Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (March 2015)
33 slides from Tonix Pharmaceuticals's investor presentation, Mar 23, 2015
Slide 1: March 2015
NASDAQ: TNXP March 2015 © 2015 Tonix Pharmaceuticals Holding Corp.
Slide 2: Safe harbor statement
Safe harbor statement 2 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 may be identified by the us
Slide 3: New approaches to treating CNS disorders
New approaches to treating CNS disorders First - in - class medicines for common disorders of the central nervous system (CNS) Innovative treatment paradigms Late stage candidates Large unmet medical needs Entered 2015 with three clinical development programs 3 Fibromyalgia Post - Traumatic Stress D
Slide 4: Pipeline led by TNX-102 SL for fibromyalgia
Pipeline led by TNX - 102 SL for fibromyalgia 4 Preclinical Phase 1 NDA Market Candidate Indication Phase 2 Phase 3 TNX - 102 SL Began Phase 2 trial - Jan ‘15 TNX - 102 SL Fibromyalgia Start Phase 3 trial - 2Q15 Start Phase 2 trial - 2Q15 TNX - 201 TNX - 102 SL (cyclobenzaprine HCl sublingual tablet
Slide 5: Fibromyalgia market opportunity
Fibromyalgia market opportunity * Lawrence et al, Arthritis Rheum 2008;58:26 - 35; Vincent et al, Arthritis Care Res 2013;65:786 - 792. ** Estimates based on information from publicly - available sources SNRI = Serotonin Norepinephrine Reuptake Inhibitor Three FDA approved prescription medications:
Slide 6: AFFIRM Phase 3 trial of TNX-102 SL in Fibromyalgia
“AFFIRM” Phase 3 trial of TNX - 102 SL in Fibromyalgia Randomized, double - blind, placebo - controlled trial in f ibromyalgia N=500; 30 - 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 – primary efficacy en
Slide 7: Restorative sleep improves pain and other fibromyalgia symptoms
Restorative sleep improves pain and other fibromyalgia symptoms >90% of fibromyalgia patients complain of poor sleep quality* Sleep quality improvement is not a feature of approved medications Phase 2a study with low - dose cyclobenzaprine (CBP) capsule showed proof - of - concept** TNX - 102 SL is
Slide 8: BESTFIT Phase 2b trial in fibromyalgia
BESTFIT Phase 2b trial in fibromyalgia 8 BESTFIT = BEdtime Sublingual TNX - 102 SL as Fibromyalgia Intervention Therapy Randomized, double - blind, placebo - controlled trial 2010 American College of Rheumatology diagnostic criteria for fibromyalgia 205 participants were randomized 1:1 at 17 U.S. si
Slide 9: TNX-102 SL improved pain in fibromyalgia in the BESTFIT study
TNX - 102 SL improved pain in fibromyalgia in the BESTFIT study 9 Outcome Measure at Week 12 Intent - to - Treat Population † p value Method Daily Pain Diary, NRS Mean Change** 0.086 0.172 MMRM JTC - MI Daily Pain Diary, NRS Proportion Achieving 30% Improvement* 0.033 LR Clinic NRS 7 - day pain reca
Slide 10: TNX-102 SL improved sleep quality in fibromyalgia in the BESTFIT study
TNX - 102 SL improved sleep quality in fibromyalgia in the BESTFIT study 10 Outcome Measure at Week 12 Intent - to - Treat Population p value Method Daily Sleep Quality Diary, NRS Mean Change* <0.001 MMRM PROMIS Sleep Disturbance T - score Change* 0.005 MMRM FIQ - R Sleep Quality Item Mean Change <0
Slide 11: TNX-102 SL broadly improved fibromyalgia symptoms in the BESTFIT study
TNX - 102 SL broadly improved fibromyalgia symptoms in the BESTFIT study 11 Outcome Measure at Week 12 Intent - to - Treat Population p value Method Patient Global Impression of Change Responder Analysis* 0.025 LR FIQ - R Total Score Mean Change* 0.014 MMRM FIQ - R Symptom Domain Mean Change 0.004 M
Slide 12: TNX-102 SL was well-tolerated in the BESTFIT Study
TNX - 102 SL was well - tolerated in the BESTFIT Study 12 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 tongue numb
Slide 13: Phase 2b BESTFIT study confirmed activity and tolerability
Phase 2b BESTFIT study confirmed activity and tolerability Statistically - significant improvements across key fibromyalgia symptoms were observed Systemic tolerability similar to placebo 2.8 mg daily dose confirmed for future development Phase 3 AFFIRM study to commence in 2Q 2015 Randomized, doubl
Slide 14: Phase 2 trial of TNX-102 SL for PTSD is recruiting
Phase 2 trial of TNX - 102 SL for PTSD is recruiting 14 Preclinical Phase 1 NDA Market Candidate Indication Phase 2 Phase 3 Post - Traumatic Stress Disorder TNX - 102 SL Began Phase 2 trial - Jan ‘15 TNX - 102 SL Start Phase 3 trial - 2Q15 Start Phase 2 trial 2Q15 TNX - 201 TNX - 102 SL (cyclobenzap
Slide 15: Post-traumatic stress disorder is a chronic, debilitating condition
Post - traumatic stress disorder is a chronic, debilitating condition High incidence among soldiers and veterans, but experiencing any trauma can lead to PTSD Associated with suicide and unpredictable, violent behaviors Patients desperate despite two FDA approved drugs; no new treatment in >10 years
Slide 16: Poor sleep quality after trauma is linked to onset of PTSD
Poor sleep quality after trauma is linked to onset of PTSD PTSD patients complain of poor sleep quality as a core symptom Distressing dreams (nightmares) are part of “re - experiencing” Restless sleep is part of “hyper - arousal” Correlated with depression, substance abuse and suicide Military - rel
Slide 17: AtEase Phase 2 trial of TNX-102 SL in PTSD
“AtEase” Phase 2 trial of TNX - 102 SL in PTSD 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 2.8 mg and place
Slide 18: PTSD program with TNX-102 SL
PTSD program with TNX - 102 SL 18 Fibromyalgia program informs development of TNX - 102 SL in PTSD Safety data from fibromyalgia studies are potentially supportive for PTSD program Efficacy data support potential for activity in PTSD Improvements in several outcomes analyses of BESTFIT that relate t
Slide 19: TNX-201 in development for episodic tension-type headache
TNX - 201 in development for episodic tension - type headache 19 Preclinical Phase 1 NDA Market Candidate Indication Phase 2 Phase 3 TNX - 102 SL Began Phase 2 trial – Jan ‘15 TNX - 102 SL Start Phase 3 trial - 2Q15 Start Phase 2 trial - 2Q15 TNX - 201 Episodic Tension - Type Headache TNX - 102 SL (
Slide 20: Episodic tension-type headache (ETTH)
Episodic tension - type headache (ETTH) * Schwartz et al., JAMA 1998;279:381 - 383; Chowdhury, Ann Ind Acad Neurol 2012;15:83 - 88; company analysis of public literature. ** Scher et al., Cephalalgia 2010;30:321 - 328; company analysis of public literature. 75 million adults in the U.S. experience f
Slide 21: TNX-201 in clinical development for ETTH
TNX - 201 in clinical development for ETTH TNX - 201 is (R) - isometheptene mucate Tonix is developing TNX - 201 for ETTH Phase 2 study to begin in 2Q 2015 Racemic isometheptene mucate is a mixture of (R) and (S) isomers Had been widely prescribed for many decades in the U.S. as: a single - agent me
Slide 22: Phase 2 trial of TNX-201 in ETTH to begin in 2Q15
Phase 2 trial of TNX - 201 in ETTH to begin in 2Q15 22 TNX - 201 ((R) - isometheptene mucate ) 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 site
Slide 23: Isometheptene isomers have distinct pharmacological activities
Isometheptene (IMH) isomers have distinct pharmacological activities 23 (R) isomer (S) isomer • Analgesic • Binds to imidazoline - 1 receptor • Inactive on adrenergic receptors • Sympathomimetic The (R) and (S) isomers of IMH have different pharmacologies Previously marketed IMH drugs were a mixture
Slide 24: Phase 1 study of TNX-201 completed
Phase 1 study of TNX - 201 completed 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.) 24 TNX - 201 ((R) - isometheptene mucate ) is an Investigational New Drug and is not approv
Slide 25: The imidazoline-1 receptor is a novel target for the treatment of pain
The imidazoline - 1 receptor (I 1 - R) is a novel target for the treatment of pain 25 Hot Plate Test Characteristics 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 and cytokine rece
Slide 26: Racemic isometheptene combination prescriptions had been commonly written
Racemic isometheptene combination (RIC) prescriptions had been commonly written 26 Usage of RIC Prescriptions for All Diagnoses Number of RIC prescriptions peaked at 2.5 million 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
Slide 27: ETTH is the most common type of headache
ETTH is the most common type of headache 27 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)* * Estimated from 2013 U.S. Census Schwartz et al., JAMA 1998;279:381 - 383; Stovner et al, Cephalalgia 2007;2
Slide 28: Patients with ETTH seek medical attention
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 28 Heath Care Utilization Project data, 2011; IMS National Disease and Therapeutic Index™ 2013 Non - migrai
Slide 29: Intellectual property
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 Headache C
Slide 30: Financial summary
Financial summary NASDAQ: TNXP Cash reported at December 31 , 2014 $ 38.2 million Net proceeds from common stock offering in 1Q15 $ 29.0 million Shares outstanding (Feb 27, 2015) 16.1 million 30
Slide 31: Management team
Bruce Daugherty, PhD Chief Scientific Officer Seth Lederman, MD Chief Executive Officer Management team Leland Gershell, MD, PhD Chief Financial Officer 31
Slide 32: Milestones – recent and upcoming
TNX - 102 SL – Fibromyalgia □ September 2014 – Reported top line results from Phase 2b BESTFIT study □ January 2015 – Reported FDA acceptance of 30% responder analysis as Phase 3 primary endpoint □ 2Q 2015 – Begin Phase 3 AFFIRM study □ 2H 2016 – R eport top - line results from AFFIRM study TNX - 10
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 (March 2015) on 6ix