VistaGen Therapeutics (VTGN) TD Cowen 46th Annual Health Care Conference summary

Platform and pipeline overview

• Five clinical-stage candidates developed from a pherine platform, all delivered as non-systemic nasal sprays targeting chemosensory neurons in the nasal passage.

• Lead asset, Fasedienol, is in phase III for acute treatment of social anxiety disorder; other assets target major depressive disorder, mental fatigue, women's health (vasomotor symptoms, PMDD), and cancer cachexia.

• Non-systemic delivery avoids typical side effects and abuse liability, offering rapid onset and patient agency in use.

Clinical development and trial insights

• Fasedienol's PALISADE program uses a public speaking challenge paradigm to assess efficacy in social anxiety disorder, with endpoints based on the Subjective Units of Distress Scale.

• PALISADE-2 showed statistically significant separation from placebo, marking the first positive phase III study in this setting; PALISADE-4 is ongoing with readout expected by end of H1 2026.

• Open label studies and exploratory endpoints (e.g., Liebowitz Social Anxiety Scale) indicate sustained improvement in real-world settings and strong safety profile.

• AI and machine learning are being used to analyze vocal biomarkers and covariates to refine statistical analysis and mitigate placebo effects in ongoing trials.

Regulatory and strategic pathways

• Multiple regulatory strategies are under consideration: conventional two-pivotal trial approach, single pivotal trial with compelling evidence, or complementary study designs (e.g., LSAS-based study).

• PALISADE-4 enrollment and trial logistics are on track, with operational and analytical adjustments being made based on learnings from prior studies.

• For Refisolone (PH80), IND clearance is targeted by end of H1 2024 to enable further U.S. development.