Vistagen Therapeutics, Inc. · NASDAQ:VTGN
Vistagen Therapeutics — Investor Presentation (May 2019)
45 slides from Vistagen Therapeutics's investor presentation, May 2, 2019
Slide 1: Looking Beyond Current Treatments for CNS Diseases and Disorders
Ke) VistaGene Therapeutics LOOKING BEYOND CURRENT TREATMENTS FOR CNS DISEASES AND DISORDERS WITH HIGH UNMET NEED £ 8 e & & £ x 3 3 3 g® Nasdaq:vTen Spring 2019
Slide 2: Forward-looking Statements
www.vistagen.com Forward-looking Statements This presentation contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements concern our product candidates, our development efforts, our collaborations, our intellectu
Slide 3: Key Highlights
Key Looking beyond current therapies for CNS Me tionts diseases and disorders with high unmet need G) 3 late-stage CNS product candidates f COS A Multiple large target markets where current treatments fall short We www.vistagen.com J New generation MOAs Rapid-onset potential ks S Co) Exceptional saf
Slide 4: Pipeline
www.vistagen.com Ke) Pipeline VistaGen» ‘Therapeutics Product Candidate Indication Preclinical Phase 1 I94E Social Anxiety Disorder Major Depressive Disorder* (inadequate response to current ADT) Major Depressive Disorder (ketamine therapy responders) | RELAY Phase 2 Study preparation in pro.es# > S
Slide 5: Most Advanced Clinical Program: PH94B for Social Anxiety Disorder
Most Advanced Clinical Program * Preparing to Enter Pivotal Phase 3 * PH94B for Social Anxiety Disorder Ke) VistaGenoe Therapeutics JM nasdaq: vten LOOKING BEYOND CURRENT TREATMENTS FOR CENTRAL NERVOUS SYSTEM (CNS) DISEASES AND DISORDERS WITH HIGH UNMET NEED
Slide 6: Social Anxiety Disorder in the U.S.
www.vistagen.com National Institute of Mental Health = one heal Social Anxiety Disorder in the U.S. SAD is More than Just Shyness of the most prevalent me, \th conditions in the Ug noi $0! Affects as many as a Biving 22 million a eech TI meeting new people 7% and 9% i) eating/drinking jduals feel an
Slide 7: Current SAD Drug Treatments Fall Short
www.vistagen.com Current SAD Drug Treatments Fall Short ae ED YA Not FDA-Approved APPROVED * Used Off-label * Antidepressants Benzodiazepines & Beta Blockers x Slow onset, chronic administration x Addiction risk * May worsen anxiety initially x Sedation x Significant side effects x Cognitive impairm
Slide 8: PH94B: Rapid-Onset Efficacy, Exceptional Safety
PH94B ie Rapid-Onset Efficacy, Exceptional Safety uo i i H : = Novel CNS neuroactive steroid nasal spray = Activates nasal chemosensory receptors that trigger neural circuits in the brain that suppress fear and anxiety = PRN treatment prior to a fear/anxiety-producing situation = Rapid-onset efficac
Slide 9: PH94B Mechanism of Action
www.vistagen.com PH94B Mechanism of Action Activates Neural Circuits that Suppress Fear and Anxiety = Fundamentally different from all current SAD treatments = Activates nasal chemosensory receptors that trigger neural circuits in the brain that suppress fear and anxiety = Engages nasal chemosensory
Slide 10: PH94B Phase 2 Study - Public Speaking
PH94B Phase 2 POC Study - Public Speaking (n = 91) www.vistagen.com Visit 3 (Treatment Phase) 80 70 Placebo Subjective Units of Distress Score 69 (SuDsS) PHO4B 50 ao R& 1 240 141 2 3 4 5 (min); Anticipation| Performance ‘Anxiety Dosi = Challenge Challenge esn8 | 15-min” | instructions Starts Patient
Slide 11: PH94B Phase 2 Study - Social Interaction
www.vistagen.com PH94B Phase 2 POC Study - Social Interaction (n = 91) \y] Visit 3 (Treatment Phase) 60 55 a Place! Subjective Units of Distress Score 45 (suDs) 40 PH94B 35 3014 rR? a 2%6 2 2 3 4 = 5(min) Anticipatiot Performance ‘Anxiety ‘Anxiety ing [yea >| Challen; Challenge D ge 8 esiné J 15-min
Slide 12: PH94B Pilot Phase 3 Crossover Study
www.vistagen.com PH94B Pilot Phase 3 Crossover Study (n = 22) Thats Cross-ovi Phase 1 Phase 2 Screening Safety Period Follow-up 4 Period Visit 1 v2 v3 v4 V5 V6 Visit 7 Screening Randomization Follow-Up Adult subjects were randomized on a 1:1 basis to PH94B or placebo for two weeks, and then crossed
Slide 13: PH94B Pilot Phase Crossover Study: Peak SUDS
www.vistagen.com Ke) PH94B Pilot Phase 3 Crossover Study VistaGen- Peak Suds at Baseline and Weeks 1-4 (Ss ME Placebo ‘Therapeutics * Early suggestions of drug/placebo differences were seen in Week 1 and 2 SUDS scores: average change in SUDS at week 1 was 16.1 for PH94B versus 3.4 for placebo (t=1.8
Slide 14: PH94B Pilot Phase Crossover Study: LSAS
www.vistagen.com Ke) PH94B Pilot Phase 3 Crossover Study agate * In the sample as a whole, drop in LSAS scores after treatment did not differ between groups because subjects receiving PH94B before receiving placebo continued to improve when crossed over to placebo LSAS at Baseline and Weeks 1-4 * Af
Slide 15: PH94B Pilot Phase Crossover Study: LSAS vs SUDS
www.vistagen.com PH94B Pilot Phase 3 Crossover Study F z 5 £ 3 2 € & 5 = é 2 3 Predicting Change in LSAS from Change in SUDS During Week 1 0 10 20 30 ‘SUDS Change from Baseline Week 1 Ke) VistaGens ‘Therapeutics Changes in total LSAS scores were closely associated with change in SUDS peak anxiety sc
Slide 16: PH94B Development for SAD: Next Step
www.vistagen.com Ke) VistaGens PH94B Development for SAD — Next Step Therpets First U.S. Pivotal Phase 3 Study Principal Investigator: Dr. Michael Liebowitz, Columbia University, New York * Randomized, double-blind, placebo-controlled, monotherapy study * As-needed (PRN) self-administration of PH94B
Slide 17: First U.S. Pivotal Phase 3 Study design
www.vistagen.com PH94B Development for SAD — Next Step First U.S. Pivotal Phase 3 Study (n = ca. 200) Treatment Week 14 Screening Safety Follow-up Ke) VistaGens Therapeutics 7
Slide 18: AV-101 and PH10 for Major Depressive Disorder
Phase 2 Clinical Programs le) 4 AV-101 and PH10 for Major Depressive Disorder VistaGenoe Therapeutics PM nasdaq: VTGN LOOKING BEYOND CURRENT TREATMENTS FOR CENTRAL NERVOUS SYSTEM (CNS) DISEASES AND DISORDERS WITH HIGH UNMET NEED
Slide 19: Major Depressive Disorder - A Global Challenge
www.vistagen.com Major Depressive Disorder - A Global Challenge 300 million Depression is the leading cause of disability worldwide? suffer from depression worldwide? 1. World Health Organization The total estimated number of people living with depression increased by 18.4% between 2005 and 2015! q
Slide 20: Major Depressive Disorder in the U.S.
www.vistagen.com Ke) VistaGens Major Depressive Disorder in the U.S. meen 1in 4 women 1in6men 1in8 * r i) f p > ip ee ee @ & @ ov ar RETREAT fF THT diagnosed with depressive disorders age 12 and over takes an antidepressant’ LARGE ADDRESSABLE MARKET WITH HIGH UNMET NEED23 11.6M 7.3M 5.1M Drug-treate
Slide 21: Current FDA-Approved MDD Drug Treatments Fall Short
www.vistagen.com Ke) VistaGens Therapeutics Current FDA-Approved MDD Drug Treatments Fall Short Antidepressants Atypical Antipsychotics * Often do not work; slow to work * Often do not work — Initial ADT effective in 1 of 3 patients — Only ca. 20% of patients respond to — May take 4 to 6 weeks or mo
Slide 22: Ketamine-based Therapy for MDD
www.vistagen.com Ky] VistaGens <i ‘Therapeutics Ketamine-based Therapy for MDD Intravenous ketamine Intranasal ketamine Ls: J The New Mork Cimes fr \ “Fast-Acting Depression ‘Ketamine HCl Drug, Newly Approved, ae Could Help Millions” 's etd +—£ %% CNBC “gy fare “Ketamine offers J&J’s new ketamine-li
Slide 23: Ketamine-based Therapy: Long-term Solution?
www.vistagen.com Ke) VistaGens Ketamine-based therapy for MDD offers new hope to Therapeutics millions, but is it a long-term solution? “...[I]t is necessary to recognize the major gaps that remain in our knowledge about the longer-term efficacy and safety of ketamine . . 1 infusions.” American Psyc
Slide 24: AV-101 for MDD: Transformative Potential
om + AV-101 for MDD: Transformative Potential ie. ‘Therapeutics Rapid-onset Potential, Exceptional Safety www.vistagen. = Oral prodrug of 7-Cl-KYNA = NMDA receptor glycine site antagonist (a full antagonist) = Rapid-onset antidepressant effects and neurogenesis in preclinical studies = Well-tolerate
Slide 25: AV-101's Mechanism of Action
AV-101’s Mechanism of Action Inhibits NMDA Receptor Activity www.vistagen.com NMDA Receptor Pharmacology Kt fo) 7-CI-KYNA ee (full antagonist) @-CHKYN) (ora livery to CS) Activated * Astyoetes Classic channel-blocking antagonists: —_ 7-chlorokynurenic acid ° © ae prin Nat Ca2+ ‘etamine (low adverse
Slide 26: AV-101's Mechanism of Action
www.vistagen.com AV-101’s Mechanism of Action Inhibits NMDA Receptor Activity Ketamine completely blocks the ion channel of the NMDA receptor AV-101’s active metaboli not block NMDA receptor activity; Ke) VistaGens ‘Therapeutics (7-Cl-KYNA) does t inhibits it 26
Slide 27: AV-101 vs. Ketamine in Published Preclinical Studies
www.vistagen.com AV-101 vs. Ketamine in Published Preclinical Studies Ke) VistaGens Therapeutics ”The Prodrug 4-Chlorokynurenine Causes Ketamine-Like Antidepressant Effects, but Not Side Effects, by NMDA/GlycineB-Site Inhibition.” Zanos, P,, etal. (2015). J Pharmacol Exp Ther 355(1): 76-85. Antidepr
Slide 28: The ELEVATE Study
www.vistagen.com Ko) The ELEVATE Study Visage. AV-101 Phase 2 Adjunctive Treatment Study for MDD Principal Investigator: Dr. Maurizio Fava, Harvard Medical School * Adjunctive treatment for MDD patients with an inadequate response to a current FDA- approved oral antidepressant (with a current depres
Slide 29: Initial Objective for AV-101 in MDD
www.vistagen.com Ky] Initial Objective for AV-101 in MDD agate Displace atypical antipsychotics in current MDD drug treatment paradigm SSRI/SNRI SSRI/SNRI SSRI/SNRI 4-6 Weeks or more 4-6 Weeks or more 4-6 Weeks or more L/L LL / soc siosyenstis ///// / /
Slide 30: Proposed Next Step: The RELAY Study
www.vistagen.com Ko) Proposed Next Step: The RELAY Study Visage. Prevent relapse following ketamine-based therapy Multi-center, double blind, placebo-controlled, adjunctive treatment study AV-101 + standard antidepressant vs. placebo + standard antidepressant in patients who have responded to IV ket
Slide 31: PH10 for MDD
( PH10 for MDD on Rapid-Onset, Exceptional Safety = Novel CNS neuroactive steroid nasal spray = Activates nasal chemosensory receptors that trigger neural circuits in the brain leading to antidepressant effects = Rapid-onset antidepressant efficacy in Phase 2A = Microgram dose, no systemic exposure
Slide 32: PH10 Mechanism of Action
www.vistagen.com PH10 Mechanism of Action on Activates Neural Circuits Leading to Antidepressant Effects Fundamentally different from all current treatments Activates nasal chemosensory receptors that trigger neural circuits in the brain leading to antidepressant effects = Engages nasal chemosensory
Slide 33: PH10 Phase 2A MDD Study - Monotherapy
www.vistagen.com PH10 Phase 2A MDD Study — Monotherapy (n = 22) Hamilton Depression (HAM D) Score Reduction From Baseline -10.0 712.5 -15.0 Reduction in HAM D from Baseline -17.5 (n=30) Txwki TxwWk2 Txwk3 Txwk4 TxwWkS Txwk6 Txwk7 Txwks 3.2419 ( 6.4 41g ( Placebo rf ne fr FF f B Dose) 01 .74 Dose) 22
Slide 34: PH10 Development for MDD - Next Step
www.vistagen.com \} PH10 Development for MDD — Next Step agate Phase 2B Study Principal Investigator: Dr. Michael Liebowitz, Columbia University, New York * Randomized, double-blind, placebo-controlled, multi-center monotherapy study * MDD patients with zero or 1 prior failure on a standard antidepr
Slide 35: PH10 Phase 2B MDD Monotherapy Study
2 j : : PH10 Phase 2B MDD Monotherapy Study 4-week study in MDD patients after zero or one standard ADT failure MDD N=200 PH10 1.6 1g N=50 PH10 3.2 1g N=50 PH10 6.4 ng N=50 35
Slide 36: Summary: AV-101 and PH10 for MDD
www.vistagen.com Summary: AV-101 and PH10 for MDD Multiple Shots on Goal Augmentation for inadequate response to SSRIs/SNRIs ¢ AV-101 ELEVATE Study (ongoing, topline results in 2019) Relapse prevention after successful ketamine-based therapy ¢ AV-101 RELAY Study (preparing for launch in 2H 2020) Fir
Slide 37: AV-101 for Suicidal Ideation
Phase 1B Clinical Program Ke) ‘A VistaGenoe Therapeutics AV-101 for Suicidal Ideation PM nasdaq: VTGN LOOKING BEYOND CURRENT TREATMENTS FOR CENTRAL NERVOUS SYSTEM (CNS) DISEASES AND DISORDERS WITH HIGH UNMET NEED
Slide 38: AV-101 for Suicidal Ideation: Baylor / VA Phase 1B Study
www.vistagen.com Ke) Medicine aylor VistaGens AV-101 for Suicidal Ideation US. Department Amma VS. of Teens Affairs ee Baylor / VA Phase 1B Clinical Study Primary Objective: Target engagement relevant to NMDA antagonism and suicidal ideation Sponsored by U.S. Department of Veteran’s Affairs (VA) Ong
Slide 39: Additional Potential Programs
Additional Potential Programs _ Ke) AV-101 ' VistaGene for £ Therapeutics Neuropathic Pain Je and Parkinson’s LID PM nasdaq: VTGN LOOKING BEYOND CURRENT TREATMENTS FOR CENTRAL NERVOUS SYSTEM (CNS) DISEASES AND DISORDERS WITH HIGH UNMET NEED
Slide 40: AV-101 for Neuropathic Pain and Parkinson's LID
www.vistagen.com Ke) VistaGens Therapeutics AV-101 for Neuropathic Pain AV-101 for Parkinson’s LID Potential oral non-opioid treatment option * Non-addictive and non-sedating in published preclinical studies * FDA Fast Track designation “[WJe’ve undertaken ... steps to advance the development of non
Slide 41: Experienced Team
www.vistagen.com Ke) Experienced Team VistaGens Leading Execution Shawn K. Singh Chief Executive Officer + 25 years of experience with biopharmaceutical companies, a healthcare venture capital firm and a profitable CRO * Artemis Neuroscience; SciClone Pharmaceuticals; Cato BioVentures; Cato Research
Slide 42: Board of Directors
Board of Directors www.vistagen.com Jerry Gin, Ph.D., MBA + 45 years of healthcare industry experience; Co-Founder of Oculex (acquired by Allergan for $230M) * Co-Founder, President and CEO of Nuvora Shawn Singh, JD, CEO + 25 years of experience with biopharmaceutical companies, a venture capital fi
Slide 43: Distinguished Clinical and Regulatory Advisors
www.vistagen.com HARVARD UNIVERSITY Couwmmia Usivenstry DEPARTMENT OF PSYCHIATRY FDA, Maurizio Fava, M.D. Professor of Psychiatry, Harvard Medical School; Director, Division of Clinical Research, Massachusetts General Hospital (MGH) Research Institute; Executive Director, MGH Clinical Trials Network
Slide 44: Looking Beyond Current Therapies for CNS Diseases
(e) Looking beyond current therapies for CNS -. diseases and disorders with high unmet need G) 3 late-stage CNS product candidates f COS A Multiple large target markets where current treatments fall short We www.vistagen.com J New generation MOAs Rapid-onset potential ks S Co) Exceptional safety as
Slide 45: VistaGen Therapeutics
Ke) VistaGene Therapeutics LOOKING BEYOND CURRENT TREATMENTS FOR CNS DISEASES AND DISORDERS WITH HIGH UNMET NEED £ 8 e & & £ 2 3 3 3 g® Nasdaq:vTen
VistaGen Therapeutics — Investor Presentation (May 2019) on 6ix