Results from Phase III Study (AML-001) of VIDAZA® (Azacitidine for Injection) in Acute Myeloid Leukemia Presented at EHA

Sat Jun 14, 2014 8:58am EDT

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Results from Phase III Study (AML-001) of VIDAZA® (Azacitidine for Injection) in Acute Myeloid Leukemia Presented at EHA

Celgene International Sàrl, a wholly-owned subsidiary of Celgene Corporation (NASDAQ:CELG) today announced that results from AML-001, its phase III study of VIDAZA® (azacitidine for injection) compared to conventional care regimens (CCR) in elderly subjects with newly-diagnosed acute myeloid leukemia (AML - >30% blasts) were presented in a late-breaking abstract oral session at the 19th European Hematology Association annual congress. The study was presented by Pr. Hervé Dombret of the Hôpital Saint-Louis in Paris, France.

In the global, multi-center, randomized, open-label pivotal study, patients at least 65 years old with newly diagnosed or secondary AML with > 30% bone marrow blasts were pre-selected to receive one of three regimens per investigator’s choice from intensive chemotherapy (standard 7+3 regimen), low-dose Ara-C (20 mg SC twice per day for 10 days of each 28-day cycle) or best supportive care only. Patients then randomized to receive either azacitidine (n=241) (75 mg/m2/d SC for 7 days of each 28-day cycle) or their predetermined CCR (n=247).

Median overall survival (OS), the primary endpoint of the study, was 10.4 months (95% CI 8.0-12.7 months) for patients receiving azacitidine compared to 6.5 months (5.0-8.6) for patients receiving CCR, which did not achieve statistical significance (unstratified HR=0.84 [95% CI 0.69, 1.02], p=0.0829).

Additionally, a pre-specified sensitivity analysis for OS that censored patients at the start of subsequent AML therapy was conducted. Results of this analysis showed a longer median overall survival for patients receiving azacitidine (median 12.1 months 95% CI, range 9.2-14.2 months) compared to patients receiving CCR (median 6.9 months 95% CI range 5.1-9.6 months) (stratified HR=0.76 [95% CI 0.60, 0.96], p=0.019).

One-year survival was 47% for patients receiving azacitidine compared to 34% for patients receiving CCR.

“AML in older patients is an area of significant unmet medical need, and this study provided insight into the use of azacitidine in this population,” said Professor Dombret.

Grade 3-4 anemia, neutropenia, febrile neutropenia, and thrombocytopenia rates, respectively, were 16%, 26%, 28%, and 24% with azacitidine; 5%, 5%, 28%, 5% with best supportive care; 23%, 25%, 30%, 28% with low-dose Ara-C; and 14%, 33%, 31%, 21% with intensive chemotherapy.

VIDAZA® is not indicated for patients with acute myeloid leukemia.

In the U.S., VIDAZA® (azacitidine for injection) is indicated for treatment of patients with the following French-American-British (FAB) myelodysplastic syndrome subtypes: 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).



• VIDAZA is contraindicated in patients with a known hypersensitivity to azacitidine or mannitol and in patients with advanced malignant hepatic tumors


Anemia, Neutropenia and Thrombocytopenia:

• Because treatment with VIDAZA is associated with anemia, neutropenia, and thrombocytopenia, complete blood counts should be performed as needed to monitor response and toxicity, but at a minimum, prior to each dosing cycle

Severe Pre-existing Hepatic Impairment:

• Because azacitidine is potentially hepatotoxic in patients with severe preexisting hepatic impairment, caution is needed in patients with liver disease.

Renal Toxicity:

Patients with renal impairment may be at increased risk for renal toxicity. Also, azacitidine and its metabolites are primarily excreted by the kidney. Therefore, these patients should be closely monitored for toxicity

Use in Pregnancy:

• VIDAZA may cause fetal harm when administered to a pregnant woman. Women of childbearing potential should be apprised of the potential hazard to the fetus. Men should be advised not to father a child while receiving VIDAZA


Nursing Mothers:

• Nursing mothers should be advised to discontinue nursing or the drug, taking into consideration the importance of the drug to the mother


• In Studies 1 and 2, the most commonly occurring adverse reactions by SC route were nausea (70.5%), anemia (69.5%), thrombocytopenia (65.5%), vomiting (54.1%), pyrexia (51.8%), leukopenia (48.2%), diarrhea (36.4%), injection site erythema (35.0%), constipation (33.6%), neutropenia (32.3%), and ecchymosis (30.5%). Other adverse reactions included dizziness (18.6%), chest pain (16.4%), febrile neutropenia (16.4%), myalgia (15.9%), injection site reaction (13.6%), and malaise (10.9%). In Study 3, the most common adverse reactions by IV route also included petechiae (45.8%), weakness (35.4%), rigors (35.4%), and hypokalemia (31.3%)

• In Study 4, the most commonly occurring adverse reactions were thrombocytopenia (69.7%), neutropenia (65.7%), anemia (51.4%), constipation (50.3%), nausea (48.0%), injection site erythema (42.9%), and pyrexia (30.3%). The most commonly occurring Grade 3/4 adverse reactions were neutropenia (61.1%), thrombocytopenia (58.3%), leukopenia (14.9%), anemia (13.7%), and febrile neutropenia (12.6%)

Please see full Prescribing Information, including CONTRAINDICATIONS, WARNINGS AND PRECAUTIONS, and ADVERSE REACTIONS.

About Celgene

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 Follow us on Twitter @Celgene as well.

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