Celgene has announced that the Phase III QUAZAR AML-001 clinical trial of CC-486 as maintenance therapy for acute myeloid leukaemia (AML) met primary and key secondary endpoints.
CC-486 is an investigational cytidine nucleoside that can integrate into DNA and RNA. It is believed to work by causing hypomethylation that could restore the normal function of genes required for differentiation and proliferation.
The molecule is also thought to cause cytotoxicity of abnormal hematopoietic cells in the bone marrow.
QUAZAR AML-001 is a randomised, double-blind, placebo-controlled trial conducted in 472 newly diagnosed patients who experienced first complete response (CR) or CR with incomplete blood count recovery (CRi) when treated with induction chemotherapy.
It assessed 300mg CC-486 versus placebo once daily in conjunction with best supportive care over 14 days of a 28-day cycle until disease progression.
The trial’s primary endpoint was overall survival, while key secondary endpoints tracked by the study included relapse-free survival, safety, tolerability, utilisation of healthcare resources, and patient-reported outcomes.
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Results demonstrated a statistically significant and clinically meaningful improvement in overall survival for patients treated with CC-486 compared to placebo.
A statistically significant improvement was also observed for relapse-free survival. Moreover, CC-486 was well-tolerated without unexpected safety events.
Celgene chief medical officer Jay Backstrom said: “AML remains a deadly blood cancer where most patients are not curable and less than 30% of patients survive five years.
“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.”
The company is planning to submit applications in the first half of next year for regulatory approval of the candidate.