Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP

Tonix Pharmaceuticals — Investor Presentation (January 2017)

39 slides from Tonix Pharmaceuticals's investor presentation, Jan 3, 2017

  1. Slide 1: Investor Presentation January 2017

    Investor Presentation January 2017 Version P0045 1-3-17 © 2017 Tonix Pharmaceuticals Holding Corp

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

    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 Litigation Reform Act of 1995. These statements may be i

  3. Slide 3: Tonix (TNXP): Developing Innovative Pharmaceutical Products to Address Emerging Public Health Issues

    Tonix (TNXP): Developing Innovative Pharmaceutical Products to Address Emerging Public Health Issues Targeting central nervous system conditions • Phase 3-ready drug, TNX-102 SL* for posttraumatic stress disorder (PTSD) with Breakthrough Therapy designation • Encouraging evidence of safety and effic

  4. Slide 4: Product Pipeline

    Product Pipeline Candidate Indication Preclinical Phase 1 Phase 2 Phase 3 NDA/BLA Market Registration Plan 505(b)(2) TNX-102 SL PTSD Phase 2 results reported expected to enroll New route of May, 2016 1Q 2017 Cyclobenzaprine HCl sublingual tablets administration and new indication Novel polymorph and

  5. Slide 5: Tonix Pharmaceuticals PTSD Program

    Tonix Pharmaceuticals PTSD Program Phase 3 Ready Program TNX-102 SL (cyclobenzaprine HCl sublingual tablets) • A unique, innovative product designed for bedtime administration • Targeting a chronic and serious psychiatric disorder: PTSD Therapeutic dose identified in Phase 2 study Phase 3 clinical a

  6. Slide 6: PTSD characteristics

    PTSD characteristics • Unmet medical need • PTSD is a serious condition and the prevalence is increasing, especially combat PTSD • Military-related PTSD is not satisfactorily treated by existing FDA approved therapies • Two selective serotonin reuptake inhibitors (SSRIs) are approved for PTSD, but h

  7. Slide 7: Breakthrough Therapy designation

    Breakthrough Therapy designation • FDA granted TNX-102 SL Breakthrough Therapy designation – reported December 19, 2016 • PTSD is a serious condition • TNX-102 SL has the potential to have advantages over existing therapies in military-related PTSD • Benefits of Breakthrough Therapy designation • El

  8. Slide 8: What is PTSD?

    What is PTSD? A chronic response to traumatic event(s) • A majority of people will experience a traumatic event at some point in their lifetime1 • 20% of women and 8% of men in the U.S. who experience significant trauma develop PTSD1 • Lifetime prevalence: 6.8%2 (~ 17 million adults in the U.S.) • P

  9. Slide 9: What Are the Symptoms of PTSD?

    What Are the Symptoms of PTSD? Symptoms of PTSD fall into four clusters: 1. Intrusion (aversive memories, nightmares, flashbacks) 2. Avoidance (avoiding persons, places or situations) 3. Mood/cognitions (memory block, emotional numbing, detachment from others) 4. Hyperarousal (anxiety, agitation & s

  10. Slide 10: What Are the Consequences of PTSD?

    What Are the Consequences of PTSD? Consequences: • Impaired daily function and substantial interference with work and social interactions • Reckless or destructive behavior • Increased health care utilization and greater medical morbidity PTSD as a risk factor for: • Depression • Alcohol or substanc

  11. Slide 11: PTSD Prevalence and Market Characteristics

    PTSD Prevalence and Market Characteristics Prevalent Population (U.S.) ~8.6 million1 Rx 510,000 6% No Rx 128,000 Undiagnosed 2% 6,788,000 79% Rx 872,000 10% No Rx 288,000 3% Diagnosed population Large population (~1.8 million) VeteransMajority receive drug treatment TreatedCivilians: ~75%2 in Vetera

  12. Slide 12: What Drug Classes are Used to Treat PTSD?

    What Drug Classes are Used to Treat PTSD? Market highly fragmented, with benzodiazepines being widely prescribed (but not indicated1) • Multiple medications per patient (or “Polypharmacy”) is the norm • Approximately 55% of patients receive a benzodiazepine, and 53% receive an selective serotonin re

  13. Slide 13: Relevance of Sleep Disturbances for PTSD

    Relevance of Sleep Disturbances for PTSD Sleep disturbances: • Core symptom of PTSD (component of intrusion and hyperarousal symptom clusters) • Believed to play roles in the pathophysiology of PTSD om Sleep Pharmacological Therapeutic As a Core Pathophysiology Action Benefit Symptom 2o Clinical 1o

  14. Slide 14: TNX-102 SL: Innovative and Unique By Design

    TNX-102 SL: Innovative and Unique By Design Active Pharmaceutical Ingredient (API) is cyclobenzaprine (CBP), is structurally related to tricyclic antidepressants (TCAs) • TCAs and their metabolites differ widely in their receptor binding and pharmacokinetic profiles1 • CBP is more selective for high

  15. Slide 15: Why Initially Target Military-Related PTSD?

    Why Initially Target Military-Related PTSD? Military-related PTSD not well-served by existing FDA-approved therapies • No clear treatment response observed in U.S. military population Sertraline: failed to show efficacy in a large multicenter trial in U.S. military (placebo numerically better)1 Paro

  16. Slide 16: Phase 2 AtEase Study in Military PTSD

    Phase 2 AtEase Study in Military PTSD • Randomized, double-blind, placebo- • Enrolled patients with baseline controlled trial in military-related PTSD CAPS-5 29 • Analysis from 231 patients; 24 U.S. clinical sites TNX-102 SL at bedtime once-daily • TNX-102 SL was active at 5.6 mg dose 2.8 mg N= 90 T

  17. Slide 17: AtEase Study Demographics and Characteristics

    AtEase Study Demographics and Characteristics 93% of the randomized patients were male 98% had trauma during military service Deployed an average of 2.3 times Mean time since index trauma was 7 years Race and ethnicity generally consistent with U.S. military distribution Current Major Depressive Dis

  18. Slide 18: AtEase Study: Traumas Associated with PTSD

    AtEase Study: Traumas Associated with PTSD Index Trauma During Military ServicePatient Count* Being involved in an IED explosion or suicide bombing 35 Witness death or injury of fellow soldiers 33 Witnessing IED explosion 30 Receiving incoming artillery, rocket, or mortar fire 29 Being wounded or in

  19. Slide 19: AtEase Study Efficacy Analysis p-Values

    Assessment Domain Analysis p-Values 2.8 mg (N=90) 5.6 mg (N=49) CAPS-5 Total MMRM 0.259^ 0.053 Total MMRM with Multiple Imputation 0.211 0.031* Total MMRM w/ Hybrid LOCF/BOCF 0.172 0.037* Total ANCOVA 0.090 0.038=" CAPS-5 clusters/items Arousal & Reactivity cluster (E) MMRM 0.141 0.048* Sleep item (

  20. Slide 20: AtEase Study: Safety and Tolerability Profile

    AtEase Study: Safety and Tolerability Profile No serious adverse events reported with TNX-102 SL deemed related to treatment Placebo TNX-102 SL 2.8 mg TNX-102 SL 5.6 mg Systemic Adverse Events* (N=94) (N=93) (N=50) Somnolence 6.4% 11.8% 16.0% Dry Mouth 10.6% 4.3% 16.0% Headache 4.3% 5.4% 12.0% Insom

  21. Slide 21: AtEase: Retrospective Analysis for TNX-102 SL 5.6 mg

    AtEase: Retrospective Analysis for TNX-102 SL 5.6 mg Prior pharmacotherapy trials in PTSD used earlier versions of CAPS (e.g. CAPS-2) • Different scoring range from CAPS-5 • Most frequently used a score of 50 for entry (similar to CAPS-5 331) • FDA has accepted this higher entry criteria (CAPS-5 33)

  22. Slide 22: Retrospective Analysis of CAPS-5 with entry CAPS-5 ≥ 33

    Retrospective Analysis of CAPS-5 in patients with entry CAPS-5 ≥ 331 -30.0 -25.0 -20.0 -15.0 -10.0 -5.0 0.0 Wk 0 Wk 2 Wk 4 Wk 6 Wk 8 Wk 10 Wk 12 LS Mean (SE) of CAPS-5 Total Score Placebo TNX-102 SL 2.8 mg TNX-102 SL 5.6 mg 6.8 point difference in CAPS-5 change from baseline between placebo and TNX-

  23. Slide 23: Retrospective Analyses of Week 12 outcome measures for TNX-102 SL 5.6 mg

    © 2017 Tonix Pharmaceuticals Holding Corp 23 Retrospective Analyses of Week 12 outcome measures for TNX-102 SL 5.6 mg vs. placebo in military-related or combat PTSD subset and for CAPS-5 ≥ 29 and CAPS-5 ≥ 33 at baseline Military-relatedPTSD CombatPTSD CAPS-5 at Baseline ≥ 29 ≥ 33 ≥ 29 ≥ 33 N:PBO/TNX

  24. Slide 24: Phase 2 AtEase Study Conclusions

    Phase 2 AtEaseStudy Conclusions First large multi-center trial demonstrating efficacy of an Investigational New Drug (IND) in military-related PTSD TNX-102 SL therapeutic dose (5.6 mg) identified Symptom reduction (CAPS-5) Functional improvement (Sheehan Disability Scale domains) Clinical global imp

  25. Slide 25: Planned Phase 3 Program in PTSD

    Planned Phase 3 Program in PTSD To confirm AtEase findings in military-related PTSD: • Larger adaptive design study • Targeting start in 1Q 2017 TNX-102 SL once-daily at bedtime 5.6 mg N ~ 275 (90*) Placebo once-daily at bedtime N ~ 275 (90*) 12 weeks * First interim analysis General study character

  26. Slide 26: Commercialization Options

    Commercialization Options Tonix is exploring a variety of options to commercialize TNX-102 SL, including commercializing on our own or partnering all or some indications in specific regions of the world. Commercial considerations: • Primary physician audience is well defined: psychiatrists (~30,000

  27. Slide 27: Intellectual Property

    Intellectual Property TNX-102 SL Portfolio of international patents filed under the Patent Cooperation Treaty (PCT) Composition-of-matter (eutectic) • Patents filed • Protection expected to 2034 Pharmacokinetics (PK) • Patents filed • Protection expected to 2033 Method-of-use • PTSD: patents filed ©

  28. Slide 28: Product Pipeline

    Product Pipeline Preclinical Phase 2 NDA/BLA Market Candidate Indication Phase 3 TNX-102 SL PTSD 505(b)(2) Phase 2 results reported May, 2016 PTSD TNX-601 505(b)(1) Novel polymorph and salt discovered and characterized TNX-801 Biologic Novel virus vaccine discovered and characterized Smallpox vaccin

  29. Slide 29: TNX-601 - A Potential Clinical Candidate for PTSD

    TNX-601- A Potential Clinical Candidate for PTSD TNX-601 (tianeptine oral formulation) • Targeted as the 1st line monotherapy for PTSD: daytime dosing Leverages expertise in PTSD (clinical and regulatory experience, market analysis, etc.)Mechanism of Action (MOA) is different from TNX-102 SL Preclin

  30. Slide 30: TNX-601 - A Potential Clinical Candidate for PTSD

    TNX-601- A Potential Clinical Candidate for PTSD TNX-601 (tianeptine oral formulation) • Targeted as the 1st line monotherapy for PTSD: daytime dosing Leverages expertise in PTSD (clinical and regulatory experience, market analysis, etc.)Mechanism of Action (MOA) is different from TNX-102 SL Preclin

  31. Slide 31: TNX-601 Mechanism of Action and Unique Characteristics

    . Tianeptinemodulates the glutamatergic system indirectlyDoes not have significant affinity (Ki>10μM) for NMDA2or AMPA3receptors . . Tianeptine is a weak μ-opioid receptor (MOR) agonistControlled substance in France, Belgium and Singapore . . Tianeptine has been shown to oppose deleterious effects o

  32. Slide 32: TNX-801 - A Potential Smallpox Preventing Vaccine

    TNX-801 -A Potential Smallpox Preventing Vaccine Pre-IND Candidate TNX-801 (live virus vaccine) •Targeting a biological warfare threat: smallpox xLeverages government affairs effort •Tonix has identified a new vaccine with improved properties xPatent application on novel vaccine submitted Regulatory

  33. Slide 33: TNX-801 Mechanism of Action and Unique Characteristics

    . Live virus vaccines stimulate cross-reactive immunityProtects from consequences of infection with variolavirus (smallpox agent) . Renders recipient “immune” and protects population (even un-immunized) by decreasing spread of infection . TNX-801 -A Potential Smallpox Preventing Vaccine . Potential

  34. Slide 34: Financial overview

    Financial overview NASDAQ: TNXP Cash, cash equivalents, and marketable $26.7 million securities reported at September 30, 2016 Net proceeds from underwritten offering in 4Q16 $4.6 million Shares outstanding (January 3, 2017) 39.2 million © 2017 Tonix Pharmaceuticals Holding Corp

  35. Slide 35: Management Team

    Management Team Seth Lederman, MD President & CEO Gregory Sullivan, MD Chief Medical Officer Bradley Saenger, CPA Chief Financial Officer Jessica Morris EVP, Operations © 2017 Tonix Pharmaceuticals Holding Corp

  36. Slide 36: Board of Directors

    Board of Directors Seth Lederman, MD Ernest Mario, PhD Chairman ALZA, Glaxo, Reliant Pharma Stuart Davidson Charles Mather Labrador Ventures, Alkermes, Combion BTIG, Janney, Jefferies, Cowen, Smith Barney Patrick Grace John Rhodes Apollo Philanthropy, WR Grace, Chemed NYSERDA, NRDC, Booz Allen Hamil

  37. Slide 37: Milestones – recent and upcoming

    Milestones – recent and upcoming TNX-102 SL – Posttraumatic Stress Disorder December 2015 Entered into Collaborative Research and Development Agreement (CRADA) with the United States Army Medical Materiel Development Activity (USAMMDA)May 2016 Reported results from AtEase studyAugust 2016 End-of-Pha

  38. Slide 38: Summary

    Summary Strong position for value growth with Phase 3 trial in a major medical indication: PTSD including military-related PTSD • Phase 3 asset not previously well-known to the investor marketplace TNX-102 SL for PTSD is designated as a Breakthrough Therapy by FDA • Accelerated development and appro

  39. Slide 39: Thank you!

    NASDAQ: TNXP Thank you! © 2017 Tonix Pharmaceuticals Holding Corp

Tonix Pharmaceuticals Holding — Investor Presentation (January 2017) on 6ix