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.

 


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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.

 

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

 

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:

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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:

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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

 

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:

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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.

C-Path’s Polycystic Kidney Disease Outcomes Consortium Secures EMA Qualification Opinion for Enrichment Biomarker in ADPKD

November 13, 2015

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C-Path’s Polycystic Kidney Disease Outcomes Consortium Secures EMA Qualification Opinion for Enrichment Biomarker in ADPKD

TUCSON, Ariz., November 13, 2015 – The Critical Path Institute (C-Path) announced today that the European Medicines Agency (EMA) rendered a positive qualification opinion to C-Path’s Polycystic Kidney Disease Outcomes Consortium (PKDOC) for total kidney volume (TKV) as a prognostic biomarker to select patients for clinical trials of new therapies for Autosomal Dominant Polycystic Kidney Disease (ADPKD).

ADPKD is a debilitating genetic disease affecting approximately 600,000 Americans and 12 million people worldwide. There is only one medication developed to treat ADPKD, called tolvaptan, which has been approved in Europe, Japan, and Canada, but has not yet been approved in the United States.

TKV is a measurement of the impact of ADPKD on the size of the kidneys and is considered to be predictive of a future decline in kidney function. The EMA opinion states, “CHMP [Committee for Medicinal Products for Human Use] supports baseline total kidney volume, in combination with patient age and eGFR [estimated glomerular filtration rate] as a prognostic biomarker to identify patients likely to experience a progressive decline in renal function, as characterized by a decline in eGFR or progression to end-stage renal disease.

“From the data provided it is reasonable to expect that baseline TKV can predict disease progression and is a biomarker valuable for risk stratification.”

”This qualification, along with a similar one from FDA, confirms the relationship between TKV and ADPKD disease progression, and will help in the design of clinical trials for new therapies for ADPKD” says C-Path Chief Operating Officer and PKDOC Co-Director Steve Broadbent.

The PKDOC created a Clinical Data Interchange Standards Consortium (CDISC) data standard for ADPKD and used it to remap the data from several patient registries and observational studies. The database was then used to develop a joint model linking baseline TKV with clinical outcomes.

About PKDOC:
The PKDOC is a successful collaboration between C-Path, the PKD Foundation, CDISC, four leading academic medical centers (Tufts University, University of Colorado Denver, Emory University, and Mayo Clinic), and three pharmaceutical companies. Its mission is to develop tools and promote research that will lead to the discovery of treatments for PKD and improve the lives of all it affects. The consortium is led by C-Path and funded through a grant from the PKD Foundation and philanthropic donations.

About the organizations:

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 1,300 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.


 

The PKD Foundation is the only organization in the United States solely dedicated to finding treatments and a cure for polycystic kidney disease (PKD) to improve the lives of those it affects. This is done through promoting programs of research, education, advocacy, support, and awareness on a national level, along with direct services in local communities across the country. Their vision is that one day no one will suffer the full effects of PKD. Visit pkdcure.org to learn more about PKD and the Foundation.

C-Path Contact:
 Kissy Black
+1.615.298.1144
kissyblack@lotosnile.com
PKD Foundation Contact:
 Angela Connelly
816.931.2600 ext. 212
angelac@pkdcure.org

 

PD Imaging Biomarker Team at European Medicines Agency (EMA)

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Experts of the Parkinson’s disease imaging biomarker team participated in a Scientific Advice meeting at the European Medicines Agency on October 7th 2015. The purpose of this meeting was to review the evidence provided to the Agency that supports the use of Molecular Imaging of the Dopamine Transporter as an enrichment biomarker in clinical trials targeting subjects at first signs and symptoms of PD. Team members from left: Steve Arneric, C-Path; Derek Hill, Ixico; Klaus Romero, C-Path; Patricia Cole, Takeda; Arthur Roach, Parkinson’s UK; Ken Marek, MNI; Diane Stephenson, C-Path; Dawn Matthews, ADm Diagnostics; Mark Gordon, Boerhinger Ingelheim.

Critical Path Institute Secures Regulatory Support For Skeletal Muscle Safety Biomarkers

April 13, 2015
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Critical Path Institute Secures Regulatory Support For Skeletal Muscle Safety Biomarkers

FDA and EMA Letters of Support Suggest Valuable Research Potential

TUCSON, Ariz., April 8, 2015 – The Critical Path Institute (C-Path) announced today that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have issued Biomarker Letters of Support for four skeletal muscle safety biomarkers identified and evaluated by C-Path’s Predictive Safety Testing Consortium (PSTC)’s Skeletal Myopathy Working Group. This is the second time the two agencies have provided a Biomarker Letter of Support to PSTC in the past six months, following FDA and EMA support for the kidney safety biomarkers osteopontin (OPN) and neutrophil gelatinase-associated lipocalin (NGAL).

“This Letter of Support intends to encourage scientists to collect data from exploratory studies, which may lead to qualification of these types of biomarkers,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “We are optimistic about how this effort will further advance biomarker development.”

The skeletal safety biomarkers – myosin light chain (Myl3), skeletal muscle troponin I (sTnI), fatty acid binding protein 3 (FABP3), and creatine kinase muscle type (CK-M, the homodimer CK-MM) – are proteins that can be measured in plasma or serum. Higher levels of these biomarkers could indicate that skeletal muscle injury is occurring.

“The development of accurate, reliable biomarkers continues to be one of the most productive methods of aligning and streamlining the research and regulatory processes,” says C-Path President and CEO Martha A. Brumfield, PhD. “The support of these skeletal muscle safety indicators by the FDA and EMA is an encouraging development in the PSTC’s larger mission of facilitating scientific validation and regulatory qualification of novel safety biomarkers.”

Each agency’s Letter of Support for Myl3, sTnI, FABP3, and CK-M is intended to encourage the biomarkers’ use in both nonclinical and exploratory clinical studies as markers of skeletal muscle injury. Combined FDA and EMA support indicates that these biomarkers have strong potential for use in humans and warrants additional exploration and gathering of data. With this milestone, work will continue in earnest on the qualification of Myl3, sTnI, FABP3, and CK-M for use in clinical trials.

Letters of Support for these skeletal muscle biomarkers are posted on the FDA DDT website and the EMA website, and can also be accessed via the C-Path PSTC website, 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.

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

 

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.