Keryx Biopharmaceuticals, Inc. Announces Positive Phase I and Phase II Data on KRX-0401 in Patients with Relapsed/Refractory Mul
June 29 2007 - 10:53AM
PR Newswire (US)
Data presented at the 11th International Myeloma Workshop held in
Kos, Greece demonstrates clinical activity of perifosine in
combination with bortezomib, dexamethasone and lenalidomide plus
dexamethasone, in patients with relapsed/refractory multiple
myeloma, and as a single agent in patients with relapsed/refractory
Waldenstrom's macroglobulinemia NEW YORK, June 29
/PRNewswire-FirstCall/ -- Keryx Biopharmaceuticals, Inc.
(NASDAQ:KERX) today announced that 4 posters were presented at the
11th International Myeloma Workshop demonstrating the clinical
activity of KRX-0401 (perifosine) in patients with advanced
multiple myeloma and Waldenstrom's macroglobulinemia. Perifosine is
currently being evaluated in 3 ongoing, multi-center clinical
studies for patients with relapsed or relapsed/refractory multiple
myeloma, including in combination with dexamethasone, in
combination with Velcade(R) (bortezomib) and in combination with
Revlimid(R) (lenalidomide) plus dexamethasone. Perifosine is also
being evaluated in an ongoing phase II single agent study in
patients with relapsed and/or refractory Waldenstrom's
macroglobulinemia. Data from each of these ongoing studies was
presented during the conference and demonstrates perifosine's
activity, both as a single agent and as part of a combination, in
patients with multiple myeloma and Waldenstrom's macroglobulinemia.
Perifosine in the treatment of Patients with Relapsed/Refractory
Multiple Myeloma: In a poster entitled "A Multicenter Phase I/II
Trial of Perifosine (KRX- 0401) + Bortezomib in Relapsed and
Refractory Multiple Myeloma in Patients Previously Treated with
Bortezomib: Preliminary Results", Dr. Paul Richardson, Clinical
Director of the Jerome Lipper Multiple Myeloma Center at the Dana-
Farber Cancer Institute in Boston, MA reported an overall response
rate (partial response (PR) + minimal response (MR)) of 31%. In 16
evaluable patients treated with perifosine plus bortezomib, there
were 2 PR's and 1 MR. Two additional MR's were observed on this
combination after dexamethasone was added; both of these patients
were previously refractory to the combination of bortezomib +
dexamethasone without perifosine. Eleven patients remain on study
and the phase II portion of the study is now open and enrolling. A
second poster presented by Dr. Richardson, entitled " Perifosine
(KRX- 0401) + Low Dose Dexamethasone is Active in Patients with
Relapsed and Refractory Multiple Myeloma (MM): Perifosine MM
Investigator Group Phase II Multicenter Study Update" demonstrated
that 78% of patients treated with perifosine plus low dose
dexamethasone had at least stable disease (SD), including 26% that
had a PR or MR. Patients continue on study now ranging from 6
months up through 1 year. Dr. Andrzej Jakubowiak, Director of the
myeloma program at the University of Michigan Cancer Center,
presented a poster entitled "A Multicenter Phase I Trial of
Perifosine (KRX-0401) in Combination with Lenalidomide and
Dexamethasone in Patients with Relapsed or Refractory Multiple
Myeloma: Preliminary Results. Multiple Myeloma Research Consortium
(MMRC) Trial", demonstrating early encouraging results, with 50% of
patients achieving a partial response or better. Commenting on the
data, Dr. Richardson stated, "Perifosine's potential in combination
with bortezomib, dexamethasone and other novel therapies to
overcome drug-resistant multiple myeloma is promising. Our team of
investigators, including top centers across the country and now
incorporating the considerable resources of the MMRC for one of the
trials, is committed to completing enrollment in our studies and
evaluating future clinical strategies to improve patient outcome."
Perifosine in the treatment of Patients with Relapsed/Refractory
Waldenstrom's Macroglobulinemia: A poster entitled "A Phase II
Trial of Perifosine (KRX-0401) in Relapsed and/or Refractory
Waldenstrom's Macroglobulinemia: Preliminary Results" demonstrated
single agent activity of perifosine with 36% of patients having a
PR or MR. Dr. Irene Ghobrial of the Dana-Farber Cancer Institute,
who presented the poster, stated, "We are excited at the efficacy
and safety results with perifosine in a patient population for
which there is no FDA approved therapy. We look forward to
reporting updated results at future meetings." I. Craig Henderson,
MD, President of Keryx Biopharmaceuticals, commented "We are very
enthusiastic about the potential of perifosine in the treatment of
multiple myeloma and Waldenstrom's macroglobulinemia and the
prospects of using perifosine to improve upon the current care for
these patients." In addition to the clinical posters, three
pre-clinical posters were presented, as follows: Perifosine, an
Oral Bioactive Novel Akt Inhibitor, Induces In Vitro and In Vivo
Anti-tumor Activity in Waldenstrom's Macroglobulinemia Lead
Investigator: Xavier Leleu, MD, Dana-Farber Cancer Institute,
Boston, MA The Combination of Perifosine with Bortezomib and
Rituximab Provides Synergistic Anti-tumor Activity in Waldenstrom's
Macroglobulinemia Lead Investigator: Xavier Leleu, MD, Dana-Farber
Cancer Institute, Boston, MA Combination of the AKT Inhibitor
Perifosine with the HSP90 Inhibitor 17-
(Dimethylaminoethylamino)-17-Demethoxygeldanamycin (17-DMAG) Has
Synergistic Activity in tumor cell and its microenvironment in
Multiple Myeloma (MM) Lead Investigator: Alissa Huston, MD,
University of Pittsburgh Cancer Center Copies of all posters can be
obtained from Keryx Biopharmaceuticals, Inc. 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 1,500
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 I/II
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 III and Phase IV clinical program under a Special
Protocol Assessment with the Food & Drug Administration.
Additionally, Keryx is developing Zerenex(TM), an oral, inorganic,
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
modulator of Akt, a pathway associated with tumor survival and
growth, and other important signal transduction pathways. KRX-0401
is currently in Phase II 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. Contact: Lauren Fischer Director of Investor Relations Keryx
Biopharmaceuticals, Inc. (212)531-5965 DATASOURCE: Keryx
Biopharmaceuticals, Inc. CONTACT: Lauren Fischer, Director of
Investor Relations for Keryx Biopharmaceuticals, Inc.,
+1-212-531-5965 Web site: http://www.keryx.com/
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