VistaGen Therapeutics, Inc. (NASDAQ: VTGN) is a clinical-stage biopharmaceutical company developing new generation medications for anxiety, depression and other central nervous system, or “CNS,” diseases and disorders where current treatments are inadequate to address high unmet need. Our CNS portfolio includes three differentiated CNS drug candidates, PH94B, PH10 and AV-101, each with a novel mechanism of action, an exceptional safety profile, and therapeutic potential for multiple indications.
PH94B Neuroactive Nasal Spray
PH94B is a first-in-class, odorless, rapid-onset (approximately 10 to 15 minutes) CNS neuroactive nasal spray. Administered in non-systemic microgram doses, PH94B binds to nasal chemosensory receptors which trigger neural circuits in the brain that suppress fear and anxiety associated with everyday social situations, as well as school, work, or other performance situations. In December 2019 the U.S. Food and Drug Administration (FDA) granted VistaGen’s request for FDA Fast Track designation for development of PH94B for treatment of social anxiety disorder (SAD), the first such designation ever granted by the FDA for a SAD drug candidate. PH94B has the potential to be the first FDA-approved, as-needed, on-demand treatment for millions of Americans who suffer from SAD. In addition, PH94B has potential in pre/post-partum anxiety, pre/post-operative anxiety, post-traumatic stress disorder, panic disorder, and generalized anxiety disorder. Following successful Phase 2 and pilot Phase 3 studies, VistaGen is preparing for Phase 3 clinical development of PH94B for treatment of SAD.
PH10 Neuroactive Nasal Spray
PH10 is a first-in-class, odorless, rapid-onset CNS neuroactive nasal spray in clinical development with potential as an innovative stand-alone and adjunctive treatment for major depressive disorder (MDD), treatment-resistant depression (TRD) and suicidal ideation (SI). Administered in non-systemic microgram doses, PH10 binds to nasal chemosensory receptors which activate neural circuits that lead to rapid antidepressant effects without psychological side effects or safety concerns that may be caused by current oral antidepressants and ketamine-based therapy (KBT). In an exploratory (n=30) randomized, double-blind, placebo-controlled Phase 2a clinical study in MDD, at microgram doses, rapid-onset antidepressant effects were observed and sustained for 8 weeks, without psychological side effects or safety concerns associated with KBT. VistaGen is preparing for planned Phase 2b clinical development of PH10 for MDD.
AV-101, an Oral NMDAR Glycine Site Antagonist
AV-101 (4-Cl-KYN) belongs to a new generation of investigational medicines in neuropsychiatry and neurology known as NMDAR (N-methyl-D-aspartate receptor) modulators. The NMDAR is a pivotal receptor in the brain and abnormal NMDAR function is associated with numerous CNS diseases and disorders. AV-101 is an oral prodrug of 7-chloro-kynurenic acid (7-Cl-KYNA), a potent and selective full antagonist of the glycine site of the NMDAR. With its exceptional side effect and safety profile in all studies to date (no psychological side effects or safety concerns similar to those that may be caused by amantadine and ketamine), AV-101 has potential to be a new oral, at-home, non-sedating treatment for multiple large market CNS indications where current treatments are inadequate to meet high unmet patient needs. The FDA has granted Fast Track designation for development of AV-101 as both a potential adjunctive treatment for MDD and as a non-opioid treatment for neuropathic pain. AV-101 also has potential as a new treatment for dyskinesia in Parkinson’s disease patients receiving levodopa therapy, epilepsy, and suicidal ideation.