Financial Data and Key Metrics Changes - In 2024, SKYTROFA revenue reached approximately €200 million, with a 6.5% market share of the total growth hormone market in the U.S. and around 45% of the total U.S. long-acting growth hormone market [10][11] - Total revenue for the fourth quarter was €173.9 million, including a $100 million upfront fee from Novo Nordisk, while total revenue for the full year 2024 was €363.6 million [35][36] - For the fourth quarter, SKYTROFA revenue was €58.5 million, down from €64.2 million in the same quarter of 2023, but benefited from a favorable sales adjustment of €4.6 million [32][34] Business Line Data and Key Metrics Changes - SKYTROFA volume increased 84% in 2024, with a premium net pricing of 3x compared to daily growth hormone [18][10] - YORVIPATH revenue for the fourth quarter increased to €13.6 million, bringing total 2024 revenue to €28.7 million [33][34] - TransCon CNP demonstrated significant improvements in linear growth and body proportionality compared to placebo in clinical trials [25][26] Market Data and Key Metrics Changes - The U.S. market for chronic hypoparathyroidism is estimated to have about 70,000 to 90,000 patients, with a significant portion currently using conventional therapy [19][20] - Initial demand for YORVIPATH is strong, with 908 patients prescribed as of February 7, 2025, including 539 unique prescribers across 44 states [21][22] Company Strategy and Development Direction - The company aims to expand SKYTROFA's addressable market and plans to submit an NDA for adult growth hormone deficiency this year [17][31] - YORVIPATH is expected to become the standard-of-care for hypoparathyroidism, with plans for further commercial launches in multiple international markets [34][22] - The company is expanding its TransCon technology platform beyond endocrine rare diseases into metabolic diseases and ophthalmology [14][29] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong launch of YORVIPATH and its potential to become a multi-billion-dollar product [11][34] - The company is optimistic about the growth potential of SKYTROFA and YORVIPATH, with expectations for sustained revenue growth [10][31] - Management highlighted the importance of ongoing discussions with payers and the evolving reimbursement landscape for YORVIPATH [61][62] Other Important Information - The company ended 2024 with cash, cash equivalents, and marketable securities totaling €560 million, compared to €399 million at the end of 2023 [38] - The company plans to submit an IND application for a combination treatment of TransCon CNP and TransCon Growth Hormone for achondroplasia [27][26] Q&A Session Summary Question: Can you confirm the 908 figure is unique patients and not cumulative scripts? - Yes, the 908 figure represents unique patient enrollments, with about 80% being new to YORVIPATH and 20% from EAP or clinical trials [41][44] Question: How many of the 908 scripts have converted to actual therapy? - The company is still gathering data on this, but many of the patients are expected to be from the uncontrolled group [50][54] Question: What type of doctors are prescribing YORVIPATH? - Prescribers include a broad range of physicians, with over 50% reach among priority physicians, and many are new prescribers not previously associated with NATPARA [82][87] Question: What are the expectations for prior authorizations for YORVIPATH? - The company anticipates some prior authorization requirements, but discussions with payers have been productive [61][96] Question: What is the market opportunity for adult growth hormone deficiency? - The penetration rate is currently low, presenting a significant growth opportunity, especially with the upcoming PDUFA date [126][127]

Financial Performance - The company reported a net loss of €481.4 million for the year ended December 31, 2023, and a net loss of €378.1 million for the year ended December 31, 2024[28]. - As of December 31, 2024, the company's total equity was negative by €105.7 million, an improvement from a negative balance of €145.7 million as of December 31, 2023[28]. - The company estimates that its existing cash and cash equivalents of €559.5 million will be sufficient to fund operations for at least the next twelve months[35]. - The company may incur substantial operating losses for the foreseeable future as it executes its operating plan[29]. Product Development and Regulatory Approvals - The company received FDA approval for TransCon hGH (SKYTROFA) on August 25, 2021, and for TransCon PTH (YORVIPATH) on August 9, 2024[27]. - The European Commission granted marketing authorization for SKYTROFA on January 11, 2022, and for YORVIPATH in November 2023[27]. - The FDA approved YORVIPATH for the treatment of hypoparathyroidism in adults on August 9, 2024, which includes a pen-injector device developed with Ypsomed[163]. - The FDA issued a Complete Response Letter in April 2023 regarding TransCon PTH, citing concerns about the manufacturing control strategy[165]. - Regulatory authorities may impose additional studies or trials, which can delay the approval of product candidates[158]. - The approval process for combination products may involve additional delays due to the need for coordination between drug and device components[164]. Market and Competition - The company has limited revenue from commercial product sales and relies significantly on its TransCon technologies and products[31]. - The company faces uncertainties regarding the successful commercialization and regulatory approval of its product candidates, which may hinder revenue generation[45]. - The company faces intense competition in the biotechnology and pharmaceutical industries, with competitors potentially commercializing products faster[75]. - Several companies are developing competing products in the same therapeutic areas, including long-acting growth hormones and cancer immunotherapies[76][77]. - The company may face technology-based competition as other firms develop enhanced drug delivery and sustained release technologies[78]. Collaboration and Partnerships - The company relies on collaboration partners for clinical studies and regulatory approvals, with recent partnerships including VISEN Pharmaceuticals for endocrinology rare disease therapies in Greater China and Teijin Limited for certain products in Japan[88][92]. - The company has formed Eyconis, Inc. to develop and commercialize TransCon ophthalmology products globally, receiving an equity position in the new company[93]. - The company may seek orphan designation for some product candidates, which could provide market exclusivity, but there is no guarantee of success in obtaining or maintaining this designation[95]. - The timing and amount of milestone and royalty payments from collaboration partners depend on their successful development and commercialization efforts[91]. Manufacturing and Supply Chain - The company relies on third-party manufacturers for the production of its products and product candidates, which poses risks related to manufacturing capacity and regulatory compliance[102][103]. - The company has secured agreements with third-party manufacturers for sufficient capacity for SKYTROFA and YORVIPATH, but market demand estimates may be inaccurate[103]. - The company is highly dependent on single-source suppliers for its drug products, which poses a risk to its business if these suppliers fail to deliver[134]. - The company has agreements with Vetter Pharma Fertigung for the manufacturing of TransCon hGH and TransCon PTH drug products, which are critical for clinical trials and commercialization[134]. Regulatory Compliance and Risks - The company faces significant costs and management time devoted to compliance as a public entity, which may hinder its ability to achieve business objectives[115]. - The company is subject to Section 404 of the Sarbanes-Oxley Act, requiring annual assessments of internal control effectiveness over financial reporting[116]. - The company may face significant penalties for non-compliance with regulatory requirements, including product recalls and withdrawal of approvals[169]. - Non-compliance with global anti-corruption laws, such as the U.S. Foreign Corrupt Practices Act, could result in criminal or civil liability, negatively impacting financial condition and operations[195]. - The company must adhere to various healthcare laws, including the U.S. Anti-Kickback Statute and false claims laws, with violations potentially leading to significant penalties and operational restrictions[203]. Economic and Political Environment - The potential abandonment of the Euro by EU member states could harm the company's business and financial condition[128]. - The UK's withdrawal from the EU may negatively impact global economic conditions and the company's operations[129]. - Global economic and political instability, including trade disputes and natural disasters, could lead to increased costs and operational disruptions for the company[148]. - Legislative changes, such as the BIOSECURE Act, could restrict the company's ability to collaborate with certain foreign entities, impacting its operations[106]. Cybersecurity and Data Protection - Cybersecurity threats pose a risk to the company's information technology systems, which could disrupt product development and critical business functions[140]. - The company maintains cyber liability insurance, but it may not be sufficient to cover potential losses from cybersecurity incidents[142]. - Data protection and privacy law compliance is critical, as failures could result in legal liabilities and damage to reputation, affecting financial performance[206]. - The company may face increased scrutiny from regulatory authorities as operations grow, necessitating adherence to evolving data protection laws[207]. Clinical Development Challenges - Clinical drug development is lengthy and expensive, with uncertain outcomes that may lead to delays in obtaining marketing approvals[52]. - Delays in clinical trials can arise from various factors, including patient enrollment challenges and regulatory requirements[53]. - The company may encounter difficulties in managing growth and expanding operations, requiring improvements in operational and financial controls[113]. - The ongoing military conflict in Ukraine has led to the need for alternative clinical trial sites, impacting patient enrollment and potentially increasing product development costs[151].

Core Viewpoint - Ascendis Pharma A/S has announced a share repurchase program and net settlement of restricted stock units (RSUs) to preserve approximately 200,000 American Depositary Shares (ADSs) held as treasury shares, with a total expected use of approximately $25 million in the first quarter of 2025 [1][3]. Share Repurchase Program - The Board of Directors has authorized the repurchase of up to $18.25 million of the Company's ADSs, which represent ordinary shares of Ascendis Pharma A/S [2][4]. - The repurchases will be executed in compliance with U.S. securities regulations and may include various methods such as open market purchases and block trades [4]. - The timing and amount of repurchases will depend on market conditions and other factors, and the program can be modified or terminated at any time without notice [4]. Net Settlement of RSUs - The Company plans to apply net settlement for tax-withholding obligations related to the vesting of approximately 450,000 RSUs, amounting to about $9 million, which will preserve approximately 75,000 ADSs as treasury shares [3]. - Together with the share repurchase program, this initiative aims to maintain a total of approximately 200,000 ADSs held as treasury shares [3]. Company Overview - Ascendis Pharma is focused on developing innovative therapies using its TransCon technology platform, aiming to make a significant impact on patients' lives [5]. - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [5].

Core Viewpoint - Ascendis Pharma A/S has announced a share repurchase program and net settlement of restricted stock units (RSUs) to preserve approximately 200,000 American Depositary Shares (ADSs) held as treasury shares, with a total expected expenditure of around $25 million [1][3]. Share Repurchase Program - The Board of Directors has authorized the repurchase of up to $18.25 million of the Company's ADSs, which represent ordinary shares of Ascendis Pharma A/S [2][4]. - The repurchases will be conducted in compliance with U.S. securities regulations and may utilize various methods including open market purchases and block trades [4]. Net Settlement of RSUs - The Company plans to net settle tax-withholding obligations related to the vesting of approximately 450,000 RSUs, amounting to about $9 million, which will help preserve around 75,000 ADSs as treasury shares [3]. Overall Strategy - The combined efforts of the Share Repurchase Program and the Net Settlement of RSUs are aimed at maintaining a total of approximately 200,000 ADSs in treasury [1][3]. Company Overview - Ascendis Pharma is focused on utilizing its TransCon technology platform to develop innovative therapies, with a commitment to improving patients' lives [5].

– Strong early U.S. YORVIPATH® launch with 908 prescriptions as of Feb. 7, 2025; YORVIPATH full year 2024 revenue of €28.7 million – Following pre-NDA meeting with FDA, on track to submit TransCon™ CNP NDA for achondroplasia in children in Q1 2025, followed by MAA in Q3 2025 – SKYTROFA® full year 2024 revenue was ~€202 million, excluding sales deductions related to prior years, (€197.0 million plus ~€5 million of sales deductions related to prior years) – Total 2024 operating expenses of €598 million – Con ...

(CVR-nr. 29918791) (Registration no 29918791) Name, Registered Office and Objects of the Company: Articles of Association of Ascendis Pharma A/S Exhibit 1.1 REF. K003583-0002 – 2788-8926-9516.v.2 11 February 2025 Article 1 The object of the company is to develop ideas and preparations for the combating of disease medically, to manufacture and sell such preparations or ideas, to own shares of companies with the same objects and to perform activities in natural connection with these objects. Company Capital a ...

COPENHAGEN, Denmark, Feb. 05, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (NASDAQ:ASND) today announced it will report full year 2024 financial results and provide a business update on Wednesday, February 12, 2025, after the close of the U.S. financial markets. Ascendis Pharma will also host a conference call and live webcast on February 12, 2025, at 4:30 p.m. Eastern Time (ET) to discuss 2024 financial results. Those who would like to participate may access the live webcast here, or register in advance fo ...

I’m a stock analyst with an MBA and a background in healthcare, bringing years of experience as a Registered Nurse dedicated to patient care. I've had the privilege of sharing my insights on Seeking Alpha since 2017. I am most interested in identifying underlying assumptions in stock valuations by emphasizing financial modeling techniques like DCF analysis. I then provide scenario-based forecasts to help readers gauge reasonable outcomes. I am influenced by books like Superforecasting and Antifragile. As su ...

This is my first Ascendis Pharma ( ASND ) article. I became interested in this name as a consequence of researching my recent article on BioMarin ( BMRN ). As it turns out Ascendis isWriting under the pseudonym "out of ignorance", I very much regard investing as a learning process. Investing failures are tuition paid. Investing successes enter the trove of lessons learned. In my Seeking Alpha articles I share my experience from decades of investing and from ~5 years of focused research on a variety of stock ...

Business and Strategic Roadmap - Ascendis Pharma is positioned for rapid revenue growth driven by the launch of its first two Endocrinology Rare Disease medicines, SKYTROFA and YORVIPATH, and planned filings for its third product, TransCon CNP for achondroplasia [2] - The company's expanding TransCon technology platform and partnerships demonstrate a business model focused on fast, successful drug development, aiming to create substantial stakeholder value [2] Key Updates and Milestones TransCon hGH (SKYTROFA) - SKYTROFA full-year 2024 revenue expected to be ~€202 million, excluding sales deductions related to prior years [3][7] - U.S. SKYTROFA volume (mg) increased 84% year-over-year in 2024, capturing an estimated 6.5% market share of the total U.S. growth hormone market [3] - PDUFA goal date of July 27, 2025, for FDA review of supplemental BLA for adult growth hormone deficiency treatment, with a planned U.S. commercial launch in Q4 2025 [3] - Plan to submit an IND application or similar for a basket trial evaluating TransCon hGH in additional indications during Q3 2025 [3] TransCon PTH (YORVIPATH) - YORVIPATH full-year 2024 unaudited preliminary revenue estimate of ~€29 million [3][7] - Commercially available in Germany and Austria since January 2024, with ~700 patients on treatment in Europe Direct and International Markets by the end of 2024 [3] - Available for prescription in the U.S. since late December 2024, with 324 patients enrolled in the Ascendis Signature Access Program or direct with specialty pharmacy as of January 9, 2025 [3] - Expect commercial launch in at least five additional Europe Direct countries in 2025 [3] TransCon CNP (navepegritide) - Plan to submit NDA for the treatment of children with achondroplasia in Q1 2025 and Marketing Authorisation Application to the European Medicines Agency in Q3 2025 [3] - New data from the ApproaCH Trial showed significant improvements in leg bowing with TransCon CNP compared to placebo [3] - Topline Week 26 results from Phase 2 COACH Trial expected in Q2 2025 [3] - Plan to submit an IND or similar for the treatment of hypochondroplasia in Q4 2025 [3] Expanding the TransCon Platform & Pipeline - Development of a new TransCon protein degrader platform designed to enable efficient clearance of hormones, cytokines, and other targets [3] - First planned TransCon protein degrader product candidate aims to normalize excess FGF-23 hormone levels for patients with X-linked hypophosphatemia [3] Financial Update - Unaudited preliminary estimate of total full-year 2024 product revenue of ~€226 million [3] - Total full-year 2024 revenue, including a $100 million Novo Nordisk milestone payment, estimated at ~€364 million [7] - Pro forma cash balance as of December 31, 2024, estimated at ~€655 million, including the expected Novo Nordisk payment [7] Presentation at J.P. Morgan Healthcare Conference - Ascendis Pharma's President and CEO Jan Mikkelsen will present the business and strategic roadmap update at the 43rd Annual J.P. Morgan Healthcare Conference on January 13, 2025 [1][4] - A live webcast of the event will be available on the company's Investor Relations website, with a replay accessible for 30 days [4]