Description of Business	3 Months Ended
Jun. 30, 2015
Notes to Financial Statements
Description of Business	Overview VistaGen Therapeutics, Inc., a Nevada corporation, is a clinical-stage biopharmaceutical company committed to developing and commercializing innovative product candidates for patients with depression, other diseases and various disorders related to the central nervous system (CNS), as well as cancer. 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. VistaGen Therapeutics, Inc., a California corporation incorporated on May 26, 1998 (VistaGen California), is our wholly-owned subsidiary. Pursuant to a strategic merger transaction on May 11, 2011, we acquired all outstanding shares of VistaGen California in exchange for 341,823 shares of our common stock (Merger), and assumed all of VistaGen California’s pre-Merger obligations. 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. AV-101 and Major Depressive Disorder 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 2012, the NIH estimated 16 million adults aged 18 or older in the U.S. had at least one major depressive episode. This represented 6.9 percent of all U.S. adults. According to the U.S. Centers for Disease Control and Prevention (CDC), one in ten Americans take an antidepressant medication. Unfortunately, approximately two-thirds of depression sufferers do not benefit from the first round treatment of currently approved antidepressant agents, including selective serotonin reuptake inhibitors (SSRIs) and serotonin-norepinephrine reuptake inhibitors (SNRIs). And, even when they do relieve depressive symptoms and induce remission of a major depressive episode, because of their mechanism of action, SSRIs and SNRIs take many weeks to achieve those therapeutic benefits. During the multiple-week lag before onset of therapeutic benefits, risks of side effects, including suicidal thoughts and behaviors, may be considerable. Ultimately, after as many as four treatment cycles involving several different antidepressant medications, approximately two-thirds of patients may find an antidepressant drug or drug combination that induces remission of depressive symptoms. This trial and error period to find an effective antidepressant medication can take many months to more than a year to achieve, with an increasing rate of potentially significant side effect risks with each successive treatment attempt. Our lead product candidate, AV-101, is an orally available small molecule prodrug in Phase 2 clinical development for Major Depressive Disorder (MDD). AV-101’s mechanism of action (MOA), as an N-methyl-D-aspartate receptor (NMDAR) antagonist binding selectively at the glycine-binding (GlyB) co-agonist site of the NMDAR, is fundamentally different from all antidepressants approved by the U.S. Food and Drug Administration (FDA). In four preclinical studies utilizing well-validated animal models of depression, AV-101 was shown to induce fast-acting, dose-dependent, persistent and statistically significant antidepressant-like responses, following a single treatment, which were equivalent to responses seen with a control single sub-anesthetic dose of ketamine (an FDA-approved drug sometimes used by clinicians off-label to treat suicidal behavior). In the same preclinical studies, fluoxetine (Prozac) did not induce rapid onset antidepressant-like responses. 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. Following two successful randomized, double-blind, placebo-controlled Phase 1 safety studies funded by the NIH, in February 2015, we entered into a Cooperative Research and Development Agreement (CRADA) with the U.S. National Institute of Mental Health (NIMH), part of the NIH. Under the CRADA, we are collaborating with the NIMH on the initial Phase 2 clinical efficacy study of AV-101 in subjects with treatment-resistant MDD. Pursuant to the CRADA, the study will be conducted at the NIMH and be fully funded by the NIMH. It is contemplated that this clinical study will begin in the second half of 2015 under the direction of 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. In addition to developing AV-101 for MDD and other CNS indications, we are applying our stem cell technology for drug rescue to identify and develop proprietary new chemical entities (NCEs) for our internal drug candidate pipeline and selected regenerative medicine opportunities. Drug rescue involves (1) using our customized in vitro bioassay systems to predict potential heart and liver toxicity of NCEs, (2) leveraging 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 or liver toxicity and are now available in the public domain, and (3) applying medicinal chemistry to produce safer proprietary NCEs for our internal development pipeline. Our CardioSafe 3D™ bioassay system uses our human pluripotent stem cell (hPSC)-derived cardiomyocytes, or human 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. We use our hPSC-derived hepatocytes, or human liver cells, in our LiverSafe 3D™ bioassay system to predict potential liver toxicity of NCEs, including potential drug metabolism issues and adverse drug-drug interactions. CardioSafe 3D and LiverSafe 3D offer a new paradigm for evaluating and predicting potential heart and liver toxicity of NCEs, including drug rescue NCEs, early in the development process, long before costly, high risk animal studies and human clinical trials. We intend to develop internally for our pipeline each lead drug rescue NCEs we produce. Our focus in regenerative medicine is on innovative therapeutic opportunities involving blood, cartilage, heart and liver cells.

Description of Business

3 Months Ended

Jun. 30, 2015

Overview

VistaGen Therapeutics, Inc., a Nevada corporation, is a clinical-stage biopharmaceutical company committed to developing and commercializing innovative product candidates for patients with depression, other diseases and various disorders related to the central nervous system (CNS), as well as cancer. 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.

VistaGen Therapeutics, Inc., a California corporation incorporated on May 26, 1998 (VistaGen California), is our wholly-owned subsidiary. Pursuant to a strategic merger transaction on May 11, 2011, we acquired all outstanding shares of VistaGen California in exchange for 341,823 shares of our common stock (Merger), and assumed all of VistaGen California’s pre-Merger obligations. 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.

AV-101 and Major Depressive Disorder

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 2012, the NIH estimated 16 million adults aged 18 or older in the U.S. had at least one major depressive episode. This represented 6.9 percent of all U.S. adults. According to the U.S. Centers for Disease Control and Prevention (CDC), one in ten Americans take an antidepressant medication. Unfortunately, approximately two-thirds of depression sufferers do not benefit from the first round treatment of currently approved antidepressant agents, including selective serotonin reuptake inhibitors (SSRIs) and serotonin-norepinephrine reuptake inhibitors (SNRIs). And, even when they do relieve depressive symptoms and induce remission of a major depressive episode, because of their mechanism of action, SSRIs and SNRIs take many weeks to achieve those therapeutic benefits. During the multiple-week lag before onset of therapeutic benefits, risks of side effects, including suicidal thoughts and behaviors, may be considerable. Ultimately, after as many as four treatment cycles involving several different antidepressant medications, approximately two-thirds of patients may find an antidepressant drug or drug combination that induces remission of depressive symptoms. This trial and error period to find an effective antidepressant medication can take many months to more than a year to achieve, with an increasing rate of potentially significant side effect risks with each successive treatment attempt.

Our lead product candidate, AV-101, is an orally available small molecule prodrug in Phase 2 clinical development for Major Depressive Disorder (MDD). AV-101’s mechanism of action (MOA), as an N-methyl-D-aspartate receptor (NMDAR) antagonist binding selectively at the glycine-binding (GlyB) co-agonist site of the NMDAR, is fundamentally different from all antidepressants approved by the U.S. Food and Drug Administration (FDA). In four preclinical studies utilizing well-validated animal models of depression, AV-101 was shown to induce fast-acting, dose-dependent, persistent and statistically significant antidepressant-like responses, following a single treatment, which were equivalent to responses seen with a control single sub-anesthetic dose of ketamine (an FDA-approved drug sometimes used by clinicians off-label to treat suicidal behavior). In the same preclinical studies, fluoxetine (Prozac) did not induce rapid onset antidepressant-like responses. 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.

Following two successful randomized, double-blind, placebo-controlled Phase 1 safety studies funded by the NIH, in February 2015, we entered into a Cooperative Research and Development Agreement (CRADA) with the U.S. National Institute of Mental Health (NIMH), part of the NIH. Under the CRADA, we are collaborating with the NIMH on the initial Phase 2 clinical efficacy study of AV-101 in subjects with treatment-resistant MDD. Pursuant to the CRADA, the study will be conducted at the NIMH and be fully funded by the NIMH. It is contemplated that this clinical study will begin in the second half of 2015 under the direction of 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.

In addition to developing AV-101 for MDD and other CNS indications, we are applying our stem cell technology for drug rescue to identify and develop proprietary new chemical entities (NCEs) for our internal drug candidate pipeline and selected regenerative medicine opportunities. Drug rescue involves (1) using our customized in vitro bioassay systems to predict potential heart and liver toxicity of NCEs, (2) leveraging 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 or liver toxicity and are now available in the public domain, and (3) applying medicinal chemistry to produce safer proprietary NCEs for our internal development pipeline. Our CardioSafe 3D™ bioassay system uses our human pluripotent stem cell (hPSC)-derived cardiomyocytes, or human 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. We use our hPSC-derived hepatocytes, or human liver cells, in our LiverSafe 3D™ bioassay system to predict potential liver toxicity of NCEs, including potential drug metabolism issues and adverse drug-drug interactions. CardioSafe 3D and LiverSafe 3D offer a new paradigm for evaluating and predicting potential heart and liver toxicity of NCEs, including drug rescue NCEs, early in the development process, long before costly, high risk animal studies and human clinical trials. We intend to develop internally for our pipeline each lead drug rescue NCEs we produce. Our focus in regenerative medicine is on innovative therapeutic opportunities involving blood, cartilage, heart and liver cells.