Transplant Therapeutics Consortium Receives FDA Acceptance of the Qualification Plan for IBox Scoring System
Transplant Therapeutics Consortium Receives FDA Acceptance of the Qualification Plan for IBox Scoring System
Improving the long-term survival of transplanted kidneys is an important area of unmet need for transplantation recipients, and clinical trials testing new immunosuppressive agents are needed to improve kidney transplant outcomes. Although the current efficacy failure endpoint has typically shown the non-inferiority of immunosuppressive regimens, the iBox Scoring System is proposed to be used to demonstrate the superiority of a new agents compared to the standard of care (SOC) as a co-primary endpoint in pivotal drug therapeutic studies. As a reasonably likely surrogate endpoint, the iBox scoring system would be used in the Accelerated Approval Pathway at the FDA designed to allow for earlier approval of drugs that treat serious conditions and fill an unmet medical need.The iBox Scoring System is a novel endpoint predictive of long-term survival after kidney transplantation
iBox评分系统是一种新颖的终点预测，可预测肾移植后的长期存活
MOUNT LAUREL, N.J., Nov. 20, 2024 /PRNewswire/ -- The American Society of Transplant Surgeons (ASTS) and American Society of Transplantation (AST) announced today that the Transplant Therapeutics Consortium (TTC) received notification from FDA of acceptance of the Qualification Plan for the iBox Scoring System, a novel reasonably likely surrogate efficacy endpoint for kidney transplant clinical trials. This takes us one step closer to having new therapies that better the lives of transplant recipients and marks the successful completion of step two in the three-step process for FDA biomarker qualification. The iBox scoring system is the first transplant endpoint to reach this stage of qualification.
新泽西州莫林特市，2024年11月20日 / PRNewswire/ - 美国移植外科医生学会（ASTS）和美国移植学会（AST）今天宣布，移植治疗联盟（TTC）收到FDA关于iBox评分系统资格计划的认可通知，这种新型而有可能的委托有效终点已经成为肾移植临床试验的合理替代终点。这让我们离拥有改善移植受体生活的新疗法更近了一步，也标志着FDA生物标记资格三步流程中的第二步成功完成。iBox评分系统是第一个达到这一资格阶段的移植终点。
"With the iBox Scoring System on the pathway to FDA qualification, we are closer than ever to cutting-edge clinical tools that could extend the lives of transplant recipients. iBox is opening the door to using artificial intelligence and machine learning. This achievement signals a transformative shift in how we approach clinical trials for transplant patients and is a critical step forward in modernizing the field," said AST President Jon Kobashigawa, MD.
“随着iBox评分系统走上FDA资格之路，我们比以往任何时候都更接近尖端临床工具，这些工具有望延长移植受体的生命。iBox正在开启使用人工智能和机器学习的大门。这一成就标志着我们在如何对待移植患者的临床试验方面发生了变革，并且是现代化该领域的关键步骤，”美国移植学会会长乔恩·小林川川博士说。
Improving the long-term survival of transplanted kidneys is an important area of unmet need for transplantation recipients, and clinical trials testing new immunosuppressive agents are needed to improve kidney transplant outcomes. Although the current efficacy failure endpoint has typically shown the non-inferiority of immunosuppressive regimens, the iBox Scoring System is proposed to be used to demonstrate the superiority of a new agents compared to the standard of care (SOC) as a co-primary endpoint in pivotal drug therapeutic studies. As a reasonably likely surrogate endpoint, the iBox scoring system would be used in the Accelerated Approval Pathway at the FDA designed to allow for earlier approval of drugs that treat serious conditions and fill an unmet medical need.
改善移植肾的长期存活是移植受体一个重要的未满足需求领域，需要进行测试新的免疫抑制药物的临床试验，以改善肾移植结果。尽管当前的有效性失败终点通常表明免疫抑制方案的非劣效性，但建议使用iBox评分系统来证明新药物与护理标准（SOC）相比的优越性作为关键药物治疗研究中的共同主要终点。作为合理可能的替代终点，iBox评分系统将在FDA的加速批准路径中使用，该路径旨在允许提前批准治疗严重疾病并填补未满足医疗需求的药物。
"We are delighted that the iBox which received endorsement and qualification by the European Medicines Agency (EMA) in 2022 now also has received acceptance of the FDA Biomarker Qualification Plan as an efficacy endpoint for kidney transplant clinical trials. This acceptance will facilitate more rapid assessment of novel Immunotherapeutics for kidney transplant recipients and harmonization of multinational clinical trials. We are grateful to our many partners and collaborators in the Transplant Therapeutics Consortium for their steadfast support of this important initiative to facilitate innovation in transplantation," said ASTS President Ginny Bumgardner.
“我们非常高兴，2022年欧洲药品管理局（EMA）认可并资格认证的iBox现在也已获得FDA生物标志物资格计划的接受，作为肾移植临床试验的有效终点。这一接受将促进更快地评估肾移植受体的新型免疫治疗药物，并协调跨国临床试验。我们感谢移植治疗学联盟中许多合作伙伴和合作者对这一促进移植创新的重要举措的坚定支持，”ASTS主席吉尼·邦加德纳博士表示。
"We desperately need new regulatory pathways which bring investment into transplantation. New therapies are essential to prolong the life of transplant recipients and take the best care of the gift of life provided by our donors" said Dr. Karin Hehenberger, President Lyfebulb, and a kidney and pancreas transplant recipient. "This step is critical to enable the dream of 'One Kidney for Life' to become a reality."
“我们迫切需要引入投资进入移植领域的新监管路径。新疗法对延长移植受体的生命至关重要，并最好地照顾我们的捐赠者提供的生命礼物，”Lyfebulb主席、肾脏和胰腺移植受体卡琳·海恩贝格尔博士表示。“这一步骤对实现‘一生只要一个肾’的梦想至关重要。”
The iBox scoring system is a composite endpoint used as a risk prediction tool that utilizes multiple clinically relevant features (estimated glomerular filtration rate (eGFR), proteinuria, anti-human leukocyte antigen donor-specific antibody, and kidney graft biopsy histopathology) at one-year post-transplant to predict 5-year graft survival. The iBox Qualification Plan is supported by strong mechanistic and epidemiologic data of the association with late kidney functional decline and failure and includes data from single center and randomized controlled trials representing over 2,500 de novo kidney transplant recipients.
iBox评分系统是一个综合终点，作为风险预测工具，利用多个临床相关特征（估算的肾小球滤过率（eGFR）、蛋白尿、抗人白细胞抗原特异性抗体和肾脏移植活检病理学）在移植后一年预测5年移植物存活率。iBox资格计划得到了与晚期肾功能下降和衰竭相关的强有力机制和流行病学数据的支持，并包括来自代表2500多个新的肾脏移植受体的单个中心和随机对照试验的数据。
The iBox scoring system (NCT03474003), has been developed and validated in a landmark publication released in the BMJ by a team of researcher led by Pr Alexandre Loupy in Paris French NIH (Inserm), APHP and Université Paris Cité, comprising 7557 patients from 10 academic centres from Europe and the US and 3 RCT's. iBox leveraged high quality longitudinal data to build a robust predictive system for long-term kidney allograft loss. iBox has been extensively validated for use in the treatment of individual patients in the clinical care setting and common clinical scenarios including response to treatment in in T-cell mediated rejection, antibody-mediated rejection and immunosuppressive regimen minimization (BMJ 2019; 366:l4923).
iBox评分系统（NCT03474003），由巴黎法国国立卫生研究院（Inserm）、APHP和巴黎城市大学的Alexandre Loupy教授领导的团队在BMJ上发布的一篇重要文章中开发和验证，共涵盖了来自欧洲和美国的10个学术中心和3个RCT的7557名患者。iBox利用高质量的纵向数据构建了一个强大的预测系统，用于预测长期肾移植物丧失。iBox已广泛验证，可用于在临床护理环境中治疗个体患者和常见的临床病例，包括对T细胞介导的排斥、抗体介导的排斥和免疫抑制方案最小化治疗反应的检查（BMJ 2019；366：l4923）。
The Transplant Therapeutics Consortium (TTC) is a public-private partnership founded in 2017 by the American Society of Transplantation (AST) and the American Society of Transplant Surgeons (ASTS). This collaboration unites the transplant community, industry leaders, and regulatory agencies to foster consensus-driven, data-focused research aimed at enhancing the development of innovative tools and methods for evaluating the safety and efficacy of solid organ transplants (SOT).
移植治疗财团（TTC）是美国移植学会（AST）和美国移植外科医生学会（ASTS）于2017年创立的公私合作伙伴关系。这种合作将整合移植社区、行业领袖和监管机构的力量，推动以共识为基础、数据为导向的研究，旨在提升固体器官移植（SOT）安全性和有效性评估方法的创新工具和方法的发展。
This regulatory qualification achievement was accomplished by employing the resources of the TTC members and engaging with FDA throughout the biomarker qualification process. TTC is supported through funding and input from the AST*, ASTS*, argenx*, Arkana Laboratories*, Bristol Myers Squibb*, CareDx*, Critical Path Institute, CSL, CTI Clinical Trial Services, Inc.*, Eledon*, European Society of Organ Transplantation, the U.S. Food and Drug Administration (FDA), Hansa Biopharma*, HUS Helsinki University Hospital, Immucor, KU Leunven, Memo Therapeutics AG*, Natera, National Institutes of Health, Novartis, Paris Transplant Group, Pirche, Sanofi*, Takeda*, Talaris Therapeutics, Thermo Fisher Scientific, The Transplantation Society, Transplant Genomics*, University of Manitoba, and Veloxis Pharmaceuticals*.
这一监管资格取得是通过利用TTC成员的资源，并在整个生物标志物资格过程中与FDA合作而实现的。TTC得到AST*、ASTS*、argenx*、Arkana Laboratories*、Bristol Myers Squibb*、caredx*、Critical Path Institute、CSL、CTI临床试验服务公司*、Eledon*、欧洲器官移植学会、美国食品药品监督管理局（FDA）、海莎生物制药*、HUS赫尔辛基大学医院、Immucor、KU列文大学、Memo Therapeutics AG*、natera、美国国家卫生研究所、诺华、巴黎移植小组、Pirche、赛诺菲安万特*、武田*、Talaris Therapeutics、赛默飞世尔、移植学会、移植基因组学*、曼尼托巴大学和Veloxis Pharmaceuticals*的资金支持和意见。
TTC is working on its next regulatory milestone, the submission of the Full Qualification Package for regulatory endorsement of the iBox Scoring System as a reasonably likely surrogate endpoint by the FDA.
TTC正在努力实现下一个监管里程碑，即提交iBox评分系统的全面资格包，以获得FDA对其作为相对可能的替代终点的认可。
As part of the 21st Century Cures Act, passed into law in December 2016, public-private partnerships consisting of government entities, including FDA, the biopharmaceutical industry, health care providers, academic researchers, and patient advocacy organizations, have been encouraged to work together to foster innovation in the development of new therapies by qualifying new drug development tools that can accelerate the process of making new therapies available to patients. For more information, click here. Any groups that would like to join in this effort or have information or data that may contribute to further advances, can contact JoAnn Gwynn at [email protected].
作为2016年12月通过的《21世纪医治法案》的一部分，政府实体、包括FDA、生物制药行业、医疗保健提供者、学术研究人员和患者倡导组织等组成的公私合作伙伴关系得到鼓励，共同促进新疗法开发的创新，通过评定新药开发工具，以加速使新疗法面向患者。欲了解更多信息，请点击这里。任何希望加入此努力或具有可能促进进一步进展的信息或数据的团体，可以通过[email protected]联系JoAnn Gwynn。
The Transplant Therapeutics Consortium (TTC) was launched in April 2017 and co-founded by the American Society of Transplantation (AST) and the American Society of Transplant Surgeons (ASTS). TTC brings together pharmaceutical companies, diagnostic companies, academic and nonprofit partners working toward a common goal of moving the field forward toward drug development solutions in transplantation.
移植治疗协会(TTC)于2017年4月启动，由美国移植学会(AST)和美国移植外科医师学会(ASTS)共同创立。TTC汇集了药品公司、诊断公司、学术和非营利伙伴，共同致力于向前推进领域，推动移植领域的药物研发解决方案。
American Society of Transplantation
Founded in 1982, the American Society of Transplantation (AST) is a non-profit, 501(c)3 organization dedicated to advancing the field of transplantation and improving patient care by promoting research, education, advocacy, organ donation, and service to the community through a lens of equity and inclusion. The society is the largest transplant organization in North America (consisting of nearly 5,000 professional members), representing a majority of the nation's medical professionals engaged in the field of transplantation.
美国移植学会
成立于1982年，美国移植学会(AST)是一家非营利性的501(c)3组织，致力于推动移植领域的发展，并通过促进研究、教育、倡导、器官捐献和为社区提供服务来改善患者护理，以平等和包容的视角。该学会是北美最大的移植组织(拥有近5000名专业会员)，代表着该领域从事工作的绝大多数全国医疗专业人员。
Learn more at myAST.org.
请在myASt.org了解更多。
American Society of Transplant Surgeons
The American Society of Transplant Surgeons (ASTS) was founded in 1974 by 127 surgeons to unite the professionals who were advancing the fledgling field of organ transplantation.
美国移植外科医师学会
美国移植外科医师学会(ASTS)成立于1974年，由127名外科医师联合成立，旨在团结那些正在推动新生器官移植领域发展的专业人员。
From this small group of dedicated individuals, we have grown to represent over 2,000 transplant professionals who are dedicated to saving and improving lives through the transformative power of organ donation and transplant surgery. Learn more about ASTS by visiting our website at ASTS.org.
从这批敬业的小团体发展壮大，我们现在代表着2000多名致力于通过器官捐献和移植手术拯救和改善生命的移植专业人士。了解更多关于ASTS的信息，请访问我们的网站asts.org。
*Denotes Current TTC Members
*标记当前TTC成员
MEDIA CONTACTS:
媒体联系人：
Shauna O'Brien – AST: [email protected]
Shauna O'Brien – 医院: [email protected]
Anna Shults – ASTS: [email protected]
Anna Shults – 医院S: [email protected]
SOURCE American Society of Transplantation
来源 美国移植协会
