Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (September 2015)
34 slides from Tonix Pharmaceuticals's investor presentation, Sep 10, 2015
Slide 1: Investor Presentation September 2015
NASDAQ: TNXP Investor Presentation September 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) Late - stage candidates supported by human experience Capitalized to achieve key readouts in all of our clinical - stage programs 2016 to feature Phase 3 and Phase 2 clini
Slide 4: Pipeline led by Tonmya for fibromyalgia
4 Candidate Indication Near - term Catalyst Tonmya Fibromyalgia Top line data 2H 2016 TNX - 102 SL Post - Traumatic Stress Disorder Top line data 1H 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 NDA
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 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, Arthritis Rheum 2015; 67 :568. 2 Robinson RL et al, Pain Med 2012;13:1366. 3 Independent study conducted by IMS Consulting Group,
Slide 7: Fewer than half of those treated receive sustained benefit from FDA-approved drugs
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 fail therapy due to lack of a response or p
Slide 8: Pervasive treatment dissatisfaction creates an opportunity for a differentiated option
8 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 medi
Slide 9: Tonmya in Phase 3 clinical development for fibromyalgia
9 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 - Designed for chronic bedtime administration, no titration Tonmya demonstrated broad
Slide 10: CBP is detected in plasma within minutes following sublingual administration
10 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 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 were ra
Slide 12: BESTFIT: Tonmya broadly improved fibromyalgia
12 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 13: Tonmya was well tolerated in the BESTFIT study
13 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 14: Phase 3 AFFIRM Study is underway
14 Phase 3 AFFIRM Study is underway Second Phase 3 clinical trial expected to begin in 2Q 2016 - E xpected 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; approximately 35
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 1H 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 (cyclobenzaprine HCl sublingual table
Slide 16: PTSD: An important and growing public health problem
16 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 - 20% of women and 8 % of men who experience significant trauma develop PTSD 1 Associated
Slide 17: PTSD is a large problem for both civilians and the military
17 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 18: Significant gap in current therapeutic landscape for PTSD
18 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 efficacy evidence in those with a
Slide 19: TNX-102 SL's potential as a treatment for PTSD is supported by clinical evidence
19 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 - Shares pharmacology with drugs used off - label to treat sleep dysfunction in PTSD Efficacy of “tricycli
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 (cyclobenzaprine HCl sublingual tablet, 2.8 mg) an
Slide 22: Episodic tension-type headache (ETTH) – the most common form of headache
22 Episodic tension - type headache ( ETTH) – the most common form of headache 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 attention, mainly
Slide 23: Current treatment market for non-migraine headaches (ETTH)
23 Current treatment market for non - migraine headaches (ETTH) “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 ref
Slide 24: Isometheptene was used for ETTH, but no longer approved
24 Isometheptene was used for ETTH, but no longer approved Isometheptene – a brief history - Isometheptene mucate was commonly used in the past - Indicated for tension - type headache, but also used for migraine 1 - Single - agent medicine (pre - 1962) - Component of prescription combination drug pr
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 mechanism of action (imidazoline - 1 receptor agonist) - Differentiated pharmacology as a single isomer of isometheptene - May offer effective, safe, and non - addictive treatment option for ETTH May serve patients who: - Do not adeq
Slide 27: Phase 2 proof-of-concept trial of TNX-201 in ETTH is ongoing
27 Phase 2 proof - of - concept trial of TNX - 201 in ETTH is ongoing Randomized, double - blind, placebo - controlled trial in episodic tension - type headache N=200 enrollment goal at approximately 10 U.S . clinical sites Top line data expected 1Q 2016 Placebo TNX - 201 N = 100 N = 100 140 mg A pr
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 Tonmya / TNX - 102 SL Fibromyalgia, PTSD
Slide 29: Financial overview
29 Financial overview NASDAQ: TNXP Cash reported at June 30, 2015 $ 48.7 million Net proceeds from July 2015 financing 18.7 million Cash used in operations in 1H 2015 $ 18.3 million Shares outstanding (September 9, 2015) 18.8 million
Slide 30: Management team
30 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 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 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 33: Developing innovative medicines for large and growing markets
33 Developing innovative medicines for large and growing markets Common and chronic disorders of the central nervous system (CNS) Late - stage candidates supported by human experience Capitalized to achieve key readouts in all of our clinical - stage programs 2016 to feature Phase 3 and Phase 2 clin
Slide 34: Contact: Madison Avenue, New York
NASDAQ: TNXP 509 Madison Avenue New York, NY 10022 (212) 980 - 9155 www.tonixpharma.com
Tonix Pharmaceuticals Holding — Investor Presentation (September 2015) on 6ix