Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (April 2014)
27 slides from Tonix Pharmaceuticals's investor presentation, Apr 1, 2014
Slide 1: Investor Presentation April 2014
Investor Presentation April 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: Fibromyalgia Phase 2b/3 trial underway; clinical-stage pipeline
TONIX PHARMACEUTICALS 3 CONFIDENTIAL TONIX PHARMACEUTICALS Fibromyalgia: Phase 2b/3 trial of TNX - 102 SL underway (BESTFIT) Top line results to be reported in 4Q 2014 Strong evidence of clinical benefit in Phase 2a $ 1.5B U.S. market; 5M patients in U.S.; large unmet need Clinical - stage pipeline
Slide 4: Pipeline by candidate and indication
TONIX PHARMACEUTICALS 4 CONFIDENTIAL TONIX PHARMACEUTICALS Preclinical Phase 1 Proof - of - Concept Pivotal NDA Market Candidate Indication POC = Proof - of - Concept TNX - 102 SL is an Investigational New Drug and is not approved for any indication Phase 2a Phase 2b/3 PTSD TNX - 102 SL 2018E To ent
Slide 5: Repurposed, reformulated and single-isomer drug strategy
TONIX PHARMACEUTICALS 5 CONFIDENTIAL TONIX PHARMACEUTICALS Active Ingredient Safety Risk to Develop Cost to Develop Time to Develop New Unknown Higher Higher Longer Repurposed Reformulated Single - Isomer Known Lower Lower Shorter New Drugs Face high hurdles on demonstrating safety Many target niche
Slide 6: Repurposing, single-isomer and reformulating examples
TONIX PHARMACEUTICALS 6 CONFIDENTIAL TONIX PHARMACEUTICALS 1x / day 3x / day cremaphore a lbumin particle omeprazole esomeprazole citalopram escitalopram m ultiple sclerosis p soriasis t raveler’s d iarrhea hepatic encephalopathy Repurposing Single - Isomer Reformulating
Slide 7: Fibromyalgia U.S. market and approved products
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 SNRI = Serotonin - Norepinephrine Reup
Slide 8: Change in pain with approved treatments
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 Normalized e
Slide 9: Fibromyalgia patients remain unsatisfied
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: Healthcare resource utilization and productivity in fibromyalgia
TONIX PHARMACEUTICALS 10 CONFIDENTIAL TONIX PHARMACEUTICALS * Robinson et al, Pain Med. 2012; 13 :1366. 10 Resource utilization over preceding 12 months Outpatient visits 82.9 % Any emergency room visit 40.2 % Mean number of emergency room visits † 2.4 Productivity measures over preceding 12 months
Slide 11: Poor sleep quality in fibromyalgia
TONIX PHARMACEUTICALS 11 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 12: Phase 2a study design
TONIX PHARMACEUTICALS 12 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 13: Phase 2a pain and sleep quality results
TONIX PHARMACEUTICALS 13 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% Mean TNX - 102 dose at trial end = 3.5 mg 26% reduction in pain vs. placebo Improvement in objective s
Slide 14: Phase 2a safety and adverse events
TONIX PHARMACEUTICALS 14 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, % (N=18) Placebo, % (N=18) Any adverse event 83 83 Heada
Slide 15: SL sublingual tablet formulation
TONIX PHARMACEUTICALS 15 CONFIDENTIAL TONIX PHARMACEUTICALS 15 t ransmucosal absorption • efficacy • tolerability/safety • chronic use • bedtime therapy • compliance • metabolism Optimized for: TNX - 102 SL (sublingual) tablets Phase 2b/3 10x faster absorption* * Absorption lag time ( t lag ) based
Slide 16: TNX-102 SL product profile
TONIX PHARMACEUTICALS 16 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 17: FDA path and remaining clinical work
TONIX PHARMACEUTICALS 17 CONFIDENTIAL TONIX PHARMACEUTICALS 17 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 18: BESTFIT trial design
TONIX PHARMACEUTICALS 18 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 19: Overlap between PTSD and fibromyalgia
TONIX PHARMACEUTICALS 19 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 20: Tension headache and TNX-201
TONIX PHARMACEUTICALS 20 CONFIDENTIAL TONIX PHARMACEUTICALS Affects >20% of the global adult population* Constant band of pressure on the back/sides of head; “squeezed in a vice” feeling Over - the - counter medications are inadequate for many The only prescription medications approved for tension h
Slide 21: Intellectual property
TONIX PHARMACEUTICALS 21 CONFIDENTIAL TONIX PHARMACEUTICALS 21 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 22: Corporate and clinical milestones
TONIX PHARMACEUTICALS 22 CONFIDENTIAL TONIX PHARMACEUTICALS Corporate □ Jan 2014 – $40.7 million net proceeds from common stock offering TNX - 102 SL □ 3Q 2013 – Began BESTFIT trial in FM □ 4Q 2013 – Began open - label extension study in FM □ 3Q 2014 – Start clinical trial in PTSD □ 4Q 2014 – Report
Slide 23: Management team
TONIX PHARMACEUTICALS 23 CONFIDENTIAL TONIX PHARMACEUTICALS Bruce Daugherty, PhD CSO Seth Lederman, MD CEO Leland Gershell, MD, PhD CFO Don Kellerman, PharmD SVP, Clinical Development & Regulatory Affairs
Slide 24: Board of directors
TONIX PHARMACEUTICALS 24 CONFIDENTIAL TONIX PHARMACEUTICALS 24 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 25: Cash and capitalization
TONIX PHARMACEUTICALS 25 CONFIDENTIAL TONIX PHARMACEUTICALS 25 NASDAQ: TNXP Cash reported at December 31, 2013 $ 8.2 million Proceeds from warrant exercises* $ 4.8 million Net proceeds from common stock offering** $ 40.7 million Net cash used in operations in 2013 $ 8.5 million Shares outstanding †
Slide 26: Summary of investment highlights
TONIX PHARMACEUTICALS 26 CONFIDENTIAL TONIX PHARMACEUTICALS 26 • 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 • Fibromyalgia is a current focus of the FDA •
Slide 27: Tonix Pharmaceuticals
PHARMACEUTICALS
Tonix Pharmaceuticals Holding — Investor Presentation (April 2014) on 6ix