Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP

Tonix Pharmaceuticals — Investor Presentation (August 2018)

43 slides from Tonix Pharmaceuticals's investor presentation, Aug 13, 2018

  1. Slide 1: Investor Presentation

    © 2018 Tonix Pharmaceuticals Holding Corp. August 2018 Version P0122 8 - 7 - 18 (Doc 0376) Investor Presentation

  2. Slide 2: Cautionary Note on Forward-Looking Statements

    © 2018 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

  3. Slide 3: Tonix Pharmaceuticals

    © 2018 Tonix Pharmaceuticals Holding Corp. 3 Tonix Pharmaceuticals Who we are: • A clinical stage pharmaceutical company dedicated to developing innovative treatments for patients and making meaningful contributions to society What we do: • Target therapeutics with high need for improvement − Condit

  4. Slide 4: Candidates in Development

    © 2018 Tonix Pharmaceuticals Holding Corp. 4 Candidates in Development Preclinical Phase 2 NDA 1 /BLA 2 Market Pipeline Product Indication/ Description Phase 3 Tonmya Bedtime Treatment for PTSD Daytime Treatment for PTSD TNX - 601 Novel polymorph and salt discovered and characterized TNX - 801 Horse

  5. Slide 5: Lead Program: TNX-102 SL – Product Concept

    © 2018 Tonix Pharmaceuticals Holding Corp. 5 Lead Program: TNX - 102 SL – Product Concept Sleep disturbances are associated with a constellation of disorders • Considered co - morbid or a key symptom in these disorders • Believed to have a role in the onset, progression and severity of these disorde

  6. Slide 6: Lead Program Tonmya – FDA Breakthrough Therapy in PTSD

    © 2018 Tonix Pharmaceuticals Holding Corp. 6 Lead Program Tonmya ®1 – FDA Breakthrough Therapy in posttraumatic stress disorder (PTSD) 2 – Bedtime treatment in Phase 3 Development • Results from 2 efficacy studies improve the new pivotal efficacy study design • FDA feedback and agreement are expecte

  7. Slide 7: Tonmya for PTSD

    © 2018 Tonix Pharmaceuticals Holding Corp. 7 Tonmya for PTSD Breakthrough Therapy (BT) designation from the FDA • Expedited development and accelerated approval are expected One Phase 2 study completed and one Phase 3 study stopped early due to inadequate separation from placebo • Both studies were

  8. Slide 8: Breakthrough Therapy Designation

    © 2018 Tonix Pharmaceuticals Holding Corp. 8 Breakthrough Therapy Designation FDA granted Tonmya Breakthrough Therapy designation – reported December 19, 2016 • PTSD is a serious condition • Tonmya has potential advantages over existing therapies in military - related PTSD Benefits of Breakthrough T

  9. Slide 9: Tonmya: Features in PTSD Therapy

    © 2018 Tonix Pharmaceuticals Holding Corp. 9 Tonmya: Features in PTSD Therapy Designed for bedtime use • Every night, sublingual therapy Targets sleep quality 1 • The active ingredient cyclobenzaprine, interacts with receptors that regulate sleep quality: strongly binds and potently blocks 5 - HT 2A

  10. Slide 10: No Recognized Abuse Potential in Clinical Studies

    © 2018 Tonix Pharmaceuticals Holding Corp. 10 No Recognized Abuse Potential in Clinical Studies Active ingredient is cyclobenzaprine, which is structurally related to tricyclic antidepressants • Cyclobenzaprine interacts with receptors that regulate sleep quality: 5 - HT 2A ; a 1 - adrenergic and hi

  11. Slide 11: TNX-102 SL Intellectual Property – U.S. Protection until 2034

    © 2018 Tonix Pharmaceuticals Holding Corp. 11 TNX - 102 SL Intellectual Property – U.S. Protection until 2034 Composition of matter (eutectic) : Protection expected to 2034 • United States Patent and Trademark Office (USPTO) issued U.S. Patent No. 9,636,408 in May 2017 and U.S. Patent No. 9,956,188

  12. Slide 12: TNX-102 SL: Sublingual Formulation Designed for Bedtime Administration

    © 2018 Tonix Pharmaceuticals Holding Corp. 12 TNX - 102 SL: Sublingual Formulation is Designed for Bedtime Administration TNX - 102 SL: Proprietary sublingual formulation of cyclobenzaprine (CBP) with transmucosal absorption • Innovation by design with patent protected CBP/mannitol eutectic • Rapid

  13. Slide 13: Tonmya: Novel Mechanism Targets Sleep Quality for Recovery from PTSD

    © 2018 Tonix Pharmaceuticals Holding Corp. 13 Tonmya: Novel Mechanism Targets Sleep Quality for Recovery from PTSD PTSD is a disorder of recovery • Most people exposed to extreme trauma recover over a few weeks • In PTSD, recovery process impeded due to insufficient sleep - dependent memory processi

  14. Slide 14: Overview of PTSD

    © 2018 Tonix Pharmaceuticals Holding Corp. 14 Overview of PTSD PSTD is a chronic disabling disorder in response to experiencing traumatic event(s) Symptoms of PTSD fall into four clusters: 1. Intrusion (aversive memories, nightmares, flashbacks) 2. Avoidance (avoiding persons, places or situations)

  15. Slide 15: Impact of PTSD on People

    © 2018 Tonix Pharmaceuticals Holding Corp. 15 Impact of PTSD on People Consequences: • Impaired daily function and substantial interference with work and social interactions • Reckless or destructive behavior • Increased health care utilization and greater medical morbidity PTSD as a risk factor for

  16. Slide 16: PTSD: Not Well-Served by Approved Treatments

    © 2018 Tonix Pharmaceuticals Holding Corp. 16 PTSD: Not Well - Served by Approved Treatments FDA - approved SSRIs, paroxetine and sertraline, are indicated as a treatment for PTSD • Neither drug has shown efficacy in military - related PTSD • Majority of patients unresponsive or intolerant to curren

  17. Slide 17: High Prevalence of PTSD Among Combat Veterans

    © 2018 Tonix Pharmaceuticals Holding Corp. 17 High Prevalence of PTSD Among Combat Veterans 1 Goldstein et al., 2016 ; 2 Norris, PTSD Res Quar . 2013; 3 Analysis of VA Health Care Utilization among Operation Enduring Freedom, Operation Iraqi Freedom, and Operation New Dawn Veterans, from 1st Qtr FY

  18. Slide 18: HONOR Study – Evidence of Efficacy at Week 4

    © 2018 Tonix Pharmaceuticals Holding Corp. 18 HONOR Study – Evidence of Efficacy at Week 4 Discontinued Due to High Placebo Response at Week 12 Primary e ndpoint CAPS - 5 1 : • Mean change from baseline at w eek 12 (Tonmya 5.6 mg vs. placebo ) Unblinded interim analysis (IA) at ~50% randomized parti

  19. Slide 19: HONOR Study Stopped in July 2018

    © 2018 Tonix Pharmaceuticals Holding Corp. 19 HONOR Study Stopped in July 2018 HONOR was a large adequate well - controlled study in military - related PTSD • Separation on primary endpoint at Week 12 did not cross pre - specified study continuation threshold at Week 12 • No safety or tolerability i

  20. Slide 20: Phase 2 AtEase Study in Military-Related PTSD

    © 2018 Tonix Pharmaceuticals Holding Corp. 20 Phase 2 AtEase Study in Military - Related PTSD • Randomized, double - blind, placebo - controlled trial in military - related PTSD • Efficacy analysis from 231* patients; 24 U.S. clinical sites • Enrolled patients with baseline CAPS - 5 ≥ 29 • Primary E

  21. Slide 21: AtEase Study: Traumas Associated with PTSD

    © 2018 Tonix Pharmaceuticals Holding Corp. 21 AtEase Study: Traumas Associated with PTSD *Some patients experienced more than one trauma 0 5 10 15 20 25 30 35 40 Other Knowing someone seriously injured or killed Being responsible for the death of an enemy combatant Seeing dead bodies or human remain

  22. Slide 22: AtEase Study – Summary of Primary and Secondary Analyses (Week 12)

    © 2018 Tonix Pharmaceuticals Holding Corp. 22 AtEase Study – Summary of Primary and Secondary Analyses (Week 12)

  23. Slide 23: AtEase Study: Safety and Tolerability Profile

    © 2018 Tonix Pharmaceuticals Holding Corp. 23 AtEase Study: Safety and Tolerability Profile No serious adverse events reported with Tonmya deemed related to treatment Systemic Adverse Events* Placebo (N=94) Tonmya 2.8 mg (N=93) Tonmya 5.6 mg (N=50) Somnolence 6.4% 11.8% 16.0% Dry Mouth 10.6% 4.3% 16

  24. Slide 24: AtEase Study Total CAPS-5 for ITT Population and Subgroup

    © 2018 Tonix Pharmaceuticals Holding Corp. 24 AtEase Study Total CAPS - 5 for Intention - to - Treat Population and Retrospective Analysis for Subgroup with Entry CAPS - 5 ≥33 A baseline CAPS - 5 score ≥33 was set as the PTSD severity inclusion criterion in Phase 3 HONOR study Intention - to - Treat

  25. Slide 25: AtEase Study Effect Sizes for Total CAPS-5 and Symptom Clusters

    © 2018 Tonix Pharmaceuticals Holding Corp. 25 0.53 0.46 0.12 0.39 0.52 0 0.1 0.2 0.3 0.4 0.5 0.6 Effect Size AtEase Study Effect Sizes for Total CAPS - 5 and Symptom Clusters for Intention - to - Treat Population and Subgroup with Entry CAPS - 5 ≥33 Effect Sizes of Tonmya 5.6 mg Subgroup with Entry

  26. Slide 26: AtEase Study Retrospective Analysis: Remission in Subgroup

    © 2018 Tonix Pharmaceuticals Holding Corp. 26 AtEase Study Retrospective Analysis: Remission in Subgroup with Entry CAPS - 5 ≥33 Remission = Loss of Diagnosis and CAPS - 5 < 11 Asterisk and hashmark represent pairwise comparisons between Tonmya and Placebo; # p=0.08, Odds Ratio 3.01 (0.89, 10.18) *p

  27. Slide 27: Commercialization Options

    © 2018 Tonix Pharmaceuticals Holding Corp. 27 Commercialization Options Tonix is exploring a variety of options to commercialize TNX - 102 SL, including commercializing on our own or partnering all or some indications in specific regions of the world. Tonix has participated in numerous partnering me

  28. Slide 28: TNX-102 SL – Multiple Potential Indications

    © 2018 Tonix Pharmaceuticals Holding Corp. 28 TNX - 102 SL – Multiple Potential Indications Management of Fibromyalgia (FM) – chronic pain condition • TNX - 102 SL clinical development in FM was halted after near miss in Phase 3 at low dose (2.8 mg) – half the dose being developed for PTSD • Imbalan

  29. Slide 29: Consequences of Agitation in Alzheimer's Disease

    © 2018 Tonix Pharmaceuticals Holding Corp. 29 Consequences of Agitation in Alzheimer’s Disease Outcomes • Agitation is associated with significant poor outcomes for Alzheimer’s patients and challenges for their caregivers Common reason for institutionalization • Development of agitation, or its wors

  30. Slide 30: Agitation in Alzheimer's Disease – Potential New Indication for TNX-102 SL

    © 2018 Tonix Pharmaceuticals Holding Corp. 30 Agitation in Alzheimer’s Disease – Potential New Indication for TNX - 102 SL Phase 2 IND cleared April 2018 Significant unmet need • No FDA approved drugs for the treatment of agitation in Alzheimer’s Mechanism of improving sleep quality • Sleep disturba

  31. Slide 31: Scientific Rationale for Developing TNX-102 SL for Agitation in Alzheimer's

    © 2018 Tonix Pharmaceuticals Holding Corp. 31 Scientific Rationale for Developing TNX - 102 SL for Agitation in Alzheimer’s Disease Connection between Sleep Disturbance and Agitation • Agitation in Alzheimer’s Disease is associated with sleep disturbance 1,2 • Evidence that improving sleep could imp

  32. Slide 32: Candidates in Development

    © 2018 Tonix Pharmaceuticals Holding Corp. 32 Candidates in Development Preclinical Phase 2 NDA 1 /BLA 2 Market Pipeline Product Indication/ Description Phase 3 Tonmya Bedtime Treatment for PTSD Daytime Treatment for PTSD TNX - 601 Novel polymorph and salt discovered and characterized TNX - 801 Hors

  33. Slide 33: TNX-601 (Tianeptine Oxalate): A Potential Clinical Candidate for PTSD

    © 2018 Tonix Pharmaceuticals Holding Corp. 33 TNX - 601 ( Tianeptine Oxalate): A Potential Clinical Candidate for PTSD Pre - IND Candidate Targeted as a 1 st line monotherapy for PTSD: oral formulation for daytime dosing x Leverages expertise in PTSD (clinical and regulatory experience, market analy

  34. Slide 34: TNX-801 (Synthesized Live Horsepox Virus): Smallpox-Preventing Vaccine Candidate

    © 2018 Tonix Pharmaceuticals Holding Corp. 34 TNX - 801 (Synthesized Live Horsepox Virus): A Smallpox - Preventing Vaccine Candidate Pre - IND Stage Potential improvement over current biodefense tools against smallpox ✓ Leverages Tonix’s government affairs effort ✓ Collaboration with Professor David

  35. Slide 35: TNX-801: Synthesis and Relation to Modern Vaccines

    © 2018 Tonix Pharmaceuticals Holding Corp. 35 TNX - 801 (Synthesized Live Horsepox Virus): A Smallpox - Preventing Vaccine Candidate Synthesis 1 from sequence of a 1976 Mongolian isolate 2 In mice, TNX - 801 behaved like attenuated vaccinia virus • Vaccinia is the term used to classify the live poxv

  36. Slide 36: Biological Properties of HPXV: Less Virulent than a Dryvax Clone

    36 © 2018 Tonix Pharmaceuticals Holding Corp. PBS VACV WR (5x10 3 PFU) Dryvax clone (10 7 PFU) HPXV (10 7 PFU) Challenge (10 6 PFU VAC strain WR) Biological Properties of HPXV: Less Virulent than a Dryvax Clone, but Produces Protective Immunity Noyce, RS, Lederman S, Evans DH. PLoS ONE. 2018; 13(1):

  37. Slide 37: Current Needs to Vaccinate Against Smallpox

    © 2018 Tonix Pharmaceuticals Holding Corp. 37 Ongoing vaccination of U.S. troops • Troops in the Global Response Force Threat of smallpox re - introduction • Strategic National Stockpile & public health policy Re - emergence of monkey pox 1 • Believed to resurgent because of vaccinia - naïve populat

  38. Slide 38: Potential for Use of HPXV as a Vector for Vaccines

    © 2018 Tonix Pharmaceuticals Holding Corp. 38 Potential for Use of HPXV as a Vector for Vaccines to Infectious Disease or Cancer Poxviruses like HPXV can be engineered to express foreign genes and are well recognized platforms for vaccine development • Large packaging capacity for exogenous DNA inse

  39. Slide 39: Management Team

    © 2018 Tonix Pharmaceuticals Holding Corp. 39 Management Team Seth Lederman, MD President & CEO Jessica Morris Chief Operating Officer Gregory Sullivan, MD Chief Medical Officer Bradley Saenger, CPA Chief Financial Officer

  40. Slide 40: Board of Directors

    © 2018 Tonix Pharmaceuticals Holding Corp. 40 Board of Directors Seth Lederman, MD Chairman Ernest Mario, PhD ALZA, Glaxo, Reliant Pharma John Rhodes Chair, NYS Public Service Commission, CEO, NYS Dept. of Public Service, Booz Allen Samuel Saks, MD Jazz Pharma, ALZA, Johnson & Johnson Charles Mather

  41. Slide 41: Milestones – Recently Completed and Upcoming

    © 2018 Tonix Pharmaceuticals Holding Corp. 41 Milestones – Recently Completed and Upcoming T onmya – Posttraumatic Stress Disorder □ July 201 8 HONOR study discontinued – interim analysis result did not support study continuation □ August 201 8 Presentation of HONOR study results at scientific meeti

  42. Slide 42: Summary

    © 2018 Tonix Pharmaceuticals Holding Corp. 42 Summary Phase 3 Breakthrough Therapy development for PTSD focused on military - related PTSD • Major unmet need; ~11 million Americans affected • Potential single - study NDA submission New indication in development for agitation in Alzheimer’s Disease •

  43. Slide 43: Thank you!

    © 2018 Tonix Pharmaceuticals Holding Corp. Thank you ! NASDAQ: TNXP

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