Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (December 2020)
57 slides from Tonix Pharmaceuticals's investor presentation, Dec 14, 2020
Slide 1: Investor Presentation
© 2020 Tonix Pharmaceuticals Holding Corp. 1 December 2020 Version P0264 12 - 14 - 2020 (Doc 0747) Investor Presentation NASDAQ:TNXP
Slide 2: Cautionary Note on Forward-Looking Statements
© 2020 Tonix Pharmaceuticals Holding Corp. 2 Cautionary Note on Forward - Looking Statements Certain statements in this presentation regarding strategic plans, expectations and objectives for future operations or results are “forward - looking statements” as defined by the Private Securities Litigat
Slide 3: Tonix Pharmaceuticals
© 2020 Tonix Pharmaceuticals Holding Corp. 3 Tonix Pharmaceuticals © 2020 Tonix Pharmaceuticals Holding Corp . • Clinical - stage biopharmaceutical company • Committed to discovering and developing innovative and proprietary new therapeutics • Focus on developing biologics and small molecules • Cent
Slide 4: Our Pipeline – CNS Portfolio
© 2020 Tonix Pharmaceuticals Holding Corp. 4 Our Pipeline – CNS Portfolio CANDIDATES INDICATION STATUS CNS Portfolio TNX - 102 SL 1 Fibromyalgia (FM) - Lead Program Mid - Phase 3 – ongoing PTSD Phase 3 – ongoing Agitation in Alzheimer’s Phase 2 ready Alcohol Use Disorder Phase 2 ready TNX - 1300 2 C
Slide 5: Our Pipeline – Immunology & Biodefense Portfolio
© 2020 Tonix Pharmaceuticals Holding Corp. 5 Our Pipeline – Immunology & Biodefense Portfolio CANDIDATES INDICATION STATUS Immunology Portfolio TNX - 1800 Covid - 19 vaccine – Prioritized Program 1 Preclinical TNX - 1810, TNX - 1820, TNX - 1830 Covid - 19 vaccine 1 Preclinical TNX - 2300 Covid - 19
Slide 6: Overview of TNX-102 SL
© 2020 Tonix Pharmaceuticals Holding Corp. 6 Overview of TNX - 102 SL* Protectic ® proprietary formulation of cyclobenzaprine that supports sublingual administration TNX - 102 SL is a non - opioid, centrally - acting analgesic that works by improving sleep quality Scientific Rationale for Protectic
Slide 7: TNX-102 SL: Differentiation from Oral Formulations
© 2020 Tonix Pharmaceuticals Holding Corp. 7 TNX - 102 SL: Differentiation from Oral Formulations FEATURE BENEFIT ADVANTAGE Cyclobenzaprine 40+ years as oral medication Established safety record Formulation: Protectic ® Allows submucosal absorption Not achievable with oral formulation Administration
Slide 8: Fibromyalgia: Disease Overview and Unmet Need
© 2020 Tonix Pharmaceuticals Holding Corp. 8 Volkswagen Check Engine [Photograph]. (2011, October 14). Wikipedia When the check engine light malfunctions, the light is on even though the car is not malfunctioning • Fibromyalgia is considered a central nervous system disorder with symptoms that inclu
Slide 9: TNX-102 SL: Results from Completed FM Trials
© 2020 Tonix Pharmaceuticals Holding Corp. 9 TNX - 102 SL: Results from Completed FM Trials Completed Trials in FM: • Phase 2 (F202 BESTFIT) – 205 participants randomized • Phase 3 (F301 AFFIRM) – 519 participants randomized • Phase 3 (F304 RELIEF) – 503 participants randomized Topline Efficacy Resu
Slide 10: Phase 3 F304/RELIEF Study: Design
© 2020 Tonix Pharmaceuticals Holding Corp. 10 Phase 3 F304/RELIEF Study: Design Primary e ndpoint (Week 14) : • Daily diary pain severity score change (TNX - 102 SL 5.6 mg vs. placebo) from baseline in the weekly average as measured by the numerical rating scale (NRS), using mixed model repeated mea
Slide 11: F304/RELIEF Topline Data: Primary Endpoint Achieved
© 2020 Tonix Pharmaceuticals Holding Corp. 11 F304/RELIEF Study Topline Data: Statistical Significance Achieved on Pre - specified Primary Efficacy Endpoint (p=0.01) ◊ ◊ 2 TNX - 102 SL is in clinical stage of development and not approved for any indication Primary Outcome Measure at Week 14 Placebo
Slide 12: F304/RELIEF Primary Efficacy Endpoint Results (continued)
© 2020 Tonix Pharmaceuticals Holding Corp. 12 F304/RELIEF Study: Primary Efficacy Endpoint Results (continued) -2.2 -2 -1.8 -1.6 -1.4 -1.2 -1 -0.8 -0.6 -0.4 -0.2 0 0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 LS Mean Change from Baseline (SE) in NRS Pain Score Study Week RELIEF Study Mean Change from Baseline
Slide 13: F304/RELIEF Key Secondary Efficacy Endpoints
© 2020 Tonix Pharmaceuticals Holding Corp. 13 F304/RELIEF Study: Key Secondary Efficacy Endpoints ◊ ◊ *TNX - 102 SL is in clinical stage of development and not approved for any indication Outcome Measure at Week 14 Intent - to - Treat Analysis 1 P - value Non - Specific Patient Global Impression of
Slide 14: F304/RELIEF Continuous Responder Analysis Graph
© 2020 Tonix Pharmaceuticals Holding Corp. 14 P e r c e n t o f S u b j e c t s 0 10 20 30 40 50 60 70 80 90 100 Percentage Redution in Pain >=0% >=10% >=20% >=30% >=40% >=50% >=60% >=70% >=80% >=90% =100% Placebo TNX-102 SL Proportion of Subjects in Arm Cut - Off Points for Percentage Pain Responde
Slide 15: Adverse Events in F304/RELIEF Study
© 2020 Tonix Pharmaceuticals Holding Corp. 15 Adverse Events* (AEs) in F304/ RELIEF Study TNX - 102 SL (N=248) Placebo (N=255) Total (N=503) Administration Site Reactions N % N % N % Tongue/mouth numbness 43 17.3 2 0.8 45 8.9 Tongue/mouth pain/discomfort 29 11.7 5 2.0 34 6.8 Taste impairment 16 6.5
Slide 16: Safety and Tolerability in F304/RELIEF Study
© 2020 Tonix Pharmaceuticals Holding Corp. 16 Safety and Tolerability in F304/RELIEF Study • No new safety signals in RELIEF at TNX - 102 SL 5.6 mg dose • 82.3% in active arm and 83.5% in placebo arm completed the study • 8.9% in active arm and 3.9% in placebo arm discontinued due to adverse events
Slide 17: Second Phase 3 F306/RALLY Study – Enrollment Ongoing
© 2020 Tonix Pharmaceuticals Holding Corp. 17 TNX - 102 SL 5.6 mg for Fibromyalgia: 2 nd Phase 3 F306/RALLY Study – Enrollment Ongoing Primary e ndpoint (Week 14) : • Daily diary pain severity score change (TNX - 102 SL 5.6 mg vs. placebo) from baseline in the weekly average as measured by the numer
Slide 18: Approved Fibromyalgia Pharmacotherapies
© 2020 Tonix Pharmaceuticals Holding Corp. 18 Approved Fibromyalgia Pharmacotherapies Pfizer • Drug: Lyrica® or pregabalin (U.S. patent expired in 2018) • Approved: 2004 • Mechanism: modulates nerve impulses involved in the transmission of pain through selective binding to the alpha2 - delta protein
Slide 19: Other Fibromyalgia Pharmacotherapies in Development in the U.S.
© 2020 Tonix Pharmaceuticals Holding Corp. 19 Other Fibromyalgia Pharmacotherapies in Development in the U.S. Axsome Therapeutics - AXS - 14 • Drug: esreboxetine • Mechanism: Selective norepinephrine reuptake inhibitor • Developmental Stage: At least mid - Phase 3 (Phase 2 and Phase 3 trial positive
Slide 20: TNX-102 SL for PTSD: Three Recent Trials
© 2020 Tonix Pharmaceuticals Holding Corp. 20 TNX - 102 SL for Posttraumatic Stress Disorder (PTSD): Three Recent Trials Phase 2 P201 “ AtEase ” – Military - related PTSD 1 • Reported May 2016 ( mITT , N=231) • 3 groups: Placebo (n= 92), TNX 2.8 mg (n= 90) and TNX 5.6 mg (n=49) • Primary endpoint (2
Slide 21: Effect of Dose on Adverse Events in PTSD Studies
© 2020 Tonix Pharmaceuticals Holding Corp. 21 Effect of Dose on Adverse Events (AEs) at 5.6 mg in PTSD Studies # Only adverse events (AEs) are listed that are at a rate of ≥ 5% in any TNX - treated group *No values in a row for either study means the AE in the active group(s) in that study was at a
Slide 22: Common Themes from Three Recent PTSD Trials
© 2020 Tonix Pharmaceuticals Holding Corp. 22 Common Themes from Three Recent PTSD Trials Testing TNX - 102 SL • Consistent nominal improvement on sleep item on CAPS - 5 • E6 sleep disturbance item • Supported by nominal benefits in PROMIS Sleep Disturbance • Evidence of “target engagement”, appropr
Slide 23: Multi-dimensional Analyses
© 2020 Tonix Pharmaceuticals Holding Corp. 23 Multi - dimensional Analyses “21 st Century Cures Act” addressed use of novel trial designs, simulations and analyses • Requiring guidance and encouraging increased reliance on novel and adaptive clinical trial designs, including use of modeling and simu
Slide 24: Proposed Analysis: RHNPCOT
© 2020 Tonix Pharmaceuticals Holding Corp. 24 Proposed Analysis: Randomization Honoring Non - Parametric Combination of Tests (RHNPCOT) 1 Honors the actual randomization method of the study • Uses modern analyses and simulations which are advocated by 21 st Century Cures Act Preserves information fr
Slide 25: Future Plans: TNX-102 SL for PTSD
© 2020 Tonix Pharmaceuticals Holding Corp. 25 Future Plans: TNX - 102 SL for PTSD Pharmacogenomics on study participants • P302 had high percentage of participant DNA collected • P301 has a subset of participant DNA available • Exome sequencing to focus on: • Drug metabolizing enzymes • Neurotransmi
Slide 26: TNX-102 SL Intellectual Property – U.S. Protection expected until 2035
© 2020 Tonix Pharmaceuticals Holding Corp. 26 TNX - 102 SL Intellectual Property – U.S. Protection expected until 2035 • United States Patent and Trademark Office (USPTO) issued United States Patent No. 9636408 in May 2017, Patent No. 9956188 in May 2018, Patent No. 10117936 in November 2018, Patent
Slide 27: Opportunities to Expand TNX-102 SL to Other Indications
© 2020 Tonix Pharmaceuticals Holding Corp. 27 Opportunities to Expand TNX - 102 SL to Other Indications Growing recognition that there are many disorders where sleep disturbances may have a role in the pathophysiology (cardiovascular, metabolic, neurologic) • Sleep quality plays a homeostatic role i
Slide 28: TNX-1800: a COVID-19 Vaccine Candidate
© 2020 Tonix Pharmaceuticals Holding Corp. 28 TNX - 1800 1 : a COVID - 19 Vaccine Candidate • Utilizes Tonix’s proprietary horsepox virus as a vector • Encodes a protein from SARS - CoV - 2, the cause of COVID - 19 • Developed in collaboration with University of Alberta, Canada • Animal testing with
Slide 29: COVID-19 Vaccine Landscape
© 2020 Tonix Pharmaceuticals Holding Corp. 29 COVID - 19 Vaccine Landscape • We expect more than one vaccine will be approved by FDA • Different vaccines for different individuals • More than 150 vaccines in development • Diversity of approaches is important since protective immunity is not yet unde
Slide 30: Live, Attenuated Virus Vaccines for Other Infectious Diseases
© 2020 Tonix Pharmaceuticals Holding Corp. 30 Live, Attenuated Virus Vaccines for Other Infectious Diseases 1 • Long term, durable immunity • Expected to stimulate T cells and provide years to decades of protection • Single administration, scalable manufacturing • Low dose is amplified by replicatio
Slide 31: TNX-1800 Vaccination of Non-Human Primates Elicited Neutralizing Antibodies
© 2020 Tonix Pharmaceuticals Holding Corp. 31 TNX - 1800 Vaccination of Non - Human Primates Elicited Anti - SARS - CoV - 2 Neutralizing Antibodies and Skin Reaction or “Take” in All Eight Animals STUDY DESIGN : Compares TNX - 1800 (modified horsepox virus encoding CoV - 2 spike protein) to TNX -
Slide 32: TNX-1800 Non-Human Primates: Findings, Conclusions and Next Phase
© 2020 Tonix Pharmaceuticals Holding Corp. 32 TNX - 1800 Vaccination of Non - Human Primates Findings, Conclusions and Next Phase TOLERABILITY : TNX - 1800 and TNX - 801 were well tolerated at both doses. DOSE: Supports the expectation that TNX - 1800 at the low dose of 1 x 10 6 PFU is an approp
Slide 33: TNX-1800: Engineered for Long-term Immunity
© 2020 Tonix Pharmaceuticals Holding Corp. 33 TNX - 1800 1 : Engineered for Long - term Immunity • Based on “vaccinia” vaccine developed more than 200 years ago by Dr. Edward Jenner to prevent smallpox • TNX - 1800 has 99.7% colinear identity with circa 1860 smallpox vaccine 2 • Eradicated smallpox
Slide 34: Why Use a Horsepox Platform for a Vaccine?
© 2020 Tonix Pharmaceuticals Holding Corp. 34 Why Use a Horsepox Platform for a Vaccine? Horsepox can be engineered to express foreign genes • Lack of persistence or genomic integration in the host • Strong immunogenicity as a vaccine • Readily manufacture at scale • Live, attenuated vaccine – direc
Slide 35: TNX-1800 is Based on a Horsepox Virus Vector
© 2020 Tonix Pharmaceuticals Holding Corp. 35 TNX - 1800 is Based on a Horsepox Virus (HPXV) Vector Designed to Express SARS - CoV - 2 S Protein *TNX - 1800 is at the pre - IND stage of development Horsepox sHPXV ~200,000 Bp TNX - 1800 * rHPXV/SARS - CoV - 2S ~200,000 Bp Homologous Recombination
Slide 36: Vaccinia Induces a Skin Reaction Called “Take”
© 2020 Tonix Pharmaceuticals Holding Corp. 36 Vaccinia Induces a Skin Reaction Called “Take” – Described by Dr. Edward Jenner 1. Fulginiti VA, et al. Clin Infect Dis. 2003;37(2):241 - 250. 2. Liu L, et al. Nature Med. 2010;16(2):224 - 228. 3. Centers for Disease Control and Prevention. Accessed Apri
Slide 37: Unique Challenges of SARS-CoV-2
© 2020 Tonix Pharmaceuticals Holding Corp. 37 Unique Challenges of SARS - CoV - 2 SARS Rate of death 1,2 Deaths 2 Rate of infectivity 1 Incubation time 2 Asymptomatic 3 0.4 2 - 7 days ~13% ~10% SARS - CoV - 2 Rate of death 1,4 Deaths (as of Nov. 2020) 5 Rate of infectivity 1,4 Incubation time 2,4 As
Slide 38: Infection of Type II Pneumocytes Can Lead to Lethal Respiratory Illness
© 2020 Tonix Pharmaceuticals Holding Corp. 38 Infection of Type II Pneumocytes Can Lead to Lethal Respiratory Illness Capillary Type II pneumocytes Inhalation Exhalation Type II pneumocytes in the alveoli secrete pulmonary surfactants that are necessary for effective gas exchange. 2 SARS - CoV - 2 I
Slide 39: SARS-CoV-2 Hijacks the Respiratory System to Spread Contagious Virus
© 2020 Tonix Pharmaceuticals Holding Corp. 39 SARS - CoV - 2 Hijacks the Respiratory System to Spread Contagious Virus Respiratory epithelium Capillary Airway Resting CD8 T cell Endothelial cell Exhalation spreads virus • Virus factories release virions by continuous budding • Breathing, speaking or
Slide 40: CoV-Specific T Cells Kill the Virus Factories
© 2020 Tonix Pharmaceuticals Holding Corp. 40 CoV - 2 Specific T Cells Kill the Virus Factories • Natural immunity or vaccine protection has the potential to decrease forward transmission • T cells specifically kill virally infected cells Bar - On YM, et al. eLife . 2020;9:e57309 . Respiratory epith
Slide 41: Contrasting T Cell and Antibody Immunity
© 2020 Tonix Pharmaceuticals Holding Corp. 41 Contrasting T cell and Antibody Immunity • T cell immunity • Durable or long - lived (many years) • Recognize fragments of pathogens on the surfaces of infected cells • Cannot recognize pathogens directly • Potential to clear viral infections (by killing
Slide 42: TNX-1800 Upcoming Milestones
© 2020 Tonix Pharmaceuticals Holding Corp. 42 TNX - 1800 Upcoming Milestones Southern Research studies will address two key questions: • Will vaccination of animals elicit an immune response to the S protein? • 4 th Quarter 2020 - Non - human primate immune response positive results reported • Will
Slide 43: Second SARS-CoV-2 Vaccine Platform: Bovine Parainfluenza Virus
© 2020 Tonix Pharmaceuticals Holding Corp. 43 2 nd SARS - CoV - 2 Vaccine Platform: Bovine Parainfluenza (BPI) Virus Collaboration with Kansas State University to develop a vaccine candidate for the prevention of COVID - 19 • Utilizes a novel live attenuated vaccine vector platform and the CD40 - li
Slide 44: TNX-1300 for the Treatment of Cocaine Intoxication
© 2020 Tonix Pharmaceuticals Holding Corp. 44 TNX - 1300* for the Treatment of Cocaine Intoxication Recombinant protein that degrades cocaine in the bloodstream 1 • Double - mutant cocaine esterase ( CocE ) • CocE was identified in a bacterium ( Rhodococcus ) that use cocaine as its sole source of c
Slide 45: TNX-1300 Is a Fast-acting Cocaine Antidote
© 2020 Tonix Pharmaceuticals Holding Corp. 45 TNX - 1300 (Cocaine Esterase or CocE ) Is a Fast - acting Cocaine Antidote CocE Rhodococcus bacteria living in the roots of the coca plant use CocE to metabolize cocaine 1 CocE cleaves chemical bonds in cocaine and disintegrates it 800 times faster than
Slide 46: TNX-1300 Development Plan
© 2020 Tonix Pharmaceuticals Holding Corp. 46 TNX - 1300 Development Plan 46 • Targeting to initiate a Phase 2 open - label, randomized pilot study of TNX - 1300 in the first quarter of 2021 • Emergency department (ED) setting with patients coming in for treatment of cocaine and/or polysubstance int
Slide 47: TNX-1900 for Migraine and Craniofacial Pain – Overview
© 2020 Tonix Pharmaceuticals Holding Corp. 47 TNX - 1900 for the Treatment of Migraine and Craniofacial Pain – Overview Novel intranasal oxytocin formulation being developed as a prophylactic treatment for chronic migraine • Based on a propriety formulation of oxytocin*, a naturally occurring human
Slide 48: TNX-1900 for the Treatment of Migraine – Prevalence
© 2020 Tonix Pharmaceuticals Holding Corp. 48 TNX - 1900 for the Treatment of Migraine – Prevalence One billion individuals worldwide suffer from migraines (~14% of population) 1 Migraine is the second leading cause of years lived with disability 1 In U.S., the estimated cost of all migraine headach
Slide 49: TNX-1900 for Migraine – Mechanism of Action
© 2020 Tonix Pharmaceuticals Holding Corp. 49 TNX - 1900 for the Treatment of Migraine – Mechanism of Action Preclinical research showed that nasally applied TNX - 1900 selectively inhibits the activity of trigeminal pain - sensing nerve cells and blocks the release of CGRP • TNX - 1900 is believed
Slide 50: TNX-1900 Mechanism of Action (continued)
TNX - 1900 for the Treatment of Migraine – Mechanism of Action (continued) HEAD PAIN PATIENT USES TNX - 1900 TARGETED DELIVERY Permeates nasal mucosa Transported to trigeminal system and brain Proprietary Nasal to Brain Delivery CGRP: NEUROTRANSMITTER THAT HAS BEEN VALIDATED AS KEY MIGRAINE TARGET T
Slide 51: TNX-1900: Mechanism of Action (continued)
TNX - 1900: Mechanism of Action (continued) In animal models, intranasal oxytocin concentrates in the trigeminal system I nhibits trigeminal neuronal firing, and decreases CGRP (and PACP) release onto meningeal vasculature and within the brainstem - Believed to have effects on: - Neurogenic inflamma
Slide 52: TNX-1900 for Migraine – Development Status
© 2020 Tonix Pharmaceuticals Holding Corp. 52 TNX - 1900 for the Treatment of Migraine – Development Status In June 2020, Tonix acquired a proprietary formulation of nasal oxytocin solution for intranasal delivery from Trigemina Also acquired migraine and pain treatment technologies of Trigemina , I
Slide 53: Pipeline Summary – by Select Therapeutic Areas
© 2020 Tonix Pharmaceuticals Holding Corp. 53 Pipeline 1 Summary – by Select Therapeutic Areas Oncology • TNX - 1700 (rTFF2 2 ) for treatment of gastric and pancreatic cancer Preclinical Public Health • TNX - 1800, TNX - 1810, TNX - 1820 & TNX - 1830 (live modified horsepox vaccine) for preventing C
Slide 54: Pipeline Summary – by Select Therapeutic Areas (continued)
© 2020 Tonix Pharmaceuticals Holding Corp. 54 Pipeline 1 Summary – by Select Therapeutic Areas (continued) Pain Psychiatry Addiction Medicine • TNX - 102 SL (sublingual cyclobenzaprine) for fibromyalgia Phase 3/RELIEF Phase 3/RALLY • TNX - 102 SL (sublingual cyclobenzaprine) for PTSD Phase 3/RECOVER
Slide 55: Milestones – Recently Completed and Upcoming
© 2020 Tonix Pharmaceuticals Holding Corp. 55 Milestones – Recently Completed and Upcoming 1 □ September 2020 Interim analysis of TNX - 102 SL Phase 3 F304/RELIEF study in fibromyalgia completed □ 4 th Quarter 2020 Non - human primate immune response positive results reported □ 4 th Quarter 2020 Pos
Slide 56: Management Team
© 2020 Tonix Pharmaceuticals Holding Corp. 56 Management Team Seth Lederman, MD President & CEO Jessica Morris Chief Operating Officer Gregory Sullivan, MD Chief Medical Officer Bradley Saenger , CPA Chief Financial Officer
Slide 57: Thank You!
© 2020 Tonix Pharmaceuticals Holding Corp. 57 Thank You!
Tonix Pharmaceuticals Holding — Investor Presentation (December 2020) on 6ix