Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP

Tonix Pharmaceuticals — Investor Presentation (March 2022)

60 slides from Tonix Pharmaceuticals's investor presentation, Mar 8, 2022

  1. Slide 1: Investor Presentation

    © 2022 Tonix Pharmaceuticals Holding Corp. INVESTOR PRESENTATION NASDAQ: TNXP Version P0338 March 8, 2022 (Doc 0963)

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

    2 © 2022 Tonix Pharmaceuticals Holding Corp. 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: Advancing the Science and Understanding of Diseases

    3 © 2022 Tonix Pharmaceuticals Holding Corp. ADVANCING THE SCIENCE AND UNDERSTANDING OF DISEASES by developing innovative therapies that improve population health by focusing on unmet needs in patient care WHAT WE DO Using our integrated development engine, we advance innovative programs across mult

  4. Slide 4: Pipeline Portfolio: BioDefense and COVID Candidates

    4 © 2022 Tonix Pharmaceuticals Holding Corp. *All of Tonix’s product candidates are investigational new drugs or biologics and have not been approved for any indication. 1 anti - CD40L humanized monoclonal antibody 2 Recombinant trefoil factor 2 (rTFF2) based protein; licensed from Columbia Universi

  5. Slide 5: CNS Portfolio Candidates

    5 © 2022 Tonix Pharmaceuticals Holding Corp. Candidates* INDICATIONS STATUS / NEXT MILESTONE CNS TNX - 1300 1 Cocaine Intoxication / Overdose FDA Breakthrough Designation Phase 2, Targeted 1H 2022 Start TNX - 102 SL 2 Fibromyalgia (FM) Posttraumatic Stress Disorder (PTSD) Long COVID (PASC 3 ) Mid -

  6. Slide 6: Immunology: Key Candidates

    © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY: KEY CANDIDATES

  7. Slide 7: TNX-1500: A Potential Treatment for Organ Transplant Rejection and Autoimmune Conditions

    7 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 1500 ( anti - CD40L m A b ): A POTENTIAL TREATMENT FOR ORGAN TRANSPLANT REJECTION AND AUTOIMMUNE CONDITIONS Pre - IND Candidate Significant Unmet Need Targeted as a first - line monotherapy for autoimmunity and add - on therapy for preventing and tr

  8. Slide 8: TNX-1500 Market Opportunity

    8 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 1500 MARKET OPPORTUNITY Autoimmune Disease $149.4 billion 6 OPPORTUNITY Kidney transplants: 24,000/year/US 2 $5.54 billion 3 Autoimmune Lupus: 1.5 M patients in US 4 1.87 billion 5 Organ transplant rejection drugs $4.7 billion 1 IMMUNOLOGY PORTFOLIO

  9. Slide 9: CD40L is a Transiently Expressed T Cell Surface Molecule

    9 © 2022 Tonix Pharmaceuticals Holding Corp. CD40L is a transiently expressed T cell surface molecule and is also called CD154 1 - 4 ‒ Predominantly expressed by T cells and interacts with CD40 on B cells and macrophages Mediates T cell helper function 1 - 4 ‒ Activates B cells for humoral (antibody

  10. Slide 10: Next Generation Anti-CD40L Antibody TNX-1500

    10 © 2022 Tonix Pharmaceuticals Holding Corp. NEXT GENERATION anti - CD40 LIGAND (CD40L) ANTIBODY TNX - 1500*: PREVENTION OF ALLOGRAFT REJECTION THE CD40 - CD40L PATHWAY IS A PIVOTAL IMMUNE SYSTEM MODULATOR AND IS A WELL - ESTABLISHED AND PROMISING TREATMENT TARGET TO MORE SAFELY PREVENT ALLOGRAFT R

  11. Slide 11: Third-Generation Anti-CD40L Engineered to Decrease Risk of Thrombosis

    11 © 2022 Tonix Pharmaceuticals Holding Corp. THIRD - GENERATION anti - CD40L ENGINEERED TO DECREASE RISK OF THROMBOSIS First - generation anti - CD40L mAbs Constant fragment (Fc) domain interacted with FcγRIIA (CD32A), which suggested a mechanism for the increased risk of thrombosis. 1,2 Ruplizumab

  12. Slide 12: Anti-CD40L Treatment to Prevent Allograft Rejection

    12 © 2022 Tonix Pharmaceuticals Holding Corp. anti - CD40L TREATMENT TO PREVENT ALLOGRAFT REJECTION • Allotransplantation is limited by a critical shortage of human organs • Costimulation blockade (anti - CD40L in particular) is more effective at protecting allografts than calcineurin inhibitors (CN

  13. Slide 13: Non-Human Primate Heart Heterotopic Allograft Study

    13 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 1500 monotherapy consistently (4/5 heart transplants) prevents heart transplant rejection 1 ‒ Graft acceptance without acute cellular injury 2 or chronic antibody injury 3 through day 180 ‒ Prolonged acceptance after cessation of therapy (in progre

  14. Slide 14: Non-Human Primate Kidney Allo-Transplantation Study

    14 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 1500 monotherapy consistently (5/6 kidney transplants) prevents kidney transplant rejection 1 ‒ Six recipients were treated with TNX - 1500 monotherapy 1 ‒ No rejection was observed in 5/6 recipients through day 180 ‒ Superior to results with conve

  15. Slide 15: Tolerance Induction with Donor Bone Marrow Transplantation

    15 © 2022 Tonix Pharmaceuticals Holding Corp. TOLERANCE INDUCTION WITH DONOR BONE MARROW TRANSPLANTATION Induction of “mixed chimerism” induces allograft tolerance ‒ Long - lasting, durable tolerance — specifically to donor tissues ‒ Initial protocols required that the recipient’s mature T cells be

  16. Slide 16: Non-Human Primate Combined Kidney and Bone Marrow Transplantation with TNX-1500

    16 © 2022 Tonix Pharmaceuticals Holding Corp. 0 20 40 60 80 100 5 12 20 27 33 40 47 % Chimerism Days Post CKBMT Chimerism Gran Mono Lymph Keys: 1. Bone marrow transplant 2. Kidney transplant 3. Total Body Irradition 4. Venclexta ® 5. Thymoglobulin® NON - HUMAN PRIMATE COMBINED KIDNEY AND BONE MARROW

  17. Slide 17: Anti-CD40L Beyond Allografts: Xenografts

    17 © 2022 Tonix Pharmaceuticals Holding Corp. anti - CD40L BEYOND ALLOGRAFTS: XENOGRAFTS • Allotransplantation is limited by a critical shortage of human organs ; pig - to - human xenotransplantation offers a promising alternative 1,2 • Costimulation blockade (anti - CD40L in particular) is more eff

  18. Slide 18: Recent Xenotransplant Headlines

    18 © 2022 Tonix Pharmaceuticals Holding Corp. “In a First, Surgeons Attached a Pig Kidney to a Human, and It Worked” Roni Caryn Rabin “Pig - Heart Transplant Jolts Doctors Confronting Lack of Organ Donors” Amy Dockser Marcus “Saved by a Pig’s Heart” The Editorial Board “Pig Kidneys Transplanted Into

  19. Slide 19: Anti-CD40L Beyond Allografts: Autoimmunity

    19 © 2022 Tonix Pharmaceuticals Holding Corp. anti - CD40L BEYOND ALLOGRAFTS: AUTOIMMUNITY • Autoimmune diseases are also characterized by immune system activity that attacks “self,” which can damage various parts of the body 1,2 • First - generation anti - CD40L Abs showed evidence of efficacy in a

  20. Slide 20: TNX-1500: Key Considerations

    20 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 1500: KEY CONSIDERATIONS • TNX - 1500 may be used in large markets that are not currently well served • There is a long history of use of monoclonal antibodies • Tonix has engineered a safer, potentially more efficacious molecule than previous anti

  21. Slide 21: Development and Regulatory Strategy

    21 © 2022 Tonix Pharmaceuticals Holding Corp. DEVELOPMENT AND REGULATORY STRATEGY • 1 st Indication – Kidney allotransplantation (human to human) ‒ Replacement for nephrotoxic CNI’s (calcineurin inhibitors, e.g. Prograf ® (tacrolimus) 1 , Neoral ® (cyclosporin) 2 ‒ Similar development path to the su

  22. Slide 22: TNFα Superfamily Members Are Targeted by mAbs

    22 © 2022 Tonix Pharmaceuticals Holding Corp. TNF ߙ  SUPERFAMILY MEMBERS ARE TARGETED BY mAbs • CD40L is a member of the Tumor Necrosis Factor (TNF α ) Superfamily 1 • Other TNF α Superfamily members have proven to be effective targets for antagonist (blocking) mAbs 2 anti - TNFα mAbs for the treat

  23. Slide 23: Immunology Biotech Acquisitions

    23 © 2022 Tonix Pharmaceuticals Holding Corp. 2020 September October November December 2021 January Immunogenics acquired by Gilead for $21B 1 • TRODELVY TM ( sacituzu mab govitecan - hziy ) is an anti - Trop - 2 antibody - drug conjugate (ADC) approved for triple - negative breast cancer Momenta ac

  24. Slide 24: Monoclonal Antibody Market Growth

    24 © 2022 Tonix Pharmaceuticals Holding Corp. • Over 100 mAbs have been approved by the US FDA, and significant growth potential remains 1 • Global mAb market is projected to grow from $179B in 2021 to $452B in 2028 at a CAGR of 14.1% 2 1 Mullard A. May 5, 2021. Accessed February 24, 2022. (https://

  25. Slide 25: TNX-1700: Gastric, Colorectal and Pancreatic Cancers

    25 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 1700*: GASTRIC, COLORECTAL AND PANCREATIC CANCERS STABILIZED RECOMBINANT TREFOIL FACTOR 2 ( r TFF2) POTENTIAL NEW CANCER TREATMENT • TNX - 1700 (rTFF2) has effects on cancer by altering the tumor micro - environment • Mechanism of action: suppresse

  26. Slide 26: CNS: Key Candidates

    © 2022 Tonix Pharmaceuticals Holding Corp. CNS: KEY CANDIDATES

  27. Slide 27: TNX-1300: Cocaine Intoxication

    27 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 1300*: COCAINE INTOXICATION COCAINE ESTERASE ( CoCe ) PROFILE Cocaine is the main cause for drug - related ED visits 1 Cocaine use can cause irreversible structural damage to the heart and accelerate cardiovascular disease 2 • In one survey of 94 l

  28. Slide 28: TNX-102 SL: Fibromyalgia

    28 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 102 SL*: FIBROMYALGIA CYCLOBENZAPRINE PROTECTIC ® SUBLINGUAL TABLETS CNS PORTFOLIO A unique formulation of cyclobenzaprine designed to optimize delivery and absorption Innovative and proprietary PROTECTIC ® Rapid drug exposure following nighttime a

  29. Slide 29: TNX-102 SL: Fibromyalgia Program Update

    29 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 102 SL: FIBROMYALGIA CYCLOBENZAPRINE PROTECTIC ® SUBLINGUAL TABLETS PROGRAM UPDATE Phase 3 Study, RALLY (F306) • July 2021: Tonix stopped enrollment in the RALLY study following an unblinded, pre planned interim analysis by the Independent Data Mon

  30. Slide 30: TNX-102 SL: Long COVID (PASC)

    30 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 102 SL*: LONG COVID (PASC) CYCLOBENZAPRINE PROTECTIC ® SUBLINGUAL TABLETS PROFILE Long COVID or Post - acute Sequelae of COVID - 19 (PASC 1 ) • Symptoms can include fatigue, sleep disorders, pain, fevers, shortness of breath, cognitive impairment d

  31. Slide 31: TNX-1900: Migraine

    31 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 1900*: MIGRAINE INTRANASAL POTENTIATED OXYTOCIN (OT) WITH MAGNESIUM CNS PORTFOLIO Intranasal OT has potential utility in treating migraine 1 • Intranasal OT reaches the trigeminal ganglion • Preclinical evidence of OT blocking CGRP release and supp

  32. Slide 32: TNX-2900: Prader-Willi Syndrome

    32 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 2900*: PRADER - WILLI SYNDROME INTRANASAL POTENTIATED OXYTOCIN (OT) WITH MAGNESIUM PROFILE CNS PORTFOLIO Prader - Willi Syndrome is the most common genetic cause of life - threatening childhood obesity • Orphan disease occurring in 1 in 15,000 birt

  33. Slide 33: TNX-601 CR: Psychiatry

    33 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 601 CR*: PSYCHIATRY TIANEPTINE OXALATE AND NALOXONE CNS PORTFOLIO A novel, oral, controlled release once - daily tablet Mechanistically different from traditional monoaminergic treatments for depression Indirectly modulates the glutamatergic system

  34. Slide 34: Infectious Diseases: Key Candidates

    © 2022 Tonix Pharmaceuticals Holding Corp. INFECTIOUS DISEASES: KEY CANDIDATES

  35. Slide 35: COVID-19: Entering Endemic Phase

    35 © 2022 Tonix Pharmaceuticals Holding Corp. • Delta and Omicron variant waves are waning in most parts of the US ‒ Leaving a path of morbidity and mortality, including “breakthrough” infection and disease among vaccinated and convalescent • U.S. states are rolling back state pandemic restrictions

  36. Slide 36: COVID-19: The Missing Pieces

    36 © 2022 Tonix Pharmaceuticals Holding Corp. • Vaccines : early vaccines slowed pandemic, but are showing limitations ‒ Short term protection – requirement for boosters with mRNA vaccines; ‒ Increasing focus on preventing hospitalization and death • Anti - viral drugs : Veklury ® (remdesivir), Paxl

  37. Slide 37: COVID-19 Vaccines: Where We Are Today

    37 © 2022 Tonix Pharmaceuticals Holding Corp. Durability of protection ‒ mRNA vaccinated people lose protection, starting at 4 - 6 months 1 ‒ High rates of “breakthrough” COVID during Delta and Omicron waves ‒ Booster vaccinations with mRNA vaccines recommended at 4 - 6 months Effect on forward tran

  38. Slide 38: COVID-19 Vaccines: Where Do We Go From Here?

    38 © 2022 Tonix Pharmaceuticals Holding Corp. mRNA vaccines have slowed pandemic, but may not be a long - term solution ‒ Vaccinated people lost protection and showed high rates of “breakthrough” COVID during Delta and Omicron waves ‒ COVID is becoming endemic in the US; vaccination of entire world

  39. Slide 39: Live Virus Vaccines: Development Rationale

    39 © 2022 Tonix Pharmaceuticals Holding Corp. • Control of smallpox, measles, mumps, rub ella, chickenpox and other viral conditions ‒ Prevent forward transmission • Effective in eliciting durable or long - term immunity • Economical to manufacture at scale ‒ Low dose because replication amplifies d

  40. Slide 40: Vaccinia Induces a Skin Reaction Called Take

    40 © 2022 Tonix Pharmaceuticals Holding Corp. VACCINIA INDUCES A SKIN REACTION CALLED “TAKE” – DESCRIBED BY DR. EDWARD JENNER *Example of major cutaneous reaction, or “take,” resulting from a replication - competent live - virus vaccine with intradermal deli very, indicating successful vaccination 1

  41. Slide 41: Live Virus Vaccine Platform: Recombinant Pox Vaccine Technology

    41 © 2022 Tonix Pharmaceuticals Holding Corp. LIVE VIRUS VACCINE PLATFORM: NEW RECOMBINANT POX VACCINE (RPV) TECHNOLOGY FOR EMERGING INFECTIOUS DISEASES AND ONCOLYTICS COVID - 19 Future Pandemics Infectious Disease Biodefense Using Proven Science To Address Challenging Disease States, We Have Create

  42. Slide 42: TNX-801: Smallpox and Monkeypox Vaccine

    42 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 801: SMALLPOX AND MONKEYPOX VACCINE LIVE VIRUS PLATFORM DEVELOPMENT PROGRAM APPLICATION OF LIVE VIRUS PLATFORM • TNX - 801 is a cloned version of horsepox 1 (without any insert) purified from cell culture • In addition to being a potential addition

  43. Slide 43: Virus-Based Science Is Well Established

    43 © 2022 Tonix Pharmaceuticals Holding Corp. VIRUS - BASED SCIENCE IS WELL ESTABLISHED • Streamlined development • Ability to vertically integrate development and manufacturing • Multi - dose packaging, standard cold - chain products LIVE VIRUS RECOMBINANT POX VACCINE ( RPV ) PLATFORM PROFILE POTEN

  44. Slide 44: TNX-1840 and TNX-1850: COVID Vaccine

    44 © 2022 Tonix Pharmaceuticals Holding Corp. CNS PORTFOLIO TNX - 1840 AND TNX - 1850*: COVID - 19 VACCINE LIVE VIRUS PLATFORM DEVELOPMENT PROGRAM APPLICATION OF LIVE VIRUS PLATFORM • First version TNX - 1800 encodes spike protein from SARS - CoV - 2, Wuhan strain • Planned new versions TNX - 1840 a

  45. Slide 45: What Makes TNX-1840 and TNX-1850 Different from mRNA Vaccines

    45 © 2022 Tonix Pharmaceuticals Holding Corp. LIVE VIRUS PLATFORM: WHAT MAKES TNX - 1840 AND TNX - 1850 DIFFERENT FROM m RNA VACCINES CRITERIA mRNA VACCINES TNX - 1840/TNX - 1850 Number of shots Two One Duration 6 months Years / decades Boosters Recommended Likely not required Protection from varian

  46. Slide 46: Internal Development and Manufacturing Capabilities

    46 © 2022 Tonix Pharmaceuticals Holding Corp. LIVE VIRUS RPV P LATFORM & COVID - 19 VACCINE INTERNAL DEVELOPMENT & MANUFACTURING CAPABILITIES Infectious Disease R&D Center (RDC) – Frederick, MD • Function : Accelerated development of vaccines and antiviral drugs against COVID - 19, its variants and

  47. Slide 47: American Pandemic Preparedness Plan

    47 © 2022 Tonix Pharmaceuticals Holding Corp. • “Platforms” – Foundation of Pandemic Response ‒ Key element of AP3 from White House Office of Science and Technology Policy or OSTP 1,2 ▪ 100 days to human trials ▪ Technologies that do not require sterile injection • TNX - 801/ - 1840/ - 1850 (live vi

  48. Slide 48: Two Types of Immunity

    48 © 2022 Tonix Pharmaceuticals Holding Corp. TWO TYPES OF IMMUNITY • Antibodies – can be measured in a blood test, but anti - SARS - CoV - 2 antibodies are not predictive of protection • T cell – can be measured in a blood test, but requires sophisticated lab, unknown if predictive ASSESSING anti -

  49. Slide 49: TNX-2100: SARS-CoV-2 Diagnostic to Measure T Cell Immunity

    49 © 2022 Tonix Pharmaceuticals Holding Corp. DESIGNED TO MEASURE THE PRESENCE AND STRENGTH OF FUNCTIONAL IN VIVO T CELL IMMUNITY • Designed to elicit delayed - type hypersensitivity in individuals who have been exposed to SARS - CoV - 2 or successfully vaccinated • SARS - CoV - 2 epitope peptide mi

  50. Slide 50: TNX-2100: Potential Uses and Development Plan

    50 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 2100*: POTENTIAL USES AND DEVELOPMENT PLAN *TNX - 2100 has not been approved for any indication. † Intracellular cytokine staining (ICS) measured by flow cytometry after in vitro stimulation of purified peripheral blood mono nuc lear cells. DEVELOP

  51. Slide 51: Small Molecule COVID-19 Therapeutics

    51 © 2022 Tonix Pharmaceuticals Holding Corp. The only COVID - 19 antiviral that is FDA approved is Remdesivir/ Veklury ® ‒ Gilead – Intravenous ( i.v. ) medicine ‒ FDA approved for patients who are at least 12 years old and require hospitalization ‒ May shorten the time to recover from acute COVID

  52. Slide 52: TNX-3500: COVID Antiviral Treatment

    52 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 3500*: COVID - 19 ANTIVIRAL TREATMENT SANGIVAMYCIN PROFILE New variants heighten need for therapeutics NIH Treatment Guidelines for COVID - 19 are mixed on use of remdesivir Potential monotherapy antiviral 1,2 • 65 times more potent than remdesivir

  53. Slide 53: Monoclonal Antibodies for COVID-19

    53 © 2022 Tonix Pharmaceuticals Holding Corp. Monoclonal antibodies ( mAbs ) (EUA) – 3 with US Emergency Use Authorization 1 ‒ Vir /GSK – XEVURDY® ( sotrovimab ) 1 – ONLY mAb ACTIVE AGAINST OMICRON ‒ Lilly - bebtelovimab – EUA for treatment of mild or moderate COVID 2 ‒ AstraZeneca – Evusheld ( Tixa

  54. Slide 54: TNX-3600: COVID Therapeutic

    54 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 3600*: COVID - 1 9 THERAPEUTIC FULLY HUMAN MONOCLONAL ANTIBODY PLATFORM PROFILE Collaboration with Columbia University Human monoclonal antibodies ( mAbs ) generated from COVID - 19 convalescent patients Potential monotherapies • Plan to seek indic

  55. Slide 55: TNX-3700: COVID Vaccine Zinc Nanoparticle

    55 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 3700*: COVID - 19 VACCINE ZINC NANOPARTICLE (ZNP) FORMULATION FOR m RNA VACCINE PROFILE Collaboration with Kansas State University ZNP technology is a potential replacement for the Lipid Nanoparticle (LNP) technology of current mRNA vaccines Potent

  56. Slide 56: Future Outlook

    © 2022 Tonix Pharmaceuticals Holding Corp. FUTURE OUTLOOK

  57. Slide 57: Key Development Programs and Partners

    57 © 2022 Tonix Pharmaceuticals Holding Corp. TNX - 1300: COCAINE INTOXICATION TNX - 1700: GASTRIC AND PANCREATIC CANCERS TNX - 3600: MONOCLONAL ANTIBODIES FOR COVID - 19 TREATMENT KEY DEVELOPMENT PARTNERS TNX - 1500: ALLOGRAFT REJECTION TNX - 1900: MIGRAINE & OTHER INDICATIONS TNX - 801: SMALLPOX A

  58. Slide 58: Milestones: Recently Completed and Upcoming

    58 © 2022 Tonix Pharmaceuticals Holding Corp. Expected Data □ 1 st Quarter 2022 Topline data from TNX - 102 SL Phase 3 F306/RALLY study in fibromyalgia □ 1 st Half 2022 Topline data from first - in - human TNX - 2100 skin test study Expected Clinical Trial Initiations □ 1 st Half 2022 Phase 2 OL saf

  59. Slide 59: Management Team

    59 © 2022 Tonix Pharmaceuticals Holding Corp. MANAGEMENT TEAM Seth Lederman, MD Co - Founder, CEO & Chairman Jessica Morris Chief Operating Officer Gregory Sullivan, MD Chief Medical Officer Bradley Saenger, CPA Chief Financial Officer

  60. Slide 60: Thank You

    © 2022 Tonix Pharmaceuticals Holding Corp. THANK YOU

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