KRX-0401 is a novel, oral, anticancer agent that modulates Akt and several other important signal transduction pathways. Keryx is currently in the process of finalizing late-stage protocols for Perifosine in the treatment of Multiple Myeloma and Metastatic Colon Cancer.
Additionally, combination studies of perifosine with novel agents in patients with advanced brain tumors are expected to commence later this year. Such studies also to be funded by external grants.
STUDY DESIGN:
The single-center, open-label, Phase 1 study is entitled “Study of Single Agent Perifosine for Recurrent Pediatric Solid Tumors.” In this study, perifosine is being evaluated as a single-agent in pediatric patients with any solid tumor that has failed standard therapy. Patients up to 18 years of age with a performance status of greater than 40% are eligible for this study. The study has been designed as a dose escalation study to determine the maximum tolerated dose (MTD) of perifosine alone in recurrent/progressive pediatric tumors. A standard 3+3 dose escalation design will be employed with 3 to 6 patients at each dose level. All patients will receive perifosine at a loading dose on the first day, followed by a maintenance dose to start on day two until progression of disease. A minimum of 4 and a maximum of 24 patients will be required to complete the study.
KRX-0401 (perifosine) is in-licensed by Keryx from Aeterna Zentaris, Inc. in the United States, Canada and Mexico.
Approximately 12,400 children and adolescents younger than 20 years of age are diagnosed with some type of cancer each year. Among these, approximately 2,300 children and adolescents die of cancer each year making cancer the most common cause of disease-related mortality for children 1 to 19 years of age. (NCI SEER Data) There are 12 major types of childhood cancers, and of these leukemias, brain and other central nervous system tumors (ie, gliomas and medulloblastomas) account for over 50% of the new cases. The remainder of new cases include extracranial tumors such as neuroblastomas, Wilms tumors, and rhabdomyosarcomas. Although improvements in imaging equipment and treatments have resulted in an overall decline in mortality and increased survival, approximately half of all children with malignant solid tumors will experience relapse and, of these children, less than 10% will achieve long-term survival after standard multimodal therapy.
Keryx Biopharmaceuticals is focused on the acquisition, development and commercialization of medically important, novel pharmaceutical products for the treatment of life-threatening diseases, including renal disease and cancer. Keryx is developing Zerenex(TM)(ferric citrate), an oral, iron-based compound that has the capacity to bind to phosphate and form non-absorbable complexes. Zerenex has recently completed a Phase 2 clinical program as a treatment for hyperphosphatemia (elevated phosphate levels) in patients with end-stage renal disease. The Company is also developing KRX-0401 (perifosine), a novel, potentially first-in-class, oral anti-cancer agent that modulates Akt, a protein in the body associated with tumor survival and growth. KRX-0401 also modulates 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. The Company also actively engages in business development activities that include seeking strategic relationships for its product candidates, as well as evaluating compounds and companies for in-licensing or acquisition. Keryx is headquartered in New York City.
SOURCE Keryx Biopharmaceuticals, Inc.
CONTACT: Lauren Fischer, Director, Investor Relations, Keryx
Biopharmaceuticals, Inc., +1-212-531-5962, lfischer@keryx.com
Web site: http://www.keryx.com/
