Transcript Presentation - American Society for Experimental NeuroTherapeutics

Alzheimer’s Association Efforts
February 2014
Maria C. Carrillo, Ph.D.
Vice President
Medical & Scientific Relations
Alzheimer’s Association
American Society for Experimental NeuroTherapeutics | 16th Annual Meeting
Disclosure
NO DISCLOSURES
American Society for Experimental NeuroTherapeutics | 16th Annual Meeting
Learning Objectives
• Federal Agency Guidelines and Decisions
increase awareness for the need to synergize
activities across the ecosystem.
• Alzheimer’s Association initiatives in response
to recent federal statement and decisions
American Society for Experimental NeuroTherapeutics | 16th Annual Meeting
Overview
• Activities driving need for commonalities in clinical
trials for Alzheimer’s disease and neurodegenerative
disease
– NIA-AA and IWG Criteria
– FDA Draft Guidance for Early Stage Alzheimer’s
Assessment in Drug Development
– CMS coverage decision for amyloid imaging/CED
• AARR Efforts
• Biomarker Initiatives and CAMD Collaborations
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Reality of Alzheimer’s
• More than 5 million
people are living with
Alzheimer’s disease.
⁻ Including 200,000
under the age of 65
with younger-onset.
• Every 68 seconds,
someone develops
Alzheimer’s in US; by
2050, will be every 33
seconds.
2013 Facts & Figures
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Alzheimer’s and Mortality
• 6th leading cause of
death across all ages
• 5th leading cause of
death for those aged 65
and older
• Only cause of death
among the top 10 in
America without a way
to prevent, cure or even
slow its progression.
Alzheimer's
Disease
68%
• Change in the
Number of
Deaths: 68%
• Between 2000
and 2010
Breast Cancer
-2%
Prostate
Cancer Heart Disease
-8%
-16%
Stroke
-23%
HIV
-42%
Based on preliminary 2010
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Modernizing Diagnosis of Alzheimer’s
2011
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Modernizing the Diagnosis of
Alzheimer’s
Normal
Adapted from Sperling et al. 2011
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Alzheimer’s disease
FDA Draft Guidance: Drug Development
of Early Stage Alzheimer’s
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AARR Oct 2014: Cognitive and
Functional Outcome Measures for AD
• CDR-SB: Strengths/ weaknesses
– James Galvin, M.D.
• Eisai Composite Score
– Veronika Logovinsky, M.D., Ph.D.
• J&J Statistical Framework
– Nandini Raghavan, Ph.D.
• Retrofitting existing tools for
MCI/Preclinical AD
– Alette Wessels, Ph.D.
• CAMD & Biomarker Qualification of
Cognitive Endpoints
– Diane Stephenson, Ph.D.
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Collaboration for Alzheimer’s
Prevention
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Prevention Trial Early Detection
Outcome Measures
• A4 Computerized Cognitive Composite
– CogState Card Playing tasks on iPAD
– Face-name Association and Pattern Separation
• API Composite Cognitive Score based on ROS/DIAN data
• TOMORROW Operationalization of the NIA-AA diagnostic
criteria for "MCI due to AD“ required
• - Specific cognitive instruments were selected and decision
rules developed to reliably capture the relevant domains
affected in cognitive decline and in MCI-AD
• DIAN – Cogstate based testing, some additional
neuropsychological tests
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Alzheimer’s Association Global
Biomarkers Standardization Consortium
• WW-ADNI
• AA Hippocampal Volumetry
• AA CSF QC Program
– IRMM/ IFCC Project
• Amyloid Imaging
– SNMMI – Alz Assoc
Health.rush.edu
• CAMD Biomarker Qualification Efforts
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Synergy with Current Efforts:
CAMD
• Biomarker qualification vMRI
– Frisoni/Jack standardization effort
– Jack (2011) proposed 3rd party oversight
committee
• Biomarker qualification CSF
– AA QC Program demonstrates stability
– IRMM Project to certify materials/methods
• GAAIN & CAMD Data sharing effort
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Recent CMS Decision on Amyloid
Imaging
• CMS decision:
– Allow Coverage with Evidence
Development (CED) to would
improve diagnostic accuracy
and certainty in challenging
cases.
– Discussions now around
infrastructure needed for CED,
learning from NOPR
• Alzheimer’s Association
disagreement with blanket non
coverage decision..
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Image adapted from Clark et al. (2011) JAMA 305(1).
AICED Workgroup
Executive Committee Members:
• Brian Carey
• Charles Apgar - ACR
• Constantine Gatsonis, PhD – Brown Univ.
• Lisa Baird- WMIS
• Steve Salloway, MD- Brown Univ.
• Peter Herscovitch, MD- SNMMI
• Bruce Hilner, MD- Virginia Univ.
• Gary Dillehay, MD- Northwestern
• Barry Siegel, MD- Washington Univ.
• Satoshi Minoshima, MD, PhD- UWash
• Keith Johnson, MD- Harvard MGH
• Maria Carrillo, PhD- Alzheimer’s Association
AICED ISG Members:
• Gail Rodriguez- Medical Imaging
• Mark Mintun- Avid
• Steve Sandor- Piramal
• Meridith Johnson- GE Healthcare
• Thom Tulip-Navidea
• Antonella Favit-Vanpelt
• Susan De Santi- GE Healthcare
• Craig Hunter- Eli Lilly
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AICED Scientific Advisory Board (SAB)
Members:
• Satoshi Minoshima- MD, PhD- UWash
• Gil Rabinovici, MD- UCSF
• Barry Siegel, MD- Washington Univ.
• Bruce Hilner, MD- Virginia Univ.
• Keith Johnson, MD (Co-Chair) – Harvard MGH
• Jason Karlawish, MD
• Norman Foster, MD- Univ. of Utah
• Kevin Donahoe, MD – Beth Israel
• Steve Salloway, MD – Brown University
• Peter Herscovitch, MD- SNMMI
• Constantine Gatsonis, PhD – Brown Univ.
• Maria Carrillo, PhD- Alzheimer’s Assoc.
FDA Fellowship in the Division of
Neurology Products
• New liaison position approved by FDA, sponsored
through the Reagan-Udall Foundation
• Fellow will receive input from various stakeholders in
Alzheimer’s drug development and share that input
with the FDA.
• Liaison for stakeholders who are developing or
conducting clinical trials by giving them greater insight
into FDA strategic thinking and current guidelines
• Launch Spring/ Summer 2014
• First of its kind disease-specific
fellowship
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Biomarkers Across Neurodegenerative
Diseases (BAND)
• GOAL: To stimulate analyses across
Alzheimer’s and Parkinson’s to
engage in further data analysis of
existing research information to
advance biomarker discovery,
standardization of assays, genetic
profiles, and imaging modalities.
• Leverage data and/or samples from
ADNI and PPMI
• First of its kind collaboration with
Michael J. Fox Foundation & G.
Weston Garfield Foundation
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Alzheimer's Disease Steering Committee
Name
Affiliation
Neil Buckholtz
NIH/NIH
Mike Hutton
Lilly
Mike Decker
AbbVie
Xiaoming Guan
GSK
Tim Harris
Biogen Idec
Maria Carrillo
Alzheimer's Association
Walter Koroshetz
NIH/NINDS
Nick Kozauer
FDA
Pat Walicke
NIH/NINDS
Co-Chairs
Industry
Academia,
Government, &
Non-profit
EC Liaison
• Richard Hodes, NIH/NIA
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White Paper

Detailed
Project Plan
Projected AMP funding
contributions
Disease area
Total project
funding ($M)
Total NIH
funding ($M)
Total
industry
funding ($M)
AD
129.5
67.6
61.9
T2D
58.4
30.4
28.0
RA/SLE
41.6
20.9
20.7
Total
229.5
118.9
110.6
Industry is also providing AMP with additional in-kind
contributions, e.g., clinical trials, drug, tracer, databases, etc.
© The Boston Consulting Group, Inc.
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SUMMARY
Pathway to Common Clinical Trial Measures
• NIA-Alzheimer’s Association guidelines for
“preclinical” stages of Alzheimer’s disease
• FDA draft guidance on Drug Development for early
stages of Alzheimer’s disease
• CMS Decisions will need to be considered
• Collaborative Environment
• URGENCY
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alz.org