NEW YORK, Dec. 13 /PRNewswire-FirstCall/ -- Keryx Biopharmaceuticals, Inc. announced today the initiation of a corporate-sponsored phase II clinical program to evaluate KRX-0401 (perifosine) as a treatment for rare sarcomas. This Phase II study will be conducted by the Sarcoma Alliance for Research through Collaboration (SARC) multi-center network, which includes nationally recognized sarcoma centers and investigators throughout the United States. Dr. Dejka M. Steinert, Assistant Professor in the Department of Sarcoma at MD Anderson Cancer Center in Houston, Texas will act as Principal Investigator for the study.
This clinical trial is entitled "A Phase II Trial of Perifosine in Patients with Chemo-Insensitive Sarcomas." In this Phase II study, the single agent activity of KRX-0401 is being evaluated in patients with chondrosarcoma, alveolar soft part sarcomas and extra-skeletal myxoid chondrosarcomas. Patients will be treated with KRX-0401 (100 mg oral daily) until disease progression. This study follows previous phase I and phase II trials of perifosine in patients with chemo-insensitive sarcoma that showed responding patients experienced very little toxicity and the duration of responses observed on both weekly and daily dosing schedules varied from 6 months to more than 18 months. Furthermore, some of the partial responses occurred in patients with sarcoma subtypes that have been traditionally unresponsive to conventional therapy.
Dr. Steinert stated, "Perifosine is a novel compound with a unique mechanism of action that in previous studies has demonstrated some activity in patients with sarcoma subtypes that have been traditionally unresponsive to conventional chemotherapy. We are eager to explore its potential in treating and hopefully benefiting patients with chemo-insensitive sarcomas."
Laurence Baker, DO, the Executive Director of the Sarcoma Alliance for Research through Collaboration (SARC), remarked, "SARC is pleased to participate in this evaluation, bringing together the strongest sarcoma clinical and research programs within the United States. We seek to improve the therapeutic options for patients with sarcomas and are unique in that adolescents, young and older adults can participate in our clinical trials. We appreciate the opportunity to partner with Keryx Biopharmaceuticals on this important clinical trial."
Craig Henderson, MD, President of Keryx Biopharmaceuticals, commented, "We've now seen objective responses in several tumors types, including sarcoma and renal cell carcinoma, and what appears to be a prolongation in the time to progression in many more. The responses in these rare sarcomas are of particular interest because they so rarely respond to chemotherapy. We are pleased to be working with SARC because of their access to and experience in conducting trials in this patient population."
KRX-0401 (perifosine) is in-licensed by Keryx from Aeterna Zentaris, Inc. in the United States, Canada and Mexico.
About KRX-0401 (Perifosine)
KRX-0401 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.
KRX-0401 has been shown to inhibit or otherwise modify signaling through a number of different signal transduction pathways including Akt, MAPK, and JNK. Akt isoforms have been found to be overexpressed in renal, breast, prostate, and pancreatic cancers. Elevated levels of pAkt 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.
The effects of KRX-0401 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.
To date, over 900 patients have been treated with KRX-0401 in trials conducted both in the US and Europe. Its safety profile is distinctly different from that of most cytotoxic agents. It does not cause myelosuppression (depression of the immune system) or alopecia (hair loss) like many currently available treatments for cancer. In phase I/II trials it 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. Responding patients, including stable disease, have been treated for months to almost 3 years, on both the daily and weekly schedule.
ABOUT SARCOMA ALLIANCE FOR RESEARCH THROUGH COLLABORATION (SARC)
Sarcoma Alliance for Research through Collaboration (SARC) is a leading advocate for sarcoma medical research and conduct of clinical trials relating to potential treatments for sarcoma. SARC has been instrumental in facilitating dialogue and collaboration among sarcoma researchers and clinicians, assisting in the development and dissemination of protocols for clinical trials and information relating to sarcoma research and the results of clinical trials, administering research grants and funding for clinical trials, and acting as a primary resource for those treating sarcoma.
ABOUT KERYX BIOPHARMACEUTICALS, INC.
Keryx Biopharmaceuticals, Inc. is focused on the acquisition, development and commercialization of novel pharmaceutical products for the treatment of life-threatening diseases, including diabetes and cancer. Keryx's lead compound under development is KRX-101 (sulodexide), a first-in-class, oral heparinoid compound for the treatment of diabetic nephropathy, a life- threatening kidney disease caused by diabetes. KRX-101 is in a pivotal Phase 3 and Phase 4 clinical program under a Special Protocol Assessment with the Food & Drug Administration. Additionally, Keryx is 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 2 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 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 Commissions. 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.
Ron Renaud, CFO, Keryx Biopharmaceuticals, Inc. 212-531-5965 rrenaud@keryx.com
Keryx Biopharmaceuticals, Inc.CONTACT: Ron Renaud, CFO, Keryx Biopharmaceuticals, Inc., +1-212-531-5965,rrenaud@keryx.com
Web site: http://www.keryx.com//
