity Incentive Plan Awards(1) | | | Estimated Future Payouts Under Equity Incentive Plan Awards (2) | | | All Other Stock Awards: Number of Shares of Stock or Units(#) | All Other Option Awards: Number of Securities Underlying Options(#) | Exercise or Base Price of Option Awards ($/Sh) | Grant Date Fair Value of Stock and Option Awards ($) (3) | | | Grant Date | Threshold ($) | Target ($) | Maximum ($) | Threshold (#) | Target (#) | Maximum (#) | | | | | | David P. Meeker | — | — | 588,750 | 963,195 | — | — | — | — | — | — | — | | | 2/14/2025 | — | — | — | — | — | — | — | 200,000 | $56.69 | $7,688,560 | | | 2/14/2025 | — | — | — | — | — | — | 133,350 | — | — | $7,559,612 | | | 2/14/2025 | — | — | — | — | 6,665 | 13,330 | — | — | — | $715,173 | | Hunter C. Smith | — | — | 262,100 | 428,796 | — | — | — | — | — | — | — | | | 2/14/2025 | — | — | — | — | — | — | — | 62,500 | $56.69 | $2,402,675 | | | 2/14/2025 | — | — | — | — | — | — | 41,650 | — | — | $2,361,139 | | | 2/14/2025 | — | — | — | — | 5,332 | 10,664 | — | — | — | $572,139 | | Jennifer Lee | — | — | 232,425 | 380,247 | — | — | — | — | — | — | — | | | 2/14/2025 | — | — | — | — | — | — | — | 55,000 | $56.69 | $2,114,354 | | | 2/14/2025 | — | — | — | — | — | — | 36,650 | — | — | $2,077,689 | | | 2/14/2025 | — | — | — | — | 5,332 | 10,664 | — | — | — | $572,139 | | Yann Mazabraud | — | — | 252,760 | 413,515 | — | — | — | — | — | — | — | | | 2/14/2025 | — | — | — | — | — | — | — | 60,000 | $56.69 | $2,306,568 | | | 2/14/2025 | — | — | — | — | — | — | 40,000 | — | — | $2,267,600 | | | 2/14/2025 | — | — | — | — | 5,332 | 10,664 | — | — | — | $572,139 | | Alastair Garfield | | — | 222,750 | 364,419 | — | — | — | — | — | — | $— | | | 2/14/2025 | — | — | — | — | — | — | — | 40,000 | 56.69 | $1,537,712 | | | 2/14/2025 | — | — | — | — | — | — | 26,650 | — | — | 1,510,789 | | | 2/14/2025 | — | — | — | — | 5,332 | 10,664 | — | — | $— | $572,139 |

  55. 2025 Summary Compensation Table

    ________________________________________________________ (1)Amounts reflect potential payouts under our 2025 annual bonus program. The target amount is based on a percentage of the individual’s fiscal year 2025 base salary. Please see the description of the annual bonus program under “Cash Incentive Compensation” in the CD&A above. The maximum amount reflects the maximum corporate achievement opportunity of 148% for 2025 for the corporate achievement component and assumes a 200% cap on the individual achievement component. (2)PSUs are eligible to vest upon the achievement of financial, commercial and strategic performance goals and are reflected in the table based on the probable outcomes of the performance conditions as of the grant date. (3)Amounts reflect the full grant-date fair value of stock options, RSUs and PSUs granted during fiscal year 2025 computed in accordance with ASC Topic 718, rather than the amounts paid to or realized by the named individual. We provide information regarding the assumptions used to calculate the value of all stock awards made to our NEOs in Note 8 to the consolidated financial statements included in our Annual Report on Form 10-K for the year ended December 31, 2025. When the Compensation & Management Development Committee approved the PSU awards in April 2024, it elected to defer finalization of the 2025 Net Product Revenue and 2026 Net Product Revenue milestones. As a result and consistent with ASC Topic 718, a portion of the PSU awards was deemed for accounting purposes to have been granted in February 2025 when the 2025 Net Product Revenue Milestones were established. The grant date fair value for the PSUs was calculated based on the probable outcome of the performance condition as of the grant date.

  56. NARRATIVE TO SUMMARY COMPENSATION TABLE AND GRANTS OF PLAN-BASED AWARDS TABLE

    NARRATIVE TO SUMMARY COMPENSATION TABLE AND GRANTS OF PLAN-BASED AWARDS TABLE

  57. Outstanding Equity Awards at 2025 Fiscal Year-End Table

    Outstanding Equity Awards at 2025 Fiscal Year-End Table The following table summarizes the number of shares of Common Stock underlying outstanding equity incentive plan awards for each NEO as of December 31, 2025.

  58. Outstanding Equity Awards at 2025 Fiscal Year-End Table

    | | Option Awards | | | | | Stock Awards | | | | | Name | Vesting Commencement Date | Number of Securities Underlying Unexercised Options (#) Exercisable | Number of Securities Underlying Unexercised Options (#) Unexercisable | Option Exercise Price ($) | Option Expiration Date | Number of Shares or Units of Stock That Have Not Vested (#) | Market Value of Shares or Units of Stock That Have Not Vested ($)(1) | Equity Incentive Plan Awards: Number of Unearned Shares, Units or Other Rights That Have Not Vested (#) | Equity Incentive Plan Awards: Market or Payout Value of Unearned Shares, Units or Other Rights That Have Not Vested ($) (1) | | David P. Meeker, M.D. | 1/6/2017(2) | 65,430 | | $6.05 | 02/7/2027 | | | | | | | 6/6/2018(3) | 10,000 | | $34.89 | 06/06/2028 | | | | | | | 6/19/2019(3) | 15,000 | | $22.93 | 06/19/2029 | | | | | | | 6/17/2020(3) | 18,500 | | $21.37 | 06/17/2030 | | | | | | | 7/20/2020(4) | 900,000 | | $22.53 | 07/17/2030 | | | | | | | 2/11/2021(5) | 220,000 | | $30.66 | 02/11/2031 | | | | | | | 2/9/2022(5) | 478,125 | 31,875 | $6.80 | 02/9/2032 | | | | | | | 2/1/2023(5) | 157,850 | 71,750 | $27.35 | 02/01/2033 | | | | | | | 2/1/2023(6) | | | | | 28,250 | 3,023,880 | | | | | 2/16/2024(5) | 113,312 | 145,688 | $49.23 | 2/16/2034 | | | | | | | 2/16/2024(6) | | | | | 63,750 | 6,823,800 | | | | | 4/26/2024(8) | | | | | | | 43,330 | 4,638,043 | | | 2/14/2025(5) | 37,500 | 162,500 | $56.69 | 2/14/2035 | | | | | | | 2/14/2025(6) | | | | | 133,350 | 14,273,784 | | | | Hunter C. Smith | 2/14/2018(5) | 61,000 | | $25.79 | 02/14/2028 | | | | | | | 2/13/2019(5) | 70,000 | | $29.78 | 02/13/2029 | | | | | | | 2/14/2020(5) | 82,000 | | $17.87 | 02/14/2030 | | | | | | | 7/17/2020(5) | 25,000 | | $22.53 | 07/17/2030 | | | | | | | 2/11/2021(5) | 56,250 | | $30.66 | 02/11/2031 | | | | | | | 2/9/2022(5) | 91,406 | 6,094 | $6.80 | 02/9/2032 | | | | | | | 2/9/2022(6) | | | | | 4,062 | 434,796 | | | | | 2/1/2023(5) | 36,850 | 16,750 | $27.35 | 02/1/2033 | | | | | | | 2/1/2023(6) | | | | | 9,850 | 1,054,344 | | | | | 2/16/2024(5) | 35,175 | 45,225 | $49.23 | 2/16/2034 | | | | | | | 2/16/2024(6) | | | | | 19,800 | 2,119,392 | | | | | 4/26/2024(8) | | | | | | | 34,664 | 3,710,435 | | | 2/14/2025(5) | 11,719 | 50,781 | $56.69 | 02/14/2035 | | | | | | | 2/14/2025(6) | | | | | 41,650 | 4,458,216 | | | | Yann Mazabraud | 10/5/2020(7) | 25,000 | | $22.28 | 10/5/2030 | | | | | | | 2/11/2021(5) | 48,750 | | $30.66 | 02/11/2031 | | | | | | | 2/9/2022(5) | 77,344 | 5,156 | $6.80 | 02/9/2032 | | | | | | | 4/14/2023(6) | | 20,750 | | | 20,750 | 2,221,080 | | | | | 2/16/2024(5) | 29,313 | 37,687 | $49.23 | 02/16/2034 | | | | | | | 2/16/2024(6) | | | | | 16,500 | 1,766,160 | | | | | 4/15/2024(6) | | | | | 7,500 | 802,800 | | | | | 4/26/2024(8) | | | | | | | 34,664 | 3,710,435 | | | 2/14/2025(5) | 11,250 | 48,750 | $56.69 | 02/14/2035 | | | | | | | 2/14/2025(6) | | | | | 40,000 | 4,281,600 | | | | Jennifer Lee | 11/9/2020(7) | 71,283 | | $24.29 | 11/9/2030 | | | | |

  59. Outstanding Equity Awards at 2025 Fiscal Year-End Table

    | | 2/11/2021(5) | 37,108 | | $30.66 | 02/11/2031 | | | | | | | 2/9/2022(5) | 16,445 | 5,156 | $6.80 | 02/9/2032 | | | | | | | 2/9/2022(6) | | | | | 3,437 | 367,896 | | | | | 2/1/2023(5) | 32,244 | 14,656 | $27.35 | 02/1/2033 | | | | | | | 2/1/2023(6) | | | | | 9,024 | 965,929 | | | | | 2/16/2024(5) | 29,312 | 37,688 | $49.23 | 02/16/2034 | | | | | | | 2/16/2024(6) | | | | | 16,500 | 1,766,160 | | | | | 4/26/2024(8) | | | | | | | 34,664 | 3,710,435 | | | 2/14/2025(5) | 10,312 | 44,688 | $56.69 | 02/14/2035 | | | | | | | 2/14/2025(6) | | | | | 36,650 | 3,923,016 | | | | Alastair Garfield | 7/1/2024(5) | 16,666 | 36,667 | $43.76 | 07/8/2034 | | | | | | | 7/1/2024(6) | | | | | 13,333 | 1,427,164 | | | | | 7/1/2024(8) | | | | | | | 34,664 | 3,710,435 | | | 2/14/2025(5) | 7,500 | 32,500 | $56.69 | 2/14/2035 | | | | | | | 2/14/2025(6) | | | | | 26,650 | 2,852,616 | | |

  60. Outstanding Equity Awards at 2025 Fiscal Year-End Table

    (1)Amounts are calculated based on multiplying the number of shares shown in the table by the per share closing price of our Common Stock on December 31, 2025 (the last trading day of 2025), which was $107.04. (2)These options vest in 3 equal annual installments following the vesting commencement date, except that the last installment, if necessary, may be smaller. (3)These options vest in full upon the earlier of the first anniversary of the date of grant or the date of the next annual meeting of stockholders. (4)25% of the shares subject to these options vest on the first anniversary of the vesting commencement date, and the remaining shares vest in 36 monthly installments thereafter, except that the last installment, if necessary, may be smaller. (5)These options vest in 16 equal quarterly installments following the vesting commencement date, except that the last installment, if necessary, may be smaller. (6)These RSUs will vest in 4 equal annual installments beginning on the first anniversary of the vesting commencement date. In December 2025, to simplify administration of the Company’s equity incentive programs, all outstanding Company RSUs were amended so that vesting of any portion of an award that was otherwise scheduled to vesting during a given month was accelerated to the first day of the applicable month. (7)25% of the shares subject to this option vest on the first anniversary of the vesting commencement date, and the remaining shares vest in 12 quarterly installments thereafter, except that the last installment, if necessary, may be smaller. (8)These PSUs are eligible to vest upon the Compensation & Management Development Committee’s determination of performance following the end of the performance period ending on December 31, 2026, subject to continued service through such determination date. For additional information about the PSUs, see the section of our CD&A above titled “2024-2026 Special PSU Grants and Determination of Performance”.

  61. Option Exercises and Stock Vested in Fiscal 2025

    Option Exercises and Stock Vested in Fiscal 2025 The following table shows the stock options exercised and the stock awards vested during 2025.

  62. Option Exercises and Stock Vested in Fiscal 2025

    | | Option Awards | | Stock Awards | | | Name | Number of Shares Acquired on Exercise (#) | Value Realized on Exercise(1) ($) | Number of Shares Acquired on Vesting (#) | Value Realized on Vesting(2) ($) | | David P. Meeker | 43,620 | $2,637,265 | 35,375 | $2,062,104 | | Hunter C. Smith | 100,594 | $7,303,001 | 17,930 | $1,044,839 | | Jennifer Lee | 4,189 | $218,289 | 15,481 | $902,549 | | Yann Mazabraud | 75,000 | $2,190,000 | 27,280 | $1,648,614 | | Alastair Garfield | — | $— | 4,445 | $290,881 |

  63. Option Exercises and Stock Vested in Fiscal 2025

    ________________________________________________________ (1)Calculated by multiplying (i) the number of shares exercised by (ii) the difference between the closing price of the Company’s Common Stock on the date of exercise and the exercise price of the option. (2)Calculated by multiplying the shares vested by the closing prices of the Company’s Common Stock on the applicable dates of vesting.

  64. Potential Payments Upon Termination or Change in Control

    Potential Payments Upon Termination or Change in Control In accordance with SEC rules, the following table summarizes the payments that would be made to certain of our NEOs upon the occurrence of certain qualifying terminations of employment, assuming such NEO’s termination of employment with the Company occurred on December 31, 2025 and, where relevant, that a change in control of the Company occurred on December 31, 2025. Amounts shown in the table below do not include (1) accrued but unpaid salary and (2) other benefits earned or accrued by the named executive officers during the NEO’s employment that are available to all salaried employees, such as accrued vacation. We have entered into certain letter agreements with each of our NEOs that provide our NEOs with severance protections. The letter agreements provide that the NEOs will be eligible for severance benefits in certain circumstances following a termination of employment without cause or with good reason, whether or not in connection with a change in control. Additionally, the PSUs granted in 2024 provide for accelerated vesting upon a change in control, with such PSUs vesting at 100% or such greater percentage if the Committee determines that sufficient milestones have been attained or that it is probable such milestones will be attained. Under the employment letter agreements for the NEOs, other than Mr. Mazabraud, in the event that such executive’s employment is terminated by the Company without “cause,” or by the executive for “good reason” (each, as defined in the applicable agreement, and referred to herein as a qualifying termination), then such executive will be entitled to the severance payments and benefits described below. If Dr. Meeker experiences a qualifying termination of employment, then, in addition to any accrued amounts, he will be entitled to receive 12 months of continued base salary, paid in substantially equal installments over a period of 12 months, and reimbursement for continued healthcare benefits for up to 12 months following the date of termination. However, in the event that Dr. Meeker experiences a qualifying termination of employment within three months prior to, or within 12 months following a change in control of the Company (referred to herein as the Change in Control Period), then Dr. Meeker will be entitled to receive (i) a cash severance amount equal to (A) 18 months of his then-current base salary and (B) 150% of his then-current target annual bonus, paid in substantially equal installments over a period of 18 months (unless the Company elects to pay such amount in lump sum), (ii) reimbursement for continued healthcare benefits for up to 18 months following the date of termination and (iii) accelerated vesting of each outstanding and unvested equity award held by him. If Messrs. Smith or Garfield or Ms. Lee experiences a qualifying termination of employment, then, in addition to any accrued amounts, such individual will be entitled to receive 9 months of continued base salary, paid in substantially equal installments over a period of 9 months, and reimbursement for continued healthcare benefits for up to 9 months following the date of termination. However, in the event that Messrs. Smith or Garfield or Ms. Lee experiences a qualifying termination of employment within the Change in Control Period, such individual will be entitled to receive (i) a cash severance amount equal to (A) 12 months of his then-current base salary and (B) 100% of such individual’s then-current target bonus, payable over 12 months following such termination, (ii) reimbursement for continued healthcare benefits for up to 12 months following the date of termination and (iii) accelerated vesting of each outstanding and unvested equity award held by such individual. If Mr. Mazabraud experiences a termination of employment that is not due to serious misconduct (“faute grave”) or gross misconduct (“faute lourde”), Mr. Mazabraud is entitled to a three-month notice period or three months’ compensation in lieu of notice. Notwithstanding the accelerated vesting in the event of a qualifying termination of employment within the Change in Control Period described above, the PSUs granted in 2024 provide that in the event the individual experiences a qualifying termination of employment within three months prior to a change in control, the PSUs will remain outstanding and eligible to vest in connection with such change in control. All severance payments and benefits under the letter agreements are subject to the NEO’s execution of a release of claims against us and continued compliance with certain restrictive covenants. The letter agreements also include a Section 280G “best pay” provision, which provides that if any amount received by the NEO pursuant to the agreement or otherwise that would be subject to the excise tax imposed by Section 4999 of the Code, the NEO would receive the full amount of the payments and benefits or an amount reduced so that no portion would be subject to the excise tax, whichever would result in the largest payment to the NEO on an after-tax basis.

  65. Estimated Potential Payments

    Estimated Potential Payments

  66. Estimated Potential Payments

    | Name | Benefit | Termination Without Cause or for Good Reason / Cause (no Change in Control) ($) | Change in Control (no Termination) ($)(1) | Termination Without Cause or for Good Reason / Cause in Connection with a Change in Control ($) | | David P. Meeker | Cash | 785,000 | — | 2,060,625 | | | Equity Acceleration(2) | — | 5,584,491 | 49,638,470 | | | Continued Healthcare | 35,031 | — | 52,546 | | | Total(3) | 820,031 | 5,584,491 | 51,751,641 | | Hunter C. Smith | Cash | 393,150 | — | 786,300 | | | Equity Acceleration | — | 4,467,528 | 15,183,699 | | | Continued Healthcare | 26,213 | — | 34,950 | | | Total(3) | 419,363 | 4,467,528 | 16,004,949 | | Jennifer Lee | Cash | 387,375 | — | 748,925 | | | Equity Acceleration | — | 4,467,528 | 13,136,560 | | | Continued Healthcare | 17,605 | — | 23,473 | | | Total(3) | 404,980 | 4,467,528 | 13,908,958 | | Yann Mazabraud | Cash | 421,266 | — | 814,448 | | | Equity Acceleration | — | 4,467,528 | 14,221,725 | | | All Other Payments or Benefits | — | — | — | | | Total(3) | 421,266 | 4,467,528 | 15,036,173 | | Alastair Garfield | Cash | 371,250 | — | 717,750 | | | Equity Acceleration | — | 4,467,528 | 8,236,443 | | | Continued Healthcare | 9,210 | — | 12,280 | | | Total(3) | 380,460 | 4,467,528 | 8,966,473 |

  67. Estimated Potential Payments

    (1)Under the award agreements for the 2024 PSU awards, the PSUs would have vested upon a change in control occurring on December 31, 2025 at 100% of the PSUs or a greater percentage if the Compensation & Management Development Committee determined that sufficient milestones have been attained or that it was probable such milestones will be attained. In addition, under our 2017 Plan, to the extent the surviving entity declined to continue, convert, assume or replace outstanding awards in the change in control, all outstanding options and RSUs would have vested upon the change in control. The amounts shown in this column assume that outstanding options and RSUs would be continued by the surviving entity and, accordingly, no value has been included for them in this column. For PSUs, the amount shown was calculated by multiplying the number of outstanding PSUs by $107.04, the closing trading price of our Common Stock on December 31, 2025. (2)With respect to options, the value of equity acceleration was calculated by multiplying (i) the number of accelerated shares of Common Stock underlying the options by (ii) the excess, if any, of $107.04, the closing trading price of our Common Stock on December 31, 2025, over the exercise price for the options. With respect to RSUs and outstanding PSUs, the value of equity acceleration was calculated by multiplying the number of accelerated RSUs and/or PSUs, as applicable, by $107.04, the closing trading price of our Common Stock on December 31, 2025. The PSUs would remain outstanding and eligible to vest in connection a change in control if the NEO experiences a qualifying termination within three months prior to the change in control. (3)Amounts shown are the maximum potential payment the NEO would have received as of December 31, 2025. Amounts of any reduction pursuant to the 280G best pay provision, if any, would be calculated upon actual termination of employment.

  68. CEO Pay Ratio

    CEO Pay Ratio As required by Section 953(b) of the Dodd-Frank Wall Street Reform and Consumer Protection Act and Item 402(u) of Regulation S-K, we are providing the following information regarding the ratio of our Chief Executive Officer’s annual total compensation to the annual total compensation of our median employee. The annual total compensation for 2025 for our Chief Executive Officer was $17,544,525, as reported in the Summary Compensation Table. The annual total compensation for 2025 for our median employee, identified as discussed below, was $484,876, calculated using the methodology below. Based on this information, for 2025, the ratio of the annual total cash compensation of our Chief Executive Officer to the median of the annual total compensation of our other employees was approximately 36 to 1.

  69. Methodology, Assumptions and Estimates Used in Determining our Pay Ratio Disclosure

    Methodology, Assumptions and Estimates Used in Determining our Pay Ratio Disclosure We analyzed fiscal year 2025 data to identify the median employee from our entire employee population (other than our Chief Executive Officer), whether employed on a full-time or part-time basis, as of December 31, 2025, which consisted of 400 employees. Within this group, the median employee was identified using a consistently applied compensation measure (“CACM”) of total cash and equity compensation received, which we calculated by aggregating all cash compensation received and equity compensation granted during the fiscal year ending December 31, 2025, for each employee consisting of: (1) base pay, (2) bonuses, (3) commissions, and (4) the grant date fair value of equity awards granted in 2025. In identifying the median employee, we converted compensation amounts paid in foreign currencies based on the applicable year-to-date average exchange rate as of December 31, 2025 and annualized the compensation values of individuals that joined our Company during 2025. We did not exclude workers in non-U.S. countries and did not make any cost-of-living adjustments. Once the median employee was identified, we calculated the median employee’s annual target total direct compensation in accordance with the requirements of the Summary Compensation Table. This information is being provided for compliance purposes and should be viewed as a reasonable estimate calculated in a manner consistent with the SEC rules, based on our internal records and the methodology described above. The SEC rules for identifying the median compensated employee allow companies to adopt a variety of methodologies, to apply certain exclusions and to make reasonable estimates and assumptions that reflect their employee populations and compensation practices. Accordingly, the pay ratio reported by other companies may not be comparable to the pay ratio reported above, as other companies have different employee populations and compensation practices and may use different methodologies, exclusions, estimates and assumptions in calculating their own pay ratios. Neither the Compensation & Management Development Committee nor management of the Company used the CEO Pay Ratio measure in making compensation decisions for 2025.

  70. Pay Versus Performance

    Pay Versus Performance In accordance with the SEC’s disclosure requirements regarding pay versus performance, this section presents the SEC-defined “Compensation Actually Paid,” or CAP. Also required by the SEC, this section compares CAP to various measures used to gauge our performance. CAP is a supplemental measure to be viewed alongside performance measures as an addition to the philosophy and strategy of compensation-setting discussed elsewhere in this proxy statement, not in replacement.

  71. 2025 Pay Versus Performance Table

    2025 Pay Versus Performance Table The following table sets forth information concerning the compensation of our NEOs for each of the fiscal years ended December 31, 2021, 2022, 2023, 2024 and 2025 and our financial performance for each such fiscal year:

  72. 2025 Pay Versus Performance Table

    | Year | Summary Compensation Table Total for PEO ($) | | Compensation Actually Paid to PEO ($)(1) | Average Summary Compensation Table Total for Non-PEO NEOs ($) | Average Compensation Actually Paid to Non-PEO NEOs ($)(1) | Value of Initial Fixed $100 Investment Based on: | | Net Income ($) | Total Revenues ($)(3) | | | | | | | | Total Stockholder Return ($) | Peer Group Total Stockholder Return ($)(2) | | | | 2025 | 17,544,525 | 17,544,525 | 54,157,885 | 5,594,181 | 14,695,734 | 360.00 | 93.71 | (196,539) | 189,757 | | 2024 | 14,663,742 | | 18,680,784 | 4,600,098 | 6,662,641 | 188.27 | 93.49 | (260,602) | 130,126 | | 2023 | 7,230,457 | | 19,005,711 | 1,974,174 | 4,813,432 | 154.61 | 94.03 | (184,678) | 77,428 | | 2022 | 3,325,758 | | 24,142,912 | 1,110,455 | 4,805,393 | 97.94 | 89.90 | (181,119) | 23,638 | | 2021 | 6,458,360 | | (10,017,699) | 2,069,493 | (775,040) | 33.57 | 100.02 | (69,612) | 3,154 |

  73. 2025 Pay Versus Performance Table

    _______________________________________________________ (1)The significant variability in Compensation Actually Paid across periods is primarily driven by changes in the fair value of equity awards, which are sensitive to stock price performance and vesting conditions. Amounts represent compensation actually paid to our PEO and the average compensation actually paid to our remaining NEOs for the relevant fiscal year, as determined under SEC rules (and described in Note 2 below), which includes the individuals indicated in the table below for each fiscal year:

  74. 2025 Pay Versus Performance Table

    | Year | PEO | Non-PEO NEOs | | 2025 | David P. Meeker | Hunter C. Smith, Jennifer Lee, Yann Mazabraud and Alastair Garfield | | 2024 | David P. Meeker | Hunter C. Smith, Jennifer Lee, Yann Mazabraud and Joseph Shulman | | 2023 | David P. Meeker | Hunter C. Smith, Jennifer Lee, Yann Mazabraud and Joseph Shulman | | 2022 | David P. Meeker | Hunter C. Smith, Jennifer Lee, Yann Mazabraud and Joseph Shulman | | 2021 | David P. Meeker | Hunter C. Smith, Murray Stewart, Jennifer Lee, Yann Mazabraud and Joseph Shulman |

  75. 2025 Pay Versus Performance Table

    The 2025 Summary Compensation Table totals reported for the PEO and the average of the Other NEOs for each year were subject to the following adjustments per Item 402(v)(2)(iii) of Regulation S-K to calculate “compensation actually paid”:

  76. 2025 Pay Versus Performance Table

    | | 2025 | | | | PEO | Average for Other NEO’s | | Summary Compensation Table Total | 17,544,525 | 5,594,181 | | ADJUSTMENTS(a): | | | | Deduction for Amounts reported under the “Stock Awards” and “Option Awards” Columns in the Summary Compensation Table(a) | (15,963,345) | (4,716,770) | | Increase based on ASC 718 Fair Value of Awards Granted during Applicable FY that Remain Unvested as of Applicable FY End, determined as of Applicable FY End | 27,463,032 | 7,464,689 | | Increase based on ASC 718 Fair Value of Awards Granted during Applicable FY that Vested during Applicable FY, determined as of Vesting Date | 2,236,519 | 608,054 | | Increase/deduction for Awards Granted during Prior FY that were Outstanding and Unvested as of Applicable FY End, determined based on change in ASC 718 Fair Value from Prior FY End to Applicable FY End | 17,766,871 | 4,961,562 | | Increase/deduction for Awards Granted during Prior FY that Vested During Applicable FY, determined based on change in ASC 718 Fair Value from Prior FY End to Vesting Date | 5,110,282 | 784,018 | | Deduction of ASC 718 Fair Value of Awards Granted during Prior FY that were Forfeited during Applicable FY, determined as of Prior FY End | — | — | | Increase based on Dividends or Other Earnings Paid during Applicable FY prior to Vesting Date | — | — | | Increase based on Incremental Fair Value of Options/SARs Modified during Applicable FY | — | — | | Adjustments Related to Defined Benefit and Actuarial Pension Plans | — | — | | Compensation Actually Paid | 54,157,885 | 14,695,734 |

  77. 2025 Pay Versus Performance Table

    Measurement date equity fair values are calculated with assumptions derived on a basis consistent with those used for grant date fair value purposes. RSUs and PSUs are valued based on the closing stock price on the relevant measurement date. Stock options are valued using a Black-Scholes model as at the relevant measurement dates. (2)For the relevant fiscal year, represents the cumulative TSR (the “Peer Group TSR”) of the NASDAQ Biotechnology Index (the “Peer Group”). The dollar amounts reported represent the amount of net loss reflected in the Company's audited financial statements for the applicable fiscal year. (3)Revenue is the Company’s selected GAAP measure.

  78. Narrative Disclosure to Pay Versus Performance Table

    Narrative Disclosure to Pay Versus Performance Table

  79. Pay Versus Performance Tabular List

    Pay Versus Performance Tabular List We believe the following performance measures represent the most important financial performance measures used by us to link compensation actually paid to our NEOs for the fiscal year ended December 31, 2025: •Total revenues; •Total stockholders return; and •Cash used in operations.