Researching Novel Diabetes Gene Therapy Using
Potentially Re-Dosable Non-Viral Delivery System
Strategic Collaboration with CDMO Partner
Positions Genprex as a Thought-Leader in Diabetes Market
AUSTIN,
Texas, Feb. 19, 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 the
advancement of its diabetes program through a strategic
collaboration with a contract development and manufacturing
organization (CDMO) to research an alternative second generation
approach using a non-viral lipid nanoparticle delivery of the
Company's diabetes gene therapy drug candidate.
"This collaboration with our CDMO partner is separate from our
existing preclinical research programs and seeks to evaluate
potential next generation construct optimization," said
Ryan Confer, President and Chief
Executive Officer at Genprex. "This exploratory research represents
our forward-thinking approach within our diabetes program to
determine if our novel diabetes gene therapy could be delivered
using a non-viral delivery system, similar to Genprex's oncology
drug candidate. While AAV delivery is a well understood delivery
mechanism, there are many benefits to a non-viral delivery system,
including the potential for re-dosing patients to optimize
treatment. This collaboration positions Genprex as a thought-leader
in gene therapy, specifically within the diabetes market where
there are only symptom managing therapies but no disease modifying
technologies."
Separate from the ongoing preclinical studies, Genprex and its
CDMO partner are researching and exploring the potential delivery
of these genes using a non-viral lipid nanoparticle. Many
gene therapies rely on viral based delivery systems. The
benefit of the viral system is that viruses are skilled at
penetrating cells. However, viruses cannot be readministered, due
to the development of an immune response against the virus
capsid.
"Prior studies have shown that Non-Obese Diabetic (NOD) mice
became hyperglycemic again a median of 17 days after treatment with
syngeneic islet cell transplants," said Mark Berger, Chief Medical Officer at Genprex.
"Our recent preclinical studies of Type 1 NOD mouse models treated
with GPX-002 have shown durability of glucose control to
approximately 120 days, or 4 months. This suggests that that the
new insulin producing beta-like cells that our therapy produced are
poorly recognized by the autoimmune process in NOD mice. Our team
continues to explore ways to enhance durability, and expanding our
research to include a non-viral delivery system could allow for
multiple treatments to be administered."
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, which
can produce insulin but may be distinct enough from beta cells to
evade the body's immune system. In vivo, preclinical studies show
that GPX-002 restored normal blood glucose levels for an extended
period of time in T1D mouse models. In T2D, where autoimmunity is
not at play, GPX-002 is believed to rejuvenate and replenish
exhausted beta cells.
About Diabetes
According to the U.S. Center for
Disease Control as of 2024, 38.4 million Americans, or
approximately 11.6% of the U.S. population, have diabetes.
Approximately 10% of this total has T1D and approximately 90-95% of
this total has T2D. It is also believed that more than 97 million
Americans aged 18 years or older have prediabetes. In 2021,
approximately 537 million adults (20-79 years) worldwide were
living with diabetes, and the total number of people living with
diabetes is projected to rise to 643 million by 2030 and 783
million in 2045. Approximately 7.7 million adults over the age of
20 live with T1D worldwide, and approximately 1.6 million children
and adolescents under the age of 20 live with T1D worldwide. Also
in 2021, diabetes caused more than 6.7 million deaths globally and
diabetes resulted in approximately $966
billion dollars in health expenditures, a 316% increase over
the preceding fifteen years.
About Genprex, Inc.
Genprex, Inc. is a clinical-stage
gene therapy company focused on developing life-changing therapies
for patients with cancer and diabetes. Genprex's technologies are
designed to administer disease-fighting genes to provide new
therapies for large patient populations with cancer and diabetes
who currently have limited treatment options. Genprex works with
world-class institutions and collaborators to develop drug
candidates to further its pipeline of gene therapies in order to
provide novel treatment approaches. Genprex's oncology program
utilizes its systemic, non-viral Oncoprex® Delivery System which
encapsulates the gene-expressing plasmids using lipid-based
nanoparticles in a lipoplex form. The resultant product is
administered intravenously, where it is taken up by tumor cells
that then express tumor suppressor proteins that were deficient in
the tumor. The Company's lead product candidate, Reqorsa® Gene
Therapy (quaratusugene ozeplasmid), is being evaluated in two
clinical trials as a treatment for NSCLC and SCLC. Each of
Genprex's lung cancer clinical programs has received a Fast Track
Designation from the FDA for the treatment of that patient
population, and Genprex's SCLC program has received an FDA Orphan
Drug Designation. Genprex's diabetes gene therapy approach is
comprised of a novel infusion process that uses an AAV vector to
deliver Pdx1 and MafA genes directly to the pancreas. In models of
Type 1 diabetes, GPX-002 transforms alpha cells in the pancreas
into functional beta-like cells, which can produce insulin but may
be distinct enough from beta cells to evade the body's immune
system. In a similar approach for Type 2 diabetes, where
autoimmunity is not at play, GPX-002 is believed to rejuvenate and
replenish exhausted beta cells.
Interested investors and shareholders are encouraged to sign up
for press releases and industry updates by visiting
the Company Website, registering for Email
Alerts and by following Genprex on Twitter, Facebook and
LinkedIn.
Cautionary Language Concerning Forward-Looking
Statements
Statements contained in this press release
regarding matters that are not historical facts are
"forward-looking statements" within the meaning of the Private
Securities Litigation Reform Act of 1995. These forward-looking
statements are made on the basis of the current beliefs,
expectations and assumptions of management, are not guarantees of
performance and are subject to significant risks and uncertainty.
These forward-looking statements should, therefore, be considered
in light of various important factors, including those set forth in
Genprex's reports that it files from time to time with the
Securities and Exchange Commission and which you should review,
including those statements under "Item 1A – Risk Factors" in
Genprex's Annual Report on Form 10-K for the year ended
December 31, 2023.
Because forward-looking statements are subject to risks and
uncertainties, actual results may differ materially from those
expressed or implied by such forward-looking statements. Such
statements include, but are not limited to, statements regarding:
Genprex's ability to advance the clinical development,
manufacturing and commercialization of its product candidates in
accordance with projected timelines and specifications; the timing
and success of Genprex's clinical trials and regulatory approvals,
including, but not limited to, the Company's diabetes clinical
development program; the effect of Genprex's product candidates,
alone and in combination with other therapies, on cancer and
diabetes; the effects of any strategic research and development
prioritization initiatives, and any other strategic alternatives or
other efforts that Genprex takes or may take in the future that are
aimed at optimizing and re-focusing Genprex's diabetes, oncology
and/or other clinical development programs including prioritization
of resources, and the extent to which Genprex is able to implement
such efforts and initiatives successfully to achieve the desired
and intended results thereof; Genprex's future growth and financial
status, including Genprex's ability to maintain compliance with the
continued listing requirements of The Nasdaq Capital Market and to
continue as a going concern and to obtain capital to meet its
long-term liquidity needs on acceptable terms, or at all; Genprex's
commercial and strategic partnerships, including those with its
third party vendors, suppliers and manufacturers and their ability
to successfully perform and scale up the manufacture of its product
candidates, including the results of our early research to extend
durability of glucose control and the potential results of further
research with a CDMO to utilize a non-viral delivery system that
would allow multiple treatments to be administered; and Genprex's
intellectual property and licenses.
These forward-looking statements should not be relied upon as
predictions of future events and Genprex cannot assure you that the
events or circumstances discussed or reflected in these statements
will be achieved or will occur. If such forward-looking statements
prove to be inaccurate, the inaccuracy may be material. You should
not regard these statements as a representation or warranty by
Genprex or any other person that Genprex will achieve its
objectives and plans in any specified timeframe, or at all. You are
cautioned not to place undue reliance on these forward-looking
statements, which speak only as of the date of this press release.
Genprex disclaims any obligation to publicly update or release any
revisions to these forward-looking statements, whether as a result
of new information, future events or otherwise, after the date of
this press release or to reflect the occurrence of unanticipated
events, except as required by law.
Genprex, Inc.
(877) 774-GNPX (4679)
GNPX Investor
Relations
investors@genprex.com
GNPX Media Contact
Kalyn
Dabbs
media@genprex.com
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SOURCE Genprex, Inc.
