Vistagen Therapeutics, Inc. · NASDAQ:VTGN
Vistagen Therapeutics — Investor Presentation (September 2026)
49 slides from Vistagen Therapeutics's investor presentation, September 2026
Slide 1: Corporate Presentation
Nasdaq: VTGN Corporate Presentation Harnessing the therapeutic power and potential of brain regulation with nose-to-brain neurocircuitry September 2026 1
Slide 2: Forward-looking Statements
Forward-looking Statements This presentation contains certain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are within the meaning of federal securities laws. These forward-looking statements involve known and unknown risks that are diffic
Slide 3: Company Highlights
Company Highlights Advancing a Robust and Diverse Neuroscience Pipeline Phase 3 product candidate in social anxiety disorder Four product candidates with Phase 2a PoC Differentiated rapid onset, non-systemic putative MOAs Targeting multiple large markets with inadequate standards of care Experienced
Slide 4: Novel Class of Rapid-Onset, Non-Systemic Neurocircuitry-modulators
Novel Class of Rapid-Onset, Non-Systemic Neurocircuitry- modulators with the Potential to Address Multiple Conditions1 Proposed Mechanism of Action Design Attributes Rapid-onset neuromodulatory Favorable safety and tolerability mechanisms of action targeting reported in all clinical trials to date d
Slide 5: Differentiated Rapid-Onset, Non-Systemic Putative MOAs
Differentiated Rapid-Onset, Non-Systemic Putative MOAs Each pherine is designed to modulate distinct neurocircuits in the brain Shared Aspects of Proposed MOAs 1 Intranasal delivery 5 2 Direct activation of nasal chemosensory neurons (NCNs) Hypothalamus 3 3 NCNs project to olfactory bulb neurons Olf
Slide 6: Lead Clinical-Stage Product Candidates
Lead Clinical-Stage Product Candidates Advancing programs in neuropsychiatry and women’s health Candidate Indication Preclinical Phase 1 Phase 2 Phase 3 Acute Treatment of Fasedienol Social Anxiety Disorder Major Depressive Itruvone Disorder Moderate to Severe VMS Refisolone (hot flashes) due to Men
Slide 7: Lead Product Candidates Target Highly-Prevalent Unmet Need
Lead Product Candidates Target Highly-Prevalent Unmet Need Fasedienol Itruvone Refisolone Social Anxiety Disorder Major Depressive Disorder Menopausal Hot Flashes ~30 million people in the U.S. with ~21 million people in the U.S. ~27 million women in the U.S. social anxiety disorder 1 with major dep
Slide 8: Fasedienol
Fasedienol Acute Treatment of Social Anxiety Disorder 8
Slide 9: Social Anxiety Disorder: A serious and debilitating mental health condition
Social Anxiety Disorder: A serious and debilitating mental health 1 condition characterized by intense fear of being judged and embarrassed Emotional Symptoms Real-Life Impact on Patients2 • Overwhelming fear • Missing out on life moments with family and friends • Surges of anxiety • Extreme self-co
Slide 10: There is No U.S. FDA-Approved Acute Treatment for Social Anxiety Disorder
There is No U.S. FDA-Approved Acute Treatment for Social Anxiety Disorder Available Rx therapies fall short of addressing the acute, in-the-moment needs of SAD patients in anxiety-provoking social and performance situations in everyday life. FDA-Approved SSRIs, SNRIs (Zoloft®, Paxil®, Effexor®) Benz
Slide 11: High Unmet Need for a Novel Acute Treatment for Social Anxiety Disorder
There is a High Unmet Need for a Novel Acute Treatment for Social Anxiety Disorder <50% of social anxiety disorder patients are diagnosed by a healthcare ~30M provider1 adults, or ~12% of the U.S. adult population, suffer from Social Anxiety Disorder1 <23% of social anxiety disorder patients are bei
Slide 12: Fasedienol May Bring New Optimism for Patients with Social Anxiety Disorder
Fasedienol May Bring New Optimism for Patients with Social Anxiety Disorder1,2 Fasedienol Design Traits Rapid-onset effect on both emotional and physical Fasedienol is designed to trigger neural symptoms of peak anxiety, as needed, without sedation or signals in the olfactory bulb, which travel dizz
Slide 13: Physician Adoption: Fasedienol's Target Product Profile
Physician Adoption: Fasedienol’s Target Product Profile (TPP) elicited a high intent-to-prescribe among Health Care Providers (HCPs) Evaluation by Psychiatrists and HCP Reaction2 Primary Care Physicians (PCPs)1 • When given fasedienol’s TPP anonymously % of your social anxiety as “Product X”, prescr
Slide 14: Fasedienol: Clinical Development Program
Fasedienol Clinical Development Program 14
Slide 15: Fasedienol PALISADE Phase 3 Program Clinical Trials
Fasedienol PALISADE Phase 3 Program Clinical Trials 2021-2023 2024-2026 Phase 3 PALISADE-1 Phase 3 PALISADE-3 n=224, 18-65 YO n=238, 18-65 YO Phase 3 PALISADE-2 Phase 3 PALISADE-3 OLE n=140, 18-65 YO n=341, 18-65 YO PALISADE-2 Open Label Long-term Safety Study Phase 3 PALISADE-4 n=238, 18-65 YO n= 4
Slide 16: PALISADE-2 Phase 3 Trial Positive Primary Efficacy
PALISADE-2 Phase 3 Trial Positive Primary Efficacy: Statistically significant relief of acute anxiety in single-dose public speaking challenge trial as measured by the Subjective Units of Distress Scale (SUDS) 20 Absolute Change in Least-squares Mean Primary Efficacy Endpoint SUDS (Visit 2 to 3) • T
Slide 17: PALISADE-2 Positive Secondary & Exploratory Endpoints
PALISADE-2 Positive Secondary & Exploratory Endpoints: Statistically significant and supportive of primary efficacy Secondary & Exploratory Endpoints • On CGI-I proportion of responders vs placebo, rating of 1 (very much less anxious) or rating of 2 (much less anxious) from Baseline Speech #1 to Ran
Slide 18: Fasedienol Across Phase 3 Public Speaking Challenge Trials
Fasedienol Across Phase 3 Public Speaking Challenge Trials Absolute Change in LS Mean SUDS (Visit 2 to Visit 3) 17.3 15.6 13.8 13.6 14.0 11.4 9.5 8.0 PALISADE-1 1* PALISADE-2 2* PALISADE-3 1* PALISADE-4 1* 2* PH94B Placebo PH94B Placebo PH94B Placebo PH94B Placebo (n=111) (n=111) (n=70) (n=71) (n=11
Slide 19: PALISADE-4 Post-Hoc Analysis: Very Severe (LSAS ≥95) Subjects
PALISADE-4 Post-Hoc Analysis: Very Severe (LSAS ≥95) Subjects Statistically Significant LS Mean SUDS Change vs Placebo (P=0.036) Excluding: one site for potentially unreliable data, subjects with V1 LSAS <95, and subjects with ceiling SUDS values 1 All P-Values calculated using ANCOVA model per PALI
Slide 20: Post-Hoc Stratification Analyses of Very Severe SAD Subjects
1* Post-Hoc Stratification Analyses of Very Severe SAD Subjects PALISADE 3/4 Pooled PALISADE-1/3/4 Pooled PALISADE-1/2/3/4 Pooled N=132 N=139 N=192 N=199 N=239 N=241 -9.5 -10.3 -10.1 -14.6 N=7 -16.0 1 -16.6 N=7-7.2 N=7 -5.7 N=7 -4.5 0 0 0 P = 0.013 P = 0.020 P = 0.049 Fasedienol Mean Change PBO Mean
Slide 21: Exploratory Phase 2 Repeat Dose Study Primary Endpoint
Exploratory Phase 2 Repeat Dose Study Primary Endpoint1 Change in Average SUDS from Visit 2 to Visit 3 Primary Endpoint: ITT Analysis (2-sided T-tests) 0 -5 Cohen’s Cohen’s Cohen’s d=0.47 d=0.44 d=0.46 -6.8 -10 -15 -14.7 -15 -15.4 P=0.14 P=0.17 P=0.10 -20 Placebo (n=21) Single Dose (n=21) Double Dos
Slide 22: Exploratory Phase 2 Repeat Dose Study Secondary Endpoints
Exploratory Phase 2 Repeat Dose Study Secondary Endpoints CGI-I and PGI-C % Responders CGI-I Responders PGI-C Responders 50% 47% 50% 42% 40% 40% 40% 33% 33% 29% 30% 30% 19% 19% 20% 20% 10% 10% 0% 0% CGI-I Responder PGI-C Responders Placebo (n=21) Single Dose (n=21) Placebo (n=21) Single Dose (n=21)
Slide 23: Exploratory Phase 2a Repeat Dose Study Minute-by-Minute Anxiety
Exploratory Phase 2a Repeat Dose Study Minute-by-Minute Anxiety SUDS Mean Changes from Visit 2 to Visit 3 Minute by Minute Mean Change from Visit 2 to Visit 3 1 10 0 -10 -20 -30 0 15 16 17 20 21 22 23 24 25 SUDS Timepoint (Minutes) Double Dose (n=19) Single Dose (n=21) Placebo (n=21) A rapid, pronou
Slide 24: PALISADE-1/2 Long Term Safety Study LSAS Results
PALISADE-1/PALISADE-2 Long Term Safety Study Exploratory LSAS Efficacy Results from As-needed Use in Everyday Life Clinically relevant improvements in social anxiety observed over time Mean LSAS Improvement from Baseline2 3rd month 1st month 2nd month Your Title Here Mean LSAS Improvement from Basel
Slide 25: PALISADE-3 Preliminary Open Label Extension LSAS Results
PALISADE-3 Preliminary Open Label Extension Exploratory LSAS Efficacy Results from As-needed Use in Everyday Life Clinically relevant improvements in social anxiety observed over time1 Mean LSAS Improvement from Baseline2 3rd month 4th month 1st month 2nd month Your Title Here Mean LSAS Improvement
Slide 26: PALISADE-4 Preliminary Open Label Extension LSAS Results
PALISADE-4 Preliminary Open Label Extension Exploratory LSAS Efficacy Results from As-needed Use in Everyday Life Clinically relevant improvements in social anxiety observed over time 1 Mean LSAS Improvement from Baseline2 3rd month 4th month 1st month 2nd month Your Title Here Mean LSAS Improvement
Slide 27: Phase 2 Real-Life Crossover Study Results (SUDS & LSAS)
Phase 2 Real-Life Crossover Study Results (SUDS & LSAS) Acute use of fasedienol in anxiety-provoking social and performance situations in everyday life Peak SUDS at Baseline and Weeks 1-4 LSAS at Baseline and Weeks 1-4 70 100 Fasedienol Fasedienol Placebo Placebo 65 90 60 80 SUDS LSAS 55 70 50 60 Ba
Slide 28: PALISADE Open Label Long-term Safety Study (LTSS)
1 PALISADE Open Label Long-term Safety Study (LTSS) Across open label safety studies, TEAEs were generally mild or moderate in severity TEAE by Preferred Term1 N=481, n (%) Safety1 Headache 82 (17.0) 1. Subjects enrolled from PALISADE-1, PALISADE-2, or de novo COVID-19 infection 55 (11.4) (N=481) 2.
Slide 29: Totality of Clinical Evidence Inform Proposed Next Step in Phase 3 Development
Totality of Clinical Evidence Inform Proposed Next Step in Registration-directed U.S. Phase 3 Development Program Positive Phase 3 SUDS PSC (PALISADE-2) Phase 3 Post-hoc Analyses SUDS PSC (PALISADE-4) 1 PALISADE-2 showed statistically significant separation 2 Post-hoc stratification analysis of pati
Slide 30: Fasedienol: Potential Registrational Path Forward
Fasedienol Potential Registrational Path Forward 30
Slide 31: LSAS is Relevant to Measuring Treatment Benefit in Everyday Life
LSAS is Relevant to Measuring Treatment Benefit in Everyday Life LSAS Aligns with How Fasedienol Regulatory & Clinical Precedent is Intended to be Used in Everyday Life ✓ LSAS served as primary efficacy endpoint in • PRN dosing in multiple outpatient anxiety provoking registrational trials for the o
Slide 32: Phase 2 and Open Label Studies Support Use of LSAS as Primary Endpoint
Phase 2 and Open Label Studies Support Use of LSAS as the Primary Efficacy Endpoint for Potential Phase 3 Path Forward Further evaluation of fasedienol for repeated, as-needed use is supported by continued reductions in LSAS scores observed in prior studies from as early as 2 weeks of treatment Stud
Slide 33: Proposed Path Forward: LSAS-Based Phase 3 Study
Proposed Path Forward: LSAS-Based Phase 3 Study 1 Design Population U.S. multicenter, randomized, double-blind, (N=350) parallel-design, placebo-controlled, Adults 18-70 multiple-dose Primary Endpoint Duration Change in LSAS total score from baseline to Double-blind treatment period: six weeks week
Slide 34: Itruvone: Major Depressive Disorder
Itruvone Major Depressive Disorder 34
Slide 35: Major Depressive Disorder Market is Large and Underserved
Major Depressive Disorder Market is Large and Underserved Oral Antidepressants3 U.S. Global (Drug classes: SSRIs, SNRIs, NDRI, 5-HT1A, TCAs, MAOIs) ~21 million ~300 million • Often do not work or are slow to work • STAR*D showed ADT# effective in 1 of 3 patients3 Adults had at least one major depres
Slide 36: Itruvone: A novel, non-systemic product candidate for MDD patients
Itruvone: A novel, non-systemic product candidate with transformative potential for MDD patients Itruvone Design Traits Itruvone Rapid-onset antidepressant effects have been Itruvone is designed to modulate olfactory- observed amygdala circuits and activate local GABAergic inhibitory pathways, which
Slide 37: Itruvone Exploratory Positive Phase 2a Study
Itruvone Exploratory Positive Phase 2a Study Major Depressive Disorder Phase2a Study Results • 6.4 µg dose significantly reduced depressive symptoms as early as Week 1 and sustained through Week 8 compared to placebo (p=0.022) • Rapid onset numerical improvements observed for 3.2 µg and 6.4 µg vs pl
Slide 38: Itruvone 6-Week Double-blind Phase 2b Study Plan
Itruvone 6-Week Double-blind Phase 2b Study Plan ➢Key Inclusions ➢Key Exclusions • No history of other significant psychiatric disorders • Male or female, 18-65 years of age • No other psychotropic drugs w/in 14 days of • Major Depressive Disorder, as defined by DSM-5 screening • HAMD-17 > 21 at scr
Slide 39: Refisolone: Vasomotor Symptoms (Hot Flashes) due to Menopause
Refisolone Vasomotor Symptoms (Hot Flashes) due to Menopause 39
Slide 40: A Significant Portion of Menopausal Women Experience Vasomotor Symptoms
A Significant Portion of Menopausal Women Experience Vasomotor Symptoms (Hot Flashes) Approximately 75% of all women in the US experience hot flashes during Hot flashes are the most common the menopausal transition1,2,3 symptoms of menopause for which women seek treatment5 ~27M ~9M Symptoms persist
Slide 41: Refisolone: A novel, on-demand, non-hormonal, non-systemic product candidate
Refisolone: A novel, on-demand, non-hormonal, non-systemic product candidate designed for the treatment of Menopausal Hot Flashes Refisolone Refisolone Design Traits Designed as a potential on-demand, Novel and differentiated proposed MOA from all currently FDA- fast-acting, non-hormonal, non- appro
Slide 42: Refisolone Positive Exploratory Phase 2a Study
Refisolone Positive Exploratory Phase 2a Study Menopausal Hot Flashes Frequency of Hot Flashes1 Severity of Hot Flashes1 Phase 2a Study Results1 • Refisolone was administered at 3.2 µg up to 5 times daily. • Significantly reduced the frequency of hot flashes after Week 1 of treatment, with sustained
Slide 43: Refisolone Positive Phase 2a Study: PMDD
Refisolone Positive Phase 2a Study Premenstrual Dysphoric Disorder (PMDD) Phase 2a Study Results • Refisolone was administered at 0.9 µg up to 4 times daily • Refisolone showed statistically and clinically significant improvement vs placebo in symptoms of PMDD at study endpoint after 6 days of treat
Slide 44: PH15: Psychomotor / Cognitive Impairment due to Mental Fatigue
PH15 Psychomotor / Cognitive Impairment due to Mental Fatigue 44
Slide 45: PH15: A novel, fast-acting, non-systemic clinical-stage product candidate
PH15: A novel, fast-acting, non-systemic clinical-stage product candidate designed for the acute treatment of cognitive/psychomotor impairment due to mental fatigue PH15 PH15 Design Traits PH15 is designed to modulate Novel neurocircuitry-focused proposed MOA differentiated from all hippocampal acti
Slide 46: PH284: Cancer Cachexia (Cancer Supportive Care)
PH284 Cancer Cachexia (Cancer Supportive Care) 46
Slide 47: PH284: An innovative non-systemic clinical stage product candidate
PH284: An innovative non-systemic clinical stage product candidate designed for the acute treatment of cancer cachexia PH284 PH284 Design Traits Potential to non-systemically Novel neurocircuitry-focused proposed MOA differentiated from modulate hypothalamic all approved treatments pathways that reg
Slide 48: Advancing a Differentiated Neuroscience Pipeline
Advancing a Differentiated Neuroscience Pipeline • Differentiated intranasal mechanism Harnessing the power and • Phase 3 opportunity in social anxiety disorder therapeutic potential of brain regulation. • Multiple Phase 2 neuroscience assets with established clinical proof-of-concept • Near-term re
Slide 49: Contact us
Contact us: Investors: IR@vistagen.com Media: media@vistagen.com Business Development: busdev@vistagen.com 49
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