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Updated Single Agent Phase 2 Data of KRX-0401 (Perifosine) in the Treatment of Advanced Metastatic Renal Cell Carcinoma Reported at the Eighth International Kidney Cancer Symposium

KRX-0401 (Perifosine) Continues to Demonstrate Impressive Efficacy, Safety and Tolerability in Metastatic Renal Cell Carcinoma Patients who Progressed after Failing Treatment with both a VEGF Receptor Inhibitor and an mTOR Inhibitor

NEW YORK, Sept. 29 /PRNewswire-FirstCall/ -- Keryx Biopharmaceuticals, Inc. (NASDAQ:KERX) today announced updated clinical results from a Phase 2 study of KRX-0401 (perifosine), the Company's PI3K/Akt pathway inhibitor for cancer, as a single-agent treatment for advanced metastatic renal cell carcinoma (RCC). Dr. Thomas E. Hutson, Director of the Genitourinary Oncology Program at Baylor-Sammons Cancer Center in Dallas, Texas, presented the data on Saturday, September 26th, in an oral presentation featured at the Eighth International Kidney Cancer Symposium held in Chicago.

Dr. Hutson's presentation, entitled "Phase 2 study of perifosine in patients with metastatic renal cell carcinoma progressing after prior therapy with both a VEGF Receptor Inhibitor and an mTOR inhibitor," included results from a subgroup of patients who failed both a VEGF receptor inhibitor (sunitinib or sorafenib) and an mTOR inhibitor (temsirolimus or everolimus). Evaluable patients (n=16) were defined as those who had greater than 7 days of treatment (2 additional patients withdrew consent within 7 days). Patients received 100 mg of perifosine daily until progression or unacceptable toxicity. The primary endpoint of this study was clinical benefit, defined as response rate (CR / PR by RECIST) or percent of patients progression-free for at least 3 months. Median progression-free survival (PFS) and overall survival were also analyzed for efficacy. Safety was a secondary endpoint. Perifosine was well-tolerated with the most common adverse events being gastrointestinal discomfort and fatigue. Best response to single agent perifosine was as follows:

           PR    SD > 12 wks  PD < 12 wks                        Overall
    N     N (%)      N (%)       N (%)         Median PFS        Survival
  ----   ------  -----------  -----------  ------------------- -------------
   16     1 (6%)     7 (44%)       8 (50%)    16 wks [95% CI
                                               (11.7, 28)]      Not Reached
  ----   ------  -----------  -----------  ------------------- (14/16 alive)
         Median PFS for patients SD or >      33 wks [95% CI   at 22+ months
                                                (19, NR)]
  ---------------------------------------  ------------------- -------------

  PR: Partial response;  SD: Stable disease;  PD: Progressive disease

This multi-center Phase 2 study was led by both Dr. Hutson and Dr. Nicholas Vogelzang, Chair and Medical Director of Developmental Therapeutics, Comprehensive Cancer Centers of Nevada / US Oncology Research. Drs. Hutson and Vogelzang also serve as co-chairs of the Genitourinary Committee for US Oncology Research. Data from this study was first presented by Dr. Vogelzang at the American Society of Clinical Oncology (ASCO) annual meeting in May 2009.

Commenting on the data, Dr. Hutson stated, "Perifosine continues to demonstrate impressive single agent activity and safety in an advanced group of RCC patients, with 50% of patients progression-free for a median of 8 months. Given the convenient oral schedule, with a manageable safety profile and demonstrated efficacy, future studies with perifosine in the 2nd or 3rd line setting (single agent or in combination with an mTOR inhibitor) should be considered."

Ron Bentsur, Chief Executive Officer of Keryx Biopharmaceuticals, remarked, "We continue to be encouraged by the single agent activity of perifosine in the treatment of advanced renal cell carcinoma. While preliminary, perifosine's response and PFS data presented appear equivalent to that of other agents for advanced RCC, which were approved in a less heavily pre-treated patient population. We look forward to evaluating future perifosine trial designs with Dr. Hutson, Dr. Vogelzang and other RCC experts."

KRX-0401 (perifosine) is in-licensed by Keryx from Aeterna Zentaris, Inc. (Nasdaq: AEZS; TSX: AEZ) in the United States, Canada and Mexico.

About Renal Cell Carcinoma (RCC)

Of all kidney tumors, 85% are classified as renal cell carcinoma (RCC), and of all patients with RCC, 25% present with advanced disease. Advanced RCC is resistant to such standard treatments as radiation therapy and chemotherapy, and the initial treatment for most patients is surgical removal of the kidney. If the cancer is confined to the kidney, the five-year survival rate is 60 to 70 percent; but the survival rate is considerably lower after the cancer has metastasized to other parts of the body. Even with the recent approval of several biological therapies for the treatment of advanced metastatic RCC (VEGF inhibitors, sunitinib and sorafenib, and mTOR inhibitors, everolimus and temsirolimus), the National Cancer Institute reports a rising incidence of RCC with incidence and mortality rates more than twice as high in men as in women. In 2009, an estimated 49,000 new cases of RCC and 11,000 deaths attributable to RCC are expected in the United States.

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 The information in our website is not incorporated by reference into this press release and is included as an inactive textual reference only.

  Lauren Fischer
  Director, Investor Relations
  Keryx Biopharmaceuticals, Inc.
  Tel: 212.531.5962

Source: Keryx Biopharmaceuticals, Inc.

CONTACT: Lauren Fischer, Director, Investor Relations, Keryx Biopharmaceuticals, Inc., +1-212-531-5962,

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Posted: September 2009