September 20, 2023 -- InvestorsHub NewsWire --
via NetworkNewsWire
Editorial Coverage: More than 30 million people in the
United States suffer with a rare disease; most of them are
receiving no current treatment. Supporting the development and
evaluation of new treatments, particularly for rare diseases, is a
key priority for the U.S. Food & Drug Administration. As part
of that process, the agency can grant Orphan Drug
Designation ("ODD") to a drug or biological product being
developed to prevent, diagnose or treat a rare disease or
condition. This designation is intended to spark innovation among
biotech companies that are developing treatments for these patient
populations, which by definition affect fewer than 200,000 people
in the U.S., by providing incentives such as tax credits, user-fee
exemptions and up to seven years of market exclusivity after FDA
approval. Last month, Genprex Inc. (NASDAQ:
GNPX) (Profile) was granted FDA orphan drug
designation for its REQORSA(R) immunogene therapy in development
for the treatment of small cell lung cancer ("SCLC"). The Phase 1/2
clinical trial, expected to dose the first patient in the fourth
quarter of 2023, uses a combination of REQORSA and Genentech Inc.'s
Tecentriq(R) as maintenance therapy 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. Genprex
joins other companies — including Roche Holding AG ADR
(OTCQX:
RHHBY), Jazz
Pharmaceuticals PLC (NASDAQ:
JAZZ), ALX Oncology
Holdings Inc. (NASDAQ:
ALXO) and Achilles Therapeutics
PLC (NASDAQ:
ACHL) — that have been granted ODD status or may
seek ODD status as they work to develop treatments for rare
diseases.
- Four decades after the Orphan Drug Act
was passed, hundreds of 'orphan' drugs have been approved for
use
- Genprex's REQORSA Immunogene Therapy has
gained both Orphan Drug Designation and Fast Track Designation
- With recent ODD and FTD designations,
Genprex is on the verge of commencing patient treatment in small
cell lung cancer in Q4 2023
Click here to view the custom infographic of
the Genprex editorial.
Launching Point for Revolution
More than 30 million Americans struggle with rare diseases.
A comprehensive
study published by the National Library of Medicine
("NLM") states that the 1983 passage of the Orphan Drug Act
("ODA"), which created the Orphan Drug Designation Program,
represented a launching point for a rare-disease drug-development
revolution for these patients.
"Drug development for rare diseases can be commercially risky,
and prior to this legislation, only approximately two drugs per
year had been approved by the U.S. Food and Drug Administration for
rare diseases," the report observed. "Now, four decades later,
hundreds of 'orphan' drugs have been approved for use in the
7,000–10,000 diseases and conditions that are considered
rare."
Specifically, the report noted that 6,340 orphan drug
designations were granted, representing drug development for 1,079
rare diseases. "Additionally, 882 of those designations resulted in
at least one FDA approval for use in 392 rare diseases," the report
continued. "Much of this development has been concentrated in
oncology as seven of the top ten most designated and approved
diseases were rare cancers. Based on our study, we can conclude
that around 5% of rare diseases have an FDA-approved drug and up to
15% of rare diseases have at least one drug that has been developed
and shown promise in their treatment, diagnosis or prevention.
Funding of basic and translational science for rare-disease drug
development should continue in order to bring therapies to the
millions of affected patients who remain without treatment
options."
Bringing Hope
As the NLM article observed, there is a growing recognition of
the potential of gene therapies to transform cancer
care. Genprex
(NASDAQ:
GNPX), a clinical-stage gene therapy company focused on
developing life-changing therapies for patients with cancer and
diabetes, is among those companies leveraging this trend. The
company is currently working in both the non-small cell lung cancer
("NSCLC") and the small cell lung cancer ("SCLC") space, where its
REQORSA Immunogene Therapy (quratusugene ozeplasmid)
has gained both Orphan Drug Designation and Fast
Track Designation ("FTD").
"We are excited to receive Orphan Drug Designation from the FDA
for REQORSA for patients with SCLC," said Rodney Varner, Genprex
president, chair and CEO. "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 ES (extensive-stage)-SCLC and non-small cell lung
cancer ("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."
Meeting the SCLC Need
In the United States, lung cancer is the most lethal cancer in
both men and women, with the American Cancer Society estimating
that almost 130,000
people will die this year alone. SCLC comprises approximately
14% of all lung cancers. Limited stage SCLC (LS-SCLC) accounts
for approximately 33% of all SCLC and is potentially curable with a
combination of cytotoxic chemotherapy and radiation
therapy. However, extensive stage (ES-SCLC), which comprises
approximately 66% of all SCLC, is not considered curable. There is
an unmet need for more effective maintenance therapy options for
ES-SCLC, as well as for better combination treatments for SCLC
overall.
This is the space Genprex is looking to fill with its REQORSA
Immunogene Therapy. The company received the FTD approval for
REQORSA Immunogene Therapy in combination with Genentech Inc.'s
Tecentriq(R). Genentech is the U.S.-based unit of Swiss pharma
giant Roche.
With ES-SCLC, cancer has spread from one lung to the other, or
to other parts of the body. Genprex will be combining its drug with
Genentech's Tecentriq to focus on this type of cancer during the
upcoming Acclaim-3 clinical trial. The phase 1/2 open-label, dose
escalation and clinical response study will evaluate 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.
Genprex has also been granted FTD approval for two other
indications of REQORSA Immunogene Therapy: REQORSA in combination
with Tagrisso for 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.
Transforming Lives, Redefining Treatments
Genprex operates in the dynamic fields of gene therapy,
specifically targeting cancer and diabetes. With a focus on
developing groundbreaking treatments for patients, the company's
market is characterized by the urgent need for innovative
solutions. The cancer and diabetes sectors are vast and rife with
unmet medical needs, offering significant opportunities for
pioneering therapies that can transform lives and redefine
treatment paradigms.
Genprex's potential for growth is underscored by its promising
pipeline of advanced gene therapies in development. REQORSA is the
company's lead candidate, which it hopes will revolutionize the
treatment of lung cancers. With the recent ODD and FTD
designations, along with a robust clinical-development program,
Genprex is poised to make a significant impact in the market.Bottom
of Form
Elevating Innovation
Finding solutions in the rare disease sector can be challenging
but certainly fulfilling as companies research, explore and strive
to help those who need it the most. Add the incentives that come
with an Orphan Drug Designation, and those efforts can become even
more rewarding for these companies.
Roche Holding AG ADR
(OTCQX:
RHHBY), through Genentech, has received several Orphan
Drug Designations. In addition to atezolizumab for the treatment of
adult patients with ES-SCLC, Genentech most
recently received
Orphan Drug Designation for pralsetinib in the treatment
of certain types of solid tumors. The ODD designation covers
the use of pralsetinib in metastatic RET fusion-positive solid
tumors. According to the terms, Roche has exclusive rights to
commercialize the treatment outside the U.S. except for Greater
China.
Jazz
Pharmaceuticals PLC (NASDAQ:
JAZZ), a global biopharmaceutical company dedicated to
developing life-changing medicines for people with serious
diseases, has also developed a drug for the SCLC market. Its
Zepzelca(TM) (lurbinectedin), which was developed in collaboration
with PharmaMar, was granted
Orphan Drug Designation in 2018, and in 2020,
it received
accelerated FDA approval based on overall response rate
("ORR") and duration of response.
ALX Oncology
Holdings Inc. (NASDAQ:
ALXO), a clinical-stage immuno-oncology company
developing therapies that block the CD47 checkpoint
pathway, received ODD
from both the FDA and the European Commission for its
evorpacept, a next-generation CD47 blocker. The ODD indication
includes both gastric cancer and gastroesophageal junction
adenocarcinoma. "Receiving orphan drug designation from both the EC
and the FDA represents a significant regulatory achievement for ALX
Oncology and signifies the growing recognition of evorpacept as a
potential new drug to improve clinical outcomes in patients with
GC," said ALX Oncology chief medical officer Sophia Randolph, MD.,
PhD.
Achilles
Therapeutics PLC (NASDAQ:
ACHL) is a biopharmaceutical company developing
precision T cell therapies that target clonal neoantigens, or
protein markers unique to each individual. Lead indications,
including advanced NSCLC and recurrent metastatic melanoma,
were selected for their high mutational burden, high
level of T cell infiltration and high unmet medical need. Deaths
from non-small cell lung cancer outnumber those resulting from
breast cancer, colon cancer and prostate cancer combined, and there
are limited approved treatment options for lung cancer and melanoma
patients whose disease has not responded well to other therapies.
In its March 25, 2021, SEC
filing, the company noted that it "may in the future seek
orphan drug designation for ATL001 and any future product
candidates."
For companies looking to help those in the underserved
rare-disease space, an Orphan Drug Designation is the ideal pathway
to progress. When a company such as Genprex, which truly is
committed to innovation and patient-centric approaches, receives
the designation, it becomes an even more distinctive
differentiator. As investors explore opportunities in the biotech
sector, Genprex stands out with its cutting-edge therapies,
potential market disruption and dedication to transforming lives
through innovative science.
For more information about Genprex Inc., please
visit Genprex.
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Editorial Coverage
Roche (QX) (USOTC:RHHBY)
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Roche (QX) (USOTC:RHHBY)
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