Article demonstrates single agent activity of KRX-0401 in neuroblastoma tumor preclinical models

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 http://www.ncbi.nlm.nih.gov/pubmed/20463309.

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.