VistaGen Therapeutics is a clinical-stage biopharmaceutical company focused on developing new generation medicines for depression and other central nervous system (CNS) disorders. Our lead CNS product candidate, AV-101, is an orally available prodrug in Phase 2 development, initially for the adjunctive treatment of major depressive disorder (MDD) in patients with inadequate response to standard FDA-approved antidepressants. AV-101 has the potential to be faster-acting and safer than standard FDA-approved antidepressants, as well as atypical antipsychotics commonly used adjunctively to augment them AV-101 is a NMDA receptor glycine-binding (Glyв) site antagonist and has been shown in published preclinical studies to provide fast-acting, ketamine-like, antidepressant effects without serious CNS side effects typically associated with ketamine and FDA-approved drug therapies for depression.
AV-101 is being evaluated in an ongoing Phase 2a monotherapy study in MDD, a trial being conducted at and funded by the U.S. National Institute of Mental Health (NIMH). The Principal Investigator (PI) of this study is Dr. Carlos Zarate, Chief, Section on the Neurobiology and Treatment of Mood Disorders and Chief of Experimental Therapeutics and Pathophysiology Branch at the NIMH, and one of the world's foremost experts on the use of low dose IV ketamine and other NMDA receptor antagonists to treat MDD. Completion of this study is currently anticipated for year end 2017. We are also preparing to launch a Phase 2b clinical study of AV-101 for the adjunctive treatment of MDD in the first half of 2017.
Based on preclinical studies and results from our Phase 1a and Phase 1b safety studies, we believe AV-101 also has the potential to address multiple neuropsychiatric, neurological and neurodegenerative diseases beyond MDD, including chronic neuropathic pain, epilepsy, Huntington's disease and Parkinson's disease.
In addition to our AV-101 programs, we are applying our human pluripotent stem cell (hPSC) technology platform and CardioSafe 3D™, our customized in-vitro human cardiac cell bioassay system, to predict potential heart toxicity of new chemical entities (NCEs) long before they are tested in preclinical animal studies and human clinical studies. Having successfully assessed AV-101 and numerous other drug candidates to establish the clinically predictive capabilities of CardioSafe 3D, we are now using CardioSafe 3D™ to expand our small molecule pipeline through cardiac liability-focused drug rescue, and to participate, together with a select group of companies, in the FDA's Comprehensive in vitro Proarrhythmia Assay (CIPA) initiative designed to change the landscape of preclinical drug development by providing a more complete and accurate assessment of potential drug effects on cardiac risk. We are also evaluating strategic alternatives to advance our stem cell technology platform for potential commercial applications involving small molecule regenerative medicine and cell therapy.