Keryx Biopharmaceuticals, Inc. Announces Positive Preliminary Phase 2 Data on KRX-0401 in Patients with Chemo-Insensitive Rare S
November 05 2007 - 8:29AM
PR Newswire (US)
Data presented at the 13th Annual Meeting of Connective Tissue
Oncology Society held in Seattle, Washington demonstrates clinical
activity and tolerability of perifosine as a single agent in
patients with chemo-insensitive sarcomas NEW YORK, Nov. 5
/PRNewswire-FirstCall/ -- Keryx Biopharmaceuticals, Inc.
(NASDAQ:KERX) today announced that preliminary data demonstrating
the tolerability and clinical activity of KRX-0401 (perifosine) in
patients with refractory, rare sarcomas was presented on Saturday,
November 3, 2007 at the Annual Meeting of the Connective Tissue
Oncology Society. In an oral presentation entitled "A PHASE II
TRIAL OF PERIFOSINE IN PATIENTS WITH CHEMO-INSENSITIVE SARCOMAS:
PRELIMINARY RESULTS," Dr. Joseph Ludwig Assistant Professor, Dept
of Sarcoma, MD Anderson Cancer Center reported on preliminary Phase
2 data in which single agent perifosine demonstrated a 40% overall
clinical benefit (Stable Disease > 3 months) in these subsets of
patients. Assessment of response was completed by both, RECIST and
Choi criteria, and results by sarcoma subtype as follows: Sarcoma
Histology Evaluable PR (Choi) SD > 12 wks Patients N (%) N (%)
Chondrosarcoma 25 1 (4%) 5 (20%) Extra-Skeletal Myxoid 13 2 (15%) 5
(38%) Alveolar Soft Part 10 3 (30%) 3 (30%) TOTAL 48 6 (13%) 13
(27%) Perifosine was also well tolerated with the most common grade
1 & 2 adverse events reported as nausea, vomiting, diarrhea and
fatigue. Commenting on the data, Dr. Craig Henderson, President of
Keryx Biopharmaceuticals stated, "We continue to remain encouraged
by the demonstrated clinical activity of perifosine in these rare
but unresponsive sarcomas. We are excited to be working with the
team of SARC investigators on this important clinical study and
look forward to completing accrual to all study arms in 2008. In
prior studies, KRX-0401 (perifosine) has been shown to be quite
active in the treatment of soft tissue sarcoma (ASCO 2007).
Responding patients experienced very little toxicity and the
duration of responses observed varied from 6 months to more than 18
months. Several types of sarcomas that responded to perifosine in
prior trials are particular interesting because they are generally
thought to be unresponsive to chemotherapy and no treatment has
been approved for them by the FDA. In this ongoing study, the
single agent activity of perifosine is being evaluated in patients
with chondrosarcoma, alveolar soft part sarcomas and extra-skeletal
myxoid chondrosarcomas. Patients are being treated with KRX-0401
(100 mg oral daily) until disease progression. Each of the three
sarcoma histology arms has met response criteria during the early
phases of the study to continue enrollment up to 37 patients. In
these studies, partial responses occurred in patients with sarcoma
subtypes that have been traditionally unresponsive to conventional
therapy. This Phase II study was initiated in December 2006 and is
being conducted by the Sarcoma Alliance for Research through
Collaboration (SARC) multi-center network, which includes
nationally recognized sarcoma centers and investigators throughout
the United States. Dr. Dejka M. Araujo, Assistant Professor in the
Department of Sarcoma at MD Anderson Cancer Center in Houston,
Texas is acting as Principal Investigator for the study. KRX-0401
(perifosine) is in-licensed by Keryx from Aeterna Zentaris, Inc.
(Nasdaq: AEZS; TSX: AEZ) in the United States, Canada and Mexico.
ABOUT KRX-0401 (Perifosine) KRX-0401 (perifosine) is a novel,
first-in-class, oral anticancer agent that modulates AKT and a
number of other key signal transduction pathways, including the
MAPK and JNK pathways all of which are pathways associated with
programmed cell death, cell growth, cell differentiation and cell
survival. High levels of activated Akt (pAkt) are seen frequently
in many types of cancer and have been correlated with poor
prognosis in patients with soft- tissue sarcoma, gastric,
hepatocellular, endometrial, prostate, renal cell, head and neck
cancers and hematological malignancies, as well as glioblastoma.
The majority of tumors expressing high levels of pAkt were
high-grade, advanced stage or had other features associated with
poor prognosis. High pAkt is often seen in tumors that are
resistant to conventional cancer treatments, including
radiotherapy, chemotherapy, endocrine therapy, and especially
therapy with some of the newer biologicals. The effects of
perifosine on Akt are of particular interest because of 1) the
importance of this pathway in the development of most cancers; 2)
the evidence that it is often activated in tumors that are
resistant to other forms of anticancer therapy; and 3) and the
difficulty encountered thus far in the discovery of drugs that will
inhibit this pathway without causing excessive toxicity. It is
plausible that perifosine may be useful in combination to reduce
the resistance to other cancer treatments. To date, over 1,200
patients have been treated with perifosine in trials conducted both
in the United States and Europe. Its safety profile is distinctly
different from that of most cytotoxic agents. It does not appear to
cause myelosuppression (depression of the immune system that may
lead to life threatening infections), thrombocytopenia (a decrease
in platelets that may result in bleeding), skin rash, flu-like
symptoms or alopecia (hair loss); all of these toxicities occur
frequently with many of the currently available treatments for
cancer. The main side effects of perifosine are nausea, vomiting,
diarrhea and fatigue, but these are either mild or non-existent in
doses that are known to induce tumor regression. In Phase 1/2
trials, perifosine has induced tumor regressions and/or caused
disease stabilization in a variety of tumor types. Perifosine has
shown single agent partial responses or long term disease
stabilizations in solid tumors including, renal, hepatocellular,
sarcoma and prostate cancer. There is also evidence of activity in
hematological malignancies, especially multiple myeloma. Responding
patients, including those with stable disease, have been treated
for months to more than three years. ABOUT KERYX
BIOPHARMACEUTICALS, INC. Keryx Biopharmaceuticals, Inc. is focused
on the acquisition, development and commercialization of medically
important, novel pharmaceutical products for the treatment of
life-threatening diseases, including diabetes and cancer. Keryx's
lead compound under development is Sulonex(TM) (sulodexide oral
gelcap), previously referred to as KRX-101, a first-in-class, oral
heparinoid compound for the treatment of diabetic nephropathy, a
life-threatening kidney disease caused by diabetes. Sulonex is in a
pivotal Phase 3 and Phase 4 clinical program under a Special
Protocol Assessment with the Food & Drug Administration.
Additionally, Keryx is developing Zerenex(TM), an oral, iron- based
compound that has the capacity to bind phosphate and form
non-absorbable complexes. Zerenex is currently in Phase II clinical
development for the treatment of hyperphosphatemia (elevated serum
phosphorous levels) in patients with end- stage renal disease.
Keryx is also developing clinical-stage oncology compounds,
including KRX-0401, a novel, first-in-class, oral anti- cancer
agent that modulates Akt, a protein in the body associated with
tumor survival and growth, and a number of other key signal
transduction pathways, including the JNK and MAPK pathways, which
are pathways associated with programmed cell death, cell growth,
cell differentiation and cell survival. KRX-0401 is currently in
Phase 2 clinical development for multiple tumor types. Keryx also
has an active in-licensing and acquisition program designed to
identify and acquire additional drug candidates. Keryx is
headquartered in New York City. Cautionary Statement Some of the
statements included in this press release, particularly those
anticipating future the financial performance and clinical and
business prospects for KRX-0401, may be forward-looking statements
that involve a number of risks and uncertainties. For those
statements, we claim the protection of the safe harbor for
forward-looking statements contained in the Private Securities
Litigation Reform Act of 1995. Among the factors that could cause
our actual results to differ materially are the following: our
ability to successfully complete the Phase 1 and Phase 2 clinical
trials for KRX-0401; we may not be able to meet anticipated
development timelines for KRX-0401 due to recruitment, clinical
trial results, manufacturing capabilities or other factors; and
other risk factors identified from time to time in our reports
filed with the Securities and Exchange Commission. Any
forward-looking statements set forth in this press release speak
only as of the date of this press release. We do not intend to
update any of these forward-looking statements to reflect events or
circumstances that occur after the date hereof. This press release
and prior releases are available at http://www.keryx.com/. The
information in our website is not incorporated by reference into
this press release and is included as an inactive textual reference
only. KERYX CONTACT: Lauren Fischer Director - Investor Relations
Keryx Biopharmaceuticals, Inc. Tel: 212.531.5965 E-mail:
DATASOURCE: Keryx Biopharmaceuticals, Inc. CONTACT: Lauren Fischer,
Director - Investor Relations of Keryx Biopharmaceuticals, Inc.,
+1-212-531-5965, Web site: http://www.keryx.com/
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