Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (November 2015)
34 slides from Tonix Pharmaceuticals's investor presentation, Nov 19, 2015
Slide 1: Investor Presentation November 2015
NASDAQ: TNXP Investor Presentation November 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 Targeting common pain conditions and a serious psychiatric disorder - Non - opiate, non - barbiturate, non - benzodiazepine medicines - Two clinical - stage proprietary medicines targeting three unique indications - Highly differentiate
Slide 4: Pipeline led by Tonmya for fibromyalgia
4 Candidate Indication Near - term Catalyst 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 Tonmya™ for fibromyalgia Preclinical Phase 1 Phase 2 Phase 3 ND
Slide 5: Fibromyalgia is a chronic, debilitating disorder with significant burden
5 Fibromyalgia is a chronic, debilitating disorder that imposes a significant social and economic burden Fibromyalgia is a neurobiological disorder characterized by : Believed to result from amplified sensory and pain signaling in central nervous system 1 Causes significant impairment in all areas o
Slide 6: Fibromyalgia is a prevalent disorder but remains under diagnosed
6 Fibromyalgia is a prevalent disorder 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, Arthritis Rheum 2015; 67 :568. 2 Robinson RL et al, Pain Med 2012;13:1366. 3 Product sales derived from IMS MIDAS; IMS NDTI use
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 with better tolerability
8 Large need for new fibromyalgia therapies that provide broad symptom relief with better tolerability Currently - approved medications have side effects that limit long - term use 1 - Many patients skip doses or discontinue altogether within months of treatment initiation Medication - related side
Slide 9: Dissatisfaction with current therapies results in inappropriate drug usage
9 Dissatisfaction with current fibromyalgia therapies results in inappropriate drug usage out of desperation 1 Halpern et al, Pain Practice 2015 (10.1111/ papr . 12364). 2 Vincent et al, BMJ Open 2015;5:e006681. 3 Peng et al, Clin J Pain 2015;31:7013. 4 Painter et al, Am J Pharm Benefits 2014;6:e177
Slide 10: Tonix is developing Tonmya for fibromyalgia
10 Tonix is developing Tonmya for fibromyalgia Advanced sublingual tablet containing cyclobenzaprine (CBP) 2.8 mg - Eutectic formulation rapidly delivers a low dose of CBP for transmucosal absorption - A voids first - pass metabolism and r educes long - lived metabolite relative to oral administrati
Slide 11: Phase 2b BESTFIT study of Tonmya in fibromyalgia
11 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 randomi
Slide 12: Tonmya provided broad clinical benefit in BESTFIT
12 Tonmya provided broad clinical benefit in BESTFIT 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 anal
Slide 13: Tonmya was well tolerated in BESTFIT and open-label extension
13 Tonmya was well tolerated in the BESTFIT study and in a 12 - month open - label extension study Most frequent local adverse events were administration site reactions - Previously reported in Phase 1 studies; no detectable bias on efficacy results - Transient tongue numbness ( 44% Tonmya vs . 2% p
Slide 14: Phase 3 AFFIRM Study is underway
14 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 Tonmya in Phase 3 clinical development for fibromyalgia Randomized, double - blind, placebo - controlled study in fibromyalgia N=500; approximatel
Slide 15: TNX-102 SL in Phase 2 development for PTSD
15 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 / T NX - 102 SL and TNX - 201 are Investigational New
Slide 16: PTSD is a chronic stress disorder triggered by a traumatic event
16 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 17: PTSD is a prevalent problem for civilians and the military
17 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 18: Significant gap in current therapeutic landscape for PTSD
18 Significant gap in current therapeutic landscape for PTSD M edicines approved for PTSD often provide inadequate and/or inconsistent benefit - Limited to two SSRIs, both of which carry suicidality warnings - US Institute of Medicine (IOM) concluded that evidence of treatment effect is low 1 - Lack
Slide 19: TNX-102 SL's potential for PTSD supported by clinical evidence and pharmacology
19 TNX - 102 SL’s potential as a treatment for PTSD is supported by clinical evidence and pharmacology TNX - 102 SL* targets mechanisms associated with treating disturbed sleep in PTSD - Active on receptor sites believed to have treatment potential for sleep problems in PTSD (including 5 - HT2a, alp
Slide 20: Phase 2 AtEase trial of TNX-102 SL in PTSD is ongoing
20 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 21: TNX-201 in Phase 2 development for episodic tension-type headache
21 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 Tonmya / T NX - 102 SL and TNX - 201 are Investigational New Drugs and ar
Slide 22: Episodic tension-type headache – the most common form of headache
22 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 23: Many patients do not receive adequate relief from OTC treatments
23 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 24: Prescription options for acute therapy are limited and potentially addictive
24 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 25: Isometheptene previously used for ETTH without an approved NDA
25 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 26: Scientific rationale for development of TNX-201
26 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 27: Current value proposition for TNX-201
27 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 28: Phase 2 proof-of-concept trial of TNX-201 in ETTH is ongoing
28 Phase 2 proof - of - concept trial of TNX - 201 in ETTH is ongoing 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 = 100 N =
Slide 29: Intellectual property
29 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 Tonmya / TNX - 102 SL Fibromyalgia, PTSD
Slide 30: Financial overview
30 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 (November 18, 2015) 18.8 million
Slide 31: Management team
31 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 32: Board of directors
32 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 33: Milestones – recent and upcoming
33 Milestones – recent and upcoming Tonmya – Fibromyalgia □ May 2015 Began Phase 3 AFFIRM study □ November 2015 Presented additional data from Phase 2b BESTFIT study at ACR □ 3Q 2016 Report top - line results from AFFIRM study TNX - 102 SL – Post - Traumatic Stress Disorder □ January 2015 Began Phas
Slide 34: 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 (November 2015) on 6ix