Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (June 2015)
38 slides from Tonix Pharmaceuticals's investor presentation, Jun 4, 2015
Slide 1: June 2015
NASDAQ: TNXP June 2015 © 2015 Tonix Pharmaceuticals Holding Corp.
Slide 2: Cautionary note on forward-looking statements
2 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 Reform Act of 1995 . These statements m
Slide 3: Developing innovative medicines for large and growing markets
3 Developing innovative medicines for large and growing markets Common and chronic disorders of the central nervous system ( CNS) Next - generation medicines with transformative treatment potential Late - stage candidates supported by human experience C apitalized to achieve key readouts in all of o
Slide 4: Pipeline led by TNX-102 SL for fibromyalgia
4 Pipeline led by TNX - 102 SL for fibromyalgia Preclinical Phase 1 NDA Market Candidate Indication Phase 2 Phase 3 TNX - 102 SL (cyclobenzaprine HCl sublingual tablet) and TNX - 201 ( dexisometheptene mucate ) are Investigational New Drugs and are not approved for any indication. TNX - 102 SL TNX -
Slide 5: Fibromyalgia: a chronic, multi-symptom disorder that generates frustration
5 Fibromyalgia: a chronic, multi - symptom disorder that generates frustration for patients and physicians 1 Phillips K & Clauw DJ, Best Pract Res Clin Rheumatol . 2011;25:141. 2 Robinson et al, Pain Medicine. 2013;14:1400. 3 White et al, J Occupational Environ Med 2008;50:13. Fibromyalgia is charac
Slide 6: Affects ~6% (5 million) Americans and typically persists for years to decades
6 Affects 2 - 6% (5 - 15 million) Americans 1 and typically persists for years to decades Onset most frequent in the 30’s - 40’s, predominantly in females Diagnosis rate of 1.1% = 2.7 million U.S. adults ; suggests under - diagnosis Among those diagnosed, 85% receive treatment 2 = 2.3 million U.S. a
Slide 7: Fewer than half of those treated receive sustained benefit from approved drugs
7 Receive long - term benefit Respond, but intolerant of side effects Do not respond 25% 35% 40% 60% 1 Market research by Frost & Sullivan, commissioned by Tonix (2011). FDA = U.S. Food and Drug Administration …and fewer than half of those treated receive sustained benefit from the three FDA - appro
Slide 8: Side effects are the most common driver of treatment discontinuation
8 Side effects are the most common driver of treatment discontinuation 1 Robinson et al., Pain Medicine 2013;14:1400. 63% 30% 15% 7% 5% 1% 0% 10% 20% 30% 40% 50% 60% 70% Adverse Events Lack of Efficacy Other reasons Too Costly No answer Felt Better Reasons for Discontinuation (results from large lon
Slide 9: Relief of several symptoms is important to patients
9 0 1 2 3 4 5 6 7 8 Symptom Intensity During Past Week (Mean ) Relief of several symptoms is important to patients Source: Bennett RM et al, BMC Musculoskelet Disord 2007;8:27.
Slide 10: High rates of discontinuation, switching and augmentation
10 1 Robinson RL et al, Pain Medicine 2012;13:1366. 2 “Patient Trends: Fibromyalgia”, Decision Resources, 2011. High rates of discontinuation, switching and augmentation Patients cycle through different medications ; a ttempt to treat multiple symptoms and/or avoid intolerable side effects Two or mo
Slide 11: Advanced sublingual tablet containing cyclobenzaprine 2.8 mg
11 Advanced sublingual tablet containing cyclobenzaprine ( CBP) 2.8 mg Eutectic formulation rapidly delivers a low dose of CBP Avoids first - pass metabolism ; reduces exposure to long - lived active metabolite Designed for chronic bedtime administration, no titration TNX - 102 SL demonstrated broad
Slide 12: Phase 2b BESTFIT study in fibromyalgia
12 Phase 2b “BESTFIT” study 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 13: BESTFIT: TNX-102 SL 2.8 mg broadly improved fibromyalgia
13 BESTFIT: TNX - 102 SL 2.8 mg broadly improved fibromyalgia Category Endpoint – week 12 1 p value Pain 30% responder analysis 2 0.033 Sleep Daily Sleep Quality PROMIS Sleep Disturbance <0.001 0.005 Overal l response to therapy PGIC 0.025 Assessment of disease impact FIQ - R total score 0.014 1 Int
Slide 14: TNX-102 SL 2.8 mg was very well tolerated in the BESTFIT study
14 TNX - 102 SL 2.8 mg 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 t
Slide 15: Phase 3 AFFIRM study of TNX-102 SL is underway
15 Phase 3 “AFFIRM” study of TNX - 102 SL is underway Randomized, double - blind, placebo - controlled study in f ibromyalgia N=500; approximately 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
Slide 16: TNX-102 SL in development for PTSD
16 TNX - 102 SL in development for PTSD Preclinical Phase 1 NDA Market Candidate Indication Phase 2 Phase 3 TNX - 102 SL TNX - 102 SL TNX - 201 Post - Traumatic Stress Disorder Top line data 1 H 2016 PTSD = post - traumatic stress disorder TNX - 102 SL and TNX - 201 are Investigational New Drugs and
Slide 17: PTSD is a chronic disorder following a traumatic event
17 PTSD is a chronic disorder following a traumatic event and is characterized by : Considered a stress response, but prolonged and does not resolve with time Of those who experience significant trauma, ~15% develop PTSD (20% of women, 8% of men) 1 Associated with significant life disruption Social
Slide 18: PTSD is a large problem for both civilians and the military
18 Affects 3.5% of adult Americans = 8.5 million individuals 1 ~70% are considered to have moderate to severe symptoms Of those diagnosed, ~50% utilize professional healthcare (psycho/pharmacotherapy) 2 Higher prevalence in military population 2 0 % of veterans from recent conflicts will have potent
Slide 19: Medicines for PTSD often provide inadequate and/or inconsistent benefit
19 Medicines for PTSD often provide inadequate and/or inconsistent benefit FDA - approved medications are limited to two SSRIs, approved >10 years ago Weak evidence of treatment effect in men 1 Lack of evidence of efficacy in those with a history of combat - related trauma 2 Carry suicidality warnin
Slide 20: TNX-102 SL's potential as a treatment for PTSD is supported by clinical evidence
20 TNX - 102 SL’s potential as a treatment for PTSD is supported by clinical evidence and non - clinical activities Outcome Measure at Week 12 in BESTFIT 1 p value PROMIS Sleep Disturbance 0.005 FIQ - R Anxiety Item 0.015 FIQ - R Sensitivity Item 0.017 TNX - 102 SL targets mechanisms associated with
Slide 21: Phase 2 AtEase trial of TNX-102 SL in PTSD is ongoing
21 Phase 2 “AtEase” trial of TNX - 102 SL in PTSD is 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 22: TNX-201 in development for episodic tension-type headache
22 TNX - 201 in development for episodic tension - type headache Preclinical Phase 1 NDA Market Candidate Indication Phase 2 Phase 3 TNX - 102 SL TNX - 102 SL TNX - 201 Episodic Tension - Type Headache Top line data 4Q 2015 TNX - 102 SL and TNX - 201 are Investigational New Drugs and are not approve
Slide 23: Episodic tension-type headache (ETTH)
23 Episodic tension - type headache (ETTH) 1 Schwartz et al., JAMA 1998;279:381 - 383; Chowdhury, Ann Ind Acad Neurol 2012;15:83 - 88; Tonix analysis of public literature. 2 Scher et al., Cephalalgia 2010;30:321 - 328; Tonix analysis of public literature . 3 Based on independent study conducted by T
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 - mi
Slide 25: ETTH is the most common type of headache
25 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) 1 1 Schwartz et al., JAMA 1998;279:381; U.S. Census Bureau, 2013 Projection. 2 Stovner et al, Cephalalgia 2007
Slide 26: TNX-201 is a modern form of a medicine with a long history of use
26 TNX - 201 is a modern form of a medicine with a long history of use TNX - 201 ( dexisometheptene mucate ) a single 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 27: Racemic isometheptene combination prescriptions had been commonly written
27 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 28: Optical isomers of IMH have distinct pharmacological activities
28 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 29: TNX-201 was well tolerated in Phase 1 study
29 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 is an Investigational New Drug and is not approved for any indicati
Slide 30: Proof-of-concept Phase 2 trial of TNX-201 in ETTH
30 Proof - of - concept Phase 2 trial of TNX - 201 in ETTH TNX - 201 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 sites Top line data expected 4
Slide 31: TNX-201 is active on the imidazoline-1 receptor
31 TNX - 201 is active on the imidazoline - 1 receptor ( I 1 - R): a novel target for the treatment of pain Hot Plate Test Characteristics 1 Transmembrane receptor Distinct from α 2 AR and MAO receptor subtypes No sequence similarity to GPCRs or ATP - sensitive K+ channels Shares similarities to rya
Slide 32: Intellectual property
32 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 33: Financial overview
33 Financial overview NASDAQ: TNXP Cash reported at March 31 , 2015 $ 58.2 million Shares outstanding ( June 3, 2015) 16.1 million
Slide 34: Management team
34 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 Management team
Slide 35: Board of directors
35 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 Davidson Labrador Ventures, Alkermes , Combion Patrick Gr
Slide 36: Milestones – recent and upcoming
36 TNX - 102 SL – Fibromyalgia □ May 2015 Began Phase 3 AFFIRM study □ June 2015 Present additional data from Phase 2b BESTFIT study at EULAR □ 2H 2016 Report top - line results from AFFIRM study TNX - 102 SL – Post - Traumatic Stress Disorder □ January 2015 Began Phase 2 AtEase study in military -
Slide 37: Developing innovative medicines for large and growing markets
37 Developing innovative medicines for large and growing markets Common and chronic disorders of the central nervous system ( CNS) Next - generation medicines with transformative treatment potential Late - stage candidates supported by human experience C apitalized to achieve key readouts in all of
Slide 38: 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 (June 2015) on 6ix