Study of Vidaza in Higher-Risk Myelodysplastic Syndrome Patients Demonstrates Improved Overall SurvivalSUMMIT, N.J.--(BUSINESS WIRE)--Jun 3, 2008 - Celgene Corporation (Nasdaq:CELG) today announced updated results from a Phase III international clinical trial of VIDAZA in patients with myelodysplastic syndromes demonstrated an extension of overall survival compared to conventional care regimens (CCR). The data were presented at the 44th Annual American Society of Clinical Oncology meeting in Chicago, IL.
In the study, the two-year survival rate for patients treated with VIDAZA was almost doubled with 50.8 percent compared vs. 26.2 percent for CCR. This improved survival extended across all patient subgroups. The median overall survival for patients treated with VIDAZA in the study was 24.4 months compared to 15 months for CCR, demonstrating a survival benefit of 9.4 months.
The updated results at ASCO reported that the survival impact was particularly impressive in patients with the deletion 7q cytogenic abnormality, which is considered to be a patient sub-group with particularly poor prognostics.
It was also shown that the survival benefit of Vidaza is not attributable exclusively to those patients that experienced complete responses.
"The results of the AZA-001 trial are the first to show that achievement of CR is not an obligate endpoint to extend overall survival in higher risk MDS," said Alan F. List, MD, Chief, Division of Hematologic Malignancies of the Moffitt Cancer Center. "Azacitidine is a disease modifying agent that prolongs overall survival by effects that are distinct from its cytotoxic or anti-leukemia action alone."
In the study, the most commonly occurring major adverse events for patients receiving VIDAZA were thrombocytopenia (69.7%), neutropenia (65.7%) and anemia (51.4%).
The increasing volume of data, including this recent study continues to provide the foundation for clinical progress, including the ongoing review of VIDAZA by the EMEA.
In May 2004, VIDAZA became the first drug approved by the FDA for the treatment of patients with Myelodysplastic Syndromes (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 (CMMoL).
VIDAZA is believed to exert its antineoplastic effects by causing hypomethylation of DNA and direct 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 for IV administration in January 2007.
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. According to the American Cancer Society, 10,000 to 20,000 new cases of MDS are diagnosed each year in the United States, with median? survival rates ranging from approximately six months to six years for the different classifications of MDS. 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.
Celgene Corporation, based in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of novel 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.
David Gryska, 908-673-9059
Sr. Vice President and Chief Financial Officer
Brian P. Gill, 908-673-9530
Vice President, Corporate Communications
Posted: June 2008