Global Biopharmaceutical Leader to Usher C-Path in Next Phase of Growth and Innovation

Board names Dr. Daniel Jorgensen as Chief Executive Officer

TUCSON, Ariz., Sept. 27, 2022 — Critical Path Institute (C-Path) today named Daniel M. Jorgensen, M.D., MPH, MBA, its Chief Executive Officer (CEO). An experienced leader in drug development, healthcare and business, Jorgensen will continue C-Path’s distinguished reputation of collaboration with global stakeholders, including patient groups, academic institutions, the pharmaceutical industry and regulatory agencies, to set a vision for C-Path’s next phase of growth and innovation. Jorgensen’s tenure as CEO will commence on the first of October.

“Dr. Jorgensen has long been a visionary in drug development, applying his vast insight, experience and passion for healthcare to advancing every organization he has been a part of,” said Kristen Swingle, M.S., C-Path’s COO and President. “We’re excited to welcome him to C-Path and to be able to benefit from his leadership and expertise.”

Jorgensen is an accomplished physician executive with more than 24 years of experience in the biopharmaceutical industry, in both small and large companies, public and private, including C-level positions for the past 11 years. Before C-Path, he was CEO at AxoProtego, guiding the neuroscience startup through funding and partnership activities. AxoProtego has an exclusive worldwide license from Johns Hopkins University for a biotechnology platform addressing neurodegeneration in areas of high unmet need.

Jorgensen began his career as a pediatrician with a deep interest in vaccines and public health. He worked at the Centers for Disease Control & Prevention (CDC) and other public health organizations before taking his first job in the industry, developing vaccines for Pasteur Merieux Connaught. During his 10 years at Pfizer, Jorgensen was the Global Clinical Leader for azithromycin and was named Pfizer’s first Vaccine Development Team Leader, playing a key role in multiple FDA meetings, submissions and approvals. Thereafter, he served as Chief Medical Officer at several biotechnology companies, developing drugs across multiple therapeutic areas.

“Dan’s deep pharmaceutical and regulatory expertise makes him the perfect choice to drive C-Path into the future,” said Wainwright Fishburn, C-Path Board Chair. “We were searching for a bold leader with an outstanding background to execute our strategic roadmap for continued organizational growth globally, and we’re thrilled that he’ll be leading the organization.”

“C-Path has an excellent reputation in advancing medical innovation and global health in a way that is uniquely collaborative, and I am thrilled to be part of its mission,” Jorgensen said. “We have a lot of exciting goals for the years ahead and I’m looking forward to hitting the ground running, with the support of C-Path’s outstanding staff and extended stakeholders.”

Jorgensen holds a B.S. from Yale University, an M.D. from the University of Wisconsin Medical School and a Master of Public Health from the University of Washington School of Public Health. He also holds an MBA from the Yale School of Management, as a member of the charter class for Yale’s MBA for Executives: Leadership in Healthcare.

“We look forward to building new C-Path partnerships within the national and international scientific, industry and regulatory communities, with the goal of improving the health of patients and populations,” Jorgensen added.


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 hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, C-Path in Europe is headquartered in Amsterdam, Netherlands and C-Path Ltd. operates from Dublin, Ireland with additional staff in multiple other locations. For more information, visit c-path.org.

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

Contact:

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

C-Path’s European Nonprofit Appoints Managing Director and New Board Member to Expand Global Efforts

TUCSON, Ariz. and AMSTERDAM, September 20, 2022 — Critical Path Institute’s (C-Path) European nonprofit today announced the appointment of Cécile Ollivier M.S. as Managing Director – Europe and Robert Hemmings, M.S., to its Board of Directors.

“We are pleased to appoint Cécile Ollivier as the Managing Director for our European activities, along with welcoming Robert Hemmings to C-Path’s European Board of Directors,” said C-Path Chairman of the Board Wainwright Fishburn. “Their wealth of knowledge and experience in the regulatory sector, plus Hemmings’ clinical trial methodology expertise, will be invaluable as we look toward the future and continue to accelerate drug development across the world.”

Ollivier is a senior health engineer with more than 15 years’ experience in global drug development and regulatory science, particularly for pediatric and rare diseases. Prior to joining C-Path, she was with a medical technology company developing digital endpoints, and for 11 years served as a scientific officer at the European Medicines Agency (EMA) where she provided technical and regulatory expert guidance on pediatric developments across multiple therapeutic areas.

Ollivier has been recognized for her work leading the EMA pediatric extrapolation global strategy and other global activities including the EMA/FDA harmonization for Gaucher disease for which she received an FDA award and the global harmonization of criteria for development in pediatric pulmonary arterial hypertension (PAH) with FDA and Health Canada. She was also an expert in the E11 R(1) working group and the pediatric standing group for the International Conference of Harmonization (ICH).

Hemmings is currently partner at Consilium Salmonson & Hemmings and has deep expertise in global clinical trial design, critical appraisal of clinical trial data and regulatory affairs. He was head of the group of statisticians and pharmacokineticists at the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK for nearly 20 years. He has been a member of the Committee for Medicinal Products for Human Use (CHMP) at the EMA for 11 years, has chaired EMA’s Scientific Advice Working Party 8 years, and chaired and served on EMA’s groups for biostatistics, modelling and simulation and extrapolation. Hemmings also represented the EU and was the Rapporteur for the revision of the ICH guideline E9 (R)1 addendum on estimands and sensitivity analysis in clinical trials, to the guideline on statistical principles for clinical trials. Additionally, he has involvement in multiple initiatives related to innovation in clinical trial design and regulatory strategy including, EMA’s Priority Medicines (PRIME) scheme for unmet medical needs, adaptable pathways, and accelerated access pathways in the UK.

Ollivier has been involved with C-Path since April 2021 and Hemmings’ Board appointment is effective September 1, 2022, joining Tomas Salmonson and Kevin Perkins appointed in February 2022.

 


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 hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, C-Path in Europe is headquartered in Amsterdam, Netherlands with additional staff in multiple other locations. For more information, visit c-path.org.

Contact:

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

C-Path Awarded FDA Grant to Establish Public-Private Partnership to Advance Treatments for Rare Neurodegenerative Diseases

TUCSON, Ariz., Sept. 14, 2022 — Critical Path Institute (C-Path) has announced it will serve as the convener of the Critical Path for Rare Neurodegenerative Diseases (CP-RND), a new public-private partnership (PPP) to benefit people across multiple rare neurodegenerative diseases, supported by a grant from the U.S. Food and Drug Administration (FDA). The Agency announced the PPP today in a press release.

“These innovative and collaborative approaches are needed to accelerate medical product development for the benefit of individuals living with these rare diseases, and their families,” said Wainwright Fishburn, C-Path Board Chair. “I am thrilled about this partnership that will strengthen collaborations between C-Path, FDA and NIH, and bring together a diverse group of stakeholders.”

C-Path’s proven proficiencies will be leveraged to ensure the success of this effort, specifically the organization’s track record in generating tangible solutions that have accelerated drug development in numerous diseases. This, together with the capabilities of its Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP ®) to integrate multiple patient-level data sources across rare diseases, will provide the foundation for leveraging advances in basic and clinical sciences, supported by NIH, and innovative regulatory science, supported by FDA.

In June, the FDA unveiled its Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis (ALS) — a five-year strategy for improving and extending the lives of people with rare neurodegenerative diseases, including ALS, by advancing the development of safe and effective medical products and facilitating patient access to novel treatments. The plan was developed in accordance with the provisions of the Accelerating Access to Critical Therapies for ALS Act, including the requirement to establish a public-private partnership this fiscal year.

“It is an honor for C-Path to be part of this transformative partnership with FDA and NIH. CP-RND will leverage all five of C-Path’s core competencies — data management and standards, quantitative analytics and modeling, biomarkers, clinical outcome assessments and regulatory science — as well as C-Path’s concentration areas from its neuroscience/neurology, pediatrics, and rare disease programs,” explained Klaus Romero, M.D., M.S., F.C.P., C-Path’s Chief Science Officer and Executive Director of Clinical Pharmacology. “This partnership with FDA and NIH will positively transform medical product development and make CP-RND a template for comprehensive and meaningful collaborative science.”

As with previous PPPs at C-Path, CP-RND will provide a neutral environment for pre-competitive collaboration including, but not limited to industry, patients, advocacy groups, academia, non-profit organizations, and others, to come together and actively contribute to advance promising solutions for the benefit of these communities. To learn more about C-Path’s CP-RND, visit https://c-path.org/programs/cp-rnd and contact the team at CP-RND@c-path.org.

 


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 hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, C-Path in Europe is headquartered in Amsterdam, Netherlands and C-Path Ltd. operates from Dublin, Ireland with additional staff in multiple other locations. For more information, visit c-path.org.

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

Contact:

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

C-Path’s Kristen Swingle Named President

Swingle joined C-Path in 2019 and was named interim president in 2021

TUCSON, Ariz., Sept. 8, 2022 — Critical Path Institute (C-Path) today announced that its Chief Operating Officer (COO) and Interim President, Kristen Swingle, M.S., has been named COO and President, effective immediately. Swingle has filled the interim President role since 2021, stewarding C-Path through growth and innovation, including the establishment of multiple national and global programs, a new European entity, a 50% staff increase, and a 90% increase in revenue.

Swingle, a leading voice in the Arizona bioscience community with two decades of experience in the medical and molecular sciences, has served as COO since July 2019, leading C-Path’s daily operations along with the development and implementation of its global strategy. Furthermore, she has served as Chairwoman for the AZ Bio Board of Directors for nearly five years and is a member of Women Business Leaders of the U.S. Health Care Industry Foundation.

“The Board recognizes the importance of this decision, as it represents its commitment to make deeper investments in C-Path’s leadership knowing that we are moving into very exciting times,” said C-Path Board Chairman Wainwright Fishburn. “The phenomenal momentum and traction over this last period of high growth for C-Path has a lot to do with the role Kristen has played. We have every confidence in her, and our incredible C-Path staff, as we take on the complex work ahead to support the great opportunities before us.”

“I thank the Board for this opportunity. I am incredibly honored to serve as President during a time of important and significant expansion of C-Path’s programs,” Swingle said. “My commitment to C-Path is always amplified by working with our dedicated and experienced executive leaders and staff — they are the driving force for C-Path’s success. I look forward to continuing to build on C-Path’s strengths and core competencies alongside my talented team.”

“Kristen has proven to shine in times of growth and change, and is a recognized leader with broad life science experience who has done a tremendous job leading C-Path operations,” said founding Board Member and Vice-Chairman Jeffery E. Jacob, SM. “The Board continues to work closely with Kristen, Chief Science Officer, Dr. Klaus Romero, Chief Financial Officer Kevin Perkins, and the Executive Team, to advance C-Path’s mission to improve the medical product development and regulatory process to benefit patients worldwide.”

The Institute and its Board have carefully deliberated its most advantageous steps forward to sustain the exponential growth it has undergone in the last three years, and anticipates announcing a new Chief Executive Officer this fall. “I look forward to partnering with our new CEO as we pursue C-Path’s ambitious agenda ahead,” Swingle said.


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 hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, C-Path in Europe is headquartered in Amsterdam, Netherlands and C-Path Ltd. operates from Dublin, Ireland with additional staff in multiple other locations. For more information, visit c-path.org.

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

Contact:

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

C-Path Receives DIA 2022 Americas Inspire Award for Outstanding Contribution to Health

TUCSON, Ariz., June 22, 2022 — Critical Path Institute (C-Path) is pleased to announce it has received the DIA 2022 Americas Inspire Award for Outstanding Contribution to Health. This prestigious honor is conferred upon an individual, group or organization that has made significant and innovative contributions to advancing global health.

“The work that Critical Path Institute has done through its programs has led to the development of tools and approaches to solve some of the most critical challenges in the product development pathway, including the need for aggregated data, and a platform for communication and collaboration,” wrote DIA representatives Anna McDermott-Vitak, Senior Vice President and Managing Director, Americas; and Stephanie Crawford, Ph.D., Chair, Americas Regional Advisory Council.

As also articulated by DIA, “One C-Path program, the eCOA Consortium, for example, has advanced the science of trial endpoints, particularly through a focus on patient reported outcomes through its ‘[eCOA:] Getting Better Together Initiative.’ Further, the eCOA Consortium has been a trusted and invaluable partner in the development of the DIA Digital Technology in Clinical Trials Conference that was founded in 2020.” Scottie Kern, Executive Director of the eCOA Consortium, accepted the award on behalf of C-Path at the Awards Celebration hosted by DIA’s Board of Directors in Chicago, Illinois on June 20, 2022.

“C-Path is honored to receive this prestigious award, affirming we are accomplishing our mission to accelerate the path to a healthier world,” said Kristen Swingle, M.S., C-Path Interim President and COO. “The eCOA Consortium team and everyone at C-Path would like to thank the DIA for this recognition.”



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 hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, C-Path in Europe is headquartered in Amsterdam, Netherlands and C-Path Ltd. operates from Dublin, Ireland with additional staff in multiple other locations. For more information, visit c-path.org.

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

About C-Path’s Electronic Clinical Outcome Assessment (eCOA) Consortium
The eCOA Consortium provides a pre-competitive environment in which a critical mass of experts can collaborate to generate measurement equivalence data, develop specification documents and data standards, and provide guidance on methodological considerations related to eCOA applications. All of these activities are aimed at enhancing the quality, practicality and acceptability of electronic capture of clinical trial endpoint data.

For more information on C-Path’s eCOA Consortium, visit:
https://c-path.org/programs/ecoac

Media Contact:

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

C-Path Launches Unique Translational Therapeutics Accelerator with Support from Cottrell Foundation

The global accelerator will transform academic discoveries to drug development opportunities

TUCSON, Ariz., June 9, 2022 — Critical Path Institute (C-Path) today announced it has been awarded a grant from the Frederick Gardner Cottrell Foundation, a non-profit organization established by Research Corporation Technologies, Inc. (RCT) that provides financial support for scientific research and educational programs, to create the C-Path Translational Therapeutics Accelerator (TRxA). The accelerator is a unique, global effort focused on supporting academic scientists in defining optimal strategies for advancing new, cutting-edge therapeutics from the lab to patients.

Advancing an idea from academic drug discovery research to human clinical trials is time and resource intensive, and, too frequently, unsuccessful. This transition from preclinical to clinical studies is therefore often referred to as the drug development ‘valley of death.’ Many aspects contribute to this valley of death, but key opportunities exist for TRxA to have a positive impact to bridge this gap.

One such opportunity is to leverage C-Path’s proficiencies in translational and regulatory science. In keeping with C-Path’s role as a unique, neutral convener between academia, industry, regulatory agencies and patients, TRxA will provide university-based researchers with advice to define optimal regulatory strategies to advance promising new drug candidates. Additionally, TRxA will have funding available to complete critical studies needed to validate the solidity of the drug product for further development.

By harnessing C-Path’s expertise in accelerating the medical product development process, grant awardees will receive guidance and mentoring to enhance the efficiency and effectiveness of advancing potential therapies from early discovery to clinical trials — starting with a clear end in mind.

Leading TRxA is newly hired Executive Director Maaike Everts, Ph.D., a pharmaceutical scientist with broad experience in drug delivery, discovery and development. Everts previously led a drug discovery program at the University of Alabama at Birmingham, and also has expertise in a CDMO environment for drug formulation and manufacturing. She will apply her firsthand experience to provide pivotal leadership, as TRxA begins accepting applications and issuing awards.

“I am honored and excited to have the opportunity to shepherd TRxA through its growth into a world-class drug accelerator program,” said Everts. “Helping academic researchers move their innovative ideas for new drugs from concept to reality is a personal passion, and a multidisciplinary team approach is critical to bringing new therapies to patients as quickly as possible.”

“C-Path is exceptionally well-suited to offer a novel translational accelerator program that will support a comprehensive strategic approach for academic groups facing the valley of death,” explained Klaus Romero, M.D., M.S., F.C.P., C-Path’s Chief Science Officer and Executive Director of Clinical Pharmacology. “In this accelerator model, C-Path will leverage our core competencies to provide strategic and tactical regulatory science guidance, engage relevant stakeholders when needed and bring diverse expertise to pivotal early-stage study design and implementation.”

“Our Cottrell Foundation has been a supporter of C-Path from its inception, and we have watched it develop into a world-class organization that catalyzes the development process of new medicines working with the FDA, EMA, industry, academia and patients. TRxA represents a unique opportunity to harness the strengths of C-Path to optimize the advancement of new therapies originating at universities and research institutions worldwide, and we’re proud to partner with C-Path on this innovative venture,” said Shaun Kirkpatrick, President, Biotechnologies, at RCT. “TRxA will provide resources and expertise to support academic scientists in developing a robust research plan and validating comprehensive data packages for potential drug candidates, a key to garnering interest from biotechnology and pharmaceutical companies who would further work with the academic scientist to bring promising drug candidates to the health care setting.”

To learn more about TRxA, be informed when the award application period opens and how to apply for an award, visit c-path.org/programs/trxa or contact C-Path at TRxA@c-path.org.


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

About the Frederick Gardner Cottrell Foundation
Research Corporation Technologies, Inc. (RCT) established the Frederick Gardner Cottrell Foundation in 1998 to provide financial support for scientific research and educational programs at qualified nonprofit organizations. RCT named the foundation in honor of the university professor and inventor who championed the transfer of academic innovation to public use. The Cottrell Foundation receives its support from donations made by RCT and is a private, non-operating entity.

Media Contact:

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

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

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

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

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

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

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

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

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

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


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

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

Contact:

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

C-Path Receives Largest Data Transfer to Date from UK’s National Neonatal Research Database

The EPR data transfer is the first from the United Kingdom and includes data from nearly 200 hospitals


TUCSON, Ariz., June 1, 2022 — Critical Path Institute (C-Path) has received fully anonymized electronic patient record (EPR) data from the National Neonatal Research Database (NNRD). This is part of efforts to better understand and find treatments for a chronic lung disease which frequently affects premature infants. This initiative, funded by a grant from the U.S. Food and Drug Administration (FDA), is being executed through a neonatal pilot project within C-Path’s International Neonatal Consortium (INC). It is the first time C-Path has received EPR data from the United Kingdom and includes data from a network of nearly 200 hospitals in England.

The NNRD data transfer received ethics approval from the UK Health Research Authority, and none of the data can be traced back to individual patients.

“We have been working with the NNRD team for more than five years and are very excited that extensive discussions have brought us to this significant milestone in data sharing,” said Mark Turner, Ph.D., Professor of Neonatology and Research Delivery, University of Liverpool, UK and INC Co-director. “This EPR data, coupled with previous clinical trial data transfers from other international centers, will shed new light on the causes and treatments of a major cause of death and disability among babies born very prematurely.”

The data transfer is the largest ever for C-Path to date, with nearly 200,000 fully anonymized patient-level data points to be used to understand how the lung condition bronchopulmonary dysplasia (BPD) develops in premature infants. The aim is also to identify stages when doctors can intervene to reduce the risk of BPD, to help find treatments, and to help understand the long-term effects of BPD.

The generation of these real-world evidence solutions will be led in partnership between INC and C-Path’s Quantitative Medicine Program with support from its Data Collaboration Center. C-Path’s INC Executive Director Kanwaljit Singh, M.D., MPH, added, “C-Path has extensive experience in real-world data project collaborations and we’re ecstatic to welcome the NNRD’s data into this initiative. We can’t thank Professor Neena Modi enough for recognizing the unmet need in neonatal drug development and accelerating this important collaboration.”

The EPR data shared by the NNRD will be integrated with other patient-level real world data national registries and networks, as well as clinical trial datasets contributed by INC industry members.

Neena Modi, FMedSci, who established the NNRD and is a Professor of Neonatal Medicine at Imperial College London, said, “The NNRD is a one-stop source of clinical data to improve newborn care, developed with wide stakeholder and parent involvement. We are exceptionally grateful to the families who agree to share data, and to the healthcare staff who carefully record this. By combining the NNRD data with data from other international registries and clinical trials, C-Path has created a novel resource for researchers to study BPD in detail and hopefully reduce the risk of a preterm baby developing the condition. I hope that in time this will improve treatments and the outlook for infants with BPD.”

C-Path’s INC will continue to partner with collaborators and new data contributors to integrate additional patient-level datasets.

Learn more about Imperial College of London’s Neonatal Data Analysis Unit, which supports the management and development of the NNRD, here:
https://web.www.healthdatagateway.org/search?search=NNRD&tab=Datasets.

For more information on collaborating with INC, and how to contribute data, please email Christine Barry at
cbarry@c-path.org.

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


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

 

Contact:

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

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

 

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

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

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

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

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

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

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

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


About C-Path

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

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

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


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

About the Janssen Pharmaceutical Companies of Johnson & Johnson. 

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

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

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

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

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

Contact:

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

C-Path European Nonprofit Established in Amsterdam

C-Path in Europe works seamlessly with its U.S. counterpart to advance the development of novel regulatory-endorsed methodologies to accelerate medical product development

AMSTERDAM and TUCSON, Ariz., March 23, 2022 — In support of Critical Path Institute’s (C-Path) mission to catalyze innovation that accelerates the path to a healthier world, C-Path is excited to announce the opening of its EU nonprofit headquarters in the Netherlands, which will focus on the development of existing and future activities in Europe.

C-Path is recognized globally as a leader in regulatory science, with extensive knowledge and experience in seeking and obtaining regulatory endorsement of drug development tools (DDTs) or novel methodologies in cooperation with the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA). All C-Path regulatory-endorsed tools and methodologies are freely available to the broader scientific community.

The nonprofit’s headquarters in Amsterdam will extend and complement C-Path’s activities to improve public health, as well as share expertise, data, risks and costs to expedite advancements in global regulatory science by facilitating public-private partnerships with members from the biopharmaceutical industry, government regulatory agencies, academic institutions and patient groups in Europe.

“C-Path already has an established and longstanding presence in Europe,” said C-Path Board Member, Tomas Salmonson, Ph.D., M.S., and former Chair of the EMA Committee for Medicinal Products for Human Use (CHMP). “We look forward to facilitating existing and new collaborations to productively support European Regulatory Science research programs and also contribute to the delivery of the EMA Regulatory Science Strategy to 2025.”

Since its inception in 2005, C-Path has worked to forge relationships and collaborations in Europe in tandem with U.S. efforts to facilitate global collaboration. Since then, C-Path has received a significant number of Qualification Opinions and Letters of Support from EMA and has a substantial track record of successful DDT and novel methodology endorsements by FDA, EMA and PMDA. These regulatory milestones have contributed to addressing different unmet needs in medical product development, which have transformed drug development in different areas (among others, tuberculosis, polycystic kidney disease, Alzheimer’s, Parkinson’s, and irritable bowel syndrome).

“Advances in drug development are rapidly playing out on a global stage, and C-Path’s extensive background in accelerating the drug development and regulatory processes will fuel innovation for the development of novel approaches that can be endorsed by regulators in many countries,” said C-Path Chief Science Officer Klaus Romero, M.D., M.S., F.C.P. “Working together with European organizations remains a key C-Path priority. We’re excited to have an office in Amsterdam, which we know will serve to enhance our collaborative efforts and speed the development of treatments for some of the world’s most complex health issues.”

An informative webinar, C-Path in Europe: Moving Global Regulatory Science Forward, is scheduled for April 20, 2022. Register here.

To learn more, visit: https://c-path.org/c-path-in-europe.


 

About C-Path

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

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

 

Contact:

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