Celgene's AML drug clears late-stage test
Celgene is gearing up for regulatory submissions after a pivotal acute myeloid leukemia (AML) study showed its drug helped patients live longer.
The drugmaker — which is in the process of being swallowed by Bristol-Myers Squibb $BMY — tested its experimental drug, CC-486, in newly-diagnosed patients with AML who fully or partially benefited from chemotherapy.
In the 472-patient QUAZAR AML-001 trial, patients on supportive care were either given CC-486 or a placebo once daily for two weeks. Maintenance treatment with CC-486 conferred a statistically significant improvement in overall survival — meeting the main goal — as well as the endpoint of relapse-free survival, Celgene $CELG said on Friday.
“AML remains a deadly blood cancer where most patients are not curable and less than 30% of patients survive five years,” Celgene’s chief medical officer Jay Backstrom said in a statement. “The CC-486 QUAZAR AML-001 study is the first phase 3 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.”
Detailed data on CC-486 — which is also being tested in other forms of cancer — are expected at a future medical conference, and the company is planning regulatory submissions next year.
AML is a form of cancer in which the bone marrow produces abnormal myeloblasts (a type of white blood cell), red blood cells, or platelets. About 21,450 cases of AML will be diagnosed in 2019, and 10,920 will succumb to the disease, estimates the American Cancer Society.
Last November, Japan’s Astellas secured FDA approval for its AML drug, gilteritinib, for use in patients that carry a mutation that accounts for about a third of all AML cases. In July 2018, Agios also scored FDA approval for its targeted AML drug, Tibsovo, for patients that carry a particular mutation.