Anavex Life Sciences Corp. · NASDAQ:AVXL
Anavex Life Sciences — Investor Presentation (April 2021)
37 slides from Anavex Life Sciences's investor presentation, April 2021
Slide 1: Corporate Presentation
Corporate Presentation Christopher U Missling, PhD | President & CEO Nasdaq: AVXL | April 2021
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,”
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
Slide 4: Targeting Large Market: Global Dementia
Targeting Large Market: Global Dementia Worldwide Patient Numbers Source: World Alzheimer’s Report 4
Slide 5: Worldwide Dementia Cases Projected to Grow to Over 130M by 2050
Worldwide Dementia Cases Projected to Grow to Over 130M by 2050 Source: Alzheimer's Disease International 5
Slide 6: Costs Associated with Alzheimer's Treatment and Care in the U.S. are Unsustainable
Costs Associated with Alzheimer’s Treatment and Care in the U.S. are Unsustainable Age Prevalence of Global Alzheimer’s Disease and Dementias >$20 trillion Cumulative costs of Alzheimer’s and dementia care from 2015 to 2050 1 in 3 Medicare dollars will be spent on people living with Alzheimer’s and
Slide 7: Aiming to Change the Course of Dementia
Aiming to Change the Course of Dementia … … Dementia is Progressive and over time a Patient’s Cognition will worsen 4.0-point calculated ADAS-Cog score improvement Direction of (p = 0.015) Improvement Visualize the improvement in calculated ADAS-Cog Parkinson’s disease dementia (PDD) patients improv
Slide 8: 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 orphaned 8
Slide 9: 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
Slide 10: 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 Q2 2021 ! Complete data ANAVEX®2-73 Parkinson’s disease dementia (PDD) Phase 2 study – expected Q2 2021 ! Top-line data AVATAR: Potentially pivotal Phase 2/3
Slide 11: Clinical Trials, Mechanism of Action and Clinical Data
Clinical Trials Mechanism of Action (MoA) and Clinical Data: • Rett Syndrome (RTT) • Alzheimer’s Disease (AD) • Parkinson’s Disease Dementia (PDD)
Slide 12: ANAVEX 2-73 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
Slide 13: 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
Slide 14: ANAVEX 2-73 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
Slide 15: Interface of ANAVEX 2-73-activated Pathway Analysis and Disease Biology
Interface of ANAVEX®2-73-activated Pathway Analysis and Disease Biology Using mRNA Differential Gene Expression with Focus on SIGMAR1 Pathway Differential Gene Expression Baseline t1 Genes of interest ~700 Patients X 26K genes X 2 timepoints mRNA0 mRNA1 ANAVEX®2-73 Molecular Patient Pathway Biology
Slide 16: 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
Slide 17: 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
Slide 18: 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-
Slide 19: 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
Slide 20: 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
Slide 21: 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
Slide 22: AI Powered, Biomarker Driven, Accelerated Development
AI Powered, Biomarker Driven, Accelerated Development Built on in- depth Molecular Understanding of SIGMAR1 Pathway Goal: Data from ~700 study participants, Expedited 3 indications, 6 trials Process Pr ec isi Increased Probability MMSE, ADCS-ADL on M of Phase II / III MoCA, CDR ed MDS-UPDRS ici Succ
Slide 23: ANAVEX 2-73-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
Slide 24: ANAVEX 2-73-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
Slide 25: ANAVEX 2-73-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
Slide 26: 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
Slide 27: ANAVEX 2-73 Demonstrated Improved MMSE and ADCS-ADL Scores in Phase 2a AD
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
Slide 28: ANAVEX 2-73 Biomarker Driven Development Strategy in Alzheimer's Disease
ANAVEX®2-73 Biomarker Driven Development Strategy in Alzheimer’s Disease Demonstrated Improved MMSE and ADCS-ADL through Patient Clinical Data 148 weeks for all patients Cognitive Scores Sleep Questionnaires Other Scores Dose, PK KEM® Genomic Data Novel Genomic Biomarkers of Response Identified in P
Slide 29: ANAVEX 2-73 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
Slide 30: 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
Slide 31: ANAVEX 2-73 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
Slide 32: 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
Slide 33: 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
Slide 34: 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
Slide 35: 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
Slide 36: 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
Slide 37: 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 [email protected] www.anavex.com NASDAQ: AVXL 37
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