Core Insights - Ascendis Pharma A/S plans to report its third quarter 2025 financial results and provide a business update on November 12, 2025, after U.S. market close [1] - A conference call and live webcast will be held on the same day at 4:30 p.m. Eastern Time to discuss the financial results [1][2] Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on its innovative 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]

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]

Regulatory Overview - The FDA has approved 34 novel drugs from January to October 2025, compared to 38 during the same period in 2024 [2] - Several biotech stocks are highlighted as they approach key regulatory decisions in November 2025 [2] Arrowhead Pharmaceuticals Inc. (ARWR) - ARWR's New Drug Application for Plozasiran is under FDA review, with a decision expected on November 18, 2025 [3] - Plozasiran targets familial chylomicronemia syndrome, a condition characterized by extremely high triglyceride levels, which can lead to acute pancreatitis [3] - Clinical trials showed significant reductions in triglycerides and the incidence of acute pancreatitis, potentially providing a new treatment option alongside Tryngolza, the only FDA-approved drug for this condition [4] Otsuka Holdings Co. Ltd. (OTSKF) - OTSKF is awaiting the FDA's decision on Sibeprenlimab, scheduled for November 28, 2025 [6] - Sibeprenlimab is an investigational monoclonal antibody for treating immunoglobulin A nephropathy (IgAN), a chronic kidney disease [6] - Clinical trials demonstrated a significant reduction in urine protein-to-creatinine ratio, indicating its potential as a blockbuster drug if approved [8] Bayer (BAYRY) - Bayer's investigational compound Sevabertinib is under priority review by the FDA, with a decision expected on November 28, 2025 [9] - Sevabertinib is proposed for treating advanced non-small cell lung cancer (NSCLC) with HER2 mutations, providing an additional option for previously treated patients [10] Ascendis Pharma A/S (ASND) - The FDA decision on Navepegritide, aimed at treating children with achondroplasia, is due on November 30, 2025 [11] - Achondroplasia is a rare genetic condition affecting over 250,000 people globally, leading to serious complications [12] - Navepegritide is designed for weekly subcutaneous administration, targeting the overactive FGFR3 pathway [13] Kura Oncology Inc. (KURA) - KURA is expecting FDA approval for Ziftomenib on November 30, 2025, for treating relapsed or refractory acute myeloid leukemia (AML) with NPM1 mutations [15] - Ziftomenib has shown significant efficacy in clinical trials, with a market opportunity estimated at $350 million to $400 million annually in the U.S. if approved [17]

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]

Group 1 - Ascendis Pharma A/S (NASDAQ:ASND) is recognized as one of the top biotech stocks to invest in, with UBS reaffirming a price target of $307 and a Buy rating on September 23 due to MBX's clinical study for treating persistent hypoparathyroidism [1][2] - Yorvipath shows higher placebo-adjusted response rates of 52.9% at 4 weeks and 69% at 26 weeks in clinical trials, indicating that Ascendis Pharma's medication remains a leader in the hypoparathyroidism treatment market despite competition [2] - UBS estimates the hypoparathyroidism market to be over $10 billion, suggesting significant sales potential for Yorvipath in the short and medium term, while the MBX data release alleviates concerns for Ascendis Pharma [2][3] Group 2 - Ascendis Pharma A/S is a biopharmaceutical company focused on developing and distributing innovative treatments for unmet medical needs, particularly in oncology and endocrinology [3]

Financial Data and Key Metrics Changes - The company has achieved revenue generation that is increasing quarter by quarter, indicating a positive cash contribution [3] - The financial independence from the market is highlighted, with consistent revenue growth expected [3] Business Line Data and Key Metrics Changes - The YORVIPATH launch is progressing well, with a potential peak sales opportunity of $5 billion in the hypoparathyroidism market [4][10] - The company is focusing on label expansion and developing new compounds based on TransCon technology [3] Market Data and Key Metrics Changes - In the U.S., there are approximately 80,000 to 100,000 diagnosed patients with hypoparathyroidism, indicating a significant unmet medical need [5] - The company has established a diversified strategy for ex-U.S. markets, with plans to expand into 14-15 countries by 2026 [7][8] Company Strategy and Development Direction - The company aims to develop three independent product opportunities addressing major unmet medical needs [2] - There is a focus on building a strong commercial infrastructure and expanding into cardiovascular diseases [44] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term growth of YORVIPATH, with a steady increase in prescriptions expected as the launch progresses [12][14] - The company is preparing for the FDA's priority review of TransCon CNP, which is expected to differentiate itself from competitors [22][25] Other Important Information - The partnership with Novo Nordisk remains strong, with no changes impacting the collaboration [36] - The company is exploring new indications and targets for the TransCon platform, with a focus on rare diseases [43] Q&A Session Summary Question: What drives the uptake of YORVIPATH in the U.S.? - The uptake is driven by a steady flow of new prescribers and prescriptions, with improvements expected in the transfer process from prescription to treatment [11][12] Question: How does the company view competition in the market? - The competitive landscape is clearing, with no other products in clinical development providing the same benefits as YORVIPATH [6][15] Question: What is the differentiation of TransCon CNP compared to competitors? - TransCon CNP offers continuous exposure and reduced injection site reactions, providing a significant clinical benefit beyond linear growth [23][24] Question: What is the company's strategy for the TransCon platform? - The company plans to expand its pipeline significantly, aiming for multiple clinical trials and product opportunities in the next few years [50][43] Question: How does the company view its cost base and earnings power moving forward? - The company has been cost-efficient and is looking to invest more in its pipeline while generating cash from operations [45][51]

Financial Data and Key Metrics Changes - The company is experiencing increasing revenue generation quarter by quarter, indicating a positive cash contribution and independence from financial markets [3][4] - The peak sales potential for YORVIPATH is estimated at $5 billion, with a significant portion expected from the U.S. in the initial years before shifting to ex-U.S. markets [10][11] Business Line Data and Key Metrics Changes - YORVIPATH is positioned as a replacement therapy for hypoparathyroidism, with strong intellectual property protection extending to 2042, and a stable growth trajectory observed since its launch [4][6] - The company has a diversified strategy for ex-U.S. markets, with direct market efforts in 14 countries and partnerships in others, including Japan [7][8] Market Data and Key Metrics Changes - In the U.S., there are approximately 80,000 to 100,000 diagnosed patients with hypoparathyroidism, indicating a substantial unmet medical need [5] - The competitive landscape for YORVIPATH is clearing, with no other products in clinical development offering similar benefits [6][7] Company Strategy and Development Direction - The company aims to develop three independent product opportunities addressing major unmet medical needs, leveraging its TransCon technology [2][3] - Future plans include label expansion for existing products and the development of new compounds targeting both rare diseases and broader indications [3][42] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing growth of YORVIPATH and the potential for TransCon CNP, with a priority review by the FDA expected soon [21][29] - The company is focused on building a sustainable pipeline and expanding its commercial infrastructure, with a goal of increasing clinical trial efforts in the coming years [42][44] Other Important Information - The partnership with Novo Nordisk remains strong, with no changes impacting the collaboration on TransCon semaglutide [35] - The company is exploring new therapeutic areas, including cardiovascular diseases, while continuing to focus on rare disease endocrinology [43][44] Q&A Session Summary Question: What drives the uptake of YORVIPATH in the U.S.? - The uptake is driven by a steady flow of new prescribers and prescriptions, with improvements expected in the infrastructure for patient transfers from prescription to treatment [11][12] Question: How does the company differentiate its products from competitors? - The differentiation lies in the continuous exposure provided by TransCon technology, which minimizes injection site reactions and maintains stable hormone levels [22][24] Question: What is the company's strategy for TransCon CNP? - The strategy involves positioning TransCon CNP as a transformative treatment for achondroplasia, focusing on both naive and switch patients [26][29] Question: How does the company view its cost base and earnings power moving forward? - The company has maintained cost efficiency and is looking to expand its pipeline, with expectations of turning a corner in cash generation in the near future [44][50]

Financial Data and Key Metrics Changes - The company has achieved revenue generation that is increasing quarter by quarter, indicating a positive cash contribution [3] - The peak sales potential for YORVIPATH is estimated at $5 billion, with a significant portion expected from the U.S. in the first four to five years [10] Business Line Data and Key Metrics Changes - YORVIPATH is positioned as a replacement therapy for hypoparathyroidism, with strong intellectual property protection extending to 2042 [4] - The company has a diversified strategy for ex-U.S. markets, with direct market efforts in 13-14 countries and plans to expand further [7] Market Data and Key Metrics Changes - In the U.S., there are approximately 80,000 to 100,000 diagnosed patients with hypoparathyroidism, highlighting a significant unmet medical need [5] - The competitive landscape for YORVIPATH is clearing, with no other products in clinical development offering similar benefits [6] Company Strategy and Development Direction - The company aims to develop three independent product opportunities addressing major unmet medical needs, leveraging its TransCon technology [2] - Future plans include label expansion and the development of new compounds targeting both rare diseases and broader indications [3][42] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's ability to generate positive cash flow and remain independent of financial markets [3] - The company is focused on building a sustainable growth model through its pipeline and partnerships, particularly in rare diseases and metabolic disorders [43] Other Important Information - The company is preparing for the FDA's priority review of TransCon CNP, which is expected to differentiate itself from competitors like BioMarin's VOXZOGO [21] - The partnership with Novo Nordisk remains strong, with no changes impacting the collaboration on TransCon semaglutide [35] Q&A Session Summary Question: What drives the uptake of YORVIPATH in the U.S.? - The uptake is driven by a steady flow of new prescribers and prescriptions, with improvements expected in the infrastructure for patient transfers from prescription to treatment [11][12] Question: How does the company view competition for longer-acting formulations? - The company has opted for a daily formulation for YORVIPATH to ensure patient stability before considering a weekly product [15][16] Question: What is the differentiation of TransCon CNP compared to competitors? - TransCon CNP offers continuous exposure without injection site reactions and has shown meaningful clinical effects beyond linear growth [22][24] Question: How does the company plan to expand its TransCon platform? - The company is focused on developing multiple product opportunities across various therapeutic areas, including cardiovascular diseases [42][43] Question: What is the company's approach to cost management and earnings power? - The company has maintained cost efficiency and is looking to expand its pipeline while generating cash from operations [44][50]

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]

Group 1 - Ascendis Pharma A/S will participate in a fireside chat at the 2025 Wells Fargo Healthcare Conference on September 3, 2025, at 8:00 a.m. Eastern Time [1] - A live webcast of the presentation will be available on the Ascendis Pharma website, with a replay accessible for 30 days after the event [2] - Ascendis Pharma is a global biopharmaceutical company focused on its innovative TransCon technology platform to develop new therapies addressing unmet medical needs [3] Group 2 - The company is headquartered in Copenhagen, Denmark, and has additional facilities in Europe and the United States [3] - Ascendis Pharma emphasizes its core values of Patients, Science, and Passion in its operations [3]