Genprex Signs Sponsored Research Agreement with the University of Pittsburgh to Study Diabetes Gene Therapy in Type 1 and Type 2 Diabetes

Rhea-AI Summary

Genprex (NASDAQ: GNPX) has signed a new Sponsored Research Agreement (SRA) with the University of Pittsburgh to study GPX-002, its gene therapy treatment for Type 1 (T1D) and Type 2 diabetes (T2D) in animal models. Previous preclinical data showed statistically significant improvements in insulin requirements, c-peptide levels, and glucose tolerance. GPX-002 uses an AAV vector containing Pdx1 and MafA genes administered into the pancreatic duct. In T1D, it transforms alpha cells into functional beta-like cells that produce insulin while potentially evading immune response. For T2D, the therapy aims to rejuvenate exhausted beta cells. The treatment can be administered in humans through routine endoscopy.

Positive

• Preclinical data showed statistically significant improvements in insulin requirements and glucose tolerance
• Treatment can be administered through routine endoscopy procedure, suggesting ease of delivery
• Single therapy approach works for both Type 1 and Type 2 diabetes, expanding market potential
• Continued research partnership with prestigious University of Pittsburgh strengthens development pipeline

Negative

• Still in preclinical stage with no human trials yet
• Success in animal models may not translate to human efficacy
• Timeline to human clinical trials remains undefined

Insights

Biotechnology and Gene Therapy Expert

Genprex renews University of Pittsburgh research partnership to advance promising diabetes gene therapy in animal studies before human trials.

This announcement represents a strategic continuation in Genprex's diabetes gene therapy program through a renewed research agreement with the University of Pittsburgh following their initial two-year collaboration. This sustained partnership suggests the preliminary results were sufficiently encouraging to warrant expanded investigation.

The scientific approach behind GPX-002 is particularly noteworthy. The therapy utilizes an adeno-associated virus (AAV) vector containing Pdx1 and MafA genes delivered directly into the pancreatic duct - a procedure that can be performed via routine endoscopy in humans. The mechanism of action differs slightly between diabetes types:

• For Type 1 diabetes: Converting pancreatic alpha cells into insulin-producing beta-like cells that may evade autoimmune destruction
• For Type 2 diabetes: Rejuvenating exhausted beta cells to restore insulin production capability

The preclinical results referenced show three critical improvements in diabetes metrics: decreased insulin requirements, increased c-peptide levels (indicating endogenous insulin production), and improved glucose tolerance. These represent precisely the endpoints one would want to see in early diabetes therapy development.

Using the same construct for both major forms of diabetes represents an efficient development strategy that could maximize return on research investment while addressing two substantial patient populations. The direct pancreatic delivery method potentially overcomes distribution challenges seen with other gene therapies.

This announcement indicates progression through the typical development pathway toward eventual human trials, but investors should recognize that the program remains in animal testing stages. Many promising preclinical therapies face challenges in translation to human subjects, and the timeline to potential approval typically spans multiple years from this stage. This research agreement represents steady advancement rather than an imminent clinical or commercial breakthrough.

Advances Relationship and Collaboration with the University of Pittsburgh of the Commonwealth System of Higher Education

AUSTIN, Texas, May 7, 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 that following the recent completion of its two-year Sponsored Research Agreement (SRA) with the University of Pittsburgh of the Commonwealth System of Higher Education (Pitt) to study Type 1 diabetes (T1D) and Type 2 diabetes (T2D), the parties have entered into a new SRA to study GPX-002, the Company's gene therapy for diabetes, in T1D and T2D in animal models.

"We continue to make advancements in our studies of GPX-002 in both T1D and T2D, including mouse models, construct optimization and studies in other animal models," said Ryan Confer, President and Chief Executive Officer at Genprex. "As we advance into new animal studies in both T1D and T2D, we look forward to exploring how GPX-002 may decrease insulin requirements and improve glucose tolerance. We believe these studies set us on a path toward human clinical trials."

Genprex has entered into a new SRA with Pitt in which the Company will sponsor preclinical studies of GPX-002 for the treatment of T1D and T2D in animal models. Preclinical data from an animal study of GPX-002 in T1D showed statistically significant decreases in insulin requirements, increases in c-peptide levels and improvements in glucose tolerance in the treated animals compared to baseline. These groundbreaking results support the hypothesis that this disruptive gene therapy approach developed by Pitt researchers may be a promising treatment for both T1D and T2D.

"We are excited to continue our pioneering gene therapy work in diabetes through this SRA," said Mark Berger, MD, Chief Medical Officer of Genprex. "Our preclinical studies have produced compelling data that have encouraged us to support the expansion of this promising gene therapy approach to treat T2D with GPX-002 in animal models."

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 and Prevention 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.

Interested investors and shareholders are encouraged to sign up for press releases and industry updates by visiting the Company Website, registering for Email Alerts and by following Genprex on Twitter, Facebook and LinkedIn.

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, 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 and regulatory approvals, including, but not limited to, the Company's diabetes clinical development program; Genprex's plans, intentions, and expections for GPX-002 and the new SRA with Pitt, including the Company's goals and plans for preclinical studies and potential future clinical trials of GPX-002, and the Company's beliefs about the anticipated effects of GPX-002 and its potential as a therapeutic approach; 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; and Genprex's intellectual property and licenses.

These forward-looking statements should not be relied upon as predictions of future events and Genprex cannot assure you that the events or circumstances discussed or reflected in these statements will be achieved or will occur. If such forward-looking statements prove to be inaccurate, the inaccuracy may be material. You should not regard these statements as a representation or warranty by Genprex or any other person that Genprex will achieve its objectives and plans in any specified timeframe, or at all. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. Genprex disclaims any obligation to publicly update or release any revisions to these forward-looking statements, whether as a result of new information, future events or otherwise, after the date of this press release or to reflect the occurrence of unanticipated events, except as required by law.

Genprex, Inc.
(877) 774-GNPX (4679)

GNPX Investor Relations
investors@genprex.com

GNPX Media Contact
Kalyn Dabbs
media@genprex.com

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SOURCE Genprex, Inc.

FAQ

What is GPX-002 and how does it work for diabetes treatment?

GPX-002 is Genprex's gene therapy that uses an AAV vector containing Pdx1 and MafA genes, administered into the pancreatic duct. For T1D, it transforms alpha cells into insulin-producing beta-like cells, while in T2D it rejuvenates exhausted beta cells.

What results has GNPX's GPX-002 shown in preclinical studies?

Preclinical studies showed statistically significant decreases in insulin requirements, increases in c-peptide levels, and improvements in glucose tolerance compared to baseline in animal models.

How is Genprex's GPX-002 diabetes treatment administered?

GPX-002 is administered directly into the pancreatic duct through a routine endoscopy procedure in humans.

What is the difference in how GPX-002 treats Type 1 vs Type 2 diabetes?

In Type 1 diabetes, GPX-002 transforms alpha cells into insulin-producing beta-like cells that may evade immune response. In Type 2 diabetes, it works by rejuvenating and replenishing exhausted beta cells.

What is the current development stage of GNPX's diabetes gene therapy?

GPX-002 is currently in preclinical development, with ongoing animal studies through a research agreement with the University of Pittsburgh. Human clinical trials have not yet begun.