BOUDRY, Switzerland – (December 8, 2008) – Celgene International Sàrl (NASDAQ: CELG) reports that an analysis presented today of a phase III international clinical trial (AZA-001) of higher-risk patients with myelodysplastic syndromes (MDS), demonstrated that continued treatment with VIDAZA (azacitidine) can improve patient responses. The data were presented at the 50th Annual American Society of Hematology meeting in San Francisco, CA.
In the study, 51 percent (91/179) of patients treated with VIDAZA responded with complete remission, partial remission or improvements in blood counts. The median number of cycles to first response was two; 87 percent of patients who responded achieved a first response by six cycles, and 91 percent of responders achieved a first response by nine cycles. For 52 percent of the responders, their first response was their best response; 48 percent had an increase in their response category after additional treatment cycles. Response was assessed by the International Working Group (IWG) 2000 criteria for MDS.
"While many patients achieve their best response to VIDAZA early on in the treatment, this analysis shows that additional cycles of treatment can further improve outcomes for almost half of patients," said Lewis R. Silverman, MD, Associate Professor of Medicine, Hematology and Medical Oncology at Mount Sinai, "Treatment with VIDAZA should be continued, even for those patients who do not respond initially, since continued therapy can lead to better patient responses."
Pierre Fenaux, M.D., of the University of Paris, added, "The landmark AZA-001 trial which was the first trial to show a significant survival benefit continues to show us how we can better treat patients with this life-threatening disease." Dr. Fenaux is the lead investigator on AZA-001. In the AZA-001 study, the most commonly occurring adverse reactions were thrombocytopenia (69.7%), neutropenia (65.7%) and anemia (51.4%).
About VIDAZA®
In May 2004, VIDAZA became the first drug approved by the FDA for the treatment of patients with MDS. The FDA approved VIDAZA, the first in a new class of drugs called demethylation agents, for treatment of all five MDS subtypes, which include both low-risk and high-risk patients. These subtypes include: refractory anemia (RA) or refractory anemia with ringed sideroblasts (RARS) if accompanied by neutropenia or thrombocytopenia or requiring transfusions; refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T), and chronic myelomonocytic leukemia (CMML). Additionally, in October 2008, VIDAZA received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use.
VIDAZA is believed to exert its antineoplastic effects by causing hypomethylation of DNA and cytotoxicity on abnormal hematopoietic cells in the bone marrow. The concentration of VIDAZA required for maximum inhibition of DNA methylation in vitro does not cause major suppression of DNA synthesis. Hypomethylation may restore normal function to genes that are critical for differentiation and proliferation. The cytotoxic effects of VIDAZA cause the death of rapidly dividing cells, including cancer cells that are no longer responsive to normal growth control mechanisms. Non-proliferating cells are relatively insensitive to VIDAZA. VIDAZA was approved by FDA for IV administration in January 2007 and the results of the VIDAZA Survival trial (AZA-001) were added in August 2008.
About Myelodysplastic Syndromes
Myelodysplastic syndromes (MDS) are a group of hematologic malignancies that affect approximately 300,000 people worldwide. Myelodysplastic syndromes occur when blood cells remain in an immature or "blast" stage within the bone marrow and never develop into mature cells capable of performing their necessary functions. Eventually, the bone marrow may be filled with blast cells suppressing normal cell development. MDS patients must often rely on blood transfusions to manage symptoms of anemia and fatigue and may develop life-threatening iron overload and/or toxicity from frequent transfusions, thus underscoring the critical need for new therapies targeting the cause of the condition rather than simply managing its symptoms.
About Celgene International Sàrl
Celgene International Sàrl, located in Boudry, in the Canton of Neuchâtel, Switzerland, is a wholly owned subsidiary and international headquarters of Celgene Corporation. Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation. For more information, please visit the Company's website at www.celgene.com.
This release contains certain forward-looking statements which involve known and unknown risks, delays, uncertainties and other factors not under the Company's control, which may cause actual results, performance or achievements of the Company to be materially different from the results, performance or other expectations implied by these forward-looking statements. These factors include results of current or pending research and development activities, actions by the FDA and other regulatory authorities, and those factors detailed in the Company's filings with the Securities and Exchange Commission such as Form 10-K, 10-Q and 8-K reports.