Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (August 2016)
39 slides from Tonix Pharmaceuticals's investor presentation, Aug 8, 2016
Slide 1: Investor Presentation August 2016
© Copyright 2016 Tonix Pharmaceuticals - Confidential - Do not duplicate or distribute NASDAQ: TNXP Investor Presentation August 2016 © 2016 Tonix Pharmaceuticals Holding Corp. Version: P0026 08 - 08 - 2016
Slide 2: Cautionary note on forward-looking statements
2 © Copyright 2016 Tonix Pharmaceuticals 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
Slide 3: Developing innovative medicines for large and growing markets
3 © Copyright 2016 Tonix Pharmaceuticals Developing innovative medicines for large and growing markets • Targeting two common central nervous system disorders ‒ One clinical - stage proprietary candidate targeting two indications ‒ Differentiated product with potential for sustainable competitive ad
Slide 4: Pipeline led by TNX-102 SL for fibromyalgia
4 © Copyright 2016 Tonix Pharmaceuticals Candidate Indication Near - term Catalyst TNX - 102 SL 2.8 mg ( Tonmya ® *) Fibromyalgia Top line data September 2016 TNX - 102 SL 5.6 mg Post - Traumatic Stress Disorder Phase 3 starting 1Q 2017 Pipeline led by TNX - 102 SL for fibromyalgia Preclinical Phase
Slide 5: Fibromyalgia is inappropriate central pain signaling without peripheral injury
5 © Copyright 2016 Tonix Pharmaceuticals Concept: Fibromyalgia is inappropriate central pain signaling in the absence of peripheral injury Volkswagen Check Engine [Photograph]. (2011, October 14). Wikipedia • Pain is a sensor system in the brain similar to a check engine light on a car’s dashboard •
Slide 6: Fibromyalgia imposes a significant societal and economic burden
6 © Copyright 2016 Tonix Pharmaceuticals Fibromyalgia is a chronic, debilitating disorder that imposes a significant societal and economic burden • Fibromyalgia is considered neurobiological disorder characterized by 1 : • Believed to result from amplified sensory and pain signaling in central nervo
Slide 7: Fibromyalgia is prevalent but underdiagnosed
7 © Copyright 2016 Tonix Pharmaceuticals Fibromyalgia is a prevalent disorder but remains underdiagnosed 1 Lawrence et al, Arthritis Rheum 2008;58:26; Vincent et al, Arthritis Care Res 2013;65:786; Jones et al, Arthritis Rheum 2015;67:568; U.S. Census Bureau, 2013 Projection . 2 Robinson RL et al, P
Slide 8: Fewer than half of treated patients receive sustained benefit
8 © Copyright 2016 Tonix Pharmaceuticals Fewer than half of those treated for fibromyalgia receive sustained benefit from the three currently marketed drugs • The treatment objective is to restore functionality and quality of life by broadly improving symptoms while avoiding significant side effects
Slide 9: Large need for new fibromyalgia therapies with better tolerability
9 © Copyright 2016 Tonix Pharmaceuticals Large need for new fibromyalgia therapies that provide broad symptom improvement with better tolerability • Currently - approved medications may have side effects that limit long - term use 1 ‒ Many patients skip doses or discontinue altogether within months
Slide 10: Tonix is developing TNX-102 SL for fibromyalgia
10 © Copyright 2016 Tonix Pharmaceuticals Tonix is developing TNX - 102 SL for fibromyalgia • Advanced sublingual tablet containing low - dose cyclobenzaprine (CBP) ‒ Designed for daily bedtime administration with no titration ‒ Efficient transmucosal absorption ‒ Avoidance of first - pass metabolis
Slide 11: Phase 2b BESTFIT study in fibromyalgia
11 © Copyright 2016 Tonix Pharmaceuticals Phase 2b “BESTFIT” study of TNX - 102 SL in fibromyalgia • BESTFIT = BE dtime S ublingual T NX - 102 SL as F ibromyalgia I ntervention T herapy ‒ Randomized, double - blind, placebo - controlled trial ‒ 2010 American College of Rheumatology diagnostic criter
Slide 12: BESTFIT results on key clinical endpoints
12 © Copyright 2016 Tonix Pharmaceuticals BESTFIT results on key clinical endpoints Category Endpoint – week 12 1 p value Pain Relief 30% responder analysis 2 0.033 Sleep Quality PROMIS Sleep Disturbance 0.005 Overal l response to therapy PGIC 0.025 Assessment of disease impact FIQ - R Total score 0
Slide 13: Safety and tolerability profile in the BESTFIT study
13 © Copyright 2016 Tonix Pharmaceuticals TNX - 102 SL safety and tolerability profile in the BESTFIT study • No serious adverse events (SAE) reported with TNX - 102 SL • Systemic adverse events reported by at least 3% of the total BESTFIT population • Most frequent local adverse events were adminis
Slide 14: Clinical phase completed in Phase 3 trial for fibromyalgia
14 © Copyright 2016 Tonix Pharmaceuticals • Phase 3 AFFIRM study is fully enrolled • Second Phase 3 Study (“RE - AFFIRM”) enrollment initiated in July 2016 - Expected to be similar to AFFIRM in design and sample size Clinical phase completed in Phase 3 trial of TNX - 102 SL for fibromyalgia • Random
Slide 15: TNX-102 SL in development for PTSD
15 © Copyright 2016 Tonix Pharmaceuticals TNX - 102 SL in Phase 2 development for PTSD Phase 1 Phase 2 Phase 3 NDA * Tonmya ® has been conditionally accepted by the FDA as the proposed tradename of TNX - 102 SL for fibromyalgia. Candidate Indication Near - term Catalyst TNX - 102 SL ( Tonmya ® *) 2.
Slide 16: PTSD is a chronic stress disorder triggered by a traumatic event
16 © Copyright 2016 Tonix Pharmaceuticals PTSD is a chronic stress disorder triggered by a traumatic event • PTSD is characterized by: • Considered a stress response, but prolonged and does not resolve with time ‒ 20% of women and 8% of men who experience significant trauma develop PTSD 1 • Associat
Slide 17: PTSD is prevalent for civilians and the military
17 © Copyright 2016 Tonix Pharmaceuticals PTSD is a prevalent problem for both civilians and the military • ~70% are considered to have moderate to severe symptoms • Of those diagnosed, ~50% utilize professional healthcare (psychotherapy/pharmacotherapy) 2 1 Kessler RC at al, Arch Gen Psychiatry 201
Slide 18: Limitations of current FDA-approved pharmacotherapies for military-related PTSD
18 © Copyright 2016 Tonix Pharmaceuticals Limitations of current FDA - approved pharmacotherapies for military - related PTSD • No treatment response observed in U.S. military population ‒ Sertraline: negative large multicenter trial in U.S. military veterans 1 ‒ Placebo numerically superior on CAPS
Slide 19: Phase 2 AtEase trial in PTSD
19 © Copyright 2016 Tonix Pharmaceuticals Phase 2 AtEase trial of TNX - 102 SL in PTSD • Randomized, double - blind, placebo - controlled trial in military - related PTSD • Analysis from 231 patients; 24 U.S. clinical sites • Primary efficacy endpoint: ‒ Difference in Clinician - Administered PTSD S
Slide 20: AtEase study design
20 © Copyright 2016 Tonix Pharmaceuticals Phase 2 AtEase study of TNX - 102 SL in PTSD AtEase Study • Randomized, double - blind, placebo - controlled trial • DSM - 5 diagnostic criteria for PTSD • 231 participants studied 2:1:2 at 24 U.S. sites ‒ 1 x TNX - 102 SL 2.8 mg: 2 x TNX - 102 SL 2.8 mg: pl
Slide 21: Key demographics and characteristics of AtEase
21 © Copyright 2016 Tonix Pharmaceuticals AtEase Study Key Demographics / Characteristics of AtEase • 93% of the sample was male • 98% had trauma during military service and were deployed on average 2.3 times • Mean time since index trauma was 7 years • Race and ethnicity generally consistent with U
Slide 22: AtEase results on key clinical endpoints
22 © Copyright 2016 Tonix Pharmaceuticals AtEase results on key clinical endpoints Category Endpoint – week 12 1 p value PTSD Symptoms CAPS - 5 (MMRM with MI) 0.031 Global improvement CGI - I 0.041 Arousal and reactivity CAPS - 5 cluster 0.048 Sleep Quality CAPS - 5 sleep 0.010 AtEase pre - specifie
Slide 23: AtEase Study Results: CAPS-5 total score
23 © Copyright 2016 Tonix Pharmaceuticals AtEase Study Results -25 -20 -15 -10 -5 0 Wk 0 Wk 2 Wk 4 Wk 6 Wk 8 Wk 10 Wk 12 Placebo TNX-102 SL 2.8 mg TNX-102 SL 5.6 mg * * p=0.031 , comparing placebo and TNX - 102 SL 5.6 mg, * p<0.05, comparing placebo and TNX - 102 SL 2.8 mg, by MMRM with MI; LS Mean,
Slide 24: AtEase Study Results: CAPS-5 sleep disturbance
24 © Copyright 2016 Tonix Pharmaceuticals AtEase Study Results -1.6 -1.4 -1.2 -1 -0.8 -0.6 -0.4 -0.2 0 Wk 0 Wk 2 Wk 4 Wk 6 Wk 8 Wk 10 Wk 12 LS Mean Change from Baseline Placebo TNX-102 SL 2.8 mg TNX-102 SL 5.6 mg Change from baseline * * * * * p<0.05, 5.6 mg v. placebo, MMRM * p<0.05, 2.8 mg v. plac
Slide 25: AtEase Study Results: Sheehan Disability Scale
25 © Copyright 2016 Tonix Pharmaceuticals AtEase Study Results 1.5 2 2.5 3 3.5 4 4.5 5 5.5 Wk 0 Wk 2 Wk 4 Wk 6 Wk 8 Wk 10 Wk 12 The symptoms have disrupted your work/school work Placebo TNX-102 SL 2.8 mg TNX-102 SL 5.6 mg 2.5 3 3.5 4 4.5 5 5.5 6 6.5 7 7.5 Wk 0 Wk 2 Wk 4 Wk 6 Wk 8 Wk 10 Wk 12 The sym
Slide 26: AtEase Study Results: Clinician Global Impression-Improvement
26 © Copyright 2016 Tonix Pharmaceuticals AtEase Study Results 44.6% 53.3% 63.3% 0% 10% 20% 30% 40% 50% 60% 70% Placebo TNX-102 SL 2.8 mg TNX-102 SL 5.6 mg * *p=0.041 , Logistic regression comparing placebo and TNX - 102 SL 5.6 mg Responders are those rated as “much improved” or “very much improved”
Slide 27: Safety and tolerability profile in the AtEase study
27 © Copyright 2016 Tonix Pharmaceuticals TNX - 102 SL safety and tolerability profile in the AtEase study No serious adverse events reported with TNX - 102 SL deemed related to treatment Systemic Adverse Events* Placebo (N=94) TNX - 102 SL 2.8 mg (N=93) TNX - 102 SL 5.6 mg (N=50) Total TNX - 102 SL
Slide 28: AtEase Study Conclusions
28 © Copyright 2016 Tonix Pharmaceuticals AtEase Study Conclusions • This is the first large, multicenter trial that demonstrated efficacy in a population with military - related PTSD ‒ Male predominant (93%) ‒ Low incidence of co - morbid FM (7%) ‒ Low incidence of current major depression (14%) •
Slide 29: Phase 3 program in PTSD being planned
29 © Copyright 2016 Tonix Pharmaceuticals Phase 3 program in PTSD being planned General Study Characteristics : • Randomized, double - blind, placebo - controlled study in PTSD • N~500; approximately 35 U.S. clinical sites Primary Efficacy Endpoint: • Difference in total CAPS - 5 analysis at Week 12
Slide 30: Intellectual property
30 © Copyright 2016 Tonix Pharmaceuticals Intellectual property • Composition - of - matter (eutectic) ‒ Patents filed ‒ Protection expected to 2034 • Pharmacokinetics (PK) ‒ Patents filed ‒ Protection expected to 2033 • Method - of - use ‒ Fibromyalgia : patents issued, 2020 expiry ‒ PTSD: patents
Slide 31: Cyclobenzaprine: active pharmaceutical ingredient
31 © Copyright 2016 Tonix Pharmaceuticals TNX - 102 SL: active pharmaceutical ingredient • Cyclobenzaprine is a tricyclic molecule that binds to a number of central nervous system (CNS) receptor types • Highest affinity for three receptors believed to have a role in treating sleep disturbances ‒ 5 -
Slide 32: Pharmacokinetic profile after sublingual administration
32 © Copyright 2016 Tonix Pharmaceuticals TNX - 102 SL: Pharmacokinetic profile after sublingual route of administration • Very rapid absorption after dosing (measurable in plasma within 3 minutes of administration) ‒ Maintains T max at ~4 hours after administration ‒ Approximately 50% of exposure t
Slide 33: Cyclobenzaprine detected in plasma within minutes following sublingual dosing
33 © Copyright 2016 Tonix Pharmaceuticals Cyclobenzaprine is detected in plasma within minutes following sublingual TNX - 102 SL Plasma Concentration Versus Time of TNX - 102 SL Compared to oral Cyclobenzaprine IR 0 0.5 1.0 1.5 Concentration (ng/L) 2500 1500 1000 500 2000 0 TNX - 102 SL 2.8 mg (subl
Slide 34: Somnolence and dry mouth with oral and sublingual cyclobenzaprine
34 © Copyright 2016 Tonix Pharmaceuticals 2% 4% 7% 4% 0% 15% 30% Somnolence Dry Mouth TNX-102 SL Placebo Somnolence and dry mouth with oral and sublingual cyclobenzaprine in fibromyalgia patients • ORAL – Phase 2a 1 : ‒ Dose TNX - 102 (CBP capsules, 1 mg) ‒ Dosing regimen ‒ titrated from 1 mg to a r
Slide 35: Financial overview
35 © Copyright 2016 Tonix Pharmaceuticals Financial overview NASDAQ: TNXP Cash, cash equivalents, and marketable securities reported at June 30 , 2016 $ 31.2 million Net proceeds from overallotment in 3Q16 $1.4 million Shares outstanding (August 8, 2016) 25.9 million
Slide 36: Management team
36 © Copyright 2016 Tonix Pharmaceuticals Management team Seth Lederman, MD President & CEO Jessica Edgar Morris EVP, Operations Bruce Daugherty, PhD, MBA Chief Scientific Officer Ronald Notvest, PhD EVP, Commercial Planning & Development Gregory Sullivan, MD Chief Medical Officer Bradley Saenger, C
Slide 37: Board of directors
37 © Copyright 2016 Tonix Pharmaceuticals Board of directors Seth Lederman, MD Chairman Ernest Mario, PhD ALZA, Glaxo, Reliant Pharma John Rhodes NYSERDA, NRDC, Booz Allen Hamilton Samuel Saks, MD Jazz Pharma, ALZA, Johnson & Johnson Charles Mather BTIG, Janney, Jefferies, Cowen, Smith Barney Stuart
Slide 38: Milestones – recent and upcoming
38 © Copyright 2016 Tonix Pharmaceuticals Milestones – recent and upcoming TNX - 102 SL – Fibromyalgia □ May 2015 Began Phase 3 AFFIRM study □ November 2015 Presented additional data from Phase 2b BESTFIT study at ACR Meeting □ May 2016 Reported completion of enrollment in Phase 3 AFFIRM study □ Jul
Slide 39: - - Do not duplicate or distribute Madison Avenue New York, NY 10022 (212)
© Copyright 2016 Tonix Pharmaceuticals - Confidential - Do not duplicate or distribute NASDAQ: TNXP 509 Madison Avenue New York, NY 10022 (212) 980 - 9155 www.tonixpharma.com
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