Phase 2 Study of KRX-0401 (Perifosine) in Relapsed or Refractory Chronic Lymphocytic Leukemia / Small Lymphocytic Lymphoma Open

Phase 2 Study of KRX-0401 (Perifosine) in Relapsed or Refractory Chronic Lymphocytic Leukemia / Small Lymphocytic Lymphoma Open for Enrollment at Duke Comprehensive Cancer Center

NEW YORK, Oct. 8 /PRNewswire-FirstCall/ -- Keryx Biopharmaceuticals, Inc. (Nasdaq: KERX) announced today the initiation of a Phase 2 clinical study to evaluate KRX-0401 (perifosine) as a single agent treatment for relapsed or refractory Chronic Lymphocytic Leukemia (CLL) and Small Lymphocytic Lymphoma (SLL). This Phase 2 study was designed by Daphne Friedman, MD, Instructor and Principal Investigator, in coordination with J. Brice Weinberg, Professor, and Mark Lanasa, Assistant Professor, Divisions of Medical Oncology and Hematology, Duke University Medical Center, and is currently open for enrollment at Duke University. This study is being externally funded.

KRX-0401 (perifosine) is a novel, potentially first-in-class, oral anti-cancer agent that inhibits the phosphoinositide 3-kinase (PI3K)/Akt pathway and several other key signal transduction pathways in both hematologic and solid tumor cancers. Keryx is preparing to initiate a Phase 3 trial by year-end, under Special Protocol Assessment (SPA), for patients with advanced multiple myeloma.

Ron Bentsur, Chief Executive Officer of Keryx Biopharmaceuticals, commented, "We're very excited to explore the potential of perifosine in this advanced CLL/SLL setting, based on the pre-clinical work completed by Dr. Weinberg." Mr. Bentsur continued, "We are extremely grateful to the Duke Comprehensive Cancer Center for conducting this study, and we look forward to working with Drs. Friedman, Weinberg, Lanasa and their team of renowned oncologists on this Phase 2 study."

STUDY RATIONALE:

CLL is characterized by the accumulation of circulating B cells which are resistant to apoptosis. CLL has been found to have aberrant signaling in several pathways including NF-kappaB, Akt/PI3K, and JNK/STAT pathways. Published data has demonstrated that Akt is important in promoting CLL survival and viability, as seen in in vitro experiments where blocking its activity results in apoptosis. The effect of perifosine on CLL cells was first tested in the laboratory of Dr. Brice Weinberg, which demonstrated the in vitro cytotoxicity of perifosine on primary CLL cells. This data proving that perifosine is an active agent against primary CLL cells, coupled with its demonstrated safety profile in the clinical setting, provided the rationale that perifosine should further be evaluated as a single agent in an advanced CLL/SLL clinical setting.

STUDY DESIGN:

The single-center, open-label, study is entitled, "Phase 2 Trial of Perifosine in Patients with Relapsed or Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma," and will enroll approximately 30 patients. In this study, perifosine will be given orally at a dose of 50 mg twice daily, for a total of six 28-day cycles. The patients will be formally restaged upon completion of the trial. Overall Response Rate is the primary endpoint with overall survival, progression-free survival and safety as secondary endpoints. Correlative studies will also be conducted and evaluated as a secondary endpoint.

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

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 the phosphoinositide 3-kinase (PI3K)/Akt pathway, a key signaling cascade that has been shown to induce cell growth and cell transformation. 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 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, with a Phase 3 in multiple myeloma, under Special Protocol Assessment (SPA), pending commencement by year-end. 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. Zerenex has recently completed a Phase 2 clinical program as a treatment for hyperphosphatemia (elevated phosphate levels) in patients with end-stage renal disease, and Keryx is in the process of finalizing the U.S. Phase 3 program for Zerenex in consultation 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 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 complete clinical trials for KRX-0401; our ability 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 http://www.keryx.com. The information in our website is not incorporated by reference into this press release and is included as an inactive textual reference only.

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