Positive results for CC 486 inphase III QUAZAR AML-001 trial for newly diagnosed acute myeloid leukemia

Celgene Corporation announced top-line results from the international phase III, randomized, double-blind, placebo-controlled study, QUAZAR AML-001 . The study evaluated the efficacy and safety of investigational therapy CC 486 as maintenance therapy in patients with newly diagnosed acute myeloid leukemia (AML) who achieved first complete response (CR) or complete response with incomplete blood count recovery (CRi) with induction chemotherapy (with or without consolidation). The study demonstrated that maintenance treatment with CC 486 resulted in a highly statistically significant and clinically meaningful improvement in overall survival compared to placebo. The key secondary endpoint of relapse-free survival (RFS) also showed a statistically significant improvement. CC 486 was well-tolerated and there were no unexpected safety events in QUAZAR AML-001.

This phase III study enrolled 472 patients, randomized 1:1 to receive either oral CC-486 300mg or placebo once daily for 14 days of a 28-day cycle plus best supportive care until disease relapse. Data from QUAZAR AML-001 will be submitted to a future medical meeting. Celgene also plans regulatory submissions for CC 486 beginning in the first half of 2020.

About QUAZAR AML-001 : Phase III, randomized, double-blind, placebo-controlled study of CC 486 as AML maintenance therapy in patients who achieved first CR or complete response with incomplete blood count recovery (CRi) with induction chemotherapy (with or without consolidation) The primary endpoint of the study was overall survival. Key secondary endpoints included relapse-free survival (RFS), safety and tolerability, healthcare resource utilization and patient-reported outcomes per the FACIT-Fatigue Scale and EQ-5D questionnaire. The study enrolled 472 patients, randomized 1:1 to receive either oral CC 486 300mg or placebo once daily for 14 days of a 28-day cycle plus best supportive care until disease progression.

Comment: The CC 486 QUAZAR AML-001 study is the first phase III trial to demonstrate that the addition of maintenance therapy has the potential to extend overall survival in a broad population of patients with newly diagnosed AML who have achieved remission with induction chemotherapy.

Comment: In prior studies of CC 486 in 23 patients with AML, the overall response rate was 48%, which included hematologic improvement in 32% and red blood cell transfusion independence in 20%. The most common grade 3/4 adverse events seen with the oral azacitidine agent were febrile neutropenia in 30%, anemia in 22%, neutropenia in 17%, and thrombocytopenia in 17%.