Alzheimer s Disease: A Priority of the Innovative Medicines Initiative. Elisabetta Vaudano PhD DVM

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1 Alzheimer s Disease: A Priority of the Innovative Medicines Initiative Elisabetta Vaudano PhD DVM

2 Alzheimer s Disease Facts & Challenges Alzheimer s Disease in Europe The world's population is ageing. There are an estimated 35.6 million people in the world with dementia, and this number is expected to increase to 65.7 million in 2030 and million in Approximately 60% of people with dementia live in developing countries and numbers are increasing.

3 Alzheimer s Disease Facts & Challenges There are no drug treatments available that can provide a cure for Alzheimer's disease. Medicines have been developed that can improve symptoms, or temporarily slow down their progression, in some people. The last drug approved by the FDA for Alzheimer's disease was memantine in 2003.

4 Alzheimer s Disease Facts & Challenges Currently there is no way as yet to predict who will respond and who will get little or no benefit from one of the five drugs approved to treat Alzheimer's disease. The list of drugs that have been promising in mouse models of Alzheimer's but disappointing in humans is long. Some have been too toxic, while others have failed to outperform a sugar pill.

5 Alzheimer s Disease Facts & Challenges Research suggests the best targets for exploring treatments are individuals who do not have full-blown Alzheimer's disease, but experience mild symptoms. It is clear that in most if not all health systems dementia is under-diagnosed, and when diagnosis occurs this is typically at a relatively late stage in the disease process. Signs of Alzheimer's may develop in the brain 10 to 20 years before any symptoms begin.

6 Alzheimer s Research in Europe

7 Individuals with Alzheimer s Need Innovative Treatments The right drug To the right individual At the right stage At the right dose Need for a for Real Innovation to Happen New Research Tools, Models and Methodologies to Translate from Bench to Bed and Back New Research Collaborative Models

8 All Stakeholders Must be Involved DATA Regulators Patient Groups = best approach

9 Innovative Medicines Initiative: Joining Forces in the Healthcare Sector

10 What is IMI doing for Alzheimer s? Prediction of Cognitive Properties of New Drug Candidates for Neurodegenerative Diseases in Early Clinical Development European Medical Informatics Framework Alzheimer s Europe is a full consortium member in both projects

11 PHARMACOG Prediction of Cognitive Properties of New Drug Candidates for Neurodegenerative Diseases in Early Clinical Development

12 PHARMACOG Unsuccessful Alzheimer s Drugs in Development drugs failed PhRMA 2012 RESEARCHING ALZHEIMER S MEDICINES: SETBACKS AND STEPPING STONES

13 PHARMACOG in a nut-shell TOOLS AND METHODS NEW TREATMENTS FOR AD SYMPTOMS THE BEST TREATMENT AND DOSE NEW TREATMENTS TO MODIFY AD Cognitive testing Blood analysis Brain talk (EEG + electrophysiology) Brain scans

14 PHARMACOG EEG is a simple not invasive, cheap and translational tool for AD research.

15 EMIF European Medical Informatics Framework

16 EMIF EHRs Access to information on 40 million patients EMIF PLATFORM Secure (privacy, legal) modular platform Metabolics research on > 20,000 obese & T2DM subjects EMIF METABOLICS EMIF AD AD research on 10- times more subjects than ADNI

17 EMIF-AD AD diagnosis is too late for modify its course Available biomarkers are of limited help for early diagnosis or to follow disease progression

18 EMIF-AD: Towards a Better Understanding of AD in Europe An understanding of the variability of AD onset and progression in the general population (EHRs) and in individuals with mild cognitive impairment Effects of constitutional, environmental and disease-specific risk factors Genetic, epigenetic and metagenomic susceptibility markers Identification of high-risk individuals and development of an algorithm for a diagnostic test Possibly novel targets for future therapeutic interventions

19 EMIF-AD: First Results Preclinical AD can be defined by CSF biomarkers Is common in individuals aged at least 65yrs Is associated with increased risk of cognitive decline Is associated with increased risk of progression to clinical AD Is associated with increased mortality

20 Ongoing IMI Efforts for Alzheimer s Research EMIF-AD The right drug To the right patient At the right stage At the right dose PHARMACOG PHARMACOG

21 IMI Efforts for Alzheimer s Research: Future Activities Literature Open source data Aetionomy hypothesis In-house data from PD and AD cohorts towards a better classification of individuals with Alzheimer s and Parkinson s Disease based on mechanism of disease: Biological pre-validation in existing cohorts Optimise benefit risk of therapies (remove individuals with no chance of responding) Clinical validation in a prospective cohort Alzheimer s Europe is a full consortium member Validation in independent cohorts already recruited Identify individuals likely to benefit from the same new therapy despite different clinical features

22 IMI Efforts for Alzheimer s Research: Future Activities A potential new topic for IMI: To establish a public-private partnership to conduct a standing adaptive trial that will enable disease modification in preclinical and early Alzheimer's disease, reducing the costs and time to initiate clinical trials of therapeutic agents designed to stem the rising tide of dementia 1. Whom should we treat? 2. With what should we treat? 3. How do we observe a potential treatment effect? 4. When should we initiate the treatment intervention?

23 IMI Efforts for Alzheimer s Research: IMI2

24 IMI Efforts for Alzheimer s Research: IMI2 Alzheimer s Disease is one of the Research Areas of the Drafted IMI2 Strategic Research Agenda

25 Questions?