Keryx Biopharmaceuticals Announces Phase 1 Data of Single Agent Perifosine in the Treatment of Recurrent Pediatric Solid Tumors, Including Patients with Advanced Brain Tumors and Neuroblastoma
Perifosine demonstrated to be safe and tolerable, with early signs of clinical benefit in advanced refractory pediatric neuroblastoma patients
NEW YORK, June 7 /PRNewswire-FirstCall/ -- Keryx
Biopharmaceuticals, Inc. (NASDAQ:KERX) today announced that Phase 1 data
of KRX-0401 (perifosine) in recurrent pediatric solid tumors was
presented yesterday in the pediatric solid tumor poster discussion
session held at the 46th Annual Meeting of the American Society of
Clinical Oncology (ASCO) taking place in Chicago, IL. Perifosine is
the Company's novel, potentially first-in-class, oral anti-cancer
agent that inhibits Akt activation in the phosphoinositide 3-kinase
(PI3K) pathway. This study, conducted by the Memorial
Sloan-Kettering Cancer Center pediatric group, marks the first time
that perifosine has been administered in a pediatric patient
setting.
Study Design: This Phase I Study of Perifosine for Recurrent
Pediatric Solid Tumors is a single center, open-label,
dose-escalating study to assess safety, tolerability,
pharmacokinetics (PK), and to identify any dose limiting toxicity
(DLT) of single agent perifosine in pediatric patients with any
solid tumor that has failed standard therapy. Eleven patients (4
males, 7 females), at a median age of 13 years (5-18) were treated
in this study to date. The following tumor types were treated thus
far: high-grade glioma (5), medulloblastoma (2), neuroblastoma (3),
and ependymoma (1). Most patients were heavily pretreated, with a
median of three prior lines of therapy. Cohorts of three patients
were treated at three dose levels: 25mg/m2/day, 50mg/m2/day and
75mg/m2/day using 50mg tablets of perifosine after a loading dose
on day 1, and taking into account the drug's long half-life
(>100hrs). No DLTs were observed at any of the three dose
levels; dose level 4 is currently open for accrual. PK data thus
far suggests similar drug absorption by pediatric patients relative
to adult patients treated with single agent perifosine.
Of particular interest are the early signs of clinical activity
observed in two of the three patients with Stage 4 refractory
neuroblastoma. Both patients were refractory to prior treatments
upon entering the study and achieved stable disease for 48 weeks
and 55+ weeks (ongoing). The investigators concluded that
perifosine is well-tolerated in children with recurrent solid
tumors and that these early signals of activity warrant further
investigation in patients with advanced neuroblastoma and select
brain tumors. Recently, NCI investigators published in vitro and in
vivo data demonstrating that perifosine targets the activation of
Akt in neuroblastoma cells and xenografts, significantly inhibits
tumor growth in vivo and improves the survival of mice bearing
neuroblastoma tumors.
Ron Bentsur, Chief Executive Officer of Keryx
Biopharmaceuticals, commented, "We are pleased at the safety and
tolerability of single agent perifosine in pediatric patients and
encouraged by the early signals of clinical benefit in
neuroblastoma, as noted by the investigators. We are grateful to
the researchers and we look forward to exploring perifosine's
potential in future pediatric settings, including
neuroblastoma."
Perifosine is currently in Phase 3 clinical development for
refractory advanced colorectal cancer and multiple myeloma, both of
these Phase 3 programs being conducted under Special Protocol
Assessment (SPA) agreements with the FDA with Fast Track
designations obtained for both indications. Perifosine is also in
Phase 1 and 2 clinical development for several other tumor
types.
A copy of the related abstract, #9540, entitled "Phase I Study
of Single Agent Perifosine for Recurrent Pediatric Solid Tumors,"
lead author Oren J. Becher, M.D., can be accessed through the ASCO
website, www.asco.org. A copy of the poster may be obtained by
contacting the Company.
KRX-0401 (perifosine) is in-licensed by Keryx from Aeterna
Zentaris Inc. in the United States, Canada and Mexico.
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 KRX-0401 (perifosine), 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 KRX-0401; the risk that the data (both safety and
efficacy) from ongoing clinical trials will not coincide with the
data analyses from prior pre-clinical and 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 ASCO 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: 212.531.5965 E-mail: lfischer@keryx.com
Source: Keryx Biopharmaceuticals, Inc.
CONTACT: Lauren Fischer, Director - Investor Relations,
Keryx
Biopharmaceuticals, Inc., +1-212-531-5965, lfischer@keryx.com
Web Site: http://www.keryx.com/
Posted: June 2010
