Announcement • May 26
Genprex, Inc. Announces Positive Clinical Data on Biomarkers in Patients Receiving Reqorsa® Gene Therapy Published at the 2026 ASCO Annual Meeting Genprex, Inc. announced that its research collaborators' abstract was published at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting. The abstract details positive clinical data from studies of predictive biomarkers in patients receiving its lead drug candidate, Reqorsa® Gene Therapy (quaratusugene ozeplasmid), for the treatment of lung cancer. Quaratusugene ozeplasmid is a gene therapy that delivers a plasmid coding for the TUSC2 tumor suppressor gene to lung cancer cells, as >80% of lung cancers have been shown to have decreased or absent TUSC2 protein. TUSC2 protein levels have not correlated with PFS, presumably because of the complexities of TUSC2 protein regulation. Preclinical studies have identified higher levels of Trop-2 protein in organoids and lower levels of PTEN protein in lung cancer cell lines as correlating with response (AACR 2026). Tumor tissue from patients in clinical trials with quaratusugene ozeplasmid were evaluated for Trop-2 and PTEN protein expression. Monoclonal antibodies against Trop-2 (BSB148 from BioSB) and PTEN (138G6 from Cell Signaling Technology) were used for immunohistochemistry in paraffin sections from archival tumor samples in patients enrolled in three clinical trials with quaratusugene ozeplasmid and results expressed as H-scores. H-scores were calculated by evaluating diaminobenzidine staining intensity using the formula [1 × (% cells 1+) + 2 × (% cells 2+) + 3 × (% cells 3+)]. Data on Trop-2 and PTEN protein expression and data on PFS were available from 18 patients enrolled in clinical trials with quaratusugene ozeplasmid. Six patients with NSCLC were enrolled in the Acclaim-1 trial in combination with osimertinib. One patient was enrolled in the Acclaim-2 trial in combination with pembrolizumab. Eleven patients with small cell lung cancer (SCLC) were enrolled in the Acclaim-3 trial in combination with atezolizumab. In patients with NSCLC, Trop-2 H-scores above 100 correlated with prolonged PFS (p=0.05), and PTEN H-scores below 100 correlated with prolonged PFS (p=0.03). In patients with SCLC, Trop-2 H-scores were universally low, and thus non-evaluable. PTEN H-scores in patients with SCLC did not correlate with prolonged PFS (p=0.53). Following up on preclinical cell line and organoid models indicating that Trop-2 and PTEN protein expression correlated with response, levels of Trop-2 and PTEN protein were evaluated in patients treated with quaratusugene ozeplasmid. In conclusion, both Trop-2 H-scores above 100 and PTEN H-scores below 100 correlated with longer PFS in patients with NSCLC, but not in patients with SCLC. Following the clinical studies outlined above, Genprex completed additional analysis to evaluate the relationship between NSCLCs with high intensity staining (3+) and PFS. NSCLCs with 3+ Trop-2 staining had a strong relationship with PFS that was just outside the bounds for significance (p=0.053) and those with 3+ PTEN staining exhibited a trend for a negative relationship with PFS that was not statistically significant (p=0.309). These results are consistent with the H-score analysis regarding a strong positive relationship between Trop-2 expression and PFS. High (3+) Trop-2 expression will be investigated further as a potential biomarker for REQORSA. Announcement • May 15
Genprex Inc. Collaborators Present Positive Preclinical Data on Diabetes Gene Therapy for Type 2 Diabetes Genprex, Inc. announced that its research collaborators presented positive preclinical data on the Company's diabetes gene therapy drug candidate at the 2026 American Society of Gene and Cell Therapy Annual Meeting. The collaborators presented preclinical data demonstrating that the diabetes gene therapy (Pdx1/MafA gene therapy, PM or GPX-002) can reverse hyperglycemia in Type 2 diabetic (T2D) mouse models. The featured Genprex-supported abstract and poster at the 2026 ASGCT Annual Meeting: Title: "Pancreatic Delivery of AAV-Pdx1/MafA Reverses Hyperglycemia in a Preclinical Model of Type 2 Diabetes" Abstract ID: 2419 Topic: Gene-Based Therapies in Pre-Clinical Models of Genetic Disease Poster Presentation Date: Wednesday, May 13, 2026 Poster Presentation Time: 5-6:30 p.m. ET. In this study, eight-week-old male C57BL/6 mice were maintained on a regular diet (RD) or high fat diet (HFD) for 24 weeks. HFD mice then either remained unoperated or underwent retrograde infusion into the pancreatic duct of adeno-associated virus (AAV-8) encoding Pdx1 and MafA (PM) cassettes under the CMV promoter (global–islet cell targeting) or the rat insulin promoter (RIP) (ß-cell–specific targeting) or received a control virus. The diet remained unchanged after surgery. At two and/or four weeks after surgery, researchers performed intraperitoneal glucose tolerance testing (IPGTT), insulin tolerance testing (ITT), glucose-stimulated insulin secretion (GSIS), calculated HOMA-IR and assessed glucagon secretion. Mice were then euthanized for pancreatic histology, quantification of ß- and a-cell mass, electron microscopy (EM), and islets were isolated for ex-vivo glucose-stimulated insulin secretion (GSIS) and single-cell RNA sequencing. The results at four weeks showed major improvements in the control of diabetes. At four weeks after surgery, ex-vivo GSIS showed that islets isolated from HFD+CMV-PM-GFP treated mice had insulin secretion similar to islets from RD mice, and both groups had increased insulin secretion compared to islets from the control HFD groups, indicating improved ß-cell function with PM treatment. Similarly, and importantly, treatment of HFD mice with RIP-PM-GFP, which selectively targets ß-cells, reversed hyperglycemia and improved ex-vivo GSIS. In addition, EM imaging showed that PM treatment in HFD mice increased the number of total and mature insulin granules and decreased the number of immature insulin granules compared with HFD controls. Furthermore, transcriptomic pseudotime analysis demonstrated a shift in ß-cells from an immature state toward a more mature state after PM treatment. PM gene therapy reverses hyperglycemia, likely in large part by specifically enhancing ß-cell function and maturation. This approach is technically translatable to humans using endoscopic retrograde cholangiopancreatography to deliver PM gene therapy to the pancreas. Announcement • May 02
Genprex Inc Receives Patent Grant from the Israel Patent Office for Combination of Reqorsa Gene Therapy and PD-1 Antibodies to Treat Cancer Genprex, Inc. announced that The Israel Patent Office (ILPO) has granted Genprex a patent covering the use of Reqorsa Gene Therapy (quaratusugene ozeplasmid) in combination with PD-1 antibodies for the treatment of cancer. This patent will expand on the previously granted patents for REQORSA in combination with PD-1 antibodies, which have been granted in the U.S., Japan, Mexico, Russia, Australia, Chile, China, Singapore and Europe. REQORSA is initially being developed in combination with prominent, approved cancer drugs to treat lung cancer. In preclinical studies, REQORSA has been shown to be complementary with targeted drugs and immunotherapies. The Company believes REQORSA's unique attributes position it to provide potential treatments that improve on these current therapies for patients with lung cancer and possibly other cancers. According to the Israeli Ministry of Health, in 2020, the number of cases of lung cancer in Israel increased by 33% (not per capita) within a decade, with 200 new cases diagnosed every month. In 2015, the lung cancer mortality rate reached 26% of the mortality rate from all cancers. According to the Israel Cancer Association, in 2021 lung cancer was the number one cancer death cause in Israeli population, responsible for 21.1% of all cancer deaths among Israeli men and 11.8% cancer deaths among Israeli women. Israel is ranked 42 in terms of lung cancer incidence (number of new cases diagnosed) and 66 in lung cancer mortality worldwide. 
