文章核心观点 - 公司的TransCon CNP(navepegritide)在1年治疗中显示了与安慰剂相比在健康状况和身体功能方面的显著改善[1][2] - 这些改善不仅仅可以解释为线性生长的变化,支持TransCon CNP可能具有超越线性生长的直接治疗效果[1][2] - TransCon CNP作为针对achondroplasia(侏儒症)健康和生活质量并发症的首个获得统计学显著改善的候选药物,有潜力满足这一需求[3] 公司概况 - 公司正在利用其创新的TransCon技术平台建立一家领先的、全面整合的生物制药公司,致力于为患者的生活带来积极影响[6] - 公司以"患者、科学和热情"为核心价值观,利用TransCon技术创造新的潜在最佳疗法[6] - 公司总部位于丹麦哥本哈根,在欧洲和美国也有其他设施[6] 临床试验情况 - ACcomplisH是一项2期、多中心、随机、双盲、安慰剂对照、剂量递增试验,评估了每周一次的TransCon CNP与安慰剂在2-10岁achondroplasia患儿中的疗效和安全性[3] - 在100μg/kg/周的剂量组,患者在日常生活功能和情感健康等方面显示出统计学显著改善,5岁及以上患者的身体功能综合评分也有显著提高[4] - 整个试验人群在100μg/kg/周剂量下52周的生长结果与随机期间该剂量组一致[4] - 未观察到新的安全信号,也未报告与研究药物相关的严重不良事件[4] - 大多数治疗期不良事件为1-2级,注射部位反应发生率较低[5]

COPENHAGEN, Denmark, June 04, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (NASDAQ:ASND) today announced that company executives will participate in a fireside chat at the Goldman Sachs 45th Annual Global Healthcare Conference on Tuesday, June 11, 2024, at 8:00 a.m. ET in Miami, Florida. A live webcast of the fireside chat will be available via the Investors & News section of the Ascendis Pharma website at investors.ascendispharma.com. A webcast replay will also be available on this website shortly after co ...

-  40% of efficacy-evaluable patients (2 out of 5) in the initial cohort of patients with anti-PD-1 refractory melanoma treated with TransCon IL-2 β/γ in combination with TransCon TLR7/8 Agonist exhibited confirmed clinical responses with no new safety signals -  45% of efficacy-evaluable patients (5 out of 11) whose disease progressed on check-point inhibitors exhibited confirmed clinical responses when treated with TransCon IL-2 β/γ as monotherapy or in combination treatment COPENHAGEN, Denmark, June 03, ...

-  40% of efficacy-evaluable patients (2 out of 5) in the initial cohort of patients with anti-PD-1 refractory melanoma treated with TransCon IL-2 β/γ in combination with TransCon TLR7/8 Agonist exhibited confirmed clinical responses with no new safety signals -  45% of efficacy-evaluable patients (5 out of 11) whose disease progressed on check-point inhibitors exhibited confirmed clinical responses when treated with TransCon IL-2 β/γ as monotherapy or in combination treatment COPENHAGEN, Denmark, June 03, ...

COPENHAGEN, Denmark, May 30, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (NASDAQ:ASND) today announced that it will host informational booths, events, and presentations showcasing use of its innovative TransCon technology in the areas of growth hormone deficiency (GHD), hypoparathyroidism, achondroplasia, and Turner syndrome during ENDO 2024, the annual meeting of the Endocrine Society being held June 1-4, 2024, in Boston. "In addition to physician and caregiver presentations in pediatric GHD, we look forw ...

Shares of Ascendis Pharma (ASND) lost more than 5% on Tuesday after management announced that the FDA extended the review period for its regulatory filing for a hormone replacement therapy by an additional three months. A final decision is now expected by Aug 14, 2024.The company’s filing seeks approval for TransCon PTH (palopegteriparatide) to treat adults with hypoparathyroidism, a rare condition in which the body produces abnormally low levels of parathyroid hormone (PTH). Low production of this hormone ...

- Prescription Drug User Fee Act (PDUFA) goal date extended by three months for further review of submission to August 14, 2024 COPENHAGEN, Denmark, May 14, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (NASDAQ:ASND) today announced that the U.S. Food and Drug Administration (FDA) notified the Company that information submitted in response to the FDA's ongoing review of the New Drug Application (NDA) for TransCon PTH (palopegteriparatide) for adults with hypoparathyroidism constituted a major amendment to th ...

COPENHAGEN, Denmark, May 13, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (NASDAQ:ASND) today announced 2-year results from a post-hoc analysis of the Company's Phase 3 PaTHway Trial demonstrating significant and sustained improvements in renal function in adults with chronic hypoparathyroidism treated with TransCon PTH (palopegteriparatide). The data were shared in an oral presentation on May 12 by Peter Schwarz, M.D., Professor of Clinical Medicine at the University of Copenhagen, during the European Cong ...

COPENHAGEN, Denmark, May 09, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (NASDAQ:ASND) today announced that new TransCon PTH (palopegteriparatide) data supporting clinical and quality-of-life benefits in adult patients with chronic hypoparathyroidism will be presented at the European Congress of Endocrinology being held May 11-14, 2024, in Stockholm. Topics include an oral presentation of new 2-year results from a post-hoc analysis of the Phase 3 PaTHway Trial demonstrating sustained improvements in kidney ...

财务数据和关键指标变化 - 第一季度SKYTROFA收入为6500万欧元,同比增长106% [24] - 第一季度YORVIPATH收入为150万欧元,为首次贡献收入 [25] - 第一季度总收入为9590万欧元,包括2480万欧元的非现金许可收入和300万欧元的服务收入 [26] - 研发费用下降33%至7070万欧元,主要由于TransCon Growth Hormone和TransCon PTH以及肿瘤项目的外部开发成本降低 [27] - 销售及一般管理费用基本持平至6680万欧元 [28] - 总运营费用下降20%至13750万欧元 [29] - 第一季度经营亏损为4910万欧元,去年同期为14370万欧元 [30] - 财务费用大幅增加至7720万欧元,主要由于可转换票据相关的非现金衍生工具损失 [31] - 截至3月31日,现金及现金等价物为32亿欧元 [32] 各条业务线数据和关键指标变化 - SKYTROFA在美国的渗透率达到17%,预计全年收入将达到3.2-3.4亿欧元,同比增长80%-90% [15] - 正在推进SKYTROFA在成人生长激素缺乏症适应症的补充新药申请 [34] - 计划在第四季度报告SKYTROFA在特纳综合征适应症的II期试验结果 [34] - YORVIPATH在德国和奥地利的发售已有8周,已有55名医生开具处方,约占目标处方医生的25%,约100名患者已获得商业产品 [16] - 计划在2024年内将YORVIPATH推广至公司的欧洲直销和国际市场分部 [16] 各个市场数据和关键指标变化 - 公司正在为YORVIPATH在美国的上市做准备,包括扩大美国销售团队 [16] - 公司正在多个欧洲国家推进YORVIPATH的注册和获得报销,计划在2024-2025年内完成覆盖 [35] 公司战略和发展方向及行业竞争 - 公司致力于成为一家领先的集成生物制药公司,利用TransCon技术平台在内分泌罕见病等领域取得进展 [12][13] - 公司有信心在2024年底前实现季度经营现金流平衡 [12][13] - 公司计划在2024年第四季度提交TransCon CNP的新药申请,并报告ApproaCH试验的关键结果 [36] - 公司正在评估TransCon CNP联合TransCon Growth Hormone治疗软骨发育不全,以期能够实现追赶性生长 [18] - 公司在肿瘤领域的TransCon IL-2 beta/gamma和TransCon TLR7/8 Agonist项目也取得了积极的临床数据,计划在2024年第四季度提供更新 [19] - 公司正在评估利用TransCon技术开发GLP-1模拟剂,并计划在未来公布相关进展 [95][96] - 公司有信心SKYTROFA有潜力成为美国单一市场的重磅产品 [15] 管理层对经营环境和未来前景的评论 - 公司认为已具备实现2024年底经营现金流平衡的所有要素,在不影响发展和商业化进度的情况下实现了精简 [12][13] - 公司对SKYTROFA和YORVIPATH的商业化进展感到满意,认为这验证了公司的商业化方法 [14] - 公司对TransCon CNP在软骨发育不全治疗中的潜力感到乐观,认为该产品有望成为最佳-in-class [18] - 公司对肿瘤项目TransCon IL-2 beta/gamma和TransCon TLR7/8 Agonist的临床数据感到鼓舞 [19] - 公司认为SKYTROFA有潜力成为美国增长激素市场的重磅产品 [15] 问答环节重要的提问和回答 问题1 **Jessica Fye 提问** 询问YORVIPATH在4月份的表现情况 [40][41][42][43] **Jan Mikkelsen 和 Scott Smith 回答** 公司对YORVIPATH在德国和奥地利的发售初期表现感到满意,已有25%的目标医生开具处方,约100名患者获得商业产品。公司将在今年晚些时候提供YORVIPATH全年收入的更新。[43][44][45] 问题2 **David Lebowitz 提问** 询问YORVIPATH在美国上市前后的市场环境和公司的准备情况 [50][51][52][53] **Jan Mikkelsen 回答** 公司认为目前的市场环境要好于一年前,因为对低钙性甲状旁腺功能减退症的认知度有所提高,医生和患者都更加意识到未满足的医疗需求。公司有信心能够复制在德国和奥地利的成功经验,在其他欧洲国家推广YORVIPATH。[51][52][53] 问题3 **Li Watsek 提问** 询问在美国和欧洲,支付方在准予YORVIPATH报销时是否对临床获益数据如住院率或肾脏保护效果提出质疑 [55][56][57][59][60] **Jan Mikkelsen 回答** 在欧洲,公司在制定YORVIPATH的健康经济学影响时,已经将短期症状改善、钙平衡恢复正常以及生活质量改善等各方面因素纳入考虑,并评估了长期并发症风险的降低。公司相信这种全面的健康经济学分析有助于获得合理的定价和报销。[57][59][60]