Phase 2 Data Presented at the 52nd Annual Meeting of the American Society of Hematology Demonstrates Promising Efficacy of KRX-0401 (Perifosine) in the Treatment of Advanced Leukemia, Lymphoma and Multiple Myeloma

NEW YORK, Dec. 6, 2010 /PRNewswire-FirstCall/ -- Keryx Biopharmaceuticals, Inc. (Nasdaq: KERX) today announced data presented for the first time at the 52nd Annual Meeting of the American Society of Hematology showing promising clinical activity, safety and tolerability of perifosine in patients with advanced chronic lymphocytic leukemia (CLL) and Hodgkin's lymphoma (HL).  KRX-0401 (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. Key highlights from the two Phase 2 poster presentations are as follows:

Abstract # 2861: "Clinical Activity and Safety of the Combined Therapy with the AKT Inhibitor Perifosine and the Multikinase Inhibitor Sorafenib In Heavily Pretreated Patients with Relapsed/Refractory Lymphomas: Preliminary Results of a Phase II Trial"  

Study Background:  In this Phase 2 study, 26 patients were enrolled with advanced lymphoma (6 NHL, 4 CLL, 1 Waldenstroms and 15 Hodgkin's lymphoma).  73% of patients were previously refractory to their prior therapy, with 85% of patients having had 4 or more prior therapies.  Perifosine (50 mg BID) was started as a single agent for 28 days; After 28 days, patients achieving partial response (PR) or better were continued on single agent perifosine.  Patients achieving less than a PR were given the combination of perifosine (50 mg BID) plus sorafenib (Nexavar®) at 400 mg BID.

Results:  All of the 4 CLL patients in the study achieved a partial response on single-agent perifosine within one month of treatment and remained on perifosine single agent.  Response durations for each of the 4 patients were 4, 8, 9+ and 12 months.  The remaining 22 patients were administered the combination with sorafenib, where 5 of the 15 (33%) Hodgkin's lymphoma patients achieved a partial response with a median response duration of 9 months.  An additional 6 patients receiving the combination (40%) achieved stable disease.  The combination was well tolerated with no unexpected safety events.

The investigators concluded that perifosine in combination with sorafenib has significant anti-lymphoma activity in relapsed/refractory HL, and that perifosine as a single agent induced prolonged responses in high-risk, heavily pretreated CLL patients.

Abstract # 1842:  "Pre-Clinical and Interim Results of a Phase II Trial of Perifosine In Patients with Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL)"

Study Background:  In this Phase 2 study, 12 patients with advanced CLL began treatment with single agent perifosine at 50 mg BID. Patients on study were heavily pre-treated having had a median of 4 prior lines of therapy with 75% of patients classified as Rai stage IV.

Results:  1 patient achieved a partial response (5 months on treatment) and 5 additional patients achieved stable disease (median duration of 4.25 months), for an overall 50% clinical benefit rate (PR + SD).  Perifosine was well tolerated with minimal dose modifications.

Abstract #3064: "Final Phase I Results of Perifosine In Combination with Lenalidomide and Dexamethasone In Patients with Relapsed or Refractory Multiple Myeloma (MM)"

Additionally, the final dataset from the Phase 1 study of perifosine + lenalidomide (Revlimid®) + dexamethasone was also presented during the ASH meeting.  The final data showed a 73% objective response rate (minimal response or better) with a 50% PR or better, a median Progression-Free Survival of 10.8 months, and a median duration for Overall Survival of 30.6 months.  The myeloma investigators concluded that perifosine in combination with lenalidomide + dexamethasone was well tolerated even at the highest doses used, and demonstrated encouraging clinical activity and survival.

Ron Bentsur, CEO of Keryx, commented, "The data presented at the ASH meeting further demonstrate perifosine's potential beyond the ongoing Phase 3 registration trials in metastatic colorectal cancer and multiple myeloma. It is encouraging to see perifosine's activity as a single agent and in combination with sorafenib in heavily pre-treated, advanced CLL and HL patients, respectively. Moreover, the perifosine-Revlimid® (+ dexamethasone) combination data in multiple myeloma also presented at ASH indicate that the combination is well tolerated demonstrating promising clinical activity."

Perifosine is currently in Phase 3 clinical trials for refractory advanced colorectal cancer and multiple myeloma.  Both of these Phase 3 programs are being conducted under Special Protocol Assessment (SPA) agreements with the FDA, and with Fast Track designations obtained for both indications. 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 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 studies is being conducted under a Special Protocol Assessment (SPA) agreement with the FDA. Keryx is also developing Zerenex (ferric citrate), an oral, ferric 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 (perifosine); 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 http://www.keryx.com. The information found on our website and the ASH website is not incorporated by reference into this press release and is included for reference purposes only.

SOURCE Keryx Biopharmaceuticals, Inc.