Healthcare Industry News:  lenalidomide 

Biopharmaceuticals Oncology

 News Release - June 29, 2007

Keryx Biopharmaceuticals, Inc. Announces Positive Phase I and Phase II Data on KRX-0401 in Patients with Relapsed/Refractory Multiple Myeloma and Waldenstrom's Macroglobulinemia

Data presented at the 11th International Myeloma Workshop held in Kos, Greece demonstrates clinical activity of perifosine in combination with bortezomib, dexamethasone and lenalidomide plus dexamethasone, in patients with relapsed/refractory multiple myeloma, and as a single agent in patients with relapsed/refractory Waldenstrom's macroglobulinemia

NEW YORK, June 29 (HSMN NewsFeed) -- Keryx Biopharmaceuticals, Inc. (Nasdaq: KERX ) today announced that 4 posters were presented at the 11th International Myeloma Workshop demonstrating the clinical activity of KRX-0401 (perifosine) in patients with advanced multiple myeloma and Waldenstrom's macroglobulinemia.

Perifosine is currently being evaluated in 3 ongoing, multi-center clinical studies for patients with relapsed or relapsed/refractory multiple myeloma, including in combination with dexamethasone, in combination with Velcade® (bortezomib) and in combination with Revlimid® (lenalidomide) plus dexamethasone. Perifosine is also being evaluated in an ongoing phase II single agent study in patients with relapsed and/or refractory Waldenstrom's macroglobulinemia. Data from each of these ongoing studies was presented during the conference and demonstrates perifosine's activity, both as a single agent and as part of a combination, in patients with multiple myeloma and Waldenstrom's macroglobulinemia.

Perifosine in the treatment of Patients with Relapsed/Refractory Multiple Myeloma:

In a poster entitled "A Multicenter Phase I/II Trial of Perifosine (KRX- 0401) + Bortezomib in Relapsed and Refractory Multiple Myeloma in Patients Previously Treated with Bortezomib: Preliminary Results", Dr. Paul Richardson, Clinical Director of the Jerome Lipper Multiple Myeloma Center at the Dana- Farber Cancer Institute in Boston, MA reported an overall response rate (partial response (PR) + minimal response (MR)) of 31%. In 16 evaluable patients treated with perifosine plus bortezomib, there were 2 PR's and 1 MR. Two additional MR's were observed on this combination after dexamethasone was added; both of these patients were previously refractory to the combination of bortezomib + dexamethasone without perifosine. Eleven patients remain on study and the phase II portion of the study is now open and enrolling.

A second poster presented by Dr. Richardson, entitled " Perifosine (KRX- 0401) + Low Dose Dexamethasone is Active in Patients with Relapsed and Refractory Multiple Myeloma (MM): Perifosine MM Investigator Group Phase II Multicenter Study Update" demonstrated that 78% of patients treated with perifosine plus low dose dexamethasone had at least stable disease (SD), including 26% that had a PR or MR. Patients continue on study now ranging from 6 months up through 1 year.

Dr. Andrzej Jakubowiak, Director of the myeloma program at the University of Michigan Cancer Center, presented a poster entitled "A Multicenter Phase I Trial of Perifosine (KRX-0401) in Combination with lenalidomide and Dexamethasone in Patients with Relapsed or Refractory Multiple Myeloma: Preliminary Results. Multiple Myeloma Research Consortium (MMRC) Trial", demonstrating early encouraging results, with 50% of patients achieving a partial response or better.

Commenting on the data, Dr. Richardson stated, "Perifosine's potential in combination with bortezomib, dexamethasone and other novel therapies to overcome drug-resistant multiple myeloma is promising. Our team of investigators, including top centers across the country and now incorporating the considerable resources of the MMRC for one of the trials, is committed to completing enrollment in our studies and evaluating future clinical strategies to improve patient outcome."

Perifosine in the treatment of Patients with Relapsed/Refractory Waldenstrom's Macroglobulinemia:

A poster entitled "A Phase II Trial of Perifosine (KRX-0401) in Relapsed and/or Refractory Waldenstrom's Macroglobulinemia: Preliminary Results" demonstrated single agent activity of perifosine with 36% of patients having a PR or MR. Dr. Irene Ghobrial of the Dana-Farber Cancer Institute, who presented the poster, stated, "We are excited at the efficacy and safety results with perifosine in a patient population for which there is no FDA approved therapy. We look forward to reporting updated results at future meetings."

I. Craig Henderson, MD, President of Keryx Biopharmaceuticals, commented "We are very enthusiastic about the potential of perifosine in the treatment of multiple myeloma and Waldenstrom's macroglobulinemia and the prospects of using perifosine to improve upon the current care for these patients."

In addition to the clinical posters, three pre-clinical posters were presented, as follows:

Perifosine, an Oral Bioactive Novel Akt Inhibitor, Induces In Vitro and
In Vivo Anti-tumor Activity in Waldenstrom's Macroglobulinemia
Lead Investigator: Xavier Leleu, MD, Dana-Farber Cancer Institute,
Boston, MA

The Combination of Perifosine with Bortezomib and Rituximab Provides
Synergistic Anti-tumor Activity in Waldenstrom's Macroglobulinemia
Lead Investigator: Xavier Leleu, MD, Dana-Farber Cancer Institute,
Boston, MA

Combination of the AKT Inhibitor Perifosine with the HSP90 Inhibitor 17-
(Dimethylaminoethylamino)-17-Demethoxygeldanamycin (17-DMAG) Has
Synergistic Activity in tumor cell and its microenvironment in Multiple
Myeloma (MM)
Lead Investigator: Alissa Huston, MD, University of Pittsburgh
Cancer Center

Copies of all posters can be obtained from Keryx Biopharmaceuticals, Inc.

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

ABOUT KRX-0401 (Perifosine)

KRX-0401 (perifosine) is a novel, first-in-class, oral anticancer agent that modulates AKT and a number of other key signal transduction pathways, including the MAPK and JNK pathways all of which are pathways associated with programmed cell death, cell growth, cell differentiation and cell survival.

High levels of activated Akt (pAkt) are seen frequently in many types of cancer and have been correlated with poor prognosis in patients with soft- tissue sarcoma, gastric, hepatocellular, endometrial, prostate, renal cell, head and neck cancers and hematological malignancies, as well as glioblastoma. The majority of tumors expressing high levels of pAkt were high-grade, advanced stage or had other features associated with poor prognosis. High pAkt is often seen in tumors that are resistant to conventional cancer treatments, including radiotherapy, chemotherapy, endocrine therapy, and especially therapy with some of the newer biologicals.

The effects of perifosine on Akt are of particular interest because of 1) the importance of this pathway in the development of most cancers; 2) the evidence that it is often activated in tumors that are resistant to other forms of anticancer therapy; and 3) and the difficulty encountered thus far in the discovery of drugs that will inhibit this pathway without causing excessive toxicity. It is plausible that perifosine may be useful in combination to reduce the resistance to other cancer treatments.

To date, over 1,500 patients have been treated with perifosine in trials conducted both in the United States and Europe. Its safety profile is distinctly different from that of most cytotoxic agents. It does not appear to cause myelosuppression (depression of the immune system that may lead to life threatening infections), thrombocytopenia (a decrease in platelets that may result in bleeding), skin rash, flu-like symptoms or alopecia (hair loss); all of these toxicities occur frequently with many of the currently available treatments for cancer. The main side effects of perifosine are nausea, vomiting, diarrhea and fatigue, but these are either mild or non-existent in doses that are known to induce tumor regression.

In Phase I/II trials, perifosine has induced tumor regressions and/or caused disease stabilization in a variety of tumor types. Perifosine has shown single agent partial responses or long term disease stabilizations in solid tumors including, renal, hepatocellular, sarcoma and prostate cancer. There is also evidence of activity in hematological malignancies, especially multiple myeloma. Responding patients, including those with stable disease, have been treated for months to more than three years.


Keryx Biopharmaceuticals, Inc. is focused on the acquisition, development and commercialization of medically important, novel pharmaceutical products for the treatment of life-threatening diseases, including diabetes and cancer. Keryx's lead compound under development is Sulonex(TM) (sulodexide oral gelcap), previously referred to as KRX-101, a first-in-class, oral heparinoid compound for the treatment of diabetic nephropathy, a life-threatening kidney disease caused by diabetes. Sulonex is in a pivotal Phase III and Phase IV clinical program under a Special Protocol Assessment with the Food & Drug Administration. Additionally, Keryx is developing Zerenex(TM), an oral, inorganic, iron-based compound that has the capacity to bind phosphate and form non-absorbable complexes. Zerenex is currently in Phase II clinical development for the treatment of hyperphosphatemia (elevated serum phosphorous levels) in patients with end- stage renal disease. Keryx is also developing clinical-stage oncology compounds, including KRX-0401, a novel, first-in- class, oral modulator of Akt, a pathway associated with tumor survival and growth, and other important signal transduction pathways. KRX-0401 is currently in Phase II clinical development for multiple tumor types. Keryx also has an active in-licensing and acquisition program designed to identify and acquire additional drug candidates. Keryx is headquartered in New York City.

Cautionary Statement

Some of the statements included in this press release, particularly those anticipating future the financial performance and clinical and business prospects for KRX-0401, 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 the Phase 1 and Phase 2 clinical trials for KRX-0401; we may not be able 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.

Source: Keryx Biopharmaceuticals

Issuer of this News Release is solely responsible for its content.
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