Ambit Biosciences presents study results of its AC220 FLT3 inhibitor for AML

Ambit Biosciences Corporation announced today that preliminary results from a clinical trial of its lead product candidate, AC220, in acute myeloid leukemia (AML) were presented at the 51st Annual Meeting of the American Society of Hematology (ASH) in New Orleans. AC220 is a novel, orally available, small molecule that was expressly optimized as a FMS-like tyrosine kinase-3 (FLT3) inhibitor for the treatment of AML.

The open-label, dose-escalation study, "AC220, a Potent, Selective, Second Generation FLT3 Receptor Tyrosine Kinase Inhibitor, in a First-in-Human Phase I AML Study," was designed to evaluate the safety, tolerability, and pharmacokinetic profile of AC220 in AML patients with predominantly relapsed or refractory disease. AC220 was generally well-tolerated in AML patients, and importantly, there was no treatment-related mortality observed in this study. Clinical response and median survival were assessed as secondary endpoints and analyzed for the overall study population, in addition to patient subsets based on the presence or absence of the FLT3 genotype for the internal tandem duplication (ITD) mutation (FLT3 ITD+ and FLT3 ITD-, respectively). Following treatment with AC220, responses were observed in both FLT3 ITD+ and FLT3 ITD- patients, and responders in each group had at least a doubling in median survival versus non-responders.

"AML represents a challenging hematological malignancy to treat. A significant portion of AML patients have activating FLT3 mutations, and these patients have a particularly poor prognosis and are often refractory to current treatment options," said Jorge Cortes MD, Internist and Professor, Deputy Chair, Department of Leukemia, Division of Cancer Medicine, The University of Texas M. D. Anderson Cancer Center, Houston, TX, and primary investigator for the Phase I Study. "These encouraging results with AC220 warrant further studies of this promising compound as a monotherapy and in combination with other treatments in patients with AML."