ANAVEX 2-73, a Clinical Candidate for Neurodevelopmental Disorders: New data Including AnC-Seizure data in Angelman Syndrome
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1 ANAVEX 2-73, a Clinical Candidate for Neurodevelopmental Disorders: New data Including AnC-Seizure data in Angelman Syndrome Christopher U Missling, PhD, President & CEO AEDD Trials XIV Conference May 2017
2 2
3 Sigma-1 Receptor AcCvaCon and NeurodegeneraCve Diseases 3
4 Sigma-1 Receptor AcCvaCon Related to AnC-Seizure Effects 4
5 Confirmed Effects of Sigma-1 Receptor AcCvaCon ü Synaptogenesis ü Restores Ca 2+ imbalance ü Reduces InflammaCon ü Reduces OxidaCve stress ü Reduces Tau hyper-phosporylacon ü Restores Mitochondrial dysfuncion effects relevant in both neurodevelopmental as well as neurodegeneracve diseases 5
6 Sigma-1R Agonists MoA: Restoring Homeostasis ANAVEX 2-73 Restoring Homeostasis Sigma-1R helping / scmulacng own body to regain funcconality Su et al., Trends Pharmacol Sci Villard et al., J. Psychopharmacol [σ1 Antagonist] Source: Adapted from Miki et al, Dec 9. doi: /neup Neuropathology 2013 Glembotski et al., CirculaHon Research. 2007;101:
7 ANAVEX 2-73 Shown to be Safe in Phase 2a Clinical Trial of Mild-to-Moderate Alzheimer s PaCents Phase 2a results demonstrate a favorable safety, bioavailability, dose-response curve and tolerability/risk profile at doses between 10mg and 50mg of oral daily ANAVEX 2-73 Primary endpoints met with favorable safety and tolerability Secondary endpoints met with supporcve exploratory biomarker, cognicon and funccon measures correlacng Low-High dose was stacsccally significant to affect MMSE-Δ and EEG/ERP-Δ scores with MMSE-Δ (p=0.0285) and EEG/ERP-Δ (p=0.0168), respeccvely 7
8 Angelman Syndrome A rare neuro-genecc disorder Majority of cases due to mutacons or delecon of UBE3A gene (Chromosome 15) Affects 1 in 15,000 Symptoms include: Seizures (presented in over 80% of affected individuals) Ataxia CogniCve impairment Speech impairment Sleep disorders 8
9 Audiogenic Seizures in an Angelman Syndrome Model ANAVEX 2-73 (10 mg/kg ip dosed daily for 14 days) was evaluated for audiogenic-induced seizures in 3-4 month old 129- background mice with mutacon in Ube3a The study was sponsored by FoundaCon for Angelman Syndrome. The work was carried out at the Anderson Lab at Baylor University in Houston, TX Audiogenic seizures are hypothesized to model tonic-clonic seizures with brainstem origin Audiogenic seizures are thought to best model temporal lobe epilepsy (TLE) and reflex seizures # # Kandratavicius L et al. Neuropsychiatr Dis Treat Sep 9;10:
10 ParCal Rescue of Audiogenic Seizure with ANAVEX 2-73 ANAVEX 2-73 administracon significantly reduced audiogenicinduced seizures (p<0.01, KO vehicle vs. KO ANAVEX 2-73) p=
11 ANAVEX 2-73: Dose-Dependent AnC-Seizure Effects % of Seizure reduccon Significant Seizure ReducCon with ANAVEX2-73 in both MES and PTZ-Induced Seizure Models Vehicle 10 mg/kg (p.o.) p< mg/kg (p.o.) 100 mg/kg (p.o.) MES-induced convulsions # PTZ-induced convulsions % of Seizure reduccon Long-LasCng Effect Shown in PTZ-Induced Seizures Vehicle p< mg/kg (p.o.) 4 hours 6 hours ANAVEX 2-73 also shows synergiscc accvity with three generacons of epilepsy drugs currently on the market: ETS (ZaronCn ), VPA (Depakene ) and GabapenCn (NeuronCn ) Presented at AES Mee.ng 2015, # results have been confirmed by the ETSP screening program 11
12 ANAVEX 2-73 Significantly Reduces the Number of Spasms in a Pre-Treatment Experiment of InfanCle Spasms in Infant Rat Model 150 Spasm count p< hour pre-treatment with ANAVEX 2-73 (30mg/kg ip) before trigger of spasms with NMDA (postnatal day 15) 0 Treatment with ANAVEX 2-73 significantly reduced the number of spasms by 55% compared to vehicle 12
13 Fragile X Syndrome Rare neurodevelopmental disorder MutaCon, CGG trinucleocde repeats (typically >200), in the FMR1 gene cause Fragile X syndrome AffecCng approximately 1 in 4,000 males and 1 in 6,000 females Symptoms include: Seizures (13-44% of pacent populacon) 1 Comorbidity with epilepsy has been found to be associated with polymorphisms in the brain-derived neurotrophic factor (BDNF) gene 2 AtenCon Problems Anxiety Developmental Delay HyperacCvity 1 Berry-Kravis E. Dev Med Child Neurol Nov;44(11): Louhivuori V et al. Epilepsy Res Jul;85(1):
14 BDNF Levels in Hippocampus as Biomarker BDNF under-expression has been observed in many neurodevelopmental as well as neurodegeneracve pathologies. BDNF signaling promotes maturacon of both excitatory and inhibitory synapses #, and normalizacon of BDNF expression could be beneficial in both neurodevelopmental and neurodegeneracve disorders ANAVEX 2-73 (1 mg/kg ip dosed twice daily for 14 days) was evaluated for its effect on potencal biomarkers in 2-month old Fragile X mental retardacon 1 knockout (Fmr1 KO) mice BDNF protein levels in the hippocampus were measured by ELISA The study was sponsored by FRAXA and performed by Fraunhofer Chile Research, SanCago, Chile # Castrén ML & Castrén E. Neuropharmacology Jan;76 Pt C:
15 ANAVEX 2-73 Normalizes Hippocampal BDNF Expression p<0.05 AdministraCon of ANAVEX 2-73 in the Fragile X Fmr1 KO mouse significantly restores hippocampal BDNF expression to the same levels observed in vehicle-treated wildtype mice (p<0.05, KO vehicle vs. KO ANAVEX 2-73) 15
16 Ret Syndrome Rare, non-inherited genecc postnatal progressive neurodevelopmental disorder 95% of cases due to mutacons in MECP2 Gene (X Chromosome) Occurs almost exclusively in girls Affects 1 in 10,000 to 15,000 Symptoms include: Seizures (86% in the adult pacent populacon) Anxiety CogniCve impairment Apraxia (motor speech disorder) Loss of purposeful hand movement, stereotypic hand movement Balance and coordinacon issues, decrease or loss of ability to walk Respiratory dysfunccons "Re` Syndrome Fact Sheet", NINDS, PublicaHon date November 2009.NIH PublicaHon No Image Source: Re`Syndrome.org, pahent Jilly 16
17 Ret Syndrome Data: OptokineCc Response (OKR) ANAXEX 2-73 (30 mg/kg po dosed daily for 4 weeks) was evaluated in the MECP2 Ret syndrome model using 7 months old mice, an age in which advanced pathology is evident The mice were tested for changes in optokinecc (automacc visual) response # RaConale This method depends on the automacc visual response of head-tracking to a moving verccal stripe patern presented on a rotacng drum to an animal placed at its center # The study was sponsored by Re`syndrome.org and performed by PsychoGenics, Inc. 17
18 ANAVEX 2-73 Rescues OptokineCc Response Vehicle-treated HET mice showed fewer responses than vehicle-treated WT mice at 1.5 RPM, 2.8 RPM as well as total of both drum speeds HET mice treated with AV2-73 (30 mg/kg po) showed an increased response compared to the vehicle-treated HET mice at 1.5 RPM as well as total of both drum speeds #p<0.05 compared to WT vehicle group *p<0.05 compared to HET vehicle group 18
19 ANAVEX 2-73 Demonstrated a Trend related to the amount of Apneas Apneas were defined as expiratory Cme greater than 1 second A trend was observed in MECP2 mice treated with ANAVEX 2-73 that is, a reduccon in apnea counts to levels comparable to those observed in wild-type animals 19
20 The Effects of ANAVEX 2-73 in Neurodevelopmental Disorders Previous results have demonstrated that administracon of ANAVEX 2-73 results in both significant and dose related improvements in an array of movement paradigms in the MECP2 HET Ret syndrome disease model and in the Fragile X Fmr1 KO disease model The new results further suggest that ANAVEX 2-73 could be of potencal value for the treatment of various neurodevelopmental diseases including Ret syndrome, Fragile X and Angelman syndrome Coupled with posicve human safety and clinical cognicon data, as well as preclinical anc-seizure and anc-anxiety data, ANAVEX 2-73 will be tested as a potencal drug candidate in Ret syndrome 20
21 ANAVEX 2-73 Clinical Trial Strategy ANAVEX Study: Phase 1 (oral) Single Ascending Dose (SAD) Healthy subjects Completed More informacve trial ANAVEX Study # : Phase 2a (iv/oral) Mild-to-moderate AD pacents AdapCve trial with PopulaCon PK Bioavailability, dose finding (PART A), and exploratory efficacy with 52 week open-label extension (PART B) ANAVEX Study: 104-week extension study ayer PART B PART A: Completed PART B: Completed PopulaCon PK analysis: Upcoming readout ANAVEX Study: Phase 2 (oral) Ret syndrome pacents Double-blind, placebo controlled study <3 month efficacy ANAVEX Study: Phase 2/3 (oral) Mild-to-moderate AD pacents Double-blind, placebo controlled study 6/12 month efficacy PreparaCon underway # ClinicalTrials.gov IdenHfier: NCT
22 ANAVEX 2-73 Phase 2a: 57 Week Safety Profile (MTD Study) 32 mild-to-moderate AD pacents (baseline MMSE: 16-28) The most common AEs at highest doses were mild dizziness followed by mild headache Consistent with Blood Brain Barrier (BBB) penetracon 98% of all AEs were mild or moderate and reversible with 76% being Grade 1 2% were Grade 3 There were no Grade 4 and 5 events AE profile similar to that of healthy volunteer Phase 1 data No differences in blood pressure or rescng heart rate Clinical laboratory parameters, vital signs, and 12-lead ECG did not show any clinically relevant or dose-dependent changes Voges et al., presented at CNS Summit 2014; Macfarlane, presented at CTAD
23 2017 Clinical funding: 23
24 Contact Us Corporate Office: Anavex Life Sciences Corp. 51 West 52nd Street, 7th floor New York, NY (212) Shareholder & Media RelaCons: Toll-free: Outside North America: + 1 (917) ir@anavex.com NASDAQ: AVXL ANAVEX is a trademark of Anavex Life Sciences Corp. 24
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