Vistagen Therapeutics, Inc. · NASDAQ:VTGN
Vistagen Therapeutics — Investor Presentation (June 2015)
22 slides from Vistagen Therapeutics's investor presentation, Jun 16, 2015
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Slide 2: Forward-looking Statements
Forward-looking Statements ‘This presentation contains forward-looking statements. These statements relate to future events and involve known and unknown risks, uncertainties and other factors which may cause our actual results, performance or achievements to be materially different from any future
Slide 3: Company Overview
Company Overview ¢ Clinical-stage biopharmaceutical company in South San Francisco + AV-101 (4-CI-KYN or L-4-chlorokynurenine) - Orally-available CNS small molecule prodrug candidate - Potential to transform treatment of depression - Fundamentally novel mechanism of action; unlike all approved antid
Slide 4: Recent Developments
Recent Developments NIH Cooperative Research and Development Agreement for Phase 2 Study of AV-101 in Major Depressive Disorder - Study to be fully-funded and conducted by the NIH - Prior NIH awards for AV-101 preclinical and Phase 1 = $8.8 million Gerard Sanacora, PhD, MD appointed to Clinical Advi
Slide 5: Management
Management Chief xecutive Officer, Director Founder, President, CSO, Director Cato BioVentures, Cato Research; SciClone; Echo; Artemis Progenitor; Lineberger Comprehensive Cancer Center; Basel Institute for Immunology Calypte Biomedical Corporation (OTCBB: CBMC); Discovery Foods Ret., CEO or senior
Slide 6: Depression: Global Concern and Multi-Billion Dollar Market
Depression: Global Concern and Multi-Billion Dollar Market + 350 million worldwide suffer from depression World Health Organization . 6.7% of U.S. adults experience major depression each year U.S. National Institutes of Health + 1-in-10 in U.S. over age 12 takes antidepressant medication U.S. Center
Slide 7: Problems with Current Antidepressants
Problems with Current Antidepressants Most FDA-approved antidepressants target the neurotransmitters, serotonin and serotonin/norepinephrine (SSRIs and SNRIs) SSRIs and SNRIs take weeks to months to work and fail to help millions - Initial SSRI/SNRI treatments fail in over 67% of depression patients
Slide 8: VistaGen's Solution: AV-101 (L-4-chlorokynurenine)
VistaGen’s Solution: AV-101 (L-4-chlorokynurenine) * NMDA receptor glycine-binding site antagonist - Fundamentally different biochemical pathway compared to SSRIs and SNRIs - Functionally (not structurally) similar to ketamine as an NMDA receptor antagonist, but substantially safer due to selective
Slide 9: NIH Ketamine Study Revolutionizes Depression Treatment Paradigm
NIH Ketamine Study Revolutionizes C _" Depression Treatment Paradigm . @, GO * Ketamine - NMDA receptor antagonist (channel blocker) - FDA-approved dissociative anesthetic used medically since the 1970s - Used privately and recreationally — aka the club drug “Special K” - Causes hallucinations, sens
Slide 10: Mechanism of Action
Mechanism of Acti AV-101 and clinically-important NMDA teceptot modulators Glutamate Glycine ir binding site “oO binding site { Prodrug wD Neo Extracellular a side DOOOOOK 4-chlorokynurenine AV-101 (antagonist) (oral my to CNS) LD 61-13 (ariaagons (Activated astrocytes [petra 7-chlorokynurenic acid
Slide 11: AV-101 is positioned to become a transformative advancement in the treatment of depression
AV-101 is positioned to become a transformative advancement in the treatment of depressio: Nares Negative Clinical Attributes Ketamine Requires IV delivery o Narrow therapeutic range not anticipated Hallucinogenic Oo Schizophrenia-like effects a Positive Clinical Attributes } van | Ketamine Positive
Slide 12: Naurex comparison of NMDA receptor modulators
Naurex Naurex Compound Description Ketamine-like antidepressant effects Indication Clinical-stage Value/Recent Raise 12 AV-101 Novel small molecule modulator of NMDA receptor; NMDA receptor glycine-binding site antagonist; orally-active; potential first-line oral therapy for MDD Yes Major Depressive
Slide 13: AV-101 NIH-funded Phase 1a and 1b Clinical Safety Studies
AV-101 NIH-funded Phase 1a and 1b Clinical Safety Studies NIH-funded Single-site Progressive Medical Research), randomized, double-blind, placebo controlled Single oral dose (capsule) with sequential dose- escalation Six single dose levels: 30, 120, 360, 720, 1,080, and 1440 mg 36 subjects: 18 treat
Slide 14: AV-101 Phase 2 Study Major Depressive Disorder
AV-101 Phase 2 Study Major Depressive Disorder * 'To be fully-funded and conducted by NIH, beginning in 2H 2015 * Principal Investigator - Carlos Zarate, MD, Chief of Experimental Therapeutics and Pathophysiology at the NIHs National Institute of Mental Health - Principal investigator on NIH studies
Slide 15: AV-101 Neurology Platform
AV-101 Neutology Platform Product Indication Opportunity * Potential new generation first-line oral therapy * Secured NIH-sponsored Phase 2 study to begin in 2H 2015 + Reduced chronic neuropathic pain due to inflammation and nerve damage in three well- established live animal pain models + Reduced f
Slide 16: AV-101 Development Pipeline
AV-101 Development Pipeline Preclinical] Phase ta Phase tb Phase 2a Phase 2b Phase 3 Major Depressive Disorder Neuropathic Pain Neurodegenerative Diseases - Parkinson’s, Huntington’s 16 VistaGen
Slide 17: NCE Drug Rescue: CardioSafe 3D and LiverSafe 3D
NCE Drug Rescue: CardioSafe 3D™ and LiverSafe 3D!™ ¥Y3 of new drug candidates fail due to unexpected heart or liver toxicity Our clinically-predictive, customized bioassay systems use pluripotent stem cell- derived human heart cells and liver cells to predict potential heart and liver toxicity of ne
Slide 18: CardioSafe 3D: Going Far Beyond the hERG Assay
CardioSafe 3D™ Going Far Beyond the hERG Assay hERG potassium ion channels v other potassium ion channels = Calcium ion channels = Sodium ion channels _ Interactions between ion channels _ Channel regulatory proteins = Cell viability = Apoptosis _ Mitochondria = Energy _ Sy NS] SSSA SSS TESTES IES O
Slide 19: VSTA-heps (human hepatocytes) vs. Primary (cadaver) hepatocytes
VSTA-heps™ (human hepatocytes) vs. Primary (cadaver) hepatocytes Human cells v Liver enzyme activity ue Within batch reproducibility v Batch-to-batch reproducibility = Long term culture = Maintenance of function in culture —_— Parental cells can be expanded into large batches _ Uniform genetic backg
Slide 20: Regenerative Medicine and Cell Production Opportunities
Regenetative Medicine and Cell Production Opportunities Cardiovascular disease ac infarct ronary heart disease Liver dis: * Acute liver failure * Chronic he Fatty liver disease * Drug-induced liver failure 20 Joint repair Non-union bone repair VistaGen
Slide 21: VistaGen Summary
VistaGen Summaty + AYV-101 is orally-available and could transform treatment of depression + NIH-funded CRADA for AV-101 Phase 2 study in Major Depressive Disorder + NIH Phase 2 study of AV-101 to be conducted Dr. Carlos Zarate, Chief of Experimental ‘Therapeutics and Pathophysiology at the NIH’s Na
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