VistaGen Therapeutics (VTGN) Stifel 2025 Virtual CNS Forum summary

Strategic focus and clinical development

• Advancing a new class of fast-acting, non-systemic intranasal drugs (pherines) for neuropsychiatric disorders, with lead focus on acute treatment of social anxiety disorder (SAD).

• Lead asset has shown positive results in both phase II and phase III studies, with two additional phase III readouts expected by year-end.

• Enhancements in trial execution for PALISADE-3 and 4 include in-person training, direct PI administration, and rigorous protocol adherence via audio recordings.

• Study design leverages a standardized public speaking challenge and the SUDS scale for minute-to-minute anxiety assessment.

• Alignment with regulatory agencies on study design, endpoints, and repeat dosing safety studies.

Regulatory and clinical considerations

• PALISADE-2 is considered more representative due to post-pandemic operational improvements; PALISADE-1 seen as an outlier.

• Effect size expectations are based on a Cohen’s D of 0.4, consistent with neuropsych studies.

• Repeat dose studies address safety and inform potential labeling for acute, repeat use.

• Open-label and placebo-controlled studies suggest repeated use may improve patient outcomes and reduce avoidance behaviors.

• PALISADE-3 or 4, if positive, will complement PALISADE-2 for regulatory submission; safety database targets are being met.

Operational and commercial outlook

• Both PALISADE-3 and 4 are on track for top-line data readouts in 2024, with quality enrollment and protocol adherence emphasized.

• Acute treatment is episodic, with expected use patterns varying by individual lifestyle and needs.

• No direct analog exists for acute SAD treatment; digital and telehealth channels are expected to drive access and demand.

• Favorable payer feedback suggests no step therapy required due to lack of approved acute treatments.

• Cash runway extends through Q1 2026, with NDA submission targeted for the first half of 2026 if trials are positive.