Data From KRX-0401 (Perifosine) Phase II Single-Agent Soft-Tissue Sarcoma Study Presented at the 16th Annual EORTC-NCI-AACR Conf
September 30 2004 - 9:40AM
PR Newswire (US)
Data From KRX-0401 (Perifosine) Phase II Single-Agent Soft-Tissue
Sarcoma Study Presented at the 16th Annual EORTC-NCI-AACR
Conference Further Evidence of Single Agent Activity Observed NEW
YORK, Sept. 30 /PRNewswire-FirstCall/ -- Keryx Biopharmaceuticals
(NASDAQ:KERX) announced today that Phase II data presented at the
16th Annual EORTC-NCI-AACR symposium on "Molecular Targets and
Cancer Therapeutics" demonstrated the tolerability and potential
efficacy of KRX-0401 (perifosine) in the treatment of patients with
advanced soft tissue sarcoma. This study was conducted by the
National Cancer Institute (NCI) pursuant to a Collaborative
Research and Development Agreement (CRADA) between Keryx and the
NCI. In this single-agent Phase II multi-center study of KRX-0401,
23 patients with advanced soft-tissue sarcoma received a loading
dose of 150mg, every 6 hours starting on day 1, followed by 100mg
daily thereafter. The patients enrolled in this study had prior
treatment, including 1-2 chemotherapy regimens, surgery and/or
radiotherapy. Nineteen patients received more than one course of
treatment. There was one confirmed partial response lasting more
than 5 months and 2 patients that remained progression free at 6
months. This follows the evidence of single agent activity of the
drug in refractory sarcoma patients as seen in the earlier Phase I
program. KRX-0401 was also shown to be well tolerated at the doses
used. All 23 patients were evaluable for toxicity and notable
toxicities seen included Grade 4 ileus (1 patient), Grade 3
toxicity (6 patients) including fatigue (2 pts) and 1 patient each
of anemia, infection, muscle weakness, pain, rash, anorexia,
dehydration, and diarrhea. Of the grade 3 and 4 toxicities seen, it
is unclear which ones were related to the disease or the drug.
There was no Grade 3 or 4 nausea or vomiting seen in this trial.
The authors concluded in summary that the regimen was tolerable and
that the preliminary observation of another responder in
soft-tissue sarcoma, such as was seen in the Phase I program,
raises the question of whether specific histologies or tumor
characteristics might predict a more sensitive sub-population of
soft tissue sarcoma patients. I. Craig Henderson, M.D., President
of Keryx Biopharmaceuticals, commented, "The level of activity seen
in this study, combined with the previous Phase I single-agent
experience where 2 partial responses were reported out of 10
sarcoma patients enrolled, provides us with very strong evidence
for the potential single-agent activity of this drug in soft tissue
sarcoma, particularly in one or more subtypes." Dr. Henderson
added, "Soft tissue sarcoma is a very aggressive disease
characterized by many heterogeneous subtypes or histologies, for
which few, if any, drugs work across multiple subtypes. The overall
response rate of sarcomas to the most effective front-line
chemotherapy treatments is about 10-20%, and some subtypes are
totally unresponsive to any form of chemotherapy." Dr. Henderson
also stated, "We believe that our correlative science program will
help us to potentially identify at least one subtype of soft-tissue
sarcoma for which we may be able to conduct a single agent
registration trial with KRX-0401 in the near future." Michael S.
Weiss, Chairman and Chief Executive Officer of Keryx, added, "We
are very pleased with the results of this study. These data confirm
KRX-0401's potential anti-cancer activity as a single agent. In
addition, we believe that KRX-0401, as an oral AKT inhibitor, is
ideally suited for combination therapy with currently available
anti-cancer treatments such as chemotherapy, radiation therapy,
EGFR inhibitors and endocrine therapy. Our goal is that our
recently-started program combining KRX-0401 with other anti-cancer
therapies will lead to additional approaches to regulatory
approval." To access the abstract, entitled "Tolerability and
limited activity of perifosine in patients with advanced soft
tissue sarcoma (STS): a multi-center phase 2 consortium (P2C)
study," please click here: http://tinyurl.com/3mdlh. KRX-0401
(Perifosine) is in-licensed by Keryx from Aeterna Zentaris, Inc.
(TSX: AEZ, Nasdaq: AEZS), which holds ex-North American rights to
the drug. ABOUT KRX-0401 (Perifosine) KRX-0401 (Perifosine), a
novel, first-in-class, oral AKT-inhibitor, is believed to be the
only AKT inhibitor in clinical development primarily for the
treatment of cancer. Activation of the AKT pathway is believed to
play an important role in cell survival and cell proliferation.
KRX-0401 has demonstrated single agent anti-tumor activity in Phase
I studies and is currently being studied as a single agent for the
treatment of refractory non-small cell lung cancer in a
corporate-sponsored Phase II program. The Company is also
investigating KRX- 0401 in combination with chemotherapy as a
treatment for several tumor types, including non-small cell lung
cancer and breast cancer. In addition to the corporate-sponsored
clinical program, KRX-0401 is currently in 9 Phase II single agent
clinical trials in 6 tumor types, including, breast, prostate,
melanoma, pancreatic, sarcoma and head and neck cancer, being
conducted by the NCI under a CRADA arrangement. ABOUT KERYX
BIOPHARMACEUTICALS, INC. Keryx Biopharmaceuticals, Inc.
(NASDAQ:KERX) is focused on the acquisition, development and
commercialization of novel pharmaceutical products for the
treatment of life-threatening diseases, including diabetes and
cancer. Keryx is developing KRX-101 (sulodexide), a novel
first-in-class oral heparinoid compound, for the treatment of
diabetic nephropathy, for which Keryx has commenced a U.S.-based
Phase II/III clinical program. Additionally, Keryx is developing
three clinical-stage oncology compounds including KRX- 0401, a
novel, first-in-class oral AKT inhibitor in Phase II. Keryx also
has an active in-licensing and acquisition program designed to
identify and acquire clinical-stage drug candidates. Keryx
Biopharmaceuticals is headquartered in New York City. Some of the
statements included in this press release, particularly those
anticipating future financial performance, characteristics of and
prospects for the KRX-0401 clinical trial programs, operating
strategies and similar matters, are 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. Important factors may cause our actual results to
differ materially, including: our ability to obtain regulatory
approval of the use of KRX-0401 as a single-agent in soft tissue
sarcoma; the ability of our correlative science and pre-clinical
research program to identify at least one subtype of the disease
for which we will be able to conduct a single agent registration
trial with KRX-0401 in the near future; our success in combining
KRX-0401 with approved anti-cancer therapies in a manner that will
lead to additional approaches to regulatory approval; and other
risk factors identified from time to time in our SEC reports,
including, but not limited to, the report on Form 10-K for the year
ended December 31, 2003, and our quarterly report on Form 10-Q for
the quarter ended June 30, 2004. Any forward-looking statements set
forth in this news release speak only as of the date of this news
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 Keryx's website is not
incorporated by reference into this press release and is included
as an inactive textual reference only. KERYX CONTACT: Ron Bentsur
Vice President - Finance & Investor Relations Keryx
Biopharmaceuticals, Inc. Tel: 212.531.5965 E-mail: DATASOURCE:
Keryx Biopharmaceuticals, Inc. CONTACT: Ron Bentsur, Vice President
- Finance & Investor Relations of Keryx Biopharmaceuticals,
Inc., +1-212-531-5965, or Web site: http://www.keryx.com/
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