NEW YORK, May 6 /PRNewswire-FirstCall/ -- Keryx
Biopharmaceuticals, Inc. (Nasdaq: KERX) announced today the
initiation of its short-term Phase 3 study of Zerenex™ (ferric
citrate), the Company's iron-based phosphate binder for the
treatment of elevated serum phosphorous levels, or
hyperphosphatemia, in patients with end-stage renal disease on
dialysis. The initiation of this study marks the commencement
of the Company's Phase 3 registration program for Zerenex, which is
being conducted in accordance with a Special Protocol Assessment
(SPA) agreement with the FDA. Pursuant to the Company's SPA
agreement, the Zerenex Phase 3 registration program will consist of
the short-term efficacy study initiated today, and 58-week
long-term safety and efficacy study, to be initiated in the third
quarter of 2010. Patients completing the short term study are
eligible to be enrolled into the long-term study.
The short-term efficacy study initiated today is a multicenter,
randomized, open-label clinical trial with a planned enrollment of
approximately 150 patients on hemodialysis. All patients will
undergo a 2-week washout period, following which the patients will
be randomized 1:1:1 to receive a fixed dose of Zerenex (1 gram, 6
grams or 8 grams per day) for a treatment period of 28 days.
The primary endpoint of the study is to demonstrate a dose
response in the change of serum phosphorous from baseline (end of
washout period) to end of the treatment period (day 28).
Approximately 15 sites in the U.S. will participate in the study.
Patient enrollment is expected to take up to 6 months, with
study completion expected by the end of 2010.
Dr. Julia Lewis, Professor of
Medicine, Department of Nephrology, Vanderbilt
University School of Medicine, and member of the Executive
Committee of the Collaborative Study Group, will be the Study Chair
of the Zerenex Phase 3 registration program. Dr. Samuel S. Blumenthal, Professor of Medicine at
Medical College of Wisconsin, will
serve as the study's Co-Principal Investigator.
Dr. Julia Lewis commented, "I am
pleased to be leading the registration program for Zerenex.
The clinical data generated to date suggests that Zerenex is
an effective, safe and well-tolerated phosphate binder which we
expect is differentiated from other marketed therapies by its iron
composition and potential dosing convenience. We are
hopeful that this Phase 3 registration program of Zerenex will lead
to a new treatment option for hyperphosphatemia benefiting patients
with end-stage renal disease."
Ron Bentsur, Chief Executive
Officer of Keryx , commented, "We are very excited about the
initiation of the Zerenex Phase 3 program and look forward to
generating Phase 3 data from this study later this year." Mr.
Bentsur, continued, "We believe that Zerenex could emerge with the
attributes to capture meaningful market share in a worldwide
phosphate binder market approaching $1.5
billion. Finally, I want to thank the study
investigators for their tremendous support and guidance."
Keryx expects to complete the Zerenex Phase 3 program and file a
New Drug Application for Zerenex for the treatment of
hyperphosphatemia in the first half of 2012.
Keryx Biopharmaceuticals retains a worldwide exclusive license
(except for the Asian Pacific Region) to Zerenex (ferric citrate)
from Panion & BF Biotech, Inc. The Company has
sublicensed the development of ferric citrate in Japan to Japan Tobacco Inc. and Torii
Pharmaceutical Co., Ltd.
PHASE 3 TRIAL DESIGN:
In accordance with the Company's SPA agreement with the FDA, the
Phase 3 clinical program for Zerenex will consist of two clinical
studies, as follows:
Short-term efficacy study: A multicenter, randomized, open-label
clinical trial with a planned enrollment of approximately 150
patients on hemodialysis, who will be randomized to fixed doses of
Zerenex, ranging from 1 gram per day to 8 grams per day, for a
treatment period of 28 days. Patients will undergo a 2-week washout
period prior to randomization. The primary endpoint of the
study will be to demonstrate a dose response in the change of serum
phosphorous from baseline (end of washout period) to end of the
treatment period (day 28).
Long-term safety and efficacy study: A multicenter, randomized,
open-label, safety and efficacy clinical trial with a planned
enrollment of approximately 300 patients on hemodialysis or
peritoneal dialysis. The long term study will consist of a
2-week washout period followed by a 52-week safety assessment in
which patients will be randomized 2:1 to receive either Zerenex or
another phosphate binder. The 52-week safety assessment will
be followed by a 4-week efficacy assessment in which only patients
randomized to treatment with Zerenex during the safety assessment
will be randomized to continue treatment with either Zerenex or
placebo for a 4-week period.
About Special Protocol Assessments
The Special Protocol Assessment (SPA) process is a procedure by
which the FDA provides official evaluation and written guidance on
the design and size of proposed protocols that are intended to form
the basis for a new drug application.
Final marketing approval depends on the results of efficacy, the
adverse event profile and an evaluation of the benefit/risk of
treatment demonstrated in the Phase 3 clinical program. The
SPA agreement may only be changed through a written agreement
between the sponsor and the FDA, or if the FDA becomes aware of a
substantial scientific issue essential to product efficacy or
safety. For more information on Special Protocol Assessment,
please visit:
http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm080571.pdf.
About Hyperphosphatemia
In the United States, according
to data from the U.S. Renal Data System, there are approximately
485,000 patients with end-stage renal disease, or ESRD, and the
number of ESRD patients is projected to rise 60% to approximately
785,000 by 2020. The majority of ESRD patients, close to 400,000,
require dialysis. Phosphate retention and the resulting
hyperphosphatemia in patients with ESRD on dialysis are usually
associated with secondary hyperparathyroidism (and its related
cardiovascular complications), renal osteodystrophy and soft tissue
mineralization. ESRD patients usually require treatment with
phosphate-binding agents to lower and maintain serum phosphorus at
acceptable levels. The need for alternative phosphate-binding
agents has long been recognized, especially given the increasing
prevalence of ESRD as well as shortcomings with current therapies.
Zerenex has the potential to be an effective and safe treatment in
lowering and/or maintaining normal serum phosphorus levels in
patients with ESRD and hyperphosphatemia.
The market for phosphate binders to treat hyperphosphatemia in
ESRD patients in 2009 was approximately $750
million and $1.3 billion in
the U.S. and worldwide, respectively.
About Keryx Biopharmaceuticals, Inc.
Keryx Biopharmaceuticals is focused on the acquisition,
development and commercialization of medically important
pharmaceutical products for the treatment of life-threatening
diseases, including cancer and renal disease. Keryx is developing
KRX-0401 (perifosine), a novel, potentially first-in-class, oral
anti-cancer agent that inhibits Akt activation in the
phosphoinositide 3-kinase (PI3K) pathway, and also affects a number
of other key signal transduction pathways, including the JNK
pathway, all of which are pathways associated with programmed cell
death, cell growth, cell differentiation and cell survival.
KRX-0401 has demonstrated both safety and clinical efficacy in
several tumor types, both as a single agent and in combination with
novel therapies. KRX-0401 is currently in Phase 3 clinical
development for both refractory advanced colorectal cancer and
multiple myeloma, and in Phase 1 and 2 clinical development for
several other tumor types. Each of the KRX-0401 Phase 3 programs
are being conducted under Special Protocol Assessment (SPA)
agreements with the FDA. Keryx is also developing Zerenex(TM)
(ferric citrate), an oral, iron-based compound that has the
capacity to bind to phosphate and form non-absorbable complexes.
The Phase 3 clinical program of Zerenex in the treatment for
hyperphosphatemia (elevated phosphate levels) in patients with
end-stage renal disease is being conducted pursuant to an SPA
agreement with the FDA. Keryx is headquartered in New York City.
Cautionary Statement
Some of the statements included in this press release,
particularly those anticipating future clinical trials and business
prospects for Zerenex™ 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
and cost-effectively complete clinical trials for Zerenex™; the
risk that the data (both safety and efficacy) from the Phase 3
trials will not coincide with the data analyses from the Phase 2
clinical trials previously reported by the Company; 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 undertake 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 found on our
website and the FDA website is not incorporated by reference into
this press release and is included for reference purposes only.
KERYX
CONTACT:
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Lauren
Fischer
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Director - Investor
Relations
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Keryx
Biopharmaceuticals, Inc.
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Tel:
212.531.5965
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E-mail:
lfischer@keryx.com
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SOURCE Keryx Biopharmaceuticals, Inc.