Celgene announces results from azacitidine Phase 1 study on AML

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 53^rd 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.