Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP

Tonix Pharmaceuticals — Investor Presentation (August 2019)

49 slides from Tonix Pharmaceuticals's investor presentation, Aug 21, 2019

  1. Slide 1: Investor Presentation August 2019

    © 2019 Tonix Pharmaceuticals Holding Corp. 1 August 2019 Version P0192 8 - 21 - 19 (Doc 0520) Investor Presentation

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

    © 2019 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: Who we are, what we do

    © 2019 Tonix Pharmaceuticals Holding Corp. 3 Tonix Pharmaceuticals Who we are: • A clinical stage biopharmaceutical company dedicated to developing innovative treatments for patients and making meaningful contributions to society • Focusing on small molecules and biologics to treat psychiatric, pain

  4. Slide 4: CNS Candidates in Development

    © 2019 Tonix Pharmaceuticals Holding Corp. 4 CNS Candidates in Development Psychiatry, Pain and Addiction Preclinical Phase 2 NDA 3 /BLA 4 Market Pipeline Product Indication Phase 3 TNX - 102 SL 1 Bedtime Treatment for PTSD – Tonmya ®2 Daytime Treatment for PTSD TNX - 601 TNX - 1300 6 Cocaine intoxi

  5. Slide 5: Preclinical Pipeline

    © 2019 Tonix Pharmaceuticals Holding Corp. 5 Preclinical Pipeline 1 1 Programs owned outright with no royalties due Category Pipeline Product Indication TNX - 801 Smallpox - preventing vaccine Live horsepox virus (HPXV) vaccine from cell culture percutaneous (scarification) formulation TNX - 701 Pro

  6. Slide 6: The focus of TNX-102 SL development is unique and innovative

    © 2019 Tonix Pharmaceuticals Holding Corp. 6 The focus of TNX - 102 SL development is both unique and innovative • Testing the therapeutic benefit of sleep (‘sleep quality’) − Restorative sleep, in contrast to time spent sleeping (‘sleep quantity’) • Targeting clinical conditions for which improved

  7. Slide 7: TNX-102 SL Intellectual Property

    © 2019 Tonix Pharmaceuticals Holding Corp. 7 TNX - 102 SL Intellectual Property – U.S. Protection expected until 2035 • United States Patent and Trademark Office (USPTO) issued U.S. Patent No. 9636408 in May 2017, U.S. Patent No. 9956188 in May 2018, U.S. Patent No. 10117936 in Nov 2018, and U.S. Pa

  8. Slide 8: Prevalence of PTSD Among Civilians and Veterans

    © 2019 Tonix Pharmaceuticals Holding Corp. 8 Prevalence of PTSD Among Civilians and Veterans 1 Goldstein et al., 2016 (adjusted for 2019) ; 2 Norris, PTSD Res Quar. 2013; 3 Analysis of VA Health Care Utilization among Operation Enduring Freedom, Operation Iraqi Freedom, and Operation New Dawn Vete r

  9. Slide 9: PTSD Prevalence and Market Characteristics

    © 2019 Tonix Pharmaceuticals Holding Corp. 9 PTSD Prevalence and Market Characteristics 1 Goldstein et al., 2016 (adjusted for 2019) 2 Veterans: VA/DOD Clinical Practice Guidelines for the Managements of PTSD and Acute Stress Disorder, 2017, page 15 (619,493 vets diagnosed with PTSD in VA for 2016)

  10. Slide 10: Unmet Need for Effective and Safe Therapies for PTSD

    © 2019 Tonix Pharmaceuticals Holding Corp. 10 Unmet Need for Effective and Safe Therapies for Treatment of PTSD No FDA - approved products for PTSD since Pfizer’s Zoloft ® (sertraline) and GSK’s Paxil ® (paroxetine) circa 2000 • Neither has shown efficacy in military - related PTSD • Male PTSD patie

  11. Slide 11: Tonmya: a Potential Bedtime Treatment for PTSD

    © 2019 Tonix Pharmaceuticals Holding Corp. 11 Tonmya : a Potential Bedtime Treatment for PTSD First investigational new drug to show treatment effect in military - related PTSD in two potential pivotal efficacy studies • Phase 2 study (P201/ AtEase ) showed Tonmya 5.6 mg had a strong signal of treat

  12. Slide 12: Potential Therapeutic Advantages of Tonmya

    © 2019 Tonix Pharmaceuticals Holding Corp. 12 Potential Therapeutic Advantages of Tonmya Tonmya is believed to treat PTSD by improving sleep quality • The brain naturally processes memories during sleep • PTSD sufferers’ emotionally charged memories disturb sleep and disrupt the natural processing o

  13. Slide 13: Tonmya Phase 2 Dose-Effect in Military-Related PTSD

    © 2019 Tonix Pharmaceuticals Holding Corp. 13 Tonmya Phase 2 Dose - Effect in Military - Related PTSD 1 1 Completed Phase 2 P201/AtEase study: Retrospective analysis of Tonmya 5.6 mg on CAPS - 5 ≥33 (high - moderate) subgroup. Primary analysis of P 201/ AtEase , based on Tonmya 2.8 mg in participant

  14. Slide 14: Primary Outcome (CAPS-5) in Phase 3 and Time Since Trauma Subgroups

    © 2019 Tonix Pharmaceuticals Holding Corp. 14 Primary Outcome (CAPS - 5) in Phase 3 ( mITT ) and ≤9 Years Time Since Trauma Subgroups Modified intent to treat ( mITT ) population Phase 3 P301/HONOR Study 1 ** p=0.004, * p=0.039, # p=0.069, TNX - 102 SL 5.6 mg group v. placebo, using MMRM with MI ~50

  15. Slide 15: Retrospective Comparison of Time Since Trauma in P201 versus P301

    © 2019 Tonix Pharmaceuticals Holding Corp. 15 Retrospective Comparison of Time Since Trauma in P201/AtEase versus P301/HONOR (Tonmya 5.6 mg Groups) P301 study was initiated approximately two years later than Phase 2 P201 • The median time since trauma in P301 was 9.5 years compared to the median tim

  16. Slide 16: Sustained Remission in Phase 2 and Phase 3 Studies

    © 2019 Tonix Pharmaceuticals Holding Corp. 16 Sustained Remission in Phase 2 and Phase 3 Studies: Retrospective Analyses of P201 Entry CAPS - 5 ≥33 and P301 ≤9 Years Since Trauma Subgroups Remission is a clinical state that is essentially asymptomatic In order to confirm remission: • Determined rate

  17. Slide 17: Adverse Events in P201/AtEase and P301/HONOR Studies

    © 2019 Tonix Pharmaceuticals Holding Corp. 17 Adverse Events (AEs) in P201/AtEase and P301/HONOR Studies No serious or unexpected AEs in P201 or P301 related to Tonmya • Systemic AEs comparable between studies and also consistent with those described in approved oral cyclobenzaprine product labeling

  18. Slide 18: Response to Tonmya for Female Participants in P301/HONOR

    © 2019 Tonix Pharmaceuticals Holding Corp. 18 Response to Tonmya for Female Participants in P301/HONOR Study 1 Females made up only 11% of the P301/HONOR study mITT population Difference in mean change from baseline in CAPS - 5 in females between placebo (N=17) and Tonmya 5.6 mg (N=10) was: • At 4 w

  19. Slide 19: Response to Tonmya for Non-Combat Traumas in P301/HONOR

    © 2019 Tonix Pharmaceuticals Holding Corp. 19 Non - combat traumas studied are similar to traumas experienced in civilian populations with PTSD To determine the therapeutic effects of Tonmya 5.6 mg in a mixed civilian and military population, difference in MCFB in CAPS - 5 was assessed in non - comb

  20. Slide 20: New Phase 3 P302/RECOVERY Study

    © 2019 Tonix Pharmaceuticals Holding Corp. 20 New Phase 3 P302/RECOVERY Study – Initiated 1Q 2019 Primary e ndpoint: • CAPS - 5 1 mean change from baseline at Week 4 ( Tonmya 5.6 mg vs. placebo) Key Secondary e ndpoint s include: • CAPS - 5 m ean change from baseline at W eek 12 (Tonmya 5.6 mg vs. p

  21. Slide 21: Late-Stage PTSD Drug Candidates

    © 2019 Tonix Pharmaceuticals Holding Corp. 21 Late - Stage PTSD Drug Candidates Tonmya • Phase 3 development focused on military - related and civilian PTSD; showed activity in treatment of military - related PTSD in large multi - center trials MDMA - assisted psychotherapy • Indication – “drug assi

  22. Slide 22: Opportunities to Expand to Other Indications

    © 2019 Tonix Pharmaceuticals Holding Corp. 22 Opportunities to Expand to Other Indications Growing recognition that there are many disorders where sleep disturbances may have a role in the pathophysiology (cardiovascular, metabolic, neurologic) • Homeostatic role of sleep quality in several disorder

  23. Slide 23: Fibromyalgia as a Neurobiological Disorder

    © 2019 Tonix Pharmaceuticals Holding Corp. 23 Volkswagen Check Engine [Photograph]. (2011, October 14). Wikipedia • Pain is a sensor system in the brain similar to a check engine light on a car’s dashboard • When the check engine light malfunctions, the light is on even though the car is not malfunc

  24. Slide 24: Fibromyalgia: Market Characteristics

    © 2019 Tonix Pharmaceuticals Holding Corp. 24 Fibromyalgia: Market Characteristics Undiagnosed ~2.7 million Drug Treated 85% 3 2,366,400 No Drug 15% 3 353,600 U.S. Prevalence Rate 2 - 4% 1 (~5 - 10 million adults) Diagnosed 2 ~2.7 million Market Characteristics Prevalence • One of the more common ch

  25. Slide 25: Fewer than Half Receive Sustained Benefit from FDA-Approved Drugs

    © 2019 Tonix Pharmaceuticals Holding Corp. 25 Fewer than Half of Those Treated for Fibromyalgia Receive Sustained Benefit from the Three FDA - Approved Drugs 1 • The treatment objective is to restore functionality and quality of life by broadly improving symptoms while avoiding significant side effe

  26. Slide 26: Large Need for New Fibromyalgia Therapies

    © 2019 Tonix Pharmaceuticals Holding Corp. 26 Large Need for New Fibromyalgia Therapies that Provide Broad Symptom Improvement with Better Tolerability • Currently - approved medications may have side effects that limit long - term use 1 • Many patients skip doses or discontinue altogether within mo

  27. Slide 27: TNX-102 SL for Fibromyalgia: Phase 3 F301 Study Summary

    © 2019 Tonix Pharmaceuticals Holding Corp. 27 TNX - 102 SL for Fibromyalgia: Summary of completed Phase 3 F301 study General s tudy c haracteristics: • Randomized, 12 - week, double - blind, placebo - controlle d Phase 3 study of TNX - 102 SL 2.8 mg (half the dose being developed for PTSD) taken dai

  28. Slide 28: TNX-102 SL for Fibromyalgia: F301 Results and Program Updates

    © 2019 Tonix Pharmaceuticals Holding Corp. 28 TNX - 102 SL for Fibromyalgia: F301 Study Results and Program Updates Safety results: • Good tolerability and low rates of systemic AEs. • The most common AEs were generally mild and transient events related to the sublingual administration of the study

  29. Slide 29: TNX-102 SL: Potential Treatment for Agitation in Alzheimer's Disease

    © 2019 Tonix Pharmaceuticals Holding Corp. 29 TNX - 102 SL: Potential Treatment for Agitation in Alzheimer’s Disease (AAD) Agitation is one of the most distressing and debilitating of the behavioral complications of Alzheimer’s disease • Includes emotional lability, restlessness, irritability and ag

  30. Slide 30: TNX-102 SL: Potential Treatment for Alcohol Use Disorder

    © 2019 Tonix Pharmaceuticals Holding Corp. 30 TNX - 102 SL: Potential Treatment for Alcohol Use Disorder (AUD) AUD is a chronic relapsing brain disease • Characterized by compulsive alcohol use, loss of control over alcohol intake, and a negative emotional state when not using Sleep disturbance is e

  31. Slide 31: TNX-1300 for the Treatment of Cocaine Intoxication

    © 2019 Tonix Pharmaceuticals Holding Corp. 31 TNX - 1300* for the Treatment of Cocaine Intoxication Recombinant protein that degrades cocaine in the bloodstream 1 • Double - mutant cocaine esterase Phase 2 study completed by Rickett Benckiser (TNX - 1300 was formerly RBP - 8000) 2 • Volunteer cocain

  32. Slide 32: About TNX-1300

    © 2019 Tonix Pharmaceuticals Holding Corp. 32 About TNX - 1300 Produced through rDNA technology in non - disease - producing strain of E. coli. • Cocaine Esterase ( CocE ) was identified in bacteria ( Rhodococcus ) that use cocaine as its sole source of carbon and nitrogen and that grow in soil surr

  33. Slide 33: About Cocaine and Cocaine Intoxication

    © 2019 Tonix Pharmaceuticals Holding Corp. 33 About Cocaine and Cocaine Intoxication Cocaine: an illegal recreational drug taken for its pleasurable effects and associated euphoria. • Cocaine blocks the reuptake of the neurotransmitter dopamine (DA) in the CNS • Results in accumulation of DA within

  34. Slide 34: Cocaine Intoxication Is the Result of Activity at Multiple Targets

    Cocaine Intoxication Is the Result of Cocaine’s Activity at Multiple Targets 1. Brim et al. Mol Pharmacol . 2011. Psychosis Seizures Arrythmia Hypertension Hyperthermia The effects of cocaine intoxication include 1 : In addition to dopamine transporters, cocaine also blocks other targets 1 Dopamine

  35. Slide 35: Pharmacotherapies for Cocaine Intoxication Have Not Been Effective

    © 2019 Tonix Pharmaceuticals Holding Corp. 35 Pharmacotherapies for Cocaine Intoxication Have Not Been Effective 1. Brim et al. Mol Pharmacol . 2011. 2. Melichar et al. Eur J Phamacol . 2003. 3. Narasimhan et al. Future Medicinal Chemistry. 2012. Opiates Simple; mediated by opioid receptors 2 Agonis

  36. Slide 36: TNX-1300 Is a Fast-acting Cocaine Antidote

    © 2019 Tonix Pharmaceuticals Holding Corp. 36 TNX - 1300 (Cocaine Esterase or CocE ) Is a Fast - acting Cocaine Antidote CocE Rhodococcus bacteria living in the roots of the coca plant use CocE to metabolize cocaine 1 CocE cleaves chemical bonds in cocaine and disintegrates it 800 times faster than

  37. Slide 37: TNX-1300 cleaves cocaine in humans and removes it from the blood

    © 2019 Tonix Pharmaceuticals Holding Corp. 37 TNX - 1300 cleaves cocaine in humans and removes it from the blood circulation 1 (N=29) Time Required to Return to Baseline (minutes) 120 0 240 Pulse Rate Diastolic Blood Pressure Systolic Blood Pressure 60 180 Time (minutes) 120 240 0 Cocaine Plasma Con

  38. Slide 38: The Prevalence of Cocaine Usage and Overdose

    © 2019 Tonix Pharmaceuticals Holding Corp. 38 The Prevalence of Cocaine Usage and Overdose (U.S.) Cocaine Usage in the U.S. 5.07 million individuals estimated to have used cocaine in past year 1 • 2.2 million “current” (i.e. users in the past month) of cocaine (2017) 2 • 966,000 had cocaine use diso

  39. Slide 39: Cocaine Intoxication Is a Growing Problem in the U.S.

    Cocaine Intoxication Is a Growing Problem in the U.S. National Drug Overdose Deaths Involving Cocaine 0 5000 10000 15000 Cocaine is involved in more emergency department (ED) visits than any other illicit substance 1 Drug overdose deaths involving cocaine have increased dramatically in recent years

  40. Slide 40: Treatment for Cocaine Intoxication

    © 2019 Tonix Pharmaceuticals Holding Corp. 40 Treatment for Cocaine Intoxication Current Standard of Care • Patients present with acute agitation, hyperthermia, tachycardia, arrhythmias, and hypertension • Potential life - threatening sequalae of myocardial infarction, cerebrovascular accident, rhab

  41. Slide 41: Value of TNX-1300 to Tonix

    © 2019 Tonix Pharmaceuticals Holding Corp. 41 Value of TNX - 1300 to Tonix Features of the Acquired Asset: • Full rights to the IP and to develop and commercialize TNX - 1300 worldwide • An inventory of investigational drug product • Clinical trial results from previous Phase 2 study in which TNX -

  42. Slide 42: TNX-601 (Tianeptine Oxalate): Potential Daytime Treatment for PTSD

    © 2019 Tonix Pharmaceuticals Holding Corp. 42 TNX - 601 (Tianeptine Oxalate): A Potential Daytime Treatment 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 analysi

  43. Slide 43: TNX-801: Smallpox-Preventing Vaccine Candidate

    © 2019 Tonix Pharmaceuticals Holding Corp. 43 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

  44. Slide 44: TNX-1500 (Anti-CD154 Monoclonal Antibody)

    © 2019 Tonix Pharmaceuticals Holding Corp. 44 T NX - 1500 (Anti - CD154 Monoclonal Antibody): Candidate to Prevent and Treat Organ Transplant Rejection and Treat Autoimmune Conditions Pre - IND Stage Third generation anti - CD154 mAB with potential improvement over first and second generations in te

  45. Slide 45: Management Team

    © 2019 Tonix Pharmaceuticals Holding Corp. 45 Management Team Seth Lederman, MD President & CEO Jessica Morris Chief Operating Officer Gregory Sullivan, MD Chief Medical Officer Bradley Saenger, CPA Chief Financial Officer

  46. Slide 46: Board of Directors

    © 2019 Tonix Pharmaceuticals Holding Corp. 46 Board of Directors Seth Lederman, MD Chairman Adeoye “Oye” Olukotun, MD Squibb, BMS, Mallinckrodt, Esperion John Rhodes Chair, NYS Public Service Commission, CEO, NYS Dept. of Public Service, Booz Allen James Treco First Chicago, Salomon Brothers/Citigro

  47. Slide 47: Milestones – Recently Completed and Upcoming

    © 2019 Tonix Pharmaceuticals Holding Corp. 47 Milestones – Recently Completed and Upcoming □ November 201 8 Received FDA minutes confirming agreement on the design of P302/RECOVERY study □ March 2019 Met with FDA to discuss new FM Phase 3 study design using TNX - 102 SL 5.6 mg □ March 2019 P302/RECO

  48. Slide 48: Summary

    © 2019 Tonix Pharmaceuticals Holding Corp. 48 Summary Two Phase 3 Programs in indications affecting millions of Americans • Tonmya for PTSD: affects 12 million adults in U.S.; currently conducting Phase 3 trial with data expected next year; bedtime treatment • TNX - 102 SL for Fibromyalgia: affects

  49. Slide 49: Thank you!

    © 2019 Tonix Pharmaceuticals Holding Corp. 49 Thank you ! NASDAQ: TNXP

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