VistaGen Therapeutics, Inc. (NASDAQ: VTGN), 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 a new generation oral antidepressant drug candidate in Phase 2 development. AV-101's mechanism of action is fundamentally differentiated from all FDA-approved antidepressants and atypical antipsychotics used adjunctively to treat major depressive disorder (MDD), with potential to drive a paradigm shift towards a new generation of safer and faster-acting antidepressants.
AV-101 is currently being evaluated by the U.S. National Institute of Mental Health (NIMH) in a Phase 2 monotherapy study in MDD being fully funded by the NIMH and conducted by Dr. Carlos Zarate Jr., 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. VistaGen is also preparing to launch a 180-patient Phase 2 study of AV-101 as an adjunctive treatment for MDD patients with inadequate response to standard, FDA-approved antidepressant therapies. Dr. Maurizio Fava of Harvard University will be the Principal Investigator of the Phase 2 adjunctive treatment study.
AV-101 may also have the potential to treat multiple CNS disorders and neurodegenerative diseases in addition to MDD, including chronic neuropathic pain, epilepsy, Parkinson's disease and Huntington's disease, where modulation of the NMDAR, AMPA pathway and/or key active metabolites of AV-101 may achieve therapeutic benefit.
In addition to our AV-101 programs, VistaStem, VistaGen’s wholly owned subsidiary, is 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 pipeline through cardiac liability-focused small molecule 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 focused on collaborating with others to advance development and commercialization of medicine and cell therapy applications of our stem cell technology across a range of cell types, including blood, bone, cartilage, heart and liver cells. In December 2016, we entered into an exclusive sublicense agreement with BlueRock Therapeutics L.P, a next generation regenerative medicine company established by Bayer AG and Versant Ventures, for our rights to proprietary technologies relating to the production of cardiac stem cells for the treatment of heart disease.