Ascendis Pharma A/S 6-K, filed Apr 29, 2025
Ascendis Pharma A/S (ASND)
About this 6-K
Ascendis Pharma A/S filed this 6-K on April 29, 2025.
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 6-K
REPORT OF FOREIGN PRIVATE ISSUER
PURSUANT TO SECTION 13a-16 OR 15d-16
UNDER THE SECURITIES EXCHANGE ACT OF 1934
For the month of April, 2025
Commission File Number: 001-36815
Ascendis Pharma A/S
(Exact Name of Registrant as Specified in Its Charter)
Tuborg Boulevard 12
DK-2900 Hellerup
Denmark
(Address of principal executive offices)
Indicate by check mark whether the registrant files or will file annual reports under cover of Form 20-F or Form 40-F.
Form 20-F ☒ Form 40-F ☐
Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(1): ☐
Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(7): ☐
Furnished as Exhibit 99.1 to this Report on Form 6-K is the convening notice for the Annual General Meeting of Ascendis Pharma A/S (the “Company”), providing notice to the Company’s shareholders of the Company’s Annual General Meeting to be held on May 27, 2025 at 2:00 pm CET.
Furnished as Exhibit 99.2 to this Report on Form 6-K is an annual report of the Company for the year ended December 31, 2024, prepared in accordance with the disclosure requirements of the Danish Financial Statements Act.
Furnished as Exhibit 99.3 to this Report on Form 6-K is the Sustainability and P-ESG Report of the Company for the year ended December 31, 2024, prepared in accordance with Section 99a (CSR) and Section 99b (Diversity) of the Danish Financial Statements Act.
Exhibits
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| Ascendis Pharma A/S | ||||||
| Date: April 29, 2025 | By: | /s/ Michael Wolff Jensen | ||||
| Michael Wolff Jensen | ||||||
| Executive Vice President, Chief Legal Officer | ||||||
Exhibit 99.1
Notice to convene Annual General Meeting 2025
Notice is hereby given that the annual general meeting of Ascendis Pharma A/S (the “Company”) will be held on:
May 27th, 2025 at 2:00 pm CET
The annual general meeting will be held at:
Mazanti-Andersen, Amaliegade 10, DK-1256 Copenhagen K, Denmark
The agenda for the annual general meeting is as follows:
| 1. | Election of Chairman of the Meeting |
| 2. | Report on the Company’s Activities during the Past Year |
| 3. | Presentation of Audited Annual Report with Auditor’s Statement for Approval and Discharge of the Board of Directors and Management |
| 4. | Resolution on Application of Profits or Covering of Losses as per the Adopted Annual Report |
| 5. | Election of Board Members |
| 6. | Election of State-authorized Public Auditor |
| 7. | Other proposals from the Board of Directors and/or Shareholders |
Complete Proposals
Re 1
The board of directors proposes that attorney-at-law Lars Lüthjohan is elected as chairman of the general meeting.
Re 2
Chairman of the board of directors, Albert Cha, and Chief Executive Officer, Jan Møller Mikkelsen or Chief Legal Officer, Michael Wolff Jensen will report on the Company’s activities for the year ended December 31, 2024.
Re 3
The board of directors recommends that the audited annual report (including Sustainability and P-ESG report) will be adopted and that a resolution be passed to discharge the board of directors and management from liability.
Re 4
The board of directors proposes that the consolidated loss for the year of EUR 378.1 million be carried forward to next year through recognition in accumulated deficit.
Re 5
All members of the board of directors are up for election.
Currently, the board of directors is composed of the following members:
Albert Cha
Lars Holtug
Jan Møller Mikkelsen
Lisa Jane Morrison
William Carl Fairey Jr
Siham Imani
The board of directors proposes that all current members of the board of directors are re-elected. All current members have accepted to stand for reelection in accordance with the above. Information about the current board members is available on the Company’s website www.ascendispharma.com.
Re 6
The board of directors proposes that Deloitte Statsautoriseret Revisionspartnerselskab be appointed as the Company’s auditor in respect of statutory financial and sustainability reporting.
Re 7
7.a Proposal from the board of directors
The current authorization contained in article 4 d (2) to the board of directors to increase the Company’s share capital by up to 3,825,000 shares without pre-emptive subscription rights for the Company’s shareholders expires 27 May 2026. The board of directors proposes that the authorization is prolonged and adopted with the following wording:
The board of directors is until 27 May 2030 authorized at one or more times to increase the company’s share capital by up to nominal DKK 3,825,000 without pre-emptive subscription rights for the company’s shareholders. Capital increases according to this authorisation can be carried out by the board of directors by way of contributions in kind, conversion of debt and/or cash contributions and must be carried out at market price. The board of directors is authorised to make the required amendments to the articles of association if the authorization to increase the share capital is used and to cause such shares to be deposited with a depositary bank and the simultaneous issuance of American Depositary Shares representing such shares.
Reference is made to the existing article 4 d (3), which shall apply to shares issued pursuant to the above authorization if adopted, and shall remain unchanged with the following wording:
§ 4 d (3) For shares issued pursuant to article 4 d (1) or 4 d (2) the following shall apply: The new shares shall be non-negotiable instruments issued in the name of the holder and registered in the name of the holder in the company’s register of shareholders. The new shares shall not have any restrictions as to their transferability and no shareholder shall be obliged to have the shares re-deemed fully or partly. No partial payment is allowed. The shares shall be with the same rights as the existing share capital. The new shares shall give rights to dividends and other rights in the company from the time which are determined by the board of directors in connection with the decision to increase the share capital.
7.b Proposal from the board of directors
The board of directors proposes to amend the articles of association by renewing the authorisation to the board of directors to issue warrants.
The board of directors specifically proposes that the following wording is inserted as a new section 4h in the articles of association:
The board of directors is authorized, in accordance with the Danish Companies Act, Section 169, cf. Section 155, Subsection 2, during the period until 27 May 2030 on one or more occasions to issue warrants to new members of the board of directors of the company or its subsidiaries entitling the cumulative holders to subscribe shares for a total of up to nominal value of DKK 100,000 without pre-emptive rights for the company’s shareholders. The exercise price for the warrants shall be determined by the board of directors in consultation with the company’s advisors and shall at least be equal to the market price of the shares at the time of issuance. The warrants issued pursuant to this authorisation shall be governed by the terms and conditions set out in appendix 1a to articles of association. The board of directors shall determine the distribution of warrants, provided that this authorisation may only be exercised in connection with grants to new members of the board of directors in connection with their first election (i.e. not in connection with re-election for another term) and no single board member shall receive more than 10,000 warrants pursuant to this authorisation.
At the same time, the board of directors is authorized in the period until 27 May 2030, on one or more occasions to increase the company’s share capital by up to a total nominal value of DKK 100,000 without pre-emptive rights for the existing shareholders by cash payment in order to implement the capital increase related to exercise of the warrants. In accordance with this clause the board of directors may increase the share capital with a minimum nominal value of DKK 1 and a maximum nominal value of DKK 100,000. The board is authorized to cause such shares to be deposited with a depositary bank and the simultaneous issuance of American Depositary Shares.
The new shares issued based on exercise of warrants shall be non-negotiable instruments issued in the name of the holder and registered in the name of the holder in the Company’s register of shareholders. The new shares shall not have any restrictions as to their transferability and no shareholder shall be obliged to have the shares redeemed fully or partly. No partial payment is allowed. The shares shall be with the same rights as the existing share capital. The new shares shall give rights to dividends and other rights in the Company from the time which is determined by the board of directors in connection with the decision to increase the share capital.
7.c Proposal from the board of directors
Pursuant to article 12 of the articles of association of the Company, the Company is bound by the joint signature of either the Chairman of the board of directors and one member of the executive management, or by 3 members of the board of directors.
The board of directors proposes to amend article 12 of the articles of association by replacing the existing article 12 in its entirety with the following proposed updated article 12, which is in line with market practice for listed companies:
The company shall be bound by the chairman of the board of directors and one member of the executive management jointly, by two members of the executive management jointly, or by all members of the board of directors jointly.
The board of directors may issue individual or joint powers of procuration.
The proposed update to article 12 is motivated by the increased number of documents, applications and filings to be signed as part of the Company’s global commercial expansion.
—oo0oo—
The proposals contained in items 1, 3-6 may be adopted by a simple majority of the votes cast. The proposals contained in item 7 may be adopted by a majority of 2/3 of the votes cast and of the voting share capital represented at the general meeting.
The Company’s nominal share capital currently amounts to DKK 60,970,565 consisting of 60,970,565 shares of DKK 1 nominal value. At the general meeting, each share amount of DKK 1 nominal value carries one vote.
Information: The following information is available at the Company’s website www.ascendispharma.com as of April 29, 2025.
| • | Notice to convene the annual general meeting |
| • | The aggregate number of shares and voting rights as at the date of the notice to convene the general meeting |
| • | The documents that will be submitted at the general meeting, including the audited annual report |
| • | The agenda and the complete proposals for adoption |
| • | Forms for voting by proxy or by mail |
The convening notice will also be forwarded in writing to all shareholders recorded in the register of shareholders who have requested such notification.
Shareholders may submit questions to the Company in writing regarding the agenda and/or the documents prepared for the general meeting.
A shareholder’s right to attend general meetings and to vote at general meetings is determined on the basis of the shares that the shareholder owns on the registration date. The registration date is May 20, 2025. The shares which the individual shareholder owns are calculated on the registration date on the basis of the registration of ownership in the register of shareholders as well as notifications concerning ownership which the company has received with a view to update the ownership in the register of shareholders.
In addition, any shareholder who is entitled to attend a general meeting and who wishes to attend must have requested an admission card from the Company as described below.
Language: The meeting will be conducted in English according to section 7 of the articles of association.
Shareholders, proxies and any accompanying adviser must have an admission card to attend the general meeting. Admission cards may be ordered on the Company’s website, www.ascendispharma.com or on the website of Computershare A/S, www.computershare.dk.
Admission cards must be ordered no later than May 23, 2025 at 23.59 p.m. (CET).
Proxy: For the general meeting, shareholders may vote by proxy by presenting an instrument of proxy, duly signed and dated. Proxy forms can be downloaded from the website of the Company, www.ascendispharma.com, and must be forwarded to Computershare A/S, Lottenborgsvej 26 D, 1st floor, DK-2800 Kgs. Lyngby, Denmark, by mail or by fax no. + 45 45 46 09 98. Computershare must receive completed proxy forms no later than May 26, 2025 at 23.59 p.m. (CET).
Proxies may also be granted electronically on the Company’s website on the website of Computershare A/S, www.computershare.dk, by using a Computershare username and password. Usernames and passwords will be sent to all shareholders by email. Electronic proxies must be granted no later than May 26, 2025 at 23.59 p.m. (CET).
Voting by mail: Shareholders may - instead of voting in person at the ordinary general meeting - choose to vote by mail, i.e. voting in writing prior to the general meeting. Any shareholder who chooses to vote by mail shall send the absentee vote to Computershare A/S, Lottenborgsvej 26 D, 1st floor, DK-2800 Kgs. Lyngby, Denmark, by mail or by fax no. + 45 45 46 09 98.
Electronic voting: It is also possible to vote electronically on the website of Computershare A/S, www.computershare.dk, by using Computershare username and password.
In order to stay valid, the absentee vote, whether sent by mail or made electronically, must be received by Computershare A/S no later than May 26, 2025 at 10.00 a.m. (CET).Absentee voting forms can also be downloaded from the website of the Company, www.ascendispharma.com. Please note that an absentee vote cannot be withdrawn.
Please note that letters may be in the mail for several days or weeks.
Hellerup, April 29, 2025
On behalf of the board of directors
Albert Cha
Chair
Exhibit 99.2
Ascendis Pharma A/S
Tuborg Boulevard 12
DK-2900 Hellerup
Central Business Registration No. 29 91 87 91
Annual Report 2024
(January 1 – December 31)
Adopted at the Annual General Meeting of Shareholders on May 27, 2025.
Lars Lüthjohan Jensen
Chairman of the General Meeting
Contents
| Company Information |
3 | |||
| Statement by Management on the Annual Report |
4 | |||
| Independent Auditor’s Report |
5 | |||
| Management Commentary |
8 | |||
| Statements of Profit or Loss and Other Comprehensive Income for the Years Ended December 31 |
37 | |||
| Statements of Financial Position as of December 31 |
38 | |||
| Statements of Changes in Equity - Group |
40 | |||
| Statements of Changes in Equity - Parent |
41 | |||
| Cash Flow Statements for the Years Ended December 31 |
42 | |||
| Notes to the Financial Statements |
43 | |||
| Company Information | 3 |
Company Information
Ascendis Pharma A/S
Tuborg Boulevard 12
DK-2900 Hellerup
Central Business Registration No. 29 91 87 91
Registered in: Gentofte
Phone: +45 70 22 22 44
Internet: www.ascendispharma.com
E-mail: info@ascendispharma.com
Board of Directors
Albert Cha, Chairman
Lisa Jane Morrison
William Carl Fairey Jr.
Lars Holtug
Siham Imani
Jan Møller Mikkelsen
Executive Board
Jan Møller Mikkelsen, Chief Executive Officer
Michael Wolff Jensen, Chief Legal Officer
Scott Thomas Smith, Chief Financial Officer
Anni Lotte Kirstine Pedersen, Chief Administration Officer
External Auditors
Deloitte Statsautoriseret Revisionspartnerselskab
Weidekampsgade 6
DK-2300 Copenhagen S
| Statement by Management on the Annual Report | 4 |
Statement by Management on the Annual Report
The Board of Directors and the Executive Board have today considered and approved the annual report of Ascendis Pharma A/S for the financial year January 1 to December 31, 2024.
The annual report is presented in accordance with the IFRS Accounting Standards (“IFRS”), as issued by the International Accounting Standards Board (“IASB”), and as adopted by the European Union (“EU”). The financial statements include additional disclosures for reporting class C large sized enterprises as required by the Danish Executive Order on Adoption of IFRS as issued in accordance with the Danish Financial Statements Act.
In our opinion, the consolidated financial statements and the parent financial statements give a true and fair view of the Group’s and the Parent’s financial position at December 31, 2024, and of their financial performance and cash flows for the financial year January 1 to December 31, 2024.
We believe that the management commentary contains a fair review of the affairs and conditions referred to therein.
We recommend the annual report for adoption at the Annual General Meeting.
Hellerup, February 12, 2025
Executive Board
| Jan Møller Mikkelsen | Scott Thomas Smith | |
| Chief Executive Officer | Chief Financial Officer | |
| Michael Wolff Jensen | Anni Lotte Kirstine Pedersen | |
| Chief Legal Officer | Chief Administration Officer | |
Board of Directors
| Albert Cha Chairman |
William Carl Fairey Jr. | Lisa Jane Morrison | ||
| Siham Imani | Lars Holtug | Jan Møller Mikkelsen | ||
| Independent Auditor’s Report | 5 |
Independent Auditor’s Report
To the shareholders of Ascendis Pharma A/S
Opinion
We have audited the consolidated financial statements and the parent financial statements of Ascendis Pharma A/S for the financial year January 1 to December 31, 2024, which comprise statements of profit or loss and other comprehensive income, statements of financial position, statements of changes in equity, cash flow statement and notes, including material accounting policy information, for the Group as well as the Parent. The consolidated financial statements and the parent financial statements are prepared in accordance with IFRS Accounting Standards as adopted by the EU and additional requirements of the Danish Financial Statements Act.
In our opinion, the consolidated financial statements and the parent financial statements give a true and fair view of the Group’s and the Parent’s financial position at December 31, 2024, and of the results of their operations and cash flows for the financial year January 1 to December 31, 2024 in accordance with IFRS Accounting Standards as adopted by the EU and additional requirements of the Danish Financial Statements Act.
Basis for opinion
We conducted our audit in accordance with International Standards on Auditing (ISAs) and the additional requirements applicable in Denmark. Our responsibilities under those standards and requirements are further described in the “Auditor’s responsibilities for the audit of the consolidated financial statements and the parent financial statements” section of this auditor’s report. We are independent of the Group in accordance with the International Ethics Standards Board for Accountants’ International Code of Ethics for Professional Accountants (IESBA Code) and the additional ethical requirements applicable in Denmark, and we have fulfilled our other ethical responsibilities in accordance with these requirements and the IESBA Code. We believe that the audit evidence we have obtained is sufficient and appropriate to provide a basis for our opinion.
Statement on the management commentary
Management is responsible for the management commentary.
Our opinion on the consolidated financial statements and the parent financial statements does not cover the management commentary, and we do not express any form of assurance conclusion thereon.
In connection with our audit of the consolidated financial statements and the parent financial statements, our responsibility is to read the management commentary and, in doing so, consider whether the management commentary is materially inconsistent with the consolidated financial statements and the parent financial statements or our knowledge obtained in the audit or otherwise appears to be materially misstated.
Moreover, it is our responsibility to consider whether the management commentary provides the information required by relevant law and regulations.
Based on the work we have performed, we conclude that the management commentary is in accordance with the consolidated financial statements and the parent financial statements and has been prepared in accordance with requirements of the relevant law and regulations. We did not identify any material misstatement of the management commentary.
| Independent Auditor’s Report | 6 |
Management’s responsibilities for the consolidated financial statements and the parent financial statements
Management is responsible for the preparation of consolidated financial statements and parent financial statements that give a true and fair view in accordance with IFRS Accounting Standards as adopted by the EU and additional requirements of the Danish Financial Statements Act, and for such internal control as Management determines is necessary to enable the preparation of consolidated financial statements and parent financial statements that are free from material misstatement, whether due to fraud or error.
In preparing the consolidated financial statements and the parent financial statements, Management is responsible for assessing the Group’s and the Parent’s ability to continue as a going concern, for disclosing, as applicable, matters related to going concern, and for using the going concern basis of accounting in preparing the consolidated financial statements and the parent financial statements unless Management either intends to liquidate the Group or the Entity or to cease operations, or has no realistic alternative but to do so.
Auditor’s responsibilities for the audit of the consolidated financial statements and the parent financial statements
Our objectives are to obtain reasonable assurance about whether the consolidated financial statements and the parent financial statements as a whole are free from material misstatement, whether due to fraud or error, and to issue an auditor’s report that includes our opinion. Reasonable assurance is a high level of assurance, but is not a guarantee that an audit conducted in accordance with ISAs and the additional requirements applicable in Denmark will always detect a material misstatement when it exists. Misstatements can arise from fraud or error and are considered material if, individually or in the aggregate, they could reasonably be expected to influence the economic decisions of users taken on the basis of these consolidated financial statements and these parent financial statements.
As part of an audit conducted in accordance with ISAs and the additional requirements applicable in Denmark, we exercise professional judgement and maintain professional scepticism throughout the audit. We also:
| • | Identify and assess the risks of material misstatement of the consolidated financial statements and the parent financial statements, whether due to fraud or error, design and perform audit procedures responsive to those risks, and obtain audit evidence that is sufficient and appropriate to provide a basis for our opinion. The risk of not detecting a material misstatement resulting from fraud is higher than for one resulting from error, as fraud may involve collusion, forgery, intentional omissions, misrepresentations, or the override of internal control. |
| • | Obtain an understanding of internal control relevant to the audit in order to design audit procedures that are appropriate in the circumstances, but not for the purpose of expressing an opinion on the effectiveness of the Group’s and the Parent’s internal control. |
| • | Evaluate the appropriateness of accounting policies used and the reasonableness of accounting estimates and related disclosures made by Management. |
| • | Conclude on the appropriateness of Management’s use of the going concern basis of accounting in preparing the consolidated financial statements and the parent financial statements, and, based on the audit evidence obtained, whether a material uncertainty exists related to events or conditions that may cast significant doubt on the Group’s and the Parent’s ability to continue as a going concern. If we conclude that a material uncertainty exists, we are required to draw attention in our auditor’s report to the related disclosures in the consolidated financial statements and the parent financial statements or, if such disclosures are inadequate, to modify our opinion. Our conclusions are based on the audit evidence obtained up to the date of our auditor’s report. However, future events or conditions may cause the Group and the Entity to cease to continue as a going concern. |
| • | Evaluate the overall presentation, structure and content of the consolidated financial statements and the parent financial statements, including the disclosures in the notes, and whether the consolidated financial statements and the parent financial statements represent the underlying transactions and events in a manner that gives a true and fair view. |
| Independent Auditor’s Report | 7 |
| • | Plan and perform the group audit to obtain sufficient appropriate audit evidence regarding the financial information of the entities or business units within the group as a basis for forming an opinion on the consolidated financial statements and the parent financial statements. We are responsible for the direction, supervision and review of the audit work performed for purposes of the group audit. |
We remain solely responsible for our audit opinion. We communicate with those charged with governance regarding, among other matters, the planned scope and timing of the audit and significant audit findings, including any significant deficiencies in internal control that we identify during our audit.
Copenhagen, February 12, 2025
Deloitte
Statsautoriseret Revisionspartnerselskab
Business Registration No 33 96 35 56
| Niels Skannerup Vendelbo | Rie Merete Kjær Larsen | |
| State-Authorised Public Accountant | State-Authorised Public Accountant | |
| Identification No (MNE) 34532 | Identification No (MNE) 43596 |
| Management Commentary | 8 |
Management Commentary
Unless the context otherwise requires, references to the “Company,” “Group,” “we,” “us” and “our” refer to Ascendis Pharma A/S and its subsidiaries.
Information and disclosure specifically addressing the parent company Ascendis Pharma A/S are described separately in the notes. Additionally, references to “Ascendis Pharma A/S” and “Parent Company” solely refer to the parent company Ascendis Pharma A/S.
Consolidated Key Figures
| (EUR’000) | 2024 | 2023 | 2022 | 2021 | 2020 | |||||||||||||||
| Revenue |
363,641 | 266,718 | 51,174 | 7,778 | 6,953 | |||||||||||||||
| Operating Profit/(Loss) |
(278,763 | ) | (455,541 | ) | (561,814 | ) | (451,792 | ) | (330,620 | ) | ||||||||||
| Finance Income/(Expenses) (net) |
(74,418 | ) | (208 | ) | 1,694 | 55,807 | (79,030 | ) | ||||||||||||
| Profit/(Loss) for the Year |
(378,084 | ) | (481,447 | ) | (583,194 | ) | (383,577 | ) | (418,955 | ) | ||||||||||
| Cash and Cash Equivalents |
559,543 | 392,164 | 444,767 | 446,267 | 584,517 | |||||||||||||||
| Total Assets |
1,179,495 | 825,587 | 1,089,738 | 1,084,921 | 979,793 | |||||||||||||||
| Equity |
(105,706 | ) | (145,697 | ) | 263,348 | 883,635 | 838,711 | |||||||||||||
| Investments in Property, Plant & Equipment |
1,427 | 2,442 | 14,489 | 23,704 | 19,860 | |||||||||||||||
| Return on Equity (%)* |
(300.8 | ) | (818.4 | ) | (101.7 | ) | (44.5 | ) | (58.4 | ) | ||||||||||
| Equity Ratio (%)* |
(9.0 | ) | (17.6 | ) | 24.2 | 81.4 | 85.6 | |||||||||||||
* Key ratios are calculated as follows:
Return on Equity: (Profit / (Loss) for the Year x 100) / Average Equity
Equity Ratio: (Equity x 100) / Total Assets
Ascendis Pharma in Brief
We are a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we develop TransCon-based therapies that demonstrate best-in-class potential to address unmet medical needs.
Our Organization
Certain of our operations are conducted through our following wholly-owned subsidiaries:
| Wholly-owned subsidiaries |
Domicile | |
| Ascendis Pharma GmbH | Germany | |
| Ascendis Pharma Endocrinology GmbH | Germany | |
| Ascendis Pharma, Inc. | USA | |
| Ascendis Pharma Endocrinology, Inc. | USA | |
| Ascendis Pharma Ophthalmology Division A/S | Denmark | |
| Ascendis Pharma Endocrinology Division A/S | Denmark | |
| Ascendis Pharma Bone Diseases A/S | Denmark | |
| Ascendis Pharma Growth Disorders A/S | Denmark | |
| Ascendis Pharma Oncology Division A/S | Denmark | |
| Ascendis Pharma Europe A/S | Denmark | |
| Ascendis Pharma UK Limited | United Kingdom | |
| Ascendis Pharma Iberia S.L. | Spain | |
| Ascendis Pharma France SASU | France | |
| Ascendis Pharma Italia SRL | Italy | |
| Ascendis Pharma Sverige AB | Sweden | |
| Ascendis Pharma Switzerland GmbH | Switzerland |
| Management Commentary | 9 |
The Company has increased its number of employees to 1,017 at the end of 2024 compared to 879 at the end of 2023. Employees engaged with selling, general, and administration increased, primarily due to commercial activities, and extension of corporate functions to support those activities. Further, in connection with formation of Eyconis, a separate company, certain employees of Ascendis Pharma, engaged with research and development in the U.S., joined the newly formed company in 2024.
Our Vision
As announced in January 2024, Vision 2030 is our vision to achieve blockbuster status for multiple products and expand our engine for future innovation. This includes:
| • | Be the Leading Endocrinology Rare Disease Company |
| • | Achieve blockbuster status (>$1B) for each of TransCon PTH, TransCon hGH, and TransCon CNP through worldwide commercialization |
| • | Be the leader in growth disorders and hypoparathyroidism, pursuing clinical conditions, innovative life cycle management, and complementary patient offerings |
| • | Expand pipeline with Endocrinology Rare Disease blockbuster product opportunities |
| • | Create Value in Additional Therapeutic Areas through Innovative Business Models |
| • | Obtain accelerated approval in oncology with registrational trials ongoing |
| • | Pursue TransCon product opportunities in >$5B indications |
| • | Maximize value creation of these product opportunities through collaboration with therapeutic area market leaders |
| • | Differentiate with Ascendis Fundamentals |
| • | Outperform industry drug development benchmarks with Ascendis’ product innovation algorithm |
| • | Remain independent as a profitable biopharma through lean and flexible ways of working |
| • | Let our values Patients, Science, Passion drive our decisions to success |
Our products and product candidates combine our TransCon technologies with clinically validated parent drugs or pathways, with the goal of optimizing safety, efficacy, tolerability, and convenience.
We apply these technologies using our algorithm with the goal of creating product candidates with the potential to be best-in-class. Using this approach, we plan to expand our pipeline with Endocrinology Rare Disease product opportunities in large addressable markets. In addition, our vision is to pursue TransCon product opportunities in >$5B indications in other therapeutic areas and maximize value creation of these product opportunities through collaboration with therapeutic area market leaders. We believe our approach to product innovation may reduce the risks associated with traditional drug development.
Ascendis Algorithm for Product Innovation
| Management Commentary | 10 |
When we apply our TransCon technologies to clinically validated parent drugs or pathways, we may benefit from established clinical safety and efficacy data, which we believe increases the probability of success compared to traditional drug development. As illustrated above, our algorithm for product innovation focuses on identifying indications that have an unmet medical need, have a clinically validated parent drug or pathway, are suitable to our TransCon technologies, have potential for creating a clearly differentiated product, have a potential established development pathway, and have the potential to address a large market.
Program Summaries
We currently have two marketed products and a diversified portfolio of four product candidates in clinical development in the areas of Endocrinology Rare Disease and Oncology, and we are working to apply our TransCon technology platform in additional therapeutic areas such as the glucagon-like peptide 1 (“GLP-1”) class, where we believe we have designed a potentially best-in-class, once-monthly program.
| • | SKYTROFA – Our first marketed product is SKYTROFA® (lonapegsomatropin-tcgd), developed as TransCon Growth Hormone (“TransCon hGH”), which has received regulatory approval in the United States for the treatment of pediatric patients one year and older who weigh at least 11.5 kg and have growth failure due to inadequate secretion of endogenous growth hormone, also known as growth hormone deficiency (“GHD”). SKYTROFA has been commercially available for prescription in the United States since October 2021. In addition, SKYTROFA (lonapegsomatropin) was granted marketing authorization in the European Union (“EU”), Norway, Iceland, Liechtenstein, and Great Britain (covering England, Wales, Scotland) as a once-weekly subcutaneous injection for the treatment of children and adolescents aged 3 to 18 years with growth failure due to insufficient secretion of endogenous growth hormone. In the EU, SKYTROFA has been commercially available for prescription in Germany since September 2023. |
| • | YORVIPATH – Our second marketed product is YORVIPATH® (palopegteriparatide), developed as TransCon PTH. In the EU, Norway, Iceland, Liechtenstein, and Great Britain (covering England, Wales, and Scotland), YORVIPATH has been granted marketing authorization as a once-daily subcutaneous injection for the treatment of adults with chronic hypoparathyroidism. In the EU, YORVIPATH has been commercially available for prescription in Germany and Austria since January 2024 and has also been available to patients in other countries under named patient programs. In August 2024, YORVIPATH received regulatory approval in the U.S. for the treatment of hypoparathyroidism in adults. YORVIPATH has been commercially available for prescription in the United States since December 2024. As of February 7, 2025, 908 new prescriptions by 539 unique prescribing healthcare providers in the U.S. |
| • | Endocrinology Rare Disease Pipeline – Two product candidates in our Endocrinology Rare Disease portfolio are currently in development spanning multiple indications and geographies. These product candidates are TransCon hGH (lonapegsomatropin) for adult GHD and children with Turner syndrome and TransCon CNP (navepegritide) for infants, children, and adolescents with achondroplasia. We are also investigating TransCon CNP in combination with TransCon hGH in children with achondroplasia. In addition, we plan to investigate TransCon hGH in other established daily growth hormone indications. Through our strategic collaboration, Teijin Limited is developing and, if approved, plans to commercialize TransCon hGH, TransCon PTH, and TransCon CNP for endocrinology rare disease in Japan. In addition, through our strategic investment, VISEN Pharmaceuticals (“VISEN”) is developing and, if approved, plans to commercialize TransCon hGH, TransCon PTH, and TransCon CNP for endocrinology rare diseases in Greater China. |
| Management Commentary | 11 |
| • | Oncology Pipeline – In Oncology, we are leveraging our TransCon technologies with the goal of enhancing the anti-tumor effects of clinically-validated parent drugs and pathways and to provide sustained modulation of tumor microenvironments and activate cytotoxic immune cells. We initiated clinical development of two programs: TransCon TLR7/8 Agonist, an investigational, long-acting prodrug of resiquimod, a small molecule agonist of Toll-like receptors (“TLR”) 7 and 8, for intratumoral delivery, and TransCon IL-2 b/g (onvapegleukin alfa) for systemic delivery, which is designed for prolonged exposure to an IL-2 variant that selectively activates IL-2 b/g with minimal binding to IL-2Rα. During the fourth quarter of 2024, we closed enrollment in our BelieveIT-201 clinical trial and to dose expansion cohorts involving TransCon TLR7/8 Agonist in the transcendIT-101 and IL-Believe trials to prioritize our efforts on TransCon IL-2 b/g. |
TransCon Product Candidates Pipeline
Other than the rights we have granted to Eyconis, Novo Nordisk, Teijin Limited, and VISEN as noted in this annual report, we hold worldwide rights to our TransCon technologies and, other than our royalty financing arrangements with Royalty Pharma as noted in this annual report, we owe no third-party royalty or milestone payment obligations with respect to our TransCon technologies, TransCon hGH, TransCon PTH or any of our other product candidates. The following chart lists our product candidates.
| 1. | Pivotal ApproaCH Trial (NCT05598320). |
| 2. | sBLA submitted to U.S. FDA, PDUFA goal date July, 27th 2025. |
| 3. | New InsiGHTS Trial (NCT05690386). |
| 4. | reACHin Trial (NCT06079398). |
| 5. | teACH Trial (NCT06732895). |
| 6. | COACH Trial (NCT06433557). |
| 7. | VISEN Pharmaceuticals’ Phase 3 trial. |
| 8. | Japanese riGHt Trial. |
| 9. | PaTHway China (NCT05387070). |
| 10. | PaTHway Japan. |
| 11. | ACcomplisH China (NCT05246033). |
| 12. | BelieveIT-201 Trial (NCT05980598). |
| 13. | IL-Believe Trial (NCT05081609). |
| Management Commentary | 12 |
We maintain an intellectual property portfolio comprising over 425 issued patents and over 525 patent applications as of December 31, 2024, which includes patents and patent applications applicable to our products and product candidates with claims directed to composition of matter, process, formulation and/or methods-of-use for our products and product candidates, including a product-specific device and core TransCon technologies. While our TransCon prodrugs may incorporate already approved parent drugs, TransCon hGH, TransCon PTH, and each of our other product candidates are new molecular entities and therefore eligible to be granted new intellectual property rights, including new composition of matter patents.
Global Commercialization Strategy
We are establishing a global presence to commercialize TransCon products and product candidates, where approved, to address patients’ unmet medical needs.
In the U.S., we have established an integrated organization to commercialize our approved Endocrinology Rare Disease products, SKYTROFA and YORVIPATH. Our U.S. organization includes sales, market access, patient support, and medical affairs teams. The sales team engages with healthcare providers and present products, usage and safety guidelines in accordance with the label. Our market access team engages with health authorities, insurance companies, and payers to help patients in need of our products gain access to them. Our patient support team facilitates reimbursement support, out of pocket assistance, provides educational resources and product training. Our medical affairs team provides scientific exchange to the physician and medical community. We have also established a network of specialty pharmacies to support product distribution.
In Europe, we are expanding our presence by building integrated organizations in select countries, which we call Europe Direct, beginning with Germany, where we have launched our Endocrinology Rare Disease products, SKYTROFA and YORVIPATH. We are establishing commercial infrastructure in other Europe Direct country clusters, including DACH (Germany, Austria, Switzerland), France & BeNeLux (Belgium, the Netherlands, and Luxembourg), Iberia (Portugal and Spain), Italy, Nordics (Denmark, Norway, Sweden, Iceland, Finland), and the United Kingdom & Ireland.
Beyond the U.S. and Europe Direct, we are expanding global reach for our Endocrinology Rare Disease programs through exclusive distribution agreements with geographic market leaders, which we call International Markets. As of December 31, 2024, we have established eight such regional agreements:
| • | Acino Pharma Proprietary Limited (South Africa) |
| • | Adium Pharma S.A. (Argentina, Brazil, Colombia and Mexico) |
| • | Er-Kim İlac Sanayi ve Ticaret A.S (Central & Eastern Europe, Turkey, and certain countries in Eurasia) |
| • | Neopharm (Israel) 1996 Ltd (Israel, Armenia, Azerbaijan, Georgia, Kazakhstan, Kyrgyzstan, Tajikistan, Turkmenistan and Uzbekistan) |
| • | Pendopharm, a division of Pharmascience Inc. (Canada) |
| • | Specialised Therapeutics Asia Pte Ltd. (Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand, and Vietnam) |
| • | SRS Pharmaceuticals Pvt. Ltd. (India and Philippines) |
| • | Vector Pharma FZCO (Saudi Arabia, United Arab Emirates, Kuwait, Oman, Qatar, and Bahrain) |
Finally, we are making our products commercially available in select markets through exclusive license agreements with partners with local development and commercialization expertise and infrastructure. In China, VISEN has exclusive license rights to develop and commercialize TransCon hGH, TransCon PTH, and TransCon CNP. In Japan, Teijin Limited has exclusive license rights to develop and commercialize TransCon hGH, TransCon PTH, and TransCon CNP.
| Management Commentary | 13 |
TransCon Technologies
Overview
Our TransCon technologies are designed to combine the benefits of conventional prodrug and sustained release technologies to solve the fundamental limitations seen in other approaches to extending duration of a drug’s action in the body, with the goal of developing highly differentiated product candidates based on efficacy, safety, tolerability, and convenience. In addition to retaining the original mode of action of the parent drug and potentially supporting dosing frequency from daily up to six months or more, we believe that predictable release over time can improve treatment safety and efficacy, increase the likelihood of clinical development success, and provide intellectual property benefits.
TransCon molecules can have up to three components: a parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic pH and temperature conditions initiate the release of the active, unmodified parent drug in a predictable release manner.
Depending upon the type of TransCon carrier we employ, we can design our TransCon prodrugs for sustained localized or systemic delivery.
TransCon Technology Components
TransCon Carriers
Our TransCon technologies incorporate three carrier platforms that can be used to provide sustained localized or systemic drug exposure. These biocompatible carrier platforms include our TransCon systemic carriers and TransCon localized carriers (self-eliminating hydrogels). Our carriers inactivate and protect the drug through a shielding effect, which may prevent rapid excretion and degradation of the parent drug and enable benefits that include improved injection site tolerability, reduced systemic adverse effects, and low immunogenicity.
| • | Systemic – Our TransCon systemic carriers are used to provide systemic drug exposure and are based on soluble compounds such as methoxypolyethylene glycol (“mPEG”) or other natural or synthetic polymers, as well as our albumin avidity approach, where 2 or more albumin binding moieties are incorporated into the drug molecule to facilitate sustained exposure. Prodrugs created using our systemic carriers are readily absorbed into the bloodstream after administration, thus minimizing exposure of the subcutaneous tissue to active drug, which we believe may improve injection site tolerability. TransCon hGH, TransCon PTH, and TransCon CNP utilize mPEG as a carrier molecule. mPEG is widely used to improve the pharmacokinetic or pharmacodynamic properties of marketed therapeutics. Below is an illustration of our systemic carrier: |
| Management Commentary | 14 |
| • | Localized – Our TransCon localized carriers include TransCon hydrogels based on PEG, hyaluronic acid, or other biopolymers. TransCon hydrogel is designed to self-eliminate to soluble, biocompatible molecules after the drug payload has been released. When applied for localized delivery, the TransCon hydrogel enables the release of a parent drug at high local concentrations within the target area while minimizing systemic exposure. We believe this may widen the therapeutic window for parent drugs that suffer from significant systemic side effects and toxicities, facilitating the development of highly efficacious product candidates with improved safety and tolerability profiles. Below is an illustration of our hydrogel carrier: |
TransCon Linkers
Our reversible TransCon linkers are designed to enable the transient conjugation of a broad range of therapeutics, including proteins, peptides, and small molecules, to our TransCon carriers. We have a large library of TransCon linkers that may be applicable to various types of parent drugs, and that can be tailored to potentially achieve half-life extension enabling daily, weekly, monthly, and half-yearly dosing and to customize the potential pharmacokinetic profile for each individual product candidate with the goal of optimizing the potential therapeutic effect. TransCon linkers are self-cleaving through a process called intra-molecular assisted cleavage, which causes the linker to release the unmodified parent drug. We can tailor the release properties of the linker to a given therapeutic indication and parent drug by modifying the linker structures. We believe the self-cleaving process of our linker avoids many of the shortcomings of conventional prodrug technologies, which often depend on metabolic processes, such as enzymatic degradation, to convert the prodrug into the active drug. The rate of metabolic conversion of prodrugs in these types of processes may differ between patients, and even within different tissues in the same patient. As a result, conventional prodrugs do not always offer predictable release of the parent drug. Our TransCon linkers are designed to predictably release an unmodified active parent drug at predetermined rates governed by physiological pH and temperature conditions, which are tightly regulated in the body. Consequently, we believe we can design our prodrugs to release the unmodified parent drug at predictable rates.
| Management Commentary | 15 |
Parent Drugs
Our TransCon technologies are applicable across a broad range of therapeutic classes and are currently used to create long-acting product candidates with best-in-class potential based on proteins, peptides, and small molecules. By primarily focusing on biological targets that have been clinically validated, we can leverage available knowledge regarding a target’s activity. Based on this selective approach, we know what drug levels must be maintained in the body for optimal efficacy and safety, and we can design the release half-life and dosing frequency of our TransCon prodrugs to maintain these levels to achieve the desired pharmacological effect. We move a product candidate into development after it demonstrates the desired profile in non-clinical models. Furthermore, based on the established translational relationships between preclinical animal models and clinical efficacy, we believe experimental results generated in animal models are highly predictive of clinical results and reduce the development risk for our TransCon prodrugs. This strategy is designed to reduce risk and increase productivity.
This approach has enabled us to develop two approved products and generate a pipeline of product candidates designed to address significant unmet medical needs and to become potential sources of significant revenue for our company. Because our TransCon technologies leverage clinically validated parent drugs or pathways, we believe we may benefit from a higher development and regulatory success rate compared to development of drug compounds without established biology.
TransCon Products – Endocrinology Rare Disease
TransCon Growth Hormone (hGH)
Market Opportunity for Recombinant Human Growth Hormone
Growth hormone deficiency is a serious orphan disease that affects both children and adults. Children with GHD are characterized by short stature, metabolic and cardiovascular abnormalities, cognitive deficiencies, and poor quality of life. GHD in adults is associated with increased adiposity, or fat mass, as well as psychiatric-cognitive, cardiovascular, muscular, metabolic and skeletal abnormalities. In childhood and adolescence, growth hormone plays an essential role in normal longitudinal growth, muscle and bone strength, and distribution of body fat. In adults, growth hormone contributes to body composition, cardiovascular function, and bone health. The current standard of care for GHD has been daily subcutaneous injections of somatropin, a recombinant human growth hormone (“hGH”). These daily hGH therapies have been shown to be safe and well-tolerated.
In both therapy-compliant children and adults with GHD, daily subcutaneous injections of hGH have resulted in improved body composition parameters, bone density, cardiovascular outcomes, and quality of life. Growth hormone-deficient children who are fully adherent to their daily hGH treatment regimen may achieve a height in adulthood that is comparable to that of their family members and national norms.
Despite the demonstrated benefits of daily hGH therapy, many GHD patients are not adequately treated, and adherence continues to be a challenge, as reported in a 2021 paper published by Kaplowitz et al. (Kaplowitz P, Manjelievskaia J, Lopez-Gonzalez L, et al. Economic burden of growth hormone deficiency in a US pediatric population. J Manag Care Spec Pharm. 2021; 27(8):1118-1128). The observational retrospective cohort analysis utilized administrative claims data from two databases on over 20,000 pediatric patients diagnosed with GHD. Approximately 68% of commercial patients and approximately 63% of Medicaid patients received daily growth hormone treatment, whereas approximately 32% of commercial patients and approximately 37% of Medicaid patients were untreated. In addition, mean adherence as measured by proportions of days covered, which is defined as the number of days covered by any daily growth hormone prescription during the follow-up period, was approximately 60% in the commercial cohort and approximately 50% in the Medicaid cohort. Only 32% of commercial and 18% of Medicaid patients reported adherence rates greater than 80%.
For adult patients with GHD, underdiagnosis and undertreatment are also a concern. Untreated adult GHD (“AGHD”) patients can experience reduced quality of life and increased risk of morbidity and mortality. A retrospective cohort study presented at ENDO 2023 analyzed an electronic health records database and selected adult patients with suspected AGHD. Of the 51,588 patients with suspected AGHD, fewer than 4% were treated with growth hormone.
| Management Commentary | 16 |
Since the introduction of hGH in 1981, a number of the world’s largest pharmaceutical companies have developed and marketed daily-administered hGH products. All currently marketed daily hGH products in the United States – Norditropin® (Novo Nordisk A/S), Humatrope® (Eli Lilly and Company), Genotropin® (Pfizer Inc.), Zomacton® (Ferring Pharmaceuticals, Inc.) and Omnitrope® (Sandoz GmbH) – contain unmodified somatropin and are administered by subcutaneous injections. The global market for daily hGH products is largely composed of products from Novo Nordisk, Pfizer, Eli Lilly, Sandoz, and Merck KGaA, which together account for most of the global market share. However, according to the FDA drug shortage website, Humatrope has been discontinued due to a business decision which might impact the hGH global market share in the future.
Primary indications for hGH in children are GHD, idiopathic short stature, chronic kidney disease, Prader-Willi syndrome, small for gestational age, and Turner syndrome. In adults, primary indications for hGH include GHD and AIDS-induced weight loss. We estimate pediatric indications comprise up to 90% of the total hGH market, of which approximately half is for pediatric GHD.
Competitive Landscape for Long-Acting Growth Hormone Therapies
Since the 1990s, the pharmaceutical industry has employed various approaches to develop long-acting growth hormone products to reduce the burden of daily injections on patients and increase patient compliance with the dosing regimen. These approaches generally fall into two categories: unmodified somatropin and permanent modification of growth hormone:
| • | Unmodified somatropin: Two long-acting growth hormone products using encapsulation technologies previously received regulatory approval in the U.S. and Europe, but were subsequently discontinued due to commercial challenges. These include Nutropin Depot®, formerly marketed by Genentech, and Somatropin Biopartners, developed by LG Life Sciences and Biopartners GmbH. Nutropin Depot was approved by the FDA in 1999 and later withdrawn; Somatropin Biopartners (LB03002) was approved by the European Medicines Agency (“EMA”) in 2013, and later withdrawn. We believe that the lack of market acceptance was a result of the various safety and tolerability issues that tend to arise with encapsulation technologies. |
| • | Permanent modification of growth hormone: Modification technologies prolong activity in the body by creating analogs of growth hormone through permanent modification of the growth hormone molecule. This modification may alter the molecular size and interaction with the growth hormone receptor and/or change the natural association affinity to endogenous proteins, as well as the distribution in the body. These changes may alter and reduce the efficacy of these drugs compared to unmodified daily somatropin and may also negatively impact the drug’s safety. |
Novo Nordisk received regulatory approval in various countries and regions including the U.S., Japan, and EU for once-weekly somapacitan (SOGROYA®) in adult and pediatric patients with GHD.
Pfizer (in collaboration with OPKO Health Inc.) received regulatory approval of once-weekly somatrogon (NGENLA™) in various countries and regions including the U.S., Japan, and EU for pediatric GHD.
A permanently PEGylated long-acting growth hormone developed by GeneScience Pharmaceuticals Co., Ltd. (Jintrolong®) is available in China for pediatric GHD, Turner syndrome and idiopathic short stature and the Somatropin Biopartners product (LB03002) is available in Korea. Other experimental growth hormone therapies based on permanent modification are in different stages of clinical development by various companies, including Genexine Inc., I-MAB, Amoytop, UnionGene, Anhui Anke Biotechnology, Alteogen, and JCR Pharmaceuticals Co., Ltd.
Our Solution: TransCon hGH
TransCon hGH is a prodrug composed of somatropin that is transiently bound to a carrier by a proprietary linker. TransCon hGH is administered once weekly and is designed to maintain the same mode of action as daily therapies by providing sustained release of active, unmodified somatropin, the same recombinant growth hormone molecule used in the daily hGH therapies that have historically been the standard of care.
| Management Commentary | 17 |
TransCon Growth Hormone (hGH) for Pediatric GHD
TransCon hGH, marketed under the brand name SKYTROFA® (lonapegsomatropin-tcgd), received regulatory approval in the U.S. for the treatment of pediatric patients one year and older who weigh at least 11.5 kg and have growth failure due to inadequate secretion of endogenous growth hormone, also known as GHD. SKYTROFA has been commercially available for prescription in the United States since October 2021. In the EU, Norway, Iceland, Liechtenstein, and Great Britain (covering England, Wales, Scotland), we received marketing authorization for TransCon hGH – known by its brand name SKYTROFA (lonapegsomatropin) – as a once-weekly subcutaneous injection for the treatment of children and adolescents aged 3 to 18 years with growth failure due to insufficient secretion of endogenous growth hormone. SKYTROFA has been commercially available for prescription in Germany since September 2023.
In September 2023, we announced top-line results from the completed enliGHten Trial, an open-label extension trial evaluating the long-term safety and efficacy of TransCon hGH as a once-weekly treatment for children and adolescents with GHD. The enliGHten Trial enrolled 298 participants (mean age 10.3 years) from the Phase 3 heiGHt Trial of treatment-naïve pediatric GHD patients and the Phase 3 fliGHt Trial of pediatric GHD patients switching from daily somatropin treatment. Patients in these trials received a total of up to 6 years of treatment with TransCon hGH. At the time of the enliGHten Trial closure, 81 participants were designated as treatment completers, based on their physician’s determination that treatment for pediatric GHD was no longer required. Of these treatment completers, 59% met or exceeded their average parental height standard deviation score (“SDS”), with mean TransCon hGH treatment duration of 3.2 years.
Clinical Trial of TransCon hGH in Japanese Pediatric GHD
In the Phase 3 riGHt Trial, we are evaluating TransCon hGH (N=15) compared to somatropin (N=16) as a treatment in Japanese children with GHD. The trial achieved its primary objective with Week 52 top-line results consistent with our pivotal heiGHt Trial and VISEN’s Phase 3 trial. In the riGHt Trial, TransCon hGH was generally well tolerated with a safety profile that was similar to that of somatropin’s. Trial subjects continue in the extension period.
Proprietary Auto-Injector
SKYTROFA includes the SKYTROFA®Auto-Injector and cartridges. The auto-injector provides for room temperature storage, includes an empty-all design, and is expected to last for at least four years. The device enables a single, low-volume injection of less than 0.6 mL for the majority of patients with a thin, 31-gauge needle that is only 4 millimeters in length, which is comparable to needles used to administer daily hGH. We are also working on strategies that will enable the auto-injector to integrate with the digital healthcare system, including Bluetooth connectivity features to allow for easy tracking of dosing adherence over time.
| Management Commentary | 18 |
Figure: Our state-of-the-art auto-injector is designed to improve treatment compliance.
TransCon Product Candidates – Endocrinology Rare Diseases
TransCon Growth Hormone (hGH) for Other Indications
Clinical Development in Adult GHD
In September 2024, we announced the submission of a supplemental Biologics License Application (“sBLA”) to the FDA for TransCon hGH to expand its currently approved label to include eligible adults for the treatment of GHD. The submission was based on results from foresiGHt, a Phase 3 randomized, parallel-arm, placebo-controlled (double-blind), and active-controlled (open-label) trial that compared the efficacy and safety of weekly TransCon hGH with weekly placebo and daily hGH in adults with GHD. In December, we announced the FDA accepted the sBLA for review and set a Prescription Drug User Fee Act (“PDUFA”) goal date of July 27, 2025.
In December 2023, we announced positive top-line results from foresiGHt. The trial aims to evaluate the metabolic benefits of TransCon hGH in adults, with the primary objective to evaluate change in trunk fat percentage.
The foresiGHt Trial evaluated 259 adults with GHD aged 23 to 80 years old, randomized 1:1:1, titrated to receive a target fixed dose of TransCon hGH, placebo, or daily hGH based on age and oral estrogen intake, with approximately equivalent hGH mg/week for TransCon hGH and daily hGH.
| • | TransCon hGH demonstrated superiority on its primary efficacy endpoint at Week 38: |
| • | Change from baseline in trunk percent fat as measured by dual x-ray absorptiometry (TransCon hGH -1.67% vs. placebo +0.37%, LS mean difference = -2.04%, p < 0.0001) |
| • | TransCon hGH demonstrated superiority on its key secondary efficacy endpoints at Week 38: |
| • | Change from baseline in total body lean mass (TransCon hGH +1.60 kg vs placebo - 0.10 kg, LS mean difference = 1.70 kg, p < 0.0001) |
| • | Change from baseline in trunk fat mass (TransCon hGH -0.48 kg vs placebo +0.22 kg, LS mean difference = -0.70 kg, p = 0.0053) |
| Management Commentary | 19 |
| • | Exploratory post-hoc analysis at Week 38 demonstrated comparable treatment effect of TransCon hGH and daily hGH on target tissues. For patients with average insulin-like Growth Factor 1 (“IGF-1”) SDS levels ≤ 1.75 at Week 38: |
| • | Change from baseline in trunk percent fat (TransCon hGH -2.42% vs. daily hGH - 2.59%) |
| • | Change from baseline in total body lean mass (TransCon hGH +1.70 kg vs daily hGH +1.37 kg) |
| • | Change from baseline in trunk fat mass (TransCon hGH -0.90 kg vs daily hGH -0.94 kg) |
| • | TransCon hGH was generally safe and well tolerated, with no discontinuations related to study drug and with comparable safety and tolerability to daily hGH. |
Clinical Development in Turner Syndrome
In December 2024, we announced positive top-line results from the Phase 2 New InsiGHTS Trial. New InsiGHTS randomized and dosed 49 children with Turner syndrome aged 1 to 10 years old into one of four treatment groups 1:1:1:1 – one of three starting doses of TransCon hGH (0.24, 0.30, or 0.36 mg/kg/week) or an active comparator of daily somatropin with a starting dose of 0.35 mg/kg/week. Doses were individualized based on IGF-1. On the primary endpoint of annualized height velocity and secondary endpoint of change from baseline in height SDS, children treated with TransCon hGH demonstrated improved growth similar to daily somatropin at Week 26, independent of starting dose. TransCon hGH was generally safe and well tolerated, with no discontinuations related to study drug and with comparable safety and tolerability to daily somatropin.
Other Development Plans
During the third quarter of 2025, we plan to submit an Investigational New Drug (“IND”) application or similar for a basket trial evaluating other indications (planned for small for gestational age without catch-up growth (“SGA”); idiopathic short stature (“ISS”); SHOX deficiency (including Turner syndrome)). In addition, we are investigating other potential indications for TransCon hGH where we believe a long-acting hGH therapy may offer benefits to patients with rare growth disorders, including in combination with our TransCon CNP product candidate in our Phase 2 COACH Trial for children with achondroplasia.
TransCon PTH
Market Opportunity in Hypoparathyroidism
Hypoparathyroidism is a rare endocrine disease caused by insufficient levels of parathyroid hormone (“PTH”). As reported in a 2016 paper, most patients with hypoparathyroidism (70-80% of cases) develop the disease following damage to or accidental removal of the parathyroid glands during thyroid surgery. Other etiologies include autoimmune disorders, genetic disorders such as autosomal dominant hypocalcemia type 1, and idiopathic causes. Conventional therapy with oral calcium and active vitamin D (also called calcitriol) does not effectively address the short-term symptoms, long-term complications, or quality-of-life impacts of hypoparathyroidism.
Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications. Short-term symptoms of hypoparathyroidism include weakness; severe muscle cramps (tetany); abnormal sensations such as tingling, burning, and numbness (paresthesia); memory loss; impaired judgment; and headache. A survey published in 2014 of 374 individuals with hypoparathyroidism showed that 72% experienced more than ten symptoms in the preceding twelve months, with symptoms experienced for a mean of 13 ± 9 hours a day. Prolonged use of conventional therapy may increase the risk of major complications, such as calcium deposits in the brain, blood vessels, eyes, and soft tissues. According to a systematic review by Gosmanova et al. published in 2021, chronic hypoparathyroidism treated with conventional therapy is associated with higher rates of renal complications compared to the general population, including nephrolithiasis (up to 36%), nephrocalcinosis (up to 38%), and chronic kidney disease (up to 41%).
| Management Commentary | 20 |
Studies have found that the burden of hypoparathyroidism negatively impacts health-related quality of life (“QoL”), physical functioning, and psychological well-being. Compared with an age-matched general population sample, individuals with hypoparathyroidism have reported markedly lower health-related QoL, irrespective of serum calcium level, as measured by the physical (P<0.001) and mental (P<0.001) component scores of the 36-Item Short Form Health Survey (SF-36) as well as the EuroQol-5 Dimensions Visual Analogue Scale. As reported in a 2021 paper, in interviews conducted on 42 individuals with hypoparathyroidism, 98% reported reduced functioning and well-being, including anxiety (81%), feeling sad or depressed (62%), and feeling irritable or short-tempered (43%) despite management with conventional therapy.
Hypoparathyroidism also imposes a substantial burden on the healthcare system despite the use of conventional therapy. For example, individuals with hypoparathyroidism may require hospitalizations or emergency department visits due to acute severe hypocalcemia (calcium crashes) and those with post-surgical hypoparathyroidism have an increased risk of hospitalization due to infection than age- and sex-matched controls from the general population. Individuals with hypoparathyroidism also have an increased risk of hospitalization due to renal complications, such as chronic kidney disease and renal failure, compared to age- and sex-matched controls. A retrospective review published in 2019 of clinical burden and healthcare resource utilization showed that 90.7% of individuals had ≥1 hypoparathyroidism-related healthcare utilization event during a 12-month period, including 87.8% with ≥1 outpatient visit, 41% with ≥1 emergency department visit, and 19.5% with ≥1 hospitalization. The management of hypoparathyroidism is also associated with substantial economic burdens and consequences of hypoparathyroidism may negatively impact employment status and work productivity.
The 2022 Guidelines from the Second International Workshop addressing the prevention, diagnosis, and management of hypoparathyroidism was published in September 2022 in the Journal of Bone and Mineral Research and authored by leading clinicians from North America, Europe, and Asia. The authors suggest consideration of PTH replacement therapy in patients whose hypoparathyroidism is inadequately controlled with conventional therapy. Inadequate control is considered to be any one of the following: symptomatic hypocalcemia, hyperphosphatemia, renal insufficiency, hypercalciuria, or poor quality of life. In addition, the guideline indicates that individuals with poor compliance, malabsorption, or intolerant of large doses of calcium and active vitamin D may also benefit from PTH replacement therapy. Based on this current guideline, we believe PTH replacement therapy could be applicable to most patients with hypoparathyroidism.
In 2015, Takeda’s NATPARA® (parathyroid hormone) was approved in the U.S. for once-daily subcutaneous injection as an adjunct to vitamin D and calcium in patients with hypoparathyroidism. NATPARA was voluntarily recalled in September 2019 in the U.S. and is now only available to a limited number of patients through a Special Use Program offered by its manufacturer, Takeda. In October 2022, Takeda announced that it would discontinue manufacturing NATPARA/NATPAR globally by the end of 2024.
We are also aware of several academic groups and companies working on making longer-acting agonists of the PTH receptor. In addition, other companies and groups are developing therapies for hypoparathyroidism at the clinical stage, including Calcilytix (a BridgeBio company), Entera Bio, Extend Biosciences, Massachusetts General Hospital, AstraZeneca, MBX Biosciences, and Septerna.
Forteo® (teriparatide, PTH [1-34]), approved since 2002 for the treatment of osteoporosis, has sometimes been used for treatment of hypoparathyroidism using multiple daily injections, despite not being approved for this indication. Clinical research conducted by the U.S. National Institutes of Health in subjects receiving continuous exposure to PTH (1-34), administered by an infusion pump demonstrated simultaneous normalization of serum calcium and urinary calcium, as well as normalization of bone turnover.
We estimate hypoparathyroidism affects over 250,000 patients in the U.S. and Europe. In the U.S., we estimate hypoparathyroidism affects approximately 70,000 to 90,000 patients, including 4,000 to 5,000 patients who we estimate have previously been treated with PTH therapy. In Germany, we estimate hypoparathyroidism affects approximately 70,000 patients. Outside of Germany, we estimate hypoparathyroidism affects over 100,000 patients in the rest of Europe.
| Management Commentary | 21 |
Our Solution: TransCon PTH
TransCon PTH (palopegteriparatide) is a prodrug of PTH (1-34) that is administered once-daily to achieve and maintain a steady concentration of PTH in the bloodstream within the physiological range. TransCon PTH is designed to provide PTH in the physiological range for 24 hours per day, thereby more fully addressing aspects of the disease, including maintaining normal serum calcium and phosphate levels and normalizing urinary calcium.
By providing steady levels of PTH in the physiological range, we believe TransCon PTH may be a highly differentiated therapy for hypoparathyroidism.
TransCon PTH for the Treatment of Hypoparathyroidism
In August 2024, the FDA approved YORVIPATH® (palopegteriparatide; developed as TransCon PTH) for the treatment of hypoparathyroidism in adults. In September 2024, the FDA granted Orphan Drug exclusivity to YORVIPATH, providing seven years of market exclusivity for YORVIPATH in the United States for the treatment of hypoparathyroidism in adults. YORVIPATH has been commercially available for prescription since late December 2024 in the United States.
In November 2023, TransCon PTH received regulatory approval in the EU and European Economic Area and is marketed as YORVIPATH® (palopegteriparatide), a parathyroid hormone replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism. In addition, YORVIPATH was granted Orphan status in the EU in November 2023. In January 2024, we announced commercial availability of YORVIPATH in Germany and Austria, and we began shipping to customers in February 2024.
In April 2024, TransCon PTH received regulatory approval in Great Britain as a PTH replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism. In addition, in April 2024, we announced that the United Kingdom’s Medicines & Healthcare products Regulatory Agency granted YORVIPATH Orphan Drug status.
In December 2022, the FDA allowed us to initiate a U.S. expanded access program (“EAP”) for TransCon PTH for eligible adult patients with hypoparathyroidism with prior PTH treatment experience. The EAP closed to new patients on November 29, 2024, in preparation for the U.S. commercial availability of YORVIPATH. Beginning in December 2024, we started enrolling EAP patients into the Ascendis Signature Access Program Ascendis’ patient support program. Patients enrolled in the EAP are now working with their healthcare providers (“HCPs”) to transition to the commercially available YORVIPATH.
In July 2021, the Ministry of Health, Labour and Welfare in Japan granted Orphan Drug Designation (“ODD”) to TransCon PTH for the treatment of hypoparathyroidism.
In October 2020, we were granted Orphan Designation (“OD”) by the European Commission (“EC”) for TransCon PTH for the treatment of hypoparathyroidism.
Clinical Development of TransCon PTH for Treatment of Hypoparathyroidism in Adults
TransCon PTH is being evaluated for the treatment of hypoparathyroidism in adults in the Phase 3 PaTHway Trial, Phase 3 PaTHway Japan Trial, and the Phase 2 PaTH Forward Trial. Last patient, last visit (“LPLV”) in the Phase 3 PaTHway Trial occurred in January 2024. LPLV in the Phase 2 PaTH Forward Trial is expected in March of 2025.
In September 2024, we announced results from the ongoing Phase 2 PaTH Forward Trial of adults with hypoparathyroidism showing that long-term treatment with TransCon PTH (palopegteriparatide; marketed as YORVIPATH) through Week 162 drove bone remodeling into the normal range. Deficiency of parathyroid hormone is associated with low rates of bone remodeling, accumulation of overly mature bone, and higher-than-average bone mineral density that may correspond with poorer overall bone quality compared to that seen in the general population. In contrast, these results suggest that long-term palopegteriparatide treatment promotes attainment of skeletal health parameters in line with those expected with states of parathyroid sufficiency. As of December 31, 2024, 10 patients successfully completed the trial, while 46 out of the 59 patients originally enrolled in the trial continued in the open label (“OLE”) portion and had exceeded four and a half years of follow-up. Three patients withdrew from the trial for reasons unrelated to safety or efficacy of the study drug.
| Management Commentary | 22 |
In May 2024, we announced two-year (Week 104) results from a post-hoc analysis of the Phase 3 PaTHway Trial demonstrating sustained improvements (nominal p-value <0.05) in renal function in adults with chronic hypoparathyroidism treated with TransCon PTH. The post-hoc analysis examined the impact of treatment with TransCon PTH on renal function using estimated glomerular filtration rate (“eGFR”) through Week 104 (n=76) of PaTHway, a Phase 3, double-blind, placebo-controlled trial of 82 dosed adults with chronic hypoparathyroidism randomized 3:1 (TransCon PTH:placebo; both arms initially co-administered with conventional therapy of active vitamin D and calcium), with a 26-week blinded period followed by an ongoing 156-week open-label extension period. Across both treatment arms, TransCon PTH treatment resulted in a mean eGFR increase of 8.9 mL/min/1.73m2 (p<0.0001) from baseline at Week 52, sustained at Week 104 with a mean change from baseline of 9.0 mL/min/1.73m2 (p<0.0001). Treatment was generally well-tolerated, with no new safety signals.
eGFR* Change from Baseline by Study Arm
| Baseline | Week 26 | Week 52 | Week 104 | |||||||||||
| Study Arm |
eGFR (mL/min/1.73m2) |
N | Mean (p value) |
N | Mean (p value) |
N | Mean (p value) | |||||||
| TransCon PTH / TransCon PTH |
eGFR < 60 | 19 | +11.4 (p=0.0002) |
19 | +11.5 (p=0.0003) |
18 | +13.4 (p<0.0001) | |||||||
| eGFR ≥ 60 | 41 | +6.3 (p=0.0002) |
40 | +8.6 (p<0.0001) |
40 | +6.9 (p<0.0001) | ||||||||
| All | 60 | +7.9 (p<0.0001) |
59 | +9.3 (p<0.0001) |
58 | +8.9 (p<0.0001) | ||||||||
| Placebo (first 26 weeks) / TransCon PTH** |
eGFR < 60 | 4 | +0.1 (p=0.9877) |
4 | +11.7 (p=0.0018) |
4 | +15.6 (p=0.0067) | |||||||
| eGFR ≥ 60 | 15 | -2.4 (p=0.3280) |
15 | +6.5 (p=0.0199) |
14 | +7.6 (p=0.0121) | ||||||||
| All | 19 | -1.9 (p=0.3468) |
19 | +7.6 (p=0.0014) |
18 | +9.4 (p=0.0006) | ||||||||
| * | eGFR (an assessment of kidney filtering capacity) was calculated by the trial’s central lab using the Modification of Diet in Renal Disease Study Group (MDRD) equation (Levey, Ann Intern Med 2006). An eGFR level <60 mL/min/1.73m2 is considered the threshold for impaired kidney function. |
| ** | Patients in the placebo arm switched to TransCon PTH following the Week 26 visit. |
TransCon PTH treatment was associated with clinically meaningful increases (≥ 5 mL/min/1.73 m2) in eGFR within 26 weeks that were sustained through Week 104 of PaTHway:
Proportion of Participants (%) with ≥ 5 and ≥ 10 mL/min/1.73 m2 Increases in eGFR from Baseline through Week 104*
| All Participants | ||||||||||||||||||||||||
| TransCon PTH / TransCon PTH (n=61) |
Placebo (first 26 weeks) / TransCon PTH** (n=21) |
|||||||||||||||||||||||
| Week 26 | Week 52 | Week 104 | Week 26 | Week 52 | Week 104 | |||||||||||||||||||
| eGFR Change from Baseline |
PTH | PTH | PTH | Placebo | Switch to PTH | Switch to PTH | ||||||||||||||||||
| ≥ 5 mL/min/1.73 m2 |
57 | % | 64 | % | 61 | % | 24 | % | 52 | % | 62 | % | ||||||||||||
| ≥ 10 mL/min/1.73 m2 |
43 | % | 43 | % | 46 | % | 10 | % | 29 | % | 38 | % | ||||||||||||
| Management Commentary | 23 |
| Participants with Baseline eGFR < 60 mL/min/1.73 m2 | ||||||||||||||||||||||||
| TransCon PTH / TransCon PTH (n=19) |
Placebo (first 26 weeks) / TransCon PTH** (n=4) |
|||||||||||||||||||||||
| Week 26 | Week 52 | Week 104 | Week 26 | Week 52 | Week 104 | |||||||||||||||||||
| eGFR Change from Baseline |
PTH | PTH | PTH | Placebo | Switch to PTH | Switch to PTH | ||||||||||||||||||
| ≥ 5 mL/min/1.73 m2 |
74 | % | 68 | % | 74 | % | 25 | % | 100 | % | 100 | % | ||||||||||||
| ≥ 10 mL/min/1.73 m2 |
47 | % | 42 | % | 53 | % | 0 | % | 75 | % | 75 | % | ||||||||||||
| * | Percentages were calculated based on all participants. Patients who did not have an eGFR assessment at the visit were still included in the denominator. |
| ** | Patients in the placebo arm switched to TransCon PTH following the Week 26 visit. |
As of December 31, 2024, 58 patients successfully completed the 182 week follow-up in the PaTHway Trial while 15 of the 82 originally enrolled in the trial continue in the OLE portion of the trial. Nine patients withdrew from the trial for reasons unrelated to safety or efficacy of the study drug.
On January 8, 2023, we announced top-line data from PaTHway Japan, a single-arm Phase 3 trial to evaluate the safety, tolerability, and efficacy of TransCon PTH in adults with hypoparathyroidism. The study achieved its primary objective, with top-line results consistent with our trials in North America and the EU. Twelve out of thirteen patients met the primary multi-component endpoint, which was defined as serum calcium levels in the normal range (8.3–10.6 mg/dL) and independence from conventional therapy (no active vitamin D and ≤600 mg/day of calcium). In this trial, TransCon PTH was generally well-tolerated, with no discontinuations related to study drug. As of December 31, 2024, 12 patients continue in the ongoing 3-year extension portion of the PaTHway Japan Trial.
In March 2022, we announced that top-line data from the randomized, double-blind, placebo-controlled portion of the Phase 3 PaTHway Trial of TransCon PTH in adults with hypoparathyroidism demonstrated statistically significant higher proportion of participants treated with TransCon PTH achieved the primary multi-component endpoint compared to placebo. The primary endpoint, defined as serum calcium levels in the normal range (8.3–10.6 mg/dL) and independence from conventional therapy (no active vitamin D and ≤600 mg/day of calcium) with no increase in prescribed study drug within the 4 weeks prior to the Week 26 visit, was achieved by 78.7% of TransCon PTH-treated patients (48 of 61), compared to 4.8% for patients (1 of 21) in control group (p-value <0.0001). In addition, all key pre-specified secondary endpoints were met with statistical significance. TransCon PTH was generally well tolerated, with no discontinuations related to study drug. Three patients discontinued during the treatment period, two from the placebo arm and one from the TransCon PTH arm. TransCon PTH-treated patients showed a mean decrease in 24-hour urine calcium excretion into the normal range.
TransCon CNP
Market Opportunity in Achondroplasia
Achondroplasia is the most common genetic form of skeletal dysplasia leading to disproportionate short stature and is associated with a well-delineated range of clinical complications and manifestations, occurring in about one in 10,000 to 30,000 newborns or approximately 250,000 worldwide. Achondroplasia results in severe skeletal complications and comorbidities including spinal stenosis due to premature fusion of the foramen magnum, sleep apnea, chronic ear infections, and muscular complications. Patients often face multiple surgeries to alleviate its many complications. There is significant unmet need for treatments that ameliorate complications and improve quality of life in achondroplasia.
Achondroplasia is primarily caused by gain-of-function variants of the FGFR3 gene resulting in constitutive activation of FGFR3 that leads to an imbalance in the effects of the FGFR3 and C-type natriuretic peptide (“CNP”) signaling pathways. In achondroplasia, mutations in FGFR3 result in constitutive activation, suppressing the proliferation and differentiation of chondrocytes resulting in improper cartilage to bone conversion in the growth plate, and dysfunction in the skeletal muscle. Preclinical and clinical data show that the CNP pathway helps to counteract the effects of the FGFR3 mutation downstream.
In November 2021, BioMarin Pharmaceutical Inc.’s daily VOXZOGO® (vosoritide) was approved by the FDA to increase linear growth in pediatric patients with achondroplasia with open epiphyses. Additionally, BioMarin is developing a long-acting CNP to build on VOXZOGO. Other companies that are developing therapies for achondroplasia include QED Therapeutics (a BridgeBio company), Sanofi, Ribomic, Tyra Biosciences, GeneScience and ProLynx.
| Management Commentary | 24 |
Changing the Treatment Paradigm of Achondroplasia
Clinical manifestations of achondroplasia are associated with significant, potentially life-threatening complications and reduced quality of life. While achondroplasia has historically been considered a growth disorder, secondary manifestations beyond linear growth, including reduced muscle strength and stamina, suggest that achondroplasia is also a muscle disorder.
ACH-AE: Increased incidence of Achondroplasia-related Adverse Events.
HRQoL: Reduced Health-Related Quality of Life; Height; Reduced height. Muscle Strength/Stamina; Reduced muscular functionality, including reduced strength and stamina.
Our Solution: TransCon CNP
TransCon CNP (navepegritide) is an investigational prodrug of CNP administered once weekly and designed to provide sustained release of active CNP supporting continuous exposure for the treatment of achondroplasia. TransCon CNP is designed to provide effective shielding of CNP from neutral endopeptidase degradation in subcutaneous tissue and the blood compartment, minimize binding of CNP to the NPR-C receptor to decrease clearance, reduce binding of CNP to the NPR-B receptor in the cardiovascular system to avoid hypotension, and release unmodified CNP, which is small enough in size to allow effective penetration into growth plates. Shorter-acting CNP and CNP analogs in development have resulted high maximum serum concentration (“Cmax”) levels that may cause adverse hypotensive events. We believe the therapeutically sustained release of TransCon CNP offers advantages that may mitigate this issue, leading to continuous CNP exposure with a lower Cmax to correlate with better therapeutic outcomes.
In February 2019, we were granted ODD by the FDA for TransCon CNP for the treatment of achondroplasia. In July 2020, we received OD from the EC for TransCon CNP for the treatment of achondroplasia.
Clinical Development of TransCon CNP for Achondroplasia.
Our ongoing pivotal ApproaCH Trial, our long-term extension trial AttaCH, and COACH, are evaluating the safety and efficacy of TransCon CNP in children with achondroplasia. The reACHin Trial is evaluating the safety, tolerability, and efficacy of TransCon CNP in infants with achondroplasia (aged 0 to < 2 years at the time of randomization). The TeACH Trial is evaluating the safety, tolerability, and efficacy of TransCon CNP in adolescents with achondroplasia (aged 12 to 18).
In January 2025, we announced data demonstrating improvements in leg bowing, a common complication in achondroplasia, observed with TransCon CNP compared to worsening observed with placebo in pivotal ApproaCH Trial.
| Management Commentary | 25 |
In September 2024, we announced top-line data from ApproaCH, a pivotal, multicenter, randomized, double-blind, placebo-controlled trial of once-weekly TransCon CNP versus placebo in 84 children (aged 2 to 11 years) with achondroplasia. Participants were randomized 2:1 to receive TransCon CNP 100µg/kg/week or placebo for 52 weeks in the double-blind period, after which all participants could choose to receive TransCon CNP at the 100µg/kg/week dose in an ongoing open-label extension. In the trial, children treated with once-weekly TransCon CNP demonstrated annualized growth velocity (“AGV”) superior to those treated with placebo. TransCon CNP also demonstrated statistically significant improvements in other growth parameters, including height Z-score and change from baseline AGV.
Highlights of the ApproaCH Trial Top-line Data
Primary Endpoint
| • | For the primary endpoint of AGV at Week 52, children treated with TransCon CNP (n=57) demonstrated an LS mean AGV of 5.89 cm/year compared to 4.41 cm/year in the placebo arm (n=27), an LS mean difference of 1.49 cm/year (p<0.0001). |
| • | Sub-group analyses: |
| • | Children aged 2 to <5 years treated with TransCon CNP (n=21) demonstrated an LS mean AGV at Week 52 of 6.07 cm/year compared to 5.06 cm/year in the placebo arm (n=10), an LS mean difference of 1.02 cm/year (p=0.0084). |
| • | Children aged 5-11 years treated with TransCon CNP (n=36) demonstrated an LS mean AGV at Week 52 of 5.79 cm/year compared to 4.02 cm/year in the placebo arm (n=17), an LS mean difference of 1.78 cm/year (p<0.0001). |
AGV Change from Baseline
| • | Children aged 2 to <5 years, treated with TransCon CNP (n=19) demonstrated a change from baseline AGV at Week 52 of 1.57 cm/year compared to 0.43 cm/year in the placebo arm (n=10), an LS mean difference of 1.15 cm/year (p=0.0047). |
| • | Children aged 5-11 years, treated with TransCon CNP (n=35) demonstrated a change from baseline AGV at Week 52 of 2.29 cm/year compared to 0.52 cm/year in the placebo arm (n=17), an LS mean difference of 1.78 cm/year (p<0.0001). |
Secondary Endpoints
| • | For the secondary endpoint of change in ACH Height Z-score, children treated with TransCon CNP (n=57) demonstrated an LS mean change from baseline ACH Height Z-score of 0.30 compared to 0.01 in the placebo arm (n=27), an LS mean difference of 0.28 (p<0.0001). |
| • | For the secondary endpoint of change in CDC Height Z-score, children treated with TransCon CNP (n=55) demonstrated an LS mean change from baseline CDC Height Z-score of 0.15 compared to -0.15 in the placebo arm (n=27), an LS mean difference of 0.30 (p=0.0003). |
| Management Commentary | 26 |
Safety Results Summary
| • | TransCon CNP was generally well-tolerated and demonstrated safety profile comparable to those observed in the placebo arm, with generally mild treatment emergent adverse events (TEAEs), no evidence of hypotensive effect, and a low frequency of injection site reactions (0.41 events per patient year), all mild. |
| • | No adverse events (“AEs”) led to discontinuation of TransCon CNP or withdrawal from the trial and no serious adverse events (“SAEs”) were assessed as related to TransCon CNP. |
Following our pre-NDA meeting with FDA, we plan to submit a New Drug Application (“NDA”) for the treatment of children with achondroplasia during the first quarter of 2025 and submit a Marketing Authorisation Application (“MAA”) for the treatment of children with achondroplasia to the EMA during the third quarter of 2025.
In December 2023, we announced new analyses demonstrating benefits beyond linear growth from the blinded and ongoing OLE periods of ACcomplisH, a Phase 2 randomized, double-blind, placebo-controlled, dose-escalation trial of TransCon CNP in children aged 2 to 10 years with achondroplasia. In the trial, all 57 patients have now completed one year of treatment with TransCon CNP at 100 µg/kg/week, the dose agreed with regulatory agencies for the active arm in our pivotal ApproaCH Trial.
We analyzed available data for patients who only received TransCon CNP at the 100 µg/kg/week dose in either the blinded or OLE period and were treated for one year (n=19), compared to those administered placebo for one year (n=15). Results showed that these TransCon CNP-treated patients (data available for 9-16 patients) showed improvements (nominal p-value <0.05) in health-related quality of life and disease impacts compared to those receiving placebo (data available for 5-13 patients).
Assessments were performed with the SF-10 (a 10-item non-disease specific survey of a child’s functional health and well-being that has been validated to assess children aged 5 years and older) and the Achondroplasia Child Experience Measure (“ACEM”) a condition-specific clinical outcome measure that assesses the impact of achondroplasia on a child’s health-related quality of life, with statistically significant improved outcome in TransCon CNP-treatment versus placebo for:
| • | SF-10 Physical Summary (p=0.002, aged 5 years and older) |
| • | ACEM Daily Living Function (p=0.047) |
| • | ACEM Emotional Well-being (p=0.045) |
The 46 children switching from placebo or a lower dose of TransCon CNP to the 100 µg/kg/week dose in the OLE demonstrated improved growth after one year of treatment, similar to the growth benefits seen in the 11 children treated with 100 µg/kg/week in the one-year randomized, double-blind period of ACcomplisH.
| Management Commentary | 27 |
While the CNP pathway may restore normal growth and skeletal muscle function, we believe delayed initiation of therapy could lead to a permanent height deficit. Clinical use of daily growth hormone injection has consistently demonstrated growth improvements in children with achondroplasia, including catch up growth; however, without reports of benefits beyond linear growth. We believe the combination of TransCon hGH and TransCon CNP, taken together once per week, could enable catch up growth beyond normal growth while maintaining potential benefits on skeletal muscle of continuous CNP exposure. COACH, a Phase 2 open-label single-arm trial, is evaluating TransCon CNP and TransCon hGH in children with achondroplasia (age 2 to 11 years). The primary objective is to evaluate the treatment effect on linear growth and safety. Secondary objectives are to evaluate treatment effect on quality of life, radiological endpoints, physical functioning, and body composition. The trial plans to enroll approximately 18 patients (treatment naïve, n=12; prior treatment with TransCon CNP (100 µg/kg/week) for at least 1 year, n=6). Week 26 top-line data from the COACH Trial are expected in the second quarter of 2025.
During the third quarter of 2023, we filed an IND amendment with the FDA to initiate reACHin, a Phase 2, multicenter, double-blind, randomized, placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 µg/kg of TransCon CNP once-weekly for 52 weeks in infants with achondroplasia, aged 0 to < 2 years at the time of randomization.
In November 2022, we announced top-line results from ACcomplisH, a Phase 2 randomized, double-blind, placebo-controlled, dose-escalation trial evaluating the safety and efficacy of once-weekly TransCon CNP compared to placebo in children with achondroplasia aged 2 to 10 years old.
The ACcomplisH Trial evaluated 57 children with achondroplasia aged 2 to 10 years old, randomized in a 3:1 ratio to receive either sequential ascending doses of once-weekly TransCon CNP (6 µg/kg/week, 20 µg/kg/week, 50 µg/kg/week, 100 µg/kg/week) or placebo for 52 weeks. The trial met its primary objectives, demonstrating that TransCon CNP at 100 µg/kg/week (n=11) was superior to placebo (n=15) on the primary efficacy endpoint of AGV at 52 weeks (p=0.0218). All 57 randomized children completed the blinded portion of ACcomplisH and continued in the OLE portion of ACcomplisH at the 100 µg/kg/week dose.
As of December 31, 2024, 55 patients completed the OLE portion of the ACcomplisH Trial; 2 patients prematurely withdrew during the OLE. From ACcomplisH OLE, 53 patients transitioned into the Phase 2 AttaCH Trial, a multicenter, long-term, open-label extension trial to continue treatment with TransCon CNP 100 µg/kg/week and 2 patients transitioned into COACH, a TransCon CNP/TransCon hGH combination trial. As of December 31, 2024, 43 patients continue in AttaCH, on long-term OLE treatment; 3 patients prematurely withdrew from treatment and 7 patients transitioned into COACH. With the last subject completing the OLE portion of the ACcomplisH Trial, the ACcomplisH Trial has completed.
In 2019, we initiated the ACHieve Study, a five-year, multi-center natural history study designed to gain insight into the experiences of pediatric patients with achondroplasia. ACHieve was designed to evaluate growth velocity, body proportionality, and comorbidities over time in children with achondroplasia up to eight years old. No study medication was administered in the ACHieve Study. The study ended in the first quarter of 2024. We plan to make the results available in 2025.
TransCon Product Candidates — Oncology
Market Opportunity in Oncology
Cancer continues to be one of the leading causes of mortality. Improved understanding of the cellular and molecular mechanisms involved in anti-tumor immune responses has fueled the rapid growth of immuno-oncology therapeutics. Immune checkpoint inhibitors, such as anti-PD-(L)1 and anti-CTLA-4 antibodies, have provided new therapeutic options for patients.
| Management Commentary | 28 |
Despite recent advances, a high need for new treatment options remains for patients who do not respond to, or who respond inadequately to, current therapies. In addition to insufficient efficacy, many current treatments are limited by toxicities that result in dose reductions, treatment discontinuations, or long-term health risks to patients.
We believe that one approach to improving efficacy while limiting adverse events is to create long-acting product candidates using our sustained systemic release TransCon technology, allowing for more consistent circulating drug levels and potentially avoiding high peak concentrations that are often associated with toxicity.
We are currently developing TransCon technology in oncology for a variety of solid tumors, with encouraging early data in platinum resistant ovarian cancer and melanoma. Aside from Proleukin being the only approved IL-2, TransCon IL-2 b/g may face competition from other IL-2 type drug candidates in development, including those being developed by Mural, Anaveon, Medicenna, Roche, Innovent, Synthekine, Dragonfly, Sotio, and Aulos. In addition, TransCon IL-2 b/g may face competition from drug candidates in development for platinum resistant ovarian cancer, including Merck, Corcept, Novartis, Advenchen Laboratories, Genelux, Daiichi Sankyo and Mereo/OncXerna. In melanoma, TransCon IL-2 b/g may face competition from drug candidates in development including from Replimune, Philogen, OncoSec, Immunocore, and Regeneron.
Our Solution: TransCon Technologies for Oncology
We believe prolonging the therapeutic activity and targeting the drug activity to the relevant cell types and tissues have the potential to improve treatment outcomes. We believe TransCon is well-suited to improve cancer treatments given the large number of validated targets with known limitations. By applying our unique algorithm for product innovation to clinically validated targets and pathways, we believe TransCon has the potential to improve outcomes currently limited by suboptimal efficacy and systemic toxicity.
We believe TransCon technologies may have the potential to increase the efficacy of small molecules, peptides and proteins without increasing toxicity, which could offer the potential to treat more patients with new combination and multi-agent regimens that would not otherwise be feasible.
We are currently investigating one clinical-stage product candidate designed to activate the patient’s own immune system to eradicate malignant cells. We believe our approach, if successfully developed, has the potential to improve the efficacy of systemically administered, clinically validated therapies while limiting adverse effects.
Our TransCon product candidate currently being investigated in solid tumors is designed to provide sustained systemic administration, which we believe could provide potent and durable anti-tumor efficacy. Our nonclinical studies have shown sustained activation of cytotoxic immune cells that resulted in robust anti-tumor responses by TransCon product candidates using infrequent administration.
TransCon IL-2 b/g for Sustained Systemic Release
TransCon IL-2 b/g (onvapegleukin alfa) is an investigational long-acting prodrug designed to improve cancer immunotherapy through sustained release of an IL-2 variant that selectively activates IL-2 b/g, with minimal binding to IL-2Rα. The IL-Believe Trial, a Phase 1/2 clinical trial to evaluate the safety and efficacy of TransCon IL-2 b/g in locally advanced or metastatic solid tumors, alone or in combination with pembrolizumab or standard of care chemotherapy, has completed dose escalation and is enrolling patients in multiple indication-specific dose expansion cohorts, including platinum-resistant ovarian cancer (“PROC”), cervical cancer, melanoma, non-small cell lung cancer (“NSCLC”), and small cell lung cancer (“SCLC”) at the RP2D.
In September 2024, we announced initial data showing signs of clinical activity in heavily pre-treated patients with PROC treated (cohort 3) with TransCon IL-2 b/g in combination with chemotherapy in the ongoing Phase 1/2 IL-Believe Trial of TransCon IL-2 b/g. As of a cutoff date of July 29, 2024, of the 18 patients (median age 64 years) included in the initial assessment, 14 were efficacy evaluable patients who had one or more post-baseline tumor assessment(s), plus an additional four who discontinued treatment before the first post-baseline tumor assessment due to disease progression or death.
| Management Commentary | 29 |
As of the data cutoff, clinical responses were observed in 29% (4/14) of the efficacy evaluable patients (two confirmed and two unconfirmed partial responses in patients who had received three to seven prior lines of treatment – including patients whose disease had previously progressed on mirvetuximab soravtansine-gynx), suggesting the potential for clinical activity in heavily pre-treated patients. The data suggest that TransCon IL-2 b/g was generally well-tolerated: the most common TEAEs related to combination therapy with TransCon IL-2 b/g plus chemotherapy were fatigue, thrombocytopenia, neutropenia, and anemia. Most TransCon IL-2 b/g-related TEAEs were grade 1 or 2.
In June 2024, we reported updated results from our ongoing Phase 1/2 IL-Believe Trial of TransCon IL-2 b/g. Data included the first presentation of Phase 2 dose expansion Cohort 4 (TransCon IL-2 b/g in combination with TransCon TLR7/8 Agonist) in post anti-PD-1 melanoma and new analyses of patients from dose escalation cohorts with prior disease progression on checkpoint inhibitors, along with biomarker studies correlating cytotoxic immune cell expansion and observed clinical benefit. As of the April 16, 2024, data cutoff, confirmed clinical partial responses were observed in 40% (two out of five) of efficacy-evaluable patients from Cohort 4, suggesting potential synergy of our two novel immunotherapy candidates in patients who did not derive sufficient benefit from checkpoint inhibitors. Of efficacy-evaluable patients with prior disease progression on checkpoint inhibitors to date (from Phase 1 dose escalation cohorts) in the IL-Believe Trial, confirmed clinical responses (per RECIST v1.1) were observed in 45% (five out of eleven) administered TransCon IL-2 b/g doses ≥80 µg/kg every 3 weeks, suggesting clinical benefit in treatment-resistant settings (monotherapy (n=4): 1 confirmed partial response (“PR”) in colorectal cancer; combination with pembrolizumab (n=2): 1 confirmed complete response and 1 confirmed PR in small-cell lung cancer; combination with TransCon TLR7/8 Agonist (n=5): 2 confirmed PRs in melanoma). In this trial, TransCon IL-2 b/g alone or in combination with pembrolizumab or TransCon TLR7/8 Agonist was generally well tolerated with no new safety signals.
In October 2023, we announced updated data from the ongoing Phase 1 dose escalation cohort from IL-Believe Trial. Forty-six patients were enrolled into dose escalation cohorts: 25 to monotherapy and 21 to combination therapy. As of the August 15, 2023, data cutoff, anti-tumor clinical responses were observed with TransCon IL-2 b/g monotherapy (colorectal cancer with PR) or in combination with pembrolizumab (small cell lung cancer, one with confirmed PR and one ongoing with unconfirmed complete response) in heavily pre-treated patients who previously progressed on checkpoint inhibitors. TransCon IL-2 b/g every three weeks was generally well-tolerated, with no meaningful effect on Tregs and eosinophils.
In September 2023, we announced completion of Phase 1 dose escalation in combination with pembrolizumab of the IL-Believe Trial with a total of 21 patients enrolled and RP2D determined at 120 µg/kg IV every three weeks. Twenty-one patients were enrolled.
In May 2023, we announced completion of the Phase 1 monotherapy dose escalation of the IL-Believe Trial with RP2D determined at 120 µg/kg IV every three weeks with 25 heavily pre-treated patients enrolled and a median of four prior lines of systemic therapies.
Strategic Collaborations
We also engage in strategic collaborations to further leverage our TransCon technologies in certain geographies and therapeutic areas with market-leading biopharmaceutical companies. These collaborations aim to make promising treatment options available to more patients and to further monetize both our TransCon technologies and our internal product candidates, particularly into therapeutic areas where we believe a partner may have more expertise, capability, and capital. In addition, we may choose to pursue a collaboration to develop and market our internal, wholly owned product candidates in geographic markets outside our core focus areas of the United States and Europe.
| Management Commentary | 30 |
Novo Nordisk A/S
In November 2024, we entered into a research and development collaboration and license agreement with Novo Nordisk A/S (“Novo Nordisk”) pursuant to which we granted Novo Nordisk an exclusive worldwide license to the TransCon technology platform to develop, manufacture and commercialize Novo Nordisk proprietary products (including Semaglutide) in metabolic diseases (including obesity and type 2 diabetes) and a product-by-product exclusive license in cardiovascular diseases.
The agreement includes provisions requiring at least one TransCon Semaglutide product and at least one other TransCon technology-based product to be identified, developed and commercialized in metabolic diseases to maintain certain exclusivities in the field, with additional provisions for cardiovascular diseases. Under the terms of the agreement, Novo Nordisk also receives exclusive rights to expand any resulting metabolic disease products into other therapeutic areas. The lead program in the collaboration is a once-monthly TransCon Semaglutide product candidate that will initially target obesity and type 2 diabetes.
Under the agreement, we have the potential to receive total payments of up to $285 million in upfront, development and regulatory milestone payments for the lead program. In addition, we have the potential to receive sales-based milestone payments and tiered royalties on global net sales.The $285 million includes an upfront fee of $100 million for the exclusive license. For each additional metabolic or cardiovascular disease product candidate, we will be eligible to receive payments of up to $77.5 million in development and regulatory milestone payments. In addition, we have the potential to receive sales-based milestone payments and tiered royalties on global net sales. Novo Nordisk agreed to pay royalties for each potential licensed product developed under the agreement that are an escalating tiered, mid-single digit percentage of the annual net sales of such licensed product and are subject to reduction due to patent valid claim expiration, biosimilar product market share, payment made under certain licenses for third party intellectual property and Inflation Reduction Act price negotiations.
Under the agreement, we have agreed to conduct certain pre-agreed early research and development of TransCon product candidates under the collaboration and we are eligible to receive cost reimbursement from Novo Nordisk for its performance of such research and development activities under the agreement with respect to such TransCon product candidates. Novo Nordisk is responsible for any other non-clinical and clinical development, regulatory, commercial manufacturing, and commercialization of such TransCon product candidates, and all costs associated with such activities.
Subject to the terms of the agreement, we granted Novo Nordisk an exclusive, worldwide, royalty-bearing license, with the right to grant sublicenses, to use its proprietary TransCon technology platform to develop, manufacture and commercialize Novo Nordisk proprietary products in metabolic diseases (including obesity and type 2 diabetes) and a product-by-product exclusive license in cardiovascular diseases. Additionally, we granted Novo Nordisk an exclusive, worldwide, royalty-bearing license, with the right to grant sublicenses, to use its proprietary TransCon technology platform to develop, manufacture and commercialize GLP-1 receptor products using the TransCon technology for all indications, except for (i) certain pre-agreed rare endocrine indications, (ii) all indications in respect of the eye and adnexa and (iii) all indications in respect of oncology.
Until expiry of the last royalty term and for one-year thereafter, we are not permitted to research, develop, manufacture, commercialize, or otherwise exploit outside of the collaboration, any GLP-1 receptor product or any other licensed products that have been subject to the collaboration. We are also not permitted to undertake any research, development, manufacture, commercialization, or other exploitation of products outside of the collaboration in the metabolic field until expiry of the last royalty term of any licensed products that have been subject to the collaboration in metabolic diseases.
Unless earlier terminated, the agreement has a royalty term that continues, on a per licensed product and per country basis, until the later of (i) the expiration of the last valid patent claim for any of our patents, joint improvement patents, licensed product patents as well as any improvements made by Novo Nordisk covering the licensed product’s dosage regimen or target product profile, or (ii) 11 years after the first commercial sale of such licensed product in such country.
Novo Nordisk has the right to terminate the agreement without cause in its entirety or on a per licensed product basis. We have the right to terminate the agreement in its entirety in case Novo Nordisk brings patent challenges with respect to our patents. The agreement may also be terminated by either party based on an uncured material breach by the other party or the bankruptcy of the other party.
| Management Commentary | 31 |
Upon termination of the agreement due to Novo Nordisk’s default, some or all of the licenses granted by us to Novo Nordisk to develop, manufacture and commercialize any of the licensed products will automatically terminate.
Upon termination of the agreement due to certain defaults by us, Novo Nordisk may choose to either (i) have the license granted by us to Novo Nordisk to develop, manufacture and commercialize licensed products terminate in its entirety or on a product-by-product basis; or (ii) continue with respect to the affected licensed product at a reduced payment rate.
Teijin Limited
In November 2023, we announced that we entered into an exclusive license agreement with Teijin Limited for the further development and commercialization of TransCon hGH, TransCon PTH, and TransCon CNP for endocrinology rare disease in Japan. Under the terms of the agreement with Teijin Limited, we received an upfront payment of $70 million, with additional development and regulatory milestones of up to $175 million, transfer pricing and commercial milestones. In addition, we are eligible to receive royalties on net sales in Japan, of up to a mid-20’s percentage, varying by product.
In December 2024, Teijin Pharma Limited announced the submission of an application for manufacturing and marketing approval of palopegteriparatide for the treatment of hypoparathyroidism in Japan.
Strategic Investments
VISEN Pharmaceuticals
In November 2018, we announced the formation of VISEN, a company established to develop and commercialize our endocrinology rare disease therapies in the People’s Republic of China, Hong Kong, Macau, and Taiwan (“Greater China”). In connection with the formation of VISEN, we granted VISEN exclusive rights to develop and commercialize certain product candidates based on our proprietary TransCon technologies, including TransCon hGH, TransCon PTH, and TransCon CNP, in Greater China for use in all human indications, subject to certain exceptions. As consideration for the rights granted to VISEN, we received 50.0% ownership in the outstanding shares of VISEN and concurrently with the rights we granted to VISEN, entities affiliated with Vivo Capital and Sofinnova Ventures purchased shares in VISEN for an aggregate purchase price of $40 million in cash. In January 2021, we invested additional $12.5 million in VISEN as part of VISEN’s $150 million Series B financing. As of December 31, 2024, our ownership in VISEN was 43.9%.
In August 2024, VISEN announced top-line data from the 26-week randomized, double-blind, placebo-controlled portion of the Phase 3 PaTHway China Trial of Palopegteriparatide (TransCon PTH) in adults with chronic hypoparathyroidism. VISEN reported a statistically significant higher proportion of patients treated with palopegteriparatide achieved the primary multi-component endpoint compared to placebo. The primary multi-component endpoint was achieved by 77.6% of palopegteriparatide-treated patients (45 of 58), compared to 0.0% of patients (0 of 22) in the placebo group (p-value <0.0001). Results were consistent with those announced by us for its palopegteriparatide Phase 3 trial.
In March 2024, VISEN announced that the BLA for lonapegsomatropin (TransCon hGH) was accepted by the China National Medical Products Administration.
In November 2023, VISEN announced top-line results from the Phase 2 ACcomplisH China Trial in children with achondroplasia aged 2 to 10 years. VISEN reported that patients dosed with TransCon CNP at the 100 µg CNP/kg/week showed significantly higher AGV than placebo at Week 52.
| Management Commentary | 32 |
In November 2022, VISEN announced data from its pivotal Phase 3 study of TransCon hGH in children with GHD in China. VISEN reported that patients dosed with TransCon hGH demonstrated an annualized height velocity (“AHV”) of 10.66 cm/year compared to 9.75 cm/year for the daily hGH at 52 weeks (treatment difference at 0.91 cm/year with a 95 percent confidence interval: 0.37 – 1.45 cm/year, p=0.0010), reaching its primary objective, demonstrating that TransCon hGH is non-inferior to the daily hGH.
Market Opportunity in China
China is the second largest pharmaceutical market in the world after the United States and represents one of the fastest growing pharmaceutical markets worldwide. In recent years, the Chinese government has initiated a number of regulatory reforms that are expected to accelerate drug development, as well as drive growth and demand for new therapeutics in China. In addition to joining an international organization that standardizes regulations for clinical development, the National Medical Products Administration has introduced initiatives such as fast track review for drugs for unmet medical needs and adopted new rules that streamline the drug approval process in China for global companies.
The purpose of our investment in VISEN is to support our strategy to extend our endocrinology rare disease portfolio globally and establish a presence in China in partnership with collaborators who have significant experience and knowledge of the biopharmaceutical opportunity in China.
Eyconis, Inc
In January 2024, we announced the formation and launch with Frazier Life Sciences of Eyconis, Inc., a separate company created to develop, manufacture, and commercialize TransCon ophthalmology assets globally, together with a $150 million commitment from an investor syndicate that included Frazier, RA Capital Management, venBio, and HealthQuest Capital.
We have granted Eyconis exclusive rights to develop and commercialize TransCon ophthalmology products globally and received an equity position in the newly formed company. In addition, we are eligible to receive development, regulatory, and sales milestone payments, plus single digit royalties on global net sales of commercialized products, if any. As of December 31, 2024, our ownership in Eyconis was 41.6%.
Financial Review
We had a net loss of €378.1 million for the year ended December 31, 2024 compared to a net loss of €481.4 million for the year ended December 31, 2023. Operating loss was €278.8 million, representing an improvement of €176.8 million compared to December 31, 2023, which, in addition to an increase in revenue, was driven by reduced operating expenses. Our total equity presented a negative balance of €105.7 million as of December 31, 2024, compared to a negative balance of €145.7 million as of December 31, 2023. Further details about our results of operations are described in the following sections.
All employees in Denmark (domicile country) are employed by the Parent Company, and accordingly, neither of the Danish subsidiaries have employees. Furthermore, all external, project related expenses, as well as site costs incurred by foreign subsidiaries are being financed by the Parent Company. All direct related project expenses are invoiced to subsidiaries that holds the license rights for the product candidates. In addition, the Parent Company provides services to subsidiaries, which are disclosed as revenue in the Parent Company’s separate financial statements. All intergroup transactions are made on an arms-length basis and eliminated in the consolidated financial statements.
Accordingly, operating results in the Parent Company highly depend on project related activities in the Group.
Income and Expenses
Revenue from sale of commercial products and clinical trial supply is recognized when the customer has obtained control of the goods and it is probable that we will collect the consideration to which we are entitled for transferring the goods. Control is transferred upon delivery. Cost of sales are recognized when the sales takes place. Rendering of services is recognized as revenue over the service period as stipulated under the applicable agreement. License agreements which transfer rights to our intellectual property (“IP”) with significant stand-alone value are classified as “right-to-use,” with revenue recognized at the point in time when the customer can use and benefit from the IP.
| Management Commentary | 33 |
Our operating expenses relate to research and development activities and to selling, general, and administration activities. Research and development costs (“R&D costs”) consist primarily of product development and pre-commercial manufacturing costs, preclinical and clinical study costs and costs for process optimizations and improvements performed by Clinical Research Organizations (“CROs”) and Contract Manufacturing Organizations (“CMOs”), salaries and other personnel costs including pension and share-based payment, the cost of facilities, professional fees, cost of obtaining and maintaining our IP portfolio, and depreciation of non-current assets used in research and development activities. Selling, general, and administrative expenses (“SG&A expenses”) comprise salaries and other personnel costs including pension and share-based payment, office supplies, cost of facilities, professional fees, and depreciation and amortization of non-current assets related to selling, general, and administrative activities, and pre-commercial and commercial activities.
A material portion of our operating expenses are denominated in other currencies than the Euro, which expose our operating expenses to volatility. We do not currently enter into derivative financial instruments to manage our exposure to foreign exchange risks.
Revenue
Revenue for the year ended December 31, 2024 was €363.6 million, representing an increase of €96.9 million compared to last year. This increase was primarily attributable to higher revenue from licenses associated with our exclusive license agreement with Novo Nordisk, signed in November 2024, which includes a $100 million upfront payment, recognized in 2024, and our exclusive license agreement with Eyconis in January 2024 of €27.1 million (non-cash) upfront payment adjusted for internal profit, compared to the $70 million upfront payment received from our exclusive license agreement with Teijin in 2023.
In addition, revenue was positively impacted from increased sales of SKYTROFA in the U.S. and the commercial launch of YORVIPATH in Europe. Revenue for SKYTROFA was negatively impacted by an adjustment to estimates and assumptions for sales deductions of €4.7 million, which were attributable to years prior to January 1, 2024. The adjustment was primarily attributable to a different payer and rebate mix than anticipated, and on which provisions for prior years were based.
Cost of Sales
Cost of sales for the year ended December 31, 2024 was €44.3 million, representing a decrease of €0.1 million compared to last year. This decrease was primarily attributable to lower costs under our license and collaboration agreements, partly offset by increased sales of commercial products.
Research and Development Costs
R&D costs for the year ended December 31, 2024 were €307.0 million representing an decrease of €106.5 million compared to last year. This decrease was primarily due to:
| • | Maturity of our endocrinology rare disease pipeline, especially regarding TransCon hGH and TransCon PTH. In addition, 2024 include reversal (income) of prior period write-downs of pre-launch inventories for TransCon PTH of €12.6 million, due to the commercial launch of YORVIPATH in Europe and the U.S. in 2024; |
| • | Lower product development activities on our TransCon TLR7/8 Agonist and TransCon IL-2 b/g programs, partly offset by increased clinical trial activities. During the fourth quarter of 2024, we closed enrollment in our BelieveIT-201 clinical trial and to dose expansion cohorts involving TransCon TLR7/8 Agonist in the transcendIT-101 and IL-Believe trials to prioritize our efforts on TransCon IL-2 b/g ; and |
| • | Cessation of ophthalmology expenses, including related employee costs, due to the grant of exclusive rights to develop and commercialize TransCon ophthalmology to Eyconis in January 2024. |
| Management Commentary | 34 |
Selling, General and Administrative Expenses
SG&A expenses for the year ended December 31, 2024 was €291.1 million representing an increase of €26.7 million compared to last year. This increase was primarily due to higher employee related expenses and other general and administrative expenses attributable to organizational growth in support of launch of YORVIPATH in Europe and the U.S., partly offset by lower administrative expenses.
Finance Income and Finance Expenses
Finance income and Finance Expenses for the year ended December 31, 2024 was a net expense of €74.4 million representing a decrease of €74.2 million. This decrease was primarily driven foreign exchange losses from translation of U.S. dollar denominated monetary positions into Euro, primarily cash and cash equivalents, convertible notes and royalty funding liabilities. The development was further driven by amortization charges, accruals, and other items, primarily due to our royalty funding liabilities which we entered into in September 2023 and September 2024, and from lower remeasurement gains from financial liabilities.
For further details, please refer to Note 16, “Financial Assets and Liabilities”.
Cash Flows from / (used in) Operating Activities
Cash flows used in operating activities for the year ended December 31, 2024 were €306.2 million, representing a decrease of €161.2 million compared to last year.
This improvement was primarily related to higher revenue and reduced operating expenditures, and to further adjustment for changes to non-operating financial income and expenses, taxes and non-cash items of total €168.5 million, partly offset by negative impact from working capital balances with €7.4 million. The negative impact from working capital balances includes $100 million plus indirect taxes related to upfront payment from our exclusive license agreement with Novo Nordisk, which was subsequently settled in January 2025.
Cash Flows from / (used in) Investing Activities
Cash flows from investing activities for the year ended December 31, 2024 were €6.9 million, representing a decrease of €279.6 million compared to last year. This increase was primarily attributable to higher net settlement of marketable securities.
Cash Flows from / (used in) Financing Activities
Cash flows from financing activities for the year ended December 31, 2024, were €443.9 million, representing an increase of €309.6 million compared to the last year. This increase was primarily attributable to our follow-on public offering of ADSs completed in September 2024 with net proceeds of €290.6 million and increased warrant exercise activity of €20.2 million. Similar to 2023, we entered into a $150.0 million capped synthetic royalty funding agreement with Royalty Pharma.
Liquidity and Capital Resources
Our liquidity and capital resources comprise cash and cash equivalents. As of December 31, 2024, these amounted to €559.5 million.
Our expenditures primarily relate to research and development activities and selling, general, and administrative activities to support our business, including our continued development of product and product candidates within Endocrinology Rare Disease and Oncology portfolios, the commercialization of SKYTROFA and YORVIPATH, and expenses made in anticipation of potential future product launches. We manage our liquidity risk by maintaining adequate cash reserves and banking facilities.
| Management Commentary | 35 |
We monitor the risk of a shortage of funds through a liquidity planning tool to ensure sufficient funds are available to settle liabilities as they become due.
As of December 31, 2024, the consolidated statements of financial position presented a negative balance of equity of €105.7 million. Under Danish corporate law, as Ascendis Pharma A/S, the parent company of the Company, holds a positive balance of equity, the Company is currently not subject to legal or regulatory requirements to re-establish the balance of equity. There is no direct impact from the negative balance of equity to the liquidity and capital resources.
Based on our current operating plan, we believe that our existing capital resources as of December 31, 2024 will be sufficient to meet our projected cash requirements for at least twelve months from the date of this annual report. However, our operating plan may change as a result of many factors currently unknown to us, and we may need to seek additional funds sooner than planned.
Historically, we have funded our operations primarily through the issuance of preference shares, ordinary shares (including public offerings and exercise of warrants), convertible debt securities, payments to us made under collaboration agreements, and our royalty funding agreements. Including our initial public offering, since February 2015, we have completed public offerings of American Depositary Shares (“ADSs”), latest in September 2024, with total net proceeds of $2,580.2 million (or €2.259.0 million at the time of the offerings).
Uncertainty Relating to Recognition and Measurement
When preparing the annual report, it is necessary that Management, in accordance with legislative provisions, makes a number of accounting judgements and estimates which form the basis for the annual report. The accounting judgements and estimates made by Management are described in Note 3, “Significant Accounting Judgements and Estimates.”
Risk Management
Business Risks
The Group is exposed to certain risks that are common across the biopharmaceutical industry, including but not limited to risks that pertain to research and development, regulatory approval, commercialization, intellectual property rights and access to financing, and some risks that are specific to the Group’s development programs and technology platform.
Some of these risks may significantly affect the Group’s ability to execute its strategy and in order to mitigate such risks, the Group has identified and categorized these risks as critical risks and has a program in place to ensure proactive identification, management and mitigation of such risks.
Financial Risks
We regularly monitor the access to domestic and international financial markets, manage the financial risks relating to our operations, and analyze exposures to risk, including market risk, such as currency risk and interest rate risk, credit risk and liquidity risk. Financial risk management is further described in Note 17, “Financial Risk Management”.
Corporate Responsibility
Ascendis Pharma A/S has established a framework of corporate policies and rules which governs compliance by the Company, employees and business partners with applicable laws and regulations, and the Ascendis Pharma Code of Business Conduct & Ethics.
The Ascendis Pharma A/S Sustainability & P|ESG Report 2024 defines our compliance with Section 99a (CSR) and Section 99d (Data ethics) of the Danish Financial Statements Act.
Find more detailed information in the Ascendis Pharma Sustainability & P|ESG Report 2024 at: https://investors.ascendispharma.com/financial-and-filings/annual-general-meetings/sustainability-and-p-esg-report-2024
| Management Commentary | 36 |
Events after the Balance Sheet Date
No events have occurred after the reporting date that would influence the evaluation of these financial statements.
Outlook
Having achieved pivotal milestones in 2024, Ascendis is positioned to continue strong revenue growth in 2025 and beyond. We believe YORVIPATH is well on its way to establishing itself as the new global standard for the treatment of hypoparathyroidism in adults. SKYTROFA has achieved a leading position in value in the U.S. growth hormone market. And for TransCon CNP, we have a clear path to submit our NDA and MAA as a differentiated treatment of achondroplasia in children.
We expect that our SG&A expenses may increase over the years as we expand our commercial organization. We expect stable R&D investments as our current portfolio matures and early stage pipeline renews.
The Company is listed under the symbol “ASND” in the United States on The Nasdaq Global Select Market, where the Company at the time of approval of this annual report, has not provided a quantitative financial outlook. Accordingly, and due to equal information to investors, no further outlook is disclosed in this annual report.
| Financial Statements | 37 |
Statements of Profit or Loss and Other Comprehensive Income for the Years Ended December 31
| Group | Parent | |||||||||||||||||||
| (EUR’000) | Notes | 2024 | 2023 | 2024 | 2023 | |||||||||||||||
| Statement of Profit or (Loss) |
||||||||||||||||||||
| Revenue |
4 | 363,641 | 266,718 | 405,780 | 302,712 | |||||||||||||||
| Cost of sales |
6,11 | 44,258 | 44,395 | 45,777 | 51,942 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Gross profit |
319,383 | 222,323 | 360,003 | 250,770 | ||||||||||||||||
| Research and development costs |
6,11 | 307,004 | 413,454 | 80,596 | 75,026 | |||||||||||||||
| Selling, general and administrative expenses |
6,11 | 291,142 | 264,410 | 153,273 | 178,935 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Operating profit/(loss) |
(278,763 | ) | (455,541 | ) | 126,134 | (3,191 | ) | |||||||||||||
| Share of profit/(loss) of associates |
12 | (20,060 | ) | (18,395 | ) | — | — | |||||||||||||
| Finance income |
16 | 25,609 | 43,857 | 94,173 | 77,624 | |||||||||||||||
| Finance expenses |
16 | 100,027 | 44,065 | 45,567 | 34,714 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Profit/(loss) before tax |
(373,241 | ) | (474,144 | ) | 174,740 | 39,719 | ||||||||||||||
| Income taxes (expenses) |
9 | (4,843 | ) | (7,303 | ) | (40 | ) | (3 | ) | |||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Net profit/(loss) for the year |
(378,084 | ) | (481,447 | ) | 174,700 | 39,716 | ||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Attributable to owners of the Company |
(378,084 | ) | (481,447 | ) | 174,700 | 39,716 | ||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Basic and diluted earnings/(loss) per share |
(6.53 | ) | (8.55 | ) | — | — | ||||||||||||||
| Number of shares used for calculation (basic and diluted) (1) |
57,891,570 | 56,287,060 | — | — | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Statement of Comprehensive Income or (Loss) |
||||||||||||||||||||
| Net profit/(loss) for the year |
(378,084 | ) | (481,447 | ) | 174,700 | 39,716 | ||||||||||||||
| Other comprehensive income/(loss) |
||||||||||||||||||||
| Items that may be reclassified subsequently to profit or (loss): |
||||||||||||||||||||
| Exchange differences on translating foreign operations |
1,062 | (2,731 | ) | — | — | |||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Other comprehensive income/(loss) for the year, net of tax |
1,062 | (2,731 | ) | — | — | |||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Total comprehensive income/(loss) for the year, net of tax |
(377,022 | ) | (484,178 | ) | 174,700 | 39,716 | ||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Attributable to owners of the Company |
(377,022 | ) | (484,178 | ) | 174,700 | 39,716 | ||||||||||||||
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|
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|
|
|
|
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| (1) | As of December 31, 2024 and 2023 a total of 6,204,122 and 6,523,784 warrants outstanding, respectively, each carrying the right to subscribe for one ordinary share, and 575,000 convertible senior notes which can potentially be converted into 3,456,785 ordinary shares, can potentially dilute earnings per share in the future but have not been included in the calculation of diluted earnings per share because they are antidilutive for the years presented. |
| Financial Statements | 38 |
Statements of Financial Position as of December 31
| Group | ||||||||||||||||
| (EUR’000) | Notes | December 31, 2024 |
December 31, 2023 (Restated)(1) |
January 1, 2023 (Restated)(1) |
||||||||||||
| Assets |
||||||||||||||||
| Non-current assets |
||||||||||||||||
| Intangible assets |
5, 10 | 4,028 | 4,419 | 4,828 | ||||||||||||
| Property, plant and equipment |
5, 11 | 98,714 | 110,634 | 129,095 | ||||||||||||
| Investments in associates |
12 | 13,575 | 5,686 | 22,932 | ||||||||||||
| Other receivables |
16 | 2,317 | 2,127 | 1,920 | ||||||||||||
| Marketable securities |
16, 17 | — | — | 7,492 | ||||||||||||
|
|
|
|
|
|
|
|||||||||||
| 118,634 | 122,866 | 166,267 | ||||||||||||||
|
|
|
|
|
|
|
|||||||||||
| Current assets |
||||||||||||||||
| Inventories |
13 | 295,609 | 208,931 | 130,673 | ||||||||||||
| Trade receivables |
16 | 166,280 | 35,874 | 11,910 | ||||||||||||
| Income tax receivables |
1,775 | 802 | 883 | |||||||||||||
| Other receivables |
16 | 9,385 | 19,097 | 12,833 | ||||||||||||
| Prepayments |
28,269 | 38,578 | 31,717 | |||||||||||||
| Marketable securities |
16, 17 | — | 7,275 | 290,688 | ||||||||||||
| Cash and cash equivalents |
16, 17 | 559,543 | 392,164 | 444,767 | ||||||||||||
|
|
|
|
|
|
|
|||||||||||
| 1,060,861 | 702,721 | 923,471 | ||||||||||||||
|
|
|
|
|
|
|
|||||||||||
| Total assets |
1,179,495 | 825,587 | 1,089,738 | |||||||||||||
|
|
|
|
|
|
|
|||||||||||
| Equity and liabilities |
||||||||||||||||
| Equity |
||||||||||||||||
| Share capital |
17 | 8,149 | 7,749 | 7,675 | ||||||||||||
| Distributable equity |
(113,855 | ) | (153,446 | ) | 255,673 | |||||||||||
|
|
|
|
|
|
|
|||||||||||
| Total equity |
(105,706 | ) | (145,697 | ) | 263,348 | |||||||||||
|
|
|
|
|
|
|
|||||||||||
| Non-current liabilities |
||||||||||||||||
| Borrowings |
16, 17 | 365,080 | 222,996 | 95,400 | ||||||||||||
| Contract liabilities |
14 | 5,000 | 5,949 | 14,213 | ||||||||||||
| Deferred tax liabilities |
9 | 7,258 | 5,830 | — | ||||||||||||
|
|
|
|
|
|
|
|||||||||||
| 377,338 | 234,775 | 109,613 | ||||||||||||||
|
|
|
|
|
|
|
|||||||||||
| Current liabilities |
||||||||||||||||
| Convertible notes, matures in April 2028 |
||||||||||||||||
| Borrowings |
16, 17 | 458,207 | 407,095 | 399,186 | ||||||||||||
| Derivative liabilities |
16, 17 | 150,670 | 143,296 | 157,950 | ||||||||||||
|
|
|
|
|
|
|
|||||||||||
| 608,877 | 550,391 | 557,136 | ||||||||||||||
|
|
|
|
|
|
|
|||||||||||
| Other current liabilities |
||||||||||||||||
| Borrowings |
16, 17 | 33,329 | 14,174 | 13,791 | ||||||||||||
| Contract liabilities |
14 | 936 | 1,184 | — | ||||||||||||
| Trade payables and accrued expenses |
16, 17 | 96,394 | 94,566 | 101,032 | ||||||||||||
| Other liabilities |
67,956 | 41,176 | 31,989 | |||||||||||||
| Income tax payables |
1,222 | 2,299 | 5,490 | |||||||||||||
| Provisions |
15 | 99,149 | 32,719 | 7,339 | ||||||||||||
|
|
|
|
|
|
|
|||||||||||
| 298,986 | 186,118 | 159,641 | ||||||||||||||
|
|
|
|
|
|
|
|||||||||||
| 907,863 | 736,509 | 716,777 | ||||||||||||||
|
|
|
|
|
|
|
|||||||||||
| Total liabilities |
1,285,201 | 971,284 | 826,390 | |||||||||||||
|
|
|
|
|
|
|
|||||||||||
| Total equity and liabilities |
1,179,495 | 825,587 | 1,089,738 | |||||||||||||
|
|
|
|
|
|
|
|||||||||||
| (1) | Restatement relates to adoption of amendments to IAS 1 “Presentation of Financial Statements.” Refer to Note 2 for further details. |
| Financial Statements | 39 |
Statements of Financial Position as of December 31
| Parent | ||||||||||||||||
| (EUR’000) | Notes | December 31, 2024 |
December 31, 2023 (Restated)(1) |
January 1, 2023 (Restated)(1) |
||||||||||||
| Assets |
||||||||||||||||
| Non-current assets |
||||||||||||||||
| Intangible assets |
5, 10 | 444 | 889 | 1,333 | ||||||||||||
| Property, plant and equipment |
5, 11 | 23,889 | 28,414 | 25,344 | ||||||||||||
| Investments in group enterprises |
20 | 156,306 | 146,267 | 122,759 | ||||||||||||
| Receivables from group enterprises |
16 | 1,992,224 | 1,759,806 | 1,372,347 | ||||||||||||
| Other receivables |
16 | 1,429 | 1,425 | 1,303 | ||||||||||||
| Marketable securities |
16, 17 | — | — | 7,492 | ||||||||||||
|
|
|
|
|
|
|
|||||||||||
| 2,174,292 | 1,936,801 | 1,530,578 | ||||||||||||||
|
|
|
|
|
|
|
|||||||||||
| Current assets |
||||||||||||||||
| Inventories |
13 | 295,603 | 208,931 | 130,673 | ||||||||||||
| Trade receivables |
16 | 120,643 | — | 281 | ||||||||||||
| Income tax receivables |
738 | 739 | 740 | |||||||||||||
| Receivables from group enterprises |
16 | 28,204 | — | — | ||||||||||||
| Other receivables |
16 | 8,952 | 18,414 | 10,949 | ||||||||||||
| Prepayments |
24,552 | 35,916 | 27,261 | |||||||||||||
| Marketable securities |
16, 17 | — | 7,275 | 290,688 | ||||||||||||
| Cash and cash equivalents |
16 | 492,174 | 263,909 | 407,184 | ||||||||||||
|
|
|
|
|
|
|
|||||||||||
| 970,866 | 535,184 | 867,776 | ||||||||||||||
|
|
|
|
|
|
|
|||||||||||
| Total assets |
3,145,158 | 2,471,985 | 2,398,354 | |||||||||||||
|
|
|
|
|
|
|
|||||||||||
| Equity and liabilities |
||||||||||||||||
| Equity |
||||||||||||||||
| Share capital |
17 | 8,149 | 7,749 | 7,675 | ||||||||||||
| Distributable equity |
2,384,422 | 1,793,109 | 1,678,334 | |||||||||||||
|
|
|
|
|
|
|
|||||||||||
| Total equity |
2,392,571 | 1,800,858 | 1,686,009 | |||||||||||||
|
|
|
|
|
|
|
|||||||||||
| Non-current liabilities |
||||||||||||||||
| Borrowings |
16, 17 | 9,223 | 12,011 | 13,362 | ||||||||||||
|
|
|
|
|
|
|
|||||||||||
| 9,223 | 12,011 | 13,362 | ||||||||||||||
|
|
|
|
|
|
|
|||||||||||
| Current liabilities |
||||||||||||||||
| Convertible notes, matures in April 2028 |
||||||||||||||||
| Borrowings |
16, 17 | 458,207 | 407,095 | 399,186 | ||||||||||||
| Derivative liabilities |
16, 17 | 150,670 | 143,296 | 157,950 | ||||||||||||
|
|
|
|
|
|
|
|||||||||||
| 608,877 | 550,391 | 557,136 | ||||||||||||||
|
|
|
|
|
|
|
|||||||||||
| Other current liabilities |
||||||||||||||||
| Borrowings |
16, 17 | 3,173 | 3,176 | 2,950 | ||||||||||||
| Trade payables and accrued expenses |
16, 17 | 84,117 | 85,784 | 95,174 | ||||||||||||
| Payables to group enterprises |
16, 17 | 2,085 | — | 6,558 | ||||||||||||
| Other liabilities |
44,998 | 19,765 | 37,165 | |||||||||||||
| Provisions |
15 | 114 | — | — | ||||||||||||
|
|
|
|
|
|
|
|||||||||||
| 134,487 | 108,725 | 141,847 | ||||||||||||||
|
|
|
|
|
|
|
|||||||||||
| 743,364 | 659,116 | 698,983 | ||||||||||||||
|
|
|
|
|
|
|
|||||||||||
| Total liabilities |
752,587 | 671,127 | 712,345 | |||||||||||||
|
|
|
|
|
|
|
|||||||||||
| Total equity and liabilities |
3,145,158 | 2,471,985 | 2,398,354 | |||||||||||||
|
|
|
|
|
|
|
|||||||||||
| (1) | Restatement relates to adoption of amendments to IAS 1 “Presentation of Financial Statements.” Refer to Note 2 for further details. |
| Financial Statements | 40 |
Statements of Changes in Equity - Group
| Distributable Equity | ||||||||||||||||||||||||
| (EUR’000) | Share Capital |
Share Premium |
Treasury Shares |
Foreign Currency Translation Reserve |
Accumulated Deficit |
Total | ||||||||||||||||||
| Equity at January 1, 2023 |
7,675 | 2,112,863 | (149 | ) | 3,452 | (1,860,493 | ) | 263,348 | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Net profit / (loss) for the period |
— | — | — | — | (481,447 | ) | (481,447 | ) | ||||||||||||||||
| Other comprehensive income/(loss), net of tax |
— | — | — | (2,731 | ) | — | (2,731 | ) | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Total comprehensive income/(loss) |
— | — | — | (2,731 | ) | (481,447 | ) | (484,178 | ) | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Transactions with Owners |
||||||||||||||||||||||||
| Share-based payment (Note 7) |
— | — | — | — | 66,660 | 66,660 | ||||||||||||||||||
| Transfer under stock incentive programs |
— | — | 3 | — | (3 | ) | — | |||||||||||||||||
| Net settlement under stock incentive programs |
— | — | — | — | (1,812 | ) | (1,812 | ) | ||||||||||||||||
| Capital increase |
74 | 10,211 | — | — | — | 10,285 | ||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Equity at December 31, 2023 |
7,749 | 2,123,074 | (146 | ) | 721 | (2,277,095 | ) | (145,697 | ) | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Net profit / (loss) for the period |
— | — | — | — | (378,084 | ) | (378,084 | ) | ||||||||||||||||
| Other comprehensive income/(loss), net of tax |
— | — | — | 1,062 | — | 1,062 | ||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Total comprehensive income/(loss) |
— | — | — | 1,062 | (378,084 | ) | (377,022 | ) | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Transactions with Owners |
||||||||||||||||||||||||
| Share-based payment (Note 7) |
— | — | — | — | 95,512 | 95,512 | ||||||||||||||||||
| Transfer under stock incentive programs |
— | — | 33 | — | (33 | ) | — | |||||||||||||||||
| Capital Increase (Note 17) |
400 | 340,392 | — | — | — | 340,792 | ||||||||||||||||||
| Cost of capital increase |
— | (19,291 | ) | — | — | — | (19,291 | ) | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Equity at December 31, 2024 |
8,149 | 2,444,175 | (113 | ) | 1,783 | (2,559,700 | ) | (105,706 | ) | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Financial Statements | 41 |
Statements of Changes in Equity - Parent
| Distributable Equity | ||||||||||||||||||||||||
| (EUR’000) | Share Capital |
Share Premium |
Treasury Shares |
Foreign Currency Translation Reserve |
Accumulated Deficit |
Total | ||||||||||||||||||
| Equity at January 1, 2023 |
7,675 | 2,112,863 | (149 | ) | (53 | ) | (434,327 | ) | 1,686,009 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Net profit /(loss) for the period |
— | — | — | — | 39,716 | 39,716 | ||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Total comprehensive income/(loss) |
— | — | — | — | 39,716 | 39,716 | ||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Transactions with Owners |
||||||||||||||||||||||||
| Share-based payment (Note 7) |
— | — | — | — | 66,660 | 66,660 | ||||||||||||||||||
| Transfer under stock incentive programs |
— | — | 3 | — | (3 | ) | — | |||||||||||||||||
| Net settlement under stock incentive programs |
— | — | — | — | (1,812 | ) | (1,812 | ) | ||||||||||||||||
| Capital increase |
74 | 10,211 | — | — | — | 10,285 | ||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Equity at December 31, 2023 |
7,749 | 2,123,074 | (146 | ) | (53 | ) | (329,766 | ) | 1,800,858 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Net profit /(loss) for the period |
— | — | — | — | 174,700 | 174,700 | ||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Total comprehensive income/(loss) |
— | — | — | — | 174,700 | 174,700 | ||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Transactions with Owners |
||||||||||||||||||||||||
| Share-based payment (Note 7) |
— | — | — | — | 95,512 | 95,512 | ||||||||||||||||||
| Acquisition of treasury shares |
— | — | 33 | — | (33 | ) | — | |||||||||||||||||
| Capital Increase (Note 17) |
400 | 340,392 | — | — | — | 340,792 | ||||||||||||||||||
| Cost of capital increase |
— | (19,291 | ) | — | — | — | (19,291 | ) | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Equity at December 31, 2024 |
8,149 | 2,444,175 | (113 | ) | (53 | ) | (59,587 | ) | 2,392,571 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Financial Statements | 42 |
Cash Flow Statements for the Years Ended December
| Notes | Group | Parent | ||||||||||||||||||
| (EUR’000) | 2024 | 2023 | 2024 | 2023 | ||||||||||||||||
| Operating activities |
||||||||||||||||||||
| Net profit/(loss) for the year |
(378,084 | ) | (481,447 | ) | 174,700 | 39,716 | ||||||||||||||
| Reversal of finance income |
(25,609 | ) | (43,857 | ) | (94,173 | ) | (77,624 | ) | ||||||||||||
| Reversal of finance expenses |
100,027 | 44,065 | 45,567 | 34,714 | ||||||||||||||||
| Reversal of (gain)/loss on disposal of property, plant and equipment |
(91 | ) | 5 | — | — | |||||||||||||||
| Reversal of income taxes (expenses) |
4,843 | 7,303 | 40 | 3 | ||||||||||||||||
| Adjustments for non-cash items: |
||||||||||||||||||||
| Non-cash consideration relating to revenue |
(27,069 | ) | (2,354 | ) | — | — | ||||||||||||||
| Share of (profit)/loss of associates |
20,060 | 18,395 | — | — | ||||||||||||||||
| Share-based payment |
95,512 | 66,660 | 61,038 | 43,259 | ||||||||||||||||
| Depreciation |
17,247 | 18,428 | 4,918 | 4,267 | ||||||||||||||||
| Impairment of property, plant and equipment |
— | 7,834 | — | — | ||||||||||||||||
| Amortization |
467 | 483 | 445 | 444 | ||||||||||||||||
| Changes in working capital: |
||||||||||||||||||||
| Inventories |
(86,678 | ) | (78,258 | ) | (86,678 | ) | (78,258 | ) | ||||||||||||
| Receivables |
(118,607 | ) | (32,773 | ) | (111,199 | ) | (8,130 | ) | ||||||||||||
| Receivables from group enterprises |
— | — | (145,433 | ) | (350,885 | ) | ||||||||||||||
| Prepayments |
10,392 | (11,413 | ) | 11,363 | (13,158 | ) | ||||||||||||||
| Contract liabilities |
(1,197 | ) | (7,080 | ) | — | — | ||||||||||||||
| Trade payables, accrued expenses and other liabilities |
26,965 | 3,551 | 23,563 | (26,791 | ) | |||||||||||||||
| Payables to group enterprises |
— | — | 2,072 | (6,558 | ) | |||||||||||||||
| Increase/(decrease) in provisions |
61,968 | 26,187 | 114 | — | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Cash flows generated from/(used in) operations |
(299,854 | ) | (464,271 | ) | (113,663 | ) | (439,001 | ) | ||||||||||||
| Finance income received |
14,374 | 17,048 | 11,825 | 15,283 | ||||||||||||||||
| Finance expenses paid |
(15,205 | ) | (15,672 | ) | (12,196 | ) | (12,489 | ) | ||||||||||||
| Income taxes received/(paid) |
(5,512 | ) | (4,466 | ) | 738 | 738 | ||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Cash flows from/(used in) operating activities |
(306,197 | ) | (467,361 | ) | (113,295 | ) | (435,469 | ) | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Investing activities |
||||||||||||||||||||
| Investment in group enterprises |
— | — | (67 | ) | (107 | ) | ||||||||||||||
| Proceeds from disposal of property, plant and equipment |
950 | 51 | — | — | ||||||||||||||||
| Acquisition of intangible assets and property, plant and equipment |
(1,427 | ) | (2,442 | ) | (394 | ) | (1,230 | ) | ||||||||||||
| Settlement of marketable securities |
7,353 | 288,865 | 7,353 | 288,865 | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Cash flows from/(used in) investing activities |
6,876 | 286,474 | 6,892 | 287,528 | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Financing activities |
||||||||||||||||||||
| Repayment of borrowings |
(11,365 | ) | (10,438 | ) | (2,834 | ) | (2,714 | ) | ||||||||||||
| Net proceeds from borrowings |
16 | 134,158 | 136,256 | — | — | |||||||||||||||
| Proceeds from exercise of warrants |
30,514 | 10,286 | 30,514 | 10,286 | ||||||||||||||||
| Proceeds from follow-on public offering |
309,913 | — | 309,913 | — | ||||||||||||||||
| Costs of follow-on public offering |
(19,291 | ) | — | (19,291 | ) | — | ||||||||||||||
| Payment of withholding taxes under stock incentive programs |
— | (1,812 | ) | — | (1,812 | ) | ||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Cash flows from/(used in) financing activities |
443,929 | 134,292 | 318,302 | 5,760 | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Increase/(decrease) in cash and cash equivalents |
144,608 | (46,595 | ) | 211,899 | (142,181 | ) | ||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Cash and cash equivalents at January 1 |
392,164 | 444,767 | 263,909 | 407,184 | ||||||||||||||||
| Effect of exchange rate changes on balances held in foreign currencies |
22,771 | (6,008 | ) | 16,366 | (1,094 | ) | ||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Cash and cash equivalents at December 31 |
559,543 | 392,164 | 492,174 | 263,909 | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Cash and cash equivalents include |
||||||||||||||||||||
| Bank deposits |
559,543 | 392,164 | 492,174 | 263,909 | ||||||||||||||||
| Cash and cash equivalents at December 31 |
||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| 559,543 | 392,164 | 492,174 | 263,909 | |||||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Financial Statements | 43 |
Notes to the Financial Statements
Note 1 – General Information
Ascendis Pharma A/S, together with its subsidiaries, is a global biopharmaceutical company focused on applying its innovative TransCon technology platform to make a meaningful difference for patients.. Ascendis Pharma A/S was incorporated in 2006 and is headquartered in Hellerup, Denmark. Unless the context otherwise requires, references to the “Company,” “we,” “us,” and “our,” refer to Ascendis Pharma A/S and its subsidiaries.
The address of the Company’s registered office is Tuborg Boulevard 12, DK-2900 Hellerup, Denmark. The Company’s registration number in Denmark is 29918791.
On February 2, 2015, the Company completed an initial public offering, which resulted in the listing of American Depositary Shares (“ADSs”), representing the Company’s ordinary shares, under the symbol “ASND” in the United States on The Nasdaq Global Select Market.
The Company’s Board of Directors (or “Board”) approved these financial statements on February 12, 2025. The financial statements can be obtained from https://datacvr.virk.dk/
Note 2 – Summary of Material Accounting Policies
Basis of Preparation
The financial statements, which include the consolidated financial statements and the parent financial statements of Ascendis Pharma A/S, are prepared in accordance with the IFRS Accounting Standards (“IFRS”), as issued by the International Accounting Standards Board (“IASB”), and as adopted by the European Union (“EU”). The financial statements include additional disclosures for reporting class C large sized enterprises as required by the Danish Executive Order on Adoption of IFRS as issued in accordance with the Danish Financial Statements Act.
The accounting policies applied when preparing the financial statements are described in detail below and are applied for all group entities. Significant accounting judgements and sources of estimation uncertainties used when exercising the accounting policies are described in Note 3, “Significant Accounting Judgements and Estimates.”
These financial statements have been prepared under the historical cost convention, apart from certain financial instruments that are measured at fair value at initial recognition.
New and Amended IFRS Accounting Standards and Interpretations
The Company has applied amendments to paragraphs 69 to 76 of IAS 1, “Presentation of Financial Statements,” which was effective for annual reporting periods beginning on or after January 1, 2024 and must be applied retrospectively. The amendments to IAS 1 specify the requirements for classifying liabilities as current or non-current. The amendments clarify:
| • | What is meant by a right to defer settlement; |
| • | That a right to defer must exist at the end of the reporting period; |
| • | That classification is unaffected by the likelihood that an entity will exercise its deferral right; and |
| • | That only if an embedded derivative in a convertible liability is itself an equity instrument would the terms of a liability not impact its classification. |
| Financial Statements | 44 |
The convertible senior notes (“convertible notes”) include an embedded equity conversion option which is not deemed closely related to the financial liability and was initially recognized and measured separately at fair value as derivative liabilities based on the stated terms upon issuance of the convertible notes. The conversion option is classified as a foreign currency conversion option and thus not convertible into a fixed number of shares for a fixed amount of cash. Accordingly, the conversion option is subsequently recognized and measured as a derivative liability at fair value through profit or loss, with any subsequent remeasurement gains or losses recognized as part of finance income or expenses.
Since the embedded derivative is not an equity instrument under IFRS, the amendments require the convertible notes (presented as part of borrowings in the statement of financial position) and derivative liabilities, presented as non-current liabilities as of December 31, 2023, to be presented as current liabilities. The amendments require presentation of the convertible notes as current liabilities even though: the initial conversion price of $166.34 per ADS is not met; the conversion would not require cash settlement; and, the convertible notes do not mature until April 1, 2028. Further details, including (cash) maturity analysis are provided in Note 16, “Financial Assets and Liabilities.” On December 31, 2023, the carrying amount of convertible notes and derivative liabilities were €407.1 million and €143.3 million, respectively. Comparative amounts have been reclassified to reflect the change to presentation, including a third statement of financial position as of the beginning of the preceding period i.e. January 1, 2023.
On December 31, 2023, lease liabilities were presented separately in the consolidated statements of financial position. As of December 31, 2023, carrying amount of lease liabilities was €84.6 million and €14.2 million, for non-current liabilities and current liabilities, respectively. Since March 31, 2024, lease liabilities are presented as part of borrowings in the consolidated statements of financial position. Comparative amounts have been reclassified to reflect the change in presentation.
Accordingly, since March 31, 2024, borrowings comprise convertible notes, royalty funding liabilities, and lease liabilities, where convertible notes are presented separately as “Current liabilities—Convertible notes, matures in 2028.” The change to presentation had no other impact on the consolidated financial statements. Refer to Note 16, “Financial Assets and Liabilities” for a specification of borrowings.
The applied amendments had no other impact on the consolidated financial statements.
Other IFRS Accounting Standards and Interpretations
Other amendments apply for the first time in 2024, but do not have an impact on the financial statements.
Going Concern
The Company’s Board of Directors has, at the time of approving the financial statements, a reasonable expectation that the Company has adequate resources to continue in operational existence for the foreseeable future. Thus, the Company continues to adopt the going concern basis of accounting in preparing the financial statements.
Basis of Consolidation
The consolidated financial statements include the parent company, Ascendis Pharma A/S, and all enterprises over which the parent company has control. Control of an enterprise exists when the Company has exposure, or rights to, variable returns from its involvement with the enterprise and has the ability to control those returns through its power over the enterprise. Accordingly, the consolidated financial statements include Ascendis Pharma A/S and the subsidiaries listed in Note 20, “Investments in Group Enterprises”.
Consolidation Principles
Subsidiaries, which are enterprises the Company controls, are fully consolidated from the date upon which control is transferred to the Company. They are deconsolidated from the date control ceases.
| Financial Statements | 45 |
Control over an enterprise is reassessed if facts and circumstances indicate that there are changes to one or more of the three elements of control, respectively:
| • | the contractual arrangement(s) with the other vote holders of the enterprise; |
| • | the Company’s voting rights and potential voting rights; and |
| • | rights arising from other contractual arrangements. |
All intra-group assets and liabilities, equity, income, expenses and cash flows relating to transactions between group enterprises are eliminated in full on consolidation.
Subsidiaries apply accounting policies in line with the Company’s accounting policies. When necessary, adjustments are made to bring the entities’ accounting policies in line with those of the Company.
Investments in Associates
An associate is an entity over which the Company has significant influence over financial and operational decisions but without having control or joint control. The Company’s associates are accounted for using the equity method and initially recognized at cost. Thereafter, the carrying amount of the investment is adjusted to recognize changes in the Company’s share of net assets of the associates since the acquisition or establishment date.
The consolidated statements of profit or loss include the Company’s share of result after tax of the associates after any adjustments made to bring the associates accounting policies in line with those of the Company. Transactions between the associates and the Company are eliminated proportionally according to the Company’s interest in the associates. Unrealized gains and losses resulting from transactions between the Company and its associates are eliminated to the extent of the Company’s interest in the associates.
On each reporting date, the Company determines whether there are indications that the investment is impaired. If there is such evidence, the amount of impairment is calculated as the difference between the recoverable amount of the associate and its carrying amount. Any impairment loss is recognized in the consolidated statements of profit or loss.
Foreign Currency
Functional and Presentation Currency
Items included in the consolidated financial statements are measured using the functional currency of each group entity. Functional currency is the currency of the primary economic environment in which the entity operates. The financial statements are presented in Euros (or “EUR”), which is also the functional currency of the parent company.
Translation of Transactions and Balances
On initial recognition, transactions in currencies other than the individual entity’s functional currency are translated applying the exchange rate in effect at the date of the transaction. Receivables, payables and other monetary items denominated in foreign currencies that have not been settled at the reporting date are translated using the exchange rate in effect at the reporting date. Monetary items carried at fair value that are denominated in foreign currencies are translated at the rates prevailing at the date when the fair value was determined.
Exchange rate differences that arise between the rate at the transaction date and the rate in effect at the payment date, or the rate at the reporting date, are recognized in profit or loss as finance income or finance expenses. Property, plant and equipment, intangible assets and other non-monetary items that are measured in terms of historical cost in a foreign currency are translated using the exchange rates as of the dates of the initial transactions.
| Financial Statements | 46 |
Currency Translation of Group Enterprises
When subsidiaries or associates present their financial statements in a functional currency other than EUR, their statements of profit or loss are translated at average exchange rates. Balance sheet items are translated using the exchange rates at the reporting date. Exchange rate differences arising from translation of foreign entities’ balance sheet items at the beginning of the year to the reporting date exchange rates as well as from translation of statements of profit or loss from average rates to the exchange rates at the reporting date are recognized in other comprehensive income. Similarly, exchange rate differences arising from changes that have been made directly in a foreign subsidiary’s equity are recognized in other comprehensive income.
Revenue
Revenue from Commercial Products
Revenue is recognized when the customer has obtained control of the goods and it is probable that the Company will collect the consideration to which it is entitled for transferring the goods. Control is transferred upon delivery.
Revenue is measured at the contractual sales price, reflecting the consideration received or receivable from customers, net of value added taxes, and provisions for a variety of sales deductions such as prompt pay discounts, shelf stock adjustments and applicable sales deductions attributable to various commercial arrangements, managed healthcare organizations, government programs and co-pay arrangements. In addition, goods are principally sold on a “sale-or-return” basis, where customers may return products in line with the Company’s return policy. Sales deductions and product returns are considered variable consideration and are estimated at the time of sale using the expected value method. The amount of variable consideration that is included in the transaction price may be constrained and is included in the net contractual price only to the extent that it is probable that a significant reversal will not occur.
Unsettled sales deductions and product returns are recognized as provisions when timing or amount is uncertain. Payable amounts that are absolute are recognized as other liabilities. Sales discounts and deductions that are payable to customers are offset in trade receivables.
Other Revenue
Other revenue relates to collaboration and license agreements. When contracts with customers are entered into, the goods and/or services promised in the contract are assessed to identify distinct performance obligations. A promise in the agreement is considered a distinct performance obligation if both of the following criteria are met:
| • | the customer can benefit from the good or service either on its own or together with other resources that are readily available to the customer (i.e., the good or service is capable of being distinct); and |
| • | the entity’s promise to transfer the good or service to the customer is separately identifiable from other promises in the contract (i.e., the promise to transfer the good or service is distinct within the context of the contract). |
For collaboration and license agreements that contain multiple promises to the customer, the promises are identified and accounted for as separate performance obligations if these are distinct. If promises are not distinct, those goods or services are combined with other promised goods or services until a bundle of goods or services that is distinct is identified.
The transaction price in the contract is measured at fair value and reflects the consideration the Company expects to be entitled to in exchange for those goods or services. Under license agreements, the transaction price may include up-front payments, royalty and milestone payments.
Sales-based royalty and sales-based milestone income promised in exchange for a license of intellectual property is recognized as revenue at the later of the occurrence of subsequent sale or satisfaction of the performance obligation to which some or all of the royalty or milestone has been allocated. Milestone income related to regulatory activities is included in the transaction price at the point in time that it is highly probable that the applicable milestone criteria is met.
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The transaction price is allocated to each performance obligation according to their stand-alone selling
prices and is recognized when control of the goods or services is transferred to the customer, either over time or at a point in time, depending on the specific terms and conditions in the contracts. License agreements, which transfer rights to the
Company’s intellectual property (“IP”), are classified as “right-to-access”, with revenue recognized over time, or as “right-to-use” with revenue recognized at a point in time.
Sale of clinical trial supply is recognized as revenue when the customer has obtained control of the goods and it is probable that the Company will collect the consideration to which it is entitled for transferring the goods. Control is transferred upon delivery. Rendering of services is recognized as revenue over the service period as stipulated under the applicable agreement.
Research and Development Costs
Research and development costs consist primarily of manufacturing costs, preclinical and clinical study costs and costs for process optimizations and improvements performed by Clinical Research Organizations (“CROs”) and Contract Manufacturing Organizations (“CMOs”), salaries and other personnel costs including pension and share-based payment, the cost of facilities, professional fees, cost of obtaining and maintaining the Company’s intellectual property portfolio, and depreciation of non-current assets related to research and development activities.
Research costs are incurred at the early stages of the drug development cycle from the initial drug discovery and include a variety of preclinical research activities in order to assess potential drug candidates in non-human subjects, prior to filing an Investigational New Drug Application (“IND”), or equivalent. Research costs are recognized in the statement of profit or loss when incurred.
Development activities relate to activities following an IND, or equivalent, and typically involve a single product candidate undergoing a series of studies to illustrate its safety profile and effect on human beings, prior to obtaining the necessary approval from the appropriate authorities. Development activities comprise drug candidates undergoing clinical trials starting in phase I (first time drug is administered in a small group of humans), and further into Phase II and III, which include administration of drugs in larger patient groups. Following, and depending on clinical trial results, a Biologic License Application (“BLA”) or New Drug Application (“NDA”) may be submitted to the authorities, to apply for marketing approval, which, with a positive outcome will permit the Company to market and sell the products. Long-term extension trials may be ongoing following submission of a BLA or NDA.
Development costs also include product development and pre-commercial manufacturing costs related to development product candidates, and write-downs of inventories manufactured for late-stage development product candidates prior to marketing approval being obtained (pre-launch inventories).
Due to the risk related to the development of pharmaceutical products, the Company cannot estimate the future economic benefits associated with individual development activities with sufficient certainty until the development activities have been finalized and the necessary market approval of the final product has been obtained. As a consequence, all development costs are recognized in the statement of profit or loss when incurred.
Selling, General, and Administrative Expenses
Selling, general, and administrative expenses comprise salaries and other personnel costs including pension and share-based payment, office supplies, cost of facilities, professional fees, and depreciation of non-current assets related to selling general and administrative activities, including pre-commercial and commercial activities. Selling, general and administrative expenses are recognized in the statement of profit or loss when incurred.
Share-based Incentive Programs
Share-based incentive programs comprise warrant programs, Restricted Stock Unit programs (“RSU-programs”) and Performance Stock Unit Programs (“PSU-programs”) which are classified as equity-settled share-based payment transactions.
| Financial Statements | 48 |
The cost of equity-settled transactions is determined by the fair value at the date of grant. For warrant programs, the fair value of each warrant granted is determined using the Black-Scholes option pricing model. For RSU-programs and PSU-programs, the fair value of each RSU or PSU granted is equal to the closing share price on the date of grant of the underlying ADS. Any social security contributions payable in connection with the grant or exercise of the warrants are recognized as expenses when incurred. The assumptions used for estimating the fair value of share-based payment transactions are disclosed in Note 7, “Share-based Payment.”
The cost is recognized together with a corresponding increase in equity over the period in which the performance and/or service conditions are fulfilled (i.e., the vesting period). The fair value determined at the grant date of the equity-settled share-based payment is expensed on a straight-line basis over the vesting period for each tranche, based on the best estimate of the number of equity instruments that will ultimately vest. No expense is recognized for grants that do not ultimately vest.
Where an equity-settled grant is cancelled other than upon forfeiture when vesting conditions are not satisfied, the grant is treated as if it vested on the date of the cancellation, and any expense not yet recognized for the grant is recognized immediately.
Where the terms and conditions for an equity-settled grant are modified, the services measured at the grant date fair value over the vesting period are recognized, subject to performance and/or service conditions that were specified at the initial grant date(s). Additionally, at the date of modification, unvested grants are re-measured and any increase in the total fair value is recognized over the vesting period. If a new grant is substituted for the cancelled grant and designated as a replacement grant on the date that it is granted, the cancelled and new grants are treated as if they were a modification of the original grant.
The Parent Company, together with its subsidiaries have entered into group share-based payment arrangements. The Parent Company incurs share-based payment transactions, whereas subsidiaries receive the services, and the Parent Company incur an obligation to settle the transaction with the subsidiaries. While the obligations are settled in the Parent Company’s own equity instruments, group share-based payments are in the Parent Company’s separate financial statements recognized as cost of investment in subsidiaries with a corresponding increase in equity over the vesting period. Where applicable, subsidiaries settles share-based payment transactions with the Parent Company, which are off-set to investments in subsidiaries.
Finance Income and Expenses
Finance income and expenses comprise interest income and expenses, realized and unrealized exchange rate gains and losses on transactions denominated in foreign currencies, fair value remeasurement gains and losses on derivative liabilities, and remeasurement gains and losses on royalty funding liabilities.
Interest income and interest expenses are stated on an accrual basis using the principal and the effective interest rate. The effective interest rate is the discount rate that is used to discount expected future cash payments or receipts through the expected life of the financial asset or financial liability to the amortized cost (the carrying amount) of such asset or liability.
Income Taxes
Tax for the year, which consists of current tax for the year and changes in deferred tax, is recognized in the statement of profit or loss by the portion attributable to the profit or loss for the year and recognized directly in equity or other comprehensive income by the portion attributable to entries directly in equity and in other comprehensive income. The current tax payable or receivable is recognized in the statement of financial position, stated as tax computed on this year’s taxable income, adjusted for prepaid tax.
When computing the current tax for the year, the tax rates and tax rules enacted or substantially enacted at the reporting date are used. Current tax payable is based on taxable profit or loss for the year. Taxable profit or loss differs from net profit or loss as reported in the statements of profit or loss because it excludes items of income or expense that are taxable or deductible in prior or future years. In addition, taxable profit or loss excludes items that are never taxable or deductible.
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Deferred tax is recognized according to the balance sheet liability method of all temporary differences between carrying amounts and tax-based values of assets and liabilities, apart from deferred tax on all temporary differences occurring on initial recognition of goodwill or on initial recognition of a transaction which is not a business combination, and for which the temporary difference found at the time of initial recognition neither affects profit or loss nor taxable income.
Deferred tax liabilities are recognized on all temporary differences related to investments in subsidiaries and/or associates, unless the Company is able to control when the deferred tax is realized, and it is probable that the deferred tax will not become due and payable as current tax in the foreseeable future.
Deferred tax assets, including the tax base of tax loss carry forwards, are recognized in the statement of financial position at their estimated realizable value, either as a set-off against deferred tax liabilities or as net tax assets for offset against future positive taxable income. Deferred tax assets are only offset against deferred tax liabilities if the entity has a legally enforceable right to offset, and the deferred tax assets and deferred tax liabilities relate to income taxes levied by the same tax jurisdiction. Deferred tax is calculated based on the planned use of each asset and the settlement of each liability, respectively.
Deferred tax is measured using the tax rates and tax rules in the relevant countries that, based on acts in force or acts in reality in force at the reporting date are expected to apply when the deferred tax is expected to crystallize as current tax. Changes in deferred tax resulting from changed tax rates or tax rules are recognized in the statement of profit or loss unless the deferred tax is attributable to transactions previously recognized directly in equity or other comprehensive income. In the latter case, such changes are also recognized in equity or other comprehensive income. On every reporting date, it is assessed whether sufficient taxable income is likely to arise in the future for the deferred tax asset to be utilized.
Intangible assets
Goodwill
Goodwill acquired in a business combination is initially measured at cost, being the excess of the aggregate of the consideration transferred and the amount recognized for non-controlling interests over the net identifiable assets acquired and liabilities assumed.
After initial recognition, goodwill is measured at cost less any accumulated impairment losses. Goodwill is not amortized but is subject to impairment testing at least on a yearly basis. For the purpose of impairment testing, goodwill acquired in a business combination is allocated to each of the cash-generating units, or group of cash-generating units, that are expected to benefit from the synergies of the combination. Each cash-generating unit or group of cash-generating units to which goodwill is allocated represents the lowest level within the Company at which the goodwill is monitored for internal management purposes.
Software
Software assets comprise administrative applications and serve general purposes to support the Company’s operations.
Development costs that are directly attributable to the design, customization, implementation, and testing of identifiable and unique software assets controlled by the Company are recognized as intangible assets from the time that; (1) the software asset is clearly defined and identifiable; (2) technological feasibility, adequate resources to complete, and an internal use of the software asset can be demonstrated; (3) the expenditure attributable to the software asset can be measured reliably; and (4) the Company has the intention to use the software asset internally. The Company does not capitalize software with no alternative use, or where economic benefit depends on marketing approvals of drug candidates and where marketing approvals have not been obtained.
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Following initial recognition of the development expenditure as an asset, the asset is carried at cost less any accumulated amortization and accumulated impairment losses. Amortization of the asset begins when the development is complete, and the asset is available for use.
Software assets are amortized over the period of expected future benefits. Amortization is recognized in research and development costs, and selling, general and administrative expenses, as appropriate. Expenditures that do not meet the criteria above are recognized as an expense as incurred.
Other Intangible Assets
Intangible assets comprise acquired intellectual property rights in the form of patents and licenses, which are measured at cost less accumulated amortization and accumulated impairment losses. Cost comprises the acquisition price and costs directly attributable to the acquisition of the asset. The amortization period is determined based on the expected economic and technical useful life of the asset, and amortization is recognized on a straight-line basis over the expected useful life of 5-10 years depending on the planned use of the specific asset and the lifetime of the patents protecting the intellectual property rights. Subsequent costs to maintain the intangible assets are recognized as expenses in the period to which they relate.
Property, Plant and Equipment
Property, plant and equipment primarily comprises leasehold improvements, office facilities, and process equipment and tools which are located at CMOs. Property, plant and equipment also includes right-of-use assets. Refer to the separate section “Leases.”
Property, plant and equipment is measured at cost less accumulated depreciation and impairment losses. Cost comprises the acquisition price, costs directly attributable to the acquisition and preparation costs of the asset until the time when it is ready to be used in operation. Subsequent costs are included in the carrying amount of the asset or recognized as a separate asset, as appropriate, only when it is probable that future economic benefits associated with the assets will flow to the Company and costs of the items can be measured reliably. All repair and maintenance costs are charged to the statement of profit or loss during the financial periods in which they are incurred.
Plant and equipment acquired for research and development activities with alternative use, which is expected to be used for more than one year, is capitalized and depreciated over the estimated useful life as research and development costs. Plant and equipment acquired for research and development activities, which has no alternative use, is recognized as research and development costs when incurred.
If the acquisition or use of the asset involves an obligation to incur costs of decommissioning or restoration of the asset, the estimated related costs are recognized as a provision and as part of the relevant asset’s cost, respectively.
The basis for depreciation is cost less estimated residual value. The residual value is the estimated amount that would be earned if selling the asset today net of selling costs, assuming that the asset is of an age and a condition that is expected after the end of its useful life.
Cost of a combined asset is divided into smaller components, with such significant components depreciated individually if their useful lives vary. Depreciation commences when the asset is available for use, which is when it is in the location and condition necessary for it to be capable of operating in the manner intended.
Depreciation is calculated on a straight-line basis, based on an asset’s expected useful life, being within the following ranges:
| Process plant and machinery |
5-10 years | |||
| Other equipment |
3- 5 years | |||
| Leasehold improvements |
3-11 years | |||
| Right-of-use assets |
2-11 years |
Depreciation methods, useful lives and residual amounts are reassessed at least annually.
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Property, plant and equipment is written down to the lower of recoverable amount and carrying amount, as described in the “Impairment” section below. Depreciation and impairment losses of property, plant and equipment is recognized in the statement of profit or loss as cost of sales, research and development costs or as selling, general, and administrative expenses, as appropriate.
Gains and losses on disposal of property, plant and equipment are recognized in the consolidated statement of profit or loss at its net proceeds, as either research and development costs or as selling, general, and administrative expenses, as appropriate.
Investments in Group Enterprises – Parent Company
Investments in group enterprises are recognized and measured at cost. Investments that are measured in terms of historical cost in a foreign currency are translated using the exchange rates as of the dates of the initial transactions.
Investments are written down to the lower of recoverable amount and carrying amount which is further described below in the section “Impairment.”
Impairment of Non-current Assets
The recoverable amount of goodwill is estimated annually irrespective of any recorded indications of impairment. Property, plant and equipment and finite-lived intangible assets are reviewed for impairment whenever events or circumstances indicate that the carrying amount may not be recoverable.
An impairment loss is recognized for the amount by which the asset’s carrying amount exceeds its recoverable amount. The recoverable amount is the higher of an asset’s fair value less costs of disposal and value in use.
For the purpose of assessing impairment, assets are grouped at the lowest levels for which there are largely independent cash inflows, or cash-generating units, which for goodwill represent the lowest level within the enterprise at which the goodwill is monitored for internal management purposes. Prior impairments of non-current assets, other than goodwill, are reviewed for possible reversal at each reporting date.
Inventories
Inventories comprise raw materials, work in progress and finished goods. The cost of work in progress and finished goods comprise service expenses incurred at CMOs, raw materials consumed, incremental storage and transportation, other direct materials, and a proportion of manufacturing overheads based on normal operation capacity.
Inventories are measured at the lower of cost incurred in bringing it to its present location and condition, and net realizable value. Net realizable value is the estimated selling price in the ordinary course of business, less estimated costs of completion and the estimated costs necessary to make the sale. Cost is measured using the first-in, first-out method.
Work in progress and finished goods are measured under a standard cost method that takes into account normal levels of consumption, yields, labor, efficiency and capacity utilization. Production processes are complex, where actual yields and consumptions are sensitive to a wide variety of manufacturing conditions. Standard cost variances are reviewed regularly and adjusted to ensure inventories approximate actual cost of production.
If net realizable value is lower than cost, a write-down is recognized as the excess amount by which cost exceeds net realizable value, as part of cost of sales, or selling, general, and administrative expenses, as appropriate. The amount of reversal of write-down of inventories arising from an increase in net realizable value is recognized as a reduction in cost of sales in the period in which the reversal occurs.
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Manufacturing of pre-launch inventories is initiated for late-stage product candidates where manufacturing costs are recognized as inventories. However, since pre-launch inventories are not realizable prior to obtaining marketing approval, pre-launch inventories are immediately written down to zero through research and development costs. If marketing approval is obtained, prior write-downs of pre-launch inventories are reversed through research and development costs.
Cost of inventories is recognized as part of cost of sales in the period in which the related revenue is recognized.
Receivables
Receivables comprise trade receivables, income tax receivables and other receivables.
Trade receivables are classified as financial assets at amortized cost, as these are held to collect contractual cash flows and thus give rise to cash flows representing solely payments of principal and interest. Trade receivables are initially recognized at their transaction price and subsequently measured at amortized cost. Income tax receivables and other receivables related to deposits, VAT and other indirect taxes are measured at cost less impairment. Carrying amounts of receivables usually equals their nominal value less provision for impairments.
Prepayments
Prepayments comprise advance payments relating to a future financial year. Prepayments are measured at cost.
Marketable Securities
Marketable securities may comprise government bonds, treasury bills, commercial papers, and other securities traded on established markets.
At initial recognition (trade-date), contractual terms of individual securities are analyzed to determine whether these give rise on specified dates to cash flows that are solely payments of principal and interest on the principal outstanding (“SPPI-test”). All marketable securities held at the reporting date have passed the SPPI-test.
Marketable securities are initially recognized at fair value at trade-date, and subsequently measured at amortized cost under the effective interest method. Interest income is recognized as finance income in the statement of profit or loss. Marketable securities are subject to an impairment test to accommodate expected credit loss. Gains and losses are recognized as finance income or expenses in the statement of profit or loss when the specific security or portfolio of securities is derecognized, modified or impaired.
Marketable securities, having maturity profiles of three months or less after the date of acquisition are presented as cash equivalents in the statements of financial position, where securities having maturities of more than three months after the date of acquisition are presented separately as marketable securities as current (i.e., those maturing within twelve months after the reporting date) or non-current assets, as appropriate.
Cash and Cash Equivalents
Cash and cash equivalents comprise cash and on-demand deposits with financial institutions, and highly liquid marketable securities with a maturity of three months or less after the date of acquisition (trade-date). Cash and cash equivalents are measured at amortized cost.
Allowance for Expected Credit Losses on Financial Assets
Financial assets comprise receivables (excluding receivables relating to VAT, other indirect tax and income tax), marketable securities, and cash and cash equivalents. Impairment of financial assets is determined on the basis of a forward-looking Expected Credit Loss (“ECL”) model. ECLs are based on the difference between the contractual cash flows due in accordance with the contract and the cash flows expected to be received, discounted by an approximation of the original effective interest rate.
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For receivables, a simplified approach in calculating ECLs is applied. Therefore, changes in credit risks are not tracked, but instead, a loss allowance based on lifetime ECL is assessed at each reporting date. Lifetime ECLs are assessed on historical credit loss experience, adjusted for forward-looking factors specific to the counterparts and the economic environment.
For cash, cash equivalents and marketable securities, ECLs are assessed for credit losses that result from default events that are possible within the next twelve months (12-month ECL). Credit risk is continuously tracked and monitored in order to identify significant deterioration. For those credit exposures for which there have been a significant increase in credit risk since initial recognition, an allowance is recognized for credit losses expected over the remaining life of the exposure, irrespective of the timing of the default.
Shareholders’ Equity
The share capital comprises the nominal amount of the parent company’s ordinary shares, each at a nominal value of DKK 1, or approximately €0.13. All shares are fully paid.
Share premium comprises the amounts received, attributable to shareholders’ equity, in excess of the nominal amount of the shares issued at the parent company’s capital increases, reduced by any expenses directly attributable to the capital increases. Under Danish legislation, share premium is an unrestricted reserve that is available to be distributed as dividends to a company’s shareholders. Also, under Danish legislation, the share premium reserve can be used to offset accumulated deficits.
Treasury shares reserve comprise nominal amounts of holding of own equity instruments. No gain or loss is recognized in profit or loss on the purchase, sale, transfer or cancellation of the Company’s own equity instruments. The treasury shares reserve is part of unrestricted reserves and accordingly, reduce the amount available to be distributed as dividends to the Company’s shareholders.
Foreign currency translation reserve includes exchange rate adjustments relating to the translation of the results and net assets of foreign operations from their functional currencies to the presentation currency.
The accumulated reserve of a foreign operation is reclassified to the statement of profit or loss at the time the Company loses control, and thus cease to consolidate such foreign operation. The foreign currency translation reserve is an unrestricted reserve that is available to be distributed as dividends to the Company’s shareholders.
Retained earnings/(accumulated deficit) represents the accumulated profits or losses from the Company’s operations, including corresponding entries to share-based payments recognized in the statement of profit or loss. In addition, premiums from acquisition and sale of treasury shares are recognized as part of this reserve. A positive reserve is available to be distributed as dividends to the Company’s shareholders.
Convertible Senior Notes and Embedded Derivative Liabilities
Convertible senior notes (“convertible notes”) are separated into a financial liability and an embedded derivative component based on the terms and conditions of the contract. The embedded derivative component is accounted for separately if it is not deemed closely related to the financial liability.
The convertible notes include an embedded equity conversion option which is not deemed closely related to the financial liability, and initially recognized and measured separately at fair value as derivative liabilities based on the stated terms upon issuance of the convertible notes. The conversion option is classified as a foreign currency conversion option and thus not convertible into a fixed number of shares for a fixed amount of cash. Accordingly, the conversion option is subsequently recognized and measured as a derivative liability at fair value through profit or loss, with any subsequent remeasurement gains or losses recognized as part of finance income or expenses.
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In addition, the convertible notes include a redemption option, which entitle the Company to redeem the notes at a cash amount equal to the principal amount of the convertible notes, plus accrued and unpaid interest. The redemption option is closely related to the financial liability, and not separately accounted for. The initial carrying amount of the financial liability component including the redemption option is the residual amount of the proceeds, net of transaction costs, after separating the derivative component.
Transaction costs are apportioned between the financial liability and derivative component based on the allocation of proceeds when the instrument is initially recognized. Transaction costs apportioned to the financial liability component form part of the effective interest and are amortized over the expected lifetime of the liability. Transaction costs allocated to the derivative component are expensed as incurred.
The financial liability is subsequently measured at amortized cost until it is extinguished on conversion, upon optional redemption or repayment at maturity. Convertible notes are presented as borrowings, together with the derivative liabilities on the statement of financial position, separately under current liabilities as “Convertible notes, matures in April 2028.”
Royalty Funding Liabilities
Royalty funding liabilities relate to the Company’s contractual obligations to pay a predetermined percentage of future revenue from sale of commercial products until reaching a predetermined multiple of proceeds received, pursuant to the detailed provisions of the capped synthetic royalty funding agreements.
Where relevant, royalty funding liabilities are separated into a financial liability and embedded derivative components based on the terms and conditions of the applicable royalty funding agreement. Embedded derivative components are accounted for separately, unless these are deemed closely related to the financial liability. The royalty funding agreements include a buy-out option where the value is dependent on non-financial variables that are specific to the Company. Accordingly, the buy-out option is not accounted for separately as a derivative.
The financial liability is recognized when the Company becomes party to the contractual provisions of the royalty funding agreement and measured at amortized cost until it is extinguished upon exercising a buy-out option or upon achieving the predetermined multiple of proceeds received.
The effective interest rate is estimated at initial recognition and takes into account incremental transaction costs and anticipated amount and timing of future cash flows, which further depends on future commercial revenue forecasts and the probability of exercising the buy-out option. The amortized cost is remeasured prospectively when there is a material change in expectations to amount and timing of future cash flows, which will increase or decrease future interest expenses. Remeasurement gain or losses are recognized through the profit or loss as finance income or expenses, respectively.
Royalty funding liabilities that are classified as a financial liability are presented as part of borrowings in the statement of financial position.
Leases
Right-of-use Assets
Right-of-use assets are recognized at the lease commencement date, defined as the date the underlying asset is available for use. Right-of-use assets are measured at cost, less any accumulated depreciation and impairment losses, and adjusted for any remeasurement of lease liabilities. The cost of right-of-use assets include the amount of lease liabilities recognized, initial direct costs incurred, and lease payments made at or before the commencement date less any incentives received. In addition, right-of-use assets also include an estimate of costs to be incurred by the Company in dismantling or restoring the underlying asset to the condition if required by the terms and condition of the lease, if any.
Right-of-use assets are presented as part of property, plant and equipment, and depreciated on a straight-line basis over the shorter of the lease term and the estimated useful lives of the assets.
| Financial Statements | 55 |
Lease Liabilities
At the lease commencement date, lease liabilities are recognized and measured at the present value of fixed lease payments and variable lease payments that depend on an index or a rate, whereas variable lease payments and payments related to non-lease components are excluded. Variable lease payments that do not depend on an index or a rate are recognized as expenses in the statement of profit or loss when incurred.
When interest rates implicit in the lease contracts are not readily available, the present value of lease payments are calculated by applying the incremental borrowing rate of the relevant entity holding the lease. Following the commencement date, the incremental borrowing rate is not changed unless the lease term is modified, or if the lease payments are modified and this modification results from a change in floating interest rates. From the lease commencement date and over the lease term, the carrying amount of lease liabilities is increased to reflect the accretion of interest and reduced for the lease payments made. In addition, the carrying amount of lease liabilities is remeasured if there is a modification, a change in lease term, or a change in lease payments, including changes to future payments resulting from a change in an index used to determine such lease payments.
Lease liabilities are presented as part of borrowings in the statement of financial position.
Provisions
Provisions comprise unsettled sales deductions and product returns regarding revenue from sale of commercial products where amount or timing of payment is uncertain.
Provisions for sales deductions attributed to various commercial arrangements, managed healthcare organizations, government programs, and co-pay arrangements are recognized when the related sales takes place and measured using the expected value method. Payable amounts for managed healthcare organizations and government programs are generally settled within 180 days from the transaction date.
Provisions for estimated product returns are measured according to the contractual sales price based on expected product returns.
Trade Payables and Accrued Expenses
Trade payables and accrued expenses are measured at amortized cost.
Other Liabilities
Other liabilities comprise payables to public authorities, short-term employee benefits, and sales deductions. Other liabilities are measured at their net-realizable values.
Contract Liabilities
Contract liabilities comprise deferred income from collaboration and license agreements, where consideration received does not match the individual deliverables with respect to amount and satisfied performance obligations.
Contract liabilities are measured at the fair value of the consideration received and is recognized as revenue in the statement of profit or loss when the relevant performance obligation, to which the deferred income relates, is satisfied.
Cash Flow Statement
The cash flow statement shows cash flows from operating, investing and financing activities as well as cash and cash equivalents at the beginning and the end of the financial year.
| Financial Statements | 56 |
Cash flows from operating activities are presented using the indirect method and calculated as the profit or loss adjusted for non-cash items, working capital changes as well as finance income, finance expenses and income taxes paid.
Cash flows from investing activities include payments in connection with acquisition, development, improvement and sale, etc., of property, plant and equipment, investments in associates and marketable securities.
Cash flows from financing activities comprise payments related to the capital structure of the Company, including changes in the share capital and treasury shares and issuance and repayments under the Company’s borrowing activities.
The effect of exchange rate changes on cash and cash equivalents held or due in a foreign currency is presented separately from cash flows from operating, investing and financing activities. Cash flows in currencies other than the functional currency are recognized in the cash flow statement, using the average exchange rates.
Cash and cash equivalents comprise cash and on-demand bank deposits with financial institutions and highly liquid marketable securities with a maturity of three months or less after the date of acquisition.
Basic Earnings per Share
Basic Earnings per Share (“EPS”) is calculated as the consolidated net income or loss from continuing operations for the period divided by the weighted average number of ordinary shares outstanding. The weighted average number of shares takes into account the weighted average number of treasury shares during the year.
Diluted Earnings per Share
Diluted EPS is calculated as the consolidated net income or loss from continuing operations for the period divided by the weighted average number of ordinary shares outstanding adjusted for the weighted average effect of changes in treasury shares during the year, and the dilutive effect of outstanding warrants and convertible notes. If the consolidated statement of profit or loss shows a net loss, no adjustment is made for the dilutive effect, as such effect would be anti-dilutive.
New IFRS Accounting Standards Not Yet Effective
The IASB has issued a number of new or amended standards, which have not yet become effective or have not yet been adopted by the EU. Therefore, these new standards have not been incorporated in these financial statements.
IFRS 18, “Presentation and Disclosure in Financial Statements”
In April 2024, the IASB issued IFRS 18, “Presentation and Disclosure in Financial Statements” (“IFRS 18”), which replaces IAS 1, “Presentation in Financial Statements.” IFRS 18 introduces new categories and subtotals in the statement of profit or loss, into:
| • | Operating activities; |
| • | Investing activities; |
| • | Financing activities; |
| • | Income taxes; and |
| • | Discontinued operations. |
In addition, IFRS 18 includes new requirements for the location, aggregation and disaggregation of financial information, and disclosure of management-defined performance measures, as defined, if any. IFRS 18 does not include any measurement changes.
| Financial Statements | 57 |
If approved by the EU, the amendments will be effective for annual reporting periods beginning on or after January 1, 2027, and must be applied retrospectively, with early adoption permitted. While IFRS 18 will change the structure and subtotal in the statement of profit or loss, the full impact from implementing IFRS 18 is currently being analyzed by the Company.
The financial statements are not expected to be affected by other new or amended standards.
Note 3 – Significant Accounting Judgements and Estimates
In the application of the Company’s accounting policies, management is required to make judgements, estimates and assumptions about the carrying amounts of assets and liabilities that are not readily apparent from other sources. Judgements, estimates and assumptions applied are based on historical experience and other factors that are relevant, and which are available at the reporting date. Uncertainty concerning estimates and assumptions could result in outcomes that require a material adjustment to assets and liabilities in future periods.
Estimates and underlying assumptions are reviewed on an ongoing basis. Revisions to accounting estimates are recognized prospectively. While the application of critical accounting estimates is subject to material estimation uncertainties, management’s ongoing revisions of critical accounting estimates and underlying assumptions have not revealed any material impact in any of the years presented in these financial statements.
There have been no changes to the application of significant accounting judgements, or estimation uncertainties regarding accounting estimates compared to December 31, 2023.
Significant Accounting Judgements
Significant accounting judgements which have a material impact on the financial statements are described in the following sections.
Internally Generated Intangible Assets
Development of Drug Candidates
IAS 38, “Intangible Assets” prescribes that intangible assets arising from development projects must be recognized in the statements of financial position if the criteria for capitalization are met. That means (1) that the development project is clearly defined and identifiable; (2) that technological feasibility, adequate resources to complete and a market for the product or an internal use of the project can be documented; (3) that the expenditure attributable to the development project can be measured reliably; and (4) that the Company has the intent to produce and market the product. Such an intangible asset shall be recognized if it can be demonstrated that the future income from the development project will exceed the aggregate cost of development, production, sale and administration of the product.
Due to the risk associated with drug development, future income from development projects related to drug candidates cannot be determined with sufficient certainty until the development activities have been completed and the necessary marketing approvals have been obtained. Accordingly, the Company does not recognize internally generated intangible assets at this time.
Significant Estimation Uncertainties
The key assumptions concerning the future and other key sources of estimation uncertainty at the reporting date, that have a risk of causing a material adjustment to the carrying amounts of assets and liabilities within the next financial year, are described below.
Revenue and Provisions
Provisions for Sales Deductions and Product Returns
Sales deductions and product returns are considered variable consideration and constrained to the extent that a significant reversal in the amount of recognized revenue will not occur when the uncertainties associated with the rebate or chargeback item are subsequently resolved, or for product returns, when the products are distributed to patients.
| Financial Statements | 58 |
Provisions for unsettled sales deductions and product returns are estimated on the basis of a percentage of sales as defined by individual agreements and contracts, and for government rebates by individual state- and plan agreements. Further inputs to the calculations are based on payer channel mix, current contract prices under eligible programs and current inventory levels in the distribution channels. Inputs to the calculations are subject to estimation and assumptions and are based on historical experience and other factors that are relevant, and which are available at the reporting date. Provisions are adjusted to absolute amounts and recognized as other liabilities when estimated sales deductions are processed.
As of December 31, 2024, provisions for sales deductions and product returns were €99.1 million compared to €32.7 million, as of December 31, 2023. The development in total provisions is disclosed in Note 15, “Provisions.” Due to the nature of these provisions, it is not practicable to give meaningful sensitivity estimates due to the large volume of variables that contribute to the overall rebates, chargebacks, product returns and other sales deductions. Provisions are reviewed and adjusted regularly considering contractual terms, regulatory obligations, payer trends, historical experiences and market projections.
Share-based Payment
Warrant Compensation Costs
IFRS 2, “Share-Based Payment” requires an entity to reflect in its statement of profit or loss and financial position, the effects of share-based payment transactions. Warrant compensation costs are recognized as cost of sales, research and development costs or selling, general and administrative expenses, as appropriate, over the vesting period, based on management’s best estimate of the number of warrants that will ultimately vest, which is subject to uncertainty.
Warrant compensation costs are measured according to the grant date fair value of the warrants granted. Estimating fair values requires the Company to apply generally accepted valuation models and apply these models consistently according to the terms and conditions of the specific warrant program. Under all warrant programs, the Black-Scholes option-pricing model has been applied to determine the fair value of warrants granted. Subjective judgements and assumptions, which are subject to estimation uncertainties, need to be exercised in determining the appropriate input to the valuation model. These inputs include expected volatility of the Company’s share price for a historic period equaling the expected lifetime of the warrants, reflecting the assumption that the historical volatility over a period similar to the life of the warrants is indicative of future trends, expected forfeitures and expected lifetime of warrants.
Warrant compensation cost recognized in the consolidated statement of profit or loss was €19.7 million, and €28.8 million for the years ended December 31, 2024, and 2023, respectively. Changes to inputs applied to the Black-Scholes option pricing model could affect the warrant compensation cost. Refer to Note 7, “Share-based Payment,” for additional details.
Valuation of Embedded Derivatives
Foreign currency conversion options embedded in the convertible notes are accounted for separately as derivative liabilities at fair value through profit or loss.
Fair value cannot be measured based on quoted prices in active markets, or other observable input, and accordingly, derivative liabilities are measured by use of valuation techniques in the form of the Black-Scholes option pricing model. Subjective judgements and assumptions, which are subject to estimation uncertainties, need to be exercised in determining the appropriate unobservable input to the valuation model (Level 3 in the fair value hierarchy). This includes volatility of the Company’s share price for a historic period, reflecting the assumption that the historical volatility is indicative of a period similar to the expected lifetime of the options.
As of December 31, 2024, the valuation of the derivative liabilities was €150.7 million compared to €143.3 million as of December 31, 2023. Changes in assumptions relating to these factors could affect the reported fair value of derivative liabilities. Refer to Note 16 “Financial Assets and Liabilities”, for additional details.
| Financial Statements | 59 |
Measurement of Royalty Funding Liabilities
The carrying amount of royalty funding liabilities is measured according to anticipated future cash flows, which further depends on the amount and timing of future revenue from sale of commercial products. Assumptions that impact the amount and timing of future sale of commercial products are subject to estimation uncertainties, and are subject to a number of factors which are not within the Company’s control.
As of December 31, 2024, the carrying amount of the royalty funding liabilities was €305.4 million compared to €138.4 million as of December 31, 2023. As of December 31, 2024, royalty funding liabilities comprised two agreements compared with one agreement as of December 31, 2023. The Company will periodically revisit the anticipated amount and timing of future sale of commercial products and to the extent such amount or timing is materially different from the previous estimates, a remeasurement gain or loss is recognized through the profit or loss as finance income or expenses, as appropriate, which would further increase or decrease future interest expenses. Refer to Note 16 “Financial Assets and Liabilities” for additional details.
Note 4 – Revenue
Revenue has been recognized in the statements of profit or loss with the following amounts:
| Group | Parent | |||||||||||||||
| (EUR’000) | 2024 | 2023 | 2024 | 2023 | ||||||||||||
| Revenue |
||||||||||||||||
| Commercial products |
225,728 | 178,663 | 47,940 | 54,710 | ||||||||||||
| Rendering of services |
11,794 | 21,659 | 252,566 | 243,002 | ||||||||||||
| Clinical supply |
3,776 | 319 | — | — | ||||||||||||
| Licenses |
122,343 | 66,077 | 105,274 | 5,000 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Total revenue |
363,641 | 266,718 | 405,780 | 302,712 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Attributable to |
||||||||||||||||
| Commercial customers |
225,728 | 178,663 | 400 | — | ||||||||||||
| Collaboration partners and license agreements |
137,913 | 88,055 | 95,397 | — | ||||||||||||
| Group enterprises |
— | — | 309,983 | 302,712 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Total revenue |
363,641 | 266,718 | 405,780 | 302,712 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Specified by timing of recognition |
||||||||||||||||
| Recognized over time |
11,794 | 21,659 | 252,566 | 243,002 | ||||||||||||
| Recognized at a point in time |
351,847 | 245,059 | 153,214 | 59,710 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Total revenue |
363,641 | 266,718 | 405,780 | 302,712 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Specified per geographical area |
||||||||||||||||
| Europe (1) |
123,336 | 869 | 405,386 | 302,712 | ||||||||||||
| North America |
233,220 | 191,677 | 394 | — | ||||||||||||
| Rest of world |
7,085 | 74,172 | — | — | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Total revenue |
363,641 | 266,718 | 405,780 | 302,712 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| (1) | For the year ended December 31, 2024 Denmark, the country of domicile, contributed with €95.4 million of revenue (parent €405.0 million and €302.7 million for 2023). |
| Financial Statements | 60 |
Commercial Customers
Revenue attributable to commercial customers by product were as follows:
| Group | ||||||||
| 2024 | 2023 | |||||||
| (EUR’000) | ||||||||
| Revenue from commercial products |
||||||||
| SKYTROFA® |
197,001 | 178,663 | ||||||
| YORVIPATH® |
28,727 | — | ||||||
|
|
|
|
|
|||||
| Total revenue from commercial products |
225,728 | 178,663 | ||||||
|
|
|
|
|
|||||
Revenue from sales of SKYTROFA® is primarily sold to specialty pharmacies and specialty distributors in the U.S. market, and to wholesalers and pharmacies in Germany. Revenue from sale of SKYTROFA for the year ended December 31, 2024 was negatively impacted by an adjustment to estimates and assumptions for sales deductions of €4.7 million, which was related to periods prior to January 1, 2024. The adjustment was primarily attributable to a different payer and rebate mix than anticipated, and on which provisions for prior years were based.
In November 2023, TransCon PTH received marketing approval in the EU and European Economic Area countries and is marketed as YORVIPATH®. In August 2024, TransCon PTH received marketing approval in the U.S. and is marketed in the U.S. as YORVIPATH. The Company began selling YORVIPATH, primarily to pharmacies in Europe in the first quarter of 2024, and to specialty pharmacies in the U.S. in December 2024. In addition, the Company began selling YORIVPATH to distributors in the second quarter of 2024.
For the years ended December 31, 2024 and 2023, four commercial customers represented more than 10% of sales to commercial customers.
Collaboration Partners and License Agreements
Revenue attributable to collaboration partners and license agreements relates to Novo Nordisk A/S (“Novo Nordisk”), Eyconis, Inc. (“Eyconis”), Teijin Limited (“Teijin”) and VISEN Pharmaceuticals (“VISEN”). Under the collaboration agreements, the Company also provides various research and development services which are invoiced to collaboration partners. Revenue for these activities is presented as part of “Rendering of services.” Employment costs related to these activities are presented as Research and Development Costs in the consolidated statement of profit or loss.
Novo Nordisk
In November 2024, the Company entered into a research and development collaboration and license agreement (the “Novo Nordisk Agreement”) with Novo Nordisk pursuant to which the Company granted Novo Nordisk an exclusive worldwide license to the TransCon technology platform to develop, manufacture and commercialize Novo Nordisk proprietary products (including Semaglutide) in metabolic diseases (including obesity and type 2 diabetes) and a product-by-product exclusive license in cardiovascular diseases (the “IP”).
The Novo Nordisk Agreement includes provisions requiring at least one TransCon Semaglutide product and at least one other TransCon technology-based product to be identified, developed and commercialized in metabolic diseases to maintain certain exclusivities in the field, with additional provisions for cardiovascular diseases. Under the terms of the Novo Nordisk Agreement, Novo Nordisk also receives exclusive rights to expand any resulting metabolic disease products into other therapeutic areas. The lead program in the collaboration is a once-monthly TransCon Semaglutide product candidate that will initially target obesity and type 2 diabetes.
Under the Novo Nordisk Agreement, the Company has the potential to receive total payments of up to $285 million in upfront, development and regulatory milestone payments for the lead program. In addition, the Company has the potential to receive sales-based milestone payments and tiered royalties on global net sales. The $285 million includes an upfront fee of $100 million for the exclusive license. For each additional metabolic or cardiovascular disease product candidate, the Company will be eligible
| Financial Statements | 61 |
to receive payments of up to $77.5 million in development and regulatory milestone payments. In addition, the Company has the potential to receive sales-based milestone payments and tiered royalties on global net sales. Novo Nordisk agreed to pay royalties for each potential licensed product developed under the agreement that are an escalating tiered, mid-single digit percentage of the annual net sales of such licensed product and are subject to reduction due to patent valid claim expiration, biosimilar product market share, payment made under certain licenses for third party intellectual property and Inflation Reduction Act price negotiations.
Under the Novo Nordisk Agreement, the Company agreed to conduct certain pre-agreed early research and development of TransCon product candidates under the collaboration and is eligible to receive cost reimbursement from Novo Nordisk for its performance of such research and development activities under the Novo Nordisk Agreement with respect to such TransCon product candidates. Novo Nordisk is responsible for any other non-clinical and clinical development, regulatory, commercial manufacturing, and commercialization of such TransCon product candidates, and all costs associated with such activities.
Subject to the terms of the Novo Nordisk Agreement, the Company granted Novo Nordisk an exclusive, worldwide, royalty-bearing license, with the right to grant sublicenses, to use its proprietary TransCon technology platform to develop, manufacture and commercialize Novo Nordisk proprietary products in metabolic diseases (including obesity and type 2 diabetes) and a product-by-product exclusive license in cardiovascular diseases. Additionally, the Company granted Novo Nordisk an exclusive, worldwide, royalty-bearing license, with the right to grant sublicenses, to use its proprietary TransCon technology platform to develop, manufacture and commercialize GLP-1 receptor products using the TransCon technology for all indications, except for (i) certain pre-agreed rare endocrine indications, (ii) all indications in respect of the eye and adnexa and (iii) all indications in respect of oncology.
Unless earlier terminated, the Novo Nordisk Agreement has a royalty term that continues, on a per licensed product and per country basis, until the later of (i) the expiration of the last valid patent claim for any of our patents, joint improvement patents, licensed product patents as well as any improvements made by Novo Nordisk covering the licensed product’s dosage regimen or target product profile, or (ii) 11 years after the first commercial sale of such licensed product in such country.
The IP comprises the patent protected TransCon technology platform, where future activities do not affect its existing stand-alone functionalities. Accordingly, the IP is classified as “right-to-use” licenses, with revenue recognized at a point in time, where the licensee is granted access to the IP.
For the year ended December 31, 2024, “Licenses” includes revenue of €95.3 million related to the upfront payment, which is allocated to transfer of the Company’s intellectual property.
Eyconis
In January 2024, the Company announced the formation and launch with Frazier Life Sciences of Eyconis, a separate company created to develop, manufacture, and commercialize TransCon ophthalmology assets globally, together with a $150 million commitment from an investor syndicate that includes Frazier, RA Capital Management, venBio, and HealthQuest Capital.
The Company has granted Eyconis exclusive rights to develop and commercialize TransCon ophthalmology products globally (the “Eyconis Agreement”) and received, as consideration, an equity position in the newly formed company. In addition, the Company is eligible to receive development, regulatory, and sales milestone payments, plus single digit royalties on global net sales of commercialized products, if any.
The Company is expected to provide various research and development services, which are subject to separate remuneration, and which will be recognized as revenue over time as rendering of services or reimbursement revenue, as applicable.
| Financial Statements | 62 |
For the year ended December 31, 2024, “Licenses” includes revenue of €27.1 million related to the non-cash upfront payment through an equity position in Eyconis, adjusted for internal profit, which is allocated to transfer of the Company’s intellectual property (the “IP”). The internal profit relates to the Company’s share of the non-cash upfront payment which is recognized as part of “Investments in associates” and recognized as revenue from “Licenses” as the IP is amortized in the associate.
No revenue from royalties or milestones has been recognized under the Eyconis agreement.
Teijin
In November 2023, the Company entered into an exclusive license agreement (the “Teijin Agreement”) with Teijin for the further development and commercialization of TransCon hGH, TransCon PTH, and TransCon CNP for endocrinology rare disease (the “Licensed Products”) in Japan. Under the terms of the Teijin Agreement, the Company received an upfront payment of $70 million, with additional development and regulatory milestones of up to $175 million and commercial milestones. In addition, the Company is eligible to receive royalties on net sales of the Licensed Products in Japan, of up to mid-20’s percent.
Further, the Company will provide clinical and commercial supply, and development services for joint activities, which are subject to separate remuneration, and which will be recognized as revenue over time as rendering of services or reimbursement revenue, as applicable.
At December 31, 2024, none of the Licensed Products have received marketing authorization in Japan. The Licensed Products (the “IP”) are patent protected, where future activities do not affect their existing stand-alone functionalities. Accordingly, all three licenses are classified as “right-to-use” licenses, with revenue recognized at a point in time, where the licensee is granted access to the IP.
For the year ended December 31, 2023, “Licenses” includes revenue of €63.7 million related to the upfront payment, which is allocated to transfer of the Company’s IP.
No revenue from royalties or milestones has been recognized under the Teijin Agreement.
VISEN
In November 2018, the Company entered into three exclusive license agreements with VISEN. Revenue from collaboration partners and license agreements also includes license income, rendering of services and sale of clinical supply under the VISEN Agreements.
Note 5 – Segment Information
The Company is managed and operated as one business unit. Accordingly, no additional information on business segments or geographical areas is disclosed apart from revenue on geographical areas as disclosed in Note 4, “Revenue.” Revenue is specified on geographical areas according to the location of the customer.
The Company’s intangible assets and property, plant and equipment located by region are specified below:
| Group | ||||||||
| (EUR’000) | 2024 | 2023 | ||||||
| Intangible assets and property, plant and equipment |
||||||||
| Europe (1) |
39,640 | 46,464 | ||||||
| North America |
63,102 | 68,589 | ||||||
|
|
|
|
|
|||||
| Total intangible assets and property, plant and equipment |
102,742 | 115,053 | ||||||
|
|
|
|
|
|||||
| (1) | As of December 31, 2024 and December 31, 2023, intangible assets and property, plant and equipment of €27.9 million and €32.9 million, respectively, is located in Denmark, the country of domicile |
The Parent Company has no non-current segment assets outside Denmark (domicile country).
| Financial Statements | 63 |
Note 6 – Employee costs
| Group | Parent | |||||||||||||||
| (EUR’000) | 2024 | 2023 | 2024 | 2023 | ||||||||||||
| Employee costs |
||||||||||||||||
| Wages and salaries |
173,474 | 170,278 | 76,448 | 70,561 | ||||||||||||
| Share-based payment |
95,512 | 66,660 | 61,038 | 43,259 | ||||||||||||
| Pensions (defined contribution plans) |
4,485 | 4,403 | 1,766 | 2,086 | ||||||||||||
| Social security costs |
15,003 | 12,877 | 633 | 559 | ||||||||||||
| Other employee costs |
4,061 | 4,238 | 2,133 | 2,605 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Total employee costs |
292,535 | 258,456 | 142,018 | 119,070 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Included in the profit or loss |
||||||||||||||||
| Cost of sales (1) |
16,487 | 15,748 | 16,487 | 15,748 | ||||||||||||
| Research and development costs |
131,867 | 127,002 | 66,073 | 57,756 | ||||||||||||
| Selling, general, and administrative expenses |
144,181 | 115,706 | 59,458 | 45,566 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Total employee costs |
292,535 | 258,456 | 142,018 | 119,070 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Average number of employees |
892 | 851 | 445 | 404 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| (1) | Includes employee costs capitalized as part of inventories. |
Key Management Personnel comprises the Board of Directors, the Executive Board and Non-executive Senior Management. Compensation to Key Management Personnel comprises salaries, participation in annual bonus schemes, pensions (defined contributions plans), and share-based compensation. Share-based compensation is elaborated in further details in Note 7, “Share-based Payment.”
Compensation to Key Management Personnel included in total employee costs is summarized below:
| Board of Directors (1) |
Executive Board (2) | Non-executive Senior Management |
||||||||||||||||||||||
| (EUR’000) | 2024 | 2023 | 2024 | 2023 | 2024 | 2023 | ||||||||||||||||||
| Compensation |
||||||||||||||||||||||||
| Wages and salaries |
482 | 543 | 4,148 | 4,375 | 3,286 | 4,673 | ||||||||||||||||||
| Share-based payment |
2,169 | 1,276 | 18,334 | 13,243 | 10,266 | 9,529 | ||||||||||||||||||
| Pensions (defined contribution plans) |
— | — | 57 | 54 | 98 | 122 | ||||||||||||||||||
| Social security costs |
— | — | 118 | 103 | 52 | 45 | ||||||||||||||||||
| Other employee cost |
— | — | 20 | 20 | 25 | 40 | ||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| Total compensation |
2,651 | 1,819 | 22,677 | 17,795 | 13,727 | 14,409 | ||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
| (1) | The Board of Directors comprised six persons in 2024 and six to seven persons 2023. |
| (2) | The Executive Board comprised four persons in 2024 and 2023. |
Note 7 – Share-based Payment
As an incentive to the senior management, other employees, members of the Board and select consultants, Ascendis Pharma A/S has established warrant programs, a RSU program (adopted in December 2021), and a PSU program (adopted in February 2023), which are all classified as equity-settled share-based payment transactions.
Restricted Stock Unit Program
RSUs are granted by the Board in accordance with authorizations given to it by the shareholders of Ascendis Pharma A/S to members of senior management, certain other employees and certain members of the Board (“RSU-holders”) in accordance with the Company’s RSU Program adopted in December 2021. Further, RSUs may be granted to select consultants. One RSU represents a right for the RSU-holder to receive one ADS of Ascendis Pharma A/S upon vesting if the vesting conditions are met or waived by the Board at its discretion.
| Financial Statements | 64 |
Performance Stock Unit Program
PSUs are granted by the Board in accordance with authorizations given to it by the shareholders of Ascendis Pharma A/S to certain members of senior management (the “PSU-holders”). In addition, PSUs may be granted to other employees, select consultants and members of the Board. PSUs were granted for the first time in March 2023. One PSU represents a right for the PSU-holder to receive one ADS of Ascendis Pharma A/S upon vesting if the vesting conditions are met or waived by the Board at its discretion.
Vesting Conditions
RSUs granted vest over a predetermined service period, and accordingly require RSU-holders to be employed, or provide a specified period of service (“service conditions”). RSUs vest over three years with 1/3 of the RSUs vesting on each anniversary date from the date of grant. RSUs generally cease to vest from the date of termination of employment, or for the Board, termination of board membership, whereas unvested RSUs will lapse.
One PSU represents a right for the PSU-holder to receive one ADS representing ordinary shares of Ascendis Pharma A/S upon vesting. PSUs vest in a manner similar to the service conditions of the RSUs. For the March 2023 PSU grant, in addition to service conditions, vesting is also contingent upon achievement of performance-based targets as determined by the Board, provided that no more than 10% of each tranche may be directly attributable to accomplishment of financial results achieved in the financial year prior to the vesting date. For the March 2024 PSU grant, in addition to service conditions, vesting is also contingent upon achievement of long-term strategic goals as evaluated by the Board no later than two weeks prior to each vesting date. Exceeding performance targets will not result in vesting of more PSUs than 100%, nor will it result in additional grants.
RSUs and PSUs generally cease to vest from the date of termination of employment or board membership, as applicable, whereas unvested RSUs or PSUs will be forfeited. The Board may at its discretion and on an individual basis decide to deviate from the vesting conditions, including deciding to accelerate vesting in the event of termination of employment or board membership, as applicable.
Settlement Options
All RSUs and PSUs are settled at the time of vesting by transfer of treasury shares that are ADSs repurchased in the market. In jurisdictions where the Company is required to withhold and settle tax with the tax authority on behalf of the RSU/PSU-holders, the Company withholds the number of RSUs or PSUs that are equal to the estimated monetary value of the RSU/PSU-holders tax obligation from the total number of RSUs or PSUs that otherwise would have been transferred to the RSU/PSU holder upon vesting. These settlements are presented as “Net settlement under stock incentive programs” in the consolidated statement of equity. The Company may at its sole discretion choose to make a cash settlement instead of delivering ADSs.
Adjustments
RSU-holders and PSU-holders are entitled to an adjustment of the number of RSUs or PSUs granted, in the event of certain corporate changes, including among other events, increases or decreases to the share capital at a price below or above market value, the issuance of bonus shares, and changes in the nominal value of each share. In addition, the RSU and PSU Programs contain provisions to accelerate vesting, or compensate with grant of new equity instruments, in the event of restructuring events including change in control events.
| Financial Statements | 65 |
RSU and PSU Activity
The following table specifies the number of RSUs and PSUs granted and outstanding:
| Restricted | Performance | |||||||||||
| Stock Units | Stock Units | Total | ||||||||||
| (Number) | ||||||||||||
| Outstanding at January 1, 2022 |
148,148 | — | 148,148 | |||||||||
| Transferred during the year |
(41,685 | ) | — | (41,685 | ) | |||||||
| Forfeited during the year |
(23,971 | ) | — | (23,971 | ) | |||||||
|
|
|
|
|
|
|
|||||||
| Outstanding at December 31, 2022 |
82,492 | — | 82,492 | |||||||||
|
|
|
|
|
|
|
|||||||
| Granted during the year (1) |
609,860 | 112,268 | 722,128 | |||||||||
| Settled during the year |
(18,132 | ) | — | (18,132 | ) | |||||||
| Transferred during the year |
(20,098 | ) | — | (20,098 | ) | |||||||
| Forfeited during the year |
(77,497 | ) | (7,245 | ) | (84,742 | ) | ||||||
|
|
|
|
|
|
|
|||||||
| Outstanding at December 31, 2023 |
576,625 | 105,023 | 681,648 | |||||||||
|
|
|
|
|
|
|
|||||||
| Granted during the year (1) |
717,980 | 92,655 | 810,635 | |||||||||
| Transferred during the year |
(212,160 | ) | (35,007 | ) | (247,167 | ) | ||||||
| Forfeited during the year |
(88,638 | ) | (6,004 | ) | (94,642 | ) | ||||||
|
|
|
|
|
|
|
|||||||
| Outstanding at December 31, 2024 |
993,807 | 156,667 | 1,150,474 | |||||||||
|
|
|
|
|
|
|
|||||||
| Specified by vesting date |
||||||||||||
| 2025 |
384,098 | 62,304 | 446,402 | |||||||||
| 2026 |
384,438 | 63,478 | 447,916 | |||||||||
| 2027 |
225,271 | 30,885 | 256,156 | |||||||||
|
|
|
|
|
|
|
|||||||
| Outstanding at December 31, 2024 |
993,807 | 156,667 | 1,150,474 | |||||||||
|
|
|
|
|
|
|
|||||||
| (1) | The fair value of RSUs and PSUs is determined on the basis of the closing ADS price on the grant date. The fair value of one RSU and one PSU at the date of grant was €141.01 and €105.96 for the years ended December 31, 2024 and December 31, 2023, respectively. |
Warrant Program
Warrants are granted by the Board in accordance with authorizations given to it by the shareholders of Ascendis Pharma A/S to all employees, members of the Board and select consultants (“warrant holders”). Each warrant carries the right to subscribe for one ordinary share of a nominal value of DKK 1. The exercise price is equal to the fair market value of the Company’s ordinary shares at the time of grant as determined by the Board. Apart from exercise prices, exercise periods and vesting conditions for board members, the programs are similar.
Vesting Conditions
Warrants granted vest over a predetermined service period and require warrant-holders provide a specified period of service. Warrants generally cease to vest from the date of termination. For warrants granted until November 2021, special vesting conditions apply in the event of termination. In relation to board members, the vesting shall cease on the termination date of the board membership regardless of the reason. In relation to consultants, the vesting shall cease on the termination date of the consultancy relationship. The warrant-holder will, however, be entitled to exercise vested warrants in the exercise periods after termination.
In the event that the employment contract is terminated, and the employee has not given the Company good reason to do so, the warrant-holder may keep the right to continued vesting and exercise of warrants as if the employment was still in effect. In such case, any expense not yet recognized for the outstanding warrants is recognized immediately.
Warrants Granted Until November 2021
Warrants granted from 2012 until November 2021, generally vest over 48 months with 1/48 of the warrants vesting per month from the date of grant. However, effective from January 2015, certain warrants granted to board members vest over 24 months with 1/24 of the warrants vesting per month from the date of grant.
| Financial Statements | 66 |
Warrants Granted From December 2021
For warrants granted to employees and consultants, 25% of the warrants vest one year after the date of grant, and the remaining 75% of the warrants granted vest over 36 months, with 1/36 of the warrants vesting per month, from one year after the date of grant.
For warrants granted to board members upon the board members accession, 25% of the warrants granted vest one year after the date of grant, and the remaining 75% of the warrants granted shall vest over 36 months, with 1/36 per month from one year after the date of grant. Regarding subsequent grants of warrants to board members, 50% of the warrants vest one year after the date of grant, and the remaining 50% of the warrants vest over 12 months, with 1/12 per month from one year after the date of grant.
Exercise Periods
Vested warrants may be exercised during certain exercise periods each year, within certain periods after publication of earnings data of a fiscal quarter, interim and annual reports, as per each program’s terms and conditions.
Warrants expire ten years after the grant date. Warrants not exercised by the warrant holder during the last exercise period shall become null and void without further notice or compensation or payment of any kind to the warrant-holder. If the warrant-holder is a consultant, advisor or board member, the exercise of warrants is conditional upon the warrant-holder’s continued service to the Company at the time the warrants are exercised. If the consultant’s, advisor’s or board member’s relationship with the Company should cease without this being attributable to the warrant-holder’s actions or omissions, the warrant-holder shall be entitled to exercise vested warrants in the pre-defined exercise periods.
Adjustments
Warrant-holders are entitled to an adjustment of the number of warrants issued and/or the exercise price applicable in the event of certain corporate changes.
Events giving rise to an adjustment include, among other things, increases or decreases to the share capital at a price below or above market value, the issuance of bonus shares, changes in the nominal value of each share, and payment of dividends in excess of 10% of the Company’s equity.
Warrant Activity
The following table specifies the number and weighted average exercise prices of, and movements in warrants:
| Weighted | ||||||||
| Average | ||||||||
| Warrants | Exercise Price | |||||||
| (number) | (EUR) | |||||||
| (Number) | ||||||||
| Outstanding at January 1, 2023 |
6,864,011 | 81.30 | ||||||
|
|
|
|
|
|||||
| Granted during the year |
395,275 | 91.07 | ||||||
| Exercised during the year (1) |
(555,144 | ) | 17.76 | |||||
| Forfeited during the year |
(180,358 | ) | 115.79 | |||||
|
|
|
|
|
|||||
| Outstanding at December 31, 2023 |
6,523,784 | 86.38 | ||||||
|
|
|
|
|
|||||
| Vested at the reporting date |
5,273,056 | 80.02 | ||||||
|
|
|
|
|
|||||
| Granted during the year |
504,105 | 122.48 | ||||||
| Exercised during the year(1) |
(682,048 | ) | 43.35 | |||||
| Forfeited during the year |
(141,719 | ) | 107.85 | |||||
|
|
|
|
|
|||||
| Outstanding at December 31, 2024 |
6,204,122 | 93.25 | ||||||
|
|
|
|
|
|||||
| Vested at the reporting date |
5,226,643 | 89.33 | ||||||
|
|
|
|
|
|||||
| (1) | The weighted average share price (listed in $) at the date of exercise was €135.86 and €98.10 for the years ended December 31, 2024 and 2023, respectively. |
| Financial Statements | 67 |
At December 31, 2024, the Board was authorized to grant up to 2,060,116 additional warrants to employees, board members and select consultants without preemptive subscription rights for the shareholders of Ascendis Pharma A/S.
The following table specifies the weighted average exercise prices and weighted average remaining contractual life for outstanding warrants at December 31, 2024, per grant year.
| Weighted | ||||||||||||
| Weighted | Average | |||||||||||
| Outstanding | Average | Remaining | ||||||||||
| Warrants | Exercise | Life | ||||||||||
| (number) | Price (EUR) | (months) | ||||||||||
| Granted before January 1, 2022 |
5,185,556 | 89.67 | 57 | |||||||||
| Granted in 2022 |
235,408 | 100.71 | 90 | |||||||||
| Granted in 2023 |
292,648 | 90.66 | 101 | |||||||||
| Granted in 2024 |
490,510 | 122.34 | 114 | |||||||||
|
|
|
|
|
|
|
|||||||
| Outstanding at December 31, 2024 |
6,204,122 | 93.25 | 65 | |||||||||
|
|
|
|
|
|
|
|||||||
At December 31, 2024, the exercise prices of outstanding warrants under the Company’s warrant programs range from €11.98 to €145.50 depending on the grant dates.
The range of exercise prices for outstanding warrants was €11.98 to €145.50 for the year ended December 31, 2023. The weighted average remaining life for outstanding warrants was 71 months for the financial year ended December 31, 2023.
Warrant Compensation Costs
Warrant compensation costs are recognized in the statements of profit or loss over the vesting period of the warrants granted.
Warrant compensation costs are determined with basis in the grant date fair value of the warrants granted and recognized over the vesting period. Fair value of the warrants is calculated at the grant dates by use of the Black-Scholes option pricing model with the following assumptions: (1) an exercise price equal to the estimated market price of the Company’s shares at the date of grant; (2) an expected lifetime of the warrants determined as a weighted average of the time from grant date to date of becoming exercisable and from grant date to expiry of the warrants; (3) a risk-free interest rate equaling the effective interest rate on a Danish government bond with the same lifetime as the warrants; (4) no payment of dividends; and (5) an expected volatility using the Company’s own share price (from 2021).
The following table summarizes the input to the Black-Scholes option pricing model and the calculated fair values for warrant grants in 2024 and 2023:
| 2024 | 2023 | |||||||
| Expected volatility |
50 | % | 49-51 | % | ||||
| Risk-free interest rate |
1.71 - 2.57 | % | 2.40 - 2.97 | % | ||||
| Expected life of warrants (years) |
6.0 | 6.0 | ||||||
| Weighted average exercise price |
€ | 122.48 | € | 91.07 | ||||
| Fair value of warrants granted in the year |
€ | 50.86 - 70.39 | € | 37.34 - 52.03 | ||||
Note 8 – Principal Accountant Fees and Services
The following table sets forth, for each of the years indicated, the fees billed by the Company’s independent public accountants and the proportion of each of the fees out of the total amount billed by the accountants.
| Financial Statements | 68 |
| Group | ||||||||
| (EUR’000) | 2024 | 2023 | ||||||
| Principal accountant fees and services |
||||||||
| Audit fees |
811 | 739 | ||||||
| Audit-related fees |
147 | — | ||||||
| Tax fees |
91 | 122 | ||||||
|
|
|
|
|
|||||
| Total principal accountant fees and services |
1,049 | 861 | ||||||
|
|
|
|
|
|||||
Note 9 – Tax on Profit/(Loss) for the Year and Deferred Tax
| Group | Parent | |||||||||||||||
| (EUR’000) | 2024 | 2023 | 2024 | 2023 | ||||||||||||
| Tax on profit/(loss) for the year: |
||||||||||||||||
| Current tax (expense)/income |
(3,289 | ) | (5,377 | ) | — | 40 | ||||||||||
| Current tax, adjustments to prior years |
(126 | ) | 3,904 | (40 | ) | (43 | ) | |||||||||
| Deferred tax, movement for the year |
(2,035 | ) | (1,044 | ) | — | — | ||||||||||
| Deferred tax, adjustments to prior years |
607 | (4,786 | ) | — | — | |||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| (4,843 | ) | (7,303 | ) | (40 | ) | (3 | ) | |||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Tax for the year can be explained as follows: |
||||||||||||||||
| Profit/(loss) before tax |
(373,241 | ) | (474,144 | ) | 174,740 | 39,719 | ||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Tax at the Danish corporation tax rate of 22% |
82,113 | 104,312 | (38,443 | ) | (8,738 | ) | ||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Tax effect of: |
||||||||||||||||
| Non-deductible costs |
(9,740 | ) | (8,494 | ) | (14,900 | ) | (10,644 | ) | ||||||||
| Additional tax deductions |
3,161 | 9,077 | 670 | 348 | ||||||||||||
| Impact from associates |
(4,413 | ) | (4,047 | ) | — | — | ||||||||||
| Prior year adjustments |
481 | (1,294 | ) | — | — | |||||||||||
| Other effects including effect of different tax rates |
182 | (882 | ) | (40 | ) | (43 | ) | |||||||||
| Deferred tax asset, not recognized |
(76,627 | ) | (105,975 | ) | 52,673 | 19,074 | ||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Tax on profit/(loss) for the year |
(4,843 | ) | (7,303 | ) | (40 | ) | (3 | ) | ||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Effective tax rate |
1.30 | % | 1.54 | % | 0.02 | % | (0.01 | )% | ||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Group | Parent | |||||||||||||||
| (EUR’000) | 2024 | 2023 | 2024 | 2023 | ||||||||||||
| Specification of Deferred Tax Assets/(Liabilities) |
||||||||||||||||
| Tax deductible losses |
434,997 | 521,697 | 9,522 | 102,921 | ||||||||||||
| Other temporary differences, assets |
164,479 | 16,256 | 38,391 | 3,522 | ||||||||||||
| Deferred tax asset, not recognized |
(599,476 | ) | (537,953 | ) | (47,913 | ) | (98,635 | ) | ||||||||
| Other temporary differences, liabilities |
(7,258 | ) | (5,830 | ) | — | (7,808 | ) | |||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Total Deferred Tax Assets/(Liabilities) at December, 31 |
(7,258 | ) | (5,830 | ) | — | — | ||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
At December 31, 2024 a deferred tax liability has been recognized in relation to taxable temporary differences in one jurisdiction, as we do not believe we will have any deductible temporary differences nor tax losses to deduct the taxable difference in, when they are expected to reverse.
Deferred tax assets have not been recognized in the statements of financial position as of December 31, 2024 due to uncertainty relating to future utilization. The deferred tax asset can be carried forward without timing limitations. For parent the deferred tax liabilities can be offset in deferred tax assets within the Danish joint taxation group.
The Company had tax losses carried forward of €1,946.2 million (Parent Company: €43.3 million) and €2,371.3 million (Parent Company: €467.8 million) at December 31, 2024 and December 31, 2023, respectively. Tax losses can be carried forward infinitely, where certain limitations exist for amounts to be utilized each year. Under Danish tax legislation, tax losses may be partly refunded by the tax authorities to the extent such tax losses arise from research and development activities the jointly taxed Danish entities had a negative taxable income, and accordingly were entitled to a tax refund of approximately €0.7 million for each of the years ended December 31, 2024 and 2023, respectively.
| Financial Statements | 69 |
The tax losses carried forward at December 31, 2023 (€2,371.3 million) has been reduced with €456.8 million (Parent Company €130.5 million). The reduction is as a consequence of two changes of tax principles made in the Danish tax returns for the taxable year 2023 (filed in June 2024). The changes are related to R&D costs (depreciations) and inventory write-down (cost price). This reduction in tax losses is still available as deferred tax assets, that can be recognized for future utilization; only not as a “Tax deductible losses,” but as “Other temporary differences, assets.”
The Company is entitled to additional tax deductions related to share based payments (Warrants, RSUs and PSUs). Tax deductions can be taken when the warrants and RSUs/PSUs are exercised/transferred. For the year ended December 31, 2024, the Company was entitled to additional tax deductions with a tax value of €21.7 million, (Parent company:€7.5 million) compared to €10.6 million (Parent company: €3.4 million) for the year ended December 31, 2023. These future tax deductions depend on the timing and amounts of warrants/RSUs/PSUs exercises/transfers, and accordingly, future additional tax deductions are subject to uncertainties. Refer to Note 7, “Share-based Payment,” regarding a description of warrant and RSU/PSU programs.
The Company is entitled to additional tax deductions for which deferred tax asset cannot be recognized due the initial recognition exception in IAS 12, “Income Taxes.” For the year ended December 31, 2024, the Company is entitled to additional future tax deduction with a tax value of €11.0 million (parent € 2.5 million), compared to €0.3 million (parent € 0.3 million) for the year ended December 31, 2023. In addition, the Company is entitled to additional tax deductions related to tax credits. Tax credits can be taken in future taxes. For the year ended December 31, 2024, the Company was entitled to additional tax deduction with a tax value of €13.8 million (parent € — million), compared to €8.4 million (parent €— million) for the year ended December 31, 2023.
On May 23, 2023, the IASB issued International Tax Reform—Pillar Two Model Rules—Amendments to IAS 12 which clarify that IAS 12 applies to income taxes arising from tax law enacted or substantively enacted to implement the Pillar Two model rules published by the OECD, including tax law that implements Qualified Domestic Minimum Top-up Taxes. The Company has adopted these amendments; however, they are not applicable for the year ended December 31, 2024, as the Company’s consolidated revenue is currently below the threshold of €750 million.
The Parent Company Ascendis Pharma A/S, is jointly taxed with its Danish subsidiaries. The current Danish corporation tax is allocated between the jointly taxed Danish companies in proportion to their taxable income (full absorption with refunds for tax losses). The jointly taxed companies are included in the on-account tax scheme.
Note 10 – Intangible Assets
| Group | ||||||||||||
| (EUR’000) | Goodwill | Software | Total | |||||||||
| Cost |
||||||||||||
| January 1, 2023 |
3,495 | 2,222 | 5,717 | |||||||||
| Additions |
— | 53 | 53 | |||||||||
| Transferred |
— | 21 | 21 | |||||||||
|
|
|
|
|
|
|
|||||||
| December 31, 2023 |
3,495 | 2,296 | 5,791 | |||||||||
|
|
|
|
|
|
|
|||||||
| Additions |
— | 76 | 76 | |||||||||
|
|
|
|
|
|
|
|||||||
| December 31, 2024 |
3,495 | 2,372 | 5,867 | |||||||||
|
|
|
|
|
|
|
|||||||
| Amortization and impairment |
||||||||||||
| January 1, 2023 |
— | (889 | ) | (889 | ) | |||||||
| Amortization charge |
— | (483 | ) | (483 | ) | |||||||
|
|
|
|
|
|
|
|||||||
| December 31, 2023 |
— | (1,372 | ) | (1,372 | ) | |||||||
|
|
|
|
|
|
|
|||||||
| Amortization charge |
— | (467 | ) | (467 | ) | |||||||
|
|
|
|
|
|
|
|||||||
| December 31, 2024 |
— | (1,839 | ) | (1,839 | ) | |||||||
|
|
|
|
|
|
|
|||||||
| Carrying amount |
||||||||||||
| December 31, 2023 |
3,495 | 924 | 4,419 | |||||||||
|
|
|
|
|
|
|
|||||||
| December 31, 2024 |
3,495 | 533 | 4,028 | |||||||||
|
|
|
|
|
|
|
|||||||
| Financial Statements | 70 |
| Parent | ||||||||||||
| Acquired | ||||||||||||
| intellectual | ||||||||||||
| (EUR’000) | Software | property | Total | |||||||||
| Cost |
||||||||||||
| January 1, 2023 |
2,222 | 1,326 | 3,548 | |||||||||
| Additions |
— | — | — | |||||||||
|
|
|
|
|
|
|
|||||||
| December 31, 2023 |
2,222 | 1,326 | 3,548 | |||||||||
|
|
|
|
|
|
|
|||||||
| Additions |
— | — | — | |||||||||
|
|
|
|
|
|
|
|||||||
| December 31, 2024 |
2,222 | 1,326 | 3,548 | |||||||||
|
|
|
|
|
|
|
|||||||
| Amortization and impairment |
||||||||||||
| January 1, 2023 |
(889 | ) | (1,326 | ) | (2,215 | ) | ||||||
| Amortization charge |
(444 | ) | — | (444 | ) | |||||||
|
|
|
|
|
|
|
|||||||
| December 31, 2023 |
(1,333 | ) | (1,326 | ) | (2,659 | ) | ||||||
|
|
|
|
|
|
|
|||||||
| Amortization charge |
(445 | ) | — | (445 | ) | |||||||
|
|
|
|
|
|
|
|||||||
| December 31, 2024 |
(1,778 | ) | (1,326 | ) | (3,104 | ) | ||||||
|
|
|
|
|
|
|
|||||||
| Carrying amount |
||||||||||||
| December 31, 2023 |
889 | — | 889 | |||||||||
|
|
|
|
|
|
|
|||||||
| December 31, 2024 |
444 | — | 444 | |||||||||
|
|
|
|
|
|
|
|||||||
At the reporting date, no internally generated intangible assets from development of pharmaceutical drug candidates have been recognized. Thus, all related research and development costs incurred for the years ended December 31, 2024, and 2023, were recognized in the statements of profit or loss.
Goodwill relates to the acquisition of Complex Biosystems GmbH (now Ascendis Pharma GmbH) in 2007. Goodwill was calculated as the excess amount of the purchase price to the fair value of identifiable assets acquired, and liabilities assumed at the acquisition date. Ascendis Pharma GmbH was initially a separate technology platform company but is now an integral part of the Company’s research and development activities. Accordingly, it is not possible to look separately at Ascendis Pharma GmbH when considering the recoverable amount of the goodwill. Goodwill is monitored and tested for impairment on a consolidated level as the Company is considered to represent one cash-generating unit.
The recoverable amount of the cash-generating unit is determined based on an estimation of the Company’s fair value less costs of disposal. The fair value of goodwill has been determined after taking into account the market value of the Company’s ADSs as of the reporting date. The computation of the market value including an estimation of selling costs, significantly exceeded the carrying amount of the net assets, leaving sufficient value to cover the carrying amount of goodwill. Considering the excess value, no further assumptions are deemed relevant to be applied in determining whether goodwill is impaired.
| Financial Statements | 71 |
Note 11 – Property, Plant and Equipment
| Group | ||||||||||||||||||||
| Leasehold | Right-of- | |||||||||||||||||||
| Plant and | Other | Improve- | Use | |||||||||||||||||
| (EUR’000) | Machinery | Equipment | ments | Assets | Total | |||||||||||||||
| Cost |
||||||||||||||||||||
| January 1, 2023 |
24,944 | 11,203 | 20,130 | 119,466 | 175,743 | |||||||||||||||
| Additions |
2,580 | 503 | 228 | 7,547 | 10,858 | |||||||||||||||
| Disposals |
(383 | ) | (57 | ) | — | — | (440 | ) | ||||||||||||
| Transferred |
504 | (21 | ) | (504 | ) | — | (21 | ) | ||||||||||||
| Foreign exchange translation |
(209 | ) | (208 | ) | (479 | ) | (3,093 | ) | (3,989 | ) | ||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| December 31, 2023 |
27,436 | 11,420 | 19,375 | 123,920 | 182,151 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| Additions |
299 | 951 | 76 | 861 | 2,187 | |||||||||||||||
| Disposals |
(5,995 | ) | (1,635 | ) | — | (89 | ) | (7,719 | ) | |||||||||||
| Transferred |
66 | (66 | ) | — | — | — | ||||||||||||||
| Foreign exchange translation |
127 | 306 | 847 | 5,462 | 6,742 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| December 31, 2024 |
21,933 | 10,976 | 20,298 | 130,154 | 183,361 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| Depreciation and impairment |
||||||||||||||||||||
| January 1, 2023 |
(7,584 | ) | (5,023 | ) | (4,497 | ) | (29,544 | ) | (46,648 | ) | ||||||||||
| Depreciation charge |
(2,569 | ) | (1,899 | ) | (2,085 | ) | (11,875 | ) | (18,428 | ) | ||||||||||
| Impairment charge |
(2,869 | ) | (405 | ) | (4,560 | ) | — | (7,834 | ) | |||||||||||
| Disposals |
146 | 54 | — | — | 200 | |||||||||||||||
| Foreign exchange translation |
92 | 98 | 196 | 807 | 1,193 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| December 31, 2023 |
(12,784 | ) | (7,175 | ) | (10,946 | ) | (40,612 | ) | (71,517 | ) | ||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| Depreciation charge |
(2,330 | ) | (1,322 | ) | (1,283 | ) | (12,312 | ) | (17,247 | ) | ||||||||||
| Disposals |
5,296 | 1,501 | — | 88 | 6,885 | |||||||||||||||
| Foreign exchange translation |
(112 | ) | (194 | ) | (556 | ) | (1,906 | ) | (2,768 | ) | ||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| December 31, 2024 |
(9,930 | ) | (7,190 | ) | (12,785 | ) | (54,742 | ) | (84,647 | ) | ||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| Carrying amount: |
||||||||||||||||||||
| December 31, 2023 |
14,652 | 4,245 | 8,429 | 83,308 | 110,634 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| December 31, 2024 |
12,003 | 3,786 | 7,513 | 75,412 | 98,714 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
The impairment charge for the year ended December 31, 2023, relates to change in activities at one of our R&D sites and is determined according to its estimated value in use. The R&D site is subleased and is recognized as a right-of-use asset with a carrying amount of €11.2 million and €12.5 million as of December 31, 2024 and 2023, respectively.
Depreciation charges are specified below:
| Group | ||||||||
| (EUR’000) | 2024 | 2023 | ||||||
| Depreciation charges |
||||||||
| Cost of sales(1) |
3,197 | 2,509 | ||||||
| Research and development costs |
7,453 | 10,296 | ||||||
| Selling, general and administrative expenses |
6,597 | 5,623 | ||||||
|
|
|
|
|
|||||
| Total depreciation charges |
17,247 | 18,428 | ||||||
|
|
|
|
|
|||||
| (1) | Includes depreciation charges capitalized as part of inventories. |
| Financial Statements | 72 |
| Parent | ||||||||||||||||||||
| Leasehold | Right-of- | |||||||||||||||||||
| Plant and | Other | Improve- | Use | |||||||||||||||||
| (EUR’000) | Machinery | Equipment | ments | Assets | Total | |||||||||||||||
| Cost |
||||||||||||||||||||
| January 1, 2023 |
6,539 | 2,594 | 2,991 | 24,577 | 36,701 | |||||||||||||||
| Additions |
1,191 | — | 39 | 6,107 | 7,337 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| December 31, 2023 |
7,730 | 2,594 | 3,030 | 30,684 | 44,038 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| Additions |
— | 268 | 76 | 49 | 393 | |||||||||||||||
| Disposals |
— | — | — | (88 | ) | (88 | ) | |||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| December 31, 2024 |
7,730 | 2,862 | 3,106 | 30,645 | 44,343 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| Depreciation and impairment |
||||||||||||||||||||
| January 1, 2023 |
(268 | ) | (1,845 | ) | (439 | ) | (8,805 | ) | (11,357 | ) | ||||||||||
| Depreciation charge |
(370 | ) | (314 | ) | (288 | ) | (3,295 | ) | (4,267 | ) | ||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| December 31, 2023 |
(638 | ) | (2,159 | ) | (727 | ) | (12,100 | ) | (15,624 | ) | ||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| Deprecation charge |
(787 | ) | (247 | ) | (296 | ) | (3,588 | ) | (4,918 | ) | ||||||||||
| Disposals |
— | — | — | 88 | 88 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| December 31, 2024 |
(1,425 | ) | (2,406 | ) | (1,023 | ) | (15,600 | ) | (20,454 | ) | ||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| Carrying amount |
||||||||||||||||||||
| December 31, 2023 |
7,092 | 435 | 2,303 | 18,584 | 28,414 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| December 31, 2024 |
6,305 | 456 | 2,083 | 15,045 | 23,889 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
Depreciation charges are specified below:
| Parent | ||||||||
| (EUR’000) | 2024 | 2023 | ||||||
| Depreciation charges |
||||||||
| Cost of sales(1) |
3,197 | 2,509 | ||||||
| Research and development costs |
1,053 | 1,128 | ||||||
| Selling, general and administrative expenses |
668 | 630 | ||||||
|
|
|
|
|
|||||
| Total depreciation charges |
4,918 | 4,267 | ||||||
|
|
|
|
|
|||||
| (1) | Includes depreciation charges capitalized as part of inventories. |
Note 12 – Investments in Associates
The Company’s associates relate to investments in Eyconis (principal place of business; U.S.), and VISEN (principal place of business; China). The Company’s investments in Eyconis and VISEN are accounted for using the equity method in the consolidated financial statements as the Company has determined that it has significant influence over the investments.
In January 2024, the Company announced the formation and launch with Frazier Life Sciences of Eyconis, a separate company created to develop, manufacture, and commercialize TransCon ophthalmology assets globally, together with a $150 million commitment from an investor syndicate that includes Frazier, RA Capital Management, venBio, and HealthQuest Capital. As of December 31, 2024, the Company’s present ownership in Eyconis was 41.6%.
In November 2018, the Company entered into three exclusive license agreements with VISEN for the further development and commercialization of TransCon hGH, TransCon PTH and TransCon CNP in Greater China, and as consideration for the granting of such rights has received a 50.0% ownership of VISEN’s issued and outstanding shares. As of December 31, 2024, the Company’s present ownership in VISEN was 43.9%.
The aggregated profit or loss, total comprehensive income, transactions and outstanding balances with associates as of December 31, 2024 and 2023 were as follows.
| Financial Statements | 73 |
| Group | ||||||||
| (EUR’000) | 2024 | 2023 | ||||||
| Statement of profit or (loss) |
||||||||
| Profit/(loss) for the year from continuing operations |
(59,235 | ) | (41,873 | ) | ||||
|
|
|
|
|
|||||
| Total comprehensive income |
(59,218 | ) | (41,859 | ) | ||||
|
|
|
|
|
|||||
| Transactions and outstanding balances as of December 31 |
||||||||
| Invoicing of goods and services to associates |
18,225 | 15,026 | ||||||
|
|
|
|
|
|||||
| Trade receivables from associates |
1,759 | 991 | ||||||
|
|
|
|
|
|||||
| Contract liabilities |
5,936 | 7,133 | ||||||
|
|
|
|
|
|||||
Note 13 – Inventories
Inventories are specified below:
| Group and Parent | ||||||||
| (EUR’000) | 2024 | 2023 | ||||||
| Inventories |
||||||||
| Raw materials and consumables |
17,596 | 18,566 | ||||||
| Work in progress |
235,688 | 171,030 | ||||||
| Finished goods |
42,325 | 19,335 | ||||||
|
|
|
|
|
|||||
| Total inventories |
295,609 | 208,931 | ||||||
|
|
|
|
|
|||||
Due to production lead time, work in progress includes inventories that are not sellable before more than twelve months after the reporting date.
Inventories were reduced by write-downs of €15.7 million and €22.9 million for the years ended December 31, 2024 and 2023 respectively, which include write-downs on pre-launch inventories.
Note 14 – Contract Liabilities
At December 31, 2024, contract liabilities comprise unsatisfied performance obligations related to delivery of commercial supply under one of the Company’s license agreements. Non-current contract liabilities are expected to be recognized as revenue within 1-2 years.
Revenue recognized from contract liabilities were €1.4 million (Parent Company: €— million) and €13.3 million (Parent Company: €— million) for the years ended December 31, 2024 and 2023, respectively, and primarily related to delivery of clinical supply and research and development services.
Note 15 – Provisions
Development in provisions is specified below:
| Group | Parent | |||||||
| (EUR’000) | 2024 | |||||||
| Provisions |
||||||||
| January 1 |
32,719 | — | ||||||
| Additions related to prior years |
4,684 | — | ||||||
| Net additions for the year |
57,284 | 114 | ||||||
| Foreign exchange translation |
4,462 | — | ||||||
|
|
|
|
|
|||||
| December 31 |
99,149 | 114 | ||||||
|
|
|
|
|
|||||
| Financial Statements | 74 |
Note 16 – Financial Assets and Liabilities
Financial assets and liabilities comprise the following:
| (EUR’000) | Group | Parent | ||||||||||||||
| 2024 | 2023 | 2024 | 2023 | |||||||||||||
| Financial assets by category |
||||||||||||||||
| Trade receivables |
166,280 | 35,874 | 120,643 | — | ||||||||||||
| Receivables from group enterprises |
— | — | 2,020,428 | 1,759,806 | ||||||||||||
| Other receivables (excluding indirect tax receivables) |
3,964 | 3,909 | 3,035 | 3,111 | ||||||||||||
| Marketable securities |
— | 7,275 | — | 7,275 | ||||||||||||
| Cash and cash equivalents |
559,543 | 392,164 | 492,174 | 263,909 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Financial assets measured at amortized cost |
729,787 | 439,222 | 2,636,280 | 2,034,101 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Total financial assets |
729,787 | 439,222 | 2,636,280 | 2,034,101 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Classified in the statement of financial position |
||||||||||||||||
| Non-current assets |
2,317 | 2,127 | 1,993,653 | 1,761,231 | ||||||||||||
| Current assets |
727,470 | 437,095 | 642,627 | 272,870 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Total financial assets |
729,787 | 439,222 | 2,636,280 | 2,034,101 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Financial liabilities by category |
||||||||||||||||
| Borrowings |
||||||||||||||||
| Convertible senior notes |
458,207 | 407,095 | 458,207 | 407,095 | ||||||||||||
| Royalty funding liabilities |
305,379 | 138,377 | — | — | ||||||||||||
| Lease liabilities |
93,030 | 98,793 | 12,396 | 15,187 | ||||||||||||
| Trade payables and accrued expenses |
96,394 | 94,566 | 84,117 | 85,784 | ||||||||||||
| Payables to group enterprises |
— | — | 2,085 | — | ||||||||||||
| Other liabilities (excluding indirect tax and employee related payables) |
311 | — | — | — | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Financial liabilities measured at amortized cost |
953,321 | 738,831 | 556,805 | 508,066 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Derivative liabilities |
150,670 | 143,296 | 150,670 | 143,296 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Financial liabilities measured at fair value through profit or loss |
150,670 | 143,296 | 150,670 | 143,296 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Total financial liabilities |
1,103,991 | 882,127 | 707,475 | 651,362 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Classified in the statement of financial position |
||||||||||||||||
| Non-current liabilities |
365,080 | 222,996 | 9,223 | 12,011 | ||||||||||||
| Current liabilities |
738,911 | 659,131 | 698,252 | 639,351 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Total financial liabilities |
1,103,991 | 882,127 | 707,475 | 651,362 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
Finance income and expenses are specified below:
| (EUR’000) | Group | Parent | ||||||||||||||
| 2024 | 2023 | 2024 | 2023 | |||||||||||||
| Finance income |
||||||||||||||||
| Interest income |
14,361 | 16,857 | 11,812 | 15,096 | ||||||||||||
| Interest income from group enterprises |
— | — | 82,361 | 37,313 | ||||||||||||
| Remeasurement gain of financial liabilities |
11,248 | 14,654 | — | 14,654 | ||||||||||||
| Foreign exchange translation (net) |
— | 12,346 | — | 10,561 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Total finance income |
25,609 | 43,857 | 94,173 | 77,624 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Finance expenses |
||||||||||||||||
| Interest expenses |
65,504 | 44,065 | 36,493 | 34,714 | ||||||||||||
| Interest expenses to group enterprises |
— | — | 13 | — | ||||||||||||
| Remeasurement loss of financial liabilities |
7,374 | — | 7,374 | — | ||||||||||||
| Foreign exchange translation (net) |
27,149 | — | 1,687 | — | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Total finance expenses |
100,027 | 44,065 | 45,567 | 34,714 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
Interest income and interest expenses relate to financial assets and liabilities measured at amortized cost. Net exchange rate gains and losses primarily relate to U.S. Dollar/Euro fluctuations pertaining to the Company’s cash, cash equivalents, marketable securities and borrowings.
| Financial Statements | 75 |
Borrowings
Convertible Senior Notes
In March 2022, the Company issued an aggregate principal amount of $575.0 million of fixed rate 2.25% convertible notes. The net proceeds from the offering of the convertible notes were $557.9 million (€503.3 million), after deducting the initial purchasers’ discounts and commissions, and transaction costs. The convertible notes rank equally in right of payment with all future senior unsecured indebtedness. Unless earlier converted or redeemed, the convertible notes will mature on April 1, 2028.
The convertible notes accrue interest at a rate of 2.25% per annum, payable semi-annually in arrears on April 1 and October 1 of each year, beginning on October 1, 2022. At any time before the close of business on the second scheduled trading day immediately before the maturity date, noteholders may convert their convertible notes at their option into the Company’s ordinary shares represented by ADSs, together, if applicable, with cash in lieu of any fractional ADS, at the then-applicable conversion rate. The initial conversion rate is 6.0118 ADSs per $1,000 principal amount of convertible notes, which represents an initial conversion price of $166.34 per ADS. The conversion rate and conversion price will be subject to customary adjustments upon the occurrence of certain events.
The convertible notes will be optionally redeemable, in whole or in part (subject to certain limitations), at the Company’s option at any time, and from time to time, on or after April 7, 2025, but only if the last reported sale price per ADS exceeds 130% of the conversion price on each of (i) at least 20 trading days, whether or not consecutive, during the 30 consecutive trading days ending on, and including, the trading day immediately before the date the Company sends the related optional redemption notice; and (ii) the trading day immediately before the date the Company sends such notice.
Royalty Funding Liabilities
The Company has entered into capped synthetic royalty funding agreements with Royalty Pharma (the “Purchaser”), which is presented as royalty funding liabilities, and represents the Company’s contractual obligations to pay a predetermined percentage of future commercial revenue until reaching a predetermined multiple of proceeds received, according to the detailed provisions of the synthetic royalty funding agreements.
YORVIPATH Agreement
In September 2024, the Company entered into a $150.0 million capped synthetic royalty funding agreement (the “Royalty Pharma Yorvipath Agreement”) with the Purchaser. The net proceeds were $148.2 million (€134.2 million) after deducting offering expenses.
Under the terms of the Royalty Pharma Yorvipath Agreement, the Company received an upfront payment of $150.0 million (the “Yorvipath Purchase Price”) in exchange for a 3% royalty on net revenue from sales of YORVIPATH in the U.S. (the “Yorvipath Revenue Payments”). The Yorvipath Revenue Payments to the Purchaser will cease upon reaching a multiple of the Yorvipath Purchase Price of 2.0 times, or 1.65 times if the Purchaser receives Yorvipath Revenue Payments in that amount by December 31, 2029.
The Royalty Pharma Yorvipath Agreement includes a buy-out option, which provides the Company with the right to settle all outstanding liabilities at any time by paying a buy-out amount equal to 2.0 times the Yorvipath Purchase Price minus the Yorvipath Revenue Payments paid to the Purchaser as of the effective date of the buy-out notice. However, if the buy-out notice is provided on or prior to September 30, 2028, and the Company has paid the Purchaser, Yorvipath Revenue Payments equal to the Yorvipath Purchase Price as of the date of the buy-out notice, then the buy-out amount is equal to 1.65 times the Yorvipath Purchase Price minus the Yorvipath Revenue Payments paid to the Purchaser as of the effective date of the buy-out notice.
SKYTROFA Agreement
In September 2023, the Company entered into a $150.0 million capped synthetic royalty funding agreement (the “Royalty Pharma Skytrofa Agreement”) with the Purchaser. The net proceeds were $146.3 million (€136.3 million) after deducting offering expenses.
| Financial Statements | 76 |
Under the terms of the Royalty Pharma Skytrofa Agreement, the Company received an upfront payment of $150.0 million (the “Skytrofa Purchase Price”) in exchange for a 9.15% royalty on net revenue from sales of SKYTROFA in the U.S., beginning on January 1, 2025 (the “Skytrofa Revenue Payments”). The Skytrofa Revenue Payments to the Purchaser will cease upon reaching a multiple of the Skytrofa Purchase Price of 1.925 times, or 1.65 times if the Purchaser receives Skytrofa Revenue Payments in that amount by December 31, 2031.
The Royalty Pharma Skytrofa Agreement includes a buy-out option, which provides the Company with the right to settle all outstanding liabilities at any time by paying a buy-out amount equal to 1.925 times the Skytrofa Purchase Price minus the Skytrofa Revenue Payments paid to the Purchaser as of the effective date of the buy-out notice. However, if the buy-out notice is provided on or prior to December 31, 2028, and the Company has paid the Purchaser, Skytrofa Revenue Payments equal to the Skytrofa Purchase Price as of the date of the buy-out notice, then the buy-out amount is equal to 1.65 times the Skytrofa Purchase Price minus the Skytrofa Revenue Payments paid to the Purchaser as of the effective date of the buy-out notice.
Leases
The Company primarily leases office and laboratory facilities. Lease arrangements contain a range of different terms and conditions and are typically entered into for fixed periods. In order to improve flexibility to the Company’s operations, lease arrangements may provide the Company with option to extend the lease or terminate the lease within the enforceable lease term. In the Company’s current lease portfolio, extension and termination options are up to five years, in addition to the non-cancellable periods.
The following expenses related to lease activities were recognized in the statements of profit or loss:
| Group | Parent | |||||||||||||||
| (EUR’000) | 2024 | 2023 | 2024 | 2023 | ||||||||||||
| Lease expenses |
||||||||||||||||
| Depreciation |
12,312 | 11,875 | 3,588 | 3,295 | ||||||||||||
| Short term leases and leases of low value assets |
285 | 353 | 78 | 164 | ||||||||||||
| Lease interest |
3,303 | 3,581 | 376 | 425 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
| Total lease expenses |
15,900 | 15,809 | 4,042 | 3,884 | ||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||
In February 2022, the Company entered into a facility lease in Germany with an enforceable lease term of 15 years, and comprises total lease cash-outflows of €81.1 million. In addition, in December 2024, the Company entered into additional leases in Denmark with enforceable lease terms of 12 years with option to terminate earlier and comprise total lease cash-outflows of €16.2 million if not terminated earlier. The leases are expected to commence in 2025.
| Financial Statements | 77 |
Financing Activities
The development in borrowings related to financing activities is specified below:
| Group | ||||||||||||||||||||||||||||||||
| Cash payments | Non-cash items | |||||||||||||||||||||||||||||||
| (EUR’000) | Beginning of year |
Repayments | Net proceeds |
Additions/ (disposals) |
Remeasure- ments |
Accretion of interest |
Foreign exchange translation |
End of year |
||||||||||||||||||||||||
| Financing activities December 31, 2024 |
||||||||||||||||||||||||||||||||
| Borrowings (excluding lease liabilities) |
545,472 | (11,819 | ) | 134,158 | — | (11,248 | ) | 62,116 | 44,907 | 763,586 | ||||||||||||||||||||||
| Lease liabilities |
98,793 | (14,677 | ) | — | 861 | — | 3,303 | 4,750 | 93,030 | |||||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||||||
| Total financing activities |
644,265 | (26,496 | ) | 134,158 | 861 | (11,248 | ) | 65,419 | 49,657 | 856,616 | ||||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||||||
| December 31, 2023 |
||||||||||||||||||||||||||||||||
| Borrowings (excluding lease liabilities) |
399,186 | (12,054 | ) | 136,256 | — | — | 40,386 | (18,302 | ) | 545,472 | ||||||||||||||||||||||
| Lease liabilities |
109,191 | (14,006 | ) | — | 2,973 | — | 3,581 | (2,946 | ) | 98,793 | ||||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||||||
| Total financing activities |
508,377 | (26,060 | ) | 136,256 | 2,973 | — | 43,967 | (21,248 | ) | 644,265 | ||||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||||||
| Parent | ||||||||||||||||||||||||||||||||
| Cash payments | Non-cash items | |||||||||||||||||||||||||||||||
| (EUR’000) | Beginning of year |
Repayments | Net proceeds |
Additions/ (disposals) |
Remeasure- ments |
Accretion of interest |
Foreign exchange translation |
End of year |
||||||||||||||||||||||||
| Financing activities December 31, 2024 |
||||||||||||||||||||||||||||||||
| Borrowings (excluding lease liabilities) |
407,095 | (11,819 | ) | — | — | — | 36,116 | 26,815 | 458,207 | |||||||||||||||||||||||
| Lease liabilities |
15,187 | (3,216 | ) | — | 49 | — | 376 | — | 12,396 | |||||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||||||
| Total financing activities |
422,282 | (15,035 | ) | — | 49 | — | 36,492 | 26,815 | 470,603 | |||||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||||||
| December 31, 2023 |
||||||||||||||||||||||||||||||||
| Borrowings (excluding lease liabilities) |
399,186 | (12,054 | ) | — | — | — | 34,227 | (14,264 | ) | 407,095 | ||||||||||||||||||||||
| Lease liabilities |
16,312 | (3,085 | ) | — | 1,535 | — | 425 | — | 15,187 | |||||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||||||
| Total financing activities |
415,498 | (15,139 | ) | — | 1,535 | — | 34,652 | (14,264 | ) | 422,282 | ||||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||||||
| Financial Statements | 78 |
Derivative Liabilities
Derivative liabilities relate to the foreign currency conversion option embedded in the convertible notes.
Fair value cannot be measured based on quoted prices in active markets or other observable inputs and accordingly, derivative liabilities are measured by using the Black-Scholes option-pricing model. Fair value of the option is calculated, applying the following assumptions: (1) conversion price; (2) the Company’s share price; (3) maturity of the option; (4) a risk-free interest rate equaling the effective interest rate on a U.S. government bond with the same lifetime as the maturity of the option; (5) no payment of dividends; and (6) an expected volatility using the Company’s share price (49.6% and 50.5% as of December 31, 2024 and December 31, 2023, respectively).
For additional description of fair values, refer to the following section “Fair Value Measurement.”
Sensitivity Analysis
On December 31, 2024, all other inputs and assumptions held constant, a 10% relative increase in volatility, will increase the fair value of derivative liabilities by approximately €15.0 million and indicates a decrease in profit or loss and equity before tax. Similarly, a 10% relative decrease in volatility indicates the opposite impact.
Similarly, on December 31, 2024, all other inputs and assumptions held constant, a 10% increase in the share price, will increase the fair value of derivative liabilities by approximately €30.8 million and indicates a decrease in profit or loss and equity before tax. Similarly, a 10% decrease in the share price indicates the opposite impact.
Fair Value Measurement
Because of the short-term maturity for cash and cash equivalents, receivables and trade payables, their fair value approximate carrying amount. Fair value compared to carrying amount of marketable securities, convertible notes, royalty funding liabilities and derivatives and their level in the fair value hierarchy is summarized in following table, where;
Level 1 inputs are quoted prices (unadjusted) in active markets for identical assets or liabilities that the entity can access at the measurement date;
Level 2 inputs are inputs other than quoted prices included within Level 1 that are observable for the asset or liability, either directly or indirectly; and
Level 3 inputs are unobservable inputs for the asset or liability.
| Group | ||||||||||||||||||||
| 2024 | 2023 | |||||||||||||||||||
| (EUR’000) | Carring Amount |
Fair Value |
Carring Amount |
Fair Value |
Fair Value Level |
|||||||||||||||
| Financial assets |
(1-3 | ) | ||||||||||||||||||
| Marketable securities |
— | — | 7,275 | 7,266 | 1 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Financial assets measured at amortized cost |
— | — | 7,275 | 7,266 | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Financial liabilities |
||||||||||||||||||||
| Convertible senior notes |
458,207 | 438,288 | 407,095 | 385,410 | 3 | |||||||||||||||
| Royalty funding liabilities |
305,379 | 305,673 | 138,377 | 143,975 | 3 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Financial liabilities measured at amortized cost |
763,586 | 743,961 | 545,472 | 529,385 | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Derivative liabilities |
150,670 | 150,670 | 143,296 | 143,296 | 3 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Financial liabilities measured at fair value through profit or loss |
150,670 | 150,670 | 143,296 | 143,296 | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Financial Statements | 79 |
| Parent | ||||||||||||||||||||
| 2024 | 2023 | |||||||||||||||||||
| (EUR’000) | Carring Amount |
Fair Value |
Carring Amount |
Fair Value |
Fair Value Level |
|||||||||||||||
| Financial assets |
(1-3 | ) | ||||||||||||||||||
| Marketable securities |
— | — | 7,275 | 7,266 | 1 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Financial assets measured at amortized cost |
— | — | 7,275 | 7,266 | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Financial liabilities |
||||||||||||||||||||
| Convertible senior notes |
458,207 | 438,288 | 407,095 | 385,410 | 3 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Financial liabilities measured at amortized cost |
458,207 | 438,288 | 407,095 | 385,410 | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Derivative liabilities |
150,670 | 150,670 | 143,296 | 143,296 | 3 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
| Financial liabilities measured at fair value through profit or loss |
150,670 | 150,670 | 143,296 | 143,296 | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|||||||||||||
The following table specifies movements in level 3 fair value measurements:
| Group and Parent | ||||||||
| (EUR’000) | 2024 | 2023 | ||||||
| Derivative liabilities |
||||||||
| January 1 |
143,296 | 157,950 | ||||||
| Remeasurement recognized in financial income or expense |
7,374 | (14,654 | ) | |||||
|
|
|
|
|
|||||
| December 31 |
150,670 | 143,296 | ||||||
|
|
|
|
|
|||||
Note 17 – Financial Risk Management
The Company manages capital to ensure that all group enterprises will be able to continue as a going concern while maximizing the return to shareholders through the optimization of debt and equity balances.
Capital Structure
The Company’s capital structure consists of equity and external debt obtained through issuance of convertible notes and royalty funding liabilities. The Company is not subject to any contractually imposed capital requirements or financial covenants. The capital structure is reviewed on an ongoing basis for the adequacy of the Company’s capital compared to the resources required for carrying out ordinary activities.
Development in the Company’s share capital and treasury shares reserves are described in the following sections. Other equity reserves are described in Note 2, “Summary of Material Accounting Policies.”
Share Capital
The share capital of Ascendis Pharma A/S consists of 60,689,487 fully paid shares at a nominal value of DKK 1, all in the same share class, and which includes 845,887 ordinary shares represented by ADSs held by Ascendis Pharma A/S.
The development in outstanding shares of the Company was as follows:
| (EUR’000) | 2024 | 2023 | 2022 | 2021 | 2020 | |||||||||||||||
| Changes in share capital |
||||||||||||||||||||
| January 1 |
57,707,439 | 57,152,295 | 56,937,682 | 53,750,386 | 47,985,837 | |||||||||||||||
| Increase through cash contributions |
2,982,048 | 555,144 | 214,613 | 3,187,296 | 5,764,549 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| December 31 |
60,689,487 | 57,707,439 | 57,152,295 | 56,937,682 | 53,750,386 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| Financial Statements | 80 |
Treasury Shares Reserve
The development in holding of treasury shares was as follows:
| Nominal value |
Holding | Holding in % of total outstanding shares |
||||||||||
| (EUR’000) | (Number) | |||||||||||
| Treasury shares |
||||||||||||
| January 1, 2023 |
149 | 1,113,152 | ||||||||||
| Transferred under stock incentive programs |
(3 | ) | (20,098 | ) | ||||||||
|
|
|
|
|
|
|
|||||||
| December 31, 2023 |
146 | 1,093,054 | 1.9 | % | ||||||||
|
|
|
|
|
|
|
|||||||
| Transferred under stock incentive programs |
(33 | ) | (247,167 | ) | ||||||||
|
|
|
|
|
|
|
|||||||
| December 31, 2024 |
113 | 845,887 | 1.4 | % | ||||||||
|
|
|
|
|
|
|
|||||||
Financial Risk Management Objectives
The Company regularly monitors the access to domestic and international financial markets, manages the financial risks relating to its operations, and analyzes exposures to risk, including market risk, such as foreign currency risk and interest rate risk, credit risk and liquidity risk.
The Company’s financial risk exposure and risk management policies are described in the following sections.
Market Risk
The Company’s activities expose the group enterprises to the financial risks of changes in foreign currency exchange rates and interest rates. Derivative financial instruments are not applied to manage exposure to such risks.
Foreign Currency Risk Management
The Company is exposed to foreign currency exchange risks arising from various currency exposures, primarily with respect to the U.S. Dollar (“USD”). Foreign currency exchange risks to the USD are unchanged to prior year, and primarily relate to sales and purchases in foreign currencies, convertible notes and royalty funding liabilities, countered by cash and cash equivalents. The exposure from foreign currency exchange risks is managed by maintaining cash positions in the currencies in which the majority of future expenses are denominated, and payments are made from those reserves.
Foreign Currency Sensitivity Analysis
The following table details how a strengthening of the USD against the EUR would impact profit or loss, and equity before tax at the reporting date. A similar weakening of the USD would have the opposite effect. A positive number indicates an increase in profit or loss and equity before tax, while a negative number indicates the opposite. The sensitivity analysis is deemed representative of the inherent foreign currency exchange risk associated with the operations.
| Group | ||||||||||||||||
| Hypothetical impact on consolidated financial statements |
||||||||||||||||
| (EUR’000) | Nominal position |
Increase in foreign currency exchange rate |
Profit/(loss) before tax |
Equity before tax |
||||||||||||
| USD/EUR |
||||||||||||||||
| December 31, 2024 |
(735,064 | ) | 10 | % | (73,506 | ) | (73,506 | ) | ||||||||
| December 31, 2023 |
(369,091 | ) | 10 | % | (36,909 | ) | (36,909 | ) | ||||||||
| Financial Statements | 81 |
| Parent | ||||||||||||||||
| Hypothetical impact on separate financial statements |
||||||||||||||||
| (EUR’000) | Nominal position |
Increase in foreign currency exchange rate |
Profit/(loss) before tax |
Equity before tax |
||||||||||||
| USD/EUR |
||||||||||||||||
| December 31, 2024 |
(374,691 | ) | 10 | % | (37,469 | ) | (37,469 | ) | ||||||||
| December 31, 2023 |
(403,063 | ) | 10 | % | (40,306 | ) | (40,306 | ) | ||||||||
Interest Rate Risk Management
Outstanding convertible notes comprise a 2.25% coupon fixed rate structure. Further, the effective interest rate on royalty funding liabilities is estimated at initial recognition and takes into account anticipated amount and timing of future cash flows, which further depends on future commercial revenue forecasts and the probability of exercising the embedded buy-out option. Material changes to anticipated future cash flows could potentially increase or decrease future interest expense. In addition, the interest rate on lease liabilities is fixed at the lease commencement date.
Future indebtedness, including those related to lease arrangements, if any, may be subject to higher interest rates. In addition, future interest income from interest-bearing bank deposits may fall short of expectations due to changes in interest rates.
Derivative liabilities are measured at fair value through profit or loss. Since the fair value is exposed from the development in interest rates, the profit or loss is exposed to volatility from such development. The effects of interest rate fluctuations are not considered a material risk to the Company’s financial position. Accordingly, no interest sensitivity analysis has been presented.
Credit Risk Management
The Company has adopted an investment policy with the primary purpose of preserving capital, fulfilling liquidity needs and diversifying the risks associated with cash, cash equivalents and marketable securities. This investment policy establishes minimum ratings for institutions with which the Company holds cash and cash equivalents, as well as rating and concentration limits for marketable securities held.
The exposure to credit risk primarily relates to cash and cash equivalents. The credit risk on bank deposits is limited because the counterparties, holding significant deposits, are banks with minimum credit-ratings of A3/A- assigned by international credit-rating agencies. The banks are reviewed on a regular basis and deposits may be transferred during the year to mitigate credit risk. In order to mitigate the concentration of credit risks on bank deposits and to preserve capital, a portion of the bank deposits may be placed into investment grade rated marketable securities. The Company’s investment policy, approved by the Board, only allows investment in marketable securities having investment grade credit-ratings, assigned by international credit-rating agencies. Accordingly, the risk from probability of default is low. On each reporting date, the risk of expected credit loss on bank deposits and marketable securities, if any, including the hypothetical impact arising from the probability of default is considered in conjunction with the expected loss caused by default by banks or securities with similar credit-ratings and attributes. In line with previous periods, this assessment did not reveal a material impairment loss, and accordingly no provision for expected credit loss has been recognized.
At the reporting dates, there are no significant overdue trade receivable balances. As a result, write-down to accommodate expected credit-losses is not deemed material.
Liquidity Risk Management
Historically, the risk of insufficient funds has been addressed through proceeds from sale of the Company’s securities in private and public offerings, through issuance of convertible notes in 2022, and through royalty funding liabilities in 2024 and 2023.
| Financial Statements | 82 |
Liquidity risk is managed by maintaining adequate cash reserves and banking facilities. The risk of shortage of funds is monitored, using a liquidity planning tool, to ensure sufficient funds are available to settle liabilities as they fall due. Besides long term deposits on leases, the Company’s financial assets are recoverable within twelve months after the reporting date.
Maturity Analysis
The following table summarizes maturity analysis (on an undiscounted basis) for non-derivative financial liabilities recognized in the consolidated statements of financial position:
| Group | ||||||||||||||||||||
| (EUR’000) | <1 year | 1-5 years | >5 years | Total contractual cashflows |
Carrying amount |
|||||||||||||||
| Financial liabilities |
||||||||||||||||||||
| December 31, 2024 |
||||||||||||||||||||
| Borrowings (excluding lease liabilities) |
32,303 | 1,027,558 | 13,660 | 1,073,521 | 763,586 | |||||||||||||||
| Lease liabilities |
15,482 | 52,007 | 39,127 | 106,616 | 93,030 | |||||||||||||||
| Trade payables, accrued expenses and other liabilities |
96,705 | — | — | 96,705 | 96,705 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| Total financial liabilities |
144,490 | 1,079,565 | 52,787 | 1,276,842 | 953,321 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| Group | ||||||||||||||||||||
| (EUR’000) | <1 year | 1-5 years | >5 years | Total contractual cashflows |
Carrying amount |
|||||||||||||||
| Financial liabilities |
||||||||||||||||||||
| December 31, 2023 |
||||||||||||||||||||
| Borrowings (excluding lease liabilities) |
11,708 | 742,925 | 42,397 | 797,030 | 545,472 | |||||||||||||||
| Lease liabilities |
14,385 | 51,426 | 49,056 | 114,867 | 98,793 | |||||||||||||||
| Trade payables, accrued expenses and other liabilities |
94,566 | — | — | 94,566 | 94,566 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| Total financial liabilities |
120,659 | 794,351 | 91,453 | 1,006,463 | 738,831 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| Parent | ||||||||||||||||||||
| (EUR’000) | <1 year | 1-5 years | >5 years | Total contractual cashflows |
Carrying amount |
|||||||||||||||
| Financial liabilities |
||||||||||||||||||||
| December 31, 2024 |
||||||||||||||||||||
| Borrowings (excluding lease liabilities) |
12,453 | 584,603 | — | 597,056 | 458,207 | |||||||||||||||
| Lease liabilities |
3,203 | 6,532 | 3,814 | 13,549 | 12,396 | |||||||||||||||
| Payables to group enterprises |
2,085 | — | — | 2,085 | 2,085 | |||||||||||||||
| Trade payables, accrued expenses and other liabilities |
84,117 | — | — | 84,117 | 84,117 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| Total financial liabilities |
101,858 | 591,135 | 3,814 | 696,807 | 556,805 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| Financial Statements | 83 |
| Parent | ||||||||||||||||||||
| (EUR’000) | <1 year | 1-5 years | >5 years | Total contractual cashflows |
Carrying amount |
|||||||||||||||
| Financial liabilities |
||||||||||||||||||||
| December 31, 2023 |
||||||||||||||||||||
| Borrowings (excluding lease liabilities) |
11,708 | 561,340 | — | 573,048 | 407,095 | |||||||||||||||
| Lease liabilities |
3,206 | 8,607 | 4,905 | 16,718 | 15,187 | |||||||||||||||
| Trade payables, accrued expenses and other liabilities |
85,784 | — | — | 85,784 | 85,784 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
| Total financial liabilities |
100,698 | 569,947 | 4,905 | 675,550 | 508,066 | |||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
“Borrowings (excluding lease liabilities)” comprise convertible notes and royalty funding liabilities. Further details regarding classification of convertible notes as current liabilities in the consolidated statement of financial position are provided in Note 2, “Summary of Material Accounting Policies,” section “New and Amended IFRS Accounting Standards and Interpretations.” Expected maturity for royalty funding liabilities is based on anticipated amount and timing of future revenue from sale of commercial products. Further details regarding the payment structure of the royalty funding agreements are provided above.
Note 18 – Commitments and Contingencies
Contractual commitments for the acquisition of property, plant and equipment were €0.3 million and €1.2 million for the years ended December 31, 2024 and 2023, respectively. Further, with certain suppliers, the Company has agreed minimum commitments related to the manufacturing of product supply, subject to continuous negotiation and adjustments according to the individual contractual terms and conditions. Cost of product supply is recognized when the Company obtains control of the goods. In addition, the Company has commitments related to short-term leases and leases of low value assets, contracts of various lengths in respect of research and development with CROs, and IT and facility related services. Costs relating to those commitments are recognized as services are received.
The Company is not aware of any significant legal claims or disputes.
The Parent company is jointly registered for VAT purposes with its Danish subsidiaries and is jointly liable for the payment thereof.
Letter of Support – Parent Company
The Parent Company has provided letters of support to its five wholly-owned subsidiaries Ascendis Pharma Ophthalmology Division A/S, Ascendis Pharma Endocrinology Division A/S, Ascendis Pharma Bone Diseases A/S, Ascendis Pharma Growth Disorders A/S and Ascendis Pharma Oncology Division A/S.
At December 31, 2024, Ascendis Pharma Ophthalmology Division A/S, Ascendis Pharma Endocrinology Division A/S, Ascendis Pharma Bone Diseases A/S, Ascendis Pharma Growth Disorders A/S and Ascendis Pharma Oncology Division A/S reported negative net assets of €31.9 million, €896.8 million, €507.2 million, €498.0 million and €391.5 million, respectively. To support the five companies, the Parent Company has confirmed the technical and financial support that it has committed and further will commit for the period until at least June 30, 2026.
Ascendis Pharma A/S undertakes to make all reasonable technical efforts to support the companies to conduct all pre-clinical, manufacturing, clinical and regulatory activities with their product candidates for the period. In addition, Ascendis Pharma A/S undertakes to provide the companies with the necessary funds to ensure that the companies can conduct their activities for the period in compliance with Danish company regulation and to ensure that the companies can meet their financial obligations as they fall due during the period.
| Financial Statements | 84 |
Applied Exception - Subsidiary
Ascendis Pharma Bone Disease A/S has prepared its statutory financial statements for 2024 pursuant to section 78(a) of the Danish Financial Statements Act, thereby reporting under the requirements for enterprises of reporting class B instead of reporting class C medium.
Note 19 – Related Party Transactions
The Board of Directors, the Executive Board and non-executive Senior Management (“Key Management Personnel”) are considered related parties as they have authority and responsibility for planning and directing the Company’s operations. Related parties also include undertakings in which such individuals have a controlling or joint controlling interest. Additionally, all group enterprises and associates are considered related parties.
Neither the Company’s related parties nor major shareholders hold a controlling, joint controlling, or significant interest in the Group.
The Company has entered into employment agreements with and issued warrants, RSUs and PSUs to Key Management Personnel. In addition, the Company pays fees for board tenure and board committee tenure to the independent members of the Board of Directors. For further details, refer to Note 6, “Employee Costs.” Indemnification agreements have been entered with members of the Board of Directors, the Executive Board and Non-executive Senior Management.
Transactions between the parent company and group enterprises comprise management and license fees, research and development services, administration services and clinical and commercial supplies. These transactions have been eliminated in the consolidated financial statements. Transactions and outstanding balances with the associates are disclosed in Note 12, “Investments in Associates.”
In addition, the parent company Ascendis Pharma A/S is jointly taxed with its Danish subsidiaries, where the current Danish corporation tax is allocated between the jointly taxed Danish companies. For further details, refer to Note 9, “Tax on Profit/(Loss) for the Year and Deferred Tax.”
Except for the information disclosed above, the Company has not undertaken any significant transactions with members of the Key Management Personnel, or undertakings in which the identified related parties have a controlling or joint controlling interest.
Transactions with subsidiaries are specified below:
| Parent | ||||||||
| (EUR’000) | 2024 | 2023 | ||||||
| Rendering of services |
252,443 | 243,002 | ||||||
| Sale of products |
47,540 | 54,710 | ||||||
| Milestone payments |
10,000 | 5,000 | ||||||
|
|
|
|
|
|||||
| Total revenue |
309,983 | 302,712 | ||||||
|
|
|
|
|
|||||
| Milestone payments (expenses) |
— | (100 | ) | |||||
| License expenses |
(100 | ) | (100 | ) | ||||
| Purchase of services |
(28,901 | ) | (56,161 | ) | ||||
|
|
|
|
|
|||||
| Total expenses |
(29,001 | ) | (56,361 | ) | ||||
|
|
|
|
|
|||||
| Interest income |
82,361 | 37,313 | ||||||
| Interest expenses |
(13 | ) | — | |||||
|
|
|
|
|
|||||
| Net financial income |
82,348 | 37,313 | ||||||
|
|
|
|
|
|||||
| Financial Statements | 85 |
Note 20 – Investments in Group Enterprises
Ascendis Pharma A/S’s (parent company) investments in Group enterprises at December 31, 2024, comprise:
| Subsidiaries |
Domicile | Ownership | ||
| Ascendis Pharma GmbH |
Germany | 100% | ||
| Ascendis Pharma Endocrinology GmbH |
Germany | 100% | ||
| Ascendis Pharma, Inc. |
USA | 100% | ||
| Ascendis Pharma Endocrinology, Inc. |
USA | 100% | ||
| Ascendis Pharma Ophthalmology Division A/S |
Denmark | 100% | ||
| Ascendis Pharma Endocrinology Division A/S |
Denmark | 100% | ||
| Ascendis Pharma Bone Diseases A/S |
Denmark | 100% | ||
| Ascendis Pharma Growth Disorders A/S |
Denmark | 100% | ||
| Ascendis Pharma Oncology Division A/S |
Denmark | 100% | ||
| Ascendis Pharma Europe A/S |
Denmark | 100% | ||
| Ascendis Pharma UK Limited |
United Kingdom | 100% | ||
| Ascendis Pharma Iberia S.L. |
Spain | 100% | ||
| Ascendis Pharma France SASU |
France | 100% | ||
| Ascendis Pharma Italia SRL |
Italy | 100% | ||
| Ascendis Pharma Sverige AB |
Sweden | 100% | ||
| Ascendis Pharma Switzerland GmbH |
Switzerland | 100% | ||
| Associate |
Domicile | Ownership | ||
| VISEN Pharmaceuticals |
Cayman Island | 43.9% | ||
| Eyconis, Inc. |
USA | 41.6% | ||
| Financial Statements | 86 |
Note 21 – Ownership
The following investors, or groups of affiliated investors, are known by us to beneficially own more than 5% of the Company’s outstanding ordinary shares at December 31, 2024:
| • | Entities affiliated with RA Capital Management, LLC, USA |
| • | Westfield Capital Management Company, L.P., USA |
| • | Entities affiliated with FMR LLC, USA |
| • | Avoro Capital Advisors LLC, USA |
| • | Entities affiliated with Artisan Partners LP, USA |
| • | Entities affiliated with Janus Henderson Group plc, United Kingdom |
The Company’s American Depository Shares are held through BNY (Nominees) Limited as nominee, of The Bank of New York Mellon, UK (as registered holder of the Company’s outstanding ADSs).
Note 22 – Subsequent Events
No events have occurred after the reporting date that would influence the evaluation of these financial statements.
Exhibit 99.3 Sustainability & P|ESG Report 2024 www.ascendispharma.com
Patients|Environmental, Social and Governance Report (P|ESG) Content Introduction Introduction Progress 03 A message from our Executive Management Team 13 Patients 04 Our Business Model 18 Environmental P|ESG 05 A Brief History 22 Social Approach 07 2024 Sustainability & P|ESG Highlights 28 Governance Ambitions P|ESG Approach Progress 08 Our Sustainability & P|ESG Approach and Reporting 35 2025 Sustainability & P|ESG Ambitions 10 Key Topics 11 P|ESG Risks Ambitions As part of the Ascendis Pharma A/S 2024 Annual Report, this report outlines the Sustainability and what we refer to as Patient, Environmental, Social and Governance (P|ESG) reporting activities for all Ascendis Pharma Group entities. Through this report, we fulfill our compliance with Section 99a (CSR), Section 99b (Gender Diversity) and Section 99d (Data Ethics) of the Danish Financial Statements Act. 2
Introduction P|ESG Approach A message from our Executive Management Team At Ascendis Pharma, we remain dedicated to advancing our TransCon® technology platform as we continue to grow as a biopharmaceutical company with a clear focus on improving patients' lives. Guided by our values and vision, we are determined to fulfill our mission of developing therapies that address unmet medical needs. Progress Our commitment to responsible corporate governance is a key part of our work to deliver safe and effective medicines to those who rely on our products. As we continue to grow, we also recognize that our impact on local and global communities, as well as the environment, is expanding. This ongoing development encourages us to continuously assess and refine our approach to ESG reporting so that Ambitions our P|ESG framework evolves in line with both our growth and the expectations of our stakeholders, and that focus is placed on areas where we can make the most meaningful difference. This report reflects our performance in 2024, as well as our commitment to continue progressing on our P|ESG journey in alignment with current and emerging regulations. We extend our gratitude to all those who have contributed to our efforts and look forward to building on these foundations in the years ahead. 3
Our Business Model Ascendis Pharma was founded in 2007. We are support our research initiatives and clinical France, the UK, and the Nordic countries. Through applying our innovative TransCon platform to trials, and we use Contract Development and exclusive sales and distribution partners covering build a leading, fully integrated, global biopharma Manufacturing Organizations (CDMOs) to more than 50 countries, we now have a global company focused on making a meaningful manufacture finished drug products intended presence and are preparing to support patients difference in patients' lives. Guided by our core for clinical or commercial use. We do not around the world. Introduction values of Patients, Science and Passion, we use manufacture any finished drug products in- our TransCon technologies to create new and house. As a company, we strive to make meaningful potentially best-in-class therapies. improvements in patients' lives. We make We currently have two marketed products, business decisions based on patient needs, and TransCon is a technology platform that uses Skytrofa® and Yorvipath®, and a diversified we do our best every day to realize our products' advanced knowledge of chemistry to overcome portfolio of product candidates in clinical benefits for the patients. We are driven by challenges of developing new therapeutics. It development in endocrinology rare diseases science and data and dedicated to being curious combines known biology with the benefits of and one product candidate in clinical and diligent when innovating, developing and P|ESG prodrug and sustained-release technologies with development in oncology. improving products and processes. Approach the goal of optimizing the therapeutic effect. This could mean enhancing safety, tolerability, efficacy With an expanding global presence, we are Our business model is built on fast, successful and convenience. positioned to reach patients worldwide. In drug development and commercial therapeutic the United States, we have established a synergies. Today, our teams are advancing programs in multifaceted organization to support ongoing endocrinology rare diseases and oncology, and commercialization efforts and serve as a we are also collaborating on the development foundation for future endocrinology rare Progress of product candidates based on TransCon in disease product launches. Similarly, we have other therapeutic areas and markets. We rely expanded our direct presence in Europe by on Contract Research Organizations (CROs) to building integrated organizations, in addition to Germany, in Austria, Switzerland, Spain, Italy, Ambitions 4
2007 A Brief History Ascendis Pharma is established. 2008 TransCon hGH is created. 2009 Transition from pre-clinical to clinical company. 2014 TransCon CNP is created. 2015 U.S. Nasdaq listing. TransCon PTH is created. 2018 Vision 2020 established. Strategic investment and exclusive license agreement with Visen Pharmaceuticals. 2019 Vision 3x3 established. Oncology pipeline announced. 2021 TransCon hGH U.S. approval. Establishment of commercial presence in the U.S. 2022 TransCon hGH EU approval. 2023 TransCon PTH EU approval. Establishment of commercial presence in Germany. 2024 Exclusive license agreement with Teijin Ltd. TransCon PTH US approval. Presence established in Italy, France, Spain, the UK, Austria, Switzerland and the Nordic countries. International sales and distribution partnerships established. Strategic investment and exclusive license agreement with Eyconis, Inc. Novo Nordisk A/S exclusive license agreement.
Global Presence Introduction P|ESG Approach Progress Ambitions Strategic investment and Direct markets Exclusive distribution agreement exclusive license agreement Exclusive license agreement 6
2024 Sustainability & P|ESG Highlights In 2024, we conducted our pilot Double Materiality Assessment and continued improving our methodology in order to align our 2025 reporting with the material impacts, risks and opportunities identified. Introduction Patients Environmental • Launched Yorvipath in Germany and the • Successfully reported on our Scope 1 and 2 emissions data, representing climate U.S. impacts related to on-site energy consumption and purchased electricity and heat. • Launched a Named Patient Program across Europe and International Markets for TransCon PTH. P|ESG Social Approach • Established collaborative relationships with Patient Advocacy groups. • Successfully hired and onboarded 304 employees. • Contracted six new sales and distribution • Continued focus on the attraction and retention of talent. partners covering 50 countries. • Ascendis Employeeship sessions focusing on ownership and collaboration were • Continued activities for Skytrofa conducted throughout the year to emphasize individual self-development and team registries in the United States and performance. initiated support to German registry Progress INSIGHTS-GHT. • Launched the Ascendis Care umbrella brand in Germany for all our patient- Governance centric initiatives, including support materials for patients. • Carried out due diligence on sales and distribution partners. • Launched the Patient Support Program in • Implemented various policies and procedures to support future commercial launches. Germany and Austria for patients under Ambitions • Established an approved supplier list for biological matrices to support high welfare Yorvipath treatment simultaneous to standards for animals used to generate these. market launch. • Signed the Marseille Declaration on the worldwide implementation of high standards • Accepted over 60 participating sites for animals housed and used internally and externally by the industry for scientific across the United States for the U.S. purposes to maintain continuous focus on raising the standards for the welfare of Expanded Access Program for TransCon animals internally, and externally through collaboration with the other signatories. PTH. 7
Our Sustainability & P|ESG Approach Our 2024 Sustainability & P|ESG reporting focuses on four key areas: Patients, Environmental, Social and Governance. It is designed to ensure our environmental and social license to operate through responsible governance practices, ultimately benefiting the business and the patients. Introduction our ongoing commitment to improvement. on the interests of various stakeholders About this Report While some ambitions were not fully met, this and other relevant definitions, which are often The Ascendis Pharma 2024 Sustainability & report emphasizes our focus on transparency different than the concept for U.S. federal P|ESG Report reflects our ongoing commitment and continuous progress in adapting to the securities law purposes. Moreover, certain P|ESG to transparency and accountability as we work ever-changing landscape of our business. We information is based on methodologies or data towards aligning with emerging regulations, appreciate your understanding as we continue that are complex and continue to evolve and thus including the European Union's Corporate to navigate our journey toward corporate are subject to uncertainty or change over time. Sustainability Reporting Directive (CSRD) and responsibility. P|ESG meeting the expectations of the users of this Approach Method & Disclaimer report and our affected stakeholders. Please note that while some topics discussed This report, included in the Ascendis Pharma A/S in this report are significant, their importance 2024 Annual Report, covers the Sustainability To this end, this year we conducted a pilot should not be interpreted as rising to the level & P|ESG activities across all Ascendis Pharma Double Materiality Assessment (DMA) where we of materiality as defined under U.S. federal Group entities for the year 2024. We use this identified and assessed our potentially material securities laws and regulations. The concept report to comply with Section 99a (CSR), Section impacts, risks and opportunities. The full list of of materiality used in this report, including any 99b (Gender Diversity) and Section 99d (Data topics is not reflected in this report, but we will references to material or materiality , is based Progress Ethics) of the Danish Financial Statements Act. continue improving our methodology to complete our 2025 DMA and report accurately on the topics most material to Ascendis Pharma and our stakeholders. Vision & Values Patients Through careful due diligence of our business, Patient & Caregiver this report highlights our key P|ESG topics, Clinical Trial Diversity Product Safety Access & Affordability Engagement including the key risks we face, as well as Environmental Social Governance our policies, processes and performance. It Ambitions • Climate Impact• Employee Engagement and • Business Conduct & Ethics outlines forward-looking goals across Patients, Development • Partners & Suppliers• Anti-Corruption & -Bribery • Health & Safety Environmental Stewardship, Social Impact, and • Responsible Marketing • Diversity, Equity & Inclusion • Data Governance Responsible Corporate Governance. • People in Value Chain • Animal Welfare • Human Rights Each area includes updates on our progress Due Diligence, Double Materiality toward the ambitions we set last year, reflecting Reporting & Communication 8
P|ESG Governance Board of Directors Sustainability and P|ESG Reporting at Ascendis Pharma is guided by a governance structure that fosters a collaborative and integrated approach Introduction across the organization. The Ethics & Compliance Committee, including members of Executive Management, plays a key role in overseeing Sustainability & P|ESG efforts Audit Committee at a strategic level. With the upcoming CSRD requirements taking P|ESG effect in 2025, the Audit Committee will provide Approach oversight of non-financial reporting processes, including preparedness for future assurance Sustainability requirements. Members of the Executive Ethics & Compliance Committee & Management Team are also engaged in shaping P|ESG Team our approach to identifying and addressing key P|ESG impacts, risks and opportunities. Progress The Sustainability & P|ESG Team drives the agenda across the organization, working collaboratively with relevant business units and subject matter experts to develop and implement ESG Leadership Team the Sustainability & P|ESG Reporting framework. This cross-functional effort supports alignment with organizational priorities and consistent data collection and accurate reporting. Ambitions This governance framework reflects our commitment to advancing Sustainability & P|ESG Business Areas integration throughout Ascendis Pharma while Involved stakeholders from the entire organization preparing for evolving regulatory and stakeholder expectations. 9
Key Topics As part of our ongoing commitment to aligning our reporting with the priorities set out in the Ascendis strategy and by our stakeholders, this year we reassessed our key topics to confirm that they accurately reflect the most pressing issues to both our business and the patients we serve. Improving diversity and inclusion in clinical trials to ensure that the effectiveness and safety of medicines are tested across Clinical T rial Diversity Page 14 various demographic groups. Introduction Maintaining the highest standards in the development, manufacturing and distribution of pharmaceutical products to ensure Product Safety Page 14 their safety, efficacy and consistency. Accecss & Affordability Page 15 Managing the cost of pharmaceutical products and establishing fair pricing strategies while ensuring sustainable operations. Actively involving patients and their caregivers in decision-making processes, clinical trials and healthcare solutions to Patient & Caregiver Engagement Page 15 improve patient-centered care. P|ESG Mitigating the impacts of climate change, including eventually reducing greenhouse gas emissions and promoting Climate Page 19 Approach sustainability in operations and supply chains. Nurturing a workforce that is engaged, skilled and motivated by offering opportunities for professional growth, fostering Employee Engagement & Development Page 23 innovation and promoting a positive workplace culture. Fostering an inclusive workplace culture that values diversity, ensures equal opportunities for all and promotes a sense of Diversity, Equity & Inclusion Page 24 belonging for all employees. Prioritizing the well-being of employees by implementing comprehensive health and safety measures in all aspects of our Progress Health & Safety Page 25 business. Addressing the impact of the operations of Ascendis Pharma on various stakeholders within the value chain, including People in our Value Chain Page 26 suppliers, distributors and local communities. Respecting and upholding fundamental human rights in all business activities and supply chain operations to ensure fair Human Rights Page 26 treatment and ethical conduct. Upholding high standards of ethical behavior and integrity in all aspects of business, including interactions with customers, Business Conduct & Ethics Page 29 competitors and stakeholders. Ambitions Implementing applicable anti-bribery and anti-corruption measures to ensure transparent, ethical and lawful business Anti-Corruption & -Bribery Page 31 practices. Promoting pharmaceutical products in an honest, ethical and responsible manner, adhering to industry standards and Responsible Marketing Page 31 regulations. Ensuring the responsible collection, storage and use of data, while safeguarding the privacy and security of sensitive Data Governance Page 32 information. Animal Welfare Page 33 Ensuring the ethical treatment and welfare of animals involved in research, development and testing processes. 10 Governance Social Environmental Patients
Introduction P|ESG Risks As part of our business model, we are exposed to various environmental, social and governance (ESG) risks. Effectively P|ESG managing these risks, particularly those that could impact our Approach operations, helps us minimize business disruptions and protect the reputation of Ascendis Pharma. For each of these risk areas, we carefully assess their potential impact and work to define appropriate mitigation strategies. In identifying our priority P|ESG risk areas, we align with our Progress Enterprise Risk Management (ERM) framework. Ambitions 11
Risk Description Risk Impact Mitigating Actions At Ascendis Pharma, effective product innovation is central Ineffective innovation management may result in slower We foster product innovation through rigorous research and to our mission of addressing unmet medical needs. A development timelines and reduced availability of vital development efforts. We actively engage with patients, ensuring key risk to our operations and strategic objectives is the treatments, affecting patient outcomes and satisfaction. our innovations are aligned with their best interests, enabling us challenge of managing product innovation effectively. to bring the right solutions to market. Our laboratory operations have the potential to generate Potential negative environmental impacts could result We take steps to comply with applicable environmental Introduction negative environmental impacts. in regulatory non-compliance, environmental damage regulations and standards. In addition, we regularly maintain and harm to our reputation, affecting our credibility as a laboratorial equipment and implement safety plans to minimize responsible corporate citizen. any potential adverse environmental impacts. Our rapid growth relies on attracting and retaining top Inability to attract and retain employees could hinder our We prioritize employee development and well-being, cultivating a talent. The competitive talent landscape poses a risk to our capacity for innovation and our ability to meet strategic dynamic, inclusive workplace. We emphasize equal opportunities, ability to continue scaling with the necessary expertise. objectives, affecting our growth trajectory. talent retention through career development programs and reward employee contributions with responsibilities and advancement opportunities. We moreover offer competitive P|ESG compensation packages, including both short- and long-term Approach incentives. Employee misconduct, such as failure to adhere to Unethical or illegal behavior by employees could lead to We focus on educating all employees about our internal and applicable legislation or ethical codes, could result in legal, civil or criminal penalties, industry sanctions, and damage external business conduct requirements through comprehensive financial and reputational risks for Ascendis Pharma. to our reputation, impacting both Ascendis Pharma and onboarding and ongoing training in our Code of Business those associated with our organization. Conduct & Ethics. Employees with higher risk exposure due to their work responsibilities undergo specialized training to ensure ethical conduct is maintained across the organization. Progress A key risk involves instances of corruption and bribery, Improper marketing practices could lead to legal, We provide all employees training on interactions with HCPs such as improper gifts, interactions or payments to regulatory and reputational consequences, potentially as part of the Code of Business Conduct & Ethics training. Healthcare Professionals (HCPs), Healthcare Organizations jeopardizing our relationships with HCPs, HCOs and Specialized training is furthermore provided to employees who (HCOs) and patients. These could be perceived as attempts patients, as well as our ability to engage with these groups interact directly with HCPs, HCOs, government officials, patients to influence decision-making regarding the use or and market our products effectively. and caregivers. Our compliance framework within the commercial recommendation of our products. organization reinforces our commitment to responsible marketing, maintaining high ethical standards in all interactions. A significant portion of our operations is outsourced Potential non-compliance by suppliers and partners could By maintaining compliance requirements within our contractual to Contract Manufacturing Organizations (CMOs) and result in legal, financial and reputational consequences, agreements, we ensure responsible sourcing and comply with Ambitions Contract Research Organizations (CROs). The risk jeopardizing the reliability of our product supply and our applicable requirements. Additionally, our Whistleblower Hotline associated with these collaborations lies in ensuring that standing in the market. provides a platform for employees and partners to report any our suppliers and partners maintain full compliance with illegal or unethical behavior. applicable laws and regulations. Given that all our manufacturing is outsourced, the While our risk level is low due to most suppliers being in To prevent human rights violations, we engage with employees primary risk lies within our supply chain. Ensuring OECD countries, any breach of human rights standards in regular training and awareness programs and maintain an compliance with human rights standards in our external could damage our reputation, negatively affect our license accessible Whistleblower Hotline for employees and business collaborations is crucial for maintaining our ethical to operate and undermine stakeholder trust. partners to report illegal and unethical behavior. standing. 12 Human Supply Responsible Business Employee Attraction Environmental Patients Rights Chain Marketing Conduct and Retention Impact
Introduction Patients P|ESG Approach Progress Ambitions 13
At the core of our decision-making is our difficult due to the small numbers affected by Product Safety unwavering commitment to addressing the rare diseases. However, our work in this area has At Ascendis Pharma, we put patient safety unmet medical needs of patients. To this end, led us to expand recruitment efforts, allowing first. We diligently adhere to applicable health we are dedicated to unlocking the full potential us to reach patients from a variety of social and safety laws to ensure that our products and benefit of our products. Our patient-centric backgrounds, ethnicities and nationalities to Introduction consistently meet high standards of safety, approach is not only central to our mission and further strengthen the outcomes of our clinical efficacy and quality. core values, but also a guiding principle that trials. shapes everything we do. This commitment We are committed to maintaining patient safety drives us to make a positive, lasting impact Because rare disease patients are frequently by closely monitoring and managing any on patients' lives, from developing safe and seen at centralized treatment centers, we provide adverse events associated with our products and effective medicines to ensuring their responsible support, such as travel expense reimbursement, services. To achieve this, we have established commercialization. to make participation more accessible. a comprehensive Global Safety Database for Additionally, we seek to leverage the close P|ESG collecting and processing adverse event data. We The Patients chapter in this report highlights our relationships Ascendis Pharma has with patient Approach analyze aggregated reports and engage in signal dedication to improving patient outcomes and communities to better understand and address detection to proactively identify potential safety covers the following key areas: their needs and priorities, fostering patient- risks. When risks are detected, they undergo centered trial designs. thorough assessment, and we implement • Clinical Trial Diversity appropriate actions to address them, including • Product Safety We are also committed to exploring decentralized notifying healthcare professionals, regulatory • Access & Affordability clinical trials to further enhance inclusivity. bodies and the public as needed. • Patient & Caregiver Engagement Decentralization strategies, like in-home nurse Progress support, direct-to-patient shipping of study Our safety measures include continuous Additionally, we provide insights into our overall medications, and virtual site visits, allow patients monitoring of reported adverse events with business governance and policies. For more to participate from a distance, reducing barriers regular updates and guidance provided as detailed information, we invite you to explore tied to travel and proximity to trial sites. However, necessary. Additionally, we maintain product our Code of Business Conduct & Ethics, which these models introduce risks and compliance safety through thorough investigations and the includes a range of publicly available policies. considerations that we carefully assess to uphold implementation of corrective and preventative patient safety and data integrity. actions. Pharmacovigilance training is provided Clinical Trial Diversity regularly to both internal teams and external Ambitions Lastly, we recognize that the broader challenge We recognize the important role of diversity partners, reinforcing our commitment to safety. of finding trial sites that serve a diverse range and representation in clinical trials, including in of populations is a global issue that calls for the rare disease area, which in itself presents Through these efforts, we work to protect patient collaborative action between industry and unique challenges and opportunities. Recruiting well-being and equip healthcare professionals national regulatory agencies to improve a meaningful diverse patient population can be with accurate, timely information on the safety inclusivity in clinical research. profile of our products. 14
We adhere to the following quality and safety hypoparathyroidism, providing them with We have diligently augmented our resources to policies and procedures: access to potentially life-changing treatment. prepare for the seamless introduction of new In collaboration with the respective health products to the market in the United States and • Good Clinical Practice (GCP) authorities and trial investigators, this program to other countries. Simultaneously, we have • Good Laboratory Practice (GLP) ensured that patients who have participated redoubled our efforts to reduce access-related Introduction • Good Manufacturing Practice (GMP) in our TransCon PTH clinical trial programs obstacles that continue to challenge both patients • Good Distribution Practice (GDP) could continue treatment while we awaited and healthcare providers. • Good Pharmacovigilance Practice (GVP) reimbursement in the respective countries. • Requirements for the development of Patient & Caregiver Engagement combination products With Yorvipath available in the U.S. since Our commitment to improving lives starts with December, the program is now closed. Our truly understanding the journeys of patients, their For further information and specific policies, commitment to this program underscores our families and caregivers. Their lived experiences kindly see our Code of Business Conduct & relentless pursuit of innovative solutions for rare P|ESG provide invaluable insights that shape how Ethics. diseases and our unwavering support for the Approach we develop, deliver and support innovative patients who rely on us. healthcare solutions. To better connect with these Access & Affordability communities, we engage with patient advocacy Name Patient Program At Ascendis Pharma, we are dedicated to groups, convene advisory panels and conduct As per regulatory approvals of Yorvipath, accelerating the availability of our innovative detailed research so that their voices are reflected physicians across the world have expressed medicines to patients. To this end, over the in our work to achieve our mission of developing great interest in having specific identified course of 2024 we have expanded our resources new and potentially best-in-class therapies that patients immediately treated. In these programs to better accommodate physicians' requests to address unmet medical needs. Progress it is the physicians who drive the process to have Yorvipath made available before launch ensure access for their patients together with approval by the competent authorities in the In 2024, we focused further on fostering their respective healthcare systems. We have respective countries. We aim to help facilitate meaningful relationships with advocacy established the logistics to accommodate those removal of access-related obstacles that could organizations by not only listening to their requests where possible. act as barriers for patients and their healthcare needs but also supporting them to amplify their providers, where possible. impact. By cultivating partnerships and enabling TM Ascendis Signature Access Program collaboration across stakeholder groups, we seek Expanded Access Program In the end of 2024, we added Yorvipath to our to address common challenges and advance Ambitions portfolio of supported products in the United patient-centered initiatives. Since the initiation of the Expanded Access States. This milestone reflects our unwavering Program (EAP) for TransCon PTH investigational dedication to expanding patient access and As we continue to launch our commercial therapy in 2022, we have accepted over 60 delivering innovative solutions to meet the needs products in select European countries, we participating sites across the United States. of patients and healthcare providers. are prioritizing the needs of under-served This robust network was dedicated to communities. These efforts are guided by addressing the unmet needs of patients with a commitment to ethical collaboration, 15
transparency and respect for regulatory and The PSP provides patients with guidance on Follow-up on enrolled patients will continue industry standards. Every interaction with effectively and safely using our treatments, for up to an additional five years from the last patient organizations is grounded in a spirit of covering everything from preparation to correct participating patient enrollment. Over the 10- partnership, aimed at driving real-world outcomes injection techniques. It facilitates the accurate year period, we expect that the data collected and creating lasting value for the communities we conduct of the titration phase and provides will increase the understanding of longer-term Introduction serve. continuous support throughout the treatment benefits/risks, potentially contribute to clinical journey. The program offers detailed guidance guidelines and provide value to multiple Looking ahead, we are dedicated to deepening on the effective and safe use of our treatments, stakeholders. our understanding of patient needs and finding covering all aspects from preparation to correct innovative ways to bridge gaps in care. By staying usage techniques. Central to this support are live This year, Ascendis Pharma began support for aligned with our core values of trust, integrity training sessions, which utilize demonstration the INSIGHTS-GHT patient registry, a prospective and collaboration, we will continue to evolve our devices and are supplemented by visual aids such observational study, and the first cross-product approach to making a meaningful difference in as Instructions for Use, Quick Reference Guides registry study on growth hormone therapy P|ESG healthcare. and training videos. worldwide collecting long-term data. It records Approach extensive experience of everyday treatment Patient Support Program We are furthermore in the process of establishing with long-acting growth hormones, in which compliant PSP in other direct markets to the experience with products such as Skytrofa is also Ascendis Pharma has introduced a Patient benefit of the patients. Through such programs, being gathered. The aim is to learn more about Support Program (PSP) in Germany and Austria Ascendis Pharma aims to empower patients the use of somatropin preparations in routine to address medication adherence challenges and caregivers to overcome practical barriers, clinical practice and to collect data on long-term and support optimal treatment outcomes. This enhancing adherence to prescribed treatments for efficacy and safety, including patient-relevant program was created in response to observed Progress better health outcomes and quality of life. outcomes such as quality of life. difficulties patients face in consistently following prescribed medication routines, particularly at the Patient Registry onset of a new treatment. In 2023, Ascendis Pharma initiated recruitment Research highlights that overcoming practical for a non-interventional observational patient barriers is a key factor in adherence, especially registry, SkybriGHt, recruiting via treating in injectable therapies where the complexity physicians pediatric patients that have been of administration can be a significant hurdle prescribed Skytrofa. The purpose of the registry Ambitions for some patients. Our goal is to increase the is to advance the understanding of Skytrofa in a perceived ease and convenience of using our diverse and larger real-world population. treatments to improve adherence and treatment experience. Recruitment of patients to the registry will continue for up to five years from initiation. 16
2024 and 2025 Sustainability and P|ESG Reporting Ambitions 2024 Ambitions Status Progress in 2024 2025 Ambitions Review the material impacts, Conduct a double materiality assessment We conducted a pilot Double Materiality risks and opportunities related Introduction to identify key patient priorities within Ongoing Assessment (DMA) and continued to our patients as part of our the scope of our Sustainability & P|ESG improving our methodology. 2025 DMA. Reporting framework. Monitor the regulatory Launch in Germany and Austria of a Launched the Patient Support Program in landscape and seek input from Patient Support Program for chronic Germany and Austria for patients under industry peers and patient hypoparathyroidism patients to significantly Achieved Yorvipath treatment simultaneous to market advocacy groups on the improve patient support and treatment launch. developing field of population outcomes. diversity in clinical trials with P|ESG Approach specific focus on rare diseases, Launch an Ascendis Pharma umbrella brand including oncology. overarching our patient-centric initiatives Launched the Ascendis Care umbrella to reflect our values and commitment and brand in Germany for all our patient-centric Establish access pathways Achieved emphasize our dedication to prioritizing initiatives, including support materials for to our entire endocrinology patients and their well-being at the heart of patients. portfolio across the world to all our efforts. patients in need. Supported Patient Listening Program, Add innovative technology Progress maintained Expanded Access Program based on TransCon to the Build the reputation of Ascendis Pharma as with support from patient organizations HCPs' armamentarium, a rare disease industry leader by identifying Achieved to ensure access to qualifying patients, establishing it as the gold patient-centric, multi-stakeholder-inclusive developed diseases state awareness standard of care for treating priorities. campaign and provided financial support to rare endocrine diseases. patient organizations. Champion ethical, meaningful and evidence-driven scientific Conducted a Patient Journey workshop in Advance initiatives within the advocacy Ambitions exchange with HCPs, anchored Germany, initiated compliant engagements environment by listening, learning and in our deep knowledge of Achieved with patient organizations and strengthened leveraging insights to uplift rare disease medical science, to improve relationships with existing advocacy communities. outcomes in individuals with groups. rare diseases. 17
Introduction Environmental P|ESG Approach Progress Ambitions 18
At Ascendis Pharma, we recognize the Climate Impact importance of understanding and managing Our commitment to climate responsibility spans our environmental impact. While we do not across all our operations and the spaces we own manufacturing sites, we collaborate closely occupy. Our office and laboratory spaces, which with Contract Development and Manufacturing Introduction are located in leased facilities, are central to our Organizations (CDMOs), logistics providers and sustainability initiatives. Over the past year, we third-party partners to align with environmental have collected our Scope 1 and 2 emissions data, principles and comply with applicable laws, enabling us to better assess our climate impact. industry standards and internal guidelines. Although we do not own or directly manage While we continue to assess our environmental manufacturing sites, we are diligent in footprint, we are working to better understand measuring our impact. We partner with Contract our impacts in order to identify potential P|ESG Development and Manufacturing Organizations opportunities for future improvements. This Approach (CDMOs), suppliers and third-party entities to approach extends to our research partnerships achieve our objectives. with Contract Research Organizations (CROs) and our broader supply chain. We encourage our In 2024, we continued our efforts to collect partners and suppliers to demonstrate a shared greenhouse gas emissions data from our largest commitment to environmental responsibility. CMOs and logistics providers. This project targets the upstream and downstream value chain of As we grow, we strive to explore opportunities commercially approved products, as we work Progress for greater environmental responsibility across towards disclosing applicable Scope 3 emissions. our supply chain and business activities, while adhering to applicable regulatory standards. Our ongoing efforts in climate responsibility focus on improving data robustness and strengthening This year's Environmental chapter includes our commitment to regulatory compliance. insights on: • Climate Impact Ambitions • Suppliers & Business Partners For more details, please refer to our full Environmental Policy in our Code of Business Conduct & Ethics. 19
Suppliers Energy Unit 2024 Contract Manufacturing Organizations (CMOs) and logistics providers are Energy Consumption vital to our business model, as we outsource all product manufacturing Total energy consumption MWh 5,780 activities. We expect our suppliers to meet the same standards we set for Introduction ourselves, operating with integrity and adhering to all applicable laws, regulations and the principles outlined in the Ascendis Pharma Code of Scope 1 and 2 GHG Emissions Unit 2024 Business Conduct & Ethics. Scope 1 We have completed the second round of supplier questionnaires. Initially, Gross Scope 1 GHG emissions tCO e 62 2 we launched these questionnaires to gather information from our Scope 2 suppliers. This effort was aimed at enhancing our understanding of their Gross location-based Scope 2 GHG emissions tCO e 1,047 2 practices and their impact on the environment. The insights gained from Gross market-based Scope 2 GHG emissions tCO e 672 2 P|ESG these questionnaires have been invaluable and will serve as the foundation Approach for more extensive assessments in the coming years. The second round Total Scope 1 and 2 Total Scope 1 and 2 emissions (location-based) tCO e 1,110 2 has allowed us to delve deeper into specific areas, fostering a more Total Scope 1 and 2 emissions (market-based) tCO e 734 2 comprehensive evaluation of our suppliers' sustainability practices in alignment with CSRD reporting requirements. Accounting principles As we assess potential suppliers, we consider a range of factors, Energy Consumption Energy consumption for our operations is measured based on the usage of electricity, heat, including their approach to ESG. Our key focus remains on deepening our cooling and fuel, with data derived from meter readings and invoices. For new office spaces with understanding of the environmental impacts, risks and opportunities in our Progress leases starting in 2024, we estimated energy consumption by extrapolating the energy usage per commercial supply chain, particularly regarding climate, water, waste and square meter from our Danish office sites, assuming similar usage patterns. For some German office sites, where heating and cooling consumption data was limited, we applied a similar resource management. extrapolation method based on the usage patterns of our German office sites with complete data. Scope 1 Scope 1 emissions comprise direct CO e emissions from sources that are controlled by Ascendis 2 Pharma. Scope 2 Scope 2 emissions comprise CO e emissions from purchased electricity, heat and cooling 2 Ambitions calculated using both the Market-based and Location-based methods from the GHG Protocol. Total Scope 1 and 2 The sum of our CO e emissions for Scope 1 and 2, calculated using both the Market-based and 2 the Location-based methods. Total Energy Consumption The sum of our total energy consumption for Scope 1 and 2 across operations. 20
2024 and 2025 Sustainability and P|ESG Reporting Ambitions 2024 Ambitions Status Progress in 2024 2025 Ambitions Validate and report on our Scope 1 & 2 We have successfully reported on our Scope Achieved greenhouse gas emissions data. 1 & 2 emissions data. Introduction Expand the collection of greenhouse gas emissions data throughout our upstream We continue our efforts to collect and downstream commercial value chain Not greenhouse gas emissions data from our and report on 2023 Scope 3 greenhouse achieved Skytrofa supply chain, as we work towards Review the material gas emissions for our commercial supply disclosing Scope 3 emissions as required. environmental impacts, risks chains. and opportunities within our operations and commercial Conduct a double materiality assessment to We conducted a pilot Double Materiality supply chains as part of our P|ESG Approach identify key environmental priorities within Ongoing Assessment (DMA) and continued 2025 DMA. our operations. improving our methodology. As part of our pilot Double Materiality Identify key environmental impacts, risks In Assessment (DMA), we have identified and opportunities in our commercial supply progress environmental impacts and risks in our chains. commercial supply chains. Progress Ambitions 21
Introduction Social P|ESG Approach Progress Ambitions 22
In 2024, we recruited and onboarded more For further information and specific policies, We invite Ascendis Pharma employees to share than 300 new employees worldwide, reflecting kindly see our Code of Business Conduct & Ethics. their feedback through various surveys, including another year of substantial growth at Ascendis onboarding and offboarding surveys, as well as Pharma. In connection with the divestment of Employee Engagement & Development targeted pulse surveys on topics like remote work our ophthalmology pipeline, 30 employees left satisfaction. Introduction With a diverse team of ambitious talents, our Ascendis Pharma to join Eyconis. As a result of company culture remains dynamic and fast- our expansion, we now have more than 1,000 We also believe that maintaining and attracting an paced. This year, we continued to enhance our highly skilled and dedicated employees working engaged workforce rests on our ability to be an global recruitment process to emphasize not together across functions and locations to attractive and fair employer. This year, we began only professional skills but also team dynamics advance our mission. a project emphasizing pay equity across the and cultural alignment, reinforcing our efforts to organization to assess and act upon any potential attract and retain top talent. Driven by science, we are focused on developing, instances of pay inequity. Our initial analysis innovating and refining products and processes is promising, indicating little to no systemic P|ESG In line with our Leadership Principles, Ascendis that make a meaningful difference in patients' variation in our pay policies towards our female Approach Pharma prioritizes building leadership capabilities lives. Our employees are central to achieving our and male employees. This is only the beginning, across all organizational levels, recognizing this goals, and we prioritize attracting, onboarding however, as we continue our investigation and as key to creating a motivated and engaged and retaining the right talent to support our efforts in the coming year to promote a clear, workforce. To drive this, we continuously conduct ambitions. objective and transparent pay approach. dedicated leadership training for our leaders across the organization, and we continue to We are also committed to conducting our Lastly, at the discretion of our Board of Directors implement our Let's Talk framework, which business with respect for all individuals and upon management's recommendation, promotes quarterly, in-depth conversations Progress connected to Ascendis Pharma, including those employees are eligible to participate in our short- between managers and employees on critical throughout our value chain. We expect our term and long-term incentive programs. topics such as Impact, Growth, Well-Being, and suppliers and business partners to uphold the Collaboration—all of which we view as essential same standards outlined in our Respecting People to employee development and retention. Policy. Furthermore, we have introduced Ascendis Employeeship , a training initiative designed to In this year's report, the Social chapter support employees in taking ownership of their encompasses the following topics: work situation and enhancing collaboration to Ambitions increase impact across the organization. With this • Employee Engagement & Development initiative, we aim to provide all our people with an • Health & Safety opportunity to develop themselves - and, in turn, • Corporate Diversity, Equity & Inclusion Ascendis Pharma. • People in our Value Chain • Human Rights 23
strives for an equal representation of gender, Corporate Diversity, Equal Opportunity Corporate Diversity, Equity & Inclusion with an acceptable range of 40/60 split to either and Non-Discrimination Policy We are committed to building an inclusive gender in compliance with the guidelines issued workplace where every individual can thrive by the Danish Business Authority. The distribution At Ascendis Pharma, we respect and foster and contribute fully. Diversity, equity and is monitored continuously with a formal biannual Introduction corporate diversity and inclusion and we take inclusion are fundamental principles within our evaluation, so that new initiatives can be pride in being an equal opportunity workplace. organization, woven into our culture, policies and discussed and initiated if necessary to ensure We believe that corporate diversity and inclusion daily practices. compliance. As a result of these, we currently among our workforce is critical to our success as a global company. maintain equal gender representation not only Ascendis Pharma is committed to providing at the Board of Directors level but also across all equal opportunities and fair treatment for We are committed to providing equal management levels within our organization. all individuals based strictly on their merits, opportunity and fair treatment to all individuals free from any discrimination based on race, on the basis of merit without discrimination due Aligned with our commitment to being an P|ESG color, religion, national origin, gender identity to race, color, religion, national origin, gender, attractive and fair employer, our reward Approach sexual orientation, age, disability or other or expression, including pregnancy, sexual philosophy focuses on offering a competitive characteristic protected by law. orientation, age, disability, veteran status, or any and equitable compensation structure. other characteristic protected by law. Compensation decisions are guided by role We also prohibit harassment based on these evaluations, individual qualifications, experience, characteristics in any form, whether physical or Focus on diversity is embedded in processes, and performance assessments, promoting fair verbal and whether committed by supervisors, including recruiting, people development, recognition and rewards for our employees. non-supervisory personnel, or non-employees. leadership development and succession planning Harassment may include, but is not limited to, in compliance with applicable guidelines. Progress offensive sexual flirtations, unwanted sexual Reflecting this commitment, we hold a policy advances or propositions, verbal abuse, sexually regarding our Board of Directors, emphasizing or racially degrading words, or the display in the importance of the best qualifications to drive the workplace of sexually suggestive or racially our business. The overall gender diversity in degrading objects or pictures. leadership positions at Ascendis Pharma meets Making Ascendis Pharma a pleasant and the Danish gender diversity requirements. When rewarding place to work, free from any type of defining equal representation, Ascendis Pharma discrimination or harassment, is fundamental. Ambitions Variations may apply to ensure compliance with local legislation. 24
Headcount 2021 2022 2023 2024 Selling, General and Administration* 236 305 354 492 Health and Safety R&D, Commercial Manufacturing 403 492 525 525 At Ascendis Pharma, we prioritize conducting business in a way that protects the health, safety, Total** 639 797 879 1,017 and well-being of our employees, fully adhering Introduction *Selling, General and Administration includes business and corporate development and commercial activities. to all relevant health and safety laws and **All permanent employees, including part-time and excluding temporary employees and student assistants. regulations. Health and safety considerations are a key part of Unit Gender distribution 2021 2022 2023 2024 our daily operations, and we regularly incorporate (%) feedback from both our employees and external Men/ stakeholders to keep our work environment safe Board of Directors 67/33 57/43 60/40 60/40 Women and compliant. P|ESG Men/ Approach Executive Management 67/33 60/40 50/50 56/44 Our local Danish Environment, Health & Safety Women organization (Arbejdsmiljøorganisation) oversees Men/ efforts to maintain and improve workplace safety. Senior Management 68/32 61/39 59/41 63/37 Women Comprised of a Health & Safety Committee and a Health & Safety Group, this team actively Executive Management and Senior Management correspond to Other Management Levels as defined by Section 99b. monitors our processes and makes adjustments as needed to uphold a safe and healthy workplace. Progress Employee turnover 2023 2024 In other countries where we operate, we take Employee turnover ratio 10.9% 14% relevant steps to comply with applicable national and local health and safety requirements, aligning our practices with regional regulations to protect Work-related accidents 2022 2023 2024 our employees globally. Accidents* (total) 20 26 12 Ambitions Accidents resulting in sick-leave/absence 0 1 2 Accidents resulting in loss of life 0 0 0 *An undesired registered event or exposure that gives rise to personal injury. Registered accident data covers permanent, part-time and temporary staff whilst on duty for Ascendis Pharma. The metrics showcased on this page pertain to Group Level data. 25
People in our Value Chain Human Rights Human Rights and Labor Rights Policy As outlined in the Environmental chapter, Our commitment to human rights is grounded in At Ascendis Pharma, we respect human rights our business relies on a network of suppliers internationally recognized standards, including as well as labor rights, and we expect the same responsible for manufacturing our products. We the Universal Declaration of Human Rights from our suppliers and business partners. We Introduction expect these suppliers to adhere to applicable (UNDHR), the International Covenant on Civil and work proactively for a positive and inclusive laws, regulations and principles of integrity in Political Rights (ICCPR) and its second optional work environment that respects the individual their operations. protocol, and the International Covenant on and is free from any form of discrimination Economic, Social, and Cultural Rights (ICESCR). or harassment. In addition, we unequivocally We aim to enhance our understanding of the oppose any form of trafficking in human beings, compulsory labor and child labor. Respecting social impacts, risks and opportunities within Furthermore, we align our commitment with the each employee's integrity and always treating our commercial supply chain. By doing so, we fundamental conventions of the International everyone with respect are key. foster alignment with our broader commitment to Labor Organization (ILO). These conventions are P|ESG ethical practices and uphold high standards in our crucial for protecting the rights of workers and Approach We set high standards on performance collaborations. are integral components of our human rights management, compensation, rewards and approach. development. Our corporate culture is Our focus is on maintaining strong, transparent characterized by our passion and commitment to relationships with suppliers and identifying areas Guided by the UN Guiding Principles, we help people grow. where improvements can align with our values, integrate human rights considerations into our such as compliance with human rights laws, third-party compliance approach. Our objective is We respect the freedom of association and the workplace safety regulations and responsible to ensure that our partners and suppliers uphold effective recognition of the right to collective business practices. This approach reflects applicable human rights standards and principles. bargaining, applicable laws regarding wages, Progress benefits and working hours as well as the our dedication to building a supply chain that promotion of the fair treatment of Ascendis supports ethical and compliant operations. Our Respecting People Policy, including our Pharma employees as well as those in our value comprehensive Human Rights Policy, can be chain. accessed in our Code of Business Conduct & Ethics. This policy reaffirms our commitment to human rights and serves as a key resource for our workforce and all those engaged with our organization. Ambitions 26
2024 and 2025 Sustainability and P|ESG Reporting Ambitions 2024 Ambitions Status Progress in 2024 2025 Ambitions Continued to enhance our global recruitment Introduction process to prioritize not only professional competencies but also team and culture fit. Attraction, onboarding and retention of talent Furthermore, we continue to implement a to ensure we have the right people to deliver on Ongoing structured process for dialogue between our ambitions. employees and managers called Let's Talk which focuses on Impact, Collaboration, Growth and Well-Being. Review the material social impacts, Leadership development is a continued focus risks and opportunities within our area in Ascendis Pharma which we believe is P|ESG operations and commercial supply Approach critical to ensuring a motivated and engaged chains as part of our 2025 DMA. workforce. We provide dedicated training to newly hired or promoted managers. We also Attraction, onboarding and Continuous focus on leadership and conduct continuous leadership training sessions retention of talent to ensure we employeeship development to enable the across the organization and ensure access to have the right people to deliver on Ongoing development, performance and well-being of our dedicated leadership resources for all people our ambitions. people. leaders to support them in their leadership role. Ascendis Employeeship sessions were Continuous focus on leadership also conducted throughout the year focused and employeeship development Progress on ownership and collaboration, emphasizing to enable the development, individual self-development and team performance and well-being of our performance. people. Conduct a double materiality assessment We conducted a pilot Double Materiality Investigation of our pay policies, to identify key social priorities within our Ongoing Assessment (DMA) and continued improving our emphasizing pay equity across the operations. methodology. organization and taking actionable steps towards ensuring a clear, objective and transparent pay As part of our pilot Double Materiality approach. Identify key social risks in our commercial supply Assessment (DMA) we have identified social Achieved Ambitions chains. impacts and risks in our commercial supply chains. Establish and formalize criteria for categorizing the risk level of our CDMOs and conduct due We have assessed risks in our supply chain and In diligence on high-risk CDMOs using self- continue to collect information from our CMOs in progress assessment questionnaires and resources from order to better understand their risk level. the PSCI. 27
Introduction Governance P|ESG Approach Progress Ambitions 28
At Ascendis Pharma, we are committed to associated policies and procedures. Any procedures aligned with these global minimum conducting our business in line with high ethical identified or potential breaches are subject to requirements while incorporating local-specific standards and in accordance with applicable laws, thorough review and investigation, followed by requirements. and Business Conduct & Ethics plays a central appropriate corrective measures. Consequences role in our Sustainability & P|ESG Reporting for employees can range from additional training Oversight of the Compliance Program is led by Introduction framework. to disciplinary actions, including written warnings the Ethics & Compliance Committee, consisting and, if necessary, termination of employment. of Senior Management representatives, including Acting with integrity in everything we do our CEO. In 2024, the Committee held quarterly is paramount to our license to operate as a Business partners are required to comply with meetings to review the general status of the biopharmaceutical company and is one of the the principles of the Code of Business Conduct Compliance Program and to approve proposals enablers of our success. & Ethics. Any identified or potential breaches are aimed at supporting its continuous improvement. subject to thorough review and investigation. In this year's report, the Governance chapter Violations by business partners may result Effective and T ailored Compliance T raining P|ESG encompasses our commitment to integrity, in contract termination and legal actions, as Approach Education, training and re-training are vital covering the following topics: permitted by applicable laws. to cultivating a strong compliance culture. Employees are required to complete various • Business Conduct & Ethics In 2024, the Code of Business Conduct & Ethics compliance training programs tailored to their • Anti-Bribery & -Corruption was reviewed, updated and approved by the roles, addressing specific topics and associated • Responsible Marketing Board of Directors, reflecting our ongoing risks to ensure relevance and effectiveness. • Data Governance commitment to ethical business practices. • Animal welfare In 2024, employees, including new hires, Progress The Ascendis Pharma Compliance Program completed mandatory training in our Code of All specific compliance and ethics policies as well The Compliance Program underpins our Business Conduct & Ethics. The training involved as detailed governance descriptions can be found dedication to maintaining high ethical standards an e-learning and an assessment requiring a in our Code of Business Conduct & Ethics. and ensuring adherence to applicable laws and 100% passing score. For existing employees, this regulations. This Program includes our Code of served as a refresher, while for new employees, it Business Conduct & Ethics Business Conduct & Ethics, the development was part of their onboarding process. The overall The Code of Business Conduct & Ethics outlines and execution of policies and procedures, completion rate for this training was 99%, with our global compliance framework and policies proactive risk identification and mitigation, and the remaining 1% attributed to employees on Ambitions across our entire value chain. It translates our comprehensive training initiatives on various leave or other extenuating circumstances. values into actionable guidelines, ensuring compliance topics. consistent adherence to high standards of In 2025, we will continue enhancing global and business ethics worldwide. The Compliance Program sets out the minimum affiliate-specific policies and training to support requirements throughout the entire Ascendis employees in making ethical decisions that align Employees are required to comply with the Pharma organization. Each affiliate is in the with applicable laws, regulations and industry Code of Business Conduct & Ethics and its process of establishing compliance policies and standards. 29
Speak Up Policy & Whistleblower Hotline Our policy strictly prohibits retaliation against any Whistleblower Hotline Policy employee who, in good faith, seeks assistance or At Ascendis Pharma, we actively encourage reports suspected misconduct. The Whistleblower Hotline offers employees and expect our employees to voice concerns and externals, e.g., former or potential about unethical behavior or potential and actual To uphold our commitment to transparency employees, members of the Board of Directors, Introduction misconduct related to the Code of Business consultants and third parties, the possibility and accountability, we share key insights into Conduct & Ethics, as well as associated policies to report concerns through a safe channel. In the outcomes of the Speak Up Policy and and procedures. the system you can communicate directly with Whistleblower Hotline over the past four years. the investigation team, and it is possible to be These figures underscore our dedication to We recognize that reporting misconduct to a anonymous. creating a safe environment where employees direct manager may not always feel comfortable, can confidently raise concerns without fear of despite our commitment to fostering open and The Whistleblower Hotline is available 24/7, and retribution. serious concerns can be filed as a written report transparent communication. In such instances, via a secure web form or alternatively via a call P|ESG employees are encouraged to reach out Our Whistleblower Hotline can be accessed 24/7 Approach through the secure hotline. confidentially to HR or Compliance. Additionally, through our website. we provide a Whistleblower Hotline, which allows No retaliation of good faith reporters. for anonymous reporting of concerns. The hotline Third-Party Compliance is accessible to anyone who suspects or has Ascendis Pharma does not accept retaliation At Ascendis Pharma, our dedication to knowledge of a potential or actual violation of our against employees who, in good faith, seek help responsible business conduct extends to our Code of Business Conduct & Ethics, applicable or report concerns or violations. Any reprisal partnerships. We expect all partners to uphold laws, regulations, or internal policies. or retaliation against reporters will be subject the same ethical standards that guide our own Progress to disciplinary action, including potential operations. To this end, we perform relevant due termination of employment. diligence on our sales and distribution partners to ensure compliance with our ethical and legal We will protect your confidentiality in alignment Whistleblower Hotline Reports with applicable laws. If an employee knowingly expectations. makes false reports, this is considered a very serious offense and can warrant disciplinary 2024 0 actions. 2023 0 Ambitions 2022 1 2021 2 30
established processes for reviewing external Anti-Corruption & -Bribery Policy Anti-Corruption & -Bribery medical and promotional materials prior to their As part of the Ascendis Pharma Compliance use. In 2024, these processes were updated to The Ascendis Pharma Anti-Corruption Program, all employees undergo annual training & -Bribery Policy prohibits all forms of align with the evolving global and direct market in the Anti-Corruption & Bribery Policy, which corruption, bribery and kickbacks, whether structure and product reach of Ascendis Pharma. Introduction outlines the expected behavior as representatives they involve a government official or a person of the company. Due to the nature of our or company within the private sector, or they Our ongoing commitment supports our marketing business, we have not identified high actual risks are carried out directly or indirectly through activities to deliver up-to-date, equitable, precise, in relation to corruption and bribery. However a third party. Under this policy, employees impartial and comprehensive information about are strictly forbidden from offering, paying employees with the potential of exposure our products, fostering trust and transparency or authorizing payments to influence the to corruption and bribery risks, such as field across all markets. actions of government officials or healthcare personnel, receive specialized training tailored to professionals, seek preferential treatment their specific challenges. Transparent Interactions or express gratitude for favorable actions. P|ESG Approach Similarly, employees cannot solicit or accept Collaborating with healthcare professionals, To address and monitor potential incidents, the any form of payment or valuable item intended healthcare organizations and patients is a Whistleblower Hotline is available for anonymous to sway their responsibilities or express fundamental aspect of our mission to develop reporting of corruption or bribery concerns. In gratitude for acting in a way that improperly innovative technologies and products that benefit 2024, no incidents were reported. benefited that person. patients worldwide. Looking ahead to 2025, we will continue to refine Indirect payments made through third parties At Ascendis Pharma, our interactions with these our policies and training programs to ensure that would otherwise be impermissible if made stakeholders are always guided by legitimate directly by Ascendis Pharma are also strictly alignment with our risk profile as well as the Progress purposes and conducted in full compliance with prohibited. latest requirements across relevant markets. the applicable regulations governing the Ascendis This policy underscores our commitment Pharma entity, the participants and the location of Responsible Marketing to transparency, integrity and adherence to the interaction. Promotional Review & Compliance ethical standards and legal requirements in all interactions. For more details, please refer Responsible marketing is a cornerstone of the We are deeply committed to transparency in all to the Anti-Corruption & -Bribery Policy in our operations of Ascendis Pharma, and it is therefore our interactions, and we report transfers of value Code of Business Conduct & Ethics. important that all information disseminated about provided to healthcare professionals, healthcare Ambitions products with marketing authorization complies organizations and patient organizations with stringent regulatory standards and ethical in accordance with legal and regulatory practices at all times. requirements. Our marketing activities are conducted in alignment with applicable laws, regulations, and internal guidelines. To support this, we have 31
Data Ethics Policy Data Privacy Data Governance With the evolving landscape of data privacy laws Data Ethics We recognize the fact that our utilization of data, and regulations worldwide, we remain committed whether it is personal or non-personal, can As an innovative biopharma company focused to respecting and protecting personal data across introduce potential risks to individuals that fall on patients and science, data is central to our Introduction all areas of our business. As such we continue beyond the scope of existing laws. To mitigate mission to make meaningful improvements in to operate in strict adherence to these laws and these risks, we apply our key data ethical patients' lives. We are committed to handling regulations, while further enhancing our data principles: data ethically, respecting individuals' rights, privacy practices. and upholding societal values while ensuring • We ensure that the economic benefits of our compliance with relevant laws, regulations and work with data do not outbalance ethical Our comprehensive data privacy policies and guidelines. considerations. procedures mandate that both our employees and our business partners adhere to these strict • We only gather, process and retain data With recent advances in artificial intelligence, P|ESG standards. We support the security, accuracy as long as we have a legitimate business we are taking steps to integrate responsible Approach purpose. and transparency of personal data processing AI principles into our data governance. We by establishing clear protocols for collection, • We make sure that data is kept secure and are actively developing an AI framework that storage, use, transfer and disposal. shared in a way where individuals' rights are promotes transparent, ethical and safe use of AI protected. systems in alignment with our commitment to For any third-party data processing, we undertake data ethics. • We only use secure systems and processes thorough assessments to confirm that our when sharing or obtaining data from third business partners are equally committed to parties. Our long-term commitment is to continually safeguarding personal data. We implement Progress enhance our data ethics and AI awareness • We are transparent when we engage with data processing agreements as well as data programs, ensuring employees and partners those who have a legitimate stake in the transfer agreements (if relevant) to enforce data understand our standards when gathering, data we process, and we will inform and, protection, as well as regular monitoring to verify processing and managing data. We stress quality, where relevant, obtain consent from any compliance. integrity, transparency and security in data persons or legal entities. handling. Additionally, our data ethics standards • To the extent we leverage technologies like extend to third parties who work with data on our AI, we do this in a responsible manner. behalf. Ambitions See our full Data Ethics Policy in our Code of Business Conduct & Ethics. 32
Ascendis Pharma further displayed the Animal Welfare commitment to animal welfare by signing Animal studies are required by regulatory the Marseille Declaration on the worldwide authorities and play an important role in the implementation of high standards for animals development of new pharmaceuticals. The housed and used internally and externally by the Introduction Ascendis Pharma Internal Committee for Animal industry for scientific purposes . This declaration Welfare reinforces our continued commitment is a joint commitment statement by the to apply the best possible welfare for animals signatories to prioritize and request high animal used in the development of safe and effective welfare standards from CROs and suppliers. human treatments. Our principles reflect the Furthermore, it is a collaboration to improve 3R framework: Replacement, Reduction and animal welfare and health globally. Refinement. We emphasize the conduct of only necessary and scientifically sound animal studies, With the aim of increasing the company-wide P|ESG minimizing the use of animals in our research awareness of the work of the Committee, the Approach whenever possible. 3R principles and animal welfare initiatives in 2024, our Internal Committee for Animal Welfare All non-clinical studies conducted by Ascendis published articles on the company intranet, Pharma are performed at external Contract prepared training material to be included in the Research Organizations (CROs). These CROs are onboarding of employees and hosted an internal audited regularly for compliance with our high knowledge-sharing session with focus on animal standards for animal welfare in the housing welfare initiatives within the company. and care of experimental animals. In addition, Progress all animal study protocols are reviewed by the internal Committee for Animal Welfare ahead of signing to ensure consistent focus on animal welfare in the conduct of studies. To address animal welfare at suppliers of animal- derived biological matrices to Ascendis Pharma studies, comprehensive questionnaire-based Ambitions audits were conducted in 2024. The purpose was to identify suppliers that adhere to the same high standards of housing and care of animals as our approved CROs do, A vendor short-list of approved suppliers has been generated. 33
2024 and 2025 Sustainability and P|ESG Reporting Ambitions 2024 Ambitions Status Progress in 2024 2025 Ambitions Continuous enhancement of our current third- party compliance approach as well as further In Carried out due diligence on sales and Introduction development of the due diligence process for progress distribution partners. high-risk business partners. Further strengthen our Global Conduct a double materiality assessment, We conducted a pilot Double Materiality Compliance Framework. which will include KPIs to track in relation to our Ongoing Assessment (DMA) and continued improving our governance performance and identify key P|ESG methodology. Review our material governance priorities. impacts, risks and opportunities as part of our 2025 DMA. Ensure local implementation in new markets of Various policies and procedures have been Ongoing our Global Compliance Framework. implemented to support future launches. P|ESG Further strengthen our compliance Approach frameworks in our EU direct Expand animal welfare audits to include Relevant suppliers of biological matrices were markets to prepare for future suppliers of biological materials to ensure that identified across the organization and audited launches. Achieved they comply with the Ascendis Pharma global based on a detailed animal welfare questionnaire animal welfare standards. developed for this purpose. Generate an audit plan of vendors for biological matrices and initiate Establish an approved supplier list for animal Successfully established an approved supplier the conduct of on-site audits of matrices used in-house and at external contract Achieved list for animal matrices used in-house and at these vendors. laboratories. external contract laboratories. Progress Implement use of approved vendor list for biological matrices across Employees across the organization participated organization and at external in external and internal meetings and Increase internal awareness and knowledge of vendors. conferences to increase knowledge of NAMs Novel Approach Methodologies (NAMs) such as Achieved and subsequently initiated processes to identify in silico, in vitro and ex vivo approaches. relevant in silico, in vitro and ex vivo methods for implementation. Ambitions 34
2025 Sustainability & P|ESG Ambitions In 2025, we will further align our Sustainability & P|ESG framework to upcoming regulations by completing our Double Materiality Assessment to identify P|ESG impacts, risks and opportunities in our operations and value chain, and integrating our Sustainability Statement in our Annual Report. Introduction Patients Environmental • Monitor the regulatory • Review the material environmental impacts, risks and opportunities within our commercial landscape and seek input from supply chains, as part of our 2025 DMA. industry peers and patient advocacy groups on the developing field of population P|ESG Social Approach diversity in clinical trials with specific focus on rare diseases, • Attraction, onboarding and retention of talent to ensure we have the right people to deliver on including oncology. our ambitions. • Establish access pathways • Continuous focus on leadership and employeeship development to enable the development, to our entire endocrinology performance and well-being of our people. portfolio across the world to all patients in need. • Investigation of our pay policies, emphasizing pay equity across the organization and taking Progress actionable steps towards ensuring a clear, objective and transparent pay approach. • Add innovative technology based on TransCon to the HCPs' armamentarium, Governance establishing it as the gold standard of care for treating rare endocrine diseases. • Further strengthen our Compliance Program. • Champion ethical, meaningful • Further strengthen our compliance frameworks in our EU direct markets to prepare for future and evidence-driven scientific launches. Ambitions exchange with HCPs, anchored • Generate an audit plan of vendors for biological matrices and initiate the conduct of on-site in our deep knowledge of audits of these vendors. medical science, to improve outcomes in individuals with • Implement use of approved vendor list for biological matrices across organization and at rare diseases. external vendors. 35
This report may contain forward-looking statements concerning our business, operations and financial performance and condition, as well as our plans, ob- jectives and expectations for our business operations and financial performance and condition, including with relation to our sustainability efforts. Any state- Introduction ments contained herein that are not statements of historical facts may be deemed to be forward-look- ing statements. In some cases, you can identify forward-looking statements by terminology such as aim , believe , continue , commit , dedicate , ensure , could , due , estimate , expect , goal , may , objective , plan , potential , positioned , seek , should , target , will , would and other P|ESG similar expressions that are predictions or indicate Approach future events and future trends, or the negative of these terms or other comparable terminology. These forward-looking statements include our plans for 2023 and onwards with respect to our Sustainability and P|ESG strategy and ambitions. These forward-look- ing statements are based on senior management's current expectations, estimates, forecasts and projec- tions about our business and the industry in which Progress we operate and involve known and unknown risks, uncertainties and other factors that are in some cases beyond our control. As a result, any or all of our forward-looking statements in this report may turn out to be inaccurate, perhaps materially so. The for- ward-looking statements speak only as of the date of this report. Except as required by law, we assume no obligation to update or revise these forward-looking Ambitions statements for any reason, even if new information becomes available in the future. Given these risks and uncertainties, you are cautioned not to rely on such forward-looking statements as predictions of future events. 36
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