Phase 1 study data of Keryx' perifosine for recurrent pediatric solid tumors presented at ASCO

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.