Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (September 2014)
28 slides from Tonix Pharmaceuticals's investor presentation, Sep 26, 2014
Slide 1: Investor Presentation September 2014
Investor Presentation September 2014 © 2014 Tonix Pharmaceuticals Holding Corp. NASDAQ: TNXP
Slide 2: Safe harbor statement
Safe harbor s tatement 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 identified by the use
Slide 3: Tonix - Overview
Tonix - Overview 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, strong balance sheet Track record of succe
Slide 4: Development programs
Development programs Preclinical Phase 1 Proof - of - Concept Pivotal NDA Market Candidate Indication *To conduct comparative pharmacokinetic and safety study in 1Q 2015 TNX - 102 SL (cyclobenzaprine HCl sublingual tablet) 2.8 mg is an Investigational New Drug and is not approved for any indication.
Slide 5: Targeting sleep quality in fibromyalgia and PTSD
Targeting sleep quality in fibromyalgia (FM) and in post - traumatic stress disorder (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 molecular t
Slide 6: Fibromyalgia market opportunity
CONFIDENTIAL Fibromyalgia market opportunity * 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 SNRI = Serotonin - Norepinephrine Reuptake Inhibitor 5 million U
Slide 7: Fibromyalgia: many dissatisfied patients
Fibromyalgia: many dissatisfied patients 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 Patients remain uns
Slide 8: Fibromyalgia has a high economic impact
CONFIDENTIAL Fibromyalgia has a high economic impact Robinson et al, Pain Med. 2012;13(10):1366 - 76. 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 months Misse
Slide 9: Poor sleep quality in fibromyalgia patients
CONFIDENTIAL >90% of fibromyalgia patients complain of poor sleep quality* Restorative sleep improves pain and other FM symptoms Sleep quality of fibromyalgia patients can be objectively measured: Cyclic Alternating P attern (CAP) A1 patterns indicate sleep stability A2, A3 patterns indicate sleep i
Slide 10: Phase 2a trial of TNX-102 capsules in FM
CONFIDENTIAL Phase 2a trial of TNX - 102 capsules in FM 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 - escalating
Slide 11: Positive efficacy results from Phase 2a trial in FM
CONFIDENTIAL -30 -25 -20 -15 -10 -5 0 % Change from Baseline at Week 8 Pain TNX - 102 Placebo Positive efficacy results from Phase 2a trial of TNX - 102 capsules in FM † 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
Slide 12: Safety results from Phase 2a trial in FM
CONFIDENTIAL Safety results from Phase 2a trial of TNX - 102 capsules in FM 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
Slide 13: Sublingual tablet formulation optimized for bedtime use
CONFIDENTIAL TNX - 102 SL is a sublingual tablet formulation optimized for chronic use at bedtime 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* * A
Slide 14: Registration program in FM
CONFIDENTIAL R egistration program for TNX - 102 SL in FM Two adequate and well - controlled efficacy and safety trials in fibromyalgia patients Primary efficacy endpoint = pain □ First trial has completed – “BESTFIT”* □ Report top line results from BESTFIT by October 15, 2014 Long - term exposure d
Slide 15: BESTFIT potential pivotal trial - completed
CONFIDENTIAL “BESTFIT” potential pivotal trial - completed 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) If
Slide 16: PTSD market opportunity
CONFIDENTIAL * 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 8.4 million U.S. patients* 4.2 million receiving medical treatment** Two FDA approved prescription medications
Slide 17: PTSD is a chronic debilitating condition
CONFIDENTIAL Post - traumatic s tress d isorder 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 has suffered from PTSD in past 12 months* E
Slide 18: PTSD patients complain of poor sleep quality
CONFIDENTIAL 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 correlates with depression, substance abuse an
Slide 19: AtEase Phase 2 trial in PTSD
CONFIDENTIAL “AtEase” Phase 2 trial of TNX - 102 SL in PTSD Randomized , double - blind, placebo - controlled; to begin enrollment in 4Q14 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 m
Slide 20: Episodic tension-type headache (ETTH)
CONFIDENTIAL TNX - 201 – Episodic tension - type headache (ETTH) * 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., Cephalalgia 2010;30(3):321 - 328; due to the lack of prescription product
Slide 21: TNX-201 to enter clinical development in 2015
CONFIDENTIAL TNX - 201 to enter clinical development in 2015 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 1Q 2015 Pr
Slide 22: Intellectual property
CONFIDENTIAL Intellectual property 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 TNX - 201 H eadache Composit
Slide 23: Milestones – recent and upcoming
CONFIDENTIAL Milestones – recent and upcoming Corporate □ July 2014 – $7.2 million in net proceeds from common stock offering TNX - 102 SL - Fibromyalgia □ August 2014 – Completed BESTFIT trial □ Report top line results from BESTFIT by October 15, 2014 TNX - 102 SL - PTSD □ June 2014 – Received IND
Slide 24: Management team
CONFIDENTIAL Bruce Daugherty, PhD CSO Seth Lederman, MD CEO Management team Leland Gershell, MD, PhD CFO Don Kellerman, PharmD SVP, Clinical Development & Regulatory Affairs 24
Slide 25: Board of directors
CONFIDENTIAL 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 ALZA Jazz Pharmaceuticals Patrick Grace WR Grace
Slide 26: Financial summary
CONFIDENTIAL Financial summary NASDAQ: TNXP Cash reported at June 30 , 2014* $ 43.9 million Net cash used in operations in 2Q14 $ 5.7 million Shares outstanding † 10.6 million * Does not include $7.2 million in net proceeds from stock offering in July 2014. † As of September 24, 2014 26
Slide 27: Why invest in Tonix now?
CONFIDENTIAL Why invest in Tonix now? • 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 • Multiple opportunities (fibro
Slide 28: Thank you
Thank you © 2014 Tonix Pharmaceuticals Holding Corp. NASDAQ: TNXP
Tonix Pharmaceuticals Holding — Investor Presentation (September 2014) on 6ix