Vidaza study reports unprecedented survival benefit in higher-risk MDS extends to AML patients

December 09, 2008

BOUDRY, Switzerland - (December 8, 2008) - Celgene International Sàrl (NASDAQ: CELG) today announced results from a subset analysis of the international phase III trial (AZA-001) demonstrating that the overall survival benefit observed in higher-risk MDS patients extended to patients with acute myeloid leukemia (AML). The data, presented at the 50th Annual Meeting of the American Society of Hematology (ASH), reported that patients with WHO-defined AML who were treated with VIDAZA (azacitidine) achieved significantly improved overall survival compared to those treated with a conventional care regimen (CCR).

The AZA-001 trial recently showed that treatment with VIDAZA provides an unprecedented survival benefit in patients with higher-risk myelodysplastic syndromes (MDS). Based on these results, VIDAZA received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) for certain patients with higher-risk MDS and WHO-defined AML and gained an approval from the U.S. Food and Drug Administration for an expanded label to include the survival data in higher-risk MDS.

Almost one third of the patients (113 of 358) enrolled in the AZA-001 study met the WHO criteria for AML (median 23% bone marrow blasts), which has a poor prognosis and does not respond well to conventional chemotherapy. This subset analysis of the AZA-001 study showed the median overall survival was 24.5 months with VIDAZA compared to 16.0 months with CCR (p=0.005). Additionally, 50 percent of the AML patients who were treated with VIDAZA survived at least two years, compared to only 16 percent of AML patients treated with CCR.

The subset analysis also showed that patients treated with VIDAZA had fewer infections requiring intravenous antibiotics and reduced rates of hospitalization and red blood cell transfusions.

"Patients with AML have an extremely poor prognosis," said Pierre Fenaux, M.D., of the University of Paris and lead investigator of the AZA-001 survival trial. "The results of this pivotal study show that the unprecedented survival benefit obtained by VIDAZA in higher-risk MDS extend to AML. I look forward to the benefits of this drug becoming available to patients in Europe and to further evaluation across all categories of AML."

In the AZA-001 study, the most commonly occurring adverse reactions were thrombocytopenia (69.7%), neutropenia (65.7%) and anemia (51.4%).
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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 (CMMoL). 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 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 by FDA for IV administration in January 2007 and the results of the VIDAZA Survival trial (AZA-001) were added in August 2008.

About Acute Myeloid Leukemia

Acute Myeloid Leukemia (AML) is a cancer of myeloid blood cells that often transforms from MDS upon disease progression. AML is the proliferation of abnormal cells that accumulate in the bone marrow and interfere with all types of normal blood cell production (multi-lineage dysplasia). AML has traditionally been treated with high intensity chemotherapy, which is poorly tolerated by the majority of the patients who are afflicted - the elderly. Many of these patients may go untreated and because they are ineligible for curative therapy, life expectancy is short and often measured in weeks to months.

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 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 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.

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