Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP

Tonix Pharmaceuticals — Investor Presentation (March 2019)

42 slides from Tonix Pharmaceuticals's investor presentation, Mar 5, 2019

  1. Slide 1: Investor Presentation March 2019

    © 2019 Tonix Pharmaceuticals Holding Corp. March 2019 Version P0160 3 - 5 - 19 (Doc 0438) Investor Presentation

  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: Tonix Development Highlights

    © 2019 Tonix Pharmaceuticals Holding Corp. 3 Posttraumatic Stress Disorder (PTSD) – Lead program; new bedtime treatment - Tonmya ®1 • P302/RECOVERY Phase 3 clinical study with Week 4 primary endpoint to initiate in 1Q2019 • Results from 2 efficacy studies improve the new Phase 3 study design New Pha

  4. Slide 4: Candidates in Development

    © 2019 Tonix Pharmaceuticals Holding Corp. 4 Candidates in Development Preclinical Phase 2 NDA 1 /BLA 2 Market Pipeline Product Indication Phase 3 TNX - 102 SL Bedtime Treatment for PTSD – Tonmya ® Daytime Treatment for PTSD TNX - 601 Novel polymorph and salt discovered and characterized; Preliminar

  5. Slide 5: Tonmya: a Potential Bedtime Treatment for PTSD

    © 2019 Tonix Pharmaceuticals Holding Corp. 5 Tonmya : a Potential Bedtime Treatment for PTSD First investigational new drug to show treatment effect in military - related PTSD in two potential pivotal efficacy studies • Phase 2 study (P201/ AtEase ) showed Tonmya 5.6 mg had a strong signal of treatm

  6. Slide 6: No Recognized Abuse Potential in Clinical Studies

    © 2019 Tonix Pharmaceuticals Holding Corp. 6 No Recognized Abuse Potential in Clinical Studies Active ingredient is cyclobenzaprine, which is structurally related to tricyclic antidepressants • Cyclobenzaprine interacts with receptors that regulate sleep quality: 5 - HT 2A, a 1 - adrenergic and hist

  7. Slide 7: TNX-102 SL Intellectual Property – U.S. Protection until 2034

    © 2019 Tonix Pharmaceuticals Holding Corp. 7 TNX - 102 SL Intellectual Property – U.S. Protection until 2034 Composition of matter (eutectic) : Protection expected to 2034 • United States Patent and Trademark Office (USPTO) issued U.S. Patent No. 9,636,408 in May 2017 U.S. Patent No. 9,956,188 in Ma

  8. Slide 8: TNX-102 SL: Sublingual Formulation Designed for Bedtime Administration

    © 2019 Tonix Pharmaceuticals Holding Corp. 8 TNX - 102 SL: Sublingual Formulation is Designed for Bedtime Administration TNX - 102 SL: Proprietary sublingual formulation of cyclobenzaprine (CBP) with transmucosal absorption • Innovation by design with patent protected CBP/mannitol eutectic • Rapid s

  9. Slide 9: Tonmya: Hypothesized Novel Mechanism Targets Sleep Quality

    © 2019 Tonix Pharmaceuticals Holding Corp. 9 Tonmya: Hypothesized Novel Mechanism Targets Sleep Quality for Recovery from PTSD PTSD is a disorder of recovery • Most people exposed to extreme trauma recover over a few weeks • In PTSD, recovery process impeded due to insufficient sleep - dependent mem

  10. Slide 10: PTSD: Not Well-Served by Approved Treatments

    © 2019 Tonix Pharmaceuticals Holding Corp. 10 PTSD: Not Well - Served by Approved Treatments FDA - approved SSRIs, paroxetine and sertraline, are indicated as a treatment for PTSD • Neither drug has shown efficacy in military - related PTSD • Majority of male PTSD patients unresponsive or intolerant

  11. Slide 11: Prevalence of PTSD Among Civilians and Veterans

    © 2019 Tonix Pharmaceuticals Holding Corp. 11 Prevalence of PTSD Among Civilians and Veterans 1 Goldstein et al., 2016 (adjusted for 2019) ; 2 Norris, PTSD Res Quar . 2013; 3 Analysis of VA Health Care Utilization among Operation Enduring Freedom, Operation Iraqi Freedom, and Operation New Dawn Vete

  12. Slide 12: Phase 2 P201/AtEase Study in Military-Related PTSD

    © 2019 Tonix Pharmaceuticals Holding Corp. 12 Phase 2 P201/AtEase 1 Study in Military - Related PTSD • Randomized, double - blind, placebo - controlled trial in military - related PTSD • Efficacy analysis from 231 * patients; 24 U.S. clinical sites • Enrolled patients with baseline CAPS - 5 2 ≥ 29 •

  13. Slide 13: P201/AtEase Study

    © 2019 Tonix Pharmaceuticals Holding Corp. 13 P201/AtEase Study P201 was a large adequate well - controlled Phase 2 study in military - related PTSD • Primary endpoint (Week 12 CAPS - 5) did not separate from placebo for TNX - 102 SL 2.8 mg • No safety or tolerability issue discovered • Retrospectiv

  14. Slide 14: P301/HONOR Study Design

    © 2019 Tonix Pharmaceuticals Holding Corp. 14 P301/HONOR 1 Study – Evidence of Efficacy at Week 4 Discontinued Due to High Placebo Response at Week 12 Primary e ndpoint CAPS - 5 2 : • Mean change from baseline at W eek 12 (Tonmya 5.6 mg vs. placebo ) Unblinded interim analysis at 274 randomized part

  15. Slide 15: P301/HONOR Study Stopped After Interim Analysis

    © 2019 Tonix Pharmaceuticals Holding Corp. 15 P301/HONOR Study Stopped After Interim Analysis (July 2018) P301 was a large adequate well - controlled Phase 3 study in military - related PTSD • Separation on primary endpoint at Week 12 did not cross pre - specified study continuation threshold at Wee

  16. Slide 16: Tonmya Phase 2 Dose-Effect in Military-Related PTSD

    © 2019 Tonix Pharmaceuticals Holding Corp. 16 Tonmya Phase 2 Dose - Effect in Military - Related PTSD 1 1 Completed Phase 2 P201/AtEase study: Retrospective analysis of Tonmya 5.6 mg on CAPS - 5 ≥33 (high - moderate) subgroup. Primary analysis of P 201/AtEase was on Tonmya 2.8 mg in participants wit

  17. Slide 17: Primary Outcome (CAPS-5) in Phase 3 mITT and ≤9 Years Subgroups

    © 2019 Tonix Pharmaceuticals Holding Corp. Primary Outcome (CAPS - 5) in Phase 3 ( mITT ) and ≤9 Years Time Since Trauma (TST) Subgroups Modified intent to treat ( mITT ) population Phase 3 P301/HONOR Study 1 ** p=0.004, * p=0.039, # p=0.069, TNX - 102 SL 5.6 mg group v. placebo, using MMRM with MI

  18. Slide 18: Retrospective Comparison of Time Since Trauma in P201 versus P301

    © 2019 Tonix Pharmaceuticals Holding Corp. 18 Retrospective Comparison of Time Since Trauma in P201/AtEase versus P301/HONOR (Tonmya 5.6 mg Groups) P301 study was initiated approximately two years later than Phase 2 P201 • The median time since trauma in Phase 3 was 9.5 years compared to the median

  19. Slide 19: CAPS-5 Difference from Placebo in TST Subgroups in P301

    © 2019 Tonix Pharmaceuticals Holding Corp. 19 CAPS - 5 Mean Change from Baseline Difference from Placebo of Tonmya 5.6 mg in TST Subgroups in P301 1 MCFB=mean change from baseline; ‘N’=number of participants in group; PBO=placebo; TST=time since trauma • The mITT population was divided into subgroup

  20. Slide 20: PTSD Treatment Response in Phase 2 and Phase 3 Subgroups

    © 2019 Tonix Pharmaceuticals Holding Corp. P201 Baseline CAPS - 5 ≥33 (majority TST 1 ≤9 yr ) **p<0.01, *p=0.017, TNX - 102 SL 5.6 mg group v. placebo, using mixed model repeated measures (MMRM) with multiple imputation (MI) P301 TST ≤9 yr ** p=0.004, * p=0.039, # p=0.069 TNX - 102 SL 5.6 mg group v

  21. Slide 21: Sustained Remission in Phase 2 and Phase 3 Studies

    © 2019 Tonix Pharmaceuticals Holding Corp. 21 Sustained Remission in Phase 2 and Phase 3 Studies: Retrospective Analyses of P201 Entry CAPS - 5 ≥33 and P301 ≤9 Years Since Trauma Subgroups Remission is a clinical state that is essentially asymptomatic In order to confirm remission: • Determined rate

  22. Slide 22: Retrospective Analyses of ≤9 Years Since Trauma Subgroup

    © 2019 Tonix Pharmaceuticals Holding Corp. BOLDED p - values are all p<0.05; BDI - II=Beck Depression CAPS - 5=Clinician - Administered PTSD Scale for DSM - 5; CGI - I=Clinical Global Impression – Improvement scale; mITT =modified Intent - to - Treat sample; MMRM=mixed model repeated measures analys

  23. Slide 23: Adverse Events in P201/AtEase and P301/HONOR Studies

    © 2019 Tonix Pharmaceuticals Holding Corp. 23 Adverse Events (AEs) in P201/AtEase and P301/HONOR Studies No serious or unexpected AEs in P201 or P301 related to Tonmya • Systemic AEs comparable between studies and also consistent with those described in approved oral cyclobenzaprine product labeling

  24. Slide 24: Time Since Trauma – Review of Published Studies

    © 2019 Tonix Pharmaceuticals Holding Corp. 24 Time Since Trauma – Review of Published Studies Published studies of prazosin suggested effects in military - PTSD prior to 9 years • Loss of treatment effect >9 years Paroxetine and sertraline studies supporting FDA approval were conducted on PTSD > 9 y

  25. Slide 25: Time Since Trauma – Remitting and Persistent Phases of PTSD

    © 2019 Tonix Pharmaceuticals Holding Corp. 25 Time Since Trauma – Remitting and Persistent Phases of PTSD Kessler et al 1 studied remission in PTSD with and without therapy • Identified remitting and persistent phase of PTSD – with transition at approximately 6 years post trauma • Supported by other

  26. Slide 26: Response to Tonmya for Female Participants in P301/HONOR

    © 2019 Tonix Pharmaceuticals Holding Corp. 26 Response to Tonmya for Female Participants in P301/HONOR Study 1 Females made up only 11% of the P301/HONOR study mITT population Difference in mean change from baseline in CAPS - 5 in females between placebo (N=17) and Tonmya 5.6 mg (N=10) was: • At 4 w

  27. Slide 27: Non-Combat Traumas Similar to Civilian PTSD Traumas

    © 2019 Tonix Pharmaceuticals Holding Corp. 27 Non - combat traumas studied are similar to traumas experienced in civilian populations with PTSD To determine the therapeutic effects of Tonmya 5.6 mg in a mixed civilian and military population, difference in MCFB in CAPS - 5 was assessed in non - comb

  28. Slide 28: Summary of Clinical Experience with Tonmya in PTSD

    © 2019 Tonix Pharmaceuticals Holding Corp. 28 Summary of Clinical Experience with Tonmya/ TNX - 102 SL in PTSD Median time since trauma (TST) in TNX - 102 SL 5.6 mg group in the P301/HONOR study (9.5 years) was longer than P201/AtEase study (6 years) • Both studied military - related PTSD • Time has

  29. Slide 29: New Phase 3 P302/RECOVERY Study – Expected to Start 1Q 2019

    © 2019 Tonix Pharmaceuticals Holding Corp. 29 New Phase 3 P302/RECOVERY Study – Expected to Start 1Q 2019 Primary e ndpoint: • CAPS - 5 1 mean change from baseline at Week 4 ( Tonmya 5.6 mg vs. placebo) Key Secondary e ndpoint s include: • CAPS - 5 m ean change from baseline at W eek 12 (Tonmya 5.6

  30. Slide 30: Late-Stage PTSD Drug Candidates

    © 2019 Tonix Pharmaceuticals Holding Corp. 30 Late - Stage PTSD Drug Candidates Tonmya • Phase 3 development focused on military - related and civilian PTSD; showed activity in treatment of military - related PTSD in large multi - center trials MDMA - assisted psychotherapy • Breakthrough therapy th

  31. Slide 31: TNX-102 SL – Bedtime Treatment for Multiple Potential Indications

    © 2019 Tonix Pharmaceuticals Holding Corp. 31 TNX - 102 SL – Bedtime Treatment for Multiple Potential Indications Ma nagement of Fibromyalgia (FM) – chronic pain condition • TNX - 102 SL studied at low dose (2.8 mg) – half the dose being developed for PTSD – did not separate from placebo on primary

  32. Slide 32: Agitation in Alzheimer's Disease – Additional Indication

    © 2019 Tonix Pharmaceuticals Holding Corp. 32 Agitation in Alzheimer’s Disease – Additional Indication Being Developed for TNX - 102 SL FDA designated Fast Track development program Significant unmet need • No FDA approved drugs for the treatment of agitation in Alzheimer’s Mechanism of improving sl

  33. Slide 33: TNX-102 SL for Agitation in Alzheimer's – Regulatory Status and Strategy

    © 2019 Tonix Pharmaceuticals Holding Corp. 33 TNX - 102 SL for Agitation in Alzheimer’s – Regulatory Status and Registration Strategy FDA confirmed no additional study was needed prior to IND submission • Pre - IND meeting established open dialogue with the FDA on pivotal clinical study design and e

  34. Slide 34: Candidates in Development

    © 2019 Tonix Pharmaceuticals Holding Corp. 34 Candidates in Development Preclinical Phase 2 NDA 1 /BLA 2 Market Pipeline Product Indication Phase 3 TNX - 102 SL Bedtime Treatment for PTSD – Tonmya ® Daytime Treatment for PTSD TNX - 601 Novel polymorph and salt discovered and characterized; Prelimina

  35. Slide 35: TNX-601 (Tianeptine Oxalate): A Potential Clinical Candidate for PTSD

    © 2019 Tonix Pharmaceuticals Holding Corp. 35 TNX - 601 ( Tianeptine Oxalate): A Potential Clinical Candidate for PTSD Pre - IND Candidate Targeted as a 1 st line monotherapy for PTSD: oral formulation for daytime dosing x Leverages expertise in PTSD (clinical and regulatory experience, market analy

  36. Slide 36: TNX-801 (Synthesized Live Horsepox Virus): A Smallpox-Preventing Vaccine Candidate

    © 2019 Tonix Pharmaceuticals Holding Corp. 36 TNX - 801 (Synthesized Live Horsepox Virus): A Smallpox - Preventing Vaccine Candidate Pre - IND Stage Potential improvement over current biodefense tools against smallpox ✓ Leverages Tonix’s government affairs effort ✓ Collaboration with Professor David

  37. Slide 37: Financial Overview

    © 2019 Tonix Pharmaceuticals Holding Corp. 37 Financial Overview NASDAQ: TNXP Cash and cash equivalents, September 30, 2018 $14.8 million Net proceeds from equity offerings in 4Q2018 $17.3 million Common Stock outstanding post December 2018 underwritten equity offering ( a s of March 1, 2019) 4.7 mi

  38. Slide 38: Management Team

    © 2019 Tonix Pharmaceuticals Holding Corp. 38 Management Team Seth Lederman, MD President & CEO Jessica Morris Chief Operating Officer Gregory Sullivan, MD Chief Medical Officer Bradley Saenger, CPA Chief Financial Officer

  39. Slide 39: Board of Directors

    © 2019 Tonix Pharmaceuticals Holding Corp. 39 Board of Directors Seth Lederman, MD Chairman Adeoye “Oye” Olukotun, MD Squibb, BMS, Mallinckrodt, Esperion John Rhodes Chair, NYS Public Service Commission, CEO, NYS Dept. of Public Service, Booz Allen James Treco First Chicago, Salomon Brothers/Citigro

  40. Slide 40: Milestones – Recently Completed and Upcoming

    © 2019 Tonix Pharmaceuticals Holding Corp. 40 Milestones – Recently Completed and Upcoming □ July 201 8 Completed P301/HONOR study interim analysis - result did not support study continuation but strengthened new Phase 3 study □ August 201 8 Presentation of P301/HONOR study results at Military Healt

  41. Slide 41: Summary

    © 2019 Tonix Pharmaceuticals Holding Corp. 41 Summary Phase 3 development of new bedtime treatment for PTSD, including military - related PTSD • Major unmet need; ~12 million Americans annually • Benefited from FDA 505(b)(2) NDA approval requirement Complimentary day - time PTSD treatment in develop

  42. Slide 42: Thank you!

    © 2019 Tonix Pharmaceuticals Holding Corp. Thank you ! NASDAQ: TNXP

Tonix Pharmaceuticals Holding — Investor Presentation (March 2019) on 6ix