Celgene Announces Topline Results for Maintenance Therapy in Acute Myeloid Leukemia

Celgene Corporation has announced achievement of primary and secondary end points in a phase 3 study of an investigational therapy for maintenance of patients with newly diagnosed acute myeloid leukemia (AML) who achieve a complete response or a complete response with incomplete blood count recovery with induction chemotherapy.

The study, QUAZAR AML-001, of the therapy CC-486, showed that the maintenance therapy resulted in highly statistically significant and clinically meaningful improvement in overall survival compared with placebo. In addition, the study met the key secondary end point of relapse-free survival and also showed statistically significant improvement, according to a statement from the company.

Full data will be presented at a future scientific meeting, and regulatory submissions are planned for the first half of 2020, the company said.

According to the National Cancer Institute, AML is the most common type of acute cancer in adults and occurs when the bone marrow makes abnormal myeloblasts (a type of white blood cell), red blood cells, or platelets. If not treated, this cancer can get worse very quickly. Leukemia cells build up in the blood marrow and crowd out healthy blood cells, causing infection or anemia.

The investigational therapy was well tolerated and demonstrated no unexpected safety events in QUAZAR AML-001. The study involved 472 patients randomized 1:1 to receive either oral 300 mg CC-486 or placebo once daily for 14 days of a 28-day cycle, as well as supportive care until relapse.

“AML remains a deadly blood cancer where most patients are not curable and less than 30% of patients survive 5 years,” Jay Backstrom, MD, MPH, chief medical officer at Celgene, said in the statement. He said the study was the first phase 3 trial to show that adding maintenance therapy can extend overall survival in patients with newly diagnosed AML who have achieved remission with chemotherapy.

It is expected that 21,450 new cases of AML will be diagnosed in the United States this year, and 10,920 deaths will result from the disease. There are 61,000 people living with AML in the United States, according to CDC statistics.