ODD is in addition to Three FDA Fast Track
Designations
Genprex expects to dose the first patient in
the Acclaim-3 clinical trial in the fourth quarter of
2023
AUSTIN,
Texas, Aug. 10, 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 the United
States Food and Drug Administration (FDA) has granted Orphan Drug
Designation (ODD) to the Company's lead drug candidate,
REQORSA® Immunogene Therapy (quratusugene ozeplasmid),
for the treatment of small cell lung cancer (SCLC).
In addition to ODD for the treatment of SCLC, in June 2023, the FDA granted Fast Track Designation
(FTD) for REQORSA Immunogene Therapy, in combination with
Genentech, Inc's Tecentriq® in patients with
extensive-stage small cell lung cancer (ES-SCLC) who did not
develop tumor progression after receiving Tecentriq and
chemotherapy as initial standard treatment. With ES-SCLC, the
cancer has spread from one lung to the other, or to other parts of
the body. Extensive-stage is the most common type of SCLC. FDA has
also granted Genprex FTD for two other indications of REQORSA
Immunogene Therapy, including REQORSA in combination with Tagrisso
for non-small cell lung cancer (NSCLC) in patients who have
progressed after Tagrisso treatment, and REQORSA in combination
with Keytruda for NSCLC in patients who have progressed after
Keytruda treatment.
"We are excited to receive Orphan Drug Designation from the FDA
for REQORSA for patients with SCLC," said Rodney Varner, President, Chairman and Chief
Executive Officer at Genprex. "This FDA Orphan Drug Designation in
combination with our recently received FDA Fast Track designation
underscores the great need for better treatment options for
patients with SCLC,ES-SCLC and NSCLC. We look forward to
initiating the Acclaim-3 clinical trial expected in the fourth
quarter of 2023 in order to bring hope of an effective new therapy
to patients suffering with this life-limiting cancer."
The FDA grants ODD status to investigational therapies being
developed to treat, diagnose, or prevent a rare disease or
condition affecting fewer than 200,000 people in the United States. Further, ODD provides
benefits to drug developers, including assistance in the drug
development process, tax credits for qualified trials, waiver of
certain FDA fees, and potential for seven years of post-approval
marketing exclusivity.
Genprex's method of treating cancer is to reexpress tumor
suppressor genes in cancers. Tumor suppressor genes are
deleted or inactivated early in the process of cancer development.
REQORSA contains a plasmid that expresses a tumor suppressor gene
protein called TUSC2. Virtually 100% of small cell lung cancers
have reduced or no TUSC2 protein expression, and 41% completely
lack TUSC2 protein expression. Pre-clinical studies in mice suggest
that re-expressing the TUSC2 protein may lead to clinical efficacy.
ES-SCLC has a very poor prognosis, with a median progression free
survival (PFS) of only 5.2 months. Importantly, median PFS for
patients receiving Tecentriq as maintenance therapy is only 2.6
months from the start of maintenance treatment, so there is a great
need for improvement in maintenance therapy.
About Acclaim-3 Clinical Trial
The Acclaim-3 clinical trial is a Phase 1/2 open-label,
dose escalation and clinical response study of maintenance therapy
evaluating REQORSA in combination with Tecentriq® in
patients with ES-SCLC who did not develop tumor progression after
receiving Tecentriq and chemotherapy as initial standard
treatment.
Patients in the Acclaim-3 clinical trial will be enrolled after
receiving initial treatment with 3-4 cycles of carboplatin,
etoposide, and Tecentriq, and achieving complete response, partial
response or stable disease. They will then receive treatment with
REQORSA and Tecentriq as maintenance therapy every 21 days until
disease progression.
The Phase 1 dose escalation portion of the Acclaim-3 clinical
trial is expected to enroll up to 12 patients at 3-5 U.S. clinical
sites to determine the Maximum Tolerated Dose (MTD). If no dose
limiting toxicities occur during Phase 1, then the highest dose
evaluated will be the Recommended Phase 2 Dose. The Phase 2
portion of the study will then enroll approximately 50 patients at
5-10 sites. Patients will be treated with REQORSA and Tecentriq
until disease progression or unacceptable toxicity is
experienced.
The primary endpoint of the Phase 2 portion of the trial is to
determine the 18-week progression-free survival rate from the time
of the start of maintenance therapy with REQORSA and Tecentriq in
patients with ES-SCLC. Patients will also be followed for survival.
A Phase 2 futility analysis will be performed after the
25th patient enrolled and treated reaches 18
weeks of follow up.
About Genprex, Inc.
Genprex 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 proprietary, non-viral ONCOPREX®
Nanoparticle Delivery System which encapsulates the gene-expressing
plasmids using lipid nanoparticles. 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® (quaratusugene ozeplasmid), is being evaluated
in three clinical trials as a treatment for non-small cell lung
cancer (NSCLC) and small cell lung cancer (SCLC). Each of Genprex's
three lung cancer clinical programs have received a Fast Track
Designation from the Food and Drug Administration (FDA), and its
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 adeno-associated virus (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, GPX-003 for Type 2 diabetes, where
autoimmunity is not at play, 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, 2022.
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; the timing and success of
Genprex's clinical trials and regulatory approvals; the effect of
Genprex's product candidates, alone and in combination with other
therapies, on cancer and diabetes; Genprex's future growth and
financial status, including Genprex's ability to continue 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; 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.