Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Investor Presentation (February 2014)
27 slides from Tonix Pharmaceuticals's investor presentation, Feb 10, 2014
Slide 1: Investor Presentation February 2014
Investor Presentation February 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: Cash and capital summary
TONIX PHARMACEUTICALS 3 CONFIDENTIAL TONIX PHARMACEUTICALS 3 NASDAQ: TNXP Cash reported at September 30, 2013 $ 7.4 million Proceeds from warrant exercises* $ 8.6 million Net proceeds from common stock offering** $ 40.7 million Net operating cash burn (January 1 – September 30, 2013) $ 5.0 million S
Slide 4: Fibromyalgia Phase 2b/3 trial underway; clinical-stage pipeline
TONIX PHARMACEUTICALS 4 CONFIDENTIAL TONIX PHARMACEUTICALS Fibromyalgia: Phase 2b/3 trial of TNX - 102 SL underway Top line results to be reported in 2H 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 Post - tra
Slide 5: Development pipeline by candidate and indication
TONIX PHARMACEUTICALS 5 CONFIDENTIAL TONIX PHARMACEUTICALS Preclinical Phase 1 Proof - of - Concept Pivotal NDA Market Candidate Indication T NX - 102 SL is an Investigational New Drug and is not approved for any indication . Phase 2a Phase 2b/3 PTSD TNX - 102 SL 2018E Phase 2 to start 3Q14 TNX - 10
Slide 6: Repurposed, reformulated and pure-isomer drug strategy
TONIX PHARMACEUTICALS 6 CONFIDENTIAL TONIX PHARMACEUTICALS Active Ingredient Safety Risk to Develop Cost to Develop Time to Develop New Unknown Higher Higher Longer Repurposed Reformulated Pure - Isomer Known Lower Lower Shorter New Drugs Face high hurdles on demonstrating safety Many target niche m
Slide 7: Repurposing, pure-isomer and reformulating examples
TONIX PHARMACEUTICALS 7 CONFIDENTIAL TONIX PHARMACEUTICALS 1x / day 3x / day allergenic solvent a lbumin particle omeprazole esomeprazole citalopram escitalopram m ultiple sclerosis p soriasis t raveler’s d iarrhea hepatic encephalopathy Repurposing Pure - Isomer Reformulating
Slide 8: Fibromyalgia market and approved products
TONIX PHARMACEUTICALS 8 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 9: Change in pain with approved treatments
TONIX PHARMACEUTICALS 9 CONFIDENTIAL TONIX PHARMACEUTICALS 9 * 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 10: Fibromyalgia unmet need
TONIX PHARMACEUTICALS 10 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
Slide 11: Healthcare resource utilization and productivity burden
TONIX PHARMACEUTICALS 11 CONFIDENTIAL TONIX PHARMACEUTICALS * Robinson et al, Pain Med. 2012; 13 :1366. 11 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 12: Poor sleep quality in fibromyalgia
TONIX PHARMACEUTICALS 12 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 13: Phase 2a study design with cyclobenzaprine
TONIX PHARMACEUTICALS 13 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 14: Phase 2a pain and sleep quality results
TONIX PHARMACEUTICALS 14 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 15: Phase 2a safety and adverse events
TONIX PHARMACEUTICALS 15 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 16: Sublingual tablet formulation
TONIX PHARMACEUTICALS 16 CONFIDENTIAL TONIX PHARMACEUTICALS 16 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 17: TNX-102 SL product profile
TONIX PHARMACEUTICALS 17 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 18: FDA path and remaining clinical work
TONIX PHARMACEUTICALS 18 CONFIDENTIAL TONIX PHARMACEUTICALS 18 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 19: BESTFIT Phase 2b/3 trial design
TONIX PHARMACEUTICALS 19 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 20: PTSD program and overlap with fibromyalgia
TONIX PHARMACEUTICALS 20 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 21: Tension-type headache and TNX-201
TONIX PHARMACEUTICALS 21 CONFIDENTIAL TONIX PHARMACEUTICALS Tension - type Headache Constant band of pressure on the back/sides of head; “squeezed in a vice” feeling Frequent episodic type affects >20% of the adult population* Over - the - counter medications are inadequate for many The only prescri
Slide 22: Intellectual property protection
TONIX PHARMACEUTICALS 22 CONFIDENTIAL TONIX PHARMACEUTICALS 22 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 23: Milestones and upcoming events
TONIX PHARMACEUTICALS 23 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 Phase 2a trial in PTSD □ 2H 2014 – Report
Slide 24: Management team
TONIX PHARMACEUTICALS 24 CONFIDENTIAL TONIX PHARMACEUTICALS Bruce Daugherty, PhD CSO Seth Lederman, MD CEO Leland Gershell, MD, PhD CFO
Slide 25: Board and advisors
TONIX PHARMACEUTICALS 25 CONFIDENTIAL TONIX PHARMACEUTICALS 25 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 26: Summary
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 • Multiple opportunities (fibromyalgia , PTSD,
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Tonix Pharmaceuticals Holding — Investor Presentation (February 2014) on 6ix