Anavex Life Sciences Corp. · NASDAQ:AVXL
Anavex Life Sciences — Investor Presentation (February 2020)
28 slides from Anavex Life Sciences's investor presentation, February 2020
Slide 1: Corporate Presentation
Corporate Presentation Christopher U Missling, PhD | President & CEO Nasdaq: AVXL | February 2020
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: Anavex Expanding Pipeline: Potential for Significant Value Creation
Anavex Expanding Pipeline: Potential for Significant Value Creation Near and Long Term CANDIDATE PRECLINICAL PHASE 1 PHASE 2 PHASE 3 ALZHEIMER’S DISEASE AD ANAVEX®2-73-AD-004 ANAVEX®2-73 (Blarcamesine) PARKINSON’S DISEASE DEMENTIA ANAVEX®2-73-PDD-001 *RETT SYNDROME U.S. ANAVEX®2-73-RS-001 Fast Track
Slide 4: Overview
Overview • Four Key Clinical Trials Enrolling: Two Phase 2 Rett Syndrome Trials, Phase 2b/3 Alzheimer’s Disease, Phase 2 Parkinson’s Disease Dementia with Several Readouts in 2020 • Rett syndrome* program Fast Track Designation and eligible for Pediatric Priority Review Voucher • Pursuing Large Mark
Slide 5: Catalysts to Drive Value
Catalysts to Drive Value The company expects to achieve key clinical milestones Full enrollment Phase 2 Parkinson’s disease dementia (PDD) FDA Fast Track designation for Rett syndrome program (RTT) Full enrollment U.S. Phase 2 Rett syndrome (RTT) Full enrollment AVATAR Phase 2 Rett syndrome (RTT) To
Slide 6: Clinical Trials – MoA and First Clinical Data
Clinical Trials – MoA and First Clinical Data: • Rett Syndrome (RTT) • Alzheimer’s Disease (AD)
Slide 7: ANAVEX 2-73 MoA: S1R Activation is Upstream from other Therapeutic Targets
ANAVEX®2-73 MoA: S1R Activation is Upstream from other Therapeutic Targets Neural cells suffer functional loss in neurological disorders which causes cellular stress Pathologies include: ü Aβ, Tau and ApoE fragmentation and dysfunction ü Proteinopathy ü Microglia activation, migration, and dysregula
Slide 8: 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 9: 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 10: 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 11: Phase 2 U.S. Rett Syndrome Part A: Improvement in All Key Domains
Phase 2 PART A: Improvement in All Key Domains U.S. Rett Syndrome ANAVEX®2-73-RS-001 Trial (NCT03758924) SUMMARY EFFICACY* • Phase 2, safety, tolerability, efficacy for 7 weeks, oral, liquid formulation, • Global severity RSBQ and CGI-I 5 mg daily (relatively low dose) RSBQ = Rett Syndrome Behavior
Slide 12: Precision Medicine
Precision Medicine Patient Selection with Biomarker Increases Probability of Success 76% 55% 46% 28% Phase II to Phase III Phase III to NDA/BLA Without Biomarkers With Patient Selection Biomarkers Thomas DW et al. Clinical Development Success Rates 2006-2015. BIO Industry Analysis 12
Slide 13: MeCP2 deficiency leads to increased glutamate in RTT
In patients with RTT, MeCP2 deficiency leads to increased levels of Glutamate, Loss of synaptic in comparison to homeostasis can healthy controls1,2,3, impair nerve cells which results in (neurons) and excitatory-inhibitory their connections imbalance and further synaptic dysfunction Glutamate as po
Slide 14: Part A: Reported Improvements Correlate with Biomarkers
Phase 2 PART A: Reported Improvements Correlate with Biomarkers U.S. Rett Syndrome ANAVEX®2-73-RS-001 Trial (NCT03758924) Glutamate REPORT on PART A: INTENSIVE PK SUBCOHORT Baseline Week 7 • Plasma levels of the biomarker Glutamate decreased significantly (Week 0 vs. Week 7; 2-tailed 0 Wilcoxon sign
Slide 15: ANAVEX 2-73 Phase 2 U.S. Rett Syndrome Study N=21
ANAVEX®2-73 Phase 2 U.S. Rett Syndrome Study N=21 * 7 RTT pa&ent popula&on ANAVEX®2-73 Primary and Secondary Endpoints Active dose# • PK, Safety and tolerability of ANAVEX®2-73 • Diagnosis of • Behavioral symptoms confirmed RTT • Sleep function • Patients age >18 • Seizure activity Randomiza0on • DN
Slide 16: ANAVEX 2-73 Phase 2 Rett Syndrome AVATAR Study N=33
ANAVEX®2-73 Phase 2 Rett Syndrome AVATAR Study N=33 * 7 RTT pa&ent popula&on ANAVEX®2-73 Primary and Secondary Endpoints Active dose# • Safety and tolerability of ANAVEX®2-73 • Diagnosis of • Behavioral symptoms confirmed RTT • Sleep function • Patients age >18 • Seizure activity Randomiza0on • DNA
Slide 17: ANAVEX 2-73-RS-003 Rett Syndrome EXCELLENCE Study N=69
ANAVEX®2-73-RS-003 Rett Syndrome EXCELLENCE Study N=69 12 RTT pa&ent popula&on ANAVEX®2-73 Primary and Secondary Endpoints Active dose# • Safety and tolerability of ANAVEX®2-73 • Diagnosis of • Behavioral symptoms confirmed RTT • Sleep function • Patients age 5-18 • Seizure activity Randomiza0on • D
Slide 18: Alzheimer's Disease (AD)
Alzheimer’s Disease (AD) Alzheimer’s disease, progressive, irreversible neurological disease and 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 worldw
Slide 19: 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 < 0.0001 p-value < 0.0001 Source: Hampel H., Afshar M., Parmentier F. et al., CTAD 2018; Hampel et al., Alzh
Slide 20: Phase 2b/3 Alzheimer's Disease and ATTENTION-AD OLE Study N=450
ANAVEX®2-73 Phase 2b/3 Alzheimer's Disease and ATTENTION-AD OLE Study N=450 48 … and Open Label Extension (OLE) 96weeks Primary Endpoints Early AD pa&ent popula&on • ADAS-Cog ANAVEX®2-73 • ADCS-ADL High dose# • Confirmed amyloid • Safety and tolerability of pathophysiology ANAVEX®2-73 (CSF/amyloid P
Slide 21: 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 (PDD) § Parkinson’s disease is a fairly common neurological disorder in older adults, estimated to affect nearly 2 percent of those ol
Slide 22: ANAVEX 2-73 Phase 2 Parkinson's Disease Dementia Study N=120
ANAVEX®2-73 Phase 2 Parkinson’s Disease Dementia (PDD) Study N=120 14 PDD pa&ent popula&on Primary Endpoints • CDR Continuity of Attention ANAVEX®2-73 • Safety and tolerability of • Diagnosis of High dose# ANAVEX®2-73 probable Parkinson’s disease dementia Key Secondary Endpoints • MDS-UPDRS (PDD) •
Slide 23: Primary Endpoint CDR Continuity of Attention of PDD Ph2 Study
Primary Endpoint ‘CDR Continuity of Attention’ of PDD ANAVEX®2-73 Ph2 Study: Confirmed Beneficial Effect in Previous Ph2a AD Study Identification (IDN) in Cogstate battery assessed in ANAVEX®2-73 Ph2a AD Study comparable to CDR Continuity of Attention (choice reaction time paradigm) CDR baBery Cogst
Slide 24: ANAVEX 2-73 Ph2 AD Study - Cogstate IDN Improves within Weeks
ANAVEX®2-73 Ph2 AD Study - Cogstate IDN Improves within Weeks Cogstate IDN from Baseline Average Delta Reaction time (ms) ANAVEX®2-73 Cogstate IDN Weeks from Baseline Bars: Standard Error 24
Slide 25: Cogstate IDN: Ph2a AD Patients Improve vs Standard of Care
Cogstate IDN: ANAVEX®2-73 Ph2a AD Patients Improve within Weeks vs Standard of Care AD Patients AIBL-ROCS-AD* cohort as standard of care comparator Average Delta Reaction time (ms) SoC (AIBL- ANAVEX® Baseline data: Cogstate IDN ROCS-AD) 2-73 Participants (n) 35 32 MMSE (mean) 21.2 21.0 (p<0.01) ANAV
Slide 26: Anavex is the only Company pursuing Large Markets by Applying Precision Medicine
Anavex is the only Company PRECISION MEDICINE IMPROVES CHANCE OF CLINICAL SUCCESS pursuing Large Markets by Testing for biomarkers demonstrated improved clinical response to ANAVEX ® 2-73 in Rett syndrome correlated with glutamate and Applying Precision Medicine to for Alzheimer’s patients carrying
Slide 27: 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 28: 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 28
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