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Publication in the Journal of the National Cancer Institute Demonstrates KRX-0401 (Perifosine) Single-Agent Potential in Neuroblastoma Tumors

Perifosine observed to be an effective novel agent in neuroblastoma cells in vitro and in vivo

Phase 1 data of single agent perifosine as a treatment for recurrent solid tumors in pediatric patients, including neuroblastoma patients, to be presented at ASCO

NEW YORK, May 17 /PRNewswire-FirstCall/ -- Keryx Biopharmaceuticals (Nasdaq: KERX) today announced the publication of an article in the May 12th Journal of the National Cancer Institute, entitled "In Vitro and In Vivo Inhibition of Neuroblastoma Tumor Cell Growth by AKT Inhibitor Perifosine," demonstrating the single agent activity of KRX-0401 (perifosine) in neuroblastoma tumor preclinical models. Neuroblastoma is the most common pediatric solid tumor. Perifosine, the Company's novel, potentially first-in-class, oral anti-cancer agent that inhibits AKT activation in the phosphoinositide 3-kinase (PI3K) pathway, is currently being investigated in a Phase 1 study as a single agent treatment for recurrent solid tumors, including neuroblastoma, in pediatric patients.

The article states that activated AKT is a marker of decreased event-free or overall survival in neuroblastoma patients, and that the aim of this study was to investigate the effect of perifosine, an AKT inhibitor, as a single agent on neuroblastoma cell growth in vitro and in vivo. The preclinical study investigated the activity of perifosine on four human neuroblastoma cell lines, as well as on the survival, tumor growth, and activation status of AKT in mice bearing human neuroblastoma xenograft tumors. Perifosine showed a statistically significant reduction in neuroblastoma cell survival, slowed or regressed tumor growth, and increased survival in mice bearing neuroblastoma tumors. A decreased level of activated AKT was also observed in perifosine-treated neuroblastoma cells and xenograft tumors.

The investigators concluded that perifosine inhibited the activation of AKT and was an effective cytotoxic agent in neuroblastoma cells in vitro and in vivo, and that this data supports the future clinical evaluation of perifosine for the treatment of neuroblastoma tumors.

Ron Bentsur, Chief Executive Officer of Keryx, commented, "We find this data to be encouraging, and we look forward to our upcoming ASCO presentation in which we will have Phase 1 single agent perifosine data in pediatric patients, including patients with neuroblastoma. Mr. Bentsur added, "While our Phase 3 programs for perifosine in metastatic colorectal cancer and multiple myeloma are moving forward, it is also exciting to see data that can potentially provide important clinical paths for the treatment of pediatric cancer patients worldwide."

A copy of the article can be obtained at

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 studies. Perifosine is also in Phase 1 and 2 clinical development for several other tumor types.

KRX-0401 (perifosine) is in-licensed by Keryx from Aeterna Zentaris Inc. in the United States, Canada and Mexico.

About Neuroblastoma

Neuroblastoma is the most common pediatric solid tumor and is also the most frequently diagnosed neoplasm during infancy. Neuroblastoma accounts for more than 7% of malignancies in patients younger than 15 years and causes 15% of all pediatric oncology deaths. Activated AKT is believed to be highly expressed in poor prognosis neuroblastoma tumors. Infants, even those with metastatic disease, may experience complete regression of their disease with single low-dose chemotherapy or observation alone in carefully selected circumstances. However, poor prognosis patients, usually older than 18 months and who have extensive metastatic disease, may initially respond to intensive multimodality chemotherapy, but the tumors eventually recur and become resistant to chemotherapy. Approximately half of all neuroblastoma patients are diagnosed with high-risk poor prognosis disease, and these patients have an overall survival rate of less than 40%. Therefore, a major challenge is to improve the treatment efficacy in high-risk neuroblastoma patients.

There are currently no FDA approved drugs for the treatment of neuroblastoma.

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 The information found on our website and the NIH website is not incorporated by reference into this press release and is included for reference purposes only.


Lauren Fischer

Director - Investor Relations

Keryx Biopharmaceuticals, Inc.

Tel: 212.531.5965




SOURCE Keryx Biopharmaceuticals, Inc.

Posted: May 2010