Core Insights - Ascendis Pharma announced positive interim results from the COACH Trial, demonstrating that the combination of TransCon CNP and TransCon hGH significantly improves growth and body proportionality in children with achondroplasia after 26 weeks of treatment [2][4][12] Group 1: Clinical Trial Results - For treatment-naïve children, the combination treatment resulted in a mean annualized growth velocity (AGV) of 9.14 cm/year, with a height Z-score improvement of +0.53 over 26 weeks [1][12] - For children previously treated with TransCon CNP, the mean AGV was 8.25 cm/year, with a height Z-score improvement of +0.44 over the same period [1][12] - The combination treatment led to accelerated improvement in body proportionality, aligning with increased linear growth [1][12] Group 2: Safety and Tolerability - The safety and tolerability profile of the combination treatment was consistent with that of the individual monotherapies, with generally mild treatment-emergent adverse events (TEAEs) reported [1][4][12] Group 3: Trial Design and Future Plans - The COACH Trial is a Phase 2 open-label trial investigating the efficacy, safety, and tolerability of the combination treatment in children aged 2 to 11 years with achondroplasia [5] - The interim analysis will be followed by Week 52 data, expected in Q4 2025, with plans to initiate a Phase 3 trial in the same timeframe [5][11] Group 4: Product Information - TransCon CNP is under priority review by the FDA as a monotherapy for children with achondroplasia, while TransCon hGH is already approved and marketed as SKYTROFA for pediatric growth hormone deficiency [3][10] - Both products utilize Ascendis Pharma's innovative TransCon technology platform, which aims to provide sustained release of active compounds [3][10]

Core Insights - The FDA has accepted Ascendis Pharma's New Drug Application for TransCon CNP for treating children with achondroplasia, granting it Priority Review status with a PDUFA goal date of November 30, 2025 [1][2] - TransCon CNP has shown significant annualized growth velocity and multiple benefits beyond linear growth in clinical trials compared to placebo, with a safety profile similar to that of placebo [1][2] - Achondroplasia affects over 250,000 people globally and leads to various complications, necessitating new treatment options [3] Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform to address unmet medical needs [4][5] - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [5] Industry Context - Achondroplasia is a rare genetic condition caused by a variant in the FGFR3 gene, leading to serious complications beyond skeletal dysplasia, including muscular, neurological, and cardiorespiratory issues [3] - Current therapies for achondroplasia are limited, and there is a significant demand for new treatments that can improve health outcomes [2][3]

Core Insights - Ascendis Pharma announced positive long-term data from its Phase 2 PaTH Forward Trial for TransCon PTH, indicating a durable response in adults with hypoparathyroidism [1][5] - The trial demonstrated sustained normalization of serum calcium levels and significant improvements in kidney function and bone health [3][5] Study Design and Results - The PaTH Forward Trial included a 4-week randomized, double-blind, placebo-controlled phase followed by an open-label extension through Week 266, with 95% of original participants remaining in the trial at Week 214 [2] - At Week 214, 98% of patients maintained normal serum calcium levels, and 93% were independent from conventional therapy [3] - Bone turnover markers showed an initial increase, peaking by Week 26, and then stabilizing above baseline levels through Week 214 [3] - A clinically meaningful increase in estimated glomerular filtration rate (eGFR) was observed in 67.8% of participants [3] Safety Profile - TransCon PTH treatment was well-tolerated, with no new safety signals identified; most treatment-emergent adverse events (TEAEs) were mild or moderate [4] Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform to address unmet medical needs [7]

Financial Data and Key Metrics Changes - Ascendis Pharma reported a significant increase in first quarter revenue for Europath, reaching €44.7 million, up from €13.6 million in the previous quarter [28] - Total revenue for the first quarter was €101 million, which includes non-product revenue from collaboration partners [30] - R&D costs for the first quarter totaled €86.6 million, compared to €70.7 million in the same quarter of the previous year [30] - SG&A expenses increased to €101 million from €66.8 million year-over-year, primarily due to global commercial expansion [31] Business Line Data and Key Metrics Changes - Europath's global revenue grew to €45 million in the first quarter, reflecting strong U.S. demand and a growing patient base [13][28] - Skytrofa generated €51.3 million in revenue for the quarter, with stable pricing and market share despite seasonal impacts [29][19] - The company expects Europath to significantly contribute to revenue in 2025, with a potential to become a multi-billion euro product [18] Market Data and Key Metrics Changes - In the U.S., Europath was prescribed by over 1,000 unique prescribers for more than 1,750 patients, marking a successful launch [11] - The company estimates there are over 400,000 patients globally with chronic hypoparathyroidism, with 70,000 in the U.S. alone [18] - Skytrofa holds approximately 7% market share in the total growth hormone market in the U.S. and around 43% in the non-active growth hormone market [19] Company Strategy and Development Direction - Ascendis Pharma aims to be a leading biopharma company, focusing on the commercialization and development of Skytrofa and Europath [10] - The company is committed to expanding its product portfolio, with TransCon CNP being a key component in treating achondroplasia [12] - Ascendis Pharma is exploring market opportunities beyond endocrinology and rare diseases, indicating a strategic shift towards broader therapeutic areas [5] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong launch of Europath and its potential to become cash flow positive [10] - The company anticipates substantial revenue growth driven by the global launch of Europath and continued contributions from Skytrofa [32] - Management highlighted the importance of addressing unmet medical needs and the positive trends in reimbursement for Europath [38][40] Other Important Information - Ascendis Pharma's proprietary TransCon technology platform is fundamental to the development of its medicines, allowing for differentiated treatment benefits [26] - The company is collaborating with Novo Nordisk for the development of TransCon technology-based products in metabolic and cardiovascular diseases [27] Q&A Session Summary Question: What is the expected reimbursement rate for Yorvapath? - Management estimates that 17% to 18% of patients will ultimately get reimbursed once Yorvapath reaches steady state [38] Question: Can you provide the split between U.S. and ex-U.S. revenue for Yorvapath? - Management indicated that while specific numbers are not disclosed, there is steady growth expected outside the U.S. with an acceleration anticipated in the second half of the year [48] Question: What is the depth of prescribing for Yorvapath? - Management noted that they cannot definitively categorize patients as well-controlled or uncontrolled but expect a steady flow of patients transitioning from conventional therapies [66][70] Question: How is the company addressing payer dynamics? - Management emphasized that they are well-equipped to navigate reimbursement challenges, leveraging experience from previous product launches [75] Question: What are the competitive dynamics with new entrants in the market? - Management expressed confidence that their product addresses significant unmet medical needs and that new entrants may not significantly impact their market position [114]

Financial Data and Key Metrics Changes - Ascendis Pharma reported a significant increase in first quarter revenue for Europath, reaching €44.7 million, up from €13.6 million in the previous quarter [28] - Total revenue for the first quarter was €101 million, which includes non-product revenue from collaboration partners [30] - R&D costs for the first quarter totaled €86.6 million, compared to €70.7 million in the same quarter of the previous year [30] - SG&A expenses increased to €101 million from €66.8 million year-over-year, primarily due to global commercial expansion [31] Business Line Data and Key Metrics Changes - Europath's first quarter global revenue grew to €45 million, reflecting strong U.S. demand and a growing patient base [13][28] - Skytrofa revenue for the quarter was €51.3 million, with stable pricing and market share, although impacted by seasonal dynamics [29][18] - The company expects Europath to significantly contribute to revenue in 2025, with ongoing growth in both U.S. and international markets [16][28] Market Data and Key Metrics Changes - As of March 31, Europath was prescribed by over 1,000 unique prescribers for more than 1,750 patients in the U.S. [11] - The company estimates over 400,000 patients globally and around 70,000 in the U.S. are living with chronic hypoparathyroidism, with a significant portion being candidates for treatment with Europath [17] - Skytrofa holds approximately 7% market share in the total growth hormone market in the U.S. and around 43% in the non-active growth hormone market [18] Company Strategy and Development Direction - Ascendis Pharma aims to be a leading biopharma company, focusing on the global launch of Europath and the development of TransCon CNP for growth disorders [10][12] - The company is committed to expanding its product portfolio and addressing unmet medical needs in endocrinology and rare diseases [25] - Ascendis Pharma plans to submit an MAA for TransCon CNP in the EU in Q3 2025, with ongoing clinical trials to support its growth strategy [23][24] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong launch performance of Europath and its potential to become a multi-billion euro product [17] - The company anticipates substantial revenue growth driven by the global launch of Europath and continued contributions from Skytrofa [32] - Management highlighted the importance of favorable reimbursement dynamics and ongoing patient adherence as key factors for future success [41][42] Other Important Information - Ascendis Pharma recognized a non-cash gain of €33.6 million from its share of profit loss of associates due to the Visa IPO [31] - The company ended the first quarter with cash and cash equivalents totaling €518 million, down from €560 million at the end of 2024 [32] Q&A Session Summary Question: Expectations for reimbursement of Yorvapath - Management estimates that 17% to 18% of patients prescribed Yorvapath will ultimately get reimbursed, with ongoing positive trends in payer policies [39][40] Question: Split between U.S. and ex-U.S. revenue for Yorvapath - Management indicated steady growth in ex-U.S. markets, with expectations for acceleration in the second half of the year as more countries achieve full reimbursement [48] Question: Status of negotiations with commercial payers - Conversations with payers have been positive, with multiple favorable policies in place, and the company expects to continue gaining traction [54] Question: Proportion of new prescriptions from NATPARA or PTH naive patients - The majority of new patients are coming from conventional therapy, with about 10% to 15% having been on some form of PTH previously [58][62] Question: Depth of prescribing for Yorvapath - Management noted that they cannot definitively categorize patients as well-controlled or uncontrolled, but they are focusing on those frequently seeing endocrinologists [66][70] Question: Feedback on the titration process for Yorvapath - Adherence rates are consistent with clinical trials, and dropout rates are under 1%, indicating strong patient retention [100][101] Question: Plans for clinical utility trials for milder patients - Management is evaluating the long-term benefits of treatment for society and considering how to demonstrate the economic value of Yorvapath [108]

Financial Performance - Revenue for the three months ended March 31, 2025, was €100,954,000, an increase of 5.4% compared to €95,894,000 for the same period in 2024[15] - Gross profit decreased to €83,437,000 from €88,325,000, reflecting a decline of 5.1% year-over-year[15] - Research and development expenses rose significantly to €86,603,000, up 22.5% from €70,687,000 in the prior year[15] - Selling, general, and administrative expenses increased to €101,046,000, a 51.4% rise compared to €66,783,000 in the same quarter of 2024[15] - The net loss for the period was €94,626,000, compared to a net loss of €131,035,000 in the same period last year, representing a 27.8% improvement[15] - The company reported a basic and diluted loss per share of €(1.58) for the period, improving from €(2.30) in the previous year[15] - Revenue from commercial products reached €96.028 million, up 44.5% from €66.499 million in Q1 2024, driven by significant sales of YORVIPATH® and SKYTROFA®[45] - The net loss for the period was €94.626 million, an improvement from a net loss of €131.035 million in Q1 2024[19] - Cash and cash equivalents at March 31, 2025, totaled €517.923 million, compared to €320.239 million at the same date in 2024, reflecting an increase of 62%[19] Assets and Liabilities - Total assets decreased to €1,061,438,000 as of March 31, 2025, down from €1,179,495,000 at the end of 2024[16] - Total equity as of March 31, 2025, was €(189,807,000), compared to €(105,706,000) at the end of 2024, indicating a decline in equity[16] - Cash and cash equivalents decreased to €517,923,000 from €559,543,000, reflecting a reduction of 7.4%[16] - Total financial liabilities increased to €1,110.1 million as of March 31, 2025, compared to €1,104.0 million at the end of 2024, marking a rise of 0.1%[61] - Total financial liabilities as of March 31, 2025, amount to €935,512,000, with contractual cash flows totaling €1,227,447,000, indicating a significant portion of liabilities maturing within 1-5 years[78] Shareholder Information - The company had 60,018,550 shares used for the calculation of basic and diluted earnings per share, an increase from 56,883,257 shares in the prior year[15] - The company has authorized a share repurchase program of up to $18.25 million, which was fully executed by March 4, 2025[39] - The exercise price of outstanding warrants ranged from €11.98 to €145.50, with a total of 6,017,157 warrants outstanding as of March 31, 2025[58] Product Development and Pipeline - The company aims to achieve blockbuster status (>$1B) for TransCon PTH, TransCon hGH, and TransCon CNP through worldwide commercialization[95] - The current pipeline includes two marketed products and four product candidates in clinical development, focusing on Endocrinology Rare Disease and Oncology[94] - The company plans to expand its pipeline with Endocrinology Rare Disease blockbuster product opportunities, targeting unmet medical needs[95] - The oncology pipeline includes TransCon IL-2 b/g, currently in Phase 2 trials for various tumor types, aiming to enhance anti-tumor effects and immune activation[98] - The company is focused on applying its TransCon technology platform to develop differentiated product candidates, aiming to address unmet medical needs in large markets[90] Clinical Trials and Regulatory Approvals - TransCon PTH (palopegteriparatide) received FDA approval in August 2024 for the treatment of hypoparathyroidism in adults, with seven years of market exclusivity in the U.S.[147] - The Phase 3 PaTHway Trial demonstrated a mean increase in estimated glomerular filtration rate (eGFR) of 8.9 mL/min/1.73m² at Week 52, sustained at Week 104 with a mean change of 9.0 mL/min/1.73m²[154] - The company has submitted a New Drug Application (NDA) for TransCon CNP for achondroplasia in children aged 2-11, and a supplemental Biologics License Application (sBLA) for TransCon hGH for adult GHD[98] - The ongoing reACHin Trial is evaluating TransCon CNP in infants aged 0 to <2 years, with an IND amendment filed in Q3 2023[183] Market and Competitive Landscape - The global market for daily hGH products is primarily dominated by Novo Nordisk, Pfizer, Eli Lilly, Sandoz, and Merck KGaA, which together account for most of the market share[120] - Pediatric indications comprise up to 90% of the total hGH market, with approximately half attributed to pediatric GHD[121] - Approximately 68% of commercial patients and 63% of Medicaid patients with growth hormone deficiency (GHD) received daily growth hormone treatment, while 32% of commercial and 37% of Medicaid patients remained untreated[118] Technology and Innovation - TransCon technologies aim to provide sustained drug release, potentially extending dosing frequency from daily to six months or more, improving treatment safety and efficacy[107] - Early clinical studies indicate sustained activation of cytotoxic immune cells, leading to robust anti-tumor responses with infrequent administration of TransCon product candidates[199] - The company believes that TransCon technologies may enhance the efficacy of existing therapies without increasing toxicity, potentially allowing for new combination regimens[197]

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 Viewpoint - Ascendis Pharma A/S will report its first quarter 2025 financial results and provide a business update on May 1, 2025, after U.S. market close, followed by a conference call and live webcast [1] Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on its innovative TransCon technology platform to develop new therapies addressing unmet medical needs [3] - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [3] Event Details - The live webcast for the financial results will be accessible through a provided link, and a replay will be available for 30 days post-event [2]

Group 1 - The article expresses a beneficial long position in the shares of ASND, indicating a positive outlook on the company's stock performance [1] - The author emphasizes that the opinions presented are personal and not influenced by any business relationships with the mentioned companies [1] - There is a clear distinction made that the commentary does not constitute personalized investment advice, urging readers to conduct their own due diligence [2] Group 2 - The article highlights that past performance is not indicative of future results, which is a common disclaimer in investment discussions [3] - It is noted that the views expressed may not reflect those of Seeking Alpha as a whole, indicating a diversity of opinions among analysts [3] - The article clarifies that Seeking Alpha does not act as a licensed securities dealer or investment adviser, reinforcing the need for independent research by investors [3]

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]