Preliminary Preclinical Data Demonstrates In Vivo Proof-of-Concept for Novel Diabetes Gene Therapy in Type 2 Diabetic Non-Human Primates
GPX-002 Rejuvenated Exhausted Beta Cells and Normalized Glucose Levels in Type 2 Diabetic Mice
AUSTIN, Texas, Jan. 6, 2026 /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 preliminary in vivo preclinical proof-of-concept data for GPX-002, the Company's diabetes gene therapy drug candidate which may have the potential for long-term control of Type 1 diabetes (T1D) and Type 2 diabetes (T2D), in a non-human primate (NHP) model of T2D and in a mouse model of T2D.
Genprex's research collaborators are conducting preclinical studies of GPX-002 in naturally occurring T2D NHP models. The first NHP was treated with a novel infusion process whereby GPX-002 is administered directly into the pancreatic duct using an adeno-associated virus (AAV) vector containing the Pdx1 and MafA genes and an insulin promoter. The second NHP was treated with GPX-002 by direct injection into the pancreas.
"The progression from mouse model studies into NHP studies demonstrates the progression of preclinical drug development of GPX-002 toward human clinical trials," said Mark Berger, MD, Chief Medical Officer at Genprex. "We have demonstrated proof-of-concept in Type 1 diabetic mouse models, where GPX-002 converted alpha cells into beta-like cells that appropriately produced insulin and normalized blood glucose levels, as well as in Type 2 diabetic mouse models, where beta cells are rejuvenated. Moving into NHP studies in both Type 1 and Type 2 diabetes has enabled us to establish critical proof-of-concept advancements in models that share significant metabolic similarities with humans."
GPX-002 in Type 2 Diabetic Non-Human Primates
Based on the study of the two T2D NHPs, key preliminary data include support for the hypothesis that GPX-002 can rejuvenate exhausted beta cells in T2D. AAV infusion into the pancreatic duct was performed on the first NHP that resulted in improvements in glucose tolerance testing over several months, and at seven months the NHP had normal glucose tolerance testing. This same NHP had severe T2D and abnormally high glucose tolerance testing at baseline just prior to the infusion of the GPX-002 AAV vector. Improvements in glucose tolerance testing, accompanied by decreases in insulin requirements, are consistent with the reprogramming of exhausted beta cells that were rejuvenated by GPX-002 treatment.
Direct pancreas injection in the second NHP resulted in significant improvements in glucose tolerance testing at three months but did not achieve normal glucose tolerance testing results. Pancreatic injection of GPX-002 still shows evidence of efficacy against T2D, however, intraductal infusion appears to be a highly favorable method of delivery to alpha and beta cells.
Immunosuppression therapy for a defined period of time may prevent immune reaction to the AAV. Although T2D studies with AAV constructs in mice do not require immunosuppression, NHPs have an immune response against AAV constructs. AAV proteins appear to be expressed on infected cells for six months or less, as our initial research in the first NHP suggests that after six months no further immunosuppression is required. This new understanding of the need for immunosuppression in NHPs for a defined period of time may lead to better results than the shorter immunosuppression regimens used previously.
"We are highly encouraged by this preliminary preclinical data for GPX-002 in Type 2 diabetes. This work strengthens our existing research, which has already demonstrated improved glucose homeostasis by reprogramming alpha cells in Type 1 diabetes, and now demonstrates potential to unlock treatment opportunities for the remaining 90 to 95 percent of diabetes patients afflicted by Type 2 diabetes utilizing our diabetes technologies," said Ryan Confer, President and Chief Executive Officer at Genprex. "We believe that these results represent significant progress for animal studies of gene therapy for Type 2 diabetes, and we are excited to continue evaluating GPX-002 further in animal studies and move toward human clinical trials."
Researchers are continuing preclinical studies of GPX-002 therapy in NHP models of both T1D and T2D and are planning formal toxicology studies and subsequent Investigational New Drug (IND) submission. Current studies in NHPs are evaluating efficacy after six months of immunosuppression. The results from preclinical testing of GPX-002 may not predict the results that will be obtained in later phase clinical trials of GPX-002.
GPX-002 in Type 2 Diabetic Mice
Genprex's research collaborators have also conducted preclinical studies of GPX-002 in T2D mice, where no immunosuppression was required. In the mouse model, researchers treated T2D mice with a novel infusion process whereby GPX-002 is administered directly into the pancreatic duct using an AAV vector containing the Pdx1 and MafA genes. Researchers showed that in both in vivo and ex vivo studies, T2D mice and diabetic islets treated with GPX-002 demonstrated increased glucose stimulated insulin secretion, thus supporting the novel gene therapy's ability to rejuvenate and replenish exhausted beta cells. In T2D, pancreatic beta cells become unable to produce and secrete enough insulin to manage high blood sugar levels, a condition known as beta cell dysfunction. In this study after GPX-002 treatment, the once exhausted beta cells were rejuvenated, allowing for the proper function of releasing insulin and producing glucose.
At four weeks post-surgery, researchers also conducted glucose tolerance testing which showed statistically significant improvement in T2D mice compared to baseline, the reversal of hyperglycemia in T2D mice and demonstrated normal glucose levels in T2D mice. These results further strengthen GPX-002's capability to rejuvenate and replenish exhausted beta cells in T2D.
About GPX-002
GPX-002 is currently being developed for the treatment of both Type 1 diabetes (T1D) and Type 2 diabetes (T2D). The same general novel approach is used in each of T1D and T2D whereby an adeno-associated virus (AAV) vector containing the Pdx1 and MafA genes is administered directly into the pancreatic duct. In humans, this can be done with a routine endoscopy procedure. In T1D, GPX-002 is designed to work by transforming alpha cells in the pancreas into functional beta-like cells, which can produce insulin but may be distinct enough from beta cells to evade the body's immune system. In vivo, preclinical studies show that GPX-002 restored normal blood glucose levels for an extended period of time in T1D mouse models. In T2D, where autoimmunity is not at play, GPX-002 is believed to rejuvenate and replenish exhausted beta cells.
About Genprex, Inc.
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 systemic, non-viral Oncoprex® Delivery System which encapsulates the gene-expressing plasmids using lipid-based nanoparticles in a lipoplex form. The resultant product is administered intravenously, where it is taken up by tumor cells that then express tumor suppressor proteins that were deficient in the tumor. The Company's lead product candidate, Reqorsa® Gene Therapy (quaratusugene ozeplasmid), is being evaluated in two clinical trials as a treatment for NSCLC and SCLC. Each of Genprex's lung cancer clinical programs has received a Fast Track Designation from the FDA for the treatment of that patient population, and Genprex's SCLC program has received an FDA Orphan Drug Designation. Genprex's diabetes gene therapy approach is comprised of a novel infusion process that uses an AAV vector to deliver Pdx1 and MafA genes directly to the pancreas. In models of Type 1 diabetes, GPX-002 transforms alpha cells in the pancreas into functional beta-like cells, which can produce insulin but may be distinct enough from beta cells to evade the body's immune system. In a similar approach for Type 2 diabetes, where autoimmunity is not at play, GPX-002 is believed to rejuvenate and replenish exhausted beta cells.
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Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are made on the basis of the current beliefs, expectations and assumptions of management, are not guarantees of performance and are subject to significant risks and uncertainty. These forward-looking statements should, therefore, be considered in light of various important factors, including those set forth in Genprex's reports that it files from time to time with the Securities and Exchange Commission and which you should review, including those statements under "Item 1A - Risk Factors" in Genprex's Annual Report on Form 10-K for the year ended December 31, 2024.
Because forward-looking statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding: Genprex's ability to advance the clinical development, manufacturing and commercialization of its product candidates in accordance with projected timelines and specifications; the timing and success of Genprex's clinical trials, its intended regulatory submissions and any resulting regulatory approvals, including but not limited to, the Company's beliefs about the anticipated effects of GPX-002 and its potential as a therapeutic approach in T1D and T2D; the effect of Genprex's product candidates, alone and in combination with other therapies, on cancer and diabetes; Genprex's future growth and financial status, including Genprex's ability to maintain compliance with the continued listing requirements of The Nasdaq Capital Market and to continue as a going concern and to obtain capital to meet its long-term liquidity needs on acceptable terms, or at all; Genprex's commercial and strategic partnerships, including those with its third party vendors, suppliers and manufacturers and their ability to successfully perform and scale up the manufacture of its product candidates; Genprex's intellectual property and licenses; and Genprex's current expectations, estimates, forecasts and projections about the industry and markets in which it operates.
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