Group 1 - Ascendis Pharma A/S (ASND) shares have increased by 1.1% over the past four weeks, closing at $174.75, with a mean price target of $226.88 indicating a potential upside of 29.8% [1] - The mean estimate consists of 16 short-term price targets with a standard deviation of $28.42, where the lowest estimate is $194.00 (11% increase) and the highest is $289.00 (65.4% increase) [2] - Analysts show strong agreement on ASND's ability to report better earnings, with a positive trend in earnings estimate revisions correlating with potential stock upside [4][11] Group 2 - The Zacks Consensus Estimate for ASND's current year has increased by 2% over the past month, with no negative revisions [12] - ASND holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimates [13] - While consensus price targets may not be reliable for predicting exact gains, they can indicate the direction of price movement [14]

COPENHAGEN, Denmark, July 14, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced new data from Week 156 of its Phase 3 PaTHway Trial confirming that long-term treatment with TransCon PTH (palopegteriparatide) continued to provide a durable response in adults with hypoparathyroidism regardless of its cause (post-surgical, autoimmune, genetic, or idiopathic), including improvements in biochemistries, kidney function, and quality of life. Results were shared over the weekend in an oral ...

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]

Summary of Ascendis Pharma (ASND) Conference Call Company and Industry Overview - **Company**: Ascendis Pharma - **Industry**: Biotechnology, specifically focusing on treatments for growth disorders such as achondroplasia Key Findings from the COAST Trial - The COAST trial is a Phase II study assessing the efficacy of a combination therapy involving TransCon CNP and TransCon Growth Hormone in children with achondroplasia aged 2 to 11 years [2][3] - The trial reported a significant increase in annualized growth velocity (AGV) for treatment-naive children, achieving 9.14 cm/year, which is an increase of 4.2 cm/year compared to baseline [19][20] - For children previously treated with TransCon CNP, the AGV was 8.25 cm/year, reflecting a 3.1 cm/year increase from baseline [21][22] Efficacy and Safety Data - The combination therapy demonstrated a 0.53 increase in height Z score for treatment-naive patients and a 0.44 increase for treatment-experienced patients after 26 weeks [20][22] - The safety profile of the combination therapy was comparable to that of the individual therapies, with no serious adverse events related to the study drug reported [17][18] - The combination therapy resulted in a linear growth improvement associated with better body proportionality, which is crucial for children with achondroplasia [24][25] Future Development Plans - Ascendis Pharma plans to initiate a Phase III trial by the end of 2025, which will include both treatment-naive and experienced children [26][30] - The company aims to expand its treatment options for growth disorders and explore over 20 potential indications for its therapies [27][84] Market Position and Strategy - Ascendis Pharma is positioned as a leader in the growth disorder treatment market, with a focus on developing unique treatment combinations that unlock new therapeutic potentials [27][84] - The company is considering co-formulating TransCon CNP and TransCon Growth Hormone into a single injection for improved patient compliance [45][103] Additional Insights - The trial results indicate a potential for achieving catch-up growth in children with achondroplasia, which could normalize their height relative to parental expectations [80] - The combination therapy is expected to provide significant benefits beyond linear growth, addressing broader health concerns associated with achondroplasia [81][96] Conclusion - Ascendis Pharma's recent trial results highlight a promising advancement in the treatment of achondroplasia, with significant improvements in growth velocity and safety. The company is poised to expand its market presence and explore additional indications for its therapies, reinforcing its commitment to addressing growth disorders effectively.

Combination treatment with investigational TransCon CNP and TransCon hGH is investigational. For investor communication only. Not for use in product promotion. Not for further distribution. COACH Trial Interim Topline Week 26 Data June 9, 2025 Cautionary Note on Forward-Looking Statements 2 Combination treatment with investigational TransCon CNP and TransCon hGH is investigational. For investor communication only. Not for use in product promotion. Not for further distribution. Executive Summary This present ...

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]