REQORSA® Immunogene Therapy
The First Systemically Delivered Gene Therapy Used for Cancer in Humans
The company's lead product candidate, REQORSA®, immunogene therapy (quaratusugene ozeplasmid) for non-small cell lung cancer (NSCLC), uses the company’s unique, proprietary ONCOPREX® Nanoparticle Delivery System, which they believe is the first systemic gene therapy delivery platform used for cancer in humans.
In 2020, the FDA granted Fast Track Designation for REQORSA in combination with AstraZeneca’s Tagrisso® (osimertinib) in late-stage NSCLC patients with EFGR mutations whose tumors progressed after treatment with Tagrisso.
In 2021, the FDA granted Fast Track Designation for REQORSA in combination with Merck & Co’s Keytruda® (pembrolizumab) in late-stage NSCLC patients whose disease progressed after treatment with Keytruda.
The company believes that REQORSA, unlike other gene therapies, which either need to be delivered directly into tumors or require cells to be removed from the body, re-engineered and then reinserted into the body, is the first systemic gene therapy used for cancer in humans.
Diabetes Gene Therapy
GPX-002, a gene therapy for diabetes, is the most recent addition of the company's licensed technologies. GPX-002 was developed by researchers at the University of Pittsburgh. Diabetic mice studies have shown that GPX-002 restored normal blood glucose levels for an extended period of time, which could translate to decades in humans.
This gene therapy could not only become a new treatment option for millions of diabetes patients who need insulin replacement therapy, but it holds the potential to provide long-term effectiveness, or may even be a cure, for diabetic patients.
The diabetes gene therapy, GPX-002, is comprised of a novel infusion process that uses an endoscope and an adeno-associated virus (AAV) vector to deliver Pdx1 and MafA genes to the pancreas. The genes express proteins that transform alpha cells in the pancreas into functional beta-like cells, which can produce insulin but are distinct enough from beta cells to evade the body’s immune system.
Diabetic mice studies show that the gene therapy restored normal blood glucose levels for an extended period of time, typically around four months. The duration of restored blood glucose levels in mice could translate to decades in humans.
The diabetes gene therapy was developed by Dr. George Gittes, a researcher at the Rangos Research Center at UPMC Children’s Hospital of Pittsburgh, where preclinical research is ongoing. GPX-002 has been tested in vivo in mice and nonhuman primates. Once sufficient preclinical data has been generated, they expect to begin a Phase I clinical trial in diabetic patients, which could be the first-ever gene therapy tested in humans for diabetes.
ONCOPREX® Nanoparticle Delivery System
The oncology drug development program utilizes our unique, proprietary non-viral ONCOPREX Nanoparticle Delivery System, which we believe is the first systemic gene therapy delivery platform used for cancer in humans. This platform, originally developed through collaborative research between the University of Texas MD Anderson Cancer Center and the National Institutes of Health, has been optimized to work with our initial product candidate, REQORSA® immunogene therapy.
Designed to Deliver Cancer-Fighting Genes Systemically
The ONCOPREX platform has been designed and optimized to deliver cancer-fighting genes into the patient’s body systemically. Using this system, the company encapsulates plasmids that express tumor suppressor genes within lipid nanoparticles and intravenously administer the encapsulated plasmids which are taken up by the tumor cells, after which the tumor suppressor genes express proteins that are missing or found in low quantities in the tumor cells. Their nanoparticles are non-immunogenic, allowing repetitive therapeutic dosing and have been clinically shown to deliver molecular kinase inhibitors effectively.
Company Website. Company Presentation.
And right now, based on multiple potential catalysts, past champ GNPX needs to be on your radar. Check them out:
No. 1 GNPX Potential Catalyst - Dawson James Analyst Provides $3.00 Target (Major Upside Potential From Current Trading Levels)
On January 30th, Dawson James Securities' Senior Analyst, Jason Kolbert, provided some key insight into GNPX along with tagging this profile with a $3.00 target.
From it's 12:00PM EST valuation Wednesday, that target provides GNPX with over 80% potential upside.
Check out some highlights from the report:
Lead Program in Oncology: REQORSA Immunogene Therapy (GPX-001). REQORSA is a plasmid that expresses a tumor suppressor gene TUSC2, that is deleted early during lung cancer development. REQORSA appears to have a multimodal mechanism of action whereby it interrupts cell signaling pathways that cause replication and proliferation of cancer cells, reestablishes pathways for apoptosis, or programmed cell death, in cancer cells, and modulates the immune response against cancer cells. REQORSA has been shown to be complementary with targeted drugs and immunotherapies.
Clinical Status: REQORSA is currently enrolling clinical patients in two trials with a third coming. The trials are initially exploring its utility in NSCLC with a range of agents – standard of care. The Acclaim-1 clinical trial is evaluating a combination of REQORSA with Tagrisso in patients with late-stage NSCLC with activating epidermal growth factor receptor mutations, whose disease progressed after treatment. The first patient was dosed in Acclaim-1 in February 2022. A second trial, Acclaim-2 (first patient doses April 2022), is using a combination of REQORSA with Merck & Co.'s Keytruda in patients with late-stage NSCLC whose disease progressed after treatment with Keytruda. The FDA has granted Fast Track Designation for both trials. Accaim-3 is exploring REQORSA with Tecentriq in ES-SCLC patients.
Diabetes Program: The company has developed a gene therapy that is designed to transform alpha cells in the pancreas into functional beta-like cells, which can produce insulin but are distinct enough from beta cells to evade the body's immune system. The therapy utilizes a procedure in which an adeno-associated virus vector is endoscopically delivered to the pancreas to insert Pdx1 and MafA genes. GPX-002 for T1D uses a glucagon promoter in alpha cells. GPX003 for T2D uses an insulin promoter in beta cells. The planned phase 1 trial represents the first-ever gene therapy tested in humans for diabetes. In vivo, preclinical studies (mice), have shown that GPX-002 restored normal blood glucose levels for an extended period. Research collaborators at the University of Pittsburgh plan to present data in non-human primates highlighting the therapeutic potential of Genprex's gene therapy for Type 1 diabetes at the 16th International Conference on Advanced Technologies & Treatments for Diabetes (ATTD 2023) being held February 22-25 in Berlin, Germany.
Valuation: We project our model out to 2033. We apply a 30% risk cut to our projected revenues in our product model in addition to our 30% risk rate applied in our Free Cash Flow to the Firm (FCFF), discounted EPS (dEPS), and Sum-of-the-Parts (SOP) models. We use a fully diluted out year share count assuming multiple raises. The result is equal-weighted and averaged and rounded to the nearest whole number to derive our 12-month projected price target of $3.00. We note that as the company established proof of concept as a result of clinical trial data, the risk rate (r) is reduced, and valuation rises.
No. 2 GNPX Potential Catalyst - This Signed Exclusive License Could Be Game-Changing In The Fight Against Diabetes
Genprex Signs Exclusive License to Additional Diabetes Technology with the University of Pittsburgh
Technologies Licensed from University of Pittsburgh May Have the Potential to Provide Long-Term Efficacy and to Change the Course of this Disease for the Millions of Patients Around the World with Type 1 or Type 2 Diabetes
AUSTIN, Texas, Jan. 5, 2023 /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 entered into an exclusive license agreement (the Agreement) with the University of Pittsburgh (Pitt), granting Genprex a worldwide, exclusive license to a patent application and related technology and a worldwide, non-exclusive license to use certain related know-how, all related to gene therapy for Type 2 diabetes using the genes of the Pdx1 and MafA transcription factors. The preclinical technology, GPX-003, is believed to work by rejuvenating diminished beta cells to increase insulin expression by introducing transcription factors controlled by an insulin promoter.
"This marks the Company's third technology license this year supporting our diabetes program, demonstrating the continued strengthening of our intellectual property portfolio, our diabetes program, and our position as a lead innovator in emerging diabetes gene therapies," said Rodney Varner, President and Chief Executive Officer of Genprex. "We are thrilled to have secured multiple cutting edge technologies from Pitt that expand our diabetes gene therapy program and specifically target Type 1 and Type 2 diabetes."
Read the full article here.
No. 3 GNPX Potential Catalyst - 6-Month Chart Check: Trades Above 3 Key Lines Of Potential Support
Check out GNPX's 6-month chart: