Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP

Tonix Pharmaceuticals — Investor Presentation (July 2017)

44 slides from Tonix Pharmaceuticals's investor presentation, Jul 6, 2017

  1. Slide 1: Investor Presentation July 2017

    © 2017 Tonix Pharmaceuticals Holding Corp. July 2017 Version P0072 7 - 6 - 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: Tonmya for Posttraumatic Stress Disorder (PTSD)

    © 2017 Tonix Pharmaceuticals Holding Corp. 3 Tonmya ® (Cyclobenzaprine HCl Sublingual Tablets) for Posttraumatic Stress Disorder (PTSD) Phase 3 HONOR study of Tonmya 1 in military - related PTSD enrolling • Encouraging evidence of safety and efficacy was demonstrated in Phase 2 Breakthrough Therapy

  4. Slide 4: Phase 3 HONOR Study in PTSD Enrolling

    © 2017 Tonix Pharmaceuticals Holding Corp. 4 Phase 3 HONOR Study in PTSD Enrolling • 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% randomized • IA

  5. Slide 5: Breakthrough Therapy Designation

    © 2017 Tonix Pharmaceuticals Holding Corp. 5 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 Breakthrou

  6. Slide 6: No Recognized Abuse Potential in Clinical Studies

    © 2017 Tonix Pharmaceuticals Holding Corp. 6 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 ; α 1 - adrenergic and h

  7. Slide 7: Tonmya Intellectual Property – U.S. Protection until 2034

    © 2017 Tonix Pharmaceuticals Holding Corp. 7 Tonmya Intellectual Property – U.S. Protection until 2034 Composition of matter (eutectic) • U.S. Patent No. 9,636,408 issued May 2, 2017 by U.S. Patent and Trademark Office • Protection expected to 2034 • Additional claims and jurisdictions pending Pharm

  8. Slide 8: Proprietary Cyclobenzaprine Eutectic Mixture Stabilizes Sublingual Tablet

    © 2017 Tonix Pharmaceuticals Holding Corp. 8 Proprietary Cyclobenzaprine Hydrochloride Eutectic Mixture Stabilizes Sublingual Tablet Formulation Base particle (K 2 HPO 4 ) Base particle (K 2 HPO 4 ) Base particle (K 2 HPO 4 ) Eutectic formulation 1 ANGSTRO - TECHNOLOGY™ Cyclobenzaprine - HCl (CBP -

  9. Slide 9: Tonmya: Sublingual Formulation Designed for Bedtime Administration

    © 2017 Tonix Pharmaceuticals Holding Corp. 9 Tonmya : Sublingual Formulation is Designed for Bedtime Administration Tonmya : Proprietary sublingual formulation of CBP with transmucosal absorption • Innovation by design with patent protected CBP and mannitol eutectic formulation • Rapid systemic expo

  10. Slide 10: Tonmya: Novel Mechanism Targets Sleep Quality for Recovery from PTSD

    © 2017 Tonix Pharmaceuticals Holding Corp. 10 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 process

  11. Slide 11: What are the Symptoms of PTSD?

    © 2017 Tonix Pharmaceuticals Holding Corp. 11 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 from o

  12. Slide 12: What are the Consequences of PTSD?

    © 2017 Tonix Pharmaceuticals Holding Corp. 12 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

  13. Slide 13: PTSD U.S. Prevalence and Index Traumas

    © 2017 Tonix Pharmaceuticals Holding Corp. 13 PTSD U.S. Prevalence and Index Traumas 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

  14. Slide 14: PTSD Prevalence and Market Characteristics

    © 2017 Tonix Pharmaceuticals Holding Corp. 14 PTSD Prevalence and Market Characteristics 1 Kessler, et al., 2005; Prevalence rate of 3.5% applied to U.S. Census estimate of 247 million U.S. adult ( > 18) population in 2015 (www.census.gov/quickfacts/table/PST045215/00) 2 IMS Consulting, Market Sizin

  15. Slide 15: What Drug Classes are Used to Treat PTSD?

    © 2017 Tonix Pharmaceuticals Holding Corp. 15 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

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

    © 2017 Tonix Pharmaceuticals Holding Corp. 16 PTSD: Not Well - Served by Approved Treatments FDA approved selective serotonin reuptake inhibitors (SSRIs), paroxetine and sertraline, have not shown efficacy in military - related PTSD Majority of patients unresponsive or intolerant to current treatmen

  17. Slide 17: Why Initially Target Military-Related PTSD?

    © 2017 Tonix Pharmaceuticals Holding Corp. 17 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 well

  18. Slide 18: High Prevalence of PTSD Among Combat Veterans

    © 2017 Tonix Pharmaceuticals Holding Corp. 18 High Prevalence of PTSD Among Combat Veterans 1 Kessler et al., Arch Gen Psych 2005; Prevalence rate of 3.5% applied to U.S. Census estimate of 247M U.S. adult ( > 18) population in 2015 ( www.census.gov/quickfacts/table/PST045215/00 ) ; 2 Norris, PTSD R

  19. Slide 19: Growing Economic and Social Burden to Care for Veterans with PTSD

    © 2017 Tonix Pharmaceuticals Holding Corp. 19 Growing Economic and Social Burden to Care for Veterans with PTSD Health care costs associated with PTSD for OEF/OIF/OND veterans: Direct costs Indirect costs 1 CBO Report 2012; 2 Tanielan , Invisible Wounds of War . 2005; 3 Analysis of VA Health Care Ut

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

    © 2017 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 score ≥ 29 • Prim

  21. Slide 21: Results of Phase 2 AtEase Study in Military-Related PTSD

    © 2017 Tonix Pharmaceuticals Holding Corp. 21 Results of Phase 2 AtEase Study in Military - Related PTSD Tonmya 5.6 mg showed clinical benefit in military - related PTSD • CAPS - 5 scale, was statistically significant by Mixed effect Model Repeated Measures, or MMRM, with Multiple Imputation, or MI,

  22. Slide 22: AtEase Study Demographics and Characteristics

    © 2017 Tonix Pharmaceuticals Holding Corp. 22 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

  23. Slide 23: AtEase Study: Traumas Associated with PTSD

    © 2017 Tonix Pharmaceuticals Holding Corp. 23 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

  24. Slide 24: AtEase Study – Summary of Primary and Secondary Analyses (week 12)

    © 2017 Tonix Pharmaceuticals Holding Corp. 24 AtEase Study – Summary of Primary and Secondary Analyses (week 12)

  25. Slide 25: AtEase Study Results: Primary Endpoint

    © 2017 Tonix Pharmaceuticals Holding Corp. 25 AtEase Study Results: Primary Endpoint CAPS - 5 Total Score by MMRM with MI # -25 -20 -15 -10 -5 0 Wk 0 Wk 2 Wk 4 Wk 6 Wk 8 Wk 10 Wk 12 Placebo Tonmya 2.8 mg Tonmya 5.6 mg * # Primary analysis MMRM, * p=0.031 , comparing placebo and Tonmya 5.6 mg, * p<0.

  26. Slide 26: AtEase Study: Safety and Tolerability Profile

    © 2017 Tonix Pharmaceuticals Holding Corp. 26 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

  27. Slide 27: Assessing CAPS-5 Entry Threshold in AtEase

    © 2017 Tonix Pharmaceuticals Holding Corp. 27 Assessing CAPS - 5 Entry Threshold in AtEase • Score of ≥29 on CAPS - 5 (20 items) required at screening and baseline • >50 on prior versions of CAPS (17 items) typical in previous drug registration trials • Extrapolation from prior versions of CAPS: ((5

  28. Slide 28: AtEase Retrospective Analysis: Effect Sizes for Total CAPS-5 and Cluster Scores

    © 2017 Tonix Pharmaceuticals Holding Corp. 28 0.53 0.46 0.012 0.39 0.52 0 0.1 0.2 0.3 0.4 0.5 0.6 Effect Size AtEase Retrospective Analysis: Effect Sizes for Total CAPS - 5 and Cluster Scores * MMRM, mixed - effects model repeated measures, p<0.05 • Effect sizes calculated for total CAPS - 5 and clu

  29. Slide 29: AtEase Retrospective Analysis: Sustained Remission in CAPS-5 Baseline ≥33 Subgroup

    © 2017 Tonix Pharmaceuticals Holding Corp. 29 AtEase Study Retrospective Analysis: Sustained Remission in CAPS - 5 Baseline ≥33 Subgroup 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, 1

  30. Slide 30: Products in Development

    © 2017 Tonix Pharmaceuticals Holding Corp. 30 Products in Development Preclinical Phase 2 NDA/BLA 1 Market Pipeline Product Indication/ Description Phase 3 Tonmya Bedtime PTSD Treatment Daytime PTSD Treatment TNX - 601 Novel polymorph and salt discovered and characterized TNX - 801 Horsepox virus sy

  31. Slide 31: TNX-601 (Tianeptine Oxalate): A Potential Clinical Candidate for PTSD

    © 2017 Tonix Pharmaceuticals Holding Corp. 31 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 an

  32. Slide 32: Structural Comparison: TNX-102 and TNX-601

    © 2017 Tonix Pharmaceuticals Holding Corp. 32 Structural Comparison: TNX - 102 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 - chlorodiben

  33. Slide 33: TNX-601: A Potential Clinical Candidate for PTSD

    © 2017 Tonix Pharmaceuticals Holding Corp. 33 • Tianeptine modulates the glutamatergic system indirectly • Does not have significant affinity (K i >10 μ M) for NMDA or AMPA receptors • Tianeptine is a weak µ - opioid receptor (MOR) agonist • Controlled substance in France, Bahrain and Singapore • Ti

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

    © 2017 Tonix Pharmaceuticals Holding Corp. 34 TNX - 801 ( Synthesized Live Horsepox Virus ): A Potential Smallpox - Preventing Vaccine Pre - IND Candidate Potential improvement over current methods for biodefense against smallpox x Leverages government affairs effort x Collaboration with Professor D

  35. Slide 35: TNX-801 (Synthesized Live Horsepox Virus): A Potential Smallpox-Preventing Vaccine

    © 2017 Tonix Pharmaceuticals Holding Corp. 35 TNX - 801 ( Synthesized Live Horsepox Virus ): A Potential Smallpox - Preventing Vaccine Synthesis from sequence of a 1976 Mongolian isolate 1 In mice, TNX - 801 behaved like attenuated vaccinia virus (vaccinia virus is foundation of current smallpox vac

  36. Slide 36: Horsepox - Better Tolerability as a Vaccine?

    © 2017 Tonix Pharmaceuticals Holding Corp. 36 Horsepox - Better Tolerability as a Vaccine? Horsepox is caused by HPXV and is characterized by mouth and skin eruptions HXPV isolate from the 1976 outbreak later sequenced Modern vaccinia virus smallpox vaccines are associated with cardiotoxicity; may h

  37. Slide 37: A Better Smallpox-Preventing Vaccine is Important and Necessary Today

    © 2017 Tonix Pharmaceuticals Holding Corp. 37 A Better Smallpox - Preventing Vaccine is Important and Necessary Today Smallpox was eradicated as a result of global public health campaigns No cases of naturally - occurring smallpox have been reported since 1977 Accidental or intentional transmission

  38. Slide 38: TNX-801: A Potential Medical Countermeasure

    © 2017 Tonix Pharmaceuticals Holding Corp. 38 TNX - 801: A Potential Medical Countermeasure 21st Century Cures Act (2016), Section 3086 • Encouraging treatments for agents that present a national security threat Medical countermeasures are drugs or vaccines intended to treat: • Biological, chemical,

  39. Slide 39: TNX-801 Mechanism of Action and Important Characteristics

    © 2017 Tonix Pharmaceuticals Holding Corp. 39 Live virus vaccines stimulate cross - reactive immunity • Protects from consequences of infection with smallpox agent • Renders recipient “immune” • Provides additional protection of non - immunized population Potential safety advantage over existing vac

  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 – Recently Completed and Upcoming

    © 2017 Tonix Pharmaceuticals Holding Corp. 42 Milestones – Recently Completed and Upcoming T onmya – Posttraumatic Stress Disorder □ May 2016 Report ed results from Phase 2 AtEase study □ August 2016 End - of - Phase 2 meeting with FDA - Proposed Phase 3 clinical and NDA plan accepted □ December 201

  43. Slide 43: Summary

    © 2017 Tonix Pharmaceuticals Holding Corp. 43 Summary Strong position for value growth with Phase 3 development in a major medical indication: PTSD including military - related PTSD • PTSD is an important public health issue Tonmya for PTSD is designated as a Breakthrough Therapy by FDA • Accelerate

  44. Slide 44: Thank you!

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

Tonix Pharmaceuticals Holding — Investor Presentation (July 2017) on 6ix