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Keryx Biopharmaceuticals, Inc. Announces Positive Preliminary Phase 2 Data on KRX-0401 in Patients with Chemo-Insensitive Rare Sarcoma

Data presented at the 13th Annual Meeting of Connective Tissue Oncology Society held in Seattle, Washington demonstrates clinical activity and

tolerability of perifosine as a single agent in patients with

chemo-insensitive sarcomas

NEW YORK, Nov. 5 /PRNewswire-FirstCall/ -- Keryx Biopharmaceuticals, Inc. (Nasdaq: KERX) today announced that preliminary data demonstrating the tolerability and clinical activity of KRX-0401 (perifosine) in patients with refractory, rare sarcomas was presented on Saturday, November 3, 2007 at the Annual Meeting of the Connective Tissue Oncology Society.

In an oral presentation entitled "A PHASE II TRIAL OF PERIFOSINE IN PATIENTS WITH CHEMO-INSENSITIVE SARCOMAS: PRELIMINARY RESULTS," Dr. Joseph Ludwig Assistant Professor, Dept of Sarcoma, MD Anderson Cancer Center reported on preliminary Phase 2 data in which single agent perifosine demonstrated a 40% overall clinical benefit (Stable Disease > 3 months) in these subsets of patients. Assessment of response was completed by both, RECIST and Choi criteria, and results by sarcoma subtype as follows:

Sarcoma Histology Evaluable PR (Choi) SD > 12 wks

Patients N (%) N (%)

Chondrosarcoma 25 1 (4%) 5 (20%)

Extra-Skeletal Myxoid 13 2 (15%) 5 (38%)

Alveolar Soft Part 10 3 (30%) 3 (30%)

TOTAL 48 6 (13%) 13 (27%)

Perifosine was also well tolerated with the most common grade 1 & 2 adverse events reported as nausea, vomiting, diarrhea and fatigue.

Commenting on the data, Dr. Craig Henderson, President of Keryx Biopharmaceuticals stated, "We continue to remain encouraged by the demonstrated clinical activity of perifosine in these rare but unresponsive sarcomas. We are excited to be working with the team of SARC investigators on this important clinical study and look forward to completing accrual to all study arms in 2008.

In prior studies, KRX-0401 (perifosine) has been shown to be quite active in the treatment of soft tissue sarcoma (ASCO 2007). Responding patients experienced very little toxicity and the duration of responses observed varied from 6 months to more than 18 months. Several types of sarcomas that responded to perifosine in prior trials are particular interesting because they are generally thought to be unresponsive to chemotherapy and no treatment has been approved for them by the FDA. In this ongoing study, the single agent activity of perifosine is being evaluated in patients with chondrosarcoma, alveolar soft part sarcomas and extra-skeletal myxoid chondrosarcomas. Patients are being treated with KRX-0401 (100 mg oral daily) until disease progression. Each of the three sarcoma histology arms has met response criteria during the early phases of the study to continue enrollment up to 37 patients. In these studies, partial responses occurred in patients with sarcoma subtypes that have been traditionally unresponsive to conventional therapy.

This Phase II study was initiated in December 2006 and is being conducted by the Sarcoma Alliance for Research through Collaboration (SARC) multi-center network, which includes nationally recognized sarcoma centers and investigators throughout the United States. Dr. Dejka M. Araujo, Assistant Professor in the Department of Sarcoma at MD Anderson Cancer Center in Houston, Texas is acting as Principal Investigator for the study.

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 1,200 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 1/2 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.


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 3 and Phase 4 clinical program under a Special Protocol Assessment with the Food & Drug Administration. Additionally, Keryx is developing Zerenex(TM), an oral, 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 anti- cancer agent that modulates Akt, a protein in the body associated with tumor survival and growth, and a number of other key signal transduction pathways, including the JNK and MAPK pathways, which are pathways associated with programmed cell death, cell growth, cell differentiation and cell survival. KRX-0401 is currently in Phase 2 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 The information in our website is not incorporated by reference into this press release and is included as an inactive textual reference only.


Lauren Fischer

Director - Investor Relations

Keryx Biopharmaceuticals, Inc.

Tel: 212.531.5965


SOURCE Keryx Biopharmaceuticals, Inc.
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