Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (December 2016)
36 slides from Tonix Pharmaceuticals's investor presentation, Dec 19, 2016
Slide 1: Investor Presentation
Investor Presentation TONIX PHARMACEUTICALS December 2016 Version P0044 12-19-16
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
Slide 3: Posttraumatic Stress Disorder (PTSD) program
Posttraumatic Stress Disorder (PTSD) program On May 19, 2016; we reported encouraging topline data for TNX-102 SL* 5.6 mg in a military-related PTSD (“AtEase”) PTSD was our cond neal program using our proprietary formulation TNX- 102 SL Prior to the AtEase trial, no other investigational new drug or
Slide 4: Tonix (TNXP): Value proposition
Tonix (TNXP): Value proposition On September 6, 2016 our lead Phase 3 program TNX-102 SL for fibromyalgia narrowly missed its primary endpoint in the first Phase 3 study (“AFFIRM”) + Received strong negative investor market response + Reassuring safety profile and activity of TNX-102 SL at 2.8 mg fo
Slide 5: Pivot to PTSD: Rationale
© Pivot to PTSD: Rationale Unmet medical need + PTSD is a serious condition and the prevalence is increasing, especially combat-related + Military-related PTSD is not satisfactorily treated by existing FDA approved therapies + Two selective serotonin reuptake inhibitors (SSRIs) are approved for PTSD
Slide 6: 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 +
Slide 7: Tonix Pharmaceuticals PTSD Program
Tonix Pharmaceuticals PTSD Program ‘TNX-102 SL (cyclobenzaprine HCI sublingual tablets) + Aunique, innovative product designed for bedtime administration Phase 3 + Targeting a chronic and serious psychiatric disorder: PTSD Ready v Therapeutic dose identified in Phase 2 study Program v Phase 3 clinic
Slide 8: What is PTSD?
What is PTSD? Kester e reser ea A chronic response to traumatic event(s) + A majority of people will experience a traumatic event at some point in their lifetime + 20% of women and 8% of men in the U.S. who experience significant trauma develop PTSD* 2 (~ 17 million adults in the U.S.) + Persistent
Slide 9: What Are the Symptoms of PTSD?
What Are the Symptoms of PTSD? ‘Symptoms of PTSD fall into four clusters: Intrusion (aversive memories, nightmares, flashbacks) Avoidance (avoiding persons, places or situations) Mood/cognitions (memory block, emotional numbing, detachment from others) Hyperarousal (anxiety, agitation & sleep distur
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
Slide 11: PTSD Prevalence and Market Characteristics
PTSD Prevalence and Market Characteristics Prevalent Population (U.S.) mi int Diagnosed population 516500 Large population (~1.8 million) 6% Veterans Majority receive drug treatment No ex ted Civilians: ~75%? 128,000 | In Veterans Veterans: ~80%* a Administration Undiagnosed ay Re 872,000 10% Civili
Slide 12: What Drug Classes are Used to Treat PTSD?
What Drug Classes are Used to Treat PTSD? Market highly ines bei ing widely ibed (but not indi ) Multiple medications per Patient (or "Polypharmacy”) is the nor Approximately 55% of patients receive a benzodiazepine, ‘and 53% receive an selective serotonin reuptake inhibitor (SSRI) SSRIs are the onl
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 Pathophysiology Stress = Hyperarousal = Sleep Disturbances Pharmacological ‘Action 2° Clinical Endpoi
Slide 14: TNX-102 SL: Innovative and Unique By Design
© TNX-102 SL: Innovative and Unique By Design antidepressants (TCAs) tricyclic + TCAsand widely pt eB ie histamine ‘a Major etatelte, soreyeaboneapine (MCB?) + Long half-life (~72 hours) : _ ay-adrenergic, histamine H,) + More selective for norepinephrine transporter from oral CBP. formulation 1, Ru
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 ES ent ty ie ya (H UTN SE Paroxetine: no largi Sertraline: FDA-conducted post-hoc analysis concluded no effect for male civilian subgroup? Paroxetine: no sex-related difference in tre
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 . 2 + TNX-102 SL was active at 5.6 mg dose + Primar
Slide 17: AtEase Study Demographics and Characteristics
AtEase Study Demographics and Characteri: © 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 Depres
Slide 18: AtEase Study: Traumas Associated with PTSD
AtEase Study: Traumas Associated with PTSD uring Military S Patient Count Being involved in an TED explosion or suicide bombing Witness death or injury of fellow soldiers 33 Witnessing TED explosion 30 Receiving incoming artillery, rocket, or mortar fire 29 Being wounded or injured 10 Being responsi
Slide 19: AtEase Study - Summary of Primary and Secondary Analyses
AtEase Study - Summary of Primary and Secondary Analyses (week 12) ‘Assessment Domain ‘Analysis, p-values 2.8 mg (N=90) | 5.6 mg (N=49) cAaPS-5 frotar ow 0.259% 0.053 [Total loMIRM with Multiple Imputation | 0.211 0.031" [Total lMMRM w/ Hybrid LOCF/BOCF 0.172 0.037" [Total lANCOVA 0.090 0.038" © | 0
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 Systemic Adverse Eventae__PRSHROTWXAOZSLZ8mg TWX-102SL 56mg Somnolnos 6% 11.8% 16.0% ory Mouth 10.6% 43% 16.0% Headache 43% 5.4% 120% Ineornis 85% 75% 60% Sedation 1.1% 2.2%
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 > 33%) + FDA has accepted this higher entry criteria (CAPS-5 =
Slide 22: Retrospective Analysis of CAPS-5 in patients with entry CAPS-5 ≥33
Retrospective Analysis of CAPS-5 in patients with entry CAPS-5 2 331 WkO Wk2 9 Wka = Wk6— Wk8—Wk10—Wk12 Placebo 5.0 -=-TNX-102 SL.2.8 mg TNX-102 SL5.6 mg LS Mean (SE) of CAPS-5 Total Score * *=9<0.025, comparing 5.6 mg group with placebo, MMRM **D=0.028, comparing 2.8 mg group with placebo, MMRM ‘Pr
Slide 23: Week 12 outcome measures for TNX-102 SL 5.6 mg vs. placebo
Week 12 outcome measures for TNX-102 SL 5.6 mg vs. placebo in military-related PTSD for CAPS-5 = 29 and CAPS- 5 2 33 at baseline 03 GI-r (responders) 2aL 0.041 2.29 *0.042 SDs Total Score 0.33 0.079 0.35 0.093 ‘Work/School item 034 0.050 oat *0.040 ‘Social/Leisure item 0.38 *0.031 0.35 0.116 Family
Slide 24: Week 12 outcome measures in combat PTSD subset
Week 12 outcome measures for TNX-102 SL 5.6 mg vs. placebo in combat PTSD subset for CAPS-5 2 29 and CAPS-5 2 33 at baseline CAPS-5 Total score Cluster B (intrusion) Cluster ¢ (avoi Cluster D (mood/cognition) luster E (arousal and rea 6 (Sleep item) E2 (Reckless/Self Destruct) CGI-I (responders) sos
Slide 25: Analysis Using CAPS-5 ≥33 As Entry Criteria
© Analysis Using CAPS-5 2 33 As Entry Criteria Aechless or Selt-Destructive Mean Score wk10 Wk Placebo -2-1Hx-102 51.2.8 mo ATNX-102 SL 5.6 mo *pe0.012, 104-102 St 5.6 ma. placebo, mixed model repeated measures Item on CAPS-5 Reckless or self-destructive behavior can include dangerous driving, high-
Slide 26: Phase 2 AtEase Study Conclusions
Phase 2 AtEase Study Conclusions ‘rst large multi trial ing efficacy of an 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 impression of improvement (C
Slide 27: Hypothesized mechanism of action in PTSD
i Hypothesized mechanism of action in PTSD © Attenuation of Sleep Disruption by TNX-102 SL Leads to Recovery [__ ¥ steep dependent J ‘ecnctonnasraton” | xf eo = PTS Recovery (over weeks) Diagrams adapted from Pace-Schott et al. Biol Mood Anxiety Disord 2015;5:3
Slide 28: 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 19 2017 ‘TINX-102 SL once-daily at bedtime 5.6mg N~ 275 (90*) Placebo once-daily at bedtime N ~ 275 (90*) 22 weeks —> * First interim analysis {© 2016 Tonix Pharma
Slide 29: 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
Slide 30: 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
Slide 31: Financial overview
Financial overview NASDAQ: TNXP Cash, cash equivalents, and marketable i securities reported at September 30, 2016 $26.7 million Net proceeds from underwritten offering in 4Q16 $4.6 million Shares outstanding (December 19, 2016) 39.2 million
Slide 32: Management Team
Management Team ‘Seth Lederman, MD President & CEO TARGENT~ &Fusiley’ vela__ Gregory Sullivan, MD Chief Medical Officer Cownata Unvessiry New York State Department of Psychiatry Psychiatric Institut Bradley Saenger, CPA Chief Financial Officer hire vittex all Jessica Morris EVP, Operations MW Americ
Slide 33: Board of Directors
© Board of Directors Seth Lederman, MD Chairman Ernest Mario, PhD ALZA, Glaxo, Reliant Pharma Stuart Davidson Labrador Ventures, Alkermes, Combion Charles Mather BTIG, Janney, Jefferies, Cowen, Smith Barney Patrick Grace Apollo Philanthropy, WR Grace, Chemed John Rhodes NYSERDA, NRDC, Booz Allen Ham
Slide 34: Milestones - recent and upcoming
Milestones - recent and upcoming INX-102 SL = Posttraumatic Stress Disorder W December 2015 __ Entered into Collaborative Research and Development Agreement (CRADA) with the United States Army Medical Materiel Development Activity (USAMMDA) & May 2016 Report results from AtEase study W& August 2016
Slide 35: Summary
Summary Strong position for value growth with Phase 3 trial in a major medical indication: PTSD including military-related PT: + Phase 3 asset not previously well-known ie the investor marketplace ‘TNX-102 SL for PTSD is designed as a Breakthrough Therapy + Accelerated development and approval proce
Slide 36: Thank you!
TONIX PHARMACEUTICALS NASDAQ: TNXP Thank you! {© 2016 Tonix Pharmaceuticals Holding Corp.
Tonix Pharmaceuticals Holding — Investor Presentation (December 2016) on 6ix