VistaGen Therapeutics (VTGN) Study update summary

Overview of PALISADE-4 study results

• PALISADE-4 Phase 3 trial evaluating intranasal fasedienol for acute treatment of social anxiety disorder did not achieve its primary or secondary endpoints in the overall population of 238 adults.

• The primary endpoint was SUDS score during a simulated public speaking challenge; the difference between fasedienol and placebo was not statistically significant.

• Safety and tolerability of fasedienol remained favorable and consistent with previous trials, with no new safety signals identified.

• Placebo responses showed greater variability than in prior studies, impacting overall results.

• Post-hoc analyses were conducted to further understand the data and inform future development.

Post-hoc and subpopulation analyses

• Subpopulation analysis focused on patients with very severe social anxiety disorder (LSAS ≥95), comprising just over half the study population (n=123).

• In this subgroup, fasedienol showed a nominally statistically significant improvement over placebo on the primary efficacy measure (SUDS), with a treatment difference of -9.1 points (p=0.036).

• Patient-reported secondary endpoint (PGIC) also showed nominal statistical significance in this subgroup.

• Analysis excluded data from a disqualified site and certain subjects to address irregularities and placebo effects.

• Pooled post-hoc analyses across PALISADE trials support the hypothesis that fasedienol's effect is most detectable in patients with high baseline anxiety.

Implications for regulatory pathway and future plans

• The totality of data, including positive PALISADE-2 results and favorable safety, supports discussions with the FDA for a new registrational pathway.

• Plans are underway to transition from acute symptom treatment to overall treatment of social anxiety disorder, using LSAS as the primary endpoint in a future multi-dose Phase 3 trial.

• The company aims to pursue a single trial registrational pathway with confirmatory evidence, leveraging recent FDA draft guidance.

• Confirmatory evidence will include PALISADE-2, Phase 2 trials, open label data, and aggregate safety data.

• Top-line results from a Phase 2 repeat dose study are expected next quarter and will inform further FDA discussions.