Ambit Biosciences begins dosing in leukemia trial

The phase I trial is a dose-escalation study that will enroll patients with relapsed or refractory acute myeloid leukemia (AML). AC220 will be administered daily via oral solution, beginning at 12 mg for 14 days and then increasing until the maximum tolerated dose is established. In addition to evaluating the safety, tolerability and pharmacokinetics of AC220, the study will measure pharmacodynamics by monitoring FLT3 receptor phosphorylation and peripheral blood blast counts.

AC220 was discovered using Ambit Biosciences' kinase profiling technology KinomeScan.

“Alone and in collaboration with partners, we continue to exploit the discovery advantages KinomeScan provides, and we anticipate advancing several more programs into the clinic over the next few years,” said Scott Salka, CEO of Ambit.

AC220 potently inhibits FLT3, a kinase that is mutated in approximately one-third of AML cases, and patients with FLT3 mutations are less responsive to traditional therapies. AC220 has previously produced marked inhibition of tumor growth when administered in mouse xenograft tumor models.