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Transplant Therapeutics Consortium Receives FDA Acceptance of the Qualification Plan for iBox Scoring System
The iBox Scoring System is a novel endpoint predictive of long-term survival after kidney transplantation
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.
"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.
Improving the long-term survival of transplanted kidneys is an important area of un
met 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.
"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.
"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."
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.
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).
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).
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*.
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.
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 jgwynn@myast.org.
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.
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.
Learn more at myAST.org.
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.
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.
*Denotes Current TTC Members
MEDIA CONTACTS:
Shauna O'Brien – AST: sobrien@myAST.org
Anna Shults – ASTS: anna.shults@asts.org
argenx* is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. Argenx developed and is commercializing the first-and-only approved neonatal Fc receptor (FcRn) blocker in the U.S., Japan and the E.U. The Company is evaluating efgartigimod in multiple serious autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit www.argenx.com and follow us on LinkedIn, Twitter and Instagram.
Arkana Laboratories* is a center for esoteric pathology focused on one thing: improving care for patients. It is our mission to improve patient care by advancing understanding of disease and providing world-class diagnostics. We are committed to providing doctors with the highest quality, clinicopathologic discussion possible. We recognize that in order to best serve the needs of our clinicians we must work together with them, not just as a lab, but as an extension of their practice. It is our goal to help equip doctors with the results they need to deliver the most effective patient care possible. As pathologists, we recognize that the longest period in a patient’s life is the timeframe between the day he/she has a biopsy, and the day the result arrives.
Bristol Myers Squibb* is a leading global biopharmaceutical company focused on discovering, developing, and delivering innovative medicines for patients with serious diseases in areas including oncology, hematology, immunology, cardiovascular, and neuroscience. Our employees work every day to transform patients’ lives through science. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
CareDx* is committed to improving transplant patient outcomes by providing innovative and intelligent solutions throughout the entire patient journey. With 25 years of commitment to transplant patients and care teams, we have developed close partnerships across the transplant ecosystem that grow stronger every day. For more information, please visit: www.CareDx.com.
The Critical Path Institute (C-Path) is a catalyst in the development of new approaches to advance medical innovation and regulatory science. C-Path Institute achieves this by leading team that share data, knowledge, and expertise resulting in sound, consensus-based science.
CSL is a global biotherapeutics leader driven by our promise to save lives. Focused on serving patients' needs by using the latest technologies, we discover, develop and deliver innovative therapies for people living with conditions in the immunology, hematology, cardiovascular and metabolic, respiratory, and transplant therapeutic areas. We use three strategic scientific platforms of plasma fractionation, recombinant protein technology, and cell and gene therapy to support continued innovation and continually refine ways in which products can address unmet medical needs and help patients lead full lives. CSL operates one of the world's largest plasma collection networks, CSL Plasma. Our parent company, CSL Limited, headquartered in Melbourne, Australia, employs more than 25,000 people, and delivers its lifesaving therapies to people in more than 100 countries.
CTI Clinical Trial and Consulting Services is a global, privately held, research service organization, delivering a complete spectrum of clinical trial and consulting services throughout the lifecycle of development, from concept to commercialization. CTI’s focused therapeutic approach provides clinical and disease area expertise with nuanced regulator support. CTI is one of the 20 largest contract research organizations in the world, with associates in more than 60 countries across six continents. CTI is headquartered in the Greater Cincinnati area, with operations across North America, Europe, Latin America, MEA, and Asia-Pacific. For more information, visit www.ctifacts.com.
Eledon* is dedicated to “One Transplant For Life” to significantly improve both the longevity of the organ and the overall health and quality of life for transplant recipients so that a donor’s greatest gift will keep on giving. Our lead compound is tegoprubart, an anti-CD40L antibody with broad therapeutic potential to regulate the drivers that can lead to the immune system attacking and rejecting transplanted organs.
The European Society for Organ Transplantation (ESOT) was founded 40 years ago and is dedicated to the pursuit of excellence in organ transplantation. Facilitating a wealth of international clinical trials and research collaborations over the years, ESOT remains committed to its primary aim of improving patient outcomes in transplantation. With a community of over 8,000 members from around the world, ESOT is an influential international organisation and the facilitator of the biennial congress which hosts approximately 3,500 experts who come to meet to explore and discuss the latest scientific research. ESOT attracts the foremost transplantation experts to work in its committees and sections. ESOT works with leading transplantation experts and supports research, education, and European policy changes.
The Food and Drug Administration is responsible for protecting the public health by ensuring the safety, efficacy, and security of human and veterinary drugs, biological products, and medical devices; and by ensuring the safety of our nation's food supply, cosmetics, and products that emit radiation. FDA also has responsibility for regulating the manufacturing, marketing, and distribution of tobacco products to protect the public health and to reduce tobacco use by minors. FDA is responsible for advancing the public health by helping to speed innovations that make medical products more effective, safer, and more affordable and by helping the public get the accurate, science-based information they need to use medical products and foods to maintain and improve their health. FDA also plays a significant role in the Nation's counterterrorism capability. FDA fulfills this responsibility by ensuring the security of the food supply and by fostering development of medical products to respond to deliberate and naturally emerging public health threats.
Hansa Biopharma* is a pioneering commercial-stage biopharmaceutical company on a mission to develop and commercialize innovative, lifesaving, and life-altering treatments for patients with rare immunological conditions. Hansa has developed a first-in-class immunoglobulin G (IgG) antibody cleaving enzyme therapy, which has been shown to enable kidney transplantation in highly sensitized patients. Hansa has a rich and expanding research and development program, based on the Company's proprietary IgG-cleaving enzyme technology platform, to address serious unmet medical needs in transplantation, autoimmune diseases, gene therapy, and cancer. Hansa Biopharma is based in Lund, Sweden, and has operations in Europe and the U.S. The Company is listed on Nasdaq Stockholm under the ticker HNSA. Find out more at https://hansabiopharma.com.
At HUS Helsinki University Hospital about 680 000 patients receive medical care annually. We have almost 27,000 professionals working for the best of all patients, and we are responsible for providing specialized health care for the residents of our 24 member municipalities. In addition, the treatment of many rare and severe diseases in Finland, such as solid-organ transplantations, is nationally centralized to HUS. HUS is the biggest public health care provider and the second largest employer in Finland. Our expertise is internationally recognized and accredited. As a university hospital, we continuously develop and evaluate our treatment methods and activities. HUS Abdominal Center, Transplantation and Liver Surgery, is currently one of the largest kidney and pancreas transplant programs in Northern Europe, with a total of 268 kidney transplantations and 31 pancreas transplantations performed in 2021."
Founded in 1982, Immucor is a global leader in transfusion and transplantation diagnostics that facilitate patient-donor compatibility. We strive to create a world where anyone, anywhere in need of transfusion or transplantation gets the right blood or transplant that is safe, accessible and affordable. With the right match, we can transform a life together. To learn more, visit https://www.immucor.com or our social media channels: LinkedIn, Twitter, Facebook, Instagram, and YouTube.
K.U. Leuven is Europe's most innovative university (Reuters) and ranks 42nd in the Times Higher Education World University Rankings. As Belgium's largest university, K.U. Leuven welcomes almost 60,000 students from over 140 countries. Its 7,000 researchers are active in a comprehensive range of disciplines. K.U. Leuven is a founding member of the League of European Research Universities (LERU) and has a strong European and international orientation. University Hospitals Leuven, its network of research hospitals, provides high-quality healthcare and develops new therapeutic and diagnostic insights with an emphasis on translational research.
The University of Manitoba is the province's only research-intensive university, educating the majority of professionals in Manitoba. The U of M is a trailblazer in many areas of learning, research and discovery, well-placed to encourage debate and discussion around the understanding of health, human rights, climate change and Indigenous studies. The U of M is home of the Truth and Reconciliation Commission of Canada.
Memo Therapeutics AG* (“MTx”) is a late-stage biotech company translating unique human immune responses into superior medicines through the development of best-in-class antibodies to treat viral infections and cancer. The Company’s lead program, potravitug, is in Phase II development targeting BK viremia in kidney transplant recipients, an infection which can result in decreased kidney functionality and longevity and reduced patient survival. Potravitug has the potential to become the best and first-in-class BKV disease-modifying therapy for kidney transplant patients with a market potential of more than $1bn. Alongside potravitug, MTx is focused on discovering novel oncology targets, both alone and in partnership. The Company entered into a partnership with Ono Pharmaceutical in late 2022. Underpinning MTx’s core assets is its proprietary DROPZYLLA® technology, an antibody repertoire copying engine with high- throughput screening capabilities. MTx is a private company located in Schlieren / Zurich and backed by investors including Ysios Capital, Kurma Partners, Pureos Bioventures, Swisscanto, Vesalius Biocapital and Adjuvant Capital. Learn more at www.memo-therapeutics.com, and on LinkedIn.
NIH’s mission is to seek fundamental knowledge about the nature and behavior of living systems and the application of that knowledge to enhance health, lengthen life, and reduce illness and disability. The goals of the agency are: to foster fundamental creative discoveries, innovative research strategies, and their applications as a basis for ultimately protecting and improving health; to develop, maintain, and renew scientific human and physical resources that will ensure the Nation's capability to prevent disease; to expand the knowledge base in medical and associated sciences in order to enhance the Nation's economic well-being and ensure a continued high return on the public investment in research; and to exemplify and promote the highest level of scientific integrity, public accountability, and social responsibility in the conduct of science.
Natera provides advanced DNA technology that enables transplant providers to deliver quality, personalized care. As a diagnostics company with proprietary bioinformatics and molecular technology, we’ve performed more than 3 million cfDNA tests and are dedicated to improving kidney care for good. Learn more at natera.com/organ-health.
Novartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 108,000 people of more than 140 nationalities work at Novartis around the world. Find out more at https://www.novartis.com.
The Paris Transplant Group (PTG) is a French INSERM research team founded in 2008. The PTG is developing a personalized approach for transplant medicine that integrates multidimensional information derived from standard of care (clinical and biological data, histology and immunology) together with novel information coming from cutting edge technologies in immunology, molecular biology, genetics and biomarkers. It also leads multiple clinical trials and focuses on providing endpoints for clinical trials with insights in therapy and patient clinical management. The team includes several departments in kidney, heart, lung and liver transplantation worldwide with many different experts including clinicians, surgeons, pathologists, immunologists, epidemiologists, statisticians, mathematicians.
Pirche is a technology leader in the application of AI to molecular medicine and has developed an exclusive epitope matching technology for risk assessment which may lead to improvements in organ allocation, management of long-term immunosuppression, and selection of induction therapies. The technology is redefining compatibility risk assessment as this is the prerequisite for foreign tissues to successfully enter the body and be accepted by the recipient immune system.
Sanofi* We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people's lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions.
Takeda* is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines. More information can be found on www.clinicaltrials.takeda.com.
Talaris Therapeutics, Inc. is a late-clinical stage cell therapy company developing therapies with the potential to transform the standard of care in solid organ transplantation and severe immune and blood disorders. Talaris maintains corporate offices in Boston, MA, its cell processing facility in Louisville, KY, and additional research operations in Houston, TX.
Thermo Fisher Scientific Inc. is the world leader in serving science, with annual revenue of approximately $40 billion. Our Mission is to enable our customers to make the world healthier, cleaner and safer. Whether our customers are accelerating life sciences research, solving complex analytical challenges, increasing productivity in their laboratories, improving patient health through diagnostics or the development and manufacture of life-changing therapies, we are here to support them. Our global team delivers an unrivaled combination of innovative technologies, purchasing convenience and pharmaceutical services through our industry-leading brands, including Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific, Unity Lab Services, Patheon and PPD. For more information, please visit www.thermofisher.com.
Transplant Genomics* is a personalized diagnostics company committed to improving organ transplant outcomes worldwide through innovative tests that detect early signs of graft injury, differentiate among actionable causes, and enable the optimization of therapy. Working alongside the transplant community, Transplant Genomics is commercializing a suite of tests enabling diagnoses and prediction of transplant recipient immune status. Our flagship product is OmniGraf, a non-invasive dual-biomarker combination test that offers the earliest and most accurate detection of rejection for transplant recipients - empowering clinicians to offer patients the best possible opportunity for successful long-term outcomes. Transplant Genomics became part of the Eurofins family of companies in 2019. Learn more about Eurofins Transplant Genomics at http://www.transplantgenomics.com.
Established in 1966, The Transplantation Society serves as the principal international forum for the advancement and development of both the science and clinical practice of transplantation throughout the world. Its mandate is fulfilled through scientific advancement and communication, continuing education and guidance on the ethical practice of transplantation. The Transplantation Society is a Non-Governmental Organization (NGO) in official relations with the World Health Organization and is composed of over 6,000 professionals including but not limited to, physicians, surgeons, scientists and allied health professionals in 105 countries. TTS is one of two parent organizations of the Declaration of Istanbul, the other being the International Society of Nephrology. In addition, TTS has ten Sections specializing in various areas of transplantation: cell transplant and regenerative medicine, vascularized composite allotransplantation, pancreas and islet transplant, pediatric, organ donation and procurement, intestinal rehabilitation and transplant, xenotransplantation, pediatric liver transplantation, uterus and transplant infectious disease. TTS is a founding and sponsoring organization of the Declaration of Istanbul Custodian Group (DICG) formed to combat organ trafficking, transplant tourism, and transplant commercialism and to encourage adoption of effective and ethical transplantation practices around the world. TTS also partners with 35 national and regional transplant societies to advance the science and clinical practice of transplantation around the world.
Veloxis Pharmaceuticals, Inc.*, an Asahi Kasei company, is a fully integrated specialty pharmaceutical company committed to improving the lives of transplant patients. Headquartered in Cary, North Carolina., USA, Veloxis is focused on the global development and commercialization of medications utilized by transplant patients and by patients with serious related diseases. For further information, please visit www.veloxis.com.