Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP

Tonix Pharmaceuticals — Investor Presentation (December 2016)

35 slides from Tonix Pharmaceuticals's investor presentation, Dec 8, 2016

  1. Slide 1: Investor Presentation December 2016

    © 2016 Tonix Pharmaceuticals Holding Corp December 2016 Version P0043 12 - 8 - 16 Investor Presentation

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

    © 2016 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 Litigati

  3. Slide 3: Posttraumatic Stress Disorder (PTSD) program

    © 2016 Tonix Pharmaceuticals Holding Corp 3 Posttraumatic Stress Disorder (PTSD) program On May 19, 2016, we reported encouraging topline data for TNX - 102 SL* 5.6 mg in a military - related PTSD trial (“AtEase”) • PTSD was our “second” clinical program using our proprietary formulation TNX - 102 S

  4. Slide 4: Tonix (TNXP): Value proposition

    © 2016 Tonix Pharmaceuticals Holding Corp 4 Tonix (TNXP): Value proposition On September 6, 2016 our lead Phase 3 program TNX - 102 SL for fibromyalgia narrowly missed its primary endpoint in the first Phase 3 study (“AFFIRM”) • Received strong negative investor market response • Reassuring safety p

  5. Slide 5: Pivot to PTSD: Rationale

    © 2016 Tonix Pharmaceuticals Holding Corp 5 Pivot to PTSD: Rationale • Unmet medical need • PTSD is a serious condition and the prevalence is increasing, especially combat - related • Military - related PTSD is not satisfactorily treated by existing FDA approved therapies • Two selective serotonin r

  6. Slide 6: PTSD Program: Phase 3 Ready Program Targeting an Attractive Market

    © 2016 Tonix Pharmaceuticals Holding Corp 6 Tonix Pharmaceuticals Posttraumatic Stress Disorder (PTSD) Program Phase 3 Ready Program Targeting An Attractive Market TNX - 102 SL (cyclobenzaprine HCl sublingual tablets) • A unique, innovative pr oduct designed for bedtime administration • Targeting a

  7. Slide 7: What is PTSD?

    © 2016 Tonix Pharmaceuticals Holding Corp 7 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 prevalenc

  8. Slide 8: What Are the Symptoms of PTSD?

    © 2016 Tonix Pharmaceuticals Holding Corp 8 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 fro

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

    © 2016 Tonix Pharmaceuticals Holding Corp 9 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 f

  10. Slide 10: PTSD Prevalence and Market Characteristics

    © 2016 Tonix Pharmaceuticals Holding Corp 10 PTSD Prevalence and Market Characteristics Undiagnosed 6,788,000 79% Rx 510,000 6% No Rx 128,000 2% Rx 872,000 10% No Rx 288,000 3% 1. Kessler, et al., 2005; ; Prevalence rate of 3.5% applied to U.S. Census estimate of 247 million U.S. adult ( > 18) popul

  11. Slide 11: What Drug Classes are Used to Treat PTSD?

    © 2016 Tonix Pharmaceuticals Holding Corp 11 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 Disor

  12. Slide 12: Relevance of Sleep Disturbances for PTSD

    © 2016 Tonix Pharmaceuticals Holding Corp 12 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

  13. Slide 13: Innovative and Unique By Design

    © 2016 Tonix Pharmaceuticals Holding Corp 13 • 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, norcyclobenzap

  14. Slide 14: Why Initially Target Military-Related PTSD?

    © 2016 Tonix Pharmaceuticals Holding Corp 14 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 no

  15. Slide 15: Phase 2 AtEase Study in Military PTSD

    © 2016 Tonix Pharmaceuticals Holding Corp 15 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 - 1

  16. Slide 16: AtEase Study Demographics and Characteristics

    © 2016 Tonix Pharmaceuticals Holding Corp 16 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. milit

  17. Slide 17: AtEase Study: Traumas Associated with PTSD

    © 2016 Tonix Pharmaceuticals Holding Corp 17 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,

  18. Slide 18: AtEase Study – Summary of Primary and Secondary Analyses (week 12)

    © 2016 Tonix Pharmaceuticals Holding Corp 18 AtEase Study – Summary of Primary and Secondary Analyses (week 12)

  19. Slide 19: AtEase Study: Safety and Tolerability Profile

    © 2016 Tonix Pharmaceuticals Holding Corp 19 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 Mo

  20. Slide 20: AtEase: Retrospective Analysis for 5.6 mg

    © 2016 Tonix Pharmaceuticals Holding Corp 20 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 used

  21. Slide 21: Retrospective Analysis of CAPS-5 in patients with entry CAPS-5 ≥ 33

    © 2016 Tonix Pharmaceuticals Holding Corp 21 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

  22. Slide 22: Week 12 outcome measures for 5.6 mg vs. placebo in military-related PTSD

    © 2016 Tonix Pharmaceuticals Holding Corp 22 Week 12 outcome measures for TNX - 102 SL 5.6 mg vs. placebo in military - related PTSD for CAPS - 5 ≥ 29 and CAPS - 5 ≥ 33 at baseline PBO N=92, 5.6mg N=49; CAPS - 5 ≥ 29 PBO N=77, 5.6mg N=38; CAPS - 5 ≥ 33 Outcome Measure ES 1 p - value 2 ES 1 p - value

  23. Slide 23: Week 12 outcome measures for 5.6 mg vs. placebo in combat PTSD subset

    © 2016 Tonix Pharmaceuticals Holding Corp 23 Week 12 outcome measures for TNX - 102 SL 5.6 mg vs. placebo in combat PTSD subset for CAPS - 5 ≥ 29 and CAPS - 5 ≥ 33 at baseline PBO N=74, 5.6mg N=46; CAPS - 5 ≥ 29 PBO N= 64, 5.6mg N=35; CAPS - 5 ≥ 33 Outcome Measure ES 1 p - value 2 ES 1 p - value 2 C

  24. Slide 24: Reckless or Self-Destructive Behavior Item on CAPS-5

    © 2016 Tonix Pharmaceuticals Holding Corp 24 Analysis Using CAPS - 5 ≥ 33 As Entry Criteria Reckless or Self - Destructive Behavior Item on CAPS - 5 0 0.5 1 Wk 0 Wk 2 Wk 4 Wk 6 Wk 8 Wk 10 Wk 12 Reckless or Self - Destructive Mean Score Placebo TNX-102 SL 2.8 mg TNX-102 SL 5.6 mg * * p=0.012, TNX - 1

  25. Slide 25: Phase 2 AtEase Study Conclusions

    © 2016 Tonix Pharmaceuticals Holding Corp 25 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 d

  26. Slide 26: Hypothesized mechanism of action in PTSD

    © 2016 Tonix Pharmaceuticals Holding Corp 26 Hypothesized mechanism of action in P TSD Attenuation of Sleep Disruption by TNX - 102 SL Leads to Recovery Recovery (over weeks) Diagrams adapted from Pace - Schott et al. Biol Mood Anxiety Disord 2015;5:3

  27. Slide 27: Planned Phase 3 Program in PTSD

    © 2016 Tonix Pharmaceuticals Holding Corp 27 Planned Phase 3 Program in PTSD General Study Characteristics: • Randomized, double - blind, placebo - controlled, entrance CAPS - 5 ≥ 33 • One to two planned unblinded interim analyses (IAs) • First IA (N ~180) for efficacy stop or sample size adjustment

  28. Slide 28: Commercialization Options

    © 2016 Tonix Pharmaceuticals Holding Corp 28 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 au

  29. Slide 29: Intellectual Property Portfolio

    © 2016 Tonix Pharmaceuticals Holding Corp 29 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 203

  30. Slide 30: Financial overview

    © 2016 Tonix Pharmaceuticals Holding Corp 30 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 (December 1, 2016) 39.2 million

  31. Slide 31: Management Team

    © 2016 Tonix Pharmaceuticals Holding Corp 31 Management Team Seth Lederman, MD President & CEO Jessica Morris EVP, Operations Gregory Sullivan, MD Chief Medical Officer Bradley Saenger, CPA Chief Financial Officer

  32. Slide 32: Board of Directors

    © 2016 Tonix Pharmaceuticals Holding Corp 32 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 Stu

  33. Slide 33: Milestones – recent and upcoming – Posttraumatic Stress Disorder

    © 2016 Tonix Pharmaceuticals Holding Corp 33 Milestones – recent and upcoming TNX - 102 SL – Posttraumatic Stress Disorder □ December 2015 Entered into Collaborative Research and Development Agreement (CRADA) with the United States Army Medical Materiel Development Activity (USAMMDA) □ May 2016 Repo

  34. Slide 34: Summary

    © 2016 Tonix Pharmaceuticals Holding Corp 34 Summary Strong position for value growth with Phase 3 trial in a major medical indication: PTSD • Phase 3 asset not previously well - known to the investor marketplace Funded through 1 st interim analysis (180 patients) of Phase 3 PTSD trial expected to i

  35. Slide 35: Thank you!

    © 2016 Tonix Pharmaceuticals Holding Corp Thank you ! NASDAQ: TNXP

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