Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (September 2016)
34 slides from Tonix Pharmaceuticals's investor presentation, Sep 6, 2016
Slide 1: Investor Presentation September 2016
NASDAQ: TNXP Investor Presentation September 2016 © 2016 Tonix Pharmaceuticals Holding Corp. Version: P0028 09-06-2016
Slide 2: Cautionary note on forward-looking statements
Cautionary note on forward-looking statements Certainstatementsinthispresentationregardingstrategicplans,expectationsandobjectivesforfutureoperationsorresultsare“forward-lookingstatements”asdefinedbythePrivateSecuritiesLitigationReformActof1995.Thesestatementsmaybeidentifiedbytheuseofforward-looking
Slide 3: Developing innovative medicines for large and growing markets
Developinginnovative medicines for large and growing markets Targeting common central nervous system disordersOne clinical-stage proprietary candidate targeting posttraumatic stress disorder (PTSD) Differentiated product with potential for sustainable competitive advantages PTSD –Phase 2 trial repor
Slide 4: Overview: PTSD program
Overview: PTSD program Results from multi-center, double-blind, randomized, placebo-controlled Phase 2 trial in military-related PTSD reported May 2016TNX-102 SL 5.6 mg was active in treating military-related PTSDReduction in symptoms and disease severity [(Clinician-Administered PTSD Scale) CAPS-5]
Slide 5: Tonix is developing TNX-102 SL for PTSD
Tonix is developing TNX-102 SL for PTSD Advanced sublingual tablet containing low-dose cyclobenzaprine (CBP) Designed for daily bedtime administration with no titration Efficient transmucosalabsorption Avoidance of first-pass metabolism reduces formation of long-lived metabolite TNX-102 SL’s pharmac
Slide 6: PTSD is a chronic stress disorder triggered by a traumatic event
PTSD is a chronic stress disorder triggered by a traumatic event PTSD is characterized by: Considered a stress response, but prolonged and does not resolve with time20% of women and 8% of men in the U.S. who experience significant trauma develop PTSD1 Associated with significant life disruptionSocia
Slide 7: PTSD is a prevalent problem for both civilians and the military
PTSD is a prevalent problem for both civilians and the military Affects 3.5% (8.5 M) U.S. Adults1 ~70% are considered to have moderate to severe symptoms Of those diagnosed, ~50% utilize professional healthcare (psychotherapy/pharmacotherapy)2 Higher prevalence in military population 20% of veterans
Slide 8: Limitations of current FDA-approved pharmacotherapies for military-related PTSD
Limitations of current FDA-approved pharmacotherapies for military-related PTSD No treatment response observed in U.S. military populationSertraline: negative large multi-center trial in U.S. military veterans1Placebo numerically superior on CAPS-2 Paroxetine: not studied in military population Inco
Slide 9: Phase 2 AtEase study of TNX-102 SL in PTSD
Phase 2 AtEase study of TNX-102 SL in PTSD AtEase Study Randomized, double-blind, placebo-controlled trial DSM-5 diagnostic criteria for PTSD 231* participants studied 2:1:2 at 24 U.S. sites1 x TNX-102 SL 2.8 mg tablet: 2 x TNX-102 SL 2.8 mg tablets: placebo Evaluated the Clinician Administered PTSD
Slide 10: Phase 2 AtEase trial of TNX-102 SL in PTSD
Phase 2 AtEase trial of TNX-102 SL in PTSD Randomized, double-blind, placebo-controlled trial in military-related PTSD Analysis from 231 patients; 24 U.S. clinical sites Primary efficacy endpoint:Difference in CAPS score between TNX-102 SL 2.8 mg and placebo at week 12 TNX-102 SL was active at 5.6 m
Slide 11: Key Demographics / Characteristics of AtEase
AtEasestudy of TNX-102 SL in PTSD Key Demographics / Characteristics of AtEase 93% of the patients were male 98% had trauma during military service and were deployed on average 2.3 times Mean time since index trauma was 7 years Race and ethnicity generally consistent with U.S. military distribution
Slide 12: AtEase results on key clinical endpoints
AtEaseresults on key clinical endpoints Category Endpoint –week 121 pvalue PTSD Symptoms CAPS-5(MMRM with MI) 0.031 Global improvement CGI-I (Logistic Regression) 0.041 Arousal and reactivity CAPS-5 cluster (MMRM) 0.048 Sleep Quality CAPS-5 sleep (MMRM) 0.010 12 © Copyright 2016 Tonix Pharmaceutical
Slide 13: AtEase study results: CAPS-5 total score mean change from baseline
AtEasestudy results:CAPS-5 total score mean change from baseline -25 -20 -15 -10 -5 0 Wk 0 Wk 2 Wk 4 Wk 6 Wk 8 Wk 10 Wk 12 Placebo TNX-102 SL 2.8 mg TNX-102 SL 5.6 mg * *p=0.031, comparing placebo and TNX-102 SL 5.6 mg,*p<0.05, comparing placebo and TNX-102 SL 2.8 mg, by MMRM with MI, mixed-effect m
Slide 14: AtEase study results: CAPS-5 sleep disturbance
AtEasestudy results: CAPS-5 sleep disturbance -1.6 -1.4 -1.2 -1 -0.8 -0.6 -0.4 -0.2 0 Wk 0 Wk 2 Wk 4 Wk 6 Wk 8 Wk 10 Wk 12 LS Mean Change from Baseline Placebo TNX-102 SL 2.8 mg TNX-102 SL 5.6 mg Change from baseline * * * * *p<0.05, 5.6 mg v. placebo, MMRM *p<0.05, 2.8 mg v. placebo, MMRM 14 © Copy
Slide 15: AtEase study results: Sheehan Disability Scale
AtEasestudy results: Sheehan Disability Scale 1.5 2 2.5 3 3.5 4 4.5 5 5.5 Wk 0 Wk 2 Wk 4 Wk 6 Wk 8 Wk 10 Wk 12 The symptoms have disrupted your work/school work Placebo TNX-102 SL 2.8 mg TNX-102 SL 5.6 mg 2.5 3 3.5 4 4.5 5 5.5 6 6.5 7 7.5 Wk 0 Wk 2 Wk 4 Wk 6 Wk 8 Wk 10 Wk 12 The symptoms have disrup
Slide 16: AtEase study results: Clinical Global Impression
AtEasestudy results: Clinical Global Impression Clinical Global Impression –Improvement Scale Responders Patients (%) 44.6% 53.3% 63.3% 0% 10% 20% 30% 40% 50% 60% 70% Placebo TNX-102 SL 2.8 mg TNX-102 SL 5.6 mg *p=0.041, Logistic regression comparing placebo and TNX-102 SL 5.6 mg Responders are thos
Slide 17: TNX-102 SL safety and tolerability profile in the AtEase study
TNX-102 SL safety and tolerability profile in the AtEasestudy 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 5.6mg (N=50) Total TNX-102 SL(N=143) Somnolence 6.4% 11.8% 16.0% 13.3% DryMouth 10.6%
Slide 18: Determining CAPS-5 severity criteria for entry in Phase 3 trials
Determining CAPS-5 severity criteria for entry in phase 3 trials CAPS-5hasnotbeenemployedpreviouslyinapharmacotherapytrial;entryseveritythresholdnotclear;≥29usedinthePhase2AtEasestudy PriorpharmacotherapytrialsinPTSDusingearlierversionsofCAPSwithdifferentscoringrangehavemostfrequentlyusedascoreof≥50
Slide 19: CAPS-5 mean change from baseline for entry CAPS ≥ 33
CAPS-5 mean change from baseline for entry CAPS ≥ 33 -30.0 -25.0 -20.0 -15.0 -10.0 -5.0 0.0 Wk 0 Wk 2 Wk 4 Wk 6 Wk 8 Wk 10 Wk 12 LS Mean (SE) Placebo TNX-102 SL 2.8 mg TNX-102 SL 5.6 mg 6.8 point difference in CAPS-5 change from baseline between placebo and TNX-102 SL 5.6 mg Effect Size = 0.53 * * *
Slide 20: CAPS-5 cluster scores
CAPS-5 cluster scores Table shows the significance levels and effect sizes of the CAPS-5 cluster scores, comparing TNX-102 SL 5.6 mg and placebo, using a CAPS-5 baseline entry criterion of ≥ 33 and the per protocol threshold of ≥ 29 Entry criteria of CAPS-5 ≥ 33 Entry criteria of CAPS-5 ≥ 29 5.6 mg
Slide 21: Rate of remission from PTSD in each treatment arm
Rate of remission from PTSD in each treatment arm 14.3% 17.1% 28.9% 0% 5% 10% 15% 20% 25% 30% 35% Placebo(N=77) TNX-102 SL 2.8 mg(N=70) TNX-102 SL 5.6 mg(N=38) Percent of Remitters in Group #p=0.10 NS, comparing placebo and TNX-102 SL 5.6 mg, logistic regression # Remission is defined as CAPS-5 scor
Slide 22: AtEase study conclusions
AtEasestudy conclusions This Phase 2 trial is the first large multi-center trial of an investigational new drug product that demonstrated efficacy in a population with military-related PTSDSymptom reduction (CAPS-5) Functional improvement (Sheehan Disability Scale domains) Global improvement (CGI-I)
Slide 23: Phase 3 program in PTSD being planned
Phase 3 program in PTSD being planned General Study Characteristics: Randomized, double-blind, placebo-controlled studies in PTSD N~400-500; approximately 35 U.S. clinical sites Primary Efficacy Endpoint: Mean change from baseline in total CAPS-5 at Week 12 compared between TNX-102 SL 5.6 mg and pla
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-usePTSD: patents filed TNX-102 SL Wholly-owned by Tonixwith no obligations to others 24 © Copyright 2016 Tonix Pharmaceuticals
Slide 25: TNX-102 SL: active pharmaceutical ingredient
TNX-102 SL: active pharmaceutical ingredient Cyclobenzaprine is a tricyclic molecule that binds to a number of central nervous system (CNS) receptor types Highest affinity for three receptors believed to have a role in treating sleep disturbances 5-HT2A receptor α1 adrenergic receptor H1 receptor ©
Slide 26: Receptor binding profile: Parent and primary metabolite
Receptor binding profile: Parent and primary metabolite 0 5 10 15 20 25 30 35 40 45 50 5-HT2c H1 5-HT2a α2c , NET 5-HT2b Undesirable Characteristics of Norcyclobenzaprine (nCBP) Half-life (t1/2) of 72 hours (will accumulate) At steady-state, projected similar exposure day and night Distinct receptor
Slide 27: TNX-102 SL single-dose PK study
TNX-102 SL single-dose PK study TNX-102 SL 2.8 mg PK profile relative to 5 mg oral immediate release (IR) CBP: 0 500 1000 1500 2000 2500 3000 3500 4000 0 4 8 12 16 20 24 Concentration (ng/mL) TNX-102 SL 2.8 mg Oral IR CBP 5 mg xLower exposure of nCBP by 48% xHigher ratio of CBP relative to nCBP Rati
Slide 28: TNX-102 SL: Pharmacokinetic profile after sublingual route of administration
TNX-102 SL: Pharmacokinetic profile after sublingual route of administration Very rapid absorption after dosing (measurable in plasma within 3 minutes of administration)Reaching tmaxat ~4 hours after dosing Approximately 50% of exposure to cyclobenzaprine occurs during first 8 hours Avoids first-pas
Slide 29: Cyclobenzaprine is detected in plasma within minutes following sublingual TNX-102 SL
Cyclobenzaprine is detected in plasma within minutes following sublingual TNX-102 SL Plasma Concentration Versus Time of TNX-102 SL Compared to Oral Cyclobenzaprine IR 0 0.5 1.0 1.5 Concentration (ng/L) 2500 1500 1000 500 2000 0 TNX-102 SL 2.8 mg (sublingual) Cyclobenzaprine IR 5 mg (oral) Time (h)
Slide 30: Management team
Management team Seth Lederman, MD President & CEO Jessica Edgar Morris EVP, Operations Bruce Daugherty, PhD, MBA Chief Scientific Officer Ronald Notvest, PhD EVP, Commercial Planning & Development Gregory Sullivan, MD Chief Medical Officer Bradley Saenger, CPA Chief Financial Officer 30 © Copyright
Slide 31: Board of directors
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 Stuart Davidson Labrador Ventures, Alkermes, Com
Slide 32: Financial overview
Financial overview NASDAQ:TNXP Cash, cash equivalents, andmarketable securitiesreported at June 30, 2016 $ 31.2 million Net proceeds from overallotment in 3Q16 $1.4 million Sharesoutstanding(September 6, 2016) 25.9 million 32 © Copyright 2016 Tonix Pharmaceuticals
Slide 33: Milestones – recent and upcoming
Milestones –recent and upcoming TNX-102 SL –Posttraumatic Stress Disorder December 2015Entered into Collaborative Research and Development Agreement (CRADA) with the United States Army Medical Materiel Development Activity (USAMMDA) May 2016Report results from AtEasestudy August 2016End of Phase 2 m
Slide 34: Madison Avenue NewYork, NY 10022 (212) 980-9155 www.tonixpharma.com - - Do not duplicate or
NASDAQ: TNXP 509 Madison Avenue NewYork, NY 10022 (212) 980-9155 www.tonixpharma.com © Copyright 2016 Tonix Pharmaceuticals - Confidential - Do not duplicate or distribute
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