Biochemist, Biotechnology Expert to Lead C-Path’s Type 1 Diabetes Consortium

TUCSON, Ariz., June 2, 2022 — Critical Path Institute (C-Path) has named Elnaz Atabakhsh, Ph.D., as Executive Director of its Type 1 Diabetes Consortium (T1DC).

Atabakhsh joined C-Path this May with extensive experience in the biotechnology and biomarker development industry. Her previous work focused on biomarker discovery and screening, cancer genetics and cell therapies for autoimmune disorders, aimed at supporting patients unable to be served by traditional therapeutics. Atabakhsh has extensive experience in and a passion for leading patient-oriented, mission-driven collaborations that include diverse representation from the scientific and medical communities.

C-Path’s type 1 diabetes portfolio consists of several projects targeting relevant stages of the type 1 diabetes disease continuum, before and after diagnosis. T1DC was established in 2017 to advance medical product development for therapies intended to prevent or delay the diagnosis of T1D. The Trial Outcome Markers Initiative in Type 1 Diabetes (TOMI-T1D) is an international partnership with a mission to optimize the design of immune intervention trials in new-onset type 1 diabetes. Co-funded by JDRF and Diabetes UK, TOMI-T1D is a partnership between academic institutions, pharmaceutical industry, and independent nonprofit organizations. Collectively, through these and other efforts, C-Path’s T1D portfolio engages the type 1 diabetes community to identify challenges to the development of new therapeutics and prioritize the development of data-driven solutions that optimize T1D drug development.

“Elnaz embodies the unique combination of scientific knowledge, business development and partnership excellence, as well as the passion and experience required to successfully lead this C-Path consortium,” said C-Path Chief Science Officer Klaus Romero, M.D., M.S., F.C.P. “The challenges faced in T1D drug development, including significant patient heterogeneity across disease stages represent a tangible unmet need. Now led by Elnaz, C-Path’s type 1 diabetes work is poised to reshape and reinvigorate the T1D development landscape.”

After receiving her Ph.D. in Biochemistry from the University of Western Ontario in London, Canada, Elnaz completed postdoctoral training at the Massachusetts General Hospital Cancer Center and Harvard Medical School.

Elnaz believes that her knowledge of various aspects of the biomarker development process, building collaborative teams and interacting with various industry and academic organizations will be beneficial in leading the consortium.

“It is my privilege to join C-Path and to lead our projects aimed at improving the lives of patients living with T1D, a disease that brings significant daily burden to millions around the world,” said Atabakhsh. “The T1D community has shown a remarkable passion and willingness to collaborate at a global scale, and I look forward to joining the community as we take on this important work.”

For more information on C-Path’s T1D efforts, visit: https://c-path.org/programs/t1d/ and https://c-path.org/programs/tomi-t1d/.


Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, is headquartered in Amsterdam, Netherlands and C-Path Ltd. operates from Dublin, Ireland with additional staff in multiple other locations. For more information, visit c-path.org.

Critical Path Institute is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) and is 54.2% funded by the FDA/HHS, totaling $13,239,950, and 45.8% funded by non-government source(s), totaling $11,196,634. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government.

Contact:

Kissy Black
C-Path
615.310.1894
kblack@c-path.org

C-Path Receives Qualification Opinion from EMA on Type 1 Diabetes Biomarker Initiative

 

TUCSON, Ariz., April 5, 2022 Critical Path Institute’s (C-Path) Type 1 Diabetes Consortium (T1DC) today announced that the European Medicines Agency (EMA) has issued a positive qualification opinion for pancreatic islet autoantibodies as enrichment biomarkers for type 1 diabetes (T1D) prevention trials. The purpose of this model-based qualification is to make publicly available tools to assist in the identification and selection of patients with a likelihood of progressing to a T1D clinical diagnosis, in trials of reasonable duration.

This regulatory endorsement will provide sponsors with the confidence to use islet autoantibodies in the optimization of clinical trials evaluating novel therapies focused on the delay and/or prevention of T1D. The models used to generate the underlying evidence for this EMA qualification opinion utilize islet autoantibody status with other patient features to identify a patient’s risk of progressing to a T1D diagnosis. Positivity for two or more of the autoantibodies, together with other patient features, will be used for enrichment of clinical trials focusing on the delay or prevention of the clinical diagnosis of T1D.

In their qualification opinion, EMA reiterated the unmet drug development need that T1DC targeted with this work, stating, “There is clearly an unmet need for biomarkers to aid development in T1DM prevention, a field with a long history of failed trials.” EMA’s qualification opinion statement says, “Positivity to at least 2 of the following islet AAs; IAA, GAD65, IA-2, and ZnT8 is qualified for use as enrichment biomarker, in combination with clinical parameters (sex, baseline age, blood glucose measurements from the 120-minute timepoints of oral glucose tolerance test (OGTT), and hemoglobin A1c (HbA1c) levels) in T1D prevention trials targeting individuals at risk of developing T1D.”

The incidence of T1D is on the rise worldwide, particularly in children. In Europe, incidence rates are between 0.2 and 0.5%, with steep rises in the number of children and young people being diagnosed in certain European countries. The ability to identify individuals at risk of progressing to a clinical diagnosis of T1D is a valuable opportunity to enrich clinical trials testing interventions that can potentially delay and ultimately prevent T1D.

“This qualification from the EMA would not have been possible without the tireless dedication and collaboration of the T1D research community,” said Marjana Marinac, Pharm.D., JDRF Senior Director of Regulatory Affairs and T1DC Co-Director. “JDRF applauds EMA’s decision to support and accelerate more innovation in therapies to delay and prevent T1D.”

“This endorsement from EMA is a result of many years of extensive work and extraordinary collaboration among clinical researchers, patient advocacy groups, nonprofit organizations, and members of the biopharmaceutical industry facilitated by T1DC at C-Path,” said Klaus Romero, M.D., M.S., F.C.P., C-Path Chief Scientific Officer. “This success was only possible through collaborative patient-level data sharing across stakeholders, and we are grateful to our many collaborators for their continued support. We look forward to the impact this tool will make in the development of novel therapies to treat the early stages of T1D with the goal of delaying or preventing the clinical onset of this disease.”

The Qualification Opinion can be found on the EMA website here, or on the T1D Consortium website here.

The consortium is also working on the next regulatory milestone: the endorsement of these islet autoantibodies by the U.S. Food and Drug Administration. C-Path’s T1D Consortium will achieve the regulatory endorsement of the islet autoantibodies currently used in clinical practice to diagnose T1D by employing the resources of all its members and engaging with regulatory agencies at each step of the process with funding and input from The Leona M. and Harry B. Helmsley Charitable Trust, Janssen Research & Development, LLC, JDRF International, Novo Nordisk A/S and Provention Bio.


About C-Path

Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, C-Path in Europe is headquartered in Amsterdam, Netherlands and C-Path Ltd. operates from Dublin, Ireland with additional staff in multiple other locations. For more information, visit c-path.org.

In the U.S., Critical Path Institute is supported by the Food and Drug Administration (FDA) of the Department of Health and Human Services (HHS) and is 54.2% funded by the FDA/HHS, totaling $13,239,950, and 45.8% funded by non-government source(s), totaling $11,196,634. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government.

C-Path’s Type 1 Diabetes Consortium (T1DC) is a public-private partnership initiated in March 2017. Currently membership is composed of the following Industry and Foundation members: The Leona M. and Harry B. Helmsley Charitable Trust; Janssen Research & Development, LLC; JDRF International; Novo Nordisk; and Provention Bio. Other consortium members, participants and advisors include individuals from the following organizations: Benaroya Research Institute at Virginia Mason; Lund University, Sweden; Helmholtz Zentrum München; University of Bristol; University of Colorado Denver; University of Florida; University of Helsinki; University of Leuven; University of Munich; University of Oulu; University of Tampere; University of Turku; Critical Path Institute; FDA and NIH.


The Leona M. and Harry B. Helmsley Charitable Trust aspires to improve lives by supporting exceptional efforts in the U.S. and around the world in health and select place-based initiatives. Since beginning active grantmaking in 2008, Helmsley has committed more than $3 billion for a wide range of charitable purposes. The Helmsley Type 1 Diabetes Program is one of the largest private foundation funders of T1D in the nation focused on understanding the disease, developing better treatments, and improving care and access in the U.S and low- and middle- income countries. For more information on Helmsley and its programs, visit helmsleytrust.org

About the Janssen Pharmaceutical Companies of Johnson & Johnson. 

At Janssen, we’re creating a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular & Metabolism, Immunology, Infectious Diseases & Vaccines, Neuroscience, Oncology, and Pulmonary Hypertension.

Learn more at www.janssen.com. Follow us at www.twitter.com/JanssenGlobal. Janssen Research & Development, LLC is one of the Janssen Pharmaceutical Companies of Johnson & Johnson.

JDRF‘s mission is to accelerate life-changing breakthroughs to cure, prevent, and treat T1D and its complications. To accomplish this, JDRF has invested more than $2.5 billion in research funding since our inception. We are an organization built on a grassroots model of people connecting in their local communities, collaborating regionally for efficiency and broader fundraising impact and uniting on a national stage to pool resources, passion and energy. We collaborate with academic institutions, policymakers and corporate and industry partners to develop and deliver a pipeline of innovative therapies to people living with T1D. Our staff and volunteers throughout the United States and our five international affiliates are dedicated to advocacy, community engagement and our vision of a world without T1D. For more information, please visit jdrf.org or follow us on Twitter: @JDRF.

Novo Nordisk is a leading global healthcare company, founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat diabetes and other serious chronic diseases such as obesity and rare blood and endocrine disorders. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 47,800 people in 80 countries and markets its products in around 170 countries. Novo Nordisk’s B shares are listed on Nasdaq Copenhagen (Novo-B). Its ADRs are listed on the New York Stock Exchange (NVO). For more information, visit novonordisk.com, Facebook, Twitter, LinkedIn, YouTube.

Provention Bio, Inc. (Nasdaq: PRVB) is a biopharmaceutical company focused on advancing the development of investigational therapies that may intercept and prevent debilitating and life-threatening immune-mediated disease. The Biologics License Application (BLA) for teplizumab, its lead investigational drug candidate, for the delay or prevention of clinical type 1 diabetes in at-risk individuals, has been filed by the U.S. Food and Drug Administration (FDA). The Company’s pipeline includes additional clinical-stage product candidates that have demonstrated in pre-clinical or clinical studies proof-of-mechanism and/or proof-of-concept in other autoimmune diseases, including celiac disease and lupus. Visit www.Proventionbio.com for more information and follow us on Twitter: @ProventionBio.

Contact:

Kissy Black
C-Path
615.310.1894
kblack@c-path.org

C-Path Virtual Workshop to Feature Latest Advances in Clinical Trials for T1D

More than 300 Attendees and 20 Speakers Are Expected to Participate in the International Workshop, June 15-16

TUCSON, Ariz., May 25, 2021 — Critical Path Institute (C-Path) today announced its schedule for the Design of Clinical Trials in New-Onset Type 1 Diabetes: Regulatory Considerations for Drug Development Workshop, to be held virtually June 15-16. Together with the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA), and in collaboration with Benaroya Research Institute, INNODIA and JDRF, this 2-day public workshop will focus on the implementation of endpoints and outcome measures for clinical trials in new onset type 1 diabetes (T1D). More than 300 researchers, pharmaceutical representatives, academia members, investigators, T1D patients and regulatory experts from throughout the United States and Europe are expected to gather virtually to hear 15 presentations about T1D research and medical product development.

“An important element of C-Path’s work is to bring together leading researchers from academia and industry, alongside clinicians and patient advocates, to gather consensus on the latest scientific developments that can improve the lives of members of the T1D community,” said C-Path’s T1D Consortium Executive Director Inish O’Doherty, Ph.D. “By providing a forum for the community and regulators to publicly engage, our workshop seeks to bridge the gap between scientific understanding of outcome measures, such as C-peptide, and their implementation in registration studies for new onset T1D.”

As one main objective of the workshop, attendees can expect discussion on existing evidence regarding the role of C-peptide in clinical trials intended to support regulatory decision making, regulatory considerations from the FDA and EMA, and perspectives from the T1D drug development community.

“This meeting comes at a critical time in the development of disease-modifying therapies to address the significant unmet needs for people with type 1 diabetes,” says JDRF Vice President of Research Sanjoy Dutta, Ph.D. “We are excited to participate in this important discussion and thank the FDA, EMA, meeting organizers and presenters for bringing the community together to accelerate pathways to cures for T1D.”

The conference will include scheduled live-broadcast sessions, live panel discussions and Q&A.

The preliminary agenda is as follows:

  • Welcome and Introductory Remarks
  • Session I: Regulatory Framework for Clinical Investigations in New/Recent Onset T1D
  • Session II: Scientific Framework: The rationale for C-peptide preservation and use as a clinical trial endpoint
  • Session III: Establishing/confirming clinical benefit
  • Session IV: Overall Issues of Study Design: Considerations and panel discussion/open comment

Registration is free and open to the public. For more information and to receive additional updates leading up the event, visit the workshop event page.

Critical Path Institute is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) and is 55% funded by FDA/HHS, totaling $14,575,306, and 45% funded by non-government source(s), totaling $11,916,747. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government. For more information, please visit FDA.gov.


Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona and C-Path, Ltd. EU is headquartered in Dublin, Ireland, with additional staff in multiple other locations. For more information, visit c-path.org and  c-path.eu.

JDRF‘s mission is to accelerate life-changing breakthroughs to cure, prevent and treat T1D and its complications. To accomplish this, JDRF has invested more than $2.5 billion in research funding since our inception. We are an organization built on a grassroots model of people connecting in their local communities, collaborating regionally for efficiency and broader fundraising impact, and uniting on a national stage to pool resources, passion, and energy. We collaborate with academic institutions, policymakers, and corporate and industry partners to develop and deliver a pipeline of innovative therapies to people living with T1D. Our staff and volunteers throughout the United States and our five international affiliates are dedicated to advocacy, community engagement and our vision of a world without T1D. For more information, please visit jdrf.org or follow us on Twitter (@JDRF), Facebook (@myjdrf), and Instagram (@jdrfhq).

Contact:
Kissy Black
C-Path
615.310.1894
kblack@c-path.org

C-Path Receives Letter of Support from EMA on Type 1 Diabetes Biomarker Initiative

 

 

TUCSON, Ariz., April 28, 2020 — The Critical Path Institute (C-Path) today announced that its Type 1 Diabetes (T1D) Consortium has received a letter of support from the European Medicines Agency (EMA) to facilitate the development and validation of the proposed regulatory qualification of pancreatic islet autoantibodies commonly used in clinical practice to diagnose T1D: insulin autoantibodies, glutamic acid decarboxylase 65, and insulinoma antigen-2 autoantibodies as enrichment biomarkers for T1D clinical trials.

In their response to the T1D Consortium Letter of Intent (LOI) and Briefing Package, the EMA stated, “[Therapies that preserve endogenous β-cell function and can prevent, halt or slow T1D disease progression in a clinically meaningful way would constitute a significant advancement in T1D care. If successful, the quantitative tools proposed by this Consortium have the potential to facilitate the streamlined design, execution, and review of clinical trials targeting this goal.].”

By the year 2050, the number of people diagnosed with T1D in the U.S. is projected to quadruple from an estimated 1.6 million in 2020 to 5 million in 2050. The ability to screen for subjects with early stages of T1D prior to the appearance of clinical symptoms is a valuable opportunity to potentially delay, and ultimately prevent, symptomatic T1D. The islet autoantibodies provide a means to identify patients at risk of progressing to a clinical diagnosis of T1D.

C-Path’s T1D Consortium will achieve the regulatory qualification of the islet autoantibodies currently used in clinical practice to diagnose T1D by employing the resources of all its members and engaging with regulatory agencies at each step of the process with funding and input from The Leona M. and Harry B. Helmsley Charitable Trust, Janssen Research & Development, LLC, JDRF International, Novo Nordisk A/S, and Provention Bio.

“JDRF is pleased the T1DC received a letter of support from EMA,” says Jessica Dunne, Ph.D., Senior Director, JDRF and Co-Director of the T1DC. “This is an example of collaboration between regulators and researchers, in the public and private sectors, working together to accelerate delivery of therapies into the hands of patients.”

This model-based qualification, will provide a tool endorsed by both the EMA and U.S. Food and Drug Administration (FDA) that utilizes islet autoantibody status, along with other relevant patient features, to identify and select patients with a likelihood of progressing to a T1D clinical diagnosis. This regulatory endorsement will provide sponsors with the confidence to use islet autoantibodies in the optimization of clinical trials evaluating novel therapies focused on the delay and/or prevent T1D.

“We are delighted that the EMA is strongly supporting the development of quantitative tools that can accelerate drug development in T1D,” said C-Path’s Executive Director of the T1D Consortium Inish O’Doherty, PhD. “This work has been enabled through the collaboration of the T1D community and the sharing of patient level data. We’re excited to move forward with this important project which will help pave the way for forthcoming therapies with the ability to treat early stages of T1D and delay or prevent the clinical, currently irreversible, stage of the disease.”

The consortium is currently working on the next regulatory milestones: the execution of the modeling analysis plan to inform the full Briefing Package for submission to the EMA and development of a Qualification Plan for submission to the FDA.

The Letter of Support can be found on the EMA website here or on the T1D Consortium website here.

 

About the Organizations:

Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path US is headquartered in Tucson, Arizona and C-Path, Ltd. EU is headquartered in Dublin, Ireland, with additional staff in multiple other locations. For more information, visit c-path.org and c-path.eu.

 


 

The Type 1 Diabetes (T1D) Consortium is a public-private partnership initiated in March 2017. Currently membership is composed of the following Industry and Foundation members: The Leona M. and Harry B. Helmsley Charitable Trust; Janssen Research & Development, LLC; JDRF International; Novo Nordisk; and Provention Bio. Other consortium members, participants, and advisors include individuals from the following organizations: Benaroya Research Institute at Virginia Mason; Lund University, Sweden; Helmholtz Zentrum München; University of Bristol; University of Colorado Denver; University of Florida; University of Helsinki; University of Leuven; University of Munich; University of Oulu; University of Tampere; University of Turku; Critical Path Institute; FDA; and NIH.

 


 

The Leona M. and Harry B. Helmsley Charitable Trust aspires to improve lives by supporting exceptional efforts in the U.S. and around the world in health and select place-based initiatives. Since beginning active grantmaking in 2008, Helmsley has committed more than $2 billion for a wide range of charitable purposes. Learn more about Helmsley at helmsleytrust.org.

 


 

About the Janssen Pharmaceutical Companies of Johnson & Johnson. At Janssen, we’re creating a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular & Metabolism, Immunology, Infectious Diseases & Vaccines, Neuroscience, Oncology, and Pulmonary Hypertension.

Learn more at www.janssen.com. Follow us at www.twitter.com/JanssenGlobal. Janssen Research & Development, LLC is one of the Janssen Pharmaceutical Companies of Johnson & Johnson.

 


 

JDRF is the leading global organization funding type 1 diabetes (T1D) research. Our mission is to accelerate life-changing breakthroughs to cure, prevent and treat T1D and its complications. To accomplish this, JDRF has invested more than $2.2 billion in research funding since our inception. We are an organization built on a grassroots model of people connecting in their local communities, collaborating regionally for efficiency and broader fundraising impact, and uniting on a national stage to pool resources, passion, and energy. We collaborate with academic institutions, policymakers, and corporate and industry partners to develop and deliver a pipeline of innovative therapies to people living with T1D. Our staff and volunteers throughout the United States and our six international affiliates are dedicated to advocacy, community engagement and our vision of a world without T1D. For more information, please visit jdrf.org or follow us on Twitter: @JDRF.

 


 

Novo Nordisk is a global healthcare company with more than 95 years of innovation and leadership in diabetes care. This heritage has given us experience and capabilities that also enable us to help people defeat obesity, haemophilia, growth disorders and other serious chronic diseases. Headquartered in Denmark, Novo Nordisk employs approximately 42,700 people in 80 countries and markets its products in around 170 countries. For more information, visit novonordisk.com, Facebook, Twitter, LinkedIn, YouTube.

 


 

Provention Bio, Inc. (Nasdaq: PRVB) is a clinical-stage biopharmaceutical company leveraging a transformational drug development strategy that is focused on the prevention or interception of immune-mediated disease. Provention’s mission is to in-license, transform and develop therapeutic candidates targeting the high morbidity, mortality and escalating costs of autoimmune and inflammatory diseases including: type 1 diabetes (T1D), celiac disease and lupus. Provention’s diversified portfolio includes advanced-stage product development candidates that have undergone clinical testing by other companies. For more information, please visit proventionbio.com.

 


 

Benaroya Research Institute at Virginia Mason (BRI) is a world-renowned, non-profit research institute committed to advancing the science that will predict, prevent, reverse and cure diseases of the immune system. BRI researchers uniquely study the immune system in both health and disease, with the ultimate goal of achieving a healthy immune system for each individual. Diseases we study include type 1 diabetes, rheumatoid arthritis, lupus, multiple sclerosis, Crohn’s and colitis as well as allergies and cancer. BRI accelerates discovery through laboratory breakthroughs in immunology that can be translated to clinical therapies. A leader of collaborative initiatives such as the Immune Tolerance Network and Type 1 Diabetes TrialNet, BRI frequently partners with global research institutes, pharmaceutical and biotech companies. Visit BenaroyaResearch.org or follow us on the Autoimmune Life blog, Facebook, Instagram, YouTube, LinkedIn or Twitter.

 

C-Path Contact:
Kissy Black
C-Path
615.310.1894
kblack@c-path.org