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 ; Smith, Alden ; Legare, Janet M ; Volck, Birgitte ; Schnabel, Dirk ; Chakraborty, Mukta ; Hofman, Paul L ; Ljungberg, Merete ; 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）

2025-07-04

·E药经理人

生物药产业合力突围：一款新型长效激素的本土化落地样本

在生物药管线冲刺迈入“兑现期”前，如何将高技术壁垒的创新药物真正推向商业化，成为Biotech绕不开的现实大考。面对复杂制剂开发中的技术挑战与体系协同难题，维昇药业与药明生物通过一场围绕新型长效生长激素的深度合作，探索出一条从技术共建到产业转化的可行路径，也为行业提供了新的协同范式。过去十年，全球新药研发IND数量井喷，PD-1、CD47 、GLP-1、ADC、双抗，热门靶点与新技术密集亮相，一场由创新带动的“军备竞赛”如火如荼。但当一部分Biotech挺进临床三期，站到商业化门槛时，一个现实的问题横亘在眼前：如何将创新项目合规、高效、高质量地推向规模化生产，完成从概念验证到商业化落地的完美闭环。这成为一场系统性的挑战。特别是在复杂剂型逐步成为产品差异化竞争力的当下，Biotech面对的不再是寻找一家委托加工商，而是需要找到合作伙伴全面为其赋能，共建系统高效的体系，“一起把药做成“。维昇药业正是这一协作模式下的代表性实践者。作为一家专注内分泌治疗领域的Biotech，维昇目前已有三款创新产品稳步推进本地注册与商业化路径：其中，长效生长激素产品隆培促生长素在中国上市在即；针对甲状旁腺功能减退症（简称甲旁减，HP）的帕罗培特立帕肽和用于儿童软骨发育不全（ACH）的那韦培肽也即将完成国内的临床开发。其中，作为首个采用TransCon技术实现长效化并匹配双腔制剂设计的产品，隆培促生长素的中国生产落地不仅代表着维昇药业在复杂制剂领域的战略发力，也意味着中国市场上将出现本土生产的且具备国际同等水平的创新型长效生长激素。维昇药业与药明生物围绕隆培促生长素项目的深度合作，随着前者新药上市进程的有序推进，逐渐成为一个可参考的复杂创新制剂落地样本：管线从海外引进，到国产商业放大；从原液工艺转移，到制剂药械适配——一整套系统能力协同路径正在被跑通。这不仅是一次“服务合作”，而是一场以Biotech为中心、CRDMO平台能力为支撑、协同机制为抓手的“共创式实践”。从合作模式看，维昇药业和药明生物并非传统的“服务与被服务”关系。药明生物首席技术官 Sherry Gu这样总结：“CDMO不仅是一种服务，更是一种创新引擎。帮助客户在不确定中提速，在挑战中突围。”这对应的是维昇药业首席执行官卢安邦清晰的战略目标：“我们希望三年后（2028年）再回头看，公司第一步是核心产品隆培促生长素获批上市、第二步是核心产品进入商业化、制剂项目落地，我们的预期就算达成了。”01协同中的“确定性”：从信任到落地维昇药业在2018年成立，可以被看作是海外创新药企“反向NewCo”的代表。公司由Ascendis Pharma A/S（Nasdaq: ASND）与维梧资本（Vivo Capital）领导的联合投资人共同创立。维昇药业获得Ascendis独家授权，将其旗下的内分泌疾病治疗方案在大中华区开发、生产及商业化，其授权覆盖中国大陆及港澳台地区。具体到隆培促生长素，维昇药业的目标一方面是快速推进进口产品上市，另一面是本地化产品快速达产，凭借在中国的协同体系实现稳定供应与持续创新。由于隆培促生长素本身涉及复杂与精准的结构设计，卢安邦最初为其本土化落地设定了两个关键目标，“技术严谨性”与“交付确定性”。因此，维昇药业在选择合作伙伴时慎之又慎。众所周知，选择一个既懂国际合规，又能实现本土落地的协作平台，远比“找代工方”复杂得多——它需要合作双方在技术、质量、文化、响应机制四个维度上充分协同，且在协作中形成互信机制。从后续合作的推进来看，药明生物的系统性支持，正是维昇药业“共创合作”理念的完美体现。药明生物不仅具备复杂制剂的工艺开发能力，其深厚的批量商业化交付经验更是稀缺资源。从端到端工艺建立，到制造流程验证，药明生物不断输出成套方案，并且与维昇药业共同优化工艺数据。与此同时，药明生物对维昇药业项目管理的专业度，以及需求快速响应，让卢安邦印象深刻：“无论何时针对何种问题的沟通，都可以得到药明生物的快速响应，实现项目真正意义上的‘任务同步’”。中国生长激素市场随着新产品的不断上市，正式进入格局迭代周期。根据弗若斯特沙利文报告，于2023年，中国在全球人生长激素市场占据最大份额，超过美国，市场规模为人民币116亿元，占全球市场的34%。预计到2030年，中国人生长激素市场规模将达到286亿元，自2023年至2030年的年复合增长率为13.7%。从手握海外引进的新型长效生长激素管线，再到实现国内本地化量产。卢安邦提到：“如果今天要选择一个合作伙伴，我们肯定还是选择药明生物。”其背后的逻辑并不复杂，“第一是专业技术，第二是质量体系，第三是团队是否务实、是否能为客户多走一步。”据悉，药明生物会将此合作模式，复制到其他项目中，与创新药企共同成长。02国产复杂制剂的新路径，正在被打开隆培促生长素是一款新型长效生长激素，用于治疗儿童生长激素缺乏症（PGHD），该产品目前已在美国及欧洲获批上市，但因其使用的TransCon(暂时连接)技术转移难度高，国内具备承接能力的CDMO屈指可数。药明生物因何有能力、有资质、有体量承接隆培促生长素落产？这场合作的另一个关键节点，是药明生物成都微生物商业化生产基地。2025年6月9日，在药明生物成都微生物商业化生产基地建设正式启动活动上，维昇药业与药明生物宣布了一项重要的合作进展：维昇药业核心产品隆培促生长素商业化批次的技术转移项目正式落地成都。在这一合作中，卢安邦着重提到：共同建设国内首条双腔冻干制剂生产线。“过去很多的冻干制剂在使用时，通常需要一瓶西林瓶的冻干粉，还有一瓶复溶剂，不仅使用很麻烦，而且有污染的风险。”卢安邦介绍称，双腔技术是在密封容器系统中，将冻干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。在注射之前，冻干药物与复溶剂通过旁路进行复溶，大大提高了药物使用的便利性、安全性以及剂量准确性。凭借国内首个双腔冻干制剂生产线的落地，药明生物在国内率先建立起双腔冻干制剂生产技术。另外，这一平台不仅服务维昇，也将对其他国际复杂制剂客户开放，形成生态集聚效应。Sherry Gu指出：“目前在国内甚至国际上，（微生物）这都是非常稀缺的平台，有很多蛋白只能通过CHO细胞（中国仓鼠卵巢细胞）表达，所以我们希望通过成都微生物平台弥补这个空缺。”为了满足隆培促生长素项目的技术规格和商业化需求，药明生物也正在推进关键产线的能力扩展。公司在成都微生物商业化生产基地搭建了国内首条双腔冻干制剂生产线，并配备西林瓶生产线，制剂年产能将超1000万支。隆培促生长素预计2025年内在中国获批，计划在2028年实现本土规模化生产。与此同时，药明生物成都微生物商业化生产基地规划面积达95,000平方米，将专注于原液（DS）生产和制剂（DP）生产，覆盖多肽、抗体片段、质粒 DNA、酶、细胞因子、病毒样颗粒（VLP）等产品的商业化生产。该基地预计于 2026 年底实现GMP投产，届时将配备15,000 升的发酵罐，原液年产能可达 80-110 批次，未来最大发酵规模可拓展至 60,000 升。正如Sherry Gu所说：“我们希望成都基地不仅服务于一个项目，更是补足行业在微生物表达和复杂制剂一体化交付方面的能力短板。”这一设施的搭建，也标志着CDMO平台能力正从“流程化建设”转向“项目牵引式建设”——项目挑战驱动体系升级，体系升级反哺更多Biotech企业的落地可能。03协同型合作，打造Biotech价值共同体在维昇药业与药明生物的合作中，双方最终的收获不仅是一款产品的本地化落地，更是一次互相的能力认可。维昇药业的目标，不只是推动隆培促生长素上市，而是以一个产品为支点，建立起本土Biotech对高质量商业化制剂能力的信心。药明生物方面，则在类似的共创中不断强化自身的跃迁能力。Sherry Gu指出：“我们的质量体系能通过几十项来自于FDA、EMA等机构的检查，这背后是因为药明生物在技术上细致到每一个环节，精益求精。”在这种协同逻辑下，CDMO的角色也正在发生变化。过去，CDMO往往被视为“交付单位”或“外包执行体”。但现在，特别是在复杂制剂与商业化节点愈发交织的当下，作为业内领先的CRDMO，药明生物不仅能够成为Biotech所需要的，具备技术协同能力、交付透明度、体系建设能力的“系统合作方”，更是客户价值共同体的成员。例如，在维昇药业与药明生物就隆培促生长素建立起一套超越传统服务边界的合作机制中，双方不止共享项目时间线，更共享技术路径、质量体系甚至人才接口标准。药明生物CRDMO+的合作模式正在成为Biotech的“加速器”：已经不局限于仅是服从于项目的体系配置，而是以高度响应的协同团队，实现跨链条、跨角色的战略协作。正如访谈中卢安邦所言，要“有所为、有所不为”，选择与谁共同行动，将决定制剂项目成功的高度和速度。当中国创新药行业发展进入“新常态”，新兴Biotech早已不再执着于拿地建厂，选择将“前半程”能力做到极致。同时为其不具备的 “临门一脚”——制剂、质量、稳定性、交付能力寻找合作支持，正在成为越来越多Biotech的选择。维昇药业与药明生物的合作，正是一次对上述能力链条的“共建型验证”。在未来三到五年，CRDMO必将成为Biotech能力体系外部构建中最具战略价值的一环。而维昇药业与药明生物打造出的隆培促生长素本地化生产样本，或许就是这场产业跃迁的前排注脚。一审| 黄佳二审| 李芳晨三审| 李静芝

上市批准临床3期抗体药物偶联物核酸药物

2025-06-25

·创鉴汇

25家中国内分泌与代谢领域创新药公司获融资！

内分泌与代谢疾病因患者基数庞大且临床需求持续增长，成为创新药研发的重要方向。据公开数据统计，2025年至今，该领域至少发生25起创新药公司融资事件，已披露融资总额超125亿元。融资覆盖糖尿病、肥胖、高脂血症等疾病，涉及小分子药物、多肽、蛋白质药物及细胞疗法等技术路径。从IPO公司到新锐技术平台，资本正加速涌入这一充满潜力的治疗领域。融资亮点聚焦多肽与蛋白质药物主导代谢性疾病疗法创新：近半数获融资公司重点布局多肽与蛋白质药物。如维昇药业推进甲状旁腺功能减退症多肽疗法，派格生物开发GLP-1类多肽药物治疗肥胖，质肽生物则聚焦长效重组蛋白治疗糖尿病。此类药物因靶向性强、半衰期可控，在代谢调节领域临床优势显著。干细胞疗法剑指糖尿病治疗瓶颈：至少4家公司聚焦干细胞疗法治疗糖尿病及其并发症。星赛瑞真开发多能干细胞（iPSC）衍生胰岛细胞疗法CRG-002动物试验结果积极；睿健医药开发iPSC来源眼科通用型细胞治疗产品，有潜力拓展至糖尿病性视网膜病变等与代谢疾病相关的视网膜病变；中盛溯源则推进iPSC来源肾脏修复细胞产品治疗糖尿病肾病等。该领域融资集中于B+轮及以前，反映资本正加速早期技术转化。AI、新分子实体创新技术引领大额融资：11家公司获得亿元及以上融资。其中，英矽智能凭借其端到端AI平台，推动包括代谢紊乱和肥胖在内的管线进展，获得1.23亿美元E轮融资。达歌生物利用其独特的GlueXplorer分子胶平台开发新型蛋白降解疗法，珂阑医药聚焦胆固醇代谢新靶点，均获得亿元以上融资，体现了源头创新技术平台的高认可度。维昇药业：内分泌疾病药物研发公司3月21日，维昇药业在港交所IPO，募资约6.72亿港元。维昇药业成立于2018年11月，是一家处于研发后期、产品接近商业化的生物医药公司，专注于在中国为患者提供特定内分泌疾病的治疗方案。该公司目前拥有一款核心产品及两款其他在研候选药物，核心产品每周一次的长效生长激素隆培促生长素已经于2024年3月向中国国家药监局（NMPA）递交上市申请。隆培促生长素（lonapegsomatropin）是一款每周一次的长效生长激素替代疗法，用于治疗儿童生长激素缺乏症（PGHD，一种生长激素不足导致的18岁以下患者中常见的矮小症）。与每日一次人生长激素相比，隆培促生长素提供了方便的给药方案，注射频率为每周一次，这可能会提高儿童患者在日常生活中给药的依从性。此外，该公司研发管线中：那韦培肽（navepegritide）是一款C型利钠肽的长效前药，每周一次给药，拟开发用于治疗2至10岁软骨发育不全。帕罗培特立帕肽（palopegteriparatide)是一款每日一次的甲状旁腺激素替代疗法，拟开发用于治疗慢性甲状旁腺功能减退症。派格生物：慢病新药研发生物技术公司5月27日，派格生物（Pegbio）完成约2.32亿港元IPO融资。该公司成立于2008年，专注于自主研究及开发慢性病创新疗法，重点关注内分泌代谢领域。通过自主开发的HECTORTM技术平台设计和筛选具有新靶标、新位点、新机制的分子实体。派格生物聚焦糖尿病、肥胖等代谢疾病，建立了多肽药物、蛋白质药物及小分子药物为主的研发管线体系。其核心产品PB-119为自主开发、接近商业化阶段的长效胰高血糖素样肽-1（GLP-1）受体激动剂。PB-119主要用于二型糖尿病（T2DM）及肥胖症一线治疗。凭借聚乙二醇化技术，可实现一周一次给药。PB-119在中国用于治疗T2DM的新药上市申请（NDA）于2023年9月获药监局受理。派格生物预计于2025年获得NDA批准并在中国商业化推出用于治疗T2DM的PB119。珂阑医药：胆固醇代谢创新药研发公司2月21日消息，珂阑医药完成超亿元人民币A+轮融资。此次融资资金将主要用于加速推进多个核心管线进入临床申报和开发，以及研发平台和团队的拓展。珂阑医药立足于糖脂代谢领域的前沿科研，聚焦胆固醇代谢通路中成药性高的创新性靶点，开展针对代谢性疾病（高脂血症、肥胖症、糖尿病、动脉粥样硬化症以及MASH）等重大疾病的创新药研发工作。原研项目转化来自宋保亮院士实验室，致力于“健康代谢，健康心血管，健康生活”的理念，力争为代谢疾病患者治疗提供可选方案。针对代谢性疾病和肿瘤，珂阑医药整合了新药研发中基于靶标调控机理的报告基因筛选系统，蛋白质小分子互作系统、基于靶标功能的体外药效测试系统，构建生化与细胞测试自主研发平台，全方位进行药物优化，高效推动药物研发深入与开展。质肽生物：蛋白质药物开发公司2月17日消息，质肽生物获亿元融资。质肽生物成立于2018年9月，专注用大肠杆菌生产重组蛋白质药物，致力于开发治疗慢性代谢性疾病（如糖尿病、肥胖、非酒精性脂肪性肝炎等）创新生物药。目前质肽生物管线中有10个在研重组蛋白创新药和生物类似物品种，包括司美格鲁肽注射液生物类似药ZT001，以及有望实现仅需每月注射1次的新型超长效GLP-1R激动剂ZT002等，口服GLP-1类多肽药物ZT006片等。2024年11月，ZT006片获得临床试验默示许可，拟开发用于成人2型糖尿病患者的血糖控制。ZT006是新一代口服蛋白药品种，主要治疗糖尿病和肥胖症。经过优化的ZT006具有较高的生物利用度，有希望成为继口服司美格鲁肽之后的新一代口服GLP-1品种。口服给药是公认的、可以显著提高慢性病患者依从性的一种给药方式。星赛瑞真：干细胞疗法研发公司3月17日，星赛瑞真宣布完成近亿元人民币天使+轮融资。这是星赛瑞真自2024年6月获得复健资本及创瑞投资的天使轮投资以来，在半年时间内再次获得天使轮老股东的追加投资。本次融资资金将用于推进具有差异化的iPSC衍生的胰岛细胞管线（CRG-002）和iPSC衍生的角膜内皮细胞产品（CRG-101）进入临床研究阶段，以及用于公司其它早研管线的研发布局。星赛瑞真成立于2022年，聚焦细胞治疗和再生医学转化，构建了iPSC快速重编程技术、基于CRISPR/Cas12b的iPSC基因编辑平台以及生物信息学指导下的不同胚层分化技术平台。在代谢疾病领域，星赛瑞真主要开发胰岛替代疗法用于1型糖尿病治疗。其在研管线CRG-002管线为iPSC衍生胰岛细胞，已经动物试验验证，有望满足糖尿病患者巨大临床需求。读者们请星标⭐创鉴汇，第一时间收到推送免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转发/复制至其他平台。转发授权请在「创鉴汇」微信公众号留言联系我们。更多数据内容推荐点击“在看”，分享创鉴汇健康新动态

IPO细胞疗法

2025-06-09

·EINPresswire

TransCon® hGH Boosted Treatment Benefits of TransCon® CNP in Children with Achondroplasia at Week 26 Interim Analysis of the Phase 2 COACH Trial

- For the TransCon CNP treatment-naïve cohort, combination treatment resulted in mean annualized growth velocity (AGV) of 9.14 cm/year, with an improvement in mean ACH height Z-score of +0.53 over 26 weeks - For the TransCon CNP-treated cohort, combination treatment resulted in mean AGV of 8.25 cm/year, with an improvement in mean ACH height Z-score of +0.44 over 26 weeks - The combination of TransCon hGH and TransCon CNP demonstrated accelerated improvement in body proportionality at Week 26, aligning with the increase in linear growth - Safety and tolerability data consistent with those observed for TransCon hGH and TransCon CNP monotherapies; combination treatment was generally well tolerated, with generally mild treatment-emergent adverse events (TEAEs) - Ascendis to host conference call today at 8:00 am ET COPENHAGEN, Denmark, June 09, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced Week 26 interim analysis results from its ongoing COACH Trial, the first clinical trial to evaluate combination treatment with once-weekly investigational TransCon CNP (navepegritide) and once-weekly TransCon hGH (lonapegsomatropin) in children with achondroplasia. Results demonstrated that TransCon hGH boosted treatment benefits of TransCon CNP, resulting in significant growth and proportionality improvements in children with achondroplasia after 26 weeks of combination treatment, with a safety and tolerability profile consistent with those observed for TransCon hGH and TransCon CNP monotherapies. TransCon CNP, which is under priority review as a monotherapy for children with achondroplasia by the U.S. Food & Drug Administration (FDA), is an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly, providing continuous exposure of active CNP to receptors on tissues throughout the body, including growth plates and skeletal muscle. TransCon hGH is a prodrug of somatropin administered once weekly, providing sustained release of active, unmodified somatropin. TransCon hGH is approved and marketed as SKYTROFA ® for the treatment of pediatric growth hormone deficiency and is in development for other indications. “TransCon CNP as a monotherapy has demonstrated the potential to transform the treatment of achondroplasia, and the COACH Trial at Week 26 demonstrates that TransCon hGH has the potential to boost treatment benefits of TransCon CNP with a safety profile consistent with monotherapies,” said Aimee Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Sciences and Chief Medical Officer at Ascendis Pharma. “These results highlight the unique portfolio of once-weekly TransCon CNP and once-weekly TransCon hGH, with complementary modes of action, to improve the treatment landscape for growth disorders and physical functioning.” COACH Trial Design The COACH Trial is an ongoing proof-of-concept prospective Phase 2 open-label trial to investigate the efficacy, safety, and tolerability of combined treatment with once-weekly TransCon CNP at 100 µg/kg/week and once-weekly TransCon hGH at 0.30 mg/kg/week in children with achondroplasia aged 2 to 11 years. The trial included a cohort of TransCon CNP treatment naïve children (N=12, mean age 4.67 years) and a cohort of TransCon CNP-treated children (N=9, mean age 7.89 years) who had received TransCon CNP (100 µg/kg/week) for a mean of 2.56 years in clinical trials. The trial population is representative of children with achondroplasia, except for the observed growth benefit in the TransCon CNP-treated cohort. The interim analysis will be followed by Week 52 data, expected in Q4 2025, and Ascendis plans to initiate a Phase 3 trial in Q4 2025. Highlights of the Interim Topline Week 26 COACH Trial Results For TransCon CNP treatment-naïve children, mean annualized growth velocity (AGV) was 9.14 cm/year, representing an increase from baseline at Week 26 of 4.23 cm/year, with an improvement in mean ACH height Z-score of +0.53 over 26 weeks. For TransCon CNP-treated children, mean AGV was 8.25 cm/year, representing an increase from baseline at Week 26 of 3.10 cm/year, with an improvement in mean ACH height Z-score of +0.44 over 26 weeks. Mean AGV with TransCon CNP and TransCon hGH combination treatment exceeded the 97th percentile of average-stature children. Children treated with TransCon hGH and TransCon CNP demonstrated accelerated improvement in body proportionality at Week 26, aligning with the increase in linear growth. Bone age advanced in line with chronologic age. Safety and tolerability data were consistent with those observed for TransCon hGH and TransCon CNP monotherapies; combination treatment was generally well tolerated, with generally mild TEAEs. A slide presentation with these data can be found on the Investor Relations & News section of the Ascendis Pharma website: https://investors.ascendispharma.com . Conference Call and Webcast Information Ascendis Pharma will host a conference call and webcast today at 8:00 am Eastern Time (ET) to discuss these results. Those who would like to participate may access the live webcast here , or register in advance for the teleconference here . The link to the live webcast will also be available on the Investors & News section of the Ascendis Pharma website at https://investors.ascendispharma.com . A replay of the webcast will be available on that page shortly after the conclusion of the event for 30 days. About Achondroplasia Achondroplasia is a rare genetic condition arising from a systemic fibroblast growth factor receptor 3 (FGFR3) variant that leads to an imbalance in the effects of the FGFR3 and CNP signaling pathways, estimated to affect more than 250,000 people worldwide. While historically considered a bone growth disorder, the FGFR3 variant seen in achondroplasia is expressed in tissues throughout the body, causing serious muscular, neurological, and cardiorespiratory complications in addition to skeletal dysplasia. Medical complications of achondroplasia vary across different stages of life. Throughout infancy and childhood, observed complications include spinal deformities, enlarged brain ventricles, impaired muscle strength and stamina, hearing deficits and chronic ear infections, upper airway obstructions, sleep-disordered breathing, hip problems, leg bowing, and chronic pain; many of these persist or worsen in adulthood. These medical complications can have detrimental effects on quality of life, physical functioning, and psychosocial function. Individuals with achondroplasia often require multiple surgeries and procedures to alleviate the condition’s many complications. About Ascendis Pharma A/S Ascendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. 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 Statements This 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) TransCon CNP’s potential to transform the treatment of achondroplasia, (ii) TransCon hGH’s potential to boost treatment benefits of TransCon CNP with a safety profile consistent with monotherapies, (iii) the potential for the combination of TransCon CNP and TransCon hGH to improve the treatment landscape for growth disorders and physical functioning, (iv) the expected timing of Week 52 data from the COACH Trial and Ascendis’ plans to initiate a Phase 3 trial in Q4 2025, (v) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients, and (vi) Ascendis’ application of its TransCon technologies to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. 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, including tariffs and trade policies. 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’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 12, 2025, and Ascendis’ other future reports filed with, or submitted to, the SEC. 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, SKYTROFA, and TransCon are trademarks owned by the Ascendis Pharma group. © June 2025 Ascendis Pharma A/S. Investor Contacts: Media Contact: Sarada Weerasinghe Melinda Baker Ascendis Pharma Ascendis Pharma ir@ascendispharma.com media@ascendispharma.com Patti Bank ICR Healthcare +1 (415) 513-1284 patti.bank@icrhealthcare.com Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

临床结果临床2期优先审批临床3期

100 项与 Navepegritide 相关的药物交易

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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	申请上市	美国	Ascendis Pharma A/S	2025-03-31

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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)	鬱襯網襯壓膚蓋襯齋糧(鏇遞觸糧簾鹽遞鏇鹽築) = 淵糧選夢餘壓艱選艱淵糧範餘鹹繭糧鹽糧廠獵 (構製鹽壓鹽製獵糧鏇願 ) 更多	积极	2024-09-16
				Placebo (ages 2-11 years)	鬱襯網襯壓膚蓋襯齋糧(鏇遞觸糧簾鹽遞鏇鹽築) = 餘壓廠積夢窪衊艱鏇壓糧範餘鹹繭糧鹽糧廠獵 (構製鹽壓鹽製獵糧鏇願 ) 更多
NCT04085523 (ACcomplisH, Company_Website) 人工标引	临床2期	软骨发育不全	57	TransCon CNP	網顧積遞鏇鹹獵構觸淵(齋壓襯憲窪餘蓋夢觸鹹) = patients treated for one year at the pivotal 100μg/kg/week dose demonstrated significant improvements in well-being and physical functioning compared to placebo. 願壓膚獵範淵餘糧襯衊 (衊憲淵膚鬱簾觸艱鹹襯 ) 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)	網遞簾觸餘醖鹹廠網淵(繭範淵積遞製鏇醖齋獵) = 壓壓夢齋獵積憲夢顧積選餘糧鏇範膚顧醖築積 (構簾願選鏇獵鹹獵願遞, 遞夢繭襯鏇蓋糧顧構獵 ~ 觸蓋鑰窪製鹽廠顧範顧) 更多	-	2024-03-07
				TransCon CNP (TransCon CNP 20 mcg)	網遞簾觸餘醖鹹廠網淵(繭範淵積遞製鏇醖齋獵) = 廠蓋遞蓋觸襯繭觸蓋廠選餘糧鏇範膚顧醖築積 (構簾願選鏇獵鹹獵願遞, 夢鬱網鑰願窪構構簾蓋 ~ 蓋獵衊鑰獵夢壓積網鹹) 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	糧繭廠願襯餘廠構網簾(窪醖齋獵製淵積鹽鏇憲): 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	廠選襯廠窪簾膚艱蓋簾(糧遞觸鑰醖獵蓋遞獵願) = 願憲鑰構遞選網顧膚鬱繭觸簾簾遞鬱積觸鬱衊 (憲簾獵鏇鏇夢鑰積築糧 ) 达到	积极	2023-11-16
				TransCon CNP 100μg/kg/week	廠選襯廠窪簾膚艱蓋簾(糧遞觸鑰醖獵蓋遞獵願) = 遞艱鏇構蓋願願膚糧積繭觸簾簾遞鬱積觸鬱衊 (憲簾獵鏇鏇夢鑰積築糧 ) 达到
NCT04085523 (ACcomplisH, Pubmed)	临床2期	软骨发育不全	42	TransCon CNP 100 μg CNP/kg/week	齋範選襯構衊齋獵顧膚(齋鏇衊憲淵窪淵鏇範夢) = 膚夢選鹽簾構網範衊鏇壓構觸淵淵艱憲顧鹽繭 (襯簾獵鹹願獵衊膚顧繭, 4.74 ~ 6.11)	积极	2023-11-01
				Placebo	齋範選襯構衊齋獵顧膚(齋鏇衊憲淵窪淵鏇範夢) = 壓積積憲築網餘襯鏇鏇壓構觸淵淵艱憲顧鹽繭 (襯簾獵鹹願獵衊膚顧繭, 3.75 ~ 4.94)
NCT05598320 (ACcomplisH, ENDO2023)	临床2期	软骨发育不全	57	TransCon CNP 6 μg/kg/week	築艱選廠遞糧艱顧鬱壓(憲網觸築淵餘蓋製遞壓) = 憲窪願齋蓋築鹹憲窪顧鏇簾構積鏇鬱窪鬱觸願 (鑰積鏇繭壓醖醖蓋網願 )	积极	2023-10-05
				TransCon CNP 20 μg/kg/week	築艱選廠遞糧艱顧鬱壓(憲網觸築淵餘蓋製遞壓) = 鏇衊簾衊壓蓋顧淵膚艱鏇簾構積鏇鬱窪鬱觸願 (鑰積鏇繭壓醖醖蓋網願 )