Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (May 2015)
36 slides from Tonix Pharmaceuticals's investor presentation, May 1, 2015
Slide 1: May 2015
NASDAQ: TNXP May 2015 © 2015 Tonix Pharmaceuticals Holding Corp.
Slide 2: Safe harbor statement
2 Safe harbor statement 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 us
Slide 3: Financial summary
3 Financial summary NASDAQ: TNXP Cash reported at December 31 , 2014 $ 38.2 million Pro forma for n et proceeds from common stock offering in 1Q15 $ 67.2 million Shares outstanding (April 30, 2015) 16.1 million
Slide 4: Tonix is developing innovative medicines for large markets
4 Tonix is developing innovative medicines for large markets Common and chronic disorders of the central nervous system ( CNS) Next - generation medicines, novel treatment paradigms Late stage candidates with mitigated development risk C apitalized to achieve key readouts in all of our clinical - st
Slide 5: Pipeline led by TNX-102 SL for fibromyalgia
5 Pipeline led by TNX - 102 SL for fibromyalgia (FM) Preclinical Phase 1 NDA Market Candidate Indication Phase 2 Phase 3 TNX - 102 SL Began Phase 2 trial - Jan ‘15 TNX - 102 SL Fibromyalgia Start Phase 3 trial - 2Q15 Start Phase 2 trial - 2Q15 TNX - 201 TNX - 102 SL (cyclobenzaprine HCl sublingual t
Slide 6: Fibromyalgia is a large pharmaceutical market
6 Fibromyalgia is a larg e pharmaceutical market * Lawrence et al, Arthritis Rheum 2008;58:26 - 35; Vincent et al, Arthritis Care Res 2013;65:786 - 792. Fibromyalgia is a chronic disorder characterized by: chronic widespread pain fatigue unrefreshed sleep diminished cognition Believed to result from
Slide 7: Fewer than half of treated patients receive sustained benefit
7 Receive long term benefit Respond, but intolerant of side effects Do not respond Market research by Frost & Sullivan (commissioned by Tonix ). * Robinson RL et al, Pain Medicine 2012;13:1366 - 76. 25% 35% 40% Fewer than half of those treated receive sustained benefit from the FDA - approved drugs
Slide 8: Patients indicate that the symptom burden is high
8 Patients indicate that the symptom burden is high 0 1 2 3 4 5 6 7 8 Symptom Intensity During Past Week (Mean) Relief of many symptoms is important to patients Bennett et al, 2007; survey of 2,569 FM patients.
Slide 9: Tonix approaches fibromyalgia by targeting sleep quality
9 Tonix approaches the treatment of fibromyalgia by targeting sleep quality >90% of fibromyalgia patients complain of poor sleep quality* Sublingual tablet formulation of low - dose cyclobenzaprine (CBP) TNX - 102 SL is designed for chronic, bedtime administration 505(b)(2) regulatory pathway TNX -
Slide 10: Phase 2b BESTFIT trial in fibromyalgia
10 Phase 2b “BESTFIT” trial 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 criteria for fibromyalgia 205 participants were randomized 1:1 at
Slide 11: TNX-102 SL broadly improved fibromyalgia in the BESTFIT study
11 TNX - 102 SL broadly improved fibromyalgia in the BESTFIT study Outcome Measure at Week 12 Declared Secondary Endpoints p value Daily Pain Diary, NRS Proportion Achieving 30% Improvement* 0.033 PROMIS Sleep Disturbance T - score Change 0.005 Patient Global Impression of Change Responder Analysis
Slide 12: TNX-102 SL was very well-tolerated in the BESTFIT study
12 TNX - 102 SL was very well - tolerated in the BESTFIT study No serious adverse events (SAE) reported with TNX - 102 SL Most frequent local adverse events were administration site reactions Previously reported in TNX - 102 SL Phase 1 studies; no detectable bias on efficacy results Transient tongue
Slide 13: Phase 3 AFFIRM study of TNX-102 SL in fibromyalgia
13 Phase 3 “AFFIRM” study of TNX - 102 SL in fibromyalgia Randomized, double - blind, placebo - controlled trial in f ibromyalgia N=500; 30 - 35 U.S. clinical sites Primary efficacy endpoint: Difference in 30 % responder analysis at 12 weeks between TNX - 102 SL 2.8 mg and placebo Placebo once - dai
Slide 14: Phase 2 trial of TNX-102 SL for PTSD is recruiting
14 Phase 2 trial of TNX - 102 SL for post - traumatic stress disorder (PTSD) is recruiting Preclinical Phase 1 NDA Market Candidate Indication Phase 2 Phase 3 Post - Traumatic Stress Disorder TNX - 102 SL Began Phase 2 trial - Jan ‘15 TNX - 102 SL Start Phase 3 trial - 2Q15 Start Phase 2 trial 2Q15
Slide 15: PTSD is a chronic, debilitating condition
15 Post - traumatic stress disorder is a chronic, debilitating condition Experiencing any trauma can lead to PTSD High incidence among soldiers and veterans Associated with suicide and unpredictable, violent behaviors 8.5 million U.S. patients A pproximately half are receiving medical treatment * Li
Slide 16: High prevalence of PTSD among combat veterans
16 High prevalence of PTSD among combat veterans 1. Norris, PTSD Res Quar. 2013. 2. Gradus JL, National Center for PTSD. 3. Tanielan , Invisible wounds of war. 2005. 4. CBO Report 2012 . 19 - 31% Vietnam veterans 1 10% Gulf war veterans 2 10 - 21% Operation Enduring Freedom (Afghanistan) / Operation
Slide 17: Military PTSD
17 Military PTSD Convoy attack on Route Appaloosa: “Ambush Alley”
Slide 18: PTSD patients complain of poor sleep quality as a core symptom
18 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” Correlated with depression, substance abuse and suicide Poor sleep quality after trauma may increase the risk of developing PT
Slide 19: Fibromyalgia program informs development in PTSD
19 Fibromyalgia program informs TNX - 102 SL development in PTSD 2.8 mg dose supported by BESTFIT study results Improvements observed in BESTFIT on several metrics that relate to PTSD core symptoms TNX - 102 SL (cyclobenzaprine HCl sublingual tablet) 2.8 mg is an Investigational New Drug and is not
Slide 20: Phase 2 AtEase trial of TNX-102 SL in PTSD
20 Phase 2 “ AtEase ” trial of TNX - 102 SL in PTSD – ongoing Randomized, double - blind, placebo - controlled trial in military - related PTSD N=220; approximately 25 U.S. clinical sites Primary efficacy endpoint: Difference in Clinician - Administered PTSD Scale (CAPS) score between TNX - 102 SL 2
Slide 21: TNX-201 in development for episodic tension-type headache
21 TNX - 201 in development for episodic tension - type headache Preclinical Phase 1 NDA Market Candidate Indication Phase 2 Phase 3 TNX - 102 SL Began Phase 2 trial – Jan ‘15 TNX - 102 SL Start Phase 3 trial - 2Q15 Start Phase 2 trial - 2Q15 TNX - 201 Episodic Tension - Type Headache TNX - 102 SL (
Slide 22: Episodic tension-type headache (ETTH)
22 Episodic tension - type headache (ETTH) * Schwartz et al., JAMA 1998;279:381 - 383; Chowdhury, Ann Ind Acad Neurol 2012;15:83 - 88; Tonix analysis of public literature. ** Scher et al., Cephalalgia 2010;30:321 - 328; company analysis of public literature. 75 million adults in the U.S. experience
Slide 23: ETTH is the most common type of headache
23 ETTH is the most common type of headache 60% 38% 13% 2% 0% 20% 40% 60% All Headaches ETTH Migraine Chronic One - Year Prevalence Rate for U.S. Adult Population (18 - 65) Adults (18 - 65)* * Estimated from 2013 U.S. Census Schwartz et al., JAMA 1998;279:381 - 383; Stovner et al, Cephalalgia 2007;2
Slide 24: Patients with ETTH seek medical attention
24 Patients with ETTH seek medical attention 1.7 7.6 4.5 4.7 0 2 4 6 8 Emergency Room Office Visits Number of Patients in Millions Care - Seeking For Non - Migraine Headache Migraine Non - Migraine Health Care Utilization Project data, 2011; IMS National Disease and Therapeutic Index™ 2013 Non - mig
Slide 25: TNX-201 is a modern form of a medicine with a long history of use
25 TNX - 201 is a modern form of a medicine with a long history of use TNX - 201 ( dexisometheptene mucate ) a pure optical isomer of isometheptene (IMH) A mixture of IMH optical isomers had been widely prescribed for many decades “ Racemic isometheptene ” was a single - agent medicine (pre - 1962)
Slide 26: Racemic isometheptene combination prescriptions had been commonly written
26 Racemic isometheptene combination ( RIC) prescriptions had been commonly written Usage of RIC Prescriptions for All Diagnoses 2.44 2.43 2.50 2.45 2.45 2.35 0 1 2 3 1995 1996 1997 1998 1999 2000 Number of Prescriptions (Millions) Other Non-Migraine (eg, ETTH) Migraine Source : IMS Health, National
Slide 27: Optical isomers of IMH have distinct pharmacological activities
27 Optical isomers of IMH have distinct pharmacological activities (R) isomer (S) isomer • Analgesic • Binds to imidazoline - 1 receptor • Inactive on adrenergic receptors • Sympathomimetic Previously marketed IMH drugs contained a mixture of two mirror - image isomers (racemic IMH) Tonix is develop
Slide 28: TNX-201 was well-tolerated in Phase 1 study
28 TNX - 201 was well - tolerated in Phase 1 study Phase 1 study in healthy volunteers Single ascending dose study (N=45) – three cohorts of 15 subjects Randomized to TNX - 201, racemic IMH, or placebo (3:1:1 ratio, resp.) TNX - 201 ( dexisometheptene mucate ) is an Investigational New Drug and is n
Slide 29: Proof-of-concept Phase 2 trial of TNX-201 in ETTH
29 Proof - of - concept Phase 2 trial of TNX - 201 in ETTH TNX - 201 ( dexisometheptene mucate ) is an Investigational New Drug and is not approved for any indication. Randomized, double - blind, placebo - controlled trial in episodic tension - type headache N=200 ; approximately 10 U.S. clinical si
Slide 30: TNX-201 is active on the imidazoline-1 receptor
30 TNX - 201 is active on the imidazoline - 1 receptor ( I 1 - R): a novel target for the treatment of pain Hot Plate Test Characteristics Transmembrane receptor Distinct from α 2 AR and MAO receptor subtypes No sequence similarity to GPCRs or ATP - sensitive K+ channels Shares similarities to ryano
Slide 31: Intellectual property
31 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, 3Q 2020 expiry PTSD: patents filed TNX - 102 SL Fibromyalgia, PTSD TNX - 201 Headach
Slide 32: Management team
32 Management team Seth Lederman, MD President & CEO Leland Gershell, MD, PhD Chief Financial Officer Bruce Daugherty, PhD Chief Scientific Officer Ronald Notvest, PhD SVP, Commercial Planning & Development Gregory Sullivan, MD Chief Medical Officer
Slide 33: Board of directors
33 Board of directors Seth Lederman, MD Chairman Ernest Mario, PhD ALZA, Glaxo, Reliant Pharma John Rhodes NYSERDA, Booz Allen Hamilton Samuel Saks, MD Jazz Pharma, ALZA, Johnson & Johnson Charles Mather BTIG, Janney, Jefferies, Cowen, Smith Barney Stuart Davidson Labrador Ventures, Alkermes , Combi
Slide 34: Shareholder summary
34 Shareholder summary Based on public filings as of April 30, 2015. Broadfin Capital Kingdon Capital Management Technology Partners Fund VIII Deerfield Special Situations Fund Franklin Templeton Investments Lombard Odier Investment Management Wall Street Associates 5%+ holders
Slide 35: Milestones – recent and upcoming
35 TNX - 102 SL – Fibromyalgia □ September 2014 – Reported top line results from Phase 2b BESTFIT study □ January 2015 – Reported FDA acceptance of 30% responder analysis as Phase 3 primary endpoint □ 2Q 2015 – Begin Phase 3 AFFIRM study □ 2H 2016 – R eport top - line results from AFFIRM study TNX -
Slide 36: Madison Avenue New York, NY 10022 (212) - 9155 www.tonixpharma.com
NASDAQ: TNXP 509 Madison Avenue New York, NY 10022 (212) 980 - 9155 www.tonixpharma.com
Tonix Pharmaceuticals Holding — Investor Presentation (May 2015) on 6ix