Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (October 2012)
23 slides from Tonix Pharmaceuticals's investor presentation, Oct 26, 2012
Slide 1: Tonix Pharmaceuticals Corporate Presentation
TONIX N PHARMACEUTICALS Corporate Presentation October 2012 OTC/QB: TNXP
Slide 2: 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 identified by the use of forward-looking words
Slide 3: Developing novel medications for challenging CNS disorders
° Developing novel medications for challenging disorders of the central nervous system (CNS) - Large and underserved indications * Pivotal trial in fibromyalgia (FM) to report in 2013 - Phase 2 data demonstrated efficacy - Unique, non-addictive treatment approach — targeting sleep quality ° Capital-
Slide 4: Management team
Seth Lederman, MD CEO & Chairman Leland Gershell, MD, PhD CFO Bruce Daugherty, PhD, MBA Senior Director of Drug Development Vela Targent Validus Fontus Cowen Apothecary Capital Favus Research Madison Williams Merck Roche Institute i Fusilev’ (fevoleucovorin) for injection Ny Zolinza [vorinostat] cap
Slide 5: Board of directors
Seth Lederman, MD Chairman Stuart Davidson Patrick Grace Donald Landry, MD, PhD Ernest Mario, PhD Charles Mather John Rhodes Samuel Saks, MD Vela Targent Validus/Fontus Alkermes Combion. WR Grace Chemed Grace Institute Columbia University Chair, Dept. of Medicine Vela Glaxo Alza Reliant Janney Montg
Slide 6: Pipeline: TNX-102 SL, TNX-201, TNX-301
TNX-102 SL_ Fibromyalgia TNX-201 TNX-301 PTSD (Post-Traumatic Stress Disorder) Headache Alcoholism Cyclobenzaprine (CBP) in sublingual formulation First pivotal trial to begin 10.2013 Topline results in 4Q 2013 Cyclobenzaprine in sublingual formulation Proof of concept data in 2013 Seeking U.S. Depa
Slide 7: Chronic pain syndrome
° Chronic pain syndrome - Central pain — originates in the CNS - Despite three FDA-approved medications, patients are dissatisfied Complaint: “Hurt all over, can’t sleep” - No benefit from opiates or prescription sleep drugs FDA primary endpoint is pain Problem with sleep quality - Restorative sleep
Slide 8: ~5 million U.S. patients
° ~5 million U.S. patients* U.S. prescription drug market in 2011 ~$1.4 billion** - 2007-2011 CAGR of 17%** First approved drug for fibromyalgia in 2007 Lyrica® Pfizer 2007 $450 million Cymbalta® Eli Lilly 2008 $560 million Savella® Forest 2009 $137 million Market growth driven by on-label drugs rep
Slide 9: Fibromyalgia presents a significant economic burden
¢ Fibromyalgia presents a significant economic burden - Studies show high cost in overall care, lost productivity, and disability ¢ Physicians and payors are aware of high unmet need in pharmacological treatment of fibromyalgia - Patients take many products without evidence of efficacy ¢ All FDA-app
Slide 10: Legacy off-label and in-development treatments
Legacy Off-Label * cyclobenzaprine | * muscle relaxants Sleep * sodium oxybate (Xyrem®)* * gabapentin Pain * opioids + venlafaxine Mood fy a * bupropion In Development * TNX-102 SL (Phase 3 ready) * Prescribed off-label for treatment-refractory patients, dispensing controlled by central mail-order p
Slide 11: TNX-102 SL will be a first-in-class FDA-approved medication
¢ TNX-102 SL will be a first-in-class FDA-approved medication targeting sleep quality for the “management of FM” ¢ By targeting sleep quality, Rekinla demonstrated powerful efficacy in both Phase 3 studies (p<0.001) Sleep eotts No approved <--— Rekinla D> medications ~---" Phase 3 Ready Sublingual C
Slide 12: Phase 2 study design
Double blind, randomized, placebo controlled e 36 fibromyalgia patients; 18 per arm - Very low dose cyclobenzaprine (VLDC) or placebo taken between dinner and bedtime daily Eight-week, dose escalating study, from 1 mg to 4 mg - Average bedtime cyclobenzaprine dose at week eight was 3.1 mg - Lowest a
Slide 13: Change from baseline at week eight
° Change from baseline at week eight Percent Change from Baseline 15% 10% + 5% 5 0% + 5% | -10% 5 -15% 5 -20% -25% -30% 5 -35% HAD HAD Depression p=0.039 p=0.017 p=0.006 VLDC m Placebo
Slide 14: Not a controlled substance
¢ Not a controlled substance ¢ No recognized addictive potential 1977 * Flexeril® (Merck) FDA approved for muscle spasm 1990's * Extensive safety and efficacy studies 1994 * Publication of a randomized, double-blind, placebo-controlled, six-month clinical trial of daily cyclobenzaprine in FM* 2007 *
Slide 15: Current regimens poorly suited for FM
¢ Current daytime regimens poorly suited for FM - Chronic daytime cyclobenzaprine may confound long-term efficacy ¢ Current doses poorly suited for bedtime use - Off-label use of available doses of cyclobenzaprine at bedtime associated with next-morning grogginess ¢ Current formulations poorly suite
Slide 16: Chronic bedtime dosing
¢ Chronic bedtime dosing - Drug exposure during the night to target non-restorative sleep ¢ Lower dose than available CBP tablets - Tailored to reduce next-morning grogginess ¢ Proprietary sublingual formulation - Transmucosal delivery - Rapid systemic absorption demonstrated in humans - Avoids “fir
Slide 17: Faster absorption
¢ Faster absorption ¢ Bypasses liver “first-pass” metabolism Swallowed Oral Dose Sublingual Oral Dose j) Cyclobenzaprine " Cyclobenzaprine ‘ mh 7 A l l OO veleoten oto OC Noreycloberizaprine Absorption 480 Liver 4 Metabolism To feces a
Slide 18: First Phase 3 efficacy trial to begin in 1Q 2013
¢ First Phase 3 efficacy trial to begin in 1Q 2013 - Randomized, double blind, placebo controlled, 76 patients; 8-10 U.S. centers - 12-week treatment period, daily bedtime dosing - Pre-defined efficacy endpoint = pain (Visual Analog Scale) - Topline results expected by YE 2013 ¢ Subsequent requireme
Slide 19: PTSD program
Patients experience disturbed sleep and widespread pain - Painkiller abuse and addiction is common 3.5% of U.S. adult population has suffered from PTSD in past 12 months* - Experiencing any trauma can lead to PTSD Unsatisfied market - Only Zoloft® and Paxil® have FDA approval Phase 2 proof-of-concep
Slide 20: Milestones
Milestones Related to Fibromyalgia 4Q 2012 ¢ Manufacture commercial tablets 1Q 2013 * Commence first pivotal trial * Topline results from first pivotal trial * Evaluate partnership opportunities Milestones Related to PTSD 1H 2013 * Commence proof of concept study in PTSD patients 4Q 2013
Slide 21: Intellectual property
¢ Pharmacokinetics (PK) - Patent filed around unique PK profile * Surprising and unexpected observations © Protection expected through 2033 - Difficult patent class to circumvent ¢ Method of Use - FM: issued patent, expiration mid-2021 - PTSD: patent filed in 2010 ¢ Composition of Matter - Patent in
Slide 22: Developing novel medications for challenging CNS disorders
° Developing novel medications for challenging disorders of the central nervous system - Large and underserved indications ¢ Pivotal trial in fibromyalgia to report in 2013 - Phase 2 data demonstrated efficacy - Unique, non-addictive treatment approach — targeting sleep quality ° Capital-efficient s
Slide 23: Pharmaceuticals
PHARMACEUTICALS OTC/QB: TNXP
Tonix Pharmaceuticals Holding — Investor Presentation (October 2012) on 6ix