Anavex Life Sciences Corp. · NASDAQ:AVXL

Anavex Life Sciences — Investor Presentation (2016)

33 slides from Anavex Life Sciences's investor presentation, Jun 15, 2016

  1. Slide 1: Corporate Presentation

    NASDAQ: AVXL Corporate Presentation Christopher U. Missling, PhD President & CEO 2016

  2. Slide 2: Safe Harbor

    Safe Harbor This presentation contains forward-looking statements made within the meaning of the Private Securities Litigation Reform Act of 1995 by Anavex™ Life Sciences Corp. and its representatives. These statements can be identified by introductory words such as ``expects,'' ``plans,'' ``intends

  3. Slide 3: Anavex Mission

    Anavex™ Mission Developing Differentiated Therapeutics for the Treatment of Neurodevelopmental and Neurodegenerative Diseases 3

  4. Slide 4: Convergence of Pathological and Genetic Lesions in All Neurodegenerative Diseases

    Convergence of Pathological and Genetic Lesions in All Neurodegenerative Diseases: ER-Mitochondria Axis Disruption 4 Sources: Miller et al. Trends in Neurosciences, March 2016, Vol. 39, No. 3; Vacca et al. Neuroscience and Biobehavioral Reviews 46 (2014) 202–217; Meli et al. NATURE COMMUNICATIONS |

  5. Slide 5: ER-Mitochondria Axis Disruption … Sigma-1R Restores Association

    Common in All Neurodegenerative Diseases: ER-Mitochondria Axis Disruption … Sigma-1R Restores Association … Disease situation Normal situation ER Mitochondrion ANAVEX 2-73 ANAVEX 2-73 Endoplasmic reticulum (ER)-Mitochondria associations are disrupted in neurodegenerative diseases …# … Sigma-1R resto

  6. Slide 6: ANAVEX 2-73: Confirmed Targeted Indications

    ANAVEX™ 2-73: Confirmed Targeted Indications: From Rare Disease Indication to Largest CNS Indication … Rett Syndrome (RTT) Alzheimer’s Disease (AD) Rare neurodevelopmental disease Neurodegenerative disease Preclinical validation, RettSyndrome.org  Clinical validation Phase 2a  Planning blinded con

  7. Slide 7: Neurodegenerative Disease Pipeline

    Neurodegenerative Disease Pipeline PRECLINICAL PHASE 1 PHASE 2 PHASE 3 ANAVEX™ 2-73 ALZHEIMER’S COGNITION IN NEUROPSYCHIATRIC EPILEPSY PARKINSON’S MULTIPLE SCLEROSIS (MS) RARE DISEASE, RETT SYNDROME ANAVEX™ 3-71 (AF710B) FRONTOTEMPORAL DEMENTIA (FTD) ALZHEIMER’S PARKINSON’S ANAVEX™ 1-41 DEPRESSION S

  8. Slide 8: Positive Preclinical Data of ANAVEX 2-73 in Rett Syndrome

    Positive Preclinical Data of ANAVEX™ 2-73 in Rare Disease: Rett Syndrome What is Rett Syndrome?  Rare non-inherited genetic postnatal progressive neurodevelopmental disorder  Caused by mutation of MECP2 gene  Occurs almost exclusively in girls and leads to severe impairments  One in 10,000 to 15

  9. Slide 9: Assessment of ANAVEX 2-73 in a MECP2 Rett Syndrome Model

    Assessment of ANAVEX 2-73 in a MECP2 Rett Syndrome Model  Administration of ANAVEX 2-73 results in both significant and dose related improvements in an array of behavioral paradigms in the MECP2 HET Rett syndrome disease model  Taken together, these behavioral paradigms measure different aspects o

  10. Slide 10: Clasping as a Behavioral Paradigm

    Clasping as a Behavioral Paradigm 100 Clasping at 8 weeks 90 p<0.001 Normal Impaired 80 *** Clasping Hindlimbs (%) 70 60 50 p<0.05 40 * 30 20 10 0 Mecp2_WT Vehicle Mecp2_HET Vehicle Mecp2_HET AV2-73 (10 mg/kg) Mecp2_HET AV2-73 (30 mg/kg) 100 Clasping at 12 weeks 90 p<0.01 80 Clasping Hindlimbs (%) 7

  11. Slide 11: Multiple Behavioral Rett Syndrome Paradigms Demonstrate Significant Improvement

    Multiple Behavioral Rett Syndrome Paradigms Demonstrate Significant Improvement Startle: Wild type (WT) mice have a higher startle response compared to impaired mice: AV2-73 treated mice showed a significant increase in startle response compared to vehicle- treated mutant mice Rotarod: Motor Coordin

  12. Slide 12: Anti-Depressant and Anti-Anxiety Effect of ANAVEX 2-73

    Anti-Depressant and Anti-Anxiety Effect of ANAVEX™ 2-73 in Porsolt Swim Test (PST) and in Open Field Test No observed “sedative” effect of ANAVEX 2-73  Effect of ANAVEX2-73 on immobility time on  Effect of ANAVEX2-73 on the number of PST. P<0.01, *p<0.05 and **p<0.01 for 50 and crosses (motility-e

  13. Slide 13: ANAVEX 2-73 Pre-Clinical Epilepsy Data

    ANAVEX™ 2-73 Pre-Clinical Epilepsy Data Significant Seizure Reduction with ANAVEX2-73 in Long-Lasting Effect Shown in PTZ- both MES and PTZ-Induced Seizure Models Induced Seizures *** *** *** *** 100 *** *** 100 90 *** p<0.001 90 80 MES-induced 80 convulsions # % of Seizure reduction % of Seizure re

  14. Slide 14: ANAVEX 2-73 Potential Protective Role in Treatment of Patients with MS

    ANAVEX™ 2-73 Potential Protective Role in Treatment of Patients with MS (Multiple Sclerosis)  ANAVEX 2-73 reduces both oligodendrocytes (OL) and OL precursor cell death induced by 4 different toxic molecules by over 50%  Conclusion: ANAVEX 2-73 protects oligodendrocytes (OL) from cytotoxic mechani

  15. Slide 15: Alzheimer's Market Overview

    Alzheimer’s Market Overview Changes in Number of Deaths (All Ages) Between 2000 and 2010 $307 billion spent caring for AD patients - expected to reach $1.5 trillion by 2050 FDA has approved 4 drugs for AD that only temporarily slow worsening of symptoms for 6-12 months in ~50% of patients 15 Sourc

  16. Slide 16: Alzheimer's Disease is Complicated ...

    Alzheimer‘s Disease is Complicated ...  Removing amyloid beta (A-beta) plaques in the brain might be not sufficient to be completely effective - Recent disappointments of large Phase 3 studies: bapineuzumab (Pfizer), solanezumab (Eli Lilly), crenezumab (Roche) and gantenerumab (Roche)  More likely

  17. Slide 17: Sigma-1R Reduces Chronic Stress and Protein Misfolding

    Sigma-1R Reduces Chronic Stress and Protein Misfolding Monomer Chronically Distressed Cell: Healthy Cell: Misfolded Proteins Quick Removal Sigma-1R After Utilization Helping / Stimulating Ca2+ dysfunction ANAVEX 2-73 Own Body to Aß plays Regain important role in hippocampal Functionality – memory Mi

  18. Slide 18: ANAVEX 3-71 Significantly Decreases Hallmark Pathologies in 3xTg-AD Model

    ANAVEX™ 3-71 Significantly Decreases Hallmark Pathologies in 3xTg-Alzheimer’s Disease Model  10 month-old 3xTg-AD and WT mice  administered i.p. with tap water or ANAVEX 3-71 (10 mcg/kg/day) for 2 months 18 Source: Fisher et al., Neurodegenerative Diseases 2015 DOI: 10.1159/000440864

  19. Slide 19: FDA Guidelines for Adaptive Trial and Population PK Design

    FDA Guidelines for Adaptive Trial and Population PK Design  Fewer patients needed  Shorter duration required  More likely to demonstrate an effect of the drug (if one exists)  More informative on the treatment’s effects  Broader and better dose-response information and subgroup effects Leads to

  20. Slide 20: ANAVEX 2-73 Efficient Clinical Trial Execution Plan

    ANAVEX™ 2-73 Efficient Clinical Trial Execution Plan ANAVEX™2-73-001 Study: • Phase 1 (oral) • Single Ascending Dose (SAD) Completed • Healthy subjects ANAVEX™2-73-002 Study: • Phase 2a (oral) • Mild-to-moderate AD patients • Adaptive trial with Population PK PART A: Preliminary Data CTAD 2015 • Bio

  21. Slide 21: ANAVEX 2-73: Phase 2a with Adaptive Trial Design

    ANAVEX 2-73: Phase 2a with Adaptive Trial Design ANAVEX 2-73 starting dose 30mg/50mg (oral) or 3mg, 5mg (IV) Mild-Mod AD ANAVEX 2-73 starting dose 30mg/50mg (oral) or patients 3mg, 5mg (IV) + Donepezil Healthy control, Donepezil/ AchEI# No. of Pts:  32 mild-moderate AD patients (MMSE 16-28), M/F =

  22. Slide 22: Positive Phase 2a Results

    Positive Phase 2a Results  Phase 2a (PART A) results demonstrate a favorable safety, bioavailability, dose-response curve and tolerability/risk profile  Despite the relative small sample size of this proof-of-concept randomized open-label study, dose-response analysis seem to indicate a cognitive

  23. Slide 23: PART A: Bioavailability Adaptive Cross-Over Design

    PART A: Bioavailability Adaptive Cross-Over Design PART B: 52 Wk Open-Label Extension and New 104 Week Study ORAL ORAL/IV Screening Randomization ORAL ORAL ORAL IV IV/ORAL 5 week 52 week 104 week [time] Part A Part B PK Study - completed Extension - ongoing  PART A is 5 week with on-off-on dosing o

  24. Slide 24: ANAVEX 2-73 Safety from Phase 2a

    ANAVEX™ 2-73 Safety from Phase 2a  Safety profile  The most common AE was dizziness followed by headache  Events were mild or moderate and reversible with 80% being grade 1  There were no drug related AEs Grade 3, 4 and 5  Most (94.4%) observed within first 8 days  AE profile similar to that o

  25. Slide 25: Efficacy Data Measurements Cognition and Function

    Efficacy Data Measurements Cognition and Function  Exploratory cognitive measures (Cogstate battery); (MMSE)  Functional measures (ADCS-ADL) Supportive Biomarker Measurements  ERP (P300): fundamental measures of synaptic network performance and target engagement  ERP target detection task measur

  26. Slide 26: Comparable Baseline of SoC (AIBL-AD) and ANAVEX 2-73 Trial

    Comparable Baseline of SoC (AIBL-AD) and ANAVEX 2-73 Trial SoC (Standard of Care) data from Australian Imaging Biomarkers and Lifestyle (AIBL-AD#) study testing Cogstate battery in Alzheimer’s disease patients Baseline data: SoC (AIBL-AD) AV 2-73 Participants (n) 43 32 Age (mean) 79.6 71 MMSE (mean)

  27. Slide 27: ANAVEX 2-73 Improves Components of Cogstate Tasks at Week 5

    ANAVEX 2-73 Improves Components of Cogstate Tasks at Week 5 Cognitive Decline Cognitive Improvement Effect sizes for change  ANAVEX 2-73 improved 5 of 6 from Cogstate tasks baseline  ANAVEX 2-73 treatment is associated with improvement *p<0.05, **p<0.001 in psychomotor function (detection), attent

  28. Slide 28: ANAVEX 2-73 Positive Trend in Interim ADCS-ADL Data

    ANAVEX 2-73 Positive Trend in Interim ADCS-ADL Data At week 12 70.00 68.00 66.00 Δ = +3.21 points 64.00 62.00 60.00 58.00 ADCS-ADL (Baseline PART B) ADCS-ADL (Week 12) ANAVEX 2-73 improved ADCS-ADL signal in 11 out of 14 (78.6%) patients at week 12 Macfarlane et al., presented at CTAD 2015 28

  29. Slide 29: EEG/P300/ERP Highlights Target Engagement of ANAVEX 2-73

    EEG/P300/ERP Highlights Target Engagement of ANAVEX 2-73 and Potential Correlation with Cognitive Effect on a Cellular Level P300 Amplitude (µV) Task Accuracy (%) False Alarms (%) Reaction Time (ms) Baseline 5.99±0.58 83.8±.3.9 3.4±1.0 559.0±24.0 Week 5 7.09±0.72 92.6±2.4 1.0±0.5 492.6±23.8* Healthy

  30. Slide 30: Financial Summary

    Financial Summary Symbol: AVXL Shares Outstanding: 35.7 million 52-Week Stock Price Range: $3.16 - $14.84 Average Daily Volume: 1,261,330 Cash @ 3/31/2016: $12 million Cash Burn (est.): ~$600K/month  Well-capitalized to achieve key clinical readouts  No debt 30

  31. Slide 31: Experienced Management Team with Strong Track Record in the CNS Space

    Experienced Management Team with Strong Track Record in the CNS Space Management and Directors Christopher U. Missling, PhD, MBA Tasos Zografidis, PhD Uli Elben, PhD Kristina Capiak Bernd Metzner, PhD Elliot Favus, MD Steffen Thomas, PhD Tom Skarpelos Scientific Advisory Board Abraham Fisher, PhD Je

  32. Slide 32: Key Achievements to Date and Expected Catalysts in 2016

    Key Achievements to Date and Expected Catalysts in 2016  New 104 week extension study of Phase 2a after PART B  Preparation for Phase 2 study in Rett syndrome Double-blinded, randomized, placebo-controlled  Preparation for Phase 2/3 study in Alzheimer’s disease Double-blinded, randomized, placebo

  33. Slide 33: Contact Us

    Contact Us Corporate Office: Anavex™ Life Sciences Corp. 51 West 52nd Street, 7th floor New York, NY 10019 (212) 332 4449 Shareholder & Media Relations: Toll-free: 1-866-505-2895 Outside North America: + 1 (416) 489-0092 Email: ir@anavex.com www.anavex.com NASDAQ: AVXL 33 ANAVEX is a trademark of An

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