Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (May 2017)
39 slides from Tonix Pharmaceuticals's investor presentation, May 1, 2017
Slide 1: Investor Presentation May 2017
© 2017 Tonix Pharmaceuticals Holding Corp. May 2017 Version P0061 5 - 1 - 17 Investor Presentation
Slide 2: Cautionary Note on Forward-Looking Statements
© 2017 Tonix Pharmaceuticals Holding Corp. 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 Litigat
Slide 3: Phase 3 Breakthrough Therapy Program in PTSD
© 2017 Tonix Pharmaceuticals Holding Corp. 3 Phase 3 Breakthrough Therapy Program in Posttraumatic Stress Disorder (PTSD) Phase 3 HONOR study of TNX - 102 SL 1 in military - related PTSD enrolling • Encouraging evidence of safety and efficacy was demonstrated in Phase 2 Breakthrough Therapy designat
Slide 4: Phase 3 HONOR Study in PTSD Enrolling
© 2017 Tonix Pharmaceuticals Holding Corp. 4 Phase 3 HONOR Study in PTSD Enrolling General s tudy c haracteristics: • Randomized, double - blind, placebo - controlled, entrance CAPS - 5 ≥ 33 • One unblinded interim analys i s (IA) by an independent data monitoring committee at 50% recruitment • IA (
Slide 5: Breakthrough Therapy Designation
© 2017 Tonix Pharmaceuticals Holding Corp. 5 Breakthrough Therapy Designation • FDA granted TNX - 102 SL Breakthrough Therapy designation – reported December 19, 2016 • PTSD is a serious condition • TNX - 102 SL has potential advantages over existing therapies in military - related PTSD • Benefits o
Slide 6: No Recognized Abuse Potential in Clinical Studies
© 2017 Tonix Pharmaceuticals Holding Corp. 6 No Recognized Abuse Potential in Clinical Studies • Active ingredient is cyclobenzaprine, which is structurally related to tricyclic antidepressants • Cyclobenzaprine interacts with receptors that regulate sleep quality: 5 - HT 2a ; α 1 - adrenergic and h
Slide 7: SL Intellectual Property – U.S. protection until 2034
© 2017 Tonix Pharmaceuticals Holding Corp. 7 TNX - 102 SL Intellectual Property – U.S. protection until 2034 Composition of matter (Eutectic) • Notice of Allowance issued by U.S. Patent and Trademark Office • Protection expected to 2034 • Additional claims and jurisdictions pending Pharmacokinetics
Slide 8: Proprietary Cyclobenzaprine HCl Eutectic Mixture Stabilizes Tablet Formulation
© 2017 Tonix Pharmaceuticals Holding Corp. 8 Proprietary Cyclobenzaprine Hydrochloride Eutectic Mixture Stabilizes Tablet Formulation Base particle (K 2 HPO 4 ) Base particle (K 2 HPO 4 ) Base particle (K 2 HPO 4 ) Eutectic formulation ANGSTRO - TECHNOLOGY™ Cyclobenzaprine - HCl (CBP - HCl) Eutectic
Slide 9: SL: Sublingual Formulation Designed for Bedtime Administration
© 2017 Tonix Pharmaceuticals Holding Corp. 9 TNX - 102 SL: Sublingual Formulation is Designed for Bedtime Administration TNX - 102 SL: Proprietary sublingual formulation of CBP with transmucosal absorption • Innovation by design with patent protected CBP and mannitol eutectic formulation • Rapid sys
Slide 10: SL: Novel Mechanism Targets Sleep Quality for Recovery from PTSD
© 2017 Tonix Pharmaceuticals Holding Corp. 10 TNX - 102 SL: Novel mechanism Targets Sleep Quality for Recovery from PTSD PTSD is a disorder of recovery • Most people exposed to extreme trauma recover over a few weeks • In PTSD, recovery process impeded due to insufficient sleep - dependent memory pr
Slide 11: SL: Novel Mechanism Targets Sleep Quality for Recovery from PTSD
© 2017 Tonix Pharmaceuticals Holding Corp. 11 TNX - 102 SL : Novel Mechanism Targets Sleep Quality for Recovery from PTSD PTSD is a disorder of recovery • Most people exposed to extreme trauma recover over a few weeks • In PTSD, recovery process impeded due to insufficient sleep - dependent memory p
Slide 12: What Are the Symptoms of PTSD?
© 2017 Tonix Pharmaceuticals Holding Corp. 12 What Are the Symptoms of PTSD? Symptoms of PTSD fall into four clusters: 1. Intrusion (aversive memories, nightmares, flashbacks) 2. Avoidance (avoiding persons, places or situations) 3. Mood/cognitions (memory block, emotional numbing, detachment from o
Slide 13: What Are the Consequences of PTSD?
© 2017 Tonix Pharmaceuticals Holding Corp. 13 What Are the Consequences of PTSD? Consequences: • Impaired daily function and substantial interference with work and social interactions • Reckless or destructive behavior • Increased health care utilization and greater medical morbidity PTSD as a risk
Slide 14: PTSD: Not Well-served by Approved Treatments
© 2017 Tonix Pharmaceuticals Holding Corp. 14 PTSD: Not Well - served by Approved Treatments FDA approved selective serotonin reuptake inhibitors (SSRIs) (paroxetine and sertraline) have not shown efficacy in military - related PTSD Majority of patients unresponsive or intolerant to current treatmen
Slide 15: Why Initially Target Military-Related PTSD?
© 2017 Tonix Pharmaceuticals Holding Corp. 15 Why Initially Target Military - Related PTSD? 1. Friedman et al., J Clin Psychiatry 2007; 68:711 2. Zoloft Package Insert, August, 2014 3. Paxil Package Insert, June, 2014 4. Fava et al., Psychother Psychosom 84:72 - 81, 2015 Military - related PTSD not
Slide 16: High Prevalence of PTSD Among Combat Veterans
© 2017 Tonix Pharmaceuticals Holding Corp. 16 High Prevalence of PTSD Among Combat Veterans 1 Kessler et al., Arch Gen Psych 2005; Prevalence rate of 3.5% applied to U.S. Census estimate of 247M U.S. adult ( > 18) population in 2015 ( www.census.gov/quickfacts/table/PST045215/00 ) ; 2 Norris, PTSD R
Slide 17: Growing Economic and Social Burden to Care for Veterans with PTSD
© 2017 Tonix Pharmaceuticals Holding Corp. 17 Growing Economic and Social Burden to Care for Veterans with PTSD Health care costs associated with PTSD for OEF/OIF/OND veterans: Direct costs Indirect costs 1 CBO Report 2012; 2 Tanielan, Invisible Wounds of War . 2005; 3 Analysis of VA Health Care Uti
Slide 18: Phase 2 AtEase Study in Military-Related PTSD
© 2017 Tonix Pharmaceuticals Holding Corp. 18 Phase 2 AtEase Study in Military - Related PTSD • Enrolled patients with baseline CAPS - 5 ≥ 29 • Analysis from 231 patients; 24 U.S. clinical sites • TNX - 102 SL was active at 5.6 mg dose • Primary efficacy analysis: • Difference in CAPS - 5 score betw
Slide 19: Results of Phase 2 AtEase Study in Military-Related PTSD
© 2017 Tonix Pharmaceuticals Holding Corp. 19 Results of Phase 2 AtEase Study in Military - Related PTSD TNX - 102 SL 5.6 mg showed clinical benefit in military - related PTSD • CAPS - 5 scale, was statistically significant by Mixed effect Model Repeated Measures, or MMRM, with Multiple Imputation,
Slide 20: AtEase Study Demographics and Characteristics
© 2017 Tonix Pharmaceuticals Holding Corp. 20 AtEase Study Demographics and Characteristics 93% of the randomized patients were male 98% had trauma during military service Deployed an average of 2.3 times Mean time since index trauma was 7 years Race and ethnicity generally consistent with U.S. mili
Slide 21: AtEase Study: Traumas Associated with PTSD
© 2017 Tonix Pharmaceuticals Holding Corp. 21 AtEase Study: Traumas Associated with PTSD *Some patients experienced more than one trauma 0 5 10 15 20 25 30 35 40 Other Knowing someone seriously injured or killed Being responsible for the death of an enemy combatant Seeing dead bodies or human remain
Slide 22: AtEase Study – Summary of Primary and Secondary Analyses (week 12)
© 2017 Tonix Pharmaceuticals Holding Corp. 22 AtEase Study – Summary of Primary and Secondary Analyses (week 12)
Slide 23: AtEase Study: Safety and Tolerability Profile
© 2017 Tonix Pharmaceuticals Holding Corp. 23 AtEase Study: Safety and Tolerability Profile No serious adverse events reported with TNX - 102 SL deemed related to treatment Systemic Adverse Events* Placebo (N=94) TNX - 102 SL 2.8 mg (N=93) TNX - 102 SL 5.6 mg (N=50) Somnolence 6.4% 11.8% 16.0% Dry M
Slide 24: Products in Development
© 2017 Tonix Pharmaceuticals Holding Corp. 24 Products in Development Preclinical Phase 2 NDA 1 /BLA 2 Market Pipeline Product Indication/ Description Phase 3 TNX - 102 SL Bedtime PTSD Treatment Daytime PTSD Treatment TNX - 601 Novel polymorph and salt discovered and characterized TNX - 801 Horsepox
Slide 25: TNX-601 (tianeptine oxalate): A Potential Clinical Candidate for PTSD
© 2017 Tonix Pharmaceuticals Holding Corp. 25 TNX - 601 (tianeptine oxalate): A Potential Clinical Candidate for PTSD Pre - IND Candidate • Targeted as a 1 st line monotherapy for PTSD : oral formulation for daytime dosing x Leverages expertise in PTSD (clinical and regulatory experience, market ana
Slide 26: Structural Comparison: SL and TNX-601
© 2017 Tonix Pharmaceuticals Holding Corp. 26 Structural Comparison: TNX - 102 SL and TNX - 601 Cyclobenzaprine and tianeptine share structural similarities with classic tricyclic antidepressants (TCAs) and to each other, but each has unique pharmacological properties • Tianeptine has a 3 - chlorodi
Slide 27: TNX-601: A Potential Clinical Candidate for PTSD
© 2017 Tonix Pharmaceuticals Holding Corp. 27 • Tianeptine modulates the glutamatergic system indirectly • Does not have significant affinity (K i >10 μ M) for NMDA or AMPA receptors • Tianeptine is a weak µ - opioid receptor (MOR) agonist • Controlled substance in France, Bahrain and Singapore • Ti
Slide 28: TNX-801 (Synthetic Live Horsepox Virus): A Potential Smallpox-Preventing Vaccine
© 2017 Tonix Pharmaceuticals Holding Corp. 28 TNX - 801 ( Synthetic Live Horsepox Virus ) A Potential Smallpox - Preventing Vaccine Pre - IND Candidate Potential improvement over current methods for biodefense against smallpox x Leverages government affairs effort x Collaboration with Professor Davi
Slide 29: TNX-801: Synthetic Live Horsepox Virus
© 2017 Tonix Pharmaceuticals Holding Corp. 29 TNX - 801: Synthetic Live Horsepox Virus A Potential Smallpox - Preventing Vaccine Synthesis from sequence of a 1976 Mongolian isolate 1 In mice, TNX - 801 behaved like attenuated vaccinia virus (vaccinia virus is foundation of current smallpox vaccines)
Slide 30: Horsepox - Better Tolerability as a Vaccine?
© 2017 Tonix Pharmaceuticals Holding Corp. 30 Horsepox - Better Tolerability as a Vaccine? Horsepox is caused by HPXV and is characterized by mouth and skin eruptions HXPV isolate from the 1976 outbreak later sequenced Modern vaccinia virus smallpox vaccines are associated with cardiotoxicity; may h
Slide 31: A Better Smallpox-Preventing Vaccine is Important and Necessary Today
© 2017 Tonix Pharmaceuticals Holding Corp. 31 A Better Smallpox - Preventing Vaccine is Important and Necessary Today Smallpox was eradicated as a result of global public health campaigns No cases of naturally - occurring smallpox have been reported since 1977 Accidental or intentional transmission
Slide 32: TNX-801: A Potential Medical Countermeasure
© 2017 Tonix Pharmaceuticals Holding Corp. 32 TNX - 801: A Potential Medical Countermeasure 21st Century Cures Act (2016), Section 3086 • Encouraging treatments for agents that present a national security threat Medical countermeasures are drugs or vaccines intended to treat: • Biological, chemical,
Slide 33: TNX-801: A Potential Smallpox-Preventing Vaccine
© 2017 Tonix Pharmaceuticals Holding Corp. 33 Live virus vaccines stimulate cross - reactive immunity • Protects from consequences of infection with smallpox agent • Renders recipient “immune” • Provides additional protection of non - immunized population Potential safety advantage over existing vac
Slide 34: Financial Overview
© 2017 Tonix Pharmaceuticals Holding Corp. 34 Financial overview NASDAQ: TNXP Cash, cash equivalents, and marketable securities reported at December 31, 2016 $26.1 million Approximate net proceeds from at - the - market offering since January 1, 2017 $9.1 million Approximate net proceeds from underw
Slide 35: Management Team
© 2017 Tonix Pharmaceuticals Holding Corp. 35 Management Team Seth Lederman, MD President & CEO Jessica Morris EVP, Operations Gregory Sullivan, MD Chief Medical Officer Bradley Saenger, CPA Chief Financial Officer
Slide 36: Board of Directors
© 2017 Tonix Pharmaceuticals Holding Corp. 36 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 St
Slide 37: Milestones – Recently Completed and Upcoming
© 2017 Tonix Pharmaceuticals Holding Corp. 37 Milestones – Recently Completed and Upcoming TNX - 102 SL – Posttraumatic Stress Disorder □ May 2016 Report ed results from Phase 2 AtEase study □ August 2016 End - of - Phase 2 meeting with FDA - Proposed Phase 3 clinical and NDA plan accepted □ Decembe
Slide 38: Summary
© 2017 Tonix Pharmaceuticals Holding Corp. 38 Summary Strong position for value growth with Phase 3 development in a major medical indication: PTSD including military - related PTSD • PTSD is an important public health issue TNX - 102 SL for PTSD is designated as a Breakthrough Therapy by FDA • Acce
Slide 39: Thank you!
© 2017 Tonix Pharmaceuticals Holding Corp. Thank you ! NASDAQ: TNXP
Tonix Pharmaceuticals Holding — Investor Presentation (May 2017) on 6ix