Core Viewpoint - The approval of the long-acting growth hormone, Lonapegsomatropin (SKYTROFA®), by the National Medical Products Administration of China marks a significant advancement in the treatment of pediatric growth hormone deficiency (PGHD) in children aged 3 and above, providing a more convenient weekly dosing option compared to daily injections [1][2]. Group 1: Product Approval and Efficacy - Lonapegsomatropin is the first long-acting growth hormone that has demonstrated superior efficacy and equivalent safety compared to short-acting daily injections in a positive parallel-controlled trial [2][3]. - In a pivotal Phase 3 trial involving children aged 3 to 17 with PGHD, Lonapegsomatropin showed an annualized height velocity of 10.66 cm/year, compared to 9.75 cm/year for short-acting growth hormone, with a statistically significant difference of 0.91 cm/year (P=0.0010) [3]. - The height standard deviation score (SDS) for the Lonapegsomatropin group increased by 1.01, while the short-acting group saw an increase of 0.83, with a p-value of 0.0015, indicating significant improvement from week 13 onwards [3]. Group 2: Commercialization Strategy - Following the BLA approval, the company has initiated a comprehensive commercialization plan to ensure supply and expand market coverage for Lonapegsomatropin [4]. - The company has established a commercialization supply agreement with Ascendis Pharma to secure market launch supplies [4]. - Strategic partnerships have been formed with Shanghai Pharmaceuticals, United Family Healthcare, and Anke Bioengineering to enhance distribution and promotional activities for Lonapegsomatropin [4].

Ascendis Pharma A/S J.P. Morgan Healthcare Conference San Francisco January 2026 This presentation also contains estimates and other statistical data made by independent parties and by us relating to market size and other data about our industry. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such data and estimates. In addition, projections, assumptions and estimates of our future performance and the future performance of the markets in which w ...

Core Insights - Ascendis Pharma announced positive topline results from the Week 52 of the COACH trial, demonstrating that the combination therapy of once-weekly TransCon CNP and TransCon hGH showed durable growth without compromising safety or tolerability [2][3] Group 1: Trial Results - The combination therapy resulted in an annualized growth velocity (AGV) that exceeded the 97th percentile of average stature children [11] - For the treatment-naïve cohort, the mean AGV was 8.80 cm/year, with a mean ACH height Z-score improvement of +1.02, indicating a tripling of efficacy compared to TransCon CNP monotherapy [5][6] - The treated cohort showed a mean AGV of 8.42 cm/year, with an increase from baseline of 3.28 cm/year and a mean ACH height Z-score improvement of +0.86, increasing from 1.28 to 2.15 over 52 weeks [5][6] Group 2: Safety and Tolerability - The combination therapy was generally well tolerated, with mild treatment-emergent adverse events (TEAEs) consistent with those observed for the monotherapies [2][11] - Safety and tolerability profiles were consistent with TransCon CNP and TransCon hGH monotherapies, indicating a low incidence of injection site reactions [11] Group 3: Implications for Treatment - The results suggest that TransCon CNP may become a foundational therapy for achondroplasia, with TransCon hGH providing complementary benefits [2][3] - The trial highlights the potential for dual-agent regimens to set new standards of care in treating achondroplasia and other growth disorders [3][11] - Improvements in body proportionality and arm span were observed, aligning with the increase in linear growth, indicating benefits beyond just height [2][11] Group 4: Future Developments - Ascendis Pharma has submitted a protocol for a Phase 3 trial of TransCon CNP and TransCon hGH in pediatric achondroplasia and held an end of Phase 2 meeting with the FDA [7][11] - The U.S. FDA has granted Priority Review for TransCon CNP, with a PDUFA target action date of February 28, 2026 [3][11]

Core Insights - Ascendis Pharma reported Q3 2025 revenue of €213.6 million, a significant increase from €57.8 million in Q3 2024, driven primarily by the strong performance of YORVIPATH and SKYTROFA [6][12] - The company is progressing towards its Vision 2030 goals, with ongoing global launches and positive feedback from healthcare providers and patients [2][4] - Ascendis Pharma achieved an operating profit of €11.0 million in Q3 2025, attributed to higher revenues from YORVIPATH, despite increased operating expenses [11][10] Financial Performance - YORVIPATH revenue for Q3 2025 was €143.1 million, reflecting a negative foreign currency impact of €3.6 million compared to the previous quarter [5][6] - SKYTROFA revenue for Q3 2025 totaled €50.7 million, also impacted by a negative foreign currency effect of €1.6 million [5][6] - Total revenue from commercial products reached €193.8 million in Q3 2025, compared to €55.7 million in Q3 2024 [9][10] Product Development and Regulatory Updates - TransCon CNP is currently under FDA Priority Review for treating children with achondroplasia, with a PDUFA date set for November 30, 2025 [4][8] - The company plans to initiate a Phase 3 trial for TransCon CNP in combination with TransCon hGH following a successful end-of-Phase 2 meeting with the FDA [8][4] - Ascendis Pharma presented new analyses from the ApproaCH Trial at the ASBMR annual meeting, showing improvements in physical functioning for children treated with TransCon CNP [8] Operational Highlights - As of September 30, 2025, Ascendis Pharma had cash and cash equivalents of €539 million, a slight decrease from €560 million at the end of 2024 [13][20] - The company reported a net loss of €61.0 million for Q3 2025, an improvement from a net loss of €99.2 million in Q3 2024 [12][19] - Research and development costs decreased to €66.9 million in Q3 2025 from €73.5 million in the same period in 2024, reflecting the completion of clinical trials [10][19]

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]