Celgene's phase 3 AML test chalks up a win

Celgene is running phase 3 trials of CC-486 in two other indications beyond acute myeloid leukemia. (Celgene)

A phase 3 trial of Celgene’s acute myeloid leukemia (AML) maintenance therapy CC-486 has met its primary endpoint. Patients who took the cytidine nucleoside analog lived longer than their peers on placebo, setting Celgene up to file for approval of the drug in the first half of next year.

The trial enrolled 472 AML patients who had experienced a complete response after treatment with chemotherapy. Participants received either CC-486 or placebo orally for half of a 28-day cycle, plus best supportive care, until their disease progressed. The hope was that CC-486 would delay relapse and, in doing so, improve the survival rate in a blood cancer that kills most people within five years.

Celgene now has data it thinks validates that hope, and this will please its parent Bristol-Myers Squibb, which is relying on the company to help boost its pipeline. The big biotech is yet to share data from the trial but revealed that it met its primary overall survival endpoint and key secondary objectives, including relapse-free survival. Celgene said the drug was well tolerated.

The readout follows an earlier-phase study that linked CC-486, an oral formulation of the active ingredient in Vidaza, to disease-free survival of 15.9 months, as compared to 10.3 months in the control arm. The promise shown in the earlier study made the phase 3 readout an eagerly anticipated event in some quarters. 

“If [the phase 3] study is positive, it is likely that maintenance therapy will be accepted as a new standard of care for patients with AML,” Monash University’s Andrew Wei wrote in Blood earlier this year.

Buoyed by the phase 3 findings, Celgene plans to file for approval of CC-486 in the first half of 2020. CC-486 has a lower profile than some of the late-phase drugs in Celgene’s pipeline and has largely been absent from Bristol-Myers Squibb’s explanations of the rationale for its planned takeover of the big biotech. 

Yet there is scope for the drug to come to market in several indications in the coming years. Celgene is currently running phase 3 trials of CC-486 in patients with transfusion-dependent myelodysplastic syndrome and relapsed or refractory angioimmunoblastic T cell lymphoma.