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Opexa Therapeutics, Inc.
NASDAQ: OPXA
Precision Immunotherapy
February 2016
The Woodlands, TX
Precision Immunotherapy®
2
Forward-Looking Statements
All statements in this presentation other than those of historical fact, including statements regarding our preclinical and
clinical development plans for Tcelna® and OPX-212, our research and other development programs, our ability to
undertake certain activities and accomplish certain goals, projected timelines for our research and development
activities and possible regulatory approvals, if any, our expectations regarding the relative benefits of our product
candidates versus competitive therapies, our expectations regarding the possibility of licensing or collaborating with
third parties regarding our product candidates or research, and our expectations regarding the therapeutic and
commercial potential of our product candidates, research, technologies and intellectual property, are forward-looking
statements. The words “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “design,” “intend,” “expect,”
“potential” and similar expressions, as well as the negative version of these words and similar expressions, are intended
to identify forward-looking statements. Our forward-looking statements do not constitute guarantees of future
performance, and are subject to a number of risks and uncertainties that could cause actual results to differ materially
and adversely from those anticipated or implied in such statements. Our forward-looking statements are based upon
our current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual
results and the timing of events could differ materially from those anticipated as a result of various risks and
uncertainties which include, without limitation, risks associated with the process of discovering, developing and
commercializing drugs that are safe and effective for use as human therapeutics and risks inherent in the effort to build
a business around such drugs. Although we believe our expectations are reasonable, we do not in any way guarantee
future results, level of activity, performance or achievements. In addition, neither we nor any other person assumes
responsibility for the accuracy and completeness of any forward-looking statements. Our forward-looking statements in
this presentation speak only as of the date of this presentation. We assume no obligation or undertaking to update or
revise any statements to reflect any changes in our expectations or any change in events, conditions or circumstances on
which any such statement is based. You should, however, review additional disclosures we make that further describe
risks and uncertainties relevant to us in additional detail in our filings with the Securities and Exchange Commission
including our Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. You may get these documents for free
by visiting EDGAR on the SEC web site at http://www.sec.gov.
3
Opexa Investment Highlights
 PIPELINE
• Tcelna®: Phase 2b for secondary progressive multiple sclerosis (SPMS), limited
competition, $7BN overall market potential
• Top line results expected for Abili-T Phase 2b SPMS trial in 2H 2016
• OPX-212: Pre-IND for neuromyelitis optica (NMO), no approved therapies, orphan
indication
 TECHNOLOGY
• Personalized T-cell immunotherapy platform for autoimmune diseases
• Potential to yield multiple candidates tailored to each patient’s disease profile
• Scalable cGMP manufacturing facility
• Strong patent estate (160 domestic and international)
 VALIDATION
• Option agreement secured with Merck Serono for Tcelna – strong potential partner
• FDA Fast Track designation for Tcelna in SPMS
• Esteemed Scientific Advisory Board
OPEXA’S NOVEL APPROACH;
PRIMING THE IMMUNE SYSTEM
4
5
• Numerous autoimmune diseases are treated with immunosuppressive
drugs, despite black box warnings on the labels and significant side
effects
• Global suppression of immune cells increases patient risk, especially
with long term chronic use
• Opexa’s immunotherapy mechanism of action selectively targets only
harmful, pathogenic T-cells leaving the rest of the immune system in
tact
• Favorable benefit:risk profile of Opexa’s immunotherapy should
differentiate Opexa from competitors
Opexa is Positioned to Address High Unmet
Medical Need in Autoimmune Diseases
6
• Opexa’s T-cell immunotherapy aims to restore tolerance
– To prevent the further attack of healthy tissue by activated immune cells
– Reprograming of the immune system to selectively eliminate the
pathogenic T-cells that cause disease
• Maintains the body’s defense mechanisms
– No evidence of global suppression in previous controlled trials
• May lead to stabilization of disease and possibly allow the
body to repair damage caused by disease
– Evidence to support this in previous MS controlled trial
Restoration of Immune Tolerance:
The key to advancing the treatment of autoimmune diseases
7
Root Cause of MS: Activated T-cells Degrade Myelin and
Damage Myelin Producing Cells
Adapted by permission from Macmillan Publishers Ltd: NATURE REVIEWS
IMMUNOLOGY 3, 483-492 (June 2003), copyright (2003)
Cytokines
Damage
Damage
• In MS patients, the faulty immune system is
not able to prevent the attack of a small sub-
population of myelin reactive T-cells (MRTC)
• MRTCs cross the blood brain barrier, enter
the brain, and bind to antigen presenting
cells (APC), causing a release of pro-
inflammatory cytokines which lead to a two
pronged attack through:
1. Destruction of myelin sheath, the protective
coating of nerve fibers
2. Destruction of oligodendroglial cells, which
are responsible for producing myelin
Myelin peptide
Result
Destruction of the myelin sheath and myelin producing cells,
thereby preventing remyelination
8
Tcelna Could Address the Root Cause of MS by
Preventing Demyelination and Enabling Remyelination
Adapted by permission from Macmillan Publishers Ltd: NATURE REVIEWS
IMMUNOLOGY 3, 483-492 (June 2003), copyright (2003)
Opexa’s Strategy
Tcelna programs the immune system to specifically
recognize MRTC as pathogenic, thereby inhibiting further
destruction of the myelin sheath and potentially enabling
remyelination
• Therapeutic dose of Tcelna (attenuated T-cell
clones) is injected subcutaneously
• This triggers an immune response specifically
targeting circulating MRTC
• Immune cells, including Tregs, have been primed,
or sensitized, we believe, to specifically target the
pathogenic MRTC for elimination or regulation
• Elimination of harmful MRTC may lead to:
1. Stabilization of disease by preventing further
destruction of myelin
2. Improvement in condition by allowing
oligodendroglial cells to remyelinate axons
(neuroprotection)
IMMPATH®: OPEXA’S PROPRIETARY
T-CELL IMMUNOTHERAPY PLATFORM
9
10
ImmPath®: Harnessing the Power of the Patient’s
Own Immune System to Restore Tolerance
Administration
5 SC doses/year
Procurement
Dispensation
1 Day
Expansion of
T-cells
Attenuation
• Annual course of treatment generated from a single manufacturing run
• Therapy is personalized annually and is tailored to evolving epitope profile
Formulation &
cryopreservation
Antigen-sourced
seed material
(Blood collection)
Manufacturing and QC
35 Days
TCELNA® FOR MULTIPLE SCLEROSIS
11
12
Opexa Developing Highly Differentiated,
Precision Therapies
OPX-212
Preclinical Phase 1 Phase 2 Phase 3Programs and indications
Neuromyelitis Optica (NMO) IND enabling activities ongoing
Tcelna (imilecleucel-T) *
Research
Secondary Progressive Multiple Sclerosis (SPMS) Phase 2b trial ongoing, data expected 2H 2016
Key Inflection Point:
2H 2016
Top-line Data from Phase 2b Abili-T trial in SPMS expected
13
Tcelna in Secondary Progressive MS
SPMS
450,000 Individuals in North America diagnosed with MS;
30-45 % potentially have an SPMS diagnosis
Market Size:
$7 Billion (est.)
Relapsing Remitting MS;
Clinically Isolated Syndrome;
Primary Progressive MS
• Secondary Progressive MS market
potential in North America could
exceed $7 billion (for all therapies)
• Roughly 150,000 SPMS patients in
North America
• Only one drug approved for SPMS in
U.S. (none in EU or Asia)
- Drug not suitable for chronic use
due to severe side effects
Tcelna being developed to be a
potential therapy of choice in SPMS
14
Tcelna®
Lead Program Targeting Secondary Progressive MS patients
Fast Track Designation by FDA
• Phase 2b is fully enrolled: 190 patients with SPMS
• Top line data expected in 2H 2016
• Design
– Double-blind, 1:1 randomized, placebo-controlled
– 35 clinical sites in USA and Canada
– Two annual courses of personalized therapy
– Efficacy Endpoints: Primary-Whole Brain Atrophy, Secondary-Disease Progression
• Immune Monitoring being conducted in parallel
– Comprehensive biomarker analysis
15
Clinical Evidence for Tcelna MoA
T-cell technology demonstrated a significant reduction in reactive T-cells (52 week study results of MS)
0%
-92%
-87%
-79%
-77%
-65%
-100%
-90%
-80%
-70%
-60%
-50%
-40%
-30%
-20%
-10%
0%
Week 0 Week 5 Week 13 Week 21 Week 28 Week 52
• Results from Tcelna dose ranging studies:
 Published in Clinical Immunology
(2009) 131, 202-215
• Each dose consisting of 30-45 x 106 cells
• % reduction relative to baseline (Week 0)
ReductioninMRTC
Dose 1 Dose 2 Dose 3 Dose 4
MRTC: Myelin Reactive T-cells
16
Phase 2b RRMS Prior Clinical Trial
Tcelna showed 37% improvement over placebo (mITT)
(secondary endpoint measurement, primary MRI endpoint measure not met)
0.214
0.339
0
0.05
0.1
0.15
0.2
0.25
0.3
0.35
0.4
37% Reduction in Annualized Relapse Rate (ARR)
Tcelna
Placebo
n=94 n=48
Relapses/Patient/Year
37%
Total mITT population
n=142
17
Results of Phase 2b TERMS study in RRMS
Reversal of disability in prospective analysis in more active patients
Sub-population of patients (n=50) with more progressed/active disease
profile (baseline ARR >1)
2.4
2.23
2.2
2.39
2.10
2.15
2.20
2.25
2.30
2.35
2.40
2.45
EDSSScore(Mean)
Baseline Week 52
Tcelna Placebo
(p=0.045)
Statistically Significant
Improvement in Disability (p=0.045)
55% Reduction in ARR
Change in Disability (EDSS)
at Week 52
0.28
0.63
0
0.1
0.2
0.3
0.4
0.5
0.6
0.7
Tcelna
Placebo
55%
Relapses/Patient/Year
Annualized Relapse Rate (ARR) at week 52
p=0.07
18
Merck Serono Agreement
Agreement signed 2013; strong potential partner
• Option and License Agreement for worldwide rights to all Multiple
Sclerosis indications, excluding Japan
• If Merck Serono exercises option:
– Merck Serono to fund Phase 3, pre-commercial and commercial activities
– Opexa has potential to receive up to an additional $220 million in option
exercise and milestone payments
– Opexa has potential to receive royalties ranging from 8% to 15% of annual net
sales, with step-ups occurring if net sales exceed $500 million, $1 B & $2 B
• Merck Serono increased their commitment to Opexa’s MS program this
year
– In anticipation of the Phase IIb trial results expected in 2H 2016, Merck
Serono gave Opexa $3 million of support in March 2015 to begin planning for
Phase III studies
19
Financials
Cash and Cash Equivalents (MM) as of September 30, 2015 ~$15.6
Shares outstanding (MM) as of December 1, 2015 ~7.0
Shares issuable for Warrants (MM) outstanding as of September 30, 2015 ~3.7(1)
Shares issuable for Stock Options (MM) outstanding as of September 30, 2015 ~0.4(2)
Debt None
(1) Weighted average exercise price =$6.30 as of September 30, 2015
(2) Weighted average exercise price =$18.11 as of September 30, 2015
LEVERAGING THE T-CELL PLATFORM:
OPX-212 FOR NEUROMYELITIS OPTICA
(NMO)
20
21
NMO: A Rare Disease of the Central Nervous System
• Neuromyelitis optica (NMO) is a rare or orphan
autoimmune disease
• Immune system cells and antibodies mistakenly attack
and destroy myelin cells in the optic nerves and the
spinal cord
• Individuals with NMO develop optic neuritis, which
causes pain in the eye and vision loss, and transverse
myelitis, which causes weakness, numbness, and
sometimes paralysis of the arms and legs
• There are no FDA-approved therapies for NMO
• Affects any age varying from 3 to 90 years, the average
age of onset ~41 years
An orphan disease with no FDA-approved therapy
Image reprinted with permission from MultiView, Inc.
22
Key Operational Milestones Achieved For NMO Program
FDA feedback
• Conducted pre-IND meeting to discuss pre-clinical and clinical program to support OPX-212
development in NMO
Preclinical animal study
• Results show statistically significant dose dependent reduction of AQP4 reactive T-cells in murine
model
• Supportive of MOA
KOL support
• Engaged key thought leaders to validate scientific hypothesis and discuss clinical trial design
– Benjamin Greenberg, M.D., University of Texas, Southwestern Medical Center
– Michael Levy, M.D., Ph.D., The Johns Hopkins Hospital
Patient support
• Collected NMO patient blood samples to conduct pre-IND activities, process development and research
into mechanism of action
23
FDA Pre-IND Meeting Outlined Preclinical Plan
Bioactivity Study Supports Development Of OPX-212
• FDA request: Design a study to demonstrate biological activity in animal model
– No pharm/tox required
• Design: Based on Opexa Phase I study in MS patients
• Hypothesis: Demonstrate reduction in pathogenic
AQP4 T-cells in murine model
• Procedure: Mice were pre-treated with attenuated antigen-specific T-cells and
primed with the NMO autoantigen
• Results: Opexa demonstrated dose dependent and statistically significant
reduction in pathogenic (AQP4) T-cells in murine model
• Conclusion: Bioactivity study supports further development of OPX-212 in
NMO and supports IND
0%
-92%
-87%
-79% -77%
-65%
-100%
-90%
-80%
-70%
-60%
-50%
-40%
-30%
-20%
-10%
0%
Week 0Week 5
Week
13
Week
21
Week
28
Week
52
Dose 1 Dose 2 Dose 3 Dose 4
ReductioninMRTC
24
Experienced Management Team and Board of Directors
Neil Warma, President & CEO, Director
› 19+ years international healthcare experience with large Pharma and emerging
biotechnology companies
› Former Senior Management, Novartis Pharmaceuticals, Basel, Switzerland
› Former CEO, Viron Therapeutics, Inc.
› Co-founder and President of MedExact Inc., a company subsequently acquired
Karthik Radhakrishnan, Chief Financial Officer
› 10+ years of health care capital markets experience
› Formerly, Vice President at ING Investment Management
› MBA, MS in Engineering, CFA charter holder
Don Healey, Ph.D., Chief Scientific Officer
› 25+ years of experience in cellular immunology and immune regulation
› Former Director of Immunology, Argos Therapeutics
Donna Rill, Chief Development Officer
› 30 years in cell and gene therapy research and clinical application
› Designed and validated cGMP Cell & Gene Therapy Laboratories, Vector Production
facilities, and Translational Research Labs
Jason Kralic, Ph.D., VP, Business Development
› Former Head of Business Development for the Neurosciences Therapy Area
Unit at GlaxoSmithKline
› Ph.D. in Pharmacology from University of N. Carolina at Chapel Hill School of
Medicine
Board of Directors
Timothy Barabe
Board member of Arqule, Inc.; Former
CFO of Affymetrix, Human Genome
Sciences, Inc., Regent Medical UK and
Sandoz GmbH
Dr. Hans-Peter Hartung
Chair of Neurology at Heinrich-Heine
University, Germany; Executive Board
member of ECTRIMS
Gail J. Maderis
CEO, Antiva Biosciences,
Former CEO, BayBio, Former CEO of
Five Prime Therapeutics, Founder of
Genzyme Molecular Oncology
Michael S. Richman
Former CEO, Amplimmune
Scott B. Seaman
Executive Director, Alkek Foundation
Neil K. Warma
President & CEO, Opexa
25
SPMS Scientific Advisory Board
 Dawn McGuire, M.D., FAAN (Chair)
• Advisory Council of the Gill Heart Institute
• Former Vice President of Clinical Research at Elan Pharmaceuticals
 Hans-Peter Hartung, M.D
• Chair of Neurology at Heinrich-Heine University, Düsseldorf
• Executive Board member of ECTRIMS, World Health Organization Advisory Board on MS
 Mark S. Freedman, M.D.
• Director of the Multiple Sclerosis Research Unit at Ottawa Hospital
• Multiple Sclerosis Society of Canada, National MS Society (USA)
• ACTRIMS committee member
 Clyde Markowitz, M.D.
• Director of MS Center at the University of Pennsylvania
 Doug Arnold, M.D.
• James McGill Professor Neurology and Neurosurgery at the Montreal Neurological Institute
 Edward Fox, M.D., Ph.D.
• Director of Multiple Sclerosis Clinic of Central Texas
• Advisory Committee, Lone Star Chapter of the National Multiple Sclerosis Society
26
Opexa Investment Highlights
 PIPELINE
• Tcelna®: Phase 2b for secondary progressive multiple sclerosis (SPMS), limited
competition, $7BN overall market potential
• OPX-212: Pre-IND for neuromyelitis optica (NMO), no approved therapies, orphan
indication
 TECHNOLOGY
• Personalized T-cell immunotherapy platform for autoimmune diseases
• Potential to yield multiple candidates tailored to each patient’s disease profile
• Scalable cGMP manufacturing facility
• Strong patent estate (160 domestic and international)
 VALIDATION
• Option agreement secured with Merck Serono for Tcelna – strong potential partner
• FDA Fast Track designation for Tcelna in SPMS
• Esteemed Scientific Advisory Board

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Opexa therapeutics corporate presentation february 2016 web

  • 1. Opexa Therapeutics, Inc. NASDAQ: OPXA Precision Immunotherapy February 2016 The Woodlands, TX Precision Immunotherapy®
  • 2. 2 Forward-Looking Statements All statements in this presentation other than those of historical fact, including statements regarding our preclinical and clinical development plans for Tcelna® and OPX-212, our research and other development programs, our ability to undertake certain activities and accomplish certain goals, projected timelines for our research and development activities and possible regulatory approvals, if any, our expectations regarding the relative benefits of our product candidates versus competitive therapies, our expectations regarding the possibility of licensing or collaborating with third parties regarding our product candidates or research, and our expectations regarding the therapeutic and commercial potential of our product candidates, research, technologies and intellectual property, are forward-looking statements. The words “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “design,” “intend,” “expect,” “potential” and similar expressions, as well as the negative version of these words and similar expressions, are intended to identify forward-looking statements. Our forward-looking statements do not constitute guarantees of future performance, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those anticipated or implied in such statements. Our forward-looking statements are based upon our current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated as a result of various risks and uncertainties which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and risks inherent in the effort to build a business around such drugs. Although we believe our expectations are reasonable, we do not in any way guarantee future results, level of activity, performance or achievements. In addition, neither we nor any other person assumes responsibility for the accuracy and completeness of any forward-looking statements. Our forward-looking statements in this presentation speak only as of the date of this presentation. We assume no obligation or undertaking to update or revise any statements to reflect any changes in our expectations or any change in events, conditions or circumstances on which any such statement is based. You should, however, review additional disclosures we make that further describe risks and uncertainties relevant to us in additional detail in our filings with the Securities and Exchange Commission including our Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. You may get these documents for free by visiting EDGAR on the SEC web site at http://www.sec.gov.
  • 3. 3 Opexa Investment Highlights  PIPELINE • Tcelna®: Phase 2b for secondary progressive multiple sclerosis (SPMS), limited competition, $7BN overall market potential • Top line results expected for Abili-T Phase 2b SPMS trial in 2H 2016 • OPX-212: Pre-IND for neuromyelitis optica (NMO), no approved therapies, orphan indication  TECHNOLOGY • Personalized T-cell immunotherapy platform for autoimmune diseases • Potential to yield multiple candidates tailored to each patient’s disease profile • Scalable cGMP manufacturing facility • Strong patent estate (160 domestic and international)  VALIDATION • Option agreement secured with Merck Serono for Tcelna – strong potential partner • FDA Fast Track designation for Tcelna in SPMS • Esteemed Scientific Advisory Board
  • 4. OPEXA’S NOVEL APPROACH; PRIMING THE IMMUNE SYSTEM 4
  • 5. 5 • Numerous autoimmune diseases are treated with immunosuppressive drugs, despite black box warnings on the labels and significant side effects • Global suppression of immune cells increases patient risk, especially with long term chronic use • Opexa’s immunotherapy mechanism of action selectively targets only harmful, pathogenic T-cells leaving the rest of the immune system in tact • Favorable benefit:risk profile of Opexa’s immunotherapy should differentiate Opexa from competitors Opexa is Positioned to Address High Unmet Medical Need in Autoimmune Diseases
  • 6. 6 • Opexa’s T-cell immunotherapy aims to restore tolerance – To prevent the further attack of healthy tissue by activated immune cells – Reprograming of the immune system to selectively eliminate the pathogenic T-cells that cause disease • Maintains the body’s defense mechanisms – No evidence of global suppression in previous controlled trials • May lead to stabilization of disease and possibly allow the body to repair damage caused by disease – Evidence to support this in previous MS controlled trial Restoration of Immune Tolerance: The key to advancing the treatment of autoimmune diseases
  • 7. 7 Root Cause of MS: Activated T-cells Degrade Myelin and Damage Myelin Producing Cells Adapted by permission from Macmillan Publishers Ltd: NATURE REVIEWS IMMUNOLOGY 3, 483-492 (June 2003), copyright (2003) Cytokines Damage Damage • In MS patients, the faulty immune system is not able to prevent the attack of a small sub- population of myelin reactive T-cells (MRTC) • MRTCs cross the blood brain barrier, enter the brain, and bind to antigen presenting cells (APC), causing a release of pro- inflammatory cytokines which lead to a two pronged attack through: 1. Destruction of myelin sheath, the protective coating of nerve fibers 2. Destruction of oligodendroglial cells, which are responsible for producing myelin Myelin peptide Result Destruction of the myelin sheath and myelin producing cells, thereby preventing remyelination
  • 8. 8 Tcelna Could Address the Root Cause of MS by Preventing Demyelination and Enabling Remyelination Adapted by permission from Macmillan Publishers Ltd: NATURE REVIEWS IMMUNOLOGY 3, 483-492 (June 2003), copyright (2003) Opexa’s Strategy Tcelna programs the immune system to specifically recognize MRTC as pathogenic, thereby inhibiting further destruction of the myelin sheath and potentially enabling remyelination • Therapeutic dose of Tcelna (attenuated T-cell clones) is injected subcutaneously • This triggers an immune response specifically targeting circulating MRTC • Immune cells, including Tregs, have been primed, or sensitized, we believe, to specifically target the pathogenic MRTC for elimination or regulation • Elimination of harmful MRTC may lead to: 1. Stabilization of disease by preventing further destruction of myelin 2. Improvement in condition by allowing oligodendroglial cells to remyelinate axons (neuroprotection)
  • 9. IMMPATH®: OPEXA’S PROPRIETARY T-CELL IMMUNOTHERAPY PLATFORM 9
  • 10. 10 ImmPath®: Harnessing the Power of the Patient’s Own Immune System to Restore Tolerance Administration 5 SC doses/year Procurement Dispensation 1 Day Expansion of T-cells Attenuation • Annual course of treatment generated from a single manufacturing run • Therapy is personalized annually and is tailored to evolving epitope profile Formulation & cryopreservation Antigen-sourced seed material (Blood collection) Manufacturing and QC 35 Days
  • 11. TCELNA® FOR MULTIPLE SCLEROSIS 11
  • 12. 12 Opexa Developing Highly Differentiated, Precision Therapies OPX-212 Preclinical Phase 1 Phase 2 Phase 3Programs and indications Neuromyelitis Optica (NMO) IND enabling activities ongoing Tcelna (imilecleucel-T) * Research Secondary Progressive Multiple Sclerosis (SPMS) Phase 2b trial ongoing, data expected 2H 2016 Key Inflection Point: 2H 2016 Top-line Data from Phase 2b Abili-T trial in SPMS expected
  • 13. 13 Tcelna in Secondary Progressive MS SPMS 450,000 Individuals in North America diagnosed with MS; 30-45 % potentially have an SPMS diagnosis Market Size: $7 Billion (est.) Relapsing Remitting MS; Clinically Isolated Syndrome; Primary Progressive MS • Secondary Progressive MS market potential in North America could exceed $7 billion (for all therapies) • Roughly 150,000 SPMS patients in North America • Only one drug approved for SPMS in U.S. (none in EU or Asia) - Drug not suitable for chronic use due to severe side effects Tcelna being developed to be a potential therapy of choice in SPMS
  • 14. 14 Tcelna® Lead Program Targeting Secondary Progressive MS patients Fast Track Designation by FDA • Phase 2b is fully enrolled: 190 patients with SPMS • Top line data expected in 2H 2016 • Design – Double-blind, 1:1 randomized, placebo-controlled – 35 clinical sites in USA and Canada – Two annual courses of personalized therapy – Efficacy Endpoints: Primary-Whole Brain Atrophy, Secondary-Disease Progression • Immune Monitoring being conducted in parallel – Comprehensive biomarker analysis
  • 15. 15 Clinical Evidence for Tcelna MoA T-cell technology demonstrated a significant reduction in reactive T-cells (52 week study results of MS) 0% -92% -87% -79% -77% -65% -100% -90% -80% -70% -60% -50% -40% -30% -20% -10% 0% Week 0 Week 5 Week 13 Week 21 Week 28 Week 52 • Results from Tcelna dose ranging studies:  Published in Clinical Immunology (2009) 131, 202-215 • Each dose consisting of 30-45 x 106 cells • % reduction relative to baseline (Week 0) ReductioninMRTC Dose 1 Dose 2 Dose 3 Dose 4 MRTC: Myelin Reactive T-cells
  • 16. 16 Phase 2b RRMS Prior Clinical Trial Tcelna showed 37% improvement over placebo (mITT) (secondary endpoint measurement, primary MRI endpoint measure not met) 0.214 0.339 0 0.05 0.1 0.15 0.2 0.25 0.3 0.35 0.4 37% Reduction in Annualized Relapse Rate (ARR) Tcelna Placebo n=94 n=48 Relapses/Patient/Year 37% Total mITT population n=142
  • 17. 17 Results of Phase 2b TERMS study in RRMS Reversal of disability in prospective analysis in more active patients Sub-population of patients (n=50) with more progressed/active disease profile (baseline ARR >1) 2.4 2.23 2.2 2.39 2.10 2.15 2.20 2.25 2.30 2.35 2.40 2.45 EDSSScore(Mean) Baseline Week 52 Tcelna Placebo (p=0.045) Statistically Significant Improvement in Disability (p=0.045) 55% Reduction in ARR Change in Disability (EDSS) at Week 52 0.28 0.63 0 0.1 0.2 0.3 0.4 0.5 0.6 0.7 Tcelna Placebo 55% Relapses/Patient/Year Annualized Relapse Rate (ARR) at week 52 p=0.07
  • 18. 18 Merck Serono Agreement Agreement signed 2013; strong potential partner • Option and License Agreement for worldwide rights to all Multiple Sclerosis indications, excluding Japan • If Merck Serono exercises option: – Merck Serono to fund Phase 3, pre-commercial and commercial activities – Opexa has potential to receive up to an additional $220 million in option exercise and milestone payments – Opexa has potential to receive royalties ranging from 8% to 15% of annual net sales, with step-ups occurring if net sales exceed $500 million, $1 B & $2 B • Merck Serono increased their commitment to Opexa’s MS program this year – In anticipation of the Phase IIb trial results expected in 2H 2016, Merck Serono gave Opexa $3 million of support in March 2015 to begin planning for Phase III studies
  • 19. 19 Financials Cash and Cash Equivalents (MM) as of September 30, 2015 ~$15.6 Shares outstanding (MM) as of December 1, 2015 ~7.0 Shares issuable for Warrants (MM) outstanding as of September 30, 2015 ~3.7(1) Shares issuable for Stock Options (MM) outstanding as of September 30, 2015 ~0.4(2) Debt None (1) Weighted average exercise price =$6.30 as of September 30, 2015 (2) Weighted average exercise price =$18.11 as of September 30, 2015
  • 20. LEVERAGING THE T-CELL PLATFORM: OPX-212 FOR NEUROMYELITIS OPTICA (NMO) 20
  • 21. 21 NMO: A Rare Disease of the Central Nervous System • Neuromyelitis optica (NMO) is a rare or orphan autoimmune disease • Immune system cells and antibodies mistakenly attack and destroy myelin cells in the optic nerves and the spinal cord • Individuals with NMO develop optic neuritis, which causes pain in the eye and vision loss, and transverse myelitis, which causes weakness, numbness, and sometimes paralysis of the arms and legs • There are no FDA-approved therapies for NMO • Affects any age varying from 3 to 90 years, the average age of onset ~41 years An orphan disease with no FDA-approved therapy Image reprinted with permission from MultiView, Inc.
  • 22. 22 Key Operational Milestones Achieved For NMO Program FDA feedback • Conducted pre-IND meeting to discuss pre-clinical and clinical program to support OPX-212 development in NMO Preclinical animal study • Results show statistically significant dose dependent reduction of AQP4 reactive T-cells in murine model • Supportive of MOA KOL support • Engaged key thought leaders to validate scientific hypothesis and discuss clinical trial design – Benjamin Greenberg, M.D., University of Texas, Southwestern Medical Center – Michael Levy, M.D., Ph.D., The Johns Hopkins Hospital Patient support • Collected NMO patient blood samples to conduct pre-IND activities, process development and research into mechanism of action
  • 23. 23 FDA Pre-IND Meeting Outlined Preclinical Plan Bioactivity Study Supports Development Of OPX-212 • FDA request: Design a study to demonstrate biological activity in animal model – No pharm/tox required • Design: Based on Opexa Phase I study in MS patients • Hypothesis: Demonstrate reduction in pathogenic AQP4 T-cells in murine model • Procedure: Mice were pre-treated with attenuated antigen-specific T-cells and primed with the NMO autoantigen • Results: Opexa demonstrated dose dependent and statistically significant reduction in pathogenic (AQP4) T-cells in murine model • Conclusion: Bioactivity study supports further development of OPX-212 in NMO and supports IND 0% -92% -87% -79% -77% -65% -100% -90% -80% -70% -60% -50% -40% -30% -20% -10% 0% Week 0Week 5 Week 13 Week 21 Week 28 Week 52 Dose 1 Dose 2 Dose 3 Dose 4 ReductioninMRTC
  • 24. 24 Experienced Management Team and Board of Directors Neil Warma, President & CEO, Director › 19+ years international healthcare experience with large Pharma and emerging biotechnology companies › Former Senior Management, Novartis Pharmaceuticals, Basel, Switzerland › Former CEO, Viron Therapeutics, Inc. › Co-founder and President of MedExact Inc., a company subsequently acquired Karthik Radhakrishnan, Chief Financial Officer › 10+ years of health care capital markets experience › Formerly, Vice President at ING Investment Management › MBA, MS in Engineering, CFA charter holder Don Healey, Ph.D., Chief Scientific Officer › 25+ years of experience in cellular immunology and immune regulation › Former Director of Immunology, Argos Therapeutics Donna Rill, Chief Development Officer › 30 years in cell and gene therapy research and clinical application › Designed and validated cGMP Cell & Gene Therapy Laboratories, Vector Production facilities, and Translational Research Labs Jason Kralic, Ph.D., VP, Business Development › Former Head of Business Development for the Neurosciences Therapy Area Unit at GlaxoSmithKline › Ph.D. in Pharmacology from University of N. Carolina at Chapel Hill School of Medicine Board of Directors Timothy Barabe Board member of Arqule, Inc.; Former CFO of Affymetrix, Human Genome Sciences, Inc., Regent Medical UK and Sandoz GmbH Dr. Hans-Peter Hartung Chair of Neurology at Heinrich-Heine University, Germany; Executive Board member of ECTRIMS Gail J. Maderis CEO, Antiva Biosciences, Former CEO, BayBio, Former CEO of Five Prime Therapeutics, Founder of Genzyme Molecular Oncology Michael S. Richman Former CEO, Amplimmune Scott B. Seaman Executive Director, Alkek Foundation Neil K. Warma President & CEO, Opexa
  • 25. 25 SPMS Scientific Advisory Board  Dawn McGuire, M.D., FAAN (Chair) • Advisory Council of the Gill Heart Institute • Former Vice President of Clinical Research at Elan Pharmaceuticals  Hans-Peter Hartung, M.D • Chair of Neurology at Heinrich-Heine University, Düsseldorf • Executive Board member of ECTRIMS, World Health Organization Advisory Board on MS  Mark S. Freedman, M.D. • Director of the Multiple Sclerosis Research Unit at Ottawa Hospital • Multiple Sclerosis Society of Canada, National MS Society (USA) • ACTRIMS committee member  Clyde Markowitz, M.D. • Director of MS Center at the University of Pennsylvania  Doug Arnold, M.D. • James McGill Professor Neurology and Neurosurgery at the Montreal Neurological Institute  Edward Fox, M.D., Ph.D. • Director of Multiple Sclerosis Clinic of Central Texas • Advisory Committee, Lone Star Chapter of the National Multiple Sclerosis Society
  • 26. 26 Opexa Investment Highlights  PIPELINE • Tcelna®: Phase 2b for secondary progressive multiple sclerosis (SPMS), limited competition, $7BN overall market potential • OPX-212: Pre-IND for neuromyelitis optica (NMO), no approved therapies, orphan indication  TECHNOLOGY • Personalized T-cell immunotherapy platform for autoimmune diseases • Potential to yield multiple candidates tailored to each patient’s disease profile • Scalable cGMP manufacturing facility • Strong patent estate (160 domestic and international)  VALIDATION • Option agreement secured with Merck Serono for Tcelna – strong potential partner • FDA Fast Track designation for Tcelna in SPMS • Esteemed Scientific Advisory Board