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

Critical Path Institute Celebrates Expanded Operations with Opening of European Headquarters

 

C-Path in the EU will work to accelerate the development of new therapies through innovative methodologies for endorsement by regulators of pharmaceutical products

TUCSON, Ariz., January 17, 2019 — Critical Path Institute (C-Path) is excited to announce the launch of Critical Path Institute, Ltd. (C-Path, Ltd.) in Ireland, a fully-owned subsidiary that will enable C-Path to increase its activity in the European Union and broaden its global operations as it works to accelerate the development of therapies in a wide range of diseases and medical conditions. As a nonprofit that builds consensus among its stakeholders from around the world to improve public health, the organization shares expertise, data, risks and costs to move regulatory science forward.

As its U.S. counterpart does, C-Path, Ltd. will form consortia of scientists and clinicians from the biopharmaceutical industry, government regulatory agencies, academic institutions and patient groups. These consortia will collaborate to develop novel methodologies — such as biomarkers, clinical outcome assessment instruments, models and clinical trial simulation tools and databases — and will submit evidence packages to European regulatory agencies (as well as agencies in the U.S. and Japan) for review. The novel tools and methodologies will be made freely available to the broader scientific community.

“After our initial startup phase, we anticipate increasing C-Path activities in the European Union,” said C-Path Executive Director, European Office, Graham Higson, M.Sc. “We look forward to bringing our expertise to bear on European Research Infrastructure programmes sponsored by the European Commission, such as those led by the Innovative Medicines Initiative.”

Shortly after it was founded in 2005, C-Path began forging relationships with European entities. In 2007, C-Path’s Predictive Safety Testing Consortium (PSTC) was the first collaboration to submit through the pilot qualification procedures an evidence package for seven kidney safety biomarkers to the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan. In May 2008, EMA issued a qualification statement for these nephrotoxicity biomarkers.

Over the ensuing 10 years, C-Path has received a number of positive qualification opinions from EMA including those for an Alzheimer’s disease imaging biomarker, a disease model of mild to moderate Alzheimer’s, kidney safety biomarkers, the Hollow Fiber System model of tuberculosis, a polycystic kidney disease imaging biomarker, and a Parkinson’s disease imaging biomarker, as well as EMA Letters of Support for skeletal muscle and liver safety biomarkers, and a proposed model-based clinical trial enrichment tool for clinical trials in Amnestic Mild Cognitive Impairment (aMCI).

“Advances in drug development are rapidly playing out on a global stage, and C-Path’s expertise in applying regulatory science fuels innovation and the development of novel methodologies that can be endorsed by regulators in multiple countries like Europe, the U.S. and Asia,” said C-Path President and CEO Martha Brumfield, Ph.D. “C-Path began working together with European organizations early on, and our commitment to Europe remains solid. We’re excited to begin this new chapter, which we know will only serve to enhance our collaborative efforts and speed the achievement of our shared goals.”

 

About C-Path, Ltd., Board of Directors

The C-Path, Ltd. Board of Directors brings a wealth of experience in medical research and innovation, academic leadership, regulatory science and the pharmaceutical and biotech industries.

Enda Connolly, B.A., has held leadership roles in economic development, science and innovation in Ireland for more than 35 years. As chief executive of the Health Research Board, he helped reposition Irish health research by introducing innovative funding initiatives that place emphasis on patient-oriented research. He previously served as a member of the executive team of IDA Ireland. Connolly has worked as a strategic advisor on economic development projects for the governments of Qatar and Saudi Arabia. He currently is chairman of the Board of Beaumont Hospital, and he serves on the boards for RCSI Hospital Group and the Health Information and Quality Authority.

 

John Hegarty, Ph.D., D.Sc., has more than 40 years of experience in research, education, and university leadership. He was a professor of laser physics at Trinity College Dublin, where he served as president/provost from 2001 to 2011. More recently, Hegarty co-founded Innovation Advisory Partners, which focuses on education, research, and innovation policy. He is chair of the Irish Times Trust and a member of the Irish Times Board. He also is a member of the Fulbright Ireland Board and the Hugh Lane Gallery Board, and he chairs the Translational Medicine Institute based in St. James’s Hospital.

 

Graham Higson, M.Sc., has spent more than 35 years in the pharmaceutical industry. He is managing director and owner of GCH Pharma Consulting Ltd, which specializes in the provision of strategic regulatory advice, drug development, regulatory outsourcing and organizational development. He has been a member of both the EFPIA and PhRMA regulatory affairs committees, the CMR Regulations Advisory Board, and the TOPRA Regulatory Advisory Council. Higson will continue to serve as the Executive Director, European Office, for C-Path.

 

Caitriona O’Driscoll, Ph.D., B.Sc. Pharm., is a pharmacist, and professor and chair of pharmaceutics at the School of Pharmacy, University College Cork (UCC), Ireland. O’Driscoll’s research is focused on the design, formulation and characterization of biomimetic drug delivery systems, incorporating the products of the pharmaceutical biotechnology industry such as peptide/protein-type drugs and nucleic acids. She is a board member of the Irish Health Products Regulatory Authority and a founding member and chair of Regulatory Science Ireland.

 

Mary Teeling, M.D. Pharm., is a specialist pharmaceutical physician with more than 30 years’ experience in the areas of pharmacology and pharmaceutical medicine. She developed the Master of Science (MSc) in Pharmaceutical Medicine programme in 2004, while working in Trinity College Dublin, and was course director up until 2018. Currently, she is adjunct professor of pharmaceutical medicine within the department of pharmacology and Therapeutics. She has also worked, or held roles, with the National Medicines Information Centre, the Irish Medicines Board, the EU Committee for Human Medicinal Products, the Royal College of Physicians, the NDA Regulatory Advisory Board and the WHO Uppsala Monitoring Centre.

Visit the Critical Path, Ltd. website at: https://c-path.eu/.

 

 


About Critical Path Institute

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.

Contact:

Kissy Black
615.298.1144
kissyblack@lotosnile.com
 

First-ever biomarker qualified for Parkinson’s is a vital step toward improved clinical trials

FOR IMMEDIATE RELEASE

Contact:
Kissy Black
615.298.1144
kissyblack@lotosnile.com

 

Tucson, AZ — July 26, 2018 – The Critical Path Institute’s (C-Path) Critical Path for Parkinson’s Consortium (CPP), in partnership with Parkinson’s UK, announces that the European Medicines Agency (EMA) has issued a positive qualification opinion on an imaging test (biomarker) as a tool to enrich Parkinson’s clinical trials. The purpose of this enrichment biomarker is to serve as a measurement that can be used to select people with Parkinson’s who are most suitable to take part in clinical trials.

The biomarker is used to determine the presence of dopamine transport deficiency in the brain, and has been qualified as an enrichment biomarker for clinical trials targeting early stages of Parkinson’s soon after diagnosis. The qualified biomarker involves the intravenous injection of a small amount of a radioactive tracer before the brain images are acquired, and can be done at any one of many specialist imaging centers. The imaging agent binds very specifically to dopamine transporter sites on the neurons that are lost in Parkinson’s disease. The use of this biomarker can help better identify patients that are more likely to exhibit significant progression in their motor signs and symptoms, thus helping select patients for clinical trials. The CPP consortium is a global public-private partnership consisting of industry, academics, advocacy organizations, and government agencies collaborating to develop solutions to optimize drug development for Parkinson’s. This qualification represents a major milestone in this regard as the first biomarker for Parkinson’s to receive such a regulatory designation. In 2015, the U.S. Food and Drug Administration issued a letter of support for the use of this imaging biomarker for use in Parkinson’s clinical trials.

“This endorsement from the European Medicines Agency represents many years of hard work and incredible collaboration among companies, universities, and charities facilitated by the Critical Path Institute,” says Dr. Diane Stephenson, Executive Director of CPP, who led the work. “These brain scans in themselves are not new, but until now there has not been a clear consensus that they can and should be used to select participants for clinical trials. Through our global project, we’ve been able to bring all the data and expertise together to make a powerful case, so we’re delighted that this endorsement from the EMA will improve the quality and chances of success for future trials of Parkinson’s treatments. This success is just the first in a suite of new tools that we hope to deliver for Parkinson’s.”

Studies suggest that up to 15% of individuals taking part in clinical trials for new Parkinson’s treatments may not exhibit a measurable progression in motor signs and symptoms over the course of such trials. Furthermore, the rate of uncertainty in predicting disease progression is higher at earlier stages of the condition. These individuals are extremely unlikely to benefit from the new therapies being tested, and their inclusion can affect both the trial results and, ultimately, the future of the potential treatment.
Professor David Dexter, Deputy Research Director of Parkinson’s UK, which funds CPP, comments, “Scientific breakthroughs mean that there is now a new wave of exciting treatments that genuinely could slow, stop, or reverse the condition, but it’s crucial that we’re able to test them properly in clinical trials. Being able to rule out individuals who are unlikely to have Parkinson’s could be the difference between a successful trial and failure. This is a vital step forward in our mission to deliver, as quickly as possible, better treatments, and one day a cure, to people living with Parkinson’s.”

Because Parkinson’s is a progressive condition caused by the gradual loss of cells in the brain, the best chance to intervene with treatments that can slow, stop, or reverse damage is during the earliest stages of the condition. During these early stages, however, signs, and symptoms tend to be mild, which makes selecting the right people to participate in trials very difficult.

“The use of these brain scans is already being included in new clinical trials at Biogen.” said Dr. Michael Ehlers, Executive Vice President of Research and Development at Biogen. “We believe that this new approach will introduce greater efficiency in terms of cost and speed, while ensuring that the right patients are being included in our trials.”


About the Organizations:

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.

Every hour, someone in the UK is told they have Parkinson’s.

It affects 127,000 people in the UK – which is around one in 500 of the population.

Parkinson’s is a degenerative neurological condition, for which there currently is no cure. The main symptoms of the condition are tremor, slowness of movement and rigidity.

Parkinson’s UK is the UK’s leading charity supporting those with the condition. Its mission is to find a cure and improve life for everyone affected by Parkinson’s through cutting edge research, information, support and campaigning.

For advice, information and support, visit www.parkinsons.org.uk or call our free, confidential helpline on 0808 800 0303.

Parkinson’s UK Contact:
Molly Horsburgh
Senior Media and PR Officer
0207 963 9351
mhorsburgh@parkinsons.org.uk
Out of hours: 07961 460248

CPTR Newsletter – April 2018

 

CPTR Newsletter – April 2018

In this issue:

  1. ReSeqTB to be Adopted for WHO Surveillance of Drug-Resistant TB
  2. New Collaborations Expand TB-PACTS Database and Leverage Data for Analyses
  3. CPTR Workgroup Works to Advance Regulatory Qualification of LAM, a Promising New Biomarker
  4. TB-PBPK Model Progresses Toward EMA and FDA Qualification
  5. TB-ReFLECT Update: A Collaborative Effort to Enhance TB Clinical Research
  6. New Project Launched to Evaluate Adaptive TB Clinical Trial Designs
  7. C-Path Partners with Catalysis Foundation for Health to Establish Database with Visualization and Analytic capabilities

ReSeqTB to be Adopted for WHO Surveillance of Drug-Resistant TB

The Relational Sequencing TB Data Platform (ReSeqTB), a global TB knowledge base for predicting TB drug resistance, will be adopted as the World Health Organization (WHO) bioinformatics platform for sequence-based surveillance of drug-resistant TB (DR-TB). In this new project phase, ReSeqTB will support surveillance and global policy development for sequencing-based clinical diagnosis of DR-TB. It could also serve as a model for the broader goals of establishing global antimicrobial resistance surveillance and clinical diagnosis networks for WHO high-priority pathogens. The development of ReSeqTB has been made possible through the collaboration of WHO, Foundation for Innovative New Diagnostics (FIND), and Critical Path Institute (C-Path) through their participation in the CPTR Initiative. 

“Genetic sequencing is the future of surveillance and diagnosis of DR-TB,” said Dr. Tereza Kasaeva, Director of the WHO Global TB Programme. “With ReSeqTB, sequencing can be used to rapidly and accurately estimate prevalence of resistance to anti-TB drugs. This platform has the potential to expand our understanding of the genetic basis of drug resistance and open the way for the use of sequencing for effective clinical management of patients with DR-TB, saving millions of lives.”

“ReSeqTB is actively collecting, standardizing, and aggregating genomic, phenotypic and, when available, clinical data to support the development of global policy for use of sequencing-based diagnostics in high-burden settings” said Dr. Debra Hanna, Executive Director of C-Path’s Critical Path to TB Drug Regimens (CPTR) initiative. “Together with our partners, we are working with the US FDA to pursue regulatory clearance of ReSeqTB to support patient management decisions.”

ReSeqTB is changing the DR-TB landscape. “ReSeqTB is already informing the development of commercial TB molecular diagnostic solutions, and the confidence-graded mutations list is becoming a standard for interpretation of existing and future molecular diagnostics,” said Dr. Catharina Boehme, CEO of FIND. “Building genetic sequencing into global diagnostics policy is an exciting advance. Enhanced diagnostics mean that patients can get a diagnosis earlier, which means they can access the right treatment more quickly – and the transmission of DR-TB is curtailed.”

Read full press release here: https://c-path.org/global-health-partners-accelerate-uptake-of-genetic-sequencing-for-surveillance-and-diagnosis-of-drug-resistant-tuberculosis/

New Collaborations Expand TB-PACTS Database and Leverage Data for Analyses

The TB PACTS Steering, including representatives from C-Path, the Special Programme for Research and Training in Tropical Diseases (TDR) at WHO, TB Alliance, St. George’s University of London, University of California, San Francisco, and the Innovative Medicines Initiative (IMI), were enthusiastically in favor of integrating datasets previously consolidated by the IMI PreDiCT-TB consortium into the TB-Platform for Aggregation of Clinical TB Studies (TB-PACTS). The consolidated IMI PreDiCT-TB consortium database currently contains 32 clinical data sets, most mapped to the CDISC TB 1.0 standard.  To-date, TB-PACTS has received data transfers for thirteen of these datasets. An additional eight datasets are expected to be integrated into the platform within the year.

This collaboration will expand the aggregated Phase 3 TB clinical trial data in the TB-PACTS data platform that is publicly available to qualified researchers. Within CPTR, the data will be used to expand the ongoing modeling and simulation efforts (e.g., refine quantitative models linking dynamic changes in time to liquid culture positivity (TTP) to clinically relevant endpoints for Phase 3 trials such as culture conversion and durable cure).

“When we established TB-PACTS we did it not only to make data from TDR-sponsored studies available, but also (and mostly) to create a trusted system where others too would be confident to share the data they generated,” said Dr. Piero Olliaro, head of intervention and implementation research at TDR. “This is happening now with the IMI PreDiCT-TB datasets, and we look forward for others to follow, so that researchers can have as many data as possible to investigate and fill critical knowledge gaps in tuberculosis.”

CPTR Advances Regulatory Discussions on Promising Tools and Methodologies to Inform and Enhance Decision-making in TB Drug Development

CPTR Workgroup Works to Advance Regulatory Qualification of LAM, a Promising New Biomarker

CPTR’s Biomarker and Clinical Endpoint Workgroup continues to progress the investigation of lipoarabinomannan (LAM) as a non-culture based pharmacodynamic biomarker to assess treatment response in clinical trials evaluating novel TB drug candidates.

LAM is a major component of mycobacterium cell wall, comprising up to 1.5% of total bacterial weight. A new immunoassay (LAM-ELISA) has been developed by Otsuka Pharmaceutical Co, Ltd (Otsuka)., which uses monoclonal antibodies against novel, unique LAM epitopes with high sensitivity and specificity to quantify sputum LAM concentration. Under the proposed context of use, sputum LAM could serve as a potential real-time decision-making tool in adaptive clinical designs of new TB treatment regimens. 

Building off a successful Critical Path Innovation Meeting with the FDA earlier in the year, CPTR received a positive response from FDA to the Letter of Intent submission, formally accepting the biomarker into FDA’s Qualification Program on October 31, 2017. The team has been advancing discussions with the EMA in parallel, with plans to submit documentation on the biomarker to both agencies in 2Q 2018.

TB-PBPK Model Progresses Toward EMA and FDA Qualification

CPTR and Simcyp have successfully developed a physiologically-based pharmacokinetic model for tuberculosis (TB-PBPK).  This model incorporates relevant pathophysiological changes that occur in TB-infected individuals to describe drug distribution from the systemic circulation into the different lung compartments and tissues, including granulomas. It may be utilized for simulations with a variety of virtual populations, including a virtual South African population. This model is complete and fully deployed within the Simcyp® simulator environment.  When applied to a library of standard of care (SOC) or emerging data for new compounds, this model may be used to inform early clinical development through predictions of drug distribution to the target tissues.

CPTR and Simcyp submitted a Letter of Intent and a briefing package to the EMA on November 22, 2017 to start the formal scientific advice qualification process for the PBPK model as a TB drug development tool. CPTR received formal feedback from EMA prior to a scheduled meeting with EMA’s Scientific Advice Working Party (SAWP) to discuss qualification on February 7, 2018. The EMA endorsed the updated context of use statement, to focus on the application of the PBPK platform to inform the design healthy volunteer studies for drugs that are not P-gp substrates, administered as monotherapy, for which plasma concentrations are known.  Further refinements to the model are being investigated which will serve as the basis for a final qualification decision by EMA.

CPTR held an informal meeting with the FDA on December 1, 2017 to discuss next steps for regulatory interaction for the potential endorsement of the PBPK platform. Per FDA’s request, CPTR will initiate a Fit-For-Purpose submission to FDA, with a Letter of Intent and Briefing Book, incorporating the scientific advice from EMA. 

Application of novel tools and methodologies can inform drug development decisions throughout the process:

TB-ReFLECT Update: A Collaborative Effort to Enhance TB Clinical Research

In 2016, C-Path and the Global TB Programme of the World Health Organization (WHO) partnered with researchers from the University of California, San Francisco (UCSF), to develop leading-edge quantitative analyses of data from the TB-Platform for Aggregation of Clinical TB Studies (TB-PACTS) database. The objective of this collaboration, called TB reanalysis of fluoroquinolone clinical trials (TB-ReFLECT), is to extract from these analyses key lessons from the TB-PACTS platform, and then package such lessons as tools to optimize future TB trial design.

The TB-ReFLECT team completed sensitivity analyses in November 2017, which included different classifications of unfavorable outcomes, and efficacy analyses that examined random effects of pharmacokinetic (PK) imputations and parametric survival models. UCSF recently presented on TB-ReFLECT efficacy analyses at a WHO workshop on clinical design, and a manuscript detailing these analyses has been submitted for peer review.   

The final stage of the project, safety analysis, is in progress and will analyze proportionality approaches to identify specific adverse events or clusters of adverse events, to inform benchmarking of the HRZE regimen in terms of safety.

CPTR will hold a face-to-face Steering Committee meeting to discuss how to message major learnings from TB-ReFLECT findings and discuss extensions to the project and/or integrations of the analyses into other CPTR projects.

New Project Launched to Evaluate Adaptive TB Clinical Trial Designs

CPTR’s Biomarker and Clinical Endpoint Workgroup (BCE-WG) launched a new effort to evaluate adaptive TB clinical trial designs, via simulation.  This project aims to evaluate a set of Phase 2 and 3 clinical development strategies and compare their overall efficiency and performance in selecting potential pan-TB regimens. A Steering Committee, consisting of experts in the field from academia, NGOs, and industry, was created to develop the scope, metrics and quantitative work for this project. Quantitative analyses simulation work is scheduled to commence in 2018. 

“Adaptive clinical trial designs using biomarkers to rapidly select the most promising treatments have been used with much success in other disease areas,” said Dr. Patrick Phillips, Professor, UCSF. “We need to be using the most efficient tools to rapidly develop safer and more efficacious TB regimens that can bring real patient benefit. In this project, we will develop and critically appraise some of the most promising biomarker-driven clinical development strategies, providing guidance to help developers speed novel regimens to market.”

C-Path Partners with Catalysis Foundation for Health to Establish Database with Visualization and Analytic capabilities

C-Path and Catalysis Foundation for Health (CFH) have partnered to share data from the clinical study “Mycobacterium tuberculosis (Mtb) Biomarkers for Diagnosis and Cure.” The collaboration seeks to identify biomarkers that correspond to detailed PET/CT information collected in this study, and to ultimately identify blood, urine, or saliva biomarker(s) that could replace sputum as a means of both diagnosing a patient and tracking their treatment progress.

Read more about the corresponding transcriptomics data for this study here:

https://www.ncbi.nlm.nih.gov/geo/query/acc.cgi?acc=GSE89403.

Parkinson’s UK Announces Major Pharma Companies Sign Up to CPP

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Major pharma companies sign up to groundbreaking Parkinson’s consortium

Seven of the world’s largest pharmaceutical companies have signed up to a groundbreaking consortium aimed at accelerating the development of safe and effective therapies for Parkinson’s.

The Critical Path for Parkinson’s consortium will bring together leading academics; industry members AbbVie, AstraZeneca, Biogen, Eli Lilly and Company, Merck Sharp and Dohme (known as Merck & Co. Inc. in the United States and Canada), Pfizer, and UCB; and founders the Critical Path Institute (C-Path) and Parkinson’s UK, to share data, expertise and resources to promote and develop new treatments for Parkinson’s.

The consortium, which was launched in October 2015, was formed by Parkinson’s UK and C-Path to increase investment into research and development of new Parkinson’s treatments.

Dr Arthur Roach, Director of Research at Parkinson’s UK, the principal funder of the consortium, says:

“Despite significant advances in our understanding of the genetics, biochemistry and pathology of Parkinson’s, the development of new treatments has not kept pace. New treatments are desperately needed to deal with the devastating effects of this progressive condition.

“Investing in clinical trials for brain disorders currently carries a high cost and high risk of failure. As the world’s largest patient-led Parkinson’s charity, we know that people living with conditions such as Parkinson’s have often been disappointed when drugs that showed significant promise early on failed in late stage testing.

“We see the consortium as a crucial part of strategies to develop new treatments that work at the earliest stage of the condition, with the goal of slowing its progression, and eventually finding a cure.”

Parkinson’s affects 127,000 people in the United Kingdom, and approximately 7 million people worldwide. In 2012/13, the National Health Service (NHS) spent more than £212 million on caring for people with Parkinson’s in England (1), with the cost of the condition in the United States approximately US$25 billion per year (2). With the baby boomer generation becoming older, the number of people with neurodegenerative conditions, including Parkinson’s, is likely to increase and will become one of the biggest issues faced by healthcare.
The International Federation of Pharmaceutical Manufacturers & Associations (IFPMA) estimated that the cost of developing a single drug was US$1.3 billion, rising from US$138 million in 1978 (3), a tenfold increase attributed to a multiplicity of factors faced by drug research.

Diane Stephenson, PhD, Executive Director, Critical Path for Parkinson’s consortium, C- Path, says:

“With the increase in the costs of getting a drug to market, the design of a clinical trial is a crucial part of a drug’s success. There is a strong realisation from the industry that collaboration among industry, academia, and worldwide regulatory agencies, along with the sharing of data, has the potential to create a more efficient development process. This recognition is evidenced by the fast pace at which members of this new consortium have joined.”

Parkinson’s UK has committed over £1million to the Critical Path for Parkinson’s consortium.

For more information, and to donate to Parkinson’s UK research and the work with the Critical Path Institute, visit http://parkinsons.org.uk/research


 

Media enquiries

Please contact: Hanna Kilpin, Senior Media & PR Officer on 020 7963 9311 or email: hkilpin@parkinsons.org.uk

Out of hours: 07961 460248 Notes to editors

  1. Parkinson’s and the NHS in England: the cost of poor care. Parkinson’s UK. October 2013.
  2. Statistics on Parkinson’s. Parkinson’s Disease Foundation.
  3. The Pharmaceutical Industry and Global Health: Facts and Figures 2015.
    International Federation of Pharmaceutical Manufacturers & Associations.

 

About Parkinson’s UK

Every hour, someone in the UK is told they have Parkinson’s.

It affects 127,000 people in the UK – which is around one in 500 of the population.

Parkinson’s is a degenerative neurological condition, for which there currently is no cure. The main symptoms of the condition are tremor, slowness of movement and rigidity.

Parkinson’s UK is the UK’s leading charity supporting those with the condition. Its mission is to find a cure and improve life for everyone affected by Parkinson’s through cutting edge research, information, support and campaigning.
For advice, information and support, visit http://www.parkinsons.org.uk or call our free, confidential helpline on 0808 800 0303.

Critical Path Institute Secures Additional Regulatory Support For Kidney Safety Biomarkers

January 12, 2015

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Critical Path Institute Secures Additional Regulatory Support For Kidney Safety Biomarkers

EMA Letter of Support Opens Door for Clinical Qualification

TUCSON, Ariz., January 12, 2015 – The Critical Path Institute (C-Path) announced today that the European Medicines Agency (EMA) issued a first-of-its kind Biomarker Letter of Support for two essential kidney safety biomarkers identified and evaluated by the Predictive Safety Testing Consortium (PSTC)’s Nephrotoxicity Working Group.

The kidney safety biomarkers, osteopontin (OPN) and neutrophil gelatinase-associated lipocalin (NGAL), are proteins that can be measured in urine. Higher levels of OPN and NGAL could indicate that the kidneys are being damaged, which may result in a loss of kidney function. The Letter of Support intends to encourage scientists to collect data from nonclinical and exploratory clinical studies, which may lead to qualification of these biomarkers.

“We are pleased to continue working collaboratively with the EMA, academia, and industry to identify tools, processes, and methods to improve the drug development process,” said Martha Brumfield, Ph.D., president and chief executive officer of C-Path. “With this Letter of Support, the EMA has opened doors that encourage the generation of necessary, rigorous clinical data to determine if these biomarkers hold clinical utility.”

The EMA’s Letter of Support indicates that these biomarkers have strong potential for use in humans and warrant additional exploration and data gathering. The EMA granted the Letter of Support for OPN and NGAL to encourage their use in both nonclinical and exploratory clinical studies as markers of proximal renal tubule degeneration/necrosis. With this milestone, work will continue in earnest on the qualification of OPN and NGAL for use in clinical trials.

“We are all incredibly excited about our collaborative interactions with EMA and the creation of the Letter of Support, enabling us to further define the path to clinical biomarker qualification,” said John Michael Sauer, Ph.D., Executive Director, Predictive Safety Testing Consortium, Critical Path Institute.

The Letter of Support is posted on the EMA website and can also be accessed via the C-Path PSTC website under the Regulatory Successes tab, along with a summary data package describing the studies that support the use of these kidney safety biomarkers.

About the Critical Path Institute

The Critical Path Institute (C-Path) is an independent, non-profit organization established in 2005 with public and private philanthropic support from the Arizona community, Science Foundation Arizona, and the U.S. Food and Drug Administration (FDA). 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 seven global, public-private partnerships that currently include over 1,000 scientists from government and regulatory agencies, academia, patient advocacy organizations, and dozens of major pharmaceutical companies. C-Path is headquartered in Tucson, Arizona. For more information, visit http://www.c-path.org.

FDA, IMI, and Patient Advocates to Present at CDISC 2012 International Interchange Formal Launch of CFAST – Collaboration between C-Path and CDISC

The Clinical Data Interchange Standards Consortium (CDISC) and Critical Path Institute (C-Path) announce the launch of the Coalition For Accelerating Standards and Therapies (CFAST), a follow-up to the partnership agreement signed earlier this year. The official launch of CFAST will take place at the CDISC 2012 International Interchange in Baltimore, Maryland (24-26 October 2012).

European Medicines Agency Deems Imaging Biomarker a Qualified Measure to Select Patients with Early Stages of Cognitive Impairment for Alzheimer’s Disease Clinical Trials

Based on a request for regulatory review by Critical Path Institute’s (C-Path) Coalition Against Major Diseases (CAMD), the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use has issued a positive opinion on the use of magnetic resonance imaging (MRI) to measure hippocampal volume as a tool to enrich recruitment into regulated clinical trials in the pre-dementia stage of Alzheimer’s disease (AD). This is the first imaging-based biomarker for AD to be granted a positive opinion by a regulatory agency.

MRI.pdf