A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial Evaluating Efficacy and Safety of Subcutaneous Doses of Navepegritide Administered Once Weekly for 52 Weeks in Adolescents (12-18 Years of Age) With Achondroplasia.

The purpose of this clinical trial is to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of navepegritide 100 μg/kg compared to placebo (inactive drug) in adolescents aged 12 to 18 years with Achondroplasia. What will be measured is Annualized Growth Velocity after a 52-week treatment period.

开始日期2024-12-10

申办/合作机构

Ascendis Pharma A/S

CTIS2023-508341-40-00

/ Not yet recruiting临床2期

COACH: A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepegritide and Lonapegsomatropin in Children with Achondroplasia - ASND0042

开始日期2024-07-26

申办/合作机构

Ascendis Pharma Growth Disorders A/S

NCT06433557

/ Active, not recruiting临床2期

A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepegritide and Lonapegsomatropin in Children With Achondroplasia

This proof-of-concept trial is being conducted to evaluate the efficacy, safety and tolerability of combination treatment with navepegritide and lonapegsomatropin administered as separate subcutaneous (SC) injections once weekly in children with achondroplasia (ACH) aged 2 to 11 years.

开始日期2024-07-26

申办/合作机构

Ascendis Pharma Growth Disorders A/S

100 项与 Navepegritide 相关的临床结果

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100 项与 Navepegritide 相关的转化医学

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100 项与 Navepegritide 相关的专利（医药）

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项与 Navepegritide 相关的文献（医药）

2024-11-01·Expert Review of Endocrinology & Metabolism

Progress in managing children with achondroplasia

Review

作者: Savarirayan, Ravi ; Taylor-Miller, Tashunka

INTRODUCTION:

Achondroplasia is a heritable disorder of the skeleton that affects approximately 300,000 individuals worldwide. Until recently, treatment for this condition has been purely symptomatic. Efficacious treatment options for children are now approved or are in clinical trials.

AREAS COVERED:

This review discusses key advances in the therapeutic management of children with achondroplasia, including vosoritide, the first approved drug, and other emerging precision therapies. These include navepegritide, a long-acting form of C-type natriuretic peptide, and infigratinib, a tyrosine kinase receptor inhibitor, summarizing trial outcomes to date.

EXPERT OPINION:

The advent of the first approved precision therapy for achondroplasia in vosoritide has been a paradigm shifting advance for children affected by this condition. In addition to changing their natural growth history, it is hoped that it will decrease their medical complications and enhance functionality. These new treatment options highlight the importance of prompt prenatal identification and subsequent testing of a suspected fetus with achondroplasia and counseling of families. It is hoped that, in the near future, families will have the option to consider a range of effective targeted therapies that best suit their child with achondroplasia, starting from birth should they choose.

2023-11-01·EClinicalMedicine

Once-weekly TransCon CNP (navepegritide) in children with achondroplasia (ACcomplisH): a phase 2, multicentre, randomised, double-blind, placebo-controlled, dose-escalation trial

Article

作者: Ma, Nina S ; Zarate, Yuri A ; Savarirayan, Ravi ; Winding, Bent ; Mao, Meng ; Sousa, Sérgio B ; McDonnell, Ciara ; Bober, Michael B ; Hoernschemeyer, Daniel G ; Bacino, Carlos A ; Legare, Janet M ; Smith, Alden ; Volck, Birgitte ; Schnabel, Dirk ; Chakraborty, Mukta ; Ljungberg, Merete ; Hofman, Paul L ; Giwa, Adebola ; White, Klane K ; Shu, Aimee D ; Högler, Wolfgang ; Quattrin, Teresa ; Abuzzahab, M Jennifer

Background:

TransCon CNP (navepegritide) is an investigational prodrug of C-type natriuretic peptide (CNP) designed to allow for continuous CNP exposure with once-weekly dosing. This 52-week phase 2 (ACcomplisH) trial assessed the safety and efficacy of TransCon CNP in children with achondroplasia.

Methods:

ACcomplisH is a global, randomised, double-blind, placebo-controlled, dose-escalation trial. Study participants were recruited between June 10, 2020, and September 24, 2021. Eligible participants were prepubertal, aged 2-10 years, with genetically confirmed achondroplasia, and randomised 3:1 to once-weekly subcutaneous injections of TransCon CNP (6, 20, 50, or 100 μg CNP/kg/week) or placebo for 52 weeks. Primary objectives were safety and annualised growth velocity (AGV). ACcomplisH is registered with ClinicalTrials.gov (NCT04085523) and Eudra (CT 2019-002754-22).

Findings:

Forty-two participants received TransCon CNP at doses of 6 μg (n = 10; 7 female), 20 μg (n = 11; 3 female), 50 μg (n = 10; 3 female), or 100 μg (n = 11; 6 female) CNP/kg/week, with 15 receiving placebo (5 female). Treatment-emergent adverse events (TEAEs) were mild or moderate with no grade 3/4 events reported. There were 2 serious TEAEs that were assessed as not related to TransCon CNP. Eleven injection site reactions occurred in 8 participants receiving TransCon CNP and no symptomatic hypotension occurred. TransCon CNP demonstrated a dose-dependent improvement in AGV. At 52 weeks, TransCon CNP 100 μg CNP/kg/week significantly improved AGV vs placebo (least squares mean [95% CI] 5.42 [4.74-6.11] vs 4.35 [3.75-4.94] cm/year; p = 0.0218), and improved achondroplasia-specific height SDS from baseline (least squares mean [95% CI] 0.22 [0.02-0·41] vs -0·08 [-0.25 to 0.10]; p = 0.0283). All participants completed the randomised period and continued in the ongoing open-label extension period receiving TransCon CNP 100 μg CNP/kg/week.

Interpretation:

This phase 2 trial suggests that TransCon CNP is effective, safe, with low injection site reaction frequency, and may provide a novel, once-weekly treatment option for children with achondroplasia. These results support TransCon CNP at 100 μg CNP/kg/week in the ongoing pivotal trial.

Funding:

Ascendis Pharma, A/S.

2022-11-01·BRITISH JOURNAL OF CLINICAL PHARMACOLOGY2区 · 医学

Phase 1 safety, tolerability, pharmacokinetics and pharmacodynamics results of a long‐acting C‐type natriuretic peptide prodrug, TransCon CNP

2区 · 医学

Article

作者: Christoffersen, Eva Dam ; Breinholt, Vibeke Miller ; Mygind, Per Holse ; Ota, Sho ; Will Charlton, R. ; Zhang, Ying ; Viuff, Dorthe

Aim:

TransCon CNP is a novel prodrug designed to provide sustained release of C‐type natriuretic peptide (CNP) for once‐weekly therapy, addressing the pathology leading to aberrant skeletal development in achondroplasia. This phase 1 trial was initiated to assess the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of TransCon CNP.

Methods:

This randomized, placebo‐controlled, single‐ascending dose phase 1 trial was performed at two sites in Australia and enrolled 45 healthy adult males. Subjects received placebo or TransCon CNP (single‐ascending dose cohorts [3, 10, 25, 75 or 150 μg CNP/kg]). The primary endpoint was frequency of adverse events and other safety outcomes. Other endpoints included PK and PD measured by cyclic guanosine‐monophosphate (cGMP) and amino‐terminal propeptide of CNP (NTproCNP).

Results:

TransCon CNP provided continuous systemic exposure to CNP over at least 7 days post‐dose. Plasma and urine levels of cGMP were significantly increased in subjects administered TransCon CNP at 75‐150 μg CNP/kg, indicating target engagement of active CNP at the natriuretic peptide receptor‐B (NPR‐B) for at least 1 week post‐dose. TransCon CNP was well‐tolerated, with no serious treatment‐emergent adverse events or discontinuations. Extensive cardiac safety assessments did not reveal any clinically relevant effects on electrocardiogram parameters, including heart rate, PR, QRS and QTcF intervals.

Conclusions:

Safety and PD data from this phase 1 trial support that TransCon CNP is well tolerated, with a PK profile compatible with a once‐weekly dosing regimen. Further studies are ongoing to evaluate the potential of TransCon CNP to positively impact abnormal endochondral ossification in children with achondroplasia.

项与 Navepegritide 相关的新闻（医药）

2025-02-24

·E药经理人

起步FIC/BIC，三款在研药品临近商业化，这家Biotech为何好消息频传？

过往6年，维昇药业的核心在研产品隆培促生长素(Lonapegsomatropin)的上市申请已于2024年3月获中国药监局受理，其他两个优质管线产品那韦培肽(Navepegritide)和帕罗培特立帕肽(Palopegteriparatide)在国内开展的临床试验均达到主要临床终点；之后，维昇将逐步构建起以患者为中心的综合医疗健康解决方案，稳步推进商业化。 2021年，维昇药业获得如红杉中国、奥博资本、夏尔巴投资等顶级投资机构青睐。2025年2月，公司通过港交所聆讯，随即A股知名上市企业安科生物表达“对维昇药业未来发展潜力的认可”并宣布将对其进行战略投资。即将成为港股"生长发育第一股"的维昇药业，在向资本市场进发的同时，也正积极进行商业化布局。隆培促生长素上市申请的受理，标志着维昇药业正式进入下一个发展阶段。近期，E药经理人专访了维昇药业首席商务官陈军。从这位深耕行业二十余年的商业化“行家”的讲述中，我们深入探寻这家“行稳踏准”的Biotech，如何坚守初心与韧性，在瞬息万变的环境中始终秉持“患者为先”的承诺，聚焦FIC/BIC的“新”产品，致力于照护患者“全”生命周期。这样的企业不仅展现了其独特的创新力，更体现了对患者深切的关怀与责任。相关阅读 * 专注内分泌，穿越大周期，从入局到破局，维昇药业做对了什么？ 01 围绕患者需求，矢志不移定“起点” 2015年的药审改革，为中国医药创新吹响了号角。经过近10年的发展，我国的创新药研发、临床试验、药物可及等方面都得到了长足进步。当链条细化到疾病本身和患者需求上，现有疗法不够理想、诊疗规范仍待完善、流行病学数据不明晰等问题凸显，我国医疗创新“升级”之路上，困难与机会并存。在内分泌领域深耕20余载的陈军告诉E药经理人，破局的关键是完善“医患共建共享”的医疗生态体系，“流行病学研究的完善，可以推动社会大众甚至广泛医疗专业人士对内分泌疾病的认知，解决系统性的认知匮乏的问题。” “系统性的认知匮乏”将引发一串连锁反应：在缺乏有效登记和流行病学调查的情况下，很多内分泌系统疾病的流病信息和疾病影响评估工作都难以开展（包括发病率、患病率、地域分布特征、患者的疾病负担和生活质量等情况），这往往伴随着规范化诊疗手段缺失、诊疗经验缺少等问题。这些问题传导至患者端会使诊疗无门，传导至政策端也会造成国家的相关诊疗和保障政策缺乏科学依据，最终往往使这类患者得不到有效的诊疗保障。因此，除了提供临床急需的创新药品之外，构建系统化医疗服务与规范化诊疗流程相协同的患者照护生态，也是制药企业可以积极参与的工作。经历了资本寒冬突袭、全球局势不确定的诸多因素，作为创新主力军的Biotech，尚陷在日益严峻的环境变化中“自顾不暇”。行业虽处于下行，但已逐渐释放回温信号，维昇药业以其笃定的战略和经营的耐力“波澜不惊”，在生态赋能中迈出稳健的步伐。维昇药业自创立起，就有计划地推动与相关机构配合开展流行病学研究，这些研究与公司的药物临床试验几乎同步进行。早在2020年，维昇药业与中国罕见病联盟签署战略合作协议，双方共同发起了中国首个“软骨发育不全流行病学研究”，这是目前全球规模最大的软骨发育不全流行病学调查研究；2022年7月，维昇药业与北京大学临床研究所共同启动“甲状旁腺功能减退症临床登记研究(PaTHway R)”，也是中国首个大规模甲状旁腺功能减退症患者登记研究项目。敢于破局者，自有到来风。2023年9月，国家卫生健康委等6部门联合制定《第二批罕见病目录》，软骨发育不全和遗传性甲状旁腺功能减退症均被纳入其中。“目录式”管理作为我国罕见病管理的主要方式之一，给罕见病新药从研发到上市、生产、准入等环节提供了政策引导，被纳入《第二批罕见病目录》就意味着该类疾病将得到全方位的政策支持。陈军预计，维昇药业引进的软骨发育不全针对性治疗药物（那韦培肽）有望通过中国2期临床结果，结合国外3期临床数据在国内提交上市申请。在过去五年间，维昇药业紧密携手政、学、研等各方，从疾病与患者两方切入，赋能整个生态。围绕内分泌领域疾病，维昇药业积极推进流行病学研究、辅助规范疾病诊疗标准的建立、推动专科医生技能培训；针对患者群体，维昇药业联动行业伙伴，致力于推动疾病知识的普及、提高患者自我管理的意识和水平，并积极参与患者关爱日等项目。事实上，在中国市场，诸多围绕创新药物的疾病生态才刚刚建起，维昇药业作为一家成立6年的Biotech，始终贯彻“以患者为先”，在企业前进的步伐中履行使命，彰显担当。 02 瞄准市场趋势，有的放矢稳“基点” 维昇药业成立至今的6年，也是中国医药市场从创新勃发到复杂外界环境冲击，进而步入医药新周期的6年，制药企业们纷纷重新思辨企业战略方向。从这一角度而言，维昇药业以“强大的产品力+经验丰富的团队+精益运营体系”为底气，以“服务患者，赋能生态”为起点，在新周期中构成了其未来行稳致远的优势。如今中国医药市场的竞争环境和市场状态显示，与患者需求的“距离”，决定了产品市场潜力的上限。维昇药业所聚焦的三大领域之一——长效生长激素正是这样一块黄金高地，市场需求高、技术难突破，形成了少数有能力的破局者分享庞大市场的竞争格局。对于儿童生长激素缺乏症（PGHD）而言，目前临床中主要治疗方式仍为每天注射的短效生长激素。频繁注射也导致了治疗依从性差、平均治疗时间短等问题。陈军告诉E药经理人，短效日制剂向长效周制剂的技术性“升级”是PGHD领域的治疗趋势。不过，长效生长激素的研发并非易事。在延长生长激素药物在体内代谢时间的同时，保持理想的疗效和安全性是一个巨大的难题。在过去的几十年里，全球数十个长效生长激素研发项目的相继失败就是证明。因为这个很高的技术门槛，在国内市场，长效生长激素形成了“1个获批产品+少数临床后期药物”的竞争格局。维昇药业的隆培促生长素就是其中具有“硬实力”的竞争者。搭载了先进TransCon技术的隆培促生长素，不仅上市时间走在其它在研产品的前面，在产品特性上更具有同内源性生长激素一样的作用机理，在实现了一周给药一次的同时，其活性成分又保持了生长激素的天然结构[1]。其临床试验证实，隆培促生长素比每日注射的生长激素有更好的长高疗效[2]，在安全性和有效性方面都具有独特优势。在迫切的临床需求和新技术的突破下，国内PGHD治疗正在由短效向高价值的长效生长激素迭代升级。弗若斯特沙利文数据显示，中国已经是全球最大的生长激素市场。2023年到2027年，预计长效生长激素的市场年复合增长率为43%。预计2030年，长效生长激素将是PGHD的主要疗法，市场份额占比可达74%。值得一提的是，隆培促生长素（海外商品名：SKYTROFA）在2021年就由Ascendis Pharma递交并获得了美国FDA的批准，是美国首个用于治疗儿童生长激素缺乏症的长效生长激素，此后SKYTROFA还登陆了欧洲多国。在2024年，SKYTROFA的销售额达到2.02亿欧元（约14.7亿元人民币）其销售量同比增长达84%。陈军介绍，维昇药业的商业化核心管理团队汇聚了4位行业“六边形战士”他们在内分泌领域从业经验加起来超过百年，几年来对生长激素市场深入调研，精准洞悉国内生长激素市场特征和规律，并以此做出了针对性的商业化战略：以产品优势占领学术制高点，以患者为中心打造高效诊疗通路，让中国患儿能够最快捷地享受到全球领先的治疗方案。聚焦专科医生的专业学术推广，传递产品价值：通过一支 “专而精”的推广团队，树立隆培促生长素“Best-in-class”的长效生长激素的品牌定位；形成以患者为本(treating patients)的系统性疾病管理方案：从PGHD患儿及家长的诊疗旅程入手，逐步建立从疾病科普教育，到辅助用药服务，再到提供多种创新支付和售后服务体系的诊疗闭环。未来，在以维昇药业为代表的“先行者”们的协力下，可以预见到PGHD患者会得到更广泛的规范治疗，治疗依从性更好，长效生长激素市场规模也将呈现质的飞跃。 “维昇药业始终着眼于同类首个或同类最佳的产品管线。”陈军告诉E药经理人，那韦培肽所针对的软骨发育不全，在国内有超过5万患者；帕罗培特立帕肽治疗的甲状旁腺功能减退症有超过40万患者。而这两个疾病在国内都尚未有针对病因的突破性疗法，现有疗法存在很大的局限性和未被满足的治疗需求。随着那韦培肽和帕罗培特立帕肽这两款产品研发进程的稳步推进，预计将在不远的将来提交上市申请。在更长远的未来，维昇药业还将继续保持有序而紧密的产品上市节奏，在内分泌领域“打出”组合优势，奠定公司商业化扎实的基础。陈军表示，“维昇药业从不做‘平庸’的产品。在未来，我们也会通过更多元的方式扩充同类首个或同类最佳的产品管线，为商业化加速赋能，辐射更广阔、更深入的诊疗领域，惠及更广泛的内分泌患者，最终将维昇打造成为中国内分泌领域创新药的门户平台。” 03 关注患者需求，以新提质筑“高点” 维昇药业成立6年，依托“一号员工”卢安邦深耕行业三十年的经验，企业战略与患者需求密不可分，产品价值与商业化策略紧密相接，药品的生产供应也与患者可及环环相扣。将好药引进来、做出来、卖出去，是这位医药老兵朴素而直接的想法。鉴于隆培促生长素优秀的临床数据，维昇药业将在销售进口产品的同时，同步落实药品的本地化生产，让更多的中国患者获益于这一有同类最佳（BIC）潜力的长效生长激素。维昇在选择本地化产品的制剂给药装置时，为了增加患者用药的便利性、提高患者用药的依从性，为隆培促生长素配备了先进的双腔预充给药装置（DCD药物递送技术）。由CMC团队自主研发、优化设计，旨在以先进的药物递送技术搭载新型长效生长激素，更好地服务广大中国患者。通常意义上，生物制品主要有液体（水剂）和冻干（粉剂）两种形式，但液体制剂通常不够稳定，对储存条件要求高且效期较短；而冻干粉制剂虽然解决了稳定性问题，但给药不方便（包含繁琐的配药过程，容易在给药过程中引入外源异物，进而造成药品污染等）。为了解决水剂和粉剂两种形式在患者给药过程中的诸多不便，双腔预充给药装置（DCD）作为生物制品皮下注射给药的优选解决方案在海外应运而生，而该项技术在中国还仍处于空白。双腔装置技术在一个容器密封系统中，将药物和复溶剂分开储存在两个独立腔室，保持了药物和复溶剂在储存过程中的独立稳定性，两个腔室在使用前再进行连接复溶，提高使用的便利性。维昇药业的这一技术不仅将填补中国在药物制剂递送系统领域的技术空白，其成功研发还将为中国其他产品提供先进的药物递送解决方案。从患者需求出发，对患者可及负责，商业化策略与自主研发协同共进，铸就维昇药业践行“治疗疾病，更是治疗患者”的未来高点。 04 结语企业价值能否跟上行业变迁，是竞争力的集中体现。从精准针对内分泌市场趋势，临床试验稳步达标，流病研究踏准痛点，到给药方式优化填补空白，维昇药业的每一步“行稳踏准”，都恰恰构成了其在Biotech竞合过程中的突出优势。国务院常务会议审议通过《全链条支持创新药发展实施方案》，从成果转化、审评审批，到多元支付、投融资渠道拓宽，创新药生态全链条支持政策得到顶层设计指导，可以预见的是，越来越多的“维昇们”正在汇聚成涌泉。“涓涓细流汇成海，点点纤尘积就山”，中国的医药创新生态高质量发展场景，也在徐徐展开。 [1] Miller BS, Yuen KCJ. Spotlight on Lonapegsomatropin Once-Weekly Injection and Its Potential in the Treatment of Growth Hormone Deficiency in Pediatric Patients. Drug Des Devel Ther. 2022;16:2055-2066. Published 2022 Jun 29. doi:10.2147/DDDT.S336285 [2] 隆培促生长素的全球和中国3期临床试验均证明其优效于短效（每日注射）生长激素，安全性相当；现全球和中国共有4款通过3期临床试验的LAGH，试验结果中仅隆培促生长素的有效性指标显示优效。一审| 黄佳二审| 李芳晨三审| 李静芝

抗体药物偶联物

2025-01-12

·GlobeNewswire-Health Care

Ascendis Pharma Provides Business and Strategic Roadmap Update at 43rd Annual J.P. Morgan Healthcare Conference

- Ascendis positioned to drive rapid revenue growth COPENHAGEN, Denmark, Jan. 12, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today provided a business and strategic roadmap update, including planned 2025 key corporate milestones. Ascendis President and CEO Jan Mikkelsen will present this update tomorrow, January 13, during the 43rd Annual J.P. Morgan Healthcare Conference. “Ascendis is well-positioned for rapid revenue growth with the launch of our first two Endocrinology Rare Disease medicines, SKYTROFA and YORVIPATH, and, following our pre-NDA meeting, planned filings for our third, TransCon CNP for achondroplasia,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Our expanding TransCon technology platform and partnerships demonstrate that our business model built on fast, successful drug development can create substantial stakeholder value.” Selected Key Updates and Milestones TransCon™ hGH (lonapegsomatropin, marketed as SKYTROFA®) SKYTROFA full-year 2024 revenue, with a single indication in pediatric GHD, expected to be ~€202 million excluding sales deductions related to prior years (based on unaudited preliminary estimate of full-year 2024 SKYTROFA revenue of ~€197 million plus ~€5 million of sales deductions related to prior years).U.S. SKYTROFA volume (mg) increased 84% in 2024 year-over-year resulting in an estimated 6.5% market share of the total U.S. growth hormone market for 2024 (based on third party prescription data).Prescription Drug User Fee Act (PDUFA) goal date of July 27, 2025 for FDA review of supplemental BLA for the treatment of adults with growth hormone deficiency; pending approval, U.S. commercial launch planned in the fourth quarter of 2025.During the third quarter of 2025, plan to submit an Investigational New Drug (IND) application or similar for a basket trial evaluating TransCon hGH in additional indications. TransCon PTH (palopegteriparatide, marketed as YORVIPATH®) YORVIPATH full-year 2024 unaudited preliminary revenue estimate of ~€29 million.YORVIPATH commercially available for prescription in Germany and Austria since January 2024. Outside Germany and Austria, providing product through early access routes, such as ‘named patient,’ until commercial reimbursement established. ~700 patients on treatment in our Europe Direct and International Markets at the end of 2024.YORVIPATH commercially available for prescription since late December 2024 in the U.S. As of January 9, 2025, 324 patients enrolled into the Ascendis Signature Access Program or direct with specialty pharmacy, with over half of prescriptions for patients new to YORVIPATH.Expect commercial launch in at least five additional Europe Direct countries in 2025. TransCon CNP (navepegritide) Following pre-NDA meeting with FDA, plan to submit New Drug Application (NDA) for the treatment of children with achondroplasia during the first quarter of 2025, and submit Marketing Authorisation Application to the European Medicines Agency during the third quarter of 2025.Presented new data demonstrating significant improvements in leg bowing, a common complication in achondroplasia, observed with TransCon CNP compared to worsening observed with placebo in pivotal ApproaCH Trial.Topline Week 26 results from Phase 2 COACH Trial (TransCon CNP in combination with TransCon hGH) expected in the second quarter of 2025.During the fourth quarter of 2025, plan to submit an IND or similar for the treatment of hypochondroplasia. Expanding the TransCon Platform & Pipeline New TransCon protein degrader platform designed to enable efficient clearance of hormones, cytokines, and other targets. First planned TransCon protein degrader product candidate designed to normalize excess FGF-23 hormone levels for patients with X-linked hypophosphatemia. Financial Update Unaudited preliminary estimate of total full-year 2024 product revenue of ~€226 million: SKYTROFA full-year 2024 revenue expected to be ~€202 million excluding sales deductions related to prior years (based on unaudited preliminary estimate of full-year 2024 SKYTROFA revenue of ~€197 million plus ~€5 million of sales deductions related to prior years).YORVIPATH full-year 2024 unaudited preliminary revenue estimate of ~€29 million. Unaudited preliminary estimate of total full-year total 2024 revenue of ~€364 million Includes $100 million Novo Nordisk milestone payment as non-product revenue. December 31, 2024 pro forma cash balance of ~€655 million (based on unaudited preliminary estimate of December 31, 2024 cash balance of €560 million plus expected payment from Novo Nordisk of $100 million). Presentation at J.P. Morgan Healthcare Conference on Monday, January 13A live webcast of the event will be available via the Investors & News section of the Ascendis Pharma website at https://investors.ascendispharma.com. The presentation will begin at 11:15 a.m. Eastern Time / 8:15 a.m. Pacific Time. A webcast replay will be available for 30 days. The Company’s slides from the J.P. Morgan presentation will be available on the same Investor Relations website at https://investors.ascendispharma.com. About Ascendis Pharma A/SAscendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of Patients, Science, and Passion, Ascendis uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more. Forward-Looking StatementsThis press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) Ascendis’ potential for rapid revenue growth, (ii) planned regulatory submissions for TransCon CNP and TransCon hGH, (iii) the ability of Ascendis’ business model to create substantial stakeholder value, (iv) expected full-year 2024 SKYTROFA, YORVIPATH and total revenues, (v) the PDUFA date of the supplemental BLA for SKYTROFA for the treatment of adults with growth hormone deficiency and Ascendis’ plans for a U.S. commercial launch, if approved, (vi) Ascendis’ expectations regarding the commercial launch of YORVIPATH in additional Europe Direct Countries, (vii) the timing of topline results from the Phase 2 COACH Trial of TransCon CNP, (viii) Ascendis’ expectations regarding its TransCon protein degrader platform and first TransCon degrader product candidate, (ix) the expected payment from Novo Nordisk, (x) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (xi) Ascendis’ use of its TransCon technologies to create new and potentially best-in-class therapies. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers, distributors and service providers for Ascendis’ products and product candidates; unforeseen safety or efficacy results in Ascendis’ development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ prospectus supplement filed on September 20, 2024 and Ascendis’ current and future reports filed with, or submitted to, the U.S. Securities and Exchange Commission (SEC), including its Annual Report on Form 20-F filed with the SEC on February 7, 2024. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law. Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, TransCon, SKYTROFA®, and YORVIPATH® are trademarks owned by the Ascendis Pharma group. © January 2025 Ascendis Pharma A/S. Investor Contacts:Media Contact:Scott SmithMelinda BakerAscendis PharmaAscendis Pharmair@ascendispharma.com +1 (650) 709-8875 media@ascendispharma.comPatti Bank ICR Healthcare +1 (415) 513-1284 patti.bank@icrhealthcare.com

临床2期申请上市临床结果

2024-12-11

·Pharmaceutical Technology

Er-Kim and Ascendis’ endocrine therapy agreement extended in Eurasia

TransCon CNP, one of the therapies in the agreement, is currently in clinical development for achondroplasia. Credit: Louis Reed on Unsplash. Er-Kim has extended its exclusive agreement with Ascendis Pharma , broadening the reach of three endocrinology treatments across multiple countries in Eurasia. This follows the initial exclusive distribution arrangement announced in January 2024 which covered Turkey along with Central and Eastern Europe. The extended agreement appoints Er-Kim as the exclusive distributor for Ascendis Pharma’s endocrinology rare disease portfolio in Azerbaijan, Armenia, Georgia, Kyrgyzstan, Kazakhstan, Turkmenistan, Tajikistan and Uzbekistan. The therapies included are once-weekly human growth hormone, SKYTROFA (lonapegsomatropin), along with TransCon CNP (navepegritide) and the first-in-class parathyroid hormone (PTH) replacement therapy, YORVIPATH (palopegteriparatide). SKYTROFA received approval in the European Union for growth hormone-deficient individuals aged three to 18. It is also approved in the US for children aged one year and above with growth failure due to inadequate secretion of growth hormone. TransCon CNP is currently in clinical development for achondroplasia, the widespread form of skeletal dysplasia causing disproportionate short stature. Ascendis Pharma is advancing this treatment following the completion of its pivotal ApproaCH trial. Ascendis Pharma Europe and international president and executive vice-president Camilla Harder Hartvig stated: “At Ascendis Pharma we are driven by our commitment to make a meaningful difference in the lives of patients. “We are therefore pleased to extend our existing partnership with Er-Kim to further broaden patient access to our innovative endocrinology rare disease treatment options across Eurasia.” YORVIPATH addresses chronic hypoparathyroidism, a condition characterised by low or absent levels of PTH. It received approval in the European Union in November 2023 for adults with this condition and in the US for the treatment of hypoparathyroidism in adults. Er-Kim CFO Mert Zorlular stated: “We are proud of what we’ve achieved together over the past year and look forward to our expanded collaboration with Ascendis Pharma in broadening the distribution of its rare endocrinology disease therapies to address the significant unmet need for access to treatments in these countries. “Our success in making life-saving treatments available to patients regardless of location, one country at a time, depends on our partnerships with innovative companies such as Ascendis Pharma.”

上市批准引进/卖出

100 项与 Navepegritide 相关的药物交易

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
软骨发育不全	临床3期	美国	Ascendis Pharma Growth Disorders A/S	2023-03-03
软骨发育不全	临床3期	澳大利亚	Ascendis Pharma Growth Disorders A/S	2023-03-03
软骨发育不全	临床3期	加拿大	Ascendis Pharma Growth Disorders A/S	2023-03-03
软骨发育不全	临床3期	丹麦	Ascendis Pharma Growth Disorders A/S	2023-03-03
软骨发育不全	临床3期	爱尔兰	Ascendis Pharma Growth Disorders A/S	2023-03-03
软骨发育不全	临床3期	新西兰	Ascendis Pharma Growth Disorders A/S	2023-03-03
软骨发育不全	临床3期	西班牙	Ascendis Pharma Growth Disorders A/S	2023-03-03

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适应症

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05598320 (ApproaCH, PipelineReview) 人工标引	临床2/3期	软骨发育不全	84	TransCon CNP (ages 2-11 years)	膚網選網觸蓋憲憲衊鹽(構鑰醖積獵醖顧淵鹽觸) = 憲艱積壓範齋簾膚選鹽艱艱簾膚窪積製膚鹹選 (鏇餘製餘願膚繭選淵餘 ) 更多	积极	2024-09-16
				Placebo (ages 2-11 years)	膚網選網觸蓋憲憲衊鹽(構鑰醖積獵醖顧淵鹽觸) = 鏇獵糧蓋觸膚醖鏇範顧艱艱簾膚窪積製膚鹹選 (鏇餘製餘願膚繭選淵餘 ) 更多
NCT04085523 (ACcomplisH, Company_Website) 人工标引	临床2期	软骨发育不全	57	TransCon CNP	顧淵選艱衊衊衊獵窪構(糧艱齋鏇壓壓艱積艱壓) = patients treated for one year at the pivotal 100μg/kg/week dose demonstrated significant improvements in well-being and physical functioning compared to placebo. 醖鏇繭構構衊壓糧糧膚 (繭積簾觸糧鹹艱餘網壓 ) 更多	积极	2024-06-24
				Placebo
NCT04085523 (ACcomplisH, CTgov)	临床2期	软骨发育不全	57	TransCon CNP (TransCon CNP 6 mcg)	鏇獵餘鏇簾觸壓憲蓋簾(壓鏇廠網願窪鏇糧膚壓) = 衊範遞餘衊齋窪膚簾構觸窪鹽構簾鹽淵醖鏇壓 (顧憲鹽築製範製膚遞壓, 構觸夢衊積築範衊製構 ~ 艱範構鏇網淵醖窪壓簾) 更多	-	2024-03-07
				TransCon CNP (TransCon CNP 20 mcg)	鏇獵餘鏇簾觸壓憲蓋簾(壓鏇廠網願窪鏇糧膚壓) = 衊願積夢蓋憲願鬱鹽積觸窪鹽構簾鹽淵醖鏇壓 (顧憲鹽築製範製膚遞壓, 築鬱壓網廠鹹鹽築鏇夢 ~ 糧選糧鏇顧範糧鑰壓蓋) 更多
NCT04085523 \| NCT05598320 (ACcomplisH, GlobeNewswire) 人工标引	临床2期	软骨发育不全	-	TransCon CNP 100 µg/kg/week	壓蓋繭構淵積齋鬱糧顧(餘糧壓艱餘觸淵築鏇夢): P-Value = 0.002 更多	积极	2023-12-20
				placebo
NCT05246033 (ACcomplisH China, PRNewswire) 人工标引	临床2期	软骨发育不全	24	TransCon CNP 50μg/kg/week	構鹽觸積簾糧積糧選製(獵窪鑰鹽鏇鹹願廠觸淵) = 膚構鑰選獵鑰選鏇鬱膚夢獵網築壓淵鹹積鹽遞 (遞醖築遞觸構願網鬱蓋 ) 达到	积极	2023-11-16
				TransCon CNP 100μg/kg/week	構鹽觸積簾糧積糧選製(獵窪鑰鹽鏇鹹願廠觸淵) = 製廠衊醖窪簾蓋壓積糧夢獵網築壓淵鹹積鹽遞 (遞醖築遞觸構願網鬱蓋 ) 达到
NCT04085523 (ACcomplisH, Pubmed)	临床2期	软骨发育不全	42	TransCon CNP 100 μg CNP/kg/week	襯鏇構範製襯艱選窪窪(憲鹽簾窪積繭壓鹹衊鹹) = 鹽選醖網廠選膚鬱膚製醖繭鬱顧顧構繭顧憲構 (醖廠齋淵衊簾遞簾觸齋, 4.74 ~ 6.11)	积极	2023-11-01
				Placebo	襯鏇構範製襯艱選窪窪(憲鹽簾窪積繭壓鹹衊鹹) = 齋糧夢製糧鬱淵夢餘築醖繭鬱顧顧構繭顧憲構 (醖廠齋淵衊簾遞簾觸齋, 3.75 ~ 4.94)
NCT05598320 (ACcomplisH, ENDO2023)	临床2期	软骨发育不全	57	TransCon CNP 6 μg/kg/week	窪壓壓餘簾憲窪顧鹽觸(鏇繭醖鬱糧蓋餘製壓廠) = 範願鏇顧襯醖淵鑰夢鹹衊鑰製衊夢鏇積積壓廠 (構窪鑰構壓艱醖襯製簾 )	积极	2023-10-05
				TransCon CNP 20 μg/kg/week	窪壓壓餘簾憲窪顧鹽觸(鏇繭醖鬱糧蓋餘製壓廠) = 鹹鬱築繭製襯築願艱網衊鑰製衊夢鏇積積壓廠 (構窪鑰構壓艱醖襯製簾 )