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.

Critical Path Institute Receives Regulatory Support for Liver Injury Biomarker

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Tucson, AZ — January 22, 2018Critical Path Institute (C-Path) announced today that the European Medicines Agency (EMA) has issued a Letter of Support for measurement of glutamate dehydrogenase (GLDH) as a biomarker of hepatocellular liver injury. The letter was awarded to C-Path’s Predictive Safety Testing Consortium (PSTC) and Duchenne Regulatory Science Consortium (D-RSC) to encourage the further study of serum GLDH for monitoring hepatocellular liver injury.

The letter—in response to data submitted by the PSTC Hepatotoxicity Working Group (HWG) and D-RSC—describes EMA’s thoughts on the value of GLDH and supports further evaluation. In it, EMA encourages PSTC and D-RSC to investigate “the voluntary and complementary use of serum GLDH, in conjunction with currently used biomarkers of liver injury, as a clinical biomarker of liver injury.” EMA also supports PSTC’s generation of additional clinical safety data, and plans for further clinical studies to potentially enable formal qualification of GLDH in the future.

“Many of the proteins currently monitored to evaluate liver safety are also found in muscle tissue,” says Jane Larkindale, D.Phil., Executive Director of D-RSC. “In situations where a patient has underlying muscle injury, such as muscular dystrophies, levels of these enzymes may be high in the absence of liver injury. Monitoring of GLDH levels may allow liver injury caused by novel drugs to be detected in this population of patients.”

D-RSC is dedicated to developing tools to accelerate development of safe drugs for Duchenne, and monitoring safety of such drugs is important. PSTC is dedicated to the development of safety biomarkers, so collaboration between the two C-Path consortia accelerated development of this biomarker.

“At C-Path, we are constantly looking to work in partnership across our many consortia, and the GLDH biomarker qualification presented the perfect opportunity for such a collaboration between PSTC and D-RSC,” says John-Michael Sauer, PhD, Biomarker Program Officer and Executive Director of PSTC.

EMA support encourages the biomarker’s use in both nonclinical and exploratory clinical studies as a marker of hepatocellular liver injury, and indicates that this biomarker has strong potential for use in humans and warrants additional exploration and gathering of data.

Says C-Path President and CEO Martha A. Brumfield, PhD, “The data package submitted to EMA supporting use of this liver-injury biomarker, developed by PSTC and D-RSC in partnership, exemplifies successful cross-consortium collaboration at C-Path, and EMA’s support of this biomarker is a harbinger of future success for both consortia. Developing reliable biomarkers remains one of the most productive methods of aligning and streamlining research and regulatory processes.”

The Letter of Support for this biomarker is posted on the EMA website, and can also be accessed via the C-Path PSTC website.

 


c_path_logo
Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 with public and private philanthropic support from the Arizona community, Science Foundation Arizona, and the US 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 12 global, public-private partnerships that currently include over 1,450 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 www.c-path.org.

 

C-Path Contact:
Kissy Black
+1.615.298.1144
kissyblack@lotosnile.com

 

Critical Path for Parkinson’s Consortium 2017 Annual Meeting

The Critical Path for Parkinson’s (CPP) Consortium, cofounded by C-Path and Parkinson’s UK, held its second annual consortium member meeting on June 12 in Bethesda, MD. The meeting brought together scientists from the biopharmaceutical industry, academic institutions, and government agencies; people living with Parkinson’s; patient-advocacy associations; and U.S. Food and Drug Administration regulators to align on the collaborative ways to increase efficiency, safety, and speed in developing new therapies for Parkinson’s.

IMI SAFE-T and C-Path PSTC Obtain Regulatory Support for New Liver Safety Biomarkers

Oct 17, 2016

 

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IMI SAFE-T and C-Path PSTC Obtain Regulatory Support for New Liver Safety Biomarkers

US FDA and EMA Letters of Support Pave the Way for Clinical Qualification

 

The Innovative Medicines Initiative (IMI) SAFE-T (Safer and Faster Evidence Based Translation) Consortium and Critical Path Institute (C-Path) Predictive Safety Testing Consortium (PSTC) announced today that the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) each issued a Biomarker Letter of Support for new liver safety biomarkers investigated by the SAFE-T Drug-Induced Liver Injury Work Package and the PSTC Hepatotoxicity Working Group. The Drug-Induced Liver Injury Network (DILIN) in the US, an expert network established by The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), contributed their expertise to the research, as well as rare samples from individuals with severe liver injury.

The liver safety biomarkers—cytokeratin 18 (CK-18), high mobility group protein B1 (HMGB1), osteopontin, and macrophage colony-stimulating factor 1 receptor (MCSFR1, or CSF1R), are proteins that can be measured in human serum. Both FDA and EMA acknowledged that higher levels of these biomarkers in patients diagnosed with Drug-Induced Liver Injury (DILI) could indicate a risk for progression toward liver failure, which may result in death or the need for liver transplantation. DILI is an adverse drug reaction that has, for decades, been a major cause for late-stage failures in drug development and post-marketing withdrawals.

In addition, EMA considered results promising for serum biomarkers total HMGB1, total and caspase-cleaved keratin 18, miR-122, and GLDH in terms of possibly improving early prediction of liver injury in clinical trials with compounds having the potential to cause intrinsic liver toxicity, similar to paracetamol (acetaminophen).

The Letters of Support indicate the new biomarkers have potential for use in humans, which warrants additional exploration and data generation, and are intended to encourage scientists to collect additional data from nonclinical and exploratory clinical studies. With this milestone, in-depth research can continue toward the qualification of the new biomarkers for use in clinical trials on top of standard safety tests.

“Many current obstacles in drug development pose substantial scientific and logistical challenges to industry and public health that are impossible to tackle by individual companies or research organizations alone. Large scale public-private partnerships are an indispensable prerequisite to solve complex tasks such as development and qualification of new safety biomarkers, as exemplified by IMI’s SAFE-T and C-Path’s PSTC,” said Pierre Meulien, PhD, IMI Executive Director.

“The collaboration demonstrated by these specific IMI and C-Path programs has enabled this significant advance which encourages utilization of these novel liver biomarkers by sponsors. Acquiring more experience and data with these biomarkers will provide greater confidence and refinement in their utility, thereby assisting decision making within drug development programs and by regulatory authorities.” Martha A. Brumfield, PhD, President & CEO, Critical Path Institute.

”The success of the SAFE-T/PSTC collaboration nicely demonstrates the benefits of working together across public private partnerships on a global scale. Shared scientific enthusiasm, persistence, and team-spirit were the key foundation for this achievement”. Michael Merz, MD, Novartis Institutes for BioMedical Research, IMI SAFE-T project coordinator.

“The SAFE-T/PSTC collaboration represents the best collaborative science, bringing together experts from Europe and North America around the common goal of qualifying safety biomarkers. This relationship demonstrates how two public-private partnerships can work together in support of their members’ goals and visions.” John Michael Sauer, PhD, Critical Path Institute, PSTC Executive Director.

The FDA Letter of Support is posted on the FDA website, the EMA Letter of Support on the EMA website. Additionally, the documents can be accessed via the SAFE-T website, or via the C-Path PSTC website under the Regulatory Successes tab. The PSTC website includes a summary data package describing the studies that support the use of these liver safety biomarkers.

The research leading to these results has received support from the Innovative Medicines Initiative Joint Undertaking under grant agreement n° 115003, resources of which are composed of financial contribution from the European Union’s Seventh Framework Programme (FP7/2007-2013) and EFPIA companies’ in kind contribution.

About the organizations:

imi

The Innovative Medicines Initiative (IMI) is working to improve health by speeding up the development of, and patient access to, the next generation of medicines, particularly in areas where there is an unmet medical or social need. It does this by facilitating collaboration between the key players involved in healthcare research, including universities, pharmaceutical companies, other companies active in healthcare research, small and medium-sized enterprises (SMEs), patient organisations, and medicines regulators. This approach has proven highly successful, and IMI projects are delivering exciting results that are helping to advance the development of urgently- needed new treatments in diverse areas.
IMI is a partnership between the European Union and the European pharmaceutical industry, represented by the European Federation of Pharmaceutical Industries and Associations (EFPIA). Through the IMI 2 programme, IMI has a budget of €3.3 billion for the period 2014-2024. Half of this comes from the EU’s research and innovation programme, Horizon 2020. The other half comes from large companies, mostly from the pharmaceutical sector; these do not receive any EU funding, but contribute to the projects ‘in kind’, for example by donating their researchers’ time or providing access to research facilities or resources.
For more information, visit www.imi.europa.eu.


 

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Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 with public and private philanthropic support from the Arizona community, Science Foundation Arizona, and the US 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 12 global, public-private partnerships that currently include over 1,450 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 www.c-path.org.

For more information:

C-Path Contact:
Kissy Black
+1.615.298.1144
kissyblack@lotosnile.com
SAFE-T Contact:
Dominique Brees 
Novartis Institutes for BioMedical Research
dominique.brees@novartis.com

 

Critical Path for Parkinson’s Consortium achieves regulatory support in Europe for use of imaging biomarker in Parkinson’s disease clinical trials

Oct 10, 2016

 

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Critical Path for Parkinson’s Consortium achieves regulatory support in Europe for use of imaging biomarker in Parkinson’s disease clinical trials

 

London and Tucson, Ariz., October 10, 2016 — The Critical Path Institute (C-Path), in partnership with Parkinson’s UK, announced today that the European Medicines Agency (EMA) has issued a letter of support for Parkinson’s disease (PD). EMA is supporting the use of an imaging biomarker as a tool to help researchers select people in the earliest stages of PD to participate in clinical trials of new treatments for the condition.

The EMA issued a letter of support on Oct 7, 2016, supporting the use of neuroimaging biomarker dopamine transporter imaging as an exploratory biomarker for early PD. The public letter was issued to the Critical Path for Parkinson’s Consortium (CPP), in response to the consortium’s submission of data supporting the use of the biomarker.

Dopamine transporter activity, as assessed by single-photon emission computed tomography (SPECT) imaging, measures the expression of dopamine nerve terminal function in the living brain. Low levels of dopamine transporter binding serve as a marker of the loss of dopamine nerve terminals, a hallmark of PD. Use of this biomarker in patients at the time of early clinical presentation will help to identify patients who are likely to show clinical progression of motor symptoms.

The letter of support is a step along the way to CPP’s ultimate goal of achieving biomarker qualification with EMA and FDA. Why is EMA qualification significant? According to Dr. Arthur Roach, Director of Research at Parkinson’s UK, qualification would relieve trial sponsors of the burden of having to convince the regulators that the biomarkers are reliable and reproducible every time they run a trial. “Qualification could save both the regulators and sponsors a tremendous amount of time and money.” CPP’s executive director, Diane Stephenson, states: “Parkinson’s disease treatments are urgently needed, and shaving off time and cost serves to incentivize companies to invest in more trials…. More shots on goal mean more chances of getting approved drugs past the finish line.”

Professor Donald Grosset, University of Glasgow, a key global leader in Parkinson’s disease research that is contributing data to CPP, commented that embedding biomarkers in clinical trials, with support from regulatory agencies, could ultimately facilitate their use as both prognostic and therapeutic indicators. “This will all happen more quickly due to the significant progress we are making in sharing data across several major studies,” Grosset wrote. “This action from the EMA is certainly good news for the field.”

In 2015, the US Food and Drug Administration (FDA) issued a letter of support for this same biomarker and its application in drug development. These letters convey that the FDA and EMA recognize the potential value of a biomarker and encourage its further evaluation. A total of seven FDA and three EMA letters of support have been issued to C-Path.

Global regulatory agencies see value in compiling data from several sponsors in a noncompetitive setting, rather than receiving data from one sponsor at a time in support of biomarker qualification. Integration of data across different clinical trials and longitudinal studies is a core competency of C-Path and a key goal of the multinational CPP.

About the organizations:

pr-c-path-logo

C-Path (Critical Path Institute) is an independent, nonprofit organization established in 2005 with public and private philanthropic support from the Arizona community, Science Foundation Arizona, and the US 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 12 global, public-private partnerships that currently include over 1,450 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 www.c-path.org.


 

pr-c-path-logo

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.

C-Path Contact:
Kissy Black
+1.615.298.1144
kissyblack@lotosnile.com

 

Critical Path for Parkinson’s Consortium achieves regulatory support in Europe for use of imaging biomarker in Parkinson’s disease clinical trials

Oct 10, 2016

 

pr-c-path-logo gaain

 

Critical Path for Parkinson’s Consortium achieves regulatory support in Europe for use of imaging biomarker in Parkinson’s disease clinical trials

 

London and Tucson, Ariz., October 10, 2016 — The Critical Path Institute (C-Path), in partnership with Parkinson’s UK, announced today that the European Medicines Agency (EMA) has issued a letter of support for Parkinson’s disease (PD). EMA is supporting the use of an imaging biomarker as a tool to help researchers select people in the earliest stages of PD to participate in clinical trials of new treatments for the condition.

The EMA issued a letter of support on Oct 7, 2016, supporting the use of neuroimaging biomarker dopamine transporter imaging as an exploratory biomarker for early PD. The public letter was issued to the Critical Path for Parkinson’s Consortium (CPP), in response to the consortium’s submission of data supporting the use of the biomarker.

Dopamine transporter activity, as assessed by single-photon emission computed tomography (SPECT) imaging, measures the expression of dopamine nerve terminal function in the living brain. Low levels of dopamine transporter binding serve as a marker of the loss of dopamine nerve terminals, a hallmark of PD. Use of this biomarker in patients at the time of early clinical presentation will help to identify patients who are likely to show clinical progression of motor symptoms.

The letter of support is a step along the way to CPP’s ultimate goal of achieving biomarker qualification with EMA and FDA. Why is EMA qualification significant? According to Dr. Arthur Roach, Director of Research at Parkinson’s UK, qualification would relieve trial sponsors of the burden of having to convince the regulators that the biomarkers are reliable and reproducible every time they run a trial. “Qualification could save both the regulators and sponsors a tremendous amount of time and money.” CPP’s executive director, Diane Stephenson, states: “Parkinson’s disease treatments are urgently needed, and shaving off time and cost serves to incentivize companies to invest in more trials…. More shots on goal mean more chances of getting approved drugs past the finish line.”

Professor Donald Grosset, University of Glasgow, a key global leader in Parkinson’s disease research that is contributing data to CPP, commented that embedding biomarkers in clinical trials, with support from regulatory agencies, could ultimately facilitate their use as both prognostic and therapeutic indicators. “This will all happen more quickly due to the significant progress we are making in sharing data across several major studies,” Grosset wrote. “This action from the EMA is certainly good news for the field.”

In 2015, the US Food and Drug Administration (FDA) issued a letter of support for this same biomarker and its application in drug development. These letters convey that the FDA and EMA recognize the potential value of a biomarker and encourage its further evaluation. A total of seven FDA and three EMA letters of support have been issued to C-Path.

Global regulatory agencies see value in compiling data from several sponsors in a noncompetitive setting, rather than receiving data from one sponsor at a time in support of biomarker qualification. Integration of data across different clinical trials and longitudinal studies is a core competency of C-Path and a key goal of the multinational CPP.

About the organizations:

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C-Path (Critical Path Institute) is an independent, nonprofit organization established in 2005 with public and private philanthropic support from the Arizona community, Science Foundation Arizona, and the US 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 12 global, public-private partnerships that currently include over 1,450 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 www.c-path.org.


 

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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.

C-Path Contact:
Kissy Black
+1.615.298.1144
kissyblack@lotosnile.com

The Michael J. Fox Foundation for Parkinson’s Research Joins Multinational Critical Path for Parkinson’s Consortium

May 19, 2016

 

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The Michael J. Fox Foundation for Parkinson’s Research Joins Multinational Critical Path for Parkinson’s Consortium

  • Consortium, led by Parkinson’s UK and the Critical Path Institute, aggregates and analyzes study data to develop a model for quantitative characterization of Parkinson’s progression
  • This asset would enrich the design of trials for novel therapies
  • Consortium will work with international regulators for acceptance of a quantitative model to streamline review processes

 

NEW YORK, LONDON AND TUCSON (May 18, 2016) — The Michael J. Fox Foundation for Parkinson’s Research (MJFF), with Parkinson’s UK and the Critical Path Institute (C-Path) in Tucson, Arizona, announce that MJFF has joined the Critical Path for Parkinson’s Consortium (CPP). The consortium aims to create a quantitative model of Parkinson’s progression from its earliest stages that will allow researchers to optimize clinical trial design for faster and more effective testing of novel therapies.

Parkinson’s UK and C-Path launched the CPP in October 2015, and seven major pharmaceutical companies, the Parkinson’s Disease Foundation, the Davis Phinney Foundation and Cure Parkinson’s Trust have subsequently signed on to the initiative.

“More therapies with potential to slow or stop Parkinson’s progression in its early stages are moving through clinical trials,” said MJFF Senior Vice President of Research Programs Mark Frasier, PhD. “A quantitative characterization of that early progression would help evaluate the impact of these treatments and usher effective therapies to the people who need them.”

Currently clinical trials for potential new therapies are time-consuming and costly. Due to the variability in Parkinson’s symptoms and progression and the lack of objective biomarkers, trials must enroll hundreds of patients and follow them for many months if not years. Development of a quantitative model of Parkinson’s progression, potentially combining both biological and clinical factors, may allow for efficient trials that aim to treat the right patient with the right drug at the right time.

To create the model, C-Path will standardize and aggregate data from several large-scale Parkinson’s studies — including the MJFF-led Parkinson’s Progression Markers Initiative (PPMI) comprising nearly 1,000 participants, which has made its data available in real time since it launched in 2010. C-Path will apply modeling and computational tools using the pooled database to produce the quantitative progression model — which will be submitted to the U.S. Food and Drug Administration and the European Medicines Agency for feedback — with the ultimate goals of saving time and money and providing a platform for the best chance of a positive outcome.

In future stages, the consortium plans to develop a clinical trial simulation platform. Drug developers make educated guesses on the number of volunteers to enroll, how long to follow them and how to evaluate therapeutic impact. The simulation platform will inform trial sponsors of the required parameters when planning a trial and allow them to enter data on individual participants to determine the number of subjects and the length of the trial required. For example, with a study population of “fast progressers” as defined by the CPP progression model, sponsors may be able to plan a shorter trial. Such a tool would help drug developers plan efficient studies and avoid false negative results.

“We are very pleased to welcome The Michael J. Fox Foundation to this important collaboration,” said Martha A. Brumfield, president and CEO of C-Path. “The Foundation’s unwavering commitment to helping patients with PD, their years of experience in articulating the patient perspective, and their research priorities will enrich the work of the Critical Path for Parkinson’s Consortium.”

Arthur Roach, PhD, director of research at Parkinson’s UK, the principal funder of the consortium, says: “Parkinson’s UK is determined to get better treatments to people living with this devastating condition in years, not decades. A key part of this is joining forces with regulators, researchers, pharma and medical research charities such as MJFF to maximize the benefit of data-sharing and learnings. Working with CPP partners will play a crucial role in helping develop and evaluate the effectiveness of potential new therapies, which could one day lead us to a cure for Parkinson’s.”

The PD data platform will serve as the resource for advancing the regulatory science goals of CPP, including qualification of drug development tools, and eventually will be made available to qualified researchers. Through CPP analyses or independent investigation, the standardized, aggregated data may allow researchers to identify events that underlie trajectories of decline and biomarkers or other determinants of early Parkinson’s disease.

About the organizations:

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As the world’s largest nonprofit funder of Parkinson’s research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson’s disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson’s patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding more than $600 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson’s research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson’s disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson’s awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world. For more information, visit us on the Web, Facebook, Twitter, and LinkedIn.


 

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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.


 

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Established in 2005 as a nonprofit organization, C-Path was formed to deliver on the vision of FDA’s Critical Path Initiative. C-Path is committed to improving human health and well-being by developing new technologies and methods to accelerate the development and review of medical products. An international leader in forming collaborations around this mission, C-Path has established twelve global, public-private partnerships that include more than 1,400 scientists from regulatory and other government agencies, academia, patient advocacy organizations, and 41 major pharmaceutical companies. C-Path has headquarters in Tucson, AZ, and has been funded by public and private philanthropic support from the University of Arizona, Science Foundation Arizona (SFAz), the US Food and Drug Administration (FDA), and the Tucson community. For more information, visit www.c-path.org.

C-Path Contact:
Kissy Black
+1.615.298.1144
kissyblack@lotosnile.com

 

Applying Regulatory Science to Neonates: Second Annual Scientific Workshop at European Medicines Agency (EMA)

Date:  September 12-13, 2016

Location: European Medicines Agency, Canary Wharf, London

Sponsors: Critical Path Institute and European Medicines Agency

The International Neonatal Consortium (INC) organized a two day workshop at the European Medicines Agency (EMA) on September 12-13, 2016.  Focused on applying regulatory science to neonates, INC members explored approaches that family and neonatal nursing organizations can take to embrace a research  culture.  Workshop participants also provided an update on INC’s progress in the first year and identified additional initiatives to accelerate the development of safe and effective therapies for neonates.

The workshop was free and open to the public.

The proceedings from this meeting can be viewed here.