Promising Results From Two Trials Highlighting Developmental Oral IMiDS(R) Compound Pomalidomide Presented At The ASH

Main Category: Lymphoma / Leukemia / Myeloma
Also Included In: Blood / Hematology;  Clinical Trials / Drug Trials
Article Date: 12 Dec 2008 - 6:00 PDT

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Celgene International Sarl (Nasdaq: CELG) today announced that its next IMiDs compound, pomalidomide, has shown promising activity with manageable safety and tolerability for the treatment of relapsed/refractory multiple myeloma (MM) and myelofibrosis. The data were presented at the 50th Annual American Society of Hematology meeting in San Francisco, CA.

Early analysis of the Phase II MM study, in which half of the 60 patients with relapsed MM received combined low-dose dexamethasone with pomalidomide, showed that 76 percent of the patients experienced disease improvement or stabilization. Another key finding showed a 29 percent response rate among patients who previously did not respond to REVLIMID(R) therapy. Objective response was achieved by 58 percent of patients. Eight of the 60 patients had dose reductions. The most commonly occurring major adverse events were neutropenia (32%), thrombocytopenia (3%) and anemia (3%). Investigators concluded that for most patients, pomalidomide plus low-dose dexamethasone was generally well tolerated with manageable adverse events. However, there was one patient death due to pneumonia while neutropenic in this refractory, pre-treated population.

The study's conclusion was that pomalidomide was highly active in this segment of multiple myeloma patients.

The second Phase II trial, evaluating 84 patients with advanced myelofibrosis with myeloid metaplasia, was a four-arm blinded adaptive design trial. The study evaluated two different doses of pomalidomide with or without prednisone, with a prednisone-only arm as a control. All but one patient had failed prior therapies. The Grade 2 or greater side effects were infrequent and comparable to the prednisone control except for thrombocytopenia that was experienced in one of 22 patients and one of 19 patients treated with 2 mg of pomalidomide with or without prednisone. Thirty-five percent of the treated pomalidomide patients experienced blood cell transfusion independence. At the time of this presentation, 15 of the 16 responders remain in remission. Granulocytopenia and normal spleen size correlated with response, but the percent of abnormal cytogenetics and the presence of a JAK2 mutation did not.

"The interest in our next IMiDs compound, pomalidomide, by the hematological community was demonstrated by both clinical abstracts being selected for oral presentations at the annual meeting of the American Society of Hematology," stated Jerome B. Zeldis, Chief Medical Officer, Celgene Corporation. "Based on these findings and others, Celgene will be developing pomalidomide for relapsed multiple myeloma and other hematological conditions."

About Pomalidomide

Pomalidomide is an IMiDs(R) compound, a member of a proprietary group of novel immunomodulatory agents. These immunomodulatory agents, taken orally, have unique multiple mechanisms of action that involve the microenvironment of the cancer cell, not just the malignant cell itself. The IMiDs pipeline is covered by a comprehensive intellectual property estate of issued and pending patent applications in the US, EU and other regions.

About Multiple Myeloma

Multiple myeloma is a cancer of the blood in which malignant plasma cells are overproduced in the bone marrow. Plasma cells are white blood cells that help produce antibodies called immunoglobulins that fight infection and disease. Most patients with multiple myeloma, however, have cells that produce a form of immunoglobulin called paraprotein (or M protein), which does not benefit the body. In addition, the malignant plasma cells replace normal plasma cells and other white blood cells important to the immune system. Multiple myeloma cells can also attach to other tissues of the body, such as bone, and produce tumors. The cause of the disease remains unknown.

About Myelofibrosis

Myelofibrosis with myeloid dysplasia is often referred to as a chronic form of leukemia. It is a serious bone marrow disorder that begins in stem cells that form blood cells. Too few red cells are produced with an overproduction of white cells and platelets. That in turn results in a buildup of collagen in the bone marrow cavity, causing extensive scarring. The results are anemia, fatigue and susceptibility to infection.

About Celgene International Sarl

Celgene International Sarl, located in Boudry, in the Canton of Neuchatel, 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 http://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.

Celgene International Sarl

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