Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (January 2016)
33 slides from Tonix Pharmaceuticals's investor presentation, Jan 8, 2016
Slide 1: Investor Presentation January 2016
NASDAQ: TNXP Investor Presentation January 2016 © 2016 Tonix Pharmaceuticals Holding Corp. Version: P0003 - 01 - 08 - 16
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 Targeting common pain conditions and a serious psychiatric disorder - Two clinical - stage proprietary candidates targeting three indications - Differentiated products with potential for sustainable competitive advantages 2016 to reveal
Slide 4: Pipeline led by TNX-102 SL for fibromyalgia
4 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 TNX - 201 Episodic Tension - Type Headache Top line data 1Q 2016 Pipeline led by TNX - 102 SL for fibromyalgia Preclinical Phase
Slide 5: Fibromyalgia is a chronic, debilitating disorder with significant burden
5 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 nervous system 1 Causes significant impairment i
Slide 6: Fibromyalgia is a prevalent disorder but remains underdiagnosed
6 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. 2 Robinson RL et al, Pain Med 2012;13:1366. 3 Product sales derived from IMS MIDAS; IMS NDTI used
Slide 7: Fewer than half of those treated receive sustained benefit from approved drugs
7 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 fail therapy due to lack of a response or poor t
Slide 8: Large need for new fibromyalgia therapies
8 Large need for new fibromyalgia therapies that provide broad symptom relief 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 of treatment initiation Medication - related s
Slide 9: Tonix is developing TNX-102 SL for fibromyalgia
9 Tonix is developing TNX - 102 SL for fibromyalgia Advanced sublingual tablet containing low - dose cyclobenzaprine (CBP ) - Suitable for administration at bedtime due to efficient transmucosal absorption - Avoidance of first - pass metabolism reduces formation of long - lived metabolite - Designed
Slide 10: Phase 2b BESTFIT study of TNX-102 SL in fibromyalgia
10 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 criteria for fibromyalgia - 205 participants r
Slide 11: BESTFIT results on key clinical endpoints
11 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 0.014 1 Intent - to - treat analysis, N=
Slide 12: TNX-102 SL safety and tolerability profile in BESTFIT
12 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 administration site reactions - Previously reported
Slide 13: Phase 3 AFFIRM study is underway
13 Phase 3 AFFIRM Study is underway Second Phase 3 Study (“REAFFIRM”) expected to begin in 2Q 2016 - E xpected to be similar to AFFIRM in design and size TNX - 102 SL in Phase 3 clinical development for fibromyalgia Randomized, double - blind, placebo - controlled study in fibromyalgia N=500; approx
Slide 14: TNX-102 SL in Phase 2 development for PTSD
14 Candidate Indication Near - term Catalyst TNX - 102 SL Post - Traumatic Stress Disorder Top line data 2Q 2016 TNX - 102 SL in Phase 2 development for post - traumatic stress disorder (PTSD) Preclinical Phase 1 Phase 2 Phase 3 NDA Market * Tonmya has been conditionally accepted by the FDA as the p
Slide 15: PTSD is a chronic stress disorder triggered by a traumatic event
15 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 Associated with significant life disruption - Social
Slide 16: PTSD is a prevalent problem for civilians and the military
16 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 (psycho/pharmacotherapy) 2 1 Kessler RC at al, Arch Gen Psychiatry 2013;62:617; U.S. Census Bureau, 2013 Projection. 2 W
Slide 17: Significant gap in current therapeutic landscape for PTSD
17 Significant gap in current therapeutic landscape for PTSD M edicines 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) concluded that evidence of treatment effec
Slide 18: TNX-102 SL's potential in PTSD supported by clinical evidence and pharmacology
18 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 adrenergic, and histamine - 1 receptors Eff
Slide 19: Phase 2 AtEase trial of TNX-102 SL in PTSD is fully enrolled
19 Phase 2 “ AtEase ” trial of TNX - 102 SL in PTSD is fully enrolled Randomized, double - blind, placebo - controlled trial in military - related PTSD N=240+; approximately 25 U.S. clinical sites Primary efficacy endpoint: - Difference in Clinician - Administered PTSD Scale (CAPS) score between TNX
Slide 20: TNX-201 in Phase 2 development for episodic tension-type headache
20 Candidate Indication Near - term Catalyst TNX - 201 Episodic Tension - Type Headache Top line data 1Q 2016 TNX - 201 in Phase 2 development for episodic tension - type headache Preclinical Phase 1 Phase 2 Phase 3 NDA Market NDA = New Drug Application; FDA = U.S. Food and Drug Administration. T NX
Slide 21: Episodic tension-type headache, the most common form of headache
21 Episodic tension - type headache ( ETTH) – the most common form of headache More than 75 million adults in the U.S. experience ETTH each year 1 - Constant band of pressure on the back/sides of head; “squeezed in a vice” feeling - Most are infrequent (<1 per month) ; rarely require medical attenti
Slide 22: Many patients do not receive adequate relief from OTC treatments
22 Many patients do not receive adequate relief from OTC treatments “First - line” treatment - NSAIDs are most common - Many patients self - medicate with a course of over - the - counter NSAIDs before seeing a physician 1 - Less than 50% of treated patients are pain - free at two hours 2 - Some are
Slide 23: Prescription options for acute therapy are limited and potentially addictive
23 Prescription options for acute therapy are limited and potentially addictive Butalbital combinations are the only FDA - approved prescription products for ETTH - ~3.5 million prescriptions for these products are written per year for non - migraine headaches 1 - Butalbital is a barbiturate – and f
Slide 24: Isometheptene previously used for ETTH without an approved NDA
24 Isometheptene previously used for ETTH without an approved NDA Isometheptene (IMH) had been marketed for tension - type headache - Single agent product for migraine was voluntarily withdrawn by Knoll Pharma in 1981 - IMH - containing combination products remained on the market without approved ND
Slide 25: Scientific rationale for development of TNX-201
25 Scientific rationale for development of TNX - 201 TNX - 201 ( dexisometheptene mucate ) contains the (R) isomer of isometheptene - Pharmacologic profile is distinct from (S) isomer per preclinical studies - Represents a new class of analgesics (selective imidazoline - 1 receptor agonists) (R) iso
Slide 26: Current value proposition for TNX-201
26 Current value proposition for TNX - 201 Key attributes - Unique analgesic mechanism of action (imidazoline - 1 receptor agonist) - Differentiated pharmacology as a single isomer of isometheptene - May offer a non - addictive treatment option for ETTH May serve patients who: - Do not adequately re
Slide 27: Phase 2 proof-of-concept trial of TNX-201 in ETTH is fully enrolled
27 Phase 2 proof - of - concept trial of TNX - 201 in ETTH is fully enrolled Randomized, double - blind, placebo - controlled trial in episodic tension - type headache Goal is to assess treatment of approx. 150 headaches ~ 10 U.S. clinical sites Top line data expected 1Q 2016 Placebo TNX - 201 N = 1
Slide 28: Intellectual property
28 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 filed TNX - 102 SL Fibromyalgia, PTSD Composit
Slide 29: Financial overview
29 Financial overview NASDAQ: TNXP Cash, cash equivalents, and marketable securities reported at September 30, 2015 $ 55.0 million Cash used in operations in 2015 through September 30 $ 30.6 million Shares outstanding (January 8, 2016) 18.8 million
Slide 30: Management team
30 Management team Seth Lederman, MD President & CEO Jessica Edgar Morris Acting CFO, Chief Administrative Officer Bruce Daugherty, PhD, MBA Chief Scientific Officer Ronald Notvest, PhD SVP, Commercial Planning & Development Gregory Sullivan, MD Chief Medical Officer
Slide 31: Board of directors
31 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 32: Milestones – recent and upcoming
32 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 Annual Meeting □ 3Q 2016 Report top - line results from AFFIRM study TNX - 102 SL – Post - Traumatic Stress Disorder □ De
Slide 33: 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 (January 2016) on 6ix