6th Annual Fall Symposium "Conquering Alzheimer's Disease" presentation

CONQUERING ALZHEIMER’S DISEASE HOPE ON THE HORIZON Howard Fillit, MD | Executive Director & Chief Science Officer All Slides © 2015 Alzheimer’s Drug Discovery Foundation. Not for distribution.

WHAT WE ARE FACING

2

$226 billion annual cost in U.S.

3

28 million Baby Boomers expected to develop Alzheimer’s

4

ADDF’S APPROACH

5

HOW ADDF IS UNIQUE

% RESEARCH FUNDING BY AREA

Drug Discovery & Clinical Trials Molecular Pathogenesis Diagnosis & Assessment Epidemiology Care & Support

6

HOW ADDF IS UNIQUE % of total budget dedicated to drug discovery, clinical trials & diagnostics

ADDF 95%

$

7.3

MILLION

Alzheimer’s Association 5.6%

$

6.8

MILLION

Cure Alzheimer’s Fund 10.2%

$

650,000 7

WHAT WE KNOW: Our Strategy

8

AGING

IS THE LEADING RISK FACTOR FOR ALZHEIMER’S DISEASE

9

Genetics & Epigenetics

Neuronal Energy Failure

Vascular Disease

Oxidation

Inflammation

NEURONAL DYSFUNCTION AND DEATH

Damaged Proteins

ALZHEIMER’S DEMENTIA

10

PORTFOLIO OF DRUG TARGETS

2014 ADDF Investments

EPIGENETICS & OTHER

11

12

+

YEARS

$

2

BILLION

12

REASONS FOR HOPE: Clinical Trials

ADDF FUNDING BY STAGE OF DISCOVERY & DEVELOPMENT

14

ADDF FUNDING BY STAGE OF DISCOVERY & DEVELOPMENT

Preclinical

Clinical

15

1999-2009

86.6%

13.4%

16

2015 YTD

54.6%

45.4%

17

120

+

Treatments Now in Clinical Trials

18

20

%

ADDF Supported

19

AMYVID ACCELERATING TRIALS

PLACEBO TREATMENT

VACCINE TREATMENT

Has enabled more scientifically rigorous clinical trials • Disease starts ~20 years before symptoms begin • Anti-amyloid prevention clinical trials are underway

from Biogen study of Aducanumab 20

ADDF NOVEL DRUGS IN CLINICAL TRIALS

Neuroprotection Oxidation

Neuroendocrine aging

Systemic Inflammation

Failed Mitochondrial Energetics dysfunction

Epigenetic changes

Hypoxia

Neuroinflammation

BBB dysfunction

CELL DEATH

CELL SURVIVAL Misfolded Proteins

Neuroprotection

Frank Longo, MD, PhD Drug: LM11A-31 Phase 2 Clinical Trial

23

ADDF FUNDING OF PHARMATROPHIX

2000-2004 $521,517

2004-2005 $160,177

2005-2009 $195,000

2008-2009 $300,000

2013-2014 $250,000

2015 $500,000

2009-2010 $180,000 24

Neuroinflammation

Healthy Control

2007

Alzheimer’s

Neuroinflammation

Philip Haydon, PhD

Paolo Pevarello, PhD

GliaCure, Boston

Axxam SpA, Milan

Drug: GC021109 Phase 1 Clinical Trial

Drug: AXX00179871 Preclinical 26

Epigenetics 27

Epigenetics

Tamara Maes, MD Oryzon Genomics, Barcelona Drug: ORY-2001 Preparing for Phase 1

28

REPURPOSING FDA-APPROVED DRUGS

3

+

YEARS

$

2

MILLION

29

Neuroprotection

Ana Pereira, MD

Michela Gallagher, PhD

The Rockefeller University

John Hopkins University & Agenebio

Drug: Riluzole Phase 2 Clinical Trial

Drug: AGB10 Phase 3 Clinical Trial 30

Neuroinflammation

Clive Holmes, PhD University of Southampton, UK Drug: Etanercept Phase 2 Clinical Trial

31

Neuronal Energy Failure

Paul Edison, MD, PhD

Jeffrey L. Cummings, MD

Imperial College London

The Cleveland Clinic

Drug: Liraglutide Phase 2 Clinical Trial

Drug: Rasagiline Phase 2 Clinical Trial 32

TOOLS FOR PREVENTION

Preventing Alzheimer’s Disease Genetics & Gender

Environment

Substance Abuse

Head Trauma

Exercise

Medical Risk Factors

Major Surgery

Psychological Factors

Diet

34

Rating the Evidence for:

• Nutrition, Natural Products, and Supplements • Health Management & Drugs • Potential Risks

35

WE CAN CONQUER ALZHEIMER’S

Howard Fillit, MD

HFillit@AlzDiscovery.org

212.901.8000

All Slides © 2015 Alzheimer’s Drug Discovery Foundation. Not for distribution.