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

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

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

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 ; Högler, Wolfgang ; Shu, Aimee D ; 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）

2025-05-13

·药时空

疗效持续4年！维昇药业特立帕肽前药甲状腺功能减退2期临床长期随访结果

5月12日（当地时间），Ascendis Pharma（中国子公司：维昇药业）宣布Yorvipath（Palopegteriparatide，中文名：帕罗培特立帕肽，研发名称：TransCon PTH）成人甲状腺功能减退2期临床研究（PaTH Forward）214周最新研究结果证实其长期持久的疗效。PaTH Forward研究（NCT04009291)为在4周随机、双盲、安慰剂对照研究后进入为期266周的开放标签扩展研究。214周时，59例患者中的56例（95%)仍在接受治疗。214周研究结果如下：98%患者白蛋白校正血清钙水平继续维持正常范围内,93%患者摆脱现有疗法（定义：钙＜600mg/天&无需Vd）；骨转换标志物CTx（胶原C端肽）和P1NP（总I型胶原氨基端延长肽）从基线正常低端增加，26周到达峰值后下降并在214周维持稳定在基线水平之上；骨骼动力学改善：BMD维持在年龄和性别匹配正常范围内；67.8%患者eGFR（肾小球滤过率）具有临床意义增加（≥5ml/min/1.73m^2),eGFR从第4周明显变化。安全性方面，耐受性良好，无新的安全性信号，TEAE多为轻或中度，无重度或药物相关停药。Yorvipath（Palopegteriparatide，中文名：帕罗培特立帕肽，研发名称：TransCon PTH）为特立帕肽（PTH 1-34）前药，每天一次即可在24h内持续释放PTH。2018年，Ascendis授予Visen（维昇药业）大中华区开发和商业化TransCon hGH、TransCon PTH和TransCon CNP的独家权利。Yorvipath在2023年6月获得欧洲药品管理局批准上市，作为成人慢性甲状腺功能减退患者的替代性疗法。商品名与FDA批准的相同，规格有三种：168μg/0.56ml、294μg/0.98ml、420μg/1.4ml，剂型均为预充笔注射液。FDA的审批过程颇为曲折，2022年8月ascendis Pharma递交NDA申请获FDA受理并获得优先审评，2023年5月FDA下发完整回复函（CRL）提出药械组合的剂量传输控制策略问题，同年12月ascendis Pharma重新递交NDA申请并获FDA受理，而在2024年5月FDA又因审查过程中重大变更导致审评时限延长3月至2024年8月14日。2024年8月，FDA最终批准上市，用于甲状腺功能减退患者的替代性疗法。维昇药业2024年8月公布帕罗培特立帕肽用于治疗成人慢性甲状旁腺功能减退症的中国3期临床试验（PaTHway China），26周随机、双盲、安慰剂对照部分的数据：与安慰剂组相比，帕罗培特立帕肽组达到了主要复合终点的患者比例显著更高。识别微信二维码，可添加药时空小编请注明：姓名+研究方向！

优先审批上市批准临床2期突破性疗法临床3期

2025-05-05

·GlobeNewswire-Health Care

Ascendis to Share Its Latest Endocrinology Rare Disease Data at ESPE & ESE 2025

COPENHAGEN, Denmark, May 05, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it will share the latest data from its hypoparathyroidism, achondroplasia, and growth hormone deficiency (GHD) programs during ESPE & ESE 2025, the joint congress of the European Society for Paediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE) being held May 10-13, 2025, in Copenhagen. Four oral presentations will feature Ascendis clinical trial results, including 4-year efficacy and safety data from the Phase 2 PaTH Forward Trial of TransCon PTH (palopegteriparatide) in adults with chronic hypoparathyroidism; Week 52 growth and bone morphometry data from the pivotal ApproaCH Trial of TransCon CNP (navepegritide) in children with achondroplasia; and efficacy and safety data from the Phase 3 foresiGHt Trial of TransCon hGH (lonapegsomatropin) in adults with growth hormone deficiency. “As new treatments and clinical practices transform the outlook for rare endocrine diseases, we are pleased to partner with trial investigators and leading experts to share new data highlighting clinical benefits of our novel therapies with attendees of ESPE & ESE 2025,” said Aimee Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Science and Chief Medical Officer at Ascendis Pharma. The full range of Ascendis Pharma events and updates at ESPE & ESE 2025 include the following: Hypoparathyroidism Sunday, May 1109:55 - 10:25 CEST Congress TheaterFoyer F5Product TheaterRestoring Physiological Levels of PTH in Chronic HypoparathyroidismClick here to see the full session outline.Sunday, May 1116:55-17-55 CEST& Monday, May 1217:15-17:45 CESTPoster HallPoster P238Estimating the Risk of Chronic Kidney Disease Progression in Chronic Hypoparathyroidism: A Restrospective Matched Cohort Study, Using Real-World Data from England Monday, May 1213:00 - 14:30 CESTRoom D5SymposiumBeyond Conventional Care: Redefining Treatment Success in Chronic HypoparathyroidismClick here to see the full session outline.Monday, May 1215:55-16:05 CESTRoom C2Oral PresentationLong-Term Efficacy & Safety of Palopegteriparatide Treatment in Adults with Chronic Hypoparathyroidism: 4-Year Results from the Phase 2 PaTH Forward TrialPresented by Dr. Andrea PalermoAchondroplasiaSunday, May 1115:55-17:25 CESTRoom C2SymposiumAssessing HRQoL in Achondroplasia Across the Life CourseClick here to see the full session outline.Monday, May 1215:15-15:25 CESTRoom C2Oral PresentationEffects of Navepegritide on Bone Morphometry in Children with Achondroplasia: 52-Week Results from the ApproaCH Clinical TrialPresented by Dr. Leanne WardTuesday, May 1315:05-15:15 CESTRoom D2Oral PresentationEffects of Navepegritide on Growth in Children with Achondroplasia: 52-Week Results from the ApproaCH Clinical TrialPresented by Dr. Hanne HoveGrowth Hormone DeficiencyTuesday, May 1314:45-14:55 CESTRoom D2Oral PresentationResults of the foresiGH Trial Support the Efficacy and Safety of Once-Weekly Lonapegsomatropin in Adults with Growth Hormone Deficiency (GHD)Presented by Dr. Aleksandra Gilis-Januszewska More information about the events and presentations listed here is available to registered attendees of the Joint ESPE & ESE 2025 congress. About HypoparathyroidismHypoparathyroidism is an endocrine disease caused by insufficient levels of parathyroid hormone (PTH), the primary regulator of calcium and phosphate balance in the body, acting directly on bone and kidney and indirectly on the intestine. Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extra-skeletal calcifications, and cognitive impairment. Post-surgical hypoparathyroidism accounts for the majority of cases (70-80%), while other etiologies include autoimmune and idiopathic causes. About AchondroplasiaAchondroplasia 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 Adult Growth Hormone DeficiencyGrowth hormone plays an essential role in the health of children and adults, promoting normal growth in children and maintenance of normal body composition and cardiometabolic health throughout adulthood. In adults, growth hormone boosts protein production, promotes fat utilization, enhances muscle mass, and helps regulate blood sugar levels. Adult GHD is a condition in which an individual’s body does not produce enough growth hormone. Symptoms and morbidity can include central obesity, metabolic syndrome, decreased bone density, alterations in lipid profile and markers of cardiovascular risk, fatigue, general weakness, lack of muscle tone, and psychological symptoms such as cognitive impairment, social isolation, lack of motivation, and depression. About Ascendis Pharma A/SAscendis 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) the clinical benefits of Ascendis’ novel therapies, (ii) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients, and (iii) 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, and TransCon are trademarks owned by the Ascendis Pharma group. © May 2025 Ascendis Pharma A/S. Investor Contacts: Scott Smith Ascendis Pharma ir@ascendispharma.com Media Contact:Melinda BakerAscendis Pharmamedia@ascendispharma.com Patti BankICR Healthcare+1 (415) 513-1284patti.bank@icrhealthcare.com

临床2期临床结果

2025-04-18

·药时空

2025年有望首次获批的6款创新多肽药物

自1922年胰岛素用于治疗糖尿病，肽类药物正式登上医学舞台。发展至今，全球已有近100种肽类药物获批。据弗若斯特沙利文数据预测，到2030年，全球多肽药物市场规模将达到2108亿美元，其中中国市场规模占全球比重维持在15%左右。多肽药物市场中，糖尿病、肿瘤和罕见病是拉动多肽药物市场扩容的“三驾马车”。2025年，以下6款创新多肽药物有望首次获批。1、ElamipretideElamipretide（依拉米肽）是一种肽化合物，可轻易穿透细胞膜，靶向线粒体内膜，并可逆性地与心磷脂（cardiolipin）结合。在临床前或临床研究中观察到该疗法可增加线粒体呼吸作用、改善电子递送链功能和ATP生成，并减少致病性活性氧（ROS）的形成。这种elamipretide-心磷脂结合已被证明可以使线粒体内膜结构正常化，从而改善线粒体功能。Elamipretide已获得美国FDA授予孤儿药资格、快速通道资格和罕见儿科疾病认定，以及欧洲药品管理局（EMA）的孤儿药资格，用于治疗巴思综合征——一种由tafazzin基因突变引起的疾病，该突变导致心磷脂水平降低，心磷脂是一种在线粒体功能中起主要作用的磷脂。这种疾病的特征是心脏异常、肌肉无力、反复感染和生长迟缓。1月23日，Stealth BioTherapeutics宣布，美国食品药品监督管理局（FDA）延长了用于治疗巴思综合征的Elamipretide新药申请（NDA）的审查期。NDA提交的包括来自2/3期TAZPOWER研究和3期SPIBA-001自然史对照研究的数据。FDA需要更多时间审查补充信息，其认为这一信息是对申请的重大修改。目前，新的处方药使用者费用法案的目标日期（PDUFA日期）是2025年4月29日。据悉，若是该药成功获批上市，将成为首个FDA批准的巴思综合征治疗药物。2、IcosemaIcoSema是诺和诺德研发的复方制品，由每周一次依柯胰岛素和每周一次司美格鲁肽（semaglutide）的固定比例组合。其中，依柯胰岛素是人胰岛素类似物，其氨基酸结构有三处进行了替换，并附加了C20二十烷脂肪二酸链，使该分子能够可逆地与白蛋白结合，从而将半衰期延长至196小时（约7天），并在经过3-4次每周一次注射后达到稳定状态。该疗法被设计为一周一次皮下注射使用。依柯胰岛素已在欧盟、加拿大、澳大利亚和日本以Awiqli为商品名获得批准。司美格鲁肽则是一种胰高糖素样肽-1（GLP-1）类似物，它通过突破性的肽链结构优化，使得分子半衰期延长至7天，实现了一周一次给药且血药浓度非常平稳。目前，诺和诺德已基于IcoSema针对2型糖尿病的COMBINE系列III期研究在欧盟提交其上市申请。其中COMBINE 1研究和COMBINE 3研究的结果已公开。随后，该药又于2024年12月在华申报上市并获受理。据悉，在COMBINE 3研究中，IcoSema取得了非劣效于甘精胰岛素U100和门冬胰岛素的结果。在总体基线HbA1c为8.30%的情况下，IcoSema组HbA1c降幅为-1.47%，而甘精胰岛素U100和门冬胰岛素组HbA1c降幅为-1.40（估计治疗差异：-0.06%）。此外，在基线体重为85.8kg的基础上，接受IcoSema治疗的患者体重显著减轻，IcoSema组体重减轻了3.6kg，而甘精胰岛素U100和门冬胰岛素组体重增加了3.2kg（估计治疗差异：-6.7kg）。3、NavepegritideNavepegritide（TransCon CNP，那韦培肽）属于一款长效的C 型利钠肽前药，被用于软骨发育不全的治疗。软骨发育不全属于人类最为常见的矮小症，是由编码成纤维细胞生长因子受体3(FGFR3)基因当中的一种常见致病突变所引发，这种突变会给骨骼生长的软骨内骨化过程带来影响。针对软骨发育不全发病机制的治疗手段始终是一项重大却尚未被满足的需求。据Ascendis Pharma在JPM 2025大会上透露，Navepegritide用于治疗软骨发育不全的疗法，计划在2025年第一季度向FDA提交新药申请（NDA），并在2025年第三季度向欧洲药品管理局（EMA）提交市场授权申请。在关键的ApproaCH试验中，Navepegritide显著改善了腿部弯曲，这是一种常见的并发症，而安慰剂组则观察到恶化。计划在2025年第四季度提交治疗低磷酸酯酶血症的临床试验申请（IND）。目前，此药已同时被FDA和EMA认定为孤儿药。4、ApraglutideApraglutide（阿帕拉格鲁肽）是Ironwood Pharmaceuticals公司通过收购VectivBio公司获得的一种新一代、人工合成的长效GLP-2类似物，拟开发用于一系列GLP-2在疾病病理生理学中可以发挥中心作用的罕见胃肠疾病。目前，该药治疗短肠综合征（SBS）的临床试验也已进入3期阶段；治疗胃肠道急性移植物抗宿主病的临床试验已进入2期阶段。Ironwood正在III 期STARS试验中测试Apraglutide，该试验于2024年2月显示，SBS患者对肠外支持的需求减少了25%，而安慰剂患者则减少了12%。Apraglutide也达到了其次要终点，包括能够至少停止肠外支持一天的患者比例增加。2025年1月，Ironwood宣布了一项大幅裁员计划，以投入更多资源推动其先导分子Apraglutide通过III期试验和滚动NDA提交。Ironwood还展示了来自STARS开放标签扩展研究的额外数据，显示更多患者因长期暴露Apraglutide而退出肠外支持。据悉，Ironwood已启动Apraglutide的滚动NDA提交，预计将在今年第三季度完成申报。5、Galinpepimut-SGalinpepimut-S（GPS）是一种多肽候选药物，已被改良以增强针对WT1蛋白的免疫反应的程度和持续时间。WT1蛋白是在多种癌症类型中过度表达的蛋白。在构成GPS的多肽混合物中，四种多肽中的两种多肽在单个氨基酸残基上经过刻意突变。这些突变肽被免疫系统识别为非自身产物，因此不易引发免疫耐受。这些突变肽通过人工智能（AI）设计，用于诱导针对癌细胞中突变肽和自然存在肽的强力T细胞反应，达到杀伤过度表达WT1蛋白的肿瘤细胞的效果。2025年1月，SELLAS Life Sciences Group（SELLAS）公司宣布，独立数据监测委员会（IDMC）已完成关键性3期临床试验REGAL的预定中期分析。该试验评估了在研多肽免疫疗法Galinpepimut-S（GPS）治疗急性髓系白血病（AML）的疗效和安全性。分析结果显示，试验参与者的中位总生存期超过12个月，而类似患者群体的预期生存期约为6个月。IDMC对中期数据的审查支持试验按照原始方案继续进行。REGAL是一项开放标签注册性3期临床试验，招募通过二线挽救治疗达到完全缓解（CR2）的AML患者。该试验的主要终点为总生存期。目前试验尚未揭盲，然而部分盲态数据已显示，在入组完成约10个月后，确认死亡的患者少于一半，且中位随访时间约为13.5个月（范围为1个月至超过3年）。这一结果表明中位生存期超过12个月，而类似患者群体的预期生存期约为6个月。此外，对接受GPS治疗的随机选取患者样本的早期免疫反应进行的盲态分析显示，80%的患者产生了特异性免疫反应。这些数据与GPS先前的试验结果一致。在2期临床试验中，接受GPS治疗的患者的中位总生存期为21个月，而接受标准治疗的患者仅为5.4个月，GPS组患者的特异性免疫反应率为64%。6、GlepaglutideGlepaglutide为一款潜在治疗SBS的长效GLP-2类似物液体制剂，并将配备可皮下注射的自动给药装置，旨在降低或消除SBS患者的肠外营养支持，FDA曾授予孤儿药资格。2024年12月，Zealand Pharma A/S宣布收到了FDA对Glepaglutide治疗SBS的新药上市申请（NDA）发出完整回复函（CRL），FDA的CRL中要求增加临床试验以提供Glepaglutide的拟上市剂量规格更多的有效性和安全性证据。Zealand Pharma A/S在表达失望之余，同时表示将与FDA进一步沟通，并在2025年启动一项III期临床研究以支持美国和欧盟的上市需要。根据Zealand Pharma的说法，向FDA提交申请是基于一项名为EASE-1（NCT03690206）的安慰剂对照的3期注册试验结果。该试验确实达到了其主要终点，即在24周时，与基线相比，每周两次的格列帕肽剂量使每周肠外支持量在统计学上显著减少了5.13升。然而，每周一次的glepaglutide却并没有达到统计学意义上的显著差异。（安慰剂2.85L vs 每周一次3.13L ）另外一个问题则出在第二个主要终点——临床响应率上，在Glepaglutide组中，有66%的患者出现具有临床意义的改善，对照组的临床响应率是38.9%。参考资料：[1] 2024年多肽药物行业进展. 知识药点. 2025-02-05.[2] 肽类药物：现状、进展与未来展望. 生物制品圈. 2025-03-07.[3] 多肽专栏 | 多肽药物研发和市场格局分析与展望. 中生药协. 2025-03-13.[4] 2025年4月，3款罕见病药有望在美国获批. 罕见病信息网. 2025-04-03.[5] FDA延长对Elamipretide治疗Barth综合征的审查. 香港济民药业. 2025-01-24.[6] 1月3款创新药有望获FDA批准. 药明康德. 2025-01-02.[7] Tides 速递 | 诺和诺德「依柯胰岛素司美格鲁肽复方」在华申报上市. 多肽圈. 2024-12-09.[8] 罕见病ACH“救命药”关键实验达主要终点，每年长高6厘米！. CPHI制药在线. 2024-10-24.[9] JPM 2025 Ascendis. 药政时势. 2025-01-15.[10] 同源不同命！作为GLP-1的“姐妹”，它却被开发治疗这些疾病. 医药观澜. 2024-07-05.[11] Ironwood裁员50%. 药物探索. 2025年01月31日.[12] 癌症患者总生存期延长一倍！多肽免疫疗法关键性3期临床试验中期结果积极. 药明康德. 2025年01月24日.[13] 临床证据不足！短肠综合征长效GLP-2类似物上市被拒. 药讯随说. 2024年12月20日.[14] FDA拒批长效GLP-2. 佰傲谷BioValley. 2024年12月20日.识别微信二维码，可添加药时空小编请注明：姓名+研究方向！

孤儿药上市批准快速通道生物类似药

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	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)	廠憲襯艱糧蓋齋鹹廠構(艱襯醖選齋鹹醖憲壓鏇) = 獵衊鬱範艱糧餘糧繭廠蓋艱壓窪鏇艱膚鹹觸壓 (遞齋鏇鬱鏇製遞製夢窪 ) 更多	积极	2024-09-16
				Placebo (ages 2-11 years)	廠憲襯艱糧蓋齋鹹廠構(艱襯醖選齋鹹醖憲壓鏇) = 蓋範衊蓋鏇鹽願鹹齋觸蓋艱壓窪鏇艱膚鹹觸壓 (遞齋鏇鬱鏇製遞製夢窪 ) 更多
NCT04085523 (ACcomplisH, Company_Website) 人工标引	临床2期	软骨发育不全	57	TransCon CNP	夢範製鹹鹽窪廠顧淵糧(窪鑰網鏇憲鏇廠廠遞齋) = patients treated for one year at the pivotal 100μg/kg/week dose demonstrated significant improvements in well-being and physical functioning compared to placebo. 願衊築顧廠構觸憲醖簾 (築廠膚觸衊鹹範鹹繭廠 ) 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)	壓膚淵艱觸獵鏇網齋醖(壓淵製繭選選製憲齋築) = 蓋壓鹽鏇構鑰廠鑰繭鏇艱選壓觸願築壓糧構鬱 (壓顧鹹積築簾憲鏇糧遞, 繭襯衊窪夢簾衊範範鏇 ~ 鹽淵製壓鹹選醖鏇選繭) 更多	-	2024-03-07
				TransCon CNP (TransCon CNP 20 mcg)	壓膚淵艱觸獵鏇網齋醖(壓淵製繭選選製憲齋築) = 憲願衊膚壓膚製壓鏇鬱艱選壓觸願築壓糧構鬱 (壓顧鹹積築簾憲鏇糧遞, 糧鹽鏇網淵窪膚選鹹鬱 ~ 憲鏇壓繭遞顧衊鹹顧繭) 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	築衊夢積願築鹹顧鑰鹽(選襯艱築願選醖製鏇顧): 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	選襯壓獵築選廠艱鏇獵(積構淵觸鹹淵鬱廠獵廠) = 鏇範醖淵簾範淵衊顧窪糧襯鹹壓鹹遞構簾夢鹽 (構獵鏇顧衊齋窪淵夢範 ) 达到	积极	2023-11-16
				TransCon CNP 100μg/kg/week	選襯壓獵築選廠艱鏇獵(積構淵觸鹹淵鬱廠獵廠) = 構廠窪獵壓鏇餘築遞壓糧襯鹹壓鹹遞構簾夢鹽 (構獵鏇顧衊齋窪淵夢範 ) 达到
NCT04085523 (ACcomplisH, Pubmed)	临床2期	软骨发育不全	42	TransCon CNP 100 μg CNP/kg/week	淵壓襯壓襯壓夢廠鏇築(淵夢廠遞鏇簾糧構蓋壓) = 壓衊壓醖廠鏇醖選憲壓鏇構製範獵糧顧蓋顧網 (顧蓋糧糧簾艱襯網夢遞, 4.74 ~ 6.11)	积极	2023-11-01
				Placebo	淵壓襯壓襯壓夢廠鏇築(淵夢廠遞鏇簾糧構蓋壓) = 膚獵鹹製構積築淵鹹鑰鏇構製範獵糧顧蓋顧網 (顧蓋糧糧簾艱襯網夢遞, 3.75 ~ 4.94)
NCT05598320 (ACcomplisH, ENDO2023)	临床2期	软骨发育不全	57	TransCon CNP 6 μg/kg/week	廠繭築願艱夢醖鏇鹹製(醖壓蓋範繭選艱糧顧膚) = 鑰鏇鹽蓋鹽鬱鏇醖築築積積齋選構餘網艱積積 (艱網壓構餘鹹窪範遞簾 )	积极	2023-10-05
				TransCon CNP 20 μg/kg/week	廠繭築願艱夢醖鏇鹹製(醖壓蓋範繭選艱糧顧膚) = 網窪膚艱衊鬱鏇膚簾簾積積齋選構餘網艱積積 (艱網壓構餘鹹窪範遞簾 )