Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP

Tonix Pharmaceuticals — WVC 2025

41 slides from Tonix Pharmaceuticals's conference presentation, Apr 24, 2025

  1. Slide 1: A Novel Single-Dose, Live Attenuated, Minimally Replicative Mpox Vaccine

    A NOVEL SINGLE - DOSE, LIVE ATTENUATED, MINIMALLY REPLICATIVE MPOX VACCINE Farooq Nasar, Ph.D., M.P.H. WVC April 23, 2025 PO6066 April 23, 2025 (Doc 1583) © 2025 Tonix Pharmaceuticals Holding Corp.

  2. Slide 2: 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 Litigation Reform Act of 1995. These statements may be identified by the use of forward - looking wo

  3. Slide 3: Talk Overview

    TALK OVERVIEW 3 © 2025 Tonix Pharmaceuticals Holding Corp. 1) Background 2) TNX - 801 attenuation in vitro and in vivo 3) TNX - 801 immunogenicity and efficacy in animal models *TNX - 801 is in the pre - IND stage of development and has not been approved for any indication.

  4. Slide 4: Poxviruses

    POXVIRUSES » Double stranded DNA, ~128 - 456 kb size » Virions: enveloped, brick - shaped » Size:  220 to 450 nm long î 140 to 260 nm wide î 140 to 260 nm thick » Infect vertebrate or invertebrate hosts » Genus Orthopoxvirus : ▪ Human Pathogens: • VARV: Case fatality rate  30 to 50% • MPXV: Case f

  5. Slide 5: Poxviruses: Ubiquitous in the Environment

    POXVIRUSES: UBIQUITOUS IN THE ENVIRONMENT 5 © 2025 Tonix Pharmaceuticals Holding Corp.

  6. Slide 6: Genus Orthopoxvirus

    GENUS ORTHOPOXVIRUS 6 © 2025 Tonix Pharmaceuticals Holding Corp.

  7. Slide 7: Monkeypox Virus (Mpox)

    MONKEYPOX VIRUS (MPOX) 7 © 2025 Tonix Pharmaceuticals Holding Corp. » Endemic in Central and West Africa » Two Clades: 1) Clade I (DRC) 2) Clade IIa (West Africa) and IIb (Nigeria) » Human Case Fatality Rate: ▪ Clade I –  11% ▪ Clade IIa –  3% ▪ Clade IIb –  <0.1% » Clade IIb – 2022 Outbreak ▪ 12

  8. Slide 8: Variola Virus (Smallpox)

    VARIOLA VIRUS (SMALLPOX) 8 © 2025 Tonix Pharmaceuticals Holding Corp. » Oldest written record –  3,500 years » Oldest sequences –  1,400 years » Human Case Fatality Rate:  30% » 20 th century –  250 to 500 million deaths » Eradication: 1980

  9. Slide 9: Edward Jenner - Smallpox Vaccine (1796)

    EDWARD JENNER - SMALLPOX VACCINE (1796) » Jenner observed milkmaids were protected from smallpox, reasoned that infection with an illness similar to smallpox but less deadly could protect one against smallpox ▪ “Cowpox” was the name of a disease in cows that could transfer to humans and cause sores

  10. Slide 10: Equination - Smallpox Vaccines from Horses

    EQUINATION - SMALLPOX VACCINES FROM HORSES » Equination, the use of vaccines from horses ( equus in Latin), was successfully used in parallel with vaccination in Europe 1 » Vaccine producers may have propagated stocks by periodically supplementing or refreshing them with horsepox 2 ▪ A 1902 smallpox

  11. Slide 11: HPXV Was Used as Civil War-Era Vaccine

    HPXV WAS USED AS CIVIL WAR - ERA VACCINE TNX - 801 VK05 has the highest identity to HPXV across the whole genome and represents a true HSPV strain 99.8% Identical Brinkmann A, et al. Genome Biol . 2020;21(1):286. 99.7% Identical 212,688 bps 212,633 bps Key Points ▪ Pre - Mulford vaccines: VK05, VK12

  12. Slide 12: Smallpox Vaccines

    SMALLPOX VACCINES 1 Downie AW. 1939. Br J Exp Pathol 20:158 – 176. 12 © 2025 Tonix Pharmaceuticals Holding Corp. » Vaccine: Cowpox origin » Serial passaging: Humans, cows, and horses (143 years) » Vaccine: Vaccinia Virus (1939) closely related to cowpox but serologically distinct 1 » Multiple Vaccin

  13. Slide 13: Balance of Tolerability and Reactogenicity for Pox-Based Vaccines

    BALANCE OF TOLERABILITY AND REACTOGENICITY FOR POX - BASED VACCINES Well tolerated, Requires high dose, 2 vaccinations ~1 death per million ~10 deaths per million Non - propagating 13 © 2025 Tonix Pharmaceuticals Holding Corp. Robustly Propagating Dryvax Lister TianTan Copenhagen Tashkent MVA NYVAC

  14. Slide 14: Balance of Tolerability and Reactogenicity for Pox-Based Vaccines

    BALANCE OF TOLERABILITY AND REACTOGENICITY FOR POX - BASED VACCINES Well tolerated, Requires high dose, 2 vaccinations ~1 death per million ~10 deaths per million Non - propagating 14 © 2025 Tonix Pharmaceuticals Holding Corp. Robustly Propagating Dryvax Lister TianTan Copenhagen Tashkent MVA NYVAC

  15. Slide 15: Balance of Tolerability and Reactogenicity for Pox-Based Vaccines

    BALANCE OF TOLERABILITY AND REACTOGENICITY FOR POX - BASED VACCINES 1. Noyce RS, et al. PLoS One. 2018;13(1):e0188453. 2. Schrick L, et al. N Eng J Med. 2017;377(15):1491 - 1492. TNX - 801 scHPXV (Horsepox) 212,811 bp LITR 1A 1B 4 2 3 5 6 7 RITR MNR - 76 genome fragments MNR - 76 genome (212,633 bp)

  16. Slide 16: Prong Approach to Mpox/Smallpox Vaccine

    4 PRONG APPROACH TO MPOX/SMALLPOX VACCINE (TNX - 801) 16 © 2025 Tonix Pharmaceuticals Holding Corp. 1) Well - tolerated 2) Single dose 3) Durable 4) Protection against mpox disease (lesions)

  17. Slide 17: Attenuation In Vitro and In Vivo

    TNX - 801 ATTENUATION IN VITRO AND IN VIVO 17 © 2025 Tonix Pharmaceuticals Holding Corp.

  18. Slide 18: In Vitro Attenuation

    IN VITRO ATTENUATION OF TNX - 801 18 © 2025 Tonix Pharmaceuticals Holding Corp. » Investigate attenuation of TNX - 801 in vitro relative to VACV strains ▪ Positive Control: VACV - Western Reserve (WR) , VACV - International Health Department (IHD) ▪ Older vaccines used in smallpox eradication: 1) VA

  19. Slide 19: Displays Small Plaque Phenotype

    TNX - 801 DISPLAYS SMALL PLAQUE PHENOTYPE VACCINA VIRUSES VACV - Western Reserve (WR) VACV - International Health Department (IHD) VACV - Lister (Lis) VACV - New York City Board of Health (NYCBH) TNX - 801 MVA 19 © 2025 Tonix Pharmaceuticals Holding Corp.

  20. Slide 20: Replication in Primary Human Cells (Dermal Tract)

    TNX - 801: REPLICATION IN PRIMARY HUMAN CELLS (DERMAL TRACT) Plaque Assay qPCR VACV - Western Reserve (WR) VACV - International Health Department (IHD) VACV - Lister (Lis) VACV - New York City Board of Health (NYCBH) TNX - 801 MVA TNX - 801:  27 - to 119 - fold more attenuated than VACV based vacci

  21. Slide 21: Replication in Primary Human Cells (Respiratory Tract)

    TNX - 801: REPLICATION IN PRIMARY HUMAN CELLS (RESPIRATORY TRACT) 21 Plaque Assay qPCR TNX - 801:  20 - to 30 - fold more attenuated than VACV based vaccines VACV - Western Reserve (WR) VACV - International Health Department (IHD) VACV - Lister (Lis) VACV - New York City Board of Health (NYCBH) TNX

  22. Slide 22: In Vivo Attenuation

    IN VIVO ATTENUATION OF TNX - 801 » Investigate attenuation of TNX - 801 in vivo relative to VACV based vaccines ▪ Immunocompromised Mice (C57BL/6 ifnar - / - , C57BL/6 ifngr - / - , C57BL/6 ifnar - / - /ifngr - / - ) • Interferon receptor knockout model • Sensitive to virus infection ▪ Positive Cont

  23. Slide 23: Lacks Lethality Associated with Older Smallpox Vaccine Strains

    TNX - 801 LACKS LETHALITY ASSOCIATED WITH OLDER SMALLPOX VACCINE STRAINS (LIS, NYCBH) TNX - 801*, Mock ~10 8 PFU VACV - IHD, Lis, NYCBH ~10 6 and 10 5 PFU VACV - WR ~10 6 and 10 5 PFU TNX - 801 is 1,000 - fold more attenuated 23 © 2025 Tonix Pharmaceuticals Holding Corp.

  24. Slide 24: Lacks Lethality Associated with Older Smallpox Vaccine Strains

    TNX - 801 LACKS LETHALITY ASSOCIATED WITH OLDER SMALLPOX VACCINE STRAINS (LIS, NYCBH) TNX - 801*, Mock ~3 x 10 8 PFU VACV - Lis, NYCBH ~10 4 and 10 3 PFU VACV - WR, IHD ~10 3 PFU TNX - 801 is up to 100,000 - fold more attenuated 24 © 2025 Tonix Pharmaceuticals Holding Corp.

  25. Slide 25: Infection Displays Limited Replication

    TNX - 801 INFECTION DISPLAYS LIMITED REPLICATION VACV - IHD ~10 6 PFU ( ) VACV - NYCBH ~10 6 PFU ( ) TNX - 801 ~10 8 PFU ( ) 25 © 2025 Tonix Pharmaceuticals Holding Corp.

  26. Slide 26: Highly Attenuated with Improved Safety Profiles Compared to Other Vaccinia-Based Vaccines

    TNX - 801 IS HIGHLY ATTENUATED WITH IMPROVED SAFETY PROFILES COMPARED TO OTHER VACCINA - BASED VACCINES 26 © 2025 Tonix Pharmaceuticals Holding Corp.

  27. Slide 27: Immunogenicity and Efficacy in Animal Models

    TNX - 801 IMMUNOGENICITY AND EFFICACY IN ANIMAL MODELS 1) 2) 3) 27 © 2025 Tonix Pharmaceuticals Holding Corp.

  28. Slide 28: NHP Immunogenicity and Efficacy Study Design

    NHP IMMUNOGENICITY AND EFFICACY STUDY DESIGN rVACV = synthesized vaccinia similar to ACAM2000 (Approved Vaccine) 28 © 2025 Tonix Pharmaceuticals Holding Corp.

  29. Slide 29: NHP Immunogenicity: Neutralizing Antibody Response

    NHP IMMUNOGENICITY: NEUTRALIZING ANTIBODY RESPONSE 29 © 2025 Tonix Pharmaceuticals Holding Corp.

  30. Slide 30: Provides Protection Against Mpox Disease

    TNX - 801 PROVIDES PROTECTION AGAINST MPOX DISEASE NO LESIONS in TNX - 801 vaccinated groups 30 © 2025 Tonix Pharmaceuticals Holding Corp.

  31. Slide 31: Provides Protection Against Lethal Monkeypox Clade I Challenge

    TNX - 801 PROVIDES PROTECTION AGAINST LETHAL MONKEYPOX CLADE I CHALLENGE NO LETHALITY in TNX - 801 vaccinated groups 31 © 2025 Tonix Pharmaceuticals Holding Corp.

  32. Slide 32: Provides Durable Protection Against Lethal Rabbitpox Challenge: 6 Months

    TNX - 801 PROVIDES DURABLE PROTECTION AGAINST LETHAL RABBITPOX CHALLENGE: 6 MONTHS 32 © 2025 Tonix Pharmaceuticals Holding Corp.

  33. Slide 33: Provides Durable Protection Against Lethal Rabbitpox Challenge: 6 Months

    TNX - 801 PROVIDES DURABLE PROTECTION AGAINST LETHAL RABBITPOX CHALLENGE: 6 MONTHS 33 © 2025 Tonix Pharmaceuticals Holding Corp.

  34. Slide 34: Provides Durable Protection Against Lethal Rabbitpox Challenge: 6 Months

    TNX - 801 PROVIDES DURABLE PROTECTION AGAINST LETHAL RABBITPOX CHALLENGE: 6 MONTHS 34 © 2025 Tonix Pharmaceuticals Holding Corp.

  35. Slide 35: Protection Against Lethal Monkeypox Clade IIa Challenge: Alternative Routes

    TNX - 801 PROVIDES PROTECTION AGAINST LETHAL MONKEYPOX CLADE IIA CHALLENGE: ALTERNATIVE ROUTES 35 © 2025 Tonix Pharmaceuticals Holding Corp.

  36. Slide 36: Elicits Humoral Immune Responses: Anti-VACV IgG Titers

    TNX - 801 ELICITS HUMORAL IMMUNE RESPONSES: ANTI - VACV IGG TITERS (28 DAYS POST - VACCINATION) 36 © 2025 Tonix Pharmaceuticals Holding Corp.

  37. Slide 37: Protection Against Lethal Monkeypox Clade IIa Challenge: Alternative Routes

    TNX - 801 PROVIDES PROTECTION AGAINST LETHAL MONKEYPOX CLADE IIA CHALLENGE: ALTERNATIVE ROUTES TNX - 801 (PERCUT) TNX - 801 (SC) TNX - 801 (IM) Mock Weight Loss Survival *p <0.0001 *p <0.003 * * * * 37 © 2025 Tonix Pharmaceuticals Holding Corp.

  38. Slide 38: Safety

    TNX - 801 SAFETY 38 © 2025 Tonix Pharmaceuticals Holding Corp. » In vitro : ▪ Small plaque phenotype ▪ Up to 100 - fold lower replication than VACV strains ▪ Primary cells from dermal and respiratory tracts » In vivo : ▪ Well tolerated in mice, rabbits, hamsters, and NHPs ▪ Minimal or no disease in

  39. Slide 39: Immunogenicity and Efficacy (Single Dose)

    TNX - 801 IMMUNOGENICITY AND EFFICACY (SINGLE DOSE) 39 © 2025 Tonix Pharmaceuticals Holding Corp. » Evaluated in multiple animal models ▪ Mouse, Rabbits, and NHPs » Elicits IgG and/or neutralizing responses ▪ Various route percutaneous, subcutaneous, intramuscular ▪ Microneedle delivery » Provides 1

  40. Slide 40: Prong Approach to Mpox/Smallpox Vaccine

    4 PRONG APPROACH TO MPOX/SMALLPOX VACCINE (TNX - 801) 40 © 2025 Tonix Pharmaceuticals Holding Corp. 1) Well - tolerated 2) Single dose 3) Durable 4) Protection against mpox disease (lesions)

  41. Slide 41: Acknowledgements

    ACKNOWLEDGEMENTS 41 © 2025 Tonix Pharmaceuticals Holding Corp. » University of Alberta ▪ Ryan Noyce ▪ David Evans » Southern Research » Tonix Pharmaceuticals ▪ Scott Goebel ▪ Tinoush Moulaei ▪ Natasza Ziółkowska ▪ Siobhan Fogarty ▪ Helen Stillwell ▪ Bruce Daugherty ▪ Sina Bavari ▪ Seth Lederman » To

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