Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (May 2014)
29 slides from Tonix Pharmaceuticals's investor presentation, May 22, 2014
Slide 1: Investor Presentation May 2014
Investor Presentation May 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: High impact prescription drug candidates for common and complex disorders
TONIX PHARMACEUTICALS 3 CONFIDENTIAL TONIX PHARMACEUTICALS High impact prescription drug candidates for common and complex disorders First - in - class medicines New treatment paradigms Large unmet medical needs Fibromyalgia (FM): BESTFIT trial of TNX - 102 SL fully enrolled Top line results to be r
Slide 4: Pipeline by candidate and indication
TONIX PHARMACEUTICALS 4 CONFIDENTIAL TONIX PHARMACEUTICALS Preclinical Phase 1 Proof - of - Concept Pivotal NDA Market Candidate Indication TNX - 102 SL is an Investigational New Drug and is not approved for any indication Phase 2a Phase 2b/3 PTSD TNX - 102 SL 2018E TNX - 102 SL Fibromyalgia t op li
Slide 5: Targeting sleep quality is a new approach
TONIX PHARMACEUTICALS 5 CONFIDENTIAL TONIX PHARMACEUTICALS Targeting sleep quality is a new approach to FM Non - restorative sleep linked to pain and fatigue Restorative sleep improves FM symptoms Goal – to introduce an effective and well - tolerated new therapeutic with the potential to decrease us
Slide 6: Fibromyalgia market 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: chronic widespread pain with unmet needs
TONIX PHARMACEUTICALS 7 CONFIDENTIAL TONIX PHARMACEUTICALS Chronic , widespread pain with sleep, fatigue, mood, and memory problems Impairs daily function and productivity: poor quality of life ”Central pain" - originates in the brain Typical patient has onset at 30 - 40 years of age with persistenc
Slide 8: Healthcare resource utilization and productivity 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 mo
Slide 9: Poor sleep quality in FM patients and TNX-102
TONIX PHARMACEUTICALS 9 CONFIDENTIAL TONIX PHARMACEUTICALS >90% of FM patients complain of poor sleep quality* Non - restorative sleep linked to hyper - vigilance Restorative sleep improves FM symptoms Sleep quality of FM patients can be objectively measured: Cyclic Alternating P attern (CAP) A1 pat
Slide 10: Phase 2a study design
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 efficacy: pain and sleep quality
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 Types of adverse events consistent with approved cyclobenzaprine products Adverse Event TNX - 102, % (N=18) Placebo, % (N=18) Any adverse event 83 83 Heada
Slide 13: Sublingual tablet formulation optimized for bedtime
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: FDA pre-Phase 3 meeting and remaining work for NDA
TONIX PHARMACEUTICALS 14 CONFIDENTIAL TONIX PHARMACEUTICALS 14 Pre - Phase 3 meeting held with FDA in February 2013 Remaining clinical work to support New Drug Application: Two adequate and well - controlled efficacy and safety trials in FM patients Primary efficacy endpoint = pain □ First trial has
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 market 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 prevalence and overlap with fibromyalgia
TONIX PHARMACEUTICALS 17 TONIX PHARMACEUTICALS 3.5% of U.S. adult population has suffered from PTSD in past 12 months* Experiencing any trauma can lead to PTSD High incidence among U.S. soldiers and veterans Associated with suicide and unpredictable violent behaviors Patients desperate despite two F
Slide 18: Poor sleep quality in PTSD
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: Mechanisms of trazodone and prazosin
TONIX PHARMACEUTICALS 19 TNX - 102 SL Trazodone - Trazodone inhibits serotonin 5HT 2a receptors and serotonin reuptake (SARI) - Prazosin blocks norepinephrine α 1 receptors Prazosin SARI = S erotonin Receptor A ntagonist & R euptake I nhibitor (Stahl SM, CNS Spectrums, 2009;14(10):536 - 46). TNX - 1
Slide 20: Phase 2 PTSD study design
TONIX PHARMACEUTICALS 20 CONFIDENTIAL TONIX PHARMACEUTICALS Randomized , double - blind, placebo - controlled N=220; approximately 25 U.S . clinical sites Primary efficacy endpoint = difference in Clinician - Administered PTSD Scale (CAPS) score between TNX - 102 SL 2.8 mg and placebo R A N D O M I
Slide 21: Tension-type headache market and approved medications
TONIX PHARMACEUTICALS 21 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 22: TNX-201 single isomer isometheptene
TONIX PHARMACEUTICALS 22 CONFIDENTIAL TONIX PHARMACEUTICALS TNX - 201 (single isomer isometheptene ) Non - barbiturate, non - opioid Racemic mixture >50 years of use in combination products for headache in the U.S . Not FDA approved for any indication * Limited availability, quality concerns via com
Slide 23: Intellectual property portfolio
TONIX PHARMACEUTICALS 23 CONFIDENTIAL TONIX PHARMACEUTICALS 23 Pharmacokinetics (PK) Patents filed around unique PK profile Protection expected to 2033 Composition - of - matter Patent filed - “Eutectic” Protection expected to 2034 Method - of - use FM: patent issued, 3Q 2020 expiry PTSD: patent fil
Slide 24: Corporate and clinical milestones
TONIX PHARMACEUTICALS 24 CONFIDENTIAL TONIX PHARMACEUTICALS Corporate □ Jan 2014 – $40.7 million net proceeds from common stock offering TNX - 102 SL □ 3Q 2013 – Began BESTFIT trial in FM □ 4Q 2013 – Began open - label extension study in FM □ 3Q 2014 – Start Phase 2 efficacy study in PTSD □ 4Q 2014
Slide 25: Management team
TONIX PHARMACEUTICALS 25 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 26: Board of directors
TONIX PHARMACEUTICALS 26 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 27: Cash and shares outstanding
TONIX PHARMACEUTICALS 27 CONFIDENTIAL TONIX PHARMACEUTICALS 27 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 May 16, 2014
Slide 28: Investment summary
TONIX PHARMACEUTICALS 28 CONFIDENTIAL TONIX PHARMACEUTICALS 28 • 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 (May 2014) on 6ix