Celgene Corporation
Dec 10, 2011
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Phase I Study Evaluating Oral Azacitidine as Potential Treatment for Patients With Acute Myeloid Leukemia Presented at the American Society of Hematology Meeting

Study Demonstrates that Oral Azacitidine Was Well Tolerated in Patients with Acute Myeloid Leukemia Not Eligible for Other Therapies and with Complex Cytogenetic Abnormalities

BOUDRY, Switzerland--(BUSINESS WIRE)--Dec. 10, 2011-- Celgene International Sàrl, a subsidiary of Celgene Corporation (NASDAQ:CELG), announced results from a multi-center Phase I study that explored multiple dosing schedules of oral azacitidine in 23 patients with acute myeloid leukemia (AML), myelodysplastic syndromes or chronic myelomonocytic leukemia (CMML) who were not candidates for other therapies or who had failed previous regimens. These findings were presented during the 53rd Annual Meeting of the American Society of Hematology in San Diego, CA.

In part one of the study, patients received an initial cycle of subcutaneous azacitidine (75 mg/m2 daily for the first 7 days of a 28-day cycle). Thereafter, they were assigned to different doses of oral azacitidine (120-600 mg daily) for the first seven days of repeated 28-day schedules. In part two, patients were assigned to one of four dosing schedules of oral azacitidine: 300 mg daily or 200 mg twice per day, each for either 14 or 21 days of each 28-day cycle. Patients remained on therapy until disease progression or study withdrawal.

Of the 15 patients receiving any of the part two extended dosing schedules, four (27%, 4/15 patients) achieved hematologic improvement, including patients who had complex cytogenetics and/or had failed prior therapy for AML. One of the eight patients in part one of the study treated with 7-day once daily oral azacitidine achieved a complete response with incomplete blood count recovery (CRi).

Grade 3/4 adverse events occurring in at least 10% of the 23 patients enrolled in the study included febrile neutropenia (35%, 8/23), pneumonia (17%, 4/23), syncope (17%, 4/23), and nausea (13%, 3/23). Three patients died due to causes unrelated to oral azacitidine.

Oral azacitidine is an investigational agent and has not been approved for any indication.

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 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 (also known as 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 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.

Forward-Looking Statements

This press release contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words "expects," "anticipates," "believes," "intends," "estimates," "plans," "will," “outlook” and similar expressions. Forward-looking statements are based on management’s current plans, estimates, assumptions and projections, and speak only as of the date they are made. We undertake no obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond our control. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors, many of which are discussed in more detail in our Annual Report on Form 10-K and our other reports filed with the Securities and Exchange Commission.

Source: Celgene International Sàrl

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