Genprex, Inc. · NASDAQ:GNPX
Genprex — Investor Presentation (May 2024)
57 slides from Genprex's investor presentation, May 2024
Slide 1: Pioneering Gene Therapies for Patients in Need
Pioneering Gene Therapies for Patients in Need May 2024 w w w. g e n p r e x . c o m | N A S D A Q : G N P X
Slide 2: Forward-Looking Statements
Forward-Looking w w w. g e n p re x . c o m Statements Statements contained in this presentation regarding matters that are not historical facts candidates, and whether patents will ever be issued under patent applications filed by us are “forward-looking statements” within the meaning of the Privat
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 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 Demonstrated abili
Slide 5: Research and Development Pipeline
Research and Development Pipeline We are developing our preclinical diabetes candidate GPX-002 using the same construct for both Type 1 diabetes and Type 2 diabetes (formerly known as GPX-003 when a different construct was being considered for Type 2 diabetes).
Slide 6: Oncology
ONCOLOGY R EPR OG R AM M I NG T HE COU R S E OF CANCER w w w. g e n p re x . c o m
Slide 7: Why TUSC2?
Why TUSC2? Discovery Tumor Suppressor Gene o TUSC2 restoration in cancer cells Tumor Suppressor Candidate 2 in vitro inhibits cell growth and induces apoptosis Chromosome 3p21.3 deleted o TUSC2 is encoded by nuclear DNA but TUSC2 protein is located gene in inner membrane of mitochondria o Plays a ke
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 lung cancers express decreased amounts of TUSC2 tumor suppressor protein Loss or reduction of TUSC2 expression
Slide 9: Oncoprex Delivery System
Oncoprex® Non-viral, positively-charged lipid-based Delivery System nanoparticle in a lipoplex form is systemically delivered. Cationic lipoplex carries drug to tumors.
Slide 10: Our Cancer Treatment Approach
Our Cancer Treatment Approach Tumor Suppressor Gene in a DNA Plasmid Tumor suppressor genes are 1 We have rights to tumor suppressor genes that may have cancer-fighting functions. These genes are deleted early during cancer expressed in a DNA plasmid. development. Non-Viral Lipid Nanoparticles in a
Slide 11: Novel Platform to Treat Cancer
Novel Platform to Systemic Gene Therapy Platform: Oncoprex ® Delivery System Treat 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®, Keytruda® and Tecentriq® Cancers C
Slide 12: Reqorsa Targets Cancer At Its Core
Reqorsa® Targets Cancer Multiple anti-cancer At Its Core mechanisms of action. 1 2 3 Controls Cell Signaling Stimulates Apoptotic Pathways Modulates Immune Response Tyrosine kinase inhibition Leads to programmed Promotes immune decreases cancer cell cancer cell death activity against cancer prolifer
Slide 13: Reqorsa Reduces Glycolysis in Cancer Cells
Reqorsa® Reduces Glycolysis REQORSA Reverses Fundamental in Cancer Cells Characteristic of Cancer Cancers are detected by PET scans • PET scanning is based on increased glucose uptake in cancer cells • Due to high rate of glycolysis in cancer cells A549 cells are a NSCLC line virtually lacking TUSC2
Slide 14: Lung Cancer: By the Numbers
Lung Cancer: By the Numbers 125K 2M+ $48B 234K 26.6% 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 market is projected More than 234,000 new Rate deaths new cases per year t
Slide 15: Reqorsa Monotherapy
Reqorsa® 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 REQORSA Targets Cancer Cells of Reqorsa® Pretreatment Biopsies Posttreatment Biopsies REQORSA is designed to deliver the functioning TUSC2 gene to cancer cells while minimizing its uptake by normal tissue. Patient 1 (.02 mg/kg) Tumor biopsy studies show that, in three patients, the
Slide 17: Reqorsa + Tarceva
Reqorsa® + 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. BEST OVERALL EGFR MUTATION PRIOR PRIOR LINES NUMBER OF CYCLES RESPONSE STATUS THERAPY OF THERAPY Positive CR 11 cycles Chemo 3 (exon 18+20) SD 24% Re
Slide 18: Drug resistance to Tagrisso, Keytruda and Tecentriq is inevitable
For most patients, drug resistance to Tagrisso®, Keytruda® and Tecentriq® is inevitable.1,2,3 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
Slide 19: AACR 21: Reqorsa + Tagrisso Reduce Tumor Growth
AACR 21: Reqorsa® + Tagrisso Reduce Tumor Growth in Tagrisso Resistant Tumors TUSC2 Osimertinib TUSC2 TUSC2 21 Days Survival & tumor growth 12/16 01/07/20 01/10 01/13 Cell inoculation Enhanced Anti-Tumor Activity Osimertinib+TUSC2 combination on Osimertinib+TUSC2 combination on H1975-OsiR tumors in
Slide 20: Reqorsa + Tagrisso Clinical Trial Design
o Patients with advanced, EGFR mutant NSCLC whose disease progressed after Tagrisso® o FDA Fast Track Designation o ~10-15 U.S. sites o ~158 patients o Phase 1 Dose Escalation: 12 patients (completed) o Phase 2a Expansion: ~66 patients in 2 cohorts (opened for enrollment in Jan. 2024) o Phase 2b: ~7
Slide 21: Phase 1 Dose Escalation
Phase 1 Dose Escalation Excellent safety profile and efficacy in relapsed patients. Delayed Infusion-Related Reaction • No symptoms during 30 min infusion • Fever, chills, and muscle aches • Symptoms generally start 3-6 hours after infusion • Generally lasts 2-4 hours • Prophylaxis with dexamethason
Slide 22: Reqorsa + Keytruda Significantly Reduced Tumor Growth
Reqorsa® + Keytruda® REQORSA increases immune response against lung cancer xenografts Significantly Reduced Tumor Growth REQORSA + Keytruda® Pembrolizumab is the generic name of Keytruda. Pembrolizumab is the generic name of Keytruda. The independent immunologic effects of REQORSA and Keytruda marke
Slide 23: AACR 21: Reqorsa May Enhance First-Line Standard of Care
AACR 21: Reqorsa® May Enhance First-Line Standard of Care Reqorsa® + Keytruda® + Chemo REQORSA enhances the efficacy of chemo-immunotherapy on KRAS-LKB1 (KL)-mutant lung metastases in humanized mice. Triple combination demonstrated strong antitumor efficacy and induced robust antitumor immunity in K
Slide 24: Reqorsa + Keytruda Clinical Trial Design
o Patients with advanced NSCLC whose disease progressed after treatment with Keytruda® o FDA Fast Track Designation o ~10 U.S. sites o ~180 patients o Phase 1 Dose Escalation: Up to 18 patients o Phase 2a Expansion: 36 patients o Phase 2b Randomized: 126 patients o Phase 2b interim analysis at 50 ev
Slide 25: 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. Small
Slide 26: 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
Slide 27: 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 • 4 cycles, then atezolizum
Slide 28: 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 • All CR, PR, and SD patients received maintenance therapy • Endpoints measured from the start of maintenance therapy PFS 2.6 vs 1.8 mos
Slide 29: Reqorsa Adds Significantly to Tecentriq Treatment
Reqorsa® Adds Significantly to H841 SCLC cell xenografts in humanized mice Tecentriq Treatment CONTROL REQORSA TECENTRIQ REQORSA + TECENTRIQ Atezolizumab is the generic name of Tecentriq.
Slide 30: Increased Immune Response with Reqorsa and Tecentriq
Increased Immune Response with Reqorsa® and Tecentriq Atezolizumab (Atezo) is the generic name of Tecentriq. huNK = human natural killer cells DC = dendritic cells MDSC = myeloid derived suppressor cells TME = tumor microenvironment
Slide 31: Reqorsa + Tecentriq Clinical Trial Design
o Patients with ES-SCLC who did not develop tumor progression after receiving Tecentriq® and chemotherapy o Fast Track Designation and Orphan Drug Designation o ~10 U.S. sites o ~62 patients o Phase 1 Dose Escalation: Up to 12 patients (opened for enrollment in Jan. 2024) o Phase 2: ~50 patients Req
Slide 32: NPRL2 Induces Anti-tumor Activity in NSCLC
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 Humanized mice w
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
Slide 34: Diabetes
2 DIABETES www.genprex.com
Slide 35: Diabetes: By the Numbers
Diabetes: By the Numbers 38M 537M $966B $92B $24B Diabetes U.S.1 Diabetes Global2 Global Diabetes T2D Global Market T1D Global Market Prevalence Prevalence Expenditures (6.7% CAGR4) (17.2% CAGR5) 97M U.S. adults (38% Expected to rise to 643M by 316% increase in global $57B expected U.S. $20.3B expec
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.
Slide 37: Diabetes Causes Serious Complications
Diabetes Causes Serious Complications Heart Disease Vision Loss (Diabetic Retinopathy) Leading cause of death for men and women in U.S. Diabetic retinopathy affects almost 1/3 of adults over 40 years old. Diabetics are 2x as likely to have heart disease or a stroke. Diabetes is leading cause of new
Slide 38: Diabetic Patients Are In Need of Advanced Therapy
Diabetic Patients Are In Potential for disease modification Need of Advanced Therapy for long-term effectiveness. Patients suffer compromised quality of life Despite certain advancements in treatment, quality 1922 Islets of Langerhans Recombinant produce insulin and DNA techniques of life remains hi
Slide 39: Novel Gene Therapy Diabetes Program
Novel Gene Therapy Collaboration with University of Pittsburgh Diabetes Program intends to address both T1D and T2D. 38M or 11.6% of Americans Have Diabetes1 Type 1 Diabetes (5-10%) Type 2 Diabetes (90-95%) An auto-immune condition where the body's Inability of the pancreas to produce enough immune
Slide 40: GPX-002 Replenishes Levels Of Insulin
GPX-002 Replenishes Reprograms and restores Levels Of Insulin cell function in T1D. Delivers Genes to the Pancreas A novel infusion process uses an AAV vector to deliver the Pdx1 + MafA (PM) genes to the pancreas. Reprograms Alpha Cells GPX-002 transforms alpha cells in the pancreas into functional
Slide 41: Reversed Drug-Induced Diabetes in T1D Toxin-Induced Mouse Model
Reversed Drug-Induced Diabetes in T1D Toxin-Induced Mouse Model 600 Blood glucose (mg/dl) 400 AAV8-GFP 200 AAV8-PM 0 0 7 14 21 28 35 alloxan AAV-8 injection GFP = Green Fluorescent Protein (fluorescent marker) | PM = Pdx1 + MafA Reprogramed alpha cells into beta-like cells that appropriately produce
Slide 42: Restored Blood Glucose in T1D Autoimmune Mouse Model for Four Months
Restored Blood Glucose in T1D In Autoimmune Mouse Model for Four Months The duration of restored blood 600 glucose levels in mice could Blood glucose (mg/dl) potentially translate to decades in humans. 400 AAV8-GFP One week in a mouse tends to 200 correlate to about one year in humans. AAV8-PM 0 -2
Slide 43: Functional Insulin-Expressing Cells from Alpha Cells in Human Islets
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 1.0 600 to destroy beta-cells, then treated with either AAV-PM or AAV-GFP Graft beta cell
Slide 44: NHP Insulin/Carb Ratio
NHP 1 Insulin / Carb Viral Infusion 1.50 Insulin/Carb Ratio 1.00 Non-Human 0.50 Primate 0.00 2 6 10 14 18 22 26 30 34 38 42 46 50 54 58 62 66 70 74 78 Model of T1D STZ -34 -30 -26 -22 -18 -14 -10 -6 -2 Reduced Insulin Requirements NHP 2 Insulin / Carb Viral Infusion 2.00 Insulin/Carb Ratio 1.50 1.00
Slide 45: ATTD 23: Decreased Insulin Requirements and Improved Glucose Tolerance 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: Decreased insulin requirements (p<0.001) Increased c-peptide levels (p<0.05) Improved glucose tolerance compared to baseli
Slide 46: NHP2 Three-Month Glucose Tolerance Test
NHP2 Three-Month Glucose Tolerance Test 500 Pre Gene Therapy Post Gene Therapy (3 months) Normal NHP 400 Glucose (mg/dL) 300 200 100 0 0 20 40 60 Time (min) UNPUBLISHED DATA
Slide 47: IHC Eight Weeks After Gene Therapy
IHC Eight Weeks After Gene Therapy Need at least 20% of normal beta cell mass to maintain normoglycemia DAPI INSULIN GLUCAGON Normal NHP Diabetic NHP Without Gene Therapy Diabetic NHP After Gene Therapy
Slide 48: Corporate
Q CORPORATE www.genprex.com
Slide 49: Our Team: Company Management
Our Team: Company Management Ryan Confer, MS Mark S. Berger, MD Thomas Gallagher, JD David Schloss, JD Suzanne Thornton-Jones, PhD President, Chief Executive Officer & Chief Medical Officer Senior Vice President, Senior Vice President, Senior Vice President, Chief Financial Officer IP & Licensing Hu
Slide 50: Our Team: Scientific Advisory Board
Our Team: Scientific Advisory Board Jack A. Roth Tony S. K. Mok Pasi Antero Jänne George Simon George K. Gittes MD, FACS, Chairman MD, FRCP(C), FHKCP, FHKAM MD, PhD MD MD Professor and Bud Johnson Professor and Chair of Clinical Professor of Medicine, Chair, Department of Medical Chief of Pediatric
Slide 51: Our Team: Clinical Advisory Board
Our Team: Clinical Advisory Board Michael Morse Andrew Becker Col. George Peoples MD, MHS, FACP MD, PhD MD, FACS Professor of Medicine, President and Founder, Chief Executive Officer of Division of Medical Oncology Becker Pharmaceutical Cancer Insight, LLC, a in the Department of Surgery Consulting
Slide 52: Our Team: Board of Directors
Our Team: Board of Directors Jose A. Moreno Toscano Ryan Confer, MS Brent Longnecker William R. Wilson, Jr. James E. Rothman, PhD Chairman of the Board Board Director Board Director Board Director Strategic Advisor to the Board Chief Executive Officer, LFB 10+ years of C-Level Chief Executive Office
Slide 53: Achievements & Upcoming Milestones
Achievements & Upcoming Milestones GPX-002 Corporate Engage KOLs in Open for enrollment Complete Open for Report data from discussions on our in Phase 2a Expansion enrollment for enrollment in Phase ongoing preclinical oncology and portion of the trial in Phase 1 Dose 1 Dose Escalation NHP studies i
Slide 54: We believe in a future of transformational patient care
We believe in a future of transformational patient care. 21st Century Large Combination Three FDA Exploring New Gene Markets & Trials with Top Fast Track Indications & Therapies Unmet Need Selling Drugs Designations Partnerships
Slide 55: NASDAQ: GNPX Contact
NASDAQ: GNPX | (877) 774 - GNPX | investors@genprex.com | www.genprex.com
Slide 56: Research References
Research References Slide 15 1. Phase 1 Clinical Trial of Systemically Administered TUSC2(FUS1)-Nanoparticles Mediating Functional Gene Transfer in Humans. Lu, C. et al. PLOS One. 2012. Slide 7 1. Wistuba II, Behrens C, Virmani AK, et al. High resolution chromosome 3p allelotyping of human lung canc
Slide 57: Research References
Research References Slide 36 Slide 40 1. Centers for Disease Control: https://bit.ly/4ageILa 1. Centers for Disease Control: https://bit.ly/4ageILa 2. International Diabetes Federation: https://bit.ly/43I4h0A 3. IDF Diabetes Atlas: https://bit.ly/4aFeCwp 4. Research and Markets: https://bwnews.pr/3S