Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (November 2016)
30 slides from Tonix Pharmaceuticals's investor presentation, Nov 1, 2016
Slide 1: Investor Presentation
Investor Presentation TONIX PHARMACEUTICALS November 2016 Version P0038 11-01-16
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 b
Slide 3: Posttraumatic Stress Disorder (PTSD) program
Posttraumatic Stress Disorder (PTSD) program On May 19, 2016, we reported encouraging topline data for TNX-102 SL* 5.6 mg ina military-related PTSD trial (“AtEase”) PTSD was our “second” clinical program using our proprietary formulation TNX-102 SL Prior to the AtEase trial, no other investigational
Slide 4: Tonix (TNXP): Value proposition
Tonix (TNXP): Value proposition On September 6, 2016 our lead Phase 3 program TNX-102 SL for fibromyalgia narrowly missed its primary endpoint in the first Phase 3 study (“AFFIRM”) + Received strong negative investor market response + Reassuring safety profile and activity of TNX-102 SL at 2.8 mg fo
Slide 5: Pivot to PTSD: Rationale
© Pivot to PTSD: Rationale Unmet Medical need + PTSD is a serious condition and the prevalence is increasing, especially combat-related + Military-related PTSD is not satisfactorily treated by existing FDA approved therapies + Two selective serotonin reuptake inhibitors (SSRIs) are approved for PTSD
Slide 6: Highlights of Tonix Pharmaceuticals' Lead Asset
Highlights of Tonix Pharmaceuticals' Lead Asset ‘TNX-102 SL (cyclobenzaprine HCI sublingual tablets) + Aunique, innovative product designed for CNS indication(s) Phase 3 + Targeting a chronic and serious psychiatric disorder: PTSD Ready v Therapeutic dose identified in Phase 2 study Program v Phase
Slide 7: What is PTSD?
What is PTSD? A chronic response to traumatic event(s) + A majority of people will experience a traumatic event at some point in their lifetime + 20% of women and 8% of men in the U.S. who experience significant trauma develop PTSD* + Lifetime prevalence: 2 (~ 17 million adults in the U.S.) + Persis
Slide 8: What Are the Symptoms of PTSD?
What Are the Symptoms of PTSD? ‘Symptoms of PTSD fall into four clusters: Intrusion (aversive memories, nightmares, flashbacks) Avoidance (avoiding persons, places or situations) Mood/cognitions (memory block, emotional numbing, detachment from others) Hyperarousal (anxiety, agitation & sleep distur
Slide 9: What Are the Consequences of PTSD?
What Are the Consequences of PTSD? Consequences: + Impaired daily function and substantial interference with work and social interactions + Reckless or destructive behavior + Increased heath care utilization and greater medical morbidity PTSD as a Risk Factor for: + Depression + Alcohol or substance
Slide 10: PTSD Prevalence and Market Characteristics
PTSD Prevalence and Market Characteristics Prevalent Population (U.S.) million* Rx 510,000 6% Veterans Nor Treated 153500 inY®* Diagnosed population = Undiagnosed 2% Large population (~1.8 million) Majority receive drug treatment Re Civilians: ~75%2 \ inna Veterans: ~80%* Civilians No Rx Population?
Slide 11: What Drug Classes are Used to Treat PTSD?
What Drug Classes are Used to Treat PTSD? il es being the largest class (but contraindicated*) Market highly fragmented, with benzodiazey Multiple medications per patient (or “Polypharmacy”) is the Approximately 55% of patients recelve a benzodiazepine, and 53% receive an SSRI + SSRIs are the only F
Slide 12: Relevance of Sleep Disturbances for PTSD
Relevance of Sleep Disturbances for PTSD Sleep Disturbances: + Core symptom of PTSD (component of intrusion and hyperarousal symptom clusters) + Believed to play roles in the pathophysiology of PTSD Pharmacological | Therapeutic Pathophysiology ‘Action Benefit 2° Clinical 1° Clinical Endpoint Endpoi
Slide 13: TNX-102 SL: Innovative and Unique By Design
© TNX-102 SL: Innovative and Unique By Design antidepressants (TCAs) tricyclic + TCAsand widely pt aie ie histamine ‘a + Major metabolite, norcyclobenzaprine Tacs) + Long half-life (~72 hours) : ic, histamine Hy) + __ More selective for norepinephrine ieee formulation 1, Rudorfer and Potter, Celular
Slide 14: Why Initially Target Military-Related PTSD?
Why Initially Target Military-Related PTSD? Military-related PTSD not well-served by existing FDA-approved therapies Sancll igs ellseheocies cies) full ilsteaisii HIS. allay @secom anita Beta Paroxetine: no largi Sertraline: FDA-conducted post-hoc analysis concluded no effect for male civilian subgr
Slide 15: Phase 2 AtEase Study in Military PTSD
(Phase 2 AtEase Study in Military PTSD ‘TNX-102 SL at bedtime once-daily + Randomized, double-blind, placebo- 2.8 mg controlled trial in military-related PTSD Analysis from 231 patients; ‘TNX-102 SL at bedtime once-daily 24 U.S. clinical sites 5.6 mg (2 x 2.8 mg) 4 ies i USD) - Primary efficacy anal
Slide 16: AtEase Study Results CAPS-5 Total Score
AtEase Study Results CAPS-5 Total Score CAPS-5 LS Total Score Mean Change from Baseline Wko = Wk2 = Wk4 = Wk6)— Wk) Wk10— Wk 2 Ds 5 a 5, Placebo & =-TNX-102 SL 2.8 mg 8 —TNX-102 SL 5.6 mg Z as * y : 5.0 point aiference a CAPSS 20 fect Size = 0.26 (MMR * 25 *p=0.031, comparing placebo and TNX-102 SL
Slide 17: AtEase Study Results CAPS-5 Sleep Disturbance
© AtEase Study Results CAPS-5 Sleep Disturbance Change from baseline Wko Wk2 Wké = Wk6 WKB Wk10. Wka2 i Placebo 5 * “=-TNx-102 SL 2.8 mg ” : —-TNx-102 SL 5.6mg 3 a2 * 1s ¥ 0.05, 5.6 ma v. placebo, MMR % 0.05, 2.8 mg v. placebo, MMRM.
Slide 18: 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 Placebo TNX-102SL2.8mg _TNX-102 SL5.6 mg ) (N=50) Systemic Adverse Events* (Nooay ‘Somnolence 6.4% 11.8% 16.0% Dry Mouth 10.6% 4.3% 16.0% Headache 4.3% 5.4% 12.0% Insomnia 8
Slide 19: AtEase: Retrospective Analysis
AtEase: Retrospective Analysis Prior pharmacotherapy trials in PTSD used earlier versions of CAPS (e.g. CAPS-2) + Different scoring range from CAPS-S Most frequently used a score of > 50 for entry (similar to CAPS-5 > 33%) + FDA has accepted this higher entry criteria (CAPS-5 = 33) for Phase 3 progr
Slide 20: Analysis Using CAPS-5 As Entry Criteria
© Analysis Using CAPS-5 2 33 As Entry Criteria Retrospective subset analysis of patients with entry CAPS-5 = 33 WkO = Wk2 0 Wk4 Wk6 WKB Wk. Wk 12 0.0 Placebo “5.0 -S-TNX-102 SL2.8 mg A TNX-102 SL5.6 mg -10.0 LS Mean (SE) 20.0 * 6.8 point difference in CAPS-5 ¢ e seling 25.0 * * 30.0 * 80.025, compar
Slide 21: Phase 2 AtEase Study Conclusions
Phase 2 AtEase Study Conclusions First large multi trial efficacy of an New Drug (IND) in military-related PTSD ¥ TNX-102 SL therapeutic dose (5.6 mg) identified ¥ Symptom reduction (CAPS-5) ¥ Functional improvement (Sheehan Disability Scale domains) ¥ Clinical global impression of improvement (CGI-
Slide 22: Planned Phase 3 program in PTSD
Planned Phase 3 program in PTSD To confirm AtEase fins litary-related PTSD: Larger adaptive design study + Targeting start in 1Q 2017 TNX-102 SL once-daily at bedtime 5.6mg N~ 275 (90%) Placebo once-daily at bedtime N ~ 275 (90%) 22 weeks ——> * First interim analysis © 2016 Tonix Pharmaceuticals Ho
Slide 23: Commercialization Options
Commercialization Options Tonix is exploring a variety of options to commercialize TNX-102 SL, including commercializing on our own or partnering all or some indications in specific regions of the world. Commercial Considerations: + Primary physician audience is well defined: psychiatrists (~30,000
Slide 24: Intellectual Property
Intellectual Property TNX-102 SL PTSD Portfolio of international patents filed under the Patent Cooperation Treaty (PCT) Composition-of-matter (eutectic) + Patents filed + Protection expected to 2034 Pharmacokinetics (PK) + Patents filed + Protection expected to 2033 Method-of-use + PTSD: patents fi
Slide 25: Management Team
Management Team Seth Lederman, MD President & CEO TARGENT~ &Fusiley vela. Bruce Daugherty, PhD, MBA Chief Scientific Officer & merck ivan, MD Chief Medical Officer coumativere New York State We perme fPcany Beha Bradley Saenger, CPA Chief Financial Officer Jessica Morris EVP, Operations hire vite al
Slide 26: 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 Ha
Slide 27: Financial overview
Financial overview NASDAQ: TNXP Cash, cash equivalents, and marketable securities reported at June 30, 2016 $31.2 million Net proceeds from overallotment in 3Q16 $1.4 million Net Proceeds from At-the-market offering (ATM) in 3Q16 $2.5. million Net proceeds from underwritten offering in 4Q16 $4.6 mil
Slide 28: Milestones - recent and upcoming
Milestones - recent and upcoming INX-102 SL = Posttraumatic Stress Disorder W December 2015 _ Entered into Collaborative Research and Development Agreement (CRADA) with the United States Army Medical Materiel Development Activity (USAMMDA) & May 2016 Report results from AtEase study W& August 2016 E
Slide 29: Summary
Summary Strong position for value growth with Phase 3 trial in a major medical indication: : hose ° asset not previously well-known to the investor marketplace Funded through 1* interim analysis (180 patients) of Phase 3 PTSD trial initiating in 192017 + Topline data from 1+ interim analysis expecte
Slide 30: Thank you!
TONIX PHARMACEUTICALS NASDAQ: TNXP Thank you! {© 2016 Tonix Pharmaceuticals Holding Corp.
Tonix Pharmaceuticals Holding — Investor Presentation (November 2016) on 6ix