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 financial independence from the market is highlighted, with consistent revenue growth expected [3] Business Line Data and Key Metrics Changes - The YORVIPATH launch is progressing well, with a potential peak sales opportunity of $5 billion in the hypoparathyroidism market [4][10] - The company is focusing on label expansion and developing new compounds based on TransCon technology [3] Market Data and Key Metrics Changes - In the U.S., there are approximately 80,000 to 100,000 diagnosed patients with hypoparathyroidism, indicating a significant unmet medical need [5] - The company has established a diversified strategy for ex-U.S. markets, with plans to expand into 14-15 countries by 2026 [7][8] Company Strategy and Development Direction - The company aims to develop three independent product opportunities addressing major unmet medical needs [2] - There is a focus on building a strong commercial infrastructure and expanding into cardiovascular diseases [44] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term growth of YORVIPATH, with a steady increase in prescriptions expected as the launch progresses [12][14] - The company is preparing for the FDA's priority review of TransCon CNP, which is expected to differentiate itself from competitors [22][25] Other Important Information - The partnership with Novo Nordisk remains strong, with no changes impacting the collaboration [36] - The company is exploring new indications and targets for the TransCon platform, with a focus on rare diseases [43] 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 transfer process from prescription to treatment [11][12] Question: How does the company view competition in the market? - The competitive landscape is clearing, with no other products in clinical development providing the same benefits as YORVIPATH [6][15] Question: What is the differentiation of TransCon CNP compared to competitors? - TransCon CNP offers continuous exposure and reduced injection site reactions, providing a significant clinical benefit beyond linear growth [23][24] Question: What is the company's strategy for the TransCon platform? - The company plans to expand its pipeline significantly, aiming for multiple clinical trials and product opportunities in the next few years [50][43] Question: How does the company view its cost base and earnings power moving forward? - The company has been cost-efficient and is looking to invest more in its pipeline while generating cash from operations [45][51]

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]

Financial Data and Key Metrics Changes - Ascendis Pharma reported a significant increase in first quarter revenue for Europath, reaching €44.7 million, up from €13.6 million in the previous quarter [28] - Total revenue for the first quarter was €101 million, which includes non-product revenue from collaboration partners [30] - R&D costs for the first quarter totaled €86.6 million, compared to €70.7 million in the same quarter of the previous year [30] - SG&A expenses increased to €101 million from €66.8 million year-over-year, primarily due to global commercial expansion [31] Business Line Data and Key Metrics Changes - Europath's global revenue grew to €45 million in the first quarter, reflecting strong U.S. demand and a growing patient base [13][28] - Skytrofa generated €51.3 million in revenue for the quarter, with stable pricing and market share despite seasonal impacts [29][19] - The company expects Europath to significantly contribute to revenue in 2025, with a potential to become a multi-billion euro product [18] Market Data and Key Metrics Changes - In the U.S., Europath was prescribed by over 1,000 unique prescribers for more than 1,750 patients, marking a successful launch [11] - The company estimates there are over 400,000 patients globally with chronic hypoparathyroidism, with 70,000 in the U.S. alone [18] - Skytrofa holds approximately 7% market share in the total growth hormone market in the U.S. and around 43% in the non-active growth hormone market [19] Company Strategy and Development Direction - Ascendis Pharma aims to be a leading biopharma company, focusing on the commercialization and development of Skytrofa and Europath [10] - The company is committed to expanding its product portfolio, with TransCon CNP being a key component in treating achondroplasia [12] - Ascendis Pharma is exploring market opportunities beyond endocrinology and rare diseases, indicating a strategic shift towards broader therapeutic areas [5] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong launch of Europath and its potential to become cash flow positive [10] - The company anticipates substantial revenue growth driven by the global launch of Europath and continued contributions from Skytrofa [32] - Management highlighted the importance of addressing unmet medical needs and the positive trends in reimbursement for Europath [38][40] Other Important Information - Ascendis Pharma's proprietary TransCon technology platform is fundamental to the development of its medicines, allowing for differentiated treatment benefits [26] - The company is collaborating with Novo Nordisk for the development of TransCon technology-based products in metabolic and cardiovascular diseases [27] Q&A Session Summary Question: What is the expected reimbursement rate for Yorvapath? - Management estimates that 17% to 18% of patients will ultimately get reimbursed once Yorvapath reaches steady state [38] Question: Can you provide the split between U.S. and ex-U.S. revenue for Yorvapath? - Management indicated that while specific numbers are not disclosed, there is steady growth expected outside the U.S. with an acceleration anticipated in the second half of the year [48] Question: What is the depth of prescribing for Yorvapath? - Management noted that they cannot definitively categorize patients as well-controlled or uncontrolled but expect a steady flow of patients transitioning from conventional therapies [66][70] Question: How is the company addressing payer dynamics? - Management emphasized that they are well-equipped to navigate reimbursement challenges, leveraging experience from previous product launches [75] Question: What are the competitive dynamics with new entrants in the market? - Management expressed confidence that their product addresses significant unmet medical needs and that new entrants may not significantly impact their market position [114]

Financial Data and Key Metrics Changes - Ascendis Pharma reported a significant increase in first quarter revenue for Europath, reaching €44.7 million, up from €13.6 million in the previous quarter [28] - Total revenue for the first quarter was €101 million, which includes non-product revenue from collaboration partners [30] - R&D costs for the first quarter totaled €86.6 million, compared to €70.7 million in the same quarter of the previous year [30] - SG&A expenses increased to €101 million from €66.8 million year-over-year, primarily due to global commercial expansion [31] Business Line Data and Key Metrics Changes - Europath's first quarter global revenue grew to €45 million, reflecting strong U.S. demand and a growing patient base [13][28] - Skytrofa revenue for the quarter was €51.3 million, with stable pricing and market share, although impacted by seasonal dynamics [29][18] - The company expects Europath to significantly contribute to revenue in 2025, with ongoing growth in both U.S. and international markets [16][28] Market Data and Key Metrics Changes - As of March 31, Europath was prescribed by over 1,000 unique prescribers for more than 1,750 patients in the U.S. [11] - The company estimates over 400,000 patients globally and around 70,000 in the U.S. are living with chronic hypoparathyroidism, with a significant portion being candidates for treatment with Europath [17] - Skytrofa holds approximately 7% market share in the total growth hormone market in the U.S. and around 43% in the non-active growth hormone market [18] Company Strategy and Development Direction - Ascendis Pharma aims to be a leading biopharma company, focusing on the global launch of Europath and the development of TransCon CNP for growth disorders [10][12] - The company is committed to expanding its product portfolio and addressing unmet medical needs in endocrinology and rare diseases [25] - Ascendis Pharma plans to submit an MAA for TransCon CNP in the EU in Q3 2025, with ongoing clinical trials to support its growth strategy [23][24] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong launch performance of Europath and its potential to become a multi-billion euro product [17] - The company anticipates substantial revenue growth driven by the global launch of Europath and continued contributions from Skytrofa [32] - Management highlighted the importance of favorable reimbursement dynamics and ongoing patient adherence as key factors for future success [41][42] Other Important Information - Ascendis Pharma recognized a non-cash gain of €33.6 million from its share of profit loss of associates due to the Visa IPO [31] - The company ended the first quarter with cash and cash equivalents totaling €518 million, down from €560 million at the end of 2024 [32] Q&A Session Summary Question: Expectations for reimbursement of Yorvapath - Management estimates that 17% to 18% of patients prescribed Yorvapath will ultimately get reimbursed, with ongoing positive trends in payer policies [39][40] Question: Split between U.S. and ex-U.S. revenue for Yorvapath - Management indicated steady growth in ex-U.S. markets, with expectations for acceleration in the second half of the year as more countries achieve full reimbursement [48] Question: Status of negotiations with commercial payers - Conversations with payers have been positive, with multiple favorable policies in place, and the company expects to continue gaining traction [54] Question: Proportion of new prescriptions from NATPARA or PTH naive patients - The majority of new patients are coming from conventional therapy, with about 10% to 15% having been on some form of PTH previously [58][62] Question: Depth of prescribing for Yorvapath - Management noted that they cannot definitively categorize patients as well-controlled or uncontrolled, but they are focusing on those frequently seeing endocrinologists [66][70] Question: Feedback on the titration process for Yorvapath - Adherence rates are consistent with clinical trials, and dropout rates are under 1%, indicating strong patient retention [100][101] Question: Plans for clinical utility trials for milder patients - Management is evaluating the long-term benefits of treatment for society and considering how to demonstrate the economic value of Yorvapath [108]