A Phase 3, Multicenter, Single Arm, Open-Label Trial, Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Subcutaneously Daily in Japanese Adult Subjects with Hypoparathyroidism - PaTHway Japan Trial

开始日期2021-11-02

申办/合作机构-

NCT05387070

/ Recruiting临床3期

PaTHway CHINA TRIAL: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Trial, With an Open-Label Extension, Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Subcutaneously Daily in Adults With Hypoparathyroidism

This study is limited to conduct in China only. The primary objective is to assess the treatment effect of daily TransCon PTH on serum calcium (sCa) levels within the normal range and stopping from therapeutic doses of active vitamin D (calcitriol) or active vitamin D analogue (alfacalcidol) and calcium at 26 weeks of treatment. All subjects will start with 18 mcg of study drug and will be individually and progressively titrated to an optimal dose over a 26-week double blind period, followed by an open label extension period up to 156 weeks. TransCon PTH or placebo will be administered as a subcutaneous injection using a pre-filled injection pen. Neither trial participants nor their doctors will know who has been assigned to each group. After the 26 weeks, participants will continue in the trial as part of a long-term extension study. During the extension, all participants will receive TransCon PTH, with the dose adjusted to their individual needs.

开始日期2021-07-28

申办/合作机构

维昇药业（上海）有限公司

NCT04701203

/ Completed临床3期

PaTHway TRIAL: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Trial, With an Open-Label Extension, Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Subcutaneously Daily in Adults With Hypoparathyroidism

During the first 26 weeks of the trial, participants will be randomly assigned to one of two groups: one group will receive TransCon PTH and one group will receive placebo. All subjects will start with study drug at a dose of 18 mcg/day and will be individually and progressively titrated to an optimal dose in dose increments of 3 mcg/day. TransCon PTH or placebo will be administered as a subcutaneous injection using a pre-filled injection pen. Neither trial participants nor their doctors will know who has been assigned to each group. After the 26 weeks, participants will continue in the trial as part of a long-term extension study. During the extension, all participants will receive TransCon PTH, with the dose adjusted to their individual needs. This is a global trial that will be conducted in the United States, Canada, Germany, Denmark, Norway, Italy, and Hungary.

开始日期2021-02-16

申办/合作机构

Ascendis Pharma Bone Diseases A/S

100 项与帕罗培特立帕肽相关的临床结果

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100 项与帕罗培特立帕肽相关的转化医学

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100 项与帕罗培特立帕肽相关的专利（医药）

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7,577

项与帕罗培特立帕肽相关的文献（医药）

2025-12-31·CLINICAL AND EXPERIMENTAL HYPERTENSION

Parathyroid hormone and vitamin D modulate nocturnal blood pressure dipping: a retrospective cohort study in primary hypertension

Article

作者: Liang, Xiaohui ; Xu, Yixin ; Deng, Changjiang

BACKGROUND:

Non-dipper hypertension, defined by reduced nocturnal blood pressure (BP) decline, increases cardiovascular risk. Calcium-phosphate metabolism (parathyroid hormone [PTH], vitamin D [25(OH)D]) may influence circadian BP rhythms, but their independent roles remain unclear.

OBJECTIVE:

To investigate associations between PTH, 25(OH)D, and nocturnal BP dipping in primary hypertension, addressing prior inconsistencies.

METHODS:

This retrospective cohort included 585 hypertensive adults stratified by nocturnal systolic BP dipping (dippers [≥10% decline, n = 250]; non-dippers [<10%, n = 335]) using 24-hour ambulatory monitoring. Serum PTH, 25(OH)D, renal/metabolic parameters, and vascular indices were analyzed. Multivariate linear regression assessed associations with dipping, adjusted for age, sex, renal function, and arterial stiffness.

RESULTS:

Non-dippers showed elevated PTH (median: 5.23 vs. 4.70 pmol/L, p = .011) and lower 25(OH)D (30.89 vs. 36.79 nmol/L, p < .001). Adjusted models confirmed PTH (β=-0.21, 95% CI:-0.34--0.08; p = .002) and 25(OH)D (β = 0.03, 95% CI:0.01-0.04; p < .001) as associated factors of dipping. Although statistically significant, these correlations were modest and suggested possible trends rather than strong causal relationships. Age/sex effects on BP patterns became nonsignificant after adjusting for calcium-phosphate markers.

CONCLUSION:

Calcium-phosphate dysregulation (elevated PTH, low vitamin D) independently contributes to non-dipping, suggesting biomarker-guided approaches for circadian BP control. Demographic disparities in dipping may reflect underlying mineral metabolism disturbances. Prospective trials should explore calcium-modulating therapies (e.g. vitamin D supplementation) in non-dipper hypertension.

2025-12-31·RENAL FAILURE

Persistent hyperparathyroidism after kidney transplantation in children

Article

作者: Lee, Hyun Kyung ; Ha, Jongwon ; Kang, Hee Gyung ; Han, Ahram ; Ahn, Yo Han ; Min, Sangil ; Park, Peong Gang

BACKGROUND:

Persistent hyperparathyroidism after kidney transplantation (KT) has been reported in up to 50% of adult recipients, but pediatric data remain limited. We evaluated the prevalence, skeletal manifestations, and risk factors for persistent hyperparathyroidism in children following KT.

METHODS:

In this retrospective cohort study, 107 pediatric KT recipients (58% male; median age 10.3 years) transplanted between 2004 and 2019 were analyzed. Persistent hyperparathyroidism was defined as a median parathyroid hormone (PTH) > 65 pg/mL between 3 and 12 months post-KT. Risk factors for persistent hyperparathyroidism, post KT clinical features, and treatment status were analyzed.

RESULTS:

Thirty-six patients (33.6%) had persistent hyperparathyroidism after KT. On univariable analysis, dialysis duration of 24 months or longer (p = 0.028) and pretransplant hyperphosphatemia (p = 0.026) were significantly associated with persistent hyperparathyroidism. The multivariable model identified pretransplant hyperphosphatemia as an independent predictor (OR 2.70, 95% CI 1.10-6.87; p = 0.030). There was no significant difference in height Z score change between patients with and without persistent hyperparathyroidism (p = 0.97). However, persistent hyperparathyroidism was associated with poorer graft survival (log-rank p = 0.049). Six patients received cinacalcet and one underwent subtotal parathyroidectomy for refractory hypercalcemia.

CONCLUSIONS:

Persistent hyperparathyroidism is relatively common in pediatric KT recipients, affecting one-third of patients by one-year post-transplant. Prolonged dialysis and pre-existing hyperphosphatemia before KT may be risk factors. These findings underscore the importance of optimizing chronic kidney disease-mineral bone disease management and routine PTH monitoring before and after transplant in children.

2025-12-01·Current Pain and Headache Reports

Functional Magnetic Resonance Imaging of Post-Traumatic Headache: A Systematic Review

Review

作者: Ashina, Håkan ; Christensen, Rune H ; Szabo, Edina ; Al-Khazali, Haidar M ; Iljazi, Afrim

PURPOSE OF REVIEW:

To evaluate existing functional magnetic resonance imaging (fMRI) studies on post-traumatic headache (PTH) following traumatic brain injury (TBI).

RECENT FINDINGS:

We conducted a systematic search of PubMed and Embase databases from inception to February 1, 2024. Eligible fMRI studies were required to include adult participants diagnosed with acute or persistent PTH post-TBI in accordance with any edition of the International Classification of Headache Disorders. We identified five eligible fMRI studies: two on acute PTH and three on persistent PTH. These studies assessed resting-state functional connectivity involving comparisons with one or more of the following groups: people with migraine, those with mild TBI but no PTH, and healthy controls. In acute PTH, studies focused exclusively on functional connectivity between the periaqueductal gray or hypothalamus and other brain regions. In persistent PTH, evidence of altered functional connectivity was identified primarily within cingulate, sensorimotor, and visual regions, indicating a hypersensitivity to sensory stimuli in PTH. Despite these insights, the fMRI data remains sparse and is limited by inconsistent results and small samples. The paucity of fMRI studies on PTH limits our understanding of its neurobiological basis. The available evidence suggests that alterations in functional connectivity occur within brain areas involved in emotional and sensory discriminative aspects of pain processing. However, inconsistent results and small sample sizes underscore a critical need for larger, more rigorous fMRI studies. Future studies should also consider using task-based fMRI to investigate possible hypersensitivity to different sensory stimuli in PTH after TBI.

201

项与帕罗培特立帕肽相关的新闻（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

2025-05-29

·PRNewswire

NEW THERAPY FOR CHILDREN WITH GROWTH HORMONE DEFICIENCY APPROVED IN AUSTRALIA

SKYTROFA® (lonapegsomatropin) is now registered by the Therapeutic Goods Administration (TGA) for the treatment of paediatric growth hormone deficiency.[1] SINGAPORE, May 29, 2025 /PRNewswire/ -- Independent biopharmaceutical company Specialised Therapeutics (ST) is pleased to announce the Australian registration of SKYTROFA® (lonapegsomatropin) as a new once-weekly injectable therapy for paediatric growth hormone deficiency (GHD).[2] The Therapeutic Goods Administration (TGA) has approved SKYTROFA as a treatment for "growth failure in children and adolescents aged from 3 years up to 18 years due to insufficient endogenous growth hormone secretion".[1,2] GHD is a rare disease affecting around 2-3/10,000 Australians, with approximately 2,000 children thought to be living with the condition.[3] It occurs when the pituitary gland, located at the base of the brain, does not produce an adequate level of growth hormone, which is essential for promoting healthy growth in children.[3] In children, GHD is typically characterised by slow height growth, resulting in short stature, and may also include delayed puberty, impaired hair growth and headaches.[3] The goal of treatment for GHD is to restore normal levels of growth hormone in the body, reduce physical symptoms, enhance metabolic health and improve quality of life.[3] ST Chief Executive Officer, Mr Carlo Montagner, said the TGA approval of SKYTROFA demonstrated the company's ongoing mission to support patients with rare diseases in Australia and the Asia-Pacific region. "We are delighted to have secured TGA registration of SKYTROFA for eligible Australian children with growth hormone deficiency. Beyond short stature, children and adolescents with the condition may experience considerable physical and psychosocial impacts on their daily life, such as poor concentration, decreased strength or muscle development, fatigue, and reduced quality of life.[4] "This announcement also represents a significant milestone for Specialised Therapeutics, marking the second endocrinology therapy we have successfully registered in Australia, following the approval of YORVIPATH® (palopegteriparatide) earlier this year," said Mr Montagner. SKYTROFA is being registered in Australia by ST, under an exclusive distribution agreement with biopharmaceutical company Ascendis Pharma A/S that covers Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand, and Vietnam. The Australian registration of SKYTROFA follows approvals issued to Ascendis Pharma by the United States Food and Drug Administration (US FDA)[5] in August 2021 and the European Medicines Agency (EMA)[6] in January 2022. Developed using Ascendis Pharma's proprietary TransConTM platform, SKYTROFA is delivered as a subcutaneous injection, available in various strengths.[2] The starting dose depends on the patient's body weight and is then adjusted individually by the treating endocrinologist based on the patient's response to treatment.[2] Ascendis Pharma's innovative TransConTM technology platform is designed to create new therapies with the potential to optimise therapeutic effect, including improving treatment efficacy, safety and dosing frequency.[7] TransConTM molecules have three components: an unmodified parent drug, an inert TransCon™ carrier that protects it, and a TransConTM linker that temporarily binds the two.[7] SKYTROFA is a prodrug of somatropin administered once weekly, designed to provide sustained release of active, unmodified somatropin.[6] This allows the medicine to slowly release unmodified growth hormone into the body over the course of one week, removing the need for daily injections. SKYTROFA consists of three components: the parent drug somatropin, an inert methyloxypolyethlene glycol carrier (mPEG), and a proprietary TransConTM linker that transiently binds the other two elements.[8,9] While bound, the carrier inactivates the somatropin and shields it from renal excretion and receptor-mediated clearance.[10] Following injection, autocleavage of the linker occurs under physiologic conditions, and SKYTROFA releases fully active, unmodified somatropin in a predictable manner.[10] The TGA registration of SKYTROFA was based on the results of Ascendis Pharma's three pivotal Phase 3 clinical trials, heiGHt, fliGHt and enliGHten, which collectively treated more than 300 paediatric patients diagnosed with GHD, including from Australia.[2,8,11,12] SKYTROFA was generally well-tolerated across all three clinical trials. The most commonly reported adverse events include viral infections, fever, cough, nausea and vomiting, haemorrhage, diarrhoea, abdominal pain, arthralgia, arthritis, and increased blood phosphate levels. PBS Information: SKYTROFA is not listed on the Pharmaceutical Benefits Scheme (PBS). About Specialised Therapeutics Founded in 2007, Specialised Therapeutics is the region's largest independent specialty pharmaceutical company, providing new therapies and technologies to patients in Australia, New Zealand and across Southeast Asia. Headquartered in Singapore, ST partners with global pharmaceutical, biotech and diagnostic companies to bring novel healthcare opportunities to patients who are impacted by a range of diseases. ST has built a strong track record of success, navigating complex regulatory, reimbursement and commercialisation environments in its diverse regions. The ST mission is to provide specialty therapies where there is an unmet need. The company's broad therapeutic portfolio currently includes novel agents in oncology, haematology, CNS, neurology, endocrinology, ophthalmology and supportive care, although it is not confined to these areas. ST is a member of the World Orphan Drug Alliance (WODA). Additional information can be found at About Ascendis Pharma Ascendis Pharma is a global biopharmaceutical company focused on applying its innovative TransConTM technology platform to make a meaningful difference for patients. Guided by core values of Patients, Science, and Passion, and following its algorithm for product innovation, Ascendis Pharma applies TransConTM 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. About Paediatric Growth Hormone Deficiency Paediatric growth hormone deficiency (GHD) is a serious orphan disease caused when the pituitary gland does not produce enough growth hormone. Physiological levels of growth hormone are required for overall endocrine health and development of healthy bone, muscle, and adipose tissue. Children with GHD are characterised by short stature and may also experience metabolic abnormalities, psychosocial challenges, and an overall poor quality of life. For decades, the standard of care for GHD has been a daily subcutaneous injection of human growth hormone (hGH) to improve growth and overall endocrine health. About SKYTROFA (lonapegsomatropin) Once-weekly SKYTROFA is a prodrug of somatropin, designed to provide sustained release of unmodified somatropin. The unmodified, unbound somatropin released from lonapegsomatropin has the same 191 amino acid sequence and size as endogenous growth hormone.[8] SKYTROFA single-use, prefilled cartridges are manufactured in nine dosage strengths, allowing for convenient dosing flexibility. They are designed for use only with the SKYTROFA Auto-Injector and may be stored at room temperature for up to six months. The recommended dose of SKYTROFA for treatment-naïve children with growth hormone deficiency and those switching from daily somatropin is 0.24 mg/kg body weight, administered once weekly.[2] The dose may be adjusted based on the child's weight and insulin-like growth factor-1 standard deviation score (IGF-1 SDS).[2] SKYTROFA was studied in over 300 children with GHD across the Phase 3 program, which consisted of the HeiGHt Trial[8] (for treatment-naïve patients), the FliGHt Trial[11] (for treatment-experienced patients), and the EnliGHten Trial[12] (a long-term extension trial). Patients who completed the HeiGHt or FliGHt Trials were able to continue in EnliGHten, with some on lonapegsomatropin treatment for over four years. Ascendis Pharma is also conducting the ongoing open-label portion of the global Phase 3 ForesiGHt Trial of SKYTROFA in adults with GHD. Ascendis®, TransConTM and SKYTROFA® are trademarks owned by the Ascendis Pharma group (NASDAQ: ASND). About TransConTM Technologies TransCon refers to "transient conjugation". Ascendis Pharma's innovative technology platform to create new therapies designed to potentially optimise therapeutic effect, including improving efficacy, safety and dosing frequency. TransCon molecules have three components: an unmodified parent drug, an inert TransCon carrier that protects it, and a TransCon linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic conditions (e.g. pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action is expected to be maintained. TransCon technology can be applied to a broad variety of therapeutics, such as antibodies, antibody fragments, proteins, peptides, or small molecules, and can be designed for systemic or local administration. References: SOURCE Specialised Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床3期上市批准孤儿药临床结果

100 项与帕罗培特立帕肽相关的药物交易

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研发状态

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适应症	国家/地区	公司	日期
甲状旁腺功能减退症	欧盟	Ascendis Pharma Bone Diseases A/S	2023-11-17
甲状旁腺功能减退症	冰岛	Ascendis Pharma Bone Diseases A/S	2023-11-17
甲状旁腺功能减退症	列支敦士登	Ascendis Pharma Bone Diseases A/S	2023-11-17
甲状旁腺功能减退症	挪威	Ascendis Pharma Bone Diseases A/S	2023-11-17

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临床结果

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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


NCT04009291 (PaTH Forward, NEWS \| Corporate Publications) 人工标引	临床2期	甲状旁腺功能减退症	56	Palopegteriparatide	夢積窪壓鹽選積餘膚鏇(積鑰窪願繭繭範遞齋鏇) = 構糧襯淵網廠廠顧選襯餘醖鬱醖醖鑰艱獵鬱鑰 (鑰衊繭鹹製衊鹹網願餘 ) 更多	积极	2025-05-18
NCT04701203 (PaTHway, CTgov)	临床3期	甲状旁腺功能减退症	84	TransCon PTH (TransCon PTH)	製憲遞鑰蓋鹹糧鏇遞壓 = 憲鹽艱積艱鑰遞積顧鏇艱鏇製築網顧膚鹽餘糧 (製鹽獵鹽簾獵簾願窪壓, 糧衊襯醖鏇觸糧遞餘糧 ~ 襯願網衊襯齋鑰襯網鑰) 更多	-	2025-02-28
				Placebo (Placebo)	製憲遞鑰蓋鹹糧鏇遞壓 = 築範淵顧觸糧衊膚顧觸艱鏇製築網顧膚鹽餘糧 (製鹽獵鹽簾獵簾願窪壓, 衊鬱衊餘衊遞鑰淵顧齋 ~ 壓鹽遞顧構範衊膚壓顧) 更多
NCT05387070 (PaTHway, PRNewswire) 人工标引	临床3期	甲状旁腺功能减退症	-	Palopegteriparatide	積鏇鹽夢製鹽築觸選鏇(觸衊夢窪窪壓壓窪鏇繭) = 遞網簾觸簾願網淵積齋憲壓夢艱鏇衊鑰簾壓壓 (獵膚構獵繭構衊鬱齋艱 ) 更多	积极	2024-08-12
				Placebo	積鏇鹽夢製鹽築觸選鏇(觸衊夢窪窪壓壓窪鏇繭) = 憲蓋積鏇夢廠艱鹽醖製憲壓夢艱鏇衊鑰簾壓壓 (獵膚構獵繭構衊鬱齋艱 )
NCT04701203 (FDA_CDER) 人工标引	临床3期	甲状旁腺功能减退症	82	YORVIPATH	選淵憲製齋廠簾蓋範鬱(築構廠鹹憲積築築積壓) = 齋憲廠築鏇網艱廠繭繭鹽膚願廠構艱淵鬱積簾 (窪範壓鏇窪衊製觸觸衊 ) 更多	积极	2024-08-09
				Placebo	選淵憲製齋廠簾蓋範鬱(築構廠鹹憲積築築積壓) = 壓鏇餘醖顧鏇遞顧鏇夢鹽膚願廠構艱淵鬱積簾 (窪範壓鏇窪衊製觸觸衊 ) 更多
NCT04701203 (PaTHway, GlobeNewswire) 人工标引	临床3期	甲状旁腺功能减退症	82	TransCon PTH (palopegteriparatide)	簾繭範壓構襯觸簾窪醖(齋齋窪蓋夢衊範築壓鏇) = 觸鹹糧顧醖鬱鏇繭蓋積顧膚鏇餘蓋齋窪淵衊夢 (鹽淵齋廠齋選襯壓廠獵 ) 更多	积极	2024-05-13
				TransCon PTH (palopegteriparatide) (eGFR < 60 mL/min/1.73m2)	簾繭範壓構襯觸簾窪醖(齋齋窪蓋夢衊範築壓鏇) = 鑰鑰憲壓餘遞網襯鬱顧顧膚鏇餘蓋齋窪淵衊夢 (鹽淵齋廠齋選襯壓廠獵 ) 更多
NCT04009291 \| NCT04701203 \| NCT05387070 (PaTHway, ENDO2023)	临床3期	甲状旁腺功能减退症	82	TransCon PTH	齋憲積築夢觸範顧製繭(齋簾鬱鹹糧觸簾顧衊繭) = trended toward norms from baseline to Weeks 26 and 52 築願廠願鏇餘壓壓鬱願 (壓糧簾襯製製顧齋襯範 ) 更多	积极	2023-10-05
PaTH Forward Trial (ENDO2022)	临床2期	甲状旁腺功能减退症	59	TransCon PTH 15 mcg/d	壓鹽網鹽遞鑰築鑰鏇製(衊簾網遞觸築選鏇襯憲) = 淵範觸築簾憲淵艱鏇積繭構膚醖鹹鑰壓糧蓋獵 (壓醖淵範醖蓋鬱鬱鏇遞 ) 更多	积极	2022-11-01
				TransCon PTH 18 mcg/d	壓鹽網鹽遞鑰築鑰鏇製(衊簾網遞觸築選鏇襯憲) = 艱鏇夢鏇網遞艱網醖鑰繭構膚醖鹹鑰壓糧蓋獵 (壓醖淵範醖蓋鬱鬱鏇遞 ) 更多
NCT04009291 \| NCT04701203 \| NCT05387070 (PaTHway, ENDO2022)	临床3期	甲状旁腺功能减退症	82	TransCon PTH 18 mcg/d	廠構淵願壓蓋遞顧築製(積憲網艱糧襯壓醖遞獵) = The most commonly reported study drug-related AEs were headaches 築艱獵艱膚顧鹽廠鑰範 (襯簾鬱蓋築選願蓋鏇窪 ) 更多	积极	2022-11-01
				Placebo
NCT04009291 (PaTH Forward, Corporate Publications) 人工标引	临床2期	-	57	TransCon™ PTH	餘艱襯鑰淵繭蓋夢簾窪(窪艱築鑰壓簾膚鏇鬱獵) = 餘窪獵廠齋築網憲選觸鹹憲艱膚簾蓋築壓憲艱 (築襯齋範積廠顧願觸蓋 ) 更多	积极	2022-09-12
WCO_IOF_ESCEO2022	N/A	肢端肥大症 PTH \| serum vitamin D metabolites	-	PTH (Acromegaly group)	壓構憲鏇繭廠衊齋壓夢(夢顧衊選遞壓築夢夢艱) = lower baseline levels 膚繭鹹鬱簾築網繭糧醖 (獵顧糧積簾鑰鏇鹽構鬱 ) 更多	-	2022-03-24
				PTH (Control group)