Core Viewpoint - Ascendis Pharma A/S announced that the FDA has extended the PDUFA target action date for TransCon CNP by three months to February 28, 2026, due to a major amendment related to post-marketing requirements [1]. Group 1: FDA Interaction - The company has responded to all outstanding FDA requests, including a revised protocol for the post-marketing study, and is committed to working diligently with the FDA to finalize these elements [2]. - The extension of the PDUFA date indicates ongoing regulatory review and the need for additional information from the company [1]. Group 2: Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform to address unmet medical needs [3]. - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [3].

Financial Data and Key Metrics Changes - In Q3 2025, Ascendis Pharma achieved total revenue of €214,000,000, up from €103,000,000 in Q2 2025, with a strong contribution from Europad and Skytropha [25][26] - Operating profit for Q3 2025 was €11,000,000, indicating a positive operating income development [25][26] - Cash and cash equivalents increased to €539,000,000 from €494,000,000 at the end of Q2 2025 [28] Business Line Data and Key Metrics Changes - Europad generated revenue of €143,000,000 in Q3 2025, showing strong growth in patient prescriptions and prescribers [9][25] - Skytropha contributed €50,700,000 in revenue, with a 3% growth in demand despite a foreign currency headwind [25][26] - Collaboration revenue included €20,000,000 driven by a milestone related to Europad [26] Market Data and Key Metrics Changes - In the U.S., over 4,250 patients have been prescribed Europad by more than 2,000 unique healthcare providers, indicating strong demand [10][12] - The overall insurance approval rate for Europad is around 70%, with most approvals occurring within eight weeks [12][28] - Europad is now commercially available in over 30 countries, with full reimbursement in Germany, Austria, and Spain [15] Company Strategy and Development Direction - The company aims to achieve €5,000,000,000 or more in annual product revenue by 2030, focusing on expanding its commercial organization and product offerings [23] - Ascendis Pharma is advancing its TransCon technology platform to ensure a continuous flow of new programs and products [23] - The company is also pursuing partnerships to enhance its market presence, particularly in Japan and China [22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the stability of prescription rates and patient retention, indicating a strong foundation for future growth [33] - The company anticipates continued revenue growth driven by new patient enrollments and stable pricing in Q4 2025 [28] - Management noted that the competitive landscape does not pose significant threats to Europad's market position due to its unique properties [72] Other Important Information - TransCon CNP is under priority review by the FDA, with a PDUFA date of November 30, 2025, and is expected to become a leading treatment for children with achondroplasia [19][20] - The company is conducting a clinical trial for higher doses of TransCon PTH, which is expected to enhance treatment options for patients [100] Q&A Session Summary Question: Expectations for new patient enrollments on YorbaPath in the U.S. - Management indicated stable prescription rates and positive trends in new patient enrollments, with a strong foundation for continued growth [32][33] Question: Impact of seasonality on Q4 performance - Management does not expect significant seasonality impacts in Q4, citing a stable prescription environment [40] Question: Conversion rate for YorbaPath and future expectations - Management expects the conversion rate to improve over time, with a current approval rate of 70% [44][48] Question: Progress in different segments of the hypoparathyroidism market - Management noted strong traction among highly symptomatic patients, while emphasizing the need for patient education in less aware segments [61][63] Question: Commercial strategy for TransCon CNP - Management anticipates a significant portion of revenue will come from switching patients from existing therapies, particularly in markets with high treatment penetration [96][97]

Core Insights - Ascendis Pharma reported Q3 2025 revenue of €213.6 million, a significant increase from €57.8 million in Q3 2024, driven primarily by the strong performance of YORVIPATH and SKYTROFA [6][12] - The company is progressing towards its Vision 2030 goals, with ongoing global launches and positive feedback from healthcare providers and patients [2][4] - Ascendis Pharma achieved an operating profit of €11.0 million in Q3 2025, attributed to higher revenues from YORVIPATH, despite increased operating expenses [11][10] Financial Performance - YORVIPATH revenue for Q3 2025 was €143.1 million, reflecting a negative foreign currency impact of €3.6 million compared to the previous quarter [5][6] - SKYTROFA revenue for Q3 2025 totaled €50.7 million, also impacted by a negative foreign currency effect of €1.6 million [5][6] - Total revenue from commercial products reached €193.8 million in Q3 2025, compared to €55.7 million in Q3 2024 [9][10] Product Development and Regulatory Updates - TransCon CNP is currently under FDA Priority Review for treating children with achondroplasia, with a PDUFA date set for November 30, 2025 [4][8] - The company plans to initiate a Phase 3 trial for TransCon CNP in combination with TransCon hGH following a successful end-of-Phase 2 meeting with the FDA [8][4] - Ascendis Pharma presented new analyses from the ApproaCH Trial at the ASBMR annual meeting, showing improvements in physical functioning for children treated with TransCon CNP [8] Operational Highlights - As of September 30, 2025, Ascendis Pharma had cash and cash equivalents of €539 million, a slight decrease from €560 million at the end of 2024 [13][20] - The company reported a net loss of €61.0 million for Q3 2025, an improvement from a net loss of €99.2 million in Q3 2024 [12][19] - Research and development costs decreased to €66.9 million in Q3 2025 from €73.5 million in the same period in 2024, reflecting the completion of clinical trials [10][19]

Core Insights - Ascendis Pharma announced positive data on TransCon PTH, showing sustained improvements in renal function and safety in adults with hypoparathyroidism through Year 3 of the Phase 2 PaTH Forward and Phase 3 PaTHway trials [1][2] - The data will be presented at Kidney Week 2025, aiming to enhance awareness of TransCon PTH and its role in kidney health [2] Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform, headquartered in Copenhagen, Denmark [4] - The company aims to address unmet medical needs and improve patient outcomes through its product innovations [4] Product Information - TransCon PTH is a prodrug of parathyroid hormone (PTH), designed for once-daily administration to maintain physiological PTH levels for 24 hours [2] - It is approved as YORVIPATH® in the U.S., EU, and EEA for treating adults with hypoparathyroidism [2] Disease Context - Hypoparathyroidism is characterized by insufficient PTH levels, leading to severe complications such as renal issues and neuromuscular irritability [3] - The majority of cases (70-80%) are post-surgical, with other causes being autoimmune and idiopathic [3]

Core Insights - Ascendis Pharma has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for TransCon CNP as a treatment for children with achondroplasia, a rare genetic condition impacting skeletal development and quality of life [1][2] - TransCon CNP is designed to provide continuous inhibition of the overactive FGFR3 pathway, administered once weekly to enhance the treatment's effectiveness [1][2] - The MAA is supported by data from three randomized clinical trials, including the pivotal ApproaCH Trial, demonstrating that TransCon CNP has been generally well tolerated [2] Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform, aiming to address unmet medical needs [5] - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [5] Industry Context - Achondroplasia affects over 250,000 individuals worldwide and is associated with various medical complications beyond skeletal dysplasia, including muscular, neurological, and cardiorespiratory issues [4] - The need for early pharmacological interventions in the achondroplasia community has been emphasized, highlighting the importance of treatments like TransCon CNP [3]

Core Insights - Ascendis Pharma A/S announced three oral presentations of data related to achondroplasia and hypoparathyroidism at ASBMR 2025, highlighting the effectiveness of their therapies [1][2] Group 1: Achondroplasia - Achondroplasia is a rare genetic condition affecting over 250,000 people globally, leading to various complications beyond skeletal dysplasia, including muscular and neurological issues [3] - The company presented new analyses correlating improvements in leg bowing with enhanced physical functioning in children treated with TransCon® CNP [1] - The first medical congress presentation of clinical trial data for children treated with TransCon CNP and TransCon hGH combination therapy was also shared [1] Group 2: Hypoparathyroidism - Hypoparathyroidism is characterized by insufficient parathyroid hormone levels, leading to severe complications, with post-surgical cases accounting for 70-80% of instances [4] - Clinical trial data demonstrated sustained improvements in skeletal dynamics in adults treated for four years with TransCon PTH [1][2] Group 3: Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform to address unmet medical needs [5] - The company emphasizes its commitment to patient care, scientific innovation, and passion in its operations [5]

Core Insights - Ascendis Pharma A/S announced two oral presentations at ENDO 2025, showcasing data on the safety and efficacy of its treatments for hypoparathyroidism and achondroplasia [1][2] Group 1: Hypoparathyroidism - Dr. Aliya Khan will present data from Week 156 of the Phase 3 PaTHway Trial, highlighting the long-term safety and efficacy of TransCon PTH (palopegteriparatide) in adults with hypoparathyroidism [1][2] - The presentation will cover maintenance of serum and urine biochemistries within normal levels and sustained improvement in renal function [1] Group 2: Achondroplasia - Dr. Carlos Bacino will present safety and tolerability data from Week 52 of the pivotal ApproaCH Trial of once-weekly TransCon CNP (navepegritide) in children with achondroplasia [1][2] - The data is expected to support the transformative potential of the investigational therapy for this rare genetic condition [2] Group 3: Company Overview - Ascendis Pharma is focused on applying its innovative TransCon technology platform to develop therapies that address unmet medical needs [5] - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [5]

Core Insights - Ascendis Pharma A/S will present new data on its hypoparathyroidism, achondroplasia, and growth hormone deficiency programs at the ESPE & ESE 2025 congress in Copenhagen from May 10-13, 2025 [1][2] Hypoparathyroidism - The company will showcase 4-year efficacy and safety data from the Phase 2 PaTH Forward Trial of TransCon PTH (palopegteriparatide) in adults with chronic hypoparathyroidism [2][4] - Presentations will include a retrospective matched cohort study using real-world data from England to estimate the risk of chronic kidney disease progression in chronic hypoparathyroidism [4][6] Achondroplasia - Ascendis will present Week 52 growth and bone morphometry data from the pivotal ApproaCH Trial of TransCon CNP (navepegritide) in children with achondroplasia [2][4] - The congress will feature discussions on assessing health-related quality of life (HRQoL) in achondroplasia across the life course [4][6] Growth Hormone Deficiency - The company will present results from the Phase 3 foresiGHt Trial supporting the efficacy and safety of once-weekly lonapegsomatropin in adults with growth hormone deficiency [2][6] - The presentations will highlight the importance of growth hormone in maintaining normal body composition and cardiometabolic health throughout adulthood [9] Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform to address unmet medical needs [10] - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [10]

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]

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]