Core Viewpoint - Ascendis Pharma A/S will report its first quarter 2025 financial results and provide a business update on May 1, 2025, after U.S. market close, followed by a conference call and live webcast [1] Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on its innovative TransCon technology platform to develop new therapies addressing unmet medical needs [3] - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [3] Event Details - The live webcast for the financial results will be accessible through a provided link, and a replay will be available for 30 days post-event [2]

Group 1 - The article expresses a beneficial long position in the shares of ASND, indicating a positive outlook on the company's stock performance [1] - The author emphasizes that the opinions presented are personal and not influenced by any business relationships with the mentioned companies [1] - There is a clear distinction made that the commentary does not constitute personalized investment advice, urging readers to conduct their own due diligence [2] Group 2 - The article highlights that past performance is not indicative of future results, which is a common disclaimer in investment discussions [3] - It is noted that the views expressed may not reflect those of Seeking Alpha as a whole, indicating a diversity of opinions among analysts [3] - The article clarifies that Seeking Alpha does not act as a licensed securities dealer or investment adviser, reinforcing the need for independent research by investors [3]

Core Insights - Ascendis Pharma has submitted a New Drug Application (NDA) to the FDA for TransCon CNP, aimed at treating children with achondroplasia, supported by data from three clinical trials and up to three years of open-label extension data [1][2] - The company plans to submit a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) in Q3 2025 [3] Group 1: Clinical Data and Benefits - Clinical data indicates that TransCon CNP not only increases growth velocity but also reduces health-related burdens, enhances muscle function, and corrects leg bowing in most treated children [3] - The treatment is administered once weekly and has a safety and tolerability profile comparable to placebo, positioning it as a potential best-in-class treatment for achondroplasia [3] Group 2: Achondroplasia Overview - Achondroplasia is a rare genetic condition affecting over 250,000 people globally, leading to various complications beyond skeletal dysplasia, including muscular, neurological, and cardiorespiratory issues [4] - Complications can include spinal deformities, impaired muscle strength, and chronic pain, significantly impacting quality of life and requiring multiple surgeries [4] Group 3: Company Background - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform to address unmet medical needs [5] - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [5]

Group 1: Ascendis Pharma - Ascendis Pharma A/S (ASND) shares increased by 10.4% to close at $168.84, driven by notable trading volume and a 6.2% gain over the past four weeks [1] - The company reported strong financial results for Q4 and the full year of 2024, which likely contributed to the recent share price rally [1] - The consensus EPS estimate for the upcoming quarter has been revised marginally higher, indicating a positive trend that may lead to further price appreciation [3] Group 2: Repligen - Repligen (RGEN) shares closed 1.5% higher at $152.81, with a 2.5% return over the past month [3] - The consensus EPS estimate for Repligen has decreased by 6.6% to $0.35, but this still represents a 25% increase compared to the previous year [4] - Repligen also holds a Zacks Rank of 3 (Hold), similar to Ascendis Pharma [4]

Group 1 - Ascendis Pharma A/S will participate in a fireside chat at the TD Cowen 45th Annual Health Care Conference on March 4, 2025, at 11:10 a.m. Eastern Time [1] - A live webcast of the presentation will be available on the Ascendis Pharma website, with a replay accessible for 30 days after the event [2] - Ascendis Pharma is focused on building a fully integrated biopharma company using its innovative TransCon technology platform to create new therapies [3] Group 2 - The company is headquartered in Copenhagen, Denmark, and has additional facilities in Europe and the United States [3]

Financial Performance - Ascendis Pharma reported a fourth-quarter EPS loss of 64 cents, beating the consensus loss of 1.07 euros [1] - Total revenue for the fourth quarter of 2024 was 173.9 million euros, exceeding the consensus of 111.178 million euros and up from 137.7 million euros reported a year ago [1] - TransCon hGH (Skytrofa) sales reached 58.5 million euros [1] Product Launch and Revenue - TransCon PTH (Yorvipath) revenue for the fourth quarter of 2024 totaled 13.6 million euros [2] - The U.S. launch of Yorvipath has seen a strong start, with 908 prescriptions as of February 7, 2025, and 539 unique prescribing healthcare providers [2] Cash Position and Share Repurchase - As of December 31, 2024, cash and cash equivalents totaled 559.5 million euros, which would increase to 655 million euros including the $100 million upfront payment [3] - The board authorized the repurchase of up to $18.25 million shares [3] Analyst Insights - Early prescription trends for Yorvipath in the U.S. are strong, but insurance coverage and reimbursement challenges may slow revenue growth in the short term [4] - Analysts are focusing on the company's progress toward achieving breakeven cash flow and key milestones, with a positive outlook for Yorvipath's launch in 2025 [4] - Goldman Sachs maintains a Buy rating and raises the price target to $225 from $200 [4] Stock Performance - ASND stock is up 15.8% at $146.08 as of the last check [5]

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].