Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (July 2014)
28 slides from Tonix Pharmaceuticals's investor presentation, Jul 8, 2014
Slide 1: Investor Presentation July 2014
Investor Presentation July 2014 NASDAQ: TNXP
Slide 2: Forward-looking statements
TONIX PHARMACEUTICALS 2 CONFIDENTIAL TONIX PHARMACEUTICALS 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 sta
Slide 3: First-in-class medicines for common CNS disorders
TONIX PHARMACEUTICALS 3 CONFIDENTIAL TONIX PHARMACEUTICALS First - in - class medicines for common disorders of the central nervous system (CNS) New treatment paradigms Late stage candidates Large unmet medical needs All intellectual property owned by Tonix outright – no royalties Experienced team,
Slide 4: Development pipeline by candidate and indication
TONIX PHARMACEUTICALS 4 CONFIDENTIAL TONIX PHARMACEUTICALS Preclinical Phase 1 Proof - of - Concept Pivotal NDA Market Candidate Indication * To conduct comparative pharmacokinetic and safety study in 4Q 2014 TNX - 102 SL (cyclobenzaprine HCl sublingual tablet) 2.8 mg is an Investigational New Drug
Slide 5: Targeting sleep quality in FM and PTSD
TONIX PHARMACEUTICALS 5 CONFIDENTIAL TONIX PHARMACEUTICALS Targeting sleep quality in FM and PTSD TNX - 102 SL is designed as a chronic therapy for bedtime use Non - restorative sleep linked to pain, fatigue, hyper - vigilance, and arousals Restorative sleep improves FM and PTSD symptoms Novel molec
Slide 6: Fibromyalgia patients and approved medications
TONIX PHARMACEUTICALS 6 CONFIDENTIAL TONIX PHARMACEUTICALS * National Institutes of Health, U.S. Department of Health and Human Services ** Robinson et al, Pain 2012;13:1366 - 76 . *** Estimates based on information from publicly - available sources † EU only SNRI = Serotonin - Norepinephrine Reupta
Slide 7: Fibromyalgia is a chronic, unmet need
TONIX PHARMACEUTICALS 7 CONFIDENTIAL TONIX PHARMACEUTICALS Chronic , widespread pain with sleep, fatigue, mood, and memory problems Typical patient has onset at 30 - 40 years of age with persistence for rest of life Impairs daily function and productivity; poor quality of life Predominantly female P
Slide 8: Healthcare resource utilization and productivity loss in FM
TONIX PHARMACEUTICALS 8 CONFIDENTIAL TONIX PHARMACEUTICALS Robinson et al, Pain Med. 2012;13(10):1366 - 76. 8 Resource utilization over preceding 12 months Outpatient visits 82.9 % Any emergency room visit 40.2 % Mean number of emergency room visits † 2.4 Productivity measures over preceding 12 mont
Slide 9: Poor sleep quality in FM patients
TONIX PHARMACEUTICALS 9 CONFIDENTIAL TONIX PHARMACEUTICALS >90% of FM patients complain of poor sleep quality* Restorative sleep improves pain and other FM symptoms Sleep quality of FM patients can be objectively measured: Cyclic Alternating P attern (CAP) A1 patterns indicate sleep stability A2, A3
Slide 10: Phase 2a trial design in fibromyalgia
TONIX PHARMACEUTICALS 10 CONFIDENTIAL TONIX PHARMACEUTICALS Double - blind, randomized, placebo - controlled Conducted at two academic centers in Canada Enrolled 36 subjects with fibromyalgia; 18 per arm TNX - 102 capsules or placebo taken between dinner and bedtime daily Eight - week, dose - escala
Slide 11: Phase 2a pain and sleep quality results
TONIX PHARMACEUTICALS 11 CONFIDENTIAL TONIX PHARMACEUTICALS -30 -25 -20 -15 -10 -5 0 % Change from Baseline at Week 8 Pain TNX - 102 Placebo † Improving at least one night of CAP A2+A3(norm) ≤ 3 3% Mean TNX - 102 dose at trial end = 3.5 mg 26% reduction in pain vs. placebo Improvement in objective s
Slide 12: Phase 2a safety and adverse events
TONIX PHARMACEUTICALS 12 CONFIDENTIAL TONIX PHARMACEUTICALS No serious adverse events No discontinuations due to adverse events in treatment arm Adverse Event TNX - 102, % (N=18) Placebo, % (N=18) Any adverse event 83 83 Headache 39 17 Dry mouth 33 6 Somnolence 22 11 Constipation 17 6 Dizziness 17 6
Slide 13: Sublingual tablet formulation optimized for bedtime use
TONIX PHARMACEUTICALS 13 CONFIDENTIAL TONIX PHARMACEUTICALS 13 t ransmucosal absorption • efficacy • tolerability/safety • chronic use • bedtime therapy • compliance • metabolism Optimized for: TNX - 102 SL (sublingual) tablets Phase 2b/3 10x faster absorption* * Absorption lag time ( t lag ) based
Slide 14: Phase 2b/3 registration program in FM
TONIX PHARMACEUTICALS 14 CONFIDENTIAL TONIX PHARMACEUTICALS 14 Two adequate and well - controlled efficacy and safety trials in FM patients Primary efficacy endpoint = pain □ First trial has completed enrollment – “BESTFIT”* □ Top line BESTFIT data expected in Q4 2014 Long - term exposure data to su
Slide 15: BESTFIT trial design
TONIX PHARMACEUTICALS 15 CONFIDENTIAL TONIX PHARMACEUTICALS BESTFIT : BE dtime S ublingual T NX - 102 SL as F ibromyalgia I ntervention T herapy Randomized, double - blind, placebo - controlled; 17 U.S. sites Primary efficacy endpoint = change in pain at week 12 vs. baseline (Numeric Rating Scale) T
Slide 16: PTSD patients and approved medications
TONIX PHARMACEUTICALS 16 CONFIDENTIAL TONIX PHARMACEUTICALS * National Institutes of Health, U.S. Department of Health and Human Services ** Wang et al., Arch Gen Psych. 2005;62(6):167 - 78. SSRI = Selective Serotonin Reuptake Inhibitor 16 8.4 million U.S. patients* 4.2 million receiving medical tre
Slide 17: PTSD is a chronic debilitating condition
TONIX PHARMACEUTICALS 17 TONIX PHARMACEUTICALS Post - traumatic s tress d isorder (PTSD) is a chronic debilitating condition Patients desperate despite two FDA approved drugs; no new treatment in >10 years Associated with suicide and unpredictable, violent behaviors 3.5 % of U.S. adult population ha
Slide 18: Poor sleep quality in PTSD and mechanisms
TONIX PHARMACEUTICALS 18 CONFIDENTIAL TONIX PHARMACEUTICALS PTSD patients complain of poor sleep quality as a core symptom Distressing dreams (nightmares) are part of “re - experiencing” Restless sleep is part of “hyper - arousal” Poor sleep quality after trauma is linked to onset of PTSD Poor sleep
Slide 19: Phase 2 PTSD trial design
TONIX PHARMACEUTICALS 19 CONFIDENTIAL TONIX PHARMACEUTICALS Randomized , double - blind, placebo - controlled; to begin enrollment in 4Q14 N=220; approximately 30 U.S. clinical sites Primary efficacy endpoint = difference in Clinician - Administered PTSD Scale (CAPS) score between TNX - 102 SL 2.8 m
Slide 20: Tension-type headache market and treatments
TONIX PHARMACEUTICALS 20 CONFIDENTIAL TONIX PHARMACEUTICALS * Schwartz et al., JAMA 1998;279(5):381 - 3; Chowdhury, Ann Ind Acad Neurol 2012;15(5):83 - 88. ** Russell, J Headache Pain 2005;6(6):441 - 47. *** Scher et al., 2010; due to the lack of prescription products for tension - type headache, mo
Slide 21: TNX-201 novel molecular mechanism
TONIX PHARMACEUTICALS 21 CONFIDENTIAL TONIX PHARMACEUTICALS Novel molecular mechanism Based on proprietary discoveries by Tonix Non - barbiturate, non - opioid Mechanism of action distinct from acetaminophen and barbiturates Comparative pharmacokinetic and safety study to be conducted in 4Q 2014 Pre
Slide 22: Intellectual property wholly owned by Tonix
TONIX PHARMACEUTICALS 22 CONFIDENTIAL TONIX PHARMACEUTICALS 22 Composition - of - matter Patents filed Protection expected to 2034 Pharmacokinetics (PK) Patents filed Protection expected to 2033 Method - of - use FM: patents issued, 3Q 2020 expiry PTSD: patents filed TNX - 102 SL Fibromyalgia, PTSD
Slide 23: Milestones and corporate events
TONIX PHARMACEUTICALS 23 CONFIDENTIAL TONIX PHARMACEUTICALS Corporate □ Jan 2014 – $40.7 million net proceeds from common stock offering TNX - 102 SL - FM □ 3Q 2013 – Began BESTFIT trial in FM □ 4Q 2013 – Began open - label extension study in FM □ 4Q 2014 – Report top line results of BESTFIT trial i
Slide 24: Management team
TONIX PHARMACEUTICALS 24 CONFIDENTIAL TONIX PHARMACEUTICALS Bruce Daugherty, PhD CSO Seth Lederman, MD CEO Leland Gershell, MD, PhD CFO Don Kellerman, PharmD SVP, Clinical Development & Regulatory Affairs
Slide 25: Board of directors
TONIX PHARMACEUTICALS 25 CONFIDENTIAL TONIX PHARMACEUTICALS John Rhodes NYSERDA, NRDC Booz Allen Hamilton Donald Landry, MD, PhD Chair, Department of Medicine Columbia University Stuart Davidson Alkermes Combion Seth Lederman, MD (Chair) Targent Pharmaceuticals Vela Pharmaceuticals Samuel Saks, MD A
Slide 26: Cash and shares outstanding
TONIX PHARMACEUTICALS 26 CONFIDENTIAL TONIX PHARMACEUTICALS 26 NASDAQ: TNXP Cash reported at March 31 , 2014 $ 49.5 million Net cash used in operations in 1Q14 $ 4.0 million Shares outstanding † 9.9 million † As of July 7, 2014
Slide 27: Investment summary
TONIX PHARMACEUTICALS 27 CONFIDENTIAL TONIX PHARMACEUTICALS 27 • TNX - 102 SL: late - stage clinical program in large market indication • Strong evidence of clinical benefit in Phase 2a • Current FM treatment options leave many patients unsatisfied • Fibromyalgia is a current focus of the FDA • Mult
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Tonix Pharmaceuticals Holding — Investor Presentation (July 2014) on 6ix