Latest Research Demonstrates Promising Improved Glucose Homeostasis by Reprogramming Alpha Cells AUSTIN, Texas, June 24, 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 its research collaborators presented positive preclinical research from studies of GPX-002, the Company's diabetes gene therapy drug candidate, at the 2025 American Di ...

Novel Gene Therapy is Addressing Both Type 1 and Type 2 Diabetes AUSTIN, Texas, May 28, 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, announced that its research collaborators presented positive preclinical data and research from studies of GPX-002, the Company's diabetes gene therapy drug candidate, in an oral presentation at the American Society of Ge ...

Clinical Trials - The company is currently enrolling patients in the Phase 2a expansion portion of the Acclaim-1 clinical trial, which combines REQORSA and AstraZeneca's Tagrisso for late-stage NSCLC patients [120]. - The recommended Phase 2 dose (RP2D) of REQORSA in the Acclaim-1 trial is determined to be 0.12 mg/kg, which is twice the highest dose level delivered in previous trials [120]. - The Phase 2a expansion portion of the Acclaim-1 trial is expected to enroll approximately 33 patients who have progressed on Tagrisso or Tagrisso-containing regimens [120]. - The FDA has granted Fast Track Designation for the Acclaim-1 treatment combination of REQORSA and Tagrisso in NSCLC patients [121]. - The Acclaim-3 clinical trial is currently enrolling patients using a combination of REQORSA and Genentech's Tecentriq for extensive stage small cell lung cancer (ES-SCLC) [124]. - The RP2D of REQORSA in the Acclaim-3 trial is also set at 0.12 mg/kg, based on safety data showing no dose limiting toxicities [124]. - The Acclaim-3 trial aims to enroll approximately 50 patients across 10 to 15 U.S. sites, with a primary endpoint of determining the 18-week progression-free survival rate [124]. - The Acclaim-3 clinical trial has received FDA Fast Track Designation and Orphan Drug Designation for the patient population [125]. - The company expects interim enrollment of the Phase 2a expansion portion of the Acclaim-1 trial to be completed by the end of 2025 [153]. Financial Performance - Research and development (R&D) expense for Q1 2025 was $2,539,993, a decrease of 22% from $3,274,380 in Q1 2024, primarily due to the closure of the Acclaim-2 clinical trial and a reduction in R&D staff from 15 to 9 employees [143]. - General and administrative (G&A) expense for Q1 2025 was $1,429,299, down 47% from $2,719,667 in Q1 2024, attributed to expense reduction strategies and a decrease in G&A staff from 11 to 6 employees [144]. - Net loss for Q1 2025 was $3,964,601, a reduction of 34% compared to $5,968,869 in Q1 2024, mainly due to decreased clinical and manufacturing expenses and overall staff reduction from 26 to 15 employees [147]. - As of March 31, 2025, the company had an accumulated deficit of $158,764,044, having never generated revenue from product sales since inception [148]. - In 2024, the company sold 5,366,503 shares of common stock for net proceeds of $6.3 million and completed a registered direct offering for approximately $5.8 million [149]. - During the three months ended March 31, 2025, the company sold 13,278,666 shares of common stock for gross proceeds of approximately $6.2 million, resulting in net proceeds of $6,028,103 after offering expenses [150]. - Net cash used in operating activities decreased by $1,136,466, or 21%, from $5,297,891 in Q1 2024 to $4,161,425 in Q1 2025, primarily due to the closure of the Acclaim-2 clinical trial and expense reduction strategies [156]. - Net cash provided by financing activities was $6,028,103 for the three months ended March 31, 2025, a decrease of $765,096 compared to $6,793,199 in Q1 2024 [158]. - The company had no net cash provided by or used in investing activities for the three months ended March 31, 2025, compared to a net cash outflow of $15,822 in Q1 2024 [157]. Strategic Focus - The company has entered into a sponsored research agreement with MD Anderson to support further preclinical studies of TUSC2 and other tumor suppressor genes [119]. - The company is focusing on optimizing and re-focusing its diabetes and oncology clinical development programs to enhance the likelihood of success [116]. - The company is currently developing GPX-002 for both Type 1 and Type 2 diabetes, with preclinical studies ongoing at the University of Pittsburgh [126]. - The company plans to seek further regulatory guidance from the FDA on IND-enabling studies in the second half of 2025 [126]. - A new sponsored research agreement with the University of Pittsburgh was established in May 2025 to study Type 1 and Type 2 diabetes in animal models [126]. - The company is exploring a non-viral lipid nanoparticle delivery system for multiple treatments [126]. - The company has implemented strategies to optimize clinical and research programs and is considering various strategic alternatives to enhance stockholder value [138]. - The company anticipates needing to raise additional capital to fund future operations, including ongoing clinical trials, as it does not expect to generate revenue from product sales in the near term [153]. - The company estimates it can fund its current operations and planned clinical trial activities through the end of the second quarter of 2025 [154]. - The company may seek additional capital through strategic collaborations or transactions, but there is uncertainty regarding its ability to maintain liquidity over the next 12 months [153]. - The company has utilized all available capacity under the current prospectus supplement for the 2023 ATM Facility as of the filing date of the Quarterly Report [153].

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, wh ...

Both REQORSA Alone and in Combination with LUMAKRAS® Demonstrated Robust Anti-Tumor Effects in Ras Inhibitor Resistant Non-Small Cell Lung CancerAUSTIN, Texas, April 30, 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 its research collaborators presented positive preclinical data for Reqorsa® Gene Therapy (quaratusugene ozeplasmid), f ...

Novel Gene Therapy Program Offers a Promising Opportunity for Curative Therapy in DiabetesAUSTIN, Texas, April 29, 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 its research collaborators have been selected to present at the upcoming American Society of Gene and Cell Therapy's (ASGCT) 28th Annual Meeting being held May 13-17, 2025 i ...

Additional Gene Therapy Technology Expands Genprex's Portfolio of Oncology LicensesAUSTIN, Texas, April 28, 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 it has signed an exclusive patent license agreement with NYU Langone Health related to the drug candidate, Reqorsa® Gene Therapy (quaratusugene ozeplasmid). The therapy is under investi ...

Research Journal Features Genprex Collaborators' Abstract Detailing Positive Preclinical Data on the Use ofReqorsa® Gene Therapy for the Treatment of Lung CancerAUSTIN, Texas, April 22, 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 its research collaborators' abstract was published in the online Proceedings supplement of Cancer Rese ...

Clinical Trials and Research - The company is developing REQORSA® gene therapy for Non-Small Cell Lung Cancer (NSCLC) and Small Cell Lung Cancer (SCLC), with a recommended Phase 2 dose of 0.12 mg/kg established after the Phase 1 trial [486]. - In the Phase 1 portion of the Acclaim-1 trial, one patient achieved a partial remission after 47 courses of treatment, demonstrating prolonged progression-free survival (PFS) of approximately 35 months [486]. - The Acclaim-1 trial is expected to enroll approximately 33 patients who have progressed on Tagrisso or Tagrisso-containing regimens, with an interim analysis planned after treating 19 patients [486]. - The FDA has granted Fast Track Designation for the combination of REQORSA and Tagrisso in NSCLC patients who have progressed on Tagrisso treatment [487]. - The Acclaim-3 trial is currently enrolling patients, with an expected enrollment of approximately 50 patients, focusing on the 18-week progression-free survival rate as a primary endpoint [491]. - The Acclaim-2 trial has been closed due to slow enrollment, with no patients currently receiving study treatment [490]. - The company is collaborating with MD Anderson to identify biomarkers that may enhance patient screening and enrollment for clinical trials [485]. - The company plans to complete interim enrollment of the Phase 2a expansion portion of the Acclaim-1 trial by the end of 2025 [523]. - The company may face delays in clinical trials due to various factors, including competition for patients and transitions to new third-party CDMOs [524]. Financial Performance - Research and development (R&D) expenses decreased by $7,081,159, or 40%, to $10,535,446 for the year ended December 31, 2024, compared to $17,616,605 in 2023 [509]. - General and administrative (G&A) expenses decreased by $2,793,257, or 21%, to $10,632,028 for the year ended December 31, 2024, compared to $13,425,285 in 2023 [510]. - Net loss for the fiscal year ended December 31, 2024, was $21,111,163, a decrease of $9,749,298, or 32%, from a net loss of $30,860,461 in 2023 [513]. - As of December 31, 2024, the accumulated deficit was $154,799,443, with no revenue generated from product sales since inception [515]. - Cash balance as of December 31, 2024, was $1,601,660, with subsequent net proceeds of approximately $6.0 million received through the ATM Agreement [522]. - Net cash used in operating activities decreased by $7,589,515, or 31%, from $24,738,603 in 2023 to $17,149,088 in 2024 due to expense reduction strategies [529]. - Net cash provided by investing activities changed from a net cash used of $71,214 in 2023 to a net cash provided of $1,166 in 2024, resulting in a net change of $72,380 [530]. - Net cash provided by financing activities increased by $1,418,576, or 13%, from $10,593,377 in 2023 to $12,011,953 in 2024, attributed to differences in capital raising activities [531]. - The net decrease in cash for the year ended December 31, 2024, was $5,135,969, compared to a decrease of $14,216,440 in 2023 [529]. Future Outlook and Funding - The company expects to fund operations through equity offerings, ATM drawdowns, and potential debt financing, anticipating the need for additional capital to support ongoing clinical trials [523]. - R&D expenses are expected to increase in the future as the company advances product candidates through clinical trials and expands research programs [504]. - The company anticipates needing additional fundraising activities and cash on hand by the second quarter of 2025 to meet fixed cash obligations related to clinical studies [526]. - The company expects insufficient cash to cover ongoing clinical trials and research and development programs through fiscal year 2025 without raising additional working capital [526]. - Future capital requirements will depend on various factors, including development costs, manufacturing expenses, and costs associated with being a public company [528]. - The company may need to use liquidity for acquisitions or additional capital to fund newly acquired operations, which could lead to dilution for existing security holders [527]. - The company has ongoing obligations related to the Acclaim clinical trials, which may require adjustments to its operating plan if cash is insufficient [526]. - The company reduced headcount in R&D from 16 employees at December 31, 2023, to 10 employees at December 31, 2024, as part of expense reduction strategies [509]. - The company has reduced its workforce from 28 employees at December 31, 2023, to 16 employees at December 31, 2024, as part of its expense reduction strategies [529].

Core Viewpoint - Genprex, Inc. announced positive preclinical data for its lead drug candidate, Reqorsa® Gene Therapy, targeting KRASG12C mutant non-small cell lung cancer (NSCLC), which may provide a new treatment option for patients resistant to Ras inhibitors [1][2]. Group 1: Research and Development - The upcoming presentation at the 2025 American Association for Cancer Research (AACR) Annual Meeting will showcase preclinical data on Reqorsa® Gene Therapy [1]. - The study focuses on overcoming acquired resistance to sotorasib (Lumakras®), the first FDA-approved KRAS inhibitor, in KRASG12C mutant NSCLC [3]. - TUSC2, the active component in Reqorsa, has shown multifunctional activity, including inhibiting cancer cell growth and activating immune responses [3][4]. Group 2: Clinical Efficacy - TUSC2 transfection significantly reduced colony formation and increased apoptosis in acquired resistance (AR) cell lines [4]. - Reqorsa demonstrated a strong antitumor effect in mouse xenografts and patient-derived xenograft (PDX) models, outperforming sotorasib alone [4][5]. - A synergistic effect was observed when combining Reqorsa with sotorasib in treating TC314AR PDX tumors [4]. Group 3: Product Information - Reqorsa (quaratusugene ozeplasmid) is a gene therapy that delivers the TUSC2 gene using a non-viral lipid-based nanoparticle system, specifically targeting cancer cells [6][8]. - Laboratory studies indicate that TUSC2 uptake in tumor cells is significantly higher than in normal cells, ranging from 10 to 33 times [7]. Group 4: Company Overview - Genprex, Inc. is a clinical-stage gene therapy company focused on developing therapies for cancer and diabetes, utilizing its Oncoprex® Delivery System [8]. - The company is advancing its pipeline of gene therapies and has received Fast Track Designation from the FDA for its lung cancer programs [8].