Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (September 2016)
33 slides from Tonix Pharmaceuticals's investor presentation, Sep 19, 2016
Slide 1: Investor Presentation September 2016
© Copyright 2016 Tonix Pharmaceuticals - Confidential - Do not duplicate or distribute NASDAQ: TNXP Investor Presentation September 2016 © 2016 Tonix Pharmaceuticals Holding Corp. Version : P0029 09 - 19 - 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 common central nervous system disorders ‒ One clinical - stage proprietary candidate targeting posttraumatic stress disorder (PTSD ) ‒ Differentiated product with potential for sustaina
Slide 4: Overview: PTSD program
4 © Copyright 2016 Tonix Pharmaceuticals Overview: PTSD program • Results from multi - center, double - blind, randomized, placebo - controlled Phase 2 trial in military - related PTSD reported May 2016 ‒ Optimal therapeutic dose identified ‒ TNX - 102 SL 5.6 mg was active in treating military - rel
Slide 5: Tonix is developing TNX-102 SL for PTSD
5 © Copyright 2016 Tonix Pharmaceuticals Tonix is developing TNX - 102 SL for PTSD • Advanced sublingual tablet containing low - dose cyclobenzaprine (CBP) ‒ Designed for daily bedtime administration with no titration ‒ Efficient transmucosal absorption ‒ Avoidance of first - pass metabolism reduces
Slide 6: PTSD is a chronic stress disorder triggered by a traumatic event
6 © 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 in the U.S. who experience significant trauma develop PTSD 1
Slide 7: PTSD is a prevalent problem for both civilians and the military
7 © 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 2005
Slide 8: Limitations of current FDA-approved pharmacotherapies for military-related PTSD
8 © 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 multi - center trial in U.S. military veterans 1 ‒ Placebo numerically superior on CA
Slide 9: Phase 2 AtEase study of TNX-102 SL in PTSD
9 © 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 tablet: 2 x TNX - 102 SL 2.8
Slide 10: Phase 2 AtEase trial of TNX-102 SL in PTSD
10 © 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 analysis: ‒ Difference in CAPS - 5 score between TNX - 10
Slide 11: Key Demographics / Characteristics of AtEase
11 © Copyright 2016 Tonix Pharmaceuticals AtEase study of TNX - 102 SL in PTSD Key Demographics / Characteristics of AtEase • 93% of the patients were 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
Slide 12: AtEase results on key clinical endpoints
12 © 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 (Logistic Regression) 0.041 Arousal and reactivity CAPS - 5 cluster (MMRM) 0.048 Sleep Quality CAPS - 5 slee
Slide 13: AtEase study results: CAPS-5 total score mean change from baseline
13 © Copyright 2016 Tonix Pharmaceuticals AtEase study results: CAPS - 5 total score mean change from baseline -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 an
Slide 14: AtEase study results: sleep disturbance
14 © Copyright 2016 Tonix Pharmaceuticals AtEase study results: CAPS - 5 sleep disturbance -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, M
Slide 15: AtEase study results: Sheehan Disability Scale
15 © Copyright 2016 Tonix Pharmaceuticals AtEase study results: Sheehan Disability Scale 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
Slide 16: AtEase study results: Clinical Global Impression
16 © Copyright 2016 Tonix Pharmaceuticals AtEase study results: Clinical Global Impression 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 impro
Slide 17: Safety and tolerability profile in the AtEase study
17 © 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 18: Determining severity criteria for entry in Phase 3 trials
18 © Copyright 2016 Tonix Pharmaceuticals Determining CAPS - 5 severity criteria for entry in phase 3 trials • CAPS - 5 has not been employed previously in a pharmacotherapy trial ; entry severity threshold not clear ; ≥ 29 used in the Phase 2 AtEase study • Prior pharmacotherapy trials in PTSD usin
Slide 19: CAPS-5 mean change from baseline for entry CAPS ≥ 33
19 © Copyright 2016 Tonix Pharmaceuticals CAPS - 5 mean change from baseline for entry CAPS ≥ 33 -30.0 -25.0 -20.0 -15.0 -10.0 -5.0 0.0 Wk 0 Wk 2 Wk 4 Wk 6 Wk 8 Wk 10 Wk 12 LS Mean (SE) Placebo TNX-102 SL 2.8 mg TNX-102 SL 5.6 mg 6.8 point difference in CAPS - 5 change from baseline between placebo
Slide 20: CAPS-5 cluster scores
20 © Copyright 2016 Tonix Pharmaceuticals CAPS - 5 cluster scores • Table shows the significance levels and effect sizes of the CAPS - 5 cluster scores, comparing TNX - 102 SL 5.6 mg and placebo, using a CAPS - 5 baseline entry criterion of ≥ 33 and the per protocol threshold of ≥ 29 Entry criteria
Slide 21: Rate of remission from PTSD in each treatment arm
21 © Copyright 2016 Tonix Pharmaceuticals Rate of remission from PTSD in each treatment arm 14.3% 17.1% 28.9% 0% 5% 10% 15% 20% 25% 30% 35% Placebo (N=77) TNX-102 SL 2.8 mg (N=70) TNX-102 SL 5.6 mg (N=38) Percent of Remitters in Group # p=0.10 NS, comparing placebo and TNX - 102 SL 5.6 mg, logistic
Slide 22: AtEase study conclusions
22 © Copyright 2016 Tonix Pharmaceuticals AtEase study conclusions • This Phase 2 trial is the first large multi - center trial of an investigational new drug product that demonstrated efficacy in a population with military - related PTSD ‒ Symptom reduction (CAPS - 5) ‒ Functional improvement (Shee
Slide 23: Planned Phase 3 program in PTSD
23 © Copyright 2016 Tonix Pharmaceuticals Planned Phase 3 program in PTSD General Study Characteristics: • Randomized, double - blind, placebo - controlled, entrance CAPS - 5 > 33 • At least one unblinded interim analyses (IA) • First IA (N ~180) for efficacy stop, futility or sample size adjustment
Slide 24: Intellectual property
24 © 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 ‒ PTSD : patents filed TNX - 102 SL Wholly - owned by Tonix w
Slide 25: Cyclobenzaprine: active pharmaceutical ingredient
25 © 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 26: Receptor binding profile: Parent and primary metabolite
26 © Copyright 2016 Tonix Pharmaceuticals Receptor binding profile: Parent and primary metabolite 0 5 10 15 20 25 30 35 40 45 50 5 - HT 2c H 1 5 - HT 2a a 2 c , NET 5 - HT 2b Undesirable Characteristics of Norcyclobenzaprine (nCBP) • Half - life (t 1/2 ) of 72 hours (will accumulate) • At steady - s
Slide 27: TNX-102 SL 2.8 mg PK profile relative to oral immediate release CBP
27 © Copyright 2016 Tonix Pharmaceuticals TNX - 102 SL 2.8 mg PK p rofile relative to 5 mg oral immediate release (IR) CBP: TNX - 102 SL single - dose PK study 0 500 1000 1500 2000 2500 3000 3500 4000 0 4 8 12 16 20 24 Concentration (ng/mL) TNX - 102 SL 2.8 mg Oral IR CBP 5 mg x Lower exposure of nC
Slide 28: TNX-102 SL: Pharmacokinetic profile after sublingual administration
28 © 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) ‒ Reaching t max at ~4 hours after dosing ‒ Approximately 50% of exposure to cyclobe
Slide 29: Cyclobenzaprine is detected in plasma within minutes following sublingual TNX-102 SL
29 © 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 30: Management team
30 © 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 31: Board of directors
31 © 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 32: Milestones – recent and upcoming – Posttraumatic Stress Disorder
32 © Copyright 2016 Tonix Pharmaceuticals Milestones – recent and upcoming TNX - 102 SL – Posttraumatic Stress Disorder □ December 2015 Entered into Collaborative Research and Development Agreement (CRADA) with the United States Army Medical Materiel Development Activity (USAMMDA) □ May 2016 Report
Slide 33: - - 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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