MMRF PRESS RELEASES
The Multiple Myeloma Research Consortium (MMRC) and Keryx Biopharmaceuticals Commence Multi-Center Combination Study for the Treatment of Multiple Myeloma
Phase I Study to Explore the Safety and Efficacy of KRX-0401 (Perifosine) in Combination with Revlimid (Lenalidomide) and Dexamethasone in Patients with Relapsed or Refractory Multiple Myeloma
New York, NY & Norwalk, CT — December 28, 2006
The Multiple Myeloma Research Consortium (MMRC) and Keryx Biopharmaceuticals (Nasdaq: KERX) today announced the initiation of a multi-center Phase I clinical program to explore the convenient all-oral combination of KRX-0401 (perifosine), Revlimid (lenalidomide, Celgene Corporation) and dexamethasone for the treatment of relapsed or refractory multiple myeloma. Under the lead of Andrzej Jakubowiak, M.D., Ph.D., Director, Multiple Myeloma Center of University of Michigan Comprehensive Cancer Center (Ann Arbor, MI), the study will also be conducted at four other of the MMRC's 13 world-renowned member academic institutions.
Entitled "An Open-Label Phase I Study of the Safety of Perifosine in Combination with Lenalidomide and Dexamethasone for Patients with Relapsed or Refractory Multiple Myeloma," the clinical program represents the first time this oral triplet is being explored in the clinical setting for this incurable blood cancer. The study is being conducted to determine the safety and efficacy of the drug combination in the treatment of these patients, as well as the highest dose at which KRX-0401 can be safely administered to multiple myeloma patients when combined with Revlimid and dexamethasone.
The clinical program is being conducted entirely within the MMRC and is one of the first industry-sponsored clinical programs to open with the organization's assistance. "We are so pleased to be collaborating with Keryx Biopharmaceuticals on this groundbreaking clinical trial and believe that this novel oral combination may represent an important new option for multiple myeloma patients," said Kathy Giusti, Founder and Chief Executive Officer of the MMRC, as well as a myeloma patient. "Through this innovative program and the others we support, our organization continues to facilitate unprecedented collaborations between industry and academia that are expediting meaningful new research and drug development opportunities."
Data from pre-clinical and clinical research studies suggest that KRX-0401 has activity in patients with multiple myeloma. In addition to the clinical trial announced today, KRX-0401 is also currently being evaluated in an ongoing multi-center Phase II study as a single agent and in combination with dexamethasone as a treatment for relapsed or relapsed/refractory multiple myeloma and in an ongoing multi-center Phase I/II clinical study in combination with Velcade (bortezomib) for injection for the treatment for multiple myeloma.
Revlimid is currently approved for the treatment of certain types of myelodysplastic syndrome (another form of cancer affecting the blood) and in combination with dexamethasone for the treatment of multiple myeloma patients who have received at least one prior therapy. Dexamethasone is a corticosteroid commonly used, either alone, or in combination with other drugs, to treat multiple myeloma.
"Keryx's collaboration with the MMRC will accelerate the development of KRX-0401 as an emerging multiple myeloma therapy," said I. Craig Henderson, MD, President, Keryx Biopharmaceuticals, Inc. "We are excited to explore this novel combination of three oral agents, and in addition are appreciative of the MMRC's ongoing commitment to this trial. Hopefully the success of this collaboration will set a precedent that proves valuable to others working in the field."
KRX-0401 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. KRX-0401 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 the Multiple Myeloma Research Consortium (MMRC)
The Multiple Myeloma Research Consortium (MMRC) is a 509a3 non-profit organization that integrates leading academic institutions to accelerate drug development in multiple myeloma. It is comprised of the MMRC and 13 member institutions: City of Hope Cancer Center, Dana-Farber Cancer Institute, Emory University's Winship Cancer Institute, Hackensack University Medical Center, H. Lee Moffitt Cancer Center & Research Institute, Mayo Clinic, Ohio State University, Roswell Park Cancer Institute, St. Vincent's Comprehensive Cancer Center of Saint Vincent Catholic Medical Centers of New York, University Health Network (Princess Margaret Hospital), University of Chicago, University of Michigan, and Washington University.
The MMRC was founded in 2004 by Kathy Giusti, a myeloma patient, and with the help of the scientific community, as an optimal research model to rapidly address critical challenges in accelerating drug development and explore opportunities in the most promising areas of myeloma research-genomics, compound validation, and clinical trials. The MMRC is the only consortium to join academic institutions through membership agreements, customized IT systems, and an integrated tissue bank. For more information, please visit www.themmrc.org.
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 Sulonex™ (sulodexide), 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 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.
Vicki Vlastaris (MMRF)