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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commonalities in clinical trials for Alzheimer’s disease and neurodegenerative disease – NIA-AA and IWG Criteria – FDA Draft Guidance for Early Stage Alzheimer’s Assessment in Drug Development – CMS coverage decision for amyloid imaging/CED • AARR Efforts • Biomarker Initiatives and CAMD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 4 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 5 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 6 Modernizing Diagnosis of Alzheimer’s 2011 7 Modernizing the Diagnosis of Alzheimer’s Normal Adapted from Sperling et al. 2011 8 Alzheimer’s disease FDA Draft Guidance: Drug Development of Early Stage Alzheimer’s 9 10 AARR Oct 2014: Cognitive and Functional Outcome Measures for AD • CDR-SB: Strengths/ weaknesses – James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. 11 Collaboration for Alzheimer’s Prevention 12 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 13 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 14 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 15 16 17 18 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.. 19 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 20 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 21 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 22 23 23 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 24 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. 25 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 26 27 alz.org