此新药获批上市主要依据于中国完成对3至17岁儿童生长激素缺乏症患者的3期关键性试验。于此项试验 中，于52周时，隆培生长激素的年化身高速率为10.66厘米╱年，而短效(每日注射)人生长激素为9.75厘 米╱年，两组治疗差异为0.91厘米╱年(95% CI：0.37-1.45，P=0.0010)。隆培生长激素组于52周时的身 高标准差评分(SDS)较基线增加了1.01，短效(每日注射)人生长激素组则增加了0.83， p值为0.0015，从 治疗第13周开始，隆培生长激素组的身高SDS改善幅度较短效(每日注射)人生长激素组更为明显，且该 差异具有统计学显著性。试验所公布的结果表明了与已完成的全球3期关键性试验(该试验支持了FDA及 EMA对其治疗 PGHD的上市批准)的一致性，并证明了隆培生长激素与短效(每日注射)人生长激素相比 的优效性以及同等安全性。 隆培生长激素获BLA批准后，集团已启动全面商业化计划，以确保供应并扩大市场覆盖。为确保隆培生 长激素推出市场时的供应，集团已与合作伙伴 Ascendis Pharma订立商业化供应协议。同时，集团透过 打造一支专注及专业的商业化团队以及实施为商业化、医生认知及市 ...

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 ...

– Once-weekly TransCon® CNP and TransCon® hGH combination therapy showed durable growth without compromising safety or tolerability at 52 weeks – Annualized growth velocity (AGV) exceeded the 97th-percentile of average stature children – ACH height Z-score improvements indicated a tripling of efficacy compared to TransCon CNP monotherapy – Combination therapy demonstrated benefits beyond linear growth with improvements in body proportionality and arm span, aligning with the increase in linear growth – All ...

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]