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: Taylor-Miller, Tashunka ; Savarirayan, Ravi

INTRODUCTIONAchondroplasia 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 COVEREDThis 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 OPINIONThe 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.

2024-05-05·Cureus

Two Cases of Cardiovascular Adverse Events Following Subcutaneous Vosoritide Injection in Early Infancy

Article

作者: Tanaka, Hiroyuki ; Oda, Yoichiro ; Adachi, Natsuho ; Nishioka, Atsushi

Achondroplasia, characterized by short stature and skeletal abnormalities, is caused by a gain-of-function variant in the fibroblast growth factor receptor 3 gene. Vosoritide, a C-type natriuretic peptide analog, is an emerging treatment for achondroplasia that functions by promoting endochondral ossification. Vosoritide was approved for the treatment of achondroplasia in Europe and the United States in 2021, and in Japan, the following year. However, vosoritide is associated with a risk of hypotension and vomiting after subcutaneous injection due to its vasodilating effect. Herein, we present two cases of cardiovascular adverse events in infants following vosoritide injection. Case 1 involved a one-month-old female infant with achondroplasia who received the first subcutaneous injection of vosoritide 30 minutes after her last formula intake. Following injection, she developed transient symptomatic hypotension accompanied by vomiting. Although established guidelines recommend that injections be administered after approximately 30 minutes (Europe/Japan) or within one hour (USA) following the last feeding, an extended interval of 1.5 to two hours was required to prevent hypotension-associated vomiting. Case 2 involved a three-month-old female infant with achondroplasia. The first subcutaneous vosoritide injection was administered four hours after the last formula intake, and she subsequently developed prolonged compensated shock with marked tachycardia requiring intervention, including repetitive bolus saline injection. These cases indicate the need to monitor patients for cardiovascular adverse events following subcutaneous injection of vosoritide in early infancy.

2024-05-01·eClinicalMedicine

Vosoritide treatment for children with hypochondroplasia: a phase 2 trial

Article

作者: Castro, Meryll Grace ; Shafaei, Niusha ; Dham, Niti ; Kanakatti Shankar, Roopa ; Boucher, Kimberly ; Merchant, Nadia ; McCarthy, Tara ; Zhang, Anqing ; Seaforth, Raheem ; Dauber, Andrew

BackgroundHypochondroplasia is a rare autosomal dominant skeletal dysplasia due to activating variants in FGFR3. It presents with disproportionate short stature with a wide range of clinical severity. There are currently no approved medications to treat short stature in children with hypochondroplasia. Vosoritide is a C-type natriuretic peptide analog that was recently approved for improving growth in children with achondroplasia. We aimed to evaluate the safety and efficacy of vosoritide in children with hypochondroplasia.MethodsWe conducted a single-arm, phase 2, open-label trial at a single centre in the USA and enrolled 26 children with hypochondroplasia. The trial consists of a 6-month observation period to establish a baseline annualized growth velocity followed by a 12-month intervention period during which vosoritide is administered daily via subcutaneous injection at a dose of 15 μg/kg/day. The trial's co-primary endpoints included the incidence of adverse events and the change from baseline in age-sex standardized annualized growth velocity and height standardized deviation score (SDS) after 12 months of treatment. This trial is registered with ClinicalTrials.gov (NCT04219007).FindingsTwenty-four participants with a mean age of 5.86 years received vosoritide therapy. The first participant was enrolled on August 4, 2020, and the final participant completed the 18-month trial on September 8, 2023. Vosoritide was well tolerated with no treatment-related serious adverse events. Injection site reactions occurred in 83.3% of participants. No participants discontinued therapy due to an adverse event. Annualized growth velocity increased by 2.26 standard deviations (SD) and height SDS increased by 0.36 SD during the treatment period versus the observation period. Hypochondroplasia specific height SDS increased by 0.38 SD. There was a 1.81 cm/year increase in absolute annualized growth velocity.InterpretationVosoritide was safe and effective in increasing growth velocity in children with hypochondroplasia. Efficacy was similar to what has been reported in children with achondroplasia.FundingThis study was supported by an investigator-initiated grant from BioMarin Pharmaceutical.

项与 Navepegritide 相关的新闻（医药）

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

2024-12-09

·动脉网

反向NewCo, 维昇药业长效平台抢滩内分泌市场蓝海

2024年以来，NewCo交易成为国内Biotech资本市场最为重要的活动之一。究其本质，NewCo是一种更复杂的创新药资产交易模式，融合了资产交易和权益交易，即BD+融资。这种兼具BD和融资属性的交易，让手握早期创新药资产的Biotech跃跃欲试。 NewCo的雏形可追溯至2014年。前华尔街对冲基金经理、2024年美国总统候选人的Vivek Ramaswamy创立Roivant，定位于“拯救制药业被遗忘的药物”。Roivant的经营逻辑，正是从MNC中筛选出那些有潜力却不被重视的管线，成立新公司、推进研发，最终推动新公司被大药厂收购而赚取回报。值得注意的是，不同于常规的资产授权，近期国内生物医药企的NewCo交易具有很强的海外属性，主要运营环节聚焦在海外市场。以恒瑞为例，尽管以授权的中国创新药管线作为核心资产，但是其海外子公司Hercules是一家纯粹的美国公司；反之，海外潜力管线落地中国，往往也会配备国内管理团队，并在本土市场完成融资或者上市。近日，作为反向NewCo，港股唯一专注内分泌领域且研发长效生长激素的biotech维昇药业（VISEN Pharmaceuticals）更新了招股书，即将IPO。维昇药业是一家处于研发后期、产品接近商业化的生物制药公司，专注内分泌领域，致力于提供同类首创（First in Class）或同类最优（Best in Class）的内分泌疾病产品和治疗方案。 2018年，丹麦药企Ascendis Pharma A/S（NASDAQ：ASND，下称Ascendis）和维梧资本、Sofinnova Ventures共同在上海创立维昇药业，前者以产品入股，维梧资本和Sofinnova Ventures出资支持。根据维昇药业与Ascendis的达成独家许可协议，公司能够在相关地区开发、生产和商业化隆培促生长素（lonapegsomatropin）、那韦培肽（navepegritide）及帕罗培特立帕肽（palopegteriparatide）等内分泌领域的候选药物。 01 确定性，基于资本、授权方和管理团队产品价值的信心随着NewCo逐渐走入国内大众视野并被广泛熟知，其在市场中的确定性也日益获得认可。通常组建和投资NewCo的基金都是熟知目标市场的老牌玩家。投资者在组建NewCo时早已看好资产在目标市场的前景，基本确定IPO的募资金额，甚至能明确未来的退出路径。上市之后，当临床数据披露、产品成功商业化，股价很可能会成倍飙升；此外，凭借经验丰富的管理运营团队，产品上市的可能性和成功率均得到有效保障，进而在资本市场持续获得认可。作为采用反向NewCo的维昇药业，接棒Ascendis已经完成的临床前期研究（包括动物实验和1期临床试验的结果），快速推进国内2期和3期临床试验，从而降低研发风险，加速产品上市进程。在这个过程中，维昇药业完成B轮融资1.5亿美金，用较少的融资资金在短短6年内，专注于完成授权国内的3条管线研发工作，并取得优异的临床结果。 NewCo模式及其投资方式现在已被认可与熟知，而早在2018年通过反向NewCo模式成立的维昇药业，在与Ascendis的合作中是通过给予Ascendis一定数量的股票来换取产品的引进权。这种合作模式使得维昇药业能够最大限度地保留自己的利润和资金，有益于公司的运营和未来发展。这一切来源于资本、授权方和管理团队对产品及其市场潜力的信心。创新药商业化的成功与否很大程度上在选品环节就已注定。理想的品类应满足市场潜力大、定价话语权强势、产品力（临床和商业竞争力）突出等特点。同时具备以上特点的产品可遇不可求，但要使得商业化成功推进，选品不应有明显短板。维昇药业的3款产品搭载TransCon技术平台，在不同层面上解决了内分泌产品依从性及成药难的技术问题，有着极高的技术壁垒。通过临床试验数据证明，这3款管线所属的差异化适应症赛道，正有大量未被满足的临床需求。图片源自维昇药业 Ascendis于近日宣布已授予诺和诺德TransCon技术平台的全球独家许可，用于开发、制造和商业化诺和诺德在代谢性疾病（包括肥胖和2型糖尿病）方面的专有产品——每月一次的GLP-1受体激动剂，并授予其在心血管疾病方面的产品独家许可。根据协议，Ascendis将有资格获得高达2.85亿美元的预付款以及开发和监管里程碑付款（超20亿人民币）。这一授权的达成也肯定了在长效化技术的多样性比较方面，TransCon技术脱颖而出，成为全球长效化技术平台中的佼佼者，而维昇早已将搭载TransCon技术的3款创新药收入囊中。 02 诺和诺德超20亿押注的TransCon长效平台维昇药业所处的内分泌治疗领域涵盖多达170余种疾病，可在新生儿直至老年人的全年龄段人群中发生。但对于整个内分泌疾病领域而言，目前至少有近一半的疾病仍然缺乏有效的治疗方法，即无药可医或现有疗法不足（疗效有限、副作用大）等，仍然存在强烈且广泛的未被满足的临床需求。相较于海外市场，中国内分泌疾病领域的创新药物仍有较大进步空间，引进经过验证、具有临床效益的内分泌创新药，有利于进一步促进中国内分泌药物市场的发展。据弗若斯特沙利文数据，中国的非糖尿病类内分泌药物市场规模预计到2026年将增至68亿美元，自2022年起的年复合增长率为23.7%；并且到2030年将增至115亿美元，自2026年起的年复合增长率为13.9%。与此同时，国家实施科创战略，推行一系列利好政策，鼓励药物创新；深化医改，加快创新药的审评审批，为创新药发展创造了空前良好的发展环境。产品方面，以维昇药业核心管线之一的长效生长激素为例，传统的短效生长激素需要患者每天注射一次，一年需注射365次，给患者带来了极大的不便，长期使用的依从性也因此很难保证，进而影响的是治疗效果难达预期。这类痛点落到儿童患者群体身上，则进一步被放大。因此，将短效生长激素转化为长效产品一直是医生、患者以及制药企业努力的方向。然而，由于长效化的技术壁垒高，过往的成功案例寥寥无几。这其中的关键技术壁垒在于，一旦涉及蛋白肽类药物，长效化途径则变得极为有限，由于肽类激素在体内复杂的组织分布、受体结合以及代谢清除的作用机理，难以在不牺牲疗效和安全性的前提下实现长效化。而TransCon（Transient Conjugation，暂时连接）平台正是瞄准了这一壁垒进行攻克。TransCon分子包括3个部分：未经修饰的原型药物，保护原型药物的惰性载体分子和将两者暂时连接起来的连接体。三部分结合后形成前药，其载体分子可以使得原型药物处于无活性状态并且不被机体清除。当前药注射至人体内，在生理条件的PH和体温下，有活性的、未经修饰的原型药物将以可控的方式释放出来，从而实现了从短效到长效的突破，减轻疾病治疗的负担。由于原型药物是未经修饰的，因此可以维持其原有的组织分布和生理作用。 TransCon平台通过独特的连接结构将载体分子与有生物学活性的原型药物暂时连接。根据所用载体的不同，TransCon前药可设计为全身（身体各处）或局部（如肿瘤内部）作用，以达到特定的治疗目标。基于其创新的长效化技术原理，TransCon平台可广泛适用于多种治疗领域的蛋白质、多肽或小分子药物研发。图片源自维昇药业值得一提的是，TransCon平台不仅为生长激素的长效化提供了创新的解决方案，还为其他激素类药物的成药提供了可能。例如，一些在体内代谢迅速的激素，如甲状旁腺激素（PTH），过去因其注射后不能维持体内平稳的激素水平而无法成药，不能用于治疗与之相关的激素缺乏性疾病（甲状旁腺功能减退症）。但现在，通过TransCon长效技术平台，这类激素类药物的成药问题得到了解决。 03 同步推进临床+产业链，加速商业化落地维昇药业CEO卢安邦于2018年以首位员工的身份加入了公司，至今已超过六年。在加入维昇之前，他在多家跨国药企履职：他在法国施维雅工作了16年，期间曾在法国总部负责多个产品的全球行销，并在2006年被派回中国担任中国区总经理；而后加入武田药业，先后担任中国区与大中华区总裁，领导团队长达7年，主导武田在中国市场的关键成长期，销售收入增长了超过10倍。而在维昇药业，卢安邦认为初创企业需要看外在环境，包括所选赛道的临床需求、产品价值以及经济环境等；同时，作为创业者也必须重新审视自己的能力和所处的赛道，包括个人的内在能力、团队可转移与不可转移的能力和所持管线产品的产品力。选品确定后，创新药商业化后续能否成功，靠的是“努力”。而商业化的“努力”，并不是简单的“大力出奇迹”，而是需要一系列正确的商业决策和强大的执行力。这种商业推广上的努力需要得当，包括建立合理的短期和中长期的销售预期和业务目标，根据产品制定适合的推广策略和方式，以及合理分配推广资源等。通过Ascendis授权的技术平台与三款候选药物，维昇药业在专注于治疗内分泌相关疾病的过程中，用了不到6年时间便让产品在中国达到接近商业化的阶段。图片源自维昇药业一方面，境外NewCo的创新药落地本土，依然要按部就班遵循临床、注册、商业化等流程，遵从监管要求、确保产品尽快上市，多线并行地推进临床成为必然的经历。值得一提的是，尽管在新冠流行期间面临诸多挑战，但维昇药业的三个临床实验都实现了既定里程碑目标，并都取得了阳性结果。另一方面，维昇药业以核心产品隆培促生长素为起点，构建自身的商业化核心团队，着力于通过创新模式提升维昇产品的可及性。通过链接专家学术网络、临床医生和患者群体，维昇药业建立包括疾病科普、患者教育、疾病管理等项目，以强化内分泌治疗创新药从基础研究、临床开发、生产制造到商业化的全链条产业布局，保证国内药品供应可持续性。近期，维昇药业与上药控股有限公司（以下简称“上药”）围绕维昇药业商业化产品，开启了创新药全生命周期服务合作，明确上药的进口总经销商地位，并进一步加速维昇药业产品的商业化进程。 04 专攻未满足临床需求，3条FIC/BIC管线上市在即 ■ 核心产品：隆培促生长素维昇药业的研发管线中进展最快的当属隆培促生长素：已在在中国完成3期临床试验，上市许可申请（BLA）于2024年获得NMPA正式受理，用于治疗儿童生长激素缺乏症。与此同时，隆培促生长素已在美国及欧洲获批上市，是欧美药监首个批准用于治疗儿童生长激素缺乏症的长效生长激素，其在国外的英文商品为 Skytrofa，由丹麦Ascendis公司生产销售，该产品在2023年第四季度已成为美国销量第一的生长激素产品。隆培促生长素是目前唯一一款可在体内持续释放未经修饰的生长激素分子、具有“天然作用”的长效生长激素，该未经修饰的生长激素的分子结构与人体自身分泌的内源性生长激素相同，具有与内源性生长激素一致的组织分布和生理作用：通过血液循环抵达并进入靶组织，与靶组织的生长受体结合发挥直接作用；通过与肝脏内的生长激素受体结合，促进胰岛素样生长因子-1（IGF-1）的分泌而发挥间接作用。相较之下，其它基于蛋白修饰技术研发的长效生长激素，通常会通过大幅增加其活动分子的大小以实现长效作用，但这种对生长激素分子大小及结构的改变，会导致其与生长激素受体的亲和力发生变化，降低进入靶组织的能力，其作用机理异于内源性生长激素，进而潜在影响这些长效生长激素的有效性或安全性。隆培促生长素这种“天然作用”的独特机制，也进一步反映在其临床试验结果上：全球及中国的3期临床试验均证实其促进儿童生长的疗效显著优于生长激素日制剂，同时安全性与日制剂相当，隆培促生长素是目前全球唯一优效于日制剂的长效生长激素。另外，在已知的全球3期长期开放性研究enliGHten试验中，隆培促生长素也显示出长达5年的持续有效性和长期安全性。中国目前已是全球最大的生长激素市场，且渗透率低、增长快。弗若斯特沙利文数据显示，2022年中国儿童生长激素缺乏症患者数已经达到337.46万人。其中，父母扮演着至关重要的角色，他们对于治疗方案的选择具有极大的影响力，因此，为了降低父母的照护难度，除提供大大降低给药频率的长效制剂外，给药操作便利程度与携带储存便利性也是他们选择治疗方案时重要考量因素。维昇药业的隆培促生长素在这一方面也显示出了独特的优势，与其他只能在2-8℃条件下储存（冰箱冷藏）的生长激素产品只不同，隆培促生长激素在无需冰箱的条件下，室温可保存6个月，这显然极大提升了患者携带和使用的便利性。此外，该产品还配备获得红点奖等3项重量级行业大奖的自动注射笔，以自动注射、隐藏针头为主要特点，进一步提升了患儿的用药体验。当前，全球共有4款长效生长激素获批上市。除了仅在中国上市的金赛增（金赛药业，长春高新子公司），以及首个在欧美上市用于儿童、在中国已由维昇药业递交BLA的隆培促生长素外，诺和诺德的Sogroya、和辉瑞的Ngenla 也已于2023年先后在美国和欧盟等主要国家获批上市。其中，搭载了创新TransCon长效技术的隆培促生长素，在产品特性上具有“天然作用”的独特优势，只需一周给药一次的同时，其活性成分也保持了生长激素的天然结构；此外，相比每日注射的生长激素日制剂具有更优的长高疗效，可以给患儿和家长带来更多治疗获益，是一款极具竞争力的长效生长激素产品。 ■ 潜力单品：那韦培肽那韦培肽（TransCon C-型利钠肽）是维昇药业一款用于儿童软骨发育不全（ACH）的在研创新疗法。软骨发育不全是一种染色体遗传疾病，表现为身材矮小且伴随多种骨骼并发症，已经被纳入了国家《第二批罕见病目录》。那韦培肽是中国第一款、也是唯一一款正在进行上市临床开发的针对软骨发育不全根本病因的疗法，旨在以安全和方便的每周一次给药，持续抑制ACH患儿过度激活的FGFR3受体通路，恢复软骨细胞的正常分化和增生以及全身骨骼的生长发育，同时改善和预防软骨发育不全的并发症。在全球范围内，首款用于儿童软骨发育不全的C-型利钠肽类似物伏索利肽于2021年由FDA批准上市，但在中国尚未开展任何上市相关临床研究。相较之下，那韦培肽先后于2019年2月及2020年8月获得FDA和欧盟委员会（EC）授予用于治疗软骨发育不全的孤儿药认定。2023年11月，那韦培肽中国2期临床试验双盲期完成，达到主要终点。2024年9月，那韦培肽全球关键3期临床试验达成主要研究目标，年化生长速率显著优效于安慰剂。 ■ First & Only：帕罗培特立帕肽甲状旁腺功能减退症（简称甲旁减，HP）这一疾病是目前唯一未能用激素替代疗法治愈的内分泌疾病。维昇药业在研的帕罗培特立帕肽，就是一款可用于甲旁减激素替代治疗的创新药物，旨在恢复每天24小时内甲状旁腺激素的生理水平，从而从根本上解决应对甲旁减各个方面的问题，包括使血钙、尿钙以及血磷恢复正常水平，降低远期并发症风险。帕罗培特立帕肽先后于2023年11月、2024年6月及2024年8月获得欧盟委员会（EC）、英国药监机构（MHRA）、FDA批准，用于成人慢性甲状旁腺功能减退症的激素替代治疗。它的研发及成功上市终结了甲状旁腺功能减退症缺乏激素替代疗法的时代，也成为目前全球第一也是唯一可用于HP激素替代治疗的药物。帕罗培特立帕肽做为甲旁减的创新疗法，也将助力中国患者摆脱传统治疗的困境。2023年1月，帕罗培特立帕肽中国3期关键试验双盲期完成，达到主要复合终点。此后，维昇公布了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，26周随机、双盲、安慰剂对照的试验数据。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。帕罗培特立帕肽组77.6%（45人/58人）的患者达到了主要复合终点，而安慰剂组为0.0%（0人/22人）（p值<0.0001），更有89.7% 使用帕罗培特立帕肽的患者可停用传统治疗。这些结果与Ascendis公布的帕罗培特立帕肽全球3期临床试验结果一致。 05 写在最后 “双十定律”在前，一款新药产品在历经重重挑战走过临床前、1期、2期和3期临床，并成功赢得监管机构的批准后，也并不能保证其能够带来预想中的商业回报，因为从研发效率、差异化技术研发、精准选品到稳步推进商业化，各环节牵一发动全身，容错率极低。维昇药业通过反向NewCo落地中国，从核心产品临床数据交付满意答卷，到上市许可申请成功受理，再至企业港股上市在即，前路已行稳。在国产替代成为潮流的当下，这样的反向NewCo一路的心路历程反倒为中国创新药出海指明一条道路。 “踏上取经路，比抵达灵山更重要”，现在的维昇药业正面临商业化实战这一考验，相信“心怀一片赤诚，灵山便在脚下”，任重道远。目前维昇药业团队正在寻找融资与商业合作伙伴，如果您对维昇药业的产品感兴趣，请与我们联系。近期推荐声明：动脉网所刊载内容之知识产权为动脉网及相关权利人专属所有或持有。未经许可，禁止进行转载、摘编、复制及建立镜像等任何使用。动脉网，未来医疗服务平台

并购ASCO会议IPO临床2期

100 项与 Navepegritide 相关的药物交易

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研发状态

10 条进展最快的记录,

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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	爱尔兰	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	丹麦	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
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	美国	Ascendis Pharma Growth Disorders A/S	2023-03-03

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临床结果

适应症

分期

评价

查看全部结果

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)	(鹽憲壓膚積範憲糧蓋願) = 顧製衊衊範願鑰襯鏇鏇鏇網衊糧鹽醖築願蓋製 (憲廠鬱廠艱獵網齋繭糧 ) 更多	积极	2024-09-16
				Placebo (ages 2-11 years)	(鹽憲壓膚積範憲糧蓋願) = 顧鏇構淵蓋窪鏇鑰鑰觸鏇網衊糧鹽醖築願蓋製 (憲廠鬱廠艱獵網齋繭糧 ) 更多
NCT04085523 (ACcomplisH, Company_Website) 人工标引	临床2期	软骨发育不全	57	TransCon CNP	(齋觸憲獵獵選繭壓鹹艱) = patients treated for one year at the pivotal 100μg/kg/week dose demonstrated significant improvements in well-being and physical functioning compared to placebo. 襯蓋願齋膚獵網壓顧餘 (艱餘鬱襯醖願醖壓製鹽 ) 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)	鬱網壓鹹觸鬱遞獵憲襯(淵簾淵鏇艱糧簾廠網憲) = 壓衊廠鑰膚醖餘衊膚鬱淵鏇蓋範憲範範簾範觸 (廠壓遞獵糧廠鹹製糧簾, 鑰顧遞觸襯鬱範鬱襯遞 ~ 淵鑰襯憲齋願獵網願窪) 更多	-	2024-03-07
				TransCon CNP (TransCon CNP 20 mcg)	鬱網壓鹹觸鬱遞獵憲襯(淵簾淵鏇艱糧簾廠網憲) = 窪膚選襯願網顧網鹽鹹淵鏇蓋範憲範範簾範觸 (廠壓遞獵糧廠鹹製糧簾, 觸鹹衊繭選廠蓋構齋廠 ~ 夢鹽鑰醖築醖齋鏇鑰遞) 更多
NCT05246033 (ACcomplisH China, PRNewswire) 人工标引	临床2期	软骨发育不全	24	TransCon CNP 50μg/kg/week	(鑰製膚鑰衊鏇膚願觸繭) = 鹽選憲築築艱範淵廠積憲夢築網艱簾繭餘範簾 (廠鏇襯選糧選築鹽顧築 ) 达到	积极	2023-11-16
				TransCon CNP 100μg/kg/week	(鑰製膚鑰衊鏇膚願觸繭) = 顧齋鹹廠網齋餘膚網鹹憲夢築網艱簾繭餘範簾 (廠鏇襯選糧選築鹽顧築 ) 达到