Anavex Life Sciences Corp. · NASDAQ:AVXL
Anavex Life Sciences — Investor Presentation (April 2019)
29 slides from Anavex Life Sciences's investor presentation, April 2019
Slide 1: Corporate Presentation
Corporate Presentation Christopher U Missling, PhD | President & CEO Nasdaq: AVXL | April 2019
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,” “b
Slide 3: New Therapeutic Strategy to Treat CNS Disorders
New Therapeutic Strategy to Treat CNS Disorders • Anavex utilizes genomic biomarkers in precision medicine to treat severe and devastating neurological disorders • Anavex is focusing on rare diseases with no available therapy (Rett syndrome) and high risk CNS patient populations (Alzheimer’s disease
Slide 4: Anavex at a Glance
Anavex at a Glance APPROACH Applying precision medicine to CNS disorders Patient selection based on genomic screening and biomarkers MECHANISM SIGMAR1 restoring cellular homeostasis FOCUS Finding effective treatments for neurodevelopmental and neurodegenerative diseases $ LARGE MARKETS Addressing un
Slide 5: Overview ANAVEX 2-73 Ongoing Precision Medicine Clinical Trials
Overview ANAVEX®2-73 Ongoing Precision Medicine Clinical Trials PHASE 2 PARKINSON’S DISEASE DEMENTIA 14 120 ANAVEX®2-73-PDD-001 STUDY (NCT03774459)* PHASE 2b/3 ALZHEIMER’S DISEASE 48 450 ANAVEX®2-73-AD-004 STUDY (NCT03790709)* PHASE 2 RETT SYNDROME** 7 15 ANAVEX®2-73-RS-001 STUDY (NCT03758924)* PHAS
Slide 6: Catalysts to Drive Value
Catalysts to Drive Value The company is well positioned to achieve key clinical readouts Phase 2a – Reported 148-week data at CTAD 2018 scientific meeting Phase 2b/3 clinical trial in Alzheimer’s disease – ongoing Phase 2 clinical trial in Parkinson’s disease dementia (PDD) – ongoing Initiate Phase
Slide 7: 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 alleviates Tau Sigma-1 receptor agonists have been shown to restore pathology in neurodegenerative have a neuroprotective effect neuronal functions in disease models in
Slide 8: Autophagy as Key Protein Clearance Process: SIGMAR1 Activation
Autophagy as Key Protein Clearance Process: New Pharmacological Target in Neurodegeneration – achieved through SIGMAR1 Activation Sigma-1 receptor activation by ANAVEX2-73 enhances autophagy in C. elegans p ≤ 0.001 8
Slide 9: Genetic SIGMAR1 Variations Linked to Neurological Disorders
Genetic SIGMAR1 Variations Linked to Neurological Disorders The SIGMAR1 receptor is an integral • SIGMAR1 null mice display muscle membrane protein involved in cellular weakness, axonal degeneration homeostasis which targets restoration and motor neuron loss1 of neuroplasticity and cellular stress r
Slide 10: Patient Improvement Correlates with ANAVEX 2-73 and SIGMAR1 RNA Expression
Patients Improvement Correlates with ANAVEX®2-73 and SIGMAR1 RNA Expression Activities of Daily Living (ADCS-ADL)* Slope from Baseline to Week 57 p=0.015 ANAVEX®2-73 SIGMAR1 RNA expression (TPM) positive response in functional (ADCS- ADL) outcomes in Alzheimer’s disease patients corelate with SIGMAR
Slide 11: Significant Relationship between ANAVEX 2-73 Concentration and Patient Response
Significant Relationship between ANAVEX®2-73 Concentration and Patients Response Activities of Daily Living (ADCS-ADL)* ADCS-ADL (Delta from Baseline to Week 57) p=0.03 • High Concentration of ANAVEX®2-73 => High Delta ADCS-ADL (improved response) • # Plasma concentration of ANAVEX®2-73 is correlate
Slide 12: Higher ANAVEX 2-73 Concentration Maintains ADCS-ADL Performance
Patients Treated with Higher ANAVEX®2-73 Concentration Maintain ADCS-ADL* Performance vs Lower Concentration Cohort High Concentration cohort shows 88 % difference to low concentration cohort • High plasma concentration of ANAVEX®2-73 [>4.0 ng/ml] is correlated with the clinically administered dose
Slide 13: Higher ANAVEX 2-73 Concentration Shows Higher MMSE Performance
Patients Treated with Higher ANAVEX®2-73 Concentration Show Higher MMSE* Performance Compared to Lower Concentration High Concentration cohort shows 64 % less decline than low concentration cohort • High plasma concentration of ANAVEX®2-73 [>4.0 ng/ml] is correlated with the clinically (p < 0.0008)
Slide 14: Precision Medicine
Precision Medicine
Slide 15: Precision Medicine: Pharmacogenomics Increases Probability of Success
Precision Medicine PHARMACOLOGY + GENOMIC = Patient Selection with Biomarker PHARMACOGENOMICS Increases Probability of Success 76% The ideal pharmacogenomics test will be able to determine: 55% • Potential for a medication to be 46% effective – for this person • The best dose of a medication – 28% f
Slide 16: Gene Signature from ANAVEX 2-73-Treated Patients
Identification of Gene “Signature” from ANAVEX®2-73-Treated Patients PRECISION MEDICINE • Genomic signature (WT SIGMAR1 gene) strongest responders to ANAVEX®2-73 • This genomic “biomarker” is drug specific, not indication specific, so it applies to all indications treated with ANAVEX®2-73 16
Slide 17: SIGMAR1 Gene Plays a Role in Protein Trafficking
SIGMAR1 Gene Plays a Role in Protein Trafficking Lumenal side Transmembrane region SIGMAR1 WT rs1800866 variant structure impairs protein trafficking Cytosolic side • Majority of the population (~80%) carries SIGMAR1 WT • Majority of patients (~80%) are expected to benefit from SIGMAR1 activation wi
Slide 18: SIGMAR1 WT Gene Associated with Improved Response, Validated at 148 Weeks
SIGMAR1 WT Gene Associated with Improved Response … … and validated at 148 weeks Delta MMSE Delta ADCS-ADL (Week 57 from Baseline) (Week 57 from Baseline) p=0.023 p=0.048 SIGMAR1 SIGMAR1 WT SIGMAR1 SIGMAR1 WT Pro2 variant (n=15) Pro2 variant (n=15) (rs1800866) (rs1800866) (n=5) (n=5) Source: H Hampe
Slide 19: Clinical Studies
Clinical Studies: • Parkinson’s Disease Dementia (PDD) • Alzheimer’s Disease (AD) • Rett Syndrome (RTT)
Slide 20: Pipeline Addresses both Rare and Large Indications
Pipeline Addresses both Rare and Large Indications Anavex pipeline and precision genetic medicine approach address unmet Alzheimer’s Disease (AD) needs of neurodevelopmental and neurodegenerative diseases ~5,700,000 patients1 Parkinson’s Disease (PD) ~1,000,000 patients2 Rett Syndrome (RTT) ~11,000
Slide 21: ANAVEX 2-73 Phase 2 Parkinson's Disease Dementia Study
ANAVEX®2-73 Phase 2 Parkinson’s Disease Dementia 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) • Sleep
Slide 22: Primary Endpoint CDR Continuity of Attention Confirmed in Ph2a AD 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 ba5ery Cogst
Slide 23: 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 23
Slide 24: Cogstate IDN: ANAVEX 2-73 Patients 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 25: ANAVEX 2-73 Phase 2b/3 Alzheimer's Disease Study
ANAVEX®2-73 Phase 2b/3 Alzheimer's Disease Study N=450 48 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 PET) Key Secondary Endpoints • Patients aged 60 to • CDR-SB 85
Slide 26: ANAVEX 2-73 Phase 2 Rett Syndrome Study
ANAVEX®2-73 Phase 2 Rett Syndrome Study N=15 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 Randomiza+on • DNA and RNA 3
Slide 27: Investment Highlights
Investment Highlights • Pursuing Large Markets By Applying Precision Medicine to Develop Treatments For Neurodegenerative and Neurodevelopmental Diseases With High Unmet Needs – Novel genomic biomarker-driven programs to treat small patient populations such as Rett syndrome* as well as major public
Slide 28: 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 - SVP of Operations Emmanuel O Fadiran, RPh, PhD - SVP of Regulatory Affairs Daniel Klamer, PhD - VP of Business Development & Scientifi
Slide 29: 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 29
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