Ascendis Pharma A/S (ASND) Q3 2021 Earnings Conference Call November 10, 2021 4:30 PM ET Company Participants Scott Smith - Senior Vice President and CFO Jan Mikkelsen - President and CEO Jesper Hoiland - Global Chief Commercial Officer Dr. Dana Pizzuti - Head, Development Operations and CMO Dr. Juha Punnonen - Head, Oncology Conference Call Participants Jessica Fye - J.P. Morgan Michelle Gilson - Canaccord Genuity Josh Schimmer - Evercore Alethia Young - Cantor Joori Park - SVB Leerink Operator Good day an ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington D.C. 20549 FORM 20-F (Mark One) ☐ REGISTRATION STATEMENT PURSUANT TO SECTION 12(b) OR (g) OF THE SECURITIES EXCHANGE ACT OF 1934 OR ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2020 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _______ to _______ OR ☐ SHELL COMPANY REPORT P ...

Ascendis Pharma A/S (ASND) Q3 2020 Earnings Conference Call November 11, 2020 4:30 PM ET Company Participants Scott Smith – Senior Vice President and Chief Financial Officer Jan Mikkelsen – President and Chief Executive Officer Mark Bach – Head-Clinical Development and Medical Affairs for Endocrinology Rare Diseases Jesper Høiland – Global Chief Commercial Officer Dana Pizzuti – Head-Development Operations Conference Call Participants Jessica Fye – JPMorgan Michelle Gilson – Canaccord Genuity Andrew Anido – ...

Ascendis Pharma A/S (ASND) Q2 2020 Earnings Conference Call August 27, 2020 4:30 PM ET Company Participants Scott Smith - CFO & SVP Jan Mikkelsen - President & CEO Dana Pizzuti - SVP, Development Operations Conference Call Participants Michelle Gilson - Canaccord Genuity Joori Park - SVB Leerink Joshua Schimmer - Evercore ISI Jessica Fye - JPMorgan Chase & Co. James Birchenough - Wells Fargo Securities Tazeen Ahmad - Bank of America Merrill Lynch Alethia Young - Cantor Fitzgerald & Co. Trevor Allred - Oppen ...

ascendis pharma Ascendis Pharma A/S Cautionary Note On Forward-Looking Statements This presentation contains forward-looking statements. All statements other than statements of historical facts contained in this presentation, such as statements regarding our future results of operations and financial position, including our business strategy, prospective products, availability of funding, clinical trial results, product approvals and regulatory pathways, collaborations, licensing or other arrangements, the ...

Financial Data and Key Metrics Changes - The company reported a net loss of €63.3 million or €1.32 per share for Q1 2020, compared to a net loss of €53.6 million or €1.24 per share in Q1 2019, indicating an increase in loss year-over-year [22] - Research and development costs increased to €57.5 million in Q1 2020 from €51.3 million in Q1 2019, driven by higher personnel-related costs and oncology area build-out [22] - General and administrative expenses rose to €17.9 million in Q1 2020 from €10.4 million in Q1 2019, primarily due to increased personnel costs [23] Business Line Data and Key Metrics Changes - For TransCon PTH and TransCon CNP, costs remained relatively flat, with higher clinical trial costs offset by lower manufacturing costs [22] - Costs for TransCon growth hormone were lower compared to the same period last year due to reduced payments and activities related to validation batch preparations [22] Market Data and Key Metrics Changes - The company is on track to submit a Biologics License Application (BLA) in the U.S. in Q2 2020 and a Marketing Authorization Application (MAA) in Europe in Q3 2020 for TransCon growth hormone [23] - The company plans to initiate a Phase 3 trial for pediatric growth hormone deficiency in Japan in Q4 2020 and enroll subjects in the foresiGHt Trial for adult growth hormone deficiency [23] Company Strategy and Development Direction - The company aims to build a fully integrated biopharma company with a diverse pipeline of high-value product candidates across multiple therapeutic areas [25] - The Vision 3x3 strategic roadmap focuses on creating sustainable growth through global clinical REITS and pursuing new indications [10] - The company is preparing for the potential launch of TransCon growth hormone and has appointed Jesper Høiland as Global Chief Commercial Officer to strengthen its commercial capabilities [9][10] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in executing strategic goals despite the COVID-19 situation, noting that operations in Denmark and Germany have resumed [8] - The company is engaging with regulatory authorities for next steps, including preparing for an end of Phase 2 meeting for TransCon PTH [19] - Management highlighted the potential of TransCon PTH as a true hormone replacement therapy for hypoparathyroidism, aiming to improve patient quality of life [15][18] Other Important Information - The company has received orphan drug designation in both the U.S. and Europe for TransCon growth hormone, recognizing the need for long-acting growth hormone therapy [14] - The company is advancing multiple oncology programs and plans to submit its first IND for the TransCon TLR 7/8 agonist program in Q4 2020 [20][24] Q&A Session Summary Question: Can you provide insights on the oncology platform and the characteristics of the PLR molecule? - Management emphasized the importance of building a pipeline of oncology products, with plans to advance multiple candidates, including TransCon TLR 7/8 and a bias IL-2 compound [26][28] Question: What feedback are you receiving from investigators on TransCon PTH? - Management reported positive feedback from investigators, noting high retention rates in the trial and the transformative potential of TransCon PTH for patients [31] Question: What are the main barriers to making TransCon growth hormone a commercial success? - Management indicated that Jesper Høiland will provide insights on commercialization strategies during the next earnings call, leveraging his extensive experience in the growth hormone market [52] Question: How is the company managing the impact of COVID-19 on the launch strategy? - Management stated that they are adapting to changing circumstances and are committed to ensuring a successful launch of TransCon growth hormone [60] Question: Can you provide an update on the ACcomplisH trial for TransCon CNP? - Management confirmed that they are on track to initiate a second Phase 2 trial in China and are conducting safety and efficacy meetings for dose escalation [55][68]

[PART I](index=5&type=section&id=PART%20I) [Item 3. Key Information](index=5&type=section&id=Item%203.%20Key%20Information) This section presents selected consolidated financial data for Ascendis Pharma from 2015 to 2019, showing a consistent increase in revenue and research and development costs, leading to growing net losses, and details a comprehensive list of risks related to the company's operations, regulatory environment, intellectual property, and share ownership [A. Selected Financial Data](index=5&type=section&id=A.%20Selected%20Financial%20Data) The company's selected financial data from 2015 to 2019 shows increasing revenue but significantly rising R&D and G&A costs, resulting in growing operating and net losses, with total equity and assets substantially funded by financing activities Selected Consolidated Financial Data (2015-2019) | Indicator (€ thousands) | 2019 | 2018 | 2017 | 2016 | 2015 | | :--- | :--- | :--- | :--- | :--- | :--- | | **Revenue** | 13,375 | 10,581 | 1,530 | 4,606 | 8,118 | | **Research and development costs** | (191,621) | (140,281) | (99,589) | (66,022) | (40,528) | | **General and administrative expenses** | (48,473) | (25,057) | (13,482) | (11,504) | (9,415) | | **Operating profit / (loss)** | (226,719) | (154,757) | (111,541) | (72,920) | (41,825) | | **Net profit / (loss) for the year** | (218,016) | (130,097) | (123,897) | (68,505) | (32,922) | | **Total assets** | 676,732 | 318,968 | 210,979 | 190,071 | 131,774 | | **Total equity** | 597,114 | 280,050 | 187,211 | 176,613 | 120,329 | Selected Consolidated Cash Flow Data (2015-2019) | Indicator (€ thousands) | 2019 | 2018 | 2017 | 2016 | 2015 | | :--- | :--- | :--- | :--- | :--- | :--- | | **Cash flows from / (used in) operating activities** | (175,936) | (138,802) | (95,099) | (60,179) | (43,466) | | **Cash flows from / used in investing activities** | (5,159) | (2,648) | (941) | (672) | (1,039) | | **Cash flows from / (used in) financing activities** | 493,593 | 203,267 | 124,721 | 117,462 | 105,742 | [D. Risk Factors](index=7&type=section&id=D.%20Risk%20Factors) This section outlines significant risks across five main areas: limited operating history and financial condition, business operations, regulatory environment, intellectual property, and share ownership, including product dependency, uncertain approvals, patent issues, and stock volatility [Risks Related to Our Limited Operating History, Financial Condition and Capital Requirements](index=7&type=section&id=Risks%20Related%20to%20Our%20Limited%20Operating%20History%2C%20Financial%20Condition%20and%20Capital%20Requirements) The company's limited operating history, lack of approved products, and history of significant losses make future viability uncertain, requiring substantial additional financing that could dilute existing shareholders - The company has a limited operating history, no approved products, and has incurred significant losses, including a net loss of **€218.0 million** in 2019 and **€130.1 million** in 2018[22](index=22&type=chunk)[23](index=23&type=chunk) - The company has never generated revenue from product sales and its future revenue depends on successfully completing R&D, obtaining regulatory approvals, and commercializing its product candidates[25](index=25&type=chunk) - Substantial additional financing may be required to achieve company goals; as of December 31, 2019, cash and cash equivalents were **€598.1 million**, which is believed to be sufficient for at least the next 12 months based on the current operating plan[28](index=28&type=chunk)[29](index=29&type=chunk) [Risks Related to Our Business](index=11&type=section&id=Risks%20Related%20to%20Our%20Business) The company's success depends heavily on its product candidates, facing uncertain clinical development, regulatory approval, and commercialization, while relying on third parties for trials and manufacturing, competing intensely, and managing potential side effects and global pandemic impacts - The company is substantially dependent on the success of its lead product candidates: **TransCon hGH**, **TransCon PTH**, and **TransCon CNP**[34](index=34&type=chunk) - The company relies on collaboration partners, such as **VISEN Pharmaceuticals** in Greater China, to develop, obtain regulatory approvals for, and commercialize its endocrinology rare disease therapies[49](index=49&type=chunk)[55](index=55&type=chunk) - The company relies on third parties for manufacturing clinical and potential commercial drug supplies, and any disruption could harm the business; key single-source suppliers include Fujifilm, Vetter, and Philips Medisize for components of TransCon hGH[93](index=93&type=chunk)[125](index=125&type=chunk) - The company faces intense competition from major pharmaceutical companies with greater resources; competitors for long-acting growth hormone therapies include Novo Nordisk and OPKO Health (in collaboration with Pfizer)[70](index=70&type=chunk)[71](index=71&type=chunk) - The global pandemic caused by COVID-19 could materially adversely impact business, including potential delays in clinical trial enrollment, site initiation, and supply chain interruptions[136](index=136&type=chunk)[138](index=138&type=chunk) [Risks Related to Government Regulatory and Legal Requirements](index=34&type=section&id=Risks%20Related%20to%20Government%20Regulatory%20and%20Legal%20Requirements) The company faces lengthy, unpredictable regulatory approval processes, ongoing post-approval obligations, potential negative impacts from healthcare law changes on pricing and reimbursement, and complex data privacy compliance risks - The regulatory approval processes of the FDA and EMA are lengthy, time-consuming, and unpredictable; the company has not yet submitted an NDA, BLA, or MAA for any product candidate[139](index=139&type=chunk)[146](index=146&type=chunk) - Even if approved, products will be subject to ongoing regulatory review, including compliance with cGMP for manufacturing, restrictions on promotion, and safety reporting requirements[161](index=161&type=chunk)[162](index=162&type=chunk) - Failure to obtain adequate coverage and reimbursement from third-party payors like Medicare, Medicaid, and private insurers could limit the ability to market products and generate revenue[174](index=174&type=chunk) - Legislative healthcare reforms in the U.S. (such as changes to the ACA) and abroad may increase costs, lengthen review times, and result in lower prices for pharmaceutical products[208](index=208&type=chunk)[212](index=212&type=chunk) - The company is subject to diverse and complex laws regarding data privacy and security, such as GDPR in the EU and HIPAA in the U.S., with non-compliance potentially leading to significant fines and penalties[205](index=205&type=chunk)[206](index=206&type=chunk) [Risks Related to Our Intellectual Property](index=49&type=section&id=Risks%20Related%20to%20Our%20Intellectual%20Property) The company's success relies on protecting its intellectual property through patents and trade secrets, but faces uncertainties in the patent process, potential infringement lawsuits, and risks from changes in patent law - The company's success depends on its ability to protect its intellectual property, including its **TransCon technologies** and product candidates like TransCon hGH, PTH, and CNP[218](index=218&type=chunk) - As of December 31, 2019, the company holds **24 issued patents in the U.S.** and approximately **139 in other jurisdictions**; the patent application process is expensive and uncertain, and issued patents may be challenged or invalidated[220](index=220&type=chunk)[224](index=224&type=chunk) - The company may be sued for infringing on the intellectual property rights of third parties, which could be costly and time-consuming and could block the commercialization of its products[231](index=231&type=chunk) - Changes to patent law in the U.S. (e.g., the Leahy-Smith Act) and other jurisdictions could increase uncertainties and costs, potentially weakening the company's ability to protect its products[241](index=241&type=chunk)[242](index=242&type=chunk) [Risks Related to Our Ordinary Shares and ADSs](index=55&type=section&id=Risks%20Related%20to%20Our%20Ordinary%20Shares%20and%20ADSs) The company's ADS trading price is highly volatile, ADS holders have differing rights under Danish law, principal shareholders exert substantial control, and as a foreign private issuer, the company has less frequent reporting obligations and may face adverse U.S. tax consequences as a PFIC - The trading price of the company's ADSs is subject to **high volatility** due to various factors, including clinical trial outcomes, regulatory news, and market fluctuations[255](index=255&type=chunk) - As of March 1, 2020, senior management, board members, and 5%+ shareholders beneficially own approximately **60.5% of outstanding voting securities**, enabling them to exert significant control over shareholder matters[268](index=268&type=chunk) - As a foreign private issuer, the company is exempt from certain U.S. proxy rules and has less frequent reporting obligations than U.S. domestic companies[274](index=274&type=chunk) - The company may be classified as a **Passive Foreign Investment Company (PFIC)** for U.S. federal income tax purposes, which could result in adverse tax consequences for U.S. investors[283](index=283&type=chunk)[284](index=284&type=chunk) [Item 4. Information on the Company](index=63&type=section&id=Item%204.%20Information%20on%20the%20Company) This section details Ascendis Pharma's history, business strategy, and operations, providing an overview of its TransCon technology and product pipeline in endocrinology and oncology, strategic collaborations, manufacturing strategy, competitive landscape, intellectual property, and government regulations [A. History and Development of the Company](index=63&type=section&id=A.%20History%20and%20Development%20of%20the%20Company) Ascendis Pharma A/S was organized in Denmark in September 2006, commenced operations in December 2007 after acquiring Complex Biosystems GmbH, and has its principal executive offices in Hellerup, Denmark, with ADSs traded on Nasdaq - Ascendis Pharma A/S was organized in Denmark in September 2006 and commenced operations in December 2007 following the acquisition of Complex Biosystems GmbH, which invented the TransCon technologies[293](index=293&type=chunk) [B. Business Overview](index=63&type=section&id=B.%20Business%20Overview) Ascendis Pharma uses its TransCon technology to develop product candidates in rare disease endocrinology and oncology, with lead candidate TransCon hGH advancing towards regulatory submission, and a strategy to expand therapeutic areas through collaborations like VISEN Pharmaceuticals - The company is applying its innovative **TransCon technologies** to build a pipeline of product candidates with potential best-in-class profiles, focusing on rare disease endocrinology and oncology[295](index=295&type=chunk) - The most advanced product candidate, **TransCon hGH**, is a once-weekly treatment for Growth Hormone Deficiency (GHD); following positive Phase 3 results, a Biologics License Application (BLA) is planned for submission to the FDA in Q2 2020, and a Marketing Authorisation Application to the EMA in Q4 2020[295](index=295&type=chunk)[298](index=298&type=chunk) - The clinical pipeline also includes **TransCon PTH** for hypoparathyroidism and **TransCon CNP** for achondroplasia, both of which are in Phase 2 trials[301](index=301&type=chunk)[304](index=304&type=chunk) - In November 2018, the company formed **VISEN Pharmaceuticals** to develop and commercialize its endocrinology therapies in Greater China, for which Ascendis received a **50% ownership stake**[307](index=307&type=chunk) [C. Organizational Structure](index=106&type=section&id=C.%20Organizational%20Structure) Ascendis Pharma A/S operates through wholly-owned subsidiaries in Germany, the United States, and Denmark, with Danish subsidiaries organized by therapeutic or functional division including Ophthalmology, Endocrinology, Bone Diseases, Growth Disorders, and Oncology - The company conducts operations through wholly-owned subsidiaries in Germany (Ascendis Pharma GmbH), the United States (Ascendis Pharma, Inc.), and Denmark[568](index=568&type=chunk) [D. Property, Plant and Equipment](index=106&type=section&id=D.%20Property%2C%20Plant%20and%20Equipment) The company leases all its facilities, including headquarters in Hellerup, Denmark, research facilities in Germany, and office space in California, with a new oncology research laboratory under construction in Redwood City, California, and existing facilities deemed adequate for near-term needs - The company's headquarters are leased in Hellerup, Denmark; it also leases research facilities in Heidelberg, Germany, and office space in Palo Alto, California[569](index=569&type=chunk)[571](index=571&type=chunk)[573](index=573&type=chunk) - A new research laboratory for oncology is being constructed at a leased site in Redwood City, California, with the lease expiring in April 2030[574](index=574&type=chunk) [Item 5. Operating and Financial Review and Prospects](index=106&type=section&id=Item%205.%20Operating%20and%20Financial%20Review%20and%20Prospects) This section analyzes the company's financial performance, highlighting a 26% revenue increase to €13.4 million in 2019, significant rises in R&D and G&A costs leading to a €218.0 million net loss, strong liquidity with €598.1 million in cash from equity offerings, and covers critical accounting policies, market risks, and contractual obligations [A. Operating Results](index=107&type=section&id=A.%20Operating%20Results) For 2019, revenue increased by 26% to €13.4 million due to the Visen collaboration, while R&D costs grew 37% to €191.6 million and G&A expenses rose 93% to €48.5 million, resulting in a net loss of €218.0 million Results of Operations (2017-2019) | (€ thousands) | 2019 | 2018 | 2017 | | :--- | :--- | :--- | :--- | | **Revenue** | 13,375 | 10,581 | 1,530 | | **Research and development costs** | (191,621) | (140,281) | (99,589) | | **General and administrative expenses** | (48,473) | (25,057) | (13,482) | | **Operating profit / (loss)** | (226,719) | (154,757) | (111,541) | | **Net profit / (loss) for the year** | (218,016) | (130,097) | (123,897) | - Revenue increased by **26%** in 2019 to **€13.4 million**, primarily due to revenue recognition related to the investment in and clinical supply sales to Visen[616](index=616&type=chunk) - Research and development costs increased by **37%** in 2019 to **€191.6 million**, mainly due to manufacturing validation batches for TransCon hGH, increased personnel costs, and development in oncology[619](index=619&type=chunk)[621](index=621&type=chunk)[622](index=622&type=chunk) - General and administrative expenses increased by **93%** in 2019 to **€48.5 million**, driven by higher personnel costs, IT expenses, and pre-commercialization activities[626](index=626&type=chunk) [B. Liquidity and Capital Resources](index=119&type=section&id=B.%20Liquidity%20and%20Capital%20Resources) As of December 31, 2019, the company had €598.1 million in cash and no debt, primarily funded by equity offerings including a €480.3 million follow-on offering in March 2019, with existing cash believed sufficient for at least the next 12 months - As of December 31, 2019, the company had cash and cash equivalents of **€598.1 million** and no debt to third parties[675](index=675&type=chunk) - In March 2019, a follow-on public offering of ADSs was completed, raising net proceeds of approximately **€480.3 million** (**$539.4 million**)[28](index=28&type=chunk)[676](index=676&type=chunk)[687](index=687&type=chunk) Summary of Cash Flows (2017-2019) | (€ thousands) | 2019 | 2018 | 2017 | | :--- | :--- | :--- | :--- | | **Cash flows from/(used in) operating activities** | (175,936) | (138,802) | (95,099) | | **Cash flows from/(used in) investing activities** | (5,159) | (2,648) | (941) | | **Cash flows from/(used in) financing activities** | 493,593 | 203,267 | 124,721 | | **Net increase in cash and cash equivalents** | 312,498 | 61,817 | 28,681 | [F. Tabular Disclosure of Contractual Obligations](index=122&type=section&id=F.%20Tabular%20Disclosure%20of%20Contractual%20Obligations) As of December 31, 2019, the company's total contractual obligations are €52.1 million, primarily comprising €43.6 million in lease obligations and €8.5 million for leasehold improvements construction Contractual Obligations as of December 31, 2019 | Contractual Obligations (€ thousands) | Less Than 1 Year | 1 to 3 Years | 3 to 5 Years | More Than 5 Years | Total | | :--- | :--- | :--- | :--- | :--- | :--- | | **Lease Obligations** | 6,191 | 11,153 | 8,603 | 17,605 | 43,552 | | **Construction of leasehold improvements** | 8,523 | — | — | — | 8,523 | | **Total contractual obligations** | **14,714** | **11,153** | **8,603** | **17,605** | **52,075** | [Item 6. Directors, Senior Management and Employees](index=122&type=section&id=Item%206.%20Directors%2C%20Senior%20Management%20and%20Employees) This section details the company's two-tier governance structure, providing biographical information and compensation for directors and senior management, including significant share-based payments, and describes board committees and employee statistics, noting 330 full-time employees with 250 in R&D as of year-end 2019 [A. Directors and Senior Management](index=122&type=section&id=A.%20Directors%20and%20Senior%20Management) The company operates with a two-tier governance structure, including a seven-member Board of Directors for strategic oversight and an Executive Board for day-to-day management, comprising the President & CEO and CFO, supported by an experienced senior management team - The company has a two-tier governance structure with a separate Board of Directors and an Executive Board; the Board of Directors consists of **seven members** divided into two classes for election purposes[696](index=696&type=chunk)[698](index=698&type=chunk) - The Executive Board, responsible for day-to-day management, includes President & CEO Jan Møller Mikkelsen and SVP & CFO Scott T. Smith[707](index=707&type=chunk) [B. Compensation](index=127&type=section&id=B.%20Compensation) In 2019, senior management's aggregate compensation was approximately €21.7 million, including €15.9 million in share-based payments, with non-employee board members receiving fees and warrant grants, and employment agreements outlining notice periods and severance provisions - Aggregate compensation for senior management in 2019 was approximately **€21.7 million**, which included **€15.9 million** in share-based payments[728](index=728&type=chunk) - On December 10, 2019, non-employee board members were each granted **7,500 warrants** with an exercise price of **$108.00 per share**[725](index=725&type=chunk) - The company has a warrant incentive program for employees, consultants, and board members; warrants generally vest monthly over a **48-month period** for employees and have a **ten-year term**[734](index=734&type=chunk)[736](index=736&type=chunk)[739](index=739&type=chunk) [C. Board Practices](index=131&type=section&id=C.%20Board%20Practices) The Board of Directors, responsible for strategic management, consists of seven members, five independent, classified into two-year terms, and has established Audit, Remuneration, and Nominating and Corporate Governance Committees, following Danish home country practices for certain Nasdaq governance requirements - The Board of Directors is responsible for overall and strategic management and consists of **seven members**, with **five determined to be independent**[747](index=747&type=chunk)[758](index=758&type=chunk) - The Board has three committees: **Audit**, **Remuneration**, and **Nominating and Corporate Governance**[759](index=759&type=chunk) - As a foreign private issuer, the company follows certain home country governance practices instead of Nasdaq rules, such as not having a quorum requirement for general shareholder meetings[755](index=755&type=chunk)[763](index=763&type=chunk) [D. Employees](index=134&type=section&id=D.%20Employees) As of December 31, 2019, Ascendis Pharma employed 330 full-time employees, with 250 in research and development and 80 in general and administrative roles, and 121 employees holding advanced degrees - As of December 31, 2019, the company had **330 full-time employees**[763](index=763&type=chunk) - Employee distribution is **250 in research and development** and **80 in general and administrative functions**; **121 employees** hold a Ph.D., M.D., or equivalent degree[763](index=763&type=chunk) [Item 7. Major Shareholders and Related Party Transactions](index=134&type=section&id=Item%207.%20Major%20Shareholders%20and%20Related%20Party%20Transactions) This section discloses major shareholders owning over 5% as of March 1, 2020, including T. Rowe Price Associates, Inc. (16.2%) and FMR LLC (9.9%), and details related party transactions such as employment agreements and R&D/supply agreements with Visen Pharmaceuticals Major Shareholders (as of March 1, 2020) | Name of Beneficial Owner | Percentage of Beneficial Ownership | | :--- | :--- | | T. Rowe Price Associates, Inc. | 16.2% | | Entities affiliated with FMR LLC | 9.9% | | Entities affiliated with RA Capital Management, LLC | 8.7% | | Baker Bros. Advisors LP | 7.1% | | Entities affiliated with OrbiMed Private Investments V, L.P. | 6.9% | - Related party transactions include employment and indemnification agreements with senior management and board members, as well as R&D and clinical supply agreements with its associate, Visen Pharmaceuticals[776](index=776&type=chunk)[777](index=777&type=chunk)[778](index=778&type=chunk) [Item 8. Financial Information](index=138&type=section&id=Item%208.%20Financial%20Information) This section refers to the detailed consolidated financial statements and confirms the company is not currently involved in any material legal proceedings, nor does it plan to pay cash dividends in the foreseeable future, intending to retain earnings for growth - The company is not currently a party to any legal proceedings that are likely to have a material adverse effect on its business[779](index=779&type=chunk) - The company does not plan to pay cash dividends on its ordinary shares in the foreseeable future, intending to retain earnings for growth[780](index=780&type=chunk) [Item 10. Additional Information](index=139&type=section&id=Item%2010.%20Additional%20Information) This section details the company's corporate structure and governance, outlining Board authorizations for share capital increases and warrant issuance, key Articles of Association provisions, a comparison of Danish and Delaware corporate law, and material Danish and U.S. federal income tax considerations for ADS investors [B. Memorandum and Articles of Association](index=140&type=section&id=B.%20Memorandum%20and%20Articles%20of%20Association) The company's articles of association authorize the Board to increase share capital and issue warrants and convertible notes until 2021-2024, establish a classified board with two-year terms and one vote per share, and provide a comparison of Danish and Delaware corporate law regarding director duties, shareholder rights, and anti-takeover measures - The Board of Directors is authorized to increase share capital by up to **9,000,000 shares** without pre-emptive rights and up to **9,000,000 shares** with pre-emptive rights, with both authorizations valid until May 28, 2024[785](index=785&type=chunk) - The Board is also authorized to issue up to **1,237,525 additional warrants** and obtain loans against convertible notes for up to **9,000,000 shares**[785](index=785&type=chunk) - A detailed comparison highlights key differences between Danish and Delaware corporate law, including director duties, shareholder proposal rights, appraisal rights, and anti-takeover provisions[822](index=822&type=chunk) [E. Taxation](index=151&type=section&id=E.%20Taxation) This section outlines tax consequences for investors, noting non-Danish residents face a 27% Danish withholding tax on dividends, potentially reduced to 15% under treaties, while U.S. Holders may treat dividends as qualified income, and the company does not believe it was a PFIC for 2019, though this status is annually re-evaluated - For non-Danish residents, dividends are generally subject to a **27% Danish withholding tax**, which can often be reduced to **15%** under tax treaties, such as the one with the United States[898](index=898&type=chunk)[899](index=899&type=chunk) - For U.S. Holders, dividends are generally included in gross income and may qualify for preferential tax rates as "qualified dividend income," provided certain conditions are met, including that the company is not a PFIC[915](index=915&type=chunk)[916](index=916&type=chunk) - The company does not believe it was a **Passive Foreign Investment Company (PFIC)** for the 2019 taxable year, but its status is subject to annual re-evaluation and could change[921](index=921&type=chunk) [Item 11. Quantitative and Qualitative Disclosures About Market Risk](index=160&type=section&id=Item%2011.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company is exposed to foreign currency exchange rate risks, particularly the U.S. Dollar, managing it by holding cash in corresponding currencies, with a 10% USD strengthening against EUR increasing net profit by €47.8 million, while interest rate and credit risks are considered minimal - The company is exposed to foreign exchange risk, primarily with respect to the U.S. Dollar; as of December 31, 2019, a **10% strengthening of the USD against the EUR** would increase net profit and equity by **€47.8 million**[637](index=637&type=chunk)[638](index=638&type=chunk)[639](index=639&type=chunk) - Interest rate risk is not considered material as the company has no interest-bearing debt; credit risk is managed by holding cash reserves in banks with high credit ratings[640](index=640&type=chunk)[642](index=642&type=chunk) [Item 12. Description of Securities Other than Equity Securities](index=160&type=section&id=Item%2012.%20Description%20of%20Securities%20Other%20than%20Equity%20Securities) This section describes the American Depositary Shares (ADSs), where each ADS represents one ordinary share, administered by The Bank of New York Mellon as depositary, outlining the deposit agreement and a schedule of fees payable by ADS holders for various services - Each American Depositary Share (ADS) represents **one ordinary share** of the company; The Bank of New York Mellon serves as the depositary[940](index=940&type=chunk) ADS Holder Fees | Fee | For | | :--- | :--- | | $5.00 (or less) per 100 ADSs | Issuance or cancellation of ADSs | | $0.05 (or less) per ADS | Any cash distribution | | $0.05 (or less) per ADS per calendar year | Depositary services | [PART II](index=161&type=section&id=PART%20II) [Item 15. Control and Procedures](index=161&type=section&id=Item%2015.%20Control%20and%20Procedures) Management concluded that the company's disclosure controls and procedures were effective as of December 31, 2019, and assessed the internal control over financial reporting as effective based on the COSO framework, which was audited and confirmed by the independent registered public accounting firm - Management concluded that the company's disclosure controls and procedures were **effective** as of December 31, 2019[947](index=947&type=chunk) - Management assessed the internal control over financial reporting based on the COSO framework and concluded it was **effective** as of December 31, 2019; this was audited and confirmed by the independent registered public accounting firm[951](index=951&type=chunk)[952](index=952&type=chunk)[953](index=953&type=chunk) [Item 16. Corporate Governance and Other Matters](index=162&type=section&id=Item%2016.%20Corporate%20Governance%20and%20Other%20Matters) This section covers corporate governance, identifying Lars Holtug as the audit committee financial expert, noting the adoption of a code of business conduct and ethics, detailing principal accountant fees for 2019 where audit fees constituted 99% of €707,000, and explaining the company's adherence to Danish home country practices instead of certain Nasdaq rules [Item 16A. Audit Committee Financial Expert](index=162&type=section&id=Item%2016A.%20Audit%20Committee%20Financial%20Expert) The company's board of directors has determined that Mr. Lars Holtug, an independent director and Audit Committee member, qualifies as an "audit committee financial expert" as defined in Item 16A of Form 20-F - Mr. Lars Holtug is designated as the **"audit committee financial expert"**[956](index=956&type=chunk) [Item 16B. Code of Ethics](index=162&type=section&id=Item%2016B.%20Code%20of%20Ethics) The company has adopted a code of business conduct and ethics applicable to all employees, senior management, and board members, which is available on its website - A code of business conduct and ethics has been adopted and applies to all employees, senior management, and board members[957](index=957&type=chunk) [Item 16C. Principal Accountant Fees and Services](index=163&type=section&id=Item%2016C.%20Principal%20Accountant%20Fees%20and%20Services) For fiscal year 2019, the company's principal accountant billed a total of €707,000, with audit fees comprising 99% (€700,000) and tax fees making up the remaining 1%, and all non-audit services requiring pre-approval from the Audit Committee Principal Accountant Fees (2018-2019) | Fee Type (€ thousands) | 2019 | 2018 | | :--- | :--- | :--- | | Audit Fees | 700 | 693 | | Tax Fees | 7 | — | | All Other Fees | — | 62 | | **Total** | **707** | **755** | [Item 16G. Corporate Governance](index=163&type=section&id=Item%2016G.%20Corporate%20Governance) As a foreign private issuer, the company follows certain Danish home country practices instead of Nasdaq corporate governance standards, including not having a quorum requirement for shareholder meetings, not providing U.S. standard proxy statements, and allowing the board to issue securities without shareholder approval in specific instances - The company follows Danish home country practice in lieu of Nasdaq's quorum requirements for shareholder meetings[963](index=963&type=chunk) - The company does not follow Nasdaq requirements for proxy solicitations or for shareholder approval of all equity compensation plans and certain other security issuances, adhering instead to Danish corporate law[964](index=964&type=chunk) [PART III](index=164&type=section&id=PART%20III) [Item 18. Financial Statements](index=164&type=section&id=Item%2018.%20Financial%20Statements) This section contains the company's audited consolidated financial statements for 2017-2019, prepared under IFRS, including Deloitte's unqualified opinion on both financial statements and internal control over financial reporting, highlighting the adoption of IFRS 16 for leases and clinical trial accruals as a critical audit matter [Report of Independent Registered Public Accounting Firm](index=166&type=section&id=Report%20of%20Independent%20Registered%20Public%20Accounting%20Firm) Deloitte issued an unqualified opinion on the company's consolidated financial statements for the three years ended December 31, 2019, and on the effectiveness of internal control over financial reporting, noting the adoption of IFRS 16 and identifying clinical trial accruals as a critical audit matter - The auditor issued an **unqualified opinion** on the financial statements for the three years ended December 31, 2019, and on the company's internal control over financial reporting as of December 31, 2019[967](index=967&type=chunk)[976](index=976&type=chunk) - The report highlights a change in accounting principle due to the adoption of **IFRS 16 (Leases)** effective January 1, 2019[968](index=968&type=chunk) - Clinical trial accruals were identified as a **critical audit matter** due to the subjectivity and judgment required in estimating the progress of research and development activities[972](index=972&type=chunk)[974](index=974&type=chunk) [Consolidated Financial Statements](index=170&type=section&id=Consolidated%20Financial%20Statements) The consolidated financial statements show a net loss of €218.0 million for 2019, increased total assets to €676.7 million, and total equity to €597.1 million, primarily due to capital increases, with €598.1 million in cash at year-end, and notes detailing critical accounting policies including IFRS 16 adoption, revenue recognition, and share-based payments Consolidated Statement of Profit or Loss (2017-2019) | (€ thousands) | 2019 | 2018 | 2017 | | :--- | :--- | :--- | :--- | | **Revenue** | 13,375 | 10,581 | 1,530 | | **Research and development costs** | (191,621) | (140,281) | (99,589) | | **General and administrative expenses** | (48,473) | (25,057) | (13,482) | | **Net profit/(loss) for the year** | (218,016) | (130,097) | (123,897) | Consolidated Statement of Financial Position (as of Dec 31) | (€ thousands) | 2019 | 2018 | | :--- | :--- | :--- | | **Total assets** | 676,732 | 318,968 | | **Total equity** | 597,114 | 280,050 | | **Total liabilities** | 79,618 | 38,918 | | **Cash and cash equivalents** | 598,106 | 277,862 | - The company adopted **IFRS 16 "Leases"** as of January 1, 2019, using the modified retrospective approach, resulting in a lease liability of **€17.7 million** and right-of-use assets of **€18.4 million**[996](index=996&type=chunk)[997](index=997&type=chunk)[998](index=998&type=chunk) - The company's investment in its associate, Visen Pharmaceuticals, had a carrying amount of **€15.5 million** as of December 31, 2019, with the company's share of Visen's loss for the year being **€8.1 million**[1151](index=1151&type=chunk)[1152](index=1152&type=chunk)[1155](index=1155&type=chunk)

Financial Data and Key Metrics Changes - The company reported a net loss of €218 million or €4.69 per share for 2019, compared to a net loss of €130.1 million or €3.17 per share in 2018, indicating a significant increase in losses [28]. - Research and development costs rose to €191.6 million in 2019 from €140.3 million in 2018, reflecting ongoing advancements in the pipeline [28]. - General and administrative expenses increased to €48.5 million in 2019 from €25.1 million in 2018, primarily due to higher personnel costs [29]. Business Line Data and Key Metrics Changes - The TransCon Growth Hormone program is on track for a U.S. BLA filing in Q2 2020 and an MAA filing in Q4 2020, with positive clinical data supporting its efficacy [12][14]. - TransCon PTH is in Phase 2 trials, with an expansion of enrollment due to the recall of NATPARA, aiming to establish it as a true replacement therapy for hypoparathyroidism [17][18]. - TransCon CNP is in a Phase 2 trial, with ongoing efforts to expand clinical programs in China, indicating a strategic focus on global reach [25][26]. Market Data and Key Metrics Changes - The adult growth hormone deficiency market is under-penetrated, with estimates suggesting only 15% to 20% penetration, presenting a significant opportunity for the company [49]. - The company is leveraging its global structure to mitigate risks associated with the pandemic, ensuring continued progress across various geographic regions [10][11]. Company Strategy and Development Direction - The company aims to become a leading biopharma company, focusing on a diverse pipeline of innovative drug candidates across multiple therapeutic areas, including endocrinology and oncology [9][26]. - The strategic goal includes establishing TransCon Growth Hormone as a new benchmark in growth hormone replacement therapy, with plans for global clinical reach and new indications [14][16]. Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving 2020 milestones despite the pandemic, citing a strong organizational structure and adaptability [10][11]. - The company anticipates increased expenses in 2020 as it continues to advance its pipeline and expand its oncology activities [30][31]. Other Important Information - The company ended 2019 with cash and cash equivalents of €598.1 million, providing a solid financial foundation for ongoing projects [29]. - The company is preparing for a potential launch of TransCon Growth Hormone in the U.S. in 2021, indicating readiness for commercialization [16]. Q&A Session Summary Question: Feedback from Pitco on growth hormone and PaTH Forward results - Management indicated they received initial feedback from Pitco, requesting more information, and they expect to hear back by the end of June [38][39]. - They plan to provide analysis related to each element of the composite primary endpoint in the PaTH Forward results [42]. Question: Requirements for European application and adult growth hormone market - Additional submissions for the MAA include an approved PIP and a risk management plan [46]. - The adult growth hormone deficiency market is significantly under-penetrated, presenting a large opportunity for the company [49]. Question: Titration regimen for long-term extension study - The high level of removal of standard of care is attributed to the physiological profile of the PTH product, which mimics continuous PTH levels [52]. - The six-month follow-up data is expected to be essential for assessing treatment effects [54]. Question: Differences in patients previously on NATPARA - Patients previously on NATPARA will be analyzed separately for certain endpoints, but not for the primary endpoint [56]. Question: Frequency of clinical visits in OLE study - The company is implementing procedures to allow for remote monitoring and follow-up, ensuring patient safety during the pandemic [64].

Endocrinology Rare Diseases Pipeline - Ascendis Pharma expects to submit the BLA for TransCon hGH for pediatric growth hormone deficiency in Q2 2020 and the MAA in Q4 2020[7, 23, 50] - The Phase 3 heiGHt Trial demonstrated superior height velocity (AHV) of TransCon hGH in pediatric GHD, with 112 cm/year compared to 103 cm/year for Genotropin, a treatment difference of 086 cm/year[25, 50] - The adverse event profile of TransCon hGH was comparable to daily hGH across Phase 3 trials, with 77% of patients experiencing treatment-emergent adverse events (TEAEs) in the TransCon hGH 024 mg/kg/week group in the heiGHt trial, compared to 70% in the Genotropin group[42] - Ascendis Pharma plans to initiate a Phase 3 trial for adult GHD in Q1 2020 and a Phase 3 trial for pediatric GHD in Japan in Q4 2020[50, 97] - Top-line Phase 2 data for TransCon PTH in hypoparathyroidism is expected by the end of March 2020, with six-month data from the open-label extension expected in Q3 2020[7, 65, 97] - Preliminary data from the first 8 subjects completing 4 weeks follow-up in open-label extension of TransCon PTH trial shows that all subjects are completely off current standard of care, 8 of 8 subjects no longer require active vitamin D, and 7 of 8 subjects no longer require calcium supplements[65] - Ascendis Pharma expects to initiate a Phase 2 trial for achondroplasia (ACH) in China in Q4 2020 through VISEN Pharmaceuticals[7, 70, 97] Oncology Pipeline - Ascendis Pharma anticipates submitting the first IND or equivalent for its oncology program in Q4 2020[7, 95, 97] - Preclinical data for TransCon IL-2 b/g in cynomolgus monkeys showed a single 1 mg dose/animal (~01 mg/kg) resulted in >3-fold enhancement of lymphocyte counts, with minimal effect on eosinophils compared to Aldesleukin[88] Financial Position - As of September 30, 2019, Ascendis Pharma had approximately €659 million in cash and cash equivalents[7]