Biomarkers of Crohn’s Disease to Support the Development of New Therapeutic Interventions

C-Path’s Crohn’s Disease Biomarkers Pre-Consortium funded by The Helmsley Charitable Trust is proud to announce its first publication in the journal Inflammatory Bowel Diseases titled, “Biomarkers of Crohn’s Disease to Support the Development of New Therapeutic Interventions.” The result of collaboration among experts in Crohn’s disease, including academia, health care institutes, foundations, public advocacy groups, industry and regulatory bodies, this work has identified biomarkers that can be used as drug development tools to advance clinical trials in IBD and potentially improve patient care.

Free access: https://academic.oup.com/ibdjournal/advance-article/doi/10.1093/ibd/izaa215/5896885?guestAccessKey=70d1e3c0-2aaf-4d65-b1bb-292f7df3b625

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

 

ePRO Consortium Announces Open Access Article

 

 

In collaboration with the DIA Study Endpoints Community, C-Path’s ePRO Consortium is pleased to announce the open access article “Considerations for development of an evidence dossier to support the use of mobile sensor technology for clinical outcome assessments in clinical trials” is available online in the April 1, 2020 issue of Contemporary Clinical Trials. This article provides recommendations for the structure and content of an evidence dossier aimed at supporting a clinical trial endpoint derived from mobile sensor data as fit-for-purpose in regulatory decision making.

Link to article: https://www.contemporaryclinicaltrials.com/article/S1551-7144(20)30040-9/fulltext

 

 

C-Path Receives FDA Grant to Establish Rare Disease Clinical Outcome Assessment Consortium

 

 

TUCSON, Ariz., March 19, 2020 — The U.S. Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) has funded a cooperative agreement to establish a Rare Disease Clinical Outcome Assessment (COA) Consortium. The grant (U01FD006882)* was awarded to the Critical Path Institute (C-Path) with the National Organization for Rare Disorders (NORD) as a sub-awardee. The first step taken toward the establishment of the new consortium has been the creation of the Rare Disease Subcommittee within C-Path’s Patient-Reported Outcome (PRO) Consortium. The PRO Consortium will serve as an incubator for the maturation of a pre-competitive, multi-stakeholder consortium within C-Path’s COA Program.

The PRO Consortium’s Rare Disease Subcommittee includes representatives from C-Path, NORD, FDA, the Patient-Centered Outcomes Research Institute, the National Center for Advancing Translational Sciences, and biopharmaceutical firms within the PRO Consortium that are developing treatments for rare diseases. In addition, plans are underway to enable rare disease-focused biotech firms not currently members of the PRO Consortium to be included in the strategic planning for the new consortium. Once established, the Rare Disease COA Consortium’s activities will be aimed at accelerating the development of new medical products intended to safely and effectively treat people with rare diseases by creating and curating a resource of information on publicly available COAs identified as potentially fit-for-purpose endpoint measures in treatment trials for rare diseases. The premise is that existing COAs may be able to be used or modified for use across multiple diseases sharing common characteristics.

Along with planning the membership, governance and organizational structure of the new consortium, the Rare Disease Subcommittee has launched a multi-pronged effort aimed at tackling challenges in the assessment of clinical benefit in rare disease treatment trials. The first pilot project under this initiative is to identify COAs that can be used in children to assess activities of daily living, which is a meaningful aspect of life impacted by many rare diseases. This will be the first in a series of reviews to identify COAs aimed at symptom and functional domains that reflect important aspects of patients’ lives that are impacted by rare diseases. Concurrently, the Rare Disease Subcommittee has initiated a second pilot project that involves a literature review to explore ways in which researchers have handled heterogeneity in clinical trials including an examination of the advantages and disadvantages of the approaches to personalizing endpoints. Development of best practice recommendations for assessing clinical benefit in rare disease trials will be subsequently explored.

*Funding for this press release was made possible, in part, by the Food and Drug Administration through grant U01FD006882. Views expressed here do not necessarily reflect the official policies of the Department of Health and Human Services nor does any mention of an organization imply endorsement by the United States Government.

 


 

 

About Patient-Reported Outcome Consortium

The Patient-Reported Outcome (PRO) Consortium was formed in 2008 by Critical Path Institute in cooperation with the US Food and Drug Administration’s Center for Drug Evaluation and Research and the pharmaceutical industry. The mission of the PRO Consortium is to establish and maintain a collaborative framework with appropriate stakeholders for the qualification of PRO measures and other clinical outcome assessment (COA) tools that will be publicly available for use in clinical trials where COA-based endpoints are used to support product labeling claims.

 

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

C-Path and Lundbeck Announce a Data Sharing Collaboration to Enable Development of Advanced Drug Development Tools in Alzheimer’s Disease

 

 

TUCSON, Ariz., March 18, 2020 — The Critical Path Institute (C-Path) and H. Lundbeck A/S (Lundbeck) are proud to announce that they will work together to significantly improve the scientific community’s insight in Alzheimer’s disease (AD) through Lundbeck’s contribution of a unique set of clinical trial data from more than 2,500 AD patients to the Critical Path for Alzheimer’s Disease (CPAD) consortium’s integrated database for qualified researchers across the globe to access.

The Data Contribution Agreement (DCA) between Lundbeck and C-Path, with support from Gates Ventures, will allow for these clinical data to be integrated and managed by CPAD, with support from C-Path’s Data Collaboration Center (DCC) adding to the CPAD patient-level database in AD, which presently contains data from more than 14,500 patients. CPAD is currently exploring acquisition of contemporary datasets from other sources with an expected growth of the CPAD database to include more than 40,000 patient-level records in the coming months.

This contribution of patient-level data from Lundbeck embodies Lundbeck’s continued support to increase pre-competitive collaborations between pharmaceutical companies and academia, and to advance effective public-private partnerships. Generous contributions such as these ensure that CPAD can continue its mission to develop innovative quantitative tools and methods to de-risk and speed up the drug development process in AD.

These data stem from Lundbeck’s Phase II and III studies that evaluated the compound idalopirdine in AD and include relevant information about disease progression, drug effects and clinical trial design. The shared data are highly valuable and the contribution of this type of data is vitally important to CPAD’s work in developing a disease progression model across the entire continuum of AD, from the earliest to late stages of the disease. CPAD aims to provide advanced drug development tools that will aid in optimizing clinical trial designs and execution and facilitating the regulatory review process.

“Lundbeck and C-Path recognize the hard work to generate these data and contributions of the many patients with Alzheimer disease, their relatives and caregivers, and the investigators who took part in these trials,” said C-Path President and CEO Joseph Scheeren, Pharm.D. “We look forward to continuing to work together to advance and expedite the development of new therapies.”

“We are delighted with this opportunity to share data with CPAD, ensuring that researchers can get the most out of them in advancing the understanding of Alzheimer’s and how to best develop new treatments. We are proud to contribute to this work, not only through our own drug development activities, but also in this unique collaboration,” said Mads Dalsgaard, Senior Vice President, Experimental Medicine & Clinical Development at Lundbeck.

 


 

About Critical Path Institute
C-Path is an independent, nonprofit organization established in 2005 as a public-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’s US headquarters is located 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 H. Lundbeck A/S
H. Lundbeck A/S (LUN.CO, LUN DC, HLUYY) is a global pharmaceutical company specialized in brain diseases. For more than 70 years, we have been at the forefront of neuroscience research. We are tirelessly dedicated to restoring brain health, so every person can be their best.

An estimated 700 million people worldwide are living with brain diseases and far too many suffer due to inadequate treatment, discrimination, a reduced number of working days, early retirement and other unnecessary consequences. Every day, we strive for improved treatment and a better life for people living with brain diseases – we call this Progress in Mind.

Read more at www.lundbeck.com/global/about-us/progress-in-mind.

Our approximately 5,500 employees in more than 50 countries are engaged in the entire value chain throughout research, development, production, marketing and sales. Our pipeline consists of several R&D programs and our products are available in more than 100 countries. We have research centres in Denmark and California and our production facilities are located in Denmark, France and Italy. Lundbeck generated revenue of DKK 18,1 billion in 2018 (EUR 2,4 billion; USD 2,8 billion).
For additional information, we encourage you to visit our corporate site www.lundbeck.com and connect with us on Twitter at @Lundbeck and via LinkedIn.

 

Contact:

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

C-Path to Lead Multi-Stakeholder Engagement on FDA’s Proposed Novel Framework to Enhance the Pediatric Medical Device Ecosystem

 

TUCSON, Ariz., March 17, 2020 — The Critical Path Institute (C-Path) today announced it has been awarded a grant to conduct stakeholder engagement to garner insights, feedback and refinement of the U.S. Food and Drug Administration (FDA) Center for Devices and Radiological Health’s (CDRH) proposed framework to enhance the pediatric medical device ecosystem and to develop a strategic plan for implementation. Funded by a cooperative agreement through the FDA, C-Path will work in collaboration with CDRH and other stakeholders to organize a workshop to brainstorm on the framework and strategic plan for building a viable ecosystem.

“We are thrilled to be part of this effort and honored to have been selected to work with CDRH to optimize the ecosystem,” said C-Path President and CEO Joseph Scheeren, Pharm.D. “This initiative has our full support and fits in well with our ongoing efforts in the pediatric and neonatal space.”

Currently, there are challenges to advancing promising new pediatric devices from ideation to clinical studies, to regulatory approval, to use for pediatric patients, including:

  • The small numbers of children with any one condition, and their dispersion across the world, making it difficult to conduct clinical trials;
  • The need to consider children’s growth and variably influenced disease mechanisms;
  • The lengthy development timeline and associated high costs;
  • Lack of funding for small inventors and companies;
  • Difficulty in identifying expert resources across the development-to-commercialization continuum.

Modernized regulatory programs and processes developed by CDRH during the past few years have supported a growing number of novel technologies available to improve patient care. In the pediatric area, CDRH has led a public meeting where challenges to innovation were verified and aspects of a supportive ecosystem were discussed. Additionally, CDRH has finalized guidance documents clarifying regulatory issues relevant to pediatric populations and provided millions of dollars to fund pediatric device consortia across the U.S. However, over the past decade, fewer than 10% of high-risk and high-benefit medical devices have been designed, evaluated and labelled for children below the age of 18.

“Today’s health care system has yet to engender a solution to the complex public health issue of medical device development for children and small populations,” said CDRH Chief Medical Officer and Director for Pediatrics and Special Populations Vasum Peiris, M.D., M.P.H. “Novel regulatory options and public-private sector collaboration informed by patient and caregiver perspectives are necessary components to create a sustainable national environment for safe technology innovation that serves the unique needs of children. We welcome input from all stakeholders as we clarify and build on the framework CDRH has proposed to transform traditional thinking around engaging, sustaining and innovating in the pediatric medical device ecosystem.”

Stakeholder groups including the Advanced Medical Technology Association (AdvaMed), the American Academy of Pediatrics (AAP), the Milken Institute, and clinicians, researchers, innovators, payors and patient groups will review and refine the high-level proposed framework developed by CDRH. The framework lays out a vision for an ecosystem that would facilitate the design, development and commercialization of medical devices for children. Through stakeholder input and feedback the framework will be iterated and refined, and goals for the yet to-be-formed multi-stakeholder workgroup – whose objective would be to optimize and complete the design of the ecosystem – will be established.

“There is an opportunity for the FDA, patient advocacy groups, academia and industry to collaborate and optimize design — with the guidance and support of C-Path — of this much needed ecosystem,” said AdvaMed Vice President for Technology and Regulatory Affairs Tara Federici. “Stakeholders will review and evaluate the proposed framework to ensure it could be effective in de-risking and accelerating pediatric medical device development while maintaining existing safety and effectiveness requirements.”

“Children are not small adults — their biology is different. Even so, every day across the nation, the care of children requires the use of many medical devices that were not specifically designed for them,” said Kurt Newman, M.D., President and CEO of Children’s National Hospital and immediate past Chair of the Board of Trustees of Children’s Hospital Association. “Children should be a priority when it comes to healthcare innovation because entire lifetimes are at stake. We commend and support the FDA’s leadership in this effort to create a system that supports innovation for children.”

“Pediatricians often find themselves without the medical or surgical devices they need to care for their patients because medical devices for children can lag five to ten years behind those for adults,” said American Academy of Pediatrics CEO and Executive Vice President Mark Del Monte, J.D. “That time frame is most of an entire childhood. We must do more to get children the devices they need sooner, and innovation is essential to enhance safe and effective options. We applaud those who have come together with that goal in mind and look forward to working with the FDA, C-Path, and other stakeholders to advance device development for children.”

“It is essential to shift the balance of device innovation toward fulfilling the vast unmet needs of children,” said Christopher Lee, former Director of the Milken Institute Center for Financial Markets and co-chair of the FasterCures BRIDGE Initiative, a recently launched program that develops novel models for driving biomedical innovation. “This proposed framework seeks to take this challenge head on by realigning incentives across the system, and we look forward to contributing to that process.”

The project’s initial deliverable, to bring together interested stakeholders to share insights and further refine and develop the proposed framework at the workshop, will occur in the latter part of this year. Specific dates, location, and registration information for the meeting will be released soon. Meeting proceedings and a strategic development plan to enhance the pediatric medical device ecosystem will be developed within six months of the workshop.

This grant is funded by a cooperative agreement through the FDA [Critical Path to Public Private Partnerships Grant Number U18 FD005320]; the grant is administered by the Center for Drug Evaluation and Research (CDER); this project is funded by the Center for Devices and Radiological Health (CDRH). Views expressed in written materials or publications do not necessarily reflect the official policies of the U.S. 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 AdvaMed
AdvaMed member companies produce the medical devices, diagnostic products and health information systems that are transforming health care through earlier disease detection, less invasive procedures and more effective treatments. AdvaMed members range from the largest to the smallest medical technology innovators and companies. For more information, visit advamed.org.

 


 

About the American Academy of Pediatrics
The American Academy of Pediatrics (AAP) is an organization of 67,000 primary care pediatricians, pediatric medical subspecialists and pediatric surgical specialists dedicated to the health, safety and well-being of infants, children, adolescents and young adults. For more information, visit aap.org and follow us on Twitter @AmerAcadPeds

 


 

About Milken Institute
The Milken Institute is a nonprofit, nonpartisan think tank that helps people build meaningful lives, in which they can experience health and well-being, pursue effective education and gainful employment, and access the resources required to create ever-expanding opportunities for themselves and their broader communities. FasterCures is a center of the Milken Institute, with a mission to put patients in the center of the healthcare system, and break down the unnecessary barriers to innovation. For more information, visit milkeninstitute.org.


 

About FDA
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

 

Contact:

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

C-Path Appoints Dr. Klaus Romero as Chief Science Officer

 

 

TUCSON, Ariz., March 12, 2020 — The Critical Path Institute (C-Path) today announced the appointment of Klaus Romero, M.D., M.S., F.C.P., as its new Chief Science Officer (CSO) effective April 1, 2020. Dr. Romero, a clinical pharmacologist and epidemiologist with more than 17 years combined experience in academic and pharmaceutical clinical research, translational sciences, pharmacometrics, modeling and simulation and pharmacoepidemiology, has been with C-Path since December 2007. His most recent role has been as Executive Director of Clinical Pharmacology and Quantitative Medicine. He will continue in dual roles as CSO and head of the Quantitative Medicine Program.

As CSO, Dr. Romero will spearhead innovations in research and science by delivering strategic and operational leadership of C-Path’s data-driven nationally and internationally recognized programs in medical product development solutions. In collaboration with senior leadership, he will set the vision for C-Path’s scientific endeavors and ensure that the focus of C-Path’s public-private partnerships stay closely aligned with its mission to accelerate the pace and reduce the costs of medical product development, through innovation in data science, model-informed drug development, patient-centric drug development and precision medicine to optimize the scientific evaluation of the efficacy and safety of new therapies. This will be achieved through strengthening C-Path’s pillars in quantitative medicine, data science, biomarkers, clinical outcome assessment tools and regulatory science.

“With more than 15 years of diverse experience in the drug development space, including demonstrated leadership abilities in heading large programs and cross-functional teams, there are few people who would be equally suited to lead C-Path’s scientific initiatives than Klaus,” said C-Path President and CEO Joseph Scheeren, Pharm. D. “His combination of clinical pharmacology, epidemiology and model and simulation experience, plus his business development expertise and history of fostering meaningful interactions between regulatory agencies, industry, academia and patient groups will be key in enhancing signature C-Path consortia initiatives, while bringing strategic focus to data analytics to drive research and create efficiencies across all stages of drug development.”

During his tenure with C-Path, he has helped lead clinical pharmacology, pharmacoepidemiology and modeling and simulation projects in Alzheimer’s disease, polycystic kidney disease (PKD), tuberculosis, type 1 diabetes, Parkinson’s disease, Duchenne muscular dystrophy, kidney transplantation, Huntington’s disease and cardiovascular drug safety. His work has helped to achieve major milestones, including the first regulatory endorsement by the U.S. Food and Drug Administration and European Medicines Agency of a clinical trial simulation tool for mild and moderate Alzheimer’s disease and the regulatory qualification of the first imaging biomarker for PKD. Dr. Romero’s scientific production with C-Path has resulted in more than 60 peer-reviewed publications related to his work with the Institute.

Reporting directly to Scheeren, Dr. Romero will serve as the Institute’s spokesperson on all scientific matters.

“I am incredibly excited and honored to have been given the opportunity to further serve C-Path in this role. I am proud of our 15-year history, as we have grown from a small group of pioneers into a mature organization of innovators in solutions to expedite medical product development,” said Dr. Romero. “I look forward to advancing and growing our pipeline of collaborative pre-competitive efforts. The era of knowledge management, decision science and personalized medicine represents the right time for C-Path to grow its leadership in transforming smart data into actionable cutting-edge knowledge to de-risk and expedite medical product development.”

Dr. Romero is a fellow of the American College of Clinical Pharmacology, a founding member of the International Society of Pharmacometrics, as well as a member of the American Society for Clinical Pharmacology and Therapeutics, and the International Society for Pharmacoepidemiology. In addition to his duties at C-Path, he serves as Chairman of the Board of Directors for CredibleMeds®. He is fluent in English, Spanish, German and Portuguese, and has a passion for music, designing and playing innovative electric guitars. Dr. Romero is also a Research Associate Professor at the University of Arizona College of Medicine, Adjunct Professor at the College of Health Solutions at Arizona State University, Adjunct Professor at the University of Southern California’s School of Pharmacy and serves on the Scientific Board of Pharos Dx. Dr. Romero received his medical degree from Pontifical Xavierian University, completed his training in Clinical Pharmacology at Columbia National University and holds an MS degree in Epidemiology from the Columbia School of Medicine.

Dr. Romero succeeds C-Path CSO Lynn Hudson, Ph.D., who will retire at the end of March; a position she has held since 2011. Under Hudson’s leadership, C-Path’s scientific consortia and programs have more than doubled.

“It has been an honor to serve as C-Path’s scientific leader for the past decade,” Hudson said. “C-Path continues to reach important milestones and achieve great progress in creating drug development efficiencies that have changed the time and expense it takes to get safe medical products to those who need them. I am incredibly proud of the work done here and I know Klaus will be a key player in C-Path’s future success.”


 

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
615.310.1894
kissyblack@c-path.org

C-Path Selects Experienced Neuroscientist to Lead Alzheimer’s Consortium

 

 

TUCSON, Ariz., March 12, 2020 — The Critical Path Institute (C-Path) today announced it has named Sudhir Sivakumaran, Ph.D., as Executive Director of its Critical Path for Alzheimer’s Disease (CPAD) consortium effective immediately. Dr. Sivakumaran, a neuroscientist and research and development professional, has nearly 20 years combined experience in academic, pre-clinical and early clinical research and development, phase 1 and phase 2 clinical study design, data analysis and diligence and business development. He has been with C-Path since July 2019 as Associate Director for CPAD.

As Executive Director of CPAD, Dr. Sivakumaran will guide all operational and scientific input for the CPAD Consortium and develop and implement the strategic plan and priorities for individual working groups within CPAD. He will work with key individuals at global regulatory agencies including the U.S. Food and Drug Administration, European Medicines Agency and the Pharmaceuticals and Medical Devices Agency. Dr. Sivakumaran will also provide scientific leadership and oversee projects dealing with neuroscience, biomarker qualification, clinical outcome assessment development, data sharing and aggregation related to utility of modeling and simulation in Alzheimer’s disease (AD).

“We are thrilled that Sudhir has accepted the role of Executive Director of our long-standing Alzheimer’s consortium, CPAD,” said C-Path President and CEO Joseph Scheeren, Pharm. D. “He brings a wealth of experience to the program and in the short time Sudhir has been with C-Path, he has stepped up to continue to drive CPAD’s initiatives to create new tools and methods to increase the efficiency in the development process of new treatments for AD and related neurodegenerative disorders. With Sudhir leading CPAD, I know we’ll continue to enhance our already ample collaborative efforts with our agency, industry and patient organization partners in this area.”

Dr. Sivakumaran previously held positions as Associate Director, Research Management at Aptinyx, Inc. in Greater Chicago and Senior Manager, External Partnerships & Alliances, Neuroscience at Pfizer, Inc. in Cambridge, Mass. He has published more than a dozen peer-reviewed publications and was the recipient of multiple research grants and awards including the Epilepsy Foundation & American Epilepsy Society Post-doctoral Training Fellowship, Integrated Pilot Award from BU-CTSI, Academy of Finland Research Grant and the CURE Young Investigator Travel Award among others. He speaks English, Tamil, and Italian and his interests include traveling the world, long road trips and exploring and enjoying world cuisine. Dr. Sivakumaran holds a doctorate in neuroscience from SISSA/ISAS in Trieste, Italy and an M.Sc. and B.Sc. in microbiology from Univ. of Madras in India.

“I am very excited to be at C-Path and for the opportunity to lead the CPAD consortium,” said Dr. Sivakumaran. “Alzheimer’s disease is globally one of the most devastating diseases for millions of people and their loved ones. We are at a game changing moment in finding meaningful and effective therapeutic options in AD. An organization like C-Path, and its consortium CPAD, provides a great opportunity for us to come together within a neutral, pre-competitive environment in partnership with industry experts, regulatory agencies and patient organizations and work towards finding effective solutions for the most pressing needs and de-risk the drug development process in AD. I look forward to continuing to work with everyone at C-Path and beyond, towards refining and redefining our future course of action in AD drug development.”

Reporting directly to Scheeren, Dr. Sivakumaran will serve on C-Path’s leadership team, which is responsible for the overall strategic direction of the Institute.


 

About CPAD
CPAD’s mission is to accelerate the drug development process for patients with chronic neurodegenerative disease leading to dementia. Its primary focus is on AD. CPAD works with industry, regulatory authorities, and patient advocacy organizations to advance Drug Development Tools for evaluating drug efficacy and safety, to optimize novel clinical trial designs, and aggregating anonymized patient-level data using CDISC consensus standards to facilitate the regulatory review process.

CPAD has the following areas of pre-competitive focus: (1) regulatory qualification of biomarkers (2) development of CDISC data standards for AD endpoint assessments, (3) creation of integrated databases for observational and clinical trials data, and (4) development of quantitative model-based tools for drug development.

 

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
615.310.1894
kissyblack@c-path.org

ERA4TB, an International Consortium to Accelerate the Development of Comprehensive Treatments Against Tuberculosis

C-Path, Ltd. and more than 30 partners from 13 countries participate in the ERA4TB project.

The ERA4TB (European Regimen Accelerator for Tuberculosis) project is a public-private initiative dedicated to the development of drugs against tuberculosis. With a team of more than thirty organizations and a budget of over 200 million euros ERA4TB will focus on developing a new, improved tuberculosis treatment. The partners will share their expertise, knowledge and resources to rapidly progress new candidate drugs into clinical trials.

Tuberculosis is the leading cause of death by an infectious disease worldwide. According to the World Health Organization (WHO), an estimated 10 million people became ill with tuberculosis in 2018, and 1.6 million died. Even though the incidence of tuberculosis is declining, the drug-resistant form constitutes a growing threat to the safety of the world population. It is in this spirit that the UN has pledged to end the tuberculosis epidemic by 2030 through joint action of its member states.

David Barros-Aguirre, Project Lead for ERA4TB and VP and Head of Tuberculosis Research, Global Health R&D, GSK said: “On behalf of the ERA4TB partners, we are very excited about the start of this unique collaborative project in the field of tuberculosis where the expertise from public partners and a pipeline of promising preclinical candidates from pharmaceutical companies merge to accelerate the development of novel clinical candidates. The goal is to deliver an innovative and differentiated combination regimen for the treatment of TB, which can play a key role in the elimination agenda.”

Standard tuberculosis treatment is based on a combination regimen of four drugs that were all developed more than 60 years ago. Treatment lasts for at least six months and, in the case of resistance to the standard drugs, can be as long as two years. The current drugs are inefficient by today’s standards and a new, faster-acting and safer treatment is required to reduce the length of therapy and to overcome the menace of drug-resistant strains. Until now, the development of new drugs has been slow and their incorporation into tuberculosis treatment regimens conducted in a sequential manner.

Stewart Cole, Scientific leader of ERA4TB and President of the Institut Pasteur, said “ERA4TB has assembled an impressive array of resources to seamlessly harness the agility and innovation of academia with the pragmatism and scientific expertise of pharma. I am confident that this powerful European initiative will speed the path to TB elimination.”

ERA4TB is set to change the paradigm of tuberculosis treatment development by abandoning the sequential approach in favor of a parallel pathway, which will allow the simultaneous investigation of more than a dozen drug candidates. By implementing a standardized approach to tuberculosis drug development, that is well coordinated with the collaborations outside Europe, ERA4TB has the potential to optimize, and, more importantly, greatly reduce the development times of the new regimens needed to eliminate this epidemic.

According to Juan José Vaquero, project coordinator from UC3M (University Carlos III Madrid) and leader of the imaging team, “ERA4TB will change the paradigm for the development of new TB drugs by adopting a flexible and modular collaboration scheme in which research efforts can be rapidly mobilized to where they are most needed allowing several molecules to be investigated simultaneously. This will save both time and money.”

ERA4TB is sponsored by the European Union’s Horizon 2020 research and innovation programme and the European pharmaceutical industry (via EFPIA) under the auspices of the Innovative Medicines Initiative 2 Joint Undertaking and part of the AMR Accelerator umbrella.

About the Innovative Medicines Initiative

The Innovative Medicines Initiative (IMI) is Europe’s largest public-private initiative aiming to speed up the development of better and safer medicines for patients. IMI supports collaborative research projects and builds networks of industrial and academic experts in order to boost pharmaceutical innovation in Europe. IMI is a joint undertaking between the European Union and the European Federation of Pharmaceutical Industries and Associations, EFPIA. More information can be found at www.imi.europa.eu.

Acknowledgement

This project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking (JU) under grant agreement No 853989. The JU receives support from the European Union’s Horizon 2020 research and innovation programme, and in-kind support from EFPIA, TB Alliance, Bill & Melinda Gates Foundation and University Of Dundee.

 

Disclaimer

This communication reflects the views of the ERA4TB Consortium and neither IMI nor the European Union and EFPIA are liable for any use that may be made of the information contained herein.

 

CONTACTS:

Project Leader: David Barros-Aguirre
Project Coordinator: Juan Jose Vaquero Lopez
Scientific Leader: Stewart Cole

C-Path Selects Global Regulatory Affairs Leader as New President and CEO

TUCSON, Ariz., March 21, 2019 — After a worldwide search, the Critical Path Institute (C-Path) today announced it has appointed Joseph Scheeren, Pharm. D., as its new President and Chief Executive Officer. Scheeren previously served as Senior Advisor Research and Development for Bayer AG and has had a distinguished 36-year career in the pharmaceutical industry having held positions domestically and internationally in drug development and regulatory approval on three continents.

“Dr. Scheeren exemplifies the in-depth knowledge and inspiring leadership that C-Path, and every organization we collaborate with, deserve in a chief executive officer,” said C-Path Board Chairman Timothy R. Franson, M.D. “His global experience with drug development and the regulatory process is evidenced by a long list of first cycle approvals for new therapies and we couldn’t be more thrilled to welcome him to C-Path.”

Scheeren joined Bayer Pharmaceuticals as Senior Vice President, Head of Global Regulatory Affairs, in 2004, responsible for development in the US and in 2009 became Site Head US in Montville, NJ. In 2012, he assumed the position of Head of Global Development Asia in Beijing and in 2015, was appointed Head of Global Regulatory Affairs Pharma and Consumer Care of Bayer Healthcare, Basel, Switzerland. In January 2018, he was appointed Senior Advisor R&D, Bayer AG. Scheeren’s experience with global regulatory affairs will help C-Path expand its mission worldwide.

“C-Path and its partners define the type of innovation and solutions that healthcare needs now and into the future to provide patients hope and access to new treatments and cures by accelerating innovation in the development and approval pathway,” Scheeren said. “I am committed to continuing C-Path’s success by making new science happen through collaborations and continuing to be a change agent in healthcare.”

Scheeren succeeds C-Path President and CEO Martha Brumfield, Ph.D., who has led the organization since 2013 and is initiating a phased retirement. Under Brumfield’s leadership, C-Path’s funding and staff have doubled, the organization has established a Data Collaboration Center and a center of excellence in quantitative medicine, plus expanded its profile in Europe. Brumfield will serve as a special advisor to Scheeren for six months and will oversee select projects already in progress to ensure a seamless transition.

“C-Path has had outstanding growth since its founding in 2005, when it responded to the call of the FDA’s Critical Path Initiative program,” Brumfield said. “We are confident that this transition under Dr. Scheeren’s leadership will mark yet another key milestone toward our future success. I look forward to supporting him in the coming months.”

Scheeren has an established network of connections across the globe and holds many memberships and designations. He serves on the advisory boards at the Center for Innovation in Regulatory Science, the Center of Regulatory Excellence Singapore, the Research & Development-based Pharmaceutical Association in China, and the China Core Research and Development Committee. He is currently serving as Board chair of the Drug Information Association (DIA) and has served as Chairman of the DIA Regional Advisory Council for Europe, the Middle East and Africa; he is also a foreign member of the Academie Nationale de Pharmacie, France, a lecturer at Yale University and an adjunct Professor at Peking University.

His start date is April 15, 2019 and he and his family will be living in Tucson, Arizona.

 


 

About C-Path:

C-Path (Critical Path Institute) 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 over 1,500 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path is headquartered in Tucson, Arizona, with additional staff in multiple remote locations. For more information, visit www.c-path.org.

 

Media Contact:

Kissy Black
615.298.1144
kissyblack@lotosnile.com