Ascendis Pharma FY Conference Summary Company Overview - **Company**: Ascendis Pharma (NasdaqGS:ASND) - **Event**: 46th Annual TD Cowen Healthcare Conference - **Date**: March 02, 2026 Key Points Product Launch and Approval - Ascendis Pharma received approval for **UVEVELL**, a new treatment for hypoparathyroidism, with a clean label and minimal adverse events [3][4] - The drug is designed for weekly administration and can be stored at room temperature for up to 6 months, differentiating it from competitors like **VOXZOGO** [3] - The launch is expected in early Q2 2026, pending final packaging information from the FDA [4] Market Potential - There are approximately **2,600 patients** diagnosed with hypoparathyroidism in the U.S., with only **30%** currently receiving treatment [4][8] - Ascendis aims to target both existing patients on other therapies and new patients who are currently untreated [9][10] - The company believes that a significant portion of patients will switch to **YORVIPATH** due to its benefits over existing treatments [9][10] Competitive Landscape - **VOXZOGO** has a known risk of blood pressure changes requiring hydration, while **YORVIPATH** does not have such stringent requirements, which may enhance its appeal [12][13] - Ascendis plans to price **YORVIPATH** at a premium, reflecting its superior benefits, but will also consider contracting with payers to maximize access [19][20] Patient Engagement and Market Penetration - Ascendis emphasizes a patient-focused approach, engaging with patient groups to understand their needs and improve treatment uptake [29][30] - The company believes that addressing comorbidities will drive treatment rates, with expectations that **70-80%** of patients will eventually seek treatment [33][34] Future Developments - Ascendis is also exploring a once-weekly version of **YORVIPATH**, which may require a different regulatory pathway for approval [44][45] - The company is focused on ensuring optimal patient journeys and minimizing dropouts during the treatment initiation phase [42][43] Financial Considerations - The company anticipates that the pricing strategy and patient support systems will help maintain demand and manage gross-to-net revenue dynamics [35][37] Conclusion - Ascendis Pharma is positioned to capitalize on the unmet needs in the hypoparathyroidism market with its innovative treatment **YORVIPATH**, leveraging its patient-centric approach and strategic pricing to enhance market penetration and patient outcomes [20][29][33]

Group 1 - Ascendis Pharma reported a nearly doubled total revenue of €720 million for 2025, driven by the global adoption of its endocrinology products [1][7] - The company achieved an operating profit of €10 million in Q4 and generated €73 million in operating cash flow, primarily from YORVIPATH and SKYTROFA [1][2] - Ascendis Pharma holds a 45% market share in the US long-acting growth hormone market for SKYTROFA, with plans for growth through the anticipated FDA approval of TransCon CNP for pediatric achondroplasia [3][4] Group 2 - Despite a full-year net loss of €228 million due to commercial investments, Ascendis Pharma ended the year with a strong cash position of €616 million, bolstered by a $100 million partnership payment from Novo Nordisk [2] - The company's proprietary TransCon technology transforms established drugs into long-acting prodrugs, ensuring predictable and sustained release of medication [2]

Core Insights - Ascendis Pharma is experiencing strong commercial growth for its products, particularly YORVIPATH and SKYTROFA, with significant revenue increases reported for Q4 2025 and full-year 2025 [3][4][7]. Commercial Performance - YORVIPATH generated €187 million in Q4 2025, contributing to a total of €477 million for the full year [3][7]. - SKYTROFA reported Q4 revenue of €53 million and full-year revenue of €206 million, with a U.S. market share of approximately 7% [11]. - The company has prescribed YORVIPATH to over 5,300 patients in the U.S. through nearly 2,400 healthcare providers, indicating a substantial long-term opportunity as less than 5% of U.S. patients are currently on treatment [2][7]. Financial Outlook - Ascendis ended 2025 with €616 million in cash and expects to achieve around €500 million in operating cash flow in 2026, with a long-term goal of at least €5 billion in annual product revenue by 2030 [5][20]. - Total revenue for Q4 2025 was €248 million, with operating expenses of €214 million, resulting in an operating profit of €10 million [17]. Pipeline and Regulatory Updates - The U.S. NDA for TransCon CNP is under review with a PDUFA date set for February 28, 2026, and the company anticipates full enrollment in its trial for infants with achondroplasia by late 2026 [6][13]. - Management highlighted the potential of a once-weekly TransCon PTH candidate, which aims to match the efficacy of daily therapies while improving patient convenience [8][12]. Market Expansion and Strategy - Ascendis plans to launch YORVIPATH in 10 additional countries in 2026, building on its existing commercial presence in over 30 countries [1]. - The company is also working to expand the label for SKYTROFA and is conducting trials to evaluate its growth hormone in additional indications, which could represent a significant market opportunity [10][11].

Financial Data and Key Metrics Changes - Revenue for Q4 2025 was EUR 248 million, with full-year 2025 revenue totaling EUR 720 million, reflecting strong growth [20][21] - Europat revenue for Q4 was EUR 187 million, up from EUR 140 million in Q3, with full-year revenue reaching EUR 477 million [20][21] - SKYTROFA generated EUR 53 million in Q4, contributing to a total of EUR 206 million for the full year [20][21] - Operating profit for Q4 2025 was EUR 10 million, with operating cash flow of EUR 73 million [21] Business Line Data and Key Metrics Changes - Europat's U.S. patient prescriptions exceeded 5,300 from nearly 2,400 healthcare providers, indicating strong demand [8] - SKYTROFA holds a market share of approximately 7% in the U.S. for growth hormone deficiency treatments [10] - The company is expanding the Europat label and working on additional doses and indications, including trials for patients under 18 [9][11] Market Data and Key Metrics Changes - The overall insurance approval rate for Europat in the U.S. is about 70%, with expectations for gradual improvement [8][35] - Europat is commercially available in over 30 countries, with full reimbursement in four European countries and two international markets [9] - The company anticipates full commercial launches in 10 additional countries in 2026 [9] Company Strategy and Development Direction - The company aims to achieve EUR 500 million in operating cash flow by 2026 and at least EUR 5 billion in annual product revenue by 2030 [7][17] - Ascendis Pharma is focused on expanding its pipeline of blockbuster products and enhancing its global commercial infrastructure [7][16] - The company plans to leverage partnerships to create additional value and accelerate product development [15][16] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth potential of Europat and SKYTROFA, citing strong clinical value propositions [35] - The company is optimistic about the upcoming approval of TransCon CNP and its potential impact on revenue [22][56] - Management highlighted the importance of patient awareness and advocacy in driving product uptake [48] Other Important Information - The company is advancing its clinical trials for TransCon CNP and SKYTROFA, with significant data supporting their efficacy [12][51] - Ascendis Pharma is committed to maintaining independence and profitability as it grows, focusing on long-term value creation [93] Q&A Session Summary Question: Confidence level heading into the TransCon CNP PDUFA - Management expressed high confidence in the approval of TransCon CNP, similar to previous product approvals [28] Question: Insurance approval rate for Europat - Management is satisfied with the current 70% approval rate and noted that reaching 100% is unlikely due to natural filtering in patient eligibility [32][35] Question: Europat pricing and net pricing trends - Management does not discuss net pricing but expects stable revenue growth based on prescription increases [42][43] Question: Usage of Europat among prescribers - There is broad uptake among prescribers, with some treating multiple patients, indicating growing confidence in the product [47] Question: Confidence in Europat growth outside the U.S. - Management noted that uptake varies by country, with different speeds of penetration based on local market conditions [60] Question: TransCon CNP launch expectations - Management anticipates a strong initial uptake for TransCon CNP, leveraging U.S. approval for international markets [56] Question: Strategy for commercializing TransCon CNP globally - The company has established infrastructure through Europat to support rapid global commercialization of TransCon CNP [70] Question: Contribution of products to EUR 5 billion target by 2030 - Management emphasized a diversified product portfolio across multiple indications and regions to achieve sustainable revenue growth [76]

Financial Data and Key Metrics Changes - Revenue for Q4 2025 was EUR 248 million, with full-year 2025 revenue totaling EUR 720 million, reflecting strong performance [22][23] - YORVIPATH revenue for Q4 was EUR 187 million, up from EUR 140 million in Q3, with full-year revenue reaching EUR 477 million [22] - SKYTROFA generated EUR 53 million in Q4, contributing to a total of EUR 206 million for the full year [22] - Operating profit for Q4 2025 was EUR 10 million, with operating cash flow of EUR 73 million [23] - The company ended 2025 with EUR 616 million in cash and cash equivalents, an increase from EUR 560 million at the end of 2024 [23] Business Line Data and Key Metrics Changes - YORVIPATH saw over 5,300 patients prescribed by nearly 2,400 unique healthcare providers in the U.S., indicating strong demand [8] - SKYTROFA maintained a market share of around 7% in the U.S. for pediatric growth hormone deficiency and adult growth hormone deficiency [11] - The company initiated a phase III basket trial for TransCon Growth Hormone in established growth hormone indications, expanding its market potential [12] Market Data and Key Metrics Changes - In the U.S., the insurance approval rate for YORVIPATH is approximately 70%, with expectations for gradual improvement [8][36] - YORVIPATH is commercially available in over 30 countries, with full reimbursement in four European countries and two international markets [9] - The company anticipates full commercial launches in 10 additional countries in 2026 [9] Company Strategy and Development Direction - The company aims to achieve operating cash flow of around EUR 500 million in 2026 and at least EUR 5 billion in annual product revenue by 2030, as part of its Vision 2030 strategy [7][19] - Ascendis Pharma is focused on expanding its pipeline of blockbuster product opportunities while strengthening its global commercial infrastructure [7][19] - The company plans to invest in label expansion for current products and develop new blockbuster opportunities in rare endocrine diseases [16] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term growth potential of YORVIPATH, citing a significant opportunity as less than 5% of U.S. patients are currently on treatment [8] - The company is optimistic about the upcoming approval of TransCon CNP and its potential impact on revenue [24][57] - Management highlighted the importance of partnerships and collaborations to create additional value and support the development of differentiated product opportunities [17] Other Important Information - TransCon Growth Hormone is now approved in the U.S. for both adult and pediatric growth hormone deficiency, with ongoing trials to expand its indications [5][12] - The company is advancing its once-weekly TransCon PTH candidate for hypoparathyroidism, with expectations for significant market impact [6][21] Q&A Session Summary Question: Confidence level heading into the TransCon CNP PDUFA - Management expressed high confidence in the approval of TransCon CNP, similar to previous assurances regarding TransCon PTH [30] Question: Insurance approval rate for YORVIPATH - Management is satisfied with the current 70% approval rate and noted that reaching 100% is unlikely due to natural filtering in patient eligibility [36][39] Question: YORVIPATH pricing and net pricing trends - Management refrained from discussing net pricing but indicated a focus on revenue growth as the product matures in the market [44][45] Question: Usage of YORVIPATH among prescribers - Management noted broad uptake among prescribers, with some treating multiple patients, and emphasized the importance of awareness among patients [50] Question: Update on the COACH Trial for achondroplasia - Management reported positive feedback from regulatory meetings regarding the trial data and readiness to initiate phase III trials [52][53] Question: TransCon CNP launch expectations - Management anticipates a strong initial uptake for TransCon CNP, leveraging existing approvals to facilitate entry into international markets [57] Question: Strategy for commercializing TransCon CNP globally - Management highlighted the established infrastructure from YORVIPATH as a foundation for rapid global commercialization of TransCon CNP [73]

Core Insights - Ascendis Pharma is entering a growth phase, aiming for approximately €500 million in operating cash flow in 2026 and at least €5 billion in global annual product revenue by 2030, driven by its TransCon platform and a growing pipeline of differentiated programs [2][3]. Financial Performance - Q4 2025 product revenue reached €240 million, with full-year 2025 product revenue totaling €684 million, reflecting significant growth compared to €364 million in 2024 [4][7]. - Operating profit for Q4 2025 was €10 million, with cash flow from operating activities amounting to €73 million [4][9]. - Total revenue for 2025 was €720 million, a substantial increase from €364 million in 2024, primarily due to the growth of YORVIPATH [6][7]. - The company reported a net loss of €34 million for Q4 2025, compared to a net loss of €38 million in Q4 2024, and a full-year net loss of €228 million, an improvement from €378 million in 2024 [13][22]. Product Highlights - YORVIPATH (TransCon PTH) generated €187 million in Q4 2025 and €477 million for the full year, with over 5,300 unique U.S. patient enrollments [5][8]. - TransCon CNP is under FDA Priority Review, with a PDUFA action goal date of February 28, 2026, and a marketing authorization application submitted to the EMA [4][5]. - SKYTROFA (TransCon hGH) revenue for Q4 2025 was €53 million, with full-year revenue of €206 million [5][8]. Research and Development - R&D expenses for Q4 2025 were €78 million, slightly down from €79 million in Q4 2024, with total R&D expenses for 2025 at €304 million [10]. - The company is conducting ongoing label expansion trials for TransCon PTH and has initiated a Phase 3 basket trial for additional indications [9][10]. Strategic Collaborations - Ascendis Pharma is engaged in a multi-product collaboration with Novo Nordisk for therapies based on TransCon technology, with the lead program, TransCon Semaglutide, expected to enter clinical trials [9]. - The company has also announced that YORVIPATH is now commercially available in Japan and received approval for pediatric growth hormone deficiency treatment in China [9].

Core Insights - Ascendis Pharma A/S is focused on developing treatments for unmet medical needs, with notable products including SKYTROFA for growth hormone deficiency and other treatments in its pipeline [1] Group 1: Stock Performance and Analyst Sentiment - The consensus price target for Ascendis Pharma's stock has increased from $270.50 a year ago to $307.33 in the last month, indicating growing optimism among analysts [2] - Bank of America Securities analyst Tazeen Ahmad has set a conservative price target of $161, reflecting some caution in the market [2] Group 2: Financial Performance - Ascendis Pharma reported Q3 revenues of €213.6 million, nearly four times higher than the previous year, driven by Yorvipath and SKYTROFA [3][5] - The company achieved operating profitability for the quarter, indicating strong financial performance [3] Group 3: Upcoming Events and Growth Prospects - The FDA PDUFA date for TransCon CNP on February 28th is critical, as approval could open a multi-billion-euro market for the company [4][5] - Ascendis Pharma has a solid cash position of €539 million, which supports continued investment in its pipeline and reduces near-term dilution risk [4]

Core Viewpoint - The approval of the long-acting growth hormone, Rongpei (SKYTROFA), by the National Medical Products Administration marks a significant advancement in the treatment of growth hormone deficiency in children aged 3 and above in China [1]. Group 1: Product Approval - Weisheng Pharmaceutical has received approval for the marketing authorization of Rongpei growth hormone in China [1]. - The product is specifically indicated for treating growth retardation due to growth hormone deficiency in children and adolescents [1]. Group 2: Product Characteristics - Rongpei is a weekly administered growth hormone replacement therapy, distinguishing itself as the only long-acting growth hormone that has demonstrated superior efficacy and equivalent safety compared to short-acting daily injections in positive parallel-controlled trials [1]. - The drug has successfully completed Phase III pivotal trials in China [1].

Core Viewpoint - The approval of Lonapegsomatropin (SKYTROFA) by the National Medical Products Administration of China marks a significant advancement in the treatment of pediatric growth hormone deficiency, providing a long-acting alternative to daily injections [1][2]. Group 1: Product Approval and Efficacy - Lonapegsomatropin has received a Biologics License Application (BLA) approval for treating growth hormone deficiency in children aged 3 years and older in China [1]. - The drug has demonstrated superior efficacy in a Phase 3 trial, showing an annualized height velocity of 10.66 cm/year compared to 9.75 cm/year for daily injections, with a statistically significant difference of 0.91 cm/year (P=0.0010) [2]. - The height standard deviation score (SDS) increased by 1.01 in the Lonapegsomatropin group versus 0.83 in the daily injection group, with a p-value of 0.0015, indicating significant improvement from week 13 of treatment [2]. Group 2: Commercialization Strategy - Following the BLA approval, the company has initiated a comprehensive commercialization plan to ensure supply and expand market coverage [3]. - A commercial supply agreement has been established with Ascendis Pharma to secure the availability of Lonapegsomatropin upon market launch [3]. - The company is enhancing its commercialization capabilities through a dedicated team and tailored strategies for market access and physician awareness, alongside strategic partnerships with Shanghai Pharmaceuticals, United Family Healthcare, and Anke Bioengineering for distribution and promotion [3].

Core Viewpoint - Ascendis Pharma is participating in the 44th Annual JPMorgan Healthcare Conference, highlighting its focus on commercialization and development of its products, particularly SKYTROFA and YORVIPATH [1][3]. Group 1: Company Overview - The CEO of Ascendis Pharma, Jan Mikkelsen, is set to present at the conference, indicating the company's commitment to engaging with investors and stakeholders [1]. - Scott Smith, the CFO, has initiated the conference call, emphasizing the importance of forward-looking statements for the company's strategic direction [2][3]. Group 2: Forward-Looking Statements - The conference will include forward-looking statements related to commercialization, development expectations for SKYTROFA and YORVIPATH, and revenue growth projections [3]. - Statements will also cover pipeline candidates, their associated costs, and expectations regarding clinical trials and regulatory filings [3].