Genprex's Gene Therapy GPX-002 Shows Promise in Type 2 Diabetes Animal Studies
Key Insights
Genprex's GPX-002 gene therapy demonstrated proof-of-concept in Type 2 diabetic non-human primates, with one animal achieving normal glucose tolerance after seven months of treatment.
The therapy uses an adeno-associated virus vector to deliver Pdx1 (search) and MafA (search) genes directly into the pancreatic duct, designed to rejuvenate exhausted beta cells (search) in Type 2 diabetes (search).
In Type 2 diabetic mice, GPX-002 treatment resulted in increased glucose-stimulated insulin secretion and reversal of hyperglycemia (search), with glucose levels returning to normal four weeks after treatment.
Genprex Inc. has reported positive preliminary preclinical results for its diabetes gene therapy candidate GPX-002, demonstrating in-vivo proof-of-concept in both Type 2 diabetic non-human primates and mice. The findings support the potential of GPX-002 to restore insulin-producing function by rejuvenating exhausted beta cells (search), expanding the therapy's reach to address the much larger Type 2 diabetes (search) population.
Novel Gene Delivery Approach Shows Efficacy
GPX-002 uses an adeno-associated virus (AAV) vector to deliver the Pdx1 (search) and MafA (search) genes directly into the pancreatic duct. In Type 2 diabetes (search), where beta cells (search) lose their ability to produce and secrete insulin, the therapy is designed to replenish and restore these cells. According to 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 (search) 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."
Promising Results in Non-Human Primates
In the non-human primate study, two animals with Type 2 diabetes (search) received different delivery methods of GPX-002. The first animal received the therapy through an intraductal infusion, which led to steady improvements in glucose tolerance over several months. By seven months, this primate demonstrated normal glucose tolerance, despite having severe Type 2 diabetes at baseline.
The second primate, treated via direct pancreatic injection, also showed significant improvement in glucose tolerance testing at three months, though it did not achieve full normalization. According to Genprex, the results suggest that intraductal infusion may be a more effective delivery method for targeting alpha and beta cells (search).
Immunosuppression Requirements and Optimization
Researchers noted that non-human primates require a defined period of immunosuppression when treated with AAV-based therapies. Early observations indicate that immune responses diminish after approximately six months, potentially allowing for improved outcomes with optimized immunosuppression regimens. The company found that AAV proteins appear to be expressed on infected cells for six months or less, suggesting that after six months no further immunosuppression may be required.
Strong Activity Demonstrated in Mouse Models
In Type 2 diabetic mice, GPX-002 produced strong signals of activity without requiring immunosuppression. Treated animals showed increased glucose-stimulated insulin secretion and reversal of hyperglycemia (search), with glucose levels returning to normal four weeks after treatment. At four weeks post-surgery, glucose tolerance testing showed statistically significant improvement in Type 2 diabetic mice compared to baseline, demonstrating the reversal of hyperglycemia and normal glucose levels.
Expanding Therapeutic Potential
Ryan Confer, President and Chief Executive Officer at Genprex, stated, "We are highly encouraged by this preliminary preclinical data for GPX-002 in Type 2 diabetes (search). This work strengthens our existing research, which has already demonstrated improved glucose homeostasis by reprogramming alpha cells (search) in Type 1 diabetes (search), 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."
The results build on earlier work in Type 1 diabetes (search) models, where GPX-002 converted alpha cells (search) into beta-like cells capable of producing insulin. The company believes the new data expand the therapy's potential to address the much larger Type 2 diabetes (search) population.
Next Steps in Development
Researchers are continuing non-human primate studies in both Type 1 and Type 2 diabetes (search) and are preparing for formal toxicology studies ahead of a future Investigational New Drug submission. Current studies in non-human primates are evaluating efficacy after six months of immunosuppression. The company notes that results from preclinical testing of GPX-002 may not predict the results that will be obtained in later phase clinical trials.