OUR NEW PROFILE IS: (NASDAQ: GNPX)
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GNPX HAS ALREADY RECEIVED FAST TRACK DESIGNATION FOR ACCLAIM 2
Genprex Announces U.S. Patent for REQORSA™ Immunogene Therapy in Combination with Immune Checkpoint Inhibitors to Treat Cancers
CHECK OUT THE INVESTOR PRESENTATION HERE
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Hello Everyone,
Happy New Year to all of our readers. 2022 was our busiest in history despite a historically weak market that lacked any real key drivers and a world wide economy in recession. We were able to put a lot of companies in front of you to research. Many of them were biotechs.
Pull up GNPX right away and get it on your screen.
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 unique, 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 proteins that are deficient. The Company’s lead product candidate, REQORSA™ (quaratusugene ozeplasmid), is being evaluated as a treatment for non-small cell lung cancer (NSCLC). REQORSA has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. REQORSA has also been shown to block mechanisms that create drug resistance. In 2020, the U.S. Food and Drug Administration (FDA) granted Fast Track Designation for REQORSA for NSCLC in combination therapy with AstraZeneca’s Tagrisso® (osimertinib) for patients with EFGR mutations whose tumors progressed after treatment with Tagrisso. In 2021, the FDA granted Fast Track Designation for REQORSA for NSCLC in combination therapy with Merck & Co’s Keytruda® (pembrolizumab) for patients whose disease progressed after treatment with Keytruda.
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.
Take a look at the chart. Notice the recent rally in GNPX as it saw major support at a buck and is slingshotting right out of the high end of it’s 6 month average by a quick glance. We have seen interest pick up slightly too as of late.
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
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 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
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
AUSTIN, Texas — (December 14, 2022) — 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 Safety Review Committee (SRC) has approved continuation to the third and final cohort in the dose escalation 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). In 2020, 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.
Acclaim-1 is an open-label, multi-center Phase 1/2 clinical trial evaluating the Company’s lead drug candidate, REQORSA Immunogene Therapy, in combination with Tagrisso (osimertinib) in patients with late-stage non-small cell lung cancer (NSCLC) whose disease progressed after treatment with Tagrisso.
The SRC is comprised of three physicians who are principal investigators in the trial. The SRC may recommend that the trial continues at the same dose or at a lower dose, that it escalates to a higher dose, or that the study be terminated altogether due to safety concerns.
“The SRC’s recommendation to increase the dosing of REQORSA is further confirmation of its favorable safety profile and it enables us to advance Acclaim-1 into the final cohort of the Phase 1 dose escalation portion of the study,” said Mark Berger, M.D., Chief Medical Officer of Genprex. “We look forward to completing enrollment of this final cohort in the first quarter of 2023.”
The Accaim-1 trial includes up to three sequential dose escalation cohorts that will treat study participants with REQORSA intravenously on Day 1 in addition to osimertinib 80 mg fixed dose oral daily tablet during 21-day treatment cycles until disease progression or unacceptable toxicity. The first group received REQORSA IV infusion at 0.06 mg/kg, the second group received 0.09 mg/kg, and the third group will receive 0.12 mg/kg.
Following successful completion of the Phase 1 dose escalation portion of the Acclaim-1 study, the Company will advance into the dose expansion portion of the study, which will evaluate the toxicity profile of REQORSA in combination with Tagrisso in patients with different eligibility criteria, and will also evaluate efficacy and other endpoints.
“The principal advantage of adding the dose expansion portion to Acclaim-1 is to gain efficacy data earlier than we would otherwise have received it from the Phase 2 portion of the study. We also will receive this data in the two distinct patient populations represented by the two expansion cohorts, which we believe will further increase the likelihood of a successful Phase 2 trial,” added Dr. Berger.
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
AUSTIN, Texas — (December 15, 2022) — 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 it has entered into an exclusive license agreement (the Agreement) with
the University of Pittsburgh, granting Genprex a worldwide, exclusive license to certain patent applications and related technology and a worldwide, non-exclusive license to use certain related know-how, all related to modulating autoimmunity in Type 1 diabetes by using gene therapy. The preclinical technology transforms macrophages enabling them to reduce autoimmune activity in Type 1 diabetes and could be complementary to the Company’s existing diabetes technology.
“Gaining exclusive access to technology that modulates the immune system by transforming macrophages could prove to be significant to our broader research partnership with 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,” said Mark Berger, MD, Chief Medical Officer of Genprex. “We are making significant strides in our program with Dr. Gittes’s innovative approach to treating diabetes by the transformation of alpha cells into beta-like cells and are excited to add to our arsenal this additional technology also out of Dr. Gittes’s lab, in collaboration with the laboratory of Dr. Xangwei Xiao, Assistant Professor of Surgery, also in the Division of Pediatric Surgery at the University of Pittsburgh’s School of Medicine. Not only could this new approach be used to reduce autoimmune activity in Type 1 diabetes by modulating the immune system but potentially it could also work in conjunction with the technology we have licensed previously.”
“With diabetes reaching epidemic proportions around the world, the work Dr. Gittes is pursuing in diabetes is absolutely critical. In the U.S. alone, there are more than 37 million people with diabetes (approximately 1.9 million of whom have Type 1 diabetes) and another approximately 96 million Americans who are pre-diabetic, or have abnormally elevated blood sugar levels. The opportunity to change the course of this disease with gene therapy is extremely compelling, and increasing our exclusive access to intellectual property could prove to be pivotal to our pathway forward,” said Rodney Varner, President and Chief Executive Officer of Genprex.
The Company signed an exclusive license agreement with the University of Pittsburgh in 2020. The gene therapy approach under the original license is comprised 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, these 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 will be rejuvenated and replenished.
This gene therapy approach was developed by Dr. Gittes. His preclinical research in this area has been published in peer-reviewed scientific publications, and he is the recipient of several research grants, including a $2.59 million grant awarded by the National Institutes of Health (NIH) National Institute of Diabetes and Digestive and Kidney Diseases. Earlier studies in diabetic mouse models showed that the gene therapy restored normal blood glucose levels for an extended period of time, typically around four months. It is believed that the duration of restored blood glucose levels in mice could translate to decades in humans. Preliminary data from a more recent study in a non-human primate model of Type 1 diabetes also have been promising. Data from this study are expected to be presented at a scientific meeting during the first quarter of 2023.
NEWS
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
Genprex to Present at Upcoming December Investor Conference
December 2, 2022
Corporate Presentation to Highlight Company’s Gene Therapies for Cancer and Diabetes
Genprex to Present at Upcoming October Investor and Industry Conferences
October 7, 2022
Corporate and Clinical Presentations to Highlight Company’s Gene Therapies for Cancer and Diabetes
Genprex to Present at Upcoming September Investor Conference
September 8, 2022
Corporate Presentation to Highlight Company’s Gene Therapies for Cancer and Diabetes
Genprex Announces U.S. Patent for REQORSA™ Immunogene Therapy in Combination with Immune Checkpoint Inhibitors to Treat Cancers
August 16, 2022
Intellectual Property Protection for Therapeutic Combination in Acclaim-2 Phase 1/2 Clinical Trial
Genprex Announces Safety Review Committee Approves Dose Escalation in Acclaim-1 Phase 1/2 Trial of REQORSA™ in Combination with Tagrisso® in Non-Small Cell Lung Cancer
August 15, 2022
Recommends Advancing Acclaim-1 to Increased Dose in Second Cohort of Phase 1 Portion of Trial
Genprex to Participate in Next Generation Lipid-Based Nanoparticles Delivery Summit
July 13, 2022
Company Manufacturing Leads Selected as Thought Leaders to Discuss Advances in Lipid Nanoparticle Delivery Systems
Genprex to Present at Upcoming June Investor Conferences
June 2, 2022
Corporate Presentation to Highlight Company’s Gene Therapies for Cancer and Diabetes
Genprex to Present at Upcoming Investor Conference in May 2022
May 19, 2022
Corporate Presentation to Highlight Company’s Gene Therapies for Cancer and Diabetes
Genprex’s Chief Medical Officer to Be Featured as an Expert Panelist at the 33rd Annual Cancer Progress Conference
May 9, 2022
Live panel discussion will explore considerations with respect to positioning of cell-based and other emerging immunotherapy platforms in solid tumors
Genprex Issues Shareholder Letter and Provides 2022 Corporate Update
May 5, 2022
Company achieves major milestones in clinical development programs in 2022
Patient treatment in Acclaim-2 clinical trial commences
Genprex to Participate in Upcoming Investor and Industry Conferences in April 2022
April 18, 2022
Presentations to highlight the Company’s gene therapies for cancer and diabetes
Genprex Announces the Opening for Enrollment of its Phase 1/2 Acclaim-2 Clinical Trial of REQORSA™ Immunogene Therapy in Combination with Keytruda® to Treat Non-Small Cell Lung Cancer
March 31, 2022
Company Has FDA Fast Track Designation for Combination of REQORSA and Keytruda
Genprex to Participate in Upcoming Investor Conference in March
March 23, 2022
Investor presentation to highlight the Company’s gene therapies for cancer and diabetes
MANAGEMENT
Sincerely,