VistaGen Therapeutics (VTGN) investor relations material

Program updates and clinical development

• Fasedienol is being developed as a rapid-onset, non-systemic intranasal treatment for social anxiety disorder, currently in the PALISADE phase III program with several studies completed and PALISADE-4 ongoing, expected to read out in the first half of the year.

• Analysis of PALISADE-1, 2, and 3 showed a consistent treatment effect for fasedienol, with variability mainly due to placebo response, informing operational and statistical strategies for PALISADE-4.

• Enhanced site training, recruitment optimization, and monitoring have been implemented to improve consistency and mitigate placebo effects in PALISADE-4.

• AI and machine learning are being used to analyze vocal biomarkers and behavioral indicators from previous studies to refine statistical analysis and potentially adjust the SAP for PALISADE-4.

• PALISADE-4 incorporates cumulative learnings from prior studies, with operational and statistical adjustments based on advanced analytics and site feedback.

Mechanism of action and supporting data

• Fasedienol belongs to the pherine class, acting locally on nasal chemosensory neurons that project to brain regions involved in emotional regulation, with no detectable blood levels.

• Over 15 phase I studies, in vitro work, and autonomic data collection have demonstrated biological activity and informed dose selection.

• Dose selection was guided by receptor activity studies, including electrogram measurements of nasal receptors and olfactory bulbs, showing rapid neural activation leading to the amygdala.

• Early clinical and pharmacodynamic studies, as well as controlled trials, support anxiolytic activity.

Regulatory and future plans

• If PALISADE-4 is successful, the data package will include two adequate and well-controlled phase III studies, real-world evidence, and a robust safety database nearing 1,500 exposures.

• PALISADE-2, despite being stopped early, is considered registration-worthy and anchors the NDA submission alongside PALISADE-4.

• Secondary endpoints such as CGI and PGIC are important for demonstrating concordance but are not key for labeling.

• Open-label and real-world data are being collected to assess utilization and impact in daily life, supplementing controlled trial evidence.