Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP

Tonix Pharmaceuticals — Investor Presentation (January 2017)

44 slides from Tonix Pharmaceuticals's investor presentation, Jan 31, 2017

  1. Slide 1: Investor Presentation January 2017

    © 2017 Tonix Pharmaceuticals Holding Corp. January 2017 Version P0050 1 - 31 - 17 Investor Presentation

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

    © 2017 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: Developing Innovative Pharmaceutical Products for Public Health Challenges

    © 2017 Tonix Pharmaceuticals Holding Corp. 3 Tonix (TNXP): Developing Innovative Pharmaceutical Products to Address Current and Emerging Public Health Challenges Targeting central nervous system conditions • Phase 3 - ready drug, TNX - 102 SL* for posttraumatic stress disorder (PTSD) with Breakthrou

  4. Slide 4: Product Pipeline

    © 2017 Tonix Pharmaceuticals Holding Corp. 4 Product Pipeline Preclinical Phase 2 NDA/BLA Market Candidate Indication Phase 3 TNX - 102 SL PTSD 505(b)(2) Phase 2 results reported May, 2016 PTSD TNX - 601 505(b)(1) Novel polymorph and salt discovered and characterized TNX - 801* 601 (h) Novel virus v

  5. Slide 5: PTSD Program: Phase 3-Ready Program

    © 2017 Tonix Pharmaceuticals Holding Corp. 5 Tonix Pharmaceuticals PTSD Program Phase 3 Ready Program Targeting a Current and Emerging Public Health Challenge TNX - 102 SL (cyclobenzaprine HCl sublingual tablets) • A unique, innovative pr oduct designed for bedtime administration • Targeting a chron

  6. Slide 6: PTSD Characteristics

    © 2017 Tonix Pharmaceuticals Holding Corp. 6 PTSD Characteristics • Unmet medical need • PTSD is a serious condition and the prevalence is increasing, especially combat PTSD • Military - related PTSD is not satisfactorily treated by existing FDA approved therapies • Two selective serotonin reuptake

  7. Slide 7: Breakthrough Therapy Designation

    © 2017 Tonix Pharmaceuticals Holding Corp. 7 Breakthrough Therapy Designation • FDA granted TNX - 102 SL Breakthrough Therapy designation – reported December 19, 2016 • PTSD is a serious condition • TNX - 102 SL has potential advantages over existing therapies in military - related PTSD • Benefits o

  8. Slide 8: What is PTSD?

    © 2017 Tonix Pharmaceuticals Holding Corp. 8 What is PTSD? A chronic response to traumatic event(s) • A majority of people will experience a traumatic event at some point in their lifetime 1 • 20% of women and 8% of men in the U.S. who experience significant trauma develop PTSD 1 • Lifetime prevalen

  9. Slide 9: What Are the Symptoms of PTSD?

    © 2017 Tonix Pharmaceuticals Holding Corp. 9 What Are the Symptoms of PTSD? Symptoms of PTSD fall into four clusters: 1. Intrusion ( aversive memories, nightmares, flashbacks ) 2. Avoidance ( avoiding persons, places or situations ) 3. Mood/cognitions ( memory block, emotional numbing, detachment fr

  10. Slide 10: What Are the Consequences of PTSD?

    © 2017 Tonix Pharmaceuticals Holding Corp. 10 What Are the Consequences of PTSD? 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

  11. Slide 11: PTSD Prevalence and Market Characteristics

    © 2017 Tonix Pharmaceuticals Holding Corp. 11 PTSD Prevalence and Market Characteristics [CATEGORY NAME] [VALUE] [PERCENTAG E] Rx [VALUE] [PERCENTAG E] No Rx [VALUE] [PERCENTAG E] Rx [VALUE] [PERCENTAG E] No Rx [VALUE] [PERCENTAG E] 1. Kessler, et al., 2005; Prevalence rate of 3.5% applied to U.S. C

  12. Slide 12: What Drug Classes are Used to Treat PTSD?

    © 2017 Tonix Pharmaceuticals Holding Corp. 12 What Drug Classes are Used to Treat PTSD? * TRx = Total prescriptions 1. VA/DoD Clinical Practice Guideline for the Management of Post - Traumatic Stress, Version 2, 2010 2. IMS Consulting, Market Sizing & Treatment Dynamics: “Post - Traumatic Stress Dis

  13. Slide 13: Sleep Disturbances Are a Core Feature of PTSD

    © 2017 Tonix Pharmaceuticals Holding Corp. 13 Sleep Disturbances Are a Core Feature Of PTSD 1 Sleep disturbances are a core feature of PTSD and a component of three of the four major symptom clusters: 1 Germain A. Am J Psychiatry . 2013; 2 American Psychiatric Association. Diagnostic and statistical

  14. Slide 14: What Is Restorative Sleep?

    © 2017 Tonix Pharmaceuticals Holding Corp. 14 What Is Restorative Sleep? Restorative Sleep Hypothesis 1 : Sleep is an active , necessary state for processing emotionally charged memories , replenishing energy, and resetting homeostasis to circuitry in the brain Homeostasis Sleep Deprivation Sleep Di

  15. Slide 15: Relevance of Sleep Disturbances for PTSD

    © 2017 Tonix Pharmaceuticals Holding Corp. 15 Relevance of Sleep Disturbances for PTSD Sleep disturbances: • Core symptom of PTSD (component of intrusion and hyperarousal symptom clusters) • Believed to play roles in the pathophysiology of PTSD om Sleep As a Core Symptom P athophysiology Pharmacolog

  16. Slide 16: TNX-102 SL: Innovative and Unique by Design

    © 2017 Tonix Pharmaceuticals Holding Corp. 16 • CBP is more selective for high affinity sites believed to have a role in sleep quality 2 • 5 - HT 2A • a 1 - adrenergic • histamine H 1 • CBP undergoes extensive first - pass hepatic metabolism when administered orally • Major metabolite, norcyclobenza

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

    © 2017 Tonix Pharmaceuticals Holding Corp. 17 High Prevalence of PTSD Among Combat Veterans 1 Kessler, Arch Gen Psych 2005 2 Norris, PTSD Res Quar . 2013 3 Tanielan , Invisible wounds of war . 2005 4 CBO Report 2012 5 http://www.nimh.nih.gov/health/topics/post - traumatic - stress - disorder - ptsd/

  18. Slide 18: Growing Economic and Social Burden to Care for Veterans with PTSD

    © 2017 Tonix Pharmaceuticals Holding Corp. 18 Growing Economic and Social Burden to Care for Veterans with PTSD Health care costs associated with PTSD for OEF/OIF veterans: Direct costs Indirect costs 1 CBO Report 2012; 2 Tanielan, Invisible Wounds of War . 2005 $6,000 - 10,000 per patient per year

  19. Slide 19: Why Initially Target Military-Related PTSD?

    © 2017 Tonix Pharmaceuticals Holding Corp. 19 Why Initially Target Military - Related PTSD? 1. Friedman et al., J Clin Psychiatry 2007; 68:711 2. Zoloft Package Insert, August, 2014 3. Paxil Package Insert, June, 2014 4. Fava et al., Psychother Psychosom 84:72 - 81, 2015 Military - related PTSD not

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

    © 2017 Tonix Pharmaceuticals Holding Corp. 20 Phase 2 AtEase Study in Military PTSD • Enrolled patients with baseline CAPS - 5 ≥ 29 • Analysis from 231 patients; 24 U.S. clinical sites • TNX - 102 SL was active at 5.6 mg dose • Primary efficacy analysis: • Difference in CAPS - 5 score between TNX -

  21. Slide 21: AtEase Study Demographics and Characteristics

    © 2017 Tonix Pharmaceuticals Holding Corp. 21 AtEase Study Demographics and Characteristics 93% of the randomized patients were male 98% had trauma during military service Deployed an average of 2.3 times Mean time since index trauma was 7 years Race and ethnicity generally consistent with U.S. mili

  22. Slide 22: AtEase Study: Traumas Associated with PTSD

    © 2017 Tonix Pharmaceuticals Holding Corp. 22 AtEase Study: Traumas Associated with PTSD Index Trauma During Military Service Patient Count* Being involved in an IED explosion or suicide bombing 35 Witness death or injury of fellow soldiers 33 Witnessing IED explosion 30 Receiving incoming artillery

  23. Slide 23: AtEase Study – Summary of Primary and Secondary Analyses

    © 2017 Tonix Pharmaceuticals Holding Corp. 23 AtEase Study – Summary of Primary and Secondary Analyses (week 12)

  24. Slide 24: AtEase Study: Safety and Tolerability Profile

    © 2017 Tonix Pharmaceuticals Holding Corp. 24 AtEase Study: Safety and Tolerability Profile No serious adverse events reported with TNX - 102 SL deemed related to treatment Systemic Adverse Events* Placebo (N=94) TNX - 102 SL 2.8 mg (N=93) TNX - 102 SL 5.6 mg (N=50) Somnolence 6.4% 11.8% 16.0% Dry M

  25. Slide 25: AtEase: Retrospective Analysis for TNX-102 SL 5.6 mg

    © 2017 Tonix Pharmaceuticals Holding Corp. 25 0 0.1 0.2 0.3 0.4 0.5 0.6 Effect Size CAPS - 5 ≥ 29 0 0.1 0.2 0.3 0.4 0.5 0.6 Effect Size CAPS - 5 ≥33 AtEase: Retrospective Analysis for TNX - 102 SL 5.6 mg * MMRM, mixed - effects model repeated measures, p<0.05 Prior pharmacotherapy trials in PTSD use

  26. Slide 26: Retrospective Analysis of CAPS-5 with Entry CAPS-5 ≥ 33

    © 2017 Tonix Pharmaceuticals Holding Corp. 26 Retrospective Analysis of CAPS - 5 in Patients with Entry CAPS - 5 ≥ 33 1 -30.0 -25.0 -20.0 -15.0 -10.0 -5.0 0.0 Wk 0 Wk 2 Wk 4 Wk 6 Wk 8 Wk 10 Wk 12 LS Mean (SE) of CAPS - 5 Total Score Placebo TNX-102 SL 2.8 mg TNX-102 SL 5.6 mg 6.8 point difference in

  27. Slide 27: Retrospective Analyses of Week 12 Outcome Measures

    © 2017 Tonix Pharmaceuticals Holding Corp. 27 Retrospective Analyses of Week 12 Outcome Measures for TNX - 102 SL 5.6 mg vs. Placebo in Military - related or Combat PTSD Subset and for CAPS - 5 ≥ 29 and CAPS - 5 ≥ 33 at Baseline Military - related PTSD Combat PTSD CAPS - 5 at Baseline ≥ 29 ≥ 33 ≥ 29

  28. Slide 28: Phase 2 AtEase Study Conclusions

    © 2017 Tonix Pharmaceuticals Holding Corp. 28 First large multi - center trial demonstrating efficacy of an Investigational New Drug (IND) in military - related PTSD x TNX - 102 SL therapeutic dose (5.6 mg) identified x Symptom reduction (CAPS - 5) x Functional improvement (Sheehan Disability Scale

  29. Slide 29: Planned Phase 3 HONOR Study in PTSD

    © 2017 Tonix Pharmaceuticals Holding Corp. 29 Planned Phase 3 HONOR Study in PTSD General s tudy c haracteristics: • Randomized, double - blind, placebo - controlled, entrance CAPS - 5 ≥ 33 • One unblinded interim analys i s ( IA) by an independent data monitoring committee at 50% recruitment • IA (

  30. Slide 30: Commercialization Options

    © 2017 Tonix Pharmaceuticals Holding Corp. 30 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. Commercial considerations: • Primary physician a

  31. Slide 31: Intellectual Property

    © 2017 Tonix Pharmaceuticals Holding Corp. 31 Intellectual Property Portfolio of international patents filed under the Patent Cooperation Treaty (PCT) C omposition - of - matter (eutectic) • Patents filed • Protection expected to 2034 Pharmacokinetics (PK) • Patents filed • Protection expected to 20

  32. Slide 32: Proprietary Cyclobenzaprine Eutectic Mixture Stabilizes Tablet Formulation

    © 2017 Tonix Pharmaceuticals Holding Corp. 32 Proprietary Cyclobenzaprine Hydrochloride Eutectic Mixture Stabilizes Tablet Formulation Base particle (K 2 HPO 4 ) Base particle (K 2 HPO 4 ) Base particle (K 2 HPO 4 ) Eutectic formulation ANGSTRO - TECHNOLOGY™ Cyclobenzaprine - HCl (CBP - HCl) Eutecti

  33. Slide 33: Product Pipeline

    © 2017 Tonix Pharmaceuticals Holding Corp. 33 Product Pipeline Preclinical Phase 2 NDA/BLA Market Candidate Indication Phase 3 TNX - 102 SL PTSD 505(b)(2) Phase 2 results reported May, 2016 PTSD TNX - 601 505(b)(1) Novel polymorph and salt discovered and characterized TNX - 801* 601 (h) Novel virus

  34. Slide 34: TNX-601: A Potential Clinical Candidate for PTSD

    © 2017 Tonix Pharmaceuticals Holding Corp. 34 TNX - 601 - A Potential Clinical Candidate for PTSD Pre - IND Candidate TNX - 601 ( tianeptine oral formulation) • Targeted as a 1 st line monotherapy for PTSD : daytime dosing x Leverages expertise in PTSD (clinical and regulatory experience, market ana

  35. Slide 35: Structural Comparison: TNX-102 SL and TNX-601

    © 2017 Tonix Pharmaceuticals Holding Corp. 35 Structural Comparison: TNX - 102 SL and TNX - 601 • Cyclobenzaprine and tianeptine share structural similarities with classic tricyclic antidepressants (TCAs) and to each other, but each has unique pharmacological properties • Tianeptine has a 3 - chloro

  36. Slide 36: TNX-601: A Potential Clinical Candidate for PTSD

    © 2017 Tonix Pharmaceuticals Holding Corp. 36 • Tianeptine modulates the glutamatergic system indirectly • Does not have significant affinity (K i >10 μ M) for NMDA 2 or AMPA 3 receptors • Tianeptine is a weak µ - opioid receptor (MOR) agonist • Controlled substance in France, Belgium and Singapore

  37. Slide 37: TNX-801: A Potential Smallpox Preventing Vaccine

    © 2017 Tonix Pharmaceuticals Holding Corp. 37 TNX - 801 - A Potential Smallpox Preventing Vaccine Pre - IND Candidate TNX - 801 (live virus vaccine) • Targeting a biological warfare threat: smallpox x Leverages government affairs effort • Tonix has identified a new vaccine with improved properties x

  38. Slide 38: TNX-801: A Potential Smallpox Preventing Vaccine

    © 2017 Tonix Pharmaceuticals Holding Corp. 38 • Live virus vaccines stimulate cross - reactive immunity • Protects from consequences of infection with variola virus (smallpox agent) • Renders recipient “immune” and p rotects population (even un - immunized) by decreasing spread of infection TNX - 80

  39. Slide 39: Financial Overview

    © 2017 Tonix Pharmaceuticals Holding Corp. 39 Financial overview NASDAQ: TNXP Cash, cash equivalents, and marketable securities reported at September 30, 2016 $26.7 million Net proceeds from underwritten offering in 4Q16 $4.6 million Shares outstanding (January 31, 2017) 39.2 million

  40. Slide 40: Management Team

    © 2017 Tonix Pharmaceuticals Holding Corp. 40 Management Team Seth Lederman, MD President & CEO Jessica Morris EVP, Operations Gregory Sullivan, MD Chief Medical Officer Bradley Saenger, CPA Chief Financial Officer

  41. Slide 41: Board of Directors

    © 2017 Tonix Pharmaceuticals Holding Corp. 41 Board of Directors Seth Lederman, MD Chairman Ernest Mario, PhD ALZA, Glaxo, Reliant Pharma John Rhodes NYSERDA, NRDC, Booz Allen Hamilton Samuel Saks, MD Jazz Pharma, ALZA, Johnson & Johnson Charles Mather BTIG, Janney, Jefferies, Cowen, Smith Barney St

  42. Slide 42: Milestones – Recent and Upcoming

    © 2017 Tonix Pharmaceuticals Holding Corp. 42 Milestones – recent and upcoming TNX - 102 SL – Posttraumatic Stress Disorder □ May 2016 Report ed results from AtEase study □ August 2016 End - of - Phase 2 meeting with FDA - Proposed Phase 3 clinical and NDA plan accepted □ December 201 6 Breakthrough

  43. Slide 43: Summary

    © 2017 Tonix Pharmaceuticals Holding Corp. 43 Summary Strong position for value growth with Phase 3 trial in a major medical indication: PTSD including military - related PTSD • Phase 3 asset not previously well - known to the investor marketplace TNX - 102 SL for PTSD is designated as a Breakthroug

  44. Slide 44: Thank you!

    © 2017 Tonix Pharmaceuticals Holding Corp. Thank you ! NASDAQ: TNXP

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