Genprex, Inc. · NASDAQ:GNPX
Genprex — Investor Presentation (January 2025)
61 slides from Genprex's investor presentation, Jan 13, 2025
Slide 1: Pioneering Gene Therapies for Patients in Need
Pioneering Gene Therapies for Patients in Need January 2025
Slide 2: Forward-Looking Statements
Forward-Looking Statements Statements contained ints presentation regarding matters tat ae not hisorka fas ae Torwareookng taterens" with the meaning ofthe Penate Secures Lidgaton Reform Ac of 195, Because such statements are subject sks ang uncertainties, actual resus may afer materially from tose
Slide 3: Our Mission
Our Mission Advancing novel gene therapies for patients afflicted with cancer or diabetes. © Genprex
Slide 4: Program Highlights
Program Highlights ONCOLOGY DIABETES @) Non-virat 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 a Demons
Slide 5: Research and Development Pipeline
Research and Development Pipeline > Prcorsrnws =; —_—=» - = — —_, [>= —==yp = © Genprex
Slide 6: Oncology
Q ONCOLOGY REPROGRAMMING THE COURSE OF CANCER 000 www.genprex.com
Slide 7: Why TUSC2?
Why TUSC2? Discovery — Tumor Suppressor Candidate 2 ——~ [Chromosome 3p21.3 deleted ene —— Previously called FUS1 ———e NPRL2 is in the same area of the chromosome © Genprex Tumor Suppressor Gene TUSC2 restoration in cancer cells in vitro inhibits cell growth and induces apoptosis TUSC2 is encoded by
Slide 8: Tumor Suppressor Genes Deleted During Cancer Development
Tumor Suppressor Genes Deleted During Cancer Development ——* Tumor suppressor genes are deleted ‘early during cancer development ——» 82% of all non-small cell Lung cancers. and 100% of all small-cell tung cancers ‘express decreased amounts of TUSC2 ‘tumor suppressor protein © Loss or reduction of TU
Slide 9: Oncoprex Non-viral Delivery System
® Oncoprex Non-viral, positively-charged lipid-based Delivery System nanoparticle in a lipoplex form is systemically delivered. NON-VIRAL DELIVERY OF TUMOR SUPPRESSOR GENE O cenprex Cationic lipoplex carries drug to tumors.
Slide 10: 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. © Genprex ‘Tumor Suppressor Gene in a DNA Plasmid We have rights to tumor suppressor genes that may have cancer-fighti
Slide 11: Novel Platform to Treat Cancer
Novel Elation to aa olan raralneliv eo syaeo reat Cancer Genes Allows for delivery of TUSC2 and NPRL2 genes and potentially a variety of other genes Synergies Can be used in combination with other Cancer therapies such as Tagrisso® and Tecentriq® Cancers Could combat multiple cancers including NSCLC
Slide 12: Reqorsa Targets Cancer At Its Core
Reqorsa® Targets Cancer At Its Core { | eS Controls Cell Signaling Tyrosine kinase inhibition decreases cancer cell proliferation © Genprex Stimulates Apoptotic Pathways Leads to programmed cancer cell death Muttiple anti-cancer mechanisms of action. a) Modulates Immune Response eo Promotes immune a
Slide 13: Reqorsa Reduces Glycolysis in Cancer Cells
Regorsa® Reduces Glycolysis REQORSA Reverses Fundamental in Cancer Cells Characteristic of Cancer Glycolyic Function ©. asiocan gee Cancers are detected by PET scans ee PET scanning is based on increased glucose uptake in cancer cells Due to high rate of glycolysis in cancer cells 400 Oligo Glucose
Slide 14: Lung Cancer: By the Numbers
Lung Cancer: By the Numbers 125K S48B 25% U.S. Annual Global Lung Global U.S. Lung U.S. Average Mortality Cancer Incidence Market Cancer Incidence Lung Cancer Survival More than 125,000 ‘More than two million Global markets projected More than 234,000 new Rate deaths newcases peryear _togrowfrom $18
Slide 15: Reqorsa Monotherapy ONC-001 Trial
Regorsa® Monotherapy ONC-001 Trial DOSE ESCALATION STUDY Explored toxicity and tolerability in patients. Phase 1 monotherapy results: + 31 Stage IV lung cancer patients + 0.01 - 0.09 mg/kg * 23 patients evaluable + 5 patients had stable disease + 2 patients had tumor shrinkage + Generally well-toler
Slide 16: Selective Uptake of Reqorsa
Selective Uptake of Regorsa® Pretreatment Biopsies Posttreatment Blopsies Patient 1 (02 moka) be Patient 2 (06mors) bd © Genprex REQORSA Targets Cancer Cells ned to deliver the @—— cancer cells while REQORSA functioning TUSC2 gene minimizing its uptake by normal tissue. s, the expression of TUSC2 wa
Slide 17: Reqorsa + Tarceva ONC-002 Phase Trial
® Regorsa® + Tarceva ONC-002 Phase 2 Trial Met Simon 2-stage criteria to enroll the full 39 subjects, but was discontinued to start study with Tagrisso. ei soxte Neohe ——Chaeraniooi vm ed Pome ey Age. os ‘“ 2 orth 0 oye cite Seine, 2 » soya eenve amo 2 © Genprex’
Slide 18: Addressing Drug Resistance to Tagrisso and Tecentriq
For most patients, drug resistance to Tagrisso® and Tecentriq® evitable.1:23 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, thereby pot
Slide 19: AACR 21: Reqorsa + Tagrisso Reduce Tumor Growth
AACR 21: Regorsa® + Tagrisso Reduce Tumor Growth in Tagrisso Resistant Tumors Enhanced Anti-Tumor Activity REQORSA in combination with Tagrisso demonstrated significantly increased anti-tumor i. i efficacy in EGFR mutant Tagrisso 40 4 i resistant NSCLC tumors in H1975- Lolz Poa] ‘a OsiR mouse xenogr
Slide 20: Acclaim-1: Reqorsa + Tagrisso Trial in EGFR Mutant NSCLC
Patients with advanced, EGFR mutant NSCLC whose disease progressed after Tagrisso® FDA Fast Track Designation ~10-15 US. sites ~119 patients Phase 1 Dose Escalation: 12 patients (completed) Phase 2a Expansion: ~33 patients (opened for enrollment in Jan. 2024) Phase 2b: ~74 patients phase 2a Exctares
Slide 21: Phase 1 Dose Escalation
Phase 1 Dose Escalation 006mokg ——009maRg «OZ mpig Tota Pats 3 ry Es 2 Medin Age ane) 5915040) S186 SO BT-7H 59878 out ° ° ° ° Progression Free Survival by Dose Group PFS (days) Excellent safety profile and efficacy in relapsed patients. Delayed Infusion-Related Reaction + No symptoms during 30 min
Slide 22: Reqorsa in Small Cell Lung Cancer
Reqorsa® 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. —
Slide 23: SCLCs Express Low Levels of TUSC2 Protein
SCLCs Express Low Levels Of TUSC2 Protein IHC analysis of tumor specimens ——© 41% of SCLC have no TUSC2 protein expression —— 100% of SCLC have reduced or no TUSC2 protein expression No.of Fust score, samples mean (SO) Histology ‘of samples Since all SCLCs have reduced or no TUSC2 protein expression
Slide 24: Atezolizumab (Tecentriq) SCLC Approval Trial
Atezolizumab (Tecentriq®) ce Samet SCLC Approval Trial ‘ IMpower133 Study i i i 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 + A
Slide 25: 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 v
Slide 26: Reqorsa Adds Significantly to Tecentriq Treatment
Reqorsa® Adds Significantly to H41 SCLC cell xenografts Tecentriq Treatment in humanized mice CONTROL REQORSA REQORSA + Atezo on H841 SCLC Lung Met in Humanized Mice TECENTRIQ AAA Ae a © Genprex
Slide 27: Increased Immune Response with Reqorsa and Tecentriq
Increased Immune Response with Regorsa® and Tecentriq © Genprex
Slide 28: Acclaim-3: Reqorsa + Tecentriq Trial in ES-SCLC
ith ES-SCLC who did not devetop tumor progression after receiving Tecentriq® and chemotherapy al « FastTrack Designation and Orphan Drug Designation Accla im:3 * “62 patients ‘c Phase 1 Dose Escalation: Completed o Phase 2: ~50 patients Phase 2 interim analysis after 25th patient enrolled and treate
Slide 29: Reqorsa in Ras Inhibitor Resistant Lung Cancer
Regorsa® in Ras Inhibitor Resistant Lung Cancer 2024 EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics ‘TUSC2 transfection significantly reduced colony formation in two Acquired Resistant (AR) cell ines ‘Transfection of TUSC2 also markedly increased apoptosis in AR cells Treatmen
Slide 30: Reqorsa in ALK-EML4 Positive Lung Cancer
Regorsa® in ALK-ELM4 Positive SRA with The University of Michigan Rogel Cancer Center Translocated Lung Cancer and Collaboration with ALK- Positive Advocacy Group 2024 AACR Annual Meeting: ALK-Positive Collaboration: REQORSA induced apoptosis alectinib + Non-proft patient-driven research resistant E
Slide 31: Reqorsa in Mesothelioma
Regorsa® in Mesothelioma 2024 EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics + Researchers investigated whether + Researchers demonstrated that TUSC2 transfection could modulate REQORSA treatment resulted in: MPM aggressive properties + Significant decrease in cell + Four MPM
Slide 32: NPRL2 Induces Anti-tumor Activity in NSCLC
NPRL2 Induces wenuzsPombre on 569 Lang Met Anti-tumor Activity in NSCLC ne on; att Further Evidence of Oncoprex® Delivery System as a Platform for Treatment Using Tumor Suppressor a 5 Fi = Genes aod Study investigated the antitumor responses to NPRL2 gene 3 therapy on anti-PD1 resistant KRAS/STK11 m
Slide 33: Combined TUSC2 and NPRL2 Re-Expression in SCLC
Combined TUSC2 and NPRL2 Re-Expression in SCLC H841 lacks both TUSC2 and NPRL2 protein Increased control of xenograft growth compared to: Control + TUSC2 re-expression alone + NPRL2 re-expression alone © Genprex “Tumor Volume (mm?) TUSC2+NPRL2 Dual Gene Therapy Combination on Inflamed H841 Tumors in
Slide 34: Diabetes
Q DIABETES www.genprex.com
Slide 35: Diabetes: By the Numbers
Diabetes: By the Numbers 38M $966B $248 e e e e Diabetes U.S1 Diabetes Global? Global Diabetes T2D Global Market _T1D Global Market Prevalence Prevalence Expenditures (6.7% CAGR') (17.2% CAGR®) STMUS aduts 08% “Expected to se to ABM by 31 merase in global ss7Bepeced US $2038 expected US. population)
Slide 36: Diabetes Can Cause Serious Complications
Diabetes can cause serious complications. In 2021, there was approximately 1 death every 5 seconds caused by diabetes worldwide. Genprex
Slide 37: Diabetes Causes Serious Complications
Diabetes Causes Serious Complications eee @ © Genprex Heart Disease Leading cause of death for men and women in U.S. Diabetics are 2x as likely tohave heart disease or a stroke. Chronic Kidney Disease ‘Approximately 1 in 3 adults with diabetes have CKD. Kidney diseases are the Sth leading cause of d
Slide 38: Diabetic Patients Are In Need of Advanced Therapy
Diabetic Patients Are In Need of Advanced Therapy Islets of Langechans Recombinant produce insulnand NA techniques the destruction of these cals resulted Synthetic indiabetes humae?irsulin Inca fest introduced to Type I diabetes 1901 bi 1978 * ° The most significant advancement in the treatment of d
Slide 39: Novel Gene Therapy Collaboration with University of Pittsburgh
Novel Gene Therapy Collaboration with University of Pittsburgh Diabetes Program intends to address both T1D and T2D. 38M or 11.6% of Americans Have Diabetes? Type 1 Diabetes (5-10%) ———e o—__— Type 2 Diabetes (90-95%) Inabily of the pancreas to produce enough Insulin due largely to resistance to ins
Slide 40: GPX-002 Replenishes Insulin Producing Cells
GPX-002 Replenishes 5 : e programs and restores Insulin Producing Cells cell function in TLD. Delivers Genes to the Pancreas my ‘Annovel infusion process uses an AAV vectorto deliver the Hypersiycemia gy Euglycemia PPdxt + MafA (PM) genes to the pancreas Intraductal infusion (of Pdx1- and Mofa- ‘exp
Slide 41: Reversed Drug-Induced Diabetes in T1D Mouse Model
Reversed Drug-Induced Diabetes in T1D Toxin-Induced Mouse Model Blood glucose (mai) beta cllmass og) Hy siloman AAV econ Untested ——allons alloxan ‘croup ————*_ Reprogramed alpha cells into beta-like cells that appropriately produce insulin in response to glucose levels, Oc Normalized blood glucose
Slide 42: Restored Blood Glucose in T1D Autoimmune Mouse Model
Restored Blood Glucose in T1D In Autoimmune Mouse Model for Four Months The duration of restored blood glucose levels in mice could bas potentially translate to decades in humans. BB beta cel maze (mg) ——° One week in a mouse tends to correlate to about one year in humans. 20 2 4 6 8 1042 44 46 38 2
Slide 43: Generation of Insulin-Expressing Cells from Human 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 —o Human istets treated with streptozotocin to to destroy beta-cells, then treated with either AAV-PM or AAV-GFP H sransv-cre into
Slide 44: Viral Infusion in NHP Model of T1D
Viral Infusion + NHP 1 lin / Car nentuman = dl i Aaliiass Model of T1D Reduced Insulin Requirements ae cision = wns (2 ll la t= = Data from University of Pittsburgh researchers show a marked reduction in insulin requirements. © Genprex
Slide 45: ATTD 23: Decreased Insulin Requirements in NHPs
ATTD 23: Decreased Insulin Requirements and Improved Glucose Tolerance in NHPs Following the pancreatic intraductal infusion of the AAV engineered construct, the eight = NHPs had: possi! z Sere ast sen fea requrerrets 3 Rect 7 (p<0.001) & | uf ——o increased c-peptide levels i | iy {p<0.05) + 4 il HH
Slide 46: NHP2 Three-Month Glucose Tolerance Test
NHP2 Three-Month Glucose Tolerance Test 500 ©. Pre Gene Therapy '& Post Gene Therapy (3 months) we Normal NHP zo ‘ve 300 * £ ° ° g ° 8 200 q = - "oe 100 |- 7 Time (min) © Genprex
Slide 47: IHC Eight Weeks After Gene Therapy
IHC Eight Weeks After Gene Thera py Need at least 20% of normal beta cell mass to maintain normoglycemia Normal NHP Diabetic NHP Without Gene Therapy Diabetic NHP After Gene Therapy Genprex
Slide 48: Corporate
Q CORPORATE
Slide 49: Our Team: Company Management
Our Team: Company Management o if Mork. Berger MO
Slide 50: Our Team: Scientific Advisory Board
Our Team: Scientific Advisory Board Jack A. Roth ony SK Mok MO, FACS, Chairman MO, FRCRO, FHKCE, FHKAM.
Slide 51: Our Team: Clinical Advisory Board
Our Team: Clinical Advisory Board
Slide 52: Our Team: Board of Directors
Our Team: Board of Directors S ie wy 6h aa iS
Slide 53: Achievements & Upcoming Milestones
Achievements & Upcoming Milestones Acclaim -1 ‘Open for enrollment in Phase 2a Expansion tion ofthe tal in Jan. Complete enrolment of first 19 patientsin Phase interim analysis Expect Phase 28 interim ‘analysesin 2H 2025 © Genprex Acclaim -3 Complete Phase 1 Dose Escalation mn 2H2024 Open for enroll
Slide 54: We believe in a future of transformational patient care
We believe in a future of transformational patient care. 21% Century Large Combination Two FDA Gene Markets & Trials with Top Fast Track Therapies UnmetNeed | SellingDrugs | Designations Exploring New Indications & Partnerships
Slide 55: Slide 55
Slide 56: References
Research References Slide 7 ‘ist I, Behrens, Viemani AK, etal. High resoluton chromosome 3p allelotyping of human lung cancer and preneopastcreimasive bronchial ‘epithelium reveals multiple discontinuous stes of 3p allele lossand three regjons of frequent breakpoints. Cancer Res. 2000:60(7; 1949-196
Slide 57: References
Research References Slide 32 Centers for Disease Controt:hitasi/biLb/dazela Inurnaeora Dabetes reset. Slide 33 1. (DF Diabetes ats: tose Mate Slide 34 1. Centers for Disease Contra hitpevicee govidiabetesmanaging/prableme html 2. Amputation Prevention Centers of America: hips: apeofamerca com/diabet
Slide 58: Appendix
a) APPENDIX www.genprex.com
Slide 59: Reqorsa + Keytruda
Regorsa® + Keytruda® REQORSA increases immune response Significa ntly Reduced Tumor against lung cancer xenografts Growth — = REQORSA + Keytruda® a im =z. _ = The independent immunologic effects of REQORSA and Keytruda markedly decrease tumor growth by increasing the immunologic attack on the tumor
Slide 60: AACR 21: Reqorsa May Enhance First-Line Standard of Care
AACR 21: Regorsa® May Enhance First-Line Standard of Care Regorsa® + Keytruda® + Chemo ae mk. ——* REQORSA enhances the efficacy of ‘chemo-immunotherapy on KRAS-LKB1 (KL)-mutant lung metastases in humanized mice. “ota Fx) ——* Tripte combination demonstrated strong antitumor efficacy and induced robus
Slide 61: Acclaim-2: Reqorsa + Keytruda Trial
Acclaim-2 is no longer enrolling new patients. tye ee ee Acclaim - 2 © Patients with advanced NSCLC whose disease progressed after ‘treatment with Keytruda® » FDA Fast Track Designation Regors in combination with Merck & Co's Keytruda® for NSCLC Phase 2b: Comparing Progression Free Survival of REQOR