Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP

Tonix Pharmaceuticals — Investor Presentation (June 2019)

47 slides from Tonix Pharmaceuticals's investor presentation, Jun 3, 2019

  1. Slide 1: Investor Presentation June 2019

    © 2019 Tonix Pharmaceuticals Holding Corp. 1 June 2019 Version P0183 6 - 3 - 19 (Doc 0494) 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 Novel salt discovered and c

  5. Slide 5: Biodefense Candidates in Development

    © 2019 Tonix Pharmaceuticals Holding Corp. 5 Biodefense Candidates in Development 1 NDA - New Drug Application; 2 BLA – Biologic Licensing Application Biodefense programs owned outright with no royalties due Preclinical Phase 2 NDA 1 /BLA 2 Market Pipeline Product Indication Phase 3 TNX - 801 Horsep

  6. Slide 6: Lead Program: TNX-102 SL Product Concept

    © 2019 Tonix Pharmaceuticals Holding Corp. 6 Lead Program: TNX - 102 SL – Product Concept Sleep disturbances are associated with a constellation of disorders • Considered co - morbid or a key symptom in these disorders • Believed to have a role in the onset, progression and severity of these disorde

  7. Slide 7: Improving sleep quality across a constellation of disorders

    © 2019 Tonix Pharmaceuticals Holding Corp. 7 A unique and innovative approach (improving sleep quality), to potentially address a constellation of disorders in a variety of therapeutic areas where sleep disturbances are co - morbid and believed to have a role in the onset, progression and severity o

  8. Slide 8: TNX-102 SL Intellectual Property: U.S. Protection until 2034

    © 2019 Tonix Pharmaceuticals Holding Corp. 8 TNX - 102 SL Intellectual Property – U.S. Protection until 2034 • United States Patent and Trademark Office (USPTO) issued U.S. Patent No. 9,636,408 in May 2017, U.S. Patent No. 9,956,188 in May 2018 and U.S. Patent No. 10,117,936 in Nov 2018 • Japanese P

  9. Slide 9: Prevalence of PTSD Among Civilians and Veterans

    © 2019 Tonix Pharmaceuticals Holding Corp. 9 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

  10. Slide 10: PTSD Prevalence and Market Characteristics

    © 2019 Tonix Pharmaceuticals Holding Corp. 10 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)

  11. Slide 11: Unmet Need for Therapies for Military PTSD

    © 2019 Tonix Pharmaceuticals Holding Corp. 11 Unmet Need for Effective and Safe Therapies for Treatment of Military PTSD PTSD is signature wound of last 25 years of war • Affects servicemember health and performance, force readiness, retention • Believed to be the underlying cause of suicide in many

  12. Slide 12: Tonmya: A Potential Bedtime Treatment for PTSD

    © 2019 Tonix Pharmaceuticals Holding Corp. 12 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

  13. Slide 13: Potential Therapeutic Advantages of Tonmya

    © 2019 Tonix Pharmaceuticals Holding Corp. 13 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

  14. Slide 14: Tonmya Phase 2 Dose-Effect in Military-Related PTSD

    © 2019 Tonix Pharmaceuticals Holding Corp. 14 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

  15. Slide 15: Primary Outcome (CAPS-5) in Phase 3 mITT and Subgroups

    © 2019 Tonix Pharmaceuticals Holding Corp. 15 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

  16. Slide 16: Retrospective Comparison of Time Since Trauma in P201 vs P301

    © 2019 Tonix Pharmaceuticals Holding Corp. 16 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

  17. Slide 17: Sustained Remission in Phase 2 and Phase 3 Studies

    © 2019 Tonix Pharmaceuticals Holding Corp. 17 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

  18. Slide 18: Adverse Events in P201/AtEase and P301/HONOR Studies

    © 2019 Tonix Pharmaceuticals Holding Corp. 18 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

  19. Slide 19: Response to Tonmya for Female Participants in P301/HONOR

    © 2019 Tonix Pharmaceuticals Holding Corp. 19 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

  20. Slide 20: Response to Tonmya for Non-Combat Traumas

    © 2019 Tonix Pharmaceuticals Holding Corp. 20 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

  21. Slide 21: New Phase 3 P302/RECOVERY Study Initiated 1Q 2019

    © 2019 Tonix Pharmaceuticals Holding Corp. 21 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

  22. Slide 22: Late-Stage PTSD Drug Candidates

    © 2019 Tonix Pharmaceuticals Holding Corp. 22 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

  23. Slide 23: Opportunities to Expand to Other Indications

    © 2019 Tonix Pharmaceuticals Holding Corp. 23 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

  24. Slide 24: Fibromyalgia: Pain Alarm Overview

    © 2019 Tonix Pharmaceuticals Holding Corp. 24 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

  25. Slide 25: Fibromyalgia: Market Characteristics

    © 2019 Tonix Pharmaceuticals Holding Corp. 25 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 chr

  26. Slide 26: Fewer than Half of Treated Fibromyalgia Patients Receive Sustained Benefit

    © 2019 Tonix Pharmaceuticals Holding Corp. 26 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

  27. Slide 27: Large Need for New Fibromyalgia Therapies

    © 2019 Tonix Pharmaceuticals Holding Corp. 27 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

  28. Slide 28: TNX-102 SL for Fibromyalgia: Phase 3 F301 Study Summary

    © 2019 Tonix Pharmaceuticals Holding Corp. 28 TNX - 102 SL for Fibromyalgia: Summary of a 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 d

  29. Slide 29: TNX-102 SL for Fibromyalgia: F301 Results and Program Updates

    © 2019 Tonix Pharmaceuticals Holding Corp. 29 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

  30. Slide 30: TNX-102 SL: Potential Treatment for Agitation in Alzheimer's Disease

    © 2019 Tonix Pharmaceuticals Holding Corp. 30 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

  31. Slide 31: New Addition to Pipeline: TNX-1300 for Cocaine Intoxication

    © 2019 Tonix Pharmaceuticals Holding Corp. 31 New Addition to Tonix’s Pipeline: TNX - 1300* for the Treatment of Cocaine Intoxication Recombinant protein that degrades cocaine in the bloodstream 1 • Double - mutant cocaine esterase In - licensed from Columbia University (Univ. of Kentucky and Univ.

  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 Action

    © 2019 Tonix Pharmaceuticals Holding Corp. 34 Cocaine Action • Cocaine acts to inhibit the Dopamine Reuptake Transporter • Binds to transporter and blocks reuptake of dopamine from synapse • No antagonist has been developed for cocaine • Unlike situation for opiates which can be blocked by naloxone

  35. Slide 35: Cocaine Action: Lack of Antidote

    © 2019 Tonix Pharmaceuticals Holding Corp. 35 Cocaine Action – Lack of “Antidote” • Cocaine binding site on Dopamine Reuptake Transporter is too small or simple for a blocker • Antagonists of cocaine binding have the same function as cocaine • Naltrexone exploits the complexity of the opiate binding

  36. Slide 36: CocE Breaks Cocaine Down into Less Toxic Metabolites

    © 2019 Tonix Pharmaceuticals Holding Corp. 36 CocE is a Catalyst that Breaks Cocaine Down into Less Toxic Metabolites 1 - 4 Cocaine Ecgonine Methyl Ester Benzoic Acid • Metabolites ecgonine methyl ester and benzoic acid are neither pleasurable nor addictive 1 Bresler MM et al, Appl Environ Microbiol

  37. Slide 37: Prevalence of Cocaine Usage and Overdose

    © 2019 Tonix Pharmaceuticals Holding Corp. 37 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

  38. Slide 38: Mortality Due to Cocaine Overdose

    © 2019 Tonix Pharmaceuticals Holding Corp. 38 Mortality Due to Cocaine Overdose Cocaine is involved in 20% of overdose deaths in the U.S. • In 2016, 10,375 deaths due to cocaine overdose 1 • In 2017, about 13,900 deaths occurred in the U.S. due to cocaine overdose. 2 Overdose deaths involving cocain

  39. Slide 39: Treatment for Cocaine Intoxication

    © 2019 Tonix Pharmaceuticals Holding Corp. 39 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

  40. Slide 40: Value of TNX-1300 to Tonix

    © 2019 Tonix Pharmaceuticals Holding Corp. 40 Value of TNX - 1300 to Tonix Features of the Acquired Asset: • Full rights to the IP and to develop and commercialize TNX - 1300 worldwide • FDA Breakthrough Therapy Designated product • An inventory of investigational drug product • Clinical trial resul

  41. Slide 41: TNX-601 (Tianeptine Oxalate): Potential Clinical Candidate for PTSD

    © 2019 Tonix Pharmaceuticals Holding Corp. 41 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

  42. Slide 42: TNX-801 (Synthesized Live Horsepox Virus): Smallpox Vaccine Candidate

    © 2019 Tonix Pharmaceuticals Holding Corp. 42 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

  43. Slide 43: Management Team

    © 2019 Tonix Pharmaceuticals Holding Corp. 43 Management Team Seth Lederman, MD President & CEO Jessica Morris Chief Operating Officer Gregory Sullivan, MD Chief Medical Officer Bradley Saenger, CPA Chief Financial Officer

  44. Slide 44: Board of Directors

    © 2019 Tonix Pharmaceuticals Holding Corp. 44 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

  45. Slide 45: Milestones: Recently Completed and Upcoming

    © 2019 Tonix Pharmaceuticals Holding Corp. 45 Milestones – Recently Completed and Upcoming □ July 201 8 Completed P301/HONOR study interim analysis - result did not support study continuation but strengthened new Phase 3 study □ August 201 8 Presentation of P301/HONOR study results at Military Healt

  46. Slide 46: Summary

    © 2019 Tonix Pharmaceuticals Holding Corp. 46 Summary Two Phase 3 Programs in indications affecting millions of Americans • Tonmya for PTSD: affects 12 million adults in U.S.; currently in Phase 3 with data expected next year; bedtime treatment • TNX - 102 SL for Fibromyalgia: affects between 5 - 10

  47. Slide 47: Thank you!

    © 2019 Tonix Pharmaceuticals Holding Corp. 47 Thank you ! NASDAQ: TNXP

Tonix Pharmaceuticals Holding — Investor Presentation (June 2019) on 6ix