Genprex, Inc. (NASDAQ: GNPX) has secured an exclusive patent license agreement with UTHealth Houston for its lead drug candidate, Reqorsa® Gene Therapy (quaratusugene ozeplasmid), expanding its potential applications to include glioblastoma treatment. The agreement, announced on May 6, 2025, provides Genprex with exclusive commercial rights to a patent co-owned by both organizations.
"With this license agreement, Genprex has obtained exclusive commercial rights to REQORSA in glioblastoma while also adding to our patent estate," said Thomas Gallagher, Senior Vice President of Intellectual Property and Licensing at Genprex. "The role of TUSC2 in lung cancer has been well established, and this latest license enables Genprex to expand the use of REQORSA into a new indication, in which the cancer can be difficult to treat and there are unmet medical needs."
Promising Preclinical Results in Glioblastoma
Genprex previously reported positive preclinical data on REQORSA's efficacy in glioblastoma at the 2024 EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics in October 2024. Research collaborators from UTHealth Houston had identified TUSC2 as a novel tumor suppressor for glioblastoma, which is the most common and deadliest primary brain tumor in adults with notably poor prognosis.
In their latest study, UTHealth Houston researchers utilized patient-derived glioblastoma (GBM) cell lines and patient-derived glioma stem cell (PD-GSC) lines. REQORSA was employed to restore TUSC2 expression in these cells. The results were significant:
- REQORSA substantially reduced GBM cell viability
- Migration assays demonstrated that REQORSA suppressed GBM cell migration independent of its ability to suppress cell viability
These promising in vitro findings support further evaluation of REQORSA's anti-tumor efficacy in malignant gliomas using mouse models, potentially opening a new treatment avenue for this aggressive cancer.
The Science Behind REQORSA Gene Therapy
REQORSA consists of a plasmid containing the TUSC2 gene encapsulated in non-viral lipid-based nanoparticles in a lipoplex form, delivered through Genprex's proprietary Oncoprex® Delivery System. The therapy carries a positive charge, allowing it to be administered intravenously and specifically target cancer cells.
The technology is designed to deliver functioning TUSC2 genes to negatively charged cancer cells while minimizing uptake by normal tissue. Laboratory studies conducted at The University of Texas MD Anderson Cancer Center have demonstrated that the uptake of TUSC2 in tumor cells in vitro after REQORSA treatment was 10 to 33 times greater than in normal cells, highlighting its selective targeting capability.
Addressing a Critical Unmet Need
Glioblastoma represents a significant unmet medical need in oncology. With standard treatment, median survival is approximately 15 months, and the five-year survival rate is less than 7%. Current treatment options typically include surgical resection followed by radiation and chemotherapy with temozolomide, but recurrence is nearly universal.
The expansion of REQORSA into glioblastoma treatment could potentially provide a novel therapeutic approach for patients with limited options. The gene therapy's mechanism of action—restoring tumor suppressor function—offers a different approach from conventional treatments.
Genprex's Expanding Therapeutic Portfolio
This license agreement strengthens Genprex's position in the oncology gene therapy space. The company's lead product candidate, REQORSA, is currently being evaluated in two clinical trials for non-small cell lung cancer (NSCLC) and small cell lung cancer (SCLC). Both lung cancer programs have received Fast Track Designation from the FDA, and the SCLC program has also received an FDA Orphan Drug Designation.
Beyond oncology, Genprex is also developing gene therapies for diabetes. Their diabetes gene therapy approach uses an AAV vector to deliver Pdx1 and MafA genes directly to the pancreas, with promising results in preclinical models of both Type 1 and Type 2 diabetes.
Next Steps in Development
While the preclinical data for REQORSA in glioblastoma is encouraging, the path to clinical trials will require additional studies to establish safety and efficacy in animal models before advancing to human trials. The company has not yet announced a timeline for potential clinical trials in glioblastoma patients.
If successful in clinical development, REQORSA could potentially join the limited arsenal of treatments for glioblastoma, offering a novel mechanism of action through gene therapy that could complement existing treatment modalities.
"These results support further evaluation of its in vivo anti-tumor efficacy in malignant gliomas using mouse models," noted the company in their announcement, indicating the next logical step in the development process.
