Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (March 2017)
30 slides from Tonix Pharmaceuticals's investor presentation, Mar 20, 2017
Slide 1: Investor Presentation
Investor Presentation TONIX PHARMACEUTICALS Version POOSS 3-15-17
Slide 2: Cautionary Note on Forward-Looking Statements
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 may be i
Slide 3: Tonix (TNXP): Developing Innovative Pharmaceutical Products
Tonix (TNXP): Developing Innovative Pharmaceutical Products to Address Current and Emerging Public Health Challenges Targeting central nervous system conditions + In Phase 3 development, TNX-102 SL* for posttraumatic stress disorder (PTSD) with Breakthrough Therapy designation from the U.S. Food and
Slide 4: Product Pipeline
Product Pipeline ee ee a Bedtime PTSD TNX-102 SL_ awed Cyclobenzaprine HCI sublingual tablets Phase 2 results reported May 2016 Phase 3 study initiation in 192017 Tianeptine oxalate oral formulation Live HPXV virus vaccine from cell culture, ‘Novel polymorph and salt discovered and ‘characterized Ho
Slide 5: Tonix Pharmaceuticals PTSD Program
Tonix Pharmaceuticals PTSD Program TNX-102 SL (cyclobenzaprine HCI sublingual tablets) + Aunique, innovative product designed for bedtime administration Phases + Targeting a chronic and serious psychiatric disorder: PTSD pavalapiient ¥ Therapeutic dose identified in Phase 2 study Phase 3 clinical an
Slide 6: PTSD Characteristics
PTSD Characteristics Unmet medical need + PTSD is a serious condition and the prevalence is increasing, especially combat PTSD + Military-related PTSD is not satisfactorily treated by existing FDA-approved therapies + Two selective serotonin reuptake inhibitors (SSRIs) are approved for PTSD, but hav
Slide 7: Breakthrough Therapy Designation
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 of Breakthrough Therapy designa n Eligibility for prio
Slide 8: What Are the Consequences of PTSD?
What Are the Consequences of PTSD? Consequences: + Impaired daily function and substan + Reckless or destructive behavior + Increased health care utilization and greater medical mort interference with work and social interactions PTSD as a risk factor for: + Depression + Alcohol or substance abuse A
Slide 9: TNX-102 SL: Proprietary Patented Formulation
TNX-102 SL: Proprietary Patented’ Formulation Active Pharmaceutical Ingredient (API) is cyclobenzaprine (CBP), is structurally related to tricyclic antidepressants (TCAs) + TCAs and their metabolites differ widely in their receptor binding and pharmacokinetic profiles? ~ CBP is more selective for hi
Slide 10: High Prevalence of PTSD Among Combat Veterans
High Prevalence of PTSD Among Combat Veterans 10 3-9% 19-31% >19% General population’ Vietnam veterans? Operation Enduring Freedom (OEF; Afghanistan) / Operation Iraqi Freedom @BK_8.6 million American adults affected! (Gir) veterans / Operation: New Dawn (OND)? QC Women more likely to develop than m
Slide 11: Growing Economic and Social Burden to Care for Veterans with PTSD
Growing Economic and Social Burden to Care for Veterans with PTSD 1 Health care costs associated with PTSD for OEF/OIF/OND veterans: Direct costs Indirect costs $3,000-5,000 ion estimated yearly cost to society? ~ 2.7 million troops depl I 10/1/2001 and 3/3. js ystem? Veterans, ‘rough 2nd Qtr FY Bis
Slide 12: Phase 2 AtEase Study in Military-Related PTSD
Phase 2 AtEase Study in Military-Related PTSD Randomized, double-blind, placebo-__*_Enrolled patients with baseline controlled trial in military-related PTSD CAPS-5 2 29 + Analysis from 231 patients; 24 U.S. clinical sites TNX-102 SL at bedtime once-daily + TNX-102 SL was active at 5.6 mg dose 2.8 m
Slide 13: AtEase Study Demographics and Characteristics
AtEase Study Demographics and Characteristics 13 © 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. military distribution © Current Major
Slide 14: AtEase Study - Summary of Primary and Secondary Analyses
AtEase Study - Summary of Primary and Secondary Analyses (week 12) ‘Assessment Domain ‘Analysis p-Values 2.8 mg (N=90) | 5.6 mg (N=49) CAPSS [Total [MRM 0.259% 0.053 [rotal IMMRM with Multiple imputation 211 0.031" lrotal lMMRM w/ Hybrid LOCF/BOCF 0.172 0.037" lrotal lANCOVA 0.090 0.038" [CAPS-5 clu
Slide 15: AtEase Study: Safety and Tolerability Profile
AtEase Study: Safety and Tolerability Profile No serious adverse events reported with TNX-102 SL deemed related to treatment Systemic Adverse ventas acaho TWXHOESL28mg TWX-1G2SL5.6 mg ‘Somnolence 6.4% 11.8% 16.0% Dry Mouth 10.6% 4.3% 16.0% Headache 43% 4% 12.0% Insormia 85% 75% 6.0% Sedation 1.1% 2.
Slide 16: AtEase: Retrospective Analysis for TNX-102 SL 5.6 mg
AtEase: Retrospective Analysis for TNX-102 SL 5.6 mg Prior pharmacotherapy trials in PTSD used earlier versions of CAPS (e.g. CAPS-2) + Different scoring range from CAPS-5 + Most frequently used a score of > 50 for entry (similar to CAPS-5 > 33%) + FDA has accepted this higher entry criteria (CAPS-5
Slide 17: Retrospective Analysis of CAPS-5 in Patients with Entry CAPS-5 ≥33
Retrospective Analysis of CAPS-5 in Patients with Entry CAPS-5 2 331 WkKO | Wk2Wk4 Wk6 WKB W100 Wk 12 0.0 Placebo 2 g so -+TNX-102 SL.2.8 mg g “+-TNX-102 SL5.6 mg B 10.0 § 15.0 3 g * B 20.0 8 point difference in CAPS-5 ra thange from baseline between § * placebo and TNX-102 SL 5.6 mg £ aso * Erect s
Slide 18: Retrospective Analyses of Week 12 Outcome Measures for TNX-102 SL 5.6 mg vs. Placebo
Retrospective Analyses of Week 12 Outcome Measures for TNX-102 SL 5.6 mg vs. Placebo ‘ary-related or in M Combat PTSD Subset and for CAPS-5 2 29 and CAPS-5 2 33 at Baseline 18 Total Cluster B (intrusion) Cluster € (avoidance) Cluster D (mood/cognition) ‘Cluster € (arousal and reactivity) 6 (Sleep it
Slide 19: Phase 2 AtEase Study Conclusions
Phase 2 AtEase Study Conclusions 19 t large multi-center trial demonstrating efficacy of an Investigat New Drug (IND) in military-related PTSD ¥ TINX-102 SL therapeutic dose (5.6 mg) identified ¥ Symptom reduction (CAPS-5) ¥ Functional improvement (Sheehan Disability Scale domains) ¥ Clinical global
Slide 20: Planned Phase 3 HONOR Study in PTSD
Planned Phase 3 HONOR Study in PTSD 20 To confirm AtEase findings in military- General study characteristics: related PTSD: + Randomized, double-blind, placebo-controlled, entrance CAPS-S 233 + Larger adaptive design study + Targeting start in 19 2017 ‘One unblinded interim analysis (IA) by an indep
Slide 21: Intellectual Property
Intellectual Property TN 02 SL Portfolio of international patents filed under the Patent Cooperation Treaty (PCT) Composition-of-matter (eutectic) + Notice of Allowance issued by U.S. Patent and Trademark Office + Additional claims and jurisdictions pending + Protection expected to 2034 Pharmacokine
Slide 22: Proprietary Cyclobenzaprine Hydrochloride Eutectic Mixture Stabilizes Tablet Formulation
© Proprietary Cyclobenzaprine Hydrochloride Eutectic Mixture Stabilizes Tablet Formulation (CBP-HCI) pats i PAA » ee Eutectic eee eee On - sollicice a « ot “HPO, @ é (K,HPO,) Vv & Vv < ooo Desiariie . Soe SE Peg & fReebece om ® o eo § PRB CO He: PMeMoPeed o> fore PH hearts wth base imc ton tnt CSI r
Slide 23: Product Pipeline
Product Pipeline Candidate Indication =e 23 Bedtime PTSD TNX-102 SL_ awed Cyclobenzaprine HCI sublingual tablets Tianeptine oxalate oral formulation Live HPXV virus vaccine from cell culture, [erent fre nee 2 ne 3 Phase 2 results reported May 2016 Phase 3 study initiation in 192017 ‘Novel polymorph
Slide 24: TNX-601 - A Potential Clinical Candidate for PTSD
TNX-601 - TNX-601 (tianep' A Potential Clinical Candidate for PTSD e oxalate oral formulation) 2 Pre-IND Candidate ga Health Challenge Targeted as a 1* line monotherapy for PTSD: daytime dosing v Leverages expertise in PTSD (clinical and regulatory experience, market analysis, etc.) ¥ Mechanism of A
Slide 25: TNX-801: A Potential Smallpox-Preventing Vaccine
TNX-801: A Potential Smallpox-Preventing Vaccine 25 Pre-IND Candidate Targeting a Public Health Issue 1. aes, Act al Ong design, development and Therapy: 4:71:79 (2010) TNX-801 (live virus vaccine - synthetic Horsepox (HPXV) vaccine) + Potential improvement over current methods for biodefense agains
Slide 26: Management Team
Management Team 26 ‘Seth Lederman, MD President & CEO TARSENT™ &Fusiley’ vela. Gregory Sullivan, MD Chief Medical Officer Cowmata Univesity New York State Department of Psychiatry Psychiatric Institute Bradley Saenger, CPA Chief Financial Officer Ghire veftex a wi a Jessica Morris EVP, Operations, A
Slide 27: Board of Directors
© Board of Directors ‘Seth Lederman, MD Chairman Ernest Mario, PhD ALZA, Glaxo, Reliant Pharma ‘Stuart Davidson Labrador Ventures, Alkermes, Combion Charles Mather BTIG, Janney, Jefferies, Cowen, Smith Barney Patrick Grace Apollo Philanthropy, WR Grace, Chemed John Rhodes NYSERDA, NRDC, Booz Allen H
Slide 28: Milestones - recent and upcoming
Milestones - recent and upcoming INX-102 SL - Posttraumatic Stress Disorder © May 2016 Reported results from AtEase study & August 2016 End-of Phase 2 meeting with FDA - Proposed Phase 3 clinical and NDA plan accepted @& December 2016 _ Breakthrough Therapy designation granted by FDA & January 2017
Slide 29: Summary
Summary 29 Strong position for value growth Phase 3 development in a major medical indication: PTSD including military-related PTSD + Phase 3 asset not previously well-known to the investor marketplace ‘TNX-102 SL for PTSD is designated as a Breakthrough Therapy by FDA + Accelerated development and
Slide 30: Thank you!
TONIX PHARMACEUTICALS NASDAQ: TNXP Thank you! © 2017 Tonix Pharmaceuticals Holding Corp.
Tonix Pharmaceuticals Holding — Investor Presentation (March 2017) on 6ix