Ascendis Pharma (ASND) FY 2025 Conference September 03, 2025 08:00 AM ET Company ParticipantsJan Møller Mikkelsen - President, CEO & DirectorScott Smith - EVP & CFOConference Call ParticipantsDerek Archila - MD & Equity Research Analyst - BiotechnologyDerek ArchilaIt's a long way to say, yeah. All right. Let's get started here. Welcome everyone to the 2025 Wells Fargo Healthcare Conference. My name is Derek Archila. We're going to be kicking off today with Ascendis Pharma. From the company, we have Jan Mikk ...

Financial Data and Key Metrics Changes - The company is experiencing increasing revenue generation quarter by quarter, indicating a positive cash contribution and independence from financial markets [3][4] - The peak sales potential for YORVIPATH is estimated at $5 billion, with a significant portion expected from the U.S. in the initial years before shifting to ex-U.S. markets [10][11] Business Line Data and Key Metrics Changes - YORVIPATH is positioned as a replacement therapy for hypoparathyroidism, with strong intellectual property protection extending to 2042, and a stable growth trajectory observed since its launch [4][6] - The company has a diversified strategy for ex-U.S. markets, with direct market efforts in 14 countries and partnerships in others, including Japan [7][8] Market Data and Key Metrics Changes - In the U.S., there are approximately 80,000 to 100,000 diagnosed patients with hypoparathyroidism, indicating a substantial unmet medical need [5] - The competitive landscape for YORVIPATH is clearing, with no other products in clinical development offering similar benefits [6][7] Company Strategy and Development Direction - The company aims to develop three independent product opportunities addressing major unmet medical needs, leveraging its TransCon technology [2][3] - Future plans include label expansion for existing products and the development of new compounds targeting both rare diseases and broader indications [3][42] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing growth of YORVIPATH and the potential for TransCon CNP, with a priority review by the FDA expected soon [21][29] - The company is focused on building a sustainable pipeline and expanding its commercial infrastructure, with a goal of increasing clinical trial efforts in the coming years [42][44] Other Important Information - The partnership with Novo Nordisk remains strong, with no changes impacting the collaboration on TransCon semaglutide [35] - The company is exploring new therapeutic areas, including cardiovascular diseases, while continuing to focus on rare disease endocrinology [43][44] Q&A Session Summary Question: What drives the uptake of YORVIPATH in the U.S.? - The uptake is driven by a steady flow of new prescribers and prescriptions, with improvements expected in the infrastructure for patient transfers from prescription to treatment [11][12] Question: How does the company differentiate its products from competitors? - The differentiation lies in the continuous exposure provided by TransCon technology, which minimizes injection site reactions and maintains stable hormone levels [22][24] Question: What is the company's strategy for TransCon CNP? - The strategy involves positioning TransCon CNP as a transformative treatment for achondroplasia, focusing on both naive and switch patients [26][29] Question: How does the company view its cost base and earnings power moving forward? - The company has maintained cost efficiency and is looking to expand its pipeline, with expectations of turning a corner in cash generation in the near future [44][50]

Financial Data and Key Metrics Changes - The company has achieved revenue generation that is increasing quarter by quarter, indicating a positive cash contribution [3] - The peak sales potential for YORVIPATH is estimated at $5 billion, with a significant portion expected from the U.S. in the first four to five years [10] Business Line Data and Key Metrics Changes - YORVIPATH is positioned as a replacement therapy for hypoparathyroidism, with strong intellectual property protection extending to 2042 [4] - The company has a diversified strategy for ex-U.S. markets, with direct market efforts in 13-14 countries and plans to expand further [7] Market Data and Key Metrics Changes - In the U.S., there are approximately 80,000 to 100,000 diagnosed patients with hypoparathyroidism, highlighting a significant unmet medical need [5] - The competitive landscape for YORVIPATH is clearing, with no other products in clinical development offering similar benefits [6] Company Strategy and Development Direction - The company aims to develop three independent product opportunities addressing major unmet medical needs, leveraging its TransCon technology [2] - Future plans include label expansion and the development of new compounds targeting both rare diseases and broader indications [3][42] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's ability to generate positive cash flow and remain independent of financial markets [3] - The company is focused on building a sustainable growth model through its pipeline and partnerships, particularly in rare diseases and metabolic disorders [43] Other Important Information - The company is preparing for the FDA's priority review of TransCon CNP, which is expected to differentiate itself from competitors like BioMarin's VOXZOGO [21] - The partnership with Novo Nordisk remains strong, with no changes impacting the collaboration on TransCon semaglutide [35] Q&A Session Summary Question: What drives the uptake of YORVIPATH in the U.S.? - The uptake is driven by a steady flow of new prescribers and prescriptions, with improvements expected in the infrastructure for patient transfers from prescription to treatment [11][12] Question: How does the company view competition for longer-acting formulations? - The company has opted for a daily formulation for YORVIPATH to ensure patient stability before considering a weekly product [15][16] Question: What is the differentiation of TransCon CNP compared to competitors? - TransCon CNP offers continuous exposure without injection site reactions and has shown meaningful clinical effects beyond linear growth [22][24] Question: How does the company plan to expand its TransCon platform? - The company is focused on developing multiple product opportunities across various therapeutic areas, including cardiovascular diseases [42][43] Question: What is the company's approach to cost management and earnings power? - The company has maintained cost efficiency and is looking to expand its pipeline while generating cash from operations [44][50]

COPENHAGEN, Denmark, Sept. 02, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced three oral presentations of its latest achondroplasia and hypoparathyroidism data at ASBMR 2025, the annual meeting of the American Society for Bone & Mineral Research being held in Seattle from September 5-8, 2025. These include a new analysis of pivotal trial data correlating improvements in leg bowing with improved physical functioning in children with achondroplasia treated with TransCon® CNP (nave ...

COPENHAGEN, Denmark, Aug. 28, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that company executives will participate in a fireside chat at the 2025 Wells Fargo Healthcare Conference on Wednesday, September 3, 2025, at 8:00 a.m. Eastern Time / 5:00 a.m. Pacific Time in Boston, Massachusetts. A live webcast of the presentation will be available via the Investors & News section of the Ascendis Pharma website at investors.ascendispharma.com. A webcast replay will also be available ...

Financial Data and Key Metrics Changes - For Q2 2025, total product revenue was €153.7 million, with a negative foreign currency exchange impact of €7.6 million [27] - Skytrofa revenue for the quarter was €50.7 million, including a €1.8 million negative currency impact [27] - Eurvipath revenue more than doubled to €103 million from €44.7 million in Q1 2025, despite a negative currency headwind of €5.8 million [27] Business Line Data and Key Metrics Changes - Skytrofa continues to see growth in the number of patients treated, with Q2 revenue at €51 million [22] - The U.S. launch of Eurvipath has seen over 1,500 prescribers write prescriptions for around 3,100 unique patients [11][27] - The company expects to become cash flow positive on a quarterly basis this year [27] Market Data and Key Metrics Changes - In the U.S., the majority of patients receiving prescriptions for Eurvipath have received payer approval within three months [11] - Outside the U.S., steady revenue growth for Eurvipath is expected, with further acceleration anticipated as reimbursement becomes available in additional countries [12] Company Strategy and Development Direction - The company aims to achieve blockbuster status for multiple products and expand its innovation engine [9] - Focus on building long-term leadership in the treatment of hypoparathyroidism and growth disorders [32] - Ongoing collaboration with Novo Nordisk for the development of TransCon-based products in metabolic and cardiovascular diseases [25] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the strong global launch of Eurvipath and its potential to achieve multiple billions in peak sales [32] - The company is focused on improving patient access and reimbursement processes to enhance treatment uptake [50] - Management highlighted the importance of ongoing clinical programs to support label expansion and the potential for future growth [12][22] Other Important Information - TransCon Growth Hormone is now approved in the U.S. for adult growth hormone deficiency, with further label expansions planned [6] - The company is preparing for a Phase 3 study of combination therapy in children with achondroplasia by 2025 [22] Q&A Session Summary Question: Continuation of enrollment trends and unique patients - Management confirmed that unique patients enrolled grew from approximately 1,750 at the end of Q1 to 3,100 at the end of Q2, indicating steady state growth [36][37] Question: Improving the time from enrollment to treatment - Management acknowledged ongoing efforts to improve the time from enrollment to treatment, with expectations for improvements in the second half of the year [46][50] Question: Patient demographics and severity - Management clarified that there is no medical definition for severity in hyperparathyroidism, and they are focusing on addressing physicians who see a high number of patients [53][56] Question: Revenue expectations and patient growth - Management indicated that revenue growth in Europe is expected to continue, with a strong launch in the U.S. [61][62] Question: Conversion rates from enrollment to treatment - Management expressed that while they aim for high conversion rates, some patients may still face challenges in reimbursement [66][68] Question: Reporting enrollment forms in future quarters - Management confirmed they will continue to report necessary KPIs until a steady state is reached [70] Question: Compliance and patient retention - Management reported low discontinuation rates in Europe, indicating strong patient retention [75] Question: Titration period and costs - Management stated that the titration period is successful, but specifics on costs post-titration are still being evaluated [100][102] Question: Harmonizing U.S. and EU labels - Management is working on trials to potentially harmonize the U.S. label with the EU label for higher doses [108]

Financial Data and Key Metrics Changes - Total product revenue for Q2 2025 was €153,700,000, with a negative foreign currency exchange impact of €7,600,000 [27] - Skytrofa revenue for the quarter was €50,700,000, including a €1,800,000 negative currency impact [27] - Eurvipath revenue more than doubled to €103,000,000, up from €44,700,000 in Q1 2025, despite a negative currency headwind of €5,800,000 [27] - R&D costs decreased to €72,000,000 from €83,500,000 year-over-year, while SG&A expenses increased to €107,600,000 from €74,300,000 [29] - The company ended Q2 2025 with cash and cash equivalents totaling €494,000,000, down from €518,000,000 as of March 31 [30] Business Line Data and Key Metrics Changes - Revenues for Europad in Q2 reached €103,000,000, more than double that of Q1 [10] - Skytrofa revenues were €51,000,000, with growth driven by new patient starts and recent label expansion for adult growth hormone deficiency [24] - The company expects continued revenue growth driven by the strength of the global launch of Europad [30] Market Data and Key Metrics Changes - In the U.S., over 1,500 prescribers wrote prescriptions for around 3,100 unique patients for Europad, reflecting strong demand [11] - The company has recognized revenue from more than 30 countries and has commercial agreements covering over 75 countries [15] - The U.S. launch of Europad is progressing well, with expectations for additional commercial launches in Europe and Japan [16][32] Company Strategy and Development Direction - The company aims to achieve blockbuster status for multiple products and expand its innovation engine [8] - Focus on building long-term leadership in the treatment of hypoparathyroidism and growth disorders through differentiation, demand, and access [13] - The company is preparing for future growth opportunities and ongoing collaborations with Novo Nordisk for TransCon-based products in metabolic and cardiovascular diseases [25] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the continued strong demand for Europad and the potential for it to become a blockbuster product [10] - The company anticipates becoming cash flow positive on a quarterly basis this year [29] - Management highlighted the importance of payer approvals and the ongoing efforts to improve the patient experience and access to treatment [34] Other Important Information - TransCon Growth Hormone is now approved in the U.S. for adults with growth hormone deficiency, and the company plans further label expansions [5] - The FDA granted priority review for TransCon CNP, recognizing its potential to improve treatment for achondroplasia [9] - The company is advancing new clinical data and preparing for additional key label expansions [25] Q&A Session Summary Question: Continuation of enrollment trends - Management confirmed that the number of unique patients enrolled grew to 3,100 by the end of Q2, indicating steady state growth [36][38] Question: Improving conversion from enrollment to treatment - Management acknowledged ongoing efforts to improve the time from enrollment to treatment, with expectations for better results in the second half of the year [45][49] Question: Patient demographics and severity - Management clarified that there is no medical definition for severity in hyperparathyroidism, and they are focusing on addressing physicians who see a high number of patients [53][55] Question: Future revenue expectations - Management indicated that they expect continued strong revenue growth in the U.S. and Europe, with a focus on expanding commercial launches [61][62] Question: Compliance and patient retention - Management reported low discontinuation rates in Europe, indicating strong patient retention and satisfaction with the therapy [72][73] Question: Reporting enrollment forms - Management confirmed that they will continue to report necessary KPIs related to enrollment and treatment until a steady state is achieved [69][70] Question: Titration period and costs - Management noted that the titration period is successful, but they are still assessing real-world data on costs post-titration [98][101]

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 August, 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 ...

This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis' future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) Ascendis' ability to apply its TransCon technology platform to make a meaningful difference for patients and (ii) Ascendis' ...

"The abnormal body composition, dyslipidemia, and insulin resistance that are the hallmarks of adult GHD predispose affected individuals to serious medical complications such as metabolic syndrome, increased cardiovascular risk, and impaired quality of life – including cognitive dysfunction, depression, anxiety, sleep disturbance, and reduced physical and mental drive. Despite this, adherence to daily somatropin injections that have been the standard of care for more than 25 years remains a problem," said K ...