Ascendis Pharma A/S J.P. Morgan Healthcare Conference San Francisco January 2026 This presentation also contains estimates and other statistical data made by independent parties and by us relating to market size and other data about our industry. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such data and estimates. In addition, projections, assumptions and estimates of our future performance and the future performance of the markets in which w ...

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]