2. 2
Forward-Looking Statements
This investor presentation contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of
the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Statements contained in
this presentation, other than statements of historical fact, constitute “forward-looking statements.” The words “expects,” “believes,”
“anticipates,” “estimates,” “may,” “could,” “intends,” and similar expressions are intended to identify forward-looking statements. The
forward-looking statements in this presentation do not constitute guarantees of future performance. Investors are cautioned that
statements in this presentation which are not strictly historical statements, including, without limitation, statements regarding the
development of the Company’s product candidate, Tcelna (imilecleucel-T), constitute forward-looking statements. Such forward-looking
statements are subject to a number of risks and uncertainties that could cause actual results to differ materially from those anticipated.
These risks and uncertainties include, but are not limited to, risks associated with: market conditions; our capital position; the rights and
preferences provided to the Series A convertible preferred stock and investors in the convertible secured notes we issued in July 2012
(including a secured interest in all of our assets); our ability to compete with larger, better financed pharmaceutical and biotechnology
companies; new approaches to the treatment of our targeted diseases; our expectation of incurring continued losses; our uncertainty of
developing a marketable product; our ability to raise additional capital to continue our development programs (including to undertake
and complete any ongoing or further clinical studies for Tcelna), including in this regard our ability to satisfy various conditions required
to access the financing potentially available under the purchase agreements with Lincoln Park Capital Fund, LLC (“Lincoln Park”) (such
as the minimum closing price for our common stock and the requirement for an ongoing trading market for our stock); our ability to
regain and maintain compliance with NASDAQ listing standards; the success of our clinical trials (including the Phase IIb trial for Tcelna
in secondary progressive MS which, depending upon results, may determine whether Merck elects to exercise its Option); whether
Merck exercises its Option and, if so, whether we receive any development or commercialization milestone payments or royalties from
Merck pursuant to the Option; our dependence (if Merck exercises its Option) on the resources and abilities of Merck for the further
development of Tcelna; the efficacy of Tcelna for any particular indication, such as for relapsing remitting MS or secondary progressive
MS; our ability to develop and commercialize products; our ability to obtain required regulatory approvals; our compliance with all Food
and Drug Administration regulations; our ability to obtain, maintain and protect intellectual property rights (including for Tcelna); the
risk of litigation regarding our intellectual property rights or the rights of third parties; the success of third party development and
commercialization efforts with respect to products covered by intellectual property rights that we may license or transfer; our limited
manufacturing capabilities; our dependence on third-party manufacturers; our ability to hire and retain skilled personnel; our volatile
stock price; and other risks detailed in our filings with the SEC. These forward-looking statements speak only as of the date made. We
assume no obligation or undertaking to update any forward-looking statements to reflect any changes in expectations with regard thereto
or any change in events, conditions or circumstances on which any such statement is based. You should, however, review additional
disclosures we make in our Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K filed with
the SEC.
3. 3
Opexa’s Mission
To lead the field of
Precision ImmunotherapyTM
by
aligning the interests of
patients, employees and
shareholders.
4. 4
Tcelna™ Addressing the Root Cause of Multiple Sclerosis; the
Role of Myelin Reactive T Cells (MRTCs)
• The Oligodendroglial cells play a vital role in the
production of myelin, the protective coating of nerve
fibers
• When MRTCs cross the blood brain barrier and bind to
the Antigen Presenting Cells, there is a release of pro-
inflammatory cytokines (IL-12/23, IFN-γ, IL-17 and
TNFa)
• The cytokine release is destructive to neurons, attacking
both the Axons and the “Oligo” Cells
Result Destruction of the myelin sheath
• T cells play a vital role in our immune system. Most
competing therapies non-specifically suppress all T-
cells (including beneficial T cells) and/or focus on
preventing their entry into the central nervous system,
which may lead to serious side effects.
Antigen
MRTC
Opexa’s Strategy
TcelnaTM programs the immune system to specifically
recognize MRTCs as pathogenic thereby inhibiting further
destruction of the myelin sheath and potentially enable
remyelination
5. 5
The Immunopathology of SPMS
• Compartmentalized CNS inflammatory cells
– Microglia activation suggests ongoing chronic innate immune
responses
• Meningeal follicles close to cortical gray matter lesions suggests
that adaptive immune responses involving antibody and
complement contribute to progression in SPMS
• Chronic myelin-reactive cells may be contributing to the
development of both innate and adaptive immune responses
persisting in the CNS
• TcelnaTM immunotherapy in SPMS may reduce the
drivers of this chronic disease-driving immunity
through both peripheral and central regulatory
responses
6. 6
Mechanism of Action
• Attenuated, patient-specific (autologous) myelin reactive
T-cells (MRTC)
– MRTC expanded ex vivo in response to immunodominant peptides of MBP, MOG
& PLP
• Therapeutic sc dosing (30-45 x 106 cells) stimulates host
reactivity to the ‘over-represented’ MRTC inducing a
dominant negative ‘regulatory T-cell’ response leading to:
– Down-regulation of similar endogenous disease-causing myelin reactive T-cells
– Up-regulation of regulatory cells (Foxp3+ and Tr1 cells) to reduce inflammation
and provide possible neuroprotection, should these gain entry to the CNS
• Three Phase I/II studies conducted with Tcelna™ in RRMS
and SPMS showed a reduction in anti-myelin reactive
T-cells, lower relapse rates and stabilization of disease as
defined by EDSS
7. 7
Mechanism of Action
-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
ReductioninMRTC
Dose 2 Dose 3 Dose 4
Each dose consisting of 30-45 x 106 cells
Dose 1
Tcelna Demonstrated a Reduction in Myelin Reactive T-cells (MRTC)
8. 8
TcelnaTM in Secondary Progressive Multiple Sclerosis
• Patients initially experience a relapsing-
remitting course then transition to SPMS
• SPMS patients experience worsening QOL /
disability with or without relapses
• Over 400,000 patients in U.S. and over
1 million world wide have MS
• Approximately 30-45% of MS patients can be classified as Secondary Progressive
• TcelnaTM is being pursued for this SPMS indication
• Potential SPMS market in the U.S. alone could exceed $7 billion [150,000 SPMS
patients at average cost of $50,000 per year of treatment]
• No SPMS treatment approved by EMA; only one approved by FDA with limited use
due to toxicity
• 1/3rd market share translates to $2.5 billion dollar sales in the U.S. and $4-$5 billion
globally
9. 9
Merck Serono Agreement for MS indication
• Feb 2013 option and license agreement with Merck Serono
– Up to $220 million in additional milestone payments
• Option exercise $25 million for starting Phase III/ or $15 million if another
Phase II
• $35 million FDA filing, approval and commercialized in US
• $30 million for EU filing, approval and commercialization in at least three
countries
• RRMS development and commercialization of up to $40 million
– One time commercial milestones of up to $85 million
• Royalties ranging from 8 % to 15% of annual net sales with step-ups occurring
when net sales exceed $500 million, $1 B & $2 B
• Opexa maintains key rights:
– Development and commercialization rights to Tcelna in Japan
– Certain manufacturing rights
– Co-development funding option in exchange for increased royalties
– Rights to all other disease indications
10. 10
Financials
Income Statement Q1 2013 Q1 2012
Option Revenue $220,100 -
Research & Development $1,621,366 $1,490,097
General & Administrative $1,102,435 $816,196
Earnings Per Share $ (0.58) $ (0.41)
Balance Sheet Details March 31, 2013 Dec 31, 2013
Cash $7,834,336 $592,004
Basic Shares as of 05/10 8,070,130
Warrants as of 03/31 3,422,646 Weighted average X=$4.01
Stock Options 903,289 Weighted average X=$5.14
Market Cap ~$18 million
11. 11
Tcelna
TM
Manufacturing: A two-step process
The Epitope Profiling Assay (EPA)
• Screen peripheral blood for Myelin-Reactive T-cells (MRTCs), and
mapping of immunodominant epitopes to MBP, MOG and PLP
– 109 overlapping peptides encompassing MBP, MOG and PLP
– Interferon gamma response to individual peptide pools defines positive response
in 7 day assay
ImmPathTM Process
• Procure unit of blood from which up to six T-cell lines reactive with
immunodominant myelin peptides are generated and pooled as a
patient-specific TcelnaTM product
– Manufacturing performed under GMP/GTPs in functionally closed system
– Process generates a year of TcelnaTM doses from a single unit of blood
12. 12
TcelnaTM Manufacturing
Expansion of antigen specific T-cells
Cryopreservation
Formulation/
Irradiation of each
dose as required
Epitope
profiling
Administration: 5
subcutaneous
injections/year
Manufacturing and QC Dispensation
35 days
Epitope Profiling
1 day14 days
- Red Cross
- Blood Group Alliance
13. 13
Year 2
Year 3
TcelnaTM Manufacturing: Precision Medicine
Proprietary Assay Enables Annual Personalized Treatments
Year 1
Conduct analysis of 109 peptides from all three key myelin proteins (MBP, MOG, PLP)
Re-assess epitope profile annually to
identify epitope drift
Develop new formulation based on evolved
epitope profile
14. 14
TcelnaTM Development Program
• Completed Trials
– 5 clinical studies completed (in Relapsing Remitting and Secondary Progressive MS patients)
– 302 patients total
– 142 treated with Tcelna in Opexa-sponsored research
• Ongoing Trial
– Abili-T Study: Clinical trial ongoing in SPMS in the US and Canada
• Over 850 Tcelna™ preparations have been successfully manufactured
– reproducible and consistent
• Safety demonstrated and clinical efficacy promising across broad spectrum of patients for
relapsing and Secondary Progressive MS
SPMS
Patients Endpoints
Phase 2b * 180 Brain Atrophy,
EDSS
Phase 1/2 10 EDSS, ARR
Phase 1
(Baylor)
26 EDSS, ARR
RRMS
Patient
s
Endpoints
Phase 2b 150 MRI (lesion #),
ARR
Phase 2b (subpopulation:
ARR>1)
50 EDSS, Atrophy,
ARR
Phase 1/2 Dose Ranging Study 12 ARR, EDSS
Phase 1/2 Extension Study 12 ARR, EDS
* Ongoing SPMS Trial
15. 15
Safety Summary
Deaths • No deaths occurred
Serious Adverse Events (SAEs) • No treatment-related SAEs
Most common adverse events • Mild to moderate injection site reaction only
Summary of Safety from Phase IIb (n=150)
Terms Study - Safety
16. 16
TERMS Study Results
• Completed Phase IIb clinical trial in 150 RRMS patients; 33 sites in U.S.
• mITT population (n=142)
– 37% reduction in ARR vs. placebo
– ARR 0.214 vs. 0.339
– 77% relapse free in TcelnaTM group
• Superior safety and promising
efficacy demonstrated
• Two End-of-Phase II meetings
with FDA successfully completed
0.214
0.339
0
0.05
0.1
0.15
0.2
0.25
0.3
0.35
0.4
Tcelna
Relapses/patient/yr
Tcelna
Placebo
37%
17. 17
Terms Study : Subgroup Analysis
Patient Naïve to DMT
Annualized relapse rate in DMT naïve populations. ARR in placebo subjects without previous
disease modifying treatment (DMT) experience reflects relapse rates commonly seen in other
placebo controlled trials in MS. In this subpopulation treatments with TcelnaTM resulted in a 56–
73% reduction in ARR compared with placebo.
Tcelna Placebo
18. 18
Terms Study -Prospective Analysis
• Sub-population of patients (n=50) with more progressed/active disease profile
(baseline ARR >1) most closely mirrors SPMS patients
– 73% relapse free
– Significant improvement in disability (p=0.05)
– 56% reduction in ARR
– 88% reduction in whole-brain atrophy
-0.04
-0.32
-0.35
-0.3
-0.25
-0.2
-0.15
-0.1
-0.05
0
VolumeChange(%)
Percent Brain Volume Change at Week 52
Tovaxin Placebo
88%
0.28
0.63
0.00
0.10
0.20
0.30
0.40
0.50
0.60
0.70
Tovaxin (n = 32) Placebo (n = 18)
ARR(relpase/year)
56%
Data Provide Support for
Phase IIb Program in SPMS
2.40
2.23
2.20
2.39
2.15
2.20
2.25
2.30
2.35
2.40
2.45
2.50
Baseline EDSS Week 52 EDSS
EDSSScore(mean)
Tovaxin (9.1%
Improvement)
p=0.045
Tcelna
(n=32)
Placebo
(n=18)
Tcelna (9.1%
Improvement)
Tcelna Placebo
19. 19
TcelnaTM increases Tr1 cells in SPMS Patients
Clinical findings in TcelnaTM treated patients
• All three patients (who had experienced relapse in preceding 12-24 month) remained relapse
free during the 52-week assessment
• Showed stabilization of disease progression
• Showed a 57%-67% reduction in myelin reactive T-cell counts from baseline
Observations
• Increase in Tr1 cells from non-detectable to detectable levels in TcelnaTM treated patients (n=3)
• Increase in Tr1 cells to a level similar to those observed in healthy controls (n=4)
• p=0.971 (i.e. no statistical difference between Tr1 healthy donor levels and post-TcelnaTM treatment dose levels)
Pre-Tcelna (non-detectable)
Post -Tcelna
20. 20
-8.0%
23.8%
32.9%
27.4%
23.4%
-9.1%
18.4%
7.8%
4.5%
6.3%
-20%
-10%
0%
10%
20%
30%
40%
0 10 20 30 40 50 60
FoxP3+PercentageChangefrom
Baseline
Tovaxin
Placebo
Treatment Period Efficacy Period
(Weeks 36-52)
Abili-T study Immune Monitoring Program
Better Characterize Mechanism of Action
Change in CD4+CD25+FoxP3+ T-regulatory Cells
Data from TERMS Study: In clinical target population ARR>1
Tcelna™ statistically significant from baseline (p<0.02)
Tcelna
21. 21
• 36 patients treated in three clinical studies
• Promising efficacy observed
• Disease stabilization in 80% of patients at two years
• Significant reduction in relapse rates
• Well-tolerated, no SAEs
Secondary Progressive MS: Clinical Overview
22. 22
Tcelna™ Stabilizes Disease in SPMS
*A small percentage of patients in pooled analysis
showed an improvement (i.e. decrease in progression) **Historical control: ESIMS Study, published Hommes Lancet 2004
80%
20%
40%
0%
20%
40%
60%
80%
100%
Tovaxin (n = 35) Historical Disease Progression
PercentageofPatientsShowing
DiseaseStabilization
Stable
Progressed
Stabilization
vs.
Historical Progression
*
**
80% of subjects had no disease progression by EDSS at 2 years
Tcelna
(n=35)
23. 23
Phase I and Phase I/II Studies
Pooled - Annualized Relapse Rate (ARR)AnnualizedRelapseRate(ARR)
0.00
0.10
0.20
0.30
0.40
0.50
0.60
Baseline 12 Months 24 Months
• SPMS Patients (n=10) followed for 7,955
cumulative days (21.8 patient years)
• Only 1 patient experienced 1 relapse
One relapse observed in 21 years of cumulative patient follow up
24. 24
Phase I and Phase I/II Studies
Pooled SPMS Data - Change in MSIS Scores at 2 years
0.9%
1.2%
2.1%
-5.1%
-6.0%
-4.0%
-2.0%
0.0%
2.0%
4.0%
Physical
Psychological
12 Month (n=10) 18-24 Month (n=7)
Negative percentage change
indicates improvement
from baseline
Following two years of treatment, no worsening of physical
condition and improvement in psychological condition
PercentagechangeinMultipleSclerosisImpactScale
25. 25
Abili-TTM
: Landmark trial in SPMS
• Abili-T Phase IIb clinical trial in SPMS is ongoing
– Double-blind, 1:1 randomized, placebo-controlled
– Inclusion criteria: Secondary Progressive MS with EDSS of 3 to 6 (TERMS:
EDSS of 0 to 5.5 and 1 year trial)
– 48 patients enrolled at April 30, 2013
– Immune Monitoring program conducted on a blinded basis
• Fast Track designation granted by FDA for Tcelna in SPMS
• 180 Patients expected to be enrolled
– SPMS population
– 30 sites in USA and Canada
• 2 annual courses of personalized therapy
• 2 years of assessment
26. 26
Efficacy Assessments
• Primary Endpoint
– Whole-brain atrophy
• Secondary Endpoints
– Sustained progression measured by EDSS
– Time to sustained progression
– T2 lesions progressing to hypointense lesions (black holes)
– Change in EDSS
– Annualized Relapse Rate (ARR)
– Change in MSFC Assessment of disability
– Change in Symbol Digit Modality Test (SDMT)
• Exploratory Endpoints
– Quality of life assessment by MSQLI
– Gd-enhancing lesion volume with increasing MTR
– Gd-enhancing lesion volume with decreasing MTR
– Change in MTR in normal-appearing white matter
– Changes in T-regulatory cell repertoire and function
27. 27
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
• President ECTRIMS, World Health Organization Advisory Board on MS
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
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
28. 28
Opexa Management Team
Neil Warma, President & CEO, Director
• 19+ years international healthcare experience with large and emerging biotechnology companies
• 9 years of Senior Management with Novartis Pharmaceuticals
• Formerly, President & CEO and Member of the Board of Directors of Viron Therapeutics, a private clinical
stage biopharmaceutical company developing a novel class of protein therapeutics
• Co-founder and President of MedExact Inc., a medical Internet company, which was successfully sold
• Neuroscience and MBA degrees
Don Healey, Ph.D., Chief Scientific Officer
• Over 25 years experience in cellular immunology and immune regulation in academic and biotech
environments
• Former Director of Immunology for Argos Therapeutics, responsible for the development of novel autologous
dendritic cell therapies for the treatment of renal carcinoma and HIV
Donna Rill, Chief Development Officer
• 30 years of extensive clinical and research laboratory experience in cell and gene therapy research and clinical
application, immunological techniques and assessments
• Designed, and validated cGMP Cell & Gene Therapy Laboratories, cGMP Vector Production facilities, and
Translational Research Labs
Kenny Frazier, VP of Clinical Development and Regulatory Affairs
• 24 years of extensive clinical and regulatory experience: Formerly, Head of Clinical Operations at Lexicon
Pharmaceuticals and at Tanox, Inc.
Karthik Radhakrishnan, Chief Financial Officer
• 10+ years of health care capital markets experience; Formerly, Vice President at ING Investment Management
responsible for health care investments in small and small-mid cap core/growth funds.
• MBA from University of Michigan, MS in Engineering from University at Buffalo, BS from Indian Institute of
Technology, CFA charter holder.
29. 29
Investment Thesis
• Platform company
• Esteemed Scientific Advisory Board
• Precision therapy potentially maximizes efficacy and minimizes
safety
• Targeting an unmet medical need with a substantial potential
revenue opportunity
• Option Agreement with Merck Serono, a strong commercial
partner
• Replacement value of company is multiples of present market
cap
• Attractive potential risk-reward profile for long term/value
investors
• Goal-oriented management team focused on value creation for
patients, employees and shareholders
