Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (February 2017)
44 slides from Tonix Pharmaceuticals's investor presentation, Feb 10, 2017
Slide 1: Investor Presentation
© Investor Presentation TONIX PHARMACEUTICALS February 2017 Version P0052 2-7-17
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: Tonix: Developing Innovative Pharmaceutical Products for Public Health Challenges
Tonix (TNXP): Developing Innovative © Pharmaceutical Products to Address Current and Emerging Public Health Challenges Targeting central nervous system conditions + Phase 3-ready drug, TNX-102 SL* for Posttraumatic stress disorder (PTSD) with Breakthrough Therapy designatt Encouraging evidence of sa
Slide 4: Product Pipeline
© Product Pipeline Candidate Phase 2 results reported TNX-102 SL fier) May, 2016 H ute of Cyclobenzaprine HCI sublingual tablets 392017 | administration and ' new indication for Tianeptine oral formulation First time approval in the US,, lever H drug veo _notmime ten coatnt ayy Live virus vaccine fr
Slide 5: 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 ¥ Phase 3 clinica
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
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 potential advantages over existing therapies in military-related PTSD + Benefits of Breakthrough Therapy designa‘ n + Eligibility f
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 lifetime + 20% of women and 8% of men in the U.S. who experience significant trauma develop PTSD* + Lifetime prevalence: 2 (~ 17 million adults in the U.S.) + Persis
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 Rx 510,000 6% 128,000 Undiagnosed 2% Rx 872,000 10% No Rx 288,000 3% Diagnosed population Large population (~1.8 million) Majority receive drug treatment ted Civilians: ~75%2 in Veterans Veterans: ~80%* Civilian Population
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: Sleep Disturbances Are a Core Feature of PTSD
© Sleep Disturbances Are a Core Feature Of PTSD+ Sleep disturbances are a core feature of PTSD and a component of three of the four major symptom clusters: ( Presence of one (or more) intrusion symptoms Persistent avoidance of stimuli associated with traumatic event Negative alterations in cognition
Slide 14: What Is Restorative Sleep?
What Is Restorative Sleep? Restorative Sleep Hypothesis*: Sleep is an active, necessary state for pi charged energy, and resetting homeostasis to circuitry in the brain DL eNeRcY F A Depression, Chronic Sleep Deprivation PAM’ Depression, Chron Sleep Disturbances ak y PTSD Germain A. Am J Psychiatry.
Slide 15: 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 Pharmacological | Therapeutic Pathophysiology ‘Action Benefit 2° Clinical 1° Clinical Endpoint Endpoint S
Slide 16: 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 17: High Prevalence of PTSD Among Combat Veterans
High Prevalence of PTSD Among Combat Veterans 3-' General 9% 19-31% 10-21% population Vietnam veterans? Operation Enduring Freedom F; / ay ed wow sao nt semana 2008 2taneln insole wounds of ar, 2008 $80 Revo 2012 essen, (OEF; Operation Iraqi Freedom 8.6 million American adults affected (OIF) veteran
Slide 18: Economic and Social Burden of PTSD
Growi and Social Burden to Care 9or Veterans with PTSD Health care costs associated with PTSD for OEF/OIF/OND veterans: Direct costs $3,000- By (000 tga ah bay 331 fer F/OIF Vel Indirect costs $2-3 billion estimated yearly cost to society” ~ 2.7 million troops deplc 10/1/2001 ar 'cB0 Report 2012; "T
Slide 19: 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 20: Phase 2 AtEase Study in Military PTSD
(Phase 2 AtEase Study in Military PTSD Randomized, double-blind, placebo- controlled trial in military-related PTSD ‘TNX-102 SL at bedtime once-daily Enrolled patients with baseline CAPS-5 = 29 Analysis from 231 patients; 24 U.S. clinical sites TNX-102 SL was active at 5.6 mg dose Primary efficacy a
Slide 21: 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 22: AtEase Study: Traumas Associated with PTSD
AtEase Study: Traumas Associated with PTSD uring Being Involved in an TED explosion or suicide bombing Witness death or injury of fellow soldiers Witnessing TED explosion 30 incoming artillery, rocket, or mortar fire 29 Being wounded or injured 10 Being responsible for the death of a noncombatant 9
Slide 23: AtEase Study - Summary of Primary and Secondary Analyses
AtEase Study - Summary of Primary and Secondary Analyses (week 12) ‘Assessment Domain ‘Analysis, p-Va 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" © | 0.141
Slide 24: 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 25: 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 26: Retrospective Analysis of CAPS-5 in Patients with Entry CAPS-5 ≥ 33
Retrospective Analysis of CAPS-5 in Patients with Entry CAPS-5 2 33 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 ‘Pri
Slide 27: Retrospective Analyses of Week 12 Outcome Measures
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 2 33. at Baselin CES DUE Cluster ¢ (avoidance) Cluster D (mood/cognition) Cluster E (arousal and 6 (Sleep item) 2 (Reckless/Self Destruct) CGI
Slide 28: Phase 2 AtEase Study Conclusions
Phase 2 AtEase Study Conclusions First large multi tri New Drug (IND) in military-related PTSI ¥ TNX-102 SL therapeutic dose (5.6 mg) identified ¥ Symptom reduction (CAPS-5) unctional improvement (Sheehan Disability Scale domains) 7 Ginical global impression of improvement, Y Evidence of meaningful
Slide 29: Planned Phase 3 HONOR Study in PTSD
Planned Phase 3 HONOR Study in PTSD \gs in military- General study characteristics: To confirm AtEase fin related PTSD: + Randomized, double-blind, placebo-controlled, entrance CAPS-5 233 + Larger adaptive design study : + One unblinded interim analysis (1A) by an independent Ter ing start in 19 201
Slide 30: 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 31: 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 32: Proprietary Cyclobenzaprine Hydrochloride Eutectic Mixture Stabilizes Tablet Formulation
© Proprietary Cyclobenzaprine Hydrochloride Eutectic Mixture Stabilizes Tablet Formulation Cyclobenzaprine-HCl ooo oof oe” ee Protactic™ wart” ee mate ion 5 ms oor FoF or” Base particle (K:HPO,) go NaS ™ sasaipertce 04) Vv Pry ote. 2 Cyclobenzaprine — 968 oxtaben &* a. oe rapid absorption properties
Slide 33: Product Pipeline
© Product Pipeline Candidate TNX-102 SL fier) Phase 2 results reported May, 2016 H ute of Cyclobenzaprine HCI sublingual tablets 392017 | administration and ' new indication for Tianeptine oral formulation First time approval in the US,, lever H drug veo _notmime ten coatnt ayy Live virus vaccine fr
Slide 34: TNX-601 - A Potential Clinical Candidate for PTSD
© TNx-601 - AP. ial Clinical Candidate for PTSD TNX-601 (tianeptine oral formulation) 34 + Targeted as a 1* line monotherapy for PTSD: daytime dosing v Leverages expertise in PTSD (clinical and regulatory experience, market analysis, ete.) v Mechanism of Action (MOA) is different from TNX-102 SL + T
Slide 35: Structural Comparison: TNX-102 SL and TNX-601
Structural Comparison: TNX-102 SL and TNX-601 + Cyclobenzaprine and tianeptine share structural similarities with classic tricyclic antidepressants (TCAs) and to each other, but each has unique pharmacological properties + Tianeptine has a nucleus with an side chain Tianeptine leverages Tonix’s expe
Slide 36: TNX-601 - A Potential Clinical Candidate for PTSD
© TNX-601 - A Potential Clinical Candidate for PTSD nt (API) is a novel salt of tianeptine + Shares structural fesbires with tricyclic antidepressants, but has unique pharmacological and neurochemical proper Tianeptine modulates the glutamatergic system indirectly Joes not have significant affinity
Slide 37: TNX-801 - A Potential Smallpox Preventing Vaccine
TNX-801 - A Potential Smallpox Preventing Vaccine TNX-801 (live virus vaccine) + Targeting a biological warfare threat: smallpox v Leverages government affairs effort PIND + Tonix has identified a new vaccine with improved properties Candidate ¥ Patent application on novel vaccine submitted Regulato
Slide 38: TNX-801 - A Potential Smallpox Preventing Vaccine
(5) TNX-801 - A Potential Smallpox Preventing Vaccine 38 ive biological ingredient is live virus Shares structural characteristics to vaccinia-based vaccines, but has unique properties that suggest lower toxicity? ‘Smallpox has a ~30% mortality rate in naive populations - vaccination discontinued in
Slide 39: 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 (February 10, 2017) 39.2 million
Slide 40: Management Team
Management Team 40 ‘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 Ame
Slide 41: 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 42: Milestones - recent and upcoming
Milestones - recent and upcoming 42 INX-102 SL — Posttraumatic Stress Disorder W May 2016 & August 2016 @& December 2016 @ January 2017 1Q 2017 1Q 2017 1H 2018 2H 2018 cooo Reported results from AtEase study End-of-Phase 2 meeting with FDA = Proposed Phase 3 clinical and NDA plan accepted Breakthrou
Slide 43: 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 ‘o othe investor marketplace ‘TNX-102 SL for PTSD is designated as a Breakthrough Therapy by FDA + Accelerated development and appr
Slide 44: Thank you!
TONIX PHARMACEUTICALS NASDAQ: TNXP Thank you! (© 2017 Tonix Pharmaceuticals Holding Corp.
Tonix Pharmaceuticals Holding — Investor Presentation (February 2017) on 6ix