Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP

Tonix Pharmaceuticals — IPO Roadshow (November 2019)

26 slides from Tonix Pharmaceuticals's IPO roadshow presentation, Nov 8, 2019

  1. Slide 1: Phase 3 Trial of Bedtime Sublingual Cyclobenzaprine (TNX-102 SL) for Fibromyalgia

    © 2019 Tonix Pharmaceuticals Holding Corp. Gregory Sullivan, MD Chief Medical Officer Tonix Pharmaceuticals Inc presenting at American College of Rheumatology Annual Meeting, Atlanta, GA November 10, 2019 A Phase 3 Randomized, Double - Blind, Placebo - Controlled Trial of Bedtime Sublingual Cycloben

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

    © 2019 Tonix Pharmaceuticals Holding Corp. 2 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: Free Writing Prospectus Statement

    © 2019 Tonix Pharmaceuticals Holding Corp. 3 Free Writing Prospectus Statement This presentation highlights basic information about us and the offering to which this communication relates. Because it is a summary, it does not contain all of the information that you should consider before investing i

  4. Slide 4: Disclosure Statements

    © 2019 Tonix Pharmaceuticals Holding Corp. 4 Disclosure Statements • Gregory Sullivan MD is an employee of Tonix Pharmaceuticals Inc and owns stock and stock options in the company • The co - authors on this work are employees or consultants of Tonix Pharmaceuticals Inc • TNX - 102 SL is an investig

  5. Slide 5: Rationale for Targeting Sleep Quality

    © 2019 Tonix Pharmaceuticals Holding Corp. 5 TNX - 102 SL for the Treatment of Fibromyalgia Rationale for Targeting Sleep Quality • Inter - relationship between poor sleep quality and fibromyalgia (FM) described almost 50 years ago 1 • Moldofsky & Smythe identified anomalous alpha - rhythms in stage

  6. Slide 6: Non-restorative Sleep is Common in Fibromyalgia and May Contribute to Pain Symptoms

    © 2019 Tonix Pharmaceuticals Holding Corp. 6 Non - restorative Sleep is Common in Fibromyalgia and May Contribute to Pain Symptoms 1 » Increased number of arousals leading to fragmented sleep » Non - restorative sleep or waking feeling unrefreshed » Diffuse stiffness, aching and fatigue, even after

  7. Slide 7: What is TNX-102 SL?

    © 2019 Tonix Pharmaceuticals Holding Corp. 7 What is TNX - 102 SL? » TNX - 102 SL is a sublingual eutectic formulation 1 of cyclobenzaprine (CBP) for transmucosal absorption » CBP is a tricyclic molecule with high affinity for target receptors that play key roles in sleep physiology and restorative

  8. Slide 8: TNX-102 SL: Sublingual Cyclobenzaprine Tablet

    © 2019 Tonix Pharmaceuticals Holding Corp. 8 TNX - 102 SL: Sublingual Cyclobenzaprine Tablet » Faster Absorption » Bypasses “First - Pass” Hepatic Metabolism » Reduced metabolism of parent CBP to active metabolite nCBP

  9. Slide 9: Phase 3 AFFIRM Study Design

    © 2019 Tonix Pharmaceuticals Holding Corp. 9 Phase 3 AFFIRM Study of TNX - 102 SL 2.8 mg in Fibromyalgia TNX - 102 SL at bedtime once - daily Placebo at bedtime once - daily 12 weeks N= 262 N= 257 2.8 mg General S tudy C haracteristics: • 12 - week, r andomized, double - blind, placebo - controlle d

  10. Slide 10: Primary Analysis of Pain Response in FM

    © 2019 Tonix Pharmaceuticals Holding Corp. 10 AFFIRM Study Results: Primary Analysis » Primary Analysis of Pain Response in FM (TNX - 102 SL N=262; Placebo N=257) » Logistic regression (LR) responder analysis (≥30% pain reduction); all discontinuations considered non - responders » TNX - 102 SL 28.6

  11. Slide 11: Pre-Planned Sensitivity Analyses Assessed Impact of Missing Data Handling

    © 2019 Tonix Pharmaceuticals Holding Corp. 11 AFFIRM Study Results: Pre - Planned Sensitivity Analyses Assessed Impact of Missing Data Handling on Primary Efficacy Outcome » Sensitivity Analysis 1 – Treatment of missing data (TNX - 102 SL N=262; Placebo N=257) » Responder analysis by LR; discontinua

  12. Slide 12: Imbalance in Patient Discontinuations

    © 2019 Tonix Pharmaceuticals Holding Corp. 12 » Unexpected imbalance in patient discontinuations for reasons unrelated to efficacy or tolerability ( eg , patient relocating) created a negative bias in the primary responder analysis because any patient who left the study for any reason, prior to comp

  13. Slide 13: Supporting Primary Efficacy Analyses

    © 2019 Tonix Pharmaceuticals Holding Corp. 13 AFFIRM Study Results: Supporting Primary Efficacy Analyses » Primary Analysis in Per Protocol (PP) Population (TNX - 102 SL N=179; Placebo N=200) » Responder analysis in PP population; all discontinuations are non - responders (like primary analysis) » P

  14. Slide 14: Additional Pain Analysis and Overall Conclusions on Negative Primary Outcome

    © 2019 Tonix Pharmaceuticals Holding Corp. 14 » ≥50% Responder Analysis (all discontinuations treated as non - responders) » TNX - 102 SL 14.5% ; Placebo 8.6% ; OR (95% CI) of 1.82 (1.04, 3.17), p=0.035 » Overall Conclusion On Negative Outcome of the Primary Efficacy Analysis: » Unexpected imbalance

  15. Slide 15: Change in Pain Scores Over 12 Weeks

    © 2019 Tonix Pharmaceuticals Holding Corp. 15 Effect Size # = 0.30 *p<.05; **p<.01; ***p<.001 # Cohen’s d for observed mean (SD) changes at Week 12 -1.8 -1.6 -1.4 -1.2 -1 -0.8 -0.6 -0.4 -0.2 0 0 1 2 3 4 5 6 7 8 9 10 11 12 LS Mean Difference (SE) in NRS Pain Score Study Week Change in Pain Scores Ove

  16. Slide 16: Patient Global Impression of Change (PGIC)

    © 2019 Tonix Pharmaceuticals Holding Corp. 16 • Responder status defined as a patient rating of 2 (‘much improved’) or 1 (‘very much improved’) • Significantly greater percentage of responders to TNX - 102 SL 2.8 mg than placebo at Week 12 (p=0.038) • Activity of TNX - 102 SL 2.8 mg in FM cross - va

  17. Slide 17: Fibromyalgia Impact Questionnaire – Revised (FIQ-R)

    © 2019 Tonix Pharmaceuticals Holding Corp. AFFIRM Study Results: Fibromyalgia Impact Questionnaire – Revised (FIQ - R) -16 -14 -12 -10 -8 -6 -4 -2 0 Wk0 Wk2 Wk4 Wk8 Wk12 LS MCFB (SE) of FIQR Symtpoms Week FIQ - R Symptom Domain Placebo (N=257) TNX-102 SL 2.8 mg (N=262) ** *** *** * -12 -10 -8 -6 -4

  18. Slide 18: Early Effects on Sleep Quality

    © 2019 Tonix Pharmaceuticals Holding Corp. 18 AFFIRM Study Results: Early Effects on Sleep Quality of TNX - 102 SL -9 -8 -7 -6 -5 -4 -3 -2 -1 0 Wk0 Wk2 Wk4 Wk8 Wk12 LS Mean (SE) Improvement in T - Score PROMIS Sleep Disturbance T - Scores Placebo (N=257) TNX-102 SL 2.8 mg (N=262) *** *** *** * Daily

  19. Slide 19: Effects on Sleep Quality as Recorded on Daily Diary

    © 2019 Tonix Pharmaceuticals Holding Corp. 19 AFFIRM Study Results: TNX - 102 SL Effects on Sleep Quality as Recorded on Daily Diary -2 -1.8 -1.6 -1.4 -1.2 -1 -0.8 -0.6 -0.4 -0.2 0 0 1 2 3 4 5 6 7 8 9 10 11 12 LS Mean Difference (SE) in NRS Sleep Score Study Week Sleep Quality Diary Scores, MMRM Pla

  20. Slide 20: Effects on Fatigue in FM

    © 2019 Tonix Pharmaceuticals Holding Corp. 20 AFFIRM Study Results: TNX - 102 SL Effects on Fatigue in FM » Fatigue is a core FM symptom » Fatigue is 1 of 3 symptoms that, if experienced over the past week, can contribute up to 3 points to the symptom severity score for diagnosis 1 » TNX - 102 SL 2.

  21. Slide 21: PROMIS Global Health – Short Form

    © 2019 Tonix Pharmaceuticals Holding Corp. AFFIRM Study Results: PROMIS Global Health – Short Form *p<0.05; **p<0.01 • Higher scores reflect improved health on PROMIS Global Health • TNX - 102 SL 2.8 mg improved Global Physical Health domain by last four weeks of study, at Weeks 8 and 12 • TNX - 102

  22. Slide 22: Safety and Tolerability

    © 2019 Tonix Pharmaceuticals Holding Corp. 22 » Trial Completion Rates: 86.4% Placebo; 77.5% TNX - 102 SL 2.8 mg » TNX - 102 SL was well - tolerated in AFFIRM and AEs reported were similar to other TNX - 102 SL studies » Total of 7 serious adverse events (SAEs) were reported: 4 in placebo group and

  23. Slide 23: Dose and Tolerability Learned from PTSD Studies

    © 2019 Tonix Pharmaceuticals Holding Corp. 23 What was learned about dose and tolerability from studies of TNX - 102 SL in PTSD? • For overall PTSD symptoms, TNX - 102 SL 5.6 mg showed an efficacy signal, whereas the 2.8 mg dose did not • The most common AE with TNX - 102 SL, oral hypoaesthesia, occ

  24. Slide 24: Evidence from Military PTSD Study of Higher Dose Requirement for Pain

    © 2019 Tonix Pharmaceuticals Holding Corp. 24 Evidence from Military PTSD Study of Higher Dose Requirement for Pain Dose Effect on Past 24 - hr Average Pain in PTSD Patient with Baseline Pain ≥4 -2.5 -2 -1.5 -1 -0.5 0 Wk 0 Wk 2 Wk 4 Wk 6 Wk 8 Wk 10 Wk 12 In - Clinic 24 hr LS MCFB Pain Score Study We

  25. Slide 25: Conclusions

    © 2019 Tonix Pharmaceuticals Holding Corp. 25 Conclusions » The AFFIRM Study in fibromyalgia (FM) did not achieve significance on the primary endpoint » While the study was not significant base on the pre - specified pain responder analysis, it was significant by other standard pain analysis techniq

  26. Slide 26: Thank you! Co-Authors

    © 2019 Tonix Pharmaceuticals Holding Corp. Thank you ! Co - Authors: R. Michael Gendreau, Gendreau Consulting Judith Gendreau, Gendreau Consulting Ashild Peters, Tonix Pharmaceuticals Inc Perry Peters, Tonix Pharmaceuticals Inc Seth Lederman, Tonix Pharmaceuticals Inc

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