Description of Business	6 Months Ended
Sep. 30, 2015
Notes to Financial Statements
Description of Business	Overview VistaGen Therapeutics, Inc., (OTCQB: VSTA), a Nevada corporation, is a clinical-stage biopharmaceutical company committed to developing and commercializing innovative product candidates for patients with depression, cancer, other diseases involving the central nervous system (CNS), as well as certain neurodegenerative diseases. Our principal executive offices are located at 343 Allerton Avenue, South San Francisco, California 94080, and our telephone number is (650) 577-3600. Our website address is www.vistagen.com. Unless the context otherwise requires, the words “VistaGen Therapeutics, Inc.” “VistaGen,” “we,” “the Company,” “us” and “our” refer to VistaGen Therapeutics, Inc., a Nevada corporation. AV-101, our new generation, orally available prodrug candidate is in Phase 2 development, initially for the adjunctive treatment of Major Depressive Disorder (MDD) in patients with an inadequate response to standard antidepressants. AV-101’s novel mechanism of action, as an N-methyl D aspartate receptor (NMDAR) antagonist binding selectively at the glycine binding (GlyB) co-agonist site of the NMDAR, is fundamentally differentiated from all antidepressants currently approved by the U.S. Food and Drug Administration (FDA). A Phase 2A clinical study of AV-101 in subjects with treatment-resistant MDD is being conducted and funded by the National Institutes of Mental Health (NIMH) under a Cooperative Research and Development Agreement (CRADA). The Principal Investigator of this NIMH-funded Phase 2A study, which was initiated in October 2015, is Dr. Carlos Zarate, Jr., Chief of the NIMH’s Experimental Therapeutics & Pathophysiology Branch and its Section on Neurobiology and Treatment of Mood and Anxiety Disorders. Beyond MDD, we believe that AV-101 has therapeutic potential in additional CNS indications, including neuropathic pain and epilepsy, and in neurodegenerative diseases such as Parkinson’s disease and Huntington’s disease. In addition to our focus on CNS and neurology, we are applying our proprietary, human pluripotent stem cell (hPSC) technology for drug rescue to develop proprietary new chemical entities (NCEs) for our internal drug candidate pipeline. Initial drug rescue programs are focused on NCEs for the treatment of cancer. We are also considering regenerative medicine applications of our stem cell technology platform. Using hPSC-derived blood, cartilage, heart and liver cells in collaborative arrangements with academic research partners, including our co-founder and the Chairman of our Scientific Advisory Board, Gordon Keller, Ph.D., the Director of the University Health Network's McEwen Centre for the Regenerative Medicine in Toronto, we may pursue development of novel in vitro human disease models to produce for our pipeline NCEs and biologics with regenerative potential and nonclinical proof of concept studies focused on cell therapy. AV-101 and Major Depressive Disorder Background The World Health Organization estimates that 350 million people worldwide are affected by depression. According to the U.S. National Institutes of Health (NIH), major depression is one of the most common mental disorders in the U.S. In 2013, the NIMH estimated that 15.7 million adults aged 18 or older in the U.S. had at least one major depressive episode in the past year. This represented 6.7 percent of all U.S. adults. According to the U.S. Centers for Disease Control and Prevention (CDC), one in 10 Americans take an antidepressant medication. Unfortunately, millions of depression sufferers do not benefit from initial treatment with standard antidepressants, which generally consists of a selective serotonin reuptake inhibitor (SSRI) or serotonin-norepinephrine reuptake inhibitor (SNRI). Moreover, even when they do relieve depressive symptoms and induce remission of a major depressive episode, SSRIs and SNRIs take many weeks to achieve therapeutic benefits because of their mechanism of action. During the multiple-weeks to months before onset of therapeutic benefits, side effects of SSRIs and SNRIs, including anxiety, metabolic syndrome, sleep disturbance, sexual dysfunction and suicidal thoughts and behaviors, may be considerable. Unfortunately, even after as many as four different treatment cycles, millions of patients suffering with MDD (over 30% of drug-related MDD patients) do not achieve an adequate therapeutic response to standard antidepressant therapies. AV-101 AV-101, our orally available prodrug candidate, is in Phase 2 clinical development, initially for the adjunctive treatment of MDD patients with an inadequate response to standard antidepressant therapies. As published in the October 2015 issue of the peer-reviewed, Journal of Pharmacology and Experimental Therapeutics, in an article entitled, The prodrug 4-chlorokynurenine causes ketamine-like antidepressant effects, but not side effects, by NMDA/glycineB-site inhibition, AV-101 was shown to induce fast-acting, dose-dependent, persistent and statistically significant antidepressant-like responses, following a single treatment, which responses were equivalent to responses seen with a control single sub-anesthetic dose of ketamine (an FDA-approved anesthetic administered intravenously by clinicians off-label to treat MDD patients who have not responded adequately to standard antidepressant therapies). In the same preclinical studies, fluoxetine (Prozac), an SSRI, did not induce rapid onset antidepressant-like responses. Following two successful randomized, double-blind, placebo-controlled Phase 1A and Phase 1B safety studies funded by the NIH, we are now collaborating with the NIMH on a Phase 2A efficacy and safety study of AV-101 in subjects with treatment-resistant MDD. This NIMH-funded Phase 2A study, began in October 2015, and is expected to enroll up to 28 patients. Dr. Carlos Zarate, Jr., Chief of the NIMH's Experimental Therapeutics & Pathophysiology Branch and its Section on Neurobiology and Treatment of Mood and Anxiety Disorders, is the Principal Investigator of the study. Preclinical studies also support the hypothesis that AV-101 has the potential to treat several additional CNS disorders, including chronic neuropathic pain, epilepsy and neurodegenerative diseases, such as Parkinson’s disease and Huntington’s disease, where modulation of the NMDAR may have therapeutic benefit. NCE Drug Rescue Our drug rescue strategy involves using CardioSafe 3D, our customized in vitro bioassay system, to predict potential human heart toxicity of NCEs, long before they are ever tested in animal and human studies. Our drug rescue programs are focused on recapturing value from substantial prior investments by pharmaceutical companies and others related to screening large-scale compound libraries, optimizing and testing for efficacy NCEs that were terminated before FDA approval due to heart toxicity risks and are now amendable to drug rescue and available in the public domain. CardioSafe 3D™ includes our human pluripotent stem cell (hPSC)-derived human heart cells. Due to the high purity and functionality of our hPSC-derived heart cells, we believe CardioSafe 3D is more comprehensive and clinically predictive than the hERG assay, which is currently the only in vitro cardiac safety assay required by FDA guidelines. CardioSafe 3D offers a new paradigm for evaluating and predicting potential human heart toxicity of NCEs early in the development process, long before costly, high risk animal studies and human clinical trials. Combining our stem cell biology expertise, the clinically predictive power of CardioSafe 3D, and contract medicinal chemistry, our drug rescue programs are focused on producing and developing safe and effective proprietary NCEs for our drug candidate pipeline. Regenerative Medicine We believe that stem cell technology-based regenerative medicine has the potential to transform the U.S. healthcare system over the next two decades by altering the fundamental mechanisms of disease, and helping to slow rapidly rising healthcare costs. Using hPSC-derived blood, bone, cartilage, heart, and liver cells, our current interests in the regenerative medicine arena include developing novel human disease models for discovery of small molecule drugs and biologics that activate the endogenous growth and healing processes (enabling the body to repair tissue damage caused by certain degenerative diseases) and, through collaborative nonclinical proof of concept studies with academic research partners, exploring potential regenerative cell therapy applications. Subsidiaries and Stock Consolidation VistaGen Therapeutics, Inc., a California corporation (VistaGen California), is our wholly-owned subsidiary. Our Condensed Consolidated Financial Statements in this Report also include the accounts of VistaGen California’s two wholly-owned subsidiaries, Artemis Neuroscience, Inc., a Maryland corporation, and VistaStem Canada, Inc., a corporation organized under the laws of Ontario, Canada. Effective August 14, 2014, we consummated a 1-for-20 reverse split of our authorized, and issued and outstanding shares of common stock (Stock Consolidation). Each reference to shares of common stock or the price per share of common stock in these financial statements is post-Stock Consolidation, and reflects the 1-for-20 adjustment as a result of the Stock Consolidation.

Description of Business

6 Months Ended

Sep. 30, 2015

Overview

VistaGen Therapeutics, Inc., (OTCQB: VSTA), a Nevada corporation, is a clinical-stage biopharmaceutical company committed to developing and commercializing innovative product candidates for patients with depression, cancer, other diseases involving the central nervous system (CNS), as well as certain neurodegenerative diseases. Our principal executive offices are located at 343 Allerton Avenue, South San Francisco, California 94080, and our telephone number is (650) 577-3600. Our website address is www.vistagen.com. Unless the context otherwise requires, the words “VistaGen Therapeutics, Inc.” “VistaGen,” “we,” “the Company,” “us” and “our” refer to VistaGen Therapeutics, Inc., a Nevada corporation.

AV-101, our new generation, orally available prodrug candidate is in Phase 2 development, initially for the adjunctive treatment of Major Depressive Disorder (MDD) in patients with an inadequate response to standard antidepressants. AV-101’s novel mechanism of action, as an N-methyl D aspartate receptor (NMDAR) antagonist binding selectively at the glycine binding (GlyB) co-agonist site of the NMDAR, is fundamentally differentiated from all antidepressants currently approved by the U.S. Food and Drug Administration (FDA). A Phase 2A clinical study of AV-101 in subjects with treatment-resistant MDD is being conducted and funded by the National Institutes of Mental Health (NIMH) under a Cooperative Research and Development Agreement (CRADA). The Principal Investigator of this NIMH-funded Phase 2A study, which was initiated in October 2015, is Dr. Carlos Zarate, Jr., Chief of the NIMH’s Experimental Therapeutics & Pathophysiology Branch and its Section on Neurobiology and Treatment of Mood and Anxiety Disorders.

Beyond MDD, we believe that AV-101 has therapeutic potential in additional CNS indications, including neuropathic pain and epilepsy, and in neurodegenerative diseases such as Parkinson’s disease and Huntington’s disease.

In addition to our focus on CNS and neurology, we are applying our proprietary, human pluripotent stem cell (hPSC) technology for drug rescue to develop proprietary new chemical entities (NCEs) for our internal drug candidate pipeline. Initial drug rescue programs are focused on NCEs for the treatment of cancer.

We are also considering regenerative medicine applications of our stem cell technology platform. Using hPSC-derived blood, cartilage, heart and liver cells in collaborative arrangements with academic research partners, including our co-founder and the Chairman of our Scientific Advisory Board, Gordon Keller, Ph.D., the Director of the University Health Network's McEwen Centre for the Regenerative Medicine in Toronto, we may pursue development of novel in vitro human disease models to produce for our pipeline NCEs and biologics with regenerative potential and nonclinical proof of concept studies focused on cell therapy.

AV-101 and Major Depressive Disorder

Background

The World Health Organization estimates that 350 million people worldwide are affected by depression. According to the U.S. National Institutes of Health (NIH), major depression is one of the most common mental disorders in the U.S. In 2013, the NIMH estimated that 15.7 million adults aged 18 or older in the U.S. had at least one major depressive episode in the past year. This represented 6.7 percent of all U.S. adults. According to the U.S. Centers for Disease Control and Prevention (CDC), one in 10 Americans take an antidepressant medication.

Unfortunately, millions of depression sufferers do not benefit from initial treatment with standard antidepressants, which generally consists of a selective serotonin reuptake inhibitor (SSRI) or serotonin-norepinephrine reuptake inhibitor (SNRI). Moreover, even when they do relieve depressive symptoms and induce remission of a major depressive episode, SSRIs and SNRIs take many weeks to achieve therapeutic benefits because of their mechanism of action. During the multiple-weeks to months before onset of therapeutic benefits, side effects of SSRIs and SNRIs, including anxiety, metabolic syndrome, sleep disturbance, sexual dysfunction and suicidal thoughts and behaviors, may be considerable. Unfortunately, even after as many as four different treatment cycles, millions of patients suffering with MDD (over 30% of drug-related MDD patients) do not achieve an adequate therapeutic response to standard antidepressant therapies.

AV-101

AV-101, our orally available prodrug candidate, is in Phase 2 clinical development, initially for the adjunctive treatment of MDD patients with an inadequate response to standard antidepressant therapies. As published in the October 2015 issue of the peer-reviewed, Journal of Pharmacology and Experimental Therapeutics, in an article entitled, The prodrug 4-chlorokynurenine causes ketamine-like antidepressant effects, but not side effects, by NMDA/glycineB-site inhibition, AV-101 was shown to induce fast-acting, dose-dependent, persistent and statistically significant antidepressant-like responses, following a single treatment, which responses were equivalent to responses seen with a control single sub-anesthetic dose of ketamine (an FDA-approved anesthetic administered intravenously by clinicians off-label to treat MDD patients who have not responded adequately to standard antidepressant therapies). In the same preclinical studies, fluoxetine (Prozac), an SSRI, did not induce rapid onset antidepressant-like responses.

Following two successful randomized, double-blind, placebo-controlled Phase 1A and Phase 1B safety studies funded by the NIH, we are now collaborating with the NIMH on a Phase 2A efficacy and safety study of AV-101 in subjects with treatment-resistant MDD. This NIMH-funded Phase 2A study, began in October 2015, and is expected to enroll up to 28 patients. Dr. Carlos Zarate, Jr., Chief of the NIMH's Experimental Therapeutics & Pathophysiology Branch and its Section on Neurobiology and Treatment of Mood and Anxiety Disorders, is the Principal Investigator of the study.

Preclinical studies also support the hypothesis that AV-101 has the potential to treat several additional CNS disorders, including chronic neuropathic pain, epilepsy and neurodegenerative diseases, such as Parkinson’s disease and Huntington’s disease, where modulation of the NMDAR may have therapeutic benefit.

NCE Drug Rescue

Our drug rescue strategy involves using CardioSafe 3D, our customized in vitro bioassay system, to predict potential human heart toxicity of NCEs, long before they are ever tested in animal and human studies. Our drug rescue programs are focused on recapturing value from substantial prior investments by pharmaceutical companies and others related to screening large-scale compound libraries, optimizing and testing for efficacy NCEs that were terminated before FDA approval due to heart toxicity risks and are now amendable to drug rescue and available in the public domain.

CardioSafe 3D™ includes our human pluripotent stem cell (hPSC)-derived human heart cells. Due to the high purity and functionality of our hPSC-derived heart cells, we believe CardioSafe 3D is more comprehensive and clinically predictive than the hERG assay, which is currently the only in vitro cardiac safety assay required by FDA guidelines. CardioSafe 3D offers a new paradigm for evaluating and predicting potential human heart toxicity of NCEs early in the development process, long before costly, high risk animal studies and human clinical trials. Combining our stem cell biology expertise, the clinically predictive power of CardioSafe 3D, and contract medicinal chemistry, our drug rescue programs are focused on producing and developing safe and effective proprietary NCEs for our drug candidate pipeline.

Regenerative Medicine

We believe that stem cell technology-based regenerative medicine has the potential to transform the U.S. healthcare system over the next two decades by altering the fundamental mechanisms of disease, and helping to slow rapidly rising healthcare costs. Using hPSC-derived blood, bone, cartilage, heart, and liver cells, our current interests in the regenerative medicine arena include developing novel human disease models for discovery of small molecule drugs and biologics that activate the endogenous growth and healing processes (enabling the body to repair tissue damage caused by certain degenerative diseases) and, through collaborative nonclinical proof of concept studies with academic research partners, exploring potential regenerative cell therapy applications.

Subsidiaries and Stock Consolidation

VistaGen Therapeutics, Inc., a California corporation (VistaGen California), is our wholly-owned subsidiary. Our Condensed Consolidated Financial Statements in this Report also include the accounts of VistaGen California’s two wholly-owned subsidiaries, Artemis Neuroscience, Inc., a Maryland corporation, and VistaStem Canada, Inc., a corporation organized under the laws of Ontario, Canada.

Effective August 14, 2014, we consummated a 1-for-20 reverse split of our authorized, and issued and outstanding shares of common stock (Stock Consolidation). Each reference to shares of common stock or the price per share of common stock in these financial statements is post-Stock Consolidation, and reflects the 1-for-20 adjustment as a result of the Stock Consolidation.