REQORSA® Immunogene Therapy
The First Systemically Delivered Gene Therapy Used for Cancer in Humans
The company's lead product candidate, REQORSA®, immunogene therapy (quaratusugene ozeplasmid) for non-small cell lung cancer (NSCLC), uses the company’s unique, proprietary ONCOPREX® Nanoparticle Delivery System, which they believe is the first systemic gene therapy delivery platform used for cancer in humans.
In 2020, the FDA granted Fast Track Designation for REQORSA in combination with AstraZeneca’s Tagrisso® (osimertinib) in late-stage NSCLC patients with EFGR mutations whose tumors progressed after treatment with Tagrisso.
In 2021, the FDA granted Fast Track Designation for REQORSA in combination with Merck & Co’s Keytruda® (pembrolizumab) in late-stage NSCLC patients whose disease progressed after treatment with Keytruda.
The company believes that REQORSA, unlike other gene therapies, which either need to be delivered directly into tumors or require cells to be removed from the body, re-engineered and then reinserted into the body, is the first systemic gene therapy used for cancer in humans.
Diabetes Gene Therapy
GPX-002, a gene therapy for diabetes, is the most recent addition of the company's licensed technologies. GPX-002 was developed by researchers at the University of Pittsburgh. Diabetic mice studies have shown that GPX-002 restored normal blood glucose levels for an extended period of time, which could translate to decades in humans.
This gene therapy could not only become a new treatment option for millions of diabetes patients who need insulin replacement therapy, but it holds the potential to provide long-term effectiveness, or may even be a cure, for diabetic patients.
The diabetes gene therapy, GPX-002, is comprised of a novel infusion process that uses an endoscope and an adeno-associated virus (AAV) vector to deliver Pdx1 and MafA genes to the pancreas. The genes express proteins that transform alpha cells in the pancreas into functional beta-like cells, which can produce insulin but are distinct enough from beta cells to evade the body’s immune system.
Diabetic mice studies show that the gene therapy restored normal blood glucose levels for an extended period of time, typically around four months. The duration of restored blood glucose levels in mice could translate to decades in humans.
The diabetes gene therapy was developed by Dr. George Gittes, a researcher at the Rangos Research Center at UPMC Children’s Hospital of Pittsburgh, where preclinical research is ongoing. GPX-002 has been tested in vivo in mice and nonhuman primates. Once sufficient preclinical data has been generated, they expect to begin a Phase I clinical trial in diabetic patients, which could be the first-ever gene therapy tested in humans for diabetes.
ONCOPREX® Nanoparticle Delivery System
The oncology drug development program utilizes their unique, proprietary non-viral ONCOPREX Nanoparticle Delivery System, which they believe is the first systemic gene therapy delivery platform used for cancer in humans. This platform, originally developed through collaborative research between the University of Texas MD Anderson Cancer Center and the National Institutes of Health, has been optimized to work with their initial product candidate, REQORSA® immunogene therapy.
Company Website. Company Presentation.
And right now, based on multiple potential catalysts, past champ GNPX needs to be on your radar. Check them out:
No. 1 GNPX Potential Catalyst - GNPX Receives Validation For REQORSA Development Program As SRC Recommends Advancement To Phase 2
Genprex Receives Safety Review Committee Approval to Advance to Phase 2 Expansion Portion of Acclaim-1 Clinical Trial of REQORSA® in Combination with Tagrisso® in Advanced Non-Small Cell Lung Cancer
Results from Phase 1 Portion Indicate Favorable Safety Profile of Novel Gene Therapy in Solid Tumor Cancer
AUSTIN, Texas, May 30, 2023 /PRNewswire/ -- Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that it has completed the Phase 1 portion of the Acclaim-1 Phase 1/2 clinical trial of REQORSA® in combination with Tagrisso® (osimertinib) to treat late-stage non-small cell lung cancer (NSCLC), and the Safety Review Committee (SRC) has approved continuation to the Phase 2 expansion portion of the trial. The combination of REQORSA and osimertinib received U.S. Food and Drug Administration's (FDA) Fast Track Designation for treatment of the Acclaim-1 patient population.
Based on full safety data which showed no dose limiting toxicities, the SRC determined that the recommended Phase 2 dose of REQORSA will be 0.12 mg/kg. This was the highest dose level delivered in the Phase 1 portion and is twice the highest dose level delivered in Genprex's prior clinical trial combining REQORSA with Tarceva® for the treatment of late-stage lung cancer. The SRC also recommended the trial advance to the Phase 2 expansion portion of the study, which the Company expects to begin in the third quarter of 2023.
"We are proud of the notable progress we made during the Phase 1 portion of the Acclaim-1 clinical trial, and the SRC recommendation to move into the Phase 2 expansion portion of the trial is another validation for our REQORSA development program," said Rodney Varner, Chairman, President and Chief Executive Officer of Genprex. "As we move into the Phase 2 expansion portion of the trial, we remain steadfast in our efforts to bring new therapies to lung cancer patients with unmet medical need."
Read the full article here.
No. 2 GNPX Potential Catalyst - Dawson James Analyst Provides $3.00 Target (Major Upside Potential From Current Trading Levels)
On January 30th, Dawson James Securities' Senior Analyst, Jason Kolbert, provided some key insight into GNPX along with tagging this profile with a $3.00 target.
From its closing valuation Thursday, that target provides GNPX with over 200% potential upside.
Check out some highlights from the report:
Lead Program in Oncology: REQORSA Immunogene Therapy (GPX-001). REQORSA is a plasmid that expresses a tumor suppressor gene TUSC2, that is deleted early during lung cancer development. REQORSA appears to have a multimodal mechanism of action whereby it interrupts cell signaling pathways that cause replication and proliferation of cancer cells, reestablishes pathways for apoptosis, or programmed cell death, in cancer cells, and modulates the immune response against cancer cells. REQORSA has been shown to be complementary with targeted drugs and immunotherapies.
Clinical Status: REQORSA is currently enrolling clinical patients in two trials with a third coming. The trials are initially exploring its utility in NSCLC with a range of agents – standard of care. The Acclaim-1 clinical trial is evaluating a combination of REQORSA with Tagrisso in patients with late-stage NSCLC with activating epidermal growth factor receptor mutations, whose disease progressed after treatment. The first patient was dosed in Acclaim-1 in February 2022. A second trial, Acclaim-2 (first patient doses April 2022), is using a combination of REQORSA with Merck & Co.'s Keytruda in patients with late-stage NSCLC whose disease progressed after treatment with Keytruda. The FDA has granted Fast Track Designation for both trials. Accaim-3 is exploring REQORSA with Tecentriq in ES-SCLC patients.
Diabetes Program: The company has developed a gene therapy that is designed to transform alpha cells in the pancreas into functional beta-like cells, which can produce insulin but are distinct enough from beta cells to evade the body's immune system. The therapy utilizes a procedure in which an adeno-associated virus vector is endoscopically delivered to the pancreas to insert Pdx1 and MafA genes. GPX-002 for T1D uses a glucagon promoter in alpha cells. GPX003 for T2D uses an insulin promoter in beta cells. The planned phase 1 trial represents the first-ever gene therapy tested in humans for diabetes. In vivo, preclinical studies (mice), have shown that GPX-002 restored normal blood glucose levels for an extended period. Research collaborators at the University of Pittsburgh plan to present data in non-human primates highlighting the therapeutic potential of Genprex's gene therapy for Type 1 diabetes at the 16th International Conference on Advanced Technologies & Treatments for Diabetes (ATTD 2023) being held February 22-25 in Berlin, Germany.
Valuation: We project our model out to 2033. We apply a 30% risk cut to our projected revenues in our product model in addition to our 30% risk rate applied in our Free Cash Flow to the Firm (FCFF), discounted EPS (dEPS), and Sum-of-the-Parts (SOP) models. We use a fully diluted out year share count assuming multiple raises. The result is equal-weighted and averaged and rounded to the nearest whole number to derive our 12-month projected price target of $3.00. We note that as the company established proof of concept as a result of clinical trial data, the risk rate (r) is reduced, and valuation rises.
No. 3 GNPX Potential Catalyst - Company's ONCOPREX Platform May Be Used To Address Multiple Types Of Cancer
Genprex Unveils New Video Featuring Chief Medical Officer Discussing Positive Preclinical Data With NPRL2 Gene Therapy Utilizing Non-Viral ONCOPREX® Nanoparticle Delivery System
Chief Medical Officer Provides Overview of Preclinical Data Recently Presented at the 2023 American Association of Cancer Research (AACR) annual meeting
Video Highlights Validation of the ONCOPREX® Nanoparticle Delivery System with a Second Tumor Suppressor Gene
AUSTIN, Texas, May 23, 2023 /PRNewswire/ -- Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today released a new video where the company's Chief Medical Officer, Mark Berger, MD, discusses the recently presented positive preclinical data for the NPRL2 gene by Genprex's research collaborators. The data were presented at the 2023 American Association of Cancer Research (AACR) annual meeting.
The studies used the Company's non-viral ONCOPREX® Nanoparticle Delivery System in KRAS/STK11 mutant anti-PD1 resistant metastatic human non-small cell lung cancer (NSCLC) humanized mouse models. These positive data support the therapeutic potential of the Company's non-viral delivery system, which is being used in its current REQORSA® clinical oncology programs. REQORSA immunogene therapy is the Company's lead drug candidate for lung cancer. The preclinical data also provide further evidence that the ONCOPREX Nanoparticle Delivery System has the ability to be successful using tumor suppressor genes other than the TUSC2 gene that is already in use in clinical trials with REQORSA. Lastly, the data suggest that targeting NSCLC with the NPRL2 gene, potentially in combination with anti-PD1 treatment as well, may provide therapeutic potential for lung cancer patients progressing on anti-PD1 treatment.
"The study utilized the same delivery platform that Genprex uses with our REQORSA product candidate but with a different tumor suppressor gene," said Mark Berger, MD, Chief Medical Officer at Genprex. "The whole point of our oncology program is to identify tumor suppressor genes, which are systematically deleted during cancer development, and then re-express the tumor suppressor genes in cancers. This process has shown preclinically that it can achieve strong efficacy and is currently being evaluated in Phase 1/2 clinical trials of REQORSA. Now researchers have replicated that process in in vitro studies with the NPRL2 gene, which we believe is validation that ONCOPREX as a platform may be used with multiple tumor suppressor genes to address multiple types of cancer."
The abstract entitled, "NPRL2 gene therapy induces effective antitumor immunity in KRAS/STK11 mutant anti-PD1 resistant metastatic human non-small cell lung cancer (NSCLC) in a humanized mouse model," has been made available on Genprex's website here.
To watch the video, please visit Genprex's website at www.genprex.com/videos/.
Read the full article here.
No. 4 GNPX Potential Catalyst - A Growing Body Of Studies Evaluating TUSC2 Could Loom BIG For Company's Lead Drug Candidate, REQORSA®
Independent Researchers Find That TUSC2, the Gene Delivered by Genprex's REQORSA®, Functions as a Novel Tumor Suppressor for Glioblastoma
Pre-clinical Study results show TUSC2 overexpression reduces colony formation, neurosphere formation and promotes apoptosis in vitro, and suppresses tumor growth in vivo
Study adds to the growing body of research examining TUSC2 as a target for multiple other cancers
TUSC2 is the tumor suppressor gene used in the Company's lead drug candidate, REQORSA® (quaratusugene ozeplasmid)
AUSTIN, Texas, April 18, 2023 /PRNewswire/ -- Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that independent researchers recently presented preclinical data on the tumor suppressor gene, TUSC2, reporting that it functions as a novel tumor suppressor for glioblastoma. TUSC2 is the tumor suppressor gene that is reexpressed in tumors using REQORSA® Therapy treatment, Genprex's lead drug candidate. Genprex has no affiliation with these researchers.
The independent researchers presented their abstract, titled, "Investigating TUSC2 for its tumor suppressive functions in glioblastoma and its role in gliomagenesis," at the 2023 American Association for Cancer Research Annual Meeting on April 17. The abstract reports TUSC2 protein expression, but not mRNA expression, to be significantly decreased in glioblastoma as compared to normal brain. TUSC2 protein is destabilized in glioblastoma due to polyubiquitination by E3 ligase, neural precursor cell-expressed developmentally downregulated gene 4 (NEDD4), and degradation by the proteasome. The researchers determined that NEDD4 targets TUSC2 on lysine residue K71.
The researchers also reported that TUSC2 overexpression reduced colony formation and neurosphere formation and promoted apoptosis of glioblastoma cells in vitro, and suppressed tumor growth in vivo. Further, glioblastoma cells with CRISPR-mediated knockout (KO) of TUSC2 were highly aggressive in vitro and in vivo, demonstrating the role of TUSC2 as a novel tumor suppressor for glioblastoma.
In the study, researchers performed immunoprecipitation-mass spectrometry analysis to identify TUSC2-interacting proteins in both normal human astrocytes and glioblastoma cell lines. Results showed 95 and 88 TUSC2-interacting proteins in astrocytes and glioblastoma cell lines respectively, and 27 proteins were found to interact with TUSC2 in both astrocytes and glioblastoma.
"This research supports the growing body of studies evaluating TUSC2 as a target in oncology, and potentially an effective treatment, for many types of cancer," said Rodney Varner, Chairman, President and Chief Executive Officer of Genprex. "We are encouraged by the increasing number of research institutions and independent researchers producing positive data on TUSC2, which provide additional support for REQORSA® and its therapeutic potential against cancers."
Read the full article here.
No. 5 GNPX Potential Catalyst - "Groundbreaking" Data Could Lead To Change In Course For Diabetes Treatment
Genprex Announces Groundbreaking Data from Non-Human Primate Study Evaluating Novel Gene Therapy to Treat Type 1 Diabetes at 16th Annual International Conference on Advanced Technologies & Treatment for Diabetes 2023
Results show statistically significant decreases in insulin requirements, increases in c-peptide levels and improvements in glucose tolerance compared to baseline
Disruptive gene therapy approach developed by University of Pittsburgh researchers may be a promising treatment for Type 1 and Type 2 diabetes
AUSTIN, Texas, Feb. 23, 2023 /PRNewswire/ -- Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that data highlighting the potential of Genprex's gene therapy for Type 1 diabetes is being presented by its research collaborators at the University of Pittsburgh (Pitt) at the 16th International Conference on Advanced Technologies & Treatments for Diabetes (ATTD 2023) taking place February 22-25, 2023 in Berlin, Germany.
The abstract data were released, and the presentation will be delivered on February 25th at 2:25 pm CET by Ranjeet S. Kalsi, DO, a member of the laboratory of George Gittes, MD, from Pitt's Division of Pediatric Surgery, Pittsburgh, Pennsylvania. The complete presentation will be available on the Company's website here following the close of Dr. Kalsi's presentation.
The presentation titled, "Pancreatic Intraductal Infusion of Adeno-Associated Virus (AAV) to Treat Non-Human Primates in a Toxin-Induced Diabetes Model," will report results from eight non-human primates (NHPs) with toxin-induced diabetes after streptozocin administration. They received a novel infusion process that used an AAV vector to deliver Pdx1 and MafA genes directly to the pancreas.
"These results are compelling as they demonstrate the potential for this gene therapy to create newly formed beta-like cells that can produce insulin. They also validate earlier studies of this approach in diabetic mouse models that showed restoration of normal blood glucose levels for several months," stated Mark Berger, MD, Chief Medical Officer of Genprex. "We are eager to continue working to advance this gene therapy into human clinical trials in order to replicate these outcomes in people and potentially provide long-term replacement of beta-cells."
The statistically significant study results show that post-infusion of the AAV engineered construct, the eight NHPs had "decreased insulin requirements (p<0.001); increased c-peptide levels (p<0.05); and improved glucose tolerance compared to baseline (p<0.05) with one demonstrating reestablished normoglycemia. Immunohistochemistry revealed insulin and glucagon staining, which suggest the formation of insulin-producing cells."
The diabetes technologies licensed from Pitt by Genprex were developed in the laboratory of George Gittes, MD, Professor of Surgery and Pediatrics and Chief of the Division of Pediatric Surgery at the University of Pittsburgh School of Medicine. "We are delighted with the statistically significant outcomes from this NHP study in Type-1 diabetes as it further supports our thesis on the potential of our gene therapy approach in diabetes. Importantly, it shows a positive data progression from our earlier mouse model data to these NHP data," noted Dr. Gittes.
"These promising data are very encouraging and further support our belief that this gene therapy approach to treating diabetes has the potential to change the course of the disease in both Type 1 and Type 2 diabetes," said Rodney Varner, President and Chief Executive Officer of Genprex. "Based on data from a number of preclinical in vivo studies, we are encouraged that our novel gene therapy approach could provide long-term efficacy in humans potentially making exogenous insulin unnecessary, which would be a gamechanger for the 537 million people around the world living with diabetes."
Read the full article here.
Past Champ Recap - 5 Must-See GNPX Potential Catalysts
No. 1 - GNPX Receives Validation For REQORSA Development Program As SRC Recommends Advancement To Phase 2
No. 2 - Dawson James Analyst Provides $3.00 Target (Major Upside Potential From Current Trading Levels)
No. 3 - Company's ONCOPREX Platform May Be Used To Address Multiple Types Of Cancer
No. 4 - A Growing Body Of Studies Evaluating TUSC2 Could Loom BIG For Company's Lead Drug Candidate, REQORSA®
No. 5 - "Groundbreaking" Data Could Lead To Change In Course For Diabetes Treatment
Coverage is officially reinitiated on GNPX. When time permits, do this: