Anavex Life Sciences Corp. · NASDAQ:AVXL

Anavex Life Sciences — Investor Presentation (February 2021)

30 slides from Anavex Life Sciences's investor presentation, February 2021

  1. Slide 1: Corporate Presentation

    Corporate Presentation Christopher U Missling, PhD | President & CEO Nasdaq: AVXL | February 2021

  2. Slide 2: Forward Looking Statement

    Forward Looking Statement 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,”

  3. Slide 3: Broad Pipeline Targeting Neurodegenerative and Neurodevelopmental Diseases

    Broad Pipeline Targeting Neurodegenerative and Neurodevelopmental Diseases with Significant Unmet Medical Need CANDIDATE PRECLINICAL PHASE 1 PHASE 2 PHASE 3 ALZHEIMER’S DISEASE AD ANAVEX®2-73-AD-004 ANAVEX®2-73 (Blarcamesine) PARKINSON’S DISEASE PARKINSON’S DISEASE DEMENTIA ANAVEX®2-73-PDD-001 *RETT

  4. Slide 4: Targeting Large Market Opportunities with Significant Unmet Medical Need

    Targeting Large Market Opportunities with Significant Unmet Medical Need All U.S. Patient Numbers 1) Alzheimer’s Disease Facts and Figures. Alzheimers Dement 2018;14(3):367-429. 2) Marras C et al 2018. npj Parkinson's Disease volume 4, Article number: 21 3) Based on prevalence number on orphanet 4

  5. Slide 5: Transformative Events for Anavex

    Transformative Events for Anavex ! ANAVEX®2-73 (Blarcamesine) Rett Syndrome Program Received Fast Track Designation and is Eligible for Pediatric Priority Review Voucher ! Pursuing Large Markets with High Unmet Need by Applying Genetic Precision Medicine ! Novel CNS Mechanism of Action Upstream of N

  6. Slide 6: Catalysts to Drive Value

    Catalysts to Drive Value The company has multiple clinical milestones ! Complete data ANAVEX®2-73 U.S. Rett syndrome Phase 2 study – expected Q1/Q2 2021 ! Complete data ANAVEX®2-73 Parkinson’s disease dementia (PDD) Phase 2 study – expected Q1/Q2 2021 ! Top-line data AVATAR: Potentially pivotal Phas

  7. Slide 7: Clinical Trials

    Clinical Trials Mechanism of Action (MoA) and Clinical Data: • Rett Syndrome (RTT) • Alzheimer’s Disease (AD) • Parkinson’s Disease Dementia (PDD)

  8. Slide 8: ANAVEX2-73 MoA: SIGMAR1 Activation Prevents Cellular Stress

    ANAVEX®2-73 MoA: SIGMAR1 Activation Prevents Cellular Stress Before and After RNA Gene Transcription Source: Couly et al., Knocking Out Sigma-1 Receptors Reveals Diverse Health Problems. Cell Mol Neurobiol (2020); Lee et al., Sigma-1 receptor chaperones rescue nucleocytoplasmic transport deficit see

  9. Slide 9: ANAVEX2-73 Establishes Human Proof-of-Concept and SIGMAR1 Target Occupancy

    ANAVEX®2-73 Establishes Human Proof-of-Concept and SIGMAR1 Target Occupancy 2D [18F]FTC-146-PET imaging of ANAVEX®2-73: Dose- ANAVEX®2-73 positive response in functional dependent ANAVEX®2-73 Target Engagement (ADCS-ADL*) outcome in Alzheimer’s disease patients correlate with SIGMAR1 mRNA levels p-v

  10. Slide 10: SIGMAR1 Activation Modulates Multiple Aspects of Neurodegenerative Processes

    SIGMAR1 Activation has been Shown to Modulate Multiple Aspects of Neurodegenerative Processes Sigma-1 receptor agonists ANAVEX®2-73 enhances Sigma-1 receptor agonists have been shown to restore autophagy and alleviates Tau have a neuroprotective effect neuronal functions in pathology in neurodegener

  11. Slide 11: What is Rett Syndrome?

    What is Rett Syndrome? Devastating neuro-developmental disease in girls with both movement impairment and cognitive impairment Rett Syndrome (RTT) § Non-inherited genetic postnatal disorder caused by mutations in the MECP2 gene Ø Occurs almost exclusively in girls Ø Leads to severe impairments, affe

  12. Slide 12: First Phase 2 Study in Adult Rett Syndrome

    First Phase 2 Study in Adult Rett Syndrome U.S. Rett Syndrome ANAVEX®2-73-RS-001 Trial (NCT03758924) SUMMARY Overview EFFICACY Assessments •Phase 2, safety, tolerability, efficacy for 7 weeks, oral, •Global severity RSBQ (Rett Syndrome liquid formulation, 5 mg daily (relatively low dose) Behavior Qu

  13. Slide 13: Phase 2: Improvement in All Key Domains

    Phase 2: Improvement in All Key Domains U.S. Rett Syndrome ANAVEX®2-73-RS-001 Trial (NCT03758924) SUMMARY of Top-line ANAVEX®2-73 (blarcamesine) Placebo-Controlled Phase 2 U.S. Rett Syndrome Trial Results RSBQ CGI-I All Participants ITT (n = 25) All Participants ITT (n = 25) Treatment Responder Non-

  14. Slide 14: Phase 2: Established Safety

    Phase 2: Established Safety U.S. Rett Syndrome ANAVEX®2-73-RS-001 Trial (NCT03758924) SUMMARY of Safety and Tolerability: • ANAVEX®2-73 was found to be well tolerated with very good medication compliance during the trial. • All 25 subjects completed the study. The overall incidence of patients who e

  15. Slide 15: Phase 2: Biomarker Correlated Outcome

    Phase 2: Biomarker Correlated Outcome U.S. Rett Syndrome ANAVEX®2-73-RS-001 Trial (NCT03758924) • Statistically significant differences in patient symptoms between the active and placebo groups occurred as early as 4 weeks following the initiation of ANAVEX®2-73 administration. • ANAVEX®2-73 activat

  16. Slide 16: Precision Medicine

    Precision Medicine Biomarkers Increase Probability of Success 76% • Patient selection biomarkers 55% 46% • Higher therapeutic response 28% • Lower variability in the target population Phase II to Phase III Phase III to NDA/BLA Without Biomarkers With Patient Selection Biomarkers Thomas DW et al. Cli

  17. Slide 17: ANAVEX2-73-RS-001 Phase 2 U.S. Rett Syndrome Study

    ANAVEX®2-73-RS-001 Phase 2 U.S. Rett Syndrome Study N=31 * 7 12weeks … and Open Label Extension (OLE) ClinicalTrials.gov: NCT03758924 RTT patient population ANAVEX®2-73 Primary and Secondary Endpoints Active dose# • PK, Safety and tolerability of ANAVEX®2-73 • Diagnosis of • RSBQ, CGI-I confirmed RT

  18. Slide 18: ANAVEX2-73-RS-002 Phase 2/3 Rett Syndrome AVATAR Study

    ANAVEX®2-73-RS-002 Phase 2/3 Rett Syndrome AVATAR Study N=33 * 7 … and Open Label Extension (OLE) 48weeks ClinicalTrials.gov: NCT03941444 RTT patient population ANAVEX®2-73 Primary and Secondary Endpoints Active dose# • RSBQ, CGI-I • ADAMS, Sleep function • Diagnosis of • Seizure activity confirmed

  19. Slide 19: ANAVEX2-73-RS-003 Phase 2/3 Rett Syndrome EXCELLENCE Study

    ANAVEX®2-73-RS-003 Phase 2/3 Rett Syndrome EXCELLENCE Study N>69 12 … and Open Label Extension (OLE) 48weeks ClinicalTrials.gov: NCT04304482 RTT patient population ANAVEX®2-73 Primary and Secondary Endpoints Active dose# • RSBQ, CGI-I • ADAMS, Sleep function • Diagnosis of • Seizure activity confirm

  20. Slide 20: Alzheimer's Disease (AD)

    Alzheimer’s Disease (AD) Alzheimer’s disease is a progressive, irreversible neurological disease and the most common cause of dementia Alzheimer’s Disease (AD) § Alzheimer’s disease incidence highly correlates with age Ø AD prevalence in US:~5,700,000 Ø Estimated 50 million people live with dementia

  21. Slide 21: Improved MMSE and ADCS-ADL Scores in Phase 2a AD Study through 148 Weeks

    ANAVEX®2-73 Demonstrated Improved MMSE1 and ADCS-ADL2 Scores in Phase 2a AD Study through 148 Weeks Adjusted change in ADCS-ADL2 (±SE) Adjusted change in MMSE1 (±SE) N=8 N=8 N=13 N=13 p-value < 0.0008 p-value < 0.0001 Source: Hampel et al. A precision medicine framework using artificial intelligence

  22. Slide 22: ANAVEX2-73 Phase 2b/3 Alzheimer's Disease and ATTENTION-AD OLE Study

    ANAVEX®2-73 Phase 2b/3 Alzheimer's Disease and ATTENTION-AD OLE Study N=450 48 … and Open Label Extension (OLE) 96weeks ClinicalTrials.gov: NCT03790709 Early AD patient population ANAVEX®2-73 Primary Endpoints High dose# • Confirmed amyloid • ADAS-Cog pathophysiology • ADCS-ADL (CSF/amyloid PET) • S

  23. Slide 23: Parkinson's Disease Dementia (PDD)

    Parkinson’s Disease Dementia (PDD) Up to 80 percent of those with Parkinson’s disease eventually experience Parkinson’s disease dementia Parkinson’s Disease Dementia § Parkinson’s disease is a fairly common neurological disorder in older adults, estimated to affect nearly 2 percent of those older th

  24. Slide 24: ANAVEX2-73 PoC Phase 2 PDD Study Design

    ANAVEX®2-73 PoC Phase 2 PDD Study Design A Phase 2 trial to Assess the Safety, Tolerability and Efficacy of ANAVEX®2-73 (blarcamesine) Oral Capsules in the Treatment of Parkinson’s Disease Dementia 1:1:1 randomization 2-week baseline period 3-week up-titration period 11-week target dose treatment pe

  25. Slide 25: Key Cognitive Domains

    Key Cognitive Domains Key cognitive features addressed by ANAVEX®2-73 (blarcamesine) The criteria from the National Institute on Aging and Alzheimer’s Association (NIA-AA) workgroup mention the following five Addressed in PoC cognitive domains when diagnosing MCI-AD: Phase 2 PDD Study (a) Episodic m

  26. Slide 26: Significant Improvements in Episodic Memory with Increased Dose

    Significant Improvements in Episodic Memory with Increased Dose ANAVEX®2-73-PDD-001 Study: Dose-dependent, statistically significant improvement of Quality of Episodic Memory with ANAVEX®2-73 (blarcamesine) Quality of Episodic Memory (counts) All participants Time: 14 weeks change from baseline • A

  27. Slide 27: Summary of Topline Results

    Summary of Topline Results: Broad and Significant Effects with ANAVEX®2-73 (blarcamesine) in PDD Patients • ANAVEX®2-73 (blarcamesine): a novel, oral, investigational sigma-1 receptor (Sig-1R / SIGMAR1) agonist with multimodal activity • Data confirm SIGMAR1 as gene “signature” biomarker of response

  28. Slide 28: Anavex is pursuing Large Markets by Applying Precision Medicine

    PRECISION MEDICINE IMPROVES CHANCE OF CLINICAL Anavex is pursuing Large Markets SUCCESS Testing for biomarkers demonstrated improved clinical response by Applying Precision Medicine to to ANAVEX ®2-73 in Rett syndrome correlated with glutamate and for Alzheimer’s and Parkinson’s patients carrying wi

  29. Slide 29: Anavex Life Sciences Expertise

    Anavex Life Sciences Expertise Management Team Christopher U. Missling PhD - President & CEO Walter E Kaufmann, MD - Chief Medical Officer Stephan Toutain, MS, MBA – Chief Operating Officer Emmanuel O Fadiran, RPh, PhD - SVP of Regulatory Affairs Daniel Klamer, PhD - VP of Business Development & Sci

  30. Slide 30: Contact Us

    Contact Us Corporate Office Anavex®Life Sciences Corp. 51 West 52nd Street, 7th floor New York, NY 10019 1-844-689-3939 Shareholder & Media Relations ir@anavex.com www.anavex.com NASDAQ: AVXL 30

Anavex Life Sciences — Investor Presentation (February 2021) on 6ix