Study of Vidaza in Higher-Risk Patients with Myelodysplastic Syndromes Reports Improved Overall Survival
COPENHAGEN, Denmark--(BUSINESS WIRE)--June 13, 2008--Celgene International Sarl (Nasdaq:CELG) today announced results from a sub-analysis of a previously reported large, randomized phase III study (AZA-001), which showed that treatment with VIDAZA (azacitidine) prolonged overall survival for patients with high-risk myelodysplastic syndromes (MDS) when compared to conventional care regimens (CCR). This analysis evaluated a sub-group of patients (n=94) who were pre-selected to receive low dose Ara-C, a chemotherapy used in the treatment of MDS and acute myeloid leukemia (AML). The results confirm that the significant survival benefit originally reported with VIDAZA in the overall population was also seen when VIDAZA was directly compared to the active comparator arm of low dose Ara-C. The data were presented at the 13th European Hematology Association (EHA) Congress in Copenhagen, Denmark.
In the sub-analysis, the median overall survival for patients treated with VIDAZA was significantly longer (24.4 months compared to 15.3 months (hazard ratio 0.36) (95% Cl: 0.20-0.65 (p=0.0006)) compared to patients treated with low-dose Ara-C, reducing the risk of death by 64 percent. This improved survival with VIDAZA was supported by significant improvements in hematologic response, and improvement in transfusion independence.
"VIDAZA offers patients significantly improved overall survival with less myelosuppression than occurs with low dose Ara-C, and should be considered for first-line therapy," said Professor Pierre Fenaux, hematologist, University of Paris, and lead investigator of the trial. "Treatment with VIDAZA offers patients transfusion independence and is an innovative advancement over current standard treatments for MDS, reinforcing the role VIDAZA plays in treatment of patients."
A similar rate of thrombocytopenia was seen in each group. Higher rates of severe anemia were seen in the low-dose Ara-C group. Additionally, low-dose Ara-C provided no survival benefit when compared with best supportive care.
In the AZA-001 study recently presented at ASCO, the two-year survival rate for patients treated with VIDAZA was almost doubled with 50.8 percent compared vs. 26.2 percent for conventional care regimens (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.
In the AZA-001 study, the most commonly occurring major adverse events for patients receiving VIDAZA were thrombocytopenia (69.7%), neutropenia (65.7%) and anemia (51.4%).
In May 2004, VIDAZA became the first drug approved in the United States by the FDA for the treatment of patients with Myelodysplastic Syndromes (MDS). VIDAZA was approved for IV administration in January 2007. 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). The VIDAZA marketing authorization as a potential treatment for patients with higher -risk MDS is currently under review by the EMEA.
VIDZA is an epigenetic agent, which may restore normal expression to genes critical for cell 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 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.
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.
About Celgene International Sarl
Celgene International Sarl, located in Boudry, Switzerland, is a wholly owned subsidiary and international headquarters of Celgene Corporation. Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global pharmaceutical 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.
David Gryska, Sr. Vice President and
Chief Financial Officer, 908-673-9059
Brian P. Gill, Vice President,
Corporate Communications, 908-673-9530
Posted: June 2008