Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP

Tonix Pharmaceuticals — Investor Presentation (April 2018)

40 slides from Tonix Pharmaceuticals's investor presentation, Apr 3, 2018

  1. Slide 1: Investor Presentation

    © 2018 Tonix Pharmaceuticals Holding Corp. April 2018 Version P0107 4 - 3 - 18 (Doc 0335) 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 Development Highlights

    © 2018 Tonix Pharmaceuticals Holding Corp. 3 Lead Program Tonmya ®1 - FDA Breakthrough Therapy in PTSD 2 • Phase 3 HONOR study enrolling - bedtime treatment for PTSD TNX - 102 SL 3 - Bedtime treatment for agitation in Alzheimer’s disease • Phase 2 IND submitted in March 2018 TNX - 601 4 - Pre - IND

  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: Tonmya for PTSD

    © 2018 Tonix Pharmaceuticals Holding Corp. 5 Tonmya for PTSD Phase 3 HONOR study in military - related PTSD enrolling • Encouraging evidence of safety and efficacy was demonstrated in Phase 2 • Higher entry CAPS - 5 criterion used in Phase 3 1 Breakthrough Therapy designation from the FDA • Expedite

  6. Slide 6: Phase 3 HONOR Study Enrolling

    © 2018 Tonix Pharmaceuticals Holding Corp. 6 Phase 3 HONOR Study Enrolling • General s tudy c haracteristics: • Randomized, double - blind, placebo - controlled . • ~ 550 participants in approximately 40 U.S. sites . • Unblinded interim analysis will be reviewed by IDMC* to determine: ( i ) Early st

  7. Slide 7: Go to: thehonorstudy.com

    © 2018 Tonix Pharmaceuticals Holding Corp. 7 Go to: https://thehonorstudy.com/

  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) • U.S. Patent No. 9,636,408 issued in May 2017 by U.S. Patent and Trademark Office (USPTO) • Protection expected to 2034 • Japan Patent Office (JPO) issued N

  12. Slide 12: Sublingual Formulation is 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: 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: What are the Consequences of PTSD?

    © 2018 Tonix Pharmaceuticals Holding Corp. 14 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

  15. Slide 15: What are the Symptoms of PTSD?

    © 2018 Tonix Pharmaceuticals Holding Corp. 15 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

  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, have not shown efficacy in military - related PTSD Majority of patients unresponsive or intolerant to current treatments • Side effects relating to sexual dys

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

    © 2018 Tonix Pharmaceuticals Holding Corp. 17 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

  18. Slide 18: Phase 2 AtEase Study in Military-Related PTSD

    © 2018 Tonix Pharmaceuticals Holding Corp. 18 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 Ef

  19. Slide 19: AtEase Study: Traumas Associated with PTSD

    © 2018 Tonix Pharmaceuticals Holding Corp. 19 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

  20. Slide 20: AtEase Study – Summary of Primary and Secondary Analyses

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

  21. Slide 21: AtEase Study: Safety and Tolerability Profile

    © 2018 Tonix Pharmaceuticals Holding Corp. 21 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

  22. Slide 22: AtEase Total CAPS-5 Retrospective Analysis Subgroup

    © 2018 Tonix Pharmaceuticals Holding Corp. 22 AtEase 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 Popul

  23. Slide 23: AtEase Effect Sizes for Total CAPS-5 and Symptom Clusters

    © 2018 Tonix Pharmaceuticals Holding Corp. 23 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 Effect Sizes for Total CAPS - 5 and Symptom Clusters for Intention - to - Treat Population and Subgroup with Entry CAPS - 5 ≥33 Effect Sizes in Tonmya 5.6 mg in AtEase Subgroup with En

  24. Slide 24: AtEase Retrospective Analysis: Remission in Subgroup

    © 2018 Tonix Pharmaceuticals Holding Corp. 24 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

  25. Slide 25: TNX-102 SL – Multiple Potential Indications

    © 2018 Tonix Pharmaceuticals Holding Corp. 25 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

  26. Slide 26: Consequences of Agitation in Alzheimer's Disease

    © 2018 Tonix Pharmaceuticals Holding Corp. 26 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

  27. Slide 27: Agitation in Alzheimer's Disease – Potential New Indication

    © 2018 Tonix Pharmaceuticals Holding Corp. 27 Agitation in Alzheimer’s Disease – Potential New Indication for TNX - 102 SL Successful pre - IND meeting in November, 2017 • Phase 2 IND submitted March 2018 Significant unmet need • No FDA approved drugs for the treatment of agitation in Alzheimer’s Me

  28. Slide 28: Scientific Rationale for TNX-102 SL in Alzheimer's Agitation

    © 2018 Tonix Pharmaceuticals Holding Corp. 28 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

  29. Slide 29: Candidates in Development

    © 2018 Tonix Pharmaceuticals Holding Corp. 29 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

  30. Slide 30: TNX-601 (Tianeptine Oxalate): Potential Clinical Candidate for PTSD

    © 2018 Tonix Pharmaceuticals Holding Corp. 30 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

  31. Slide 31: TNX-801: Smallpox-Preventing Vaccine Candidate

    © 2018 Tonix Pharmaceuticals Holding Corp. 31 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

  32. Slide 32: TNX-801: Synthesis and Relation to Modern Vaccines

    © 2018 Tonix Pharmaceuticals Holding Corp. 32 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

  33. Slide 33: Biological Properties of HPXV: Protective Immunity in Mice

    33 © 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):

  34. Slide 34: Current Needs to Vaccinate Against Smallpox

    © 2018 Tonix Pharmaceuticals Holding Corp. 34 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

  35. Slide 35: Potential for Use of HPXV as a Vector for Vaccines

    © 2018 Tonix Pharmaceuticals Holding Corp. 35 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

  36. Slide 36: Management Team

    © 2018 Tonix Pharmaceuticals Holding Corp. 36 Management Team Seth Lederman, MD President & CEO Jessica Morris Chief Operating Officer Gregory Sullivan, MD Chief Medical Officer Bradley Saenger, CPA Chief Financial Officer

  37. Slide 37: Board of Directors

    © 2018 Tonix Pharmaceuticals Holding Corp. 37 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

  38. Slide 38: Milestones – Recently Completed and Upcoming

    © 2018 Tonix Pharmaceuticals Holding Corp. 38 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

  39. Slide 39: Summary

    © 2018 Tonix Pharmaceuticals Holding Corp. 39 Summary Near term milestones in Phase 3 program PTSD focused on military - related PTSD • Major unmet need; 8.6 Million Americans affected • Potential for NDA approval based on one study New indication for agitation in Alzheimer’s Disease • Phase 2 IND s

  40. Slide 40: Thank you!

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

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