Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (May 2016)
38 slides from Tonix Pharmaceuticals's investor presentation, May 10, 2016
Slide 1: Investor Presentation May 2016
© Copyright 2016 Tonix Pharmaceuticals - Confidential - Do not duplicate or distribute NASDAQ: TNXP Investor Presentation May 2016 © 2016 Tonix Pharmaceuticals Holding Corp. Version: P0017 - 05 - 9 - 16
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 ( Tonmya ® *) Fibromyalgia Top line data 3Q 2016 TNX - 102 SL Post - Traumatic Stress Disorder Top line data 2Q 2016 (May 2016) Pipeline led by TNX - 102 SL for fibromyalgia Preclinical Phase 1 Phase 2 Ph
Slide 5: Fibromyalgia is inappropriate central pain signaling in the absence of 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 is a chronic, debilitating disorder with 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 a prevalent disorder but remains 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 those treated receive sustained benefit from marketed drugs
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: Enrollment completed in Phase 3 trial for fibromyalgia
14 © Copyright 2016 Tonix Pharmaceuticals • Phase 3 AFFIRM Study is fully enrolled • Second Phase 3 Study (“REAFFIRM”) expected to begin in 2Q 2016 - Expected to be similar to AFFIRM in design and sample size Enrollment completed in TNX - 102 SL in Phase 3 trial for fibromyalgia • Randomized, double
Slide 15: TNX-102 SL in development for post-traumatic stress disorder
15 © Copyright 2016 Tonix Pharmaceuticals TNX - 102 SL in Phase 2 development for post - traumatic stress disorder (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. NDA = New Drug Application; FDA = U.
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 a prevalent problem for both 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 20
Slide 18: PTSD veteran population: recent conflicts only
18 © Copyright 2016 Tonix Pharmaceuticals PTSD veteran population: recent conflicts only Veteran Administration (VA) records indicate that 20% of veterans from recent conflicts will have potential or provisional PTSD Source: Report on VA Facility Specific Operation Enduring Freedom (OEF), Operation
Slide 19: PTSD veteran population: all conflicts
19 © Copyright 2016 Tonix Pharmaceuticals PTSD veteran population: all conflicts 1 Bernardy et al., J Clin Psychiatry, 2012, 73:297 - 303. 2 Bowe et al, J Dual Diagnosis, 2014, 11:22 - 32. The number of veterans in the VA system and diagnosed with PTSD has been rising 1,2 • Does not include veterans
Slide 20: Significant gap in current therapeutic landscape for PTSD
20 © Copyright 2016 Tonix Pharmaceuticals Significant gap in current therapeutic landscape for PTSD • Medicines approved for PTSD often provide inadequate and/or inconsistent benefit ‒ Limited to two SSRI antidepressants, both of which carry suicidality warnings ‒ U.S. Institute of Medicine (IOM) co
Slide 21: Targeting sleep quality is a novel mechanism of action in PTSD therapy
21 © Copyright 2016 Tonix Pharmaceuticals Targeting sleep quality is a novel mechanism of action in PTSD therapy • PTSD patients complain of sleep disturbance as a core symptom 1 ‒ Distressing dreams (nightmares) are part of “re - experiencing” ‒ Avoidance can be of bed/sleep due to fear of nightmar
Slide 22: AtEase study: primary endpoint and power considerations
22 © Copyright 2016 Tonix Pharmaceuticals Tonix’s AtEase study in military - related PTSD: primary endpoint and power considerations • Primary endpoint is the change from baseline of the Clinician Administered PTSD Scale for DSM - 5 (CAPS - 5) ‒ Raters are trained, certified, and monitored for relia
Slide 23: Limitations of current FDA-approved pharmacotherapies for PTSD
23 © Copyright 2016 Tonix Pharmaceuticals Limitations of current FDA - approved pharmacotherapies for PTSD • Britain’s National Institute for Clinical Excellence (NICE) National Clinical Practice Guideline for management of PTSD reported an effect size in paroxetine trials of 0.42 and for sertraline
Slide 24: TNX-102 SL potential in PTSD supported by clinical evidence and pharmacology
24 © Copyright 2016 Tonix Pharmaceuticals TNX - 102 SL’s potential as a treatment for PTSD is supported by clinical evidence and pharmacology • TNX - 102 SL is active on receptor sites believed to have treatment potential for sleep problems in PTSD ‒ Targeted receptors include 5 - HT2a, alpha - 1 ad
Slide 25: Phase 2 AtEase trial in PTSD is fully enrolled
25 © Copyright 2016 Tonix Pharmaceuticals Phase 2 “ AtEase ” trial of TNX - 102 SL in PTSD is fully enrolled • Randomized, double - blind, placebo - controlled trial in military - related PTSD • Randomized>240; approximately 25 U.S. clinical sites • Primary efficacy endpoint: ‒ Difference in Clinici
Slide 26: Intellectual property
26 © 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 27: Active pharmaceutical ingredient
27 © Copyright 2016 Tonix Pharmaceuticals TNX - 102 SL: a ctive pharmaceutical i ngredient • 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 28: Properties of cyclobenzaprine and norcyclobenzaprine
28 © Copyright 2016 Tonix Pharmaceuticals Properties of cyclobenzaprine and its metabolite norcyclobenzaprine : opportunity to modify pharmacokinetic profile 0 5 10 15 20 25 30 35 40 45 50 Receptor Affinity (Ki ( nM ) M 1 α 1b D 1 5HT 2b α 2b α 2c 5 - HT Transporter D3 NE Transporter 5 - HT 2c H 1 5
Slide 29: Cyclobenzaprine detected in plasma within minutes following sublingual dosing
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: Pharmacokinetic profile after sublingual administration
30 © 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 31: Somnolence and dry mouth with oral and sublingual cyclobenzaprine
31 © 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 c yclobenzaprine in f ibromyalgia patients • ORAL – Phase 2a 1 : ‒ Dose TNX - 102 (CBP capsules, 1 mg) ‒ Dosing regimen ‒ titrated from 1 mg to a
Slide 32: Formulation with base increases systemic absorption
32 © Copyright 2016 Tonix Pharmaceuticals Formulation with base increases systemic absorption of sublingual cyclobenzaprine 1 Concentration gradient increases diffusion of free base across oral mucosa (Le Chatelier’s Principle) CBP - HCl Cyclobenzaprine free base Oral mucosa CBP - HCl Low pH (acidic
Slide 33: Proprietary eutectic mixture stabilizes tablet formulation
33 © Copyright 2016 Tonix Pharmaceuticals Proprietary cyclobenzaprine hydrochloride eutectic mixture stabilizes tablet formulation 1 Base particle (K 2 HPO 4 ) Base particle (K 2 HPO 4 ) Base particle (K 2 HPO 4 ) Eutectic formulation Protective crystal Cyclobenzaprine - HCl (CBP - HCl) Eutectic for
Slide 34: Financial overview
34 © Copyright 2016 Tonix Pharmaceuticals Financial overview NASDAQ: TNXP Cash, cash equivalents, and marketable securities reported at March 31, 2016 $ 27.5 million Shares outstanding (May 9, 2016) 18.9 million
Slide 35: Management team
35 © Copyright 2016 Tonix Pharmaceuticals Management team Seth Lederman, MD President & CEO Jessica Edgar Morris EVP, Administration Bruce Daugherty, PhD, MBA Chief Scientific Officer Ronald Notvest, PhD EVP, Commercial Planning & Development Gregory Sullivan, MD Chief Medical Officer Bradley Saenge
Slide 36: Board of directors
36 © 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 37: Milestones – recent and upcoming
37 © 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 P3 AFFIRM study □ Q3 2016
Slide 38: - - 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
Tonix Pharmaceuticals Holding — Investor Presentation (May 2016) on 6ix