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Perifosine Phase 2 Study Results Presented at EORTC-NCI-AACR Conference

NEW YORK, Sept. 30 /PRNewswire-FirstCall/ -- Keryx Biopharmaceuticals announced today that Phase II data presented at the 16th Annual EORTC-NCI-AACR symposium on "Molecular Targets and Cancer Therapeutics" demonstrated the tolerability and potential efficacy of KRX-0401 (perifosine) in the treatment of patients with advanced soft tissue sarcoma. This study was conducted by the National Cancer Institute (NCI) pursuant to a Collaborative Research and Development Agreement (CRADA) between Keryx and the NCI.

In this single-agent Phase II multi-center study of KRX-0401, 23 patients with advanced soft-tissue sarcoma received a loading dose of 150mg, every 6 hours starting on day 1, followed by 100mg daily thereafter. The patients enrolled in this study had prior treatment, including 1-2 chemotherapy regimens, surgery and/or radiotherapy. Nineteen patients received more than one course of treatment. There was one confirmed partial response lasting more than 5 months and 2 patients that remained progression free at 6 months. This follows the evidence of single agent activity of the drug in refractory sarcoma patients as seen in the earlier Phase I program.

KRX-0401 was also shown to be well tolerated at the doses used. All 23 patients were evaluable for toxicity and notable toxicities seen included Grade 4 ileus (1 patient), Grade 3 toxicity (6 patients) including fatigue (2 pts) and 1 patient each of anemia, infection, muscle weakness, pain, rash, anorexia, dehydration, and diarrhea. Of the grade 3 and 4 toxicities seen, it is unclear which ones were related to the disease or the drug. There was no Grade 3 or 4 nausea or vomiting seen in this trial.

The authors concluded in summary that the regimen was tolerable and that the preliminary observation of another responder in soft-tissue sarcoma, such as was seen in the Phase I program, raises the question of whether specific histologies or tumor characteristics might predict a more sensitive sub-population of soft tissue sarcoma patients.

I. Craig Henderson, M.D., President of Keryx Biopharmaceuticals, commented, "The level of activity seen in this study, combined with the previous Phase I single-agent experience where 2 partial responses were reported out of 10 sarcoma patients enrolled, provides us with very strong evidence for the potential single-agent activity of this drug in soft tissue sarcoma, particularly in one or more subtypes." Dr. Henderson added, "Soft tissue sarcoma is a very aggressive disease characterized by many heterogeneous subtypes or histologies, for which few, if any, drugs work across multiple subtypes. The overall response rate of sarcomas to the most effective front-line chemotherapy treatments is about 10-20%, and some subtypes are totally unresponsive to any form of chemotherapy."

Dr. Henderson also stated, "We believe that our correlative science program will help us to potentially identify at least one subtype of soft-tissue sarcoma for which we may be able to conduct a single agent registration trial with KRX-0401 in the near future."

Michael S. Weiss, Chairman and Chief Executive Officer of Keryx, added, "We are very pleased with the results of this study. These data confirm KRX-0401's potential anti-cancer activity as a single agent. In addition, we believe that KRX-0401, as an oral AKT inhibitor, is ideally suited for combination therapy with currently available anti-cancer treatments such as chemotherapy, radiation therapy, EGFR inhibitors and endocrine therapy. Our goal is that our recently-started program combining KRX-0401 with other anti-cancer therapies will lead to additional approaches to regulatory approval."

To access the abstract, entitled "Tolerability and limited activity of perifosine in patients with advanced soft tissue sarcoma (STS): a multi-center phase 2 consortium (P2C) study," please click here: http://tinyurl.com/3mdlh. KRX-0401 (Perifosine) is in-licensed by Keryx from Aeterna Zentaris, Inc. (TSX: AEZ, Nasdaq: AEZS), which holds ex-North American rights to the drug.

ABOUT KRX-0401 (Perifosine)
KRX-0401 (Perifosine), a novel, first-in-class, oral AKT-inhibitor, is believed to be the only AKT inhibitor in clinical development primarily for the treatment of cancer. Activation of the AKT pathway is believed to play an important role in cell survival and cell proliferation. KRX-0401 has demonstrated single agent anti-tumor activity in Phase I studies and is currently being studied as a single agent for the treatment of refractory non-small cell lung cancer in a corporate-sponsored Phase II program. The Company is also investigating KRX- 0401 in combination with chemotherapy as a treatment for several tumor types, including non-small cell lung cancer and breast cancer. In addition to the corporate-sponsored clinical program, KRX-0401 is currently in 9 Phase II single agent clinical trials in 6 tumor types, including, breast, prostate, melanoma, pancreatic, sarcoma and head and neck cancer, being conducted by the NCI under a CRADA arrangement.

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