Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP

Tonix Pharmaceuticals — Investor Presentation (August 2018)

50 slides from Tonix Pharmaceuticals's investor presentation, Aug 21, 2018

  1. Slide 1: Investor Presentation

    © 2018 Tonix Pharmaceuticals Holding Corp. August 2018 Version P0125 8 - 21 - 18 ( Doc 0380) 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 3 Daytime Treatment for PTSD TNX - 601 Novel polymorph and salt discovered and characterized TNX - 801 Hor

  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 PTSD – Bedtime treatment in Phase 3 Development • Results from 2 efficacy studies can improve a new Phase 3 study design • FDA feedback and agreement are expected 4Q2018 • Pivotal efficacy study may ini

  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 his

  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 Posttraumatic Stress Disorder (PTSD)

    © 2018 Tonix Pharmaceuticals Holding Corp. 14 Overview of Posttraumatic Stress Disorder (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

  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: AtEase/P201 Study in Military-Related PTSD

    © 2018 Tonix Pharmaceuticals Holding Corp. 18 Phase 2 AtEase/P201 1 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 2 ≥ 29 •

  19. Slide 19: AtEase/P201 Study

    © 2018 Tonix Pharmaceuticals Holding Corp. 19 AtEase/P201 Study AtEase was a large adequate well - controlled study in military - related PTSD • Separation on primary endpoint at Week 12 did not meet primary endpoint at Week 12 for TNX - 102 SL 2.8 mg • No safety or tolerability issue discovered • R

  20. Slide 20: AtEase/P201 Study – Summary of Primary and Secondary Analyses (Week 12)

    © 2018 Tonix Pharmaceuticals Holding Corp. 20 AtEase/P201 Study – Summary of Primary and Secondary Analyses (Week 12)

  21. Slide 21: HONOR/P301 Study – Discontinued Due to High Placebo Response

    © 2018 Tonix Pharmaceuticals Holding Corp. 21 HONOR/P301 1 Study – Evidence of Efficacy at Week 4 Discontinued Due to High Placebo Response at Week 12 Primary e ndpoint CAPS - 5 2 : • Mean change from baseline at w eek 12 (Tonmya 5.6 mg vs. placebo ) Unblinded interim analysis (IA) at 274 randomized

  22. Slide 22: HONOR/P301 Study - Primary Analysis in mITT Population

    © 2018 Tonix Pharmaceuticals Holding Corp. 22 HONOR/P301 Study - Primary Analysis in mITT Population In P301 study both TNX - 102 SL and placebo - treated groups improved but the greater improvement on TNX - 102 SL compared with placebo diminished over time • TNX - 102 SL did not separate from place

  23. Slide 23: HONOR/P301 Study Stopped After Interim Analysis

    © 2018 Tonix Pharmaceuticals Holding Corp. 23 HONOR/P301 Study Stopped After Interim Analysis (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 •

  24. Slide 24: Differences Between AtEase/P201 and HONOR/P301 Studies

    © 2018 Tonix Pharmaceuticals Holding Corp. 24 Differences Between AtEase/P201 and HONOR/P301 Studies Design Phase 2 and 3 studies were very similar – both studied military related PTSD at multiple sites in the US • CAPS - 5 ≥ 33 entry criteria used in Phase 3 BDI - II= Beck Depression Inventory - II

  25. Slide 25: AtEase/P201 and HONOR/P301 Demographics and Characteristics

    © 2018 Tonix Pharmaceuticals Holding Corp. 25 AtEase/P201 and HONOR/P301 Demographics and Characteristics The striking difference between P201 and P301 was time since trauma • Phase 2 AtEase P201 study recruited many participants from the surge in Iraq who were mostly <9 years since trauma

  26. Slide 26: Retrospective Comparison of Time Since Trauma

    © 2018 Tonix Pharmaceuticals Holding Corp. 26 Retrospective Comparison of Time Since Trauma in AtEase/P201 versus HONOR/P301 (TNX 5.6 mg Groups) P301 study was initiated approximately two years later than Phase 2 P201 • The median time since trauma in Phase 3 was 9.5 years compared to the median tim

  27. Slide 27: Retrospective Analysis of Treatment Response by Years Since Trauma

    © 2018 Tonix Pharmaceuticals Holding Corp. 27 Retrospective Analysis of Treatment Response in ≤9 & >9 Years since Trauma in HONOR/P301 Study The ≤ 9 years since trauma group in P301 replicated results from P201 • Retrospective analysis of P201 showed 5.6 mg treatment group difference over placebo of

  28. Slide 28: Retrospective Analyses of ≤9 Years Group on Key Secondary Endpoints

    © 2018 Tonix Pharmaceuticals Holding Corp. 28 Retrospective Analyses of ≤9 Years Since Trauma Group on Key Secondary Endpoints in HONOR/P301 Key secondary endpoints showed strong treatment effects • CGI - I, PGIC and PROMIS SD were pre - specified secondary analyses • Support results on CAPS - 5 and

  29. Slide 29: Adverse Events in AtEase/P201 and HONOR/P301

    © 2018 Tonix Pharmaceuticals Holding Corp. 29 Adverse Events (AEs) in AtEase/P201 and HONOR/P301 *# No serious and unexpected AEs in P301 or P201 • Systemic AEs comparable between studies and also consistent with those described in approved product labeling • Similar severity and incidence of oral h

  30. Slide 30: Sustained Remission in HONOR/P301 and AtEase/P201 Studies

    © 2018 Tonix Pharmaceuticals Holding Corp. 30 Sustained Remission in HONOR/P301 and AtEase/P201 Studies Retrospective Analyses of Phase 2 Subgroup with Entry CAPS - 5 ≥33 and Phase 3 subgroup ≤9 Years Since Trauma Remission is a clinical state that is essentially asymptomatic In order to confirm rem

  31. Slide 31: Time Since Trauma – Review of Published Studies

    © 2018 Tonix Pharmaceuticals Holding Corp. 31 Time Since Trauma – Review of Published Studies Published studies of prazosin suggested effects in military - PTSD prior to 9 years • Loss of treatment effect >9 years Paroxetine and sertraline studies supporting FDA approval were conducted on PTSD > 9 y

  32. Slide 32: Time Since Trauma – Remitting and Persistent Phases of PTSD

    © 2018 Tonix Pharmaceuticals Holding Corp. 32 Time Since Trauma – Remitting and Persistent Phases of PTSD Kessler et al 1 studied remission in PTSD with and without therapy • Identified remitting and persistent phase of PTSD – with transition at approximately 6 years post trauma • Supported by other

  33. Slide 33: Tonmya/TNX-102 SL – Summary

    © 2018 Tonix Pharmaceuticals Holding Corp. 33 Tonmya /TNX - 102 SL – Summary Median time since trauma in TNX - 102 SL 5.6 mg group in the HONOR/P301 study (9.5 years) was longer than AtEase/P201 study (6 years) • Time has passed since the surge in Iraq In retrospective analysis, the ≤ 9 year subgrou

  34. Slide 34: Commercialization Options

    © 2018 Tonix Pharmaceuticals Holding Corp. 34 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

  35. Slide 35: TNX-102 SL – Multiple Potential Indications

    © 2018 Tonix Pharmaceuticals Holding Corp. 35 TNX - 102 SL – Multiple Potential Indications Ma nagement of Fibromyalgia (FM) – chronic pain condition • TNX - 102 SL studied at low dose (2.8 mg) – half the dose being developed for PTSD – did not separate from placebo on primary endpoint, average pain

  36. Slide 36: Consequences of Agitation in Alzheimer's Disease

    © 2018 Tonix Pharmaceuticals Holding Corp. 36 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

  37. Slide 37: Agitation in Alzheimer's Disease – Additional Indication

    © 2018 Tonix Pharmaceuticals Holding Corp. 37 Agitation in Alzheimer’s Disease – Additional Indication Being Developed for TNX - 102 SL FDA designated Fast Track development program Significant unmet need • No FDA approved drugs for the treatment of agitation in Alzheimer’s Mechanism of improving sl

  38. Slide 38: Scientific Rationale for TNX-102 SL in Agitation in Alzheimer's Disease

    © 2018 Tonix Pharmaceuticals Holding Corp. 38 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

  39. Slide 39: Candidates in Development

    © 2018 Tonix Pharmaceuticals Holding Corp. 39 Candidates in Development Preclinical Phase 2 NDA 1 /BLA 2 Market Pipeline Product Indication/ Description Phase 3 Tonmya Bedtime Treatment for PTSD 3 Daytime Treatment for PTSD TNX - 601 Novel polymorph and salt discovered and characterized TNX - 801 Ho

  40. Slide 40: TNX-601 (Tianeptine Oxalate): A Potential Clinical Candidate for PTSD

    © 2018 Tonix Pharmaceuticals Holding Corp. 40 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

  41. Slide 41: TNX-801 (Synthesized Live Horsepox Virus): A Smallpox-Preventing Vaccine Candidate

    © 2018 Tonix Pharmaceuticals Holding Corp. 41 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

  42. Slide 42: TNX-801: Smallpox-Preventing Vaccine Candidate Synthesis

    © 2018 Tonix Pharmaceuticals Holding Corp. 42 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

  43. Slide 43: Biological Properties of HPXV: Less Virulent Than a Dryvax Clone

    43 © 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):

  44. Slide 44: Current Needs to Vaccinate Against Smallpox

    © 2018 Tonix Pharmaceuticals Holding Corp. 44 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

  45. Slide 45: Potential for Use of HPXV as a Vector for Vaccines

    © 2018 Tonix Pharmaceuticals Holding Corp. 45 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

  46. Slide 46: Management Team

    © 2018 Tonix Pharmaceuticals Holding Corp. 46 Management Team Seth Lederman, MD President & CEO Jessica Morris Chief Operating Officer Gregory Sullivan, MD Chief Medical Officer Bradley Saenger, CPA Chief Financial Officer

  47. Slide 47: Board of Directors

    © 2018 Tonix Pharmaceuticals Holding Corp. 47 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

  48. Slide 48: Milestones – Recently Completed and Upcoming

    © 2018 Tonix Pharmaceuticals Holding Corp. 48 Milestones – Recently Completed and Upcoming T onmya – Posttraumatic Stress Disorder □ July 201 8 Completed HONOR/P301 study interim analysis - result did not support study continuation □ August 201 8 Presentation of HONOR/P301 study results at Military

  49. Slide 49: Summary

    © 2018 Tonix Pharmaceuticals Holding Corp. 49 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 •

  50. Slide 50: Thank you!

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

Tonix Pharmaceuticals Holding — Investor Presentation (August 2018) on 6ix