Ascendis Pharma (NasdaqGS:ASND) FY Conference March 02, 2026 11:10 AM ET Company ParticipantsJan Møller Mikkelsen - President and CEOConference Call ParticipantsYaron Werber - Managing Director and Senior Biotechnology AnalystYaron WerberWell, welcome everybody to the 46th annual TD Cowen Healthcare Conference. I'm Yaron Werber from the biotech team, and it's a great pleasure to moderate the literally virtual fireside chat with Ascendis on the heels of the YUVIWEL approval. We moved the participation to vir ...

Summary of Ascendis Pharma Conference Call Company Overview - **Company**: Ascendis Pharma (NasdaqGS:ASND) - **Product**: YUVIWEL, a treatment for achondroplasia - **FDA Approval**: YUVIWEL is the third FDA-approved drug from Ascendis Pharma, with plans for commercial launch in the U.S. in Q2 2026 [5][34] Key Points and Arguments FDA Approval and Product Details - YUVIWEL is the first and only once-weekly treatment for children with achondroplasia, utilizing TransCon technology to extend the half-life of C-type natriuretic peptide (CNP) from minutes to 5-6 days [6][7] - The FDA approval was based on data from three randomized double-blind placebo-controlled trials, including the pivotal ApproaCH Trial [7][8] - A Rare Pediatric Disease Priority Review Voucher was granted alongside the approval, allowing for priority review of a future drug application [8] Clinical Efficacy - In the pivotal ApproaCH Trial, children treated with YUVIWEL achieved an annualized growth velocity (AGV) of 5.9 cm/year compared to 4.4 cm/year for placebo, with a statistically significant treatment difference of 1.5 cm/year [20] - The treatment demonstrated improvements in height Z-scores across all predefined subgroups, including age, sex, and geographic region [21] Market Opportunity - The U.S. pediatric achondroplasia prevalence is estimated at around 2,600, with approximately 30% currently on pharmacological treatment [26] - There is a significant unmet need in the market, with many patients either not on treatment or having discontinued previous therapies due to tolerability or perceived lack of benefit [27] Commercial Strategy - Ascendis Pharma plans to engage with centers of excellence, thought leaders, and patient advocacy groups to educate stakeholders on YUVIWEL's clinical value proposition [28] - The company aims to optimize the patient experience and ensure affordability through programs that may allow eligible patients to pay as little as $0 a month [30] - Initial uptake is expected to come from both switches from existing therapies and treatment-naive patients [47] Future Plans - Ascendis Pharma has a roadmap to achieve $5 billion in product revenue by 2030, focusing on expanding the label for existing products and developing new therapies [14] - Plans include exploring indications for hypochondroplasia and adult patients with achondroplasia [70] Safety and Administration - YUVIWEL is designed to minimize injection site reactions and does not require food intake before administration, which is seen as a meaningful differentiation from existing therapies [22][55] - The product can be stored at room temperature for up to 6 months, enhancing convenience for patients [24] Additional Important Information - The company has a strong existing infrastructure from previous product launches, which will be leveraged for YUVIWEL [59] - Pricing for YUVIWEL has not yet been disclosed, but a premium pricing strategy is anticipated due to the product's differentiated benefits [45][46] - The company is prepared for potential reimbursement challenges and is engaging with payers to facilitate access [76] This summary encapsulates the critical aspects of the conference call, highlighting the company's strategic direction, product details, market potential, and future plans.

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].

Ascendis Pharma (NasdaqGS:ASND) Q4 2025 Earnings call February 11, 2026 04:30 PM ET Company ParticipantsAlex Thompson - Managing DirectorChad Fugere - VP of Investor RelationsDerek Archila - Managing Director and Co-Head of Therapeutics ResearchHeidi Jacobson - Equity Research AssociateJan Møller Mikkelsen - President and CEOJay Wu - EVP and President of US MarketLeland Gershell - Managing DirectorMaxwell Skor - VP in Biotech Equity ResearchScott Smith - CFOConference Call ParticipantsDaniel Brander - Equit ...

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 Viewpoint - OPKO Health and Entera Bio are expanding their collaboration to develop innovative oral peptide therapies, including a long-acting PTH analog for hypoparathyroidism and an oral oxyntomodulin for metabolic disorders, with IND applications expected in late 2026 [3][4][5]. Group 1: Collaboration and Development Programs - The collaboration between OPKO and Entera aims to advance the first oral long-acting PTH analog (LA-PTH) as a once-daily tablet for hypoparathyroidism, with an IND application planned for late 2026 [3][4]. - The companies are also developing an injectable and oral oxyntomodulin (OXM) for metabolic and fibrotic disorders, with initial Phase 1 data from the injectable form expected in late 2026 [1][8]. - Under the expanded collaboration agreement, both companies will hold a 50% ownership interest in the LA-PTH program and share development costs equally, while maintaining a 60%/40% ownership structure for the oral OXM program [5]. Group 2: Key Personnel Changes - Steve Rubin, an industry veteran with extensive experience in corporate governance and drug development, has joined Entera's board of directors, replacing Gerry Ostrov [2][6]. Group 3: Clinical Data and Market Potential - Preclinical data for the oral LA-PTH program suggests it could transform treatment for hypoparathyroidism, which currently relies on daily injections of the only approved PTH replacement therapy [7]. - Entera's previous clinical data for its EB612 program indicated significant reductions in calcium supplement use and maintenance of serum calcium levels, although the regimen required multiple daily doses [7]. - The oral oxyntomodulin is positioned as a dual GLP-1/glucagon agonist with potential benefits for obesity and metabolic disorders, with no approved dual agonists currently available [8].

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].

Core Insights - Ascendis Pharma is rapidly transforming into a leading global biopharma company, driven by its TransCon technology platform, strong R&D capabilities, and expanding commercial infrastructure [2][3] Business and Strategic Roadmap - The company plans to present its business and strategic roadmap, including key corporate milestones for 2026, at the J.P. Morgan Healthcare Conference on January 12, 2026 [1][6] - Ascendis aims for further global penetration and commercial expansion, with a third potential TransCon product approval expected to have blockbuster potential [2] Key Updates and Milestones - TransCon PTH (YORVIPATH) is expected to generate approximately €187 million in Q4 2025 and €477 million for the full year 2025, with over 5,300 unique U.S. patient enrollments [3] - TransCon hGH (SKYTROFA) is projected to have revenues of around €53 million in Q4 2025 and €206 million for the full year 2025, with FDA approval for adult growth hormone deficiency received in July 2025 [3] - TransCon CNP has a PDUFA goal date of February 28, 2026, for pediatric achondroplasia, with a marketing authorization application submitted to the EMA [3] - The combination therapy of TransCon CNP and TransCon hGH showed significant improvements in growth velocity in the Phase 2 COACH Trial [3] Financial Update - Ascendis reported an estimated total product revenue of approximately €683 million for 2025, with a gross margin expected to be around 87% [4] - The company has authorized a $120 million share repurchase program, with a preliminary cash balance of approximately €616 million as of December 31, 2025 [4] Strategic Collaborations - Ascendis has ongoing collaborations with Novo Nordisk for therapies in obesity and metabolic diseases, and with Eyconis, VISEN Pharmaceuticals, and Teijin Limited for various TransCon products [5]