VistaGen Therapeutics, Inc. (Nasdaq: VTGN), is a clinical-stage biopharmaceutical company developing new generation medicines for depression and other central nervous system (CNS) disorders. Our lead CNS product candidate, AV-101, is an oral N-methyl-D-aspartate (NMDA) receptor glycine B (GlyB) antagonist, belonging to a new generation of investigational medicines in neuropsychiatry known as glutamate receptor modulators having the potential to treat MDD faster than current FDA-approved antidepressants commonly known as SSRIs and SNRIs, which target the neurotransmitters serotonin and/or norepinephrine, respectively. AV-101 has a novel mechanism of action (MOA), meaning its MOA is fundamentally different from all current FDA-approved SSRIs and SNRIs for depression, most of which, if effective, take many weeks to achieve therapeutic benefits. AV-101 has the 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 also launched ELEVATE, a 180-patient Phase 2 study of AV-101 as an adjunctive treatment of MDD in patients with inadequate response to current FDA-approved antidepressants. Dr. Maurizio Fava of Harvard University is 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 neuropathic pain, Parkinson’s disease levodopa-induced dyskinesia (PD LID) and other diseases and disorders where modulation of NMDA receptors, activation of AMPA pathways 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 focused on 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 utilizing 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. 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.