Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (July 2015)
47 slides from Tonix Pharmaceuticals's investor presentation, Jul 7, 2015
Slide 1: Corporate Presentation July 2015
NASDAQ: TNXP Corporate Presentation July 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 Capitalized to achieve key readouts in all of our
Slide 4: Pipeline led by Tonmya for fibromyalgia
4 Candidate Indication Near - term Catalyst Tonmya Fibromyalgia Top line data H2 2016 TNX - 102 SL PTSD Top line data H1 2016 TNX - 201 Episodic Tension - Type Headache Top line data Q4 2015 Pipeline led by Tonmya ™ for fibromyalgia (cyclobenzaprine HCl sublingual tablets, 2.8 mg) Preclinical Phase
Slide 5: Fibromyalgia: a chronic, multi-symptom disorder
5 Fibromyalgia: a chronic, multi - symptom disorder that generates frustration for patients and physicians Fibromyalgia is characterized by : Believed to result from amplified sensory and pain signaling in CNS 1 Causes significant impairment in all areas of life - Lower levels of health - related qu
Slide 6: Fibromyalgia is a large market, but remains under-diagnosed
6 Fibromyalgia is a large market, but remains under - diagnosed… 1 Lawrence et al, Arthritis Rheum 2008;58:26; Vincent et al, Arthritis Care Res 2013;65:786; Jones et al, 2015;67:568. 2 Robinson RL et al, Pain Med 2012;13:1366. 3 Independent study conducted by IMS Consulting Group, April 2015 using
Slide 7: Fewer than half of those treated receive sustained benefit
7 …and fewer than half of those treated receive sustained benefit from the three FDA - approved drugs The treatment objective is to restore functionality and quality of life by broadly improving symptoms while avoiding significant side effects The majority discontinue therapy due to lack of a respon
Slide 8: Side effects are the most common driver of 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 Relief of several symptoms is important to patients 0 1 2 3 4 5 6 7 8 Symptom Intensity During Past Week ( Mean) 1 1 Bennett RM et al, BMC Musculoskelet Disord 2007;8:27.
Slide 10: Pervasive treatment dissatisfaction creates an opportunity
10 Pervasive treatment dissatisfaction creates an opportunity for a differentiated therapeutic option 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 more med
Slide 11: Tonmya in Phase 3 clinical development for fibromyalgia
11 Tonmya in Phase 3 clinical development for fibromyalgia 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 a
Slide 12: CBP detected in plasma within minutes following sublingual administration
12 CBP is detected in plasma within minutes following sublingual administration of Tonmya in Phase 1 studies Tonmya / TNX - 102 SL is an Investigational New Drug and is not approved for any indication. Time (hours)
Slide 13: Tonmya is a proprietary eutectic formulation of low dose CBP
13 Tonmya is a proprietary eutectic formulation of low dose CBP engineered for sublingual transmucosal absorption 1. Transmucosal absorption requires a basic excipient. 3. Eutectic composition protects cyclobenzaprine HCl from degradation caused by the basic excipient. 2. However, a basic excipient
Slide 14: Phase 2b BESTFIT study of Tonmya in fibromyalgia
14 Phase 2b “BESTFIT” study of Tonmya 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 ra
Slide 15: BESTFIT: Tonmya broadly improved fibromyalgia
15 BESTFIT: Tonmya 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 Intent - to - tr
Slide 16: BESTFIT: significantly improved 30% pain responder rate
16 BESTFIT: Tonmya significantly improved the 30% pain responder rate Source: Phase 2b BESTFIT study data. Tonmya / TNX - 102 SL is an Investigational New Drug and is not approved for any indication. A 30% responder is an individual whose pain has decreased ≥30% from baseline to week 12 In a respond
Slide 17: BESTFIT: Tonmya significantly improved sleep quality
17 BESTFIT: Tonmya significantly improved sleep quality Source: Phase 2b BESTFIT study data. Tonmya / TNX - 102 SL is an Investigational New Drug and is not approved for any indication.
Slide 18: BESTFIT: improved Daily Diary Sleep Quality scores
18 - 0.88 - 1.85 -2 -1.8 -1.6 -1.4 -1.2 -1 -0.8 -0.6 -0.4 -0.2 0 0 2 4 6 8 10 12 Placebo TNX-102 SL BESTFIT: Tonmya significantly improved weekly average of Daily Diary Sleep Quality scores Mean change from Baseline Study Week P <.001 Source: Phase 2b BESTFIT study data. Tonmya / TNX - 102 SL is an
Slide 19: BESTFIT: significantly improved FIQ-R Total scores
19 BESTFIT: Tonmya significantly improved FIQ - R T otal scores Source: Phase 2b BESTFIT study data. Tonmya / TNX - 102 SL is an Investigational New Drug and is not approved for any indication.
Slide 20: BESTFIT: More FIQ-R responders with Tonmya vs. placebo
20 BESTFIT: More FIQ - R responders with Tonmya vs. placebo at all levels Source: Phase 2b BESTFIT study data. Tonmya / TNX - 102 SL is an Investigational New Drug and is not approved for any indication.
Slide 21: BESTFIT: Tonmya improved PGIC responder rate
21 BESTFIT: Tonmya improved Week 12 PGIC responder rate Source : Phase 2b BESTFIT study data. Tonmya / TNX - 102 SL is an Investigational New Drug and is not approved for any indication.
Slide 22: Tonmya was well tolerated in the BESTFIT study
22 Tonmya was well tolerated in the BESTFIT study No serious adverse events (SAE) reported with Tonmya Most frequent local adverse events were administration site reactions - Previously reported in Phase 1 studies; no detectable bias on efficacy results - Transient tongue numbness (42% Tonmya vs . 1
Slide 23: BESTFIT: oral adverse events did not affect outcomes
23 BESTFIT: Presence of oral adverse events did not lead to meaningful differences in outcome measures Source: Phase 2b BESTFIT study data. Tonmya / TNX - 102 SL is an Investigational New Drug and is not approved for any indication.
Slide 24: Phase 3 AFFIRM Study is underway
24 Phase 3 AFFIRM Study is underway Second Phase 3 clinical trial expected to begin in 2Q 2016 - Currently expected to be similar to AFFIRM in design and size Tonmya in Phase 3 registration program in fibromyalgia Randomized, double - blind, placebo - controlled study in fibromyalgia N=500; approxim
Slide 25: TNX-102 SL in Phase 2 development for PTSD
25 Preclinical Phase 1 Phase 2 Phase 3 NDA Market TNX - 102 SL in Phase 2 development for PTSD PTSD = post - traumatic stress disorder Tonmya / T NX - 102 SL and TNX - 201 are Investigational New Drugs and are not approved for any indication . Candidate Indication Near - term Catalyst TNX - 102 SL P
Slide 26: PTSD: An important and growing public health problem
26 PTSD: An important and growing public health problem 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 me
Slide 27: PTSD is a large problem for civilians and the military
27 PTSD is a large problem for both civilians and the military ~70% are considered to have moderate to severe symptoms Of those diagnosed, ~50% utilize professional healthcare (psycho/pharmacotherapy) 2 1 Kessler RC at al, Arch Gen Psychiatry 2013;62:617; U.S. Census Bureau, 2013 Projection. 2 Wang
Slide 28: Significant gap in current therapeutic landscape for PTSD
28 Significant gap in current therapeutic landscape for PTSD 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
Slide 29: TNX potential in PTSD supported by clinical and nonclinical evidence
29 TNX - 102 SL’s potential as a treatment for PTSD is supported by clinical evidence and nonclinical activities TNX - 102 SL targets mechanisms associated with treating disturbed sleep in PTSD - 5 - HT2A receptor antagonist and reuptake inhibitor (like trazodone ) - Alpha - 1 adrenergic receptor an
Slide 30: Phase 2 AtEase trial in PTSD is ongoing
30 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 S
Slide 31: TNX-201 in Phase 2 development for episodic tension-type headache
31 TNX - 201 in Phase 2 development for episodic tension - type headache Preclinical Phase 1 Phase 2 Phase 3 NDA Market Tonmya / T NX - 102 SL and TNX - 201 are Investigational New Drugs and are not approved for any indication. Preclinical Phase 1 Phase 2 Phase 3 NDA Market Candidate Indication Near
Slide 32: Episodic tension-type headache (ETTH)
32 Episodic tension - type headache (ETTH) 75 million adults in the U.S. experience frequent episodic tension - type headaches 1 - Constant band of pressure on the back/sides of head; “squeezed in a vice” feeling - “Frequent” = one to 15 headaches per month over a three - month period - Approximatel
Slide 33: Patients with ETTH seek medical attention
33 Patients with ETTH seek medical attention Non - migraine headaches lead to 9.2 million emergency room or office visits each year 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 Uti
Slide 34: ETTH is the most common type of headache
34 ETTH is the most common type of headache More than one - third of U.S. adults experience frequent ETTH 1 38% 13% 2% 0% 20% 40% 60% ETTH Migraine Chronic One - Year Prevalence Rate for U.S. Adult Population (18 - 65) Adults (18 - 65) 2 ~198 M 1 Schwartz et al., JAMA 1998;279:381 2 U.S. Census Bure
Slide 35: TNX-201 evolved from popular headache medications
35 TNX - 201 has evolved from popular headache medications with a long history of use TNX - 201 ( dexisometheptene mucate ) - S ingle optical isomer of isometheptene (IMH) A mixture of IMH optical isomers had been widely prescribed for many decades - “Racemic isometheptene ” - Single - agent medicin
Slide 36: Racemic isometheptene combination prescriptions commonly written
36 Racemic isometheptene combination (RIC ) prescriptions had been commonly written Usage of RIC Prescriptions for All Diagnoses 1 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 1 IMS Health, National Pres
Slide 37: Optical isomers of IMH have distinct pharmacological activities
37 Optical isomers of IMH have distinct pharmacological activities Previously marketed IMH drugs contained a mixture of two mirror - image isomers (racemic IMH) (R) isomer (S) isomer • Analgesic • Binds to imidazoline - 1 receptor • Inactive on adrenergic receptors • Sympathomimetic x TNX - 201 TNX
Slide 38: TNX-201 well-tolerated in Phase 1 study
38 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 35 mg (N=9) TNX - 201 70 mg (N=9) TNX - 201 140 mg (N=9) Racemi
Slide 39: Proof-of-concept Phase 2 trial of TNX-201 in ETTH
39 Proof - of - concept Phase 2 trial of TNX - 201 in ETTH Randomized, double - blind, placebo - controlled trial in episodic tension - type headache N =200; approximately 10 U.S. clinical sites Top line data expected Q4 2015 Placebo TNX - 201 N = 100 N = 100 140 mg A proof - of - concept study to e
Slide 40: TNX-201 is active on the imidazoline-1 receptor
40 TNX - 201 is active on the imidazoline - 1 receptor (I 1 - R): a novel target for the treatment of pain 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 ryanodine a
Slide 41: Intellectual property
41 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 Tonmya / TNX - 102 SL Fibromyalgia, P
Slide 42: Financial overview
42 Financial overview NASDAQ: TNXP Cash reported at March 31 , 2015 $ 58.2 million Shares outstanding (June 6, 2015) 16.1 million
Slide 43: Management team
43 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 44: Board of directors
44 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 Davidson Labrador Ventures, Alkermes ,
Slide 45: Milestones – recent and upcoming
45 Milestones – recent and upcoming Tonmya – Fibromyalgia □ May 2015 Began Phase 3 AFFIRM study □ June 2015 Presented 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
Slide 46: Developing innovative medicines for large and growing markets
46 Developing innovative medicines for large and growing markets Common and chronic disorders of the CNS Next - generation medicines with transformative treatment potential Late - stage candidates supported by human experience Capitalized to achieve key readouts in all of our clinical - stage progra
Slide 47: 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 (July 2015) on 6ix