C-Path and Diamyd Medical Announce Data Sharing Collaboration to Develop Advanced Drug Development Tools in Type 1 Diabetes

 

TUCSON, Ariz., and STOCKHOLM, Sweden. December 16, 2020 — The Critical Path Institute (C-Path) and Diamyd Medical (DMYD B; Nasdaq First North Growth Market) are proud to announce their collaboration to significantly improve the scientific community’s insight into type 1 diabetes (T1D) through Diamyd Medical’s contribution of fully anonymized data from a European Phase III trial to the Trial Outcome Markers Initiative (TOMI-T1D) integrated database. The Phase III trial evaluated the use of the diabetes vaccine Diamyd®, an antigen-specific immunotherapy based on the auto-antigen GAD (glutamic acid decarboxylase), to induce immunological tolerance and stop the autoimmune destruction of insulin producing cells. The Data Contribution Agreement (DCA) between Diamyd Medical and C-Path will allow for this unique set of fully anonymized clinical trial data to be integrated into an ever-growing list of committed trial data sets within the TOMI-T1D project.

TOMI-T1D is an international partnership between academia, the pharmaceutical industry and nonprofit organizations. It is funded by the world’s leading charities dedicated to diabetes research, JDRF, and Diabetes UK, guided by both organizations’ strong commitment to facilitate deep interrogation of consolidated community-wide trial data as a means to accelerate clinical research and therapeutic development for T1D. TOMI-T1D aims to create a clinical trial simulation tool (CTST) with large and diverse clinical datasets from the T1D community. The project also seeks to engage the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) to identify opportunities for regulatory endorsement of such drug development tools.

The Diamyd Medical data includes relevant information about disease progression, drug effects and clinical trial design. Contribution of these robust data sets from industry led trials is critical to TOMI-T1D’s work in developing innovative and quantitative tools that can facilitate clinical development efforts and be endorsed by regulators for future use by the pharmaceutical industry to optimize the design of future clinical trials.

“Progress towards the establishment of approved therapies for people with T1D is critically reliant on participation from our partners in industry with their data,” said Simi Ahmed and Elizabeth Robertson, on behalf of the charity partnership. “This is indeed a right step in that direction,” said Colin Dayan, lead PI at Cardiff University.

“We are thrilled that Diamyd Medical is taking a leading role and championing precompetitive collaborations advancing type 1 diabetes regulatory science solutions,” said C-Path Executive Director Inish O’Doherty, Ph.D. “Their data will help in the construction and evaluation of a clinical trial simulation tool to assist in the development of novel therapies for type 1 diabetes patients.”

“We are very honored to be part of this important collaboration -involving key stakeholders within the T1D landscape,” said Ulf Hannelius, President & CEO of Diamyd Medical. “As we are moving into an era of precision medicine in type 1 diabetes, we can expect to see significant therapeutic advances, and access to high quality data will be integral to maximizing these efforts.”

To learn more about the TOMI-T1D project visit: https://c-path.org/programs/tomi-t1d/

TOMI-T1D is solely funded by JDRF and Diabetes UK. Critical Path Institute is supported by the U.S. Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) and is 69% funded by FDA/HHS, totaling $19,471,171, and 31% funded by non-government source(s), totaling $8,612,313. 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.


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.


 

Diamyd Medical develops therapies for type 1 diabetes. The diabetes vaccine Diamyd® is an antigen-specific immunotherapy for the preservation of endogenous insulin production. Significant results have been shown in a genetically predefined patient group in a large-scale metastudy as well as in the Company’s European Phase IIb trial DIAGNODE-2, where the diabetes vaccine is administered directly into a lymph node in children and young adults with newly diagnosed type 1 diabetes. A new facility for vaccine manufacturing is being set up in Umeå for the manufacture of recombinant GAD65, the active ingredient in the therapeutic diabetes vaccine Diamyd®. Diamyd Medical also develops the GABA-based investigational drug Remygen® as a therapy for regeneration of endogenous insulin production and to improve hormonal response to hypoglycaemia. An investigator-initiated Remygen® trial in patients living with type 1 diabetes for more than five years is ongoing at Uppsala University Hospital. Diamyd Medical is one of the major shareholders in the stem cell company NextCell Pharma AB. Diamyd Medical’s B-share is traded on Nasdaq First North Growth Market under the ticker DMYD B. FNCA Sweden AB is the Company’s Certified Adviser; phone: +46 8-528 00 399, e-mail: info@fnca.se.


 

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.


 

1.   Diabetes UK’s UK’s aim is creating a world where diabetes can do no harm. Diabetes is the most devastating and fastest growing health crisis of our time, affecting more people than any other serious health condition in the UK – more than dementia and cancer combined. There is currently no known cure for any type of diabetes. With the right treatment, knowledge and support people living with diabetes can lead a long, full and healthy life. For more information about diabetes and the charity’s work, visit www.diabetes.org.uk.
2.   Diabetes is a condition where there is too much glucose in the blood because the body cannot use it properly. If not managed well, both type 1 and type 2 diabetes can lead to devastating complications. Diabetes is one of the leading causes of preventable sight loss in people of working age in the UK and is a major cause of lower limb amputation, kidney failure and stroke.
3.   People with type 1 diabetes cannot produce insulin. About 10 per cent of people with diabetes have type 1. No one knows exactly what causes it, but it’s not to do with being overweight and it isn’t currently preventable. It’s the most common type of diabetes in children and young adults, starting suddenly and getting worse quickly. Type 1 diabetes is treated by daily insulin doses – taken either by injections or via an insulin pump. It is also recommended to follow a healthy diet and take regular physical activity.
4.   People with type 2 diabetes don’t produce enough insulin or the insulin they produce doesn’t work properly (known as insulin resistance). Around 90 per cent of people with diabetes have type 2. They might get type 2 diabetes because of their family history, age and ethnic background puts them at increased risk. They are also more likely to get type 2 diabetes if they are overweight. It starts gradually, usually later in life, and it can be years before they realise they have it. Type 2 diabetes is treated with a healthy diet and increased physical activity. In addition, tablets and/or insulin can be required.

For more information on reporting on diabetes, download our journalists’ guide: Diabetes in the News: A Guide for Journalists on Reporting on Diabetes (PDF, 3MB).

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

 

C-Path Selects Aridhia to Support Rare Disease Cures Accelerator-Data and Analytics Platform

 

TUCSON, Ariz., December 3, 2020 — The Critical Path Institute (C-Path) today announced it has selected Aridhia to support its Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP). The RDCA-DAP initiative, launched in September 2019 as a collaborative project between C-Path and the National Organization for Rare Disorders® (NORD), will provide a centralized and standardized infrastructure to accelerate and optimize the quantitative characterization of rare diseases, with the goal of accelerating therapy development. RDCA-DAP is designed to accept patient-level rare disease data from clinical trials, observational studies, real-world data, patient registries and other sources, to support the analysis and interpretation of those data.

Based in Scotland, Aridhia encompasses a multidisciplinary team of data scientists, information governance specialists, computer scientists, software developers and health care experts who are uniquely skilled to give researchers and innovators the ability to discover and understand data through dataset searching, classification and efficient metadata browsing capabilities. Aridhia will work closely with C-Path’s Data Collaboration Center (DCC) team of top-tier technical, scientific and project management experts to build a neutral, precompetitive data collaboration environment to support advanced research efforts in rare diseases.

“We look forward to working with Aridhia on this exciting collaboration,” said C-Path Executive Director of Data Science Amanda Borens, MS. “RDCA-DAP is an important resource for the entire rare disease community. Using Aridhia technology, RDCA-DAP will help the community come together and share data and knowledge that will revolutionize the development of therapies for rare diseases.”

Aridhia was chosen by C-Path based on its unparalleled expertise in providing secure cloud-enabled digital research environments for biomedical, precision medicine and health care researchers. Coupled with the fact that Aridhia has been on the forefront of the fair, lawful and transparent handling of personal data — prior to the implementation of the EU’s General Data Protection Regulations (GDPR) — and have continued to be an industry leader in the strict adherence to GDPR standards, Aridhia was the stand-out choice to assist with the development of the RDCA-DAP.

“We’re delighted to be working with colleagues at C-Path on such an important area of biomedical research,” said Aridhia CEO David Sibbald. “We know that improving therapy development for rare disease requires significant data discovery and data access, while preserving the governance requirements of individual data contributors. Our collaboration will help the rare disease scientific community gain access to richer, more comprehensive datasets to accelerate rare disease therapy development.”

RDCA-DAP already includes integrated rare disease data from a number of different sources — including those within NORD’s IAMRARE™ registry platform — and building in advanced analytics capabilities will allow efficient and effective interrogation of that data to generate solutions for clinical trial design and regulatory review.

Groups interested in contributing data to the effort, collaborating on rare disease research or using the platform for their own research may visit c-path.org/rdca-dap or email rdcadap@c-path.org for more information. The platform is open to accept data immediately and the user interface and analytics will be available in early 2021, with additional analytics tools being developed concurrently.

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 69% funded by FDA/HHS, totaling $19,471,171, and 31% percent funded by non-government source(s), totaling $8,612,313. 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.

 


 

About Critical Path Institute
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.

 

About Aridhia
Aridhia is a health data science company founded in 2008 and based in Glasgow’s Queen Elizabeth University hospital and central Edinburgh. Aridhia provides a cloud based digital research platform enabling both the discovery and analysis of data to take place in a trusted, secure environment at scale. Our multidisciplinary team of data scientists, information governance specialists, computer scientists, software developers and health care experts delivers research services across the UK and Europe, working collaboratively with the NHS, research organizations, life sciences companies, IT service providers and patients to transform clinical research into clinical practice.

 

Contact:

Kissy Black
C-Path
kissyblack@c-path.org

C-Path, Ltd. Announces New Contract with Innovative Medicines Initiative

 

DUBLIN, Ireland, December 3, 2020 — Critical Path Institute, Ltd. (C-Path, Ltd.) announced today a new contract with the Innovative Medicines Initiative 2 Joint Undertaking (IMI2 JU). The new project will leverage C-Path’s global expertise in developing novel product development tools.

“The mission of IMI, to improve health by speeding up the development of, and patient access to, innovative medicines, is carried out through collaborative projects, and each project has taken large steps forward towards reaching this goal,” said Pierre Meulien, IMI2 JU’s Executive Director. “IMI’s new contract with C-Path, Ltd. will identify opportunities to expand the impact of these projects for patients across Europe.”

Novel methodologies, including biomarkers, clinical outcome assessments and disease progression models, are tools that inform regulatory decision-making during the product development process. These tools can decrease clinical trial size and/or duration, inform endpoint selection, increase the patient voice, improve safety monitoring and more, depending on the tool. As a result, product developers have a more efficient process, bringing therapies to patients faster.

“C-Path’s expertise lies beyond leading collaborations that gather consensus among its member organizations,” said Acting Chief Executive of C-Path, Ltd. Graham Higson, M.Sc. “We’ve been uniquely successful in using these collaborations to identify key product development needs, assessing available data, then using that data to develop novel drug development tools.”

Often, these tools can be submitted for endorsement at the regulatory agencies, such as the European Medicines Agency. By doing so, product developers have increased confidence in the tool and are more likely to incorporate it into their development programs. Regulatory endorsed tools are also publicly available on agency websites.


Critical Path Institute, Ltd. (C-Path, Ltd.) is a wholly owned subsidiary of Critical Path Institute (C-Path), 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, Ltd. EU is headquartered in Dublin, Ireland, and C-Path US is headquartered in Tucson, Arizona, with additional staff in multiple other locations. For more information, visit www.c-path.org and  c-path.eu.

 

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

Alzheimer’s Disease Data Initiative Launches, Critical Path for Alzheimer’s Disease Consortium to Partner

 

CPAD will partner with ADDI to build better tools for researchers around the world to access, use and share dementia-related data to accelerate the development of new and more effective treatments for Alzheimer’s disease

TUCSON, Ariz., November 17, 2020 — The Critical Path Institute’s (C-Path) Critical Path for Alzheimer’s Disease (CPAD) Consortium today announced its participation as a Partner with the Alzheimer’s Disease Data Initiative (ADDI), a 501(c)(3) medical research organization which launched today. ADDI is dedicated to advancing scientific breakthroughs in the treatment of Alzheimer’s disease (AD) and related dementias.

“ADDI will be a game-changing collaboration to provide the necessary data accessibility to advance data-driven solutions to accelerate Alzheimer’s disease drug development,” said C-Path’s Chief Science Officer Klaus Romero, M.D., M.S., F.C.P. “C-Path is honored to be a key Partner with ADDI, through CPAD, and contribute our data science, model-informed drug development and regulatory science expertise to ensure the success of ADDI.”

CPAD’s integrated and standardized patient-level database comprised of randomized clinical trials from industry, which is currently available to qualified researchers across the globe, will be a relevant and crucial component of ADDI’s Alzheimer’s Disease Workbench. This will enable researchers in AD and related dementias around the world to interact with relevant data resources more efficiently and accelerate the generation of actionable scientific insights leading to innovative solutions, diagnostic tools and therapies in AD.

Emphasizing the expected impact of ADDI, CPAD’s Executive Director Sudhir Sivakumaran, Ph.D., said, “ADDI is uniquely positioned to maximize the value of each data point contributed by individuals living with AD, who participate in clinical trials, observational studies, patient registries and other research efforts.”

CPAD’s Industry Co-Director and Vice President, Clinical Research, Neurology Business Group, Eisai, Inc. Michael C. Irizarry, M.D., M.P.H., added, “In this important initiative, the CPAD partnership with ADDI aims to facilitate increased aggregation and robust analysis of high-quality AD clinical research data, to model disease progression and help accelerate the development of biomarkers and therapeutics.”

For more information, visit: https://c-path.org/programs/cpad/; https://codr.c-path.org/main/applyDatabaseSelection.html; https://www.alzheimersdata.org/; https://www.alzheimersdata.org/ad-workbench.

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 62% funded by FDA/HHS, totaling $14,448,917, and 38% percent funded by non-government source(s), totaling $8,669,646. 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.

 


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 www.c-path.org and  c-path.eu.

 

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

Rare Disease Cures Accelerator-Data and Analytics Platform First Year Milestones Highlighted at Virtual Workshop

 

 

C-Path, NORD, FDA and more come together to showcase the importance of data sharing in rare disease drug development

 

TUCSON, Ariz. and WASHINGTON, D.C. October 21, 2020 — The Critical Path Institute (C-Path) and the National Organization for Rare Disorders (NORD®) hosted the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) 2020 Virtual Workshop on Monday, October 19. The platform, funded by a cooperative agreement through the U.S. Food and Drug Administration (FDA), serves as a centralized and standardized infrastructure to support data sharing and data analysis to aid in the understanding of rare diseases.

Theresa Mullin, Ph.D., Associate Director for Strategic Initiatives, FDA, Center for Drug Evaluation and Research (CDER) opened the workshop and explained how RDCA-DAP is part of the FDA’s aggressive plan to advance drug development for rare diseases. “The concept of RDCA-DAP has been discussed by C-Path and NORD with hundreds of stakeholders through public meetings, webinars, focus groups and one on one interviews to understand what the community would like to see in an analytics platform,” Mullin said. “These learnings are being used now to make the platform as useful as possible to as many people as possible.”

The virtual workshop, attended by more than 400 individuals from patient groups, academia, industry and regulatory agencies informed the rare disease community of the initiative’s first-year progress. Topics included pilot analyses and the development of tools facilitated by RDCA-DAP to advance characterization and accelerate drug development in rare diseases. Additional updates were provided on the platform’s data sharing infrastructure and recent enhancements, including the ability to search, sort and analyze data.

C-Path RDCA-DAP Executive Director Jane Larkindale, D.Phil., detailed milestones the effort achieved in its first year and outlined plans for the future. In year one, the RDCA-DAP team worked with the community to develop a data governance structure that answers the needs of both data contributors and data users, accessed initial datasets and built actionable prototypes to demonstrate the utility of the platform.

“Our priority is not to compete, but to collaborate with other groups doing the same or similar work in rare disease drug development,” said Larkindale. “Stakeholders are open to collaboration and data sharing and we have been meeting with different groups about what is already being done and how we can interoperate with other existing projects.”

Several case studies were presented during the workshop to demonstrate the power of the platform including a demonstration of the value of the integration of data and analysis of combined datasets. In one case, Pravin Jadhav, Ph.D., global team leader at Otsuka America Pharmaceutical, Inc. shared how patient-level data aided in the acceptance of a biomarker for an accelerated approval of a new therapy for polycystic kidney disease.

C-Path’s Chief Science Officer Klaus Romero, M.D., M.S., F.C.P. shared examples of prototypes built from RDCA-DAP patient-level data that demonstrated the ability to integrate data from multiple disease areas to generate advanced models of disease progression and to optimize the characterization of rare diseases, as a means to accelerate drug development for these conditions.

Similarly, NORD’s Director of Research Vanessa Boulanger, M.Sc. used the example of the NPKUA phenylketonuria patient registry from NORD’s IAMRARE™ platform to show how patient registry data can inform drug development programs.

“The PKU case study is one example of the insights that patient registry and natural history study data can bring to RDCA-DAP,” said Boulanger. “Patient-driven data gives us an opportunity to arrive at a more comprehensive understanding of rare diseases, to learn what matters to patients, and then to apply that knowledge to the design of studies that may result in treatments that are truly meaningful to the community.”

RDCA-DAP is positioned to generate solutions to drug development bottlenecks by maximizing the utility of patient-level data and making that data available to a larger community. C-Path President and CEO Joseph Scheeren, Pharm.D. and NORD President and CEO Peter L. Saltonstall both thanked the community for coming together around the concept of RDCA-DAP, acknowledging that data contribution is key to its success. FDA/CDER Office of Neuroscience Acting Director Billy Dunn, M.D. closed the workshop congratulating the teams on the work accomplished since RDCA-DAP’s launch in September 2019 and discussing the three main areas of the FDA’s rare disease drug development efforts, including RDCA-DAP, standard core clinical outcome assessments and clinical trials network.

“The reason RDCA-DAP is so critical, is that it focuses on the characterization of the disease and if we don’t have a good characterization of the disease, we really can’t do our job,” Dunn said. “Natural history, the variability of symptoms and experiences and the opportunities for clinical trial design are all addressed by a more robust characterization of disease.”

Groups interested in contributing data to the effort, collaborating on the development of the platform or using the database may visit c-path.org/rdca-dap or email rdcadap@c-path.org for more information. The platform is open to accept data immediately, but the user interface and analytics will be available in early 2021, with additional analytics tools being developed concurrently.

A full recording of the workshop can be accessed here.

*Funding for this program was made possible, in part, by the Food and Drug Administration through grant (U18 FD 005320). Views expressed in written materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services; nor does any mention of trade names, commercial practices, or organization imply endorsement by the United States Government.

 


 

About Critical Path Institute
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.

 

About the National Organization for Rare Disorders (NORD)®
The National Organization for Rare Disorders (NORD®) is the leading independent advocacy organization representing all patients and families affected by rare diseases. NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. Since then, NORD has led the way in voicing the needs of the rare disease community, driving supportive policies and education, advancing medical research and providing patient and family services for those who need them most. NORD is made strong together with over 325 disease-specific member organizations and their communities and collaborates with many other organizations on specific causes of importance to the rare disease patient community. Visit rarediseases.org.

 

Media Contacts:

Kissy Black
C-Path
615.310.1894
kissyblack@lotosnile.com

Laura Mullen
NORD
203.304.7258
lmullen@rarediseases.org

C-Path Data Collaboration Center Analytics Team Wins Metadata Automation DREAM Challenge

TUCSON, Ariz., July 14, 2020 — Tucson-based Critical Path Institute (C-Path) today proudly announced that the analytics team from its Data Collaboration Center (DCC) program has won first place in the validation phase of the Metadata Automation DREAM Challenge, funded by the Cancer Moonshot℠ initiative. The mission of C-Path’s DCC program is to provide large-scale data collaboration solutions to support scientific research and advance medical innovation.

Designed and run by a community of researchers from a variety of organizations, DREAM (Dialogue for Reverse Engineering Assessments and Methods) Challenges invite participants to propose solutions to fundamental biomedical questions — fostering collaboration and building communities in the process. The Metadata Automation DREAM Challenge focused on oncology with the goal to have participants develop tools to automate annotation of metadata fields and values, using available research data annotations, as well as established terminologies and ontologies.

“This kind of leading edge metadata annotation and ontology integration is a new focus for DCC that will help expedite C-Path’s data and analytics efforts, including that of C-Path’s Rare Disease Cures Accelerator-Data and Analytics Platform or RDCA-DAP,” said C-Path Chief Science Officer, Klaus Romero, M.D., M.S., F.C.P. “To win among such a competitive and prestigious list of teams validates the DCC skills; skills that will be necessary for innovating our data management practices, and strengthening the FAIR Principles of data at C-Path.”

Ten teams participated in the Challenge. The teams received annotated clinical data files from The Cancer Imaging Archive (TCIA), and synthetic data from Applied Proteogenomics Organizational Learning and Outcomes (APOLLO), which they used to develop automated tools that can operate on input data sources. The DREAM C-Path Analytics team included:

Roopal Bhatnagar, M.S.
Data Analyst
Emily Hartley, B.S.
Data Analyst
Kurt Michels, Ph.D.
Data Analyst

“We are very proud of our DREAM team,” said C-Path President and CEO, Joseph Scheeren, Pharm.D. “Not only did the team demonstrate strong credentials in oncology metadata, they also developed techniques to annotate data from any disease area making this work extremely valuable to C-Path’s consortia and programs focused on multiple disease areas.”

The C-Path Analytics Team, along with other winning teams, will move on to the collaboration phase of the challenge to improve on their models. The end result is aimed at significantly lowering the burden of adding these annotations across the data ecosystem, to streamline and enable both retrospective harmonization as well as data query, discovery and interpretation.

“We are looking forward to work and shared learning with other teams in the collaborative phase of the Challenge,” said C-Path Executive Director of Data Science Amanda Borens, M.S. “Our next project internally will apply these algorithm designs with new ontologies and standards for rare diseases. The DREAM Challenge has given us a wonderful proof of concept and confidence to proceed with experience under our belts.”

Sage Bionetworks provides the expertise and infrastructure to host challenges via their Synapse platform. Work by Sage Bionetworks staff for the Challenge is supported by the NCI Cancer Systems Biology Consortium (CSBC) and Physical Sciences-Oncology Network (PS-ON).

 


 

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 www.c-path.org and  c-path.eu.

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

C-Path Launches CURE Drug Repurposing Collaboratory to Accelerate Identification of New Uses of Existing Drugs to Treat Infectious Diseases, Including COVID-19

Clinicians to report novel uses of existing drugs through FDA-NCATS CURE ID Mobile App.

TUCSON, Ariz., June 23, 2020 — As millions of patients struggle with diseases that lack adequate treatments, there is a critical need to understand how existing drugs can be used in new ways to improve clinical outcomes. Health care professionals use drugs in novel ways as a potential life-saving intervention when no specific approved therapies are available. However, without the ability to share these experiences in a systematic manner, the clinical and research communities cannot benefit from lessons learned.

To address the challenge, the Critical Path Institute (C-Path) today announced the launch of the CURE Drug Repurposing Collaboratory (CDRC) funded by the U.S. Food and Drug Administration (FDA), in collaboration with the National Center for Advancing Translational Sciences (NCATS), part of the National Institutes of Health (NIH). A public-private partnership, CDRC will provide a forum for the exchange of clinical practice data to inform potential new uses of existing drugs for areas of high unmet medical need, advancing research in these areas. The Collaboratory will also create a network connecting major treatment centers, academic institutions and researchers, private practitioners, government facilities and health care professionals around the world.

CDRC will focus on capturing relevant real-world clinical outcome data through the FDA-NCATS CURE ID platform. The objective is to accelerate the identification and development of potentially effective drugs for patients with diseases that lack adequate approved treatment options with the goal of helping identify drug candidates for additional study and potentially drug labeling in the future. Available on the web and as a mobile app, CURE ID serves as a centralized source of reliable, curated, clinician-submitted information.

“The CURE ID platform enables clinicians to provide data to a crowdsourced central repository where aggregated clinician experiences can be shared with the global scientific community to help drive innovation,” said NCATS Director Christopher P. Austin, M.D.

In a pilot project focused on COVID-19, CDRC will use data collected via the CURE ID platform to aggregate global clinician treatment experiences to identify existing drugs that demonstrate possible treatment approaches that should be studied further in randomized trials. Critical updates have been made to the CURE ID case report form for capturing relevant details related to COVID-19.

“This initiative led by C-Path, in partnership with multiple divisions and offices within the FDA as well as NCATS/NIH, will help address the scientific and regulatory challenges for drug repurposing,” said Amy Abernethy, M.D., Ph.D., FDA principal deputy commissioner. “For COVID-19 patients, time is of the essence and the contribution of cases reported directly by health care providers, followed by rapid analysis of data from the CURE ID platform, provides a much-needed accelerated strategy to generate hypotheses about the potential safety and efficacy of existing drugs and inform subsequent clinical trials.”

“While participation of patients in randomized clinical trials of potential COVID treatments is ideal, we recognize that many individuals with COVID-19 are unable to participate in trials,” said infectious disease physician and FDA Associate Director for Clinical Methodology in the Office of Medical Policy Leonard Sacks, M.D. “Broad data sharing of treatment successes and failures will provide information that can be used to inform new trials to find safe and effective therapies for COVID-19.”

The effort will rely on FDA’s experience and knowledge in the regulation of drugs and C-Path’s vast experience in regulatory innovation, as well as its proven model of creating successful and productive collaborations. C-Path Scientific Director Marco Schito, Ph.D., will serve as the Executive Director for the CDRC.

“We are encouraging health care professionals, researchers, clinicians and prescribers to download CURE ID and submit data regularly,” said C-Path President and CEO Joseph Scheeren, Pharm.D. “Clearly this is of immediate importance for global public health and we applaud the FDA and NCATS for developing the CURE ID app, which may accelerate the identification of treatments for COVID-19 and other diseases that can be further studied in randomized controlled trials.”

Visit https://c-path.org/cdrc for more information. To date, more than 5,500 health care professionals have registered on the CURE ID platform. Health care professionals who wish to download and use the CURE ID application should visit https://cure.ncats.io or download “CURE ID” from the App or Play Store. Institutions, organizations, and individuals interested in becoming CURE Drug Repurposing Collaboratory partners should email: CDRC@c-path.org.

The CURE Drug Repurposing Collaboratory is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) as part of a financial assistance award 2U18FD005320 totaling $1,103,978 funded 100% by FDA/HHS. 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.

 

Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005. 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 www.c-path.org and  c-path.eu.

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

C-Path’s Transplant Therapeutics Consortium Receives Acceptance of Letter of Intent for iBox Scoring System (Composite Biomarker Panel) as a Reasonably Likely Surrogate Endpoint

 

Biomarker aims to streamline the development of novel therapies intended to improve long-term outcomes for kidney transplant recipients.

 

TUCSON, Ariz., June 17, 2020 — Critical Path Institute (C-Path) announced today that its Transplant Therapeutics Consortium (TTC) has received a positive response to its Letter of Intent (LOI) from the U.S. Food and Drug Administration (FDA) detailing the decision to accept the Composite Biomarker Panel (iBox Scoring System) into the Center for Drug Evaluation and Research (CDER) Biomarker Qualification Program (BQP).

In its LOI, TTC provided information to support the qualification of the iBox Scoring System for its proposed context of use (COU) as a reasonably likely surrogate endpoint in clinical trials intended to evaluate immunosuppressive therapies (ISTs) for individuals living with a kidney transplant. Qualification as a surrogate or reasonably likely surrogate endpoint would allow drug sponsors to pursue accelerated approval, removing a significant barrier to kidney transplant drug development.

FDA indicated in its LOI Decision Letter that it supports the consortium’s intent to pursue biomarker qualification and invited TTC to submit a Qualification Plan, the stage two submission that details how the consortium’s will demonstrate the clinical and analytical validity of the Composite Biomarker Panel, for its intended COU. In its Decision Letter, FDA stated, “Based on our review of the LOI, we agree there is an unmet need, and the development of this composite scoring system to predict patient’s long-term outcomes in clinical trials will facilitate the development of novel immunosuppressive therapies.”

Long-term graft failure rates after kidney transplantation remain unacceptably high, despite improved short-term outcomes, with 10-year all-cause graft failure approaching 50% (Hart et al., 2019). Survival of the transplanted organ has been rated, by patients, as the most important outcome, including the overall survival of the patient (Howell et al., 2012). The iBox Scoring System, developed by the Paris Transplant Group, is the first tool of its kind to seek regulatory qualification for use in kidney transplant clinical trials. To date, no biomarkers have been qualified for use as a surrogate or reasonably likely surrogate endpoint in any therapeutic area.

“This project is a meaningful example of what can be achieved by public-private-partnerships at the interface of translation science,” said TTC Executive Director Inish O’Doherty, Ph.D. Through collaboration and data sharing in the pre-competitive space, we are elevating the entire field of drug development in transplantation and working to improve the lives of those living with a kidney transplant.”

The iBox Scoring System is a risk prediction tool that combines measurements of kidney function, immunological status, and pathological assessment of kidney biopsy histology to predict the risk of graft-loss up to seven years after the time of risk assessment. The iBox Scoring System has been extensively validated for use in the treatment of individual patients in the clinical care setting. TTC, in close collaboration with the Paris Transplant Group, is seeking to translate this work into the regulatory setting for use in drug development programs. If qualified as a reasonably likely surrogate endpoint, the Composite Biomarker Panel (iBox Scoring System) will be publicly available and facilitate any drug sponsor seeking accelerated approval of novel agents, significantly reducing the time required to bring a new therapeutic agent to patients.

The iBox Scoring System will allow for greater application in drug development and lead to better therapies for patients,” said Alexandre Loupy, M.D., Ph.D., head of the National Institute of Medical Research (Inserm) U970. Loupy is a professor of Nephrology at Necker Hospital in Paris and a founder of the Paris Transplant Group. He has led the development of the iBox Scoring System over the past 10 years.

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, healthcare providers, academic researchers, and patient advocacy organizations are encouraged to work together to foster innovation in drug development through drug development tools that facilitate patient access to life-saving medications.

“Long-term transplant outcomes are still suboptimal, and people living with a kidney transplantation need novel anti-rejection medications with better long-term graft survival,” said Mark Stegall, M.D., Clinician Investigator, Department of Surgery, Mayo Clinic. “Improving long-term kidney graft survival would mean patients are less likely to need a second transplant during their lifetime and would allow them to lead longer and healthier lives. The positive response letter from FDA takes us one step closer to having new therapies that better the lives of transplant recipients.”

Ken Newell, M.D., Ph.D., past-president of AST and co-chair for TTC’s Endpoints and Efficacy Working Group said, “TTC has been instrumental in bringing together partners from industry, academia, and professional societies for this groundbreaking endeavor. This effort is a labor of love, and I’m honored to be part of this consortium and to be working with colleagues across the globe with a shared mission.”

 

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 www.c-path.org and  c-path.eu.

 


 

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. The society is the largest transplant organization in North America (consisting of more than 4,000 professional members) and is recognized as the premier society for transplantation. AST members are sought out as transplant experts and advocates. Other transplant organizations, policy makers, regulatory agencies, payors, academic institutions, and the general public look to the AST for guidance, research, and resources related to transplantation.
To learn more, visit: www.myast.org.
Follow us: Twitter @AST_info and
Facebook www.facebook.com/AmericanSocietyofTransplantation

 


 

The American Society of Transplant Surgeons represents approximately 1,900 professionals dedicated to excellence in transplantation surgery. ASTS advances the art and science of transplant surgery through patient care, research, education, and advocacy. To learn more, visit ASTS.org.

 


 

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 communication, continuing education of its membership 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 6500 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 nine Sections specializing in various areas of transplantation: cell and regenerative medicine, Vascularized Composite Allotransplantation (VCA), pancreas and islet transplant, pediatric, organ donation and procurement, intestinal rehabilitation and transplant, xenotransplantation, pediatric liver transplantation, and transplant infectious disease. TTS also partners with 35 national and regional transplant societies to bring the science and clinical practice of transplantation around the world.

 


 

Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram. Celgene and Juno Therapeutics are wholly owned subsidiaries of Bristol-Myers Squibb Company. In certain countries outside the U.S., due to local laws, Celgene and Juno Therapeutics are referred to as, Celgene, a Bristol Myers Squibb company and Juno Therapeutics, a Bristol Myers Squibb company.

 


 

CareDx, Inc., headquartered in South San Francisco, California, is a leading precision medicine solutions company focused on the discovery, development and commercialization of clinically differentiated, high-value healthcare solutions for transplant patients and caregivers. CareDx offers testing services, products, and digital healthcare solutions along the pre- and post-transplant patient journey, and is the leading provider of genomics-based information for transplant patients. For more information, please visit: www.CareDx.com.

 


 

CSL Behring is a global biotherapeutics leader driven by its promise to save lives. Focused on serving patients’ needs by using the latest technologies, we develop and deliver innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, respiratory disease, and neurological disorders. The company’s products are also used in cardiac surgery, burn treatment and to prevent hemolytic disease of the newborn.

CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. The parent company, CSL Limited (ASX:CSL;USOTC:CSLLY), headquartered in Melbourne, Australia, employs more than 25,000 people, and delivers its life-saving therapies to people in more than 70 countries. For inspiring stories about the promise of biotechnology, visit Vita CSLBehring.com/vita and follow us on Twitter.com/CSLBehring.

 


 

Hansa Biopharma is leveraging its proprietary enzyme technology platform to develop immunomodulatory treatments for enabling transplantations and rare immunoglobulin G (IgG)-mediated autoimmune conditions, transplant rejection and cancer.

The Company’s lead product candidate, imlifidase, is an antibody cleaving enzyme being developed to enable kidney transplantation in highly sensitized patients and may be further developed for use in other organ and tissue transplantation as well as acute autoimmune indications. Imlifidase is currently under review for a potential marketing authorization by the European Medicines Agency (EMA).

Hansa’s research and development program is advancing the Company’s enzyme technology to develop the next generation of IgG-cleaving enzymes with potentially lower immunogenicity, suitable for repeat dosing in relapsing autoimmune diseases and oncology. Hansa Biopharma is based in Lund, Sweden and also has operations in other European countries and in the U.S.

 


 

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. 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 is nationally centralized to HUS.

HUS is the biggest 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 293 kidney transplantations and 39 pancreas transplantations performed in 2019.

 


 

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 109,000 people of more than 145 nationalities work at Novartis around the world. Find out more at https://www.novartis.com.

 


 

Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.

With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.
Sanofi, Empowering Life.

 


 

 


 

Transplant Genomics, Inc. (“TGI”) 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 and within the Eurofins family, TGI is commercializing a suite of tests enabling diagnoses and prediction of transplant recipient immune status. Our flagship product is TruGraf, the only blood test approved by CMS for surveillance and to rule out “silent” subclinical acute rejection in kidney transplant recipients with stable graft function. Test services are offered through TGI’s CLIA laboratory in Fremont, CA. TGI was acquired by Eurofins in 2019.

 


 

Veloxis Pharmaceuticals A/S, an Asahi Kasei company, is a commercial-stage specialty pharmaceutical company committed to improving the lives of transplant patients. Veloxis Pharmaceuticals A/S operates in the U.S. through Veloxis Pharmaceuticals, Inc., a wholly owned subsidiary headquartered in Cary, North Carolina, USA. Veloxis is focused on the direct commercialization of immunosuppression medications in the US, expansion of partnerships for markets around the world, and acquisition of assets utilized in transplant patients and by adjacent medical specialties. For further information, please visit www.veloxis.com.

 

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

 

C-Path and Provention Bio Announce Data Sharing Collaboration to Develop Advanced Drug Development Tools in Type 1 Diabetes

 

 

TUCSON, Ariz., and OLDWICK, N.J. May 13, 2020 — The Critical Path Institute (C-Path) and Provention Bio, Inc. (Nasdaq: PRVB) are proud to announce their collaboration to significantly improve the scientific community’s insight into type 1 diabetes (T1D) through Provention’s contribution of data from the Phase III Protégé study of teplizumab to the T1D Trial Outcome Measures Initiative (TOMI) integrated database. The Protégé study evaluated teplizumab on the preservation of beta cell function in newly onset T1D patients and generated the largest disease modifying interventional clinical trial dataset in T1D with more than 500 patients.

TOMI is an international partnership between academia, the pharmaceutical industry and nonprofit organizations funded by a grant from the world’s leading charities dedicated to diabetes research, JDRF and Diabetes UK. The primary goal of the TOMI is to seek the regulatory endorsement of drug development tools with the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) to accelerate the development of new therapies in T1D.

The Data Contribution Agreement (DCA) between Provention and C-Path will allow for this unique set of clinical trial data to be integrated and managed by C-Path’s Data Collaboration Center (DCC) adding to the effort of developing advanced drug development tools including a clinical trial simulation tool (CTST) for regulatory approval. This CTST will help identify optimal clinical endpoints in future studies to improve clinical interpretability of trials, shorten the time to primary outcome and/or minimize the number of participants required in trials.

The Protégé data includes relevant information about disease progression, drug effects and clinical trial design. Contribution of these data is critical to TOMI’s work in developing innovative and quantitative tools based on robust data that can be endorsed by regulators and be used in confidence by the pharmaceutical industry to optimize future clinical trial designs.

“C-Path is excited to facilitate the analysis of data with this esteemed group of T1D scientists,” said C-Path’s Executive Director of the T1D Consortium Inish O’Doherty, Ph.D. “Seeing the T1D community work together to solve challenging questions is encouraging and we are looking forward to the future of drug development in T1D.”

“We are proud to contribute such a large set of patient-level data from the Phase III Protégé study of teplizumab to the TOMI,” said Francisco Leon, M.D., Ph.D., Co-founder and Chief Scientific Officer of Provention Bio. “This collaboration embodies our continued support to increase pre-competitive collaborations and to advance effective public-private partnerships in the field of T1D to accelerate drug development.”

 

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.


 

Provention Bio, Inc. (Nasdaq: PRVB) is a clinical-stage biopharmaceutical company leveraging a transformational drug development strategy focused on the prevention or interception of immune-mediated disease. Provention’s mission is to source, transform and develop therapeutic candidates targeting the high morbidity, mortality and escalating costs of autoimmune diseases. Provention’s diversified portfolio includes PRV-031 (teplizumab), a pre-commercial-stage candidate that has been shown to delay the onset of end-stage type one diabetes (T1D) in at-risk individuals with pre-symptomatic disease. The Company’s portfolio includes additional clinical-stage product development candidates that have demonstrated proof-of-mechanism and/or proof-of-concept in other autoimmune diseases, including celiac disease and lupus. For more information, please visit proventionbio.com.


 

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.


 

1.   Diabetes UK’s aim is creating a world where diabetes can do no harm. Diabetes is the most devastating and fastest growing health crisis of our time, affecting more people than any other serious health condition in the UK – more than dementia and cancer combined. There is currently no known cure for any type of diabetes. With the right treatment, knowledge and support people living with diabetes can lead a long, full and healthy life. For more information about diabetes and the charity’s work, visit www.diabetes.org.uk.
2.   Diabetes is a condition where there is too much glucose in the blood because the body cannot use it properly. If not managed well, both type 1 and type 2 diabetes can lead to devastating complications. Diabetes is one of the leading causes of preventable sight loss in people of working age in the UK and is a major cause of lower limb amputation, kidney failure and stroke.
3.   People with type 1 diabetes cannot produce insulin. About 10 per cent of people with diabetes have type 1. No one knows exactly what causes it, but it’s not to do with being overweight and it isn’t currently preventable. It’s the most common type of diabetes in children and young adults, starting suddenly and getting worse quickly. Type 1 diabetes is treated by daily insulin doses – taken either by injections or via an insulin pump. It is also recommended to follow a healthy diet and take regular physical activity.
4.   People with type 2 diabetes don’t produce enough insulin or the insulin they produce doesn’t work properly (known as insulin resistance). Around 90 per cent of people with diabetes have type 2. They might get type 2 diabetes because of their family history, age and ethnic background puts them at increased risk. They are also more likely to get type 2 diabetes if they are overweight. It starts gradually, usually later in life, and it can be years before they realise they have it. Type 2 diabetes is treated with a healthy diet and increased physical activity. In addition, tablets and/or insulin can be required.

For more information on reporting on diabetes, download our journalists’ guide: Diabetes in the News: A Guide for Journalists on Reporting on Diabetes (PDF, 3MB).

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

 

C-Path Appoints Two New Members to Board of Directors

 

TUCSON, Ariz., April 30, 2020 — Tucson-based Critical Path Institute (C-Path) today announced the appointment of two new members to its Board of Directors: James W. Newman, CPA, former Executive Chairman of Victory Pharma, Inc. and Tomas Salmonson, Ph.D., M.Sc., former Chair of the Committee for Medicinal Products for Human Use (CHMP), European Medicines Agency (EMA).

“We are pleased to welcome both Jim and Tomas to the C-Path Board of Directors,” said Timothy R. Franson, chairman of the C-Path Board. “Their wealth of knowledge and experience in the pharmaceutical industry and regulatory sector, respectively, will be invaluable as we look toward the future and how we can continue to evolve and improve drug development across the globe.”

Newman is a longtime pharmaceutical executive with more than 30 years’ experience in pharmaceutical company senior management, finance, information systems, facilities operations and strategic planning. He founded Victory Pharma, Inc. in 2001 and served as Executive Chairman from 2001 – 2013. During his tenure at Victory, Newman was responsible for the acquisition and development of several proprietary products for the treatment of chronic pain. The company grew to approximately $100 million in annual sales prior to its sale to Shionogi in 2011. In 1998, Newman founded DJ Pharma, Inc. and served as President and CEO. The company expanded to 400 employees and $65 million in sales in two years. DJ Pharma sold at a 500 percent ROI for investors in 2001.

As Senior Vice President of Finance and Administration for Dura Pharmaceuticals, Inc., Newman redirected the company from a biotech to a pharmaceutical sales and marketing organization. He was responsible for organizing and completing its IPO and for four secondary and three private offerings to expand the market capitalization to more than $2 billion in six years. During his active retirement, Newman serves on the board of several charitable organizations and on the Executive Committee of the Pacific Crest Trail Association.

Dr. Tomas Salmonson has deep expertise in pharmacokinetics, bioequivalence, clinical trial methodology, clinical drug development and regulatory affairs and is currently partner at Consilium Salmonson & Hemmings. He left the Medical Products Agency in February 2019 after more than 30 years at the Swedish agency and the European regulatory network. He chaired the CHMP at the EMA between 2012 and 2018. Before that he was a member of CHMP representing Sweden between 1999 and 2012. During his last 10 years there, he also represented the EU at the ICH Steering /Management Committee and the ICH Assembly. Salmonson obtained his Ph.D. from Faculty of Medicine Uppsala University, Uppsala, Sweden and an M.Sc. from Uppsala University. He did postgraduate research at the University of California in San Francisco in the mid-80s.

Salmonson has published over 60 publications in different areas including pharmacokinetics and regulatory affairs. In 2016, he received the DIA Outstanding Contribution to Health Award, the Pharmacist of the Year in Sweden in 2017 and the TOPRA Lifetime Achievement Award in 2018.

“We’re fortunate to be able to leverage Jim’s previous operational and senior management experience at various, highly successful pharmaceutical companies and Tomas’ extensive background in drug development and the regulatory process through complex and challenging environments is of tremendous value, especially during these unprecedented times,” said C-Path President and CEO Joseph Scheeren, Pharm.D.

Newman and Salmonson’s Board appointments will begin June 1, 2020.

 


 

About Critical Path Institute
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.

 

Contact:

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