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]

Summary of Ascendis Pharma (ASND) Conference Call Company and Industry Overview - **Company**: Ascendis Pharma - **Industry**: Biotechnology, specifically focusing on treatments for growth disorders such as achondroplasia Key Findings from the COAST Trial - The COAST trial is a Phase II study assessing the efficacy of a combination therapy involving TransCon CNP and TransCon Growth Hormone in children with achondroplasia aged 2 to 11 years [2][3] - The trial reported a significant increase in annualized growth velocity (AGV) for treatment-naive children, achieving 9.14 cm/year, which is an increase of 4.2 cm/year compared to baseline [19][20] - For children previously treated with TransCon CNP, the AGV was 8.25 cm/year, reflecting a 3.1 cm/year increase from baseline [21][22] Efficacy and Safety Data - The combination therapy demonstrated a 0.53 increase in height Z score for treatment-naive patients and a 0.44 increase for treatment-experienced patients after 26 weeks [20][22] - The safety profile of the combination therapy was comparable to that of the individual therapies, with no serious adverse events related to the study drug reported [17][18] - The combination therapy resulted in a linear growth improvement associated with better body proportionality, which is crucial for children with achondroplasia [24][25] Future Development Plans - Ascendis Pharma plans to initiate a Phase III trial by the end of 2025, which will include both treatment-naive and experienced children [26][30] - The company aims to expand its treatment options for growth disorders and explore over 20 potential indications for its therapies [27][84] Market Position and Strategy - Ascendis Pharma is positioned as a leader in the growth disorder treatment market, with a focus on developing unique treatment combinations that unlock new therapeutic potentials [27][84] - The company is considering co-formulating TransCon CNP and TransCon Growth Hormone into a single injection for improved patient compliance [45][103] Additional Insights - The trial results indicate a potential for achieving catch-up growth in children with achondroplasia, which could normalize their height relative to parental expectations [80] - The combination therapy is expected to provide significant benefits beyond linear growth, addressing broader health concerns associated with achondroplasia [81][96] Conclusion - Ascendis Pharma's recent trial results highlight a promising advancement in the treatment of achondroplasia, with significant improvements in growth velocity and safety. The company is poised to expand its market presence and explore additional indications for its therapies, reinforcing its commitment to addressing growth disorders effectively.

Combination treatment with investigational TransCon CNP and TransCon hGH is investigational. For investor communication only. Not for use in product promotion. Not for further distribution. COACH Trial Interim Topline Week 26 Data June 9, 2025 Cautionary Note on Forward-Looking Statements 2 Combination treatment with investigational TransCon CNP and TransCon hGH is investigational. For investor communication only. Not for use in product promotion. Not for further distribution. Executive Summary This present ...

Core Insights - The FDA has accepted Ascendis Pharma's New Drug Application for TransCon CNP for treating children with achondroplasia, granting it Priority Review status with a PDUFA goal date of November 30, 2025 [1][2] - TransCon CNP has shown significant annualized growth velocity and multiple benefits beyond linear growth in clinical trials compared to placebo, with a safety profile similar to that of placebo [1][2] - Achondroplasia affects over 250,000 people globally and leads to various complications, necessitating new treatment options [3] Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform to address unmet medical needs [4][5] - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [5] Industry Context - Achondroplasia is a rare genetic condition caused by a variant in the FGFR3 gene, leading to serious complications beyond skeletal dysplasia, including muscular, neurological, and cardiorespiratory issues [3] - Current therapies for achondroplasia are limited, and there is a significant demand for new treatments that can improve health outcomes [2][3]

Core Insights - Ascendis Pharma reported Q1 2025 revenue of €101.0 million, an increase from €95.9 million in Q1 2024, driven by strong product sales, particularly from YORVIPATH and SKYTROFA [7][11] - The company anticipates significant milestones in 2025, including the submission of a New Drug Application (NDA) for TransCon CNP and a Marketing Authorisation Application (MAA) for the same product in Europe [3][5] - Ascendis Pharma's CEO highlighted the potential for cash flow breakeven in the near term, supported by the successful launch of YORVIPATH and upcoming clinical trial data [3][5] Financial Performance - YORVIPATH generated €44.7 million in revenue for Q1 2025, with over 1,750 prescriptions written and more than 1,000 unique prescribing healthcare providers [5] - SKYTROFA revenue for Q1 2025 was €51.3 million, with a PDUFA goal date set for July 27, 2025, for FDA review of its supplemental BLA [5][7] - Total operating expenses for Q1 2025 were €187.6 million, up from €137.5 million in Q1 2024, with significant increases in research and development costs and selling, general, and administrative expenses [9][10] Future Developments - The topline data from the COACH trial, evaluating the combination of TransCon CNP and TransCon hGH, is expected in Q2 2025 [5][7] - Ascendis Pharma plans to submit an IND application for additional indications of TransCon hGH in Q3 2025 and for TransCon CNP in combination with TransCon hGH in Q4 2025 [5][7] - The company holds a significant equity position in VISEN Pharmaceuticals, valued at approximately €260 million as of March 31, 2025 [6][12]