Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (January 2014)
30 slides from Tonix Pharmaceuticals's investor presentation, Jan 7, 2014
Slide 1: Investor Presentation January 2014
Investor Presentation January 2014 NASDAQ: TNXP
Slide 2: Forward-looking statements
TONIX PHARMACEUTICALS 2 CONFIDENTIAL TONIX PHARMACEUTICALS 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 sta
Slide 3: Company snapshot
TONIX PHARMACEUTICALS 3 CONFIDENTIAL TONIX PHARMACEUTICALS 3 Ticker TNXP Exchange NASDAQ Capital Market Cash at 9/30/13 $7.4 million Shares outstanding* 5.8 million Year Founded 2007 Independent Directors 7 * As of December 30, 2013
Slide 4: Investment highlights
TONIX PHARMACEUTICALS 4 CONFIDENTIAL TONIX PHARMACEUTICALS Fibromyalgia trial underway for TNX - 102 SL (sublingual cyclobenzaprine) Top line results of Phase 2b/3 trial to be reported in 2H 2014 Anticipated to be the first of two pivotal trials required for FDA approval Strong evidence of clinical
Slide 5: Repurposed and reformulated drug strategy
TONIX PHARMACEUTICALS 5 CONFIDENTIAL TONIX PHARMACEUTICALS Active Ingredient FDA Term Safety Risk to Develop Cost to Develop Time to Develop New 505(b)1 Unknown Higher Higher Longer Repurposed/ Reformulated 505(b)2 Known Lower Lower Shorter New Drugs Face high hurdles for showing safety Many target
Slide 6: Product pipeline
TONIX PHARMACEUTICALS 6 CONFIDENTIAL TONIX PHARMACEUTICALS TNX - 102 SL PTSD Fibromyalgia Preclinical Phase 1 Proof - of - Concept Pivotal NDA Market TNX - 102 SL Candidate Indication TNX - 201 Headache TNX - 301 Alcoholism T NX - 102 SL is an Investigational New Drug and is not approved for any ind
Slide 7: Fibromyalgia market and approved therapies
TONIX PHARMACEUTICALS 7 CONFIDENTIAL TONIX PHARMACEUTICALS * National Institutes of Health, U.S. Department of Health and Human Services ** Robinson et al, Pain 2012; 13 : 1366 - 76. *** Estimates based on information from publicly - available sources ‡ EU only 7 5 million U.S. patients* 2.6 million
Slide 8: Effect sizes of approved FM drugs
TONIX PHARMACEUTICALS 8 CONFIDENTIAL TONIX PHARMACEUTICALS 8 * Hauser et al., J. Pain. June 2010 ** Effect sizes normalized based on Cohen methodology: Cohen J, "A power primer". Psychological Bulletin 112 (1): 155 – 159 . - 0.27 - 0.33 - 0.19 -0.35 -0.30 -0.25 -0.20 -0.15 -0.10 -0.05 0.00 Normalize
Slide 9: Fibromyalgia unmet need
TONIX PHARMACEUTICALS 9 CONFIDENTIAL TONIX PHARMACEUTICALS Patients feel pain all over the body, but it originates in the brain Chronic, widespread pain with sleep, fatigue, mood, and memory problems Impairs daily function and productivity: poor quality of life Predominantly female Patients remain u
Slide 10: Sleep quality and fibromyalgia
TONIX PHARMACEUTICALS 10 CONFIDENTIAL TONIX PHARMACEUTICALS >90% of FM patients complain of poor sleep quality* Non - restorative sleep linked to hyper - vigilance Restorative sleep improves FM symptoms Sleep quality of FM patients can be objectively measured: Cyclic Alternating P attern (CAP) A1 pa
Slide 11: Phase 2a study design
TONIX PHARMACEUTICALS 11 CONFIDENTIAL TONIX PHARMACEUTICALS Double - blind, randomized, placebo - controlled Conducted at two academic centers in Canada Enrolled 36 subjects with fibromyalgia; 18 per arm TNX - 102 capsules or placebo taken between dinner and bedtime daily Eight - week, dose - escala
Slide 12: Phase 2a pain and sleep results
TONIX PHARMACEUTICALS 12 CONFIDENTIAL TONIX PHARMACEUTICALS -30 -25 -20 -15 -10 -5 0 % Change from Baseline at Week 8 Pain TNX - 102 Placebo † Improving at least one night of CAP A2+A3(norm) ≤ 3 3% 26% reduction in pain vs. placebo Improvement in objective sleep quality measures vs. placebo * 0 10 2
Slide 13: Phase 2a safety and adverse events
TONIX PHARMACEUTICALS 13 CONFIDENTIAL TONIX PHARMACEUTICALS No serious adverse events No discontinuations due to adverse events in treatment arm Types of adverse events consistent with approved cyclobenzaprine products Adverse Event TNX - 102, % Placebo, % Any adverse event 83 83 Headache 39 17 Dry
Slide 14: SL sublingual tablet formulation
TONIX PHARMACEUTICALS 14 CONFIDENTIAL TONIX PHARMACEUTICALS 14 t ransmucosal absorption • efficacy • tolerability/safety • chronic use • bedtime therapy • compliance • metabolism 3 0 minutes 2 hours Optimized for: TNX - 102 SL (sublingual) tablets Phase 2b/3
Slide 15: SL product profile
TONIX PHARMACEUTICALS 15 CONFIDENTIAL TONIX PHARMACEUTICALS Targets pain and poor sleep U nique mechanism of action among marketed FM products Sublingual tablet at bedtime Fast onset aligns exposure with sleeping period Designed to optimize ease - of - use, compliance Very low dose – 2.8 mg per day
Slide 16: FDA path and remaining clinical work
TONIX PHARMACEUTICALS 16 CONFIDENTIAL TONIX PHARMACEUTICALS 16 Pre - Phase 3 meeting held with FDA in February 2013 Remaining clinical work to support New Drug Application: Two adequate and well - controlled efficacy and safety trials in FM patients Primary efficacy endpoint = pain □ First trial is
Slide 17: BESTFIT Phase 2b/3 trial design
TONIX PHARMACEUTICALS 17 CONFIDENTIAL TONIX PHARMACEUTICALS BESTFIT : BE dtime S ublingual T NX - 102 SL as F ibromyalgia I ntervention T herapy Randomized, double - blind, placebo - controlled; 13 - 15 U.S. sites Primary efficacy endpoint = change in pain at week 12 vs. baseline (Numeric Rating Sca
Slide 18: PTSD and overlap with fibromyalgia
TONIX PHARMACEUTICALS 18 TONIX PHARMACEUTICALS Overlap between PTSD and FM ~50% of FM or PTSD patients meet criteria for the other disorder Patients experience disturbed sleep and widespread pain Core defining feature is hyper - vigilance – can disturb sleep Painkiller abuse and addiction are common
Slide 19: TNX-201 headache and TNX-301 alcoholism
TONIX PHARMACEUTICALS 19 CONFIDENTIAL TONIX PHARMACEUTICALS TNX - 201 (pure isomer isometheptene ) for tension - type headache Isometheptene has been used in the U.S. for >50 years as a treatment for headache, but is not FDA approved for any indication* Limited availability via compounding pharmacie
Slide 20: Intellectual property
TONIX PHARMACEUTICALS 20 CONFIDENTIAL TONIX PHARMACEUTICALS 20 Pharmacokinetics (PK) Patents filed around unique PK profile Protection expected to 2033 Composition - of - matter Patent filed - “Eutectic” Protection expected to 2034 Method - of - use FM: patent issued, 3Q 2020 expiry PTSD: patent fil
Slide 21: Milestones
TONIX PHARMACEUTICALS 21 CONFIDENTIAL TONIX PHARMACEUTICALS Corporate □ 8/9/13 – TNXP stock uplisted to NASDAQ □ 8/14/13 – Gross proceeds of $11.4 million from underwritten offering TNX - 102 SL (FM) □ 3Q 2013 – Began Phase 2b/3 trial in FM □ 4Q 2013 – Began open - label extension study in FM □ 2H 2
Slide 22: Management team
TONIX PHARMACEUTICALS 22 CONFIDENTIAL TONIX PHARMACEUTICALS Bruce Daugherty, PhD CSO Seth Lederman, MD CEO Leland Gershell, MD, PhD CFO
Slide 23: Board of directors
TONIX PHARMACEUTICALS 23 CONFIDENTIAL TONIX PHARMACEUTICALS 23 Samuel Saks, MD Ernest Mario, PhD Stuart Davidson Alkermes Combion Charles Mather Janney Montgomery Scott Cowen, Smith Barney Patrick Grace WR Grace Chemed John Rhodes NYSERDA, NRDC Booz Allen Hamilton Donald Landry, MD, PhD Chair, Depar
Slide 24: Summary
TONIX PHARMACEUTICALS 24 CONFIDENTIAL TONIX PHARMACEUTICALS 24 • TNX - 102 SL: late - stage clinical program in large market indication • Strong evidence of clinical benefit in Phase 2a • Active ingredient has established safety profile at higher doses • Multiple opportunities (fibromyalgia , PTSD,
Slide 25: Appendix
TONIX PHARMACEUTICALS 25 CONFIDENTIAL TONIX PHARMACEUTICALS Appendix
Slide 26: Effects of cyclobenzaprine
TONIX PHARMACEUTICALS 26 U ntreated Effects of TNX - 102 SL Cyclobenzaprine is a multi - functional drug - inhibits serotonin and norepinephrine reuptake - blocks serotonin 5HT 2a and norepinephrine α 1 receptors SNARI = S erotonin and N orepinephrine receptor A ntagonist and R euptake I nhibitor
Slide 27: SL trazodone and prazosin
TONIX PHARMACEUTICALS 27 TNX - 102 SL Trazodone Trazodone (disordered sleep), Prazosin (night terrors) - Trazodone inhibits serotonin 5HT 2a receptors and serotonin reuptake (SARI) - Prazosin blocks norepinephrine α 1 receptors Prazosin SARI – S erotonin Receptor A ntagonist & R euptake I nhibitor (
Slide 28: Known drugs developed for new conditions
TONIX PHARMACEUTICALS 28 CONFIDENTIAL TONIX PHARMACEUTICALS Medicis Celgene Corporation Jazz Pharmaceuticals myeloma b acterial infection acne morning sickness narcolepsy conditions Revlimid ® sales of $ 3.7B in 2012 Acquired by Valeant for $2.6B Xyrem ® sales of $380M in 2012 Tonix Pharmaceuticals
Slide 29: Reformulated drugs and acquisitions
TONIX PHARMACEUTICALS 29 CONFIDENTIAL TONIX PHARMACEUTICALS MAP Pharmaceuticals Alza Corporation New River Pharmaceuticals Migranal ® Levadex ® Dexedrine ® Vyvanse ® Tonix Pharmaceuticals Ritalin ® Concerta ® Acquired by J&J - $11 B Acquired by Allergan - $958 M Acquired by Shire - $2.6 B Known drug
Slide 30: PHARMACEUTICALS
PHARMACEUTICALS
Tonix Pharmaceuticals Holding — Investor Presentation (January 2014) on 6ix