This document provides an overview and summary of Opexa Therapeutics' rights offering. Key points include:
- Opexa is conducting a rights offering of up to 28.8 million units to fund its ongoing Phase 2b trial of Tcelna in secondary progressive multiple sclerosis and a potential Phase 1/2 trial of OPX-212 in neuromyelitis optica.
- Proceeds will also be used for general corporate purposes.
- Tcelna utilizes Opexa's proprietary precision immunotherapy platform and has received Fast Track designation from the FDA for secondary progressive MS. Prior clinical trials in both relapsing-remitting and secondary progressive MS showed encouraging results.
- The rights offering is
Kiromic BioPharma, Inc. is a target discovery and gene-editing company utilizing artificial intelligence and its proprietary neural network platform with a therapeutic focus on immuno-oncology.
SciSparc is a specialty pharmaceutical company developing unique cannabinoid technologies for treatment of central nervous system disorders. The proprietary compounds capitalize on the biochemistry of receptors that specialize in modulating the central nervous system (CNS) to create therapeutics that mitigate the adverse symptoms associated with CNS disorders.
Kiromic BioPharma, Inc. is a target discovery and gene-editing company utilizing artificial intelligence and its proprietary neural network platform with a therapeutic focus on immuno-oncology.
SciSparc is a specialty pharmaceutical company developing unique cannabinoid technologies for treatment of central nervous system disorders. The proprietary compounds capitalize on the biochemistry of receptors that specialize in modulating the central nervous system (CNS) to create therapeutics that mitigate the adverse symptoms associated with CNS disorders.
SciSparc is a specialty pharmaceutical company developing unique cannabinoid technologies for treatment of central nervous system disorders. The proprietary compounds capitalize on the biochemistry of receptors that specialize in modulating the central nervous system (CNS) to create therapeutics that mitigate the adverse symptoms associated with CNS disorders.
NeoStem, Inc. (“NeoStem” or the “Company”), a biopharmaceutical company, is capitalizing on the paradigm shift occurring in medicine by engaging in the development and manufacture of cellular therapies for oncology, immunology, and regenerative medicines in the United States. The Company anticipates that cell therapy will play a significant role in the fight against chronic disease and in lessening the economic burden that these diseases pose to modern society.
Cidara is developing long-acting therapeutics designed to improve the standard of care for patients facing serious diseases. The Company’s portfolio is comprised of drug candidates intended to transform existing treatment and prevention paradigms. Its lead Phase 3 antifungal candidate, rezafungin, will report Phase 3 data at the end of 2021. The potential peak sales opportunity for rezafungin in the US is ~$750M. In addition, the Company is developing Drug-Fc Conjugates (DFCs) targeting viral and oncology diseases from Cidara’s proprietary Cloudbreak® platform.
Lantern Pharma is a clinical stage biotechnology company focused on leveraging artificial intelligence (“A.I.”), machine learning and genomic date to streamline the drug development process and to identify patients who will benefit from their targeted oncology therapies. Their portfolio of therapies consists of compounds that others have tried, but failed, to develop into an approved commercialized drug. Additionally, they develop new compounds with the assistance of their A.I. platform (RADR) and biomarker driven approach. The Company is currently developing four therapeutic programs.
Second Sight Medical Products, a recognized global leader in neuromodulation devices for blindness, announced a definitive agreement on February 7, 2022, under which privately held Nano Precision Medical (NPM) will merge with a wholly owned subsidiary of Second Sight in an all-stock transaction. NPM will be the surviving company and owned by Second Sight. The resulting company will focus on development of innovative drug and device medical implants that treat chronic diseases with high unmet medical need.
Lantern Pharma is a clinical stage biotechnology company focused on leveraging artificial intelligence (“A.I.”), machine learning and genomic date to streamline the drug development process and to identify patients who will benefit from their targeted oncology therapies. Their portfolio of therapies consists of compounds that others have tried, but failed, to develop into an approved commercialized drug. Additionally, they develop new compounds with the assistance of their A.I. platform (RADR) and biomarker driven approach. The Company is currently developing four therapeutic programs.
G Medical Innovations Holdings Ltd is a mobile health (mHealth) and e-health company. It develops and markets clinical and consumer medical-grade health monitoring solutions and offers end-to-end support for e-health projects. The company offers a suite of both consumer and clinical grade products and platforms which are positioned to reduce inefficiencies in healthcare delivery, improve access, reduce costs, increase the quality of care, and make healthcare more personalized and precise.
Aridis Pharmaceuticals is a late-stage clinical development company, leading the creation of transformative, first-in-class anti-infectives for life-threatening viral and bacterial respiratory infections. The company’s lead drug candidate for acute pneumonia met the primary endpoint for its phase 2 clinical trial and the Company is now enrolling in a global phase 3 study. Its pipeline of novel mechanism antibacterial and antivirals, sprung from its proprietary discovery technology platforms, are designed to combat the growing public health threat of viral pandemics and antimicrobial resistant (AMR) bacteria. Its anti-toxin monoclonal antibody (mAb) approach is a proven mechanism of action and has shown preliminary trend toward efficacy as an adjunctive therapy in reducing acute pneumonia patients time in ICU as compared to the current standard of care, which is antibiotics. In addition to its phase 3 program, ARDS also has a phase 2 asset for the treatment of Bacteria HAP/VAP LPS 011. The company also has an on-going phase 2 clinical trial for the treatment of Cystic Fibrosis patients, a program funded by the Cystic Fibrosis Foundation for $7.5 million. Importantly, ARDS is also preparing to launch a Phase 1/2 clinical trial in the second half of 2021 with a self-administered broadly neutralizing mAb for emerging COVID-19 mutated variants. The expansion of COVID virus strain coverage, combined with the product's self-administered, at-home treatment modality, further differentiates the company's AR-712 COVID treatment offering.
Tiziana Life Sciences is a dual-listed clinical stage biotechnology company focused on the discovery and development of novel molecules to treat human disease in oncology and immunology. Tiziana’s two lead drug candidates, Foralumab and Milciclib, uniquely target the root cause of diseases with large unmet needs in multibillion-dollar markets. Tiziana is accelerating development of anti-IL6 receptor monoclonal antibody (mAb) for treatment of COVID-19 lung inflammation. Foralumab is the only fully human anti-CD3 monoclonal antibody in clinical development in the world. This Phase 2 compound has potential application in a wide range of autoimmune and inflammatory diseases, such as nonalcoholic steatohepatitis (NASH), ulcerative colitis, multiple sclerosis, type-1 diabetes (T1D), Crohn's disease, psoriasis and rheumatoid arthritis, where modulation of a T-cell response is desirable. Tiziana is a market leader in the invention of proprietary technology for oral, nasal and inhaled formulation to transform immunotherapies with Monoclonal Antibodies currently administered intravenously. Also, Tiziana has a robust and growing patent portfolio.
Milestone Scientific Inc. (MLSS) is a biomedical technology research and development company that patents, designs, develops and commercializes innovative diagnostic and therapeutic injection technologies and instruments for medical and dental applications. Milestone's computer-controlled systems are designed to make injections precise, efficient, virtually painless, and less expensive. Milestone’s proprietary DPS® Dynamic Pressure Sensing technology® platform advances the development of next-generation devices, regulating flow rate and monitoring pressure from the tip of the needle, through platform extensions for local anesthesia for subcutaneous drug delivery, with specific applications for epidural space identification in regional anesthesia procedures.
One in 8 U.S. women will develop invasive breast cancer over her lifetime, with approximately 266,000 new breast cancer patients and 3.1 million breast cancer survivors in 2018. Following breast cancer surgery in the adjuvant setting, a HER2/neu 3+ patient typically receives Herceptin® in the first year, with the hope that their breast cancer will not recur, and with the odds of recurrence slowly decreasing over the first 5 years after surgery. Herceptin® has been shown to reduce recurrence rates from 25% to 12% in the adjuvant setting. In the neoadjuvant setting, a patient receives treatment before surgery and based on the results of a biopsy at surgery, will receive the same or more potent treatment after surgery. Kadcyla® has been shown to reduce recurrence rates from 22% to 11% in the neoadjuvant setting. Accordingly, we believe that GP2 may be used to address the 50% of recurring patients who do not respond to either Herceptin® or Kadcyla®.
Cidara is developing long-acting therapeutics designed to improve the standard of care for patients facing serious diseases. The Company’s portfolio is comprised of drug candidates intended to transform existing treatment and prevention paradigms. Its lead Phase 3 antifungal candidate, rezafungin, will report Phase 3 data at the end of 2021. The potential peak sales opportunity for rezafungin in the US is ~$750M. In addition, the Company is developing Drug-Fc Conjugates (DFCs) targeting viral and oncology diseases from Cidara’s proprietary Cloudbreak® platform.
Genetic Technologies Limited is a diversified molecular diagnostics
company developing tools for the prediction and assessment of cancer
risk to help physicians proactively manage patient health. The
Company’s lead products, ‘GeneType for Breast Cancer’ and
‘GeneType for Colorectal Cancer’, are clinically validated risk
assessment tests that are first in their class. The Company’s
development pipeline includes new tests for COVID-19, Type 2
diabetes, cardiovascular disease, prostate cancer, and melanoma.
Listed on the ASX in 2000 and NASDAQ in 2005, Genetic
Technologies has been a leader in the development and
commercialization of genetic risk assessment technology for 20 years.
Tiziana Life Sciences is a dual-listed clinical stage biotechnology company focused on the discovery and development of novel molecules to treat human disease in oncology and immunology. Tiziana’s two lead drug candidates, Foralumab and Milciclib, uniquely target the root cause of diseases with large unmet needs in multibillion-dollar markets. Tiziana is accelerating development of anti-IL6 receptor monoclonal antibody (mAb) for treatment of COVID-19 lung inflammation. Foralumab is the only fully human anti-CD3 monoclonal antibody in clinical development in the world. This Phase 2 compound has potential application in a wide range of autoimmune and inflammatory diseases, such as nonalcoholic steatohepatitis (NASH), ulcerative colitis, multiple sclerosis, type-1 diabetes (T1D), Crohn's disease, psoriasis and rheumatoid arthritis, where modulation of a T-cell response is desirable. Tiziana is a market leader in the invention of proprietary technology for oral, nasal and inhaled formulation to transform immunotherapies with Monoclonal Antibodies currently administered intravenously. Also, Tiziana has a robust and growing patent portfolio.
SciSparc is a specialty pharmaceutical company developing unique cannabinoid technologies for treatment of central nervous system disorders. The proprietary compounds capitalize on the biochemistry of receptors that specialize in modulating the central nervous system (CNS) to create therapeutics that mitigate the adverse symptoms associated with CNS disorders.
NeoStem, Inc. (“NeoStem” or the “Company”), a biopharmaceutical company, is capitalizing on the paradigm shift occurring in medicine by engaging in the development and manufacture of cellular therapies for oncology, immunology, and regenerative medicines in the United States. The Company anticipates that cell therapy will play a significant role in the fight against chronic disease and in lessening the economic burden that these diseases pose to modern society.
Cidara is developing long-acting therapeutics designed to improve the standard of care for patients facing serious diseases. The Company’s portfolio is comprised of drug candidates intended to transform existing treatment and prevention paradigms. Its lead Phase 3 antifungal candidate, rezafungin, will report Phase 3 data at the end of 2021. The potential peak sales opportunity for rezafungin in the US is ~$750M. In addition, the Company is developing Drug-Fc Conjugates (DFCs) targeting viral and oncology diseases from Cidara’s proprietary Cloudbreak® platform.
Lantern Pharma is a clinical stage biotechnology company focused on leveraging artificial intelligence (“A.I.”), machine learning and genomic date to streamline the drug development process and to identify patients who will benefit from their targeted oncology therapies. Their portfolio of therapies consists of compounds that others have tried, but failed, to develop into an approved commercialized drug. Additionally, they develop new compounds with the assistance of their A.I. platform (RADR) and biomarker driven approach. The Company is currently developing four therapeutic programs.
Second Sight Medical Products, a recognized global leader in neuromodulation devices for blindness, announced a definitive agreement on February 7, 2022, under which privately held Nano Precision Medical (NPM) will merge with a wholly owned subsidiary of Second Sight in an all-stock transaction. NPM will be the surviving company and owned by Second Sight. The resulting company will focus on development of innovative drug and device medical implants that treat chronic diseases with high unmet medical need.
Lantern Pharma is a clinical stage biotechnology company focused on leveraging artificial intelligence (“A.I.”), machine learning and genomic date to streamline the drug development process and to identify patients who will benefit from their targeted oncology therapies. Their portfolio of therapies consists of compounds that others have tried, but failed, to develop into an approved commercialized drug. Additionally, they develop new compounds with the assistance of their A.I. platform (RADR) and biomarker driven approach. The Company is currently developing four therapeutic programs.
G Medical Innovations Holdings Ltd is a mobile health (mHealth) and e-health company. It develops and markets clinical and consumer medical-grade health monitoring solutions and offers end-to-end support for e-health projects. The company offers a suite of both consumer and clinical grade products and platforms which are positioned to reduce inefficiencies in healthcare delivery, improve access, reduce costs, increase the quality of care, and make healthcare more personalized and precise.
Aridis Pharmaceuticals is a late-stage clinical development company, leading the creation of transformative, first-in-class anti-infectives for life-threatening viral and bacterial respiratory infections. The company’s lead drug candidate for acute pneumonia met the primary endpoint for its phase 2 clinical trial and the Company is now enrolling in a global phase 3 study. Its pipeline of novel mechanism antibacterial and antivirals, sprung from its proprietary discovery technology platforms, are designed to combat the growing public health threat of viral pandemics and antimicrobial resistant (AMR) bacteria. Its anti-toxin monoclonal antibody (mAb) approach is a proven mechanism of action and has shown preliminary trend toward efficacy as an adjunctive therapy in reducing acute pneumonia patients time in ICU as compared to the current standard of care, which is antibiotics. In addition to its phase 3 program, ARDS also has a phase 2 asset for the treatment of Bacteria HAP/VAP LPS 011. The company also has an on-going phase 2 clinical trial for the treatment of Cystic Fibrosis patients, a program funded by the Cystic Fibrosis Foundation for $7.5 million. Importantly, ARDS is also preparing to launch a Phase 1/2 clinical trial in the second half of 2021 with a self-administered broadly neutralizing mAb for emerging COVID-19 mutated variants. The expansion of COVID virus strain coverage, combined with the product's self-administered, at-home treatment modality, further differentiates the company's AR-712 COVID treatment offering.
Tiziana Life Sciences is a dual-listed clinical stage biotechnology company focused on the discovery and development of novel molecules to treat human disease in oncology and immunology. Tiziana’s two lead drug candidates, Foralumab and Milciclib, uniquely target the root cause of diseases with large unmet needs in multibillion-dollar markets. Tiziana is accelerating development of anti-IL6 receptor monoclonal antibody (mAb) for treatment of COVID-19 lung inflammation. Foralumab is the only fully human anti-CD3 monoclonal antibody in clinical development in the world. This Phase 2 compound has potential application in a wide range of autoimmune and inflammatory diseases, such as nonalcoholic steatohepatitis (NASH), ulcerative colitis, multiple sclerosis, type-1 diabetes (T1D), Crohn's disease, psoriasis and rheumatoid arthritis, where modulation of a T-cell response is desirable. Tiziana is a market leader in the invention of proprietary technology for oral, nasal and inhaled formulation to transform immunotherapies with Monoclonal Antibodies currently administered intravenously. Also, Tiziana has a robust and growing patent portfolio.
Milestone Scientific Inc. (MLSS) is a biomedical technology research and development company that patents, designs, develops and commercializes innovative diagnostic and therapeutic injection technologies and instruments for medical and dental applications. Milestone's computer-controlled systems are designed to make injections precise, efficient, virtually painless, and less expensive. Milestone’s proprietary DPS® Dynamic Pressure Sensing technology® platform advances the development of next-generation devices, regulating flow rate and monitoring pressure from the tip of the needle, through platform extensions for local anesthesia for subcutaneous drug delivery, with specific applications for epidural space identification in regional anesthesia procedures.
One in 8 U.S. women will develop invasive breast cancer over her lifetime, with approximately 266,000 new breast cancer patients and 3.1 million breast cancer survivors in 2018. Following breast cancer surgery in the adjuvant setting, a HER2/neu 3+ patient typically receives Herceptin® in the first year, with the hope that their breast cancer will not recur, and with the odds of recurrence slowly decreasing over the first 5 years after surgery. Herceptin® has been shown to reduce recurrence rates from 25% to 12% in the adjuvant setting. In the neoadjuvant setting, a patient receives treatment before surgery and based on the results of a biopsy at surgery, will receive the same or more potent treatment after surgery. Kadcyla® has been shown to reduce recurrence rates from 22% to 11% in the neoadjuvant setting. Accordingly, we believe that GP2 may be used to address the 50% of recurring patients who do not respond to either Herceptin® or Kadcyla®.
Cidara is developing long-acting therapeutics designed to improve the standard of care for patients facing serious diseases. The Company’s portfolio is comprised of drug candidates intended to transform existing treatment and prevention paradigms. Its lead Phase 3 antifungal candidate, rezafungin, will report Phase 3 data at the end of 2021. The potential peak sales opportunity for rezafungin in the US is ~$750M. In addition, the Company is developing Drug-Fc Conjugates (DFCs) targeting viral and oncology diseases from Cidara’s proprietary Cloudbreak® platform.
Genetic Technologies Limited is a diversified molecular diagnostics
company developing tools for the prediction and assessment of cancer
risk to help physicians proactively manage patient health. The
Company’s lead products, ‘GeneType for Breast Cancer’ and
‘GeneType for Colorectal Cancer’, are clinically validated risk
assessment tests that are first in their class. The Company’s
development pipeline includes new tests for COVID-19, Type 2
diabetes, cardiovascular disease, prostate cancer, and melanoma.
Listed on the ASX in 2000 and NASDAQ in 2005, Genetic
Technologies has been a leader in the development and
commercialization of genetic risk assessment technology for 20 years.
Tiziana Life Sciences is a dual-listed clinical stage biotechnology company focused on the discovery and development of novel molecules to treat human disease in oncology and immunology. Tiziana’s two lead drug candidates, Foralumab and Milciclib, uniquely target the root cause of diseases with large unmet needs in multibillion-dollar markets. Tiziana is accelerating development of anti-IL6 receptor monoclonal antibody (mAb) for treatment of COVID-19 lung inflammation. Foralumab is the only fully human anti-CD3 monoclonal antibody in clinical development in the world. This Phase 2 compound has potential application in a wide range of autoimmune and inflammatory diseases, such as nonalcoholic steatohepatitis (NASH), ulcerative colitis, multiple sclerosis, type-1 diabetes (T1D), Crohn's disease, psoriasis and rheumatoid arthritis, where modulation of a T-cell response is desirable. Tiziana is a market leader in the invention of proprietary technology for oral, nasal and inhaled formulation to transform immunotherapies with Monoclonal Antibodies currently administered intravenously. Also, Tiziana has a robust and growing patent portfolio.
1. Issuer Free Writing Prospectus
Filed Pursuant to Rule 433
Registration No. 333-201731
January 28, 2015
Opexa Therapeutics, Inc.
NASDAQ: OPXA
January 2015
The Woodlands, TX
Precision Immunotherapy®
2. 2
Free Writing Prospectus Statement
• This presentationhighlights basic informationabout us and the offering. Beinga summary document, this
slide deckdoes notcontainall theinformation that youshould considerbeforeinvesting.
• We have filed a registration statement (including a preliminary prospectus) with the SEC for the offering
to which this presentation relates. The registration statement has not yet become effective. Before you
invest, you shouldread the preliminary prospectusin the registration statement(including the risk factors
described therein) and other documents, including the Company's Form 10-Ks and Form 10-Qs, that we
have filed with the SEC for more complete information about us and the offering. You may get these
documents for free by visiting the “Search EDGAR” section on the SEC web site at http://www.sec.gov.
The preliminary prospectus, dated January 28, 2015, is available on the SEC website. Alternatively, we or
the dealer-managers for this offering, Maxim Group LLC and National Securities Corporation, will
arrange to send you a preliminary prospectus if you contact Maxim Group LLC, Prospectus Department,
405 Lexington Ave.,NewYork,NY, 10174;Telephone:(212)-895-3745; Email:syndicate@maximgrp.com.
3. 3
Forward-Looking Statements
All statements inthis presentationother than those of historical fact, including statements regarding our preclinicaland 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 negativeversion 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 anticipatedor implied insuch 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 andthe timingofevents coulddiffer materially from those anticipated as a result ofvariousrisks anduncertainties
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.
Althoughwe believe ourexpectationsare reasonable,we donot inany way guarantee futureresults, levelofactivity, performance
or achievements. In addition,neither we nor any other personassumes responsibility for the accuracy and completeness of any
forward-looking statements. Our forward-lookingstatements inthis presentation speak only as of the date this presentation is
actually delivered by us in person. 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 additionaldisclosures we make that further describe risks and uncertainties relevantto us inadditional
detail in our filings with the Securities and Exchange Commission, including our registration statement on Form S-1 initially
filed on January 28,2015, as wellas in ourAnnual Report on Form 10-K, Quarterly Reports on Form 10-Q and Current Reports
onForm 8-K. You may get these documentsfor free byvisiting EDGAR onthe SECweb site at http://www.sec.gov.
4. 4
Opexa Investment Highlights
PIPELINE
• Tcelna®: Phase 2b for secondary progressive multiple sclerosis (SPMS), limited
competition, $7BN overall market potential, encouraging prior trials (Phase IIb RRMS,
Phase I/II SPMS)
• OPX-212: Pre-IND for neuromyelitis optica (NMO), no approved therapies, orphan
indication
TECHNOLOGY
VALIDATION
• Precision immunotherapy for T-cell-mediated autoimmune diseases
• Proprietary Platform: potential to yield multiple candidates personalized to each patient
• Company owned and scalable cGMP manufacturing facility
• Strong patent estate (98 domestic and international)
• Option agreement secured with Merck Serono for Tcelna – strong potential partner
• FDA Fast Track designation for Tcelna in SPMS
• Esteemed Scientific Advisory Board
5. 5
Offering Summary
Issuer
Exchange/Ticker
Structure
Offering size
Subscription Price
Warrant structure
Oversubscription privilege
Use of proceeds
Co-managers
Opexa Therapeutics, Inc.
NASDAQ / OPXA
Rights Offering
Up to 28,776,419 Units
Each Unit composed of one share of common stock and one warrant
TBD
Range expected $0.65-$0.75
Exercise price: $0.50 per share from issuance through June 30, 2016;$1.50 per share from
July 1, 2016 through 3-year expiration
Redeemable for $0.01 per share upon certain conditions
Exercisable for cash unless no effective registration statement
Allocated pro rata up to available Units not purchased by other
holders, subject to limitations
Funding of ongoing Phase IIb Abili-T study in SPMS patients
Funding of preclinical and/or clinical development for OPX-212 in NMO
For general corporate purposes and operational purposes
Maxim Group
National Securities Corp.
6. 6
Opexa Investment Thesis
Why a Rights Offering?
• A successful rights offering would give Opexa sufficient capitalto fund operations through2016.
– Completethe ongoingPhase2bclinicaltrialin SPMSandreleasedata
– Potentially initiate a Phase 1/2 trial with OPX-212 in neuromyelitis optica(NMO)
Background
• Opexa haspreviouslyraised over $90 million, with most being invested in its T-cell immunotherapyplatform.
• Opexa hasconducted two Phase 1 trials and is currently conducting its second Phase 2 trial having treated both RRMS
and SPMS patients with Tcelna. Over 400 individuals in total have been enrolledin Opexa’s clinical trials for Tcelna,
including the ongoing Phase 2b SPMS trial.
• Current Phase 2b trial is designed to show whether Tcelna could improve clinical outcomes -data expectedin 2H2016.
– Enrollment of 190 patients in a randomized, placebo-controlled trial completed
– The Company may apply for accelerated approval and breakthrough designation with the FDAfollowing the completion of the
currentPhase2b trialin theeventofpositiveresults(primaryendpoint-wholebrainatrophy;secondary-progression)
• Fast Track Designation obtained from the U.S. FDAfor Tcelna in SPMS.
• In addition, the Company has further leveraged its platform to move into neuromyelitis optica (NMO), an orphan (rare)
disease with no FDA-approved treatments. This program is in the pre-IND stage.
• Immunotherapyapproaches have been validated and are increasingly being applied in oncology.
• Opexa hasbeen refining its approach for over a decade focusing on autoimmune diseases, specifically, Tcelna for MS.
• Based, in part, ondata generated in prior MS trials, Merck Serono, a leading pharma companyin MS, entered into an
option andlicense agreement with Opexa to potentially license Tcelna for MS, worldwide, excluding Japan. Should
Merck Serono exercise its option, they would fund any future Phase III studiesand commercial activities withpotential
milestone payments and royalties coming to Opexa upon success ofthe program.
8. 8
The Root Cause of Multiple Sclerosis
Adapted by permission from Macmillan Publishers Ltd: NATURE REVIEWS
IMMUNOLOGY 3, 483-492 (June 2003), copyright (2003)
Cytokines
Damage
Damage
• MS is an inflammatory disease in which the
insulating cover of the nerve fibers (the
myelin sheath) in the brain are damaged by
the body’s own immune system. There is no
cure for MS.
• In MS patients, the faulty immune system is
unable to prevent the attack of myelin
reactive T-cells (MRTC)
• MRTC cross the blood brain barrier, enter
the brain leading 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
ActivatedT- cells degradeMyelinand
DamageMyelinProducing Cells
9. 9
Tcelna CouldAddressthe Root Cause ofMultipleSclerosisby
Preventing DemyelinationandEnabling 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 only the damaging T-
cells (MRTC), thereby inhibiting further
destruction of the myelin sheath and
potentially enabling remyelination
• Immune cells, including Tregs, have been
primed, or sensitized to specifically target
the pathogenic MRTC for elimination or
regulation
• Elimination of harmful MRTC may lead to:
o Stabilization of disease by preventing
further destruction of myelin
o Improvement in condition by allowing
Oligondendroglial cells to remyelinate
axons (neuroprotection)
10. RESULTS AND EXPLANATION OF RRMS &RESULTS AND EXPLANATION OF RRMS &
SPMS CLINICAL TRIALSSPMS CLINICAL TRIALS
10
11. 11
Business Strategy and Rationale for Pursuing
Secondary Progressive MS (SPMS)
• Opexa completed a Phase 2b clinicalstudy inRRMS patients
– Although the primary MRI endpoint was not met, the clinical signal was encouraging
– In a subset of more active RRMS subjects, patients treated withTcelna saw a statistically
significant improvement in disability, and the therapy was safe and well tolerated
– Opexa completed formal End of Phase 2 meetings with the FDA and obtained support to move
into Phase 3 studies in RRMS
• Inseparate Phase I/II trials in SPMS patients, 35 subjects treated with
Tcelna showedencouragingresults
– 80% of patients showed no disease progression following two yearsof treatment
– Substantial reduction in relapse rate following two years of treatment
• One relapse observed in 21 years of cumulative patient follow up
– No worsening in physical or psychological condition following two years of treatment
– Well tolerated, no Serious Adverse Events
• StronginterestfromKeyOpinionLeaders,clinicalinvestigators,patients
and potentialpartners toadvanceinSPMS
• Substantialunmet medicalneedinSPMS
– Only one product currentlyapproved for SPMS indication with limited use due to cardio toxicity
• Novel,personalizedT-cellapproachdifferentiatesOpexa fromcompetition
13. 13
Phase 2b RRMS Prior Clinical Trial
Reversal of Disability in Prospective Analysis in More Active Patients
Sub-population of patients(n=50)withmoreprogressed/activedisease
profile(baselineARR >1)
Statistically Significant
Improvement inDisability (p=0.045)
55%ReductioninARR
Change in Disability (EDSS)
at Week 52Annualized Relapse Rate (ARR) at week 52
2.4
2.23
2.2
2.39
2.10
2.15
2.20
2.25
2.30
2.35
2.40
2.45
Baseline Week 52
Tcelna Placebo
(p=0.045)
0.28
0.63
0
0.1
0.2
0.3
0.4
0.5
0.6
0.7
Tcelna
Placebo
55%
14. 14
Phase I/II SPMS Prior Clinical Trials
Tcelna Stabilizes Disease in SPMS at 2 Years
80% ofpatients showed no further disease progression by EDSS at 2years
Historical Disease
Progression
Tcelna Open Label
(n=35)
*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%
Stabilization vs. Historical Progression
Stable
Progressed
15. 15
Tcelna in Secondary Progressive MS
•Secondary Progressive MS market
potential in North America could
exceed $7billion (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
SPMS
450,000Individuals in NorthAmericadiagnosedwithMS;
30-45%potentially haveanSPMSdiagnosis
Market Size:
$7 Billion (est.)
Relapsing Remitting MS;
ClinicallyIsolated Syndrome;
Primary ProgressiveMS
16. 16
Tcelna
Lead Program Targeting Secondary Progressive MS patients
Fast Track Designation by FDA
• Phase 2btrialis fully enrolled: 190patientswith SPMS
• Top line dataexpectedin 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 inparallel
– Comprehensive biomarker analysis
17. 17
Merck Serono Agreement
Agreement signed 2013; strong potential partner
Option andLicenseAgreement forworldwiderights toallMultiple
Sclerosisindications,excludingJapan
• If Merck Seronoexercisesoption:
– Merck Serono to fund any Phase 3, pre-commercial and commercial activities
– Merck Serono obtains rights to develop Tcelna for all MS indications
– Worldwide rights excluding Japan
• Opexareceived$5 millionupfront optionfeeat signing
• Opexahaspotentialtoreceiveadditional$220millionin optionexerciseand
payments
• Opexahaspotential toreceiveroyaltiesranging from8%to15%ofannualnet sales, with
step-upsoccurringif net sales exceed $500million, $1B& $2B
• Opexamaintains 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
19. NMO: A Rare Disease with High Unmet Need and
Significant Market Potential
• Neuromyelitis optica (NMO) is a rare or orphan autoimmune
disease, affecting the optic nerves and the spinal cord
• There are no FDA-approved therapies for NMO
• Any therapy that could achieve tolerization of the AQP-4
reactive T-cells may offer a cure for NMO
• Opexa retains world wide rights to OPX-212. Product has
potential to generate $300-$500 million in peak sales, subject
to successful clinical development
• IND filing expected in Mid 2015
• Open label Phase I/II clinical trials expected to start in 2H
2015 (subject to IND acceptance and financing)
An orphan disease with no FDA-approved therapy
Image reprinted withpermission from MultiView, Inc.
19
20. 20
Recent and Upcoming Expected Milestones
2016 20172014 2015
1H 2017: Top lineresults
expected from Phase I/II trial
withOPX-212 inNMO (subject
to IND acceptance andfinancing)
Mid-2015: File
IND/CTAwith US FDA
and/or Canadian health
authorities
2016/17: Apply for orphan
and fast track designationfor
OPX-212 in NMO
1H 2014: Complete
enrollment in Phase
IIb SPMS study
2H 2014: Initiate
second disease
indication: NMO
2H 2015: Initiate Phase I/II
clinical study with OPX-212 in
NMO (subject to IND
acceptance and financing) 2H 2016: Results from
Immune Monitoring
(biomarker) program
expected
2H 2016: Top Line
resultsexpected in
Phase IIb SPMS trial
20
21. 21
Experienced Management Team and Board of Directors
NeilWarma,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 subsequentlyacquired
KarthikRadhakrishnan,ChiefFinancialOfficer
› 10+ years of health care capital markets experience
› Formerly, Vice President at ING Investment Management
› MBA, MS in Engineering, CFAcharter holder
DonHealey,Ph.D.,ChiefScientific Officer
› 25+ years of experience in cellular immunology and immune regulation
› Former Director of Immunology, ArgosTherapeutics
Donna Rill,ChiefDevelopment Officer
› 30 yearsin cell and gene therapy research and clinical application
› Designed andvalidatedcGMP Cell & Gene Therapy Laboratories, Vector
Production facilities, and Translational Research Labs
KennyFrazier,VP of ClinicalDev.and Regulatory
Affairs
› 25+ years of extensive clinical and regulatory experience
› Formerly, Headof Clinical Operations, Lexicon Pharmaceuticals and Tanox,
Inc.
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, BayBio, Former CEO of Five
Prime Therapeutics, Founder of
Genzyme Molecular Oncology
Michael S. Richman
CEO, Amplimmune
ScottB. Seaman
Executive Director,
Alkek Foundation
Neil K. Warma
President & CEO, Opexa
22. 22
SPMS Scientific Advisory Board
DawnMcGuire,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
ClydeMarkowitz,M.D.
• Director of MS Center at the University of Pennsylvania
DougArnold, M.D.
• James McGill Professor Neurology and Neurosurgery at the Montreal Neurological Institute
EdwardFox,M.D., Ph.D.
• Director of Multiple Sclerosis Clinic of Central Texas
• Advisory Committee, Lone Star Chapter of the National Multiple Sclerosis Society
25. 25
Personalized T-cell vaccines
Consisting of Attenuated Antigen Specific T-cell Clones
• Opexa identifies the exact T-cell
responsible for T-cell-mediated
autoimmune damage, expands the T-cells
in cGMP facility
• Therapeutic dose of attenuated T-cell
clones is injected subcutaneously
• Immune system recognizes the large
volume of cells (30-45 million) under the
skin as potential foreign pathogens
• Attenuated T-cells believed to trigger an
immune response specifically targeting
circulating pathogenic reactive T-cells
Tcelna: lead product candidate in Phase 2b
trial for secondary-progressive multiple
sclerosis
Tcelna®
Personalized T-cell Vaccine
26. 26
Business Differentiator: Manufacturing Process
Designed for commercialization, Owned and managed by Opexa
Cryopreservation
Formulation/
Irradiationofeach
doseasrequired
Administration: 5
subcutaneous
injections/year
Manufacturing and QC Dispensation
35 days
Epitope Profiling
1 day14 days
- Red Cross
- Blood Group Alliance
EpitopeProfiling ExpansionofantigenspecificT-cells
Annual course of treatment is 5 doses manufactured from a single procurement
27. 27
Comprehensive ProcesstoDetermine Optimal Additional
IndicationsBeyondSecondary-Progressive MS
EvaluationCriteria
Short List of
Diseases
PotentialDisease
Targetsoff Platform
First Disease Target
NMO
• Unmet medical need
• Competitive landscape
• Disease incidence
• Single antigen immunity
• Multiple antigen immunity
• Likely duration of Phase 1/2 clinical trial
• Likely cost of Phase 1/2 clinical trial
• Clinical endpoint
• Efficacy biomarker known
• Animal model available
Addison’sdisease
Autoimmunehemolyticanemia
Celiac Disease
Goodpasture’ssyndrome
Grave’sDisease
Hashimoto’sthyroiditis
Idiotypicthrombocytopenicpurpura
MyastheniaGravis
NeuromyelitisOptica (NMO)
Pemphigusvulgaris/foliaceus
Primary Biliary Cirrhosis
Rheumaticheartdisease
Systemiclupuserythematosus
Sjögren’ssyndrome
Type 1Diabetes