Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP

Tonix Pharmaceuticals — The Wall Street Conference March 2022

58 slides from Tonix Pharmaceuticals's conference presentation, Mar 8, 2022

  1. Slide 1: Investor Presentation, The Wall St Conference March 8, 2022

    © 2022 Tonix Pharmaceuticals Holding Corp. INVESTOR PRESENTATION THE WALL ST CONFERENCE MARCH 8, 2022 NASDAQ: TNXP Version P0339 March 8, 2022 (Doc 0967)

  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: Mission, What We Do, Our Strategy

    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: Portfolio: BioDefense and COVID candidates

    4 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO *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) ba

  5. Slide 5: CNS Portfolio Pipeline

    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 (anti-CD40L mAb): Potential Treatment for Organ Transplant Rejection

    7 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO 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 autoimmunit

  8. Slide 8: TNX-1500 Market Opportunity

    8 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO 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 rejecti

  9. Slide 9: CD40L Biology

    9 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO 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 -

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

    10 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO 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 TREATME

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

    11 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO 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

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

    12 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO 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 protectin

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

    13 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO 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 acce

  14. Slide 14: Non-Human Primate Kidney Allotransplantation Study

    14 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO 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 thro

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

    15 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO TOLERANCE INDUCTION WITH DONOR BONE MARROW TRANSPLANTATION Induction of “mixed chimerism” induces allograft tolerance ‒ Long - lasting, durable tolerance — specifically to donor tissues ‒ Initial protocols requ

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

    16 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO 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 - H

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

    17 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO 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 block

  18. Slide 18: Recent Xenotransplant Headlines

    18 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO “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 Ed

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

    19 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO 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

  20. Slide 20: TNX-1500: Key Considerations

    20 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO 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

  21. Slide 21: Development and Regulatory Strategy

    21 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO 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 ® (cyclosp

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

    22 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO 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 (blo

  23. Slide 23: Recent Immunology Acquisitions

    23 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO 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 fo

  24. Slide 24: Monoclonal Antibody Market

    24 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO • 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,

  25. Slide 25: Key Development Partners

    25 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO 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: MIGRAI

  26. Slide 26: Management Team

    26 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO 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

  27. Slide 27: Appendix

    © 2022 Tonix Pharmaceuticals Holding Corp. APPENDIX

  28. Slide 28: Molecules Targeting CD40L

    28 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO MOLECULES TARGETING CD40L Drug name Sponsor Molecule Indications Stage of development/clinical trial status Ruplizumab (BG9588) Biogen hu5c8 Fab* IgG1 mAb LN, SLE, transplantation Discontinued after Phase 2 Tor

  29. Slide 29: Molecules Blocking CD40

    29 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO MOLECULES BLOCKING CD40 BLOCKADE Drug name Sponsor Molecule Indications Stage of development/clinical trial status Bleselumab (ASKP1240) Astellas Pharma Humanized Fab IgG4 mAb Psoriasis, FSGS in Kidney Tx Disco

  30. Slide 30: About Monoclonal Antibodies

    30 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO ABOUT MONOCLONAL ANTIBODIES ( mAbs ) STRUCTURE, THERAPEUTIC & COMMERCIAL POTENTIAL 1. Patient Protection and Affordable Care Act, HR 3590, 111th Congress (2009 - 2010). mAbs have unique therapeutic benefits: •

  31. Slide 31: Rejection Is Caused by Immune Response

    31 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO REJECTION IS CAUSED BY IMMUNE RESPONSE • Transplant rejection occurs because the immune system recognizes the allograft as foreign and attacks it 1 • The immune response is triggered by the activation and proli

  32. Slide 32: T Cell Activation Requires Key Signals

    32 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO T CELL ACTIVATION REQUIRES 3 KEY SIGNALS • Three key signals are required for T cell activation 1 - 3 : Signal 1 • Antigen binding at T cell receptors (TCR) Signal 2 / Costimulation • Stimulation of CD80/86 - C

  33. Slide 33: Costimulation Involves Distinct Pathways

    33 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO COSTIMULATION INVOLVES 2 DISTINCT PATHWAYS • The CD40L - CD40 pathway induces maturation of APCs by altering gene expression, including the upregulation of CD80/86 1,2 • Stimulation of CD28 in T cells results i

  34. Slide 34: Immunosuppression Is the Standard of Care

    34 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO IMMUNOSUPPRESSION IS THE STANDARD OF CARE • Immunosuppression is the standard of care to prevent rejection 1,2 : Calcineurin Inhibitors • CNIs such as cyclosporin and tacrolimus block calcineurin Corticosteroid

  35. Slide 35: The Cornerstone of Immunosuppression

    35 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO THE CORNERSTONE OF IMMUNOSUPPRESSION • CNIs, mainly tacrolimus, are the cornerstone of immunosuppressive therapy and have helped reduce acute rejection and increase 1 - year graft survival 1,2 • Most transplant

  36. Slide 36: Nephrotoxicity Is the Achilles' Heel of CNIs

    36 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO NEPHROTOXICITY IS THE ACHILLES’ HEEL OF CNIS • CNIs cause irreversible and progressive deterioration of kidney function in all types of solid organ transplants 1,2 • CNIs can also cause hypertension, neurotoxic

  37. Slide 37: Broadening the Therapeutic Window

    37 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO BROADENING THE THERAPEUTIC WINDOW • CNIs have a narrow therapeutic window, risking drug toxicities and rejection 1,2 • Immunomodulation with next - generation treatments strives to provide a broader therapeutic

  38. Slide 38: The Search for CNI-Sparing Treatments

    38 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO THE SEARCH FOR CNI - SPARING TREATMENTS • “CNI - sparing” regimens that reduce or avoid use of CNIs are being developed • CNI - sparing regimens have been based on 2 classes of agents: mTOR Inhibitors • Sirolim

  39. Slide 39: Sparing CNIs by Targeting Costimulation

    39 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO SPARING CNI s BY TARGETING COSTIMULATION • When used to replace CNIs, belatacept shows similar rates of long - term patient and graft survival but is associated with increased acute rejection rates and increase

  40. Slide 40: Blocking Costimulation Through CD40L

    40 © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY AND INFECTIOUS DISEASE PORTFOLIO BLOCKING COSTIMULATION THROUGH CD40L • The CD40 - CD40L pathway was the first costimulation pathway explored to inhibit transplant rejection CD40L Antibodies (Abs) • Ruplizumab (hu5c8) • Toralizumab Zhang T, et

  41. Slide 41: Immunology: Key Candidate

    © 2022 Tonix Pharmaceuticals Holding Corp. IMMUNOLOGY: KEY CANDIDATE

  42. Slide 42: TNX-1700: Gastric, Colorectal and Pancreatic Cancers

    42 © 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

  43. Slide 43: CNS: Key Candidates

    © 2022 Tonix Pharmaceuticals Holding Corp. CNS: KEY CANDIDATES

  44. Slide 44: TNX-1300: Cocaine Intoxication

    44 © 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

  45. Slide 45: TNX-102 SL: Fibromyalgia

    45 © 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

  46. Slide 46: TNX-102 SL Fibromyalgia Program Update

    46 © 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

  47. Slide 47: TNX-102 SL: Long COVID (PASC)

    47 © 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

  48. Slide 48: TNX-1900: Migraine

    48 © 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

  49. Slide 49: TNX-2900: Prader-Willi Syndrome

    49 © 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

  50. Slide 50: TNX-601 CR: Psychiatry

    50 © 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

  51. Slide 51: Infectious Diseases: Key Candidates

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

  52. Slide 52: TNX-801: Smallpox and Monkeypox Vaccine

    52 © 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

  53. Slide 53: TNX-1840 and TNX-1850: COVID Vaccine

    53 © 2022 Tonix Pharmaceuticals Holding Corp. 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 and TNX - 1850

  54. Slide 54: Live Virus RPV Platform Development and Manufacturing Capabilities

    54 © 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

  55. Slide 55: TNX-3500: COVID Antiviral Treatment

    55 © 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

  56. Slide 56: TNX-3600: COVID Therapeutic

    56 © 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

  57. Slide 57: TNX-3700: COVID Vaccine

    57 © 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

  58. Slide 58: Thank You

    © 2022 Tonix Pharmaceuticals Holding Corp. THANK YOU

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