Vistagen Therapeutics, Inc. · NASDAQ:VTGN
Vistagen Therapeutics — TD Cowen 45th Annual Health Care Conference (March 2025)
34 slides from Vistagen Therapeutics's conference presentation, Mar 6, 2025
Slide 1: Pioneering neuroscience with nose-to-brain neurocircuitry
Nasdaq: VTGN Pioneering neuroscience with nose-to-brain neurocircuitry March 3, 2025 TD Cowen 45th Annual Health Care Conference
Slide 2: Forward-looking Statements
Forward-looking Statements 2 This presentation contains certain forward-looking statements that are within the meaning of federal securities laws. These forward-looking statements involve known and unknown risks that are difficult to predict and include all matters that are not historical facts. In
Slide 3: Highlights
3 Highlights - Funded registration-directed Phase 3 program in Social Anxiety Disorder underway - Multi-billion-dollar peak sales potential across several high prevalence indications - Partnering opportunities in multiple indications and territories - Harnessing the therapeutic power and potential o
Slide 4: Pherines: Harnessing the power and potential of nose-to-brain neurocircuitry
Pherines Harnessing the power and potential of nose-to-brain neurocircuitry
Slide 5: Pherines: A new class of intranasal neuroscience product candidates
Pherines 5 A new class of intranasal neuroscience product candidates - Rapidly activate nose-to-brain neurocircuits affecting multiple high-prevalence indications - Non-systemic MOAs are distinguished from all FDA-approved drugs for target indications - Microgram-level dosing without binding to neur
Slide 6: Clinical-stage Pherine Pipeline
Product Candidate Indication Preclinical Phase I Phase II Phase III Fasedienol Itruvone Acute Treatment of Social Anxiety Disorder Major Depressive Disorder (Monotherapy) • U.S. registration-directed Phase 3 program underway • First positive Phase 3 study reported in 2H 2023 • FDA Fast Track designa
Slide 7: Acute Treatment of Social Anxiety Disorder: Fasedienol
Acute Treatment of Social Anxiety Disorder Fasedienol
Slide 8: Social Anxiety Disorder (SAD)
Social Anxiety Disorder (SAD) 8 Presenting at work or school Eating/drinking in front of others Public speaking Making a phone call Emotional Symptoms • Overwhelming fear • Surges of anxiety • Extreme self-consciousness • Isolation leading to depression Source: ADAA Social Anxiety Brochure 2021 Chro
Slide 9: SAD Affects Over 10% of U.S. Adults
~31M SAD Prevalent Patients ~15M SAD Diagnosed Patients ~7M SAD Treated Patients Treatable Patients Patients suffering but unaware they may have SAD or not yet motivated to seek professional help 9 SAD Affects Over 10% of U.S. Adults Sources: Oracle Life Sciences. May 2024. U.S. National Health and
Slide 10: SAD Prevalence in the U.S. Continues to Grow
10 SAD Prevalence in the U.S. Continues to Grow Source: 1. NCS-R Survey, 2003; Kantar NHWS 2023, Internal Projections 15 31 2003 2005 2007 2009 2011 2013 2015 2017 2019 2021 2023 U.S. Adult SAD Prevalence (in Millions)1
Slide 11: There is no FDA-approved Acute Treatment of SAD
11 There is no FDA-approved Acute Treatment of SAD Off-label acute treatment options fall short of Physicians’ Preferred Product Profile Drug Fast-acting Non-systemic No Long-term Side Effects Non- sedating* No Cognitive Impairment No Withdrawal Syndrome No Abuse Potential Benzodiazepines1 Beta-bloc
Slide 12: Fasedienol Brings New Optimism for SAD Patients
Fasedienol Brings New Optimism for SAD Patients ‐ Rapid-onset efficacy and differentiated safety ‐ Potential to be the first FDA-approved acute treatment of SAD ‐ Patient-tailored administration, as needed, up to several times a day ‐ No observed systemic absorption or binding to neurons in the brai
Slide 13: Fasedienol's Novel Neurocircuitry-focused MOA
Sources: Monti L, and Liebowitz MR (2022). Neural circuits of anxiolytic and antidepressant pherine molecules. CNS Spectrums https://doi.org/10.1017/S109285292000190X Fasedienol’s Novel Neurocircuitry-focused MOA A microgram-level dose of fasedienol is administered intranasally 1 A ctivity D ecrease
Slide 14: PALISADE-2 Phase 3 Trial for Acute Treatment of SAD
14 14 PALISADE-2 Phase 3 Trial for Acute Treatment of SAD : A VISIONARY APPROACH TO MENTAL HEALTH CARE U.S. randomized, double-blind, placebo-controlled, single-dose administration Phase 3 trial to evaluate the efficacy, safety, and tolerability of fasedienol for the acute treatment of SAD induced b
Slide 15: PALISADE-2 Primary Efficacy Endpoint: Change in SUDS Scores
13.8 8.0 0 5 10 15 20 (p=0.015) Absolute Change in Least-squares Mean SUDS (V2-V3) Fasedienol Placebo PALISADE-2 Primary Efficacy Endpoint (Patient-reported): Change in Least-squares Mean SUDS Scores Met primary efficacy endpoint with a change from Baseline of 5.8 points better than placebo 15 N=70
Slide 16: PALISADE-2 Secondary Endpoint: CGI-I Responders vs. Placebo
CGI-I Score 1 = Very Much Less Anxious 2 = Much Less Anxious 3 = A Little Less Anxious 4 = No Change 5 = A Little More Anxious 6 = Much More Anxious 7 = Very Much More Anxious 37.7% 21.4% 0% 5% 10% 15% 20% 25% 30% 35% 40% (p=0.033) CGI-I % Responders* (Rating of 1 or 2) Fasedienol Placebo PALISADE-2
Slide 17: PALISADE-2 Exploratory Endpoint: PGI-C Responders vs. Placebo
PGI-C Score 1 = Very Much Less Anxious 2 = Much Less Anxious 3 = A Little Less Anxious 4 = No Change 5 = A Little More Anxious 6 = Much More Anxious 7 = Very Much More Anxious 40.6% 18.6% 0% 5% 10% 15% 20% 25% 30% 35% 40% 45% (p=0.003) PGI-C % Responders* (Rating of 1 or 2) Fasedienol Placebo PALISA
Slide 18: PALISADE-2 Tolerability Profile
No severe or serious adverse events were reported No discontinuations due to adverse events following the single dose of fasedienol Adverse events were infrequent and mild or moderate in severity There were no treatment-emergent adverse events reported above a 2% occurrence, except pyrexia in the pl
Slide 19: Fasedienol U.S. Registration-directed Phase 3 Program
19 Fasedienol U.S. Registration-directed Phase 3 Program To complement the positive PALISADE-2 Phase 3 trial, Vistagen is conducting two ongoing PALISADE Phase 3 studies as part of its U.S. registration-directed Phase 3 program for the acute treatment of SAD PALISADE-3 and PALISADE-4 Phase 3 Trials
Slide 20: PALISADE-3 and PALISADE-4 Study Enhancements
‐ No mask-wearing during the public speaking challenges ‐ Recurring in-person training of clinical site personnel ‐ Expanded subject eligibility review at screening ‐ Increased surveillance by Vistagen clinical site-facing staff, reduced reliance on CRO ‐ Treatment administration by clinical site he
Slide 21: Major Depressive Disorder: Itruvone
Major Depressive Disorder Itruvone
Slide 22: MDD is a Highly Prevalent and Unsatisfied Market
MDD is a Highly Prevalent and Unsatisfied Market Sources: 1. National Institute of Mental Health, https://www.nimh.nih.gov/health/statistics/major-depression.shtml; 2. World Health Organization, https://www.who.int/news-room/fact-sheets/detail/depression; 3. Rush AJ, et al. Am J. Psychiatry. 2006, 1
Slide 23: Itruvone has Potential to Transform Treatment of MDD
Itruvone has Potential to Transform Treatment of MDD ‐ Non-systemic, neurocircuitry-focused MOA is differentiated from all FDA-approved depression therapies ‐ Positive exploratory Phase 2A trial ‐ Designed for rapid-onset antidepressant effects ‐ Observed to be non-sedating, non-addictive ‐ Well-tol
Slide 24: Rapid-onset antidepressant effects with itruvone observed in MDD
Rapid-onset antidepressant effects with itruvone observed in MDD study participants with minimal side effects Design: Phase 2A randomized, double-blind, placebo-controlled, parallel design exploratory clinical study (n=30) Dosing: 3.2 µg or 6.4 µg of itruvone or placebo i.n., 2 times per day for 8 w
Slide 25: Itruvone Phase 2A Study in MDD
25 Itruvone Phase 2A Study in MDD Sources: Monti, L., Nicolini, H., Liebowitz, M., & Hanover, R. (2019). “A Placebo Controlled Trial of PH10: Test of a New Rap idly Acting Intranasally Administered Antidepressant.” Br J Phar Med Res 4(6): 2157-2168. Hamilton Depression (HAMD-17) Score Reduction From
Slide 26: Itruvone Phase 2B Clinical Plan
26 Itruvone Phase 2B Clinical Plan* Planning and preparation for Phase 2B development of itruvone as a non-systemic monotherapy for MDD is underway ‐ Potential Design: U.S. randomized, double-blind, placebo-controlled, parallel study in male and female subjects (18 to 65 years old) with a confirmed
Slide 27: Vasomotor Symptoms (Hot Flashes) due to Menopause: PH80
Vasomotor Symptoms (Hot Flashes) due to Menopause PH80
Slide 28: VMS (Hot Flashes): Highly Prevalent and Disrupts Daily Life
VMS (Hot Flashes): Highly Prevalent and Disrupts Daily Life 28 • Hallmark symptoms include sudden sensations of heat, night sweats, flushed skin, anxiety, and chills lasting for several minutes • On average, symptoms persist for more than 7 years, however, they may last for over a decade • Frequency
Slide 29: PH80 potential to transform treatment of VMS
PH80 potential to transform treatment of VMS (Hot Flashes) 29 PH80 Nasal Spray ‐ Neurocircuitry-focused MOA differentiated from all approved treatments ‐ Non-hormonal and non-systemic ‐ Rapid-onset potential to be taken as-needed to provide relief in the moment ‐ Potential for differentiated safety
Slide 30: PH80 Phase 2A Study in Menopausal Hot Flashes
PH80 Phase 2A Study in Menopausal Hot Flashes 30 Objective: Proof-of-principle evaluation of PH80 efficacy and tolerability for the management of vasomotor symptoms (hot flashes) due to menopause Study Details: Randomized, double-blind, placebo-controlled, Phase 2A study. Participants self- administ
Slide 31: PH80 Phase 2A Study: Met Primary Efficacy Endpoint
PH80 Phase 2A Study in Hot Flashes: Met Primary Efficacy Endpoint Statistically and clinically significant improvement vs. placebo in the number of hot flashes at 1 week and maintained through 4 weeks of treatment (p<0.001) 0 1 2 3 4 5 6 7 8 9 Baseline Week 1 Week 2 Week 3 Week 4 Follow-up Daily Mea
Slide 32: PH80 Phase 2A Study: Met Secondary Efficacy Endpoint
PH80 Phase 2A Study in Hot Flashes: Met Secondary Efficacy Endpoint H o t Fl as h es - B o th e r H o t Fl as h es - Sw ea t H o t Fl as h es - S ev er it y 0 0.5 1 1.5 2 2.5 Baseline Week 1 Week 2 Week 3 Week 4 Follow Up PH80 Placebo 0 0.5 1 1.5 2 2.5 Baseline Week 1 Week 2 Week 3 Week 4 Follow Up
Slide 33: Distinguished Clinical and Regulatory Advisors
33 Distinguished Clinical and Regulatory Advisors Maurizio Fava, M.D. Professor of Psychiatry, Harvard Medical School; Director, Division of Clinical Research, Massachusetts General Hospital (MGH) Research Institute; and Executive Vice Chair of the Department of Psychiatry Sanjay Mathew, M.D. Vice C
Slide 34: Thank you
Nasdaq: VTGN Thank you
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