Genprex, Inc. · NASDAQ:GNPX
Genprex — Investor Presentation (September 2023)
52 slides from Genprex's investor presentation, Sep 8, 2023
Slide 1: Pioneering Gene Therapies for Patients in Need
€ ) Genprex’ Pioneering Gene Therapies for Patients in Need September 2023
Slide 2: Forward-Looking Statements
Forward-Looking Statements ‘Statements contained in this presentation regarding matters that are not historical facts are “Yorwardlooking statements within the meaning of the Private Secures Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual resuts
Slide 3: Our Mission
Our Mission Advancing novel gene therapies for patients afflicted with cancer or diabetes.
Slide 4: Program Highlights
Program Highlights ONCOLOGY DIABETES iS) Non-viral gene therapy platform @ Addressing both Type 1 and Type 2 diabetes with AAV gene therapy Novel approach using systemic gene therapy to replace tumor suppressor genes for cancer in @) Novel infusion process delivers genes to pancreas humans Potential
Slide 5: Research and Development Pipeline
DiaBeres. Research and Development Pipeline | I |
Slide 6: Oncology
QO ONCOLOGY REPROGRAMMING THE COURSE OF CANCER or Yo) www.genprex.com
Slide 7: Tumor Suppressor Genes Deleted During Cancer Development
Tumor Suppressor Genes Deleted During Cancer Development Why TUSC2? © Tumor suppressor genes are deleted early during cancer development © 82% of allnon-small cell lung cancers and 100% of all small-cell lung cancers express decreased amounts of TUSC2, tumor suppressor protein © Loss or reduction of
Slide 8: REQORSA Targets Cancer At Its Core
REQORSA® Targets Cancer At Its Core | Controls Cell Signaling Pan-kinase inhibition decreases cancer cell proliferation Stimulates Apoptotic Pathways Leads o programmed cancer cell death Multiple anti-cancer mechanisms of action. 6 Modulates Immune Response Promotesimmune activity against cancer
Slide 9: Our Cancer Treatment Approach
Our Cancer Treatment Approach Tumor suppressor genes are deleted early during cancer development. Our method of treating cancer is to reintroduce tumor suppressor genes to patients. Tumor Suppressor Gene in a DNA Plasmid We have rights to tumor su have cancer-fighting functions. expressed ina DNA pl
Slide 10: ONCOPREX Nanoparticle Delivery System: Novel Platform to Treat Cancer
Novel Platform to Systemic Gene Therapy Platform: ONCOPREX® Nanoparticle Delivery System Treat Cancer Genes Allows for delivery of TUSC2 and NPRL2 genes and potentiallya variety of other genes Synergies © Can be used in combination with other cancer therapies suchas Tagrisso®, Keytruda® and Tecentri
Slide 11: Oncoprex Nanoparticle Delivery System
® . Oncoprex Nanopa rticle Non-viral, positively-charged lipid Delive ry Syste’ m nanoparticle (LNP) is systemically delivered. Plasmid containing umor Tusc2 suppressor jfene | REQORSA® IMMUNOGENE THERAPY oe ene > a gene protein Plasmid with TUSC2 gene. zi a4 Cationic lipid nanoparticle carries drug
Slide 12: Selective Uptake of REQORSA Targets Cancer Cells
Selective Uptake REQORSA Targets Cancer Cells of REQORSA® Pretreatment Biopsies Posttreatment Biopsies REQORSA is designed to deliver t functioning TUSC2 gene to cancer cells while minimizing its uptake by normal tissue. Patient 1 (02mafka) * Tumor biopsy studies conducted at MD Anderson show that,
Slide 13: Lung Cancer: By the Numbers
Lung Cancer: By the Numbers 127K $48B 18.6% e e e e U.S. Annual Global Lung Global U.S. Lung Average Lung Cancer Cancer Incidence Marke Cancer Incidence Survival Rate milion Global markStis projec More than 238,000 new The lungcancer five-year newcasesperyear togrowfrom$183billion In casesper year i
Slide 14: Drug resistance to Tagrisso, Keytruda and Tecentriq is inevitable
For most patients, drug resistance to Tagrisso®, Keytruda® and Tecentriq® is inevitable. Our approach is designed to address drug resistance. REQORSA® Immunogene Therapy may be complementary with targeted drugs and immunotherapies. REQORSA's multimodal activity may block emerging bypass pathways, th
Slide 15: AACR 21: REQORSA + Tagrisso
AACR 21: REQORSA® + Tagrisso Reduce Tumor Growth in Tagrisso Resistant Tumors Enhanced Anti-Tumor Activity oe osimetnibsTUSC2 combination on HIO75-03iR tumors in NSG mice REQORSA in combination with 250 Tagrisso demonstrated = significantly increased anti-tumor 7 efficacy in EGFR mutant Tagrisso 100
Slide 16: REQORSA + Tagrisso Phase 1/2 Trial (FDA Fast Track)
© ~15-20 US. sites © ~158 patients : FDA Fast Track Designation Patients with advanced, EGFR mutant NSCLC whose disease progressed after Tagrisso® © Phase 1 Dose Escalation:~12-18 patients (completed) © Phase 2 Expansion: ~66 patientsin 2 cohorts o Phase 2: ~74 patients Phase 2 Expansion cohort inte
Slide 17: REQORSA with Immunotherapies
REQORSA® with Immunotherapies Percent Survival Survival on Lung Metastasis Model — Control 200) — PD1+CTLAG REQORSA REQORSA+PD1 — REQORSA+PD1+CTLAS a> 50 0 10 15 20 25 Days REQORSA+anti-PD1 exhibit greater anti-tumor activity than either agent alone or control. REQORSAtanti-PD1 combination significa
Slide 18: REQORSA + Keytruda
REQORSA® + Keytruda® REQORSA increasesimmune response Significantly Reduced Tumor EAU BI i Growth REQORSA® + Keytruda® The independent immunologic effects of REQORSA and Keytruda markedly decrease tumor growth by increasing the immunologic attack on the tumor compared to PD-1 inhibition alone.
Slide 19: AACR 21: REQORSA May Enhance First-Line Standard of Care
AACR 21: Regorsa® May Enhance First-Line Standard of Care REQORSA® + Keytruda® + Chemo ‘ipl tretment on AB4S hing metin bunsnizad mice “Tumor intensity (Wk) post treatment x10" > No treatment REQORSA 4x10" | + CarborPemro + CarbsPemrosREQORSA, REQORSA enhances the efficacy of chemo-immunotherapyon K
Slide 20: REQORSA + Keytruda Phase 1/2 Trial
PHASE 1 DOSE ESCALATION FDA Fast Track Designation Patients with advanced NSCLC whose disease progressed after tit: treatment with Keytruda® | tl o ~10US. sites “guste Acclai 2 © Phase 1 Dose Escalation: Up to 18 patients © Phase 2 Expansion: 36 patients © Phase 2 Randomized: 126 patients Phase 2 in
Slide 21: REQORSA in Small Cell Lung Cancer
Regorsa® in Small Cell Lung Cancer Targeting Small Cell Lung Cancer (in addition to NSCLC) allows Genprex® to address virtually the entire lung cancer market. Small Cell Lung Cancer: © Consistently has low TUSC2 protein levels Documented to often have deletion of at least one TUSC2 gene allele. © Ex
Slide 22: SCLCs Express Low Levels of TUSC2 Protein
SCLCs Express Low Levels Of TUSC2 Protein IHC analysis of tumor specimens Since all SCLCs have reduced or no TUSC2 41% of SCLC have no TUSC2 protein expression protein expression, re-expressing TUSC2 100% of SCLC have reduced or no TUSC2 protein protein may lead to clinical efficacy. expression Fusi
Slide 23: Atezolizumab (Tecentriq) SCLC Approval Trial
Atezolizumab (Tecentriq®) SCLC Approval Trial IMpower133 Study Adding Tecentriq to standard therapy improves survival in SCLC and establishes a new standard therapy for ES-SCLC. © Untreated, extensive stage SCLC © Carboplatin & etoposide chemotherapy + atezolizumab or placebo + 4cycles, then atezoli
Slide 24: Atezolizumab Maintenance Therapy
Atezolizumab Maintenance Therapy Once patients begin maintenance therapy with Tecentriq, Progression Free Survival is very short (2.6 mos). © Atezolizumab vs placebo + AILCR, PR, and SD patients received maintenance therapy + Endpoints measured from the start of maintenance therapy PFS 2.6 vs 1.8 mo
Slide 25: Fast Track and Orphan Drug Designation: REQORSA + Tecentriq
Fast Track Designation and Orphan Drug Designation Patients with ES-SCLC who did not develop tumor progression . after receiving Tecentriq® and chemotherapy A | 3 “ous cc aim ~62 patients © Phase 1 Dose Escalation: Up to 12 patients © Phase 2: ~50 patients Phase 2 futility analysis after 25th patien
Slide 26: AACR 23: NPRL2 Induces Anti-tumor Activity in NSCLC
AACR 23: NPRL2 Induces Anti-tumor Activity in NSCLC Further Evidence of ONCOPREX® Delivery System as a Platform for Treatment Using Tumor Suppressor Genes Study investigated the antitumor responses to NPRL2 gene therapy on anti-PD1 resistant KRAS/STK11 mutant NSCLC in a humanized mouse model Humaniz
Slide 27: Diabetes
eo DIABETES www.genprex.com
Slide 28: Diabetes: By the Numbers
Diabetes: By the Numbers 37M S966B $24B e e e e Diabetes U.S.+ Diabetes Global? Global Diabetes | T2D Global Market T1D Global Market Prevalence Prevalence Expenditures (6.7% CAGR?) (17.2% CAGR*) 643M by by 2045, $57B expected US, ‘market sales in 2029. prediabetes. Caribbean
Slide 29: Diabetes can cause serious complications
Diabetes can cause serious complications. In 2021, there was approximately 1 death every 5 seconds caused by diabetes worldwide.
Slide 30: Diabetes Causes Serious Complications
Diabetes Causes Serious Complications Heart Disease Leading cause of death for men and women in US. Diabetics are 2x as likely to have heart disease or a stroke. Chronic Kidney Disease Approximately 1 in 3 adults with diabetes have CKO. Kidney diseases are the 9th leading cause of deathin US. Nerve
Slide 31: Diabetic Patients Are In Need of Advanced Therapy
Diabetic Patients Are In Need of Advanced Therapy Islets of Langerhans produce insulin and Recombinant DNA techniques the destruction of produce these cells resulted synthetic indiabetes human” insulin Insulin first introduced to Type | diabetes 1901 ‘patient - ° 1978 The most significant advancemen
Slide 32: Novel Gene Therapy Collaboration with University of Pittsburgh
Novel Gene Thera py Collaboration with University of Pittsburgh Diabetes Program intends to address both T1D and T2D. 37.3M or 11.3% of Americans Have Diabetes* Type 1 Diabetes (5-10%) ——————e e———— Type 2 Diabetes (90-95%) An auto-immune condition where the body's immune system destroys pancreatic
Slide 33: GPX-002 Replenishes Levels Of Insulin
GPX-002 Replenishes Levels Of Insulin Delivers Genes to the Pancreas ‘an AAV vector with to deliver pancreas. A novel infusion process: the Pdxt + MafA(PM) g Reprograms Alpha Cells transforms alpha cells Restores Blood Glucose Levels in vivo, preclinical studies show that GPX-002 restored normal blo
Slide 34: Reversed Drug-Induced Diabetes in T1D Toxin-Induced Mouse Model
Reversed Drug-Induced Diabetes in T1D Toxin-Induced Mouse Model Blood glucose (mala) beta cell maze (mg) aitoxan AAV-8 injection Reprogramed alpha cells into functional beta cells. Normalized blood glucose in beta cell-toxin-induced diabetic mice
Slide 35: Restored Blood Glucose in T1D Autoimmune Mouse Model
Restored Blood Glucose in T1D Autoimmune Mouse Model for Four Months The duration of restored blood glucose levels in mice could potentially translate to decades in humans. 10 600 One week in a mouse tends to fa ave see. correlate to about one year in humans. Blood glucose (mg/l) beta cell mass (ms)
Slide 36: Induced Generation of Functional Insulin Expressing Cells from Alpha Cells
Induced Generation of Functional Insulin Expressing Cells from Alpha Cells in Human Islets Provides a potential basis for further investigation in human Type 1 diabetes Human islets treated with streptozotocin 600 m to destroy beta-cells, then treated with either AAV-PM or AAV-GFP 8 > AAV treatedisl
Slide 37: Non-Human Primate Model of T1D
Non-Human Primate Model of T1D Reduced Insulin Requirements == ‘Wnsulin / Carb i: ; Ll mm fr lili Z iso Veatinton NP? srsuin care S| il re ii Preliminary data from University of Pittsburgh researchers show a marked reduction in insulin requirements and stabilization of glucose levels.
Slide 38: ATTD 23: Statistically Significant Decreases In Insulin Requirements in NHPs
ATTD 23: Statistically Significant Decreases In Insulin Requirements in NHPs Following the pancreatic intraductal infusion of the AAV engineered construct, the eight NHPs had: © Decreased insulin requirements (p<0.001) © Increased c-peptide levels (p<0.05) Improved glucose tolerance comparedto basel
Slide 39: Corporate
a) CORPORATE www.genprex.com
Slide 40: Our Team: Company Management
Our Team: Company Management Rodney Varner, JD Chairman President & Chet Executive Offcer Catherine Vaczy, JD Executive VP, General Counsel Chief Strategy Officer C2 ” Ryan Confer, MS Chief Financial Officer Mark S. Berger, MD ‘Chief Medical Offcer corporat biotech industry 20+ years of experenceln
Slide 41: Our Team: Company Management
Our Team: Company Management
Slide 42: Our Team: Scientific Advisory Board
Our Team: Scientific Advisory Board Tony S. K.Mok IMO, FRCP(O), FHKCP, FHKAM Jack A. Roth MD, FACS. Chaieman MO, Pho Professor and Chair of Clinica of Paburgh; recor ofthe Richard King Mellon lector of he Mote Cancer Center Advent Health oie Section of Thoracic Molecular Scientific Oirectorof the Ci
Slide 43: Our Team: Clinical Advisory Board
Our Team: Clinical Advisory Board \ i) Michael Morse ‘Andrew Becker Col. George Peoples MD, MHS, FACP MO, PhO MO. FACS
Slide 44: Our Team: Board of Directors
Our Team: Board of Directors Jose A, Moreno Toscano Board Director Rodney Vamer, JD Chairman of the Board William R. Wilson. J Board Director Brent Longnecker Board Director a James E Rothman, PhD Strategie Adwsor tothe Board Chief Execatve Ofc, LFB USAlne 30+ yearsofexperssein corporate governance,
Slide 45: GNPX: At a Glance
>| GNPX: | At a Glance @ Company Symbol Exchange Share Price Senprex, GNPX Nasdaq $0.44 Market Cap 30-Day Avg Vol Shares Outstanding Cash $26M 892K 59,409,822 $9.9 M* All data areas of August 21 hich are as of August 10, 2023 and cash whichis on uly 21, 2023 we completed aregistered dir
Slide 46: Achievements & Upcoming Milestones
Achievements & Upcoming Milestones Acclaim-1 Acclaim -2 Acclaim -3 D complete Y File for Orphan enrollment for Drug Designation Phase 1 Dose with the FDA Escalation portion in. 22024 1D Dose first patient in Phase 1 Dose Escalation portion in 942023 Present preclinical Present full data data on REQO
Slide 47: We believe in a future of transformational patient care
We believe in a future of transformational patient care. 21% Century Large Combination Three FDA Gene Markets & Trials with Top Fast Track Therapies Unmet Need | SellingDrugs | Designations Exploring New Indications & Partnerships
Slide 48: Slide 48
Slide 49: Research References
Research References Slide 12 1, Selective and Preferential Cancer Cell Uptake and Anti-Cancer Actity In ‘Advanced Lung Cancer Patients. Lu etal. PLOS1 (2012) Slide 13 1. American Cancer Society (2023): hitps:/bi V/3SPCMRI 2. World Health Organization (2022): https bit N/3fLGVS! 3. Fortune Business i
Slide 50: Research References
Research References Slide 32 1. Centers for Disease Control: httosu/wmnvcdcgov/dlabetes/data/satsics: repocvinde him Slides 33-36 1. Xia0X, Guo P, Shiota C. et al. Endogenous Reprogramming of AlphacCellsinto eta Cells, nduced by Viral Gene Therapy, Reverses Autoimmune Diabetes. Cell ‘Stem Cell, 2018
Slide 51: Appendix
a APPENDIX www.genprex.com
Slide 52: ONC-001 & ONC-002
ONC-001 & ONC-002 DOSE ESCALATION STUDY (ONC-001) Favorable safety profile after being treated in more than 50 patients. Metabolic responsesiin late-stage metastatic lung cancer patient REQORSA® Monotherapy & REQORSA® + Tarceva Combination REQORSA® + TARCEVA (ONC-002) Ph 2 data in subjects with or w