Celgene International Sàrl (Nasdaq:CELG) today announced the publication in the journalThe Lancet Oncology of the results from AZA-001, the largest international, randomized, Phase III study ever conducted in patients with higher-risk myelodysplastic syndromes (MDS). The data indicate that VIDAZA (azacitidine) demonstrated a significant extension of overall survival compared to conventional care regimens (CCR) for patients with Intermediate-2 and high-risk MDS and AML with 20 to 30 percent bone marrow blasts.

The median overall survival for patients treated with VIDAZA (n=179) was 24.5 months compared to 15 months for those receiving CCR treatment (n=179), an improvement of 9.5 months (p=0.0001). CCR includes best supportive care, low-dose ARA-C, and standard chemotherapy. There was a 42 percent reduction in the risk of death (0.58 hazard ratio, 95% CI). The two-year survival rate was nearly doubled at 50.8 percent for patients receiving VIDAZA versus 26.2 percent for patients receiving CCR (p=0.0001). The extension of survival was seen across the relevant patient subgroups including those greater than 65 years of age and those with poor cytogenetics, a poor prognostic factor. In addition, 34 percent of patients with AML as classified by the World Health Organization saw a survival benefit.

In addition, 45 percent of patients achieved red blood cell transfusion independence versus 11 percent of patients receiving CCR treatment (p About VIDAZA

In December 2008, VIDAZA became the first and only drug approved by the European Commission to demonstrate a significant extension of overall survival compared to conventional care regimens, for patients with Intermediate-2 and high-risk MDS and AML. Earlier in 2008 the U.S. FDA also included this extension of overall survival in its approved VIDAZA indication for treatment of all five French, American, British (FAB) MDS subtypes, which includes 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 leukaemia (CMML). The more recent WHO classification system incorporates RAEB-T patients within the AML category. VIDAZA has received orphan drug designation in several markets including the European Union, the U.S. and Japan.

Adverse reactions considered to be possibly or probably related to the administration of VIDAZA have occurred in 97 % of patients. The most commonly reported adverse reactions with VIDAZA treatment were haematological reactions (71.4 %) including thrombocytopenia, neutropenia and leukopenia (usually Grade 3-4), gastrointestinal events (60.6 %) including nausea, vomiting (usually Grade 1-2) or injection site reactions (77.1 %; usually Grade 1-2).

About Epigenetics

VIDAZA is an epigenetic compound believed to exert 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. DNA hypomethylation of aberrantly methylated genes involved in normal cell cycle regulation, differentiation and death pathways may result in gene re-expression and restoration of cancer-suppressing functions to cancer cells. 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.

About Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) are a group of haematologic 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. Patients with higher-risk MDS have a median survival of approximately 6-12 months. 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 celgene.

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.

Celgene International Sàrl

View drug information on Vidaza.