Researching Novel Diabetes Gene Therapy Using Potentially Re-Dosable Non-Viral Delivery System
Strategic Collaboration with CDMO Partner Positions Genprex as a Thought-Leader in Diabetes Market
AUSTIN, Texas, Feb. 19, 2025 /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 the advancement of its diabetes program through a strategic collaboration with a contract development and manufacturing organization (CDMO) to research an alternative second generation approach using a non-viral lipid nanoparticle delivery of the Company's diabetes gene therapy drug candidate.
"This collaboration with our CDMO partner is separate from our existing preclinical research programs and seeks to evaluate potential next generation construct optimization," said Ryan Confer, President and Chief Executive Officer at Genprex. "This exploratory research represents our forward-thinking approach within our diabetes program to determine if our novel diabetes gene therapy could be delivered using a non-viral delivery system, similar to Genprex's oncology drug candidate. While AAV delivery is a well understood delivery mechanism, there are many benefits to a non-viral delivery system, including the potential for re-dosing patients to optimize treatment. This collaboration positions Genprex as a thought-leader in gene therapy, specifically within the diabetes market where there are only symptom managing therapies but no disease modifying technologies."
Separate from the ongoing preclinical studies, Genprex and its CDMO partner are researching and exploring the potential delivery of these genes using a non-viral lipid nanoparticle. Many gene therapies rely on viral based delivery systems. The benefit of the viral system is that viruses are skilled at penetrating cells. However, viruses cannot be readministered, due to the development of an immune response against the virus capsid.
"Prior studies have shown that Non-Obese Diabetic (NOD) mice became hyperglycemic again a median of 17 days after treatment with syngeneic islet cell transplants," said Mark Berger, Chief Medical Officer at Genprex. "Our recent preclinical studies of Type 1 NOD mouse models treated with GPX-002 have shown durability of glucose control to approximately 120 days, or 4 months. This suggests that that the new insulin producing beta-like cells that our therapy produced are poorly recognized by the autoimmune process in NOD mice. Our team continues to explore ways to enhance durability, and expanding our research to include a non-viral delivery system could allow for multiple treatments to be administered."
GPX-002 is currently being developed using the same construct 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 Diabetes According to the U.S. Center for Disease Control as of 2024, 38.4 million Americans, or approximately 11.6% of the U.S. population, have diabetes. Approximately 10% of this total has T1D and approximately 90-95% of this total has T2D. It is also believed that more than 97 million Americans aged 18 years or older have prediabetes. In 2021, approximately 537 million adults (20-79 years) worldwide were living with diabetes, and the total number of people living with diabetes is projected to rise to 643 million by 2030 and 783 million in 2045. Approximately 7.7 million adults over the age of 20 live with T1D worldwide, and approximately 1.6 million children and adolescents under the age of 20 live with T1D worldwide. Also in 2021, diabetes caused more than 6.7 million deaths globally and diabetes resulted in approximately $966 billion dollars in health expenditures, a 316% increase over the preceding fifteen years.
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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Cautionary Language Concerning Forward-Looking Statements 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, 2023.
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 and regulatory approvals, including, but not limited to, the Company's diabetes clinical development program; the effect of Genprex's product candidates, alone and in combination with other therapies, on cancer and diabetes; the effects of any strategic research and development prioritization initiatives, and any other strategic alternatives or other efforts that Genprex takes or may take in the future that are aimed at optimizing and re-focusing Genprex's diabetes, oncology and/or other clinical development programs including prioritization of resources, and the extent to which Genprex is able to implement such efforts and initiatives successfully to achieve the desired and intended results thereof; 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, including the results of our early research to extend durability of glucose control and the potential results of further research with a CDMO to utilize a non-viral delivery system that would allow multiple treatments to be administered; and Genprex's intellectual property and licenses.
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