Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (October 2014)
28 slides from Tonix Pharmaceuticals's investor presentation, Oct 28, 2014
Slide 1: October 2014
October 2014 © 2014 Tonix Pharmaceuticals Holding Corp. NASDAQ: TNXP
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: 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 To enter 2015 with three clinical development programs 3 Fibromyalgia Post - Traumatic Stress
Slide 4: Pipeline led by TNX-102 SL for fibromyalgia
Pipeline led by TNX - 102 SL for fibromyalgia Preclinical Phase 1 NDA Market Candidate Indication Phase 2 Phase 3 TNX - 102 SL 2019E TNX - 102 SL Fibromyalgia 2018E TNX - 201 2019E 4 TNX - 102 SL (cyclobenzaprine HCl sublingual tablet) 2.8 mg and TNX - 201 (isometheptene mucate single isomer) are In
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: 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 therapeutic options Proof - of - concept study Phase 2a results with very low dose oral cyclobenzaprine (CBP) published
Slide 7: BESTFIT Phase 2b trial in fibromyalgia complete
BESTFIT Phase 2b trial in fibromyalgia complete 7 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 1
Slide 8: TNX-102 SL improved pain in fibromyalgia in the BESTFIT study
TNX - 102 SL improved pain in fibromyalgia in the BESTFIT study 8 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 - day pain reca
Slide 9: TNX-102 SL improved sleep quality in fibromyalgia in the BESTFIT study
TNX - 102 SL improved sleep quality in fibromyalgia in the BESTFIT study 9 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 Quality Item
Slide 10: TNX-102 SL broadly improved fibromyalgia symptoms in the BESTFIT study
TNX - 102 SL broadly improved fibromyalgia symptoms in the BESTFIT study 10 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 Symptom Domain Mean
Slide 11: TNX-102 SL was well-tolerated in the BESTFIT study
TNX - 102 SL was well - tolerated in the BESTFIT study 11 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 detectable bias on efficacy results Transient tongue numbness (41.7% TNX -
Slide 12: End-of-Phase meeting held with FDA in February 2013
End - of - Phase 2 clinical/non - clinical meeting held with FDA in February 2013 Acceptable to submit 505(b)(2) New Drug Application ( NDA) Required clinical studies to support NDA: • Two adequate and well - controlled efficacy and safety trials in fibromyalgia patients Primary endpoint based on ch
Slide 13: TNX-102 SL to enter Phase 2 for PTSD this quarter
TNX - 102 SL to enter Phase 2 for PTSD this quarter Preclinical Phase 1 NDA Market Candidate Indication Phase 2 Phase 3 Post - Traumatic Stress Disorder TNX - 102 SL 2019E TNX - 102 SL 2018E TNX - 201 2019E 13 2018E 2017E 2018E Begin 1H15 Begin 4Q14 4Q14 Begin 1H15 TNX - 102 SL (cyclobenzaprine HCl
Slide 14: 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 15: Rationale for developing TNX-102 SL for PTSD
Rationale for developing TNX - 102 SL for PTSD 15 Overlap between PTSD and fibromyalgia ~50 % of fibromyalgia or PTSD patients meet criteria for the other disorder Patients experience disturbed sleep Widespread pain is considered “co - morbid” with PTSD Opioid, benzodiazepine, other sedative - hypno
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: TNX-102 SL acts on neurotransmitter systems intrinsic to sleep physiology
17 TNX - 102 SL acts on neurotransmitter systems intrinsic to sleep physiology Serotonin and Norepinephrine Antagonist and Reuptake Inhibitor (SNARI) Blocks serotonin and norepinephrine reuptake Selectively b locks serotonin 2A and α - 1 adrenergic receptors TNX - 102 SL TNX - 102 SL (cyclobenzaprin
Slide 18: Mechanistic relationship of TNX-102 SL with trazodone and prazosin
18 Mechanistic relationship of TNX - 102 SL with trazodone and prazosin Trazodone blocks serotonin reuptake and 2A receptors Prazosin blocks α - 1 adrenergic receptors TNX - 102 SL TNX - 102 SL (cyclobenzaprine HCl sublingual tablet) 2.8 mg is an Investigational New Drug and is not approved for any
Slide 19: PTSD program with TNX-102 SL
PTSD program with TNX - 102 SL 19 P hase 2 study of TNX - 102 SL in military - related PTSD (“ AtEase ”) to begin in 4Q 2014 Fibromyalgia program informs development of TNX - 102 SL in PTSD Safety data from fibromyalgia studies are potentially supportive for PTSD program Efficacy data support potent
Slide 20: 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 i n military - related PTSD 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 pl
Slide 21: TNX-201 in development for episodic tension-type headache
TNX - 201 in development for episodic tension - type headache Preclinical Phase 1 NDA Market Candidate Indication Phase 2 Phase 3 TNX - 102 SL 2019E TNX - 102 SL 2018E TNX - 201 Episodic Tension - Type Headache 2019E 21 TNX - 102 SL (cyclobenzaprine HCl sublingual tablet) 2.8 mg and TNX - 201 (isome
Slide 22: 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. 70 million adults in the U.S. experience f
Slide 23: TNX-201 to enter clinical development in 2014
TNX - 201 to enter clinical development in 2014 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 4Q 2014 TNX - 201 IND c
Slide 24: 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 H eadache
Slide 25: Financial summary
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. 25 † As of October 27, 2014
Slide 26: Management team
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 26
Slide 27: Milestones – recent and upcoming
Milestones – recent and upcoming TNX - 102 SL – Fibromyalgia □ September 2014 – Reported top line results from Phase 2b BESTFIT study □ 1H 2015 – Begin Phase 3 program TNX - 102 SL – Post - Traumatic Stress Disorder □ June 2014 – Received IND clearance □ 4Q 2014 – Commence Phase 2 AtEase study in mi
Slide 28: October 2014
October 2014 © 2014 Tonix Pharmaceuticals Holding Corp. NASDAQ: TNXP
Tonix Pharmaceuticals Holding — Investor Presentation (October 2014) on 6ix