Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP

Tonix Pharmaceuticals — MHSRS 2024

27 slides from Tonix Pharmaceuticals's conference presentation, Aug 27, 2024

  1. Slide 1: TNX-102 SL (sublingual cyclobenzaprine) for Fibromyalgia

    TNX-102 SL (sublingual cyclobenzaprine) for Fibromyalgia MHSRS Presentation for Breakout Session: “Assuaging Agony: Novel Pain Therapeutics” NASDAQ: TNXP Version P0583 August 27, 2024 (Doc 1481) © 2024 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: Disclosure

    Disclosure Title: Efficacy and Safety of Bedtime TNX-102 SL (Sublingual Cyclobenzaprine HCl) for the Management of Fibromyalgia: Results from the Confirmatory Phase 3 Randomized, Double-Blind, Placebo-Controlled RESILIENT Trial Authors: Seth Lederman, MD,1 Mary Kelley, MPH,1 Jean Engels, MS,1 Ben Va

  4. Slide 4: About Fibromyalgia

    About Fibromyalgia CNS PORTFOLIO Fibromyalgia is a chronic pain disorder resulting from amplified sensory and pain signaling within the CNS1 Fibromyalgia is a syndrome comprised of the symptoms: chronic widespread pain, nonrestorative sleep, and fatigue Fatigue Multisite Non-Restorative Sleep pain F

  5. Slide 5: The Third Primary Type of Pain: Nociplastic Pain

    The Third Primary Type of Pain: Nociplastic Pain1-4 CNS PORTFOLIO Nociplastic syndrome includes: (1) widespread pain (2) fatigue Nociplastic Pain (3) sleep disturbances Pathological Pain (4) cognitive dysfunction (“brain fog”) Examples: Mechanism: Fibromyalgia Altered pain ME/CFS perception in the M

  6. Slide 6: Fibromyalgia is a Large, Underserved and Dissatisfied Population

    Fibromyalgia is a Large, Underserved and Dissatisfied Population CNS PORTFOLIO • ~10 million U.S. adults are affected – predominantly women1,2 ‒ Debilitating and life altering condition ‒ Significant economic impact • Patients are dissatisfied, despite three FDA approved drugs3,4 ‒ 85% of patients f

  7. Slide 7: Many Fibromyalgia Patients are Prescribed Off-Label Drugs

    Many Fibromyalgia Patients are Prescribed Off-Label Drugs; ~53% FM Patients Prescribed Opioids Off-Label1,2 CNS PORTFOLIO % FM Patients (after index3 date) 50% 45% 40% 35% Off-Label 30% Off-Label Off-Label FDA Approved 25.50% Opioid Opioid 25% 22.62% Off-Label 20.47% Off-Label 19.45% 20% Opioid 17.5

  8. Slide 8: Poor Sleep and Pain have Bi-directional Reinforcing Effects

    Poor Sleep and Pain have Bi-directional Reinforcing Effects1 CNS PORTFOLIO • Poor sleep and pain form a vicious cycle in driving fibromyalgia decompensation • Can’t sleep → worse pain / In pain → can’t sleep • Poor sleep and pain contribute to persistence, chronicity and severity • Syndrome includes

  9. Slide 9: Fibromyalgia: Unrefreshing Sleep and Cyclobenzaprine Treatment

    Fibromyalgia: Unrefreshing Sleep and Cyclobenzaprine Treatment CNS PORTFOLIO • Non-restorative sleep1,2 ‒ Harvey Moldofsky – recognition of unrefreshing/non-restorative sleep:  Symptom  Potential causative or potentiating factor • Cyclobenzaprine3,9 ‒ Potentially the earliest drug studied in fibro

  10. Slide 10: Tonmya (TNX-102 SL, Cyclobenzaprine HCl Sublingual Tablets)

    Tonmya (TNX-102 SL, Cyclobenzaprine HCl Sublingual Tablets)1 CNS PORTFOLIO • Non-opioid analgesic designed for long-term daily bedtime use in fibromyalgia patients ‒ Targets non-restorative sleep ‒ Potent binding and antagonist activities at the serotonin-5-HT2A, α1-adrenergic, histaminergic-H1, and

  11. Slide 11: Fibromyalgia Program Status

    Fibromyalgia Program Status CNS PORTFOLIO Tonmya (TNX-102 SL) Statistically Significant 2nd Phase 3 Topline Results Fibromyalgia Cyclobenzaprine Protectic® Reported 4Q’23 Sublingual Tablets First pivotal Phase 3 study (RELIEF) reported – December 20201 Second Phase 3 study (RALLY) missed primary end

  12. Slide 12: Tonmya (TNX-102 SL): Phase 3 RESILIENT Study Design

    Tonmya (TNX-102 SL): Phase 3 RESILIENT Study Design CNS PORTFOLIO General study characteristics: • Randomized, double-blind, multicenter, placebo-controlled study in fibromyalgia • 33 U.S. sites enrolled 457 participants with fibromyalgia as defined by 2016 Revisions to the 2010/2011 FM Diagnostic C

  13. Slide 13: Summary of Primary and Key Secondary Endpoints

    RESILIENT Summary of Primary and Key Secondary Endpoints CNS PORTFOLIO Endpoint P-value Effect Size (ES) Primary Endpoint Daily Diary Pain ratings p = 0.00005 ES = 0.38 Key Secondary Endpoints Patient Global Impression of Change (PGIC), responders p = 0.00013 -- Fibromyalgia Impact Questionnaire – S

  14. Slide 14: Primary Outcome Measure: Reduction in Widespread Pain

    RESILIENT Primary Outcome Measure Reduction in Widespread Pain CNS PORTFOLIO Weekly Average of Daily Diary NRS Ratings of Average Pain Over Prior 24 Hours 0 Placebo (N=225) TNX-102 SL (N=231) -0.2 LS Mean (SE) Change in NRS Pain Score -0.4 -0.6 -0.8 * -1 -1.2 *** -1.4 *** -1.6 *** -1.8 *** ** *** **

  15. Slide 15: Continuous Pain Responder Graph

    RESILIENT Continuous Pain Responder Graph CNS PORTFOLIO 100 Placebo TNX-102 SL 90 30% Responders# 80 (45.9% vs 27.1%) p < 0.001 70 Percent of Subjects 60 50 50% 40 Responders# 30 (22.5% vs. 13.3%) p = 0.011 20 10 0 ≥0 ≥ 10% ≥ 20% ≥ 30% ≥ 40% ≥ 50% ≥ 60% ≥ 70% ≥ 80% ≥ 90% ≥ 100% Percentage Redution i

  16. Slide 16: Patient Global Impression of Change

    RESILIENT Patient Global Impression of Change Key Secondary Outcome Measure CNS PORTFOLIO Patient Global Impression of Change Responder Analysis Percent Much Improved or Very Much Improved 50% Placebo (N=225) TNX-102 SL (N=231) 45% ** 40% ** ** 35.1% 35% 33.3% 30.7% 30% 25% * 19.1% 19.1% 20% 16.5% 1

  17. Slide 17: FIQ-R Symptoms Domain

    RESILIENT FIQ-R Symptoms Domain Key Secondary Outcome Measure CNS PORTFOLIO Fibromyalgia Impact Questionnaire – Revised (FIQ-R) Symptoms Domain 0 LS Mean (SE) Change in FIQ-R Symptoms Placebo (N=225) TNX-102 SL (N=231) -2 -4 -6 -8 -10 -12 -14 *** -16 *** * -18 *** -20 Week 0 Week 2 Week 6 Week 10 We

  18. Slide 18: FIQ-R Function Domain

    RESILIENT FIQ-R Function Domain Key Secondary Outcome Measure CNS PORTFOLIO Fibromyalgia Impact Questionnaire – Revised (FIQ-R) Function Domain 0 LS Mean (SE) Change in FIQ-R Function Placebo (N=225) TNX-102 SL (N=231) -2 -4 -6 -8 -10 -12 ** -14 ** ** -16 Week 0 Week 2 Week 6 Week 10 Week 14 **p<0.0

  19. Slide 19: PROMIS Sleep Disturbance Inventory

    RESILIENT PROMIS Sleep Disturbance Inventory Key Secondary Outcome Measure CNS PORTFOLIO PROMIS Sleep Disturbance 0 LS Mean (SE) Change in PROMIS SD T-score Placebo (N=225) TNX-102 SL (N=231) -1 -2 -3 -4 -5 -6 -7 -8 *** -9 *** * *** -10 Week 0 Week 2 Week 6 Week 10 Week 14 *p<0.001; ***p<0.00001 Wee

  20. Slide 20: PROMIS Fatigue Inventory

    RESILIENT PROMIS Fatigue Inventory Key Secondary Outcome Measure CNS PORTFOLIO PROMIS Fatigue 0 LS Mean (SE) Change in PROMIS Fatigue T-Score Placebo (N=225) TNX-102 SL (N=231) -1 -2 -3 -4 -5 -6 -7 *** -8 * *** *** -9 Week 0 Week 2 Week 6 Week 10 Week 14 *p<0.01; ***p<0.0001 Week 14 LS mean (SE) cha

  21. Slide 21: Sleep Quality by Daily Diary

    RESILIENT Sleep Quality by Daily Diary Key Secondary Outcome Measure CNS PORTFOLIO Weekly Average of Daily Diary NRS Ratings of Prior Night Sleep Quality 0 Placebo (N=225) TNX-102 SL (N=231) LS Mean (SE) Change in NRS Sleep Quality Score -0.2 -0.4 -0.6 -0.8 * -1 ** -1.2 -1.4 *** -1.6 *** *** -1.8 **

  22. Slide 22: Summary of Efficacy

    RESILIENT Summary of Efficacy CNS PORTFOLIO Fibromyalgia is the prototypic noicplastic syndrome composed of symptoms • Widespread pain • Fatigue • Sleep disturbance Efficacy across symptoms of pain, fatigue and sleep • Pain: primary endpoint, daily pain diary: p-value of 0.00005 • Fatigue: PROMIS fa

  23. Slide 23: Safety

    RESILIENT Safety CNS PORTFOLIO Treatment-Emergent Adverse Events (TEAEs) at Rate of ≥ 3% in Either Treatment Group System Organ Class TNX-102 SL Placebo Total* Preferred Term N=231 N=226 N=457 Systemic Adverse Events COVID-19 10 (4.3%) 7 (3.1%) 17 (3.7%) Somnolence 7 (3.0%) 3 (1.3%) 10 (2.2%) Headac

  24. Slide 24: Safety, Continued

    RESILIENT Safety, Continued CNS PORTFOLIO No Signals for Clinically Meaningful Changes in Systolic or Diastolic Blood Pressure or in Weight No clinically meaningful difference in mean systolic blood pressure between groups Week 14 mean (SD) change from baseline: TNX-102 SL = 0.7 (12.38) mmHg Placebo

  25. Slide 25: Large Need for New Fibromyalgia Therapies with Better Tolerability

    Large Need for New Fibromyalgia Therapies that Provide Broad Symptom Improvement with Better Tolerability CNS PORTFOLIO • Currently-approved medications may have side effects that limit long-term use1 • Many patients skip doses or discontinue altogether within months of treatment initiation • Medica

  26. Slide 26: Tonmya Showed Broad-Spectrum Activity and was Well Tolerated

    Tonmya Showed Broad-Spectrum Activity and was Well Tolerated CNS PORTFOLIO Cymbalta® Lyrica® Tonmya Savella® Pain + + + Activity Sleep + - + Fatigue - + + Insomnia - + - Fatigue + - - Systemic Weight + - - Tolerability Issues Blood Pressure - + - Sexual function - + - GI issues - + - • Tonmya showed

  27. Slide 27: Thank You

    THANK YOU © 2023 Tonix Pharmaceuticals Holding Corp.

Tonix Pharmaceuticals Holding — MHSRS 2024 on 6ix