Keryx Biopharmaceuticals Announces Special Protocol Assessment Agreement with FDA for Phase 3 Registration Program of Zerenex in
January 05 2010 - 7:30AM
PR Newswire (US)
Milestone Marks Attainment of SPA Agreements for Both of Keryx's
Phase 3 Drug Candidates, Zerenex and Perifosine NEW YORK, Jan. 5
/PRNewswire-FirstCall/ -- Keryx Biopharmaceuticals, Inc.
(NASDAQ:KERX) announced today that it has reached agreement with
the U.S. Food and Drug Administration (FDA) regarding a Special
Protocol Assessment (SPA) on the design of a Phase 3 clinical
program for Zerenex(TM) (ferric citrate), its iron-based phosphate
binder for the treatment of elevated serum phosphorous levels, or
hyperphosphatemia, in patients with end-stage renal disease (ESRD).
The SPA provides agreement that the Phase 3 program design
adequately addresses objectives in support of a regulatory
submission for drug approval. 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). This short-term study is expected to commence by
the end of the first quarter of 2010, with data expected in the
second half of 2010. -- 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 the same dose of phosphate binder administered
immediately prior to washout. 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. The long-term study is expected to
begin in mid-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 program. Dr.
Lewis commented, "We are extremely excited to be leading this
clinical program for Zerenex, which we believe is a differentiated,
iron-based phosphate binder. The data accumulated to date suggests
that Zerenex is an effective, tolerated phosphate binder, and,
given the growing market and limitations of the currently marketed
drugs for hyperphosphatemia, we believe that Zerenex, which is not
polymer-based and is free of aluminum, lanthanum, and calcium, will
potentially make a significant clinical addition to treating the
important universally present problem of hyperphosphatemia in
patients with end-stage renal disease." Ron Bentsur, Chief
Executive Officer of Keryx, stated, "The Zerenex SPA agreement is a
major milestone which provides us with a clearly defined
development and regulatory pathway for Zerenex in the treatment of
hyperphosphatemia, and we would like to thank the FDA for its
invaluable guidance throughout this process." Mr. Bentsur
continued, "With two Phase 3 drug candidates, Zerenex and
Perifosine, both with SPA agreements in place, we believe that
Keryx is well positioned to unlock significant shareholder value in
2010." Keryx Biopharmaceuticals retains a worldwide exclusive
license (except for the Asian Pacific Region) to Zerenex (ferric
citrate). The Company has sublicensed the development of ferric
citrate in Japan to Japan Tobacco Inc. and Torii Pharmaceutical
Co., Ltd. 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/Gui
dances/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, over
350,000, 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 the U.S.
exceeded $600 million in 2008, and has grown approximately 25% per
annum over the last five years. 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 the phosphoinositide 3-kinase
(PI3K)/Akt pathway, a key signaling cascade that has been shown to
induce cell growth and cell transformation. 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 also modulates a number of other key signal
transduction pathways, including the JNK pathway, which are
pathways associated with programmed cell death, cell growth, cell
differentiation and cell survival. KRX-0401 is currently in a Phase
3 trial, under Special Protocol Assessment (SPA), in multiple
myeloma, and in Phase 2 clinical development for several other
tumor types. 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
expected to begin this quarter under 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 (ferric citrate) and expectations regarding our
shareholder value, 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 program
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: Lauren Fischer Director, Investor Relations
Keryx Biopharmaceuticals, Inc. Tel: 212.531.5962 DATASOURCE: Keryx
Biopharmaceuticals, Inc. CONTACT: Lauren Fischer, Director,
Investor Relations, Keryx Biopharmaceuticals, Inc., +1-212-531-5962
Web Site: http://www.keryx.com/
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