Core Viewpoint - Ascendis Pharma A/S will report its second quarter 2025 financial results and provide a business update on August 7, 2025, after U.S. market close, followed by a conference call and live webcast to discuss the results [1]. Company Overview - Ascendis Pharma is a global biopharmaceutical company that utilizes its innovative TransCon technology platform to develop new therapies aimed at 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 discussion can be accessed through the company's website, and a replay will be available for 30 days post-event [2].

Core Viewpoint - The FDA has approved SKYTROFA (lonapegsomatropin-tcgd) for the treatment of growth hormone deficiency (GHD) in adults, expanding its previous approval for pediatric use, which is expected to improve treatment adherence and patient outcomes [1][2][3]. Company Overview - Ascendis Pharma A/S is a global biopharmaceutical company focused on innovative therapies for rare diseases, utilizing its TransCon technology platform to address unmet medical needs [10][12]. Product Details - SKYTROFA is a prodrug of somatropin, administered once weekly, providing sustained release of active, unmodified somatropin, which is expected to enhance patient adherence compared to daily injections [1][3]. - The approval was based on the results from the foresiGHt Phase 3 clinical trial, which demonstrated the efficacy and safety of SKYTROFA compared to placebo and daily somatropin [2]. Market Position - SKYTROFA is already recognized as the treatment of choice for pediatric GHD, and the company aims to establish it as a leading option for adult patients as well [3][6]. - Ascendis Pharma plans to initiate additional clinical trials for various conditions, including ISS, SHOX deficiency, Turner syndrome, and SGA, in Q4 2025, as part of its strategy to expand its product offerings [6][13].

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]

Core Insights - Ascendis Pharma announced positive long-term data from the Phase 3 PaTHway Trial for TransCon PTH, showing sustained efficacy in treating adults with hypoparathyroidism [1][4] - The trial demonstrated significant improvements in biochemical markers, kidney function, and quality of life for patients [1][3] Group 1: Trial Details - The PaTHway Trial was a Phase 3, randomized, double-blind, placebo-controlled study involving 82 adults with chronic hypoparathyroidism, followed by an open-label extension [2] - At Week 156, 88% of patients had normal serum calcium levels, and 96% were independent from conventional therapy [3] - The trial maintained high retention rates, with 89% of participants completing the 3.5-year study [2] Group 2: Efficacy and Safety - Mean estimated glomerular filtration rate (eGFR) increased by 8.76 mL/min/1.73 m² across all participants, with a notable increase of 13.98 mL/min/1.73 m² in those with baseline eGFR < 60 [3] - Patients reported continued improvements in hypoparathyroidism-related symptoms and health-related quality of life, with normalization of 24-hour urine calcium excretion [3] - TransCon PTH treatment was well-tolerated, with no new safety signals identified and most treatment-emergent adverse events being mild or moderate [3] Group 3: Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform [6] - The company aims to address unmet medical needs and improve patient outcomes through its product innovations [6]

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]