NEW CANAAN, CT announced today that its triphendiol (previously
known as NV-196) has been granted orphan drug status by the U.S.
Food and Drug Administration (FDA) for the treatment of pancreatic
cancer and for the treatment of cholangiocarcinoma, or bile duct
cancer.
An orphan drug refers to a product that is intended for use in a
disease or condition that affects fewer than 200,000 individuals in
the United States. A grant of orphan drug status provides 7 years
of market exclusivity for the orphan indication after approval by
the FDA, as well as tax incentives, study design assistance, and
eligibility for grant funding from the FDA during its
development.
Laboratory testing in vitro and in animals bearing human
pancreatic and bile duct tumors has demonstrated the activity of
triphendiol against cancer cells. In mice bearing a human
pancreatic cancer tumor, triphendiol administration resulted in a
mean reduction in tumor volume by 62 percent compared with
untreated control animals. In the two Phase I clinical studies
completed thus far, the investigational drug has demonstrated
acceptable pharmacokinetic profiles in healthy human volunteers
with no reported side effects.
Professor Alan Husband, Group Director of Research for Marshall
Edwards, said, "This grant of orphan drug status is a significant
step in the development program for triphendiol as a multipotent
anti-cancer agent."
"The activity seen in laboratory testing, coupled with the
favorable safety profile observed in early clinical testing,
suggest we may finally have discovered a drug which will be of
benefit in these unusually aggressive and difficult-to-treat
diseases," Professor Husband said.
Pancreatic cancer is considered an "orphan" cancer, because of
its relatively low incidence and high mortality. Pancreatic cancer
is a malignant tumor of the pancreas, which is slightly more common
in men than in women. In the U.S. it is the fourth leading cause of
cancer-related death in males and the fifth leading cause of
cancer-related deaths in women with a death rate estimated by the
National Cancer Institute as approximately 96 percent of patients
with the disease(1). The American Cancer Society estimated the
number of new cases of pancreatic cancer in the U.S. in 2007 as
37,170, with 18,830 cases diagnosed in men and 18,340 in
women(2).
Pancreatic cancer has a poor prognosis. The disease is difficult
to diagnose in its early stage, and patients usually present with
incurable disease. It has a high mortality rate, and no therapy has
been shown to significantly impact survival.
Bile duct cancer (otherwise known as cholangiocarcinoma) is a
malignant tumor that develops from the mucus glands lining the bile
duct and is also more common in men than in women. The American
Cancer Society estimated that in 2007 approximately 4,600 people in
the United States developed bile duct cancer outside the liver.
Bile duct cancer is also associated with a poor prognosis and has
limited treatment options(3).
Triphendiol is a second-generation derivative of phenoxodiol.
Phenoxodiol is an investigational drug that is currently undergoing
clinical evaluation in a Phase III study in platinum resistant
ovarian cancer patients, a study that has been approved under the
FDA's Special Protocol Assessment scheme. Patients seeking more
information about the phenoxodiol trial should visit
www.OVATUREtrial.com.
This class of drugs is derived from a proprietary phenolic drug
technology platform that has produced a number of anti-cancer lead
compounds, characterized by unusually broad activity against a
range of tumor targets in cell based studies, coupled with data
that suggest a highly satisfactory safety profile.
The CEO of Marshall Edwards, Mr. Christopher Naughton,
commenting on the Company's strategic plan for triphendiol, said,
"The Company now intends to vigorously pursue the clinical
development of triphendiol into Phase II human clinical studies in
affected patients during the course of 2008."
"With phenoxodiol now in a Phase III trial, coupled with this
latest boost in the development of its second cancer drug,
triphendiol, the Company is well placed to crystallize the value
inherent in the technology platform underpinning these novel
drugs," Mr. Naughton said.
About triphendiol
Triphendiol (NV-196) is another investigational drug in the
Marshall Edwards, Inc., oncology drug pipeline, currently being
developed as an orally-delivered chemosensitizing agent, intended
for use in conjunction with standard chemotoxic anti-cancer drugs
for the treatment of late stage pancreatic cancer,
cholangiocarcinoma, and melanoma. Triphendiol is broadly cytostatic
and cytotoxic against most forms of human cancer cells in vitro,
and has been shown to cause cell cycle arrest (or stop cells
increasing in number) and to induce apoptosis (or initiate
programmed cell death) in various cancer cell lines.
Biological studies suggest a mechanism of cytotoxicity that
involves the tumor necrosis factor-related apoptosis-inducing
ligand (TRAIL) death receptors. It exhibits high selectivity,
little effect on non-tumor cells and no observable toxicity in
animals at therapeutically effective doses. In human studies
conducted so far, no adverse events or side effects have been
reported when administered to healthy volunteers. Compared to
phenoxodiol, triphendiol has substantially greater activity in
laboratory testing against pancreatic cancer and
cholangiocarcinoma.
About Marshall Edwards, Inc. and Novogen Limited
Marshall Edwards, Inc., is a specialist oncology company focused
on the clinical development of novel anti-cancer therapeutics.
These derive from a flavonoid technology platform that has
generated a number of novel compounds characterized by broad
ranging activity in laboratory testing against a range of cancer
targets with few side effects. The ability of these compounds to
target an enzyme present on the surface of cancer cells, and
inhibit the production of pro-survival proteins within the cancer
cell suggests that they may possess a unique combination of
efficacy and safety. Marshall Edwards, Inc. has licensed rights
from Novogen Limited (ASX: NRT) (NASDAQ: NVGN) to bring three
oncology drugs -- phenoxodiol, triphendiol (NV-196) and NV-143 --
to market globally. Marshall Edwards, Inc., is majority owned by
Novogen, an Australian biotechnology company that is specializing
in the development of therapeutics based on a flavonoid technology
platform. Novogen, based in Sydney, Australia, is developing a
range of therapeutics across the fields of oncology, cardiovascular
disease and inflammatory diseases. More information on phenoxodiol
and on the Novogen group of companies can be found at
www.marshalledwardsinc.com and www.novogen.com.
Under U.S. law, a new drug cannot be marketed until it has been
investigated in clinical trials and approved by the FDA as being
safe and effective for the intended use. Statements included in
this press release that are not historical in nature are
"forward-looking statements" within the meaning of the "safe
harbor" provisions of the Private Securities Litigation Reform Act
of 1995. You should be aware that our actual results could differ
materially from those contained in the forward-looking statements,
which are based on management's current expectations and are
subject to a number of risks and uncertainties, including, but not
limited to, our failure to successfully commercialize our product
candidates; costs and delays in the development and/or FDA
approval, or the failure to obtain such approval, of our product
candidates; uncertainties in clinical trial results; our inability
to maintain or enter into, and the risks resulting from our
dependence upon, collaboration or contractual arrangements
necessary for the development, manufacture, commercialization,
marketing, sales and distribution of any products; competitive
factors; our inability to protect our patents or proprietary rights
and obtain necessary rights to third party patents and intellectual
property to operate our business; our inability to operate our
business without infringing the patents and proprietary rights of
others; general economic conditions; the failure of any products to
gain market acceptance; our inability to obtain any additional
required financing; technological changes; government regulation;
changes in industry practice; and one-time events. We do not intend
to update any of these factors or to publicly announce the results
of any revisions to these forward-looking statements.
(1) National Cancer Institute, A Snapshot of Pancreatic Cancer
(available at http://planning.cancer.gov/disease/snapshots.shtml).
(2) Cancer Facts and Figures, American Cancer Society, 2007.
(3) Cancer Facts and Figures, American Cancer Society, 2007.
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CONTACT: David Sheon +1 202 518-6321 (USA) Prof. Alan Husband
Group Director, Research Marshall Edwards, Inc. +61 2 9878 0088
(Australia)
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