Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (October 2014)
28 slides from Tonix Pharmaceuticals's investor presentation, Oct 10, 2014
Slide 1: Tonix Pharmaceuticals Holding Corp. October 2014
NASDAQ: TNXP © 2014 Tonix Pharmaceuticals Holding Corp. October 2014
Slide 2: Safe harbor statement
Safe harbor s tatement 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 use
Slide 3: Overview
Overview First - in - class medicines for common disorders of the central nervous system (CNS) Innovative treatment paradigms Late stage candidates Large unmet medical needs Unencumbered intellectual property portfolio Strong balance sheet, experienced team Multiple successes in drug approvals and v
Slide 4: Development programs
Development programs Preclinical Phase 1 NDA Market Candidate Indication TNX - 102 SL (cyclobenzaprine HCl sublingual tablet) 2.8 mg is an Investigational New Drug and is not approved for any indication. TNX - 201 (isometheptene mucate single isomer) is not approved for any indication. Phase 2 Phase
Slide 5: Targeting sleep quality in fibromyalgia and PTSD
Targeting sleep quality in fibromyalgia (FM) and post - traumatic stress disorder (PTSD) Non - restorative sleep linked to pain, fatigue, hyper - vigilance, and arousals Restorative sleep improves symptoms of fibromyalgia and PTSD TNX - 102 SL is designed as a chronic therapy for bedtime use Novel m
Slide 6: Fibromyalgia market opportunity
CONFIDENTIAL 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
Slide 7: Fibromyalgia treatments dissatisfy many patients
Fibromyalgia treatments dissatisfy many patients Chronic , widespread pain with sleep, fatigue, mood, and memory problems Poor quality of life Typical patient has onset at 30 - 40 years of age with persistence for rest of life Patients remain unsatisfied despite approved products Patients typically
Slide 8: Fibromyalgia has a highly negative economic impact
CONFIDENTIAL Fibromyalgia has a highly negative economic impact Source: Robinson et al, Pain Med 2012;13:1366 - 76. Resource utilization over preceding 12 months Outpatient visits 82.9 % Any emergency room visit 40.2 % Mean number of emergency room visits † 2.4 Productivity measures over preceding 1
Slide 9: Restorative sleep improves pain and other fibromyalgia symptoms
CONFIDENTIAL Restorative sleep improves pain and other fibromyalgia symptoms >90% of fibromyalgia patients complain of poor sleep quality* Clinical validation from proof - of - concept study Phase 2a trial showed pain and sleep benefit with very low dose oral cyclobenzaprine (CBP)** TNX - 102 SL: su
Slide 10: BESTFIT Phase 2b trial in fibromyalgia complete
CONFIDENTIAL BESTFIT Phase 2b trial in fibromyalgia complete 10 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 rando
Slide 11: Improved pain in fibromyalgia in the BESTFIT study
CONFIDENTIAL TNX - 102 SL improved pain in fibromyalgia in the BESTFIT study 11 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* 0.030 LR Clinic NRS 7 -
Slide 12: Improved sleep quality in fibromyalgia in the BESTFIT study
CONFIDENTIAL TNX - 102 SL improved sleep quality in fibromyalgia in the BESTFIT study 12 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* 0.003 0.004 MMRM JTC - MI FIQ - R Sleep
Slide 13: Broadly improved fibromyalgia symptoms in the BESTFIT study
CONFIDENTIAL TNX - 102 SL broadly improved fibromyalgia symptoms in the BESTFIT study 13 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 0.015 MMRM JTC - MI FIQ - R Sympto
Slide 14: Was well-tolerated in the BESTFIT study
CONFIDENTIAL TNX - 102 SL was well - tolerated in the BESTFIT study 14 Source: Phase 2b BESTFIT preliminary top line results No serious adverse events (SAE) reported Most frequent local adverse events were administration site reactions Previously reported in TNX - 102 SL Phase 1 studies; no detectab
Slide 15: Registration program in fibromyalgia
CONFIDENTIAL Acceptable to submit 505(b)( 2) New Drug Application (NDA) Two adequate and well - controlled efficacy and safety trials in fibromyalgia patients Requesting FDA meeting to review BESTFIT results and discuss efficacy requirements for NDA One - year exposure data to support chronic use la
Slide 16: Post-traumatic stress disorder is a chronic, debilitating condition
CONFIDENTIAL 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
Slide 17: PTSD patients complain of poor sleep quality as a core symptom
CONFIDENTIAL 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” Poor sleep quality after trauma is linked to onset of PTSD Correlated with depression, substance abuse and suicide T
Slide 18: PTSD program with Phase 2 efficacy study to begin in 4Q 2014
CONFIDENTIAL PTSD program with TNX - 102 SL 18 P hase 2 efficacy study of TNX - 102 SL to begin in 4Q 2014 Leverage fibromyalgia formulation, clinical experience, manufacturing know - how Overlap between PTSD and fibromyalgia ~50% of fibromyalgia or PTSD patients meet criteria for the other disorder
Slide 19: AtEase Phase 2 trial in PTSD
CONFIDENTIAL “AtEase” Phase 2 trial of TNX - 102 SL in PTSD Randomized , double - blind, placebo - controlled trial N=220; approximately 25 U.S. clinical sites Primary efficacy endpoint: D ifference in Clinician - Administered PTSD Scale (CAPS) score between TNX - 102 SL 2.8 mg and placebo TNX - 102
Slide 20: TNX-201 for episodic tension-type headache
CONFIDENTIAL TNX - 201 for 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. 70 million adul
Slide 21: TNX-201 to enter clinical development in 2015
CONFIDENTIAL TNX - 201 to enter clinical development in 2015 Novel molecular mechanism Based on proprietary discoveries by Tonix Non - barbiturate, non - opioid Mechanism of action distinct from acetaminophen and barbiturates Comparative pharmacokinetic and safety study to be conducted in 1Q 2015 Pr
Slide 22: Intellectual property
CONFIDENTIAL 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 - 2
Slide 23: Milestones – recent and upcoming
CONFIDENTIAL Milestones – recent and upcoming Corporate □ July 2014 – $7.2 million in net proceeds from common stock offering TNX - 102 SL – Fibromyalgia □ September 2014 – Reported top line results from Phase 2b BESTFIT study □ Meet with FDA to determine remaining requirements for NDA □ Begin Phase
Slide 24: Management team
CONFIDENTIAL Bruce Daugherty, PhD Chief Scientific Officer Seth Lederman, MD Chief Executive Officer Management team Leland Gershell, MD, PhD Chief Financial Officer Don Kellerman, PharmD SVP, Clinical Development & Regulatory Affairs 24
Slide 25: Board of directors
CONFIDENTIAL John Rhodes NYSERDA, NRDC Booz Allen Hamilton Donald Landry, MD, PhD Chair, Department of Medicine Columbia University Stuart Davidson Alkermes Combion Seth Lederman, MD (Chair) Targent Pharmaceuticals Vela Pharmaceuticals Samuel Saks, MD ALZA Jazz Pharmaceuticals Patrick Grace WR Grace
Slide 26: Financial summary
CONFIDENTIAL Financial summary NASDAQ: TNXP Cash reported at June 30 , 2014* $ 43.9 million Net cash used in operations in 2Q14 $ 5.7 million Shares outstanding † 10.8 million * Does not include $7.2 million in net proceeds from stock offering in July 2014. † As of October 8, 2014 26
Slide 27: Opportunity summary
CONFIDENTIAL Opportunity summary TNX - 102 SL: late - stage clinical program for large market opportunity Strong clinical evidence of broad activity in lead indication Well - known safety profile with excellent tolerability in patients studied Current fibromyalgia treatment options leave many patien
Slide 28: © 2014 Tonix Pharmaceuticals Holding Corp. NASDAQ: TNXP
© 2014 Tonix Pharmaceuticals Holding Corp. NASDAQ: TNXP
Tonix Pharmaceuticals Holding — Investor Presentation (October 2014) on 6ix