Phase III Study (MM-003) of Pomalidomide Plus Low-Dose Dexamethasone Demonstrates Significant Progression-Free and Overall Survival Improvement for Patients with Relapsed or Refractory Multiple Myeloma

Tue Dec 11, 2012 8:45am EST

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Phase III Study (MM-003) of Pomalidomide Plus Low-Dose Dexamethasone Demonstrates Significant Progression-Free and Overall Survival Improvement for Patients with Relapsed or Refractory Multiple Myeloma

Study presented as a late-breaking abstract at 54th American Society of Hematology annual meeting

Celgene International Sàrl, a subsidiary of Celgene Corporation (NASDAQ: CELG) today announced data from its phase III, multi-center, randomized (2:1), open-label study (MM-003, n=455) of pomalidomide plus low-dose dexamethasone compared with high-dose dexamethasone in patients with refractory multiple myeloma who have failed therapy with both bortezomib and lenalidomide, administered either alone or in combination.

MM-003 met the primary endpoint of improvement in progression-free survival (PFS). PFS was significantly longer in patients who received pomalidomide plus low-dose dexamethasone compared with those who received high-dose dexamethasone (median 3.6 months vs. 1.8 months; hazard ratio [HR], 0.45; P < 0.001).

According to the study design, an interim analysis of overall survival (OS) was also conducted. In this analysis, pomalidomide plus low-dose dexamethasone demonstrated a highly statistically significant improvement in overall survival that crossed the upper boundary for superiority (median OS not reached vs. 7.8 months; HR, 0.53; P < 0.001). As a result, the Data Monitoring Committee recommended that patients who had not yet progressed in the high-dose dexamethasone arm should be crossed over to pomalidomide plus low-dose dexamethasone.

As adjudicated by an independent committee, pomalidomide plus low-dose dexamethasone demonstrated a statistically greater overall response rate compared with high-dose dexamethasone: 21% vs. 3%, respectively (P < 0.001). Patients who were randomized for at least six months have shown a greater overall response rate of 24% vs. 3% (P < 0.001). Further evaluation of response rates is ongoing.

The most common grade 3/4 hematologic toxicities included for the pomalidomide plus low-dose dexamethasone arm and high-dose dexamethasone arms, respectively, neutropenia (42% vs. 15%), anemia (27% vs. 29%), thrombocytopenia (21% vs. 24%), and febrile neutropenia (7% vs. 0%). Other toxicities (grade 3/4) were predominantly infections (24% vs. 23%), pneumonia (9% vs. 7%), hemorrhage (3% vs. 5%), glucose intolerance (3% vs. 7%), and fatigue (5% vs. 5%). Any grade venous thromboembolism occurred in 3% vs. 2% of patients, and any grade peripheral neuropathy in 12% vs. 11%. The primary reason for discontinuation was progressive disease: 35% for patients in the pomalidomide plus low-dose dexamethasone arm and 49% for the high-dose dexamethasone arm. Percentages of patients who discontinued due to adverse events were 7% vs. 6%, respectively.

Patients in the pomalidomide plus low-dose dexamethasone arm received 4 mg of oral pomalidomide on days 1-21 of each 28-day cycle. Oral dexamethasone was given at 40 mg weekly; for patients older than 75 years, dexamethasone was administered at 20 mg weekly.

Patients in the comparator arm were treated with 40 mg oral high-dose dexamethasone on days 1-4, 9-12 and 17-20 of each 28-day cycle, until disease progression; patients older than 75 years received 20 mg oral dexamethasone on the same schedule.

A Marketing Authorisation Application (MAA) for pomalidomide in combination with low-dose dexamethasone was submitted to the EMA in May 2012, and Celgene anticipates a decision by European regulatory authorities in the second half of 2013.

Additionally, a new drug application (NDA) has been accepted for review by the U.S. Food and Drug Administration, with a Prescription Drug User Fee Act (PDUFA) date of Feb. 10, 2013.

These results are from an investigational study. Pomalidomide is not approved for the treatment of any indication.

About Pomalidomide

Pomalidomide is an IMiDs® compound. Pomalidomide and other IMiDs compounds continue to be evaluated in over 100 clinical trials. The IMiDs compound 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 (also known as myeloma or plasma cell 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. However, most patients with multiple myeloma have cells that produce a form of immunoglobulin called paraprotein (or M protein) that 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 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.

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