OUR NEW PROFILE IS: (NASDAQ: GNPX)
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GNPX HAS ALREADY RECEIVED FAST TRACK DESIGNATION FOR ACCLAIM 2
Genprex Receives U.S. FDA Fast Track Designation for REQORSA® Immunogene Therapy in Combination with Tecentriq® for the Treatment of Small Cell Lung Cancer
Independent Researchers Find That TUSC2, the Gene Delivered by Genprex’s REQORSA®, Functions as a Novel Tumor Suppressor for Glioblastoma
Genprex Collaborators Report Positive Preclinical Data With NPRL2 Gene Therapy Utilizing Non-Viral ONCOPREX® Nanoparticle Delivery System in Non-Small Cell Lung Cancer at the 2023 AACR Annual Meeting
CHECK OUT THE INVESTOR PRESENTATION HERE
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Genprex, Inc. is a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes.
Genprex’s technologies are designed to administer disease-fighting genes to provide new therapies for large patient populations with cancer and diabetes who currently have limited treatment options.
Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches.
Genprex’s oncology program utilizes its proprietary, non-viral ONCOPREX® Nanoparticle Delivery System, which the Company believes is the first systemic gene therapy delivery platform used for cancer in humans.
ONCOPREX encapsulates the gene-expressing plasmids using lipid nanoparticles. The resultant product is administered intravenously, where it is then taken up by tumor cells that express tumor suppressor proteins that are deficient in the body.
The Company’s lead product candidate, REQORSA™ is being evaluated as a treatment for non-small cell lung cancer (NSCLC) (with each of these clinical programs receiving a Fast Track Designation from the Food and Drug Administration) and for small cell lung cancer.
Genprex’s diabetes gene therapy approach consists of a novel infusion process that uses an endoscope and an adeno-associated virus (AAV) vector to deliver Pdx1 and MafA genes directly to the pancreas.
In models of Type 1 diabetes, 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.
In Type 2 diabetes, where autoimmunity is not at play, it is believed that exhausted beta cells are also rejuvenated and replenished.
Genprex Granted Chinese Patent for REQORSA® Immunogene Therapy in Combination with PD-1 Antibodies to Treat CancersProvides Additional Protection in Large Markets for Therapeutic Combination in Acclaim-2 Phase 1/2 Clinical Trial
Patent Already Granted for Same Combination in U.S. Japan, Australia, Russia and Mexico
AUSTIN, Texas — (July 5, 2023) — 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 the China National Intellectual Property Administration has granted Genprex China Patent No: 201780076886.X. The broad patent covers the use of Genprex’s lead drug candidate, REQORSA® Immunogene Therapy, in combination with PD1 antibodies through 2037.
“This is a very important patent because it provides Genprex with additional protection for our REQORSA gene therapy in combination with PD1 antibodies, such as Keyruda®, which is the subject of our ongoing Acclaim-2 clinical trial for the treatment of lung cancer,” said Thomas Gallagher, Esq., Senior Vice President of Intellectual Property and Licensing at Genprex. “Genprex has already received this patent protection in the U.S., Japan, Australia, Russia and Mexico. Now with the same patent protection in China, we have secured exclusivity for this drug combination for the treatment of cancer in many of the largest markets. This patent protection prevents others from making, using or selling this drug combination.”
Acclaim-2 is an open-label, dose escalation and clinical response study of REQORSA in combination with Keytruda in patients with advanced, metastatic non-small cell lung cancer (NSCLC) whose disease progressed after treatment with Keytruda. More information on Acclaim-2 can be found at www.clinicaltrials.gov Identifier NCT05062980.
In 2022, Genprex received a U.S. patent granting protection for the combination of REQORSA with PD1 antibodies for the treatment of cancer. In 2021, Genprex received U.S. Food and Drug Administration’s (FDA) Fast Track Designation for treatment of the Acclaim-2 patient population.
About Acclaim-2
The Acclaim-2 trial is a Phase 1/2 open-label, dose-escalation and clinical response study of REQORSA in combination with Keytruda in patients with advanced, metastatic non-small-cell lung cancer who have progressed after treatment with Keytruda. The Company anticipates enrolling patients at approximately 10 clinical sites and estimates that the Phase 1 dose escalation portion of the Acclaim-2 trial will enroll up to 18 patients, the Phase 2 expansion portion will enroll approximately 36 patients, and the Phase 2 randomized portion will enroll approximately 126 patients. Patients enrolled in the Phase 2 randomized portion of the study will be randomized 2:1 to either REQORSA and Keytruda combination therapy or to chemotherapy (docetaxel with or without ramucirumab). Patients will be treated until disease progression or unacceptable toxicity is experienced. Patients must have histologically confirmed unresectable stage III or IV NSCLC (any histology) with radiological progression on Keytruda and an ECOG performance status of 0 to 1. Genprex expects to complete enrollment for the Phase 1 dose escalation portion of Acclaim-2 by the first quarter of 2024.
Genprex Receives U.S. FDA Fast Track Designation for REQORSA® Immunogene Therapy in Combination with Tecentriq® for the Treatment of Small Cell Lung Cancer
Third FDA Fast Track Designation Further Validates the Potential of REQORSA
AUSTIN, Texas — (June 28, 2023) — 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 the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) for the Company’s lead drug candidate, REQORSA® Immunogene Therapy, in combination with Genentech, Inc’s Tecentriq® in patients with extensive-stage small cell lung cancer (ES-SCLC) who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment.
In the third quarter of 2023, Genprex expects to enroll the first patient in its Acclaim-3 clinical trial, which is a Phase 1/2 dose escalation and clinical response study of maintenance therapy evaluating REQORSA in combination with Tecentriq for this patient population. The Company has previously received two other FTDs for REQORSA, for REQORSA in combination with AstraZeneca PLC’s Tagrisso® in patients with late-stage non-small cell lung cancer (NSCLC) whose disease progressed after treatment with Tagrisso, and for REQORSA in combination with Merck & Co’s Keytruda® in patients with late-stage NSCLC whose disease progressed after treatment with Keytruda.
“We are very pleased to receive a third Fast Track Designation from the FDA for REQORSA, this time for patients with ES-SCLC in combination with the checkpoint inhibitor Tecentriq,” said Rodney Varner, President, Chairman and Chief Executive Officer at Genprex. “This is another exciting achievement in our REQORSA development program, which further validates REQORSA’s potential not only in NSCLC but also in SCLC. We look forward to accelerating the clinical development of REQORSA, and potentially providing a new treatment option for patients with SCLC.”
Tumor suppressor genes are deleted or inactivated early in the process of cancer development. REQORSA contains a plasmid that expresses a tumor suppressor gene named TUSC2. Virtually 100% of small cell lung cancers express descreased amounts of TUSC2 tumor suppressor protein, and 41% completely lack TUSC2 protein expression. ES-SCLC has a very poor prognosis, with a median progression free survival (PFS) of only 5.2 months. Importantly, median PFS for patients receiving Tecentriq as maintenance therapy is only 2.6 months from the start of maintenance treatment, so there is a great need for improvement in maintenance therapy.
“This Fast Track Designation for the Acclaim-3 patient population is another validation of REQORSA’s potential to treat lung cancer,” said Mark Berger, MD, Chief Medical Officer at Genprex. “We are very excited to soon begin treating patients in the Acclaim-3 clinical trial, which positions REQORSA as a component of initial standard therapy for SCLC rather than as treatment for relapse. That will allow us to highlight the contribution of REQORSA to an earlier stage of treatment. Based on our experience in other REQORSA trials, we have reduced the Phase 1 portion of the study to two dose levels instead of the three dose levels in our Acclaim-1 and Acclaim-2 clinical trials. We believe this will shorten the Phase 1 portion of the trial. In addition, the median PFS of only 2.6 months seen with Tecentriq maintenance treatment will also shorten the time needed to evaluate the combination of REQORSA and Tecentriq as maintenance therapy for SCLC.”
Patients in the Acclaim-3 clinical trial will be enrolled after receiving initial treatment with 3-4 cycles of carboplatin, etoposide, and Tecentriq, and achieving complete response, partial response or stable disease. They will then receive treatment with REQORSA and Tecentriq as maintenance therapy every 21 days until disease progression.
The Phase 1 dose escalation portion of the Acclaim-3 clinical trial is expected to enroll up to 12 patients at 3-5 U.S. clinical sites to determine the Maximum Tolerated Dose (MTD). If no dose limiting toxicities occur during Phase 1, then the highest dose evaluated will be the Recommended Phase 2 Dose. The Phase 2 portion of the study will then enroll approximately 50 patients at 5-10 sites. Patients will be treated with REQORSA and Tecentriq until disease progression or unacceptable toxicity is experienced.
The primary endpoint of the Phase 2 portion of the trial is to determine the 18-week progression-free survival rate from the time of the start of maintenance therapy with REQORSA and Tecentriq in patients with ES-SCLC. Patients will also be followed for survival. A Phase 2 futility analysis will be performed after the 25th patient enrolled and treated reaches 18 weeks of follow up.
FDA may award FTD if it determines that non-clinical or clinical data demonstrate the potential for a drug to address an unmet medical need for a serious or life-threatening disease or condition. This provision is intended to facilitate development and expedite review of such drugs so that a product, if approved, can reach the market expeditiously.FTD recipients may also be eligible for accelerated approval or rolling review of the recipient’s Biologics License Application (BLA) if other qualifying criteria are met. In addition, Fast Track product candidates could be eligible for priority review if supported by clinical data at the time of BLA submission.
Research And Development Pipeline
CATALYSTS
– Addressing unmet medical need in large markets through the buildout of a robust pipeline of new drug candidates and drug combinations.
– Leveraging a gene therapy platform; Genprex’s non-viral ONCOPREX® Nanoparticle Delivery System is designed to deliver a variety of therapeutic genes to fight multiple types of cancer.
– REQORSATM immunogene therapy, the first systemically delivered gene therapy used for cancer in humans, can be combined with top-selling cancer drugs and may improve their benefits.
– Genprex has demonstrated clinical achievement with REQORSA in two clinical trials, showing a favorable safety profile and evidence of efficacy in lung cancer.
– GPX-002, Genprex’s diabetes gene therapy, works to transform alpha cells in the pancreas into insulin producing beta-like cells. A Phase 1 clinical trial could be the first-ever gene therapy tested in humans for diabetes.
– Advancing novel gene therapies in diseases with large markets and unmet needs.
– Two NSCLC trials currently enrolling; both with FDA Fast Track Designations.
– World class academic partners.
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
CLINICAL TRIALS
Evaluating the Safety of REQORSA® Immunogene Therapy as a Monotherapy (Completed)
A Phase 1 dose escalation trial was conducted at The University of Texas MD Anderson Cancer Center evaluating the systemic, intravenous delivery of REQORSA® (TUSC2/FUS1) as a monotherapy in stage IV recurrent, metastatic lung cancer patients. The primary objective of this Phase 1 trial was to assess the toxicity of REQORSA administered systemically and intravenously, to determine the maximum tolerated dose, or MTD, and to determine a recommended Phase 2 dose of REQORSA alone.
The First Systemically Delivered Gene Therapy Used for Cancer in Humans
The study showed for the first time that a tumor suppressor gene can be delivered systemically, intravenously and selectively to a patient’s cancer cells using a systemic nanoparticle vector. Although this trial was not designed to show changes in outcomes, a halt in cancer growth was observed in a number of patients. REQORSA was well tolerated with tumor responses noted in lung primary and metastatic cancers in the liver, pancreas, and lymph nodes. In addition, pre- and post-treatment patient biopsies demonstrated that intravenous REQORSA selectively and preferentially targeted patients’ cancer cells.
Metabolic Tumor Response in a Metastatic Lung Cancer Subject
This subject survived after subsequent therapy more than seven years after the final treatment with REQORSA, to our knowledge, without evidence of cancer progression in the responding sites.
Combining REQORSA® Immunogene Therapy with Tarceva (erlotinib)
Phase 1 portion completed; Phase 2 portion no longer enrolling due to our change of focus to conduct Acclaim-1
A Phase 1/2 clinical trial evaluated REQORSA® in combination with Tarceva® in stage IIIB/IV lung cancer patients without an activating EGFR mutation and in patients with an activating EGFR mutation whose cancer has progressed on Tarceva therapy. Patients without the EGFR mutation represent the vast majority of lung cancer patients. However, such patients generally are not candidates for Tarceva therapy.
In the Phase 2 trial combining REQORSA with Tarceva, subjects received REQORSA in combination with Tarceva every 21 days until the occurrence of progressive disease (PD), unacceptable toxicity, withdrawal of consent, or study treatment discontinuation for other reasons, whichever occurred first. We believe that the results from the Phase 2 trial are encouraging. Out of 10 patients, 9 had received 2 or more cycles and were therefore evaluable for response. Four patients had tumor regression. The median duration of response is three months. The disease control rate (CR+PR+SD > 8weeks) was 78%, which substantially exceeds the 7% response rate (with no CRs) and 58% disease control rate reported for the LUX-Lung 1 trial, a clinical trial of Gilotrif (afatinib) in a comparable group of patients.
Combining REQORSA™ Immunogene Therapy with Tagrisso (osimertinib)
Preliminary analysis of the interim data from the Phase 2 portion of our ONC-002 trial supported our belief that REQORSA™ may provide medical benefit in several subpopulations of NSCLC patients for which there is an unmet medical need, and may provide pathways for accelerated approval by the U.S. Food and Drug Administration, or FDA. Data from our clinical trials, along with our preclinical data, provided the basis for our application for a Fast Track Designation, which was granted by FDA on January 14, 2020.
In granting our Fast Track Designation, the FDA found that REQORSA may provide a benefit over existing therapies for patients whose tumors progress on AstraZeneca’s Tagrisso. The FDA Fast Track Designation is for use of REQORSA in combination with TKI Tagrisso for the treatment of NSCLC patients with EGFR mutations whose tumors progressed after treatment with Tagrisso. We believe that the Fast Track Designation provides a clearly defined pathway toward FDA approval of the combination of REQORSA with Tagrisso.
We initiated the Acclaim-1 clinical trial in June 2021, a Phase 1/2 clinical trial of REQORSA combined with Tagrisso. To learn more about Acclaim-1, please visit ClinicalTrials.gov.
To learn more about scientific evidence and studies supporting REQORSA and the TUSC2 gene, please refer to our TUSC2 Bibliography page.
Combining REQORSA™ immunogene therapy with Keytruda (pembrolizumab)
Researchers at MD Anderson Cancer Center have conducted preclinical studies evaluating REQORSA™ in combination with anti-PD1 checkpoint inhibitors, including Merck’s Keytruda.
Positive and encouraging data indicate that REQORSA is synergistic with immunotherapies.
In April 2019, we reported that our collaborators at MD Anderson presented positive preclinical data for the combination of TUSC2 with pembrolizumab, demonstrating that TUSC2 combined with checkpoint blockade was more effective than checkpoint blockade alone in increasing the survival of mice with human immune cells, that had metastatic lung cancer.
In November 2019, we reported that our collaborators at MD Anderson presented positive preclinical data for the combination of TUSC2, pembrolizumab and chemotherapy for the treatment of some of the most resistant metastatic lung cancers. This study found that the combination of TUSC2 increases the effectiveness of pembrolizumab and chemotherapy, and thus, may improve on first-line standard of care for lung cancer.
In May 2020, we entered into a worldwide, exclusive license agreement with The Board of Regents of the University of Texas System on behalf of MD Anderson for the use of TUSC2 in combination with immunotherapies, including Keytruda, and also for the use of TUSC2 in a three-drug combination of TUSC2, immunotherapy and chemotherapy.
In December 2021, we received Fast Track Designation from the FDA for use of REQORSA in combination with the checkpoint inhibitor Keytruda for the treatment of advanced NSCLC patients whose tumors progressed after treatment with Keytruda.
In March 2022, we opened the Acclaim-2 clinical trial for patient enrollment, a Phase 1/2 clinical trial of REQORSA combined with Keytruda. To learn more about Acclaim-2, please visit ClinicalTrials.gov.
To learn more about scientific evidence and studies supporting REQORSA and the TUSC2 gene, please refer to our TUSC2 Bibliography page.
Studying GPX-002 in Diabetic Mice
Researchers at the University of Pittsburgh have conducted preclinical studies evaluating GPX-002 in diabetic mice models. In vivo mice studies have found that GPX-002 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.
These researchers are continuing to conduct preclinical studies in diabetic primates. Once sufficient preclinical data has been generated, we expect to begin a Phase 1 clinical trial in diabetic patients, which could be the first-ever gene therapy tested in humans for diabetes.
To learn more about scientific evidence and studies supporting GPX-002 and the Pdx1/MafA genes, please refer to our Pdx1/MafA Bibliography page.
Our oncology program uses a modern combinational treatment approach to fight cancer, utilizing our lead drug candidate, REQORSA™ immunogene therapy and our unique, proprietary, non-viral ONCOPREX® nanoparticle delivery system combined with approved targeted therapies and immunotherapies. This enables us to offer hope to large patient populations who would otherwise not be candidates for those therapies or who have become resistant to them.
Oncology Combination Treatment Approach
Our research indicates that when REQORSA, our lead drug candidate for non-small cell lung cancer (NSCLC), is combined with targeted therapies such as Tarceva (erlotinib) or Tagrisso (osimertinib) or with immunotherapies such as Opdivo (nivolumab) or Keytruda (pembrolizumab), REQORSA is synergistic with those drugs, meaning that the combination is more effective than either drug alone. We believe that by combining REQORSA with targeted therapies and immunotherapies, we can extend the benefit of these approved lung cancer drugs into the large majority of patients who do not now benefit from them, either because the patients’ tumors do not have the molecular profiles that indicate effectiveness of those drugs, or because the patients have developed resistance to those drugs after receiving them for some period of time.
To learn more about our combination trials utilizing our oncology combination treatment approach, refer to our Pipeline and Clinical Trials pages.
Targeted Therapies
Targeted therapies work by targeting the cancer or disease’s specific gene, protein or tissue that contributes to the disease while sparing normal tissue. Unfortunately, targeted therapies require patients to have an activating genetic mutation specific to the drug. In NSCLC, the vast majority of lung cancer patients do not have the genetic mutations that qualify them for targeted therapies, such as the EGFR or ALK gene. In addition, the few patients who do have the genetic mutation qualifying them to receive targeted therapies are likely to develop resistance to these drugs over time.
In January 2020, we received a United States FDA Fast Track Designation for use of REQORSA in combination with EGFR inhibitor Tagrisso for the treatment of NSCLC patients with EFGR mutations whose tumors progressed after treatment with Tagrisso. The median length of time that patients are treated with Tagrisso before their tumors progress is approximately eighteen (18) months.
Immunotherapies
Immunotherapy is a type of treatment that helps the immune system fight disease. Biomarker testing can help determine if high levels of PD-1/PD-L1 proteins are detected in the patient, signifying that the body’s immune system is not working to fight cancer or disease as it should. These patients could qualify for approved immunotherapies, however, not all patients benefit from immunotherapies.
Genprex is conducting preclinical studies to evaluate REQORSA in combination with checkpoint inhibitors. Preclinical data indicate that Genprex’s lead drug candidate is synergistic with checkpoint inhibitors, such as Merck’s Keytruda, meaning that the combination of drugs may be more effective than checkpoint inhibitors alone. Preclinical data also indicates that a three-drug combination of a checkpoint inhibitor, chemotherapy, and REQORSA may be more effective than the two-drug combination of a checkpoint inhibitor and chemotherapy.
Market Opportunity
AstraZeneca’s Tagrisso had more than $4 billion in worldwide gross sales in 2020, and Tagrisso is AstraZeneca’s highest grossing product. Given Genprex’s Fast Track Designation and Tagrisso’s status as the current standard of care in EGFR-mutated NSCLC, we have prioritized the clinical development of REQORSA in combination with Tagrisso. We believe this regulatory pathway positions us well in the $17.9 billion global lung cancer market, especially given the advantages of our Fast Track Designation status.
Merck’s Keytruda generated more than $14 billion in worldwide sales in 2020, and Keytruda is Merck’s highest grossing product. Keytruda is the standard of care in non-EGFR mutated NSCLC. Genprex plans to initiate a Phase I/II clinical trial evaluating REQORSA in combination with pembrolizumab.
PROGRAMS
Cancer
Cancer is a complex disease that can start in any site in the body when a tissue grows out of control and inhibits the body’s normal functioning. At the cell level, cancer often involves the dysregulation of multiple genes and cellular pathways, leading to the cell’s inability to maintain proper cellular functions. Re-establishing or blocking these pathways can be done through many therapeutic approaches, including gene therapy. More general information about cancer can be found at The American Cancer Society.
Lung Cancer
Genprex’s oncology program is focused on developing new treatments for cancer. Our initial therapeutic target is lung cancer, including non-small cell lung cancer (NSCLC) and small cell lung cancer (SCLC).
According to the World Health Organization in 2020, lung cancer was the leading cause of cancer deaths worldwide, causing more deaths than colorectal, breast, liver, or stomach cancers. In 2020, there were more than 2 million new lung cancer cases and 1.8 million deaths from lung cancer worldwide. In the United States, according to the American Cancer Society, it is estimated that in 2022 there will be more than 236,000 new cases of lung cancer and more than 130,000 deaths from this disease. NSCLC represents 84% of all lung cancers and the five-year survival rate for patients with NSCLC with distant spread is 7 percent. SCLC represents about 13% of lung cancer patients and the five-year survival rate for patients with SCLC with distant spread is 3 percent. With limited benefit from current therapies, we believe there is a significant unmet medical need for new treatments for NSCLC and SCLC in the United States and globally, and we believe REQORSA may be suitable for the majority of lung cancer patients.
Diabetes
Our diabetes gene therapy candidate, GPX-002, is being developed for the treatment of diabetes.
According to the U.S. Center for Disease Control, 37 million Americans, or approximately 11% of the population, have diabetes. It is also believed that more than 96 million Americans have prediabetes, which represents approximately 38% of the U.S. population. The prevalence of this chronic disease is continuing to rise.
Chronic diabetes conditions include Type 1 diabetes and Type 2 diabetes, both of which lead to excess sugar in the blood and can cause serious health problems. Left untreated, high blood sugar levels can damage eyes, kidneys, nerves, and the heart, and can also lead to coma and death.
TECHNOLOGY
The First Systemically Delivered Gene Therapy Used for Cancer in Humans
Our 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 we 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 active ingredient in our lead product candidate, REQORSA, is the TUSC2 gene, a tumor suppressor gene.
REQORSA consists of the TUSC2 gene encapsulated in a nanoparticle made from lipid molecules with a positive electrical charge. REQORSA is injected intravenously and can specifically target cancer cells, which generally have a negative electrical charge. Once REQORSA is taken up into a cancer cell, the TUSC2 gene is expressed into a protein that is capable of restoring certain defective functions arising in the cancer cell. REQORSA has a multimodal mechanism of action whereby it interrupts cell signaling pathways that cause replication and proliferation of cancer cells, re-establishes pathways for programmed cell death, or apoptosis, in cancer cells, and modulates the immune response against cancer cells. REQORSA has also been shown to block mechanisms that create drug resistance.
We believe 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.
Overcoming Drug Resistance
REQORSA is a pan-kinase inhibitor shown to simultaneously inhibit the EGFR and AKT oncogenic kinase pathways in vitro and in vivo. Once the cancer cell takes up the nanoparticle containing TUSC2, it is reprogrammed to die. Resistance to targeted drugs and checkpoint inhibitors develop through activation of alternate bypass pathways. For example, when PD-1 is blocked, the TIM-3 checkpoint is up-regulated. We believe that REQORSA’s multimodal activity will block emerging bypass pathways, reducing the probability that drug resistance develops.
To learn more about scientific evidence and studies supporting REQORSA and the TUSC2 gene, please refer to our Clinical Trials and TUSC2 Bibliographypages.
Diabetes Gene Therapy
GPX-002, a gene therapy for diabetes, is the most recent addition of our 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, we expect to begin a Phase I clinical trial in diabetic patients, which could be the first-ever gene therapy tested in humans for diabetes.
To learn more about scientific evidence and studies supporting GPX-002 and the Pdx1/MafA genes, please refer to our Clinical Trials and Pdx1/MafA Bibliography pages.
The First Systemic Gene Therapy Delivery Platform Used in Humans for Cancer
Our oncology drug development program utilizes our unique, proprietary non-viral ONCOPREX Nanoparticle Delivery System, which we 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 our initial product candidate, REQORSA® immunogene therapy.
Designed to Deliver Cancer-Fighting Genes Systemically
The ONCOPREX platform has been designed and optimized to deliver cancer-fighting genes into the patient’s body systemically. Using this system, we encapsulate plasmids that express tumor suppressor genes within lipid nanoparticles and intravenously administer the encapsulated plasmids which are taken up by the tumor cells, after which the tumor suppressor genes express proteins that are missing or found in low quantities in the tumor cells. Our nanoparticles are non-immunogenic, allowing repetitive therapeutic dosing and have been clinically shown to deliver molecular kinase inhibitors effectively.
Optimized Particle Size
Our systemic, nanoparticle, non-viral delivery system, which is being used in our clinical trials for the treatment of non-small cell lung cancer (NSCLC) and small cell lung cancer (SCLC), is designed to be small enough to cross tight barriers in the lungs but large enough to avoid accumulation in the liver, spleen and kidney. The nanoparticles have been shown to be taken up by tumor cells after REQORSA administration at up to 33 times the rate they are taken up by normal cells. The cationic charge of the lipid nanoparticles targets cancer cells, which facilitates endocytosis. Once inside the cancer cell, the TUSC2 gene activates signaling pathways that result in cell destruction or apoptosis.
Enhanced Safety and Efficacy Design
We have administered REQORSA to more than 50 patients in Phase 1 and 2 clinical trials using our systemic, proprietary, non-viral delivery system.
A Phase 1 clinical trial showed that systemic, intravenous therapy using the ONCOPREX Nanoparticle Delivery System was shown to selectively and preferentially target primary and metastatic tumor cells, resulting in clinically significant anticancer activity. The nanoparticles are non-immunogenic, allowing repetitive therapeutic dosing and providing extended half-life in the circulation.
Our earlier clinical trials have also shown that the ONCOPREX Nanoparticle Delivery System is well tolerated in humans and can safely deliver high therapeutic doses. We believe the ONC-001 clinical trial was the first systemic gene therapy clinical trial using a nanoparticle delivery system to deliver a tumor suppressor gene.
Genprex (NASDAQ:GNPX) Releases New Patient Video Interview Describing Positive Experience In Clinical
Independent Researchers Find That TUSC2, the Gene Delivered by Genprex’s REQORSA®, Functions as a Novel Tumor Suppressor for Glioblastoma
April 18, 2023
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) — 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.”
REQORSA® Therapy is currently in development for the treatment of non-small cell lung cancer (NSCLC) and small-cell lung cancer (SCLC). REQORSA® consists of the TUSC2 gene expressing plasmid encapsulated in non-viral nanoparticles made from lipid molecules (the Company’s ONCOPREX® Nanoparticle Delivery System) with a positive electrical charge. REQORSA® is injected intravenously and specifically targets cancer cells, taking advantage of leaky tumor vascularity, increased tumor pinocytosis, and attraction of positively charged lipid nanoparticles to negatively charged cancer cells. REQORSA is designed to deliver the functioning TUSC2 gene to cancer cells while minimizing their uptake by normal tissue.
According to the National Brain Tumor Society, glioblastoma is one of the most complex, deadly, and treatment-resistant cancers. More than 13,000 Americans were expected to receive a glioblastoma diagnosis in 2022. Glioblastoma accounts for 49.1 percent of all primary malignant brain tumors. There have only been five drugs and one device ever approved by the U.S. Food and Drug Adminstration for the treatment of glioblastoma.
Other independent researchers and studies have found that TUSC2 therapy may benefit other types of cancer, including head and neck, breast (including triple-negative breast cancer), renal cell (kidney), thyroid, and soft tissue sarcoma, as well as NSCLC and SCLC. For more information and additional studies supporting TUSC2, please refer to our Bibliography Page.
NEWS
Bell2Bell Podcast featuring CEO Rodney Varner & CFO Ryan Confer of Genprex, Inc. (NASDAQ: GNPX)
July 6, 2023
Bell2Bell’s latest podcast features Chairman, President and CEO Rodney Varner and CFO Ryan Confer of Genprex, Inc. (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients living with cancer and diabetes. In this interview, Varner and Confer discuss the journeys that led them to Genprex and the promise of the company’s drug development pipeline.
Genprex Granted Chinese Patent for REQORSA® Immunogene Therapy in Combination with PD-1 Antibodies to Treat Cancers
July 5, 2023
Provides Additional Protection in Large Markets for Therapeutic Combination in Acclaim-2 Phase 1/2 Clinical Trial
Genprex Receives U.S. FDA Fast Track Designation for REQORSA® Immunogene Therapy in Combination with Tecentriq® for the Treatment of Small Cell Lung Cancer
June 28, 2023
Third FDA Fast Track Designation Further Validates the Potential of REQORSA
AUSTIN, Texas — (June 28, 2023) — 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 the U.S.
Genprex’s ASCO Company Update: Dr. Mark Berger, Chief Medical Officer, Shares Insights on Bell2Bell Podcast
June 8, 2023
Bell2Bell’s latest podcast features Dr. Mark Berger, Chief Medical Officer of Genprex Inc. (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients living with cancer and diabetes.
Genprex (NASDAQ: GNPX) Releases New Patient Video Interview Describing Positive Experience in Clinical Trial
June 1, 2023
On the heels of the release of recent positive safety and early efficacy data related to the phase 1 portion of its Acclaim-1 phase 1/2 clinical trial for non-small cell lung cancer evaluating REQORSA in combination with Tagrisso, Genprex has released a new patient video featuring an NSCLC patient from the phase 1 portion of its Acclaim-1.
Genprex Publishes Positive Clinical Data from Phase 1 Portion of Acclaim-1 Clinical Trial Evaluating REQORSA® Immunogene Therapy in Non-Small Cell Lung Cancer at the 2023 ASCO Annual Meeting
May 25, 2023
Results Show Combination of REQORSA® and Tagrisso® was Well Tolerated at All Three Dose Levels of Phase 1 Trial with Encouraging Evidence of Efficacy Observed
Genprex Unveils New Video Featuring Chief Medical Officer Discussing Positive Preclinical Data With NPRL2 Gene Therapy Utilizing Non-Viral ONCOPREX® Nanoparticle Delivery System
May 23, 2023
Chief Medical Officer Provides Overview of Preclinical Data Recently Presented at the 2023 American Association of Cancer Research (AACR) annual meeting
Genprex to Participate and Present at Upcoming May Industry and Investor Conferences
May 8, 2023
Presentations to Highlight Company’s Gene Therapies for Cancer and Diabetes
Genprex Collaborators Report Positive Preclinical Data With NPRL2 Gene Therapy Utilizing Non-Viral ONCOPREX® Nanoparticle Delivery System in Non-Small Cell Lung Cancer at the 2023 AACR Annual Meeting
April 19, 2023
Provides Preclinical Validation of the ONCOPREX® Nanoparticle Delivery System with a Second Tumor Suppressor Gene
Independent Researchers Find That TUSC2, the Gene Delivered by Genprex’s REQORSA®, Functions as a Novel Tumor Suppressor for Glioblastoma
April 18, 2023
Pre-clinical study results show TUSC2 overexpression reduces colony formation, neurosphere formation and promotes apoptosis in vitro, and suppresses tumor growth in vivo
Genprex CEO Commends FDA’s Initiative to Accelerate Approval Process for Gene Therapies
March 23, 2023
Genprex is advancing its novel gene therapies for large patient populations with cancer and diabetes who currently have limited treatment options
Genprex to Present at Bioprocessing Summit Conference
March 13, 2023
Genprex Chief Manufacturing and Technology Officer to Provide Insight on Manufacturing Gene Therapies
Genprex to Participate in March Investor and Industry Conferences
March 8, 2023
Company to Showcase its Innovate Gene Therapy Programs in Oncology and Diabetes
Genprex, Inc. Announces $4 Million Registered Direct Offering With a Single, Healthcare-Focused Institutional Investor
February 27, 2023
The gross proceeds from the offering are expected to be approximately $4 million, before deducting placement agent fees and other estimated offering expenses.
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
February 23, 2023
Results show statistically significant decreases in insulin requirements, increases in c-peptide levels and improvements in glucose tolerance compared to baseline
Genprex Signs Exclusive License to Additional Diabetes Technology with the University of Pittsburgh
January 5, 2023
Technologies Licensed from University of Pittsburgh May Have the Potential to Provide Long-Term Efficacy and to Change the Course of this Disease for the Millions of Patients Around the World with Type 1 or Type 2 Diabetes
Genprex Announces Selection of Preclinical Data for Oral Presentation at 16th International Conference on Advanced Technologies & Treatments for Diabetes
January 4, 2023
Exciting Data from University of Pittsburgh Researchers in Non Human Primates that Underpins Genprex’s Gene Therapy Program in Diabetes to be Showcased
Genprex Strengthens Diabetes Gene Therapy Program with License of Additional Technology from University of Pittsburgh
December 15, 2022
Technology for modulating autoimmunity expands intellectual property portfolio
Genprex Receives Safety Review Committee Approval to Proceed to Final Cohort in Acclaim-1 Phase 1 Dose Escalation Trial of REQORSA® in Combination with Tagrisso® in Advanced Non-Small Cell Lung Cancer
December 14, 2022
Recommendation to Advance to Increased Dose in Third and Final Cohort of Phase 1 Portion of Trial Indicates Favorable Safety Profile of Novel Gene Therapy in Solid Tumor Cancer
Sincerely,